Innovative Therapies to Treat Rare and Underserved Eye Diseases

Innovative Therapies to Treat Rare and Underserved Eye Diseases

December 06, 2019PAP-Q4-19-CL-025

Ocugen offers a robust and diversified ophthalmology portfolio that includes novel gene therapies, biologics and small molecules targeting a range of high-need retinal and ocular surface diseases.

Rare and Underserved Ophthalmic Diseases

Ocugen is targeting inherited retinal diseases (IRDs), age-related macular degeneration (AMD) and ocular GVHD (oGVHD). The main classes of ophthalmic drugs in the marketplace include anti-allergy, anti-glaucoma, anti-inflammatory and anti-vascular endothelial growth factor (anti-VEGF) agents. Currently available FDA-approved therapies are intended for the treatment of wet AMD, diabetic retinopathy (DR) and diabetic macular edema (DME). Due to significant non-responders in these patient populations, there is an unmet medical need, with no FDA-approved therapeutics for many patients suffering with IRDs. Furthermore, there are no FDA-approved treatments available for dry AMD, which represents a population of approximately 9 million patients in the United States. alone.

oGVHD is an autoimmune disease that occurs in approximately 60% of allogeneic bone marrow transplant patients and has no FDA-approved therapy. The condition can lead to significant vision loss and irreparable ocular surface damage restricting daily activities and diminishing quality of life. Diseases like IRDs and oGVHD fall under the orphan disease category, as these are rare diseases that affect fewer than 200,000 people in the United States, and several of these diseases have no approved drugs in the marketplace today. This creates a significant unmet medical need.

Leveraging Proprietary Nanoemulsion Technology

Ocugen’s lead candidate OCU300 is an investigational steroid-free, preservative-free eye drop formulated with brimonidine tartrate using Ocugen's proprietary nanoemulsion technology OcuNanoE™. Phase III studies have been initiated for the treatment of ocular discomfort and ocular redness in patients with oGVHD for OCU300.

Because brimonidine tartrate is already FDA-approved with established safety as an eye treatment, Ocugen is able to develop OCU300 under the accelerated 505(b)(2) regulatory pathway.

Ocugen's OcuNanoE technology enhances the efficacy of this potent anti-inflammatory drug on the eye surface. In addition, it allows the products to be sterile filtered and single-use, thereby eliminating the potentially irritating effects of preservatives.

OCU300 is the only product candidate to be granted Orphan Drug Designation for oGVHD by the FDA, and Ocugen is the only company to advance a therapy into a phase III clinical trial for patients with oGVHD. More than 10 centers are recruiting patients, with top-line results that are expected in the second half of 2020.

Groundbreaking Modifier Gene Therapy Platform

Ocugen’s modifier gene therapy platform was licensed from the Schepens Eye Research Institute of Massachusetts Eye and Ear (Harvard Medical School) and involves targeted delivery and expression of one or more nuclear hormone receptors (NHRs) in disease tissues. NHRs play a vital role in regulating retinal cell development, maturation, metabolism, visual cycle function, survival and maintenance of cellular and molecular homeostatic tissues, including the retina. 

OCU400 and OCU410 are investigational candidates based on the nuclear hormone receptor genes NR2E3 and RORA for the treatment of IRDs and dry AMD, respectively. OCU400 has been granted Orphan Drug Designation by the FDA for two IRDs to date.

Ocugen is developing OCU400 as a gene therapy product for the treatment of rare/orphan IRDs, such as NR2E3 mutation–associated retinal degeneration, CEP290 mutation–associated retinal degeneration, and Rhodopsin mutation–associated retinitis pigmentosa. OCU410 achieves retinal delivery of the RORA gene, which regulates other genes associated with AMD and plays an important role in lipid metabolism and reduction of inflammation.

Given the wide applicability of our gene therapy platform and OCU400’s potential use for many IRDs, we anticipate that we will be able to conduct numerous parallel studies for different disease targets without the need to invest further in manufacturing, minimizing preclinical and safety studies and accelerating the development of additional ophthalmic treatments with reduced cost.

CanSinoBIO will provide all CMC development and clinical supplies for the development of OCU400, with their own capital and resources, allowing Ocugen to focus its capital on pre-clinical toxicology and clinical studies. CanSinoBIO maintains the option to support commercial manufacturing for Ocugen. The agreement also provides commercialization rights to CanSinoBIO in Greater China. With state-of-the-art facilities and a world-class team, CanSinoBIO provides a clear path to advance development and manufacturing processes that reach the clinic.

A Novel Fusion Protein

OCU200 is a novel fusion protein for wet AMD comprising tumstatin, which targets actively dividing epithelial cells of expanding blood vessels, leaving healthy existing vasculature unharmed, and transferrin, which transports tumstatin to the disease site. In preclinical studies, OCU200 demonstrated superior efficacy compared with anti-VEGF therapies.

Becoming a Beacon of Hope

The overall goal of Ocugen is to help all patients around the world with eye diseases. Within 5–10 years, we want patients suffering with unmet ophthalmology needs across the world to look to Ocugen as a beacon of hope.

We are initially focusing on rare and orphan diseases in order to pursue accelerated pathways to commercialization. Once our initial candidates have reached the market, we will be able to shift our focus to diseases affecting large patient populations.

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