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news.Inspire Pharmaceuticals has announced top-line results from the 352-patient, double-blind, 24-week placebo-controlled portion of its Phase III trial with denufosol tetrasodium inhalation solution for the treatment of cystic fibrosis.
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The trial demonstrated statistical significance for its primary efficacy endpoint, which was the change in FEV1 (Forced Expiratory Volume in One Second) from baseline at the trial endpoint (at 24 weeks or last observation carried forward). Patients treated with denufosol had a statistically significant improvement in FEV1 compared to placebo (45 milliliter treatment group difference, p = 0.047). On average, patients on denufosol improved relative to baseline whereas patients on placebo remained essentially unchanged.
The treatment effect of denufosol increased over the 24-week placebo-controlled period, and available preliminary data from approximately 210 patients who have completed the ongoing open-label safety extension (including the placebo patients switched to denufosol) suggest that FEV1 continued to improve during the extension period from weeks 24 to 48.
Secondary endpoints were also evaluated in the trial. There was a trend in differences in FEF (25%-75%) (Forced Expiratory Flow 25%-75%), a measure of small airway function, favoring denufosol over placebo (p = 0.072). There were no statistically significant differences between denufosol and placebo relative to pulmonary exacerbations. Data analysis is ongoing for various other pre-specified secondary endpoints and sub-groups.
This trial demonstrated that the effects of denufosol on FEV1 were beneficial in this population of patients taking multiple commonly-prescribed cystic fibrosis (CF) pulmonary medications. Denufosol was well-tolerated and had a favorable safety profile in the trial. Patient retention rates were high and similar between treatment groups with approximately 90% of patients completing the 24-week placebo-controlled portion.
Following the 24-week placebo-controlled period, approximately 315 patients continued into the open-label safety extension portion of the trial and approximately 213 of those patients have now completed the full trial for a total of 48 weeks. The patient discontinuation rate in the open-label safety extension is currently less than 4%.