Solid Biosciences Inc., a life sciences company specializing in precision genetic medicines for neuromuscular and
cardiac disorders, announced its financial results for the second quarter ending June 30, 2024, alongside a comprehensive business update.
CEO Bo Cumbo emphasized the company's achievement in starting patient dosing for the Phase 1/2 INSPIRE DUCHENNE trial of
SGT-003, a gene therapy candidate for
Duchenne muscular dystrophy. Cumbo noted that the treatment has been well-received by patients so far, with no serious adverse events reported. The company anticipates revealing initial safety and functional data by the end of 2024.
Chief Medical Officer Gabriel Brooks, M.D., highlighted that the treatment's safety data aligns with preclinical studies, and the immunosuppression achieved with steroids alone is promising. Brooks elaborated on the novel capsid, AAV-SLB101, which is designed to target and transduce muscle tissue more effectively. The specific capsid is not only integral to SGT-003 but also pivotal for future therapies, emphasizing the need for improved treatment options for Duchenne patients.
Solid Biosciences plans to expand the trial with additional clinical sites in the U.S., Canada, and Europe. They aim to initiate six sites by Q4 2024, including one in Canada, with further European sites expected to be operational in the first half of 2025. The expansion aligns with the company's commitment to delivering next-generation treatments globally.
Beyond Duchenne, the company is progressing with
SGT-501, targeting
catecholaminergic polymorphic ventricular tachycardia (CPVT), with plans to submit an Investigational New Drug (IND) application in the first half of 2025. Solid Biosciences continues to advance its early-stage drug candidates, incorporating the proprietary capsid AAV-SLB101 into its
BAG3 and TNNT2 cardiac programs. This strategic move leverages the company's clinical and manufacturing expertise across its pipeline.
Financially, Solid Biosciences ended Q2 2024 with approximately $190.3 million in cash, cash equivalents, and available-for-sale securities, providing a runway into 2026. R&D expenses for the quarter were $19.5 million, a slight decrease from the previous year, primarily due to reduced manufacturing and research costs for SGT-003. However, these were offset by increased costs related to SGT-501 and other product candidates. General and administrative expenses rose to $8.3 million, driven by higher legal fees and recruitment costs. The net loss for the quarter was $25.1 million, marginally higher than the $24.6 million loss in Q2 2023.
Solid Biosciences also reported the broader adoption of its proprietary capsid AAV-SLB101, with ten academic labs and one company utilizing it for various studies. The company's inclusion in the Russell 3000® Index on July 1, 2024, reflects its growing prominence in the financial community.
The company's presentations at the 2024 American Society of Gene and Cell Therapy (ASGCT) Annual Meeting highlighted advancements in AAV manufacturing, purification, and non-clinical data for SGT-003.
Overall, Solid Biosciences is strategically positioned to advance its diverse pipeline of gene therapy candidates, focusing on significant unmet needs in neuromuscular and cardiac diseases. With a robust financial position and ongoing clinical and preclinical developments, the company is poised to make transformative strides in precision genetic medicine.
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