PTC Therapeutics announced Thursday that its Huntington’s disease candidate PTC518 is no longer subject to an FDA partial clinical hold after it showed positive interim results from a Phase II study.
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PTC Therapeutics said on Thursday the European health regulator’s advisory panel reaffirmed a negative opinion for its drug used to treat an inherited progressive muscle-wasting disorder, sending its shares down over 11% in extended trading.
Royalty Pharma said it has purchased additional royalties on Roche and PTC Therapeutics’ oral spinal muscular atrophy (SMA) drug for an upfront payment of $1 billion.
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.
PTC Therapeutics said on Wednesday its experimental Huntington’s disease drug lowered mutated protein levels that cause the neurological disease, citing interim data from a mid-stage study.
The goal of the trial was to reduce levels of phenylalanine, an amino acid, in the blood. Patients with the inherited metabolic disorder cannot break down phenylalanine on their own, causing a build-up of the acid, which often leads to memory and neurological issues.
The gene therapy has yet to be approved in the U.S. PTC expects to submit a Biologics License Application (BLA) to the FDA in the first half of 2023, the company announced.
PTC Therapeutics reported Q4 and full-year 2022 results Tuesday ahead of a pivotal 2023 featuring three late-stage readouts in registration-directed trials.
End-to-end vector manufacturing optimizes batch success and delivers more doses at lower cost.