Dicerna Pharmaceuticals
Aug 4, 2016

Dicerna Reports Second Quarter 2016 Financial and Operational Results

Management to Host Conference Call Today at 4:30 p.m. ET

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (Nasdaq: DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, today reported financial and operational results for the second quarter ended June 30, 2016.

"This quarter marks a significant strategic step forward for Dicerna as we formally introduced our GalXC™ subcutaneous delivery platform at our June Investor Day. During that event we showed consistent, potent and durable preclinical gene silencing data from GalXC molecules against 12 different disease-associated genes, including six examples of data from non-human primates, to highlight the robust basis for further investigation of the silencing of disease-causing genes in the liver across multiple therapeutic areas," said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. "Our GalXC platform is now a fully-enabled RNAi drug discovery engine with powerful capabilities that could result in potency that is on par with or better than comparable platforms, longer duration of action versus other modalities, exquisite specificity to gene targets, and a simple, infrequent dosing regimen. We are launching three GalXC research programs in 2016, the first in primary hyperoxaluria, a second in cardiovascular disease targeting PCSK9, and a third in an undisclosed orphan genetic disease. We are also continuing to advance our DCR-PH1 and DCR-MYC clinical research programs."

Technology Update

Subcutaneous delivery to the liver with GalXC Platform: Dicerna's proprietary GalXC platform utilizes the Company's extended dicer substrate short interfering RNA (DsiRNA-EX) technology conjugated to a targeting agent to enable delivery to the liver via subcutaneous administration. Dicerna scientists attach small drug delivery agents, known as N-acetylgalactosamine (GalNAc) sugars, directly to the extended region of a DsiRNA-EX molecule, a chemically optimized, double stranded RNA developed by Dicerna. The GalNAc sugars are designed to specifically bind to receptors on hepatocyte target cells in the liver, potentially leading to effective delivery and silencing of specific gene targets within the cells. Many of the conjugates produced using the GalXC platform incorporate a folded motif known as a tetraloop, which stabilizes the RNA duplex and provides multiple conjugation points for the addition of the GalNAc sugars. The tetraloop configuration, which is proprietary and unique to Dicerna's conjugates, interfaces effectively with the RNAi machinery within target cells.

Rare Disease Program Update

DCR-PH1: DCR-PH1 is an intravenously infused DsiRNA-EX-based therapeutic candidate for primary hyperoxaluria type 1 (PH1), a severe, rare genetic disease of liver metabolism that often results in life-threatening damage to the kidneys. In a genetic mouse model of PH1, DCR-PH1 knocked down the activity of the HAO1 gene transcript that encodes for the enzyme glycolate oxidase, thereby reducing the production of oxalate, the key mediator of disease pathology in PH1. Similar results, if obtained in PH1 patients, may have significant clinical benefit. In non-human primate studies, a single dose of DCR-PH1 led to an average of 94% knockdown, with a maximum of 96% knockdown, of the HAO1 gene transcript. The DCR-PH1 clinical research program consists of the following studies:

Oncology Program Update

DCR-MYC: DCR-MYC is a being investigated as a specific inhibitor of MYC, an oncogene frequently amplified or overexpressed in a wide variety of tumor types, including hepatocellular carcinoma (HCC). MYC has long been considered "undruggable" with small molecule and antibody technologies. DCR-MYC is a DsiRNA-based therapeutic formulated in Dicerna's EnCore™ lipid nanoparticle for delivery to solid tumors. In preclinical studies, DCR-MYC knocked down MYC transcript levels and reduced tumor volume in multiple mouse tumor models, including models of HCC. DCR-MYC is currently being tested in two clinical trials.

Corporate Update

Financial Results

For more detailed information and analysis see the Company's Quarterly Report on Form 10-Q, which was filed with the Securities and Exchange Commission (SEC) on August 4, 2016.


Based on Dicerna's current cash position and operating plan, the Company reiterates its expectation that it has sufficient cash to fund operations for at least the next 12 months. This estimate assumes no additional funding from new partnership agreements or debt or equity financing events.

Conference Call

Management will conduct a conference call at 4:30 p.m. ET today to review the Company's second quarter 2016 financial results and provide a general business update. The conference call can be accessed by dialing (855) 453-3834 or (484) 756-4306 (international), and referencing conference ID 49039068 prior to the start of the call. The call will also be webcast via the Internet and will be available under the "Investors & Media" section of the Dicerna website, www.dicerna.com. A replay of the call will be available beginning at 7:30 p.m. ET on August 4, 2016. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and refer to conference ID 49039068. The webcast will also be archived on the Company's website.

About Dicerna Pharmaceuticals, Inc.

Dicerna Pharmaceuticals, Inc., is an RNA interference-based biopharmaceutical company focused on the discovery and development of innovative treatments for rare, inherited diseases involving the liver, for other therapeutic areas in which the liver plays a key role, and for cancers that are genetically defined. The Company is using its proprietary RNA interference (RNAi) technology platform to build a broad pipeline in these therapeutic areas. In many cases, Dicerna is pursuing targets that have historically been difficult to inhibit using conventional approaches, but where connections between targets and diseases are well understood and documented. The Company intends to discover, develop and commercialize these novel therapeutics either on its own or in collaboration with pharmaceutical partners. For more information, please visit www.dicerna.com.

About DCR-PH1

DCR-PH1 is being developed by Dicerna for the treatment of PH1 by addressing its pathology through the targeting and destruction of the messenger RNA (mRNA) produced by the HAO1 gene. HAO1 encodes glycolate oxidase (GO), an upstream enzyme involved in the production of oxalate, the mediator of pathogenesis and progression of PH1. In preclinical studies, DCR-PH1 inhibited HAO1 and increased levels of glycolate and reduced levels of urinary oxalate.

DCR-PH1 incorporates small interfering RNA (siRNA) formulated in a proprietary lipid nanoparticle (LNP) technology that is being investigated as a system for efficient delivery to the liver after intravenous (IV) administration. Dicerna obtained rights to this delivery technology through a licensing agreement with Arbutus Biopharma Corporation, formerly known as Tekmira Pharmaceuticals Corporation.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements, including, for example, our expected timeline of development and licensing plans. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our clinical and preclinical research and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q filing and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Dicerna's current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to update any forward-looking statements.

Dicerna Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheets (Unaudited)
(In thousands)
June 30, December 31,
2016 2015
Cash and cash equivalents $ 29,187 $ 56,058
Held-to-maturity investments $ 40,003 $ 38,551
Total assets $ 74,333 $ 100,023
Total liabilities $ 9,365 $ 9,001
Total stockholders' equity $ 64,968 $ 91,022
Dicerna Pharmaceuticals, Inc.
Condensed Consolidated Statements of Operations (Unaudited)
(In thousands, except share and per share data)

For the Three Months Ended
June 30,


For the Six Months Ended
June 30,

2016   2015 2016   2015
Revenues $ - 184 $ - 184
Operating expenses:
Research and development 11,032 $ 11,875 22,296 $ 20,567
General and administrative 4,656   4,519   9,140   9,964  
Total operating expenses 15,688 16,394 31,436 30,531
Loss from operations (15,688 ) (16,210 ) (31,436 ) (30,347 )
Interest income 66 34 121 87
Net loss $ (15,622 ) $ (16,176 ) $ (31,315 ) $ (30,260 )
Net loss per share - basic and diluted $ (0.75 ) $ (0.86 ) $ (1.51 ) $ (1.65 )
Weighted average shares outstanding - basic and diluted 20,726,108 18,852,814 20,706,388 18,337,030

Rx Communications Group
Melody Carey, 917-322-2571
Alex Van Rees, 973-442-1555 ext. 111

Source: Dicerna Pharmaceuticals, Inc.

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