Nivalis Therapeutics has reached the halfway point in dosing of the 135 patient Phase 2 clinical trial evaluating N91115, the Company’s lead investigational drug and first-in-class stabilizer of the cystic fibrosis transmembrane conductance regulator (CFTR) protein.
“We are encouraged by the progress we have made with patient enrollment in this trial and by the interest expressed by the cystic fibrosis community in a multi-mechanism approach that includes N91115 as a stabilizer of the CFTR protein,” said Jon Congleton, president and chief executive officer of Nivalis. “We are grateful to the investigators, research staff, and especially the patients who have participated, for enabling us to remain on track to report results in the fourth quarter of this year.”
The Phase 2 study is designed to investigate the efficacy and safety of two doses of N91115, 200mg and 400mg, administered twice daily versus placebo in 135 adult patients with CF who are homozygous for the F508del-CFTR mutation and being treated with Orkambi.
N91115 works through a novel mechanism of action called S-nitrosoglutathione reductase (GSNOR) inhibition that is presumed to modulate the unstable and defective CFTR protein responsible for CF. This effect is expected to be both complementary and agnostic to other CFTR modulators, like Orkambi.
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