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PTC Therapeutics Announces Publication of Preclinical Data in PNAS
Date:2/4/2008

ng the premature stop signal and reading through the complete genetic instructions, resulting in the restoration of a full-length, functional protein. Patients with CF lack the CFTR protein, a chloride channel that maintains proper hydration of epithelial cells in the lung, pancreas, and liver. The data in PNAS demonstrate that PTC124 allows CFTR to be made in cells in which it was previously absent, to be delivered to the proper cellular location, and to induce chloride channel function. Collectively, these results indicate that this new approach may ultimately be applicable to a subset of patients across a large number of genetic disorders.

The paper entitled, "PTC124 is an orally bioavailable compound that promotes suppression of the human CFTR-G542X nonsense allele in a CF mouse model," will be available in an advanced online publication of PNAS during the week of February 4, 2008 (http://www.pnas.org/papbyrecent.shtml). This publication is the result of collaborative efforts between the University of Alabama at Birmingham and PTC Therapeutics.

"These preclinical data further demonstrate the potential applicability of the PTC124 mechanism of action to multiple genetic disorders," said Stuart W. Peltz, Ph.D., President and Chief Executive Officer of PTC Therapeutics. "We look forward to further evaluating PTC124 in registration-directed clinical trials in both CF and DMD this year, as well as in studies in additional genetic disorders in the future."

About PTC124

PTC124 is an orally delivered investigational new drug in Phase 2 clinical development for the treatment of genetic disorders due to nonsense mutations. Nonsense mutations are single-point alterations in the genetic code that prematurely halt the translation process, producing a shortened, non- functional protein. PTC124 has restored production of full-length, functional proteins in preclinical genetic disease m
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SOURCE PTC Therapeutics, Inc.
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