RESTORE-FA trial modifications keep 1 mpk as the planned go-forward dose, targeting 10 patients in the 12-week cohort; 12-week data are expected in Q1 2027.
Management said four-week RESTORE-FA data showed DT-216P2 increased endogenous frataxin; dosing began in a Phase 1 MAD trial of DT-818 in DM1.
Quarterly results and cash position
Design Therapeutics posted a Q2 net loss of USD 20.2 million, widening from USD 19.1 million a year earlier.
R&D expenses rose to USD 16.4 million from USD 15.7 million, while G&A expenses edged down to USD 5.8 million from USD 5.83 million.
Cash, cash equivalents and investment securities slipped to USD 207.4 million at June 30 from USD 219.8 million at Dec. 31.
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