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 Nyxol® is being developed for the reversal of pharmacologically-induced mydriasis, presbyopia, and night (or dim light) vision disturbances (NVD). Our second product candidate, APX3330, is being developed for diabetic retinopathy (DR) and diabetic macular edema (DME).

	Product Candidate	Indication	Development Stage					Anticipated Milestones
	Product Candidate	Indication	Preclinical	Phase 1	Phase 2	Phase 3	Regulatory Approval	Anticipated Milestones
Ocuphire-Focused Development
	0.75% Nyxol® Eye Drop	Reversal of Mydriasis (RM)	Preclinical complete	Phase 1 complete	Phase 2 complete	Phase 3 in progress partnered with Viatris PDUFA Sept 28, 2023	Regulatory Approval not started	Positive MIRA Phase 3 trials (n=500+) Submitted NDA in November 2022 PDUFA September 28, 2023
	0.75% Nyxol® + 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 Nyxol 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 Late 2023 LYRA-1 Long Term Safety Phase 3 Trial (n=400+)
	0.75% Nyxol® 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+)
	APX3330 Oral Pill	Diabetic Retinopathy (DR)/ Macular Edema (DME)	Preclinical complete	Phase 1 complete	Phase 2 in progress Ongoing Trial EOP2 Meeting	Phase 3 not started	Regulatory Approval not started	ZETA-1 Phase 2 data reported in 1Q 2023 (n=103) EOP2 Meeting to Advance P3 2H 2023
Partnering-Focused Development
Partnering-Focused Development	APX2009 Intravitreal	DME, Wet Age-Related Macular Degeneration (wAMD)	Preclinical in progress	Phase 1 not started	Phase 2 not started	Phase 3 not started	Regulatory Approval not started	Seeking partner funding for IND enabling studies and further development

Lead Product Candidate: Nyxol®

Phase 3 Completed in Multiple Indications

We believe that results from Nyxol’s Phase 1, Phase 2, and Phase 3 trials, including the results from ORION-1 and the successfully completed MIRA program and MIRA-1 which were Phase 2b trials that were successfully completed in 2019, and MIRA-2 a Phase 3 registration trial (positive data reported in March 2021), support its current development plan focused on reversal of mydriasis (RM), presbyopia patients, and dim light or night vision disturbance (DLD or NVD). Specifically, patients treated with Nyxol were observed to have significantly decreased pupil diameter and improved low contrast visual acuity in light and dark conditions.

See More About Nyxol

Since 2021, we have reported positive results for five clinical trials for Nyxol® Eye Drops, including:

A Phase 3 trial in RM (MIRA-2)
A Phase 2 POC trial in Presbyopia (Nyxol alone and Nyxol 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)

Given the results of these clinical trials and on regulatory feedback, we expect to file an NDA for Nyxol® with the FDA under the 505(b)(2) drug approval pathway and make additional international filings in parallel.

Lead Product Candidate for Refractive Indications APX3330

Our second product candidate, APX3330, is a twice-a-day oral tablet designed to target multiple pathways relevant to retinal and choroidal vascular diseases such as DR and DME. 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.

In the first quarter of 2021, we initiated ZETA-1, a Phase 2 trial investigating APX3330 for the treatment of moderately severe non-proliferative DR (NPDR) and mild proliferative DR (PDR), as well as patients with DME without loss of central vision. We anticipate advancing APX3330 towards an NDA in the future.

See 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).

See More About APX2009

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 Nyxol for treatment of presbyopia, both Nyxol as a single agent and Nyxol with low dose pilocarpine (“LDP”) 0.4% as adjunctive therapy. Nyxol 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 Nyxol 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 Nyxol studies in peer-reviewed medical journals and presenting them at leading eye care conferences around the world. Nyxol’s active pharmaceutical ingredient, phentolamine mesylate, is in the alpha-adrenergic antagonist class and has a long history in the literature.

Browse Posters & Publications