Solid Biosciences has provided an update on the safety and efficacy data from their IGNITE DMD Phase I/II clinical trial:

We are pleased to share an update regarding the safety and efficacy data from our ongoing IGNITE DMD phase I/II clinical trial, as announced in the press release issued this afternoon. Additionally, we are excited to announce that we have resumed safe dosing at the 2E14 vg/kg dose, and that this patient experienced mild to moderate adverse events all of which have fully resolved. We are encouraged with the improved safety profile of SGT-001 under our previously reported amended protocol and second-generation manufacturing process, both of which were designed to enhance patient safety.

The data reported were collected from the first six patients dosed in IGNITE DMD twelve to twenty-four months after treatment and include data from three patients dosed at the low dose (5E13vg/kg) and three patients dosed at the high dose (2E14 vg/kg). Data from the delayed treatment cohort, analyzed as an untreated control cohort, were evaluated alongside representative natural history data. The six patients ranged in age from five to 14-years-old at time of dosing.

Click here to read the full Letter to the Duchenne Community.

Click here to read the Press Release.

The totality of data collected and the re-initiation of safe dosing support the continued enrollment of patients into the IGNITE DMD study.

For more information, you can visit clinicaltrials.gov (NCT03368742) for updates as they become available. If you have any questions, please feel free to contact SOLID directly at Community@solidbio.com.