FDA gives green light to study of cholangiocarcinoma drug

Microscope showing virus to depict study that finds poxvirus tricks cells

Sirnaomics, Inc., a biopharmaceutical company in discovery and development of RNAi therapeutics, announces that the U.S. Food and Drug Administration (FDA) has approved the company’s first oncology IND application. The IND approval will allow for the study of the Company’s lead product candidate, STP705, in patients with advanced cholangiocarcinoma (CCA).

Cholangiocarcinoma (CCA) is the second most common hepatic primary malignancy (accounting for 15-20% of liver cancer).  CCA is also known as bile duct adenocarcinoma and biliary tract cancer. Although it is more common in Asia, its incidence in Europe and North America has increased significantly in recent decades. CCA is characterised by late diagnosis and fatal outcome with 5-year survival ranges from 2-30%.

Sirnaomics’ lead product candidate, STP705, is an anti-cancer/anti-fibrosis siRNA (small interfering RNA) therapeutic. It takes advantage of a dual-targeted inhibitory property and a proprietary polypeptide nanoparticle (PNP)-enhanced delivery system to target cells in the liver. It acts by directly inhibiting tumourigenesis through down regulation of Cancer Associated Fibroblast (CAF) activity and cell proliferation by silencing both TGF-β1 and COX-2 gene expression within the tumor micro-environment.

In 2017, STP705 was granted Orphan Drug Designation from the FDA Office of Orphan Products Development (OOPD) for treatment of Cholangiocarcinoma (CCA) and Primary Sclerosing Cholangitis (PSC). The product has also received both US FDA and Chinese FDA IND approval for Hypertrophic Scar Reduction. The dual-targeted approach of STP705 is expected to have efficacy across many diseases in multiple therapeutic areas.

Founder and CEO of the company, Dr. Patrick Y. Lu said, “Building on our company’s anti-fibrosis therapeutic approach, Sirnaomics’ drug target selection and tumour targeting delivery should support a high rate of success for novel anticancer siRNA therapeutics. The latest success of Alnylam’s Patisiran drug validates the RNAi class of therapeutics and it is our belief that the industry will see great enthusiasm and expanded discovery and development of novel RNAi therapeutics for many disease applications.”

Chief Medical Officer, Michael Molyneaux M.D. said, “Cholangiocarcinoma is a devastating form of liver cancer with very high mortality and no effective therapy.  Based on our preclinical data, we are very hopeful that STP705 will have a positive impact on this disease. This IND approval is in line with our mission to target critical diseases with high unmet clinical need. We expect that our rigorous clinical study design will enable us to gain great insights into the impact of STP705 on CCA.”