Benzinga | Jul 28, 2021 05:01PM EDT

PTC Therapeutics Reports Results From Part 2 Of FIREFISH Study Published In New England Journal Of Medicine

PTC Therapeutics, Inc. (NASDAQ:PTCT) today announced that results from Part 2 of the FIREFISH pivotal study were published in the New England Journal of Medicine. This trial showed that symptomatic Type 1 spinal muscular atrophy (SMA) infants treated with Evrysdi(tm) (risdiplam) showed significant improvements in survival, motor milestones, and motor function compared to historical controls.

"It is exciting to see such profound results in infants treated with Evrysdi," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "We are proud that such a significant treatment for SMA patients was developed from PTC's splicing platform. We believe that the splicing platform is a disruptive technology that will continue to drive the development of new therapies in diseases with no treatment options available."

The table below depicts the primary and secondary endpoints achieved across Part 2 of the FIREFISH study after 12 months of treatment:

Percentage of Infants Achieving Milestones Historical Milestone Achievement After Controls 12 months with Evrysdi

Ability to sit without support for 29% (12/41) 5% at least five seconds

CHOP-INTEND* score of 40 or 56% (23/41) 17% more

An increase of at least 4 points in the CHOP-INTEND total score 90% (37/41) 17% from baseline

HINE-2** motor-milestone 78% (32/41) 12% responders

Event free survival? 85% (35/41) 42%

FIREFISH is an open-label, two-part, pivotal clinical trial designed to evaluate the efficacy, safety, tolerability, pharmacokinetics and pharmacodynamics in infants with type 1 SMA. In the dose-finding Part 1 of the study, treatment with Evrysdi led to an increased expression of SMN protein levels. Part 2 evaluated the efficacy and safety of Evrysdi in Type 1 SMA.

Roche leads the clinical development of Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics. Evrysdi is marketed in the United States by Genentech, a member of the Roche Group.

* CHOP-INTEND, Children's Hospital of Philadelphia Infant Test of Neuromuscular Disorders

** HINE-2, Hammersmith Infant Neurological Examination

? Event-free survival is defined as alive without need for permanent ventilation (defined as requiring tracheostomy or ventilation for ?16 hours per day continuously for >3 weeks or continuous intubation >3 weeks, in the absence of, or following the resolution of, an acute reversible event). Data cut-off: 14 Nov 2019.