Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA), (the “Company”) a leading developer of RNA interference (RNAi) therapeutics, today announced the dosing of the first human volunteer in its Phase 1 clinical trial of DCR-HBVS, the Company’s investigational GalXCTM-based therapy for the treatment of chronic hepatitis B virus (HBV) infection in adults. The Company anticipates human proof-of-concept data from the Phase 1 trial, which is known as DCR-HBVS-101, in the second half of 2019.
“The dosing of the first human in the DCR-HBVS-101 trial brings us a step closer to the potential availability of an innovative therapy for patients with chronic hepatitis B, a serious liver infection that can lead to advanced hepatic disease or liver cancer if not treated effectively,” said Ralf Rosskamp, M.D., chief medical officer of Dicerna. “We are hopeful that this three-part Phase 1 trial will validate RNA interference as a viable clinical strategy against chronic hepatitis B infection, based upon our encouraging preclinical data on DCR-HBVS.”
DCR-HBVS is comprised of a single GalXC molecule that targets HBV messenger RNAs (mRNAs) within the hepatitis B surface antigen (HBsAg) gene sequence region. Preclinical studies with a standard mouse model of HBV infection showed DCR-HBVS led to greater than 99% reduction in circulating HBsAg, suggesting superior HBsAg suppression (both in magnitude and duration of suppression), compared to targeting within the X gene sequence region.
“RNAi-based therapy has the potential to change the treatment paradigm for patients with chronic HBV infection. By silencing not only the S antigen but also other viral genes, through a powerful and long-acting mechanism, RNAi-based therapy could tip the balance toward allowing the patient’s own immune system to mount an effective immune response. This approach could help eradicate HBV and remove the need for life-long therapy,” said principal investigator Edward Gane, MBCHB, M.D., deputy director and hepatologist of the New Zealand Liver Transplant Unit at Auckland City Hospital and clinical professor of Medicine at the University of Auckland School of Medicine. “Given the encouraging inhibitory activity of DCR-HBVS in animal studies, as well as its favorable preclinical safety profile, we eagerly anticipate the first results from healthy volunteers in the DCR-HBVS-101 trial, and then in the second part of the study, from patients with chronic hepatitis B.”
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