Otonomy, Inc., a clinical stage biopharmaceutical company developing innovative therapeutics for diseases and disorders of the inner and middle ear, has enrolled its first patient in its pivotal phase IIb study of OTO-104 for the reduction of vertigo in patients with unilateral Ménière's disease. The company also announced that the US Food and Drug Administration (FDA) has granted OTO-104 Fast Track designation, a process designed to facilitate the development and expedite the review of drugs to treat serious conditions and fill an unmet medical need.
This prospective, randomized, double-blind, placebo-controlled phase IIb study is designed to assess the efficacy and safety of OTO-104 for the treatment of Ménière's disease in a total of 140 patients at approximately 50 centres in the United States and Canada. The primary endpoint is the reduction in vertigo frequency at week 12, measured during the four week interval from week nine through week 12, compared to baseline. Otonomy expects to complete patient enrollment of the trial by mid-2014 and announce top line results during the fourth quarter of 2014.
“The commencement of enrollment marks an important clinical milestone for the advancement of OTO-104 as we expect this trial will serve as one of the two studies required to demonstrate efficacy for approval in the United States,” said David A Weber, Ph.D., president and CEO of Otonomy. “The FDA’s Fast Track designation shows that the agency recognizes the potential for this product to address the important unmet medical need in Ménière's disease.”
Added Paul R Lambert, MD, professor and chair of the department of otolaryngology – head and neck surgery at Medical University of South Carolina, and clinical investigator for the phase IIb clinical trial, “Unpredictable spontaneous vertigo is life altering and a source of considerable distress for patients with Ménière's disease, and there are currently no FDA-approved drug therapies. This study may bring us closer to providing a safe and effective treatment option for the many patients with this debilitating disorder.”
The study will also assess the safety and tolerability of OTO-104, which is a proprietary, sustained-exposure otic formulation of dexamethasone. Upon screening, all subjects will enter into a 4-week observational period for a baseline assessment during which subjects will record their vertigo and tinnitus symptoms via a daily diary. Following the lead-in period, eligible subjects will be randomized 1:1 to a single intratympanic injection of 12 mg OTO-104 or placebo (gel vehicle). Tinnitus will also be assessed as a secondary endpoint in the study.
OTO-104 is a sustained-exposure formulation of the steroid dexamethasone that has been designed for intratympanic (IT) injection into the middle ear for the potential treatment of a broad range of inner ear disorders including vertigo and acute onset hearing loss. OTO-104 is based on Otonomy’s proprietary formulation technology which is intended to overcome the limitations associated with the use of unapproved short-acting solutions in the ear. These include limited and variable drug exposure, need for repeat IT injections, and potential for suboptimal efficacy. OTO-104 successfully completed a Phase 1b trial in Ménière's disease patients that demonstrated a clinically meaningful reduction in vertigo frequency and improvement in tinnitus following a single administration compared to placebo. Otonomy has initiated the first of two pivotal clinical studies for OTO-104 in patients with Ménière's disease. OTO-104 has received Fast Track designation from the FDA.
Ménière's disease is a disorder of the inner ear characterized by acute episodes of vertigo, fluctuations in hearing, tinnitus and aural fullness. The underlying cause of Ménière's disease is unknown and there are currently no FDA-approved drug treatments.