Benzinga | Apr 6, 2021 07:48AM EDT

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.