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.

It’s 100 percent fatal, hits its victims in the prime of their lives and has companies with prospective treatments either shutting down or spinning their wheels with trial holds and failures. Huntington’s disease might just top the list of intractable neurodegenerative diseases.

PTC Therapeutics forged a financing deal valued at up to $1 billion with Blackstone that will allow it to deliver one new therapy every two or three years, the New Jersey-based biotech reported.