Rare diseases
Govorestat failed to meet its primary endpoint in a Phase II/III trial for a rare form of Charcot-Marie-Tooth disease, a few months after the FDA rebuffed the same drug in a similar indication.
While sparking excitement among biopharma companies focused on rare and ultrarare indications, experts say FDA Commissioner Marty Makary’s proposal is light on details and raises potential concerns about safety, access and liability.
The star of the acquisition is the enzyme replacement therapy INZ-701, being developed for the rare disease ENPP1 deficiency.
Azafaros will use the Series B haul to push lead asset nizubaglustat into late-stage studies for Niemann-Pick disease Type C and GM1/GM2 gangliosidoses later this year.
The company discontinued development last month of its most mature asset, RLYB212, following disappointing mid-stage pharmacokinetic findings in a rare bleeding disorder.
CRISPR Therapeutics’ partner Vertex reported that more than 65 treatment centers have been activated for the gene therapy Casgevy. While Vertex handles the market, CRISPR has been focused on its clinical program.
Vertex has recorded some 25,000 prescriptions for Journavx since its January approval and is in the process of getting big PBMs to cover the non-opioid pain drug.
At the heart of the acquisition is Regulus’ farabursen, an miRNA-targeting oligonucleotide in early-stage development for rare autosomal dominant polycystic kidney disease.
The condition, recessive dystrophic epidermolysis bullosa, causes chronic wounds and has an 84% mortality rate by age 40.
In an interview with former Fox News journalist Megyn Kelly, FDA Commissioner Marty Makary introduced a new mechanism-driven pathway that could be leveraged by rare disease therapies while saying that autism could potentially be driven by certain environmental factors.
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