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Friday, on the stock market, Solid Biosciences (NASDAQ:SLDB), currently trading at $5.50 with a market capitalization of $420 million, maintained its Market Outperform rating and $15.00 price target, as reiterated by JMP analysts. The firm expressed a positive outlook on the company’s Duchenne muscular dystrophy (DMD) therapy, SGT-003, citing recent encouraging clinical data and anticipated regulatory discussions. According to InvestingPro, analyst targets range from $12 to $20, suggesting significant upside potential.
The analyst highlighted that the first-in-human (FiH) SGT-003 data, which was released in February 2025, surpassed expectations. The therapy demonstrated a notable mean microdystrophin expression, high copy numbers, and early signs of improved muscle integrity. InvestingPro data shows the company maintains a strong financial position with more cash than debt and a healthy current ratio of 7.85, providing runway for clinical development. The JMP analyst believes that the expression outcomes in more than ten patients and forthcoming regulatory updates following discussions with the FDA in the second half of 2025 will be pivotal in establishing the therapy’s potential and in clarifying the accelerated approval process.
Additionally, insights from a key opinion leader (KOL) call with Dr. Edward C. Smith, who is closely associated with DMD therapeutic trials, were shared. Dr. Smith expressed his interest in the early yet impressive results from the INSPIRE DUCHENNE study, which investigates the efficacy of SGT-003 in treating DMD.
The continued support from JMP analysts for Solid Biosciences’ stock comes at a crucial time as the company progresses with its clinical and regulatory milestones. With the therapy’s recent performance exceeding initial expectations, the company is poised to engage in further discussions with the FDA to determine the next steps towards potential accelerated approval.
In other recent news, Solid Biosciences has announced encouraging results from its Phase 1/2 INSPIRE DUCHENNE trial, showcasing promising initial clinical data for its gene therapy candidate, SGT-003. The trial reported average microdystrophin expression of 110% among the first three participants, with notable improvements in muscle health biomarkers and potential cardiac benefits. Concurrently, Solid Biosciences has set a $200 million stock and warrant offering, aiming to bolster its financial position. The offering includes shares of common stock and pre-funded warrants, with participation from investors like Adage Capital Partners (WA:CPAP) and Bain Capital Life Sciences.
JMP Securities has maintained its Market Outperform rating for Solid Biosciences, citing the company’s progress in patient dosing and the promising data from SGT-003. Analysts highlighted that the gene therapy demonstrated a significantly higher number of vector copies per nucleus at a low dose compared to competitors. Moreover, Solid Biosciences has received FDA Fast Track designation for its gene therapy candidate SGT-212, aimed at treating Friedreich’s ataxia. This designation is intended to expedite the development and review of therapies for serious conditions with unmet medical needs.
The company plans to begin a Phase 1b trial for SGT-212 in the latter half of 2025, focusing on safety and tolerability in adult patients with Friedreich’s ataxia. Solid Biosciences’ CEO, Bo Cumbo, expressed optimism about the potential of SGT-212 to address the full spectrum of symptoms associated with the disease. These developments reflect Solid Biosciences’ ongoing efforts to advance its pipeline of gene therapy candidates targeting rare diseases.
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