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DMD Race For First FDA Approved Drug: PTC Therapeutics Business Developments

June 8, 2013

Duchenne Muscular Dystrophy (DMD) has attracted a lot of interest from investors and the media in the past year. Three companies and their orphan drugs are now in a race to gain the first approval drug for DMD :

•   PTC Therapeutics’ Ataluren
•   GlaxoSmithKline/Prosensa’s Drisapersen
•   Sarepta Therapeutics’ Eteplirsen.

This is the first in this week’s three part series on recent business developments for the three competitors in the DMD space.

PTC Therapeutics, a New Jersey pharmaceutical company, is developing orphan drug Ataluren for DMD.  Ataluren, as described in a recent journal article in Expert Opinion on Orphan Drugs (see References below), is a “stop codon read-through …. gene therapy that allows cells to …. read-through or ignore certain types of mutations called nonsense mutations …. Ataluren introduces a nucleotide sequence at the transfer RNA acceptor site, allowing the translational machinery to continue.” Up to 20% of DMD patients have a premature stop codon.

Ataluren is in clinical trials for DMD, Becker Muscular Dystrophy (BMD), and Cystic Fibrosis (CF). The FDA granted Ataluren fast track and orphan drug designations for DMD and CF, due to nonsense mutations. The European Commission gave Ataluren orphan designation for CF, DMD, and BMD. The European Medicines Agency (EMA) in December 2012, accepts for review a Marketing Authorization Application (MAA) from PTC Therapeutics seeking conditional approval for Ataluren for nonsense mutation DMD.

On May 16, 2013, PTC Therapeutics announces that it has filed a registration statement on Form S-1 with the US Securities and Exchange Commission (SEC), relating to the proposed IPO of its common stock. J.P. Morgan and Credit Suisse are acting as joint book-running managers for the offering. PTC Therapeutics wants to list its shares on the NASDAQ under the symbol “PTCT”. On June 5, 2013, PTC Therapeutics announces terms for its IPO. The company plans to raise $100 million by offering 6.9 million shares at a price range of $13 – $16.  At the $14.50 midpoint of the proposed range, the company would have a market value of $322 million.

PTC Therapeutics announces on June 3, 2013, the appointment of Shane Kovacs as Chief Financial Officer (CFO), reporting to Stuart W. Peltz, Ph.D., CEO of the company. Mr. Kovacs will be responsible for all finance-related activities at the company. Prior to joining PTC Therapeutics, Mr. Kovacs is a Managing Director at Credit Suisse, focusing on advising life sciences companies.

PTC Therapeutics’ large multinational, randomized, double-blinded, and placebo-controlled, Phase III trial for Ataluren for non-sense-mutation Duchenne or Becker MD, opens its first site at Cincinnati Children’s Hospital Medical Center, in Cincinnati, Ohio. It is estimated that enrollment is to be completed by mid-2014. There are to be more than 50 trial sites in at least 18 different countries. For full details about this Phase III trial, please reference PTC Therapeutics’ “Frequently Asked Questions (FAQ)” Document. According to the PTC Therapeutics’ FAQ document :

The main goals of the Phase 3 study are to confirm the safety and efficacy of Ataluren in nonsense mutation DMD (nmDMD). Efficacy will be determined primarily by measuring changes in walking distance based on the 6-minute walk test. Additional measures of physical function and quality of life will be assessed as well. The study is intended to generate the information required to support approval of Ataluren by regulatory authorities in the US and EU.


Review of Phase II and Phase III clinical trials for Duchenne Muscular Dystrophy. Scully M, Pandya S, Moxley R. Expert Opinion on Orphan Drugs. Jan 2013; 1(1): 33-46.

Please Note: “View from starting block of a competition swimming pool” by Rufino Uribe [CC-BY-SA-2.0] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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