Phase 1b clinical study data of EBI-005 for treatment of dry eye disease presented at ARVO meeting

Eleven Biotherapeutics, a biopharmaceutical company designing and engineering novel and differentiated protein-based biotherapeutics for ocular diseases, announced today the presentation of data for EBI-005, the first IL-1 (Interleukin-1) signaling inhibitor designed for topical ocular administration, at the Association for Research in Vision and Ophthalmology (ARVO) 2013 Annual Meeting in Seattle, Washington. Eleven Biotherapeutics researchers presented data demonstrating that EBI-005 was safe and well-tolerated in both preclinical testing and in a clinical Phase 1a study in healthy volunteers. These data led to Eleven's Phase 1b clinical study of EBI-005 in subjects with dry eye disease, with top-line data from the Phase 1b study expected in the second half of 2013. In addition, these data support Eleven's new plans to evaluate the potential of EBI-005 in additional ocular surface inflammatory diseases, including severe allergic conjunctivitis.

“Preclinical Development of EBI-005: a Potent Interleukin-1 (IL-1) Receptor-1 (R1) Blocker for Topical Ocular Administration was Safe in GLP Toxicology Studies and Active in a Mouse Model of Dry Eye Disease (DED)”

"EBI-005 is a novel therapeutic that enables the topical administration of a protein on the surface of the eye, and it also represents an important mechanism of blocking IL-1, a critical mediator of the inflammatory cascade that causes both the surface disease and symptoms suffered by patients with dry eye disease," said Abbie Celniker, PhD, Chief Executive Officer of Eleven Biotherapeutics. "With these data, we have successfully established that topical administration of EBI-005 is safe and well-tolerated in healthy volunteers, safety and tolerability are important criteria for patients who suffer from painful symptoms associated with ocular inflammation. We look forward to reporting data on our Phase 1b clinical study of EBI-055 in dry eye disease and moving expeditiously to advance into Phase 2 clinical studies in multiple ocular inflammatory diseases including continuation of the clinical program in dry eye disease as well as a new study in severe allergic conjunctivitis."

In a poster presentation entitled "Early Clinical Development of EBI-005, a Potent Interleukin-1 (IL-1) Receptor-1 (R1) Blocker for Topical Ocular Treatment of Dry Eye Disease (DED)" Eleven Biotherapeutics researchers presented clinical data which demonstrated that:

• EBI-005 was safe and well-tolerated in 16 normal healthy volunteers at two dose levels, with no systemic absorption, antibody formation, effect on vision, burning and irritation or serious adverse events.
• EBI-005 was successfully administered as a topical biologic - a novel approach for applying a protein therapeutic to treat ocular surface disease.
• The safety and tolerability profile demonstrated with EBI-005 in this Phase 1a study informed the design of the ongoing Phase 1b study in patients with DED.

In a poster presentation entitled "Preclinical Development of EBI-005: a Potent Interleukin-1 (IL-1) Receptor-1 (R1) Blocker for Topical Ocular Administration was Safe in GLP Toxicology Studies and Active in a Mouse Model of Dry Eye Disease (DED)" Eleven Biotherapeutics researchers presented preclinical data which demonstrated that:

• EBI-005 distributed well to ocular surface tissues and had low systemic exposure. This feature is important to reduce the risk of immune suppression related adverse events associated with systemic IL-1 antagonism.
• EBI-005 did not accumulate in the eye, even after multiple daily doses, demonstrating the potential for chronic administration in a variety of ocular surface disorders including dry eye disease and severe allergic conjunctivitis.
• High thermal stability shown by EBI-005 creates the potential for an ambient temperature-stable product.

In a poster presentation entitled "Use of galectin-3 fusions to extend the surface residence time of proteins topically applied to the eye" Eleven Biotherapeutics researchers presented data which demonstrated that:

• Galectin-3 leads to longer residence time of model proteins, and modifications to an IL-1 antagonist may result in improved potency, potentially enabling reduced dosing frequency for future topically administered protein-based therapeutics.