Product Pipeline

Clinical Development Plan for Clearer Vision

Ocuphire’s pipeline currently includes two small molecular product candidates targeting front and back-of-the-eye indications. Our lead candidate APX3330 is being developed for diabetic retinopathy (DR) and diabetic macular edema (DME). Our late-stage candidate Phentolamine Ophthalmic Solution 0.75% is being developed for the reversal of pharmacologically-induced mydriasis, presbyopia, and dim light vision disturbances (DLD).

	Product Candidate	Indication	Development Stage					Anticipated Milestones
	Product Candidate	Indication	Preclinical	Phase 1	Phase 2	Phase 3	Regulatory Approval	Anticipated Milestones
Retina-Focused Development
	APX3330 Oral Pill	Diabetic Retinopathy (DR)/ Macular Edema (DME)	Preclinical complete	Phase 1 complete	Phase 2 in progress Completed Trial EOP2 Meeting	Phase 3 not started	Regulatory Approval not started	ZETA-1 Phase 2 data reported in 1Q 2023 (n=103) EOP2 Meeting in Q4 2023
	APX2009 Local Delivery	Retina	Preclinical in progress	Phase 1 not started	Phase 2 not started	Phase 3 not started	Regulatory Approval not started	Select retinal drug delivery technology
	APX2014 Local Delivery	Retina	Preclinical in progress	Phase 1 not started	Phase 2 not started	Phase 3 not started	Regulatory Approval not started	Select retinal drug delivery technology
Refractive-Focused Development
	Phentolamine Ophthalmic Solution 0.75% Eye Drop	Pharmacologically-Induced Mydriasis	Preclinical complete	Phase 1 complete	Phase 2 complete	Phase 3 complete	Regulatory Approval in progress partnered with Viatris	Positive MIRA Phase 3 trials (n=500+) Submitted NDA in November 2022 APPROVED in September 2023
	Phentolamine Ophthalmic Solution 0.75% + Low-Dose 0.4% Pilocarpine Eye Drops	Presbyopia	Preclinical complete	Phase 1 complete	Phase 2 complete	Phase 3 in progress partnered with Viatris Phase 3 Initiated	Regulatory Approval not started	Positive VEGA-1 Phentolamine Ophthalmic Solution 0.75% alone daa in 1Q 2022 (and in combination with LDP) VEGA-2 and VEGA-3 Phase trials for single agent and combination with LDP (n=300+); VEGA-2 Topline Data in Q4 2023 LYRA-1 Long Term Safety Phase 3 Trial (n=400+)
	Phentolamine Ophthalmic Solution 0.75% Eye Drop	Dim Light or Night Vision Disturbances (NVD)	Preclinical complete	Phase 1 complete	Phase 2 complete	Phase 3 in progress partnered with Viatris	Regulatory Approval not started	Positive LYNX-1 Phase 3 data in 2Q 2022 (n=145) LYNX-2 2nd Phase 3 trial (n=150+)

Lead Product Candidate for Retinal Indication: APX3330

APX3330 is a late-stage product candidate and 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 confirmed for APX3330 in Q4, 2023.

Prior to our in-licensing of the product candidate, APX3330 had been studied in six Phase 1 and five Phase 2 clinical trials totaling over 440 patients, for inflammatory and oncology indications, and had demonstrated promising evidence of target engagement, tolerability, pharmacokinetics, and durability.

Learn More About APX3330

Intravitreal Product Candidate: APX2009

APX2009, a second generation preclinical product candidate analog of APX3330, is being investigated for use in wet age-related macular degeneration (wAMD). In mouse models of retinal neovascularization, APX2009 was effective in decreasing lesion size as well as improved efficacy against the Ref-1 target compared to APX3330. An intravitreal formulation could present itself as an alternative to APX3330’s oral formulation (as published in the Journal of Pharmacology and Experimental Therapeutics).

Learn More About APX2009

Refractive Product Candidate: Phentolamine Ophthalmic Solution 0.75%

Phase 3 Completed in Multiple Indications

Ocuphire has a partnership with Viatris, Inc. to develop and commercialize Phentolamine Ophthalmic Solution 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. Phentolamine Ophthalmic Solution has been studied in a total of 12 clinical trials 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. Phentolamine Ophthalmic Solution’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. Phentolamine Ophthalmic Solution is currently in Phase 3 for presbyopia and DLD.

Learn More About Phentolamine Ophthalmic Solution

Since 2021, we have reported positive results for five clinical trials for Phentolamine Ophthalmic Solution Eye Drops, including:

A Phase 3 trial in RM (MIRA-2)
A Phase 2 POC trial in Presbyopia (POS alone and POS with 0.4% pilocarpine, VEGA-1)
A Phase 3 trial in DLD (LYNX-1)
A Phase 3 trial in RM (MIRA-3)
A Pediatric safety trial in RM (MIRA-4)

Clinical Trials with a Broad Range of View

Ocuphire has reported positive data from MIRA-2, MIRA-3 registration trials, and MIRA-4 pediatric safety trial for the treatment of RM.

The Company reported positive topline data from a Phase 2 trial of Phentolamine Ophthalmic Solution for treatment of presbyopia, both Phentolamine Ophthalmic Solution as a single agent and Phentolamine Ophthalmic Solution with low dose pilocarpine (“LDP”) 0.4% as adjunctive therapy. Phentolamine Ophthalmic Solution Phase 3 program for the treatment of presbyopia (VEGA-2), for single-agent and combination with Low Dow Pilocarpine (LDP), is planned to initiate by the middle of 2022.

The Company recently announced positive topline results from LYNX-1 Phase 3 trial of Phentolamine Ophthalmic Solution for NVD, and the completion of enrollment in a Phase 2b clinical trial of APX3330 to treat DR/DME (ZETA-1).

View Our Trials

Read our Posters and Publications

We are dedicated to publishing the results of Phentolamine Ophthalmic Solution or POS studies in peer-reviewed medical journals and presenting them at leading eye care conferences around the world. Phentolamine Ophthalmic Solution’s active pharmaceutical ingredient, phentolamine mesylate, is in the alpha-adrenergic antagonist class and has a long history in the literature.

Browse Posters & Publications

Ocuphire Pharma and Viatris Announce FDA Approval of RYZUMVI™ (Phentolamine Ophthalmic Solution) 0.75% Eye Drops for the Treatment of Pharmacologically-Induced Mydriasis Produced by Adrenergic Agonists (e.g., Phenylephrine) or Parasympatholytic (e.g., Tropicamide) Agents