Voici le courriel que je viens de recevoir de la Société Américaine PTC Thérapeutique qui pilote l'essai thérapeutique du PTC124 ou Ataluren et ce, pour les dystrophies musculaires de Duchenne et Becker, mais aussi pour la Fibrose Kystique (Mucoviscidose). Ce document est en anglais, mais je vais essayer de vous traduire les grandes lignes :

La firme nous annonce ces résultats préliminaires pour l'essai thérapeutique en phase 2b qui concerne les patients atteints de DMD et DMB avec une mutation non-sens. Ces premiers résultats ne sont pas trés significatifs sur une durée de 48 semaines, cependant, il ressort que les patients tolérent bien le traitement.

D'autres analyses sont en cours sur des sous-groupes de patients. Ces résultats demontrent que les essais peuvent se poursuivre en raison du profil sécuritaire de l'Ataluren.

Ces essais fournissent d'importantes informations et des données sur l'Ataluren et les dystrophies de Duchenne et de Becker qui seront utiles ultérieurement et pour de nombreuses autres analyses.

PTC THERAPEUTICS AND GENZYME CORPORATION ANNOUNCE PRELIMINARY RESULTS FROM THE PHASE 2B CLINICAL TRIAL OF ATALUREN FOR NONSENSE MUTATION DUCHENNE/BECKER MUSCULAR DYSTROPHY

SOUTH PLAINFIELD, NJ and CAMBRIDGE, MA – March 03, 2010 – PTC Therapeutics, Inc. and Genzyme Corporation (Nasdaq: GENZ) today announced preliminary results from the Phase 2b clinical trial of ataluren, an investigational new drug, in patients with nonsense mutation Duchenne/Becker Muscular Dystrophy (nmDBMD). The primary endpoint of change in 6-minute walk distance did not reach statistical significance within the 48 week duration of the study. Study results showed that ataluren was well tolerated and no clinical trial patients discontinued treatment due to an adverse event. Additional efficacy analyses are underway in patient subgroups.

"These results further demonstrate the safety profile of ataluren and support continued development," remarked Langdon Miller, M.D., PTC's chief medical officer. "DBMD is a progressive, debilitating, and life-threatening neuromuscular disorder. The variability of symptom onset, disease progression, and 6-minute walk distance creates challenges for clinical development. Importantly, this trial does provide a wealth of valuable data about ataluren and DBMD. Additional analyses will guide the overall clinical and regulatory path forward."

"PTC has a longstanding commitment to discovering and developing new treatments for DBMD and we will continue to collaborate with patients, investigators, and DBMD advocacy groups to advance these efforts," stated Stuart Peltz, Ph.D., president and chief executive officer of PTC Therapeutics. "Data from this Phase 2b study will guide further development of ataluren in nonsense mutation genetic disorders."

"The quality of the data from this well-conducted study and additional analyses will help to inform the clinical development of ataluren in other indications," stated Geoffrey McDonough, M.D., senior vice president and general manager of Genetic Diseases at Genzyme. "We are committed to the development of ataluren and will continue to collaborate with PTC to advance its development for the treatment of genetic disorders."

Ataluren is also currently being investigated for use in patients with nonsense mutation cystic fibrosis in a Phase 3 study and nonsense mutation hemophilia A and B in a Phase 2a study. Ataluren's mechanism of action offers the potential to address multiple genetic disorders with various pathophysiologies and disease manifestations.

ABOUT THE PHASE 2B CLINICAL TRIAL
The randomized, double-blind, placebo-controlled Phase 2b trial was designed to evaluate the safety and efficacy of 48 weeks of ataluren therapy in patients with nmDBMD. The study enrolled 174 participants at 37 sites in North America, Europe, Australia, and Israel. Participants were randomized to receive either a low dose of ataluren (10 mg/kg in the morning, 10 mg/kg at midday, and 20 mg/kg in the evening), a high dose of ataluren (20 mg/kg in the morning, 20 mg/kg at midday, and 40 mg/kg in the evening), or placebo (inactive drug in the morning, at midday, and in the evening). The primary outcome measure was the total distance walked during a 6-minute walk test, a standardized test of ambulation. Other outcome measures in the study evaluated activity at home, muscle and heart function, strength, cognitive ability, muscle integrity, and muscle dystrophin expression. Safety parameters, compliance, and ataluren blood levels were also monitored.

Résultats essai phase 2b PTC 124

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