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 (Nasdaq: ALNY) has published its 2021 Corporate Responsibility Report, its first full report, highlighting its GHG emissions and sustainability efforts.
Key points include the establishment of baseline GHG emissions, a strengthened corporate responsibility structure, a $1 million investment in health equity initiatives, and enhanced diversity, equity, and inclusion goals.
Alnylam Pharmaceuticals announced that the FDA has accepted its supplemental New Drug Application (sNDA) for lumasiran, an RNAi therapeutic targeting HAO1, aimed at reducing plasma oxalate in patients with advanced primary hyperoxaluria type 1 (PH1). The FDA has set a PDUFA action date for October 6, 2022. The six-month results from the ILLUMINATE-C study demonstrated significant reductions in plasma oxalate levels for patients, including those on hemodialysis. Alnylam also submitted a Type II Variation to the EMA for lumasiran's label amendment.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present company overviews at two upcoming conferences: the Cowen 42nd Annual Healthcare Conference on March 8, 2022, at 9:50 am ET, and the Barclays Global Healthcare Conference on March 15, 2022, at 8:00 am ET in Miami, Florida. Live audio webcasts will be accessible on the Investor section of Alnylam's website, with replays available within 48 hours post-event. Alnylam specializes in RNA interference therapeutics, aiming to transform treatment for rare and prevalent diseases.
Alnylam Pharmaceuticals has premiered a documentary titled Two of Me: Living with Porphyria on Rare Disease Day, highlighting the challenges faced by individuals living with acute hepatic porphyria (AHP).
The film aims to raise awareness about AHP, a rare genetic disorder that can take up to 15 years to diagnose, by encouraging viewers to tell 15 others about it.
The documentary focuses on the emotional and psychological impact of AHP, emphasizing the need for better understanding and prompt diagnosis of this debilitating condition.
Alnylam Pharmaceuticals reported impressive financial results for Q4 and the full year 2021, achieving global net product revenues of $199 million and $662 million, representing 83% annual growth compared to 2020. The company anticipates combined net product revenues of $900 million to $1 billion for 2022, indicating 44% growth at the midpoint. Positive results from the HELIOS-A Phase 3 study of vutrisiran demonstrate its potential in treating hATTR amyloidosis. Despite operating losses, Alnylam expects significant upcoming milestones in its product pipeline.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will present a company overview at the 11th Annual SVB Leerink Global Healthcare Conference on February 17, 2022, at 10:00 am ET. The event will be held virtually, and interested parties can access a live audio webcast through the Investors section of their website. Alnylam is renowned for pioneering RNAi therapeutics, with innovative products including ONPATTRO®, GIVLAARI®, OXLUMO®, and Leqvio®, and is committed to delivering transformative medicines for various diseases.
Alnylam Pharmaceuticals (Nasdaq: ALNY) will report its financial results for Q4 and year-end 2021 on February 10, 2022, before U.S. market opening. A conference call will be held at 8:30 am ET the same day to discuss results and future expectations. Notable RNAi therapeutics by Alnylam include ONPATTRO, GIVLAARI, OXLUMO, and Leqvio, with a robust pipeline including six late-stage candidates. The company aims to deliver innovative treatments for rare and prevalent diseases, ensuring strong financial performance.
Alnylam Pharmaceuticals announced that its investigational drug vutrisiran met all secondary endpoints of the HELIOS-A Phase 3 study at 18 months. The study demonstrated statistically significant improvements in neuropathy impairment, quality of life, gait speed, nutritional status, and overall disability compared to placebo. Additionally, vutrisiran showed potential for cardiac benefits, including reduced technetium uptake, suggesting possible amyloid regression. The drug is under regulatory review globally, with a potential FDA action date of April 14, 2022.
Ceptur Therapeutics, a biotechnology company focused on U1 Adaptor technology, has successfully completed a $75M Series A financing round. The round was co-led by venBio Partners and Qiming Venture Partners USA, with participation from various investors including Bristol Myers Squibb and Janus Henderson Investors. The funds will be used to advance their genetic medicine pipeline. Ceptur's U1 Adaptors aim to regulate gene expression at the pre-mRNA level, offering innovative therapeutic applications for genetic diseases.
Alnylam Pharmaceuticals has partnered with n-Lorem Foundation to support nano-rare disease patients through innovative therapies. The collaboration aims to utilize Alnylam's RNA interference technology alongside n-Lorem's experimental antisense oligonucleotide medicines. This partnership is designed to identify treatment opportunities for rare diseases affecting 1 to 30 patients globally, addressing significant healthcare challenges.