New Research Validates Translarna's™ (ataluren) Mechanism of Action to Promote Readthrough of Nonsense Mutations and Produce Full-length Functional Protein

PTC Therapeutics announced that the Proceedings of the National Academy of Sciences (PNAS) has published new results further validating Translarna's™ (ataluren) mechanism of action to promote readthrough of premature stop codons resulting from nonsense mutations in genetic disorders. The results reported in PNAS establish ataluren's ability to alter the protein production process at premature stop codons and to promote the insertion of specific amino acids and restore the production of a full-length functional protein. 

PPMD continues to urge the FDA to provide PTC with a full review and advisory committee meeting. 

Read the announcement from PTC:

New Research Validates Translarna's™ (ataluren) Mechanism of Action to Promote Readthrough of Nonsense Mutations and Produce Full-length Functional Protein


- Data published in the Proceedings of National Academy of Sciences (PNAS) -

- Results show ataluren targets the protein production machinery -

SOUTH PLAINFIELD, N.J., Oct. 4, 2016 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced that the Proceedings of the National Academy of Sciences (PNAS) has published new results further validating Translarna's™ (ataluren) mechanism of action to promote readthrough of premature stop codons resulting from nonsense mutations in genetic disorders. The results reported in PNAS establish ataluren's ability to alter the protein production process at premature stop codons and to promote the insertion of specific amino acids and restore the production of a full-length functional protein.

"These new results help us better understand ataluren's mechanism of action as well as confirm its protein restoration effect in genetic disorders," said Stuart W. Peltz, Ph.D., co-founder and Chief Executive Officer of PTC Therapeutics. "These results further support our clinical findings demonstrating the production of full-length functional protein in nonsense mutation Duchenne muscular dystrophy and cystic fibrosis. Given this mechanism, ataluren offers the potential for a new therapeutic approach for multiple nonsense mutation genetic disorders by targeting the underlying cause of the disease."

The results published by PTC Therapeutics, Dr. Allan Jacobson and his team at the University of Massachusetts Medical School, and Dr. David Bedwell and his team at the University of Alabama, demonstrate ataluren treatment produces a protein that is similar to the protein from cells that do not have a nonsense mutation. The findings were verified in multiple nonsense mutation models. In addition, there have been almost 40 publications to date, many by independent investigators, demonstrating the clinical activity of Translarna across a spectrum of rare diseases.

"These data provide new insight on ataluren's effect on protein production and validates that it targets the source of nonsense mutation genetic disorders," said Allan Jacobson, Ph.D., co-founder and Board member of PTC Therapeutics and the Gerald L. Haidak, MD and Zelda S. Haidak professor of cell biology and chair of microbiology and physiological systems at University of Massachusetts Medical School. "Therapeutic nonsense suppression is a potentially powerful approach for the treatment of the large number of genetic disorders caused by nonsense mutations."

About Translarna™ (ataluren)
Translarna, discovered and developed by PTC Therapeutics, Inc., is a protein restoration therapy designed to enable the formation of a functioning protein in patients with genetic disorders caused by a nonsense mutation. A nonsense mutation is an alteration in the genetic code that prematurely halts the synthesis of an essential protein. The resulting disorder is determined by which protein cannot be expressed in its entirety and is no longer functional, such as dystrophin in Duchenne muscular dystrophy. Translarna is licensed in the European Economic Area for the treatment of nonsense mutation Duchenne muscular dystrophy in ambulatory patients aged five years and older. Translarna is an investigational new drug in the United States . The development of Translarna has been supported by grants from Cystic Fibrosis Foundation Therapeutics Inc. (the nonprofit affiliate of the Cystic Fibrosis Foundation); Muscular Dystrophy Association; FDA'sOffice of Orphan Products Development;National Center for Research Resources; National Heart, Lung, and Blood Institute; and Parent Project Muscular Dystrophy.

Read the full press release from PTC

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