Dicerna Pharmaceuticals
Mar 30, 2017

Dicerna Reports Fourth Quarter and Full Year 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 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," 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

Financial Condition and Operating Results

For more detailed information and analysis, see Dicerna's Annual Report on Form 10-K for the year ended December 31, 2016, which was filed with the Securities and Exchange Commission (SEC) on March 30, 2017.

Subsequent Event


Assuming successful closing of the stock purchase agreement, the Company anticipates that it will have sufficient cash to fund the execution of the Company's current operating plan into 2019, including focusing its resources on advancing its first three development programs through proof of concept studies and a fourth program into formal preclinical development. This estimate assumes no additional funding from new partnership agreements or from additional financing events.

Conference Call

Management will host a conference call at 4:30 p.m. ET today to review the Company's fourth quarter and full year 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 70365614 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 March 30, 2017. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and refer to conference ID 70365614. The webcast will also be archived on the Company's website.

About Dicerna Pharmaceuticals, Inc.

Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company focused on the discovery and development of innovative RNAi-based therapeutics for diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. The Company is leveraging its proprietary GalXC™ RNAi technology platform to build a broad pipeline in these core therapeutic areas, focusing on target genes where connections between target gene and diseases are well understood and documented. The Company intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners. For more information, please visit www.dicerna.com.

About GalXC™ RNAi Technology Platform

GalXCTM is a proprietary technology platform invented by Dicerna to discover and develop next-generation RNAi-based therapies designed to silence disease-driving genes in the liver. Compounds produced via GalXC are intended to be broadly applicable across multiple therapeutic areas, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. Using GalXC, Dicerna scientists attach N-acetylgalactosamine (GalNAc) sugars directly to the extended region of our proprietary Dicer substrate short-interfering RNA molecules, yielding multiple proprietary conjugate delivery configurations. Many of the conjugates produced via GalXC incorporate a folded motif known as a tetraloop in the extended region. The tetraloop configuration, which is unique to Dicerna's GalXC compounds, allows flexible and efficient conjugation to the targeting ligands, and stabilizes the RNAi duplex which we believe will enable subcutaneous delivery of Dicerna's RNAi therapies to hepatocytes in the liver, where they are designed to specifically bind to receptors on target cells, potentially leading to internalization and access to the RNAi machinery within the cells. The technology may offer several distinct benefits, as suggested by strong preclinical data. These benefits include: potency that is on par with or better than comparable platforms; highly specific binding to gene targets; long duration of action; and an infrequent subcutaneous dosing regimen.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements, including, for example, the closing of our Preferred Stock financing, our expected timeline and plans for development, potential collaborations, and potential therapeutic benefits. 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 risks that the offering may be delayed or may not occur due to market or other conditions and the satisfaction of customary closing conditions related to the offering, risks relating to our clinical and preclinical research and other risks identified under the heading "Risk Factors" included in our most recent Form 10-K 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.
Consolidated Balance Sheet Information
(In thousands)
December 31, December 31,
  2016     2015
Cash and cash equivalents





Held-to-maturity investments





Total assets





Total liabilities





Total stockholders' equity







Dicerna Pharmaceuticals, Inc.
Consolidated Statements of Operations Information
(In thousands, except share and per share data)

For the Three Months Ended
December 31,


For the Year Ended
December 31,

2016   2015 2016   2015


133   $ -  


295   $ 184  
Operating expenses:
Research and development 9,337 11,263 41,694 43,971
General and administrative 4,871   4,418   18,349   19,240  
Total operating expenses 14,208 15,681 60,043 63,211
Loss from operations (14,075 ) (15,681 ) (59,748 ) (63,027 )
Interest income 53 46 235 188
Net loss


(14,022 )


(15,635 )


(59,513 )


(62,839 )
Net loss per share - basic and diluted


(0.68 )


(0.76 )


(2.87 )


(3.09 )
Weighted average shares outstanding - basic and diluted 20,753,001 20,592,840 20,719,761 20,320,628

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

Source: Dicerna Pharmaceuticals, Inc.

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