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Funding drugs for rare diseases
Vertex Pharmaceuticals Inc. has begun a Phase 2a clinical trial of a cystic fibrosis treatment that combines two of the company’s regimens.
The trial will test two cystic fibrosis transmembrane conductance regulator (CFTR) modulators – VX-809 as a stand-alone dose for 14 days, then a seven-day dose of VX-809 in combination with VX-770. Cystic fibrosis is caused by defective or missing CFTR proteins, Vertex (Nasdaq: VRTX) officials report. VX-809 is a CFTR corrector, which is designed to move CFTR proteins to the cell surface; VX-770 is a CFTR potentiator, which is designed to improve functioning of defective CFTR proteins.
The three-part trial will test 160 people with cystic fibrosis at 21 sites in the U.S., Europe, New Zealand and Australia.
Vertex’ VX-809 and VX-770 were developed through a collaboration with Cystic Fibrosis Foundation Therapeutics Inc., with Vertex holding the worldwide development and commercialization rights.
Vertex, which employs approximately 1,400 people worldwide, has been running at a loss that runs between $150 million and $200 million for each quarter for the last year.
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