PTC Therapeutics

PTC, OSIsoft to accelerate industrial internet of things solution development

Tuesday, March 21, 2017

PTC and OSIsoft announced their intention to integrate the ThingWorx Industrial Internet of Things (IoT) Platform with the OSIsoft PI System to enable faster development of IoT solutions from connected data sources. As planned, the integration would allow customers using the OSIsoft PI System data infrastructure to easily incorporate capabilities from ThingWorx—including application enablement, machine learning, industrial connectivity, and augmented reality—and access to applications on the ThingWorx Marketplace to more rapidly advance IoT initiatives and enable customers to expand and accelerate their return on investment.

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PTC Therapeutics to acquire Emflaza for Duchenne muscular dystrophy

Thursday, March 16, 2017

PTC Therapeutics has entered into an asset purchase agreement with Marathon Pharmaceuticals to acquire all rights to Emflaza (deflazacort). Emflaza is the first treatment approved in the U.S. for all Duchenne muscular dystrophy (DMD) patients five years and older, regardless of their genetic mutation. DMD is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death due to heart and respiratory failure. Emflaza aligns with PTC’s mission to bring therapies to patients who have rare diseases with limited or no treatment options.

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Compassionate use and the impact on clinical research

Monday, April 11, 2016

The Pulse on Global Trials by Matthew Howes

Requests for compassionate use of drugs not yet approved by the FDA have been making headlines, and experts predict this trend will continue. Last year, the FDA received 1,262 expanded access requests from drug companies, on behalf of patients, to use unapproved drugs outside of clinical trial programs.

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PTC Therapeutics cuts workforce 18%

Thursday, March 24, 2016

PTC Therapeutics is reducing its workforce by approximately 18%, which will primarily affect employees and contractors in the U.S. This reduction is part of PTC’s program intended to optimally manage operating expenses following its recent setback related to the Refuse to File letter received from the FDA for Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). 

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PTC Therapeutics’ cancer stem cell program targeting BMI1 enters phase I

Thursday, April 30, 2015

PTC Therapeutics has announced that its oncology program targeting BMI1, a protein linked to drug-resistant cancers, has entered a phase I study in patients with advanced solid tumors. The open-label, first-in-human study will investigate the safety and pharmacokinetics of PTC596, an orally available small molecule. PTC’s BMI1 program is supported by a collaboration with the Wellcome Trust.

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PTC Therapeutics, University of Pennsylvania collaborate on orphan disease research

Thursday, March 19, 2015

PTC Therapeutics, a global biopharmaceutical company, has inked a collaboration with the Orphan Disease Center at the Perelman School of Medicine at the University of Pennsylvania focused on translational research for discovering and developing new treatments for orphan disorders. The partnership reflects both parties’ commitment to developing treatments and improving the lives of those who suffer from rare diseases.

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Spinal muscular atrophy program enters phase Ib/IIa trial in patients with SMA

Thursday, November 20, 2014

PTC Therapeutics’ joint development program in spinal muscular atrophy (SMA) with Roche and the SMA Foundation (SMAF) has started a phase Ib/IIa study in adult and pediatric patients. The placebo-controlled, randomized, multiple-dose study will enroll approximately 48 patients with SMA and investigate the safety and tolerability of an investigational survival of motor neuron 2 (SMN2) gene splicing modifier (RG7800) over 12 weeks. The achievement of this milestone triggers a $10 million payment to PTC from Roche.

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PTC Therapeutics expands management team

Monday, January 27, 2014

PTC Therapeutics has expanded its senior management team with the appointment of Robert J. Spiegel, M.D., FACP, to chief medical officer and Murad Husain to vice president, Global Regulatory Affairs. PTC also announced the promotions of Joseph Colacino, Ph.D., to senior vice president, Drug Discovery and Ernest R. Dietel to senior vice president, Quality. In addition, Jay Barth, M.D., senior vice president of Clinical Development, is leaving PTC to pursue other opportunities.

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PTC Therapeutics, the SMA Foundation and Roche collaborate

Friday, January 24, 2014

PTC Therapeutics, the SMA Foundation and Roche have announced that their joint research program in Spinal Muscular Atrophy (SMA) has entered the first stage of clinical development aiming to assess safety and tolerability of an oral compound in healthy volunteers. SMA is a genetic neuromuscular disorder that leads to muscle weakness and mobility impairment and is considered the leading genetic cause of death in infants and toddlers.

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