Ocuphire Pharma submits New Drug Application to FDA for Nyxol eye drops for reversal of mydriasis

The NDA submission for phentolamine ophthalmic solution 0.75% is an important step toward the company’s goal of providing a reversal option for the millions of standard eye exams and procedures that involve dilation.

Ocuphire Pharma Inc. today announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for phentolamine ophthalmic solution 0.75% (Nyxol) for the reversal of pharmacologically-induced mydriasis (RM) produced by adrenergic agonist (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents, or a combination thereof.

In a news release, Mina Sooch, MBA, founder and CEO of Ocuphire Pharma, pointed out that the NDA submission for phentolamine ophthalmic solution 0.75% is an important step toward our goal of providing a reversal option for the millions of standard eye exams and procedures that involve dilation.

“Achieving this milestone is a testament to the commitment of the clinical trial investigators, their staff and trial participants as well as the Ocuphire team and its network of development partners who have worked diligently to advance Nyxol towards a potential approval in RM,” Sooch said in the release. “We look forward to working closely with the FDA during the review process. If approved, Nyxol is expected to be the only commercially available treatment option indicated for the reversal of dilated eyes.”

Sooch also added that the company is looking forward to adding this milestone to the company’s recently closed global license agreement with FamyGen Life Sciences (Famy) for the development and commercialization of Nyxol across three indications. With the separately announced acquisition of Famy by Viatris (Nasdaq: VTRS), a global healthcare company, we believe Viatris provides a great opportunity for all of the phentolamine ophthalmic solution 0.75% indications to realize their full commercial potential in their respective markets given its strategic commitment to ophthalmology and its global commercial infrastructure.

The company noted in the news release that the NDA is supported by positive results from the comprehensive MIRA clinical program collectively involving over 600 subjects, including the MIRA-1 Phase 2b trial, MIRA-2 and MIRA-3 Phase 3 pivotal trials, and MIRA-4 Phase 3 pediatric trial. The MIRA-2 and MIRA-3 trials successfully met their primary and key secondary endpoints, demonstrating statistically significant superiority of phentolamine ophthalmic solution 0.75% compared to placebo to rapidly return dilated eyes to their baseline pupil diameter as early as 60 and 90 minutes.

Moreover, the positive MIRA-4 pediatric trial results support a potential broader label for phentolamine ophthalmic solution 0.75% in RM to include pediatric subjects aged 3 and older. In addition, phentolamine ophthalmic solution 0.75% consistently showed a favorable safety and tolerability profile across all trials.

Ocuphire has received the $35 million upfront payment under the license agreement. Combined with its previous reported cash balance of $13.9 million at Sept 30, 2022, the Company believes that it has sufficient capital to fund operations into 2025, including advancing development of APX3330. If Nyxol is approved for RM in the US, Ocuphire is eligible to receive a $10 million milestone payment.

About Nyxol

Phentolamine ophthalmic solution 0.75% l is a proprietary, preservative-free, stable, investigational eye drop formulation of phentolamine mesylate 0.75% designed to uniquely modulate the pupil size by blocking the α1 receptors found only on the iris dilator muscle without affecting the ciliary muscle. Phentolamine ophthalmic solution 0.75% is being developed for reversal of pharmacologically-induced mydriasis (RM), presbyopia, and night (or dim light) vision disturbances (NVD) under the 505(b)(2) pathway.

The company noted that phentolamine ophthalmic solution 0.75% has been studied in a total of 12 clinical trials (3 Phase 1, 5 Phase 2, 4 Phase 3) in a total of approximately 1100 patients (with over 650 Nyxol-treated) and has demonstrated promising clinical data for use in the multiple ophthalmic indications mentioned above. Ocuphire reported positive top-line data from the MIRA-1, MIRA-2, MIRA-3 and MIRA-4 trials in RM, Phase 3 LYNX-1 trial in NVD and Phase 2b VEGA-1 trial for phentolamine ophthalmic solution 0.75% as single agent and as adjunctive therapy with 0.4% low dose pilocarpine in presbyopia.