PTC Therapeutics (NASDAQ:PTCT) and exclusive licensee Genentech, a unit of Roche (OTCQX:RHHBY), announce two-year data from Part 1 of the pivotal Phase 2/3 FIREFISH study evaluating Evrysdi (risdiplam) in infants aged 2-7 months with symptomatic Type 1 spinal muscular atrophy (SMA). Infants treated receiving the therapeutic dose of Evrysdi continued to improve and achieve motor milestones. The data were presented virtually at the World Muscle Society Annual Congress.
The exploratory analysis showed that ~88% infants were alive and required no permanent ventilation at year two. 59% (n=10/17) were able to sit without support for at least five seconds, compared to 41% (n=7/17) at year one. 65% (n=11/17) maintained upright head control, compared to 53% (n=9/17) at year one. 29% (n=5/17) could turn themselves over at year two, compared to 12% (n=2/17) at year one, and 30% (n=5/17) were able to stand either supporting weight or with support, compared to 59% (n=10/17) at year one.
All infants (n=14/14) alive at year two were able to swallow, and 93% (n=13/14) were able to feed orally.
No new safety signals were observed. The most common adverse events were pyrexia (fever) (71%), upper respiratory tract infection (52%), cough (33%), vomiting (33%), diarrhea (29%) and respiratory tract infection (29%). The most common serious adverse event was pneumonia (24%).
Related tickers: Biogen (NASDAQ:BIIB), Scholar Rock (NASDAQ:SRRK), Novartis (NYSE:NVS)
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