Spinal Muscular Atrophy
The TOPAZ Phase 2 proof of concept trial evaluates the safety and efficacy of apitegromab (SRK-015) in patients with Type 2 and Type 3 SMA.
The trial enrolled 58 patients between the ages of 2 and 21 in the U.S. and Europe across three distinct cohorts. All patients are receiving apitegromab once every four weeks either as a monotherapy or in conjunction with an approved SMN upregulator treatment over a 12-month treatment period. The primary efficacy endpoints measure motor function through clinically meaningful outcome measures validated in SMA, such as the Hammersmith Functional Motor Scale Expanded (HFMSE) in non-ambulatory SMA and the Revised Hammersmith Scale (RHS) in ambulatory SMA.
A pre-planned interim analysis was conducted following a six-month treatment period, and positive proof-of-concept data were announced in October 2020. Top-line results for the full 12-month treatment period are anticipated in the second quarter of 2021.
The DRAGON Phase 1 open-label, dose escalation and dose expansion clinical trial is evaluating the safety, tolerability, pharmacokinetics (PK), pharmacodynamics (PD), and efficacy of SRK-181 in patients with locally advanced or metastatic solid tumors.
The two-part trial consists of a dose escalation portion (Part A) for SRK-181 as both a single agent and in combination with approved anti-PD-(L)1 therapy, followed by a dose expansion portion (Part B) evaluating SRK-181 in combination with approved anti-PD-(L)1 therapy in multiple tumor-specific cohorts.
An update on dose escalation of SRK-181 is anticipated in the fourth quarter of 2020. Clinical response and safety data are anticipated in the second half of 2021.
Patients seeking more information on potential clinical trials should contact their physician.