Enrollment target reached; topline data on track for Q3 2018 announcement
NEW YORK, Feb. 09, 2018 (GLOBE NEWSWIRE) — Aquinox Pharmaceuticals, Inc. (“Aquinox”) (NASDAQ:AQXP), a clinical-stage pharmaceutical company discovering and developing novel drug candidates to treat inflammation, inflammatory pain, and blood cancers, today announced that it has met its enrollment threshold of 300 females in its LEADERSHIP 301 clinical trial of rosiptor (AQX-1125) in interstitial cystitis/bladder pain syndrome (IC/BPS) and remains on track for topline data in the third quarter of 2018. The announcement was made as part of the company’s analyst and investor event held today in New York.
“Hitting our enrollment target is a key milestone in our clinical development plan for rosiptor, our lead drug candidate, and we continue to be on track to share topline data from LEADERSHIP 301 in the third quarter of 2018,” said David Main, President & CEO of Aquinox. “We believe the majority of patients with IC/BPS don’t have effective treatment options today. We’re excited for the role rosiptor may be able to play in offering hope to the millions of adults with IC/BPS and look forward to sharing the results of this clinical trial.”
Recruitment for the LEADERSHIP 301 trial was closed at the end of January and as of February 6, 2018, a total of 410 subjects had been enrolled, including 322 females and 88 males. There remain 49 subjects who entered screening in January who will be randomized, if eligible, over the next few weeks, at which time enrollment will be complete.
In addition to the clinical trial enrollment announcement, the analyst and investor event featured a presentation from Dr. Philip Hanno, MD, MPH, clinical professor of urology at the Stanford University School of Medicine and co-chair of the Medical Advisory Board of the Interstitial Cystitis Association (ICA). Dr. Hanno presented on the treatment of IC/BPS, a debilitating condition marked by chronic bladder pain and urinary symptoms, including pain, pressure, and/or discomfort perceived to be related to the urinary bladder as well as urinary frequency, urgency, and/or nocturia.
Barbara Troupin, MD, MBA, Chief Medical Officer and Vice President of Clinical Development and Regulatory Affairs of Aquinox, provided a review of LEADERSHIP 301 enrollment, topline data planning, and the December 7, 2017 meeting minutes released by the U.S. Food and Drug Administration (FDA) Bone, Reproductive, and Urologic Drugs Advisory Committee (BRUDAC).
Abigail Jenkins, Chief Commercial Officer and US Business Head of Aquinox, provided an overview of insights on IC/BPS gathered from market research, the competitive landscape, and the commercial opportunity for rosiptor.
Aquinox’s analyst and investor event in New York was held on Friday, February 9 from 10:00am to 12:00pm EST. A webcast of the presentations is available from the “Events & Presentations” page of Aquinox’s website. Presentation slides are made available on Aquinox’s website following the event.
About the LEADERSHIP 301 Trial
The LEADERSHIP 301 trial is a three-arm, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial assessing the effect of once-daily rosiptor (AQX-1125) 100 mg and 200 mg on bladder pain and urinary symptoms in female and male subjects with IC/BPS. The primary endpoint of the LEADERSHIP 301 trial is the change from baseline at week 12 in maximum daily bladder pain based on an 11-point numeric rating scale (NRS) compared to placebo, recorded by electronic diary. Additional endpoints include urinary symptoms as well as an evaluation of overall improvement with therapy. The LEADERSHIP 301 trial has a 52-week extension period, affording all participating subjects the opportunity for treatment with rosiptor. Subjects have been enrolled at clinical research centers in the United States, Canada, and Europe. Top-line data are anticipated in the third quarter of 2018.
About Interstitial Cystitis/Bladder Pain Syndrome
Interstitial cystitis/bladder pain syndrome (IC/BPS) is a debilitating condition marked by chronic bladder pain and urinary symptoms. Patients may experience recurring pain, pressure, and/or discomfort perceived to be related to the urinary bladder as well as urinary frequency, urgency, and/or nocturia. The pain often worsens upon bladder filling and may be relieved upon bladder emptying. Many patients living with IC/BPS report that it takes a physical, emotional, and psychological toll, greatly impacting employment and social and intimate relationships. There are currently few FDA approved and/or effective treatment options for IC/BPS. Only about 1M of the 5.5M adults in the United States with symptoms of IC/BPS have been diagnosed or are receiving treatment.
Rosiptor (AQX-1125), Aquinox’s lead drug candidate, is a first-in-class, once-daily, oral treatment being studied for its effects on inflammation and inflammatory pain. Rosiptor has a novel mechanism of action, activating SHIP1 (SH2-containing inositol-5′-phosphatase 1), an enzyme that serves to down-regulate inflammation through its role in the PI3K signaling pathway. Rosiptor has been generally well tolerated in multiple completed clinical studies, with more than 395 subjects dosed.
About Aquinox Pharmaceuticals, Inc.
Aquinox Pharmaceuticals, Inc. is a clinical-stage pharmaceutical company developing novel therapeutics for chronic urological conditions marked by inflammation and pain. Aquinox’s lead drug candidate, rosiptor (AQX-1125), is in Phase 3 development for the treatment of patients with interstitial cystitis/bladder pain syndrome (IC/BPS), a condition for which there are currently few FDA approved and/or effective treatment options. Aquinox is focused on leveraging its library of novel compounds that activate SHIP1 to develop therapeutics for application in inflammation, inflammatory pain, and blood cancers. For more information, please visit www.aqxpharma.com.
Cautionary Note on Forward-looking Statements
Certain of the statements made in this press release are forward looking, such as those, among others, relating to: development of rosiptor (AQX-1125), LEADERSHIP 301 and availability of top-line data. These statements are subject to risks and uncertainties that could cause actual results and events to differ materially from those anticipated, including, but not limited to, risks and uncertainties related to: our ability to enroll patients in our clinical trials at the pace that we project; as an organization, we have never conducted a pivotal clinical trial before; the size and growth of the potential markets for rosiptor (AQX-1125) or any future product candidates and our ability to serve those markets; our ability to obtain and maintain regulatory approval of rosiptor (AQX-1125) or any future product candidates; reaching agreement on design of pivotal trials with regulatory authorities and our expectations regarding the potential safety, efficacy or clinical utility of rosiptor or any future product candidates. Actual results or developments may differ materially from those projected or implied in these forward-looking statements. More information about the risks and uncertainties faced by Aquinox is contained in the company’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2017 filed with the Securities and Exchange Commission. Aquinox disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.