Great Strides 2006
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First Vertex Drug Candidate Enters Clinical Trials

In collaboration with Cystic Fibrosis Foundation Therapeutics, Inc. (CFFT), Vertex Pharmaceuticals recently advanced into the clinic the first chemical compound to emerge from its mass testing of compounds. Identified through the use of high-throughput screening, the compound, VX-770, has been evaluated in healthy volunteers and CF patients in Phase 1 clinical trials. The data analyses from this initial safety study are currently taking place. In the meantime, plans are underway to initiate a Phase 2 trial in early 2007 to assess the efficacy and safety of multiple doses of VX-770 in CF patients.

“VX-770 is the first drug candidate to emerge from our innovative CF research efforts, and the initiation of this Phase 1 study represents an exciting new stage in the development of this compound,” said John Alam, M.D., executive vice president, medicines development, and chief medical officer of Vertex.
Vertex scientists believe that this compound may act to partially restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein, produced by the defective CF gene. Flaws in the CFTR protein alter the movement of chloride and sodium across the cell membrane, which results in the build up of the characteristic thick, sticky mucus in the lungs of people with CF. This mucus provides a fertile breeding ground for chronic infection and inflammation.

VX-770 belongs to a category of compounds called CFTR potentiators, which have been found to increase the probability that the CFTR channel is open. When the channel is open, increased chloride transport across the cell surface should occur. In previous laboratory experiments, VX-770 has partially restored the opening or “gating activity” of defective CFTR channels.

Vertex’s potentiator compound has also been granted “Fast Track Designation” by the Food and Drug Administration (FDA). Such designation indicates that the FDA will facilitate the development and may expedite the review of a drug if it is intended for the treatment of a serious or life-threatening condition and demonstrates the potential to address an unmet medical need.

“We are excited to see the first fruits of our collaboration with Vertex enter into clinical testing,” said Robert J. Beall, Ph.D, president and CEO of the CF Foundation. “Although many steps remain before this potential drug could ever become a viable therapy, we are certainly looking forward to seeing the results of this initial trial.”