Our liver cancer program targets patients in Asia with advanced or metastatic disease (BCLC stage C) that are unable to afford or obtain access to sorafenib (Nexavar®).  Asia has the unfortunate distinction to be the world leader in liver cancer incidence, reporting 80% of global cases. Liver cancer particularly affects people in Southeast Asia. Incidence rates can be as up to 10-20 times higher in Asia than those observed in the United States and Western Europe.  55% of new diagnoses in the world occur in China and Hong Kong alone. Liver cancer is the most prevalent form of cancer in Laos, Mongolia, Taiwan and Thailand.

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In the Asian-Pacific region it is estimated that up to 80% of patients present with unresectable, advanced HCC (BCLC Stage C) and are not eligible for locoregional therapy, surgery or TACE due to tumor size and/or vascular involvement. Advanced liver cancer patients also do not respond to chemotherapy. For these patients, the standard of care for over a decade has been the oral kinase inhibitor drug called sorafenib (Nexavar®) (Bayer, A.G.). 

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Sorafenib was approved based on a randomized, controlled clinical trial (SHARP study) of 602 patients randomized to sorafenib vs. placebo. Median overall survival (OS) was 10.7 months for sorafenib and 7.9 months for placebo (p<0.05).  However, the SHARP study was conducted in a Western population. A separate randomized, controlled Phase III clinical study in Asian patients (226 patients from China, South Korea and Taiwan) comparing sorafenib to placebo (Sorafenib-AP study) demonstrated a OS of 6.5 months for sorafenib compared to 4.2 months for placebo (p<0.05).

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The placebo OS difference between Asian and Western patients (4.2mo vs 7.9 mo) suggests a difference in the disease characteristics in the Asian population.  One significant difference is that the Asian population has an increased prevalence of HBV compared to Western population which may contribute to the increased incidence of HCC and worse OS outcomes observed in Asian patients compared to Western patients.

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In our Phase IIa study in 1 subject in Thailand, 5 of 15 subjects were alive at 1 year and 3 of 15 alive at 2 years. These results indicate that our next generation immunotherapy drug has life extending potential in this indication.  A Phase II/III randomized, controlled study in “real life” advanced liver cancer patients in Taiwan, Thailand, Malaysia and Hong Kong is planned to verify this survival signal.

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In “real life”, the majority of patients in Asia that present with advanced liver cancer are unable to access sorafenib. The median monthly price for sorafenib is around $10,000 and patients usually receive at least 3 months of treatment ($30,000 per treatment).  Even with availability of generic sorafenib, most physicians in “real life” are reluctant to prescribe the drug due to the high cost of treating the side-effects. 

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Accordingly, our next generation immunotherapy is being positioned to fill a high unmet medical need in Asia to have a minimally toxic and effective drug for advanced liver cancer that can be priced at less than $10,000 a treatment.