DT-216 / Design Therap - LARVOL DELTA

Design Therapeutics Highlights Progress Across Lead GeneTAC Programs and Reports Fourth Quarter and Full Year 2024 Financial Results (GlobeNewswire) - "Dosing Complete in DT-168 Phase 1 Healthy Volunteer Trial, with Data on Track for the First Half of 2025...Selection of Development Candidate for Myotonic Dystrophy Type-1 (DM1) Expected in 2025...A Phase 1/2 multiple ascending dose (MAD) clinical trial...in FA patients is anticipated to begin in mid-2025. Data based on twelve weeks of DT-216P2 dosing in patients is anticipated in 2026."

New P1/2 trial • P1 data • P1/2 data • Pipeline update • Friedreich ataxia • Myotonic Dystrophy • Ophthalmology

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia (clinicaltrials.gov) - P1 | N=32 | Completed | Sponsor: Design Therapeutics, Inc. | Recruiting ➔ Completed | Phase classification: P1b ➔ P1

Phase classification • Trial completion • Ataxia • Friedreich ataxia • Movement Disorders

Study to Evaluate DT-216 in Adult Patients With Friedreich Ataxia (clinicaltrials.gov) - P1a | N=39 | Completed | Sponsor: Design Therapeutics | Recruiting ➔ Completed | N=25 ➔ 39

Enrollment change • Trial completion • Ataxia • Friedreich ataxia • Movement Disorders

Study to Evaluate Multiple Ascending Dose and Multi-Dose of DT-216 in Adult Patients With Friedreich Ataxia (clinicaltrials.gov) - P1b | N=38 | Recruiting | Sponsor: Design Therapeutics

New P1 trial • Ataxia • Friedreich ataxia • Movement Disorders

Design Therapeutics Secures $125 Million in Series B Financing to Advance Pipeline of Genomic Medicines for Nucleotide Repeat Expansion Disorders (Odessa American) - "Design Therapeutics...today announced the successful completion of a $125 million Series B financing...'This capital raise provides important resources to efficiently advance our pipeline, including our lead programs in Friedreich ataxia and myotonic dystrophy type-1'...The company’s initial focus is on developing medicines for the treatment of nucleotide repeat expansion disorders, including Friedreich ataxia, which is expected to enter clinical development in the first half of 2022 and myotonic dystrophy type-1."

Financing • New trial • Friedreich ataxia • Genetic Disorders • Myotonic Dystrophy