Trial Outcomes & Findings for Study of the Safety and Efficacy of APX3330 in Diabetic Retinopathy (NCT NCT04692688)

Last Updated: 2026-06-08

Results Overview

Percent of subjects with a ≥ 2-step improvement in DRSS from baseline in the study eye (MITT Population- last observation carried forward). DRSS is scored on a range from 10 to 90 with 13 discrete scores given within that range and where higher scores indicate a worse outcome.

Recruitment status

COMPLETED

Study phase

PHASE2

Target enrollment

103 participants

Primary outcome timeframe

24 Weeks

Results posted on

2026-06-08

Participant Flow

Participant milestones

Participant milestones
Measure	APX3330 Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. APX3330: APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.	Placebo Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. Placebo: Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.
Overall Study STARTED	51	52
Overall Study COMPLETED	45	46
Overall Study NOT COMPLETED	6	6

Reasons for withdrawal

Withdrawal data not reported

Baseline Characteristics

Study of the Safety and Efficacy of APX3330 in Diabetic Retinopathy

Baseline characteristics by cohort

Baseline characteristics by cohort
Measure	APX3330 n=48 Participants Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. APX3330: APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.	Placebo n=50 Participants Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. Placebo: Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.	Total n=98 Participants Total of all reporting groups
Age, Continuous	54.3 years STANDARD_DEVIATION 10.66 • n=9 Participants	58.3 years STANDARD_DEVIATION 9.93 • n=27 Participants	56.3 years STANDARD_DEVIATION 10.44 • n=267 Participants
Sex: Female, Male Female	25 Participants n=9 Participants	25 Participants n=27 Participants	50 Participants n=267 Participants
Sex: Female, Male Male	23 Participants n=9 Participants	25 Participants n=27 Participants	48 Participants n=267 Participants
Ethnicity (NIH/OMB) Hispanic or Latino	26 Participants n=9 Participants	22 Participants n=27 Participants	48 Participants n=267 Participants
Ethnicity (NIH/OMB) Not Hispanic or Latino	22 Participants n=9 Participants	28 Participants n=27 Participants	50 Participants n=267 Participants
Ethnicity (NIH/OMB) Unknown or Not Reported	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Race (NIH/OMB) American Indian or Alaska Native	0 Participants n=9 Participants	4 Participants n=27 Participants	4 Participants n=267 Participants
Race (NIH/OMB) Asian	3 Participants n=9 Participants	1 Participants n=27 Participants	4 Participants n=267 Participants
Race (NIH/OMB) Native Hawaiian or Other Pacific Islander	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Race (NIH/OMB) Black or African American	4 Participants n=9 Participants	6 Participants n=27 Participants	10 Participants n=267 Participants
Race (NIH/OMB) White	38 Participants n=9 Participants	39 Participants n=27 Participants	77 Participants n=267 Participants
Race (NIH/OMB) More than one race	0 Participants n=9 Participants	0 Participants n=27 Participants	0 Participants n=267 Participants
Race (NIH/OMB) Unknown or Not Reported	3 Participants n=9 Participants	0 Participants n=27 Participants	3 Participants n=267 Participants
Region of Enrollment United States	48 participants n=9 Participants	50 participants n=27 Participants	98 participants n=267 Participants

PRIMARY outcome

Timeframe: 24 Weeks

Population: MITT

Outcome measures

Outcome measures
Measure	APX3330 n=48 Participants Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. APX3330: APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.	Placebo n=50 Participants Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. Placebo: Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.
Percent of Subjects With ≥ 2-step Improvement in Diabetic Retinopathy Severity Score (DRSS)	39 Participants	41 Participants

SECONDARY outcome

Timeframe: 24 Weeks

Population: Cumulative values; participants may fall into multiple categories therefor the counts in the categories will not add up to the number analyzed.

Percent of subjects with Binocular Improvement or worsening in DRSS of ≥ 1, ≥ 2, ≥ 3, and ≥ 4 steps from baseline at Week 24 (MITT Population- Last Observation Carried Forward). DRSS is scored on a range from 10 to 90 with 13 discrete scores given within that range and where higher scores indicate a worse outcome.

Outcome measures

Outcome measures
Measure	APX3330 n=48 Participants Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. APX3330: APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.	Placebo n=50 Participants Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. Placebo: Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with No Change (LOCF)	10 Participants	14 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 1 Steps Improvement (LOCF)	13 Participants	14 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 4 Steps Worsening (LOCF)	0 Participants	3 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 3 Steps Worsening (LOCF)	0 Participants	6 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 2 Steps Worsening (LOCF)	4 Participants	6 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 1 Steps Worsening (LOCF)	14 Participants	13 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 2 Steps Improvement (LOCF)	8 Participants	9 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 3 Steps Improvement (LOCF)	5 Participants	3 Participants
Percent of Subjects With Binocular Improvement or Worsening in DRSS at Week 24 Subjects with >= 4 Steps Improvement (LOCF)	4 Participants	1 Participants

Adverse Events

APX3330

Serious events: 4 serious events

Other events: 13 other events

Deaths: 0 deaths

Placebo

Serious events: 12 serious events

Other events: 21 other events

Deaths: 1 deaths

Serious adverse events

Serious adverse events
Measure	APX3330 n=51 participants at risk Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. APX3330: APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.	Placebo n=52 participants at risk Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. Placebo: Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.
Nervous system disorders Dyskinesia	2.0% 1/51 • 6 months	0.00% 0/52 • 6 months
Nervous system disorders Transient ischaemic attack	2.0% 1/51 • 6 months	0.00% 0/52 • 6 months
Nervous system disorders Vertigo CNS origin	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
General disorders Chest pain	2.0% 1/51 • 6 months	1.9% 1/52 • 6 months
General disorders Asthenia	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
General disorders Multiple organ dysfunction syndrome	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
Cardiac disorders Bradycardia	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
Cardiac disorders Coronary artery disease	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
Hepatobiliary disorders Cholelithiasis	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
Infections and infestations COVID-19 pneumonia	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
Infections and infestations Cellulitis	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
Respiratory, thoracic and mediastinal disorders Respiratory failure	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months
Skin and subcutaneous tissue disorders Skin ulcer	2.0% 1/51 • 6 months	1.9% 1/52 • 6 months
Vascular disorders Peripheral embolism	0.00% 0/51 • 6 months	1.9% 1/52 • 6 months

Other adverse events

Other adverse events
Measure	APX3330 n=51 participants at risk Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. APX3330: APX3330, a small-molecule oral tablet, is a Ref-1 inhibitor that can potentially reduce proinflammatory and hypoxic signaling that contributes to several eye diseases.	Placebo n=52 participants at risk Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. Placebo: Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient.
Eye disorders Cataract	5.9% 3/51 • 6 months	1.9% 1/52 • 6 months
Eye disorders Diabetic retinal oedema	3.9% 2/51 • 6 months	9.6% 5/52 • 6 months
Eye disorders Diabetic Retinopathy	2.0% 1/51 • 6 months	11.5% 6/52 • 6 months
Infections and infestations COVID-19	2.0% 1/51 • 6 months	9.6% 5/52 • 6 months
Skin and subcutaneous tissue disorders Pruritis	11.8% 6/51 • 6 months	1.9% 1/52 • 6 months
Investigations SARS-CoV-2 test positive	0.00% 0/51 • 6 months	5.8% 3/52 • 6 months

Additional Information

Barbara Withers, VP Clinical & Regulatory Strategy

Ocuphire

Phone: 248-957-9024

Email: [email protected]

Results disclosure agreements

Principal investigator is a sponsor employee
Publication restrictions are in place

Restriction type: LTE60