Dicerna Pharmaceuticals
May 9, 2016

Dicerna Reports First 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 first quarter ended March 31, 2016.

"We continue to achieve milestones across our portfolio of opportunities, including program launches with our DsiRNA-EX Conjugate technology, as well as our DCR-PH1 and DCR-MYC clinical programs," said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. "We look forward to sharing new preclinical data for our conjugate programs as well as further updates on our clinical programs at our upcoming Investor Day."

Technology Update

Subcutaneous delivery to the liver with DsiRNA-EX Conjugates: Dicerna's Dicer substrate short interfering RNA extended (DsiRNA-EX) Conjugates are proprietary RNAi therapeutic candidates rationally designed to enable convenient subcutaneous delivery for Dicerna's emerging pipeline of liver-targeted RNAi investigational therapies. These conjugates do not involve lipid nanoparticles and are built on the DsiRNA-EX platform, using an extension to one end of the double-stranded DsiRNA molecule. These extensions are unique to Dicerna and utilize proprietary RNA structures that enable a differentiated and independent approach to subcutaneous delivery of RNAi-inducing therapeutics.

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 significantly 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 84% knockdown of the HAO1 gene transcript. The DCR-PH1 clinical program consists of the following studies:

Oncology Program Update

DCR-MYC: DCR-MYC is a potent and 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 Dicer substrate short interfering RNA (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 significantly reduced tumor volume in multiple mouse tumor models, including models of HCC. DCR-MYC is currently being tested in two ongoing clinical trials.

Phase 1 DCR-MYC Trial in Solid Tumors

Phase 1 DCR-MYC Trial in Hepatocellular Carcinoma (HCC)

Corporate Update

Dicerna announces today that it plans to host an Investor Day on June 29, 2016 in New York City to present new preclinical data for its emerging pipeline of DsiRNA-EX Conjugate programs, as well as an update on its PH1 and oncology clinical programs.

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 May 9, 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 first 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 92953440 prior to the start of the call. The call will also be webcast live over the Internet and can be accessed on the "Events & Presentations" page under the "Investors & Media" section of the Dicerna Pharmaceuticals website, www.dicerna.com, prior to the event. A replay of the call will be available beginning at 7:30 p.m. ET on May 9, 2016. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and refer to conference ID 92953440. 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. Preclinical studies have shown that DCR PH1 inhibited HAO1 and significantly 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.

Add 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.
Consolidated Balance Sheets (unaudited)
(In thousands)
        March 31,     December 31,
2016     2015
Cash and cash equivalents $ 27,614 $ 56,058
Held-to-maturity investments $ 53,031 $ 38,551
Total assets $ 85,544 $ 100,023
Total liabilities $ 7,364 $ 9,001
Total stockholders' equity $ 78,180 $ 91,022
Dicerna Pharmaceuticals, Inc.
Consolidated Statements of Operations (unaudited)
(In thousands, except share and per share data)

For the Three Months Ended
March 31,

2016 2015
Operating expenses:
Research and development $ 11,264 $ 8,692
General and administrative 4,484   5,445  
Total operating expenses 15,748 14,137
Loss from operations (15,748 ) (14,137 )
Other income (expense), net 55   53  
Net loss $ (15,693 ) $ (14,084 )
Net loss per share - basic and diluted $ (0.76 ) $ (0.79 )
Weighted average shares outstanding - basic and diluted 20,646,793 17,815,515

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

Source: Dicerna Pharmaceuticals, Inc.

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