“APX3330 has potential to be the first non-invasive, early treatment to delay vision-threatening complications in millions of patients with non-proliferative diabetic retinopathy (“DR”) who otherwise remain untreated, and we are pleased with the progress on our preparations for the next clinical study. We continue to work collaboratively with the U.S. Food and Drug Administration (the “FDA”) on our submitted Special Protocol Assessment (“SPA”) for Phase 2/3 and the overall clinical development plan for APX3330,” said George Magrath, M.D., M.B.A., M.S., CEO of Ocuphire.

 

Dr. Magrath continued, “We are also making progress on additional indications for phentolamine ophthalmic solution. The VEGA-3 Phase 3 trial in presbyopia, funded by our commercial partner, has begun recruiting. Phentolamine ophthalmic solution has demonstrated a differentiated product profile in prior presbyopia studies, and we believe it could be a promising and convenient treatment option for the growing number of people with this condition. The LYNX-2 Phase 3 study in dim light disturbances (“DLD”) has seen strong enrollment and could provide a differentiated product for patients.”

 

 

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The VEGA-3 Phase 3 trial evaluating phentolamine ophthalmic solution 0.75% for the treatment of presbyopia is now recruiting. This randomized, double-masked, placebo-controlled, multi-center Phase 3 trial is expected to enroll up to 545 subjects with presbyopia at up to 40 U.S. sites. Participants will be randomized 3:2 to receive phentolamine ophthalmic solution 0.75% or placebo. The primary endpoint is the percentage of subjects with 15-letter improvement in photopic binocular distance-corrected near visual acuity (DCNVA) on the eighth day following their first visit. Topline results are expected in 2025.

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Enrollment has been strong for the LYNX-2 Phase 3 registration study evaluating phentolamine ophthalmic solution 0.75% for the treatment of decreased visual acuity under low (mesopic) light conditions following keratorefractive surgery. The LYNX-2 trial is being conducted under conditions of an SPA with the FDA.

•	The Company is also planning an additional Phase 3 study for decreased vision under mesopic (low) light conditions following keratorefractive surgery, LYNX-3, in 2024.

•	The phentolamine ophthalmic solution 0.75% development portfolio is being funded by Ocuphire’s partner in both indications (presbyopia and dim light vision disturbances).

General and administrative expenses for the three months ended June 30, 2024 were $3.4 million compared to $4.3 million for the three months ended June 30, 2023. The decrease was primarily attributable to a net reduction in payroll-related costs and a reduction in other operating expenses. Partially offsetting these reductions were increases in business development costs, legal support costs and other operating expenses, compared to the corresponding prior year period. General and administrative expenses included $0.5 million and $1.2 million in stock-based compensation expense during the three months ended June 30, 2024 and 2023, respectively.

 

About Ocuphire Pharma

 

Ocuphire Pharma, Inc. (Nasdaq: OCUP) is a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing novel therapies for the treatment of patients with retinal and refractive eye disorders. Ocuphire’s lead product candidate, APX3330, a novel small-molecule inhibitor of Ref-1 (reduction oxidation effector factor-1 protein), is in development for diabetic retinopathy. In addition, Ocuphire’s late-stage product candidate Phentolamine Ophthalmic Solution 0.75%, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size, is being developed for presbyopia and dim light vision disturbances and is currently approved and marketed as RYZUMVI™, indicated for the treatment of pharmacologically-induced mydriasis produced by adrenergic agonists (e.g., phenylephrine) or parasympatholytic (e.g., tropicamide) agents, by our commercial partner. For more information, please visit www.ocuphire.com.

 

Forward Looking Statements

 

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Such statements include, but are not limited to, statements concerning the applications of phentolamine ophthalmic solution 0.75% in ophthalmology, the registration program for phentolamine ophthalmic solution 0.75%, the LYNX-2 Phase 3 registration study, the efficacy of APX3330 in slowing the progression of diabetic retinopathy, the safety and tolerability of APX3330, logistical details regarding the upcoming VEGA-3 Phase 3 clinical trial and estimates of when data will become available from such trial, the benefits, uses and side effects of phentolamine ophthalmic solution 0.75% treatment, ongoing discussions with the U.S. Food and Drug Administration regarding our drug products, continued drug development under our agreement with our commercial partner, and the sufficiency and amount of cash on hand to meet future funding needs.

 

These forward-looking statements relate to us, our business prospects and our results of operations and are subject to certain risks and uncertainties posed by many factors and events that could cause our actual business, prospects and results of operations to differ materially from those anticipated by such forward-looking statements. Factors that could cause or contribute to such differences include, but are not limited to, those described under the heading “Risk Factors” included in our Annual Report on Form 10-K and in subsequent filings with the U.S. Securities and Exchange Commission. Readers are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date of this report. In some cases, you can identify forward-looking statements by the following words: “anticipate,” “believe,” “continue,” “could,” “estimate,” “expect,” “intend,” “may,” “ongoing,” “plan,” “potential,” “predict,” “project,” “should,” “will,” “would” or the negative of these terms or other comparable terminology, although not all forward-looking statements contain these words. We undertake no obligation to revise any forward-looking statements in order to reflect events or circumstances that might subsequently arise.

 

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The success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts;

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Regulatory requirements or developments;

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Changes to or unanticipated events in connection with clinical trial designs and regulatory pathways;

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Delays or difficulties in the enrollment of patients in clinical trials;

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Substantial competition and rapid technological change;

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Our development of sales and marketing infrastructure;

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Future revenue losses and profitability;

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Our relatively short operating history;

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Changes in capital resource requirements;

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Risks related to the inability of Ocuphire to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs;

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Domestic and worldwide legislative, regulatory, political and economic developments;

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Employee misconduct;

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Changes in market opportunities and acceptance;

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Reliance on third-parties;

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Future, potential product liability and securities litigation;

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System failures, unplanned events, or cyber incidents;

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The substantial number of shares subject to potential issuance associated with our equity line of credit arrangement;

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Risks that our partnership with our commercial partner, or our other licensing arrangements, may not facilitate the commercialization or market acceptance of Ocuphire’s product candidates;

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Future fluctuations in the market price of our common stock;

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The success and timing of commercialization of any of Ocuphire’s product candidates; and

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Obtaining and maintaining Ocuphire’s intellectual property rights.

Ocuphire Pharma, Inc.

Condensed Balance Sheets

(in thousands, except share amounts and par value)

Ocuphire Pharma, Inc.

Condensed Statements of Comprehensive Loss

(in thousands, except share and per share amounts)

(Unaudited)

 

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