Pipeline Update: Ataluren - PTC receives funds to support Phase 3 confirmatory trials

PTC Therapeutics, Inc. (PTC) today announced the successful completion of a $60 million financing led by Brookside Capital Partners Fund, L.P.

"This substantial financing enables PTC to continue to pursue the regulatory approval and delivery of ataluren to patients," stated Michael Schmertzler, Chairman of the Board. "PTC's Board of Directors welcomes the breadth of support and expertise of our new investors."

Proceeds from this financing will support continued clinical development of ataluren in Duchenne muscular dystrophy and cystic fibrosis. PTC is initiating a Phase 3 confirmatory trial of ataluren in patients with nonsense mutation Duchenne muscular dystrophy (nmDMD) with the goal of commencing enrollment in the first half of 2013. PTC also is developing ataluren for nonsense mutation cystic fibrosis (nmCF). The company has worldwide development and commercial rights to ataluren in all indications and territories.

"We are honored to have the support of our new and existing investors," said Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, Inc. "This financing is critical to expanding our efforts to develop and commercialize novel therapies that may benefit patients with nonsense mutation Duchenne muscular dystrophy and cystic fibrosis."

(Read PTC's Full Release)

Learn more about ataluren 

Ataluren is an investigational drug developed by PTC Therapeutics that is designed to allow cells to read-through or “ignore” certain types of mutations called nonsense mutations. A nonsense mutation occurs when a single DNA letter is changed in such a way that the cell stops reading the gene to create a protein too early, leading to an incomplete protein that is not functional. Many genetic diseases, including Duchenne, can be caused by a nonsense mutation. Approximately 13% of boys and men with Duchenne have this kind of mutation.

Ataluren is a small molecule that can be taken orally in a liquid. PTC believes that ataluren is potentially applicable to a broad range of other genetic disorders in which a nonsense mutation is the cause of the disease. The FDA has granted fast-track designations and orphan drug designations to ataluren for the treatment of cystic fibrosis and Duchenne due to nonsense mutations.

Duchenne Research

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