Aquinox Announces Update on Development Program for AQX-1125 Following Meeting With FDA

Aquinox Pharmaceuticals, Inc. ("Aquinox") (NASDAQ: AQXP), a clinical-stage pharmaceutical company discovering and developing targeted therapeutics in disease areas of inflammation and immuno-oncology, today announced receipt of official minutes from its December 8, 2015 meeting with the Division of Bone, Reproductive and Urologic Products (DBRUP) of the United States Food and Drug Administration (FDA) and provided an update on its plans for development of AQX-1125 for the treatment of patients with bladder pain syndrome/interstitial cystitis (BPS/IC). Discussed at the meeting were guidance and recommendations regarding proposed pivotal trial designs with AQX-1125 for BPS/IC, as well as additional supportive clinical, preclinical and manufacturing activities that could potentially be required for registration.The proposed design for Aquinox's intended first pivotal trial of AQX-1125 is a three-arm, randomized, double-blind, placebo controlled Phase 3 clinical trial, with 12 weeks dosing followed by an open label extension, to assess the efficacy and safety of AQX-1125 in both male and female BPS/IC patients. Patients will be randomized to receive one of two potential doses of AQX-1125 or placebo. DBRUP confirmed that International Council for Harmonization (ICH) requirements for the safety database would apply and recommended increasing male patient participation in Aquinox's development program to support the desired label claim. Consequently, Aquinox is evaluating the most efficient approach to meet safety database requirements and male participation, which may increase the size of the planned Phase 3 trials or require additional Phase 2 trials."The understanding of BPS/IC and its clinical management is evolving and our discussions with the FDA are helpful in planning these important development activities for AQX-1125," said David Main, President and CEO of Aquinox. "Our next steps will be to complete tasks required to support our planned Phase 3 protocol submission to the FDA in Q2 2016."Additional relevant topics discussed included the proposed use of maximum daily pain as a primary endpoint for registration, which DBRUP indicated would be potentially acceptable, subject to consultation with the Division of Anesthesia, Analgesia and Addiction Products upon receipt of a final protocol. Discussion on secondary endpoints was consistent with the company's expectations. Importantly, Aquinox shared with the FDA final audited analysis of the secondary endpoint of urinary frequency from the LEADERSHIP trial demonstrating a statistically significant reduction in urinary frequency with AQX-1125, compared to placebo (p = 0.04) after determining that the previous unaudited analysis had not been normalized for assessing frequency over a 24 hour periods. Both Aquinox and the FDA agreed that improvement in urinary frequency, in addition to reduction in pain, would be an important endpoint in assessing the benefit of AQX-1125 to BPS/IC patients.Aquinox also announced today that it has recently completed a clinical trial demonstrating bioequivalence between AQX-1125 capsules and tablets. All future clinical development will utilize AQX-1125 tablets. Aquinox is also planning to seek Scientific Advice from the European Medicines Agency in the first half of 2016 on topics similar to those discussed with the FDA.