Following a crucial regulatory milestone, Solid Biosciences Inc. (NASDAQ: SLDB) saw a notable increase in the value of its shares. Tuesday’s after-hours trade saw SLDB shares increase 17.35% to $4.60. The spike was propelled by the FDA’s approval of Investigational New Drug or IND application for SGT-212, the company’s new gene therapy candidate that targets Friedreich’s ataxia (FA).
Novel Gene Therapy
Friedreich’s ataxia is a degenerative, multisystem illness brought on by low levels of the frataxin protein. Solid Biosciences’ SGT-212 is a novel treatment for this condition. The treatment uses an AAV-based delivery method that may be administered directly into the cerebellum via intradentate nuclei (IDN) or systemically by intravenous (IV) infusion. This dual-targeting strategy aims to address both the neurological and systemic manifestations of FA, offering a comprehensive solution for disease progression.
Friedreich’s ataxia presents unique challenges for drug development due to the need for precise frataxin expression levels to prevent cardiac toxicity and targeted delivery to cerebellar tissues for neurological benefits. SGT-212 stands out as the only candidate addressing both cardiac and neurological complications by leveraging its dual-route delivery system.
Preclinical Success and a Differentiated Approach
Solid Biosciences’ preclinical studies have demonstrated the potential of SGT-212 to safely deliver frataxin to critical tissues. The results showed significant improvements in neurological function and reversal of cardiac symptoms in animal models. SLDB’s extensive research, including long-term non-human primate studies, supports its belief that targeting multiple systems via dual administration is the most effective strategy for addressing FA’s diverse impacts.
Clinical Trials on the Horizon
Solid Biosciences is in further plans to launch a first-in-human Phase 1b clinical trial of SGT-212 in the latter half of 2025. This open-label, dose-finding study will include both ambulatory and non-ambulatory adult patients with FA across up to three cohorts. The trial will evaluate the safety and tolerability of the dual-administration approach, with participants being monitored for up to five years post-treatment.
Solid Biosciences (SLDB) also announced a conference call scheduled for today before the market opens, aiming to provide further details on this milestone and its broader pipeline developments.