Rare diseases
Capricor Therapeutics’ CEO Linda Marbán believes the FDA is willing to work with the biotech as the Aug. 22 deadline approaches for its Duchenne muscular dystrophy cell therapy deramiocel, with the biotech’s stock climbing 68% on the update.
Sponsors of the pivotal study behind Amgen’s rare disease drug Tavneos saw unblinded findings for the drug and re-adjudicated data from nine patients to make the drug seem effective, according to European regulators.
The terms of the agreement with Royalty Pharma suggest the market potential of Zealand Pharma’s polycythemia vera drug candidate may be undervalued, according to Jefferies analysts.
PTC Therapeutics emerged as the winning bidder over Astellas for Sangamo Therapeutics’ Fabry disease gene therapy, while Eli Lilly is purchasing the biotech’s prion disease program and several platforms.
Reports of seven deaths in patients who had taken Neurocrine Biosciences’ Vykat and the official termination of Aardvark Therapeutics’ Phase 3 trial after cardiac complications marked a tough day for the Prader-Willi syndrome community.
Mereo BioPharma’s asset, dubbed alvelestat, is set to enter Phase 3 development for AATD-LD in 2027.
BridgeBio Pharma’s transthyretin amyloidosis medicine Attruby is off to a strong launch, while Alnylam lowered full-year sales guidance for competitor Amvuttra.
The mid-stage win of Silence Therapeutics’ siRNA drug in polycythemia vera validates the therapeutic approach of similar assets in development—but also sets a high standard for therapies from Takeda, Agios and Disc Medicine.
Vaderis is hoping that engasertib will become the first approved medicine for hereditary hemorrhagic telangiectasia, a rare condition that can cause spontaneous bleeding.
A surprise flop in Phase 2a cystic fibrosis trial sent Sionna’s shares plummeting, but for Vertex, the news reinforced its standard-of-care standing with highly successful drugs like Trikafta.
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