Dicerna Pharmaceuticals
Mar 27, 2014

Dicerna Announces Fourth Quarter and Full Year 2013 Financial and Operational Results

-DCR-M1711 to Enter Phase 1 Clinical Trials in First Half of 2014-

-Management to Host Conference Call at 4:30 PM ET-

WATERTOWN, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA), a leader in the development of RNAi-based therapeutics, today provided a corporate update and announced operational and financial results for the fourth quarter and full year ended December 31, 2013.

"In 2013, Dicerna achieved very substantial financial and operational objectives in our mission to build an innovative drug development company based on the powerful Dicer Substrate RNA interference technology," commented Douglas M. Fambrough, PhD, Dicerna's President and CEO. "With the completion of our successful initial public offering, we added significant financial resources to support the development of our pipeline of optimized Dicer Substrate siRNA (DsiRNA™) molecules, which is focused on specific indications in oncology and rare genetic diseases that we can target through the liver. We look forward to continued progress in 2014 with DCR-M1711 expected to enter a Phase 1 clinical study in the second quarter of the year. We also anticipate providing further definition to the DCR-PH1 program in Primary Hyperoxaluria as well as announcing an additional therapeutic area of development from our discovery efforts during the year."

2013 Business Highlights and Recent Developments

Upcoming Development Milestones

DCR-M1711 is a therapeutic candidate for solid tumors, including HCC. DCR-M1711 was developed using Dicer Substrate technology and EnCore™ drug delivery technology and is a potent and specific inhibitor of the MYC oncogene, a gene that causes or promotes cancer when abnormally expressed or activated and is up-regulated in a wide variety of tumor types.

DCR-PH1 is a therapeutic candidate for Primary Hyperoxaluria (PH1), a rare, inherited autosomal recessive disorder of metabolism in the liver that usually results in severe damage to the kidneys. Developed using DsiRNA and EnCore lipid nanoparticle delivery technology, DCR-PH1 inactivates the gene encoding glycolate oxidase, significantly reducing the production of oxalate, the key disease pathology of PH1.

Fourth Quarter 2014 Financial Results and Guidance

Conference Call Information

Dicerna will host a conference call and live audio webcast today at 4:30 p.m. (ET) to discuss its financial results and provide a corporate update. To participate in the conference call, please dial 855-453-3834 (domestic) or 484-756-4306 (international) and refer to conference ID 18911998. To access the live webcast please visit the "Events & Presentations" page under the "Investors & Media" tab on Dicerna's website at www.dicerna.com.

An archived copy of the webcast will be available on Dicerna's website beginning approximately two hours after the conference call, and will be available on the website for at least 30 days after the conference call.

About Dicerna Pharmaceuticals, Inc.

Dicerna is a biopharmaceutical company focused on the discovery and development of innovative treatments for rare inherited diseases involving the liver and for cancers that are genetically defined. Dicerna is using its proprietary RNA interference (RNAi) technology platform to build a broad pipeline in these therapeutic areas and intends to discover, develop and commercialize novel therapeutics either on its own or in collaboration with pharmaceutical partners.

Cautionary Note on Forward-Looking Statements

This press release includes forward-looking statements. 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 identified under the heading "Risk Factors" included in the prospectus related to the initial public offering, and in other filings that Dicerna may make with the SEC in the future. 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 (unaudited)
(Amounts in Thousands)
      December 31,
2012       2013
Cash and cash equivalents $3,670       $46,595
Total Assets $10,191 $49,794
Long-term Debt, including current portion $8,800 $4,847
Total Liabilities $10,662 $8,467
Total stockholders' deficit ($64,719) ($68,919)
Dicerna Pharmaceuticals, Inc.
Consolidated Statements of Operations (unaudited)
(Amounts in thousands except per share data)
          Three Months Ended December 31,         Years Ended December 31,
2012     2013 2012       2013
Revenue $6,254 $0 $7,015 $0
Operating expenses:
Research and development $3,327 $4,194 $11,565 $11,558
General and administrative $1,227     $2,243 $4,700       $5,820
Total operating expenses $4,554 $6,437 $16,265 $17,378
Income (loss) from operations $1,700 ($6,437) ($9,250) ($17,378)
Other income (expense), net ($187) ($282) (871) ($1,140)
Net income (loss) $1,513     ($6,719)   ($10,121)       ($18,518)
Less: Accretion and dividends on redeemable convertible preferred stock $1,506 $9 $4,097 $2,388
          -       -
Net loss attributable to common stockholders $7     ($6,728)   ($14,218)       ($20,906)
Net income (loss) per share allocable to common stockholders - basic and diluted $0 ($200) ($516) ($710)
Weighted average shares outstanding - basic and diluted 27,598 33,654 27,554 29,463

Dicerna Pharmaceuticals, Inc.
James E. Dentzer, 617-621-8097
Chief Financial Officer
MacDougall Biomedical Communications
Chris Erdman, 781-235-3060
Senior Vice President

Source: Dicerna Pharmaceuticals, Inc.

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