Eleven Biotherapeutics, a biopharmaceutical company designing and engineering novel and differentiated protein-based biotherapeutics for ocular diseases, has reported positive topline clinical results from a six-week randomized, double-masked, placebo-controlled phase Ib/IIa study of EBI-005 in patients with dry eye disease (DED). EBI-005 reflects a new approach to the treatment of DED and is the first IL-1 (Interleukin-1) signaling inhibitor designed for topical ocular administration.

In the efficacy results from the study, EBI-005 demonstrated statistically significant improvements in signs and symptoms of dry eye disease compared to baseline. In addition, EBI-005 met the predefined efficacy criteria of the study and showed a differential effect between patients who received EBI-005 and those who received only the vehicle control (EBI-005 drug formulation without the active pharmaceutical ingredient). Data from the study showed that EBI-005 was generally safe and well tolerated. The results from the phase Ib/IIa study of EBI-005 confirm similar observations from a recently-published study validating the blockade of IL-1 as an efficacious mechanism for treating dry eye disease.

The topline data from the clinical study of EBI-005 is being presented at the Masters in Ophthalmology 2013 annual meeting in Florida, and the full data set will also be presented at an upcoming ophthalmologic meeting this fall. Based on these results, Eleven plans to rapidly advance EBI-005 into late stage clinical studies in dry eye disease and additional ocular surface inflammatory diseases, including severe allergic conjunctivitis.

“It is unprecedented to see this magnitude of effect in such an early clinical study of a drug for dry eye disease,” commented Michael H Goldstein, MD, head of Medical Research at Eleven Biotherapeutics and co-director of the Cornea, External Disease and Cataract Service group at Tufts University/New England Eye Centre. “As a practicing ophthalmologist, these results are striking in comparison to both currently available treatments and those in development for dry eye disease.”

“These clinical data of EBI-005 in patients with dry eye disease confirm other recent clinical results, and further validate that IL-1 inhibition targets a fundamental inflammatory process showing that EBI-005 has the potential for beneficial results for patients with dry eye disease and other inflammatory diseases on the surface of the eye,” said Reza Dana, MD, Professor of Ophthalmology at Harvard Medical School and a founder and clinical advisor of Eleven Biotherapeutics.  “The efficacy data from this study with EBI-005 are highly encouraging given the significant effect shown in improvements in both signs and symptoms in patients with dry eye disease, and given the efficacy shown in the differentiation between patients who received EBI-005 compared to those who received the vehicle control.”

The phase Ib/IIa clinical trial study was a double-masked, multi-center, randomized, placebo-controlled study to evaluate two doses of EBI-005 over a six-week period in subjects with dry eye disease. The trial enrolled 74 subjects and was conducted in multiple centers throughout the United States. The primary objective of the study was to determine the safety and tolerability of EBI-005, along with additional assessments of efficacy of EBI-005 in patients with dry eye disease. The study was designed to assess activity of EBI-005 to determine improvements in signs and symptoms of dry eye disease with EBI-005 as compared to baseline as well as differentiation of EBI-005 as compared to vehicle control. The efficacy of EBI-005 in relation to improvement in signs and symptoms of dry eye disease from baseline and EBI-005 compared to vehicle control was measured by the Ocular Surface Disease Index (OSDI) and corneal fluorescein staining.

Dry eye disease (DED) is an ocular surface inflammatory condition induced by desiccating stress and mediated by an inflammatory cascade regulated by IL-1.

Eleven Biotherapeutics’ lead product candidate, EBI-005, is a novel, interleukin-1 (IL-1) receptor antagonist currently entering phase II clinical development as a rationally-designed topical protein for the treatment of inflammatory diseases at the surface of the eye.  Clinical studies in patients with dry eye disease, conducted by both Eleven and collaborators, have validated the blockade of IL-1 as an efficacious mechanism for treating dry eye disease. Eleven’s phase Ib/IIa clinical study of EBI-005 in patients with dry eye disease showed that EBI-005 was generally safe and well-tolerated and demonstrated statistically significant improvements in signs and symptoms of dry eye disease compared to baseline. EBI-005 has been designed to have superior physical, pharmaceutical and therapeutic properties, including topical ophthalmic administration, for the treatment of ocular diseases including dry eye and severe ocular allergy.

Eleven Biotherapeutics creates novel and differentiated biotherapeutics: first-of-a-kind protein-based drugs with significantly improved physical, pharmaceutical, and therapeutic benefits.