RegeneRx Biopharmaceuticals, Inc, and G-treeBNT Co. Ltd, a Korean biotech firm, jointly announced that G-treeBNT is preparing to file an IND and sponsor a phase III clinical trial with RGN-259 (a Thymosin Beta 4–based preservative-free eye drop) in patients with moderate to severe dry eye initially in South Korea, followed by Japan and Australia, with follow-on registrations in certain additional Asian and Pacific Rim countries if appropriate.

The proposed phase lll trial is based on data generated in a 72-patient, double-masked, placebo-controlled trial sponsored by RegeneRx and conducted by Ora, Inc., an ophthalmic CRO in the US and Japan, as well as data generated in a recently completed 9-patient physician-sponsored, double-masked, placebo-controlled clinical trial in patients with severe dry-eye syndrome, both conducted in the US. Both phase II trials resulted in statistically significant sign and symptom improvements in central cornea staining and ocular discomfort.

RGN-259 is a sterile, preservative-free topical eye drop for ophthalmic indications. Based on two phase II clinical trials in moderate and severe dry eye syndrome, RGN-259 was found to show statistically significant improvements in several signs and symptoms of dry eye, as well as positive trends in other outcome measures. The results from these trials reflect RGN-259’s mechanisms of action, appear to support the “protective” effects of RGN-259, and provide RegeneRx with FDA-approvable clinical endpoints to be targeted in future clinical trials.

In the 72-patient trial in the US, patients were diagnosed with moderate dry eye, treated with RGN-259 or placebo four times daily for 28 days, and evaluated using Ora’s proprietary controlled adverse environment (CAE) model. RGN-259 was found to be safe and well-tolerated and patients had statistically significant improvements in several signs and symptoms of dry eye at several time points during the study, offering protective effects when patients were “challenged” in the CAE model.

In a 9-patient severe dry eye study conducted at the Kresge Eye Institute in Detroit, RGN-259 was found to be safe and well-tolerated and met key efficacy objectives with statistically significant sign and symptom improvements, compared to vehicle control, at various time intervals, including 28 days post-treatment, which is consistent with the potentially protective effects observed in the Ora study.

The reduction of fluorescein staining is indicative of a reduction in ocular surface damage of the cornea while improvement in ocular discomfort reflects how the patient’s eye feels. The effects of RGN-259 were seen relatively quickly and there were no adverse events associated with the drug in phase II. These results reflect the known mechanisms of action of RGN-259.

“With two RGN-259 phase II dry eye trials completed in the US, we are very pleased that our strategic partner, G-treeBNT, intends to move into phase III,” stated J.J. Finkelstein, president & chief executive officer of RegeneRx Biopharmaceuticals. “We have been working closely with G-treeBNT on the development of RGN-259 and a successful phase III clinical trial in Asia would be a critical milestone as G-treeBNT has the rights to commercialize RGN-259 in 28 countries in Asia and Pacific Rim including South Korea, Japan and Australia.”

“We have decided to initially file an IND for a phase III clinical trial with RGN-259 in South Korea, followed by a similar strategy in Japan and Australia. If successful, this will facilitate the commercialisation of RGN-259 in other countries in Asia and the Pacific Rim that are covered by our strategic partnership with RegeneRx. Our target is to submit an IND for phase III within 2014 in order to achieve the first patient recruitment before the end of the first quarter of 2015,” stated Won S. Yang, president and chief executive officer of G-treeBNT.