Dyne Therapeutics, the company developing the antisense oligonucleotide DYNE-101 for Steinert disease, has published initial results from its ongoing ACHIEVE trial. This is a Phase I/II trial starting with a dose escalation phase.
- The treatment appears to be well tolerated at all three doses tested.
- Improvements in myotonia and in the Myotonic Dystrophy Health Index (MDHI) were measured after six months in participants receiving the lowest dose.
- Biological parameters show an effect of DYNE-101 at the cellular level (reduction in the amount of toxic DMPK RNA and in the percentage of splicing abnormalities linked to mutated DMPK RNA) in the first two cohorts treated with low and intermediate doses.
The ACHIEVE trial is continuing, and a fourth dose is due to be evaluated shortly.
Dyne Therapeutics Announces Positive Initial Clinical Data from ACHIEVE Trial in DM1 Patients and DELIVER Trial in DMD Patients Demonstrating Promise of the FORCE™ Platform in Developing Therapeutics for Rare Muscle Diseases – DyneTherapeutics – Press release, 3 January 2024
