Business
Design Therapeutics Provides Pipeline Update and Reports Second Quarter 2023 Financial Results
Initial Data from Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Showed Significant Increase in FXN mRNA Levels in Skeletal Muscle
About this update from Design Therapeutics, Inc.
[{"type":"text","content":"Initial Data from Phase 1 Multiple-Ascending Dose Trial of DT-216 for Friedreich Ataxia Showed Significant Increase in FXN mRNA Levels in Skeletal Muscle Company Expects to Resume a Multiple Dose Phase 1 Clinical Study with DT-216 with an Improved Formulation in the Second Half of 2024 and Report Data in the First Half of 2025 IND Submission for FECD GeneTAC™ Program On-track for the Second Half of 2023 $303.1 Million in Cash and Securities Expected to Support Operating Runway through 2026 CARLSBAD, Calif., Aug. 14, 2023 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today provided business updates and anticipated upcoming milestones across its clinical and research-stage pipeline of novel GeneTAC™ small molecules and reported second quarter 2023 financial results. “Throughout the first half of 2023, our team has been focused on executing our programs to bring our novel GeneTAC™ small molecules to patients who need them,” said João Siffert, M.D., president and chief executive officer of Design Therapeutics. “Today, we presented initial results from our Phase 1 multiple-ascending dose trial of DT-216 for the treatment of Friedreich ataxia, which show proof-of-concept for clinical activity and are in-line with our founding hypothesis that GeneTAC™ molecules are capable of restoring FXN transcription in patients with FA. Additionally, we are preparing to submit an IND later this year to enable clinical development of our novel DT-168 eye drop for patients with FECD, a degenerative genetic eye disease that affects millions of people. With an expert team and strong financial position afforded by efficient cost and program management, I am confident in our ability to execute our goals. We look forward to providing an update on our progress across our GeneTAC™ pipeline in the months to come.” Pipeline Updates and Anticipated Upcoming Milestones Reported Initial Data from Ongoing Phase 1 MAD Trial Demonstrating Proof-of-Concept for DT-216 for FA: Design reported initial results, based upon a data cutoff date of August 7, 2023, from the company’s Phase 1 multiple-ascending dose (MAD) clinical trial of its lead GeneTAC™ small molecule, DT-216, in patients with FA. Exploratory analyses demonstrated that DT-216 treatment ac...