In This Article:
Shares of Solid Biosciences SLDB have rallied 59.9% in the past month. The stock price increase was mainly due to positive initial data, announced in mid-Feb, from the early to mid-stage study of its next-generation gene therapy product candidate, SGT-003, for treating Duchenne muscular dystrophy (DMD).
Interim biopsy data reported in the first three participants at day 90 showed an average microdystrophin expression of 110%, as measured by western blot, along with improvements in multiple biomarkers that are indicators of muscle health and resilience.
In the past three months, shares of Solid Biosciences have gained 7.6% against the industry’s 1% decline.
Image Source: Zacks Investment Research
More on SLDB’s Phase I/II DMD Study for SGT-003
The phase I/II INSPIRE DUCHENNE study is evaluating the safety, tolerability and efficacy of SGT-003, administered as a one-time intravenous infusion in pediatric DMD patients. As of Feb. 11, 2025, six participants have received SGT-003 doses in the INSPIRE DUCHENNE study. Enrollment is currently ongoing, with at least 10 participants expected to be dosed by early in the second quarter of 2025 and around 20 by fourth-quarter 2025.
The initial data readout also showed encouraging early signals of potential cardiac benefit. SGT-003 was well-tolerated in the first six DMD patients dosed. Adverse events were mostly mild to moderate in severity and consistent with those typically observed in AAV gene therapy.
SLDB plans to request an FDA meeting in mid-2025 to discuss a potential accelerated approval pathway for SGT-003 for DMD treatment.
DMD is a progressive and degenerative disorder that leads to weakness and wasting of the body’s muscles. The candidate is being developed using SLDB’s proprietary and rationally designed capsid (AAV-SLB101). The investigational gene therapy already enjoys the FDA’s Fast Track, Rare Pediatric Disease and Orphan Drug designations in the United States for the treatment of DMD.
SLDB’s Other Clinical-Stage Pipeline Candidate
Apart from SGT-003, Solid Biosciences’ clinical-stage pipeline comprises another novel AAV-based gene therapy candidate, SGT-212, which is being developed for the treatment of Friedreich’s ataxia (FA). In January 2025, the FDA cleared SLDB’s investigational new drug (IND) application to begin clinical studies on SGT-212 for the FA indication.
Per the company, SGT-212 is the only full-length frataxin replacement gene therapy candidate that targets the cardiac manifestations of FA. The candidate is being developed to treat the neurologic and systemic clinical manifestations of FA to address the full spectrum of disease progression.