Dosing of patients has commenced in TOPAZ, the Phase 2 clinical trial of SRK-015 in Type 2 and Type 3 Spinal Muscular Atrophy (SMA)
Preliminary PK/PD data from a subset of patients are anticipated by year-end 2019
Interim safety and efficacy analysis of a subset of patients with six months of treatment expected in 1H20; top-line results for the full 12-month treatment period are expected beginning 4Q20
CAMBRIDGE, Mass., May 08, 2019 (GLOBE NEWSWIRE) -- Scholar Rock Holding Corporation (NASDAQ: SRRK), a clinical-stage biopharmaceutical company focused on the treatment of serious diseases in which protein growth factors play a fundamental role, today announced the initiation of patient dosing in the Phase 2 clinical trial of SRK-015, a highly specific inhibitor of myostatin activation, in Type 2 and Type 3 Spinal Muscular Atrophy (SMA). All patients will receive SRK-015 once every four weeks either as a monotherapy or in conjunction with an approved SMN upregulator therapy. This Phase 2 trial is supported by interim results from the Phase 1 trial in healthy volunteers that showed favorable safety and tolerability, pharmacodynamic (PD), and pharmacokinetic (PK) results as well as preclinical data that highlight the promising potential of myostatin as a drug target in SMA.
"Despite real advances improving upon the natural history of SMA with therapies that increase levels of the deficient SMN protein, individuals with SMA nonetheless experience a range of weakness from mild to profound," said Thomas Crawford, M.D., Professor of Neurology at Johns Hopkins and Lead Principal Investigator of the TOPAZ trial. "Muscle-directed therapy is the next step towards addressing residual weakness in individuals with SMA. I believe SRK-015 holds real promise to potentially improve their overall motor function."
"The initiation of patient dosing in our Phase 2 TOPAZ study marks an important milestone towards our goal of establishing SRK-015 as the first muscle-directed therapy to help address the functional deficits that continue to affect patients with SMA despite available therapies," said Yung Chyung, M.D., Chief Medical Officer of Scholar Rock. "Results from this Phase 2 trial will further our understanding on the potential clinical benefits of targeting the latent form of myostatin and open up the possibility of investigating other neuromuscular disorders."
TOPAZ Phase 2 Trial Design
The Phase 2 proof-of-concept trial will evaluate the safety and efficacy of SRK-015 dosed intravenously every four weeks (Q4W) over a 12-month treatment period. The trial is anticipated to enroll approximately 55 patients with Type 2 or Type 3 SMA in the U.S., Canada, and Europe across three distinct and parallel cohorts.
Cohort 1 has an open-label, single-arm design and will enroll approximately 20 patients ages 5 through 21 with ambulatory Type 3 SMA. Patients will be treated with 20 mg/kg of SRK-015 Q4W as monotherapy or in conjunction with an approved SMN upregulator therapy. The primary objectives of the cohort are to assess safety and the mean change from baseline in Revised Hammersmith Scale (RHS) over 12 months of treatment. Key secondary assessments include the proportion of patients attaining various thresholds of change from baseline in RHS and change from baseline in 6-minute walk test (6MWT).
Cohort 2 has an open-label, single-arm design and will enroll approximately 15 patients ages 5 through 21 with Type 2 or non-ambulatory Type 3 SMA and who are already receiving treatment with an approved SMN upregulator. Patients will be treated with 20 mg/kg of SRK-015 Q4W in conjunction with an approved SMN upregulator therapy. The primary objectives of the cohort are to assess safety and the mean change from baseline in Hammersmith Functional Motor Scale Expanded (HFMSE) over 12 months of treatment. Key secondary assessments include the proportion of patients attaining various thresholds of change from baseline in HFMSE and change from baseline in Revised Upper Limb Module (RULM).
Cohort 3 has a randomized, double-blind, parallel arm design and will evaluate the effects of SRK-015 in the setting of early intervention with an SMN upregulator therapy. This c...
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