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Dicerna Reports Third Quarter 2016 Financial and Operational Results


Dicerna Pharmaceuticals, Inc. (DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, today reported financial and operational results for the third quarter ended September 30, 2016.

“This quarter, we made the strategic decision to focus our resources on advancing our GalXC™ subcutaneous RNAi technology platform and to prioritize the product candidates emerging from this powerful product engine,” said Douglas Fambrough, Ph.D., president and chief executive officer of Dicerna. “Our decision was based on the strength of the GalXC preclinical data and broad opportunities this technology enables in our core therapeutic areas of rare disease, chronic liver diseases, cardiovascular diseases and liver infectious diseases. In preclinical models across these areas, the potency and duration of action results for the GalXC platform have consistently supported the potential for target gene silencing in the liver with a simple, infrequent, subcutaneous injection paradigm. As a result, we transitioned our primary hyperoxaluria program to the GalXC-based DCR-PHXC from DCR-PH1, as we believe DCR-PHXC has the potential to be a better therapeutic candidate for patients with this disease. We also discontinued our DCR-MYC program in oncology because early efficacy results did not meet our threshold for further development. We believe this strategic shift will put us in a stronger position to execute our development strategy more efficiently and build an exciting pipeline of subcutaneously-delivered, RNAi-based therapeutics with clear differentiation and that have the potential to be first-in-class in specific indications.”

  • Primary Hyperoxaluria: Dicerna is developing DCR-PHXC for the treatment of primary hyperoxaluria (PH), a rare inborn error of metabolism in which the liver produces excessive levels of oxalate, which in turn causes damage to the kidneys and to other tissues in the body. DCR-PHXC is in preclinical development and is advancing into Investigational New Drug (IND)-enabling studies. Dicerna intends to file an IND submission or Clinical Trial Application (CTA) for DCR-PHXC in late 2017 and commence human clinical trials shortly thereafter.

    To facilitate DCR-PHXC development, 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.
  • Cardiovascular Disease: Dicerna is using its GalXC RNAi platform to develop an investigational therapeutic that targets PCSK9 to treat statin-refractory patients with hypercholesterolemia. 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 monoclonal antibody therapies, based on smaller subcutaneous injection volumes and less frequent dosing, while providing equal or superior control of serum cholesterol. The Company is continuing to advance this program and is on track to nominate a candidate for preclinical development in 2016.
  • Undisclosed Rare Disease: Dicerna is developing a GalXC-based therapeutic that targets a liver-expressed gene involved in a rare disease associated with high morbidity and mortality. This undisclosed program is on track for formal program launch with an optimized lead candidate in 2016.

Dicerna has the capacity to launch up to three additional programs annually, with the intent to advance five programs into the clinic by the end of 2019. The Company expects to initiate programs for hepatitis B virus (HBV) as well as two additional programs from its core therapeutic areas in 2017.

Legacy Program Update

  • During the third quarter of 2016, Dicerna transitioned its PH program to DCR-PHXC from DCR-PH1. The Company discontinued the development program for DCR-PH1, an investigational therapy formulated in a lipid nanoparticle (LNP) delivery system obtained through a licensing agreement with Arbutus Biopharma Corporation (formerly known as Tekmira Pharmaceuticals Corporation). DCR-PH1 was being studied in two Phase 1 clinical trials: DCR-PH1-101 in patients with primary hyperoxaluria type 1 (PH1) and DCR-PH1-102 in normal healthy...