What happened
Shares of Design Therapeutics (DSGN 0.53%) were down more than 69% as of 2:15 p.m. ET on Tuesday. The healthcare company announced phase 1 results for its Friedreich ataxia (FA) therapy, DT-216. The stock is down more than 78% so far this year.
So what
The clinical-stage biotech company released its second-quarter earnings report on Monday after the markets closed. It also announced phase 1 results for DT-216 to treat FA, a rare, inherited disease that causes progressive damage to the nervous system. The disease affects more than 5,000 people in the U.S. and has no approved therapies yet.
The company said the safety profile for DT-216 was good, though it mentioned that some subjects in the study developed injection site reactions to DT-216. The company said a new formulation of the drug wouldn't be ready until the second half of 2024, with results from that dosage expected in the first half of 2025. That pushes back the timeline in which Design could potentially launch its lead pipeline therapy to as late as fiscal 2029.
Now what
As a clinical-stage company, Design doesn't have any revenue. It did have a net loss of $19.9 million in the quarter and said it had $303.1 million in cash as of June 30, enough to fund operations through 2026. With DT-216's likely launch after that, it's obvious the company will need more financing to get to the finish line unless one of its other pipeline therapies is approved before then.
Other key therapies in Design's pipeline could soon have their investigational new drug (IND) submission go before the Food and Drug Administration (FDA). DT-168 is an eye drop treatment for patients with the eye disease, Fuchs endothelial corneal dystrophy, and is expected to have its IND submission in the second half of this year. Several of the company's GeneTAC molecules are being examined as therapies to treat the multisystem genetic disorder, myotonic dystrophy type 1, with a possible IND submission in the second half of 2024.