Scholar Rock stock catapulted by triple digits Monday after its spinal muscular atrophy treatment succeeded in a Phase 3 study.
The company tested a drug called apitegromab in patients with the muscle-wasting disease. After a year, patients age 2 to 12 showed an average 1.8-point improvement on a 66-point scale measuring motor symptoms compared with patients who received standard treatments.
Nearly a third of patients, 30.4%, had a greater than 3-point improvement. Just 12.5% of patients given standard drugs had the same improvement.
Improvements in motor function were apparent as early as eight weeks into treatment. The benefits were consistent across all age groups, from age 2 to 21, and appeared to widen over time, Wedbush analyst David Nierengarten said in a report. Nearly all patients, 98%, continued onto an open-label study. In open-label studies, all patients receive the experimental treatment.
Scholar Rock shares launched 362% to 34.28. That put shares at their highest point since November 2021. The news also buoyed shares of Biohaven, which is working on a similar treatment option for a broader group of SMA patients. Biohaven stock jumped 10.8% to 52.78.
Scholar Rock Could Soon Ask For Approval
Scholar Rock's drug works by blocking myostatin, a protein that inhibits muscle growth. Biohaven is also testing an anti-myostatin drug in patients with spinal muscular atrophy and in obesity treatment.
But Piper Sandler analyst Christopher Raymond noted Biohaven is using a slightly different mechanism to tackle a broader population of patients with SMA.
"While this is a major win for the anti-myostatin field and the SMA community writ large, the specific read-through to Biohaven's Phase 3 (study) Resilient readout for (Biohaven's drug) taldefgrobep alfa is not straightforward," he said in a report.
For Scholar Rock, the Phase 3 success means the company could potentially ask for Food and Drug Administration approval in the first quarter, Raymond said. This would be Scholar Rock's first commercial product. Wedbush's Nierengarten projects $70 million to $80 million in 2026 sales, $300 million to $315 million in 2027, and $550 million to $575 million in 2028.
That opportunity could be even bigger pending the outcome of Scholar Rock's study called Opal, which is testing the drug in patients younger than age 2. The company is also considering expanding into other neuromuscular conditions. Nierengarten has an outperform rating on Scholar Rock stock.
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