- Bone, Reproductive, and Urologic Drugs Advisory Committee Votes Unanimously
in Favor of Unified IC/BPS Population for Clinical Trials -
SILVER SPRING, Md., Dec. 07, 2017 (GLOBE NEWSWIRE) -- 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 issued the following statements following the U.S. Food and Drug Administration (FDA) Bone, Reproductive, and Urologic Drugs Advisory Committee meeting on interstitial cystitis/bladder pain syndrome (IC/BPS).
At the meeting, the panel voted 15 to 0 that patients with interstitial cystitis and patients with bladder pain syndrome should be combined in clinical trials. This is aligned with the current American Urological Association (AUA) guideline and Aquinox's position that IC/BPS is a single symptom complex defined by bladder pain and urinary symptoms.
The following statement may be attributed to Aquinox President & CEO David Main:
"We greatly appreciate the FDA's interest in this chronic, often debilitating condition and the Agency's commitment to helping accelerate the development of new and effective treatment options, which are desperately needed for patients suffering from IC/BPS. The Committee's vote is aligned with the approach we have taken in Aquinox's clinical development program for rosiptor (AQX-1125). We look forward to additional interaction with the Agency based on today's discussion as we advance our program."
The following statement may be attributed to Aquinox Chief Medical Officer Barbara Troupin, MD, MBA:
"An estimated 5.5 million adults in the US suffer from symptoms consistent with IC/BPS, yet only about a million have been diagnosed or are receiving treatment ? evidence of the clear and pressing need for new, effective, and well-tolerated therapies. We appreciate the patients with IC/BPS who shared their experiences today, illustrating the profound impact the condition has on their social, emotional, and overall quality of life. Aquinox is committed to developing its lead drug candidate rosiptor for the treatment of bladder pain and urinary symptoms associated with IC/BPS, to address the unmet need and relieve these patients' symptoms."
The advisory committee meeting took place at the FDA's White Oak Campus in Silver Spring, MD.
About Rosiptor (AQX-1125)
Rosiptor, Aquinox's lead drug candidate, is a small molecule activator of SHIP1, which is a regulating component of the PI3K cellular signaling pathway. By increasing SHIP1 activity, rosiptor accelerates a natural mechanism that has evolved to maintain homeostasis of the immune system and reduce immune cell activation and migration to sites of inflammation. Rosiptor has demonstrated preliminary safety and favorable drug properties for once daily oral administration in multiple preclinical studies and eight completed clinical trials.
About Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS)
IC/BPS is a chronic inflammatory bladder disease characterized by pelvic pain and increased urinary frequency and/or urgency. For many sufferers, these symptoms are severe and adversely affect all major aspects of their lives, including overall physical and emotional health, employment, social and intimate relationships, and leisure activities. While the cause of the disease remains largely unknown, erosion of the bladder lining is thought to be a significant contributor. IC/BPS is estimated to affect millions of people in the United States and around the world. Most IC/BPS patients continue to suffer this debilitating condition, despite treatment with existing therapies. Most current therapies and those in development are focused solely on symptomatic relief of IC/BPS. Aquinox believes new and innovative therapies that target the underlying disease to reduce the chronic pain and urinary symptoms are needed.
About the LEADERSHIP 301 Trial
The LEADERSHIP 301 trial, which commenced enrollment in September 2016, is a three-arm, multicenter, randomized, double-blind, placebo-controlled Phase 3 clinical trial investigating the ability of 200 mg and 100 mg oral, once daily rosiptor (AQX-1125) to reduce bladder pain in patients with IC/BPS. The primary endpoint of the LEADERSHIP 301 trial is the difference in the change from baseline in the maximum daily bladder pain score based on an 11-point numeric rating scale (NRS) at twelve weeks recorded by electronic diary. Additional endpoints include urinary symptoms, including frequency and nighttime awakenings to void, as well as measures of quality of life. The LEADERSHIP 301 trial also has an additional 52-week extension period, affording all participating patients the opportunity for treatment with rosiptor. An anticipated minimum of 300 female patients, up to a maximum of 600 patients including males, are currently being enrolled at clinical research centers in the United States, Canada and Europe. Top-line data from the LEADERSHIP 301 trial is anticipated in third quarter of 2018.
About Aquinox Pharmaceuticals, Inc.
Aquinox Pharmaceuticals, Inc. is a late clinical-stage pharmaceutical company discovering and developing targeted therapeutics in disease areas of inflammation and immuno-oncology. Our primary focus is anti-inflammatory product candidates targeting SH2-containing inositol-5'-phosphatase 1, or SHIP1, which is a key regulator of an important cellular signaling pathway in immune cells, known as the PI3K pathway. Aquinox's lead drug candidate, rosiptor (AQX-1125), is a small molecule activator of SHIP1 suitable for oral, once daily dosing. In September 2016, we began enrolling patients in a Phase 3 clinical trial of rosiptor in our lead indication, Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS). Other indications are under consideration for future investigation. Aquinox has a broad intellectual property portfolio and pipeline of preclinical drug candidates that activate SHIP1. 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.
Associate Director, Investor Relations
Aquinox Pharmaceuticals, Inc.
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