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Alnylam Pharmaceuticals, Inc. (ALNY) Report Analysis

Corporate Events

Neutral

Alnylam Pharmaceuticals, Inc. - Special Call

2022-06-13 23:22:00

To discuss the FDA approval of AMVUTTRA

Positive

Alnylam Announces FDA Approval of AMVUTTRA (vut...

2022-06-13 23:22:00

Alnylam Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA (vutrisiran), an RNAi therapeutic a...

Positive

Alnylam Reports Positive Topline Results from P...

2022-06-09 11:00:00

Alnylam Pharmaceuticals, Inc. announced topline results from the Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting th...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at 2022 ...

2022-06-02 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at 2022 Jefferies Global Healthcare Conference, Jun-09-2022 11:00 AM. Venue: Marriott Marquis, New York...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Goldm...

2022-06-02 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Goldman Sachs 43rd Annual Global Healthcare Conference, Jun-13-2022 02:40 PM. Venue: Terranea Resort...

Positive

Alnylam Announces Publication of Preclinical Re...

2022-06-02 15:42:00

Alnylam Pharmaceuticals, Inc. announced that data from its preclinical research on delivery of lipophilic short interfering RNA (siRNA) conjug...

Positive

Alnylam Pharmaceuticals, Inc. Presents New Resu...

2022-05-24 11:00:00

Alnylam Pharmaceuticals, Inc. announced new positive results from the six-month primary analysis period of the ILLUMINATE-C Phase 3 open-label...

Positive

Alnylam Pharmaceuticals, Inc. Announces New Pos...

2022-05-23 11:00:00

Alnylam Pharmaceuticals, Inc. announced new positive results from an 18-month analysis of exploratory cardiac endpoints in the HELIOS-A Phase ...

Positive

Alnylam Pharmaceuticals, Inc. Announces That He...

2022-05-18 12:00:00

Alnylam Pharmaceuticals, Inc. announced that Health Canada has issued a Notice of Compliance (NOC) authorizing OXLUMO™ (lumasiran) injection f...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at H.C. ...

2022-05-04 12:00:00

Alnylam Pharmaceuticals, Inc. Presents at H.C. Wainwright Global Investment Conference 2022, May-24-2022 07:00 AM. Venue: Fontainebleau Miami ...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at UBS G...

2022-05-04 12:00:00

Alnylam Pharmaceuticals, Inc. Presents at UBS Global Healthcare Conference, May-25-2022 10:00 AM. Venue: Lotte New York Palace, New York City,...

Neutral

Alnylam Pharmaceuticals, Inc. Updates Earnings ...

2022-04-28 12:00:00

Alnylam Pharmaceuticals, Inc. updated earnings guidance for the year 2022. For the year, the company expects combined net product revenues for...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at TIDES...

2022-04-23 17:57:00

Alnylam Pharmaceuticals, Inc. Presents at TIDES (Oligonucleotide & Peptide Therapeutics), May-05-2022 . Venue: Boston, Massachusetts, United S...

Neutral

I.I.R Ltd., TIDES (Oligonucleotide & Peptide Th...

2022-04-23 07:45:00

I.I.R Ltd., TIDES (Oligonucleotide & Peptide Therapeutics), May 05, 2022 through May 12, 2022. Venue: Boston, Massachusetts, United States.

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q1, 202...

2022-04-14 12:30:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q1, 2022 results Pre-Market on Apr 28, 2022

Neutral

Alnylam Pharmaceuticals, Inc., Q1 2022 Earnings...

2022-04-14 12:30:00

Alnylam Pharmaceuticals, Inc., Q1 2022 Earnings Call, Apr 28, 2022

Neutral

Alnylam Pharmaceuticals, Inc. - Shareholder/Ana...

2022-04-05 20:19:00

ANNUAL MEETING OF STOCKHOLDERS

Neutral

Alnylam Pharmaceuticals, Inc., Annual General M...

2022-04-05 20:19:00

Alnylam Pharmaceuticals, Inc., Annual General Meeting, May 18, 2022, at 12:00 US Eastern Standard Time. Agenda: To elect three members to our ...

Negative

Alnylam Pharmaceuticals, Inc. Announces Retirem...

2022-04-05 20:00:00

Alnylam Pharmaceuticals, Inc. announced that Steven Paul, M.D. is retiring from the Board of Directors effective April 16, 2022. Dr. Paul has ...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Pharm...

2022-03-29 08:36:00

Alnylam Pharmaceuticals, Inc. Presents at Pharma Manufacturing World Summit, Mar-29-2022 . Venue: Boston Marriott Copley Place, Boston, Massac...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Bank ...

2022-03-22 05:03:00

Alnylam Pharmaceuticals, Inc. Presents at Bank of America 2022 Healthcare Conference, May-11-2022 11:20 AM. Venue: Encore Hotel, 3121 Las Vega...

Neutral

Executive Platforms Inc., Pharma Manufacturing ...

2022-03-21 14:57:00

Executive Platforms Inc., Pharma Manufacturing World Summit, Mar 29, 2022 through Mar 30, 2022. Venue: Boston Marriott Copley Place, Boston, M...

Neutral

UBS Investment Bank, UBS Global Healthcare Conf...

2022-03-03 04:07:00

UBS Investment Bank, UBS Global Healthcare Conference, May 23, 2022 through May 25, 2022. Venue: Lotte New York Palace, New York City, New Yor...

Neutral

H.C. Wainwright & Co., LLC, H.C. Wainwright Glo...

2022-02-28 18:21:00

H.C. Wainwright & Co., LLC, H.C. Wainwright Global Investment Conference 2022, May 23, 2022 through May 26, 2022. Venue: Fontainebleau Miami B...

Neutral

Bank of America Corporation, Bank of America 20...

2022-02-17 10:42:00

Bank of America Corporation, Bank of America 2022 Healthcare Conference, May 10, 2022 through May 13, 2022. Venue: Encore Hotel, 3121 Las Vega...

Neutral

Jefferies LLC, 2022 Jefferies Global Healthcare...

2022-02-14 21:00:00

Jefferies LLC, 2022 Jefferies Global Healthcare Conference, Jun 08, 2022 through Jun 10, 2022. Venue: Marriott Marquis, New York City, New Yor...

Neutral

Alnylam Pharmaceuticals, Inc. Provides Earnings...

2022-02-10 13:10:00

Alnylam Pharmaceuticals, Inc. provided earnings guidance for the year 2022. For the year, the company expects Combined net product revenues fo...

Neutral

The Goldman Sachs Group, Inc., Goldman Sachs 43...

2022-02-08 07:31:00

The Goldman Sachs Group, Inc., Goldman Sachs 43rd Annual Global Healthcare Conference, Jun 13, 2022 through Jun 16, 2022. Venue: Terranea Reso...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at SLAS ...

2022-02-05 10:28:00

Alnylam Pharmaceuticals, Inc. Presents at SLAS 2022 International Conference and Exhibition, Feb-09-2022 01:00 PM. Venue: Boston Convention an...

Neutral

Alnylam Pharmaceuticals, Inc., Q4 2021 Earnings...

2022-01-24 13:38:00

Alnylam Pharmaceuticals, Inc., Q4 2021 Earnings Call, Feb 10, 2022

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q4, 202...

2022-01-24 13:38:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q4, 2021 results Pre-Market on Feb 10, 2022

Negative

Alnylam Pharmaceuticals, Inc. Announces Promoti...

2022-01-04 12:30:00

Alnylam Pharmaceuticals, Inc. announced that Akshay Vaishnaw, M.D., Ph.D. has been appointed President. Dr. Vaishnaw joined the company in 200...

Neutral

Society for Laboratory Automation & Screening, ...

2021-12-28 18:17:00

Society for Laboratory Automation & Screening, SLAS 2022 International Conference and Exhibition, Feb 05, 2022 through Feb 09, 2022. Venue: Bo...

Positive

Alnylam Comments on FDA Approval of Leqvio, the...

2021-12-22 21:23:00

Alnylam Pharmaceuticals, Inc. highlighted the significance of the U.S. Food and Drug Administration’s (FDA) approval of Leqvio® (inclisiran), ...

Positive

Alnylam Pharmaceuticals, Inc. Submits Clinical ...

2021-12-22 12:00:00

Alnylam Pharmaceuticals, Inc. announced that the Company has submitted a Clinical Trial Authorization (CTA) application to The Medicines and H...

Negative

Yvonne L. Greenstreet Appoints as Chief Executi...

2021-12-20 21:15:00

Yvonne L. Greenstreet Alnylam Pharmaceuticals, Inc.’s current President and Chief Operating Officer, has been appointed Chief Executive Office...

Positive

Alnylam Pharmaceuticals, Inc. Initiates Phase 2...

2021-12-20 21:00:00

Alnylam Pharmaceuticals, Inc. announced that the company has initiated a global Phase 2 study to evaluate the safety and efficacy of lumasiran...

Positive

Alnylam Submits CTA Application for ALN-XDH, an...

2021-12-20 12:00:00

Alnylam Pharmaceuticals, Inc. announced that the Company has submitted a clinical trial authorization (CTA) application to The Medicines and H...

Positive

Alnylam Pharmaceuticals, Inc. Submits Regulator...

2021-12-14 12:00:00

Alnylam Pharmaceuticals, Inc. announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration ...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at 4th A...

2021-11-23 13:00:00

Alnylam Pharmaceuticals, Inc. Presents at 4th Annual Evercore ISI HealthCONx Virtual Conference, Nov-30-2021 09:40 AM.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Piper...

2021-11-22 15:06:00

Alnylam Pharmaceuticals, Inc. Presents at Piper Sandler 33rd Annual Virtual Healthcare Conference, Dec-02-2021 10:00 AM. Venue: New York, New ...

Positive

Alnylam Pharmaceuticals, Inc. Presents New Data...

2021-11-13 13:00:00

Alnylam Pharmaceuticals, Inc. announced positive interim results from the ongoing Phase 1 study of zilebesiran (formerly known as ALN-AGT), an...

Neutral

Alnylam Pharmaceuticals, Inc. - Special Call

2021-11-12 13:00:00

Alnylam Pharmaceuticals, Inc. - Special Call

Positive

Alnylam Pharmaceuticals, Inc. Initiates KARDIA-...

2021-11-08 12:00:00

Alnylam Pharmaceuticals, Inc. announced initiation of KARDIA-2, a global Phase 2 study to evaluate the efficacy and safety of zilebesiran (for...

Positive

Alnylam Pharmaceuticals, Inc. Presents Positive...

2021-11-05 17:30:00

Alnylam Pharmaceuticals, Inc. announced on November 5, 2021 positive results from the 6-month primary analysis of the ILLUMINATE-C Phase 3 ope...

Neutral

SVB Leerink LLC, 11th SVB Leerink Annual Global...

2021-10-29 14:57:00

SVB Leerink LLC, 11th SVB Leerink Annual Global Healthcare Conference 2022, Feb 14, 2022 through Feb 18, 2022. Venue: New York, New York, Unit...

Neutral

Alnylam Pharmaceuticals, Inc. Provides Earnings...

2021-10-28 12:00:00

Alnylam Pharmaceuticals, Inc. provided earnings guidance for the year 2021. For the year, the company expects net revenues from collaborations...

Negative

Alnylam Pharmaceuticals, Inc. Announces Board Changes

2021-10-28 11:55:00

Alnylam Pharmaceuticals, Inc. announced that effective immediately, Dr. Yvonne Greenstreet has been appointed to the Alnylam Board of Director...

Negative

Alnylam Announces Planned CEO Leadership Transition

2021-10-28 11:55:00

Alnylam Pharmaceuticals, Inc. announced the planned CEO transition of John Maraganore, Ph.D., to Yvonne Greenstreet, MBChB, at year-end 2021. ...

Positive

Alnylam Pharmaceuticals, Inc. Reports Positive ...

2021-10-27 20:00:00

Alnylam Pharmaceuticals, Inc. announced that the HELIOS-A phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for ...

Positive

Alnylam Pharmaceuticals, Inc. Receives Positive...

2021-10-18 12:00:00

Alnylam Pharmaceuticals, Inc. announced that GIVLAARI® (givosiran) has now received a positive recommendation for reimbursement from the Canad...

Neutral

Alnylam Pharmaceuticals, Inc., Q3 2021 Earnings...

2021-10-14 12:00:00

Alnylam Pharmaceuticals, Inc., Q3 2021 Earnings Call, Oct 28, 2021

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q3, 202...

2021-10-14 12:00:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q3, 2021 results Pre-Market on Oct 28, 2021

Positive

Alnylam Pharmaceuticals, Inc. Announces Launch ...

2021-10-13 11:30:00

Alnylam Pharmaceuticals, Inc. announced the launch of Alnylam Challengers, a signature community impact program that will support bold and div...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Inter...

2021-09-30 20:47:00

Alnylam Pharmaceuticals, Inc. Presents at International Oligonucleotide and Peptide Conference, Oct-18-2021 02:35 PM. Speakers: Muthiah Manoha...

Neutral

Tekno Scienze S.R.L., International Oligonucleo...

2021-09-30 20:01:00

Tekno Scienze S.R.L., International Oligonucleotide and Peptide Conference, Oct 18, 2021 through Oct 19, 2021.

Positive

Alnylam Pharmaceuticals, Inc. Announces New Pre...

2021-09-29 11:30:00

Alnylam Pharmaceuticals, Inc. announced new pre-clinical research results demonstrating continued RNAi therapeutics platform innovation and sc...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Chard...

2021-09-28 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Chardan's 5th Annual Genetic Medicines Conference, Oct-05-2021 02:30 PM.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Longw...

2021-09-28 18:44:00

Alnylam Pharmaceuticals, Inc. Presents at Longwood Healthcare Leaders Conference, Oct-05-2021 02:50 PM. Speakers: Kevin Fitzgerald, Senior VP ...

Neutral

Longwood Fund, Longwood Healthcare Leaders Conf...

2021-09-28 13:09:00

Longwood Fund, Longwood Healthcare Leaders Conference, Oct 04, 2021 through Oct 05, 2021.

Positive

Alnylam Pharmaceuticals, Inc. Announces Onpattr...

2021-09-28 12:00:00

Alnylam Pharmaceuticals, Inc. announced that ONPATTRO® (patisiran) is now reimbursed almost everywhere in Canada for the treatment of heredita...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Cell ...

2021-09-16 14:42:00

Alnylam Pharmaceuticals, Inc. Presents at Cell & Gene Therapy Bioprocessing & Commercialization, Sep-20-2021 . Speakers: John M. Maraganore, C...

Neutral

Informa plc, Cell & Gene Therapy Bioprocessing ...

2021-09-08 23:01:00

Informa plc, Cell & Gene Therapy Bioprocessing & Commercialization, Sep 20, 2021 through Sep 30, 2021.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at BioPr...

2021-09-08 22:27:00

Alnylam Pharmaceuticals, Inc. Presents at BioProcess International Conference & Exhibition, Sep-20-2021 . Speakers: John M. Maraganore, CEO & ...

Neutral

Informa plc, BioProcess International Conferenc...

2021-09-08 21:27:00

Informa plc, BioProcess International Conference & Exhibition, Sep 20, 2021 through Sep 30, 2021.

Negative

Alnylam Pharmaceuticals, Inc. Announces Retirem...

2021-09-08 12:36:00

On September 6, 2021, Laurie B. Keating, a named executive officer of Alnylam Pharmaceuticals, Inc., retired from the company and resigned fro...

Positive

Alnylam Pharmaceuticals, Inc. Presents Addition...

2021-09-07 11:00:00

Alnylam Pharmaceuticals, Inc. announced additional positive results from subgroup analyses and exploratory endpoints of the HELIOS-A Phase 3 s...

Positive

Alnylam Pharmaceuticals, Inc. Presents Addition...

2021-09-07 11:00:00

Alnylam Pharmaceuticals, Inc. announced additional positive results from subgroup analyses and exploratory endpoints of the HELIOS-A Phase 3 s...

Neutral

OLIGONUCLEOTIDE THERAPEUTICS SOCIETY INC, 17th ...

2021-09-04 15:00:00

OLIGONUCLEOTIDE THERAPEUTICS SOCIETY INC, 17th Annual Meeting of the Oligonucleotide Therapeutics Society, Sep 26, 2021 through Sep 29, 2021.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at 17th ...

2021-09-04 15:00:00

Alnylam Pharmaceuticals, Inc. Presents at 17th Annual Meeting of the Oligonucleotide Therapeutics Society, Sep-26-2021 . Speakers: John M. Mar...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Morga...

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Morgan Stanley 19th Annual Global Healthcare Conference, Sep-09-2021 03:30 PM. Venue: New Windsor, U...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Canto...

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Cantor Global Healthcare 2021 Conference, Sep-28-2021 10:40 AM. Venue: New York, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at H.C. ...

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at H.C. Wainwright 23rd Annual Global Investment Conference, Sep-13-2021 07:00 AM. Venue: Lotte New Yor...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Bank ...

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Bank of America Global Healthcare Conference, Sep-16-2021 10:05 AM. Speakers: John M. Maraganore, CE...

Neutral

Evercore Inc., 4th Annual Evercore ISI HealthCO...

2021-08-24 20:29:00

Evercore Inc., 4th Annual Evercore ISI HealthCONx Virtual Conference, Nov 29, 2021 through Dec 03, 2021.

Neutral

Chardan Capital Markets, LLC, Chardan's 5th Ann...

2021-08-24 14:20:00

Chardan Capital Markets, LLC, Chardan's 5th Annual Genetic Medicines Conference, Oct 04, 2021 through Oct 05, 2021.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at World...

2021-08-20 20:21:00

Alnylam Pharmaceuticals, Inc. Presents at World Orphan Drug Congress USA 2021, Aug-25-2021 through Aug-27-2021. Venue: Gaylord National Resort...

Neutral

Alnylam Pharmaceuticals, Inc. Presents at INCLU...

2021-08-20 19:06:00

Alnylam Pharmaceuticals, Inc. Presents at INCLUSION Diversity, Equity and Inclusion Summit, Sep-21-2021 01:00 PM. Speakers: Joseph Vogel, Sen...

Neutral

I.I.R Ltd., INCLUSION Diversity, Equity and In...

2021-08-20 18:44:00

I.I.R Ltd., INCLUSION Diversity, Equity and Inclusion Summit, Sep 21, 2021.

Neutral

Terrapinn Holdings Ltd, World Orphan Drug Congr...

2021-08-20 17:31:00

Terrapinn Holdings Ltd, World Orphan Drug Congress USA 2021, Aug 25, 2021 through Aug 27, 2021. Venue: Gaylord National Resort & Convention C...

Positive

Alnylam Pharmaceuticals, Inc. Completes Enrollm...

2021-08-09 11:00:00

Alnylam Pharmaceuticals, Inc. announced that it has achieved full patient enrollment in its HELIOS-B Phase 3 study of vutrisiran, an investiga...

Negative

Alnylam Pharmaceuticals, Inc. Appoints Michael ...

2021-08-03 12:11:00

Alnylam Pharmaceuticals, Inc. announced that Michael W. Bonney, the Chair of the company’s Board, had been appointed Executive Chair of the Bo...

Positive

Alnylam Pharmaceuticals, Inc. Updates Revenue G...

2021-08-03 12:00:00

Alnylam Pharmaceuticals, Inc. updated revenue Guidance for the full year of 2021. For the period, the company expects Combined net product rev...

Positive

Alnylam and PeptiDream Enter into Collaboration...

2021-07-29 21:00:00

Alnylam Pharmaceuticals, Inc. and PeptiDream, Inc. announced a license and collaboration agreement to discover and develop peptide-siRNA conju...

Positive

Alnylam Pharmaceuticals, Inc. Reports Positive ...

2021-07-29 11:00:00

Alnylam Pharmaceuticals, Inc. announced positive topline results from the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients of al...

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q2, 202...

2021-07-21 12:00:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q2, 2021 results Pre-Market on Aug 03, 2021

Neutral

Alnylam Pharmaceuticals, Inc., Q2 2021 Earnings...

2021-07-21 12:00:00

Alnylam Pharmaceuticals, Inc., Q2 2021 Earnings Call, Aug 03, 2021

Positive

Alnylam Pharmaceuticals, Inc. Initiates KARDIA-...

2021-06-30 11:00:00

Alnylam Pharmaceuticals, Inc. announced initiation of KARDIA-1, a global Phase 2 study evaluating the efficacy and safety of zilebesiran (pron...

Neutral

Alnylam Pharmaceuticals, Inc. - Special Call

2022-06-13 23:22:00

To discuss the FDA approval of AMVUTTRA

Positive

Alnylam Announces FDA Approval of AMVUTTRA (vutrisiran), an RNAi Therapeutic for the Treatment of the Polyneuropathy of Hereditary Transthyretin-Mediated Amyloidosis in Adults

2022-06-13 23:22:00

Alnylam Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) approved AMVUTTRA (vutrisiran), an RNAi therapeutic administered via subcutaneous injection once every three months (quarterly) for the treatment of the polyneuropathy of hereditary transthyretin-mediated (hATTR) amyloidosis in adults. hATTR amyloidosis is a rare, inherited, rapidly progressive, and fatal disease with debilitating polyneuropathy manifestations, for which there are few treatment options. The FDA approval is based on positive 9-month results from the HELIOS-A Phase 3 study, where AMVUTTRA significantly improved the signs and symptoms of polyneuropathy, with more than 50 % of patients experiencing halting or reversal of their disease manifestations. The FDA approval of AMVUTTRA is based on positive 9-month results from HELIOS-A, a global, randomized, open-label, multicenter, Phase 3 study that evaluated the efficacy and safety of AMVUTTRA across a diverse group of patients with hATTR amyloidosis with polyneuropathy. 164 patients with hATTR amyloidosis were randomized 3:1 to receive either 25 mg of vutrisiran (N=122) via subcutaneous injection once every three months or 0.3 mg/kg of patisiran (N=42) via intravenous infusion once every three weeks (reference group) for 18 months. The efficacy of AMVUTTRA was assessed by comparing the AMVUTTRA group in HELIOS-A with the placebo group (n=77) from the landmark APOLLO Phase 3 study of patisiran, a randomized controlled study in a comparable patient population. AMVUTTRA met the primary endpoint of the study, the change from baseline in the modified Neuropathy Impairment Score + 7 (mNIS+7) at 9 months. Treatment with AMVUTTRA (N=114) resulted in a 2.2 point mean decrease (improvement) in mNIS+7 from baseline as compared to a 14.8 point mean increase (worsening) reported for the external placebo group (N=67), resulting in a 17.0 point mean difference relative to placebo (p<0.0001); by 9 months, 50 % of patients treated with AMVUTTRA experienced improvement in neuropathy impairment relative to baseline. AMVUTTRA also met all secondary endpoints in the study at 9 months, with significant improvement in the Norfolk Quality of Life Questionnaire-Diabetic Neuropathy (Norfolk QoL-DN) score and timed 10-meter walk test (10-MWT), and improvements were observed in exploratory endpoints, including change from baseline in modified body mass index (mBMI), all relative to external placebo. Efficacy results at 18 months were consistent with 9-month data, with AMVUTTRA achieving statistically significant improvements compared to external placebo for all secondary endpoints including mNIS+7, Norfolk QoL-DN, 10-MWT and mBMI, and non-inferiority in serum TTR reduction relative to the within-study patisiran reference group.

Positive

Alnylam Reports Positive Topline Results from Phase 2 Study of Investigational Cemdisiran for the Treatment of Iga Nephropathy

2022-06-09 11:00:00

Alnylam Pharmaceuticals, Inc. announced topline results from the Phase 2 study of cemdisiran, an investigational RNAi therapeutic targeting the C5 component of the complement pathway that is in development in collaboration with Regeneron Pharmaceuticals for the treatment of immunoglobulin A nephropathy (IgAN). At Week 32, treatment with cemdisiran resulted in a 37% mean reduction from baseline in the 24-hour urine protein to creatinine ratio relative to placebo – the primary endpoint of the study and an important prognostic marker of disease progression. The results of secondary endpoints were also consistent with a therapeutic benefit of cemdisiran in IgAN. There were no significant drug-related safety signals. The Phase 2 study enrolled a total of 31 patients who were on stable doses of angiotensin converting enzyme (ACE) inhibitor or angiotensin II receptor blocker (ARB); nine were randomized to placebo and 22 to cemdisiran. The primary endpoint of the study was the percent change in the 24-hour urine protein to creatinine ratio (UPCR) at Week 32. Secondary endpoints at Week 32 included: Percent of patients with partial clinical remission (urine protein <1g/24-hours) . Percent of patients with >50% reduction in 24-hour proteinuria . Percent change from baseline in 24-hour proteinuria (g/24-hours) . Change from baseline in UPCR as measured in spot urine. Change from baseline in hematuria . Frequency of adverse events (AEs). This Phase 2 study was descriptive only and did not include statistical hypothesis testing. Treatment with cemdisiran led to a 37% (90% CI: 0, 61) mean reduction in 24-hour urine protein to creatinine ratio relative to placebo. All secondary efficacy endpoints trended in favor of cemdisiran, supporting the therapeutic hypothesis. There were no serious or severe adverse events (AEs) related to study drug, and the overall safety and tolerability profile of cemdisiran supports continued clinical development. One death was reported in the cemdisiran arm due to a post-surgical complication; this was not considered related to study drug by the investigator. There were no discontinuations from study drug due to AEs. Two of nine (22%) patients on placebo and 12 of 22 (55%) of patients on cemdisiran experienced treatment emergent AEs that were related to study drug.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at 2022 Jefferies Global Healthcare Conference, Jun-09-2022 11:00 AM

2022-06-02 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at 2022 Jefferies Global Healthcare Conference, Jun-09-2022 11:00 AM. Venue: Marriott Marquis, New York City, New York, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Goldman Sachs 43rd Annual Global Healthcare Conference, Jun-13-2022 02:40 PM

2022-06-02 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Goldman Sachs 43rd Annual Global Healthcare Conference, Jun-13-2022 02:40 PM. Venue: Terranea Resort, Rancho Palos Verdes, California, United States.

Positive

Alnylam Announces Publication of Preclinical Results Based on Novel Conjugate Technology That Facilitates Delivery of Short Interfering Rna to the Central Nervous System and Other Extrahepatic Tissues

2022-06-02 15:42:00

Alnylam Pharmaceuticals, Inc. announced that data from its preclinical research on delivery of lipophilic short interfering RNA (siRNA) conjugates to extra-hepatic tissues, including the central nervous system (CNS), were published online in Nature Biotechnology. The published data provide early evidence of a potential role for 2'-O-hexadecyl (C16)-conjugated siRNAs in treating diseases of the CNS, eye, and lung. The framework for designing siRNAs for extrahepatic applications leverages Alnylam’s decades long investment in the siRNA delivery platform. The full manuscript titled “Expanding RNAi therapeutics to extrahepatic tissues with lipophilic conjugates” will appear in the June issue of Nature Biotechnology. The published data show that conjugation of C16 to metabolically stable siRNAs enables robust and long-lasting gene silencing in the CNS, eye and lung in rodents and non-human primates (NHPs) with broad cell type specificity and a favorable nonclinical safety profile. The manuscript also provides evidence demonstrating C16-siRNA-mediated gene silencing translates into efficacy in an in vivo mouse model of neurodegenerative disease. Based on the published results, a single intrathecal (IT) administration of C16 conjugated siRNA targeting SOD1 in rodents resulted in a widespread uptake of the conjugate in neurons, astrocytes, and microglial cells and a dose dependent SOD1 knockdown with greater than 75% target knockdown at the highest dose of 0.9 mg. These results were recapitulated in NHPs treated with C16-conjugated siRNA targeting amyloid beta precursor protein (APP), with up to 70 and 80% reductions in the spinal cord and brain, respectively, at three months post a single IT dose of 60 mg. These reductions were sustained beyond three months based on biomarker measurements in the cerebrospinal fluid. The APP-siRNAs were well-tolerated in NHPs and there were no test item-related microscopic findings in the examined brain, spinal cord, and dorsal root ganglia sections. The published data also provide a pre-clinical proof-of-concept, whereby administration of a C16-siRNA targeting both intracellular and extracellular APP in a mouse model of Alzheimer’s disease produced a sufficiently potent and durable knockdown in the CNS to alter both the physiological deficits in the diseased mice, such as Aß deposition and inflammation, as well as, normalizing behavioral deficits as measured by an open field test. Reduction of APP expression was correlated with reduced expression of Iba1, a marker normally upregulated in human disease, and increased levels of glutamate, a metabolite associated with improved cognition. The safety and efficacy of ALN-APP, an investigational RNAi therapeutic conjugated to C16 and targeting APP, is currently being evaluated in a Phase 1 clinical study for the treatment of early onset Alzheimer's disease. C16 conjugation also enables ocular delivery in NHP via intravitreal administration with greater than 95% target knockdown noted in the retinal pigmented epithelium at 100 µg per eye of conjugated siRNA targeting transthyretin (TTR). Similarly, intranasal administration of C16 lipophile conjugated siRNA targeting mouse SOD1 resulted in broad uptake of the conjugate to mouse lung tissue, including bronchioles and alveoli, and 57% SOD1 mRNA knockdown sustained for two months post a 10 mg/kg dose.

Positive

Alnylam Pharmaceuticals, Inc. Presents New Results from the ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1

2022-05-24 11:00:00

Alnylam Pharmaceuticals, Inc. announced new positive results from the six-month primary analysis period of the ILLUMINATE-C Phase 3 open-label study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1– the gene encoding glycolate oxidase (GO) – that is being investigated for the treatment of patients of all ages with advanced primary hyperoxaluria type 1. As previously reported, treatment with lumasiran resulted in substantial reductions in plasma oxalate at six months. Results from new exploratory analyses provide evidence that lumasiran treatment resulted in improvements in cardiac measures, nephrocalcinosis and kidney stone events. In addition, some patients treated with lumasiran experienced improvements in their most burdensome symptoms (as assessed by the investigator), including fatigue, nausea, and bone pain, with no patients reporting worsening of those symptoms. These data were presented at the European Renal Association-European Dialysis and Transplant Association International Congress being held both in Paris, France, and as a virtual event on May 19-22. ILLUMINATE-C enrolled a total of 21 patients, including six who were not on dialysis at study start (Cohort A) and 15 who were on hemodialysis (Cohort B). As previously presented at the American Society of Nephrology (ASN) Kidney Week Meeting in 2021, in Cohorts A and B, respectively, treatment with lumasiran led to 33% and 42% mean reductions in POx from baseline to Month 6. Additionally, treatment with lumasiran led to 35 µmol/L (Cohort A) and 48 µmol/L (Cohort B) mean absolute reductions in POx from baseline to Month 6. In new exploratory analyses, left ventricular ejection fraction (LVEF) and global longitudinal strain (GLS) were measured to assess the impact of lumasiran on the cardiac manifestations of systemic oxalosis. Among patients with abnormal LVEFi at baseline across Cohort A (N=1) and Cohort B (N=4), the patient in Cohort A and two of four patients in Cohort B experienced a = 5% improvement following six months of treatment with lumasiran. Among patients with abnormal GLSii at baseline across Cohort A (N=1) and Cohort B (N=3), all four patients showed a =2% improvement at Month 6. In patients with medullary nephrocalcinosis at baseline in Cohort A (N=5), two patients remained stable, three improved (two unilateral and one bilateral improvement) and none worsened following six months of treatment with lumasiran. In Cohort A, one patient did not have nephrocalcinosis at baseline and had bilateral worsening at six months. In patients with nephrocalcinosis at baseline in Cohort B (N=2), both improved (one unilateral and one bilateral improvement) following six months of treatment with lumasiran. In Cohort A, the rate of kidney stone events per person-year decreased from 3.2 (95% CI: 2, 5.2) in the 12 months prior to consent to 1.5 (95% CI: 0.6, 3.9) during the first six months of treatment with lumasiran. As expected, given patients in Cohort B are on hemodialysis, the rate of kidney stone events was low: 0.07 (95% CI: 0.01, 0.71) in the 12 months prior to consent and 0.0 (95% CI: 0.0, 0.53) during the first six months of treatment with lumasiran. For patients in both cohorts, the most burdensome symptoms at baseline (as assessed by the investigator), including fatigue, nausea/decreased appetite, bone pain and decreased mobility, improved or remained stable following six months of treatment with lumasiran; none of the symptoms worsened. The most common adverse event related to lumasiran was injection-site reaction (ISR) reported in 5 of 21 patients (24%). All ISRs were mild and transient, and the most common symptoms included erythema, discoloration, and hematoma. A separate analysis presented at ERA-EDTA demonstrated that the recommended weight-based dosing regimens of lumasiran showed equivalent efficacy in patients of all ages and degrees of kidney function, including patients on hemodialysis.

Positive

Alnylam Pharmaceuticals, Inc. Announces New Positive Results from an 18-Month Analysis of Exploratory Cardiac Endpoints in the HELIOS-A Phase 3 Study of Vutrisiran

2022-05-23 11:00:00

Alnylam Pharmaceuticals, Inc. announced new positive results from an 18-month analysis of exploratory cardiac endpoints in the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. The 18-month findings show that in a pre-defined cardiac subpopulation, treatment with vutrisiran was associated with improvements in exploratory cardiac endpoints relative to external placebo, including levels of NT-proBNP, a measure of cardiac stress, and a trend towards improvement in echocardiographic parameters. Consistent beneficial effects on cardiac measures were observed in the modified intent-to-treat (mITT) group. Additionally, in a planned cohort of patients from the mITT population, vutrisiran treatment reduced cardiac uptake of technetium on scintigraphy imaging relative to baseline in a majority of assessable patients, including those with Perugini grade =2 at baseline, suggesting that patients with the higher degrees of cardiac amyloid burden may recognize benefit from a TTR silencer. The exploratory findings were presented in a late breaking oral session during the annual congress of the Heart Failure Association of the European Society of Cardiology (Heart Failure 2022; May 21-24, 2022). Alnylampreviously announced that vutrisiran met its primary endpoint and all secondary endpoints at nine months in HELIOS-A, as well as all 18-month secondary endpoints in patients with hereditary ATTR (hATTR) amyloidosis with polyneuropathy, which were presented at the Société Francophone du Nerf Périphérique (SFNP) annual meeting earlier this year. At 18 months, patients with pre-existing evidence of cardiac amyloid involvement (baseline LV wall thickness =1.3 cm and no aortic valve disease or hypertension in medical history; n=40/122 vutrisiran; n=36/77 external placebo group) treated with vutrisiran demonstrated improvement in NT-proBNP levels, with an adjusted geometric fold change of 0.95 compared to 1.93 in the external placebo group (p=0.0004). In the cardiac subpopulation, vutrisiran treatment was also associated with a trend towards improvement (nominal p values) in echocardiographic parameters at Month 18 relative to external placebo, including cardiac output (p=0.0426), LV end-diastolic volume (p=0.0607), global longitudinal strain (p=0.0781) and mean LV wall thickness (p=0.5397). In a planned cohort of patients from the mITT population, vutrisiran also reduced cardiac uptake of technetium on scintigraphy imaging relative to baseline. 96% of assessable patients maintained a stable grade or improved by one grade or greater in the Perugini grading scale at Month 18 relative to baseline. Among vutrisiran-treated patients with Perugini grade =1 at baseline, 50% showed a one grade or greater improvement at Month 18. Of patients with baseline Perugini grade =2 who represent those with evidence of the greater degree of cardiac amyloid burden evaluated in the analysis, the proportion with improvement in normalized left ventricle total uptake and heart-to-contralateral lung ratio was 100% (25/25) and 77% (20/26), respectively. Vutrisiran is under review by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), the Brazilian Health Regulatory Agency (ANVISA) and the Japanese Pharmaceuticals and Medical Devices Agency (PMDA). Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults. In April, Alnylam announced a 3-month extension of the review period for the new drug application for vutrisiran in the U.S. with a new action date under the Prescription Drug User Fee Act (PDUFA) of July 14, 2022.

Positive

Alnylam Pharmaceuticals, Inc. Announces That Health Canada Has Issued A Notice of Compliance (Noc) Authorizing Oxlumo™ (Lumasiran) Injection for Subcutaneous Use for the Treatment

2022-05-18 12:00:00

Alnylam Pharmaceuticals, Inc. announced that Health Canada has issued a Notice of Compliance (NOC) authorizing OXLUMO™ (lumasiran) injection for subcutaneous use for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in paediatric and adult patients. PH1 is an ultra-rare and debilitating genetic disease of the liver characterized by oxalate overproduction. Oxalate is an end-product of metabolism and high levels of it are toxic because it cannot be broken down by the human body. Oxalate overproduction results in the deposition of calcium oxalate crystals in the kidneys and urinary tract and can lead to the formation of painful and recurrent kidney stones, nephrocalcinosis (renal deposition of calcium oxalate crystals), progression to ESKD (kidney failure), and systemic organ dysfunction. There are several types of primary hyperoxaluria (PH), however, PH1 is the most common and the most severe form accounting for 70% to 80% of all PH cases.4 PH1 affects approximately four individuals per million with some regions, such as the Middle East and North Africa having a higher genetic prevalence. Symptom onset ranges from early infancy to sixty years of age with the median age being four to six years. The remainder of affected cases present in adulthood with 20% to 50% presenting late stages of chronic kidney disease when diagnosed. Until today, there were no authorized pharmaceutical therapies for PH1. The only curative treatment is a liver transplant and if the patient has already progressed to kidney failure, then a dual liver/kidney transplant is required. Unfortunately, liver transplantation is associated with high morbidity and mortality, and life-long immunosuppression, leaving patients with limited options. OXLUMO™ is a double-stranded siRNA that reduces the levels of the enzyme glycolate oxidase (GO) responsible for supporting the production of oxalate by targeting the hydroxy acid oxidase 1 (HAO1) messenger ribonucleic acid (mRNA) in major cells of the liver known as hepatocytes through RNA interference. As a result, decreased GO enzyme levels reduce the amount of urinary and plasma oxalate levels, the underlying cause of disease manifestations in patients with PH1.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at H.C. Wainwright Global Investment Conference 2022, May-24-2022 07:00 AM

2022-05-04 12:00:00

Alnylam Pharmaceuticals, Inc. Presents at H.C. Wainwright Global Investment Conference 2022, May-24-2022 07:00 AM. Venue: Fontainebleau Miami Beach Hotel, 4441 Collins Avenue, Miami Beach, Florida, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at UBS Global Healthcare Conference, May-25-2022 10:00 AM

2022-05-04 12:00:00

Alnylam Pharmaceuticals, Inc. Presents at UBS Global Healthcare Conference, May-25-2022 10:00 AM. Venue: Lotte New York Palace, New York City, New York, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Updates Earnings Guidance for the Year 2022

2022-04-28 12:00:00

Alnylam Pharmaceuticals, Inc. updated earnings guidance for the year 2022. For the year, the company expects combined net product revenues for ONPATTRO, GIVLAARI, OXLUMO and vutrisiran of $870 million to $930 million. The company expects net revenues from collaborations and royalties of $175 million to $225 million.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at TIDES (Oligonucleotide & Peptide Therapeutics), May-05-2022

2022-04-23 17:57:00

Alnylam Pharmaceuticals, Inc. Presents at TIDES (Oligonucleotide & Peptide Therapeutics), May-05-2022 . Venue: Boston, Massachusetts, United States. Speakers: Akshay K. Vaishnaw, President, Christopher Theile, Principal Scientist, Joshua Brooks, Associate Director, Joy Zeng, Associate Director, Analytical Development, Muthiah Manoharan, Senior VP of Innovation Chemistry & Distinguished Scientist and Member of Scientific Advisory Board, Punit Seth, SVP, Research, Weinong Guo, Senior Vice President, Clinical Development.

Neutral

I.I.R Ltd., TIDES (Oligonucleotide & Peptide Therapeutics), May 05, 2022 through May 12, 2022

2022-04-23 07:45:00

I.I.R Ltd., TIDES (Oligonucleotide & Peptide Therapeutics), May 05, 2022 through May 12, 2022. Venue: Boston, Massachusetts, United States.

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q1, 2022 Results on Apr 28, 2022

2022-04-14 12:30:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q1, 2022 results Pre-Market on Apr 28, 2022

Neutral

Alnylam Pharmaceuticals, Inc., Q1 2022 Earnings Call, Apr 28, 2022

2022-04-14 12:30:00

Alnylam Pharmaceuticals, Inc., Q1 2022 Earnings Call, Apr 28, 2022

Neutral

Alnylam Pharmaceuticals, Inc. - Shareholder/Analyst Call

2022-04-05 20:19:00

ANNUAL MEETING OF STOCKHOLDERS

Neutral

Alnylam Pharmaceuticals, Inc., Annual General Meeting, May 18, 2022

2022-04-05 20:19:00

Alnylam Pharmaceuticals, Inc., Annual General Meeting, May 18, 2022, at 12:00 US Eastern Standard Time. Agenda: To elect three members to our board of directors, as nominated by board of directors,each to serve as a Class III director for a term ending in 2025, or until a successor has been duly elected and qualified; to approve Alnylam's Amended and Restated 2018 Stock Incentive Plan, to, among other things, increase the number of shares authorized for issuance thereunder by 6,000,000 shares; To approve, on a non-binding advisory basis, the compensation of named executive officers, as described in the Compensation Discussion and Analysis, executive compensation tables and accompanying narrative disclosures in this proxy statement; To ratify the appointment of PricewaterhouseCoopers LLP, an independent registered public accounting firm, as independent auditors for the fiscal year ending December 31, 2022; and to transact any other business that may properly come before the annual meeting or any adjournment or postponement thereof.

Negative

Alnylam Pharmaceuticals, Inc. Announces Retirement of Steven Paul from Board of Directors

2022-04-05 20:00:00

Alnylam Pharmaceuticals, Inc. announced that Steven Paul, M.D. is retiring from the Board of Directors effective April 16, 2022. Dr. Paul has served as a member of the Alnylam Board of Directors since September 2010. He brought to the Board more than 25 years of management experience in the pharmaceutical industry and 35 years of scientific research experience during his time of service. He is widely recognized as a leader across many dimensions of medical research and drug development, including in the central nervous system (CNS), and late-stage clinical development. Dr. Paul is currently the Chief Executive Officer and Chair of the Board of Karuna Therapeutics, Inc., a clinical-stage biopharmaceutical company.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Pharma Manufacturing World Summit, Mar-29-2022

2022-03-29 08:36:00

Alnylam Pharmaceuticals, Inc. Presents at Pharma Manufacturing World Summit, Mar-29-2022 . Venue: Boston Marriott Copley Place, Boston, Massachusetts, United States. Speakers: Alfred W. Boyle, Chief Technical Operations & Quality Officer.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Bank of America 2022 Healthcare Conference, May-11-2022 11:20 AM

2022-03-22 05:03:00

Alnylam Pharmaceuticals, Inc. Presents at Bank of America 2022 Healthcare Conference, May-11-2022 11:20 AM. Venue: Encore Hotel, 3121 Las Vegas Blvd. S, Las Vegas, Nevada, United States. Speakers: Jeffrey V. Poulton, CFO & Executive VP.

Neutral

Executive Platforms Inc., Pharma Manufacturing World Summit, Mar 29, 2022 through Mar 30, 2022

2022-03-21 14:57:00

Executive Platforms Inc., Pharma Manufacturing World Summit, Mar 29, 2022 through Mar 30, 2022. Venue: Boston Marriott Copley Place, Boston, Massachusetts, United States.

Neutral

UBS Investment Bank, UBS Global Healthcare Conference, May 23, 2022 through May 25, 2022

2022-03-03 04:07:00

UBS Investment Bank, UBS Global Healthcare Conference, May 23, 2022 through May 25, 2022. Venue: Lotte New York Palace, New York City, New York, United States.

Neutral

H.C. Wainwright & Co., LLC, H.C. Wainwright Global Investment Conference 2022, May 23, 2022 through May 26, 2022

2022-02-28 18:21:00

H.C. Wainwright & Co., LLC, H.C. Wainwright Global Investment Conference 2022, May 23, 2022 through May 26, 2022. Venue: Fontainebleau Miami Beach Hotel, 4441 Collins Avenue, Miami Beach, Florida, United States.

Neutral

Bank of America Corporation, Bank of America 2022 Healthcare Conference, May 10, 2022 through May 13, 2022

2022-02-17 10:42:00

Bank of America Corporation, Bank of America 2022 Healthcare Conference, May 10, 2022 through May 13, 2022. Venue: Encore Hotel, 3121 Las Vegas Blvd. S, Las Vegas, Nevada, United States.

Neutral

Jefferies LLC, 2022 Jefferies Global Healthcare Conference, Jun 08, 2022 through Jun 10, 2022

2022-02-14 21:00:00

Jefferies LLC, 2022 Jefferies Global Healthcare Conference, Jun 08, 2022 through Jun 10, 2022. Venue: Marriott Marquis, New York City, New York, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Provides Earnings Guidance for the Year 2022

2022-02-10 13:10:00

Alnylam Pharmaceuticals, Inc. provided earnings guidance for the year 2022. For the year, the company expects Combined net product revenues for ONPATTRO, GIVLAARI, OXLUMO and vutrisiran of $900 million to $1,000 million. The company expects net revenues from collaborations and royalties of $175 million to $225 million.

Neutral

The Goldman Sachs Group, Inc., Goldman Sachs 43rd Annual Global Healthcare Conference, Jun 13, 2022 through Jun 16, 2022

2022-02-08 07:31:00

The Goldman Sachs Group, Inc., Goldman Sachs 43rd Annual Global Healthcare Conference, Jun 13, 2022 through Jun 16, 2022. Venue: Terranea Resort, Rancho Palos Verdes, California, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at SLAS 2022 International Conference and Exhibition, Feb-09-2022 01:00 PM

2022-02-05 10:28:00

Alnylam Pharmaceuticals, Inc. Presents at SLAS 2022 International Conference and Exhibition, Feb-09-2022 01:00 PM. Venue: Boston Convention and Exhibition Center, Boston, Massachusetts, United States. Speakers: Tuyen Nguyen.

Neutral

Alnylam Pharmaceuticals, Inc., Q4 2021 Earnings Call, Feb 10, 2022

2022-01-24 13:38:00

Alnylam Pharmaceuticals, Inc., Q4 2021 Earnings Call, Feb 10, 2022

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q4, 2021 Results on Feb 10, 2022

2022-01-24 13:38:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q4, 2021 results Pre-Market on Feb 10, 2022

Negative

Alnylam Pharmaceuticals, Inc. Announces Promotion of Dr. Akshay Vaishnaw to President

2022-01-04 12:30:00

Alnylam Pharmaceuticals, Inc. announced that Akshay Vaishnaw, M.D., Ph.D. has been appointed President. Dr. Vaishnaw joined the company in 2006 as Vice President, Clinical Research with roles of increasing responsibility, ultimately serving as President of Research and Development (R&D). In this new role, Dr. Vaishnaw will serve as the key scientific leader of Alnylam and will advance the company’s R&D strategy focused on sustainable innovation.

Neutral

Society for Laboratory Automation & Screening, SLAS 2022 International Conference and Exhibition, Feb 05, 2022 through Feb 09, 2022

2021-12-28 18:17:00

Society for Laboratory Automation & Screening, SLAS 2022 International Conference and Exhibition, Feb 05, 2022 through Feb 09, 2022. Venue: Boston Convention and Exhibition Center, Boston, Massachusetts, United States.

Positive

Alnylam Comments on FDA Approval of Leqvio, the First Sirna (Rnai Therapeutic) Approved to Reduce LDL-C

2021-12-22 21:23:00

Alnylam Pharmaceuticals, Inc. highlighted the significance of the U.S. Food and Drug Administration’s (FDA) approval of Leqvio® (inclisiran), the fourth small interfering RNA (siRNA) therapy (or RNAi therapeutic) approved in the U.S., and the first and only to lower low-density lipoprotein cholesterol (also known as “bad cholesterol” or LDL-C). Leqvio is indicated in the U.S. as an adjunct to diet and maximally tolerated statin therapy for the treatment of adults with clinical atherosclerotic cardiovascular disease (ASCVD) or heterozygous familial hypercholesterolemia (HeFH) who require additional lowering of LDL-C. Alnylam scientists discovered inclisiran and published the first clinical data. Alnylam also supported early clinical development. As of January 2020, Novartis has obtained global rights to develop, manufacture and commercialize inclisiran under a license and collaboration agreement. Novartis AG continues to develop inclisiran and commercialize Leqvio worldwide, with Alnylam eligible to receive tiered royalties between 10% and 20% on global sales. Leqvio is the fourth Alnylam-discovered medicine using its RNAi therapeutic platform to be approved to date. The Leqvio approval marks the first U.S. approval of an RNAi therapeutic indicated to treat a major risk factor for a highly prevalent disease. Alnylam launched its first RNAi therapeutic in 2018 with the FDA approval of ONPATTRO® (patisiran) for treatment of the polyneuropathy caused by hATTR amyloidosis, a progressive and life-threatening, rare, genetic disease. Leqvio was approved to lower LDL cholesterol and has the potential to benefit millions of people with ASCVD and also those with HeFH around the world. Inclisiran was discovered by Alnylam and, in collaboration with The Medicines Company, advanced into clinical development in 2014. Upon successful completion of Phase 1 clinical studies, the ORION clinical program was launched in 2015. The FDA approval was based on results from the comprehensive Phase III ORION-9, -10 and -11 clinical trials, where all of the 3,457 participants with ASCVD or HeFH had elevated LDL-C while being on a maximally tolerated dose of statin therapy. This comprehensive Phase 3 program represents the largest clinical program conducted to date for an investigational RNAi therapeutic program. Two complementary Phase 3 cardiovascular outcomes trials, ORION-4 and the Novartis initiated VICTORION-2-PREVENT are currently ongoing.

Positive

Alnylam Pharmaceuticals, Inc. Submits Clinical Trial Authorization Application to the Medicines and Healthcare Products Regulatory Agency

2021-12-22 12:00:00

Alnylam Pharmaceuticals, Inc. announced that the Company has submitted a Clinical Trial Authorization (CTA) application to The Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom to initiate a Phase 1 study of ALN-APP, an investigational RNAi therapeutic targeting amyloid precursor protein (APP) for the treatment of Alzheimer’s disease (AD) and cerebral amyloid angiopathy (CAA). The Company plans to initiate the Phase 1 study in patients with early-onset AD in early 2022, upon obtaining MHRA approval, and expects to report initial human data at or around year-end 2022. ALN-APP represents the first-ever RNAi therapeutic targeting CNS diseases. The clinical candidate was designed using Alnylam’s proprietary C16 conjugate technology for the delivery of short interfering RNA (siRNA) to the CNS. As such, ALN-APP represents the start of Alnylam’s expansion of RNAi therapeutic opportunities in extra-hepatic tissues. ALN-APP is being advanced in partnership with Regeneron as part of the companies’ 2019 agreement. Alnylam will lead global development and commercialization of ALN-APP, and Regeneron has exercised its 50-50 co-development and co-commercialization option under the collaboration.

Negative

Yvonne L. Greenstreet Appoints as Chief Executive Officer of Alnylam Pharmaceuticals, Inc., Effective January 1, 2022

2021-12-20 21:15:00

Yvonne L. Greenstreet Alnylam Pharmaceuticals, Inc.’s current President and Chief Operating Officer, has been appointed Chief Executive Officer of the Company effective January 1, 2022.

Positive

Alnylam Pharmaceuticals, Inc. Initiates Phase 2 Study of Lumasiran in Patients with Recurrent Kidney Stone Disease

2021-12-20 21:00:00

Alnylam Pharmaceuticals, Inc. announced that the company has initiated a global Phase 2 study to evaluate the safety and efficacy of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in patients with recurrent kidney stone disease and elevated urinary oxalate levels. Lumasiran is approved by the U.S. Food and Drug Administration (FDA) for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients and by the European Medicines Agency (EMA) for the treatment of PH1 in all age groups, in each region under the brand name OXLUMO. The Phase 2 trial is a randomized, double-blind, placebo-controlled study to evaluate the safety, efficacy, pharmacodynamics, and pharmacokinetics of lumasiran administered subcutaneously in patients with recurrent calcium oxalate kidney stone disease and elevated urinary oxalate levels. This global, multicenter trial is expected to enroll 120 adults with a documented diagnosis of recurrent calcium oxalate kidney stone disease, based on two or more stone events within five years prior to screening, and whose 24-hour urinary oxalate levels are greater than the upper limit of normal. Study participants will be randomized 1:1:1 to receive either placebo or lumasiran at 567 mg or 284 mg on Day 1, Month 3, and Month 9 of a 15-Month double-blind treatment period that includes a six-month primary analysis period followed by a nine-month treatment extension period. The primary endpoint of the Phase 2 study is the percent change in 24-hour urinary oxalate after six months of treatment. Key secondary endpoints include the percentage of patients who achieve a 20% or greater reduction in 24-hour urinary oxalate and percent change in urinary calcium oxalate supersaturation after six months of treatment.

Positive

Alnylam Submits CTA Application for ALN-XDH, an Investigational RNAi Therapeutic for the Treatment of Gout

2021-12-20 12:00:00

Alnylam Pharmaceuticals, Inc. announced that the Company has submitted a clinical trial authorization (CTA) application to The Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom to initiate a Phase 1/2 study of ALN-XDH, an investigational RNAi therapeutic targeting xanthine dehydrogenase (XDH) for the treatment of gout. Upon obtaining MHRA approval, the Company plans to initiate a Phase 1/2 study in healthy volunteers and in patients with gout in early 2022, with initial human data expected in late 2022.

Positive

Alnylam Pharmaceuticals, Inc. Submits Regulatory Applications to the U.S. Food and Drug Administration and European Medicines Agency to Support Label Expansion for Oxlumo® for the Treatment of Advanced Primary Hyperoxaluria Type 1

2021-12-14 12:00:00

Alnylam Pharmaceuticals, Inc. announced the submission of a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration (FDA) and Type II Filing Variation to the European Medicines Agency (EMA) for lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – for the reduction of plasma oxalate in the treatment of patients with advanced primary hyperoxaluria type 1 (PH1). The applications to the FDA and EMA are based on positive results from the ILLUMINATE-C single-arm, open-label Phase 3 study of lumasiran in patients of all ages with advanced PH1. The six-month primary analysis results from the study were presented in November 2021 at the American Society of Nephrology (ASN) Kidney Week annual meeting and showed a substantial reduction in plasma oxalate from baseline in PH1 patients with chronic kidney disease (CKD) stage 3b-5, with or without dialysis. The most common drug-related adverse events were injection site reactions, all of which were mild and transient. Indication: OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. Important Safety Information: Adverse Reactions: The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling. Pregnancy and Lactation: No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at 4th Annual Evercore ISI HealthCONx Virtual Conference, Nov-30-2021 09:40 AM

2021-11-23 13:00:00

Alnylam Pharmaceuticals, Inc. Presents at 4th Annual Evercore ISI HealthCONx Virtual Conference, Nov-30-2021 09:40 AM.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Piper Sandler 33rd Annual Virtual Healthcare Conference, Dec-02-2021 10:00 AM

2021-11-22 15:06:00

Alnylam Pharmaceuticals, Inc. Presents at Piper Sandler 33rd Annual Virtual Healthcare Conference, Dec-02-2021 10:00 AM. Venue: New York, New York, United States. Speakers: John M. Maraganore, CEO & Executive Director.

Positive

Alnylam Pharmaceuticals, Inc. Presents New Data for Zilebesiran, an Investigational RNAi Therapeutic for the Treatment of Hypertension, at the American Heart Association Scientific Sessions 2021

2021-11-13 13:00:00

Alnylam Pharmaceuticals, Inc. announced positive interim results from the ongoing Phase 1 study of zilebesiran (formerly known as ALN-AGT), an investigational subcutaneous RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension. Findings on the pharmacodynamic and antihypertensive effects of zilebesiran six months after a single dose were presented at the American Heart Association (AHA) Scientific Sessions 2021, taking place virtually from November 13-15, 2021. Additional results demonstrated that zilebesiran was safe and well tolerated in the Phase 1 study during salt restriction and when administered concomitantly with irbesartan, an oral renin-angiotensin-aldosterone system inhibitor (RAASi). In the Phase 1 study, 84 patients with hypertension were randomized and enrolled in ascending single dose cohorts of zilebesiran up to 800 mg (N=12 per dose cohort; 2:1 randomization of zilebesiran:placebo). As of the May 28, 2021 data cut-off date, patients treated with single doses of zilebesiran at =100 mg experienced reductions in serum AGT of =90 percent from Week 3 and sustained to Week 12. In the 800 mg dose cohort, durable reductions in serum AGT of >90 percent were maintained through six months. Dose-dependent reductions in blood pressure (BP), assessed by ambulatory blood pressure monitoring (ABPM), were observed in conjunction with AGT knockdown. Mean reductions in 24-hour systolic blood pressure (SBP) of >10 mm Hg were observed at Week 8 after single doses of zilebesiran at =200 mg, with clinically meaningful reductions in BP maintained through six months. After a single dose of 800 mg, mean reductions in 24-hour SBP of >20 mm Hg were observed at Month 6. Patients treated with zilebesiran at =200 mg experienced consistent and sustained lowering of daytime and nighttime SBP through six months, and similar improvements were seen for diastolic blood pressure (DBP), indicating early evidence of the potential for tonic BP control to be achieved by zilebesiran for an extended period. Additional parts of the ongoing Phase 1 study assessed the safety and tolerability of zilebesiran during low-salt intake or when given in combination with irbesartan, an oral RAASi. In the controlled salt diet cohort, 12 patients were randomized 2:1 to receive zilebesiran at 800 mg or placebo, with controlled salt intake delivered through a two-week low-salt/high-salt dietary subprotocol to assess the tolerability of zilebesiran during volume depletion due to sodium loss induced by a low-salt diet. As expected, a reduction in 24-hour mean SBP/DBP was observed in the absence of zilebesiran treatment for all patients on a low-salt diet, with reversal upon switching to a high-salt diet. When assessed on a background of zilebesiran treatment, changes in BP due to a low-salt diet were more profound than seen in placebo patients, consistent with augmented pharmacology. There were no hypotensive events among patients being treated with zilebesiran during a low-salt diet. A high-salt diet was shown to modulate the BP lowering effect of zilebesiran, providing early evidence that this standard intervention could be used to treat potential hypotensive adverse events should they occur. In the irbesartan add-on cohort, 10 patients who received a single dose of open-label zilebesiran at 800 mg proceeded to receive irbesartan daily at 300 mg by oral administration for two weeks. Coadministration with irbesartan reduced 24-hour mean SBP/DBP by an additional 6.4/3.2 mm Hg, with no clinically significant changes in serum creatinine or potassium. Across various cohorts in the Phase 1 study, zilebesiran was shown to be generally well tolerated with an acceptable safety profile that supports continued development. In the ascending single dose study, most adverse events (AEs) were mild or moderate in severity and resolved without intervention, with the most common AE consisting of mild and transient injection site reactions in 5 out of 56 patients (8.9 percent) receiving zilebesiran. In the salt depletion and irbesartan cohorts, all AEs were mild in severity and there were no AEs of concern for hypotensive events. Across all cohorts, there were no treatment-related serious AEs or clinically significant elevations in serum ALT, serum creatinine, or serum potassium, and no patients required intervention for low blood pressure.

Neutral

Alnylam Pharmaceuticals, Inc. - Special Call

2021-11-12 13:00:00

Alnylam Pharmaceuticals, Inc. - Special Call

Positive

Alnylam Pharmaceuticals, Inc. Initiates KARDIA-2 Phase 2 Study of Investigational Zilebesiran (ALN-AGT) in Patients with Inadequately Controlled Hypertension

2021-11-08 12:00:00

Alnylam Pharmaceuticals, Inc. announced initiation of KARDIA-2, a global Phase 2 study to evaluate the efficacy and safety of zilebesiran (formerly known as ALN-AGT), an investigational subcutaneous RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension. KARDIA-2 will evaluate the efficacy and safety of zilebesiran administered biannually as a concomitant therapy in patients whose blood pressure is not adequately controlled by standard of care antihypertensive medications. The primary endpoint of KARDIA-2 is the change from baseline in 24-hour mean systolic blood pressure (SBP) after three months of treatment, as measured by ambulatory blood pressure monitoring (ABPM). Additional endpoints will include change from baseline in blood pressure at six months and time-averaged reduction of blood pressure as a measure of tonic control. Safety will be assessed throughout the study. KARDIA-2 has been activated at clinical sites in the U.S. and will be conducted at approximately 80 clinical study centers worldwide. KARDIA-2 is the second Phase 2 study evaluating zilebesiran in hypertension, with KARDIA-1, announced earlier this year, assessing zilebesiran as monotherapy across different doses administered quarterly and biannually. These Phase 2 studies are based on encouraging Phase 1 data, including results presented earlier this year at the 2021 Joint Meeting of the European Society of Hypertension (ESH) and the International Society of Hypertension (ISH). Additional clinical results from the Phase 1 study of zilebesiran will be presented at the American Heart Association (AHA) Scientific Sessions 2021, being held November 13-15, 2021.

Positive

Alnylam Pharmaceuticals, Inc. Presents Positive Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1

2021-11-05 17:30:00

Alnylam Pharmaceuticals, Inc. announced on November 5, 2021 positive results from the 6-month primary analysis of the ILLUMINATE-C Phase 3 open-label study of lumasiran, an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – that is being investigated for the treatment of patients of all ages with advanced primary hyperoxaluria type 1 (PH1). The clinical data were presented at a late-breaking session at the American Society of Nephrology (ASN) Kidney Week being held as a virtual event on November 4-7. Lumasiran resulted in substantial reductions in plasma oxalate (POx) in PH1 patients with chronic kidney disease (CKD) stage 3b-5, with or without dialysis. Elevated plasma oxalate is directly correlated with the pathophysiology of systemic oxalosis, a life-threatening complication of PH1. Lumasiran demonstrated an acceptable safety profile through Month 6, with mild injection site reactions (ISRs) as the most common drug-related adverse event. ILLUMINATE-C enrolled 21 patients: six patients in Cohort A who did not require dialysis and 15 patients on hemodialysis in Cohort B. The primary efficacy endpoint of the study was the percent change in POx (Cohort A) and predialysis POx (Cohort B) from baseline to Month 6, averaged across Months 3 to 6. Treatment with lumasiran in Cohorts A and B, respectively, led to 33% and 42% mean reductions in POx and predialysis POx from baseline to Month 6. Reduction in POx was evident by Month 1 and persisted through the end of the 6-month primary analysis period. Lumasiran also demonstrated positive results across secondary endpoints, including patients in Cohort B achieving substantial reductions in POx area under the curve (AUC) 0-24h between dialysis sessions from baseline to Month 6, and lumasiran demonstrating consistent reductions across all measures of urinary oxalate in Cohort A. In addition, a similar magnitude of plasma glycolate increase, an exploratory endpoint in Cohorts A and B, was observed as compared to previously completed studies in patients with relatively preserved kidney function (ILLUMINATE-A and ILLUMINATE-B), suggesting similar pharmacodynamics of lumasiran regardless of kidney function. Mild and transient ISRs were the most common drug-related AE reported in 24% (5/21) of patients. There were no deaths and no serious or severe AEs related to lumasiran. Furthermore, there were no treatment discontinuations or study withdrawals. Results from a separate modeling analysis were also presented at ASN Kidney Week, predicting a long-term reduction in kidney failure risk among PH1 patients treated with lumasiran, assuming prompt treatment at diagnosis. OXLUMO is indicated for the treatment of primary hyperoxaluria type 1 (PH1) to lower urinary oxalate levels in pediatric and adult patients. The most common adverse reaction that occurred in patients treated with OXLUMO was injection site reaction (38%). Symptoms included erythema, pain, pruritus, and swelling. No data are available on the use of OXLUMO in pregnant women. No data are available on the presence of OXLUMO in human milk or its effects on breastfed infants or milk production. Consider the developmental and health benefits of breastfeeding along with the mother’s clinical need for OXLUMO and any potential adverse effects on the breastfed child from OXLUMO or the underlying maternal condition.

Neutral

SVB Leerink LLC, 11th SVB Leerink Annual Global Healthcare Conference 2022, Feb 14, 2022 through Feb 18, 2022

2021-10-29 14:57:00

SVB Leerink LLC, 11th SVB Leerink Annual Global Healthcare Conference 2022, Feb 14, 2022 through Feb 18, 2022. Venue: New York, New York, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Provides Earnings Guidance for the Year 2021

2021-10-28 12:00:00

Alnylam Pharmaceuticals, Inc. provided earnings guidance for the year 2021. For the year, the company expects net revenues from collaborations and royalties of $150 million to $200 million. Combined net product revenues for ONPATTRO, GIVLAARI and OXLUMO of $640 million to $665 million.

Negative

Alnylam Pharmaceuticals, Inc. Announces Board Changes

2021-10-28 11:55:00

Alnylam Pharmaceuticals, Inc. announced that effective immediately, Dr. Yvonne Greenstreet has been appointed to the Alnylam Board of Directors as part of the planned succession. Dr. Greenstreet joined Alnylam in 2016 and currently serves as the company’s President and COO.

Negative

Alnylam Announces Planned CEO Leadership Transition

2021-10-28 11:55:00

Alnylam Pharmaceuticals, Inc. announced the planned CEO transition of John Maraganore, Ph.D., to Yvonne Greenstreet, MBChB, at year-end 2021. Dr. Maraganore joined Alnylam in 2002 as the company’s founding CEO and has led the company’s advancement of RNAi therapeutics as a whole new class of innovative medicines, including four approved medicines marketed in over 25 countries around the world. Dr. Greenstreet joined Alnylam in 2016 and currently serves as the company’s President and COO. Dr. Maraganore will support the leadership transition in a consulting capacity through March 31, 2022 and will continue to contribute to Alnylam’s success as a member of the company’s Scientific Advisory Board.

Positive

Alnylam Pharmaceuticals, Inc. Reports Positive Topline 18-Month Results from HELIOS-A Phase 3 Study of Vutrisiran in Patients with hATTR Amyloidosis with Polyneuropathy

2021-10-27 20:00:00

Alnylam Pharmaceuticals, Inc. announced that the HELIOS-A phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of the polyneuropathy associated with hereditary transthyretin-mediated (hATTR) amyloidosis, met all secondary endpoints measured at 18 months, including statistically significant improvements in neuropathy as measured by the modified Neuropathy Impairment Score (mNIS+7), quality of life (QOL), gait speed, nutritional status and overall disability, relative to external placebo data from the APOLLO phase 3 study of patisiran. The final secondary endpoint, reduction in serum TTR levels with vutrisiran, demonstrated non-inferiority relative to the within-study patisiran arm, as expected. In addition, patients treated with vutrisiran showed improvements in exploratory endpoints, including the biomarker NT-proBNP and certain echocardiographic parameters, relative to placebo, and an improvement in technetium uptake relative to baseline in a majority of patients in a planned cohort, providing potential evidence for reduced cardiac amyloid burden. Vutrisiran continued to demonstrate an encouraging safety and tolerability profile. Alnylam previously announced that HELIOS-A met its primary and secondary endpoints at nine months and study results were presented at the 2021 American Academy of Neurology(AAN) Virtual Annual Meeting. At 18 months, patients treated with vutrisiran showed quantitative improvement across a number of exploratory endpoints. Compared to placebo, patients in the vutrisiran arm demonstrated improvement in the cardiac biomarker endpoint, NT-proBNP, a measure of cardiac stress. In addition, patients treated with vutrisiran also demonstrated improvement in certain echocardiographic parameters, relative to placebo. Finally, in a planned cohort of 48 patients, treatment with vutrisiran was associated with an improvement in technetium uptake in the heart in a majority of patients, providing potential evidence for reduced cardiac amyloid burden. Vutrisiran demonstrated an encouraging safety profile. There were three study discontinuations (2.5%) due to adverse events in the vutrisiran arm during the 18 Month treatment period; the single new discontinuation since Month 9 was an event of cardiac failure considered unrelated to study drug by the investigator. During the 18 Month treatment period, there were two deaths (neither of which was considered related to study drug) and two serious adverse events (SAEs) deemed related to vutrisiran by the study investigator; these deaths and related SAEs all occurred by Month 9 and were previously reported. Treatment emergent adverse events (AEs) occurring in 10% or more patients included fall, pain in extremity, diarrhea, peripheral edema, urinary tract infection, arthralgia and dizziness; with the exception of pain in extremity and arthralgia, each of these events occurred at a similar or lower rate as compared with external placebo. Injection site reactions (ISRs) were reported in 5 patients (4.1%) and were all mild and transient. There were no hepatic safety concerns. Vutrisiran is under review by the U.S. Food and Drug Administration (FDA), the European Medicines Agency (EMA), and the Brazilian Health Regulatory Agency (ANVISA). Vutrisiran has been granted Orphan Drug Designation in the U.S. and the European Union (EU) for the treatment of ATTR amyloidosis. Vutrisiran has also been granted a Fast Track designation in the U.S. for the treatment of the polyneuropathy of hATTR amyloidosis in adults. In the U.S., vutrisiran has received an action date under the Prescription Drug User Fee Act (PDUFA) of April 14, 2022. The company received Orphan Drug Designation in Japan for transthyretin type familial amyloidosis with polyneuropathy.

Positive

Alnylam Pharmaceuticals, Inc. Receives Positive Reimbursement Recommendation from the Canadian Agencies for Drugs and Technology in Health (CADTH) for Use of GIVLAARI® for Treatment of Acute Hepatic Porphyria (AHP) in Adults

2021-10-18 12:00:00

Alnylam Pharmaceuticals, Inc. announced that GIVLAARI® (givosiran) has now received a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH). GIVLAARI® is approved by Health Canada for subcutaneous use for the treatment of acute hepatic porphyria (AHP) in adults. AHP is an ultra-rare condition in which patients can experience debilitating attacks of severe abdominal pain, vomiting and seizures. It can be life-threatening due to the possibility of paralysis and respiratory arrest during attacks. Many patients also experience chronic symptoms, including severe pain, which continues to be present between attacks, linked to increased rates of liver cancer, kidney failure and hypertension. The positive recommendation for reimbursement was supported by the results of the ENVISION Phase 3 Study, a randomized, double-blind, placebo-controlled, global, multicenter study to evaluate the efficacy and safety of GIVLAARI® in patients with a documented diagnosis of acute hepatic porphyria (AHP). The primary endpoint was reduction relative to placebo in the annualized rate of composite porphyria attacks, defined as those requiring hospitalization, urgent healthcare visit, or intravenous hemin administration at home, in patients with acute intermittent porphyria (AIP, the most common subtype of AHP) over six months.

Neutral

Alnylam Pharmaceuticals, Inc., Q3 2021 Earnings Call, Oct 28, 2021

2021-10-14 12:00:00

Alnylam Pharmaceuticals, Inc., Q3 2021 Earnings Call, Oct 28, 2021

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q3, 2021 Results on Oct 28, 2021

2021-10-14 12:00:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q3, 2021 results Pre-Market on Oct 28, 2021

Positive

Alnylam Pharmaceuticals, Inc. Announces Launch of “Alnylam Challengers,” A Signature Community Impact Program to Improve Health Access to Underserved Communities

2021-10-13 11:30:00

Alnylam Pharmaceuticals, Inc. announced the launch of Alnylam Challengers, a signature community impact program that will support bold and diverse organizations in their pursuit of developing new and sustainable solutions to address challenges in health equity. Through this program, Alnylam will collaborate with social entrepreneurs, employees, and business partners to create innovative health solutions to tackle obstacles to accessing health care such as poverty, racial discrimination, and other social determinants of health. Alnylam’s first U.S. partnership is with Acumen America, the U.S. division of a global nonprofit that raises philanthropic dollars and invests them in social impact-driven companies, leaders and ideas that take on world poverty. Alnylam is contributing $1 million to support Acumen America in its efforts to impact 75 million people in the U.S. by 2024. The three focus areas of Alnylam Challengers include: Social Investment Portfolio: A strategic social investment model to tackle health inequities by partnering with a diverse and boldly innovative set of social entrepreneurs, enterprises, and non-profit organizations. Advisory Corps: A skills-based volunteerism initiative where Alnylam employee teams serve as subject-matter expert (SME) advisors to address health equity issues through innovative business models. Convenings: A series of cross-sector forums that embed Alnylam into industry, national, and international dialogue around pressing issues of health equity and rally others to think boldly and partner on important health issues. Alnylam’s Corporate Responsibility actions are organized across five interconnected, stakeholder-related focus areas: patients, science, employees, communities, and planet. Through Challengers, Alnylam is advancing its commitment to improve the health of humanity by investing in social organizations that are actively tackling the most pressing community and health equity challenges.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at International Oligonucleotide and Peptide Conference, Oct-18-2021 02:35 PM

2021-09-30 20:47:00

Alnylam Pharmaceuticals, Inc. Presents at International Oligonucleotide and Peptide Conference, Oct-18-2021 02:35 PM. Speakers: Muthiah Manoharan, Senior VP of Innovation Chemistry & Distinguished Scientist and Member of Scientific Advisory Board.

Neutral

Tekno Scienze S.R.L., International Oligonucleotide and Peptide Conference, Oct 18, 2021 through Oct 19, 2021

2021-09-30 20:01:00

Tekno Scienze S.R.L., International Oligonucleotide and Peptide Conference, Oct 18, 2021 through Oct 19, 2021.

Positive

Alnylam Pharmaceuticals, Inc. Announces New Pre-Clinical Research Results Demonstrating Continued RNAi Therapeutics Platform Innovation and Scientific Leadership

2021-09-29 11:30:00

Alnylam Pharmaceuticals, Inc. announced new pre-clinical research results demonstrating continued RNAi therapeutics platform innovation and scientific leadership. The research was presented at the 17th Annual Meeting of the Oligonucleotide Therapeutics Society (OTS), held virtually from September 26 – 29, 2021. Among the presentations from Alnylam scientists, new pre-clinical data were presented identifying a hexadecyl (C16) moiety as a lipophilic ligand by which conjugation to siRNAs enables the potential for safe, robust, and durable target knockdown in the central nervous system following intrathecal administration. In addition, pre-clinical platform data were presented highlighting the Company’s extended duration platform, called IKARIA™, which has the potential to enable highly potent (i.e., >90 percent) target mRNA silencing with an annual dosing regimen. New pre-clinical data were presented from Alnylam’s CNS-targeting platform. Various fatty acid ligands were conjugated to SOD1-targeting siRNAs and evaluated in rats. A hexadecyl (C16)-conjugated siRNA was shown to yield robust and durable target knockdown throughout the brain and spinal cord following intrathecal administration. Combining a 5’-vinyl phosphonate (VP) modification with C16 provided the greatest level of potency. This chemistry was therefore selected for further evaluation. In rat studies, single or multiple intrathecal doses of a SOD1-targeting siRNA led to robust, dose-dependent, and durable silencing of SOD1 throughout the brain. Further, in toxicology studies performed in rats and NHPs, no test article-related findings were seen for C16 conjugates, suggesting the potential for a clean safety and tolerability profile. Additional pre-clinical data were presented with an siRNA targeting amyloid precursor protein (APP). Alnylam is developing ALN-APP as a potential treatment for early onset Alzheimer’s Disease (AD) and cerebral amyloid angiopathy (CAA), with a planned Clinical Trial Application (CTA) filing in late 2021. An APP siRNA was shown in vitro to cause greater intracellular reductions in soluble APP beta (sAPPß) fragments compared to ß-site APP cleaving enzyme (BACE) inhibitors. Moreover, in an experiment using induced pluripotent stem cell (iPSC)-derived neurons, treatment with APP siRNA was able to reduce the size of Rab5+ early endosomes back to wild-type levels within two weeks of treatment compared to control treated cells. This result shows that siRNA-mediated APP lowering corrects an early intracellular phenotype known to be linked to neurotoxicity in AD patients. Finally, in a transgenic mouse model of human sAPP, siRNA-mediated silencing of APP led to phenotypic improvements. Further, in CTA-enabling GLP toxicology studies in rats and NHPs there were no test-article related findings with ALN-APP, revealing an encouraging non-clinical tolerability profile in support of upcoming human studies. In a separate presentation, initial pre-clinical results were presented on the Company’s IKARIA platform. Using the proprietary IKARIA design and chemistry, RNA sequencing analysis revealed that a transthyretin (TTR)-targeting siRNA delivered to hepatocytes, ALN-TTRsc04, achieved exquisite specificity. The IKARIA-derived ALN-TTRsc04 molecule also demonstrated comparable potency and durability with an analog of vutrisiran in NHP studies. Computational modeling data suggest that a single 300 mg subcutaneous dose of ALN-TTRsc04 may result in trough TTR reduction greater than 90% that is durable for approximately one year. ALN-TTRsc04 is planned to enter clinical development at or around year-end 2022 with an investigational new drug (IND) application filing. Additional OTS presentations by Alnylam included previously shown results on: Development of lumasiran for the treatment of primary hyperoxaluria type 1; Interim results from a Phase 1 study of zilebesiran (ALN-AGT), an investigational RNAi therapeutic in development for the treatment of hypertension.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Chardan's 5th Annual Genetic Medicines Conference, Oct-05-2021 02:30 PM

2021-09-28 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Chardan's 5th Annual Genetic Medicines Conference, Oct-05-2021 02:30 PM.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Longwood Healthcare Leaders Conference, Oct-05-2021 02:50 PM

2021-09-28 18:44:00

Alnylam Pharmaceuticals, Inc. Presents at Longwood Healthcare Leaders Conference, Oct-05-2021 02:50 PM. Speakers: Kevin Fitzgerald, Senior VP & Chief Scientific Officer.

Neutral

Longwood Fund, Longwood Healthcare Leaders Conference, Oct 04, 2021 through Oct 05, 2021

2021-09-28 13:09:00

Longwood Fund, Longwood Healthcare Leaders Conference, Oct 04, 2021 through Oct 05, 2021.

Positive

Alnylam Pharmaceuticals, Inc. Announces Onpattro® is Now Reimbursed Almost Everywhere in Canada for the Treatment of Hereditary ATTR Amyloidosis with Polyneuropathy

2021-09-28 12:00:00

Alnylam Pharmaceuticals, Inc. announced that ONPATTRO® (patisiran) is now reimbursed almost everywhere in Canada for the treatment of hereditary ATTR (hATTR) amyloidosis with polyneuropathy. This comes following a positive recommendation for reimbursement from the Canadian Agency for Drugs and Technologies in Health (CADTH) and the Institut national d'excellence en santé et services sociaux (INESSS). hATTR amyloidosis is a hereditary, multisystemic, progressive disease caused by mutations that interfere with the way the body manufactures a specific protein formed in the liver. Known as gene silencing, RNAi therapeutics is a new approach to the treatment of the disease; targeting the faulty protein that causes the condition. ONPATTRO is the only treatment that has demonstrated improvement, relative to baseline, in both polyneuropathy and quality of life measurements in patients with hATTR amyloidosis. The positive recommendations for reimbursement were supported by the results of the Phase III APOLLO study, which showed that ONPATTRO was associated with a statistically significant difference versus placebo in change from baseline, as well as improvements from baseline in key study end points for those receiving ONPATTRO versus placebo.5 Statistically significant differences in change from baseline versus placebo were also demonstrated favouring ONPATTRO for disability, gait speed, unintended weight loss, and autonomic symptoms.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Cell & Gene Therapy Bioprocessing & Commercialization, Sep-20-2021

2021-09-16 14:42:00

Alnylam Pharmaceuticals, Inc. Presents at Cell & Gene Therapy Bioprocessing & Commercialization, Sep-20-2021 . Speakers: John M. Maraganore, CEO & Executive Director, Muthiah Manoharan, Senior VP of Innovation Chemistry & Distinguished Scientist and Member of Scientific Advisory Board.

Neutral

Informa plc, Cell & Gene Therapy Bioprocessing & Commercialization, Sep 20, 2021 through Sep 30, 2021

2021-09-08 23:01:00

Informa plc, Cell & Gene Therapy Bioprocessing & Commercialization, Sep 20, 2021 through Sep 30, 2021.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at BioProcess International Conference & Exhibition, Sep-20-2021

2021-09-08 22:27:00

Alnylam Pharmaceuticals, Inc. Presents at BioProcess International Conference & Exhibition, Sep-20-2021 . Speakers: John M. Maraganore, CEO & Executive Director.

Neutral

Informa plc, BioProcess International Conference & Exhibition, Sep 20, 2021 through Sep 30, 2021

2021-09-08 21:27:00

Informa plc, BioProcess International Conference & Exhibition, Sep 20, 2021 through Sep 30, 2021.

Negative

Alnylam Pharmaceuticals, Inc. Announces Retirement of Laurie B. Keating as Executive Vice President, Chief Legal Officer and Secretary, Effective on September 10, 2021

2021-09-08 12:36:00

On September 6, 2021, Laurie B. Keating, a named executive officer of Alnylam Pharmaceuticals, Inc., retired from the company and resigned from her position as Executive Vice President, Chief Legal Officer and Secretary of the company, effective September 10, 2021, following 7 years of service.

Positive

Alnylam Pharmaceuticals, Inc. Presents Additional 9-Month Data from HELIOS-A Phase 3 Study of Vutrisiran at the European ATTR (EU-ATTR) Amyloidosis Meeting

2021-09-07 11:00:00

Alnylam Pharmaceuticals, Inc. announced additional positive results from subgroup analyses and exploratory endpoints of the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. Data presented at the 3rd European ATTR Amyloidosis Meeting further supports and builds on the previously reported primary and secondary endpoint results of the HELIOS-A study in hereditary ATTR amyloidosis patients with polyneuropathy, with improvements observed across important areas of patient health and function, including neuropathy impairment, Quality of Life (QoL), ability to perform daily activities and social engagement, nutritional status, and cardiac stress. The HELIOS-A study included 164 hATTR amyloidosis patients with polyneuropathy. At 9 months, vutrisiran met the primary and all secondary endpoints, with statistically significant improvements in neuropathy impairment, QoL, and gait speed, relative to external placebo. The subgroup analyses and exploratory efficacy data from HELIOS-A provide additional insights on the potential benefits of vutrisiran across important aspects of patients’ health and wellbeing, specifically: the modified Neuropathy Impairment Score (mNIS+7) and Norfolk Quality of Life-Diabetic Neuropathy Score (Norfolk QOL-DN) were consistently improved with vutrisiran treatment compared with external placebo across all pre-specified patient subgroups at month 9, including age, sex, race, geographic region, baseline neuropathy impairment, genotype, prior TTR stabilizer use, baseline Familial Amyloid Polyneuropathy (FAP) stage, and in a pre-specified cardiac subpopulation . improvements were observed in the Rasch-built Overall Disability Scale (R-ODS), a comprehensive patient-ranked 24-item scale which assesses activity and social participation limitations on a linearly weighted scale, compared with external placebo. Activity measures increase in intensity and include ability to perform activities of daily living and social engagement. [LS mean difference vs. placebo 4.3 (2.7, 6.0) p=3.26x10-7]. modified body mass index (mBMI) levels increased in patients receiving vutrisiran treatment compared with external placebo, indicating improvements in nutritional status over 9 months [LS mean difference vs. placebo 67.8 (43.0, 92.6) p=8.46×10-8] . QoL (EuroQol Visual Analog Scale, EQ-VAS, self-rated health score) and neuropathy impairment (NIS), assessing patients’ muscle strength, sensation and reflexes, also improved in patients receiving vutrisiran versus external placebo at 9 months. [EQ-VAS LS mean difference vs. placebo 9.8 (4.8, 14.9) p=0.0001; NIS LS mean difference vs. placebo -13.7 (- 17.3, -10.1) p=1.08 x 10-13] . improvement in the exploratory endpoint of the cardiac biomarker, NT-proBNP, a measure of cardiac stress, was observed in the vutrisiran arm in both the prespecified cardiac subpopulation [adjusted geometric fold change ratio: 0.60 (0.43, 0.82) p=0.0016] and the modified intent-to-treat (mITT) population [adjusted geometric fold change ratio: 0.63 (0.52, 0.75) p=9.2×10-7], relative to external placebo.

Positive

Alnylam Pharmaceuticals, Inc. Presents Additional 9-Month Data from Helios-A Phase 3 Study of Vutrisiran at the European ATTR Amyloidosis Meeting

2021-09-07 11:00:00

Alnylam Pharmaceuticals, Inc. announced additional positive results from subgroup analyses and exploratory endpoints of the HELIOS-A Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of transthyretin-mediated (ATTR) amyloidosis. Data presented at the 3rd European ATTR Amyloidosis Meeting further supports and builds on the previously reported primary and secondary endpoint results of the HELIOS-A study in hereditary ATTR amyloidosis patients with polyneuropathy, with improvements observed across important areas of patient health and function, including neuropathy impairment, Quality of Life (QoL), ability to perform daily activities and social engagement, nutritional status, and cardiac stress. Additionally, patients experienced improvements in neuropathy impairment and quality of life scores regardless of prior TTR stabilizer use. The HELIOS-A study included 164 hATTR amyloidosis patients with polyneuropathy. At 9 months, vutrisiran met the primary and all secondary endpoints, with statistically significant improvements in neuropathy impairment, QoL, and gait speed, relative to external placebo.

Neutral

OLIGONUCLEOTIDE THERAPEUTICS SOCIETY INC, 17th Annual Meeting of the Oligonucleotide Therapeutics Society, Sep 26, 2021 through Sep 29, 2021

2021-09-04 15:00:00

OLIGONUCLEOTIDE THERAPEUTICS SOCIETY INC, 17th Annual Meeting of the Oligonucleotide Therapeutics Society, Sep 26, 2021 through Sep 29, 2021.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at 17th Annual Meeting of the Oligonucleotide Therapeutics Society, Sep-26-2021

2021-09-04 15:00:00

Alnylam Pharmaceuticals, Inc. Presents at 17th Annual Meeting of the Oligonucleotide Therapeutics Society, Sep-26-2021 . Speakers: John M. Maraganore, CEO & Executive Director, Kirk Brown, Senior Director, Martin Maier, Vice President, RNAi Discover, Muthiah Manoharan, Senior VP of Innovation Chemistry & Distinguished Scientist and Member of Scientific Advisory Board, Pushkal Garg, Chief Medical Officer, Stephen Huang, Senior Director, Clinical Development.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Morgan Stanley 19th Annual Global Healthcare Conference, Sep-09-2021 03:30 PM

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Morgan Stanley 19th Annual Global Healthcare Conference, Sep-09-2021 03:30 PM. Venue: New Windsor, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Cantor Global Healthcare 2021 Conference, Sep-28-2021 10:40 AM

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Cantor Global Healthcare 2021 Conference, Sep-28-2021 10:40 AM. Venue: New York, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at H.C. Wainwright 23rd Annual Global Investment Conference, Sep-13-2021 07:00 AM

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at H.C. Wainwright 23rd Annual Global Investment Conference, Sep-13-2021 07:00 AM. Venue: Lotte New York Palace Hotel, New York City, New York, United States.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at Bank of America Global Healthcare Conference, Sep-16-2021 10:05 AM

2021-09-01 20:00:00

Alnylam Pharmaceuticals, Inc. Presents at Bank of America Global Healthcare Conference, Sep-16-2021 10:05 AM. Speakers: John M. Maraganore, CEO & Executive Director.

Neutral

Evercore Inc., 4th Annual Evercore ISI HealthCONx Virtual Conference, Nov 29, 2021 through Dec 03, 2021

2021-08-24 20:29:00

Evercore Inc., 4th Annual Evercore ISI HealthCONx Virtual Conference, Nov 29, 2021 through Dec 03, 2021.

Neutral

Chardan Capital Markets, LLC, Chardan's 5th Annual Genetic Medicines Conference, Oct 04, 2021 through Oct 05, 2021

2021-08-24 14:20:00

Chardan Capital Markets, LLC, Chardan's 5th Annual Genetic Medicines Conference, Oct 04, 2021 through Oct 05, 2021.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at World Orphan Drug Congress USA 2021, Aug-25-2021 through Aug-27-2021

2021-08-20 20:21:00

Alnylam Pharmaceuticals, Inc. Presents at World Orphan Drug Congress USA 2021, Aug-25-2021 through Aug-27-2021. Venue: Gaylord National Resort & Convention Center, Oxon Hill, Maryland, United States. Presentation Date & Speakers: Aug-25-2021, Pushkal Garg, Chief Medical Officer. Aug-26-2021, Pushkal Garg, Chief Medical Officer.

Neutral

Alnylam Pharmaceuticals, Inc. Presents at INCLUSION Diversity, Equity and Inclusion Summit, Sep-21-2021 01:00 PM

2021-08-20 19:06:00

Alnylam Pharmaceuticals, Inc. Presents at INCLUSION Diversity, Equity and Inclusion Summit, Sep-21-2021 01:00 PM. Speakers: Joseph Vogel, Senior Manager, Program & Alliance Management; D&I Core Team Member & SAGA ERN Leadership Team Member.

Neutral

I.I.R Ltd., INCLUSION Diversity, Equity and Inclusion Summit, Sep 21, 2021

2021-08-20 18:44:00

I.I.R Ltd., INCLUSION Diversity, Equity and Inclusion Summit, Sep 21, 2021.

Neutral

Terrapinn Holdings Ltd, World Orphan Drug Congress USA 2021, Aug 25, 2021 through Aug 27, 2021

2021-08-20 17:31:00

Terrapinn Holdings Ltd, World Orphan Drug Congress USA 2021, Aug 25, 2021 through Aug 27, 2021. Venue: Gaylord National Resort & Convention Center, Oxon Hill, Maryland, United States.

Positive

Alnylam Pharmaceuticals, Inc. Completes Enrollment in HELIOS-B Phase 3 Study of Investigational Vutrisiran in Patients with Transthyretin-Mediated (ATTR) Amyloidosis with Cardiomyopathy

2021-08-09 11:00:00

Alnylam Pharmaceuticals, Inc. announced that it has achieved full patient enrollment in its HELIOS-B Phase 3 study of vutrisiran, an investigational RNAi therapeutic in development for the treatment of cardiomyopathy in patients with transthyretin-mediated (ATTR) amyloidosis. Enrollment was completed, significantly ahead of schedule, with more than 600 ATTR amyloidosis patients across 123 activated sites in 32 countries. The HELIOS-B study was designed to evaluate the safety and efficacy of investigational vutrisiran, an RNAi therapeutic subcutaneously administered once every three months for the potential treatment of cardiomyopathy in ATTR amyloidosis patients. The primary endpoint will evaluate the efficacy of vutrisiran versus placebo on the composite endpoint of all-cause mortality and recurrent cardiovascular (CV) events (CV hospitalizations and urgent heart failure visits) at 30 months in ATTR amyloidosis patients with cardiomyopathy. Topline full results from HELIOS-B are expected in early 2024. The HELIOS-B protocol includes an optional interim analysis which, if pursued, would be conducted following the data readout from Alnylam’s APOLLO-B Phase 3 clinical study of patisiran in patients with ATTR amyloidosis with cardiomyopathy. The APOLLO-B study completed enrollment in June 2021 and is expected to have topline data available in mid-2022. Based on those data and subsequent regulatory interactions, the potential path forward for a HELIOS-B interim analysis will be further refined, including potential for an earlier readout of topline results. HELIOS-B is a global, Phase 3, randomized, double-blind, placebo-controlled study to evaluate the efficacy and safety of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy, which enrolled more than 600 adult patients with ATTR amyloidosis (including both hATTR and wtATTR amyloidosis) with cardiomyopathy. Patients were randomized on a 1:1 basis to receive either 25 mg of vutrisiran or placebo administered as a subcutaneous injection once every three months for up to 36 months. The primary endpoint will evaluate the efficacy of vutrisiran versus placebo on the composite endpoint of all-cause mortality and recurrent cardiovascular (CV) events (CV hospitalizations and urgent heart failure (HF) visits) at 30 months. Vutrisiran is an investigational, subcutaneously administered RNAi therapeutic in development for the treatment of ATTR amyloidosis, which encompasses both hereditary ATTR (hATTR) and wild-type ATTR (wtATTR) amyloidosis. It is designed to target and silence specific messenger RNA, with the goal of blocking the production of wild-type and variant transthyretin (TTR) protein before it is made. Quarterly, and potentially biannual, administration of vutrisiran may help to reduce deposition and facilitate the clearance of TTR amyloid deposits in tissues and potentially restore function to these tissues. Vutrisiran utilizes Alnylam’s Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate delivery platform, designed for increased potency and high metabolic stability that may allow for infrequent subcutaneous injections. The U.S. Food and Drug Administration is currently evaluating the New Drug Application (NDA) for vutrisiran for the treatment of the polyneuropathy of hATTR amyloidosis. The safety and efficacy of vutrisiran for this indication or for the treatment of cardiomyopathy of ATTR amyloidosis have not been evaluated by the U.S. Food and Drug Administration, European Medicines Agency, or any other health authority. Transthyretin-mediated (ATTR) amyloidosis is a rare, rapidly progressive, debilitating disease caused by misfolded transthyretin (TTR) proteins which accumulate as amyloid fibrils in multiple tissues including the nerves, heart, and gastrointestinal (GI) tract. There are two different types of ATTR amyloidosis – Hereditary ATTR (hATTR) amyloidosis, caused by a TTR gene variant, and wild-type ATTR (wtATTR) amyloidosis, which occurs without a TTR gene variant. hATTR amyloidosis affects approximately 50,000 people worldwide, while wtATTR amyloidosis is estimated to impact 200,000 – 300,000 people worldwide. RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as “a major scientific breakthrough that happens once every decade or so,” and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in cells, a new class of medicines, known as RNAi therapeutics, is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam’s RNAi therapeutic platform, function upstream of today’s medicines by silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.

Negative

Alnylam Pharmaceuticals, Inc. Appoints Michael W. Bonney as Chair

2021-08-03 12:11:00

Alnylam Pharmaceuticals, Inc. announced that Michael W. Bonney, the Chair of the company’s Board, had been appointed Executive Chair of the Board to further enhance the company’s ethics and compliance function and its integration with the business, in what is expected to be a temporary expansion of his role. Mr. Bonney will continue to serve in his historic role as Chair, including working with Dr. Maraganore and Management to ensure alignment with the Board on strategy. In connection with Mr. Bonney’s new role, Amy W. Schulman has been appointed by the Board to serve as Lead Independent Director of the Board. These appointments became effective August 2, 2021.

Positive

Alnylam Pharmaceuticals, Inc. Updates Revenue Guidance for the Full Year of 2021

2021-08-03 12:00:00

Alnylam Pharmaceuticals, Inc. updated revenue Guidance for the full year of 2021. For the period, the company expects Combined net product revenues for ONPATTRO, GIVLAARI and OXLUMO to be in the range of $640 million to $665 million. Net revenues from collaborations and royalties expected to be in the range of $150 million to $200 million with no change.

Positive

Alnylam and PeptiDream Enter into Collaboration Agreement to Discover and Develop Peptide-siRNA Conjugates for Targeted Delivery of RNAi Therapeutics to a Broader Range of Extrahepatic Tissues

2021-07-29 21:00:00

Alnylam Pharmaceuticals, Inc. and PeptiDream, Inc. announced a license and collaboration agreement to discover and develop peptide-siRNA conjugates to create multiple opportunities to deliver RNAi therapeutics to tissues outside the liver. Through this collaboration, the companies will collaborate to select and optimize peptides for targeted delivery of small interfering RNA (siRNA) molecules to a wide range of cell types and tissues via specific interactions with receptors expressed on the target cells. Under the terms of the alliance, Alnylam will select a set of receptors for PeptiDream’s peptide discovery platform. PeptiDream will select, optimize, and synthesize peptides for each receptor. Alnylam will then generate peptide-siRNA conjugates and perform in vitro and in vivo studies to support final peptide selection. The collaboration has the potential to yield multiple treatment opportunities by targeting disease causing mRNA transcripts in a wide variety of tissue types. Under the terms of the agreement, PeptiDream will receive an upfront payment from Alnylam as well as R&D funding over the term of the research collaboration, as provided in the agreement. PeptiDream may also receive payments based on the achievement of specified development, regulatory, and commercial milestones potentially totaling up to $2.2 billion (¥244 billion). In addition, PeptiDream is eligible to receive low-to-mid single digit royalties on sales on any such Alnylam products.

Positive

Alnylam Pharmaceuticals, Inc. Reports Positive Topline Results from ILLUMINATE-C Phase 3 Study of Lumasiran in Patients with Advanced Primary Hyperoxaluria Type 1

2021-07-29 11:00:00

Alnylam Pharmaceuticals, Inc. announced positive topline results from the ILLUMINATE-C Phase 3 open-label study of lumasiran in patients of all ages with advanced primary hyperoxaluria type 1 (PH1) associated with progressive decline in renal function. Lumasiran is an RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – that is being investigated for the treatment of adult and pediatric patients with advanced PH1. Results of the primary analysis at six months demonstrated substantial reduction in plasma oxalate from baseline in patients (N=21) with advanced disease, including those on hemodialysis. Elevated plasma oxalate is directly related to the pathophysiology of oxalosis and results in systemic deposition of oxalate in extra-renal tissues, potentially leading to bone fractures, cardiomyopathy, impaired erythropoiesis, vision loss, skin ulcers, and other serious manifestations1. The safety and tolerability profile of lumasiran following six months of treatment is encouraging across all ages, with no drug related serious adverse events (SAEs) and injection site reactions (ISRs) as the most common adverse event (AE). ILLUMINATE-C (NCT04152200) is a single arm, open-label, multinational Phase 3 study evaluating the safety and efficacy of lumasiran in PH1 patients of all ages with severe renal impairment (eGFR = 45 mL/min/1.73m2 or elevated serum creatinine for patients <12 months of age), and conducted at 13 study sites across 10 countries around the world. Cohort A enrolled six patients with advanced PH1 who do not yet require dialysis, and Cohort B enrolled 15 patients who are hemodialysis-dependent. The dosing regimen is based on weight with three monthly starting doses followed by ongoing monthly or quarterly doses. The primary efficacy endpoint for Cohort A was the percent change in plasma oxalate from baseline to month six, and the primary endpoint for Cohort B was the percent change in pre-dialysis plasma oxalate from baseline to month six. Key secondary endpoints are designed to evaluate additional measures of plasma oxalate and changes in urinary oxalate. Renal function, frequency and mode of dialysis, frequency of renal stone events, and measures of systemic oxalosis, including clinical manifestations, will also be evaluated in the extension period of the study. At six months, treatment with lumasiran resulted in a substantial reduction in plasma oxalate from baseline in both dialysis-independent and -dependent patients. Lumasiran also demonstrated positive results across key secondary endpoints, including measures of urinary oxalate (for patients in Cohort A) and additional measures of plasma oxalate. There were no deaths and no drug related SAEs among enrolled patients. There were two treatment discontinuations due to adverse events in the extension period of the study, neither of which was drug related. The most common drug related AEs (occurring in 10% or more of patients) were ISRs reported in five patients (23.8%), all of which were mild. Based on these results, the Company plans to submit a Supplemental New Drug Application (sNDA) for lumasiran with the U.S. Food and Drug Administration (FDA) and a Type II Variation with the European Medicines Agency (EMA) in late 2021. In November 2020, lumasiran was approved by the FDA for the treatment of PH1 to lower urinary oxalate levels in pediatric and adult patients and by the EMA for the treatment of PH1 in all age groups. Lumasiran is marketed in the U.S. and EU as OXLUMO®. ILLUMINATE-C topline results will be discussed during Alnylam’s Second Quarter Earnings Conference Call on August 3rd at 8:30 am ET, and full results are expected to be presented at a medical meeting later this year.

Neutral

Alnylam Pharmaceuticals, Inc. to Report Q2, 2021 Results on Aug 03, 2021

2021-07-21 12:00:00

Alnylam Pharmaceuticals, Inc. announced that they will report Q2, 2021 results Pre-Market on Aug 03, 2021

Neutral

Alnylam Pharmaceuticals, Inc., Q2 2021 Earnings Call, Aug 03, 2021

2021-07-21 12:00:00

Alnylam Pharmaceuticals, Inc., Q2 2021 Earnings Call, Aug 03, 2021

Positive

Alnylam Pharmaceuticals, Inc. Initiates KARDIA-1 Phase 2 Study of Zilebesiran (ALN-AGT) in Patients with Mild-to-Moderate Hypertension

2021-06-30 11:00:00

Alnylam Pharmaceuticals, Inc. announced initiation of KARDIA-1, a global Phase 2 study evaluating the efficacy and safety of zilebesiran (pronounced “zile-BEE-siran” and formerly known as ALN-AGT), an investigational subcutaneous RNAi therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension. KARDIA-1 will evaluate zilebesiran as monotherapy across different doses administered quarterly and biannually. The Company will host an “RNAi Roundtable” webinar today at 10:00 a.m. ET to discuss the zilebesiran program. The primary endpoint of KARDIA-1 is the change from baseline in systolic blood pressure as measured by 24-hour ambulatory blood pressure monitoring after three months of treatment. Additional endpoints will include change from baseline in blood pressure at six months, time-averaged reduction of blood pressure as a measure of tonic control, and safety. The study initiation is based on encouraging Phase 1 data, including results presented earlier this year at the 2021 Joint Meeting of the European Society of Hypertension (ESH) and the International Society of Hypertension (ISH). KARDIA-1 has been activated at clinical sites in the U.S. and will also be conducted at sites in Europe. The KARDIA-1 Phase 2 trial is a randomized, double-blind (DB), placebo-controlled, dose-ranging study to evaluate the efficacy and safety of zilebesiran as monotherapy in adults with mild-to-moderate hypertension. This global, multicenter trial will enroll approximately 375 adults with untreated hypertension or who are on stable therapy with one or more anti-hypertensive medications. Any patients taking prior anti-hypertensive medications will complete at least a four-week wash-out before randomization. Study participants will be randomized to one of five treatment arms during a 12-month DB period and DB extension period: 1) 150 mg zilebesiran subcutaneously once every six months; 2) 300 mg zilebesiran subcutaneously once every six months; 3) 300 mg zilebesiran subcutaneously once every three months; 4) 600 mg zilebesiran subcutaneously once every six months; or 5) placebo. Patients who receive placebo will be randomized to one of the four initial zilebesiran dose regimens beginning at month six. The study’s primary efficacy endpoint is the change from baseline in systolic blood pressure, assessed by 24-hour ambulatory blood pressure monitoring, after three months of treatment. In addition to the evaluation of the safety of zilebesiran, key secondary and exploratory endpoints in this study include additional measures of blood pressure reduction at six months, time-adjusted change in blood pressure, and change in daytime average and night-time average blood pressure.

Fundamental Summary

Looking at Alnylam Pharmaceuticals's financials of Q1 reflected decent results. Its growth and income factors appear positive and give support for optimism regarding the likelihood of continued positive performance. Therefore, Alnylam Pharmaceuticals received an overall score of 69, translating into a HOLD ranking.

Alnylam Pharmaceuticals reported earnings results for the first quarter ended March 31, 2022. For the first quarter, the company reported revenue was USD 213.26 million compared to USD 177.57 million a year ago. Net loss was USD 240.34 million compared to USD 200.29 million a year ago. Basic loss per share from continuing operations was USD 2 compared to USD 1.71 a year ago. Diluted loss per share from continuing operations was USD 2 compared to USD 1.71 a year ago.

Business Description

Alnylam Pharmaceuticals, a biopharmaceutical company, focuses on discovering, developing, and commercializing novel therapeutics based on ribonucleic acid interference. The company’s pipeline of investigational RNAi therapeutics focuses on genetic medicines, cardio-metabolic diseases, hepatic infectious diseases, and central nervous system (CNS)/ocular diseases. Its marketed products include ONPATTRO (patisiran), a lipid complex injection for the treatment of the polyneuropathy of hereditary transthyretin-mediated amyloidosis in adults; GIVLAARI for the treatment of adults with acute hepatic porphyria (AHP); and OXLUMO (lumasiran) for the treatment of primary hyperoxaluria type 1 (PH1). In addition, the company is developing givosiran for the treatment of adolescent patients with AHP; patisiran for the treatment of transthyretin amyloidosis, or ATTR amyloidosis, with cardiomyopathy; cemdisiran to treat complement-mediated diseases; ALN-AAT02 for the treatment of AAT deficiency-associated liver disease; ALN-HBV02 to treat chronic HBV infection; Zilebesiran to treat hypertension; and ALN-HSD to treat NASH. Further, it offers Fitusiran for the treatment of hemophilia and rare bleeding disorders, Inclisiran to treat hypercholesterolemia, lumasiran for the treatment of advanced PH1 and recurrent renal stones, and vutrisiran for the treatment of ATTR amyloidosis, which is in phase 3 clinical trial. Alnylam Pharmaceuticals has strategic collaborations with Regeneron Pharmaceuticals, Inc. to discover, develop, and commercialize RNAi therapeutics for a range of diseases by addressing therapeutic targets expressed in the eye and CNS; and Sanofi Genzyme to discover, develop, and commercialize RNAi therapeutics. It also has license and collaboration agreements with Novartis AG; Vir Biotechnology, Inc.; Dicerna Pharmaceuticals, Inc.; Ionis Pharmaceuticals, Inc.; and PeptiDream, Inc. The company was founded in 2002 and is headquartered in Cambridge, Massachusetts.

Sector Overview

Alnylam Pharmaceuticals is included in the Biotechnology according to GICS (global industry classification standard). The Biotechnology industry, which is part of the Health Care sector, includes companies primarily engaged in R&D, manufacturing, and/or marketing of products based on genetic analysis and genetic engineering. Alnylam Pharmaceuticals's industry and sector affiliation are expected to negatively affect their likelihood to overperform the market in the upcoming period, as other sectors appear more likely to benefit from the macroeconomic environment we see now.

Fundamental Highlights


Parameter Value Change Score
Assets 3,433.5 -5.8% 66
Liabilities 3,032.4 -0.7% 50
Price to Book 38.1 9.8% 61
Cash & Equivalents 534.1 -34.9% 44
Equity 401.2 -31.8% 46
EBITDA -486.2 9.1% 86
Total Revenues 880.0 4.2% 60
Parameter Value Change Score
Return on Equity -134.5 -26.6% 56
Net Cashflow 154.3 -52.3% 82
Capital Expenditure -77.1 -0.9% 70
Asset Turnover 0.3 9.9% 89
Free Cashflow -5.5 9.5% 80

* All values are TTM

The below chart reflects Alnylam Pharmaceuticals's birds-eye view on its performance with respect to its peers, the company's fillings as reported or to a similar industry, market cap, and country of origin. While Alnylam Pharmaceuticals's peer average final assessment score stands on 60.0, Alnylam Pharmaceuticals's score is 69.

  •  ALNY
  •  Peers average
Name Market Cap Balance Sheet Income Statement Cash Flow Rating Final Assessment mc_sort Hidden
Amgen Inc. 130.1B 69 78 81
Hold
77
77 0 1
Gilead Sciences, Inc. 78.0B 57 73 62
Underperform
60
60 1 1
Vertex Pharmaceuticals Incorporated 70.6B 78 75 71
Buy
79
79 2 1
Regeneron Pharmaceuticals, Inc. 64.0B 67 74 73
Hold
72
72 3 1
BioNTech SE 34.0B 75 79 72
Buy
79
79 4 1
Seagen Inc. 32.9B 61 62 58
Underperform
57
57 5 1
Biogen Inc. 29.7B 76 70 66
Hold
74
74 6 1
Incyte Corporation 16.8B 83 73 84
Buy
86
86 7 1
BioMarin Pharmaceutical Inc. 15.4B 70 82 63
Hold
74
74 8 1
United Therapeutics Corporation 10.6B 76 82 93
Strong Buy
88
88 9 1
Neurocrine Biosciences, Inc. 9.3B 68 55 63
Underperform
60
60 10 1
Exelixis, Inc. 6.7B 70 81 87
Buy
81
81 11 1
Sarepta Therapeutics, Inc. 6.5B 58 78 93
Hold
73
73 12 1
Halozyme Therapeutics, Inc. 6.3B 74 82 75
Buy
81
81 13 1
Ionis Pharmaceuticals, Inc. 5.4B 62 86 89
Buy
79
79 14 1
Ascendis Pharma A/S 5.2B 68 54 62
Underperform
60
60 15 1
Alkermes plc 5.0B 62 62 92
Hold
69
69 16 1
Cytokinetics, Incorporated 4.1B 64 66 69
Hold
65
65 17 1
Novavax, Inc. 4.0B 77 75 60
Hold
74
74 18 1
Mirati Therapeutics, Inc. 3.8B 50 64 65
Underperform
52
52 19 1
Natera, Inc. 3.6B 54 52 58
Underperform
47
47 20 1
Arrowhead Pharmaceuticals, Inc. 3.4B 71 76 70
Hold
74
74 21 1
PTC Therapeutics, Inc. 2.8B 51 73 72
Underperform
59
59 22 1
Insmed Incorporated 2.4B 55 57 82
Underperform
58
58 23 1
ACADIA Pharmaceuticals Inc. 2.3B 63 58 72
Hold
61
61 24 1
BioCryst Pharmaceuticals, Inc. 1.9B 55 66 67
Underperform
57
57 25 1
ChemoCentryx, Inc. 1.8B 61 73 87
Hold
71
71 26 1
Ironwood Pharmaceuticals, Inc. 1.8B 68 79 78
Hold
76
76 27 1
Myriad Genetics, Inc. 1.5B 52 96 59
Hold
66
66 28 1
Agios Pharmaceuticals, Inc. 1.2B 50 75 75
Hold
60
60 29 1
IVERIC bio, Inc. 1.1B 50 70 64
Underperform
54
54 30 1
FibroGen, Inc. 1.0B 73 67 84
Hold
76
76 31 1
Syndax Pharmaceuticals, Inc. 1.0B 58 62 57
Underperform
54
54 32 1
ImmunoGen, Inc. 946.1M 56 64 87
Hold
64
64 33 1
Enanta Pharmaceuticals, Inc. 922.8M 60 75 66
Hold
65
65 34 1
Deciphera Pharmaceuticals, Inc. 914.0M 61 77 94
Hold
75
75 35 1
Anavex Life Sciences Corp. 767.1M 61 75 72
Hold
67
67 36 1
Sangamo Therapeutics, Inc. 600.0M 48 76 91
Hold
65
65 37 1
AnaptysBio, Inc. 586.3M 52 68 80
Hold
60
60 38 1
Geron Corporation 566.2M 60 78 77
Hold
70
70 39 1
Mersana Therapeutics, Inc. 449.1M 58 47 65
Underperform
50
50 40 1
Inovio Pharmaceuticals, Inc. 416.9M 46 72 71
Underperform
54
54 41 1
Arbutus Biopharma Corporation 388.2M 60 67 95
Hold
71
71 42 1
Intercept Pharmaceuticals, Inc. 386.3M 57 86 96
Hold
78
78 43 1
Anika Therapeutics, Inc. 322.7M 64 77 63
Hold
73
73 44 1
Lexicon Pharmaceuticals, Inc. 321.6M 51 69 77
Hold
61
61 45 1
Precigen, Inc. 299.1M 73 79 74
Buy
79
79 46 1
bluebird bio, Inc. 288.7M 50 96 83
Hold
78
78 47 1
Rigel Pharmaceuticals, Inc. 187.5M 62 64 41
Underperform
52
52 48 1
MacroGenics, Inc. 184.6M 47 71 65
Underperform
53
53 49 1
Exact Sciences Corporation 7.2B 47 62 56
Underperform
46
46 50 1
Amicus Therapeutics, Inc. 3.0B 56 58 85
Hold
61
61 51 1
CureVac N.V. 2.6B 57 74 90
Hold
70
70 52 1
Fate Therapeutics, Inc. 2.4B 53 46 61
Underperform
45
45 53 1
Abgenix Inc. 2.1B 67 47 58
Underperform
45
45 54 1
Xenon Pharmaceuticals Inc. 1.9B 61 56 89
Hold
64
64 55 1
Iovance Biotherapeutics, Inc. 1.7B 63 71 77
Hold
68
68 56 1
Xencor, Inc. 1.6B 67 79 98
Buy
82
82 57 1
Emergent BioSolutions Inc. 1.6B 61 66 75
Hold
65
65 58 1
Dynavax Technologies Corporation 1.5B 63 67 68
Hold
64
64 59 1
Ligand Pharmaceuticals Incorporated 1.5B 56 69 84
Hold
65
65 60 1
Aurinia Pharmaceuticals Inc. 1.4B 52 70 84
Hold
62
62 61 1
Veracyte, Inc. 1.4B 57 55 82
Underperform
59
59 62 1
Sierra Oncology, Inc. 1.3B 80 61 52
Hold
68
68 63 1
Celldex Therapeutics, Inc. 1.3B 57 70 69
Hold
61
61 64 1
CareDx, Inc 1.2B 52 50 86
Underperform
54
54 65 1
Vericel Corporation 1.1B 57 56 65
Underperform
53
53 66 1
Avid Bioservices, Inc. 959.5M 67 75 66
Hold
69
69 67 1
MannKind Corporation 957.2M 57 61 66
Underperform
56
56 68 1
Merus N.V. 941.6M 70 54 71
Hold
64
64 69 1
Sorrento Therapeutics, Inc. 747.5M 74 55 52
Hold
61
61 70 1
Amarin Corporation plc 734.5M 55 63 55
Underperform
52
52 71 1
Catalyst Pharmaceuticals, Inc. 707.1M 75 77 87
Buy
84
84 72 1
Northwest Biotherapeutics, Inc. 682.9M 65 79 85
Hold
77
77 73 1
Organogenesis Holdings Inc. 612.1M 60 69 75
Hold
64
64 74 1
CTI BioPharma Corp. 603.6M 54 59 56
Underperform
50
50 75 1
Vanda Pharmaceuticals Inc. 598.8M 64 62 69
Hold
62
62 76 1
Eagle Pharmaceuticals, Inc. 546.4M 75 78 73
Buy
79
79 77 1
Immatics N.V. 538.7M 76 77 86
Buy
84
84 78 1
Agenus Inc. 520.5M 54 74 58
Underperform
57
57 79 1
Radius Health, Inc. 499.8M 54 82 57
Hold
61
61 80 1
Vaxart, Inc. 440.0M 53 68 65
Underperform
56
56 81 1
Affimed N.V. 415.0M 54 66 74
Underperform
59
59 82 1
ADMA Biologics, Inc. 396.6M 68 57 71
Hold
64
64 83 1
MiMedx Group, Inc. 391.5M 45 59 60
Underperform
45
45 84 1
Albireo Pharma, Inc. 362.9M 54 65 63
Underperform
55
55 85 1
AVEO Pharmaceuticals, Inc. 337.5M 66 74 95
Hold
78
78 86 1
Zymeworks Inc. 324.7M 62 56 57
Underperform
54
54 87 1
Heron Therapeutics, Inc. 314.6M 58 62 71
Underperform
59
59 88 1
Silence Therapeutics plc 313.4M 66 72 69
Hold
70
70 89 1
AC Immune SA 299.8M 68 70 68
Hold
68
68 90 1
Viridian Therapeutics, Inc. 293.2M 65 53 65
Underperform
56
56 91 1
Sutro Biopharma, Inc. 245.5M 61 56 74
Underperform
59
59 92 1
Chimerix, Inc. 178.4M 49 65 83
Underperform
58
58 93 1
DermTech, Inc. 165.3M 56 52 58
Underperform
47
47 94 1
Gelesis Holdings, Inc. 111.5M 78 39 80
Hold
66
66 95 1
Brooklyn ImmunoTherapeutics, Inc. 31.8M 49 61 58
Underperform
56
56 96 1
INSYS Therapeutics, Inc. 3.2M 43 70 73
Underperform
55
55 97 1

Technical Analysis

When trying to optimize the timing of an investment, it's critical to analyze whether the stock looks overbought or oversold, and in which direction the momentum is moving. Alnylam Pharmaceuticals's stock is now priced above its 50-day, but below its 5-day and 200-day moving average, while its MACD (moving average convergence divergence) indicates that the stock's price movement momentum is strengthening. Historically, this is a positive setup in the medium-term. Meanwhile, looking at the Stochastic Oscillator and RSI (relative strength index), Alnylam Pharmaceuticals's stock appears overbought. Overall, these technical indicators signal negative momentum. Therefore, this stock received a cumulative TA (technical analysis) score of 52.

Bearish 52
Close Price 142.2
52W Low 120.42
52W High 209.29
5D MA 144.81
50D MA 137.53
200D MA 162.24
MACD 2.73
RSI 70.62
STOCH 73.23

Balance Sheet Analysis

A few metrics from Alnylam Pharmaceuticals's current balance sheet were especially concerning: Cash & Equivalents and Equity. Alnylam Pharmaceuticals did a poor job related to managing cash and cash equivalents this period, which stood at 534.1, representing a -34.9% change from the previous filing. This performance is significantly less impressive than its peers and competitors. Discouraging results like these statistically lead to negative pressure in stock prices, so we rated their cash and cash equivalents with a score of 44. Also, Alnylam Pharmaceuticals published concerning equity metrics for this filing. In the current report, equity stood at 401.2, which represents a -31.8% change from the last report. This parameter often affects companies in the same industry and market capitalization by up to 7.3%. Their equity metrics appear unremarkable relative to their peers. Its equity movement component, therefore, received a grade of 46. That said, one metric, Assets, stood out as strongly positive. Alnylam Pharmaceuticals's management was effective in managing the value of the assets on their balance sheet, which now sits at 3433.5 and represents a -5.8% change from the previous report. This growth should support upward pressure on its's stock price, which better captures their intrinsic value. Therefore, its asset component earned a score of 66. Therefore, the company's balance sheet earned a grade of 54.

Parameter Value Change Score
Assets 3,433.5 -5.8% 66
Liabilities 3,032.4 -0.7% 50
Price to Book 38.1 9.8% 61
Cash & Equivalents 534.1 -34.9% 44
Equity 401.2 -31.8% 46
* All values are TTM

The below chart describes Alnylam Pharmaceuticals's performance as reflected on its balance sheet with respect to its peers. While Alnylam Pharmaceuticals received a balance sheet score of 54, the average of its peers stands on 60.0.

  •  ALNY
  •  Peers average
Name Market Cap Liabilities Movement Asset Change Equity/Intangibles Adjustments Cash & Equivalents Book Value Momentum Balance Sheet mc_sort Hidden
Amgen Inc. 130.1B 72 68 86 53 62 69 0 1
Gilead Sciences, Inc. 78.0B 48 63 61 52 62 57 1 1
Vertex Pharmaceuticals Incorporated 70.6B 69 69 58 84 92 78 2 1
Regeneron Pharmaceuticals, Inc. 64.0B 48 65 52 86 81 67 3 1
BioNTech SE 34.0B 75 69 64 97 73 75 4 1
Seagen Inc. 32.9B 59 68 55 43 66 61 5 1
Biogen Inc. 29.7B 57 72 92 50 76 76 6 1
Incyte Corporation 16.8B 71 72 95 82 79 83 7 1
BioMarin Pharmaceutical Inc. 15.4B 55 75 54 77 74 70 8 1
United Therapeutics Corporation 10.6B 61 74 58 60 88 76 9 1
Neurocrine Biosciences, Inc. 9.3B 67 69 55 51 78 68 10 1
Exelixis, Inc. 6.7B 53 66 57 84 81 70 11 1
Sarepta Therapeutics, Inc. 6.5B 68 69 49 41 58 58 12 1
Halozyme Therapeutics, Inc. 6.3B 59 68 65 72 85 74 13 1
Ionis Pharmaceuticals, Inc. 5.4B 59 68 58 43 67 62 14 1
Ascendis Pharma A/S 5.2B 51 70 85 94 54 68 15 1
Alkermes plc 5.0B 63 66 46 54 71 62 16 1
Cytokinetics, Incorporated 4.1B 49 79 42 76 62 64 17 1
Novavax, Inc. 4.0B 61 67 52 77 99 77 18 1
Mirati Therapeutics, Inc. 3.8B 53 66 57 48 42 50 19 1
Natera, Inc. 3.6B 73 65 43 95 42 54 20 1
Arrowhead Pharmaceuticals, Inc. 3.4B 72 71 63 65 73 71 21 1
PTC Therapeutics, Inc. 2.8B 57 61 80 49 37 51 22 1
Insmed Incorporated 2.4B 49 65 45 46 63 55 23 1
ACADIA Pharmaceuticals Inc. 2.3B 83 65 55 91 55 63 24 1
BioCryst Pharmaceuticals, Inc. 1.9B 46 67 46 50 62 55 25 1
ChemoCentryx, Inc. 1.8B 72 77 47 53 52 61 26 1
Ironwood Pharmaceuticals, Inc. 1.8B 49 68 87 67 63 68 27 1
Myriad Genetics, Inc. 1.5B 51 62 51 43 57 52 28 1
Agios Pharmaceuticals, Inc. 1.2B 50 66 52 38 48 50 29 1
IVERIC bio, Inc. 1.1B 50 67 46 48 48 50 30 1
FibroGen, Inc. 1.0B 79 87 49 82 56 73 31 1
Syndax Pharmaceuticals, Inc. 1.0B 85 68 41 55 55 58 32 1
ImmunoGen, Inc. 946.1M 52 67 64 61 48 56 33 1
Enanta Pharmaceuticals, Inc. 922.8M 51 86 45 38 49 60 34 1
Deciphera Pharmaceuticals, Inc. 914.0M 56 71 38 82 63 61 35 1
Anavex Life Sciences Corp. 767.1M 50 67 50 75 66 61 36 1
Sangamo Therapeutics, Inc. 600.0M 59 64 47 45 46 48 37 1
AnaptysBio, Inc. 586.3M 85 63 41 37 51 52 38 1
Geron Corporation 566.2M 69 72 37 59 62 60 39 1
Mersana Therapeutics, Inc. 449.1M 75 68 39 82 52 58 40 1
Inovio Pharmaceuticals, Inc. 416.9M 56 66 38 54 40 46 41 1
Arbutus Biopharma Corporation 388.2M 86 65 61 48 55 60 42 1
Intercept Pharmaceuticals, Inc. 386.3M 46 68 57 41 61 57 43 1
Anika Therapeutics, Inc. 322.7M 76 68 63 64 65 64 44 1
Lexicon Pharmaceuticals, Inc. 321.6M 48 68 51 43 51 51 45 1
Precigen, Inc. 299.1M 81 85 61 72 48 73 46 1
bluebird bio, Inc. 288.7M 48 67 42 38 57 50 47 1
Rigel Pharmaceuticals, Inc. 187.5M 53 67 51 90 63 62 48 1
MacroGenics, Inc. 184.6M 53 64 61 37 39 47 49 1
Exact Sciences Corporation 7.2B 57 58 45 42 52 47 50 1
Amicus Therapeutics, Inc. 3.0B 52 64 58 66 56 56 51 1
CureVac N.V. 2.6B 49 64 75 52 52 57 52 1
Fate Therapeutics, Inc. 2.4B 63 70 54 39 45 53 53 1
Abgenix Inc. 2.1B 81 57 86 92 45 67 54 1
Xenon Pharmaceuticals Inc. 1.9B 48 69 53 45 70 61 55 1
Iovance Biotherapeutics, Inc. 1.7B 52 74 72 87 45 63 56 1
Xencor, Inc. 1.6B 53 78 60 40 67 67 57 1
Emergent BioSolutions Inc. 1.6B 48 62 87 48 61 61 58 1
Dynavax Technologies Corporation 1.5B 52 69 54 38 76 63 59 1
Ligand Pharmaceuticals Incorporated 1.5B 75 63 61 49 52 56 60 1
Aurinia Pharmaceuticals Inc. 1.4B 51 67 59 41 47 52 61 1
Veracyte, Inc. 1.4B 64 68 54 66 51 57 62 1
Sierra Oncology, Inc. 1.3B 62 71 52 97 97 80 63 1
Celldex Therapeutics, Inc. 1.3B 48 69 61 44 57 57 64 1
CareDx, Inc 1.2B 54 67 52 39 51 52 65 1
Vericel Corporation 1.1B 51 65 56 53 61 57 66 1
Avid Bioservices, Inc. 959.5M 75 61 61 58 72 67 67 1
MannKind Corporation 957.2M 79 59 50 40 64 57 68 1
Merus N.V. 941.6M 59 68 50 83 81 70 69 1
Sorrento Therapeutics, Inc. 747.5M 89 71 50 97 73 74 70 1
Amarin Corporation plc 734.5M 51 65 58 72 51 55 71 1
Catalyst Pharmaceuticals, Inc. 707.1M 52 69 70 77 86 75 72 1
Northwest Biotherapeutics, Inc. 682.9M 65 63 48 38 86 65 73 1
Organogenesis Holdings Inc. 612.1M 59 66 56 66 60 60 74 1
CTI BioPharma Corp. 603.6M 84 70 37 92 37 54 75 1
Vanda Pharmaceuticals Inc. 598.8M 82 70 47 89 56 64 76 1
Eagle Pharmaceuticals, Inc. 546.4M 83 70 73 46 79 75 77 1
Immatics N.V. 538.7M 56 70 84 95 73 76 78 1
Agenus Inc. 520.5M 73 62 37 55 60 54 79 1
Radius Health, Inc. 499.8M 50 64 50 44 59 54 80 1
Vaxart, Inc. 440.0M 69 67 47 56 47 53 81 1
Affimed N.V. 415.0M 56 66 63 56 47 54 82 1
ADMA Biologics, Inc. 396.6M 74 68 49 77 72 68 83 1
MiMedx Group, Inc. 391.5M 53 66 42 57 37 45 84 1
Albireo Pharma, Inc. 362.9M 48 65 42 58 62 54 85 1
AVEO Pharmaceuticals, Inc. 337.5M 76 68 61 78 60 66 86 1
Zymeworks Inc. 324.7M 77 69 70 90 43 62 87 1
Heron Therapeutics, Inc. 314.6M 84 65 48 43 58 58 88 1
Silence Therapeutics plc 313.4M 58 71 69 63 59 66 89 1
AC Immune SA 299.8M 84 64 47 53 75 68 90 1
Viridian Therapeutics, Inc. 293.2M 56 72 44 37 76 65 91 1
Sutro Biopharma, Inc. 245.5M 82 69 56 66 49 61 92 1
Chimerix, Inc. 178.4M 48 63 46 95 40 49 93 1
DermTech, Inc. 165.3M 76 72 51 52 43 56 94 1
Gelesis Holdings, Inc. 111.5M 47 76 48 76 98 78 95 1
Brooklyn ImmunoTherapeutics, Inc. 31.8M 37 71 45 39 55 49 96 1
INSYS Therapeutics, Inc. 3.2M 48 70 44 90 37 43 97 1

Income Statement Analysis

Alnylam Pharmaceuticals's income statement showed strong financials, specifically EBITDA and Revenue Efficiency. Alnylam Pharmaceuticals reported impressive EBITDA this period. At filing, EBITDA was reported as -486.2, representing 9.1% change from the previous period. Companies in the same industry and market capitalization are typically affected by this parameter by up to 24.4%. Their EBITDA metrics are even more impressive relative to their peers and competitors, and its likely the stock price has room to grow to reflect its intrinsic value accurately. The company's EBITDA movement, therefore, received a grade of 86. Also, Alnylam Pharmaceuticals did an excellent job managing its revenue efficiency this past period. Alnylam Pharmaceuticals's revenue efficiency is 880.0 according to the metrics in the current filing, which represents a 4.2% change from the previous report. This characteristic can affect companies in the same industry and market capitalization by up to 13.1%. Their industry-leading revenue efficiency makes a strong case for upward pressure on its's stock price. Consequently, their revenue efficiency received a grade of 60. However, one discouraging result, Return Factors, stood out. In this filing, Alnylam Pharmaceuticals reported a return on equity (ROE) ratio of -134.5, which represents a growth of -26.6%. This metric might have a 3.6 percent impact on companies in the same industry and with the same market capitalization. The company's return factor metrics return on equity and return on assets are even more concerning when compared to their peers. Therefore, their return factors component earned a score of 56. Consequently, the companie's income statement earned a rank of 80.

Parameter Value Change Score
EBITDA -486.2 9.1% 86
Total Revenues 880.0 4.2% 60
Return on Equity -134.5 -26.6% 56
* All values are TTM

The below chart describes Alnylam Pharmaceuticals's performance as reflected on its income statement with respect to its peers. While Alnylam Pharmaceuticals received a income statement score of 80 , the average of its peers stands on 62.0.

  •  ALNY
  •  Peers average
Name Market Cap Revenue Momentum Earning Movement Return Factors Momentum Income Statement mc_sort Hidden
Amgen Inc. 130.1B 74 75 64 78 0 1
Gilead Sciences, Inc. 78.0B 78 71 52 73 1 1
Vertex Pharmaceuticals Incorporated 70.6B 57 80 62 75 2 1
Regeneron Pharmaceuticals, Inc. 64.0B 67 74 66 74 3 1
BioNTech SE 34.0B 41 89 75 79 4 1
Seagen Inc. 32.9B 54 65 70 62 5 1
Biogen Inc. 29.7B 90 57 73 70 6 1
Incyte Corporation 16.8B 59 77 60 73 7 1
BioMarin Pharmaceutical Inc. 15.4B 72 81 65 82 8 1
United Therapeutics Corporation 10.6B 57 91 54 82 9 1
Neurocrine Biosciences, Inc. 9.3B 53 54 77 55 10 1
Exelixis, Inc. 6.7B 54 91 54 81 11 1
Sarepta Therapeutics, Inc. 6.5B 49 89 55 78 12 1
Halozyme Therapeutics, Inc. 6.3B 53 84 85 82 13 1
Ionis Pharmaceuticals, Inc. 5.4B 62 94 53 86 14 1
Ascendis Pharma A/S 5.2B 38 68 46 54 15 1
Alkermes plc 5.0B 69 55 81 62 16 1
Cytokinetics, Incorporated 4.1B 95 50 73 66 17 1
Novavax, Inc. 4.0B 41 89 54 75 18 1
Mirati Therapeutics, Inc. 3.8B 76 55 78 64 19 1
Natera, Inc. 3.6B 52 51 78 52 20 1
Arrowhead Pharmaceuticals, Inc. 3.4B 38 92 53 76 21 1
PTC Therapeutics, Inc. 2.8B 54 78 66 73 22 1
Insmed Incorporated 2.4B 51 58 73 57 23 1
ACADIA Pharmaceuticals Inc. 2.3B 72 49 80 58 24 1
BioCryst Pharmaceuticals, Inc. 1.9B 41 74 72 66 25 1
ChemoCentryx, Inc. 1.8B 97 57 71 73 26 1
Ironwood Pharmaceuticals, Inc. 1.8B 70 79 61 79 27 1
Myriad Genetics, Inc. 1.5B 89 93 57 96 28 1
Agios Pharmaceuticals, Inc. 1.2B 99 64 54 75 29 1
IVERIC bio, Inc. 1.1B 84 58 79 70 30 1
FibroGen, Inc. 1.0B 48 74 63 67 31 1
Syndax Pharmaceuticals, Inc. 1.0B 87 47 81 62 32 1
ImmunoGen, Inc. 946.1M 40 73 71 64 33 1
Enanta Pharmaceuticals, Inc. 922.8M 90 63 72 75 34 1
Deciphera Pharmaceuticals, Inc. 914.0M 60 79 70 77 35 1
Anavex Life Sciences Corp. 767.1M 84 69 60 75 36 1
Sangamo Therapeutics, Inc. 600.0M 72 72 73 76 37 1
AnaptysBio, Inc. 586.3M 97 50 77 68 38 1
Geron Corporation 566.2M 89 67 73 78 39 1
Mersana Therapeutics, Inc. 449.1M 37 54 72 47 40 1
Inovio Pharmaceuticals, Inc. 416.9M 96 57 68 72 41 1
Arbutus Biopharma Corporation 388.2M 37 82 53 67 42 1
Intercept Pharmaceuticals, Inc. 386.3M 71 93 40 86 43 1
Anika Therapeutics, Inc. 322.7M 63 80 62 77 44 1
Lexicon Pharmaceuticals, Inc. 321.6M 98 48 82 69 45 1
Precigen, Inc. 299.1M 57 87 59 79 46 1
bluebird bio, Inc. 288.7M 99 91 61 96 47 1
Rigel Pharmaceuticals, Inc. 187.5M 99 44 82 64 48 1
MacroGenics, Inc. 184.6M 95 56 74 71 49 1
Exact Sciences Corporation 7.2B 57 67 54 62 50 1
Amicus Therapeutics, Inc. 3.0B 61 55 77 58 51 1
CureVac N.V. 2.6B 45 83 71 74 52 1
Fate Therapeutics, Inc. 2.4B 45 52 61 46 53 1
Abgenix Inc. 2.1B 47 47 47 47 54 1
Xenon Pharmaceuticals Inc. 1.9B 41 65 59 56 55 1
Iovance Biotherapeutics, Inc. 1.7B 84 60 75 71 56 1
Xencor, Inc. 1.6B 42 94 52 79 57 1
Emergent BioSolutions Inc. 1.6B 91 51 77 66 58 1
Dynavax Technologies Corporation 1.5B 51 69 77 67 59 1
Ligand Pharmaceuticals Incorporated 1.5B 92 53 78 69 60 1
Aurinia Pharmaceuticals Inc. 1.4B 38 81 68 70 61 1
Veracyte, Inc. 1.4B 45 63 57 55 62 1
Sierra Oncology, Inc. 1.3B 84 54 54 61 63 1
Celldex Therapeutics, Inc. 1.3B 96 53 78 70 64 1
CareDx, Inc 1.2B 61 44 82 50 65 1
Vericel Corporation 1.1B 76 44 82 56 66 1
Avid Bioservices, Inc. 959.5M 49 82 69 75 67 1
MannKind Corporation 957.2M 95 48 56 61 68 1
Merus N.V. 941.6M 48 60 59 54 69 1
Sorrento Therapeutics, Inc. 747.5M 50 60 60 55 70 1
Amarin Corporation plc 734.5M 95 44 82 63 71 1
Catalyst Pharmaceuticals, Inc. 707.1M 48 88 58 77 72 1
Northwest Biotherapeutics, Inc. 682.9M 43 90 65 79 73 1
Organogenesis Holdings Inc. 612.1M 89 55 79 69 74 1
CTI BioPharma Corp. 603.6M 84 50 62 59 75 1
Vanda Pharmaceuticals Inc. 598.8M 89 46 81 62 76 1
Eagle Pharmaceuticals, Inc. 546.4M 39 94 53 78 77 1
Immatics N.V. 538.7M 37 93 53 77 78 1
Agenus Inc. 520.5M 57 83 45 74 79 1
Radius Health, Inc. 499.8M 94 71 68 82 80 1
Vaxart, Inc. 440.0M 99 51 72 68 81 1
Affimed N.V. 415.0M 95 49 75 66 82 1
ADMA Biologics, Inc. 396.6M 43 65 62 57 83 1
MiMedx Group, Inc. 391.5M 87 43 83 59 84 1
Albireo Pharma, Inc. 362.9M 46 69 77 65 85 1
AVEO Pharmaceuticals, Inc. 337.5M 38 89 54 74 86 1
Zymeworks Inc. 324.7M 58 53 77 56 87 1
Heron Therapeutics, Inc. 314.6M 61 60 75 62 88 1
Silence Therapeutics plc 313.4M 83 64 69 72 89 1
AC Immune SA 299.8M 83 61 65 70 90 1
Viridian Therapeutics, Inc. 293.2M 55 50 63 53 91 1
Sutro Biopharma, Inc. 245.5M 60 47 80 56 92 1
Chimerix, Inc. 178.4M 99 50 62 65 93 1
DermTech, Inc. 165.3M 49 48 78 52 94 1
Gelesis Holdings, Inc. 111.5M 41 46 56 39 95 1
Brooklyn ImmunoTherapeutics, Inc. 31.8M 83 49 92 61 96 1
INSYS Therapeutics, Inc. 3.2M 97 48 63 70 97 1

Cash Flow Analysis

Overall, Alnylam Pharmaceuticals's critical cash flow metrics appear to signal strong support and a high likelihood of positive growth going forward. Alnylam Pharmaceuticals's published asset turnover numbers were encouraging and reflected management's balanced change strategy. Alnylam Pharmaceuticals recorded asset turnover of 0.3, which represents a 9.9% change from the previous report. This metric might have a 4.1 percent impact on companies in the same industry and with the same market capitalization. Their impressive asset turnover metrics, specifically in contrast to their industry peers' performance, should support a tailwind in the company's stock price. Therefore, its asset turnover movement earned a score of 89. Also, Alnylam Pharmaceuticals's strong net cash flow numbers, which were 154.3 in this filing, showed a -52.3% change from the last period. Their net cash flow metrics are especially remarkable relative to their peers. The company's net cash flow, therefore, received a grade of 82. That said, one metric, Capital Expenditure, stood out as particularly concerning. Alnylam Pharmaceuticals's management did not a remarkable job this period managing capital expenditures (CapEx). In terms of the raw numbers, CapEx was reported as -77.1, which represents a -0.9% change from the last period. This performance is all the more uninspiring relative to their peers and competitors. Its CapEx movement, therefore, received a grade of 70. Consequently, their cash flow earned a score of 85.

Parameter Value Change Score
Net Cashflow 154.3 -52.3% 82
Capital Expenditure -77.1 -0.9% 70
Asset Turnover 0.3 9.9% 89
Free Cashflow -5.5 9.5% 80
* All values are TTM

The below chart describes Alnylam Pharmaceuticals's performance as reflected on its cash flow with respect to its peers. While Alnylam Pharmaceuticals received a cash flow score of 85, the average of its peers stands on 68.0.

  •  ALNY
  •  Peers average
Name Market Cap Cashflow Momentum Free Cashflow Growth Capital Expenditure Growth Assets Factors Momentum Cash Flow mc_sort Hidden
Amgen Inc. 130.1B 80 74 67 74 81 0 1
Gilead Sciences, Inc. 78.0B 57 61 53 85 62 1 1
Vertex Pharmaceuticals Incorporated 70.6B 64 75 80 71 71 2 1
Regeneron Pharmaceuticals, Inc. 64.0B 69 86 64 66 73 3 1
BioNTech SE 34.0B 73 97 50 52 72 4 1
Seagen Inc. 32.9B 53 53 50 87 58 5 1
Biogen Inc. 29.7B 58 54 90 73 66 6 1
Incyte Corporation 16.8B 78 79 92 74 84 7 1
BioMarin Pharmaceutical Inc. 15.4B 59 48 66 77 63 8 1
United Therapeutics Corporation 10.6B 93 92 68 80 93 9 1
Neurocrine Biosciences, Inc. 9.3B 63 43 54 74 63 10 1
Exelixis, Inc. 6.7B 79 90 89 83 87 11 1
Sarepta Therapeutics, Inc. 6.5B 85 89 95 87 93 12 1
Halozyme Therapeutics, Inc. 6.3B 83 66 52 49 75 13 1
Ionis Pharmaceuticals, Inc. 5.4B 88 96 60 86 89 14 1
Ascendis Pharma A/S 5.2B 48 59 77 98 62 15 1
Alkermes plc 5.0B 89 95 76 80 92 16 1
Cytokinetics, Incorporated 4.1B 63 79 89 51 69 17 1
Novavax, Inc. 4.0B 62 38 63 54 60 18 1
Mirati Therapeutics, Inc. 3.8B 56 58 71 89 65 19 1
Natera, Inc. 3.6B 52 54 56 84 58 20 1
Arrowhead Pharmaceuticals, Inc. 3.4B 60 39 87 96 70 21 1
PTC Therapeutics, Inc. 2.8B 72 53 54 83 72 22 1
Insmed Incorporated 2.4B 79 80 61 89 82 23 1
ACADIA Pharmaceuticals Inc. 2.3B 61 57 98 80 72 24 1
BioCryst Pharmaceuticals, Inc. 1.9B 61 58 54 96 67 25 1
ChemoCentryx, Inc. 1.8B 84 92 97 50 87 26 1
Ironwood Pharmaceuticals, Inc. 1.8B 77 66 67 76 78 27 1
Myriad Genetics, Inc. 1.5B 50 37 82 83 59 28 1
Agios Pharmaceuticals, Inc. 1.2B 85 68 44 46 75 29 1
IVERIC bio, Inc. 1.1B 64 67 44 71 64 30 1
FibroGen, Inc. 1.0B 86 74 48 87 84 31 1
Syndax Pharmaceuticals, Inc. 1.0B 50 41 74 78 57 32 1
ImmunoGen, Inc. 946.1M 77 79 95 95 87 33 1
Enanta Pharmaceuticals, Inc. 922.8M 58 47 84 83 66 34 1
Deciphera Pharmaceuticals, Inc. 914.0M 87 79 95 91 94 35 1
Anavex Life Sciences Corp. 767.1M 69 63 74 71 72 36 1
Sangamo Therapeutics, Inc. 600.0M 85 78 93 83 91 37 1
AnaptysBio, Inc. 586.3M 80 77 92 41 80 38 1
Geron Corporation 566.2M 78 68 49 81 77 39 1
Mersana Therapeutics, Inc. 449.1M 59 69 53 92 65 40 1
Inovio Pharmaceuticals, Inc. 416.9M 67 67 92 44 71 41 1
Arbutus Biopharma Corporation 388.2M 92 93 80 87 95 42 1
Intercept Pharmaceuticals, Inc. 386.3M 87 95 98 89 96 43 1
Anika Therapeutics, Inc. 322.7M 69 67 70 51 63 44 1
Lexicon Pharmaceuticals, Inc. 321.6M 91 82 39 46 77 45 1
Precigen, Inc. 299.1M 82 75 37 85 74 46 1
bluebird bio, Inc. 288.7M 89 77 93 37 83 47 1
Rigel Pharmaceuticals, Inc. 187.5M 42 40 46 40 41 48 1
MacroGenics, Inc. 184.6M 70 53 54 46 65 49 1
Exact Sciences Corporation 7.2B 53 45 80 46 56 50 1
Amicus Therapeutics, Inc. 3.0B 81 80 71 87 85 51 1
CureVac N.V. 2.6B 87 80 90 70 90 52 1
Fate Therapeutics, Inc. 2.4B 64 52 55 48 61 53 1
Abgenix Inc. 2.1B 66 53 45 59 58 54 1
Xenon Pharmaceuticals Inc. 1.9B 82 77 87 92 89 55 1
Iovance Biotherapeutics, Inc. 1.7B 77 66 69 69 77 56 1
Xencor, Inc. 1.6B 96 95 70 95 98 57 1
Emergent BioSolutions Inc. 1.6B 70 53 87 75 75 58 1
Dynavax Technologies Corporation 1.5B 64 48 82 65 68 59 1
Ligand Pharmaceuticals Incorporated 1.5B 86 93 51 78 84 60 1
Aurinia Pharmaceuticals Inc. 1.4B 73 79 92 96 84 61 1
Veracyte, Inc. 1.4B 90 95 46 46 82 62 1
Sierra Oncology, Inc. 1.3B 47 77 38 71 52 63 1
Celldex Therapeutics, Inc. 1.3B 69 63 67 54 69 64 1
CareDx, Inc 1.2B 98 84 40 50 86 65 1
Vericel Corporation 1.1B 64 46 59 73 65 66 1
Avid Bioservices, Inc. 959.5M 80 38 40 50 66 67 1
MannKind Corporation 957.2M 77 56 43 37 66 68 1
Merus N.V. 941.6M 70 71 74 53 71 69 1
Sorrento Therapeutics, Inc. 747.5M 48 63 61 52 52 70 1
Amarin Corporation plc 734.5M 52 40 74 55 55 71 1
Catalyst Pharmaceuticals, Inc. 707.1M 77 81 98 87 87 72 1
Northwest Biotherapeutics, Inc. 682.9M 80 77 81 86 85 73 1
Organogenesis Holdings Inc. 612.1M 76 76 63 55 75 74 1
CTI BioPharma Corp. 603.6M 48 60 74 67 56 75 1
Vanda Pharmaceuticals Inc. 598.8M 66 60 87 51 69 76 1
Eagle Pharmaceuticals, Inc. 546.4M 63 40 97 92 73 77 1
Immatics N.V. 538.7M 82 95 56 99 86 78 1
Agenus Inc. 520.5M 57 43 52 77 58 79 1
Radius Health, Inc. 499.8M 64 40 37 56 57 80 1
Vaxart, Inc. 440.0M 72 56 53 38 65 81 1
Affimed N.V. 415.0M 72 57 95 43 74 82 1
ADMA Biologics, Inc. 396.6M 62 77 74 85 71 83 1
MiMedx Group, Inc. 391.5M 46 48 97 86 60 84 1
Albireo Pharma, Inc. 362.9M 59 68 41 95 63 85 1
AVEO Pharmaceuticals, Inc. 337.5M 94 87 74 83 95 86 1
Zymeworks Inc. 324.7M 52 65 45 83 57 87 1
Heron Therapeutics, Inc. 314.6M 66 74 61 84 71 88 1
Silence Therapeutics plc 313.4M 68 66 72 69 69 89 1
AC Immune SA 299.8M 66 77 79 63 68 90 1
Viridian Therapeutics, Inc. 293.2M 72 87 43 45 65 91 1
Sutro Biopharma, Inc. 245.5M 83 67 64 50 74 92 1
Chimerix, Inc. 178.4M 80 84 95 47 83 93 1
DermTech, Inc. 165.3M 60 51 45 86 58 94 1
Gelesis Holdings, Inc. 111.5M 64 90 92 98 80 95 1
Brooklyn ImmunoTherapeutics, Inc. 31.8M 58 72 43 71 58 96 1
INSYS Therapeutics, Inc. 3.2M 75 78 87 52 73 97 1

This report is intended for general guidance and information purposes only and under no circumstances is tailored to a specific factor or variable related to its reader or intended to be used or considered as financial or investment advice, a solicitation of any offer, a recommendation, or an offer to sell or buy any securities or other form of a financial asset. For the full disclaimer, click here.