Design Therapeutics' RESTORE-FA trial of DT-216P2 in Friedreich ataxia showed meaningful clinical and biomarker improvements after four weeks of IV dosing, with a 6.4‑point mean improvement on mFARS at 1 mpk and robust increases in frataxin mRNA and protein in blood and muscle, supporting a registrational path.
Vincentage Pharma’s oral GLP‑1 agonist VCT220 achieved about 12% weight loss at 52 weeks in a Phase 3 trial in overweight or obese Chinese adults, with a safety profile similar to other GLP‑1s and low discontinuation rates due to treatment‑related adverse events.
Sources:
DT-216P2 - Friedreich's Ataxia Research Alliance
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