NEW YORK – Solid Biosciences on Wednesday said it will apply to begin human trials of its Duchenne muscular dystrophy gene therapy in the US by year-end.
In reporting Q3 2023 financials, the Charlestown, Massachusetts-based drugmaker said in a statement that it will submit an investigational new drug application with the US Food and Drug Administration in the fourth quarter for the gene therapy SGT-003.
Duchenne muscular dystrophy, characterized by progressive muscle weakness, is caused by DMD mutations that result in a lack of dystrophin protein. SGT-003 is designed to slow or stop progression of the rare and inherited disorder by delivering a synthetic form of a normal DMD gene.
The natural, full-sized DMD gene is too big to fit into an adeno-associated virus vector, and multiple companies are testing gene therapies with their own version of a smaller transgene. Sarepta Therapeutics in June became the first to market a Duchenne muscular dystrophy gene therapy that received accelerated approval from the FDA.
Solid Biosciences doesn't have revenues from any commercialized products yet, and in Q3, the company did not record any revenues from collaborations. The firm spent $16.7 million on R&D in Q3 2023, up 19 percent from $14.0 million in the year-ago quarter. The increase in R&D expense was primarily due to greater study-related costs for SGT-003 and the upcoming IND submission, the company said.
The company reported a net loss of $20.9 million in Q3 2023, or $1.05 per share, compared to a net loss of $20.4 million, or $2.71 per share, during the same period last year. As of Sept. 30, it had roughly $142.9 million in cash, cash equivalents, and available-for-sale securities, which Solid Biosciences expects will fund its strategic priorities into 2025.