Eleven Biotherapeutics Announces Top-Line Results From Pivotal Phase III Study

Eleven Biotherapeutics, Inc. recently announced top-line results from the OASIS study, the company’s first pivotal Phase III study of its lead drug candidate, EBI-005, in moderate-to-severe dry eye disease. The co-primary endpoints of the Phase III study were the total corneal fluorescein staining score and the patient-reported measurement related to ocular pain and discomfort based on the ocular surface disease index (OSDI), comparing the mean change from baseline at week 12 for treatment with EBI-005 to treatment with vehicle control. In this study, EBI-005 did not meet either of these two co-primary endpoints.

There was no statistically significant difference between the EBI-005 treated group and the vehicle control group on the co-primary endpoints or any secondary endpoints. Patients with dry eye disease in both the EBI-005 and vehicle treatment groups showed statistically significant improvement from baseline on the co-primary endpoints. While the change from baseline on the co-primary endpoints was greater in the vehicle group than the EBI-005 group, the differences between the two groups were not statistically significant and the company believes the differences were not clinically meaningful. EBI-005 was generally well tolerated in the Phase III study with fewer than 5% of patients reporting eye irritation and no treatment-related serious adverse events. Approximately 13% of patients in the study reported some use of artificial tears, with no difference in artificial tear use between the EBI-005-treated and vehicle-control groups. Overall, 92% of patients completed the study, with 33 patients having dropped out of the EBI-005 group, and 20 patients having dropped out of the vehicle control group.

“We are disappointed that our Phase III study in dry eye disease did not meet its primary efficacy endpoints, but we are encouraged that we continue to see a favorable tolerability profile for EBI-005. Our key focus will be on continuing to develop EBI-005 to meet unmet medical needs in allergic conjunctivitis, based on our previously reported Phase II data and scientific rationale supporting EBI-005 as a treatment for allergic conjunctivitis,” said Abbie Celniker, PhD, President and Chief Executive Officer of Eleven Biotherapeutics. “Based on these top-line results, the company does not see an immediate path forward for EBI-005 in dry eye disease, and we will not be initiating the second Phase III study of EBI-005 in dry eye disease that we had planned to start in the second half of this year. However, we need to further assess the results from the OASIS study to fully inform our evaluation of future plans in dry eye disease, including the ongoing Phase III safety study.”

Dr. Celniker commented further, “We have sufficient capital to continue to invest in our promising pipeline and remain confident in the potential for our novel protein therapeutic drug candidates for ocular disease. Our team continues to be committed to moving forward with our upcoming plans, including advancing EBI-005 into a pivotal study for allergic conjunctivitis and continuing to progress EBI-031 for diabetic macular edema.”

Eleven continues to prepare to advance EBI-005 into late-stage clinical development for allergic conjunctivitis, with plans to initiate a pivotal Phase III study in patients with moderate-to-severe allergic conjunctivitis in the second half of 2015. Allergic conjunctivitis is a mechanistically different ocular disease from dry eye disease. The ocular surface inflammation associated with allergic conjunctivitis is initiated by a single, central mechanism of allergen stimulation, while the underlying causes of ocular surface inflammation in dry eye disease are diverse.

In October 2014, Eleven reported results from a Phase II study of EBI-005 in allergic conjunctivitis, which demonstrated statistically significant improvements in ocular itching, tearing, and nasal symptoms in the late-phase allergy response in moderate-to- severe allergic conjunctivitis patients challenged with allergen, using the conjunctival allergen provocation model. The company also plans to continue to invest in its pipeline. Eleven has approximately $59 million of cash and cash equivalents, which the company expects will finance its current plans into the second half of 2016. The company has approximately $15 million in debt.

Eleven Biotherapeutics, Inc. is a clinical-stage biopharmaceutical company with a proprietary protein engineering platform, called AMP-Rx, that it applies to the discovery and development of protein therapeutics to treat diseases of the eye. The company’s therapeutic approach is based on the role of cytokines in diseases of the eye, the company’s understanding of the structural biology of cytokines, and the company’s ability to rationally design and engineer proteins to modulate the effects of cytokines. For more information, visit www.elevenbio.com.