Rare diseases
In exchange for its investigational gene therapies, Regenxbio will receive $110 million upfront and up to $700 million in milestones. After hitting an all-time low of $6.95 at close of business yesterday, the stock surged on the news by nearly 20% before markets opened Tuesday.
Sarepta Therapeutics’ Duchenne muscular dystrophy therapy Elevidys handily beat analysts’ expectations in the fourth quarter of 2024, reflecting the biotech’s “world-class” execution, according to BMO Capital Markets analysts.
Benefiting from technological and conceptual groundwork and positive early data, gene therapies are advancing in the clinic for cardiovascular diseases including congestive heart failure, chronic refractory angina and cardiomyopathy.
Eli Lilly’s idiopathic pulmonary fibrosis deal with Mediar is centered around MTX-463, an anti-WISP1 antibody that early studies have found to be safe and capable of engaging its target.
From ADCs and radiopharmaceuticals to cell and gene therapies, eager young startups are betting on advances in biopharma’s most competitive therapeutic spaces—and attracting dollars from Big Pharma.
Among the FDA’s pending decisions for this quarter are Vertex’s non-opioid pain drug and Sanofi’s RNA interference therapy for hemophilia A and B.
The new combination, dubbed Alyftrek, is designed to improve on the Trikafta, a product that generated sales of $8.9 billion in 2023. It’s welcome good news for Vertex following last week’s subpar clinical results for its non-opioid analgesic.
Following a 90% drop in share price due to an FDA rejection and a warning letter over Applied Therapeutics’ conduct during a clinical trial, the biotech’s CEO has stepped aside.
Despite securing the industry’s first approval for familial chylomicronemia syndrome, BMO Capital Markets believes that Tryngolza’s regulatory triumph will not be a significant positive for Ionis. Instead, the firm is focusing on olezarsen’s readout in severe hypertriglyceridemia, a much larger market.
Novo’s latest investment comes just days after the U.S. FTC greenlit the highly contentious acquisition of Catalent, which analysts expect will help the pharma expand its production capacity.
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