Alnylam Pharmaceuticals develops and commercializes RNA interference (RNAi) therapeutics, with news centered on its approved medicines, TTR franchise, clinical pipeline and financial performance. Company updates commonly address AMVUTTRA/vutrisiran and ONPATTRO/patisiran for transthyretin-mediated amyloidosis, GIVLAARI/givosiran for acute hepatic porphyria, OXLUMO/lumasiran for primary hyperoxaluria type 1, and partner-commercialized RNAi medicines.
Recurring developments include quarterly product revenue reports, guidance, clinical and real-world data for ATTR-CM and other cardiovascular programs, pipeline progress across cardio-metabolic disease, neuroscience and hematology, disease-awareness collaborations, and investor conference presentations. Alnylam also reports on its Alnylam 2030 strategy and commercialization of RNAi medicines in global markets.
Alnylam Pharmaceuticals has announced its 9th annual 'RNAi Roundtable' webinar series starting September 27, 2022. This series will feature discussions on pipeline programs, RNAi platform innovations, and clinical developments. Key sessions include:
- Cemdisiran for IgA Nephropathy - September 27, 2022
- Leadership in RNAi - October 21, 2022
- CNS Delivery for Alzheimer’s - November 1, 2022
A virtual R&D Day is scheduled for December 15, 2022, focusing on RNAi pipeline advancements. More details are available on their website.
Alnylam and Regeneron have released preliminary Phase 1 results for ALN-HSD, an RNAi therapeutic targeting HSD17B13, aimed at treating nonalcoholic steatohepatitis (NASH).
The study showed promising outcomes, with robust target knockdown and reduced liver enzymes over six months in 20 patients compared to 4 in the placebo group.
Following this success, a Phase 2 study is set to commence in late 2022. Safety data indicated mild injection site reactions, with no serious adverse events reported, further validating ALN-HSD's potential in addressing NASH's significant unmet medical needs.
Regeneron (REGN) and Alnylam (ALNY) announced positive preliminary data from a Phase 1 study of ALN-HSD, aimed at treating nonalcoholic steatohepatitis (NASH). Results show robust target knockdown and lower liver enzymes in patients receiving ALN-HSD compared to placebo, with a favorable safety profile. The companies plan to advance to a Phase 2 trial in late 2022. The study highlighted a significant unmet need in NASH, which affects 16 million people in the U.S. and is projected to be a leading cause of liver transplants.
Alnylam Pharmaceuticals (NASDAQ: ALNY) will present new data from the APOLLO-B Phase 3 study of patisiran at the HFSA Annual Scientific Meeting on September 30, 2022. This investigational RNAi therapeutic targets transthyretin-mediated amyloidosis with cardiomyopathy. The company previously announced positive results from the APOLLO-B study. The presentation will include primary and exploratory analyses. CEO Mat Maurer will discuss these findings during a webcast on the same date.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has announced the pricing of a $900 million private offering of 1.00% convertible senior notes due in 2027. The offering allows initial purchasers to buy an additional $135 million of the notes. The notes will accrue interest payable semi-annually and will be convertible under specified conditions. Proceeds are projected to be approximately $883.2 million, intended for capped call transactions and to repay existing borrowings. Closing is expected on September 15, 2022, pending customary conditions.
Alnylam Pharmaceuticals (Nasdaq: ALNY) has initiated a private offering of $900 million in convertible senior notes due 2027, with an option for initial purchasers to acquire an additional $135 million. The notes, which are senior and unsecured, will mature on September 15, 2027, and interest will be paid semi-annually. Alnylam plans to use around $762 million of the proceeds to repay existing borrowings and for general corporate purposes. The offering is under Rule 144A and is contingent upon market conditions.
Alnylam Pharmaceuticals announced positive results from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic for treating ATTR amyloidosis with cardiomyopathy. The study met its primary endpoint, showing a significant improvement in functional capacity (14.7 meters in 6-MWT) and health status (3.7 points in KCCQ-OS) compared to placebo after 12 months. Safety profiles were encouraging, with no major cardiac concerns. A supplemental new drug application is planned, indicating a potential market opportunity for patisiran if approved.
Alnylam Pharmaceuticals (NASDAQ: ALNY) will present a company overview at the Morgan Stanley 20th Annual Global Healthcare Conference on September 13, 2022, at 11:10 am ET. The event will take place at the Sheraton New York Hotel. Investors can access a live audio webcast on the company’s website, with a replay available within 48 hours post-event. Alnylam is known for pioneering RNA interference (RNAi) therapeutics, addressing rare diseases with innovative products like ONPATTRO and GIVLAARI.
Alnylam Pharmaceuticals announced positive results from a Phase 2 study of cemdisiran for treating immunoglobulin A nephropathy (IgAN). The study demonstrated a 37% reduction in the 24-hour urine protein to creatinine ratio compared to placebo. Notably, 32% of patients on cemdisiran achieved a significant reduction in protein levels, compared to 13% on placebo. The treatment was well tolerated, with common side effects being injection site reactions. Alnylam aims to advance cemdisiran to Phase 3 development, addressing the significant unmet needs in IgAN treatment.
Alnylam Pharmaceuticals has appointed Dr. Elliott Sigal to its Board of Directors, enhancing its leadership at a pivotal moment. With decades of experience, including his role as Chief Scientific Officer at Bristol-Myers Squibb, Dr. Sigal contributed to the launch of 14 new medicines across various therapeutic areas. His expertise is expected to strengthen Alnylam's ongoing efforts in advancing RNAi therapeutics aimed at rare diseases. The company continues its pursuit of transforming healthcare through innovative treatments, guided by its 'Alnylam P5x25' strategy.