BUZZ-Design Therapeutics falls after early-stage data for genetic disorder treatment
Design Therapeutics, Inc. DSGN | 0.00 |
** Shares of drug developer Design Therapeutics DSGN.O fall 22.7% to $11.11
** Co says early-stage data showed its experimental drug, DT-216P2, showed meaningful improvement in symptoms of Friedreich ataxia, a rare genetic disorder, in an early-stage study
** Says patients at the highest dose showed a 6.4-point improvement in a standard disease rating scale and a 2.7-point gain in balance scores after four weeks of treatment
** Says the drug raised levels of frataxin, a protein lacking in patients with the disease, by 65% in blood and 42% in muscle
** "There are some limitations, mostly associated with the small number of patients and early timepoint," RBC Capital Markets says, adding that "overall, we think these issues do not detract from the overall impression of the data"
** "While bears may suggest the impressive magnitude at such an early timepoint may require further validation...we think the data show that DT-216P2 is able to deliver a potentially best-in-class improvement on clinical scales" - brokerage
** DSGN says it plans to pursue a registrational path, with an update expected in Q4 2026
** Including session's move, DSGN up 22.4% YTD
