Product Pipeline, Commercial, and Financial Update
- Positive Top-Line Results from Phase 3 Clinical Trial of OTIPRIO in Patients with Acute Otitis Externa:
Otonomyannounced last week positive results from its pivotal Phase 3 clinical trial of OTIPRIO in 262 pediatric and adult patients with acute otitis externa (AOE), also known as swimmer's ear. This single administration trial of OTIPRIO met the primary endpoint by showing a statistically significant increase in clinical cure rate compared to sham (no treatment) at Day 8 (p<0.001). OTIPRIO also demonstrated a statistically significant superiority to sham in clinical cure rate at all other time points assessed including Day 4 (p<0.021), Day 15 (p<0.001) and Day 29 (p<0.001), and was well-tolerated. Based on these positive results, Otonomyexpects to submit a supplemental New Drug Application (sNDA) to the U.S. Food and Drug Administration( FDA) in the first half of 2017.
- OTIPRIO® Launch Update: In
March 2016, Otonomyannounced the commercial availability of OTIPRIO (ciprofloxacin otic suspension) in the United Statesfor the treatment of pediatric patients with bilateral otitis media with effusion undergoing tympanostomy tube placement (TTP). During 2016, Otonomy'scommercial efforts focused on establishing patient "access" to OTIPRIO by building awareness and advocacy with physicians, gaining formulary approval in target facilities, and establishing reimbursement. These efforts require contact and coordination with multiple stakeholders including physicians, hospital pharmacists, facility administrators, and payors. In 2017, Otonomy'scommercial priority is "pull-through" which the company defines as the purchase and utilization of OTIPRIO in facilities where the product has received formulary approval.
Commercial launch highlights through December include:
- More than 600 physicians affiliated with over 800 facilities received OTIPRIO for use during ear tube surgery through Otonomy's product sample program.
- OTIPRIO received formulary approval or was purchased by 326 facilities, and reviews were in process at approximately 250 additional hospitals. Otonomy's success rate for formulary approval was 89%, demonstrating the importance of our methodical, multiple stakeholder approach.
- 173 facilities purchased OTIPRIO and 55% of these accounts placed multiple orders. An increasing number of facilities placed regular stocking orders.
- End-user demand totaled 1,172 vials in the fourth quarter compared to 877 in the third quarter.
- Collectively, the facilities that purchased OTIPRIO, had formulary approvals or reviews in process, or are affiliated with physicians who received product samples, account for over 40% of total annual ear tube surgeries in
the United States.
- The J code assigned to OTIPRIO by the Centers for
Medicareand Medicaid Services (CMS) became effective on January 1, 2017. This code is the permanent billing code for OTIPRIO, replacing the previously assigned C code while retaining transitional pass-through payment status. Assignment of the J code will support patient access for use of OTIPRIO during ear tube surgery and for office-based expansion indications when approved.
- Ongoing tracking of reimbursement claims by
Otonomy'sthird party vendor has confirmed payment for OTIPRIO in addition to the TTP procedure itself by multiple payors for both Medicaidand commercially-insured patients.
- Preparing for End-of-Phase 2 Discussion with the FDA Related to Second Label Expansion Indication for OTIPRIO: In
October 2016, Otonomyannounced the successful completion of a Phase 2 clinical trial that evaluated a single administration of OTIPRIO for the treatment of pediatric patients with acute otitis media with tympanostomy tubes (AOMT). The trial demonstrated that both OTIPRIO doses, 6 mg (0.1 mL) and 12 mg (0.2 mL), were well-tolerated and achieved higher and statistically significant (p<0.05) clinical cure rates over sham (no treatment). Otonomybelieves this trial supports the advancement of OTIPRIO into Phase 3 in AOMT and intends to discuss the requirements for such a program with the FDAin the first half of 2017.
- Enrollment in AVERTS-1 and AVERTS-2 Phase 3 Clinical Trials for OTO-104 in Ménière’s Disease Remains Ongoing:
Otonomyis conducting two identical, parallel Phase 3 trials for OTO-104 in patients with Ménière’s disease, AVERTS-1 in the United Statesand AVERTS-2 in Europe. Each trial is a 16-week, prospective, randomized, double-blind, placebo-controlled trial that is expected to enroll approximately 160 patients with unilateral Ménière’s disease. Results from the Phase 3 trials are expected in the second half of 2017.
- Initiated Phase 2 Clinical Trial of OTO-104 in Hearing Loss Indication: In December,
Otonomyinitiated patient screening for a Phase 2 clinical trial evaluating OTO-104 for the prevention of hearing loss in cancer patients undergoing chemotherapy with platinum-based agents. This multicenter, randomized trial is designed to assess the feasibility, safety and efficacy of OTO-104 given by intratympanic administration in subjects at risk for ototoxicity from cisplatin chemotherapy. Approximately 60 subjects will receive an administration of OTO-104 in one ear prior to each of the first three cisplatin treatment cycles with hearing assessed throughout the trial and following the last chemotherapy treatment cycle.
- Phase 1 Clinical Safety Trial of OTO-311 Remains Ongoing: OTO-311 is a single-dose, sustained-exposure formulation of the potent and selective N-Methyl-D-Aspartate (NMDA) receptor antagonist gacyclidine that is in development for the treatment of tinnitus. Several dose cohorts in the Phase 1 clinical safety trial have been completed and there have been no patient tolerability concerns. The trial remains open pending the potential evaluation of additional dose levels in order to establish a maximum tolerated dose. During 2017,
Otonomyexpects to complete the Phase 1 trial and define a plan for Phase 2.
- Financial Update and Operating Expense Guidance for 2017:
Otonomyhad cash, cash equivalents and short-term investments totaling approximately $196 millionon December 31, 2016. Based on preliminary estimates, Otonomyexpects that Non-GAAP operating expenses for 2016 were at the low end of the $100-$105 millionguidance range previously provided. In addition, Otonomyexpects Non-GAAP operating expenses for 2017 in the range of $85-$90 million. The decrease in expenses from 2016 to 2017 is primarily related to a reduction in the clinical trial activity expected this year.
“Our strong momentum continued into 2017 with the positive Phase 3 AOE results announced last week. We expect to build on this success throughout the year by focusing on driving OTIPRIO commercial adoption for ear tube surgery and by delivering top-line results for the OTO-104 AVERTS-1 and AVERTS-2 trials in Ménière’s disease,” said
OTIPRIO (ciprofloxacin otic suspension) is a fluoroquinolone antibacterial indicated for the treatment of pediatric patients with bilateral otitis media with effusion undergoing tympanostomy tube placement. OTIPRIO is administered by a physician as a single 0.1 mL (6 mg) intratympanic administration into each affected ear, following suctioning of the middle ear effusion. The thermosensitive suspension exists as a liquid at or below room temperature and gels when warmed. In two Phase 3 trials, a single intraoperative administration of OTIPRIO demonstrated a statistically significant reduction in the cumulative proportion of study treatment failures compared to tubes alone (p-value <0.001).
Important Safety Information for OTIPRIO
Contraindications: OTIPRIO is contraindicated in patients with a history of hypersensitivity to ciprofloxacin, to other quinolones, or to any of the components of OTIPRIO.
Warnings and Precautions - Potential for Microbial Overgrowth: OTIPRIO may result in overgrowth of nonsusceptible bacteria and fungi. If such infections occur, institute alternative therapy.
Adverse Reactions: Adverse reactions (incidence at least 3%) that occurred in two Phase 3 trials with OTIPRIO vs sham were: nasopharyngitis (5% vs 4%), irritability (5% vs 3%), and rhinorrhea (3% vs 2%).
Use in Specific Populations - Pediatric Use: The safety and effectiveness of OTIPRIO in infants below six months of age have not been established.
Full prescribing information can be found at www.OTIPRIO.com.
Cautionary Note Regarding 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 generally relate to future events or future financial or operating performance of
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