Otonomy, Inc. (Nasdaq:OTIC), a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the inner and middle ear, today announced the enrollment of the first patients in a multiple-dose safety study of OTO-104 initiated in the United Kingdom (U.K.) in patients with Ménière's disease.
The prospective, randomized, placebo-controlled study, designed to evaluate the safety of multiple doses of OTO-104, will enroll 125 patients across multiple trial sites in the U.K. In the first part of the study, patients will be randomized to receive two doses of either placebo or 12 mg OTO-104 by intratympanic (IT) injection given at three month intervals. Patients completing the double-blind portion of the study will be eligible to participate in an open-label extension study where all patients will receive two IT injections of OTO-104 at three month intervals.
"This study marks a number of important milestones for the OTO-104 program and Otonomy," said David A. Weber, president and CEO of Otonomy. "This study is designed to provide us with important data about the safety of repeat dosing with OTO-104 that we believe will occur in the long-term treatment of patients with Ménière's disease, and we expect that the results will support regulatory filings in this indication. Furthermore, the initiation of this study signals the start of our clinical development activities outside of North America."
OTO-104, which has been granted Fast Track designation by the FDA, is a sustained-exposure formulation of the steroid dexamethasone in development for the treatment of Ménière's disease and other inner ear conditions. Otonomy has completed a randomized, prospective, double-blind, placebo-controlled, Phase 1b clinical trial of a single IT injection of OTO-104 in patients with Ménière's disease. Results demonstrated that OTO-104 is well tolerated when administered as a single IT injection, and 12 mg of OTO-104 was associated with clinically meaningful improvements in both vertigo frequency and tinnitus compared to placebo three months after treatment. There were no serious adverse events observed during the clinical trial. A Phase 2b single-dose efficacy trial with 140 Ménière's disease patients is ongoing in the U.S. and Canada, with results expected in the first half of 2015. We believe this trial will serve as one of two pivotal, single-dose efficacy trials required to support U.S. regulatory approval.
About Ménière's Disease
Ménière's disease is a chronic condition characterized by acute vertigo attacks, tinnitus, fluctuating hearing loss and a feeling of aural fullness. Of these symptoms, the vertigo attacks are typically most troubling for patients since they disrupt daily activities and are difficult to anticipate and manage. In general, patients are diagnosed with unilateral Ménière's disease in middle age and symptoms often continue for decades. Over time, the fluctuating hearing loss becomes permanent in many patients, and a subset of patients will develop symptoms in their second ear. According to the National Institute of Deafness and Other Communication Disorders, there are more than 600,000 patients diagnosed with Ménière's disease in the United States. There is no known cure for Ménière's disease and there are currently no FDA-approved drug treatments.
Otonomy is a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the ear. Otonomy's proprietary technology provides sustained exposure of drugs to the middle and inner ear following a single intratympanic (IT) injection. Otonomy has three product candidates in development. AuriPro™ is an antibiotic that has completed Phase 3 clinical trials in pediatric patients with middle ear effusion at the time of tympanostomy tube placement surgery. OTO-104 is a steroid that is in the first of two pivotal clinical studies for the treatment of patients with Ménière's disease. OTO-311 is an NMDA receptor antagonist in development as a treatment for tinnitus. For additional information, please visit www.otonomy.com.
Forward Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements include, among others, statements concerning the multiple-dose safety study of OTO-104, the progress and results of such study, the Company's expectations with respect to such study, the Company's belief that the results of such study will support regulatory filings in this indication, and the Company's belief that the OTO-104 Phase 2b clinical trial will serve as one of two pivotal, single-dose efficacy trials required to support U.S. regulatory approval. Forward-looking statements reflect the Company's current views with respect to certain current and future events and are subject to various risks, uncertainties and assumptions that could cause actual results to differ materially. Risks, and uncertainties include, but are not limited to, the risk that the Company may fail to achieve favorable results in clinical trials or receive regulatory approval; risks of delays in enrollment of patients in clinical trials; and the possibility that product candidates may cause serious adverse side effects or have properties that delay or prevent regulatory approval or limit their commercial profile. The risks, uncertainties and assumptions referred to above are discussed in detail in our reports filed with the Securities and Exchange Commission, including our Prospectus filed on August 13, 2014. The Company does not undertake to publicly update or revise any forward-looking statements to reflect events or circumstances that may arise after the date hereof except as may be required by law.