Design Therapeutics (DSGN) reported top-line data Monday from its phase 1/2 RESTORE-FA clinical trial evaluating DT-216P2 to treat Friedreich ataxia, a recessive neurodegenerative disorder.
The biotech company said that after a four-week dosing window for 16 patients, the multi-dose cohort evaluation demonstrated dose-dependent increases in frataxin mRNA and frataxin protein.
It also said the treatment was generally well-tolerated, with no serious adverse events reported and no treatment discontinuations.
Design Therapeutics said that it will use the trial data to progress DT-216P2 into a registrational development track, with a formal regulatory update expected in Q4.
Shares of the company were up close to 7% pre-bell.
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