FDA clears Otonomy's IND for clinical trial of OTO-104 in Meniere's disease patients

Otonomy, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted clearance of the company's Investigational New Drug (IND) application for the clinical trial of OTO-104 in patients with Meniere's disease, a debilitating disorder of the inner ear affecting balance and hearing.

The FDA clearance enables Otonomy to move forward with the first clinical trial of a sustained release drug delivered by direct otic injection. Using an approach called intratympanic (IT) injection, otolaryngologists deposit the drug into the middle ear via a small perforation in the tympanic membrane (eardrum). IT drug delivery results in increased drug exposure to the inner ear where the organs for balance and hearing are located, and minimizes systemic exposure that can cause side effects.

"This marks an important milestone for the company and completes our rapid transition to a clinical-stage organization after less than eighteen months from the start of OTO-104 development," said Jay Lichter, Ph.D., CEO and co-founder of Otonomy. "Furthermore, this advancement demonstrates the utility of our novel, patent-protected formulation approach and enables us to move other development programs toward clinical trials."

The study is a prospective, randomized, placebo-controlled, multicenter, Phase 1b study of OTO-104 given as a single IT injection in subjects with unilateral Meniere's disease. While the primary endpoint of the study is safety and tolerability, a number of efficacy endpoints will be monitored, including the frequency of vertigo attacks experienced by patients pre- and post-treatment.

"Intermittent attacks of vertigo, hearing loss, tinnitus, and aural fullness can be very disruptive and debilitating for Meniere's disease patients," said Jeffrey Harris, M.D., Ph.D., chief of the Division of Otolaryngology-Head & Neck Surgery at University of California San Diego, and a co-founder of Otonomy. "Although there are no FDA-approved drug treatments to control these symptoms, IT steroid injections appear to provide relief for many patients as demonstrated in numerous independent physician-sponsored clinical studies."

OTO-104 is a proprietary formulation of the steroid dexamethasone designed to provide sustained drug release to the inner ear from a single IT injection. A key component of this formulation is a thermosensitive gel which increases residence time in the middle ear thereby enabling higher levels of drug exposure to the inner ear. Preclinical studies confirm the extended release profile of OTO-104 and significant advantage over aqueous formulations which rapidly drain from the middle ear through the eustachian tube. Sustained release is important to maximize therapeutic effect, enhance drug distribution to the inner ear, and reduce response variability.