Solid Biosciences Inc., a firm specializing in precision genetic medicines for
neuromuscular and cardiac diseases, recently shared financial results for the third quarter ending on September 30, 2024. The company also provided updates on its ongoing projects and clinical trials.
Bo Cumbo, the President and CEO of Solid Biosciences, expressed optimism regarding the progress of the INSPIRE DUCHENNE clinical trial. The trial's first three patients have been dosed with
SGT-003, and the treatment has been well tolerated with no severe adverse events reported. The positive early results have led to an expansion of the study protocol, affecting enrollment size, age range, and clinical endpoint timelines. Solid Biosciences has initiated patient enrollment under the updated protocol and plans to continue dosing through the end of 2024 and into 2025. Data from the initial three patients is expected in the first quarter of 2025.
Further development activities include expanding the study protocol in September 2024, activating additional clinical sites in the fourth quarter of 2024, and securing extra manufacturing supply to support the trial's expanded enrollment. These measures aim to accelerate the development of SGT-003 and bring a new treatment option to the Duchenne community.
Gabriel Brooks, M.D., the Chief Medical Officer of Solid Biosciences, provided updates on the company's cardiac pipeline. The IND-enabling studies for SGT-501, aimed at treating
catecholaminergic polymorphic ventricular tachycardia (CPVT), are progressing well. An IND submission for
SGT-501 is anticipated in the first half of 2025. CPVT is a severe
genetic arrhythmia syndrome with high mortality rates, and current treatments have significant side effects. Solid Biosciences aims to transform patient care for this disorder with its gene therapy.
AAV-SLB101, the capsid used in SGT-003, has also shown positive results. It has been well tolerated in the first three patients of the INSPIRE DUCHENNE study and in preclinical studies with non-human primates and mice. Additionally, 13 academic labs and one corporation are utilizing AAV-SLB101 for their research.
The company’s financial status remains robust. As of September 30, 2024, Solid Biosciences held approximately $171.1 million in cash, cash equivalents, and available-for-sale securities. This is an increase from $123.6 million as of December 31, 2023. The company anticipates that its financial resources will support its operations into 2026, covering the expansion of the INSPIRE DUCHENNE clinical trial, additional manufacturing, and other ongoing projects.
Research and development expenses for the third quarter of 2024 were $27.3 million, up from $16.7 million in the same period of 2023. This increase is attributed to higher development program expenses, research costs, and clinical and manufacturing costs for SGT-003 and SGT-501. General and administrative expenses also rose to $7.9 million from $6.4 million, mainly due to increased personnel costs and consulting fees.
The company's net loss for the third quarter of 2024 was $32.7 million, compared to a net loss of $21.0 million for the same period in 2023. The basic and diluted net loss per share was $0.79, down from $1.05 in the third quarter of 2023.
Solid Biosciences remains committed to advancing its pipeline of gene therapy candidates for rare neuromuscular and cardiac diseases. The company's efforts are driven by a focus on improving the lives of patients affected by these devastating conditions.
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