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Scholar Rock Reports Apitegromab Data In Spinal Muscular Atrophy

Scholar Rock Reports Apitegromab Data In Spinal Muscular Atrophy
  • Scholar Rock (NASDAQ: SRRK) has announced top-line data from the TOPAZ Phase 2 trial evaluating apitegromab (SRK-015) in patients with Type 2 and Type 3 spinal muscular atrophy (SMA).
  • In Cohort 1 (pooled population), the mean change from baseline in Revised Hammersmith Scale (RHS) score was a 0.3-point decline. RHS is an assessment of the physical abilities of SMA patients.
  • Patients in this cohort were treated with apitegromab either as a monotherapy or in conjunction with an approved SMN upregulator therapy (nusinersen).
  • 57% of patients observed maintenance or improvement in RHS score, and 22% of patients achieved at least a 3-point increase in RHS score from baseline.
  • Cohort 2 included patients who were already receiving nusinersen treatment initiated at age five years or older. 
  • Efficacy data demonstrated improvement of motor function from baseline. The mean change from baseline in motor function score was a 0.6-point increase. 64% of patients achieved at least a 1-point increase in the score, and 29% achieved at least a 3-point increase.
  • Potential durability of effect was observed up to 12 months of treatment.
  • Cohort 3 included patients who had initiated nusinersen treatment before five years of age. This cohort's efficacy data demonstrated further motor function improvements relative to what was observed at the six-month interim analysis.
  • The mean change from baseline in HFMSE score was a 7.1-point and a 5.3-point improvement for the 20 mg/kg and 2 mg/kg dose arms, respectively.
  • 59% of patients achieved at least a 5-point increase in motor function score and 35% achieved greater than a 10-point increase over baseline.
  • The Company has identified Becker Muscular Dystrophy (BMD) as the next potential indication for apitegromab. It is planning a proof-of-concept trial to initiate it in 2022.
  • BMD is an X-linked recessive inherited disorder characterized by slowly progressing muscle weakness of the legs and pelvis.
  • Scholar Rock will host a conference call and audio webcast to discuss the apitegromab TOPAZ Phase 2 trial results today at 8:00 a.m. E.T. 
  • Price Action: SRRK shares closed 3.2% lower at $45.13 on Monday.

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Posted-In: Briefs Spinal Muscular AtrophyBiotech News Health Care Small Cap General

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