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February 14, 2016 5:58 AM 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), 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...

100 Corporate Court

South Plainfield, NJ 07080

United States

Founded in 1998

187 Employees





Key Executives for PTC Therapeutics, Inc.

Co-Founder, Chief Executive Officer and Executive Director
Age: 55
Total Annual Compensation: $495.0K
Chief Medical Officer
Age: 65
Total Annual Compensation: $329.3K
Compensation as of Fiscal Year 2014.

PTC Therapeutics, Inc. Key Developments

PTC Therapeutics, Inc. Presents at The LEERINK Partners 5th Annual Global Healthcare Conference, Feb-11-2016 01:25 PM

PTC Therapeutics, Inc. Presents at The LEERINK Partners 5th Annual Global Healthcare Conference, Feb-11-2016 01:25 PM. Venue: The Waldorf Astoria, New York, New York, United States.

PTC Therapeutics, Inc. Announces RG7916, an Additional SMN2 Splicing Modifier Joint Development Program with Roche and the SMA Foundation

PTC Therapeutics, Inc. announced that RG7916, an additional SMN2 splicing modifier from the company's joint development program with Roche and the SMA Foundation in spinal muscular atrophy (SMA), has entered clinical development. RG7916 is designed to shift SMN2 pre-mRNA splicing toward the production of full length SMN mRNA. A Phase 1 study in healthy volunteers has been initiated to investigate the safety, tolerability, pharmacokinetics and pharmacodynamics of RG7916. Two compounds are currently in clinical development within the SMA program, RG7800 and RG7916. The most advanced compound, RG7800, is the subject of a Phase 2 randomized, double-blind, placebo-controlled trial called Moonfish in adult and pediatric patients with SMA. Dosing in the Moonfish trial was suspended in April 2015 and the trial was placed on clinical hold to investigate a non-clinical safety finding observed in a longer-term animal study. 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 RG7800 and RG7916 is being executed by Roche and overseen by a joint steering committee with members from PTC, Roche, and the SMA Foundation.

PTC Therapeutics, Inc. Presents at JPMorgan 34th Annual Healthcare Conference, Jan-13-2016 09:30 AM

PTC Therapeutics, Inc. Presents at JPMorgan 34th Annual Healthcare Conference, Jan-13-2016 09:30 AM. Venue: Westin St. Francis Hotel, 335 Powell Street, San Francisco, California, United States. Speakers: Stuart W. Peltz, Co-Founder, Chief Executive Officer and Executive Director.

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