New data further support efficacy of Scholar Rock's apitegromab for SMA patients

News coming out of Cure SMA meeting on new 36-month extension data from US biotech Scholar Rock’s (Nasdaq: SRRK) Phase II TOPAZ clinical trial reinforces long-term substantial and sustained improvement of motor function in patients with non-ambulatory spinal muscular atrophy (SMA) treated with apitegromab.

Scholar Rock, whose shares rose 10% to $7.54 on Friday, said these data showed that continued treatment with apitegromab over the extended treatment period was associated with substantial and sustained improvement in motor function, as well as improvements in patient-reported outcome measures in patients with non-ambulatory Types 2 and 3 spinal muscular atrophy (SMA) receiving survival motor neuron (SMN)-targeted therapy.

Detailed results were presented by Dr Thomas Crawford, of Johns Hopkins Medicine, and the lead principal investigator of the TOPAZ trial, during two podium presentations at the Cure SMA Research & Clinical Care Meeting in Orlando, Florida, USA.