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Also known as OCU400 Low Dose, OCU400 Med Dose, OCU400 Second Eye Dosing, Sub-Retinal Administration of OCU400-301
OCU400 · 2 trials · 2 indications
Change in functional vision from baseline to week 52 in pooled analysis from retinitis pigmentosa subjects when study eyes in treatment group were compared to study eyes in untreated control, as measured by the ability of a study participant to navigate through a maze in Luminance Dependent Navigation Assessment (LDNA)
Counts, frequencies and percentages of SDAEs. SDAE is a primary adverse event of interest and defined as AEs and SAEs that are direct subjects to the Study Drug only.
Counts, frequencies and percentages TEAEs. TEAEs are defined as an event that was not present prior to administration of the dose of study drug and present after the dose, or if it represents the exacerbation of an event that was present prior to the dose.
Counts, frequencies and percentages of SAEs including Resulted in Death, Life-threatening, Hospitalization, Disabling/incapacitating, Congenital anomaly or birth defect and medically significant AEs ( AE that did not meet any of the above criteria but could have jeopardized the subject and might have required medical or surgical intervention to prevent one of the outcomes listed above).
| Arm | Type | Description |
|---|---|---|
| RHO Arm | EXPERIMENTAL | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye |
| Gene Agnostic Arm | EXPERIMENTAL | Subjects will receive a sub-retinal injection of OCU400-301 modifier gene therapy product with a concentration of 2.5 x 10\^10 vg/eye |
| Control for RHO Arm | NO_INTERVENTION | Will not receive any active study intervention |
| Control for Gene Agnostic Arm | NO_INTERVENTION | Will not receive any active study intervention |
| Cohort 1 (Low Dose) | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup |
| Cohort 2 (Mid Dose) | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation or RHO mutations subgroup |
| Cohort 3 (High Dose) | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup |
| Pediatric Arm | EXPERIMENTAL | Pediatric subjects will receive the medium dose concentration |
| Phase 2 (High and Medium Dose) | EXPERIMENTAL | Following DSMB confirmation, adult RP subjects with Biallelic autosomal recessive NR2E3 mutations, autosomal dominant NR2E3 mutations, Autosomal dominant RHO mutations will receive a high dose concentration of OCU400 or LCA patients with CEP290 mutation will receive a medium dose concentration of OCU400. |
| Adult Arm | EXPERIMENTAL | Biallelic autosomal recessive NR2E3 mutations subgroup or Autosomal dominant NR2E3 mutation, RHO mutations subgroup will receive a high dose concentration of OCU400 and LCA patients with CEP290 will receive a medium dose concentration of OCU400 |
| Second Eye Dosing | EXPERIMENTAL | Eligible RP participants will be dosed in the untreated fellow eye with a therapeutic dose used in Phase 3 study of OCU400 (1.0x10E11vg/mL in 250 μl ) and will be followed for an additional 48 weeks. |
| Natural History Study (OCU400-104) | NO_INTERVENTION | A Prospective and Retrospective Natural History Study of RP and LCA: This is an observatory study for the prospective natural history of RP and LCA in adult and pediatric subjects. The study will also collect and review retrospective data and ophthalmology examination of natural history and progression of disease for all subjects starting with earliest timepoint on or after the date of their diagnosis of RP or LCA. Subjects will be seen up to a total of four times during the 12 months of the Observational Period, at baseline, 3 months, 6 months and 12 months. A total of up to 100 subjects will be enrolled in the study, including: Approximately 76 newly enrolled subjects consisting of 50 adult RP subjects, 6 adult LCA subjects, 20 pediatric RP/LCA subjects. Up to 24 subjects that reconsent from the OCU400-101 study (subjects from OCU400-101 will provide data on their untreated eye) |
| Name | Type | Description |
|---|---|---|
| Sub-Retinal Administration of OCU400-301 | GENETIC | Sub-Retinal Administration of OCU400-301 |
| OCU400 Low Dose | DRUG | subretinal injection of up to 1.66×10E10 vg/mL |
| OCU400 Med Dose | DRUG | subretinal injection of up to 3.33×10E10 vg/mL |
| OCU400 High Dose | DRUG | subretinal injection of up to 1.66×10E11 vg/mL |
| OCU400 Second Eye Dosing | DRUG | subretinal injection of 1.0x10E11vg/mL in 250 μl |
Inclusion Criteria: 1. Males or females ≥ 3 years of age 2. Confirmed genetic diagnosis of autosomal dominant RHO mutation with clinical diagnosis of RP 3. Clinical Diagnosis of Syndromic or Non-Syndromic RP with/without confirmed genetic diagnosis of any other RP associated mutation (except AD-NR2...
| Company | Ticker | Trials | Lead Phase | Drugs |
|---|---|---|---|---|
| Ocugen Inc | OCGN | 3 | PHASE3 | OCU400, OCU410ST |
| Johnson & Johnson | JNJ | 1 | PHASE3 | Botaretigene sparoparvovec |
| Belite Bio, Inc. ADR | BLTE | 1 | PHASE2 | Tinlarebant |
| Sanofi SA Sponsored ADR | SNY | 1 | PHASE2 | SAR422459 |
| Kiora Pharmaceuticals, Inc. | KPRX | 1 | PHASE2 | KIO-301 |
| ProQR Therapeutics N.V. | PRQR | 1 | PHASE1 | QR-1123 |
OCU400 is an investigational gene therapy being developed for retinitis pigmentosa, a rare inherited retinal disease. It is designed to target the NR2E3 gene and is administered sub-retinally. Ocugen, Inc. is running it in a Phase 3 clinical program, and it holds RMAT and Orphan Drug designations from the FDA.
OCU400 targets NR2E3, a gene that regulates retinal development and photoreceptor function. By delivering a functional copy of NR2E3, the therapy aims to restore retinal homeostasis and potentially benefit patients regardless of the specific genetic mutation causing their retinitis pigmentosa. This gene-agnostic approach is intended to address multiple forms of the disease.
OCU400 is being developed by Ocugen, Inc., a biopharmaceutical company traded on the Nasdaq under the ticker OCGN. Ocugen is running the clinical program for OCU400, including a Phase 3 trial in retinitis pigmentosa and an earlier Phase 1 study that also enrolled patients with Leber congenital amaurosis.
OCU400 is in Phase 3 clinical development for retinitis pigmentosa. The Phase 3 trial, NCT06388200, is active but not recruiting, with an enrollment of 140 patients across the United States and Canada. An earlier Phase 1 study, NCT05203939, is also active but not recruiting. OCU400 is investigational and has not been approved by the FDA.
OCU400 is being studied in two active trials. NCT06388200 is a Phase 3 study in retinitis pigmentosa enrolling 140 patients in the United States and Canada. NCT05203939 is a Phase 1 study assessing safety and efficacy in retinitis pigmentosa and Leber congenital amaurosis, with 22 patients enrolled in the United States. Both are active but not recruiting.
Yes. OCU400 Low Dose, OCU400 Med Dose, OCU400 Second Eye Dosing, and Sub-Retinal Administration of OCU400-301 are alternative names referring to the same investigational gene therapy, OCU400. These names reflect different dosing arms or administration approaches used within the clinical program rather than separate products.