Scholar Rock Reports Apitegromab Data In Spinal Muscular Atrophy

Scholar Rock 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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