RegeneRx Announces First Patient Enrolled in Phase III Trial

RegeneRx Biopharmaceuticals, Inc. recently announced the first patient has been enrolled in a Phase III clinical trial with RGN-259 (designated GBT-201 in Korea), its sterile, preservative-free eye drop formulation developed for patients with dry eye syndrome, neurotrophic keratopathy (NK), and other corneal disorders. The clinical trial is being sponsored by ReGenTree LLC, a joint venture between RegeneRx and GtreeBNT Co. Ltd. a Korean biopharmaceutical company and is being conducted by Ora Inc., an established contract research organization specializing in the field of ophthalmology.

This is the second late-stage ophthalmic clinical trial with RGN-259 that has begun recently. On September 17, RegeneRx announced that it began enrollment of a 350 patient multicenter Phase IIb/III clinical trial in the US in patients with dry eye syndrome.

The NK trial is a double-masked, placebo-controlled trial being conducted at eight sites, which include major US medical centers. The trial will enroll approximately 46 patients with chronic stage 2 and 3 NK, and is expected to be completed by the end of the first quarter of 2016, or shortly thereafter. The primary endpoint is complete corneal healing in patients using RGN-259 compared to those using placebo. Patients will use the eye drops five times daily for 28 days and will be assessed periodically during treatment, upon completion of treatment, and at 1 week and 2 weeks post-treatment. There will be numerous secondary endpoints that will also be evaluated pursuant to the trial protocol.

“Enrollment of the first patient in our NK study marks another important milestone in the advancement of RGN-259, which we believe should be of significant value for this orphan indication in the US and abroad. Positive results seen in NK patients that had previously failed all standards of care and then treated with RGN-259 supports our confidence that our product candidate can provide relief from this serious disorder,” stated J.J. Finkelstein, RegeneRx’s President and Chief Executive Officer.

Thymosin beta 4 (TB4), the active ingredient in RGN-259, is a first-in-class, naturally occurring molecule that has been the subject of numerous published animal studies in the fields of ophthalmology, dermatology, cardiology, and central nervous system disorders, among others. In addition to the animal studies, the molecule has been tested in three unique formulations in approximately 350 patients and has an excellent safety profile.

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 US 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 US 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.

RegeneRx is focused on the development of a novel therapeutic peptide, Thymosin beta 4, 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 US, and has an extensive worldwide patent portfolio covering its products. For more information, visit www.regenerx.com.