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Friday, Feb 3, 2023

Vertex’s Cystic Fibrosis Drug Shows Promise in Patient Trial

A midstage trial of an investigational drug being developed by Vertex Pharmaceuticals Inc. showed significant improvements in patients with cystic fibrosis, the company said March 27.

Vertex said its oral drug candidate, which targets a basic defect in cystic fibrosis, showed promising results in an ongoing Phase 2a clinical trial for patients who carry the genetic mutation.

Patients who took the investigational drug for 14 days showed significant improvements in several key indicators of the disease, including lung function, nasal potential difference measurements and sweat chloride levels.

The findings suggest that VX-770 improves function of what is known as the faulty CFTR protein.

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“We basically confirmed the hypothesis that if you fix CFTR function you might restore the lung function,” said Paul Negulescu, vice president of research at Vertex, a Cambridge, Mass.-based company that employs about 150 people in San Diego, where it conducts cystic fibrosis research.

The early data could have important implications for studies of other drugs in development, according to the Cystic Fibrosis Foundation. The fatal lung condition affects about 30,000 people in the United States and 70,000 people worldwide.

VX-770 was developed in San Diego in conjunction with Cystic Fibrosis Foundation Therapeutics Inc., the nonprofit drug discovery and development arm of the Cystic Fibrosis Foundation. The venture philanthropy arm of the foundation has invested $79 million in the project, and the two organizations have worked in close collaboration to advance VX-770 and a second cystic fibrosis compound, known as VX-809, through research and into development.

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Pure Regains Nasdaq Status:

Pure Bioscience, an El Cajon-based maker of disinfectants, began listing its stock on Nasdaq on April 2 under the symbol PURE.

Nasdaq delisted the company’s stock in February 2004, less than a year after it changed its name from Innovative Medical Services to Pure in an effort to refocus its water treatment business on bioscience technologies. Its stock did not comply with minimum bid price requirements of $1 a share.

The stock was trading at $6.18 in midday trading April 2. Pure traded on the Over the Counter Bulletin Board between $2 and $8.72 a share during the past year.

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Cancer Research Convention Drawing International Crowd:

The American Association for Cancer Research plans to gather at the San Diego Convention Center for its annual meeting April 12 to 16.

More than 17,000 participants from 60 countries are expected to discuss the latest happenings in basic, clinical and translational cancer research.

The Philadelphia-based association said it expects the exhibit show to be its biggest in history, gathering together an anticipated 400 companies with the latest products and services for laboratory and clinical research.

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