ChemoCentryx, Inc. (NASDAQ:CCXI) Q2 2018 Earnings Conference Call - Final Transcript
Aug 09, 2018 • 05:00 pm ET
largest randomized controlled trial of a new pharmacologic agent ever conducted in the orphan disease of ANCA vasculitis. Second, we enrolled it in record time. We set ourselves the bold, some might say, audacious goal, of enrolling 300 patients by the middle of this year, and we are proud to have attained this goal. As mentioned, ANCA vasculitis is an orphan disease, so we had to look for centers all over the world. We ended up with 316 patients and more than 200 activated clinical sites in 20 countries worldwide. We are very grateful, especially to the patients and the dedicated clinicians who took part and are continuing to take part in this study.
The most important part of landmark, though, is the treatment benefit we are attempting to render this vastly underserved ANCA patient population. ANCA-vasculitis patients suffer a serious total burden of disease and a huge diminishment in quality of life. Ironically, one of the biggest parts of this total burden of disease includes the severe health consequences resulting from the current therapy, not just the debilitating symptoms, caused by the underlying disease.
For about the last 40 years, standard therapy, especially the high chronic doses of corticosteroids over many months and the sicknesses steroids cause have given ANCA patients a lesser of 2 evils option. With avacopan, we hope to convert from a lesser of evils to a greater-of-good approach.
The ADVOCATE trial will evaluate the safety and efficacy of avacopan, following 52 weeks of continuous treatment, showing the impact of avacopan in treating active ANCA vasculitis and also testing the durability of the clinical benefit, which is one of the major problems with the current standard of care.
ADVOCATE is designed to greatly reduce or eliminate the need for chronic high doses of steroids and thus, eliminates steroid therapies' debilitating consequences and provide other marked benefits to patients' quality of life. We believe this should be achievable because of avacopan's highly precise mode of action.
Avacopan attacks directly the underlying cause of ongoing damage in ANCA vasculitis without the need of the immunosuppressive sledgehammer approach that steroid therapy represents. Our ambitious aim is nothing short of revolutionizing the standard of care, making patients better on a sustained basis and dramatically improving their quality of life. In short, avacopan's precise mode of action makes for pragmatic precision medicine.
By the way, some patients' enrollment in ADVOCATE continues in Japan in order to support a regulatory application there. We expect Japan enrollment to conclude later this year so that all Japanese patients complete treatment before our planned release of top line data. Thus, the 52 weeks of treatment clock is ticking and we plan to release top line data from the ADVOCATE trial in the fourth quarter of 2019. We expect that successful top line data would then form the basis of a new drug application, or NDA, during the first half of 2020.
Meanwhile, a Conditional Marketing Authorization application for avacopan in the treatment of ANCA vasculitis