High-dose Spinraza meets study goal; Top Dyne executives exit
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Today, a brief rundown of news from Biogen, as well as updates from Dyne Therapeutics, Denali Therapeutics and Novo Nordisk that you may have missed.
A higher dose regimen of Biogen’s drug Spinraza met the goal of a Phase 2/3 study testing it in previously untreated infants with spinal muscular atrophy, showing improvements in motor function compared to a matched control arm from a prior study. Additionally, the higher dose appeared to reduce neurofilament, a biomarker associated with neurodegeneration, by more than Spinraza’s approved dose, said Stephanie Fradette, Biogen’s head of neuromuscular development, in a Wednesday statement. The company plans to share results with regulators. — Ned Pagliarulo
Shares in Dyne Therapeutics fell by nearly one-third Tuesday after the company simultaneously disclosed the departure of several senior executives alongside new trial data for its experimental Duchenne muscular dystrophy drug. Dyne’s chief medical officer, chief operating officer and chief business officer have all stepped down, about seven months after the exit of the company’s CEO. While Dyne described the new drug data as positive and supportive of further advancement, they also showed two study participants experienced serious, treatment-emergent adverse events. — Ned Pagliarulo
Both Denali Therapeutics and Regenxbio see clear paths ahead to submit their experimental treatments for Hunter syndrome, an uncommon genetic disease. In separate statements Tuesday, Denali announced and Regenxbio confirmed plans to apply to the Food and Drug Administration for accelerated approval. Denali made its plans after reaching an agreement with the agency to use spinal fluid levels of heparan sulfate as a proxy of its drug’s efficacy. Regenxbio, which also unveiled new data Tuesday, will also use heparan sulfate levels as a surrogate marker. Denali expects to file an application early next year, while Regenxbio will do so this quarter. — Ned Pagliarulo
In a letter posted Monday by the European Medicines Agency, Novo Nordisk said intermittent shortages of its GLP-1 drugs Ozempic and Victoza could continue into the fourth quarter this year. High demand, along with capacity constraints at some manufacturing sites, have led to “out-of-stock situations,” the company said. Specifically, supply of the 0.25 milligram and 0.5 milligram doses of Ozempic is low, so Novo recommended physicians in Europe defer starting new patients on the drug until September. Both drugs are cleared for diabetes, but Ozempic contains the same ingredient as Novo’s popular obesity drug Wegovy. — Delilah Alvarado
RNA medicines developer Orbital Therapeutics has appointed biotechnology veteran Ron Philip as its next CEO, the company said Tuesday. Philip recently served as the CEO of Spark Therapeutics, and led it through marketing of its gene therapy Luxturna. Philip will succeed CEO and co-founder Giuseppe Ciaramella, who will remain a scientific and strategic adviser to the company. Orbital raised one of of the biotech industry’s largest Series A rounds last year after emerging from stealth in 2022 and partnering with Beam Therapeutics, where Ciaramella is currently serving as president. — Delilah Alvarado