Otonomy, a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the inner and middle ear, has achieved target enrollment in its multiple-dose safety study of OTO-104 in patients with Ménière's disease.
This prospective, randomized, placebo-controlled study, designed to evaluate the safety of quarterly dosing of OTO-104, enrolled a total of 128 patients across multiple trial sites in the United Kingdom.
"We would like to thank all of the patients for their participation in this trial, and the clinical investigators for their support in rapidly meeting our enrollment goals," said David A. Weber, Ph.D., president and CEO of Otonomy.
"This study will provide us with important data on the safety of quarterly dosing with OTO-104, a dosing regimen we anticipate will be relevant in the long-term treatment of Ménière's disease patients. Furthermore, we expect that the results of this trial will support future regulatory filings in this indication in conjunction with results from the ongoing phase 2b trial for which we expect to announce results this quarter, as well as a second pivotal single-dose efficacy trial that will be initiated pending positive results."
In the first part of this multiple-dose safety study, patients are randomized to receive two doses of either placebo or 12 mg OTO-104 by intratympanic (IT) injection given at quarterly intervals. Patients completing the double-blind portion of the study will receive two IT injections of OTO-104 at quarterly intervals. Each enrolled patient is expected to participate in the study for a total of one year.
OTO-104, which has been granted Fast Track designation by the US Food and Drug Administration (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 was 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 has completed enrollment with a total of 154 Ménière's disease patients, and results are expected in the second quarter of 2015.
The company believes this trial will serve as one of two pivotal, single-dose efficacy trials required to support US regulatory approval. OTO-104 is also being evaluated in a multiple-dose safety study in the United Kingdom in patients with Ménière's disease, and enrollment has recently been completed with a total of 128 patients.
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 a clinical-stage biopharmaceutical company focused on the development and commercialization of innovative therapeutics for diseases and disorders of the ear disease in the United States.
There is no known cure for Ménière's disease and there are currently no FDA-approved drug treatments.