Dicerna Pharmaceuticals
May 8, 2017

Dicerna Reports First Quarter 2017 Financial and Operational Results and Provides Corporate Update

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 first quarter ended March 31, 2017.

"While continuing to focus on our research and development activities during the first quarter, we announced a major $70.0 million convertible preferred stock financing, which subsequently closed on April 11, 2017," said Douglas Fambrough, Ph.D., president and chief executive officer of Dicerna. "The financing, led by Bain Capital Life Sciences and a syndicate of current and new investors, lends an additional level of validation to the potential of our proprietary GalXC™ RNAi technology platform. Furthermore, the funds, when combined with cash already on-hand, provide us with the necessary resources to execute our stated strategy, which includes pursuing the advancement of our core therapeutic programs. Specifically, as we look forward from today and into 2019, we expect to be able to advance our first three development programs, including DCR-PHXC, a second undisclosed rare disease program and DCR-HBV, into proof of concept studies, while progressing DCR-PCSK9 toward formal preclinical development, resulting in a number of key, value-creating inflection points. In the nearer term, I look forward to presenting new preclinical data for DCR-PHXC later this year and to our expected Investigational New Drug (IND) or Clinical Trial Application (CTA) filing at the end of 2017."

GalXC™ Program Update

Financial Condition and Operating Results

For more detailed information and analysis, see Dicerna's Quarterly Report on Form 10-Q for the quarter ended March 31, 2017, which was filed with the Securities and Exchange Commission (SEC) on May 8, 2017.

Subsequent Event


With the closing of its Preferred Stock transaction, Dicerna believes that it has sufficient cash to fund the execution of its current operating plan into 2019, which includes focusing its resources on advancing its first three development programs into 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 first quarter 2017 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 7812180 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 May 8, 2017. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and refer to conference ID 7812180. 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 GalXCTM 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 disease 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, our anticipated use of proceeds from the Preferred Stock transaction, 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 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.
Consolidated Balance Sheet Information
(In thousands)
  March 31,   December 31,
2017 2016
Cash and cash equivalents $ 22,873 $ 20,865
Held-to-maturity investments $ 10,002 $ 25,009
Total assets $ 39,436 $ 51,252
Total liabilities $ 10,347 $ 10,044
Total stockholders' equity $ 29,089 $ 41,208
Dicerna Pharmaceuticals, Inc.
Consolidated Statements of Operations Information
(In thousands, except share and per share data)

For the Three Months Ended
March 31,

2017 2016
Revenue $ 133   $ -  
Operating expenses:
Research and development 8,876 11,264
General and administrative 5,496   4,484  
Total operating expenses 14,372   15,748  
Loss from operations (14,239 ) (15,748 )
Interest income 38 55
Net loss $ (14,201 ) $ (15,693 )
Net loss per share - basic and diluted $ (0.68 ) $ (0.76 )
Weighted average shares outstanding - basic and diluted 20,791,644 20,646,793

Rx Communications Group
Paula Schwartz, 917-322-2216
Alex Van Rees, 973-442-1555 ext. 111

Source: Dicerna Pharmaceuticals, Inc.

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