ALNY Alnylam Pharmaceuticals Inc.

145.6
+0.59  (+0%)
Previous Close 145.01
Open 144.17
52 Week Low 73.32
52 Week High 167.33
Market Cap $16,885,200,405
Shares 115,969,783
Float 84,062,490
Enterprise Value $14,603,236,361
Volume 548,027
Av. Daily Volume 483,108
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Upcoming Catalysts

Drug Stage Catalyst Date
Lumasiran (ALN-GO1) ILLUMINATE-B
Primary Hyperoxaluria Type 1
Phase 3
Phase 3
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Lumasiran (ALN-GO1)
Primary Hyperoxaluria Type 1 (PH1)
PDUFA priority review
PDUFA priority review
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Inclisiran
Hyperlipidemia
PDUFA
PDUFA
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Vutrisiran - HELIOS-A
ATTR amyloidosis
Phase 3
Phase 3
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Fitusiran (ATLAS)
Hemophilia A/B
Phase 3
Phase 3
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ALN-CC5 (cemdisiran)
IgA nephropathy
Phase 2
Phase 2
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Lumasiran (ALN-GO1) ILLUMINATE-C
Impaired renal function
Phase 3
Phase 3
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Drug Pipeline

Drug Stage Notes
ALN-AAT02
alpha-1 anti-trypsin deficiency-associated liver disease
Phase 1/2
Phase 1/2
Phase 1/2 initial data released.
VIR-2218
Chronic hepatitis B virus (HBV)
Phase 2
Phase 2
Phase 2 preliminary antiviral activity and safety data presented at EASL August 28, 2020.
Patisiran APOLLO-B
Wild-type ATTR amyloidosis patients with cardiomyopathy
Phase 3
Phase 3
Phase 3 enrolment to be completed in 2021.
VIR-2703
COVID-19
Phase 1
Phase 1
Human trials to commence around the end of 2020.
ALN-TTRsc02 (vutrisiran) - HELIOS-B
ATTR amyloidosis with cardiomyopathy
Phase 3
Phase 3
Phase 3 trial has been initiated - noted November 22, 2019.
ALN-AGT
Hypertension
Phase 1
Phase 1
Phase 1 trial ongoing.
Givosiran
Acute hepatic porphyrias
Approved
Approved
FDA approval announced November 20, 2019.
Patisiran
Familial Amyloidotic Polyneuropathy (FAP) in Patients with ATTR
Approved
Approved
FDA Approval announced August 10, 2018.

Latest News

  1. – First-Ever Study to Have Evaluated the Safety and Efficacy of an Investigational RNAi Therapeutic in Infants and Children Under the Age of Six

    Lumasiran Demonstrated Clinically Significant Reduction in Urinary Oxalate Levels Relative to Baseline in Children as Young as Four Months Old

    Safety and Tolerability Profile Consistent with That Observed in ILLUMINATE-A Phase 3 Pivotal Study

    – Full Results Planned to be Presented at the American Society of Nephrology Annual Meeting in October 2020

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today positive topline results from the ILLUMINATE-B pediatric Phase 3 study of lumasiran, an investigational RNAi therapeutic targeting hydroxyacid

    – First-Ever Study to Have Evaluated the Safety and Efficacy of an Investigational RNAi Therapeutic in Infants and Children Under the Age of Six

    Lumasiran Demonstrated Clinically Significant Reduction in Urinary Oxalate Levels Relative to Baseline in Children as Young as Four Months Old

    Safety and Tolerability Profile Consistent with That Observed in ILLUMINATE-A Phase 3 Pivotal Study

    – Full Results Planned to be Presented at the American Society of Nephrology Annual Meeting in October 2020

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today positive topline results from the ILLUMINATE-B pediatric Phase 3 study of lumasiran, an investigational RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) – the gene encoding glycolate oxidase (GO) – in development for the treatment of primary hyperoxaluria type 1 (PH1). ILLUMINATE-B is now the seventh Phase 3 study of an RNAi therapeutic that has yielded positive results, and the first-ever study evaluating the safety and efficacy of this new class of medicines in children under the age of six, including infants.

    "We are pleased to report these positive topline results that we believe hold promise for many families impacted by PH1. The safety and efficacy of lumasiran are consistent with that reported for the ILLUMINATE-A study in patients six and older, demonstrating that lumasiran can significantly reduce the hepatic production of oxalate across all ages, which we believe can thereby address the underlying pathophysiology of PH1," said Pritesh J. Gandhi, PharmD., Vice President and General Manager, Lumasiran Program at Alnylam. "The current standard of care for young children and infants diagnosed with PH1 is burdensome, including the frequent need for gastrostomy tube placement to enable hyperhydration, and, for those who have progressed to advanced disease, the risks associated with performing dialysis and, ultimately, organ transplantation. Thus, we believe, a meaningful reduction in urinary oxalate levels has the potential to favorably impact disease progression and management in very young patients. We look forward to reporting complete data from the ILLUMINATE-B study at the ASN virtual congress later this fall."

    "The ILLUMINATE-B results signal hope for the many families with children whose lives are deeply impacted by PH1. This is especially encouraging given that children as young as a few months old could benefit from the therapeutic approach that lumasiran offers, curbing production of oxalate at its source," said Kim Hollander, Executive Director of the Oxalosis and Hyperoxaluria Foundation. "We are grateful to Alnylam for their continued commitment to the PH1 community and for designing and successfully conducting a study that addresses a particularly vulnerable group of patients – young children and babies."

    ILLUMINATE-B Topline Study Results

    ILLUMINATE-B (NCT03905694) is a single arm, open-label, multicenter Phase 3 trial that enrolled 18 patients with PH1 under the age of six (range: 3-72 months), with an estimated glomerular filtration rate (eGFR) of greater than 45 mL/min/1.73 m2 or normal serum creatinine if less than 12 months old, at nine study sites, in five countries around the world. Lumasiran was administered according to a weight-based dosing regimen. The primary efficacy endpoint of the study was the percent change from baseline to Month 6 in spot urinary oxalate:creatinine ratio averaged across Months 3 to 6. At six months, relative to baseline, lumasiran demonstrated a clinically meaningful reduction in spot urinary oxalate:creatinine ratio. Reduction of urinary oxalate relative to baseline was consistent across all three body weight categories (less than 10 kg; 10 kg to less than 20 kg, and 20 kg or higher). Lumasiran demonstrated positive results across secondary endpoints, including additional measures of urinary and plasma oxalate. There were no serious or severe adverse events related to study drug, and the overall safety and tolerability profile of lumasiran was consistent with that observed in the ILLUMINATE-A study. Full ILLUMINATE-B study results will be presented on October 22, 2020 at the ASN virtual congress.

    Lumasiran has received U.S. and EU Orphan Drug Designations, Breakthrough Therapy and Rare Pediatric Disease Designations from the U.S. Food and Drug Administration (FDA), and a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). Alnylam has filed a New Drug Application (NDA) for lumasiran with the U.S. FDA. The FDA has granted a Priority Review for the NDA and has set an action date of December 3, 2020 under the Prescription Drug User Fee Act (PDUFA). In addition, the Marketing Authorisation Application (MAA) for lumasiran has been submitted to and validated by the EMA and has received Accelerated Assessment designation.

    The Company is also conducting ILLUMINATE-C – a global single-arm Phase 3 study of lumasiran in PH1 patients of all ages with advanced renal disease, including patients on dialysis, with results expected in 2021.

    About ILLUMINATE-A Phase 3 Study

    ILLUMINATE-A (NCT03681184) is a six-month randomized, double-blind, placebo-controlled, global, multicenter Phase 3 study (with a 54-month extension period) to evaluate the efficacy and safety of lumasiran in 39 patients with a documented diagnosis of PH1. Patients were randomized 2:1 to receive three monthly doses of lumasiran or placebo followed by quarterly maintenance doses at 3 mg/kg. The primary endpoint was the percent change in 24-hour urinary oxalate excretion from baseline to the average of months 3 to 6 in the patients treated with lumasiran as compared to placebo. Treatment arms were stratified at randomization based upon mean 24-hour urinary oxalate during screening (≤ 1.7 or > 1.7 mmol/24hr/1.73m2). Key secondary and exploratory endpoints were designed to evaluate additional measures of urinary oxalate, plasma oxalate, estimated glomerular filtration rate (eGFR), nephrocalcinosis, renal stone events, safety and tolerability.

    About Lumasiran

    Lumasiran is an investigational, subcutaneously administered RNAi therapeutic targeting hydroxyacid oxidase 1 (HAO1) in development for the treatment of primary hyperoxaluria type 1 (PH1). HAO1 encodes glycolate oxidase (GO). Thus, by silencing HAO1 and depleting the GO enzyme, lumasiran inhibits production of oxalate – the metabolite that directly contributes to the pathophysiology of PH1. Lumasiran utilizes Alnylam's Enhanced Stabilization Chemistry (ESC)-GalNAc-conjugate technology, which enables subcutaneous dosing with increased potency and durability and a wide therapeutic index. Lumasiran has received both U.S. and EU Orphan Drug Designations, Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), and Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The safety and efficacy of lumasiran are under evaluation by the FDA and EMA.

    About Primary Hyperoxaluria Type 1 (PH1)

    PH1 is an ultra-rare disease in which excessive oxalate production 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 and nephrocalcinosis. Renal damage is caused by a combination of tubular toxicity from oxalate, calcium oxalate deposition in the kidneys, and urinary obstruction by calcium oxalate stones. Compromised kidney function exacerbates the disease as the excess oxalate can no longer be effectively excreted, resulting in subsequent accumulation and crystallization in bones, eyes, skin, and heart, leading to severe illness and death. Current treatment options are very limited and include frequent renal dialysis or combined organ transplantation of liver and kidney, a procedure with high morbidity that is limited due to organ availability. Although a small minority of patients respond to vitamin B6 therapy, there are no approved pharmaceutical therapies for PH1.

    About RNAi

    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 our 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 potently silencing messenger RNA (mRNA) – the genetic precursors – that encode for disease-causing or disease pathway 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.

    About Alnylam Pharmaceuticals

    Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam's commercial RNAi therapeutic products are ONPATTRO® (patisiran), approved in the U.S., EU, Canada, Japan, Brazil, and Switzerland, and GIVLAARI® (givosiran), approved in the U.S, EU, and Brazil. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views with respect to the safety and efficacy of lumasiran as demonstrated in the ILLUMINATE-B Phase 3 study in children under the age of six, including infants, the potential for lumasiran to have a favorable impact on PH1 disease manifestations and overall disease progression and management across all ages, its plans to present the full results of the ILLUMINATE-B study, Alnylam's expectations with respect to the review timelines for the lumasiran NDA and MAA by the FDA and EMA, respectively, Alnylam's plans, assuming favorable regulatory reviews, to bring lumasiran to patients with PH1 around the world, and expectations regarding the continued execution on its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by Alnylam products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to Alnylam's business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam's ability to execute business continuity plans to address disruptions caused by the COVID-19 or any future pandemic; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for its product candidates, including lumasiran, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all; actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing; delays, interruptions or failures in the manufacture and supply of its product candidates, including lumasiran, or its marketed products; obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, including lumasiran, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO and GIVLAARI; progress in continuing to establish a commercial and ex-United States infrastructure; successfully launching, marketing and selling its approved products globally, including ONPATTRO and GIVLAARI, and achieving net product revenues for ONPATTRO within its revised expected range during 2020; Alnylam's ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's ability to manage its growth and operating expenses within the ranges of guidance provided by Alnylam through the implementation of further discipline in operations to moderate spend and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam's ability to establish and maintain strategic business alliances and new business initiatives; Alnylam's dependence on third parties, including Regeneron, for development, manufacture and distribution of certain products, including eye and CNS products, Ironwood, for assistance with the education about and promotion of GIVLAARI, and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; the risk of government investigations; and unexpected expenditures; as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

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  2. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Chardan Virtual 4th Annual Genetic Medicines Conference on Monday, October 5, 2020 at 4:15 pm ET via webcast.

    A live audio webcast of the presentation will be available on the Investors section of the Company's website at www.alnylam.com/events. A replay will be available on the Alnylam website within 48 hours after the event.

    About Alnylam Pharmaceuticals

    Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, infectious…

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present a company overview at the Chardan Virtual 4th Annual Genetic Medicines Conference on Monday, October 5, 2020 at 4:15 pm ET via webcast.

    A live audio webcast of the presentation will be available on the Investors section of the Company's website at www.alnylam.com/events. A replay will be available on the Alnylam website within 48 hours after the event.

    About Alnylam Pharmaceuticals

    Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam's commercial RNAi therapeutic products are ONPATTRO® (patisiran), approved in the U.S., EU, Canada, Japan, Switzerland and Brazil, and GIVLAARI® (givosiran), approved in the U.S., EU, and Brazil. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

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  3. – Company Announces Potential for a Biannual Dosing Regimen Option for its Investigational RNAi Therapeutic Vutrisiran, Providing Support for Further Product Differentiation as a Potential Best-in-Class Agent –

    – New Clinical Data Presented at the European Society of Cardiology 2020 Congress Provide Further Evidence that Treatment with Patisiran may Lead to Substantial Reduction in Cardiac Amyloid Burden in ATTR Amyloidosis –

    – Company Remains on Track to Report Topline Results from HELIOS-A in Early 2021 and to Complete Enrollment in APOLLO-B in 2021; HELIOS-B Enrollment Expanding Globally –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, will host an "RNAi Roundtable" webinar today at 12:00 p.m. ET…

    – Company Announces Potential for a Biannual Dosing Regimen Option for its Investigational RNAi Therapeutic Vutrisiran, Providing Support for Further Product Differentiation as a Potential Best-in-Class Agent –

    – New Clinical Data Presented at the European Society of Cardiology 2020 Congress Provide Further Evidence that Treatment with Patisiran may Lead to Substantial Reduction in Cardiac Amyloid Burden in ATTR Amyloidosis –

    – Company Remains on Track to Report Topline Results from HELIOS-A in Early 2021 and to Complete Enrollment in APOLLO-B in 2021; HELIOS-B Enrollment Expanding Globally –

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, will host an "RNAi Roundtable" webinar today at 12:00 p.m. ET, which will focus on progress across its ATTR amyloidosis programs. The Company will discuss the potential for a biannual subcutaneous dosing regimen for vutrisiran, as well as discuss ongoing Phase 3 development for patisiran and vutrisiran across the APOLLO-B, HELIOS-A, and HELIOS-B studies. The Company also announced data from a clinical study evaluating the impact of patisiran and diflunisal, a TTR stabilizer, on cardiac amyloid burden. These data, presented at the European Society of Cardiology (ESC) 2020 Congress, suggest that treatment with patisiran may lead to cardiac amyloid regression in patients with cardiomyopathy associated with ATTR amyloidosis.

    "We believe we have the potential to build an industry-leading franchise of medicines for the treatment of ATTR amyloidosis, and we look forward to highlighting our progress in our RNAi Roundtable later today. Amongst other achievements, we will discuss the opportunity for a biannual dosing regimen option for vutrisiran providing further differentiation to achieve a potential best-in-class profile. In addition, we will update on ongoing progress for our TTR programs in our APOLLO-B, HELIOS-A, and HELIOS-B Phase 3 studies of patisiran and vutrisiran," said Eric Green, Senior Vice President and General Manager of the TTR Program. "Moreover, we will summarize new recently presented data supporting the potential for patisiran to achieve a substantial reduction in cardiac amyloid burden in ATTR amyloidosis, with associated functional and biomarker improvements. We remain committed to developing additional therapeutic options for the treatment of ATTR amyloidosis and driving innovation that can potentially help improve patient outcomes and treatment experience."

    Vutrisiran Clinical Development Program

    Vutrisiran, an investigational RNAi therapeutic in development for the treatment of ATTR amyloidosis, is currently being evaluated in the HELIOS Phase 3 program. HELIOS-A is a randomized, open-label, global multi-center study evaluating the efficacy and safety of vutrisiran in patients with hATTR amyloidosis with polyneuropathy; this study is fully enrolled. The Company reiterates that topline results from HELIOS-A are expected to be announced in early 2021. In this study, patients receive 25mg of vutrisiran subcutaneously once every 12 weeks.

    Alnylam announces today that it has obtained clinical pharmacology data supporting the potential for a biannual subcutaneous dosing regimen for vutrisiran. Specifically, new analyses show that a biannual 50mg dosing regimen is expected to achieve comparable TTR knockdown to results with a 25mg quarterly dosing regimen and to a once every three weekly intravenous dosing regimen employed with patisiran. A biannual dosing regimen option for vutrisiran is expected to support further product differentiation for a potential best-in-class profile, and the Company plans to work with regulatory authorities in late 2020 to determine the most efficient regulatory path forward.

    The Company affirmed that enrollment continues in the HELIOS-B Phase 3 study, a randomized, double-blind, placebo-controlled, global multi-center study evaluating the efficacy and safety of vutrisiran in patients with ATTR amyloidosis with cardiomyopathy. In this study, patients receive 25mg of vutrisiran subcutaneously or placebo once every 12 weeks, and the primary endpoint is mortality and CV hospitalization at 30 months with a planned interim analysis. Alnylam has implemented steps to accelerate enrollment in HELIOS-B to make up for slowed patient accrual during the COVID-19 pandemic.

    In April 2020, Alnylam announced that the U.S. Food and Drug Administration (FDA) granted Fast Track designation to vutrisiran for the treatment of the polyneuropathy of hereditary ATTR (hATTR) amyloidosis.

    Patisiran Clinical Development Program

    Patisiran is being investigated for the treatment of ATTR amyloidosis with cardiomyopathy in the APOLLO-B study. APOLLO-B is a Phase 3, randomized, double-blind, placebo-controlled, global multi-center study designed to evaluate the efficacy and safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy. Enrollment in APOLLO-B continues, and the study enrollment is expected to be completed in 2021.

    Of interest, a study of 32 patients with hATTR cardiac amyloidosis – presented at ESC 2020 held virtually August 29 - September 1, 2020 – provides encouraging evidence of the potential for substantial reduction in cardiac amyloid burden in patients treated with patisiran. The non-randomized study, led by the University College Hospital in the UK, was conducted to evaluate the impact of patisiran and diflunisal, a TTR stabilizer, on cardiac amyloid load as measured by cardiac magnetic resonance (CMR) and T1 mapping, in patients with hATTR amyloidosis. Patients were assessed with echocardiogram, CMR, NT-proBNP, and 6-minute walk test (6MWT) at baseline and at one year. At the one-year time point, there was a substantial reduction in cardiac amyloid burden in 45% of patients who received patisiran (N=16). Patients treated with patisiran also showed a reduction in extracellular volume fraction (ECV) compared to an increase in ECV in the control group (n=16). These findings show the potential for CMR to be used to track response in treated patients with ATTR cardiac amyloidosis. Patients treated with patisiran also showed an improvement in change in 6MWT at one year, compared to patients in the control group. According to the authors, combination therapy with an RNAi therapeutic against transthyretin and a TTR stabilizing agent may be synergistic given enhanced stoichiometry of TTR stabilizers in the face of markedly reduced plasma transthyretin concentration.

    Today's RNAi Roundtable webinar can be accessed at www.alnylam.com/events or on the Capella section of the Alnylam website.

    About ONPATTRO® (patisiran)

    ONPATTRO is an RNAi therapeutic that was approved in the United States and Canada for the treatment of the polyneuropathy of hATTR amyloidosis in adults. ONPATTRO is also approved in the European Union, Switzerland and Brazil for the treatment of hATTR amyloidosis in adults with Stage 1 or Stage 2 polyneuropathy, and in Japan for the treatment of hATTR amyloidosis with polyneuropathy. ONPATTRO is an intravenously administered RNAi therapeutic targeting transthyretin (TTR). It is designed to target and silence TTR messenger RNA, thereby blocking the production of TTR protein before it is made. ONPATTRO blocks the production of TTR in the liver, reducing its accumulation in the body's tissues in order to halt or slow down the progression of the polyneuropathy associated with the disease. For more information about ONPATTRO, visit ONPATTRO.com.

    ONPATTRO (patisiran) lipid complex injection Important Safety Information

    Infusion-Related Reactions

    Infusion-related reactions (IRRs) have been observed in patients treated with ONPATTRO. In a controlled clinical study, 19 percent of ONPATTRO-treated patients experienced IRRs, compared to 9 percent of placebo-treated patients. The most common symptoms of IRRs with ONPATTRO were flushing, back pain, nausea, abdominal pain, dyspnea, and headache.

    To reduce the risk of IRRs, patients should receive premedication with a corticosteroid, acetaminophen, and antihistamines (H1 and H2 blockers) at least 60 minutes prior to ONPATTRO infusion. Monitor patients during the infusion for signs and symptoms of IRRs. If an IRR occurs, consider slowing or interrupting the infusion and instituting medical management as clinically indicated. If the infusion is interrupted, consider resuming at a slower infusion rate only if symptoms have resolved. In the case of a serious or life-threatening IRR, the infusion should be discontinued and not resumed.

    Reduced Serum Vitamin A Levels and Recommended Supplementation

    ONPATTRO treatment leads to a decrease in serum vitamin A levels. Supplementation at the recommended daily allowance (RDA) of vitamin A is advised for patients taking ONPATTRO. Higher doses than the RDA should not be given to try to achieve normal serum vitamin A levels during treatment with ONPATTRO, as serum levels do not reflect the total vitamin A in the body.

    Patients should be referred to an ophthalmologist if they develop ocular symptoms suggestive of vitamin A deficiency (e.g. night blindness).

    Adverse Reactions

    The most common adverse reactions that occurred in patients treated with ONPATTRO were upper respiratory-tract infections (29 percent) and infusion-related reactions (19 percent).

    For additional information about ONPATTRO, please see the full Prescribing Information.

    About hATTR Amyloidosis

    Hereditary transthyretin (TTR)-mediated amyloidosis (hATTR) is an inherited, progressively debilitating, and often fatal disease caused by mutations in the TTR gene. TTR protein is primarily produced in the liver and is normally a carrier of vitamin A. Mutations in the TTR gene cause abnormal amyloid proteins to accumulate and damage body organs and tissue, such as the peripheral nerves and heart, resulting in intractable peripheral sensory-motor neuropathy, autonomic neuropathy, and/or cardiomyopathy, as well as other disease manifestations. hATTR amyloidosis, represents a major unmet medical need with significant morbidity and mortality affecting approximately 50,000 people worldwide. The median survival is 4.7 years following diagnosis, with a reduced survival (3.4 years) for patients presenting with cardiomyopathy.

    About RNAi

    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 our 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 potently 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.

    About Alnylam Pharmaceuticals

    Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam's commercial RNAi therapeutic products are ONPATTRO® (patisiran), approved in the U.S., EU, Canada, Japan, Switzerland and Brazil, and GIVLAARI® (givosiran), approved in the U.S., EU, and Brazil. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

    Alnylam Forward Looking Statements

    Various statements in this release, including, without limitation, Alnylam's views and plans with respect to the potential for RNAi therapeutics, including patisiran and vutrisiran, as monotherapies or in combination with a TTR stabilizing agent, Alnylam's prospects for building an industry-leading ATTR amyloidosis franchise, expected timing for completing the ongoing APOLLO B Phase 3 study and completing enrollment and availability of results in the ongoing HELIOS Phase 3 program, and expectations regarding the achievement of its "Alnylam 2020" guidance for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or a future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by Alnylam products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to Alnylam's business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam's ability to execute business continuity plans to address disruptions caused by the COVID-19 or a future pandemic; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates, including patisiran and vutrisiran; the pre-clinical and clinical results for its product candidates, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all; actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing; delays, interruptions or failures in the manufacture and supply of its product candidates or its marketed products, including ONPATTRO, GIVLAARI, inclisiran, lumasiran and vutrisiran; obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, including lumasiran and inclisiran, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO and GIVLAARI; progress in continuing to establish a commercial and ex-United States infrastructure; successfully launching, marketing and selling its approved products globally, including ONPATTRO and GIVLAARI and achieving net product revenues for ONPATTRO within its revised expected range during 2020; Alnylam's ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's ability to manage its growth and operating expenses within the reduced ranges of guidance provided by Alnylam through the implementation of further discipline in operations to moderate spend and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam's ability to establish and maintain strategic business alliances and new business initiatives ; Alnylam's dependence on third parties, including Regeneron, for development, manufacture and distribution of certain products, including eye and CNS products, Ironwood, for assistance with the education about and promotion of GIVLAARI, and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; the risk of government investigations; and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

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  4. Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following conferences being held virtually:

    • Citi 15th Annual BioPharma Virtual Conference on Thursday, September 10, 2020 at 10:45 am ET
    • Morgan Stanley 18th Annual Global Healthcare Conference on Tuesday, September 15, 2020 at 9:30 am ET
    • Cantor Virtual Global Healthcare Conference on Wednesday, September 16, 2020 at 3:20 pm ET
    • BofA Global Healthcare Conference on Thursday, September 17, 2020 at 2:10 pm BST (9:10 am ET)

    A live audio webcast of each presentation will be available on the Investors section of the Company's website at www.alnylam.com/events. A replay will be available…

    Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, announced today that management will present company overviews at the following conferences being held virtually:

    • Citi 15th Annual BioPharma Virtual Conference on Thursday, September 10, 2020 at 10:45 am ET
    • Morgan Stanley 18th Annual Global Healthcare Conference on Tuesday, September 15, 2020 at 9:30 am ET
    • Cantor Virtual Global Healthcare Conference on Wednesday, September 16, 2020 at 3:20 pm ET
    • BofA Global Healthcare Conference on Thursday, September 17, 2020 at 2:10 pm BST (9:10 am ET)

    A live audio webcast of each presentation will be available on the Investors section of the Company's website at www.alnylam.com/events. A replay will be available on the Alnylam website within 48 hours after each event.

    About Alnylam Pharmaceuticals

    Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, hepatic infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam's commercial RNAi therapeutic products are ONPATTRO ® (patisiran), approved in the U.S., EU, Canada, Japan, Switzerland and Brazil, and GIVLAARI ® (givosiran), approved in the U.S. and EU. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

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  5. Blackstone (NYSE:BX) and Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced the closing of the R&D funding component of the companies' previously announced $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics.

    Under the terms of the agreement, Alnylam will receive up to $150 million from Blackstone Life Sciences for the development of Alnylam's cardiometabolic disease programs vutrisiran and ALN-AGT. The investment includes up to $70 million to support the ongoing HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy, and up to $80 million to support Phase 2 and Phase 3 development of ALN-AGT, in development for the…

    Blackstone (NYSE:BX) and Alnylam Pharmaceuticals, Inc. (NASDAQ:ALNY), the leading RNAi therapeutics company, today announced the closing of the R&D funding component of the companies' previously announced $2 billion strategic financing collaboration to accelerate the advancement of RNAi therapeutics.

    Under the terms of the agreement, Alnylam will receive up to $150 million from Blackstone Life Sciences for the development of Alnylam's cardiometabolic disease programs vutrisiran and ALN-AGT. The investment includes up to $70 million to support the ongoing HELIOS-B Phase 3 study of vutrisiran in ATTR amyloidosis patients with cardiomyopathy, and up to $80 million to support Phase 2 and Phase 3 development of ALN-AGT, in development for the treatment of hypertension.

    In April 2020, Blackstone and Alnylam entered into a broad $2 billion strategic financing collaboration anchored by Blackstone's purchase of 50 percent of royalties owed to Alnylam on global sales of inclisiran, an investigational RNAi therapeutic for the treatment of hypercholesterolemia, currently under review by the U.S. Food and Drug Administration and other regulatory authorities. The strategic financing collaboration is expected to enable Alnylam's achievement of a self-sustainable financial profile without need for future equity financing, accelerating the commercial potential of Alnylam's rapidly advancing product portfolio.

    About RNAi

    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 our 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 potently 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.

    About Alnylam Pharmaceuticals

    Alnylam (NASDAQ:ALNY) is leading the translation of RNA interference (RNAi) into a whole new class of innovative medicines with the potential to transform the lives of people afflicted with rare genetic, cardio-metabolic, infectious, and central nervous system (CNS)/ocular diseases. Based on Nobel Prize-winning science, RNAi therapeutics represent a powerful, clinically validated approach for the treatment of a wide range of severe and debilitating diseases. Founded in 2002, Alnylam is delivering on a bold vision to turn scientific possibility into reality, with a robust RNAi therapeutics platform. Alnylam's commercial RNAi therapeutic products are ONPATTRO® (patisiran), approved in the U.S., EU, Canada, Japan, Switzerland and Brazil, and GIVLAARI® (givosiran), approved in the U.S., EU and Brazil. Alnylam has a deep pipeline of investigational medicines, including six product candidates that are in late-stage development. Alnylam is executing on its "Alnylam 2020" strategy of building a multi-product, commercial-stage biopharmaceutical company with a sustainable pipeline of RNAi-based medicines to address the needs of patients who have limited or inadequate treatment options. Alnylam is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.

    About Blackstone Life Sciences

    Blackstone Life Sciences is a private, global investment platform with capabilities to invest across the life-cycle of companies and products within the key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines and medical products that improve patients' lives.

    Alnylam Forward Looking Statements

    Various statements in this release concerning Alnylam's expectations, plans and prospects, including, without limitation, expectations regarding the direct or indirect effects on Alnylam's business, activities and prospects as a result of the COVID-19 pandemic, or delays or interruptions resulting therefrom and the success of Alnylam's mitigation efforts, Alnylam's views and plans with respect to the potential for RNAi therapeutics, including vutrisiran and ALN-AGT, expectations regarding its agreement with Blackstone for funding of certain R&D activities for vutrisiran and ALN-AGT, Alnylam's belief that the funding provided by Blackstone should enable Alnylam to achieve a self-sustainable profile without the need for future equity financing, and expectations regarding the achievement of its "Alnylam 2020" strategic plan announced in 2015 for the advancement and commercialization of RNAi therapeutics, constitute forward-looking statements for the purposes of the safe harbor provisions under The Private Securities Litigation Reform Act of 1995. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation: the direct or indirect impact of the COVID-19 global pandemic or any future pandemic, such as the scope and duration of the outbreak, government actions and restrictive measures implemented in response, material delays in diagnoses of rare diseases, initiation or continuation of treatment for diseases addressed by Alnylam products, or in patient enrollment in clinical trials, potential supply chain disruptions, and other potential impacts to Alnylam's business, the effectiveness or timeliness of steps taken by Alnylam to mitigate the impact of the pandemic, and Alnylam's ability to execute business continuity plans to address disruptions caused by the COVID-19 or any future pandemic; Alnylam's ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates, including vutrisiran and ALN-AGT; the pre-clinical and clinical results for its product candidates, including vutrisiran and ALN-AGT, which may not be replicated or continue to occur in other subjects or in additional studies or otherwise support further development of product candidates for a specified indication or at all; actions or advice of regulatory agencies, which may affect the design, initiation, timing, continuation and/or progress of clinical trials or result in the need for additional pre-clinical and/or clinical testing; delays, interruptions or failures in the manufacture and supply of its product candidates or its marketed products, including ONPATTRO, GIVLAARI, inclisiran, lumasiran and vutrisiran; obtaining, maintaining and protecting intellectual property; intellectual property matters including potential patent litigation relating to its platform, products or product candidates; obtaining regulatory approval for its product candidates, including lumasiran and inclisiran, and maintaining regulatory approval and obtaining pricing and reimbursement for its products, including ONPATTRO and GIVLAARI; progress in continuing to establish a commercial and ex-United States infrastructure; successfully launching, marketing and selling its approved products globally, including ONPATTRO and GIVLAARI and achieving net product revenues for ONPATTRO within its further revised expected range during 2020; Alnylam's ability to successfully expand the indication for ONPATTRO in the future; competition from others using technology similar to Alnylam's and others developing products for similar uses; Alnylam's ability to manage its growth and operating expenses within the reduced ranges of guidance provided by Alnylam through the implementation of further discipline in operations to moderate spend and its ability to achieve a self-sustainable financial profile in the future without the need for future equity financing; Alnylam's ability to establish and maintain strategic business alliances and new business initiatives; Alnylam's dependence on third parties, including Regeneron, for development, manufacture and distribution of certain products, including eye and CNS products and ALN-APP, Ironwood, for assistance with the education about and promotion of GIVLAARI, and Vir for the development of ALN-COV and other potential RNAi therapeutics targeting SARS-CoV-2 and host factors for SARS-CoV-2; the outcome of litigation; the risk of government investigations; and unexpected expenditures, as well as those risks more fully discussed in the "Risk Factors" filed with Alnylam's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission (SEC) and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam's views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.

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