We assume that the current public health crisis, while severe, is temporary. We intend to focus on long-lasting opportunities
At the time of this writing, the world is gripped by theCOVID-19 pandemic. The consequences for the global economy have been unprecedented, as have been the effects on the biopharmaceutical industry.
What are (and what will be) the effects of the pandemic on the CCXI programs? While this letter is being penned, the pandemic has yet to play out. Accordingly, the long term consequences are still a matter of conjecture. But perhaps CCXI programs may be somewhat less at risk than most others under the circumstances. Prior to the crisis, we had fortunately completed either key clinical work, or collected essential endpoint data (albeit still blinded) from key ongoing trials. Moreover, new clinical development initiatives have yet to be launched. Thus, timing has been perhaps less grievous for CCXI than for many other sponsors.
With avacopan, for example, the new drug application (NDA) preparation in the US for ANCA vasculitis should proceed with little impact (i.e., submitmid-year, per plan). Also, we expect minimal impact on our current drug supply, commercialization readiness processes, etc. For our CCX140 program in FSGS, the data from theLUMINA-1 trial (while still blinded as of this writing) and the large majority of patient visits were completed before the crisis. WithLUMINA-2 in nephrotic syndrome FSGS (a smaller cohort in any case), much of the essential data has already been collected at the sites as well, so we are hoping there will be minimal impact.
Even with the AURORA study of avacopan in HS, it’s likely that the majority of primary endpoint data (again, while still blinded as of this writing) have been collected. That means that even in a worst case scenario it would not necessarily impede the Company’s ability to obtain a meaningful determination of the primary HS efficacy endpoint. We believe the same is true for the ACCOLADE trial of avacopan in C3G.
Overall, I believe that even were the direst of circumstances to transpire in the current global crisis, we at CCXI would be able to proceed with meaningful clinical trial results for our major programs.
Towards the future: small steps and giant leaps
Let’s now review progress toward: A) CCXI’s forward integration plan, and B) sustainable growth and continued potential value creation via additional pipeline development.
First, to forward integration. We have a global strategy to bring our innovative orphan drug candidates to the market. With the positive data in ANCA vasculitis now in hand, we intend to file an NDA in the USmid-year; with filings to follow in Europe and in Japan thereafter. I remind the reader that CCXI owns 100% of the rights for all of our drug candidates in the United States, the largest of all pharmaceutical markets. For US marketing, we are making excellent progress towards commercial readiness and intend to be fully operational for a launch of avacopan in ANCA vasculitis upon approval. Outside the US, we have licensed the international commercial rights for avacopan (and CCX140) to our partner in renal care, Vifor Pharma. In addition to remitting to CCXI significant milestone payments (the next of which will be tied to regulatory events in their territories) Vifor would also pay tiered royalties to CCXI (between the teens and themid-twenties) on any aggregate net sales in their territories.