Clinical Trials

Date: 2016-10-04

Type of information: Publication of results in a medical journal

phase: preclinical

Announcement: publication of results in the Proceedings of the National Academy of Sciences of the United States of America (PNAS)

Company: PTC Therapeutics (USA - NJ)

Product: Translarna™ (ataluren)

Action mechanism:

protein restauration therapy/inducer of ribosomal readthrough on nonsense mutation mRNA stop codons. Ataluren (3-[5-(2-fluoro-phenyl)-[1,2,4]oxadiazole-3-yl]-benzoic acid) is an investigational new drug designed to enable the production of functional dystrophin protein in the muscle cells of patients with genetic disorders due to a nonsense mutation. Ataluren is designed to allow the ribosome to ignore the premature stop signal and continue translation of the mRNA, resulting in formation of a functioning protein. Ataluren does not cause the ribosome to read through the normal stop signal.

Disease:

Therapeutic area: Rare diseases - Genetic diseases

Country:

Trial details:

Latest news:

• On October 4, 2016, 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. 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.