Roche Reports Positive Evrysdi Data From Dose-Finding Part Of Spinal Muscular Atrophy Trial

Roche Holding AG (OTC:RHHBY) has reported positive data from the dose-finding Part 1 FIREFISH Phase 2/3 study of Evrysdi (risdiplam) in infants with symptomatic Type 1 spinal muscular atrophy (SMA).
Evrysdi helps in the survival of motor neuron 2 (SMN2) splicing modifier designed for treating SMA by boosting SMN protein production. The protein is vital to maintaining healthy motor neurons and movement.
According to the latest data from 21 infant participants, Evrysdi treatment at 12 months helped 90% of infants survive without needing permanent ventilation, and 33% sit without support for at least five seconds.
Furthermore, it raised the survival levels of SMN protein by a median 1.9-fold from baseline in the high-dose group at 12 months.
In the low- and high-dose cohorts, no infant lost the ability to swallow over 12 months, and 86% were able to feed orally, either exclusively or in combination with a feeding tube at 12 months.
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