Design Therapeutics (DSGN) investor relations material

Key program updates and milestones

• RESTORE-FA study aims to demonstrate increased endogenous frataxin in FA patients, with 12-week dosing data expected in the second half of the year.

• Multiple ascending dose studies are ongoing, with safety as the primary criterion for dose escalation.

• Preclinical and early clinical data support the 10 nanomolar exposure target for DT-216, with sustained exposure hypothesized to yield steady-state pharmacology.

• Patient enrollment criteria are broad, including both ambulatory and wheelchair-bound individuals, to assess frataxin response across a spectrum.

• Functional assessments are included but are not the primary focus; the main goal is to measure frataxin response.

Pipeline and technology platform insights

• Exploratory biomarker study in late-stage Fuchs' corneal dystrophy (FECD) is ongoing, using corneal tissue to assess spliceopathy and target engagement, with results expected in the second half of the year.

• DT-818 for DM1 is a small molecule that targets pathogenic CTG repeats, showing over 90% reduction in toxic DMPK RNA and comparable efficacy across a wide range of repeat lengths.

• Both IV and sub-Q administration routes are being explored for DT-818, potentially offering advantages over current investigational therapies.

• The GeneTAC platform enables gene-specific modulation, with different programs either relieving transcriptional blocks or repressing transcription as needed.

• Lessons from earlier programs have informed the development and biomarker strategies for newer candidates.

Financial position and strategic outlook

• Cash position exceeds $200 million, providing runway into 2029 and supporting the goal of achieving clinical proof of concept in at least one major indication.

• Business plan is structured to prioritize positive clinical data in areas of high unmet need, with the expectation that such data could drive significant therapeutic advances.