Design Therapeutics (DSGN) investor relations material

Executive summary

• Advanced four GeneTACⓇ molecule programs for severe monogenic disorders: Friedreich Ataxia (FA), Fuchs Endothelial Corneal Dystrophy (FECD), Myotonic Dystrophy Type 1 (DM1), and Huntington's Disease (HD), with three clinical-stage programs in 2026 and HD in active research.

• Lead FA candidate DT-216P2 advanced to Phase 1/2 after FDA clinical hold was lifted in December 2025; RESTORE-FA trial ongoing with interim 12-week data expected in the second half of this year and frataxin update in H2 2026.

• FECD program (DT-168) completed Phase 1 and is in Phase 2 biomarker trial, with data expected in H2 2026.

• DM1 candidate (DT-818) entered Phase 1 MAD trial, with dosing to begin in 1H 2026 and results anticipated in 2027.

• No product revenue to date; operations funded primarily through equity offerings.

Financial highlights

• Cash, cash equivalents, and investment securities totaled $222.8 million as of March 31, 2026, expected to fund planned operations into 2029.

• Net loss for Q1 2026 was $17.6 million, compared to $17.7 million in Q1 2025; operating expenses decreased year-over-year.

• R&D expenses were $14.4 million and G&A expenses $5.3 million for Q1 2026.

• $19.9 million raised via at-the-market (ATM) equity offering in Q1 2026.

Outlook and guidance

• Cash position expected to fund operations into 2029; anticipates increased expenses and operating losses as programs advance.

• Data readouts for RESTORE-FA and FECD Phase 2 trials expected in H2 2026; DM1 Phase 1 results anticipated in 2027.

• RESTORE-FA aims to establish proof of concept, inform regulatory pathways, and determine a go/no-go decision for the FA program.