PTC Therapeutics, Inc.

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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.
Huntington’s disease might just top the list of intractable neurodegenerative diseases. BioSpace spoke with the field’s foremost experts about the challenges and how to overcome them.
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