Ocuphire Provides Corporate Update and Reiterates Previous Guidance on Clinical Programs
PDUFA Date of September 28, 2023 Set for Nyxol® in its First Indication, Reversal of Pharmacologically-induced Mydriasis (RM); $10 Million Milestone Linked to Approval
Oral APX3330 End of Phase 2 Meeting with FDA Anticipated in 2023 to Align on Regulatory Path for Diabetic Retinopathy
Company Plans to Announce Q1 2023 Financial Results on May 15; Preliminary (Unaudited) Cash Balance as of March 31, 2023 of $39 Million is Expected to Fund Operations Into 2025
FARMINGTON HILLS, Mich., April 25, 2023 (GLOBE NEWSWIRE) -- Ocuphire Pharma, Inc. (Nasdaq: OCUP), a clinical-stage ophthalmic biopharmaceutical company focused on developing and commercializing therapies for the treatment of refractive and retinal eye disorders, today announced that the Company will release its first quarter 2023 financial results on Monday, May 15, 2023. The Company also provided a corporate update, reiterating its previous guidance on its clinical programs.
“All development program timelines remain unchanged from previous guidance and the Ocuphire team is committed to executing on our near-term priorities. 2023 is a pivotal year for Ocuphire with multiple potential late-stage clinical, regulatory, and product approval catalysts across the Nyxol and APX3330 programs,” said Rick Rodgers, Interim CEO and President of Ocuphire.
Anticipated 2023 Milestones
In February 2023, Ocuphire announced that U.S. Food and Drug Administration (FDA) had accepted for review a New Drug Application (NDA) for Nyxol® in RM and set a PDUFA date of September 28, 2023. The NDA was submitted in November 2022 and was supported by positive results from the four MIRA clinical trials that collectively involved over 600 subjects, including pediatric subjects over 3 years old. An NDA approval for Nyxol in RM would trigger a $10 million payment from Viatris.
The Company entered into a partnership with Viatris in November 2022 and Viatris has highlighted the Nyxol portfolio of indications as one of the key elements of its plan to create a global eye care leader. The partnership provides an externally financed pathway to complete development and all regulatory activities for Nyxol and provides a commercial partner to execute successful US and global launches. The financial elements of the agreement have strengthened Ocuphire’s balance sheet and provides additional capital to help advance APX3330 .
Topline results from the ZETA-1 Phase 2 trial of oral APX3330 for the treatment of diabetic retinopathy (DR) were announced in January 2023. Oral APX3330 achieved statistical significance on a key pre-specified secondary endpoint of binocular ≥ 3-step worsening of DRSS and demonstrated favorable safety and tolerability after 24 weeks of treatment. This binocular secondary endpoint is a potential Phase 3 registration endpoint. The Company plans an End-of-Phase 2 FDA meeting in the second half of 2023 to formally agree on this endpoint.
In January 2023, the Company announced the initiation of the VEGA-2 Phase 3 pivotal trial in presbyopia, with the first patient enrolled in late December. VEGA-2 is evaluating treatment efficacy and safety for two labels for presbyopia: Nyxol alone and Nyxol with adjunctive Low Dose Pilocarpine (LDP) therapy. The Company plans to release topline data from VEGA-2 in the second half of 2023 and to initiate a second Phase 3 pivotal trial in presbyopia (VEGA-3), and a one-year safety study (LYRA-1) in 2023.
|Partner or OCUP Name||Study||Milestone||Anticipated Indication||1H 2023||2H 2023|
|APX3330||ZETA-1||End of Phase 2 FDA meeting||Diabetic Retinopathy||•|
|MIRA program||PDUFA date Sept 28, 2023||Reversal of Pharmacologically-induced Mydriasis||•|
(Nyxol and Nyxol+LDP)
|VEGA-3||Initiate 2nd Phase 3 trial||Presbyopia||•|
|VEGA-2||Report 1st Phase 3 topline data||Presbyopia||•|
|LYNX-2||Initiate 2nd Phase 3 trial||Night Vision or Dim Light Disturbances
|MR-141 and MR-142
|LYRA-1||Initiate long-term safety trial||Presbyopia and DLD||•|
The preliminary unaudited financial information presented in this press release reflects the Company's current estimates based on information available as of the date of this press release and is subject to change as a result of the completion of the Company’s financial and operating closing procedures, customary audit procedures, and other developments that may occur before the completion of these procedures. Accordingly, you should not place undue reliance on this preliminary financial information, which may differ materially from actual results. See “Forward Looking Statements” below for a discussion of certain factors that could result in differences between the estimated unaudited financial information reported in this press release and actual results.
About Ocuphire Pharma
Ocuphire is a publicly traded (Nasdaq: OCUP), clinical-stage, ophthalmic biopharmaceutical company focused on developing and commercializing small-molecule therapies for the treatment of refractive and retinal eye disorders.
Ocuphire has a partnership with Viatris, Inc. to develop and commercialize Nyxol® eye drops as a preservative-free eye drop formulation of phentolamine mesylate, a non-selective alpha-1 and alpha-2 adrenergic antagonist designed to reduce pupil size by uniquely blocking the alpha-1 receptors found only on the iris dilator muscle without affecting the ciliary muscle. Nyxol has been studied in a total of 12 clinical trials (3 Phase 1, 5 Phase 2, 4 Phase 3) across three indications, including single-use for reversal of pharmacologically-induced mydriasis (RM), and once-daily for treatment of presbyopia and dim light (night) vision disturbances (DLD), pending regulatory approvals. Nyxol’s NDA under the 505(b)(2) pathway for the first indication, RM, has been accepted with a PDUFA date assigned of September 28, 2023. Nyxol is currently in Phase 3 for presbyopia and DLD.
Ocuphire’s other late-stage product candidate, APX3330, is a first-in-class, small molecule oral drug that blocks downstream pathways regulated by transcription factor Ref-1 – including those involving angiogenesis (VEGF) and inflammation (NFkB). These pathways are implicated in several ocular diseases, including diabetic retinopathy (DR), diabetic macular edema (DME), and age-related macular degeneration (AMD). Ocuphire recently announced topline data from the ZETA-1 Phase 2 trial in which APX3330 achieved statistical significance on a key pre-specified secondary endpoint of preventing clinically meaningful progression of DR after 24 weeks of daily treatment. APX3330 has also shown a favorable safety and tolerability profile in diabetic subjects (ZETA-1 trial) and in 11 previous clinical trials conducted in healthy, liver disease, and cancer subjects. An End-of-Phase 2 meeting with the FDA is planned for APX3330.
For more information, visit www.ocuphire.com.
Forward Looking Statements
Statements contained in this press release regarding matters that are not historical facts are “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 timing and success of identifying a permanent CEO, the potential receipt of regulatory approval for Nyxol for the treatment of RM, expected financial results for the quarter ended March 31, 2023, the ability to fund operations into 2025, the occurrence and timing of an End-of-Phase 2 meeting with the FDA, the ability of Viatris to execute successful US and global launches of Nyxol, and the ability to determine a path to registration for APX3330. These forward-looking statements are based upon Ocuphire’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, including, without limitation: (i) the success and timing of regulatory submissions and pre-clinical and clinical trials, including enrollment and data readouts; (ii) regulatory requirements or developments; (iii) changes to clinical trial designs and regulatory pathways; (iv) changes in capital resource requirements; (v) risks related to the inability of Ocuphire to obtain sufficient additional capital to continue to advance its product candidates and its preclinical programs; (vi) legislative, regulatory, political and economic developments, (vii) changes in market opportunities, (viii) the effects of COVID-19 on clinical programs and business operations; (ix) risks that the Nyxol partnership may not facilitate the commercialization or market acceptance of Ocuphire’s product candidates; (x) the success and timing of commercialization of any of Ocuphire’s product candidates; and (xi) the maintenance of Ocuphire’s intellectual property rights. The foregoing review of important factors that could cause actual events to differ from expectations should not be construed as exhaustive and should be read in conjunction with statements that are included herein and elsewhere, including the risk factors detailed in documents that have been and may be filed by Ocuphire from time to time with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made. Ocuphire undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made.
Corey Davis, Ph.D.
Released April 25, 2023