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Cell & Gene Therapy Insights

Cell & Gene Therapy Insights

Solid Biosciences to proceed gene therapy trial for DMD with increased dose

Based on the promising preliminary findings from its ongoing Phase 1/2 trial (IGNITE DMD) for Duchenne muscular dystrophy, Solid Biosciences has announced its plan to escalate the dose of its gene therapy.

Duchenne muscular dystrophy (DMD) is a progressive, X-linked degenerative disorder caused by the absence of dystrophin and is the most commonly inherited neuromuscular disease. Dystrophin protein levels are affected due to out-of-frame mutations in the dystrophin gene. Solid Biosciences’ lead gene therapy candidate SGT-001 uses an AAV9 vector to deliver microdystrophin, a shorter form of the dystrophin protein. Preclinical studies have shown that single administration of SGT-001 resulted in long-term expression of micro-dystrophin in muscle and has the potential to slow or stop the progression of DMD.

Preliminary analysis was based on initial three-month biopsy data from the first three patients dosed with 5E13 vg/kg of SGT-001, the lowest dose outlined in the trial protocol. Microdystrophin level, although present, was very low in all the three patients. SGT-001 was safe and well tolerated by all patients. Increasing the dose to the next level, the team believes, could restore protein level and potentially stop DMD progression in these patients.

SGT-001 was developed based on the work by Dr Jeffrey Chamberlain (University of Washington) and Dr Dongsheng Duan (University of Missouri). It was granted Rare Pediatric Disease Designation and Fast Track Designation in the US and Orphan Drug Designations in both the EU and US.

The IGNITE DMD is a randomized, open-label, single-ascending dose Phase 1/2 clinical trial to evaluate SGT-001 in ambulatory and non-ambulatory males with DMD aged 4 to 17 years. The primary objectives of the study are to assess the safety and tolerability of SGT-001, as well as efficacy as defined by microdystrophin expression. Six patients have been enrolled in IGNITE DMD thus far, three to the active treatment group and three to the delayed treatment control group, the latter group will receive active treatment 12 months after trial initiation.

Ilan Ganot, Co-founder, CEO and President of Solid Biosciences commented: “We have already begun working to expedite the planned dose escalation strategy outlined in our clinical trial protocol. This strategy is further supported by our scalable manufacturing process, from which we have sufficient drug product available to dose escalate without delay. We have the financial resources to execute on our plan and look forward to communicating additional data later this year.”

Source: Solid Biosciences Announces Preliminary SGT-001 Data and Intention to Dose Escalate in IGNITE DMD Clinical Trial for Duchenne Muscular Dystrophy; Press Release

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