Mina Sooch, MBA, is the CEO and founder of Ocuphire Pharma in Farmington Hills, Mich. Ocuphire’s pipeline currently includes two small-molecule product candidates, Nyxol and APX3330, targeting front and back of the eye indications. Ms. Sooch has more than 25 years of pharmaceutical and biotech experience as CEO, entrepreneur, venture capitalist and strategist.

Ophthalmology Management: What are Ocuphire’s latest products/developments for the US market?

Mina Sooch: Ocuphire has two late-stage product candidates within its portfolio. Nyxol (0.75% phentolamine ophthalmic solution) is a once-daily, preservative-free eyedrop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist that works by relaxing the iris dilator muscle to reduce the pupil size safely and effectively.

Nyxol is being developed for several refractive indications with the most advanced in Phase 3 including the reversal of pharmacologically induced mydriasis, presbyopia and dim light or night vision disturbances (a new indication to reduce halos and glares that can occur in people with post-LASIK or due to aging or dry eye). Nyxol has the potential to be the only dilation reversal drop, if approved, and a differentiated presbyopia-correcting drop in development with a unique mechanism of action (MOA) and long durability.

The second product candidate is APX3330, an oral tablet that targets a novel protein called Ref-1 (reduction oxidation effector protein-1) and has a dual mechanism of an anti-VEGF and an anti-inflammatory, both pathways that are relevant to retinal and choroidal vascular diseases, such as diabetic retinopathy (DR) and diabetic macular edema (DME). APX3330 as an oral therapy has the potential to treat over 7 million DR patients with limited treatment options. APX3330 is currently in Phase 2b clinical trial for the treatment of diabetic retinopathy.

OM: Ocuphire recently announced positive topline results in the MIRA-3 trial and MIRA-4 pediatric safety trial. Can you give us an overview?

MS: MIRA-3 was designed as a multi-center, randomized, parallel arm, double-masked, placebo-controlled Phase 3 trial evaluating the safety and efficacy of Nyxol in subjects with pharmacologically induced mydriasis. The effects of mydriasis cannot be minimized, and the visual impairment and discomfort can last anywhere from 6-24 hours. The MIRA-3 trial met its primary endpoint with 58% of subjects (study eye) treated with Nyxol returning to ≤0.2 mm of their baseline pupil diameter (PD) at 90 minutes compared to only 6% of subjects (study eye) treated with placebo. The effect was also rapid and significant with 42% response rate at 60 minutes compared to only 36% of placebo treated subjects returned to baseline PD at 6 hours. Also, MIRA-4 investigated Nyxol’s safety and efficacy in pediatric subjects 3-11 years old, and we are pleased to report the topline results were consistent with that shown in MIRA-3.

We intend to file an NDA with the FDA in late 2022, which, if approved, would position Ocuphire for commercial launch of Nyxol, a reversal drop for dilations, in the second half of 2023.

OM: Can you discuss Ocuphire’s pursuit of treating presbyopia with Nyxol?

MS: Nyxol’s differentiated mechanism of action allows us to pursue presbyopia in two ways: Nyxol as a single agent and Nyxol with low-dose pilocarpine 0.4% as an adjunctive therapy. We reported positive top-line efficacy data from a multi-center, well-controlled Phase 2 presbyopia trial both alone and in combination, and we plan to initiate a presbyopia Phase 3 program this year. A single drop off Nyxol in the evening has demonstrated 12- to 18-hour durability with a favorable safety and tolerability profile with no headaches or brow aches due to unique MOA that does not engage the ciliary muscle.

With our single agent and adjunct therapy, we are uniquely positioned to tune the pupil beyond the chart to accommodate the dynamic lifestyles and needs in the presbyopia patient journey.

OM: Ocuphire recently completed enrollment in a Phase 2b clinical trial of APX3330 to treat DR/DME (ZETA-1). Can you give us an overview?

MS: The ZETA-1 trial will evaluate the efficacy and safety of APX3330 for the treatment of DR at 25 investigational sites across the United States. The primary endpoint is a responder analysis that evaluates the percentage of subjects with a ≥2-step improvement on the DRSS score.

APX3330 has been extensively studied in humans across 11 Phase 1 and Phase 2 clinical trials in healthy, hepatitis and cancer subjects prior to ZETA-1 and has shown a favorable safety and tolerability profile. APX330 also has a dual mechanism of an anti-VEGF and an anti-inflammatory.

OM: How has your company accomplished so much in such a short period of time?

MS: I founded Ocuphire in 2018 and with a strategy to acquire two de-risked clinical-stage assets from the Midwest. Our pipeline, a highly experienced internal team and a great cadre of KOLs that spans across ophthalmology, retina and optometrists have contributed to our milestone accomplishments. With their insights, we have been able to target the right indications, trial designs and understand the needs of their patients.

In addition, what makes Ocuphire different from other publicly traded biotech companies in the ophthalmic space is that we are one of the very few companies with a late-stage retinal program and a late-stage refractive program.

Lastly, we have a brilliant and productive team who are committed to bringing innovative treatment options to millions of patients. We might be a small team, but we make up for it in grit, talent and passion. I am inspired and extremely proud of the Ocuphire team. The momentum is at an all-time high, and I encourage you to stay tuned for more. OM