CAMBRIDGE, Mass.--(BUSINESS WIRE)--Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA), a leading developer of investigational ribonucleic acid interference (RNAi) therapeutics, today reported financial and operational results for the fourth quarter and full year ended December 31, 2016.
“2016 was an important year for Dicerna as we completed the transition to our GalXC™ RNAi technology platform and focused our efforts squarely on driving the development of our GalXC pipeline programs”
“2016 was an important year for Dicerna as we completed the transition to our GalXC™ RNAi technology platform and focused our efforts squarely on driving the development of our GalXC pipeline programs,” said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. “The ability of GalXC to enable development of subcutaneously delivered RNAi therapeutics to silence any disease-causing gene in the liver provides substantial opportunities for growth. Our $70.0 million convertible preferred financing, announced today, to be led by Bain Capital Life Sciences and a syndicate of current and new investors, further supports the broad applicability of our GalXC RNAi technology and will be key to progressing our pipeline and strategic plan over the next two years.
“Our two-pronged strategy includes internal development of product candidates for rare diseases that are genetically and molecularly defined, have high unmet medical need, and offer efficient development paths. Our lead product candidate, DCR-PHXC, for primary hyperoxaluria type 1, as well as our second development program aimed at an undisclosed rare disease, fall into this category. Additionally, we are aggressively pursuing key partnerships for programs that address complex diseases with multiple gene dysfunctions and larger patient populations, including DCR-PCSK9 for hypercholesterolemia and DCR-HBV for hepatitis B virus.”
GalXC™ Program Update
- During 2016, Dicerna continued to optimize its GalXC technology platform and expand its preclinical pipeline of product candidates that are long-acting and highly specific for targets in the liver within its core therapeutic areas of rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases.
- Dicerna has prioritized four therapeutic programs where it believes the probability of success is favorable; these include DCR-PHXC, an undisclosed rare disease program, DCR-PCSK9, and DCR-HBV. Dicerna plans to file its first Investigational New Drug (IND) application and/or Clinical Trial Application (CTA) for its GalXC product candidates at the end of 2017 followed by additional INDs in 2018 and 2019.
- The Company has qualified a significant number of disease-associated genes in clinical indications where it believes an RNAi-based inhibitor may provide substantial benefit to patients, providing expansive therapeutic opportunities, and has developed hits and/or optimized GalXC conjugate inhibitors against almost 40 targets.
- Primary Hyperoxaluria Type 1 (PH1): During 2016, Dicerna announced the launch of DCR-PHXC, its first subcutaneously delivered GalXC clinical candidate for the treatment of patients with PH1. PH1 is a rare inborn error of metabolism in which the liver produces excessive levels of oxalate, which in turn causes severe damage to the kidneys and to other tissues in the body. PH1 is often fatal in the absence of a combined liver-kidney transplant.
- DCR-PHXC is in preclinical development and has advanced into IND-enabling studies. Dicerna intends to file an IND submission and/or CTA for DCR-PHXC in late 2017 and commence Phase 1 clinical trials in the first quarter of 2018.
- To facilitate development of DCR-PHXC, Dicerna continues to advance its Primary HYperoxaluria Observational Study (PHYOS), an international, multicenter, observational study in patients with a genetically confirmed diagnosis of PH1. PHYOS is collecting data on key biochemical parameters implicated in the pathogenesis of PH1. Dicerna hopes to use the data to better understand the baseline PH1 disease state, which will help guide long-term drug development plans.
- Undisclosed Rare Disease Involving the Liver: During the fourth quarter of 2016, Dicerna launched development of a GalXC-based therapeutic with an optimized lead candidate that targets a liver-expressed gene involved in a serious rare disease. Dicerna has selected this target gene and disease based on criteria that include having a strong therapeutic hypothesis, a readily-identifiable patient population, the availability of a potentially predictive biomarker, and high unmet medical need. Dicerna plans to file an IND for this program in the second quarter of 2018.
- Cardiovascular Disease: Based on the Company’s candidate development work during the fourth quarter of 2016, Dicerna is positioned to advance DCR-PCSK9, which targets the PCSK9 gene and is indicated for the treatment of statin-refractory patients with hypercholesterolemia, into formal preclinical development. PCSK9 is a validated target for hypercholesterolemia, and United States Food and Drug Administration-approved therapies targeting PCSK9 using monoclonal antibody (MAb) technology are currently on the market. Based on preclinical studies, Dicerna believes that its GalXC RNAi platform has the potential to produce a PCSK9-targeted therapy with more attractive commercial properties than existing MAb therapies, based on comparatively smaller subcutaneous injection volumes and less frequent dosing, while providing equal or superior control of serum cholesterol.
- Chronic Hepatitis B Virus (HBV): Based on the Company’s candidate development work during the fourth quarter of 2016, Dicerna is positioned to advance DCR-HBV, which targets the HBV directly, into formal preclinical development. Current therapies for HBV rarely lead to a long-term immunological cure as measured by the clearance of HBV surface antigen (HBsAg). Based on preclinical studies, Dicerna believes that its GalXC RNAi platform has the potential to produce an experimental HBV-targeted therapy that eliminates HBsAg expression in HBV patients and can be delivered in a commercially attractive subcutaneous dosing paradigm.
- Additional GalXC Programs: Dicerna has the capacity to launch up to three programs annually, and intends to advance five programs into the clinic by the end of 2019.
