Rare diseases
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.
Incyte is abandoning its ALK2 blocker zilurgisertib, which it was trialing for myelofibrosis-associated anemia, while iTeos will deprioritize the development of inupadenant after it failed to meet the biotech’s clinical bar in a Phase II study of metastatic non-small cell lung cancer.
Ocaliva recently failed to secure the FDA’s traditional approval for primary biliary cholangitis due to safety concerns.
The company had said in October said that it was looking to license out its ex vivo editor renizgamglogene autogedtemcel to focus its resources on its in vivo platform.
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.
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