Scholar Rock Announces Completion of Dosing of First Cohort in Phase 1 Clinical Trial of SRK-015 for SMA

The placebo-controlled, double-blind Phase 1 clinical trial will evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of single- and multiple-ascending doses of intravenous SRK-015 in healthy adult volunteers. Assuming the successful completion of the Phase 1 trial, the Company plans to initiate a Phase 2 proof-of-concept trial in the first quarter of 2019 to evaluate the efficacy and safety of SRK-015 in patients with later-onset SMA as a monotherapy or in conjunction with an approved SMN upregulator therapy

The initiation of this Phase 1 clinical trial of SRK-015 is a momentous milestone for Scholar Rock an they step closer to potentially bringing the first muscle-directed therapy to patients suffering from SMA,”

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About SRK-015
SRK-015 is a selective inhibitor of the activation of myostatin and is an investigational therapy for the treatment of patients with spinal muscular atrophy (SMA). Myostatin, a member of the TGF-beta superfamily of growth factors, is expressed primarily in skeletal muscle cells and the absence of its gene is associated with an increase in muscle mass and strength in multiple animal species. Scholar Rock believes the inhibition of the activation of myostatin with SRK-015 may promote a clinically meaningful increase in muscle mass and strength. A Phase 1 clinical trial in healthy volunteers is ongoing.

The U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SRK-015 for the treatment of SMA. The effectiveness and safety of SRK-015 have not been established and SRK-015 has not been approved by the FDA or any other regulatory agency.

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