Beacon raises $170M for eye gene therapy; J&J confirms Carvykti survival benefit
Today, a brief rundown of news from Beacon Therapeutics and Regeneron Pharmaceuticals, as well as updates from Vertex Pharmaceuticals, Johnson & Johnson and Codexis that you may have missed.
Gene therapy developer Beacon Therapeutics has reloaded with a $170 million Series B round, which the company said Wednesday will help advance its lead eye disease therapy further in clinical trials. The round, led by Forbion with participation from Syncona, TCGX Capital and the University of Oxford, comes about a year after the company’s launch. Beacon was built from three eye-focused biotechs and its lead program, for a condition called X-linked retinitis pigmentosa, comes from one of those. It has a second drug in development to treat dry age-related macular degeneration. — Gwendolyn Wu
The European Commission on Wednesday approved Regeneron Pharmaceuticals and Sanofi’s Dupixent as the first new treatment option in more than a decade for adults with chronic obstructive pulmonary disease, or COPD. The drug is cleared for use as add-on maintenance treatment for adults with uncontrolled COPD characterized by elevated blood eosinophils. The companies estimate 220,000 adults in the EU will be eligible for treatment. In the U.S., regulators extended their review of Dupixent for this indication by three months and will decide on approval by Sept. 27. — Delilah Alvarado
By January 2, the Food and Drug Administration will decide whether to approve Vertex Pharmaceuticals’ latest combination therapy for cystic fibrosis, a drug cocktail that contains three compounds designed to increase the quantity or function of the ion channel that’s damaged in people with the lung disease. Testing showed it matched the company’s current top-seller Trikafta, but with once-daily dosing. — Ned Pagliarulo
Johnson & Johnson on Tuesday said treatment with its CAR-T cell therapy Carvykti led to “a statistically significant and clinically meaningful improvement” in overall survival among people with previously treated multiple myeloma. The study, dubbed Cartitude-4, compared Carvykti to standard therapies. The results, which J&J did not detail further, are important as the Food and Drug Administration previously shared concerns about signs of an increased risk of early death in testing. — Ned Pagliarulo
Two gene therapies for Fabry and Pompe disease are now in the hands of Crosswalk Therapeutics, which acquired them from Codexis. The AAV-based treatments were previously licensed to Takeda, but returned to Codexis when the Japanese pharmaceutical company discontinued its discovery and preclinical work in AAV gene therapy. Crosswalk is led by former Takeda employees who are familiar with the Fabry and Pompe programs, Codexis said. — Ned Pagliarulo
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