We were heartbroken to learn that Rosiptor (AQX-1125), once believed to be a promising new therapy for interstitial cystitis and bladder pain syndrome, has failed to demonstrate success in their latest clinical trial, the Phase 3 LEADERSHIP 301 study.
In a press conference, David Main, CEO of Aquinox Pharmaceuticals, said “This is a disappointing result for Aquinox and for patients. LEADERSHIP 301 was a robust and well-conducted trial, and we believe the results are definitive. We have conducted a number of sensitivity, subpopulation, and secondary endpoint analyses and none demonstrate a benefit of rosiptor over placebo. We had hoped to deliver better news to the patients and investigators that made the personal commitment to participate in the trial.” Sadly, the company has halted all further IC research and outreach.
Since 2013, I have had the privilege of working with the Aquinox clinical trial team as well staff at regional centers across the USA. They have dedicated thousands of hours to the study of IC and the care of patients. Their passion and determination to help our patient population was undeniable and I know that they, too, were stunned by the results. In earlier studies, Rosiptor performed well but in this vital Phase 3 study it did not show any benefit above placebo. The announcement was also a devastating blow to the financial health of the company. Their shares plunged 85% of value that same day.
Is the door completely closed? No. During the press conference, Mr. Main confirmed that the company would release all of the study data for review. Perhaps, just perhaps, another researcher will connect the dots and help us understand why this study failed. A number of concepts come to mind. I suspect that it could have been a sub-typing error. As we have discussed throughout the past few years, we now understand that there are clear and distinct subtypes for IC. Could they have included too many diverse patients in the study group? Possibly.
The company is also studying Rosiptor for chronic prostatitis and is currently seeking men to participate in those clinical trials! Click here to learn more about that study.
Within an hour of the announcement, I wrote Mr. Main a letter not only to express our gratitude for their years worth of IC research but also with a plea that they reconsider the data based upon what we now believe are clear subtypes of IC.
June 27, 2018
David Main, CEO
Suite 450 – 887 Great Northern Way
Canada, V5T 4T5
Thank you! Thank you for being one of the rare companies who have taken an interest in bladder pain syndrome. Thank you for allocating millions of dollars to study a medication which had great promise. Thank you for trying. Thank you for listening. Thank you for studying. Thank you for every single hour that your team spent working on behalf of patients struggling with interstitial cystitis and bladder pain syndrome. We, the IC/BPS patient community, are grateful for all that you have done and all that you tried to do.
The news today that the latest clinical trial for Rosiptor failed to show a statistically significant reduction in bladder pain was heartbreaking. In talking with your staff, it is very clear that they are stunned. It’s certainly not what we had all hoped for.
We have long known in the IC community that a one treatment fits all approach does not work. Why? We can clearly see distinct variations in the IC patient community. Some patients have Hunner’s lesions, the most clear subtype/phenotype, while others have bladder symptoms driven by hypertonic pelvic floor muscles. Some patients have bladder wall abnormalities while others have a clear central sensitization process driving their bladder sensitivity. (See Payne C. A New Approach To Urologic Chronic Pelvic Pain Syndromes: Applying Oncologic Principles To ‘Benign’ Conditions. Current Bladder Dysfunct Rep. Topical Collection on Pelvic Pain. March 2015)
Before you abandon research with Rosiptor, I hope that your team revisit and re-evaluate the data based upon these potential subtypes. There is still a chance that Rosiptor can be beneficial to MANY patients provided that it is targeted for their specific subtype. Here are a couple of options:
- Can you isolate and study patients who also present with other CNS driven pain syndromes such as IBS and vulvodynia?
- Can you isolate (and likely exclude) patients with hypertonic pelvic floor muscles and/or pudendal neuralgia?
And, though this sounds minor to most, I’ve told every clinical trial coordinator that I’ve worked with in the past two decades that diet modification is critical. If patients participating in a study continue to drink coffees, teas, sodas and acidic juices, etc., the likelihood of any therapy driving symptom improvement is low. We have research demonstrating that certain comestibles trigger IC symptoms, particularly products high in acid and caffeine. As we ponder the concept of tissue injury and repair, the introduction of an acidic irritant to the target organ could hinder if not prevent healing. Throw in the neurostimulatory effect of caffeine, and these patients will have increased frequency regardless of the therapy being used and/or studied.
I had the privilege of working with several of your team members over the years and they were remarkably passionate and professional as they worked on the Rosiptor trials. You could not have had better representatives for the study. Though this latest round of clinical trial data is heartbreaking, I believe that Rosiptor may still be viable. It could provide the foundation for a more specific, refined and phenotype driven therapy that could help patients not only with IC, but also irritable bowel, vulvodynia and other chronic pain conditions.
If there is anything that we can do to help, please don’t hesitate to ask. You and your team have our respect, our admiration and hope for a better future.
Jill H. Osborne MA
Founder & CEO
Interstitial Cystitis Network
PO Box 2159
Healdsburg CA 95448
If you are a patient who participated in the study and improved, please consider writing the company a letter sharing your experience . They truly deserve not only our gratitude but also, perhaps, some inspirational stories to reopen their IC research studies! Working together, we can make a difference!