Otonomy Reports Meniere’s Phase III Failure

 

Otonomy shares plunged 50.1% at news that its drug to treat Meniere’s disease failed to hit the primary endpoint in its Phase III trial. Meniere’s disease is a condition of the inner ear that can cause vertigo. It can also cause ringing in the ear (tinnitus), hearing loss and a feeling of pressure in the ear. The hearing loss can be permanent.

Otividex is a sustained-exposure formulation of the steroid dexamethasone.

The trial was evaluating Otividex in patients with Meniere’s disease. The primary endpoint was the count of definitive vertigo days (DVD) in Month 3 compared to placebo. The evaluation used the Negative Binomial Model. The data did not hit statistical significance for the per protocol (PP) population.

“We are disappointed by the top-line results for the primary intent-to-treat population and are undertaking an assessment to understand the difference observed with the per protocol analysis,” said David A. Weber, president and chief executive officer of Otonomy. “We thank the many patients, clinical investigators and study site staff who supported this effort. Our focus turns to the strong pipeline we have built as recently highlighted by the successful clinical trial results for OTO-313 in tinnitus and OTO-413 in hearing loss.”

He went on to say, “OTO-313 and OTO-413 each address a large patient population with significant unmet need and no approved drug therapy. These programs provide an attractive opportunity for the company with clinical readouts anticipated in mid-2022. We expect that our existing cash balance will permit us to achieve these clinical readouts as well as advance our preclinical hearing loss programs including OTO-825, a gene therapy for congenital hearing loss.”

As of December 31, 2020, Otonomy reported cash, cash equivalents and short-term investments of $86.3 million. The GAAP operating expenses for the full year 2020 were $42.6 million and non-GAAP operating expenses, which excluded stock-based compensation, for the same period, was $36.5 million.

Otherwise, the most recent clinical results reported by the company were in December 2020. Otonomy announced positive top-line data from the Phase I/II trial of OTO-413 in patients with speech-in-noise hearing difficulty. In that trial, the drug was well-tolerated across all dose cohorts and demonstrated therapeutic activity compared to placebo in multiple speech-in-noise hearing tests at consecutive time points, notably Days 57 and 85. OTO-413 is a sustained exposure formulation of brain-derived neurotrophic factor (BDNF).

Patients in the trial self-reported hearing problems in a noisy environment. It was confirmed by speech-in-noise (SIN) testing. Subjects in the trial could also have up to moderately-severe hearing loss by standard testing in a quiet background. The SIN included Digits-in-Noise (DIN), Words-in-Noise (WIN), and American English Matrix tests. They were administered at baseline and after treatment at Day 15, 29, 57, and 85.

In the trial, six out of nine OTO-413 patients showed a clinically meaningful improvement on at least one of the three SIN tests at both Days 57 and 85 compared to zero out of eight for the placebo group. And three out of nine, or 33%, of OTO-413 subjects demonstrated a clinically-meaningful improvement by two or more different SIN tests at both Days 57 and 85 compared to zero out of eight for the placebo group.

“We are excited to announce these positive top-line clinical results for OTO-413 that support its continued development for patients with hearing loss,” Weber said at the time. “It is also a great way to build on the successful clinical trial results we announced this summer for OTO-313 in tinnitus, and further affirms our leading position in the emerging neurotology field.”