Our strategy is to develop and commercialize medicines to treat liver disease and associated fibrotic indications in areas of high unmet medical need. The key elements of our strategy are to:
- Develop emricasan as a treatment for liver diseases in orphan patient populations.
We believe that by inhibiting the caspases responsible for inflammation and apoptosis in the liver, emricasan has the potential to stabilize and improve liver function and to slow liver fibrosis progression in patients with liver disease.
We are focused on developing emricasan for orphan patient populations with high unmet medical needs, including ACLF, CLF and HCV-POLT. We believe that because these indications represent targeted patient populations, the size and cost of the required clinical trials will be manageable for a company of our size.
- Pursue accelerated pathways for regulatory approval in the United States and EU.
Based on our discussions with regulatory authorities in the United Kingdom and Germany, we have designed clinical studies for both HCV-POLT and ACLF that could suffice as single registration studies for each indication in the EU if the results are compelling. The U.S. Food and Drug Administration, or FDA, may require additional registration trials for HCV-POLT. We plan to discuss our registration strategy for ACLF and CLF with the FDA after we have received data from our planned Phase 2b ACLF study. We believe that our planned Phase 2b CLF trial may have the potential to serve as a second registration trial for the ACLF indication, should one be required.
- Build our own sales and marketing capabilities to commercialize emricasan for indications that target orphan patient populations in North America and Europe.
If emricasan is approved for ACLF, CLF and/or HCV-POLT in North America and/or Europe, we intend to build our own commercial organization to market the product for these indications. Specifically, we plan to build a focused, specialized sales force to target the key physicians who treat these indications in these geographic locations, including hepatologists and other liver specialists in tertiary care and transplant centers.
- Evaluate strategic partnerships to maximize the commercial potential of emricasan.
We plan to evaluate opportunities to partner emricasan with pharmaceutical companies that have established sales and marketing capabilities in regions outside of North America and Europe. We may also partner with a pharmaceutical company that has global capabilities to evaluate emricasan in non-orphan indications for which we believe it may also be effective, such as liver fibrosis from viral hepatitis, alcoholic hepatitis and non-alcoholic steatohepatitis, or NASH.