Interstitial Cystitis/Bladder Pain Syndrome (IC/BPS) is a chronic condition characterized by bladder pain and urinary symptoms including increased frequency and urgency. Rosiptor (AQX-1125), a SHIP1 enzyme activator, is a novel, once-daily oral medication being evaluated in a global clinical trial (LEADERSHIP 301) for its efficacy and safety in subjects with moderate to severe symptoms of IC/BPS. Preliminary blinded baseline demographics and characteristics from randomized subjects were analyzed to better understand if the population being evaluated was similar between North America and Europe.

LEADERSHIP 301 is a 12-week, randomized, double-blind, placebo-controlled, Phase 3 clinical trial evaluating the efficacy and safety of two doses of rosiptor (100 mg or 200 mg) vs placebo in subjects with moderate to severe symptoms of IC/BPS. Preliminary blinded baseline demographics and disease characteristics assessed included age, race, time since diagnosis, presence or absence of Hunner lesions, average bladder pain based on an 11-point numerical rating scale (NRS), voiding over a 24-hour period, and symptom scores based on the following questionnaires: Bladder Pain/Interstitial Cystitis Symptom Score (BPIC-SS), O’Leary-Sant Interstitial Cystitis Symptom Index (ICSI), and O’Leary-Sant Interstitial Cystitis Problem Index (ICPI).

As of December 31, 2017, 351 subjects had been randomized to either placebo, rosiptor 100 mg, or rosiptor 200 mg. The majority of subjects were female (78%) and white (89% in North America and 99% in Europe). Baseline average bladder pain, voids per 24 hours, BPIC-SS, ICPI, and ICSI scores were similar between subjects in North America and Europe (Table) and representative of a patient population with moderate to severe symptoms. Mean duration since diagnosis was longer in North America (63.5 months) than in Europe (35.1 months); presence of Hunner lesions was higher in subjects from Europe (28.2%) compared to those in North America (11.7%).

Baseline demographics and characteristics of subjects randomized into the LEADERSHIP 301 clinical trial were very similar in North America and Europe for most parameters, and in line with what would be expected based on the general population of patients with IC/BPS. The difference in time since diagnosis may be due to variations in clinical practice or health care system factors rather than in patient characteristics between regions. The higher rates of Hunner lesions in Europe may be due the respective approaches to cystoscopy between North America and Europe, and the way in which Hunner lesions are characterized. In North America, office cystoscopy under local anesthesia is typically used to identify the presence of Hunner lesions, while in Europe, cystoscopy with hydrodistension under general anesthesia is more common, with the procedure inducing bladder wall changes which are commonly characterized as Hunner lesions.

Baseline demographics and characteristics among subjects randomized into the LEADERSHIP 301 clinical trial were similar between North America and Europe. Enrolling patients with homogeneous baseline symptom presentation globally facilitates study data interpretation based on demonstrated symptom improvement, and data are then more likely to be generalizable across multiple regions. The difference in duration of diagnosis and presence of Hunner lesions may be due to variations in health system characteristics, clinical practice, or guideline interpretation between regions.