Beacon secures $170M for eye gene therapy; J&J affirms Carvykti survival benefit

Beacon Therapeutics, a gene therapy developer, has secured a $170 million Series B funding round, as announced on Wednesday. This financial boost, led by Forbion with contributions from Syncona, TCGX Capital, and the University of Oxford, aims to propel the company's leading eye disease therapy further through clinical trials. Beacon, which emerged from the merger of three eye-focused biotechs about a year ago, is concentrating its efforts on a treatment for X-linked retinitis pigmentosa. Additionally, the company is working on a second drug targeting dry age-related macular degeneration.

Regeneron Pharmaceuticals and Sanofi received approval from the European Commission for their drug Dupixent as a new treatment for chronic obstructive pulmonary disease (COPD) in adults. This marks the first new treatment option for COPD in over a decade. Dupixent is designated for use as an add-on maintenance therapy for adults with uncontrolled COPD marked by elevated blood eosinophils. The approval could impact approximately 220,000 adults in the European Union. In parallel, U.S. regulators have extended their review period for Dupixent by three months, with a final decision expected by September 27.

Vertex Pharmaceuticals is awaiting a decision from the Food and Drug Administration (FDA) on whether to approve its latest combination therapy for cystic fibrosis by January 2. This new drug cocktail includes three compounds aimed at enhancing the function or quantity of the ion channel that is defective in cystic fibrosis patients. Early testing indicated that this new therapy is comparable to Vertex’s current top-seller, Trikafta, but offers the convenience of once-daily dosing.

Johnson & Johnson has announced promising results from a study of its CAR-T cell therapy, Carvykti, in treating multiple myeloma. The study, named Cartitude-4, showed that Carvykti significantly improved overall survival rates among patients who had previously received treatment for multiple myeloma. While the company has yet to release detailed results, this development is noteworthy given earlier FDA concerns about potential increased risks of early death associated with the therapy.

Codexis has transferred the rights to two gene therapies for Fabry and Pompe disease to Crosswalk Therapeutics. These AAV-based treatments were initially licensed to Takeda, which later halted its discovery and preclinical work in AAV gene therapy, returning the rights to Codexis. Crosswalk Therapeutics, led by former Takeda employees familiar with the Fabry and Pompe programs, will now oversee their development.

This series of updates highlights significant progress and strategic shifts within the biotech and pharmaceutical industries, showcasing continued advancements in gene therapy, treatments for chronic diseases, and innovative combination therapies.