Additional analysis of phase III ARISE-3 trial of RGN 259 shows efficacy in dry eye disease.- ReGenTree

ReGenTree announced results of additional analysis of the Phase III clinical trial, ARISE-3, using RGN 259 (timbetasin) for the treatment of dry eye syndrome. The Company also pooled data from all three Phase III 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.Comment: In March 2021 the company announced that ARISE-3 Phase III clinical trial evaluating RGN 259 (timbetasin) eyedrops for the treatment of dry eye syndrome did not meet its primary outcome measures. However, based on the recent reclassification of RGN 259 to a Biologics License Application (BLA) and the draft guidance for dry eye from the FDA, the Company has started discussions with regulatory consulting firms to explore the prospects of a pre-BLA meeting with the FDA using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 III 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.