The 2023 Nobel Prize in Chemistry has been awarded to David Baker, Demis Hassabis, and John Jumper for their groundbreaking work in computational protein design and the development of AlphaFold, an artificial intelligence model capable of predicting protein structures. This achievement addresses the long-standing "protein folding" problem and holds promise for accelerating drug discovery.
Alnylam Pursues Expanded Approval for Vutrisiran
Alnylam Pharmaceuticals has submitted a supplemental New Drug Application (sNDA) to the FDA for vutrisiran (Amvuttra), seeking approval for the treatment of transthyretin amyloidosis with cardiac manifestations. Vutrisiran is already approved for nerve damage associated with the inherited form of the disease. This new application, benefiting from a priority review, positions vutrisiran to compete with Pfizer's tafamidis and a therapy under review from BridgeBio Pharma in a market with significant unmet needs.
Johnson & Johnson Discontinues TAR-200 Bladder Cancer Trial
Johnson & Johnson has halted the Sunrise-2 trial evaluating TAR-200, a drug delivery system releasing gemcitabine, in patients with muscle-invasive bladder cancer who are not candidates for surgical resection. The trial was stopped because TAR-200 in combination with chemoradiation did not demonstrate superior efficacy compared to chemoradiation alone. J&J is continuing to develop TAR-200 in other bladder cancer settings and anticipates submitting the data from a completed study for regulatory approval next year.
AstraZeneca Licenses Lp(a)-Lowering Drug from CPSC Pharmaceutical Group
AstraZeneca has acquired exclusive rights to YS2302018, a preclinical oral drug targeting lipoprotein(a) [Lp(a)], from CPSC Pharmaceutical Group for an upfront payment of $100 million. Lp(a) is a genetically determined risk factor for cardiovascular disease that is not adequately addressed by statins. This asset complements AstraZeneca's existing cardiovascular portfolio and positions them in a competitive landscape that includes Amgen's olpasiran, Eli Lilly's lepodisiran, and Ionis/Novartis' pelacarsen. The deal includes potential milestone payments of up to $1.9 billion based on development and commercial achievements.
