Today, a brief rundown of news involving Ultragenyx and Bristol Myers Squibb, as well as updates from Roche, Longeveron and Rocket Pharmaceuticals that you may have missed.
TheFood and Drug Administrationhas approved the first gene therapyfor the rare progressive neurological condition Sanfilippo syndrome Type A. CalledFayuviandpriced at $3.95 million, the treatment is now the second gene therapy Ultragenyx Pharmaceuticalshas brought to market since August. Neither of those therapies are projected by analysts to become blockbuster products. But Ultragenyx obtained sellable drug review vouchers alongside both approvals, which should “significantly bolster” the company’s balance sheet, wroteWilliam BlairanalystSami Corwin. Ultragenyxwarned of coming cost cutsfollowing the failure of an Angelman syndrome drug with bigger sales potential. Company shares climbed by double digits following Fayuvi’s approval.— Ben Fidler
Bristol Myers Squibbhas stopped development of an experimental cancer drug licensed from startupOrum Therapeuticsthree years ago. Bristol Myerspaid Orum $100 million up frontin 2023 for rights to the therapy, which is known as ORM-6151, involves a combination of antibody-drug conjugate and protein degradation technologies, and was being tested in acute myeloid leukemia. Ina regulatory filing, though, Orum said that, after reviewing the data from a Phase 1 trial, it's shelving the drug along with obligations to make additional milestone payments. Orum had been eligible to receive as much as $80 million in conditional payouts in the deal.— Ben Fidler
Roche'sdouble-barreled blood cancer drugLunsumiohassucceeded in a late-stage trialin follicular lymphoma. Roche didn't provide specifics, but said that a regimen involving Lunsumio and a standard therapy meaningfully improved progression-free survival compared toRituxanand typical treatment in people who'd received at least one prior medication. Survival data were "immature" upon an interim data check, Roche said. Lunsumio is currently approved in the third-line setting, but the latest Phase 3 study could convert that clearance to a standard nod and expand use. Roche intends to submit the findings to regulatory authorities.— Ben Fidler
Longeveronwillbegin a strategic reviewafter its experimental cell therapy for heart disease failed a Phase 2 trial. After 12 weeks of follow-up, patients receiving that therapy,laromestrocel, didn’t have a statistically significant change in a measure of the heart’s ability to pump out blood. Longeveron plans to meet with the FDA to discuss potential steps forward, but in the meantime, the company will hire an investment bank to “review all options” and intends to cut costs.— Ben Fidler
Rocket Pharmaceuticalshas come to terms with U.S. regulatorson modifications to a pivotal study testing a treatment for the rare heart condition Danon disease. Rocket previously paused that trial following the death of a study participant and then resumedtesting with an altered protocolthat, so far, has proven safein three other enrollees. On Tuesday, Rocket said the FDA endorsed that safety update as well as its path to completing the trial. The company will recruit 9 additional patients and hopes to complete dosing in the middle of 2027. Rocket will provide an update on the program during a virtual investor webinar in early October.— Delilah Alvarado
