Janux stock plunges after hours following mCRPC trial data
CARLSBAD - Design Therapeutics, Inc. (NASDAQ:DSGN) announced Wednesday it has obtained ex-US regulatory clearance for DT-818, a GeneTAC small molecule developed for the treatment of myotonic dystrophy type-1 (DM1). The clinical-stage biotech, currently valued at $374.72 million, has seen its shares trade at $6.55, near the upper end of its 52-week range of $2.60-$7.77.
The clinical-stage biotechnology company plans to begin dosing DM1 patients in a Phase 1 multiple-ascending dose trial in Australia during the first half of 2026, with splicing data expected in 2027.
According to the company, DT-818 has demonstrated promising preclinical results, including a greater than 90% reduction in toxic RNA foci in DM1 patient cells and selective targeting of mutant DMPK, the gene responsible for the disease.
"Today we are excited to unveil DT-818 as our development candidate for the treatment of DM1, a debilitating neuromuscular disease with significant unmet medical need," said Pratik Shah, chairperson and chief executive officer of Design Therapeutics.
The company also reported progress on its other clinical programs. The RESTORE-FA Phase 1/2 trial of DT-216P2 for Friedreich ataxia continues to enroll patients, with data expected in the second half of 2026. Similarly, a Phase 2 biomarker trial of DT-168 for Fuchs endothelial corneal dystrophy is ongoing with data anticipated in the second half of 2026.
Design Therapeutics reported a net loss of $17.0 million for the third quarter of 2025, with research and development expenses of $14.6 million and general and administrative expenses of $4.7 million. The company maintained a strong financial position with $206.0 million in cash, cash equivalents and investment securities as of September 30, 2025.
The company also announced the September 2025 appointment of Justin Gover to its Board of Directors, who previously served as founding CEO of GW Pharmaceuticals.
This information is based on a press release statement from Design Therapeutics.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.