Vertex Pharmaceuticals has recorded its first commercial revenue from Casgevy, its CRISPR-based gene therapy for sickle cell disease, while Intra-Cellular Therapies announced positive topline results for Caplyta in preventing relapse in schizophrenia. Moderna is also undergoing leadership changes, and Syndax Pharmaceuticals has entered into a royalty agreement for Niktimvo.
Vertex Reports Initial Casgevy Sales
Vertex Pharmaceuticals reported $2 million in revenue from the first commercial sale of Casgevy, a CRISPR gene therapy for sickle cell disease. This revenue represents a patient who received an infusion between July and September. Approximately 40 patients have undergone cell collection, a preliminary step involving the removal of stem cells for gene editing. Executives anticipate growing demand and project billions in future sales for the treatment.
Caplyta Demonstrates Efficacy in Schizophrenia Relapse Prevention
Intra-Cellular Therapies announced positive results from a clinical trial of Caplyta (lumateperone) in patients with schizophrenia. The trial demonstrated a 63% reduction in the risk of relapse compared to placebo (p<0.0001). Demand for Caplyta, approved for schizophrenia in the U.S. nearly five years ago, has been increasing, with company executives projecting annual sales to reach $5 billion within the next decade.
Moderna Leadership Changes
Moderna CEO Stéphane Bancel is relinquishing leadership of the company’s sales operations to President Stephen Hoge. This change follows a company reorganization and revised sales expectations for its RSV vaccine, mResvia. Bancel previously held the role of chief commercial officer, which became vacant after Arpa Garay's departure. Hoge will also oversee medical and research affairs.
Syndax Sells Niktimvo Royalty Rights
Syndax Pharmaceuticals has sold royalty rights to Royalty Pharma for $350 million upfront for Niktimvo (axatilimab), its drug approved for chronic graft-versus-host disease. Royalty Pharma will receive 13.8% of Niktimvo’s net sales in the U.S. The drug, approved in August, is sold by both Syndax and Incyte through a collaboration established in 2021. Syndax plans to use the funds to support the drug's launch and achieve profitability.
