Design Therapeutics (DSGN) investor relations material

Executive summary

• Clinical-stage biopharma focused on GeneTAC® molecules for nucleotide repeat expansion diseases, with lead programs in Friedreich ataxia (FA), Fuchs endothelial corneal dystrophy (FECD), myotonic dystrophy type-1 (DM1), and Huntington's disease (HD).

• Advanced RESTORE-FA trial for Friedreich ataxia, with positive four-week data showing DT-216P2 increased frataxin and improved clinical measures; DT-216P2 generally well-tolerated.

• FECD program (DT-168) achieved enrollment goals for observational study; Phase 2 biomarker trial ongoing but delayed to 2027 due to supply issues.

• DM1 program (DT-818) initiated Phase 1 MAD trial in 2026, with data expected in 2027.

• Continued preclinical work on Huntington's disease candidates.

Financial highlights

• Cash, cash equivalents, and investment securities totaled $207.4 million as of June 30, 2026, down $12.4 million from December 31, 2025.

• Net loss for Q2 2026 was $20.2 million, compared to $19.1 million in Q2 2025; net loss for the first half of 2026 was $37.8 million, compared to $36.8 million in the prior year.

• Operating expenses for Q2 2026 were $22.2 million, up from $21.6 million in Q2 2025; six-month operating expenses were $41.9 million, nearly flat year-over-year.

• R&D expenses were $16.4 million and G&A expenses were $5.8 million for Q2 2026.

• $19.9 million raised via at-the-market (ATM) equity offering in the first half of 2026.

Outlook and guidance

• Cash runway expected to fund operations for more than 12 months from the reporting date.

• RESTORE-FA 12-week cohort data expected in Q1 2027; update on registrational plans anticipated in Q4 2026.

• FECD Phase 2 biomarker trial data and DM1 Phase 1 MAD trial data expected in 2027.

• Anticipates continued net losses and increased expenses as clinical programs advance.