Ocugen to Discontinue Phase 3 oGVHD Trial

Rare Daily Staff

Ocugen said it has decided to discontinue the phase 3 trial of OCU300 for ocular graft vs. host disease (oGVHD) 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.

Graft vs. host disease is a rare disorder can occur when the immune system is deficient or suppressed and is a reaction to donor immune cells against host tissues after receiving a bone marrow transplant or a non-irradiated blood transfusion.

“This is disappointing news, especially for those who suffer from oGVHD.  Our hope was to provide the first treatment for this complex, orphan disease,” said Shankar Musunuri, chairman, CEO and co-founder of Ocugen.

Ocugen will shift its resources to focus on its modifier gene therapy programs for patients suffering from blindness diseases. The company says it remains on track to enter the clinic by next year with its first gene therapy product candidate, OCU400, which targets inherited retinal diseases, and additional pipeline products that are focused on major retinal diseases, which are targeted to enter the clinic by 2022. 

Ocugen’ modifier gene therapy platform is based on nuclear hormone receptors, which have the potential to restore homeostasis, the basic biological processes in the retina and potentially rescue photoreceptors from degeneration. The technology was licensed from the Harvard Medical School. Unlike single-gene replacement therapies, which only target one gene at a time, Ocugen says 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 vector.

OCU400 has received two Orphan Drug designations from the Food and Drug Administration, one for the treatment of NR2E3 mutation-associated retinal diseases and the other for the treatment of CEP290 mutation-associated retinal diseases.

Ocugen believes OCU400 has the potential to eliminate the need for developing more than 150 individual products and provide one treatment option for all retinitis pigmentosa patients.

Photo: Shankar Musunuri, chairman, CEO and co-founder of Ocugen