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 the presentation of data from the APOLLO-B Phase 3 study of patisiran at the 18th International Symposium on Amyloidosis on September 8, 2022. This investigational RNAi therapeutic targets transthyretin-mediated amyloidosis with cardiomyopathy. The company will also present findings from the Global Open-Label Extension study and a multicenter observational study on patients with hATTR amyloidosis. A conference call to discuss results will occur on September 8, 2022, at 8:00 a.m. ET.
Alnylam Pharmaceuticals announced that the APOLLO-B Phase 3 study of patisiran met its primary endpoint, showing a statistically significant improvement in the 6-Minute Walk Test compared to placebo at 12 months (p-value 0.0162). The study also achieved its first secondary endpoint, indicating improved quality of life via the Kansas City Cardiomyopathy Questionnaire (p-value 0.0397). The company plans to file a Supplemental New Drug Application in the U.S. in late 2022. Patisiran demonstrated a favorable safety profile, with fewer deaths in the treatment arm.
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Alnylam Pharmaceuticals reported Q2 2022 net product revenues of $214 million, a 33% increase year-over-year, driven by strong performance from ONPATTRO and GIVLAARI. The company received FDA approval for AMVUTTRA, marking its fifth RNAi therapeutic. Alnylam expects topline results from the APOLLO-B Phase 3 trial of patisiran within three weeks and reiterated its 2022 financial guidance of combined net product revenues between $870 million and $930 million.
Alnylam Pharmaceuticals has published research in Nature Communications indicating that individuals with mutations in the INHBE gene exhibit reduced abdominal fat, a favorable metabolic profile, and lower risks for cardiovascular disease and type 2 diabetes. The study, which analyzed data from over 360,000 people, suggests that targeting INHBE may provide new therapeutic options for cardiometabolic diseases. Alnylam aims to develop a candidate targeting INHBE using its IKARIA™ platform.
This breakthrough highlights the potential to address obesity and its associated health risks effectively.
Alnylam Pharmaceuticals (NASDAQ: ALNY) announced a positive opinion from the European Medicines Agency's CHMP for its RNAi therapeutic, vutrisiran, aimed at treating hATTR amyloidosis in adult patients. The European Commission's decision is expected in September 2022. Vutrisiran is notable for being the first treatment to show reversal in neuropathy impairment with quarterly subcutaneous administration. This therapy met all study endpoints in the HELIOS-A Phase 3 trial, demonstrating safety and efficacy. Vutrisiran is already approved in the U.S. and is under review in Brazil and Japan.
Alnylam Pharmaceuticals (NASDAQ: ALNY) announced it will report its Q2 financial results for the period ending June 30, 2022, on July 28, 2022, before U.S. markets open. A conference call will follow at 8:30 am ET to discuss the results and future expectations. Registered participants can access the call through a dedicated link. Alnylam, a leader in RNA interference therapeutics, continues to innovate with a pipeline of late-stage candidates aimed at addressing unmet medical needs.
Alnylam Pharmaceuticals announced FDA approval for AMVUTTRA™ (vutrisiran), the first RNAi therapeutic for hereditary transthyretin-mediated (hATTR) amyloidosis, administered subcutaneously every three months. The approval follows the HELIOS-A Phase 3 study, where over 50% of patients showed improvement or reversal in neuropathy symptoms. AMVUTTRA met all primary and secondary endpoints with significant enhancements in quality of life and gait speed. Launch is anticipated in early July, supported by value-based agreements.
Alnylam Pharmaceuticals (Nasdaq: ALNY) recently reported topline results from a Phase 2 study of cemdisiran, an investigational RNAi therapeutic for IgA Nephropathy (IgAN). The treatment led to a 37% mean reduction in the 24-hour urine protein to creatinine ratio, a key indicator for disease progression. The study demonstrated an acceptable safety profile, with no significant drug-related safety signals. Alnylam plans to advance cemdisiran into Phase 3 clinical development to address the unmet needs in IgAN.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present at major healthcare conferences, including the Jefferies Healthcare Conference on June 9, 2022, and the Goldman Sachs 43rd Annual Global Healthcare Conference on June 13, 2022. Live audio webcasts of the presentations will be accessible on the Investors section of their website, with replays available within 48 hours. Alnylam is a leader in RNA interference therapeutics, offering innovative treatments for rare diseases, with a deep pipeline and a commitment to delivering transformative medicines globally.