Business
Dyne Therapeutics Presents Data at World Muscle Society Congress Highlighting Promise of FORCE™ Platform to Address Underlying Causes of Neuromuscular Diseases
- Presentations Will Feature Recent Clinical Data From DYNE-251 and DYNE-101 as Well as Preclinical Data in FSHD and Pompe Disease - WALTHAM, Mass., Oct. 09,
About this update from Dyne Therapeutics, Inc.
[{"type":"text","content":"- Presentations Will Feature Recent Clinical Data From DYNE-251 and DYNE-101 as Well as Preclinical Data in FSHD and Pompe Disease - WALTHAM, Mass., Oct. 09, 2024 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that previously reported clinical and preclinical data across its pipeline will be featured in poster presentations at the 29th Annual Congress of the World Muscle Society, held virtually and in Prague, Czech Republic, October 8-12, 2024. The presentations highlight the promise of the FORCE™ platform to deliver targeted therapeutics to address neuromuscular diseases. “The full breadth and versatility of our FORCE platform is on display at this year’s World Muscle Society Congress, with presentations covering both of our co-lead clinical programs in DM1 and DMD, along with preclinical work in FSHD and Pompe disease,” said John Cox, president and chief executive officer of Dyne. “We’ve observed targeted delivery with the FORCE platform showing broad distribution to key tissues like skeletal, cardiac, smooth muscle and the CNS – which has the potential to transform the lives of individuals living with these diseases.” Poster Highlights The Phase 1/2 DELIVER trial evaluating DYNE-251 in males with Duchenne muscular dystrophy (DMD) mutations amenable to exon 51 skipping includes 6-month biomarker and functional data from patients enrolled in the 20 mg/kg (approximate PMO dose) cohort and 12-month functional data from the 10 mg/kg cohort. DYNE-251 demonstrated dose dependent exon skipping and dystrophin expression and improvement in multiple functional endpoints in both cohorts. Patients treated with 20 mg/kg of DYNE-251 Q4W had a mean absolute dystrophin expression of 3.7% of normal (unadjusted for muscle content) and when adjusting for muscle content, it reached 8.7%. Importantly, treatment with DYNE-251 resulted in meaningful improvements in Stride Velocity 95th Centile (SV95C), a digital objective outcome measure of ambulatory performance in a patient’s normal daily environment. The change from baseline in SV95C met the published minimal clinically important difference as defined by the European Medicines Agency (0.1 m/sec) at 6 months for both ...