Alnylam Pharmaceuticals

Vir Biotechnology to present Phase 2 SOLSTICE trial data on tobevibart and elebsiran for chronic hepatitis delta at AASLD The Liver Meeting®, highlighting efficacy, safety, and economic burden insights.

Pfizer's Hympavzi, a once-weekly injection for hemophilia A or B, received FDA approval. The drug, administered via auto-injector, aims to reduce bleeding episodes and treatment burden. Sales are projected to reach $300 million by 2030.

Pfizer's Hympavzi, a once-a-week injection, received FDA approval for treating haemophilia A or B in patients aged 12 and older. The drug aims to reduce treatment burden and showed a 92% reduction in bleeding episodes in a late-stage study. Hympavzi is Pfizer's second haemophilia treatment approved this year, with potential sales of $300 million by 2030.

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BofA Securities maintains Buy rating on Alnylam Pharmaceuticals with $307 price target, confident in Amvuttra's commercial potential for ATTR-CM. Alnylam plans to position Amvuttra as first-line treatment, leveraging HELIOS-B trial data and convenient dosing. Submitted sNDA using Priority Review Voucher, aiming for faster FDA approval. Q2 earnings exceeded expectations, updating 2024 revenue guidance to $1.575-$1.65 billion. Analysts revise earnings upwards, reflecting optimism. Alnylam's gross profit margin is 87%, indicating strong financial health.

David Baker, Demis Hassabis, and John Jumper won the 2023 Nobel Prize in chemistry for computational protein design and AlphaFold development. Alnylam Pharmaceuticals seeks FDA approval for vutrisiran in another form of transthyretin amyloidosis. Johnson & Johnson terminated TAR-200 trial in bladder cancer. Gemma Biotherapeutics partners with Brazil's Fiocruz for rare disease gene therapies. AstraZeneca acquires a preclinical cholesterol-lowering drug from CPSC Pharmaceutical Group. Astellas invests in AviadoBio's experimental gene therapy for frontotemporal dementia.

Alnylam Pharmaceuticals submits sNDA to FDA for vutrisiran, an RNAi therapeutic for ATTR amyloidosis with cardiomyopathy, based on positive HELIOS-B Phase 3 study results. The submission includes use of a Priority Review Voucher for accelerated review. Alnylam anticipates vutrisiran's potential as a first-line therapy for ATTR-CM, pending regulatory approval.

City Therapeutics, led by John Maraganore, aims to advance RNAi-based medicine through next-generation siRNA engineering, targeting novel RNAi triggers and enhanced delivery. The company secured $135 million in Series A funding and plans to initiate clinical development by 2025, aiming for one to two new INDs per year starting in 2026.

John Maraganore, former CEO of Alnylam Pharmaceuticals, launches City Therapeutics, a startup focusing on RNAi-based treatments using tiny RNA molecules called cityRNAs, aiming to target tissues beyond the liver. The company plans to begin human trials for its first drug candidate by early 2026 with $135 million in series A financing.

John Maraganore, former CEO of Alnylam Pharmaceuticals, co-founded City Therapeutics to develop next-gen RNA interference medicines. City aims to innovate RNAi triggers and delivery methods, targeting new diseases beyond the liver. Funded with $135 million, City plans to start clinical testing by end of 2024.