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Design Therapeutics Announces Third Quarter 2024 Financial Results and Reviews Near-term Milestones for GeneTACTM Portfolio

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Design Therapeutics, Inc.
Design Therapeutics, Inc.

Fuchs Endothelial Corneal Dystrophy (FECD) Phase 1 Trial Initiated with Data Expected in the First Half of 2025

Friedreich Ataxia (FA) Program on Track to Initiate Phase 1 Single Ascending Dose, Healthy Volunteer Study in the First Half of 2025

Active Research Pipeline Advancing with Programs in Myotonic Dystrophy Type-1 (DM1) and Huntington’s Disease (HD)

Well-Capitalized with Cash and Securities of $254.1 Million to Fund Operations Through Up to Four Potential Clinical Proof-of-Concept Data Sets

CARLSBAD, Calif., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced its third quarter 2024 financial results and reviewed anticipated upcoming program milestones for its portfolio of GeneTAC™ candidates.

“Thanks to our progress so far this year, the first half of 2025 will be a busy one, with data expected from our ongoing Phase 1 trial in FECD and the start of clinical activities for our FA program,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “We believe these programs lead a pipeline of GeneTAC™ small molecules capable of transforming the status quo in genomic medicines, with the potential for multiple clinical proof-of-concept data sets over the next few years.”

Corporate Highlights and Anticipated Upcoming Milestones

  • Friedreich Ataxia (FA) Design is on track to initiate the Phase 1 single ascending dose, normal healthy volunteer trial for DT-216P2 in the first half of 2025. The company anticipates beginning FA patient dosing later in 2025.

  • Fuchs Endothelial Corneal Dystrophy (FECD) The company has initiated Phase 1 development for DT-168 in normal healthy volunteers, with initial data expected in the first half of 2025. In parallel, Design continues its ongoing FECD observational study.

  • Pipeline programs Design also continues to advance preclinical characterization of several lead molecules toward the selection of development candidates for Huntington’s disease (HD) and myotonic dystrophy type-1 (DM1) in anticipation of future IND submissions.

Third Quarter 2024 Financial Results

  • R&D Expenses: Research and development (R&D) expenses were $11.9 million for the quarter ended September 30, 2024.

  • G&A Expenses: General and administrative (G&A) expenses were $4.4 million for the quarter ended September 30, 2024.

  • Net Loss: Net loss was $13.0 million for the quarter ended September 30, 2024.

  • Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $254.1 million as of September 30, 2024, which the company expects to fund its planned operating expenses into 2029.