OTO 104 narrowly misses endpoints in Phase II trial for Ménière's disease but likely to progress to Phase III - Otonomy Inc.

Otonomy, Inc. a clinical stage biopharmaceutical company developing innovative therapeutics for diseases and disorders of the inner and middle ear, announced topline results from its Phase IIb trial evaluating OTO-104 in patients with unilateral Ménière's disease. The primary endpoint of the trial was reduction in vertigo frequency during Month 3 following treatment compared to a one month baseline period. In the topline analysis, OTO-104 demonstrated a 61% reduction from baseline in vertigo frequency in Month 3 vs. 43% for placebo with a p value of 0.067, which narrowly missed achieving statistical significance. In addition to Month 3, a similar positive trend was also observed during Month 2 following treatment. The trial achieved statistical significance (p < 0.05) for multiple prospectively defined secondary vertigo endpoints at multiple time points .

"While the trial narrowly missed achieving statistical significance on the primary endpoint, we believe the clear efficacy trends observed along with the multiple secondary endpoints where statistical significance was achieved provide a solid basis to move OTO-104 forward into Phase III testing," said David A. Weber, Ph.D., president and CEO of Otonomy. "We plan to request an End-of-Phase II meeting with the FDA and intend to initiate two parallel Phase III trials in Ménière's disease with the first beginning by the end of 2015."

Comment:Analysts noted the tapering of efficacy into month 4 and suggested that the way forward may be for treatment of patients with more severe vertigo.