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CHARLESTOWN, Mass. - Solid Biosciences Inc. (NASDAQ:SLDB), a $405 million market cap biotechnology company whose stock has gained over 21% in the past six months, announced today that the U.S. Food and Drug Administration (FDA) has approved its Investigational New Drug (IND) application for SGT-501, a gene therapy targeting catecholaminergic polymorphic ventricular tachycardia (CPVT). According to InvestingPro data, analysts have set price targets ranging from $10 to $20, suggesting significant upside potential from current levels. Health Canada has also approved the company’s clinical trial application for the therapy.
SGT-501 is designed to treat CPVT, a rare genetic heart condition that can cause life-threatening abnormal heart rhythms triggered by physical activity or emotional stress. The condition affects approximately 1 in 10,000 people globally and currently has no FDA-approved therapies specifically addressing its underlying causes. With a strong balance sheet showing more cash than debt and a healthy current ratio of 10.89, Solid Biosciences appears well-positioned to advance its clinical programs.
The company plans to initiate a Phase 1b clinical trial in the fourth quarter of 2025 to evaluate the safety, tolerability, and efficacy of SGT-501.
"Despite being identified nearly 50 years ago, CPVT still lacks FDA-approved therapies," said Gabriel Brooks, Chief Medical Officer of Solid Biosciences, in the press release.
SGT-501 is an AAV-based gene therapy designed to deliver a functional copy of the human cardiac calsequestrin gene to heart muscle cells. The therapy has received both Orphan Drug Designation and Rare Pediatric Disease Designation from the FDA.
The gene therapy approach was initially developed by Dr. Silvia Priori and researchers at the IRCCS ICS Maugeri lab in Italy. Solid Biosciences licensed the technology in 2023 and has since advanced it to clinical development.
CPVT is primarily caused by mutations in the RYR2 and CASQ2 genes, which disrupt calcium regulation in heart muscle cells. The condition is often diagnosed in children and young adults and can result in fainting, seizures, cardiac arrest, and sudden death.
This development expands Solid Biosciences’ pipeline beyond its existing neuromuscular disease programs to include cardiac indications. While the company maintains a "FAIR" overall financial health score according to InvestingPro, investors should note its rapid cash burn rate. For deeper insights into Solid Biosciences’ financial health and growth prospects, including exclusive ProTips and comprehensive analysis, explore the detailed Pro Research Report available on InvestingPro, covering over 1,400 US stocks.
In other recent news, Solid Biosciences has been highlighted by several research firms for its developments in gene therapy for Duchenne muscular dystrophy (DMD). Citi initiated coverage with a buy rating and a $14 price target, citing the company’s promising position in the DMD market and the potential of its SGT-003 gene therapy. The firm noted that SGT-003 does not show liver toxicity, a significant advantage over competitors. Meanwhile, Cantor Fitzgerald maintained its Overweight rating with a $16 price target, expressing optimism following discussions with Solid Biosciences’ management about early Phase 1/2 data from SGT-003. H.C. Wainwright also reaffirmed its Buy rating, emphasizing the favorable safety profile of SGT-003 compared to Sarepta Therapeutics’ Elevidys, which has faced safety concerns. Truist Securities reiterated a Buy rating with a $16 price target, noting progress in Solid Biosciences’ gene therapy programs for DMD and other conditions. Additionally, Piper Sandler adjusted its price target to $17 from $20, maintaining an Overweight rating, following Solid Biosciences’ first-quarter financial results and business updates. The company is actively advancing its clinical programs, with significant milestones expected in the near future.
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