Phase I/II trials for Ocugen’s gene therapies, OCU410 and OCU410ST, are planned for initiation by the end of 2023 in geographic atrophy and Stargardt disease.
The plans follow the US Food and Drug Administration’s (FDA) approval of the Investigational New Drug (IND) application for the two gene therapies.
Ocugen is also planning for a Phase III trial in retinitis pigmentosa for OCU400 in Q4 2023/Q1 2024. The Phase III trial initiation is contingent on the FDA’s approval of trial plans and Phase I/II data.
OCU410 is an adeno-associated virus containing a gene that encodes human retinoic acid receptor (RAR)-related orphan receptor alpha (RORα). It is planned to be investigated in geographic atrophy, which is the chronic degeneration of the macula during the late stages of age-related macular degeneration.
OCU410ST was granted the orphan drug designation by the FDA for the treatment of Stargardt disease. Also known as ABCA4 retinopathy, the illness is a rare genetic disorder caused by pathogenic variations in the ABCA4 gene leading to fatty build-up on the macula and loss of vision.
OCU400 is an adeno-associated virus serotype 5 capsid containing the gene for human nuclear hormone receptor NR2E3. It is being developed in partnership with China-based CanSinoBIO. It is currently being evaluated in an open-label Phase II trial (NCT05203939).
An open-label Phase I trial (NCT05802329) for OCU200 in patients with diabetic macular oedema was initiated in Q2 2023. One of the cohorts in the trial will evaluate the combination therapy of OCU200 and Novartis’ Lucentis (ranibizumab).
Ocugen announced that the manufacturing facility for its regenerative cell therapy for cartilage, NeoCart, is expected for completion by the end of 2023, with a Phase III trial for the therapy planned for initiation in H2 2024.
The US-based company is looking for government funding for its inhaled mucosal vaccine platform, with the IND application for the treatment expected to be filled in 2024.
As part of their second quarter financials, Ocugen reported cash equivalents of $70.6m on 30 June 2023. The company also reported a loss of $0.10 per common share in Q2 2023.
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