OpRegen® is a therapy in development for the dry form of Age-related Macular Degeneration (dry AMD). OpRegen® cells are introduced into the subretinal space where they are expected to replace missing RPE cells. This therapy is now in a Phase I/IIa dose escalation study to evaluate its safety and efficacy in patients who have an advanced stage of the disease, and has received fast track designation by the FDA.
Currently, there are no FDA approved therapies for dry AMD, the most prevalent form of AMD and one of the major diseases of aging.
This human clinical trial is backed by significant data, including recently-reported data showing that OpRegen® demonstrates high purity, safety, and efficacy, as well as preserves vision and retinal structure when transplanted into the leading animal model of retinal disease. This data was presented at the 2015 annual meeting of the Association for Research in Vision and Opthalmology (ARVO).
Phase I/IIa Trial for Dry AMD
Regulatory clearance from the Food and Drug Administration (FDA) and the Israeli Ministry of Health was granted in order to initiate this Phase I/IIa dose escalation safety and efficacyinical trial of OpRegen® for Geography Atrophy (GA), the severe stage of the dry form of AMD.
The Phase I/IIa clinical trial will evaluate three different dose regimens of OpRegen®. Following transplantation, the patients will be followed for 12 months at specified intervals in order to evaluate the safety and tolerability of the product. Following the initial 12 month period, patients will continue to be monitored at longer intervals.
A secondary objective of the clinical trial will be to examine the ability of transplanted OpRegen® to engraft, survive, and modulate disease progression in the patients. In addition to thorough characterization of visual function, a battery of vision tests will be used to quantify improvements in reducing the progression of the disease.
Significant Unmet Need with No Approved Therapies
- AMD is the leading cause of blindness in people over age 60
- 90% of AMD prevalence is the dry form of the disease, while the wet form afflicts only about 10% of patients. Nevertheless, the market for therapeutics for the wet form sell ~$5B globally
- Dry AMD occurs when the light-sensitive cells in the macula of the eye break down due to the death of a supporting cell type called retinal pigment epithelial cells (RPE), impairing central vision and sometimes leading to blindness
- OpRegen® is designed to integrate under the retina to replace the patient’s missing RPE cells, thereby halting the progress of AMD
- There are currently no approved therapies for dry AMD
Our products and technologies are protected by a strong portfolio of intellectual property.
OpRegen® is being developed in Jerusalem, Israel, and is led by Charles Irving, Ph.D. and Benjamin Reubinoff, M.D., Ph.D.
We have significant support from Israel’s Innovation Authority (formerly known as Israel’s Office of the Chief Scientist or OCS) for the development of OpRegen®:
- In June 2016 Israel’s Innovation Authority awarded a grant of $2.2 million to help finance the development of OpRegen®
- In May 2015 Cell Cure was awarded a grant of $1.6 million from OCS to help finance the development of OpRegen®
- In December 2013 Cell Cure was awarded a grant of approximately $1.74 million from OCS to help finance the development of OpRegen®
In June 2016, the Data Safety Monitoring Board (DSMB) for the OpRegen® Phase I/IIa clinical trial for the treatment of the advanced form of dry age-related macular degeneration (AMD) completed its review of the initial safety data from the first cohort and recommended dose escalation to the second cohort. Enrollment has begun for the second patient cohort, which is receiving a higher, more clinically significant, dose of OpRegen® cells. The Company expects completion of enrollment for the second cohort in 2016 and, if the data are positive, anticipates DSMB approval to proceed to the third cohort by the end of 2016.
OpRegen is comprised of specialized retina cells which were derived from a cell line that is listed on the National Institutes of Health (NIH) Registry and are eligible for use in NIH funded research.