Tag Archives: PTC Therapeutics

PTC Therapeutics Announces Phase III Trial of Ataluren

PTC Therapeutics, Inc. (PTC) initiated a Phase III trial of ataluren, an investigational protein restoration therapy for patients with nonsense mutation cystic fibrosis (nmCF). Ataluren is the first investigational drug designed to address the underlying cause of nonsense mutation cystic fibrosis. The primary objective of the registration-directed double-blind, placebo-controlled study is to evaluate whether ataluren can improve lung function, as measured by forced expiratory volume in one second (FEV1), in patients with nmCF. An orally Read More >

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PTC Therapeutics Announces Collaboration with Roche

PTC Therapeutics, Inc. announced a research collaboration and licensing agreement with Roche for development of orally bioavailable small molecules using PTC’s technology, Gene Expression Modulation by Small-molecules (GEMS™). As part of the collaboration, Roche is paying PTC Therapeutics $12 million as part of an agreement to develop small molecule CNS disease drugs. Subject to meeting milestones, there is the potential for PTC to earn up to $239 million in research, development, regulatory and commercial milestone Read More >

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