- Post hoc analysis of the placebo arm of ACT DMD Data Published in Muscle & Nerve -
SOUTH PLAINFIELD, N.J., July 24, 2018 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced the publication of data in Muscle & Nerve comparing the efficacy and safety of deflazacort and prednisone/prednisolone from the placebo arm of the ACT DMD study. The results demonstrated a clinically differentiated benefit of deflazacort over prednisone/prednisolone in slowing disease progression as measured using physical function endpoints and the time to delay loss of ambulation. Duchenne muscular dystrophy patients treated with deflazacort had notably less decline from baseline in 6-minute walk distance at Week 48 than those treated with prednisone/prednisolone. The extrapolated time to loss of ambulation was 8.58 years for deflazacort and 4.74 years with prednisone/prednisolone.
"This publication supports the benefit of deflazacort in slowing the progression of Duchenne compared to other corticosteroids," said Stuart W. Peltz, Ph.D. Chief Executive Officer of PTC Therapeutics, Inc. "The data indicates that deflazacort should be the standard of care for all patients with Duchenne. The availability of deflazacort, a treatment that has the potential to alter the natural history of Duchenne, supports the need for early diagnosis in patients with this disease."
Study results measuring other clinical endpoints monitoring physical function in Duchenne patients are also consistent with deflazacort having a differentiated clinical benefit over prednisone/prednisolone. The time function tests, such as the 4-stair climb, 4-stair descend, rise from supine, 10-m walk/run, and the North Star Ambulatory Assessment total score notably favored deflazacort over prednisone/prednisolone. Furthermore, the safety profiles for deflazacort and prednisone/prednisolone were generally comparable.
ACT DMD Study Design
The Ataluren Confirmatory Trial in patients with nonsense mutation Duchenne muscular dystrophy (ACT DMD, NCT01826487) study was a randomized, double-blind, placebo-controlled 48-week, phase 3 trial that evaluated ataluren's treatment effect in stabilizing motor function and delaying disease progression in patients with nonsense mutation Duchenne. All patients enrolled in this study had been receiving corticosteroid therapy (deflazacort or prednisone/prednisolone) for ≥6 months at study entry, had no clinically significant change in dosage or dosing regimen for ≥3 months before study entry, and were expected to maintain a stable dose and regimen during the study. The placebo arm consisted of 114 patients, 53 of whom received deflazacort and 61 of whom received prednisone/prednisolone at entry and throughout the study. The study enrolled ambulatory male patients with phenotypic and genotypic confirmation of nonsense mutation Duchenne aged 7-16 years.
About Duchenne Muscular Dystrophy
Primarily affecting males, Duchenne muscular dystrophy (Duchenne) is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of skeletal, diaphragm, and heart muscles. Patients with Duchenne, the more severe form of the disorder, lose the ability to walk as early as age 10 and experience life-threatening lung and heart complications in their late teens and twenties. More information about Duchenne is available through the Muscular Dystrophy Association (www.mdausa.org), Parent Project Muscular Dystrophy (www.parentprojectmd.org), Action Duchenne (www.actionduchenne.org), United Parent Projects Muscular Dystrophy (uppmd.org), Muscular Dystrophy Campaign (www.muscular-dystrophy.org) and AFM (l'Association francaise contre les myopathies), (www.afm-telethon.fr).
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