Bladder Pain Syndrome / Interstitial Cystitis

Bladder Pain Syndrome / Interstitial Cystitis ("BPS/IC") is a chronic urinary bladder disease characterized by erosion of the lining and chronic inflammation of the bladder, pelvic pain and increased urinary urgency and/or frequency. For many BPS/IC sufferers, their symptoms of constant pain and urinary frequency 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. Stress or a change in diet has been known to trigger symptoms. BPS/IC affects men and women of all ages. BPS/IC currently affects an estimated 5-12 million people in the United States and is accepted to be one of the most challenging urological conditions without effective therapy.

There is no known cure for BPS/IC, although a number of therapies can relieve symptoms. The only approved oral therapy is an agent, pentosan polysulfate (Elmiron), first approved in 1996, which helps to temporarily restore the bladder lining. Other therapies include such approaches as antihistamines, low dose antidepressants to fight neurogenic pain and analgesics. Most BPS/IC 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 BPS/IC. In addition to oral therapies, direct instillation of drugs into the bladder via catheter (intravesical therapy) has been shown to provide temporary relief of symptoms.

For more information about BPS/IC visit:

Intersitial Cystitis Association (

Interstitial Cystitis Network (

Our Phase 2 trial, known as the LEADERSHIP 201 trial, was a double blind, placebo controlled, Phase 2 clinical trial investigating AQX-1125’s ability compared to placebo to reduce pain and urinary symptoms in 69 BPS/IC patients. The primary endpoint was to measure the difference in the change from baseline in the mean daily bladder pain score based on an 11-point numeric rating scale at two, four and six weeks recorded by electronic diary. Approximately 49% of patients receiving AQX-1125 had a 2-point or greater reduction in average daily pain compared to 34% of patients who received placebo. The overall reduction in average pain with AQX-1125 and placebo on average daily pain recorded by diary was a 2.4 units compared to 1.4 units, a difference of 1.0 which was very close to being statistically significant (p=0.06). However the reduction in maximum daily pain recorded by diary was statistically significant (p=0.03) as was the pain reported to clinic staff. Urinary symptoms were recorded throughout the study by having patients complete questionnaires and record their number of voids each day in the diary. The O’Leary-Sant IC Symptom and Problem Index combined score showed an improvement of 4.4 points more than placebo (p=0.008) and the Bladder Pain/IC Symptom Scale score improved by 4.8 points more than placebo (p=0.012). The results from these questionnaires demonstrate that urinary symptoms improved in patients who received AQX-1125.

For more information on the LEADERSHIP 201 study, please visit

We believe AQX-1125 is a candidate for development in BPS/IC due to the fact that it has demonstrated activity in both preclinical studies and clinical trials relevant to BPS/IC and is delivered to the bladder via the bloodstream and excreted unmetabolized into the urine thereby achieving high concentrations proximate to the inflamed bladder wall and in September of 2016 dosed the first patient in the LEADERSHIP 301 Phase 3 trial with AQX-1125 in IC/BPS. The LEADERSHIP 301 trial 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 AQX-1125 to reduce bladder pain in patients with IC/BPS. The primary endpoint of the LEADERSHIP 301 trial is to measure 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. Secondary 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 AQX-1125. 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 2018.

For more information on the LEADERSHIP 301 study, please visit

"What at first was believed to a UTI, has progressed into a very different diagnosis: interstitial cystitis (IC)"

Read a personal account about the experience of Interstitial cystitis in this The Globe and Mail article from Tuesday, Apr. 05, 2016: What it’s like to constantly feel like your bladder is full