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Mar 31, 2023
Design Therapeutics Q1 2023 Earnings Report
Provided pipeline updates and reported first quarter 2023 financial results.
Key Takeaways
Design Therapeutics reported its first quarter 2023 financial results, highlighting progress in its GeneTAC™ small molecule pipeline. Initial data from the Phase 1 multiple-ascending dose trial of DT-216 for Friedreich Ataxia is expected in the third quarter of 2023. The company's cash and securities are expected to support operating runway through 2025.
Initial data from Phase 1 multiple-ascending dose trial of DT-216 for Friedreich Ataxia expected in the third quarter of 2023.
Progress across GeneTAC™ small molecule pipeline with IND submissions for FECD and DM1 programs on-track for the second half of 2023 and 2024, respectively.
Cash and securities of $315.4 million expected to support operating runway through 2025.
Preparing to submit a second Investigational New Drug application (IND) in the second half of this year to support development of our novel DT-168 eye drop for patients with Fuchs endothelial corneal dystrophy (FECD).
Design Therapeutics
Design Therapeutics
Forward Guidance
Design Therapeutics anticipates several milestones, including initial data from the Phase 1 MAD trial of DT-216, IND submission for DT-168 for FECD, and IND submission for the DM1 program.
Positive Outlook
- Initial data from Phase 1 MAD trial of DT-216 for FA expected in Q3 2023.
- IND submission on-track for DT-168 for FECD in the second half of 2023.
- IND submission on-track for DM1 Program in the second half of 2024.
- Sufficient cash to fund operating plan through 2025.
- Advancing preclinical characterization of lead GeneTAC™ molecules for DM1.
Challenges Ahead
- An unanticipated vendor issue related to the study drug vial stopper caused a short delay in product supply for DT-216.
- Early-stage programs are subject to projections and may not yield expected results.
- Preclinical and early-stage clinical data may not translate to later stages.
- Clinical trial and patient enrollment are affected by many factors, which may cause delays.
- Reliance on third parties to conduct clinical trials and preclinical studies poses risks.