|Otonomy CEO David Weber|
Otonomy's ($OTIC) treatment for a balance-destroying ear disease missed the mark in a Phase IIb study--but it was close, and the biotech believes the drug did well enough to merit moving forward into late-stage testing.
The drug, OTO-104, is a steroid treatment for a rare inner ear disorder called Ménière's disease, which disrupts hearing and balance. In a Phase IIb trial on 154 patients, Otonomy's candidate reduced the average number of vertigo spells by 61% compared to baseline, while placebo charted a 43% reduction. The difference clocks a p value of 0.067, missing the generally accepted threshold of statistical significance, which is 0.05.
But OTO-104 did reach statistical significance on its secondary goals of lowering the net number of vertigo incidents and reducing their severity, Otonomy said. And the biotech isn't going to let a missed primary endpoint derail its plans. Otonomy is now scheduling an end-of-Phase II meeting with the FDA to discuss the results and remains on track to launch two Phase III trials in Ménière's by year's end, CEO David Weber said.
"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," Weber said in a statement.
Investors seemed not to share his optimism, however, sending Otonomy's shares down roughly 18% overnight.
Otonomy, which pulled off a $100 million IPO last year, is pressing forward with a pipeline of treatments for unmet needs in otology. The biotech's lead drug, AuriPro, is a sustained-release formulation of the antibiotic ciprofloxacin, designed to reduce postoperative complications in pediatric patients undergoing tube-placement surgery. AuriPro aced a pair of Phase III trials last year and is up for a final FDA decision by Dec. 25. The company's third candidate, a preclinical tinnitus treatment dubbed OTO-311, is in line to enter Phase I study this year.
- read the statement