Nicox Reports Positive Results from Whistler Phase 3b Trial of NCX 470 in IOP Reduction

Nicox has announced results from the Whistler Phase 3b exploratory trial, evaluating the intraocular pressure (IOP)-lowering effects of NCX 470 ophthalmic solution 0.1% in both healthy volunteers and patients with ocular hypertension. NCX 470 is the company’s lead clinical candidate, a novel nitric oxide (NO)-donating bimatoprost formulation with a dual mechanism of action targeting IOP through prostaglandin analog activity and NO-mediated trabecular meshwork outflow enhancement.

Trial Design and Population

The Whistler study was a double-masked, placebo-controlled Phase 3b trial involving 18 participants, including healthy volunteers and individuals with ocular hypertension. Measurements were conducted at baseline and after 8 days, with IOP and outflow parameters evaluated at 1 PM and, for some endpoints, at 3 PM.

Participants were primarily normotensive healthy individuals, with baseline IOP averaging 16.6 mmHg in the NCX 470 group and 16.9 mmHg in the placebo group.

Key Efficacy Findings

While aqueous humor flow rate showed a trend toward significance compared to placebo (p=0.072), other findings demonstrated stronger clinical signals:

       • Outflow facility was positive at 1 PM (p=0.081) and statistically significant at 3 PM (p=0.001)

       • Diurnal outflow was significant (p=0.004)

       • IOP reduction and uveoscleral outflow were statistically significant at all timepoints

       • Episcleral venous pressure showed no notable change

Nicox attributed the improvement in outflow to the nitric oxide component of NCX 470, which enhances trabecular meshwork outflow.

“These positive exploratory results suggest that further investigation may be warranted into the dual mechanism effect of NCX 470 on intraocular pressure,” said Doug Hubatsch, Chief Scientific Officer at Nicox. “The therapeutic characteristics of NCX 470 demonstrated in the Phase 3 program so far show that we have an approvable and differentiated asset with a promising clinical profile.”

Regulatory Pathway and Ongoing Development

Nicox confirmed that the Whistler trial is exploratory and not required for the upcoming New Drug Application (NDA) submission to the U.S. Food and Drug Administration (FDA). Therefore, it will not affect the regulatory timeline.

The company continues to advance its pivotal development program for NCX 470, which includes the completed Mont Blanc study and the ongoing Denali Phase 3 trial, with final U.S. patient visits now completed. Results from Denali are expected in Q3 2025.

Nicox intends to use data from both Mont Blanc and Denali to support its NDA submission for NCX 470. A specific NDA submission date has not yet been disclosed.