Apitegromab’s Potential in Treating SMA: Yung Chyung, MD


The chief medical officer of Scholar Rock discussed topline results from the phase 2 TOPAZ study of apitegromab in patients with spinal muscular atrophy.

“All in all, we are quite enthusiastic about advancing our program of apitegromab and further investigating the safety and efficacy of this molecule towards our aspiration of transforming the care of patients with SMA.”

Data from the phase 2 TOPAZ clinical trial (NCT03921528) presented at the 2021 Cure SMA Research & Clinical Care Meeting, June 9-11, showed that treatment with apitegromab (SRK-015; Scholar Rock) further demonstrated proof-of-concept in patients with spinal muscular atrophy (SMA) types 2 and 3. A majority of the 23 patients (57%; n = 13) with ambulatory type 3 SMA who received a 20-mg/kg dose monotherapy of apitegromab in conjunction with nusinersen (Spinraza; Biogen) across cohort 1 maintained or improved their motor function, as reflected by a greater than 0-point change from baseline in Revised Hammersmith Scale.

No new safety signals for apitegromab were identified in the 12-month topline analysis, with headache, pyrexia, upper respiratory tract infection, cough, and nasopharyngitis rounding out the most frequently reported treatment-emergent adverse events. Findings from the study helped advance the drug into a pivotal phase 3 trial, which is expected to launch by the end of 2021.

Yung Chyung, MD, chief medical officer, Scholar Rock, sat down with NeurologyLive to provide more insight on the results, as well as to discuss why there is palpable buzz surrounding the drug and its clinical pathway.

Scholar Rock announces positive 12-month top-line results from the TOPAZ phase 2 clinical trial evaluating apitegromab in patients with type 2 and type 3 spinal muscular atrophy (SMA). News release. April 6, 2021. Accessed July 1, 2021. https://investors.scholarrock.com/news-releases/news-release-details/scholar-rock-announces-positive-12-month-top-line-results-topaz
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