Design Therapeutics — Third Quarter 2026 Earnings Summary

Financial Performance:

• Net loss was $20.3 million for the quarter ended September 30, 2026, compared to a net loss of $17.0 million in the same period of 2025.
• Research and development expenses were $16.9 million for the quarter, up from $14.6 million in Q3 2025.
• General and administrative expenses were $5.3 million for the quarter, up from $4.7 million in Q3 2025.
• Total operating expenses were $22.2 million for the quarter, compared to $19.3 million in Q3 2025.
• Other income, net was $1.9 million for the quarter, down from $2.3 million in Q3 2025.
• Net loss per share (basic and diluted) was $0.32 for the quarter, compared to $0.30 in Q3 2025.
• Cash, cash equivalents, and investment securities totaled $208.6 million as of September 30, 2026, down from $219.8 million as of December 31, 2025.
• Total assets were $216.8 million as of September 30, 2026, compared to $226.2 million as of December 31, 2025.
• Total liabilities were $14.6 million as of September 30, 2026, compared to $13.7 million as of December 31, 2025.
• Total stockholders' equity was $202.2 million as of September 30, 2026, down from $212.5 million as of December 31, 2025.
• For the nine months ended September 30, 2026, net loss was $58.1 million, compared to $53.8 million in the same period of 2025.

Guidance and Future Outlook:

• Design Therapeutics expects to provide an update on registrational plans for DT-216P2 in the fourth quarter of 2026.
• 12-week data from the RESTORE-FA trial for DT-216P2 is expected in the first quarter of 2027.
• Data readouts for the Phase 1 trial of DT-818 in Myotonic Dystrophy Type-1 are anticipated in 2027.
• Data from the Phase 2 biomarker trial of DT-168 for Fuchs Endothelial Corneal Dystrophy is expected in 2027.
• The company continues to advance preclinical characterization for its Huntington's disease program.

Business Segments and Product Lines:

• Friedreich Ataxia (FA): The Phase 1/2 multiple ascending dose RESTORE-FA trial of DT-216P2 is ongoing.
• Myotonic Dystrophy Type-1 (DM1): The Phase 1 multiple-ascending dose trial of DT-818, a GeneTAC® small molecule designed to selectively reduce transcription of the mutant DMPK allele, is ongoing.
• Fuchs Endothelial Corneal Dystrophy (FECD): A Phase 2 biomarker trial of DT-168 is ongoing to evaluate safety, tolerability, and corneal endothelium biomarkers in patients scheduled for corneal transplant surgery.
• Huntington's Disease (HD): Preclinical characterization of several candidate molecules is advancing.
• Discovery efforts are underway for multiple genomic medicines.

Market and Competitive Landscape:

• The company operates in the clinical-stage biotechnology sector, developing treatments for serious degenerative genetic diseases.
• The press release notes that competitive products may make any products developed or sought to be developed obsolete or noncompetitive.

Risks and Challenges:

• Risks include the possibility that data from early clinical and nonclinical studies may impact clinical development plans.
• Pursuing a biomarker-driven clinical development strategy carries increased risks due to a limited number of approved biomarker-specific therapies.
• Challenges include patient enrollment and retention difficulties, which could delay or adversely affect clinical development plans.
• Potential for undesirable side effects or properties that could cause regulatory authorities to suspend or discontinue clinical trials.
• Risks associated with the ability to obtain and maintain intellectual property protection.
• Risks related to reliance on third parties, including contract manufacturers and contract research organizations.
• The company may need to raise additional funding to continue pursuing business and product development plans.

Management Commentary and Tone:

• Pratik Shah, Ph.D., Chairperson and CEO, stated the company remains focused on evaluating DT-216P2 over longer treatment durations and preparing for the next stage of clinical development in Friedreich ataxia.
• Management highlighted that clinical readouts expected in 2027 will help guide programs as the company pursues its vision for the GeneTAC® platform to address underlying causes of serious genetic diseases.

Other Key Points:

• The company has a strong financial runway with $208.6 million in cash and securities at quarter-end.
• The press release includes a detailed "Forward-Looking Statements" section outlining risks and uncertainties under the Private Securities Litigation Reform Act of 1995.
• The company is a clinical-stage biotechnology firm developing GeneTAC® gene targeted chimera small molecules designed to dial up or down the expression of specific disease-causing genes.