Positive Phase II SMA data for apitegromab send Scholar Rock soaring

US clinical-stage biotech Scholar Rock (Nasdaq: SRRK) today announced new data from the Phase II TOPAZ trial extension period evaluating patient outcomes after 24-months of treatment, which support sustained and continued improvement with apitegromab (SRK-015) for non-ambulatory patients with Types 2 and 3 SMA receiving a spinal muscular atrophy (SMN) therapy.

The TOPAZ trial data were presented during a podium presentation at the Cure SMA Research & Clinical Care Meeting today, following which Scholar Rock’s shares leapt almost 27% to $6.16.

"The 24-month results provide long-term data and evidence, underscoring the findings of the 12-month primary treatment period of the TOPAZ trial in which patients receiving apitegromab experienced sizable motor function gains,” said Dr George Nomikos, senior vice president of clinical sciences, head of muscle therapeutic area of Scholar Rock. “This durability and continued increase in motor function support the transformative potential of apitegromab for patients suffering with SMA,” he noted.