PRINCETON, N.J. (May 14, 2021) – ReGenTree, LLC (‘Company’), a U.S. joint venture company between GtreeBNT and RegeneRx, announced results of additional analysis of the Phase 3 clinical trial, ARISE-3, using RGN-259 for the treatment of dry eye syndrome. The Company also pooled data from all three Phase 3 clinical trials for dry eye.
In ARISE-3, a symptom of efficacy was seen in the improvement of ocular grittiness, one of the pre-specified secondary endpoints. The statistically significant difference was seen at one and two weeks after treatment, and post-exposure to an exacerbated environment after two weeks of treatment with RGN-259 compared to placebo (p = 0.0094, 0.0384, and 0.0079, respectively). Also, statistically significant differences were seen with improvement in ocular discomfort during the first week of treatment (as noted in the subject diary) and with several items of the Ocular Surface Disease Index (OSDI) at Day 15, which were other prespecified secondary symptom endpoints.
In addition, through post-hoc analysis, statistically significant differences were seen in the central corneal fluorescein staining score for sign efficacy at 2 weeks after treatment in a subpopulation of ARISE-3 and in the pooled population of three trials of ARISE-1, -2, and -3 comprised with corneal sum fluorescein staining score at the baseline (ARISE-3, p = 0.0352; Pooled data of three trials, p = 0.0074) and in a subpopulation of ARISE-2 and in the pooled population of three trials comprised with inferior corneal fluorescein staining score and Schirmer’s test score at the baseline (ARISE-2, p = 0.0057; Pooled data of three trials, p = 0.0196). In terms of safety and tolerance, it was confirmed that RGN-259 continued to demonstrate safety in the treatment of dry eye syndrome consistent with previous clinical trials.
Dry eye syndrome is a multifactorial disease with various underlying causes. For treatment, it is important not only to treat the inflammation but also to repair the damaged eye surface and restore tear film homeostasis. These multiple pathologies highlight the importance of drugs with a variety of therapeutic mechanisms along with anti-inflammatory therapeutics. Last year, the FDA released a draft guidance for developing drugs for the treatment of dry eye syndrome. The FDA recommended a number of different endpoints for an objective sign or subjective symptom. The FDA indicated that a statistically significant difference for at least one objective prespecified sign and at least one subjective prespecified symptom could be used for dry eye, even if in separate clinical trials.
ReGenTree is developing RGN-259, which contains thymosin b4 (Tβ4) as a drug substance for ophthalmic indications. RGN-259 regulates inflammation, wound healing, cell protection, cell migration, and apoptosis. The ARISE-3 trial enrolled 700 patients in 20 clinical trial sites nationwide.
A Company official said, "We think that the safety and efficacies of RGN-259 are demonstrated in both signs and symptoms for the treatment of dry eye based on ARISE-3 and the pooled data of all three Phase 3 trials."
Based on the recent reclassification of RGN-259 to a Biologics License Application (BLA) and the draft guidance for dry eye from the FDA, the Company has started discussions with regulatory consulting firms to explore the prospects of a pre-BLA meeting with the FDA using these comprehensive data from all three phase 3 trials. The Company will proceed with a request for a pre-BLA meeting and future development steps will depend on the discussion results. If RGN-259 is approved, the Company anticipates that RGN-259 will have market exclusivity for 12 years as a BLA product.
About ReGenTree, LLC
ReGenTree is a U.S. joint venture company owned by GtreeBNT Co., Ltd, and RegeneRx Biopharmaceuticals, Inc., specifically to develop RGN-259 in both the U.S. and Canada for ophthalmic indications. ReGenTree licensed the rights to RGN-259 from RegeneRx in 2015. Thus far, ReGenTree has sponsored three Phase 3 studies in the U.S. for dry eye, which are ARISE-1 (Phase 2b/3), ARISE-2 (Phase 3), and the recently completed ARISE-3 (Phase 3) for dry eye. In addition to dry eye, the Company completed a Phase 3 study (SEER-1) for neurotrophic keratopathy (NK), an orphan indication in ophthalmology. Its primary endpoint, “the ratio of corneal wound healed patients after four weeks' administration” showed a strong efficacy trend in only 18 patients (p = 0.0656 and p = 0.0400, Fisher’s exact test and Chi square test, respectively) with a similar safety profile as demonstrated in our dry eye trials. For additional information about ReGenTree, please visit www.regentreellc.com.
Any statements in this press release that are not historical facts are forward-looking statements made under the provisions of the Private Securities Litigation Reform Act of 1995. Any forward-looking statements involve risks and uncertainties that could cause actual results to be materially different from historical results or from any future results expressed or implied by such forward-looking statements. There can be no assurance that any current or future clinical trials will result in approved products or future value. There can also be no assurance that the Company will apply for a BLA in the future or that if the Company applies for a BLA, that it will be accepted by the FDA and/or that the product candidate will be approved for marketing in the U.S. Any forward-looking statements in this press release represent the Company's views only as of the date of this release and should not be relied upon as representing its views as of any subsequent date. The Company specifically disclaims any obligation to update this information as a result of future events or otherwise, except as required by applicable law.