Drugs / APX3330
Trials 2
| Phase | Registry id | Dates | Indication | Sponsor | Status | Outcome |
|---|---|---|---|---|---|---|
| Phase 11 trial | ||||||
| Phase 1 | NCT03375086 | Jan → Aug 2018 | cancer | Apexian Pharmaceuticals, Inc. | Completed | No outcome recorded |
| Phase 21 trial | ||||||
| Phase 2 | NCT04692688 | Apr 2021 → Jan 2023 | diabetic macular edema, proliferative diabetic retinopathy, severe nonproliferative diabetic retinopathy | Ocuphire Pharma, Inc. | Completed | No outcome recorded |
News releases announcing trial results or a regulatory action · 14
| Date | Issuer | Release |
|---|---|---|
| 2024-12-19 | Ocuphire Pharma, Inc. | Regulatory Opus Genetics Receives FDA Agreement Under Special Protocol Assessment for Phase 3 Trial of APX3330 in Diabetic Retinopathy opusgtx.com ↗
The SPA agreement reflects that the proposed Phase 3 trial design, endpoints, and planned analyses will be adequate to support a New Drug Application (NDA) submission for treatment of NPDR, subject to a successful outcome of the trial and review of all data in the NDA. |
| 2024-05-06 | Ocuphire Pharma, Inc. | Results Ocuphire Pharma Announces Presentation on APX3330 at the ARVO 2024 Annual Meeting opusgtx.com ↗
The presentation, titled Oral APX3330, a Ref-1 Inhibitor, Slows Progression of Diabetic Retinopathy on a Binocular DRSS Person-Level Scale , was delivered by Daniel Su, M.D., a practicing retina specialist at Retina-Vitreous Associates Medical Group (LA Retina) in Los Angeles, California. |
| 2023-11-02 | Ocuphire Pharma, Inc. | Regulatory Ocuphire Pharma Announces Successful End-of-Phase 2 Meeting with FDA for Oral APX3330 in Diabetic Retinopathy opusgtx.com ↗
We are pleased to have FDA agreement on the primary endpoint for Phase 3 pivotal trials of APX3330 which we believe is the most advanced oral therapy currently in development for diabetic retinopathy, |
| 2023-08-28 | Ocuphire Pharma, Inc. | Results Abstract on ZETA-1 Study of APX3330 Receives Award at Women in Ophthalmology (WIO) Summer Symposium opusgtx.com ↗
In the ZETA-1 trial, APX3330 achieved statistical significance in preventing clinically meaningful progression of diabetic retinopathy, as measured by the percentage of subjects with binocular 3-step worsening in Diabetic Retinopathy Severity Scale (DRSS). |
| 2023-07-26 | Ocuphire Pharma, Inc. | Results Ocuphire to Present at American Society of Retina Specialists Annual Meeting and OIS Retina Innovation Summit opusgtx.com ↗
In the ZETA-1 trial, APX3330 achieved statistical significance in preventing clinically meaningful progression of diabetic retinopathy (DR), as measured by the percentage of subjects with binocular ≥3-step worsening in DRSS, the anticipated Phase 3 primary endpoint. |
| 2023-06-27 | Ocuphire Pharma, Inc. | Results Ocuphire Presents APX3330 ZETA-1 Clinical Data in Late-Breaker Session at the American Diabetes Association’s Annual Conference opusgtx.com ↗
In this trial, APX3330 achieved statistical significance in preventing clinically meaningful progression of diabetic retinopathy (DR), as measured by the percentage of subjects with binocular 3-step worsening in DRSS, our anticipated Phase 3 primary endpoint. |
| 2023-06-12 | Ocuphire Pharma, Inc. | Results Ocuphire Presents APX3330 ZETA-1 Data at Clinical Trials at the Summit (CTS) 2023 opusgtx.com ↗
The ZETA-1 trial achieved statistical significance for APX3330 preventing clinically meaningful progression of diabetic retinopathy, as measured by the percentage of subjects with binocular improvement/worsening in DRSS. |
| 2023-02-16 | Ocuphire Pharma, Inc. | Results Ocuphire Announces APX3330 Phase 2 Data Presentations at Retina Meetings opusgtx.com ↗
today announced two presentations featuring efficacy and safety results from the Company’s recently completed ZETA-1 Phase 2 trial of oral APX3330 in diabetic retinopathy (DR) at two medical meetings in February. |
| 2023-01-25 | Ocuphire Pharma, Inc. | Results Ocuphire Announces Topline Results from ZETA-1 Phase 2 Trial of Oral APX3330 in Diabetic Retinopathy and Plans for End-of-Phase 2 Meeting with FDA opusgtx.com ↗ |
| 2022-09-08 | Ocuphire Pharma, Inc. | Results Ocuphire Pharma Announces Last Patient Completes Final Visit in ZETA-1 Phase 2b 24-Week Trial of Oral APX3330 in Diabetic Retinopathy opusgtx.com ↗
These data indicated that oral APX3330 continued to demonstrate a favorable safety profile consistent with the prior trials comprising hundreds of patients. |
| 2022-06-08 | Ocuphire Pharma, Inc. | Results Ocuphire to Present New Interim Masked Safety Data for Oral APX3330 in Diabetics at Annual Macula Society Meeting opusgtx.com ↗ |
| 2022-04-26 | Ocuphire Pharma, Inc. | Results Ocuphire Announces Upcoming Clinical Presentations at ARVO 2022 Medical Meeting and MODLive! 2022 opusgtx.com ↗
The results show a favorable ophthalmic and systemic safety and tolerability profile, consistent with 11 prior safety trials of APX3330 in non-ophthalmic indications. |
| 2022-01-31 | Ocuphire Pharma, Inc. | Results Ocuphire Presenting New Data and Updates on APX3330 and Nyxol® Clinical Programs at Virtual Investor R&D Day opusgtx.com ↗
ZETA-1 study demonstrated a favorable safety profile consistent with prior studies for the first-in-class APX3330 oral drug with additional exposure data in diabetic patients with retinal disease |
| 2020-11-12 | Ocuphire Pharma, Inc. | Results Ocuphire Pharma Announces Two Publications Supporting the APX3330 Program opusgtx.com ↗
The second publication covers a preclinical study outlining the benefits of APX3330 that were shown in mouse models of chronic colitis, an inflammatory condition, in the peer-reviewed journal Inflammatory Bowel Disease . |
All press releases naming this drug 33 releases
Evidence & citations 6 cited values
Every value below carries the sentence it was read from. 5 sources stand behind the page.
| Field | Value | Cited text |
|---|---|---|
| Known as | APX3330 | ClinicalTrials.gov intervention name — accepted as the source's own label NCT03375086 ↗ |
| Known as | E 3330 | ChEMBL registry synonym — accepted as the source's own label CHEMBL578390 ↗ |
| Action | Inhibit | “APX3330 selectively inhibits Ref-1's redox function without affecting its DNA repair role.” PMID 41423797 ↗ Jan 2026 |
| Modality | Small molecule | “small molecular inhibitor, APX3330” PMID 39729027 ↗ Dec 2024 |
| Route | Oral | “Patients will receive APX3330 orally, twice per day until disease progression” NCT03375086 ↗ |
| Target | APEX1 | “APX3330 is an oral agent targeting the redox signaling activity of APE1/Ref-1 (Ref-1)” PMID 41423797 ↗ Jan 2026 |