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The Outlook for Apitegromab and Its Phase 3 Trial: Yung Chyung, MD

The chief medical officer of Scholar Rock discussed the upcoming phase 3 trial for apitegromab in SMA and whether a successful trial would warrant FDA submission.

"We will continue to look further into the ambulatory type 3 SMA data from the TOPAZ trial as we believe there’s a potential signal there. There’s a lot of work ahead of us, but we’re excited about the progress and look forward to finding updates as they become available.”

Following the completion of the phase 2 TOPAZ clinical trial (NCT039215128), investigators concluded that the results shown for apitegromab (Scholar Rock) in patients with spinal muscular atrophy (SMA) warrant a phase 3 study. Apitegromab, a muscle-directed therapy that serves as a complimentary approach to approved SMN upregulator therapies, was proven to be safe and effective in treating multiple different SMA populations, including ambulatory SMA type 2, ambulatory SMA type 3, and nonambulatory SMA type 3.

In addition to milestones gained throughout the study, the data also suggested that increases in Hammersmith Functional Motor Scale Expanded (HFMSE) scores were attributable to the therapy, as demonstrated by no correlation between duration of prior nusinersen (Spinraza; Biogen) treatment and change in HFMSE. Yung Chyung, MD, chief medical officer, Scholar Rock, claimsthat the company is ecstatic about the progress of apitegromab, and has intentions of expanding its broad range to patients with SMA type 1.

In an interview with NeurologyLive, Chyung provided additional background on the parameters of the phase 3 trial, topline end points being evaluated, and regulatory outlook pending the trial’s success. He also noted that apitegromab has already received regulatory feedback in terms of fast track and prime designations.