Solid Adds to Differentiated Profile With New Data for Duchenne Gene Therapy

 

Solid Biosciences’ SGT-003 is the only late‑stage program to show early cardiac benefit across biomarkers and function, according to William Blair.

Solid Biosciences’ investigational gene therapy boosts the expression levels of key markers that are otherwise deficient in patients with Duchenne muscular dystrophy, according to Phase 1/2 results announced Wednesday. The findings not only support the asset’s biological mechanism but could also help the biotech make its case to the FDA, according to analysts.

“The data point to a differentiated product profile that we expect will pave the way for a productive discussions w/ the FDA,” analysts at Jefferies wrote in a note on Thursday.

William Blair analysts were likewise bullish about Solid’s data, writing that the gene therapy, dubbed SGT-003, “remains the only late‑stage program to show early cardiac benefit across biomarkers and function, which we continue to view as a meaningful differentiator in a disease where cardiac involvement is a major driver of long‑term morbidity.”

This latest readout from Solid, William Blair continued, strengthens “the biologic plausibility of SGT‑003’s treatment effect.”

In patients with Duchenne muscular dystrophy (DMD), genetic mutations render dystrophin deficient or dysfunctional, giving rise to the hallmark symptoms of the disease, such as progressive muscle weakness, body deformities and breathing and cardiac complications. Designed to be a one-time infusion, SGT-003 delivers a construct for microdystrophin that boosts levels of the dystrophin protein in the body.

In the Phase 1/2 INSPIRE DUCHENNE trial, interim data from which were presented on Wednesday, 90-day biopsies from 20 treated patients showed “robust” expression levels of the microdystrophin marker, as well as “properly localized and restored” fibers positive for certain molecular indicators, the company said in its announcement. These fibers, Solid explained, are components of the dystrophin-associated protein complex, which helps maintain muscle integrity. This complex is absent in patients with DMD.

INSPIRE DUCHENNE did not employ an untreated control arm. The gene therapy’s efficacy was measured by comparing patients’ measurements to their baseline levels.

Solid also conducted preliminary cardiac assessments of treated patients and touted stabilized or improved systolic function, particularly in patients with weaker left ventricular ejection fraction at baseline. SGT-003 also improved other biomarkers of muscle integrity, including creatinine kinase and lactate dehydrogenase.

Solid has reached an agreement with the FDA to run a Phase 3 placebo-controlled study for the gene therapy, according to an announcement last month. That trial continues to screen participants, building up to a first dose given this quarter, the biotech said on Thursday. In the meantime, Solid will continue to meet with the FDA regarding the potential to apply for accelerated approval.

https://www.biospace.com/drug-development/solid-adds-to-differentiated-profile-with-new-data-for-duchenne-gene-therapy