Shares of biotech company Solid Biosciences (NASDAQ: SLDB) skyrocketed on Feb. 18 after the firm released key clinical data. Solid shares ended the day up nearly 32%. So, what information came out resulting in this massive single-day rise? What potential do Wall Street analysts see in this stock, and can the appreciation continue? I’ll break all that down and provide my perspective on this small company that just saw its value explode.
SGT-003: Solid’s Answer to Duchenne Muscular Dystrophy
Solid’s leading drug candidate is SGT-003. It is a developmental medication that works to treat Duchenne Muscular Dystrophy (DMD). This is a degenerative muscular disorder that primarily affects boys, appearing typically between the ages of two and five years. A genetic mutation causes it, and it worsens progressively over time.
Solid Biosciences is working to create more potent medicines that can be used to treat this debilitating condition. This represents a huge opportunity to improve the lives of the approximately 15,000 people in the United States currently affected. In addition, it represents an opportunity to invest in a company like Solid, hoping to provide a new solution. The Food and Drug Administration (FDA) approved a drug called ELEVIDYS in 2024 that directly competes with Solid’s treatment. The $10 billion pharma company Sarepta Therapeutics (NASDAQ: SRPT) makes ELEVIDYS.
Breaking Down the Results That Sent Solid Shares Soaring
The results came from the company’s biopsy data of three participants in its Phase 1/2 INSPIRE DUCHENNE trial. When deciding whether to approve a drug, the FDA primarily looks at two factors: safety and efficacy. As of February 11, 2025, “SGT-003 has been well tolerated in the six participants dosed, with no serious adverse events observed.” This is important because Solid's past attempts at a DMD treatment were halted over safety concerns.
Efficacy data was also strong. Duchenne Muscular Dystrophy (DMD) is caused by a lack of functional dystrophin, a protein that stabilizes muscle cells. SGT-003 delivers a microdystrophin gene, which is a truncated version of dystrophin designed to reduce muscle deterioration. On average, the three patients produced more microdystrophin than healthy kids produce natural dystrophin. Other data showed comprehensive improvements in muscle strength and resilience. There were also early signs of cardiac benefits.
Breaking Down Solid’s Position and Potential of the Stock
Overall, Solid’s results were highly impressive, both in terms of safety and efficacy. Microdystrophin expression was “the highest mean expression data that has been shared to date of any Duchenne gene therapy." The company also said its medication delivered genetic material to muscles at a level five times higher than ELEVIDYS with a dose 25% lower. These data indicate that the treatment could be safer and more effective. However, seeing functional data is still extremely important.
From a financial perspective, the company has no approved drugs, and thus no revenue. Over the past four quarters, its average outflow in cash from operations has been $23 million. The company has $171 million in cash now. After issuing new shares, it will raise $200 million. So, it could have a total of $371 million in cash. This gives it a runway of four years to keep operating at this pace. However, shareholder dilution is still a significant long-term concern.
Overall, Wall Street analysts still see big upside in this stock. The average of price targets compiled by MarketBeat shows an upside in the shares of nearly 189% as of the Feb. 18 close. Analysts have yet to issue new targets after this trial data release, but it is possible they could raise their expectations significantly.
Solid Biosciences' recent results are highly encouraging for its future. However, the trial still had a very low number of patients. A larger sample size could reveal less robust results. The company aims to provide another update in Q3 with data from 10 to 12 patients. At the same time, it will look to engage the FDA in talks around an accelerated approval for SGT-003. It needs to move quickly with ELEVIDYS well on its way.
The drug brought in $384 million in revenue last quarter. Some questions surround whether ELEVIDYS patients could switch to SGT-003 without issues.
This highlights the huge opportunity Solid has if it succeeds. This is especially true if its treatment is actually safer and more effective. However, betting on the results of clinical trials can be particularly scrupulous and speculative. Shares could fall big on bad data just as they rose on good data. However, if successful, Solid may just be able to achieve the lofty valuation that Wall Street analysts see.
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