What happened
Shares of Scholar Rock Holding Corporation (SRRK -0.72%) were skyrocketing 74.2% higher as of 10:18 a.m. EDT on Tuesday. The huge jump came after the company announced positive interim results from a phase 2 study evaluating experimental drug SRK-015 in treating patients with type 2 and type 3 spinal muscular atrophy (SMA).
So what
As you might expect from today's big gain for the biotech stock, Scholar Rock's clinical results knocked the socks off investors. The company reported significant motor function improvements for all three treatment cohorts in the phase 2 study of SRK-015 at the six-month mark following treatment.
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The interim analysis found that six months after receiving SRK-015, 67% of all patients achieved an improvement of at least one point in Hammersmith scale scores. Thirty-five percent of patients achieved an increase of at least 3 points in Hammersmith scores. The Hammersmith Functional Motor Scale was the first metric designed to evaluate the motor functioning abilities of individuals with SMA type 2 and type 3.
Thomas Crawford, lead investigator of the study and professor of neurology at the Johns Hopkins University School of Medicine, stated, "These interim results are important because they demonstrate the potential of this muscle-directed approach to improve motor function of individuals with Type 2 and Type 3 SMA."
For Scholar Rock, the interim results from the phase 2 study appear to make it a near slam dunk that SRK-015 will advance into a pivotal study that could eventually lead to regulatory approvals. The results also improve the outlook for Scholar Rock's other pipeline candidates.
Now what
Scholar Rock expects to report top-line data from the 12-month treatment period of the phase 2 study for SRK-015 in the second quarter of 2021. The company should also soon begin discussions with regulatory authorities about advancing the experimental drug into registrational late-stage clinical testing.
