Approval Probability
TA Base Rate
Adjusted LOA
ML Risk
APX3330 · 1 trial · 4 indications
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.
| Arm | Type | Description |
|---|---|---|
| APX3330 | EXPERIMENTAL | Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. |
| Placebo | PLACEBO_COMPARATOR | Five 120 mg tablets will be taken by mouth as follows: 3 tablets every morning and 2 tablets every evening. |
| Name | Type | Description |
|---|---|---|
| APX3330 | DRUG | 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 | DRUG | Placebo tablets are identical to APX3330 tablets except for the absence of the active pharmaceutical ingredient. |
Inclusion Criteria: 1. Males or non-pregnant females ≥ 18 years of age 2. At least one eye with DR graded at least moderately severe to severe NPDR or mild PDR (corresponding to DRSS 47, 53, or 61) 3. BCVA assessed by ETDRS protocol letters score of ≥ 60 letters (Snellen equivalent ≥ 20/63) 4. Body...
Top 6 of 8 competitors
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Ocular Therapeutix Inc | OCUL | 1 | PHASE3 | OTX-TKI |
| AbbVie, Inc. | ABBV | 3 | PHASE2 | Surabgene Lomparvovec |
| Merck & Co., Inc. | MRK | 2 | PHASE3 | MK-8748 |
| Jaeb Center for Health Research | RHHBY | 3 | PHASE3 | Faricimab, RO7663498 |
| 4D Molecular Therapeutics, Inc. | FDMT | 1 | PHASE2 | 4D-150 |
| Adverum Biotechnologies, Inc. | ADVM | 1 | - | ADVM-022 |
APX3330 is an investigational oral small molecule being developed for diabetic retinopathy, including non-proliferative and proliferative forms, as well as diabetic macular edema. It is in Phase 2 clinical development and is not approved for any indication.
APX3330 targets APEX1, also known as apurinic/apyrimidinic endonuclease 1, and acts as an inhibitor of this enzyme. APEX1 is involved in DNA repair and redox regulation, and inhibiting it is the intended mechanism of action for APX3330 in diabetic retinopathy.
APX3330 is being developed by Opus Genetics, Inc., which trades under the ticker IRD. The company is advancing the drug as an oral small molecule candidate for diabetic retinopathy and related retinal conditions.
APX3330 is in Phase 2 clinical development. It has completed one Phase 2 trial and is not FDA approved. It remains an investigational therapy for diabetic retinopathy.
APX3330 has been studied in the Phase 2 trial NCT04692688, titled Study of the Safety and Efficacy of APX3330 in Diabetic Retinopathy. This completed, randomized, double-blind, placebo-controlled trial enrolled 103 participants in the United States.