Inspire Pharmaceuticals, Inc. (NDAQ: ISPH) shares jumped more than 50% in early trading after its new drug to treat cystic fibrosis met its primary goal for a Phase III trial. The 352-patient, double-blind, 24-week placebo-controlled study demonstrated statistical significance for its primary efficacy endpoint. Secondary endpoints were also evaluated in the trial, which is great news for the company.

“The data from this Phase 3 trial with denufosol are encouraging for the cystic fibrosis community because it brings us one step closer to a novel treatment that addresses the basic cystic fibrosis defect," said Robert Beall of the Cystic Fibrosis Foundation. "There remains a high unmet medical need in the area of CF treatments and denufosol could be an important addition to the treatment regimen."

The drug, denufosol, was well-tolerated and had a favorable safety profile in the trial. Patient retention rates were high and similar etween treatments groups with approximately 90% of patients completing the 24-week placebo-controlled portion. The incidence of adverse events in the group was comparable to the placebo. As in previous trials, the problems were minor - coughing.

“The results from this trial are exciting because they demonstrate that denufosol’s novel mechanism of action, activation of an alternative chloride channel that is treating the underlying defect, had a clinically-meaningful impact on lung function that progressively increased over time," said Frank Accurso. "The improvement in FEV1 was achieved on top of aggressive use of multiple concomitant medications."

In the end, this announcement definitely makes Inspire Pharmaceuticals a stock worth watching as successful drug candidates often become takeover targets once all the risk is gone. The completion of this Phase III trial means one giant leap forward in this process.

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