On Aug. 10, 2018, the United States Food and Drug Administration (FDA) approved our licensor-- Alnylam’s ONPATTRO™ (patisiran) as the first ever FDA-approved RNAi medicine. This historical approval marks the arrival of an entirely new class of therapeutics — RNAi Medicines.
RNA interference is a naturally occurring cellular mechanism of regulating gene expression and is mediated by siRNAs. We aim to develop ASC06 as the first systematically delivered therapeutic drug to treat liver cancer by using RNA interference, a breakthrough approach to drug discovery and development. ASC06 has been designed to silence two genes critical for growth of liver cancer cells — vascular endothelial growth factor (“VEGF”) and kinesin spindle protein (“KSP”). ASC06 has completed phase I and phase I extension clinical trials, which have shown that 50% of patients who received ≥0.7 mg/kg dose achieved stable disease and one patient achieved a complete response. ASC06 is safe and well-tolerated by patients. We expect to initiate phase II clinical trial of ASC06 in China in 2020.
Current Therapies and Limitations
There are several treatment options for liver cancer. Current primary therapies for liver cancer include systematic chemotherapy and targeted small molecule drugs. These therapies show relatively low clinical efficacy and significant adverse side effects. As such, none of these options is considered a cure. In particular, there are no effective systematic therapies to treat late stage liver cancer.
Advantages of ASC06
We believe that, based on clinical trials and pre-clinical studies, ASC06 has the potential to address the limitations of current liver cancer therapies in the following respects:
• Efficacy indication. Clinical trials for ASC06 have shown preliminary activity in patients with advanced solid tumors with liver involvement. One patient with metastatic liver cancer achieved a complete response (illustrated in the diagram below). A total of 12 (50%) of 24 patients who received ≥ 0.7 mg/kg dose achieved stable disease at the end of the two month assessment compared with 1 (8%) of 12 patients who received ≤ 0.4 mg/kg dose. We believe ASC06 has strong potential to be a first-in-class therapy for liver cancer.
• Favorable safety and tolerability profile. The majority of side effects experienced by patients in the phase I clinical trial were grade 1 or 2 in severity. Moreover, seven patients were treated for 11.5 months on average (including phase I), with no new toxicities reported, indicating that ASC06 has a favorable safety profile that permits chronic dosing. We believe the favorable safety and tolerability profile will allow more patients to receive long-term treatment.