July 23, 2017 5:07 PM ET


Company Overview of PTC Therapeutics, Inc.

Company Overview

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), for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients; and which is in phase III clinical trials to treat cystic fibrosis caused by nonsense mutations. It also develops Translarna, which is in Phase II clinical trials for the treatment of mucopolysaccharidosis type I caused by nonsense mutation, nonsense mutation aniridia, and nonsense mutation Dravet syndrome/CDKL5; and RG7916 that is in Phase I ...

100 Corporate Court

South Plainfield, NJ 07080

United States

Founded in 1998

298 Employees





Key Executives for PTC Therapeutics, Inc.

Co-Founder, Chief Executive Officer and Executive Director
Age: 57
Total Annual Compensation: $618.0K
Chief Commercial Officer
Age: 54
Total Annual Compensation: $438.2K
Chief Legal Officer, Executive Vice President and Secretary
Age: 56
Total Annual Compensation: $421.5K
Executive Vice President of Research, Pharmaceutical Operations & Technology
Age: 50
Total Annual Compensation: $422.3K
Compensation as of Fiscal Year 2016.

PTC Therapeutics, Inc. Key Developments

PTC Therapeutics, Inc. Announces Preliminary Phase 2 Data from Spinal Muscular Atrophy Program

PTC Therapeutics, Inc. announced the presentation of preliminary clinical data from the company's joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA) at the Cure SMA Meeting in Orlando, FL. Preliminary results from an early analysis of Part 1 of the Phase 2 SUNFISH trial evaluating oral RG7916, a small molecule modifier of Survival Mo tor Neuron 2 (SMN2) splicing, were highlighted in an oral presentation titled "Clinical Studies of RG7916 in Patients with Spinal Muscular Atrophy: Study Update." SMA is a rare genetic disorder that results in neuromuscular disability beginning in infancy and is the leading genetic cause of mortality in infants and young children. Preliminary results from an early analysis from the ongoing Part 1 of the RG7916 SUNFISH study in Type 2/3 SMA patients demonstrated a dose-dependent increase in SMN2 full length/?7 mRNA ratio of ~ 400% versus baseline, as measured in whole blood. These results provided proof of mechanism for oral small molecule SMN2 splicing modifiers. No drug-related adverse events leading to withdrawal have been observed to date for RG7916. SUNFISH is a two-part phase 2 clinical study. Part 1 is a double-blind, placebo-controlled, randomized, exploratory dose-finding study in Type 2/3 pediatric and adult SMA patients. The primary objective of the first part of the study is to evaluate the safety, pharmacokinetics, and pharmacodynamics of RG7916 in patients, and to select the dose for the second part of the study. The pivotal second part is a double-blind, placebo-controlled, randomized, confirmatory study in Type 2/3 SMA patients followed by an open-label extension. The primary objective of the pivotal second part of the study is to evaluate the safety and efficacy of RG7916 compared to placebo. SUNFISH is one of three ongoing clinical trials of RG7916, along with FIREFISH and JEWELFISH. The SMA program was initially developed by PTC Therapeutics in partnership with the SMA Foundation in 2006 to accelerate the development of a treatment for SMA. In November 2011, Roche gained an exclusive worldwide license to the PTC/SMA Foundation SMN2 alternative splicing program. The development of these compounds is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the SMA Foundation.

PTC Therapeutics Announces Update on Nonsense Mutation Dystrophinopathy Treatment from FDA

PTC Therapeutics, Inc. has announced that the FDA has notified the company of the tentative scheduling of a Peripheral and Central Nervous Systems Drugs Advisory Committee meeting on September 28, 2017 to review the new drug application (NDA) for ataluren (Translarna), protein restoration therapy for the treatment of nonsense mutation dystrophinopathy, including nonsense Duchenne muscular dystrophy (nmDMD).

PTC Therapeutics, Inc Announces Executive Promotions

PTC Therapeutics, Inc. announced the appointments of Marcio Souza to Chief Operating Officer and Christine Utter to Principal Financial Officer and Treasurer. Mr. Souza joined PTC in July 2014 as Vice President of Global Marketing and was subsequently Senior Vice President and Head of Global Product Strategy since June 2016, where he was responsible for serving as the chair the integrated program teams as well as leading the global marketing, market access and health economics areas. Utter joined PTC in 2010 as the Assistant Controller and advanced with increasing responsibilities and positions to her most recent position as Senior Vice President, Finance, in January 2017.

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