CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the ILLUMINATE-A Phase 3 study of lumasiran, an investigational RNAi therapeutic targeting glycolate oxidase (GO) in development for the treatment of primary hyperoxaluria type 1 (PH1), met its primary efficacy endpoint and all tested secondary endpoints. Specifically, lumasiran met the primary efficacy endpoint of percent change from baseline, relative to placebo, in 24-hour urinary oxalate excretion averaged across months 3 to 6 (p less than 0.0001). The study also achieved statistically significant results for all six tested secondary endpoints (p less than or equal to 0.001). Lumasiran also demonstrated an encouraging safety and tolerability profile. Based on these results, the Company plans to submit a New Drug Application (NDA) and file a Marketing Authorisation Application (MAA) for lumasiran in early 2020.
We are very pleased to report positive topline Phase 3 results for lumasiran, our third wholly owned investigational RNAi therapeutic. Patients living with PH1 and their families are faced with the burden of recurrent and painful stone events and a progressive and unpredictable decline in kidney function that ultimately results in end-stage renal disease and the need for intensive dialysis as a bridge to dual liver/kidney transplantation. The results from ILLUMINATE-A demonstrate that lumasiran can significantly reduce the hepatic production of oxalate, which we believe can thereby address the underlying pathophysiology of PH1, said Akshay Vaishnaw, M.D., Ph.D., President of R&D at Alnylam. Further, we are encouraged by the safety and tolerability profile of lumasiran and believe this investigational medicine has the potential to have a meaningful clinical impact on patients living with PH1. We look forward to submitting regulatory filings in early 2020 and advancing this highly needed medicine one step closer to patients. Finally, we extend our deepest gratitude to the patients, caregivers, investigators, and study staff who participated in ILLUMINATE-A and contributed to what we believe is an important medical advance for the treatment of PH1.
The ILLUMINATE-A results represent a significant landmark for the PH1 patient community. These patients live with the angst of not knowing when that next kidney stone will come or for how long their kidneys will keep working, and they grapple with the possibility of needing new organs. We have lived with the hope that someday patients living with PH1 and their families would finally have a treatment with the potential to have a positive impact on their health and alleviate some of that angst, said Kim Hollander, Executive Director of the Oxalosis and Hyperoxaluria Foundation. Today we are hopeful that we are much closer to that day than we have ever been.
Lumasiran results in ILLUMINATE-A mark our third positive Phase 3 study readout in 2019, positioning Alnylam with the potential for four marketed products by the end of 2020, assuming positive regulatory reviews. We believe this achievement also provides further support of our relatively high product development success rate linked to selection of genetically validated targets and a modular and reproducible platform, said John Maraganore, Ph.D., Chief Executive Officer of Alnylam. With these results in hand, we believe that were on track to exceed our Alnylam 2020 guidance, building by the end of 2020 a global, multi-product, commercial-stage company with a robust portfolio of clinical-stage programs for future growth and an organic product engine for sustainable innovation and patient impact.
ILLUMINATE-A Topline Study ResultsILLUMINATE-A (NCT03681184), a randomized, double-blind, placebo-controlled trial, designed to enroll approximately 30 patients with PH1 ages six and above, at 16 study sites, in eight countries around the world, is the largest interventional study conducted specifically in PH1. Patients were randomized 2:1 to lumasiran or placebo, with lumasiran administered at 3 mg/kg monthly for three months followed by quarterly maintenance doses. The primary endpoint for the study was the percent change from baseline in 24-hour urinary oxalate excretion averaged across months 3 to 6 in patients treated with lumasiran as compared to placebo. At six months, lumasiran met the primary endpoint in patients with PH1 (p less than 0.0001) and achieved statistically significant results for all six hierarchically-tested secondary endpoints (p less than or equal to 0.001), including the proportion of lumasiran patients that achieved near-normalization or normalization of urinary oxalate levels, relative to placebo.
There were no serious or severe adverse events in the study, and results showed that lumasiran was generally well tolerated with an overall profile generally consistent with that observed in Phase 1/2 and open-label extension studies of lumasiran. Lumasiran has received U.S. and EU Orphan Drug Designations, Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), and a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). Full ILLUMINATE-A study results will be presented in an oral plenary session on Tuesday, March 31, 2020 at OxalEurope International Congress in Amsterdam, Netherlands.
The Company is also conducting ILLUMINATE-B a global Phase 3 study of lumasiran in PH1 patients less than six years of age, with results expected in mid-2020, and ILLUMINATE-C a global Phase 3 study of lumasiran in PH1 patients of all ages with advanced renal disease, with results expected in 2021.
Conference Call InformationAlnylam Management will discuss the ILLUMINATE-A results via conference call on Tuesday, December 17, 2019 at 8:00 am ET. A webcast presentation will also be available on the Investors page of the Companys website, http://www.alnylam.com. To access the call, please dial 800-239-9838 (domestic) or +1-323-794-2551 (international) five minutes prior to the start time and refer to conference ID 6976021. A replay of the call will be available beginning at 11:00 am ET on the day of the call. To access the replay, please dial 888-203-1112 (domestic) or +1-719-457-0820 (international) and refer to conference ID 6976021.
About LumasiranLumasiran 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, a Breakthrough Therapy Designation from the U.S. Food and Drug Administration (FDA), and a Priority Medicines (PRIME) designation from the European Medicines Agency (EMA). The safety and efficacy of lumasiran have not been evaluated by the FDA, EMA or any other health authority.
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 RNAiRNAi (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 todays 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 PharmaceuticalsAlnylam (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 discovery platform. Alnylams commercial RNAi therapeutic products are ONPATTRO (patisiran), approved in the U.S., EU, Canada, Japan, and Switzerland, and GIVLAARI (givosiran), approved in the U.S. Alnylam has a deep pipeline of investigational medicines, including five product candidates that are in late-stage development. Looking forward, Alnylam will continue to execute 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 employs over 1,200 people worldwide and is headquartered in Cambridge, MA. For more information about our people, science and pipeline, please visit http://www.alnylam.com and engage with us on Twitter at @Alnylam or on LinkedIn.
Alnylam Forward Looking StatementsVarious statements in this release concerning Alnylam's future expectations, plans and prospects, including, without limitation, Alnylam's views with respect to the implications of the positive topline results from the ILLUMINATE-A study and the potential for lumasiran to have a meaningful clinical impact on patients living with PH1, its plans and expected timing for filing applications for regulatory approval of lumasiran, its plans for reporting the full results from the ILLUMINATE-A study, expectations regarding the timing for reporting results from the ILLUMINATE-B and ILLUMINATE-C clinical studies, and expectations regarding the potential to exceed 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, Alnylam's ability to discover and develop novel drug candidates and delivery approaches, successfully demonstrate the efficacy and safety of its product candidates, including lumasiran, 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, including lumasiran, obtaining, maintaining and protecting intellectual property, Alnylam's ability to enforce its intellectual property rights against third parties and defend its patent portfolio against challenges from third parties, obtaining and maintaining regulatory approval, pricing and reimbursement for products, including lumasiran, progress in establishing a commercial and ex-United States infrastructure, successfully launching, marketing and selling its approved products globally, Alnylams 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, obtain additional funding to support its business activities, and establish and maintain strategic business alliances and new business initiatives, Alnylam's dependence on third parties for development, manufacture and distribution of products, 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.
Lumasiran has not been approved by the FDA, EMA, or any other regulatory authority and no conclusions can or should be drawn regarding the safety or effectiveness of this investigational therapeutic.
See the original post here:
Alnylam Reports Positive Topline Results from ILLUMINATE-A Phase 3 Study of Lumasiran for the Treatment of Primary Hyperoxaluria Type 1 - Business...
- Department of Genetic Medicine - January 6th, 2025
- Research Services | Johns Hopkins Institute of Genetic Medicine - January 6th, 2025
- Patient Care | Johns Hopkins Department of Genetic Medicine - January 6th, 2025
- Specialty Clinics | Johns Hopkins Institute of Genetic Medicine - January 6th, 2025
- Pediatric Genetic Medicine at Johns Hopkins Children's Center - January 6th, 2025
- Research Centers | Johns Hopkins Institute of Genetic Medicine - January 6th, 2025
- About Us - Johns Hopkins Medicine - January 6th, 2025
- Graduate Programs & Training | Johns Hopkins Medicine - January 6th, 2025
- Request an Appointment | Johns Hopkins Institute of Genetic Medicine - January 6th, 2025
- Clemson professor Trudy Mackay elected to the National Academy of Medicine - Clemson News - October 22nd, 2024
- Research sheds new light on the behavior of KRAS gene in pancreatic and colorectal cancer - News-Medical.Net - October 22nd, 2024
- Pushing the boundaries of rare disease diagnostics with the help of the first Undiagnosed Hackathon - Nature.com - October 22nd, 2024
- Tailored Genetic Medicine: AAV Gene Therapy and mRNA Vaccines Redefine Healthcare's Future - Intelligent Living - October 22nd, 2024
- The Genetic Link to Parkinson's Disease - Hopkins Medicine - August 27th, 2022
- Epic Bio makes gene therapies by editing the epigenome - Labiotech.eu - August 27th, 2022
- Ovid turns to gene therapy startup to restock drug pipeline - BioPharma Dive - August 27th, 2022
- Whole-exome analysis of 177 pediatric patients with undiagnosed diseases | Scientific Reports - Nature.com - August 27th, 2022
- First Gene Therapy for Adults with Severe Hemophilia A, BioMarin's ROCTAVIAN (valoctocogene roxaparvovec), Approved by European Commission (EC) -... - August 27th, 2022
- Arbor Biotechnologies Enters into Agreement with Acuitas Therapeutics for Lipid Nanoparticle Delivery System for Use in Rare Liver Diseases - BioSpace - August 27th, 2022
- ElevateBio Partners with the California Institute for Regenerative Medicine to Accelerate the Development of Regenerative Medicines - Business Wire - August 27th, 2022
- ElevateBio and the University of Pittsburgh Announce Creation of Pitt BioForge BioManufacturing Center at Hazelwood Green to Accelerate Cell and Gene... - August 27th, 2022
- Genetic variants cause different reactions to psychedelic therapy - The Well : The Well - The Well - August 27th, 2022
- Personalized Medicine for Prostate Cancer: What It Is and How It Works - Healthline - August 27th, 2022
- Four radical new fertility treatments just a few years away from clinics - The Guardian - August 27th, 2022
- Why are Rats Used in Medical Research? - MedicalResearch.com - August 27th, 2022
- The Columns Stepping Stones in STEM Washington and Lee University - The Columns - August 27th, 2022
- Study points to new approach to clearing toxic waste from brain Washington University School of Medicine in St. Louis - Washington University School... - August 27th, 2022
- ALS Gene Therapy SynCav1 Found to Extend Survival in Mouse Model |... - ALS News Today - August 27th, 2022
- A New Kind of Chemo | The UCSB Current - The UCSB Current - August 27th, 2022
- Unraveling the mystery of who gets lung cancer and why - Genetic Literacy Project - June 16th, 2022
- How diet and the microbiome affect colorectal cancer - EurekAlert - June 16th, 2022
- Akouos Presents Nonclinical Data Supporting the Planned Clinical Development of AK-OTOF and Strategies for Regulated Gene Expression in the Inner Ear... - May 20th, 2022
- Money on the Move: SwanBio, Remix, Locus, Mirvie and More - BioSpace - May 20th, 2022
- DiNAQOR Opens DiNAMIQS Subsidiary to Partner with Gene Therapy Companies Bringing New Treatments to Patients - PR Newswire - May 20th, 2022
- Brain tumor growth may be halted with breast cancer drug - Medical News Today - May 20th, 2022
- LogicBio Therapeutics to Present at HC Wainwright Global Investment Conference - PR Newswire - May 20th, 2022
- Genascence Announces Data From Phase 1 Clinical Trial on GNSC-001, Company's Lead Program in Osteoarthritis, Presented at American Society of Gene... - May 20th, 2022
- Encoded Therapeutics Presents Nonclinical Data Showing Genomic Medicine Platform Yields Selective Expression to Optimize Gene Therapy Performance at... - May 20th, 2022
- California, Other States to Cover Rapid WGS of Newborns Under Medicaid, but Questions of Access Loom - GenomeWeb - May 20th, 2022
- Researchers Identify Role of 'Sonic the Hedgehog' Gene in Bone Repair - BioSpace - May 20th, 2022
- Targeting the Uneven Burden of Kidney Disease on Black Americans - The New York Times - May 20th, 2022
- ASC Therapeutics, U Mass Medical School, and the Clinic for Special Children Announce Podium Presentation of Safety and Efficacy in Murine and Bovine... - May 20th, 2022
- UC Davis Looks to Expand Genetic Breast Cancer Risk Education, Outreach for Hispanic Women - Precision Oncology News - May 20th, 2022
- Fly Researchers Find Another Layer to the Code of Life - Duke Today - May 20th, 2022
- CANbridge-UMass Chan Medical School Gene Therapy Research Presented at the American Society of Gene and Cell Therapy (ASGCT) Annual Meeting - Business... - May 20th, 2022
- Omicron BA.4 and BA.5: What to know about the new variants - Medical News Today - May 20th, 2022
- Krystal Biotech to Present Additional Data on B-VEC from the GEM-3 Phase 3 Study at the Society for Investigative Dermatology Annual Meeting -... - May 20th, 2022
- FDA approves Lilly's Mounjaro (tirzepatide) injection, the first and only GIP and GLP-1 receptor agonist for the treatment of adults with type 2... - May 20th, 2022
- Elucidating the developmental origin of life-sustaining adrenal glands | Penn Today - Penn Today - May 20th, 2022
- 5 questions facing gene therapy in 2022 - BioPharma Dive - January 17th, 2022
- In a First, Man Receives a Heart From a Genetically Altered Pig - The New York Times - January 17th, 2022
- Antibodies, Easy Single-Cell, Genomics for All: Notes from the JP Morgan Healthcare Conference - Bio-IT World - January 17th, 2022
- Using genetics to conserve wildlife - Pursuit - January 17th, 2022
- Genetics of sudden unexplained death in children - National Institutes of Health - January 17th, 2022
- Amicus Therapeutics Reports Preliminary 2021 Revenue and Provides 2022 Strategic Outlook and Revenue Guidance - Yahoo Finance - January 17th, 2022
- Maze Therapeutics Announces $190 Million Financing to Support the Advancement of Nine Precision Medicine Programs and Compass Platform for Genetically... - January 17th, 2022
- How The mRNA Vaccines Were Made: Halting Progress and Happy Accidents - The New York Times - January 17th, 2022
- Press Registration Is Now Open for the 2022 ACMG Annual Clinical Genetics Meeting - PRNewswire - January 17th, 2022
- A Novel Mutation in the TRPM4 Gene | RRCC - Dove Medical Press - January 17th, 2022
- Biomarkers and Candidate Therapeutic Drugs in Heart Failure | IJGM - Dove Medical Press - January 17th, 2022
- Genetic counseling program helps patients take control of their health - Medical University of South Carolina - June 24th, 2021
- One-year-old baby in UAE receives imported genetic medicine to treat rare disease - Gulf News - June 24th, 2021
- Black and non-Hispanic White Women Found to Have No Differences in Genetic Risk for Breast Cancer - Cancer Network - June 24th, 2021
- What's in your genes | The Crusader Newspaper Group - The Chicago Cusader - June 24th, 2021
- Immusoft Announces Formation of Scientific Advisory Board - Business Wire - June 24th, 2021
- Arrowhead Presents Positive Interim Clinical Data on ARO-HSD Treatment in Patients with Suspected NASH at EASL International Liver Congress - Business... - June 24th, 2021
- Pacific Biosciences and Rady Children's Institute for Genomic Medicine Announce its First Research Collaboration for Whole - GlobeNewswire - June 24th, 2021
- Despite the challenges of COVID-19, Yale-PCCSM section members continued their work on scientific papers - Yale School of Medicine - June 24th, 2021
- Veritas Intercontinental: Genetics makes it possible to identify cardiovascular genetic risk and prevent cardiac accidents such as those that have... - June 24th, 2021
- New Research Uncovers How Cancers with Common Gene Mutation Develop Resistance to Targeted Drugs - Newswise - June 24th, 2021
- Celebrate the Third Annual Medical Genetics Awareness Week April 13-16, 2021 - PRNewswire - February 14th, 2021
- How will WNY fare in the race between vaccines and coronavirus variants? - Buffalo News - February 14th, 2021
- Myriad Genetics to Participate in Multiple Upcoming Health and Technology Conferences - GlobeNewswire - February 14th, 2021
- ASCO GU 2021: The Landscape of Genetic Alterations Using ctDNA-based Comprehensive Genomic Profiling in Pat... - UroToday - February 14th, 2021
- The Human Genome and the Making of a Skeptical Biologist - Scientific American - February 14th, 2021
- Breast Cancer Gene Mutations Found in 30% of All Women - Medscape - February 1st, 2021
- Mysterious untreatable fevers once devastated whole families. This doctor discovered what caused them - CNN - February 1st, 2021
- CCMB team identifies variants of genes that metabolise drugs - BusinessLine - February 1st, 2021
- NeuBase Therapeutics Announces Acquisition of Gene Modulating Technology from Vera Therapeutics - GlobeNewswire - February 1st, 2021
- Copy number variations linked to autism have diverse but overlapping effects - Spectrum - February 1st, 2021