PTC Therapeutics, Inc., a biopharmaceutical company, focuses on the discovery, development, and commercialization of orally administered, small molecule drugs that target post-transcriptional control processes. The company’s lead product is Translarna (ataluren), which is in Phase III clinical trials for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients; and for the treatment of cystic fibrosis caused by nonsense mutations. It also intends to develop Translarna for the treatment of mucopolysaccharidosis type I caused by nonsense mutation, as well as develops spinal muscular atrophy, which is in Phase II clinical program. The company’s preclinical developme...
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PTC Therapeutics Announces Translarna™ Access Program in Duchenne Muscular Dystrophy for Siblings of Patients Participating in PTC Clinical Trials
Mar 3 15
PTC Therapeutics, Inc. announced that it will make Translarna available for siblings of patients in certain open label PTC clinical trials for nonsense mutation Duchenne muscular dystrophy (nmDMD). Translarna will be made available to these siblings in advance of commercial availability in their regions, after a treating physician and the patient or guardian make a clinical decision to use Translarna, and consistent with any other applicable regulatory requirements. Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States. The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA'sOffice of Orphan Products Development; National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.
PTC Therapeutics, Inc. Presents at Barclays Global Healthcare Conference, Mar-10-2015 08:00 AM
Feb 27 15
PTC Therapeutics, Inc. Presents at Barclays Global Healthcare Conference, Mar-10-2015 08:00 AM. Venue: Loews Miami Beach Hotel, 1601 Collins Avenue, Miami, Florida, United States. Speakers: Tuyen Ong, Senior Vice President and Head of Clinical Development & Translational Research.
PTC Therapeutics, Inc. Reports Consolidated Earnings Results for the Fourth Quarter and Full Year Ended December 31, 2014; Provides Financial Guidance for the Full Year 2015
Feb 27 15
PTC Therapeutics, Inc. reported consolidated earnings results for the fourth quarter and full year ended December 31, 2014. For the quarter, the company reported net loss and net loss attributable to common shareholders of $27,269,000 or $0.84 per basic and diluted share on total revenues of $12,658,000 against net loss and net loss attributable to common shareholders of $17,885,000 or $0.75 per basic and diluted share on total revenues of $4,411,000 a year ago. Loss from operations of $32,230,000 and loss from operations before tax benefit of $31,962,000 against loss from operations of $18,092,000 and loss from operations before tax benefit of $17,885,000 a year ago.
For the full year, the company reported net loss and net loss attributable to common shareholders of $93,753,000 or $2.97 per basic and diluted share on total revenues of $25,245,000 against net loss and net loss attributable to common shareholders of $51,574,000 or $5.18 per basic and diluted share on total revenues of $34,696,000 a year ago. Loss from operations of $99,413,000 and loss from operations before tax benefit of $98,446,000 against loss from operations of $45,398,000 and loss from operations before tax benefit of $51,574,000 a year ago.
Operating expense for the full year 2015 is anticipated to be between $160- $170 million, excluding expected non-cash stock-based compensation expense of approximately $30 million, for total operating expenses of approximately $190 million to $200 million. These expenses will be primarily in support of its ongoing and planned confirmatory Phase 3 clinical trials for Translarna in nmDMD and nmCF, commercial launch activities for Translarna in the EU as well as pre-commercial activities in the US, and the continued research and clinical development of other product pipeline candidates.