Inspire Pharmaceuticals Inc announced a collaboration with Cystic Fibrosis Foundation Therapeutics Inc (CFFT), a non-profit drug development affiliate of the Cystic Fibrosis Foundation that will provide funding of a Phase II study for INS37217 Respiratory for the treatment of cystic fibrosis (CF). The Phase II study is being conducted based on results from a recently completed Phase I/II study.
The CFFT has agreed to provide the majority of funding of external costs for a Phase II trial of INS37217 Respiratory in exchange for post-commercialization milestone payments to be made following FDA approval. Financial terms of the agreement were not disclosed.
This trial, which has been designed in collaboration with the CFFT, will be a 28-day, double-blind, placebo-controlled, parallel group study of INS37217 Respiratory inhaled solution. The study will be conducted in approximately 90 patients with mild CF lung disease at 12 U.S. sites. Inspire will work closely with the Cystic Fibrosis Therapeutics Development Network (CF TDN) and Coordinating Center in conducting the study. The study is targeted for initiation by the end of 2002.
Results from the Phase I/II study of INS37217 Respiratory for CF will be described in a podium presentation on October 5th at 3:00 PM CDT at the North American CF Conference, with a supporting poster. Inspire worked with the CF TDN to conduct the study, which was a double-blind, placebo-controlled, ascending-dose safety and tolerability study in approximately 60 patients at 8 U.S. sites. Results demonstrated that INS37217 Respiratory, delivered by inhalation over an 8-day treatment period, was well tolerated at all dose levels in children and adults with mild CF lung disease. Clear evidence of pharmacological activity was noted. Patients receiving INS37217 Respiratory showed a statistically significant increase compared to placebo (p value = 0.02) in sputum expectoration, a key indicator of lung clearance, following dosing on the first day of the study. In addition, some improvement in lung function was observed in the patients treated with INS37217 Respiratory during this short treatment period. This improvement was noted particularly in patients with mild lung disease, who accounted for the majority of patients in the study, and are the target population for the next study.
Cystic fibrosis is a life-threatening disease involving a genetic mutation that disrupts the cystic fibrosis transmembrane regulator protein. This protein acts as an ion-specific channel that moves salt and water to the surface of the lungs. The defect in this ion channel in CF patients leads to poorly hydrated, thick, mucous secretions in the airways and severely impaired mucociliary clearance. INS37217 Respiratory is believed to enhance the lung's mucosal hydration and mucociliary clearance mechanisms by activating an alternative ion channel that acts in the same way as the defective ion channel in moving salt and water to the surface of the lungs. It is well established that mucociliary clearance is impaired early in life in CF patients. Therefore, this unique, early intervention approach is different from the approach of other approved CF products and may be important in intervening in the early clinical course of CF lung disease.