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Jun 30, 2023
Design Therapeutics Q2 2023 Earnings Report
Design Therapeutics reported second quarter 2023 financial results and provided pipeline update.
Key Takeaways
Design Therapeutics reported 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. The company had $303.1 million in cash and securities, expected to support operating runway through 2026.
Reported initial data from Phase 1 MAD trial demonstrating proof-of-concept for DT-216 for FA.
Design plans 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 is on-track for the second half of 2023.
Cash and securities of $303.1 million are expected to support operating runway through 2026.
Design Therapeutics
Design Therapeutics
Forward Guidance
Design Therapeutics anticipates several milestones and continued progress in its GeneTAC™ pipeline, including IND submissions and clinical trial advancements.
Positive Outlook
- Resuming clinical development in FA and announcing data therefrom and the timing thereof
- Design’s ability to improve the formulation of DT-216 to prevent injection site thrombophlebitis or other limiting side effects
- Design’s anticipated timeline to submit an IND for DT-168 in the second half of 2023
- Design’s anticipated timeline to select a development candidate and submit an IND for its GeneTAC™ program for the treatment of DM1 in the second half of 2024
- The potential of Design’s GeneTAC™ small molecules to be a new class of therapies for patients suffering from devastating genetic diseases
Challenges Ahead
- Risks associated with the acceptance of INDs by the FDA for the conduct of planned clinical trials of our product candidates and our proposed design of future clinical trials
- Risks associated with designing and implementing investigational drug product formulation improvements
- Risks associated with conducting a clinical trial and patient enrollment, which is affected by many factors, and any difficulties or delays encountered with such clinical trial or patient enrollment may delay or otherwise adversely affect Design’s Phase 1 clinical development of DT-216
- The risk that undesirable side effects or other properties could cause Design or regulatory authorities to suspend or discontinue clinical trials which could delay or prevent Design’s product candidates’ development or regulatory approval
- Design’s ability to raise any additional funding it will need to continue to pursue its business and product development plans