Preliminary results at 6 months of the TOPAZ trial evaluating SRK-015 – a myostatin inhibitor – in 48 participants with type II and III SMA have just been communicated: SRK-015 was well tolerated and improves participants’ motor function with a dose-dependent effect.
TOPAZ trial
This trial takes place in the United States and Europe (but not France). Participants receive SRK-015 intravenously every 4 weeks for one year. They are divided into 3 cohorts:
- one of 23 participants aged 7 to 21, with type III SMA, walking, with or without treatment increasing the production of SMN (nusinersen) and treated with 20 mg / kg of SRK-015;
- one of 15 participants aged 8 to 19, with type II or type III SMA, non-ambulant, on nusinersen and also treated with 20 mg / kg of SRK-015;
- one of 20 children aged 2 to 6 years, with type II SMA, who started treatment with nusinersen before the age of 5 years, for double-blind evaluation of two doses of SRK-015 (2 mg / kg or 20 mg / kg).
This one-year trial is expected to end in January 2021. SRK-015 could be the first treatment to be taken in addition to those currently on the market and affecting the production of SMN, to improve motor function.
Press release October 27, 2020 « Scholar Rock Announces Positive Proof-of-Concept Data from TOPAZ Phase 2 Trial Interim Analysis of SRK-015 in Patients with Type 2 and Type 3 Spinal Muscular Atrophy »
TOPAZ trial on Clinicaltrials.gov: NCT03921528
