On Tuesday, Dyne Therapeutics Inc. DYN introduced new scientific knowledge from its ongoing Part 1/2 DELIVER trial of DYNE-251 in sufferers with Duchenne muscular dystrophy (DMD) amenable to exon 51 skipping.
This evaluation of the DELIVER trial evaluating DYNE-251 contains 6-month biomarker and useful knowledge from 8 male sufferers enrolled within the 20 mg/kg (approximate PMO dose) cohort who have been randomized to obtain DYNE-251 or placebo as soon as each 4 weeks and 12-month useful knowledge from 6 members within the 10 mg/kg cohort.
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At a 20-mg/kg dose degree, given each 4 weeks, DYNE-251 resulted in a imply absolute dystrophin expression of three.71% of regular, with out adjusting for muscle content material.
The corporate famous that the expression degree is greater than ten occasions greater than in a reference scientific trial assessing the weekly customary of care therapy Sarepta Therapeutics Inc’s SRPT EXONDYS 51 (eteplirsen), the place dystrophin expression was at 0.3% regular.
After controlling for sufferers’ muscle content material, the corporate stated that the imply absolute dystrophin expression jumped to eight.72%, which exceeds ranges sometimes recorded in research evaluating different investigational oligomers for DMD.
These outcomes are in keeping with six-month knowledge Dyne launched in Might for sufferers handled with the 10-mg/kg dose of DYNE-251.
Sufferers handled with 10 mg/kg of DYNE-251 Q4W had a imply absolute dystrophin degree of three.22% of regular and a 2.97% change (unadjusted for muscle content material) from baseline at six months.
When adjusting for muscle content material, the DYNE-251 handled group reached 7.64% imply absolute dystrophin.
Dyne Therapeutics says significant enhancements in a number of useful endpoints have been noticed within the 20 mg/kg and 10 mg/kg DYNE-251 Q4W teams. The ten mg/kg cohort confirmed continued enchancment in all reported measures from 6 to 12 months.
DYNE-251 demonstrated a positive security profile, and most treatment-emergent hostile occasions have been delicate or reasonable.
Dyne says it’s initiating registrational cohorts within the DELIVER trial and plans to offer an replace on the trail to registration by the top of 2024.
Value Motion: DYN inventory is down 31.90% at $31.39 finally examine Tuesday.
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