ARISE-3 Dry Eye Trial Does Not Meet Primary Outcome Measures

RegeneRx Biopharmaceuticals announced that the ARISE-3 Phase 3 clinical trial evaluating RGN-259 eyedrops for the treatment of dry eye syndrome did not meet its primary outcome measures. However, efficacy was seen in the improvement of ocular grittiness, one of the pre-specified secondary symptom endpoints in the trial. The statistically significant improvement was seen at one and two weeks after treatment, and post-exposure in a controlled adverse environment after two weeks of treatment with RGN-259 compared to placebo (p=0.0104, 0.0307, and 0.0046, respectively).

RGN-259 continued to demonstrate safety in treatment of dry eye syndrome consistent with previous clinical trials. In the ARISE-3 trial, there were no serious adverse events with only mild to moderate adverse events in both the active and placebo arms. The most common ophthalmic adverse event was mild ocular pain upon instillation (RGN-259 6.6% vs. placebo 4.6%).

Based on preliminary post-hoc analyses by GtreeBNT using all of the patients in the three phase 3 trials, the population compromised by a corneal sum fluorescein staining score showed significant improvement in sign efficacy parameters such as fluorescein staining scores in the central, corneal sum, conjunctival sum, and total sum after two weeks of treatment. Additional analyses are being conducted to define efficacies in various sign and symptom parameters using the pooled data of the three phase 3 trials. Comprehensive results will be released when all the data analyses are completed.

Data analysis is expected to be focused on the fast on-set and multifunctional effects of RGN-259 that were shown in all three clinical trials. Based on these results, GtreeBNT and RegeneRx will explore the prospects of a pre-BLA meeting with the FDA using the pooled data or discuss a detailed plan for additional clinical studies, if needed.

"While the trial did not meet its primary outcome measures, there was a statistically significant symptom effect in favor of RGN-259 in a short period of time in addition to its safety. Statistically significant clinical improvements in various signs and symptoms of dry eye syndrome have now been observed in all three phase 3 clinical trials, ARISE-1 -2 and -3, and further analyses are ongoing. We look forward to completing the analyses, reporting results, and meeting with the FDA as soon as practicable," J.J. Finkelstein, RegeneRx president and chief executive, said.