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CF Researchers Create Inspiration via Inspire Pharmaceuticals


Christy L. Shaffer, Ph.D.
President and CEO, Inspire Pharmaceuticals

Nearly 10 years ago, two leading CF researchers, Ric Boucher, M.D., and Michael Knowles, M.D., who were conducting CF Foundation-funded research at the University of North Carolina at Chapel Hill, sought to bring their research to the commercial drug discovery and development arena.  Their cutting-edge work became the foundation for Inspire Pharmaceuticals’ development of denufosol, a novel inhaled treatment to improve mucus clearance.  Denufosol targets the basic defect of the disease by stimulating the movement of chloride in and out of CF cells, thus circumventing the faulty CFTR protein channel.

The research first conducted by Boucher and Knowles—and currently advanced by Inspire—focuses on P2Y2 receptors, protein molecules that are expressed on the surface of cells.  Drugs binding to receptors are similar to a key fitting into a lock.  The drug acts as the key and the receptor as the lock.  Once the key turns the lock, particular processes within a cell are activated.  For CF patients, these processes could improve mucus hydration and clearance.       

“The ability of this product to significantly improve mucus hydration and clearance may help maintain good lung function, reduce the need for aggressive therapy, and minimize pulmonary exacerbations and hospitalizations,” concluded Christy L. Shaffer, Ph.D., Inspire’s president and chief executive officer. “We believe that denufosol has the potential to shift the treatment paradigm for young people with mild CF by treating them before significant or irreversible lung damage occurs.” 

From its inception, Inspire set out to research the P2Y2 receptors to discover, develop and commercialize new treatments for CF, as well as other diseases in the ophthalmic, respiratory and allergy fields.  

Shortly after signing its first drug discovery and development contract with the CF Foundation, Shaffer also became actively involved.  She served on a committee charged with selecting the first CF care centers to be part of the Therapeutics Development Network (TDN) and led Inspire to become the first pharmaceutical company to use the newly chosen TDN sites and coordinating center to run a clinical trial.

“Through the CF Foundation’s tremendous efforts to gain awareness of this devastating disease, there are many more companies interested in developing new CF therapies,” said Shaffer.  “There are now multiple clinical trials underway to test novel drug candidates and many new trials are expected to be launched within the next few years. This is certainly a tribute to the CF Foundation’s dedication to research.”

Inspire’s CF drug candidate, denufosol, has now entered the final stage of clinical testing.  With the acronym, TIGER-1 (Transport of Ions to Generate Epithelial Rehydration), this Phase 3 trial is currently enrolling approximately 350 patients at more than 70 sites across the United States.  For more information on Inspire’s TIGER-1 trial, please call (919) 287-1286 or visit www.inspirepharm.com.