RegeneRx Biopharmaceuticals, Inc. (OTCQB: RGRX) ("the Company" or "RegeneRx") today announced its U.S. joint venture with G-treeBNT (ReGenTree LLC) has retained Ora, Inc. to conduct a phase 3 clinical trial for the orphan disorder, neurotrophic keratopathy (NK), and a 2b/3 dose-response, confirmatory trial for dry eye syndrome (DES) in the U.S. Both trials will be testing ReGenTree's preservative-free eye drops, RGN-259/GBT201, this year. Each of these eye disorders was being targeted by RegeneRx prior to entering into the joint venture.
Earlier clinical trials with RGN-259 (thymosin beta 4 ophthalmic solution) have demonstrated the drug candidate's ability to significantly improve both signs and symptoms of DES and identified co-primary endpoints to be targeted in the DES trials. Results of a small physician-sponsored phase 2 clinical trial in patients with severe dry eye were recently published in Cornea (2015 May;34(5):491-496) demonstrating statistically significant improvements in patients' signs and symptoms. Positive results with RGN-259 have also been seen in patients with chronic neurotrophic keratopathy, a degenerative disease of the corneal epithelium resulting from impaired corneal innervation that may cause ulceration and perforation of the cornea.
Ora is a recognized leader in ophthalmic product development and has developed a Controlled Adverse Environment (CAE®) model for dry eye allowing rapid recruitment, treatment and evaluation of patients with DES. Ora has significant experience with RGN-259 having previously conducted a 72-patient phase 2 dry eye trial with the drug candidate on behalf of RegeneRx.
Neurotrophic Keratopathy (NK) is a serious degenerative disease of the corneal epithelium (the outside layer of the eye) that is designated an "orphan" disease in the U.S. and EU due to its relatively low prevalence. A reduction in corneal sensitivity or complete corneal anesthesia is the hallmark of this disease and is responsible for producing corneal ulceration, perforation, pain and impaired vision. NK typically has three stages: Stage 1 is characterized by mild, nonspecific signs and symptoms. The viscosity of the tear mucus increases. There is decreased tear break-up time, leading to dry spots on the epithelium. Stage 2 involves a non-healing corneal epithelial defect. The surrounding epithelium becomes loose and the stroma swells and becomes edematous. Characteristic of this stage, the defect forms a punched-out oval or circular shape. Stage 3 often ensues if stages 1 and 2 are not treated appropriately and is characterized by stromal melting leading to perforation and/or ulceration. Estimated U.S. and EU prevalence of all stages of neurotrophic keratopathy is less than 50,000 in each region and less than 10,000 with Stage 2 or 3.
Dry eye occurs when the eye does not produce tears properly, or when the tears are not of the correct consistency and evaporate too quickly. DES can be associated with inflammation of the surface of the eye, the lacrimal gland, or the conjunctiva; any disease process that alters the components of the tears; an increase in the surface of the eye, as in thyroid disease when the eye protrudes forward; or cosmetic surgery. There are numerous causes of DES including medications, autoimmune and other disorders, eye surgeries, allergies, workplace environments, eye injuries, and aging. DES can be temporary or chronic and, if left untreated, can lead to pain, ulcers or scars on the cornea, and some loss of vision. Studies suggest that the incidence of both chronic and acute DES in the U.S. is approximately 15% of the population or nearly 50 million people.
RegeneRx is focused on the development of a novel therapeutic peptide, thymosin beta 4 (TB4), for tissue and organ protection, repair and regeneration. RegeneRx currently has three drug candidates in clinical development for ophthalmic, cardiac and dermal indications, three active strategic licensing agreements in China, Pan Asia (Korea, Japan, and Australia, among others) and the U.S., and has an extensive worldwide patent portfolio covering its products. RGN-259, the Company's TB4-based ophthalmic drug candidate, has been designated an orphan drug for the treatment of neurotrophic keratopathy. RegeneRx was recently allowed by the FDA to move into phase 3 clinical trials with RGN-259 for the treatment of patients with NK. The drug candidate is also being studied in patients with dry eye syndrome in the U.S., China and Korea. RegeneRx, through its U.S. joint venture, ReGenTree LLC, will be developing RGN-259/GBT-201 in the United States. RGN-352, the Company's TB4-based injectable, is a phase 2-ready drug candidate designed to be administered systemically to prevent and restore tissue damage associated with acute events such as heart attacks, strokes, and other similar traumatic injuries. For additional information about RegeneRx please visit www.regenerx.com.
G-treeBNT recently entered the biopharmaceutical industry through carefully selected strategic alliances with distinguished leading bio-venture companies in the US and EU. The strategic alliance with RegeneRx was the first strategic investment in its mission to create a multi-national biopharmaceutical company. Currently the company is developing RGN-259 for Korea (GBT-201), its first drug candidate for dry eye syndrome in 28 Asian and Pacific Rim countries, has filed an IND for a phase 2b/3 clinical trial in Korea, and has assembled an accomplished pharmaceutical team to carry out its mission. G-treeBNT recently entered into a joint venture with RegeneRx to develop RGN-259/GBT-201 in the U.S. for patients with dry eye syndrome and neurotrophic keratopathy.
Ora, Inc. is a global, full-service ophthalmic clinical research and product development firm. Over the past 30 years, it has helped clients earn 39 FDA approvals. Ora supports a wide array of organizations, from start-ups to global pharmaceutical and device companies, to efficiently and successfully bring their new products from concept to market. Ora's pre-clinical and clinical models, unique methodologies and regulatory strategies have been refined and proven across thousands of projects both in the US and internationally. Ora brings together the world's most extensive and experienced network of ophthalmic experts and R&D professionals in order to maximize the value of new product initiatives.