Enrollment completed in VEGA-1 Phase 2 clinical trial for presbyopia treatment

Ocuphire Pharma Inc. announced today that it has completed enrollment in the VEGA-1 Phase 2 clinical trial (NCT04675151) evaluating the safety and efficacy of a combination kit of 0.75% phentolamine (Nyxol) and low-dose pilocarpine in presbyopia.

According to the company, VEGA-1 successfully recruited 150 subjects in just under 3 months.

According to Mina Sooch, MBA, president and CEO of Ocuphire Pharma, hitting the enrollment completion target for the VEGA-1 trial is another milestone in the company’s Nyxol program and tracks for top-line results by the end of June.

“Nyxol has the potential to address multiple unmet needs initially with the large market opportunity in reversal of mydriasis supported by our recent positive Phase 3 data results and then adding this very large presbyopia market opportunity that is estimated at over $5 billion in the US alone,” Sooch said in a statement. “We are excited to develop Nyxol with low-dose pilocarpine as a potential option for the growing number of presbyopic patients and to also realize commercial synergies if products are approved given the common targeted optometrists and ophthalmologists.”

Ocuphire noted that the global prevalence of presbyopia is estimated to be 2 billion.

An estimated 120 million Americans live with presbyopia, a large prevalence that is expected to exceed 150 million by 2034. There are currently no approved drug therapies for presbyopia.

Moreover, when it comes to payment, presbyopia corrective devices generally have been mostly out-of-pocket cash pay by patients.

Marguerite McDonald, MD, FACS, a clinical professor of ophthalmology at the NYU Langone Medical Center and an Ocuphire Medical Advisory Board member, pointed out that presbyopia is a condition that affects nearly everyone over the age of 40.

“Many patients reliant on reading glasses and contact lenses desire more flexibility and convenience and continually request alternatives or a complementary choice,” she said in a statement. “Development of eyedrop treatments for Presbyopia has recently become very active, led by Allergan having recently submitted an NDA for this indication.”

McDonald noted that there is room for many options given the large unmet need, and she said she is encouraged by Nyxol eye drops in combination with low-dose pilocarpine eye drops as a differentiated solution that works both on the iris dilator and sphincter muscles.

“This combination product should allow for moderate unopposed pupil constriction and provide the ability to see both at near and at distance without the side-effects such as brow ache, headache, blurry vision, and loss of distance night vision associated with the use of higher doses of pilocarpine,” she said.

According to a GlobalData market research report, 69% of patients would consider an eye drop as an alternative to reading glasses.

More than 70% of patients indicated that they wear reading glasses in the morning, afternoon, and evening, suggesting that patients desire a solution with durability.

More than 50% of responders also indicated that using drops 2 to 4 times per day would be moderately to very convenient to them.

In addition, 66% of surveyed eyecare providers indicated interest in a Nyxol and low-dose pilocarpine product profile as a potential therapeutic alternative to reading glasses.

VEGA-1 Phase 2 Clinical Trial
The VEGA-1 clinical trial is designed to evaluate the efficacy and safety of Nyxol in combination with low-dose pilocarpine compared to placebo in presbyopic subjects. A total of 150 subjects have been enrolled at 17 investigational sites in the US from mid-February to mid-May of this year.

The Phase 2 trial is randomly assigned, double-masked, placebo-controlled with 4 treatment arms.

At the first visit, subjects are randomized to receive either Nyxol or placebo drops that are instilled at home near bedtime for 3 to 4 days prior to Visit 2; at Visit 2 subjects then receive either low-dose pilocarpine or no treatment with efficacy and safety measurements collected at multiple timepoints through 6 hours.

The primary endpoint is the percentage of subjects with ≥ 15 letters of improvement in photopic binocular near vision (i.e. distance-corrected near visually acuity, DCNVA) at 1 hour on Visit 2 for Nyxol + low-dose pilocarpine arm compared to placebo alone arm.

Secondary endpoints at multiple timepoints include improvements of 3 lines of DCNVA without any loss of distance vision, pupil diameter, and improvements of DCNVA at 1 and 2 lines compared to placebo as well as to each Nyxol and low-dose pilocarpine alone.

Top-line results are expected by the end of Q2 2021.

Ocuphire collaborated closely with Oculos Development Services, a Tampa, Florida based clinical research organization and a subsidiary of Iuvo BioScience, on the launch and execution of the VEGA-1 trial.

“We are pleased by the rapid enrollment in this Phase 2 trial, which speaks to the unmet need of an eye drop to benefit vision correction for patients with presbyopia,” Chuck Slonim, MD, chief medical officer and medical monitor of Oculos Development Services, said in a statement. “We thank our investigators, clinical coordinators, staff, and subjects for their support and ease of trial execution at 17 sites across the US.”