PTC Therapeutics, Inc.
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Despite the “unfortunate” failure, William Blair analysts do not believe that the utreloxastat readout will heavily affect PTC, instead postulating that the upcoming FDA decision on its phenylketonuria candidate sepiapterin will be a stronger driver of the biotech’s stock.
As the Phase III amyotrophic lateral sclerosis pipeline thins out, the ALS community is placing its hopes on earlier-stage trials sponsored by Denali Therapeutics, PTC Therapeutics and more.
The deal with PTC Therapeutics increases Royalty Pharma’s stake to 13% of total royalties garnered by Evrysdi, the blockbuster drug for spinal muscular atrophy licensed and marketed by Roche.
More than a month after failing its Friedrich’s ataxia trial, PTC Therapeutics’ vatiquinone has again missed its primary endpoint, this time in a mitochondrial disease-associated seizures study.
The Huntington’s disease space saw a flurry of activity Wednesday as PTC and uniQure released data from their respective mid-phase trials.
Despite a disappointing showing in the Phase III MOVE-FA trial, PTC Therapeutics says it will move forward with regulatory talks for the Friedreich’s ataxia treatment. Meanwhile, the company is discontinuing several early-stage gene therapies and reducing its headcount.
The Phase III trial met its primary endpoint, a significant reduction of phenylalanine levels in the blood, with no severe adverse events reported, PTC Therapeutics reported Wednesday.
Upstaza is the first targeted therapy for aromatic L-amino acid decarboxylase (AADC) deficiency. PTC expects to submit a Biologics License Application to the FDA in the first half of 2023.
PTC Therapeutics reported Q4 and full-year 2022 results Tuesday ahead of a pivotal 2023 featuring three late-stage readouts in registration-directed trials.
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