Dicerna Pharmaceuticals
Mar 10, 2016

Dicerna Announces Fourth Quarter and Full Year 2015 Financial and Operational Results

-Management to Host Conference Call Today at 4:30 pm ET-

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA), a leading developer of investigational RNA interference (RNAi) therapeutics, today announced financial and operational results for the quarter and full year ended December 31, 2015.

"2015 was a pivotal year for the advancement of our RNAi platform," stated Douglas M. Fambrough, Ph.D., Dicerna's chief executive officer, "both in advancing our clinical-stage oncology and rare disease product candidates as well as advancing the proprietary subcutaneous delivery technology behind our emerging pipeline of additional liver programs. Specifically, we initiated clinical testing of DCR-PH1 for the treatment of Primary Hyperoxaluria Type 1 (PH1), and we presented preliminary efficacy and safety data from our DCR-MYC Phase 1 study in patients with advanced solid tumors that showed early signs of clinical anti-tumor activity in patients with advanced pancreatic neuroendocrine tumors (PNET). To build our future pipeline, we completed technology development of our subcutaneous DsiRNA-EX Conjugate technology, achieving preclinical potency levels that we believe enable a simple, infrequent subcutaneous treatment regimen in patients. With technology development complete, we've embarked on a series of programs in multiple therapeutic areas utilizing the DsiRNA-EX Conjugate technology which can be advanced into development by Dicerna or, potentially, in collaboration with partners."

Technology Update

Subcutaneous delivery to the liver with DsiRNA-EX Conjugates: DsiRNA-EX Conjugates are a proprietary delivery technology that is 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 a 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 disease pathology of PH1. Similar results, if obtained in PH1 patients, may have significant clinical benefit. In non-human primate studies, a single dose of DCR-PH1 can lead to an average of 84% knockdown of the HAO1 gene transcript with up to 93% knockdown observed.

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 DsiRNA-based therapeutic formulated in Dicerna's EnCore lipid nanoparticle for delivery to solid tumors. In pre-clinical 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)

Financial Results

For more detailed financial information and analysis see the Company's Annual Report on Form 10-K for the year ended December 31, 2015.


Based on our current cash position and operating plan, the Company expects that it has sufficient cash to fund operations for at least the next twelve 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 fourth quarter and full year 2015 financial results. The call can be accessed by dialing (855) 453-3834 or (484) 756-4306 (international), and referencing conference ID 42131082. The conference 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 March 10, 2016. To access the replay, please dial 855-859-2056 or 404-537-3406, and refer to conference ID 42131082. 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.

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)
December 31, December 31,
2015   2014
Cash and cash equivalents $ 56,058 $ 26,067
Held-to-maturity investments $ 38,551 $ 72,556
Total assets $ 100,023 $ 103,605
Total liabilities $ 9,001 $ 5,265
Total stockholders' equity $ 91,022 $ 98,340
Dicerna Pharmaceuticals, Inc.
Consolidated Statements of Operations (unaudited)
(In thousands, except share and per share data)

For the Three Months Ended
December 31,

For the Years Ended
December 31,

2015 2014 2015 2014
Revenues $ - $ - $ 184 $ -
Operating expenses:
Research and development 11,263 9,907 43,971 29,453
General and administrative 4,418   4,710   19,240   15,648  
Total operating expenses 15,681 14,617 63,211 45,101
Loss from operations (15,681 ) (14,617 ) (63,027 ) (45,101 )
Preferred stock warrant remeasurement - - - (2,559 )
Other income (expense), net 46   27   188   (279 )
Net loss $ (15,635 ) $ (14,590 ) $ (62,839 ) $ (47,939 )
Less: Accretion and dividends on redeemable convertible preferred stock -   -   -   204  
Net loss attributable to common stockholders (15,635 ) (14,590 ) (62,839 ) (48,143 )
Net loss per share allocable to common stockholders - basic and diluted $ (0.76 ) $ (0.82 ) $ (3.09 ) $ (3.00 )
Weighted average shares outstanding - basic and diluted 20,592,840 17,706,645 20,320,628 16,070,054

Investor Contact:
Westwicke Partners
Peter Vozzo, 443-213-0505
Media Contact:
Alex Van Rees, 973-442-1555 ext. 111

Source: Dicerna Pharmaceuticals, Inc.

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