A decision to stop the trial is based on results of a pre-planned interim sample size analysis conducted by an independent committee.
Ocugen Inc., has announced its decision to discontinue the Phase 3 trial of OCU300 for ocular Graft vs. Host Disease (oGVHD).
According to Ocugen, a clinical-stage company focused on discovering, developing and commercializing transformative therapies to treat the whole eye, the decision to stop the trial is based on results of a pre-planned interim sample size analysis conducted by an independent data monitoring committee, which indicated the trial was unlikely to meet its co-primary endpoints upon completion. The study was not stopped based on safety concerns.
The company said it will analyze the full data when it is available.
Shankar Musunuri, chairman, CEO and co-founder of Ocugen, said the news was disappointing, especially for those who suffer from oGVHD.
“Our hope was to provide the first treatment for this complex, orphan disease,” Musunuri said in a statement. “We are grateful to the patients, their families and the investigators who participated in our clinical trial.
Musunuri added that the company remains committed to advancing its transformative therapies and will shift its resources to focus on breakthrough modifier gene therapy platform and novel biologic programs for patients suffering from blindness diseases.
“We remain on track to enter the clinic by next year with our first gene therapy product candidate, OCU400, which targets inherited retinal diseases, Musunuri added in the statement. “Our additional pipeline products, OCU410 and OCU200, which are focused on major retinal diseases, are targeted to enter the clinic by 2022.”
In August, the FDA granted the fourth Orphan Drug Designation for OCI400 a novel gene therapy product candidate in the treatment of PDE6B gene mutation-associated retinal diseases.
According to the company, Ocugen is developing a modifier gene therapy platform to generate therapies designed to fulfill unmet medical needs in the area of retinal diseases, including inherited retinal diseases (“IRDs”).
This modifier gene therapy platform is based on nuclear hormone receptors (“NHRs”), which have the potential to restore homeostasis, the basic biological processes in the retina and potentially rescue photoreceptors from degeneration.
The company said in a statement that this technology was licensed from the Harvard Medical School. Unlike single-gene replacement therapies, which only target one gene at a time, the company said in a statement that it believes OCU400, through its use of NHRs, represents a novel approach in that it may address multiple retinal diseases with one product.
Consisting of a functional copy of the nuclear hormone receptor gene NR2E3, OCU400 is delivered to target cells in the retina using an adeno-associated viral (AAV) vector.
According to the company, OCU400 has the potential to eliminate the need for developing more than 150 individual products and provide one treatment option for all Retinitis Pigmentosa (RP) patients.