Solid Biosciences gets FDA Fast Track for FA gene therapy

CHARLESTOWN, Mass. - Solid Biosciences Inc. (NASDAQ:SLDB), a $130.65 million market cap biotechnology company, has been granted Fast Track designation by the U.S. Food and Drug Administration (FDA) for its gene therapy candidate SGT-212, aimed at treating Friedreich's ataxia (FA), a serious genetic neurodegenerative disease. The FDA's decision, announced today, follows the company's previous announcement on January 7, 2025, that it had received FDA Investigational New Drug (IND) clearance for SGT-212. According to InvestingPro analysis, the company currently trades at $3.27 and appears to be slightly undervalued based on Fair Value calculations.

The Fast Track designation is designed to expedite the development and review process for new therapies that treat serious conditions and fill unmet medical needs. With this status, Solid Biosciences will benefit from increased communication with the FDA and may qualify for priority review in the future. InvestingPro data shows the company maintains a strong liquidity position with a current ratio of 7.85 and holds more cash than debt on its balance sheet, though it is currently burning through cash at a notable rate.

SGT-212 is unique in its approach to treating FA as it delivers the full-length frataxin gene through both intradentate nucleus (IDN) and intravenous (IV) infusions. This dual-route delivery is intended to restore therapeutic levels of the frataxin protein, which is deficient in FA patients, potentially halting the progression of the disease's symptoms. These symptoms include neurological issues, heart disease, and other systemic problems, with heart complications being the leading cause of death among FA patients.

Bo Cumbo, President and CEO of Solid Biosciences, expressed optimism that SGT-212 could address the full spectrum of FA symptoms and provide a much-needed treatment option for those affected by this debilitating disease.

The planned Phase 1b trial, set to begin in the second half of 2025, will be a first-in-human study assessing the safety and tolerability of SGT-212 in both non-ambulatory and ambulatory adult patients with FA. Participants will be monitored for five years following treatment.

Jessie Hanrahan, Ph.D., Chief Regulatory Officer at Solid Biosciences, acknowledged the FDA's recognition of the unmet needs within the FA community and the potential of SGT-212 to make a significant impact.

Friedreich's ataxia affects approximately 5,000 people in the United States and 15,000 in Europe. Currently, there are no treatments that can cure or stop the disease's progression.

Solid Biosciences, a company dedicated to precision genetic medicine, is developing a range of gene therapies targeting rare neuromuscular and cardiac diseases. The company is also working on genetic regulators and technologies that could broadly advance gene therapy delivery.

This news article is based on a press release statement from Solid Biosciences Inc.

In other recent news, Solid Biosciences has been the focus of several analyst ratings and strategic partnerships. Truist Securities initiated coverage on the company with a Buy rating and a $16 price target, highlighting the potential upside from its ongoing Phase 1/2 study of SGT-003 in Duchenne muscular dystrophy. JMP Securities also initiated coverage with a Market Outperform rating, recognizing Solid Biosciences' potential in gene therapy and capsid engineering capabilities.

The company recently received approval from the U.S. Food and Drug Administration for its Investigational New Drug application for SGT-212, a gene therapy candidate for Friedreich’s ataxia. Solid Biosciences plans to initiate a Phase 1b clinical trial in the second half of 2025 to assess the safety and tolerability of SGT-212.

In addition, Solid Biosciences has entered into an exclusive collaboration with Mayo Clinic to develop gene therapies targeting genetic cardiac conditions. This partnership grants the company exclusive worldwide licenses to Mayo Clinic's Suppression-Replacement gene therapy platform and several cardiac gene therapy programs. These developments underscore the company's ongoing efforts to advance its gene therapy technologies and potentially commercialize its capsid engineering capabilities.

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