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CARLSBAD, Calif. - Design Therapeutics, Inc. (NASDAQ:DSGN), a clinical-stage biotechnology firm with a market capitalization of $228 million, has announced the initiation of a clinical trial for Friedreich ataxia (FA) with the first patient receiving a dose of DT-216P2. According to InvestingPro data, the company maintains a strong financial health score of 2.55, despite its stock declining 35% year-to-date. The RESTORE-FA trial aims to assess the safety, tolerability, and pharmacokinetics (PK) of the drug administered via intravenous and subcutaneous methods.
The ongoing Phase 1/2 trial has reported no adverse events for the dosed patient, including no injection site thrombophlebitis. Enrollment for the trial is currently open in Australia, with the company expecting to release data, including frataxin (FXN) expression levels, in 2026. InvestingPro analysis reveals the company holds more cash than debt on its balance sheet, though it’s currently burning through cash at a notable rate - crucial factors for investors monitoring clinical development progress.
Concurrently, Design Therapeutics has submitted an investigational new drug (IND) application to the U.S. Food and Drug Administration (FDA) for DT-216P2. However, the FDA has placed a clinical hold on the application due to nonclinical deficiencies, with further details to be provided within 30 days.
Initial data from a single-ascending dose (SAD) trial in healthy volunteers indicated that DT-216P2 was well-tolerated and showed promising pharmacokinetics, suggesting improved exposure over the first-generation formulation. Dr. Pratik Shah, CEO of Design Therapeutics, expressed surprise at the FDA’s clinical hold but affirmed the company’s commitment to addressing the FDA’s concerns and expanding the trial to U.S. sites.
DT-216P2 is an enhanced version of a GeneTAC® small molecule targeting the GAA repeat expansion mutation responsible for FA. It is designed to restore the production of endogenous frataxin proteins to therapeutic levels.
This clinical-stage biotechnology company is also advancing GeneTAC® programs for other genetic diseases, including Fuchs endothelial corneal dystrophy, myotonic dystrophy type-1, and Huntington’s disease, with additional genomic medicines in discovery phases. With analyst price targets ranging from $5 to $12, significantly above the current trading price of $4.01, investors seeking detailed financial analysis and additional insights can access over 10 more exclusive tips on InvestingPro.
The information in this article is based on a press release statement from Design Therapeutics.
In other recent news, Design Therapeutics has announced the appointment of Chris M. Storgard, M.D., as its new Chief Medical Officer. Dr. Storgard brings over 20 years of experience in drug development, having successfully guided drugs from preclinical stages to global regulatory approvals. Previously, he held the role of CMO at ADARx Pharmaceuticals and played a significant role in transitioning the company from research to development-stage. His career also includes securing U.S. and European product approvals in oncology and acute care at Heron Therapeutics. At Design Therapeutics, Dr. Storgard will advance the company’s GeneTAC® small molecules, including DT-216P2 for Friedreich ataxia and DT-168 for Fuchs endothelial corneal dystrophy. The company is also working on programs targeting myotonic dystrophy type-1 and Huntington’s disease. Dr. Storgard expressed enthusiasm for leveraging the GeneTAC® platform to impact the treatment of monogenetic disorders. This leadership change reflects Design Therapeutics’ ongoing efforts to address unmet needs in rare genetic disorders.
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