Pipeline

CHB is the most common viral infection in the world and an area of substantial unmet medical need.¹ There were over 290 million chronic carriers worldwide as of July 2020 and approximately 30 million individuals become newly infected every year despite the availability of a prophylactic vaccine. In 2015, there were more than 90 million cases of CHB in China alone, while the EU, United States and Japan accounted for nearly 8 million cases. Complications from CHB include cirrhosis, end-stage liver disease, and hepatocellular carcinoma, which collectively resulted in approximately 900,000 deaths in 2015, according to the World Health Organization. CHB is the primary cause of liver cancer worldwide, and the mortality associated with HBV-related liver cancer continues to increase.

We are developing a portfolio of wholly owned drug candidates targeting four distinct mechanisms of action: STOPS™, ASO, siRNA and CAM, each of which has been clinically validated in CHB patients. These drug candidates are designed to reduce S-antigen (HBsAg) and/or HBV DNA/RNA, which may lead to higher rates of functional cure in a significant percentage of CHB patients, relative to existing standard of care treatment options.

CHB is the most common viral infection in the world and an area of substantial unmet medical need.¹ There were over 290 million chronic carriers worldwide as of July 2020 and approximately 30 million individuals become newly infected every year despite the availability of a prophylactic vaccine. In 2015, there were more than 90 million cases of CHB in China alone, while the EU, United States and Japan accounted for nearly 8 million cases. Complications from CHB include cirrhosis, end-stage liver disease, and hepatocellular carcinoma, which collectively resulted in approximately 900,000 deaths in 2015, according to the World Health Organization. CHB is the primary cause of liver cancer worldwide, and the mortality associated with HBV-related liver cancer continues to increase.

We are developing a portfolio of wholly owned drug candidates targeting four distinct mechanisms of action: STOPS™, ASO, siRNA and CAM, each of which has been clinically validated in CHB patients. These drug candidates are designed to reduce S-antigen (HBsAg) and/or HBV DNA/RNA, which may lead to higher rates of functional cure in a significant percentage of CHB patients, relative to existing standard of care treatment options.

CHB is the most common viral infection in the world and an area of substantial unmet medical need.¹ There were over 290 million chronic carriers worldwide as of July 2020 and approximately 30 million individuals become newly infected every year despite the availability of a prophylactic vaccine. In 2015, there were more than 90 million cases of CHB in China alone, while the EU, United States and Japan accounted for nearly 8 million cases. Complications from CHB include cirrhosis, end-stage liver disease, and hepatocellular carcinoma, which collectively resulted in approximately 900,000 deaths in 2015, according to the World Health Organization. CHB is the primary cause of liver cancer worldwide, and the mortality associated with HBV-related liver cancer continues to increase.

We are developing a portfolio of wholly owned drug candidates targeting four distinct mechanisms of action: STOPS™, ASO, siRNA and CAM, each of which has been clinically validated in CHB patients. These drug candidates are designed to reduce S-antigen (HBsAg) and/or HBV DNA/RNA, which may lead to higher rates of functional cure in a significant percentage of CHB patients, relative to existing standard of care treatment options.

One of the effects of improper diet and insufficient exercise is the accumulation of fatty deposits in the liver, referred to as nonalcoholic fatty liver disease (“NAFLD”), which was estimated to occur in approximately 25% of the worldwide population as of 2015. At that time, an estimated 1.5% to 6.5% of the global population was estimated to have an ongoing inflammatory response to these excess fat deposits, which is referred to as nonalcoholic steatohepatitis (NASH).¹¹ In the United States alone, the prevalence of NASH is projected to increase from approximately 16.5 million in 2015 to 27.0 million in 2030.¹²

In the absence of changes in diet and exercise, the inflammation inherent in NASH persists and may result in progressive fibrosis of the liver, which may result in cirrhosis. These fibrotic changes are associated with numerous morbidities including recurrent hospitalization for complications of cirrhosis, hepatocellular carcinoma, need for liver transplant, and death.

The only widely accepted treatment for NASH is weight loss through behavioral modifications such as diet and exercise, which is difficult to achieve at the broad population level. As there are currently no approved drugs to treat NASH, many development programs are underway to identify drugs to address this epidemic. One of the promising MOAs in the NASH space appears to be drugs which preferentially target the beta subtype of the THR receptor (THR-β).

One of the effects of improper diet and insufficient exercise is the accumulation of fatty deposits in the liver, referred to as nonalcoholic fatty liver disease (“NAFLD”), which was estimated to occur in approximately 25% of the worldwide population as of 2015. At that time, an estimated 1.5% to 6.5% of the global population was estimated to have an ongoing inflammatory response to these excess fat deposits, which is referred to as nonalcoholic steatohepatitis (NASH).¹¹ In the United States alone, the prevalence of NASH is projected to increase from approximately 16.5 million in 2015 to 27.0 million in 2030.¹²

In the absence of changes in diet and exercise, the inflammation inherent in NASH persists and may result in progressive fibrosis of the liver, which may result in cirrhosis. These fibrotic changes are associated with numerous morbidities including recurrent hospitalization for complications of cirrhosis, hepatocellular carcinoma, need for liver transplant, and death.

The only widely accepted treatment for NASH is weight loss through behavioral modifications such as diet and exercise, which is difficult to achieve at the broad population level. As there are currently no approved drugs to treat NASH, many development programs are underway to identify drugs to address this epidemic. One of the promising MOAs in the NASH space appears to be drugs which preferentially target the beta subtype of the THR receptor (THR-β).

SARS-CoV-2, the coronavirus responsible for the ongoing COVID-19 pandemic, has caused widespread morbidity and mortality throughout the world. We have initiated multiple discovery programs to identify purpose-built drug candidates with pan-coronavirus activity, including against SAR-CoV-2. Protease inhibitors have proved highly effective in the treatment of other viral infections such as HIV and HCV and we have initiated a collaboration with Prof. Johan Neyts and CD3 at the Rega Institute to develop a protease inhibitor. We have also initiated efforts to identify oligonucleotide drug candidates.

SARS-CoV-2, the coronavirus responsible for the ongoing COVID-19 pandemic, has caused widespread morbidity and mortality throughout the world. We have initiated multiple discovery programs to identify purpose-built drug candidates with pan-coronavirus activity, including against SAR-CoV-2. Protease inhibitors have proved highly effective in the treatment of other viral infections such as HIV and HCV and we have initiated a collaboration with Prof. Johan Neyts and CD3 at the Rega Institute to develop a protease inhibitor. We have also initiated efforts to identify oligonucleotide drug candidates.