Dicerna Pharmaceuticals
Nov 2, 2017

Dicerna Reports Third Quarter 2017 Financial and Operating 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 operating results for the third quarter ended September 30, 2017.

"We are extremely pleased with the progress we made during the third quarter, and the fourth quarter is off to a great start, highlighted by the achievement of two key milestones for the Company," said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. "These include the submission of a Clinical Trial Application (CTA) in the United Kingdom for our lead program, DCR-PHXC, which we are developing for primary hyperoxaluria (PH), and the announcement of a strategic research collaboration with Boehringer Ingelheim International GmbH (BI), which, not only expands our GalXC™ pipeline to include a program for nonalcoholic steatohepatitis (NASH), but is reflective of the strength of our proprietary technology platform and our underlying intellectual property.

"As we approach year end and look ahead to 2018, we are working toward a number of other important catalysts for the Company. These include CTA submissions in additional European countries and an investigational new drug (IND) application filing in the U.S. for DCR-PHXC, as well as the commencement of our Phase 1 clinical trial of DCR-PHXC in the United Kingdom early in the first quarter of 2018. We also anticipate reporting human proof-of-concept data for this program in the second half of 2018. At the same time, we are focused on advancing our undisclosed GalXC program for a second rare disease, for which we plan to file an IND application and/or CTA in mid-2018, as well as our continued focus on DCR-HBVS for chronic hepatitis B virus, DCR-PCSK9 for hypercholesterolemia, and additional GalXC programs. Lastly, we are eager to embark on our new, joint GalXC collaboration with BI in chronic liver diseases, and we look forward to working closely with the company as we seek to discover and develop RNAi therapeutics to treat NASH."

GalXC™ Research Collaboration

On November 2, 2017, Dicerna announced a research collaboration and license agreement with BI to discover and develop novel GalXC RNAi therapeutics for the treatment of chronic liver diseases. The collaboration will initially focus on NASH, a devastating, chronic liver disease for which there is no approved treatment option. NASH is caused by the buildup of fat in the liver, potentially leading to liver fibrosis and cirrhosis. It has an especially high prevalence among obese and diabetic patients, and is an area of high unmet medical need. NASH is expected to soon become the most common cause of advanced liver disorders, and it often necessitates liver transplantation. Under the terms of the agreement, Dicerna may receive more than $200 million from BI, including an upfront payment, development and commercial milestone payments, and research and development reimbursement for a GalXC candidate product addressing an undisclosed NASH target. Dicerna is also eligible to receive royalties tiered up to double-digits on worldwide net sales.

GalXC™ Pipeline 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 September 30, 2017, which was filed with the Securities and Exchange Commission (SEC) on November 2, 2017.


Dicerna believes that it has sufficient cash to fund the execution of its current clinical and operating plan into 2019, which includes focusing its resources on advancing its first three development programs into proof-of-concept clinical studies. This estimate assumes no new funding from additional collaboration agreements or from external financing events.

Conference Call

Management will host a conference call at 4:30 p.m. ET today to review Dicerna's third 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 95620574 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 approximately two hours after the completion of the call and will remain available for seven days. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and refer to conference ID 95620574. The webcast will also be archived on Dicerna's website.

About Dicerna Pharmaceuticals, Inc.

Dicerna Pharmaceuticals, Inc., is a biopharmaceutical company focused on the discovery and development of innovative, subcutaneously delivered RNAi-based therapeutics for diseases involving the liver, including rare diseases, chronic liver diseases, cardiovascular diseases, and viral infectious diseases. Dicerna 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. Dicerna 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 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 the Company believes will enable subcutaneous delivery of its 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, Dicerna's expected timeline and plans for development of DCR-PHXC and other pipeline programs, expectations related to the collaboration with BI, and guidance related to the anticipated availability of current liquidity. 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 Dicerna's clinical and preclinical research and other risks identified under the heading "Risk Factors" included in the Company's 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)
September 30, December 31,
2017     2016
Cash and cash equivalents $ 30,960 $ 20,865
Held-to-maturity investments $ 44,959 $ 25,009
Total assets $ 82,726 $ 51,252
Total liabilities $ 10,522 $ 10,044
Redeemable convertible preferred stock $ 75,983 $ -
Total stockholders' (deficit) equity $ (3,779 ) $ 41,208
Dicerna Pharmaceuticals, Inc.
Consolidated Statements of Operations Information
(In thousands, except share and per share data)

For the Three Months Ended
September 30,

For the Nine Months Ended
September 30,

2017 2016 2017 2016
Revenue $ 474 162 $ 859 162
Operating expenses:
Research and development 9,001 10,061 27,197 32,357
General and administrative 6,685   4,338   18,481   13,478  
Total operating expenses 15,686   14,399   45,678   45,835  
Loss from operations (15,212 ) (14,237 ) (44,819 ) (45,673 )
Interest income 179   61   360   182  
Net loss $ (15,033 ) $ (14,176 ) $ (44,459 )



(45,491 )

Dividends on redeemable
convertible preferred stock

(4,111 ) - (6,733 ) -

Deemed dividend related to
beneficial conversion feature
of redeemable convertible
preferred stock

-   -   (6,144 ) -  

Net loss attributable to
common stockholders

$ (19,144 ) $ (14,176 ) $ (57,336 )



(45,491 )
Net loss per share - basic and diluted $ (0.92 ) $ (0.68 ) $ (2.76 )



(2.20 )

Weighted average shares
outstanding - basic and diluted

20,841,728 20,752,416 20,809,372 20,708,600

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

Source: Dicerna Pharmaceuticals, Inc.

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