Dicerna Pharmaceuticals
Aug 10, 2017

Dicerna Reports Second 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 second quarter ended June 30, 2017.

"We are pleased to have reported a number of significant events during this quarter that support the execution of our business strategy," stated Douglas Fambrough, president and chief executive officer of Dicerna. "Specifically, the completion of our $70 million convertible preferred stock financing and the key addition of Dr. Ralf Rosskamp as chief medical officer, have served to further solidify our financial and leadership capacity. As a result, we are in a strong position to continue to advance DCR-PHXC, our lead GalXC™-based product candidate, into Phase 1 clinical studies for primary hyperoxaluria (PH) early next year and to continue to pursue Investigational New Drug (IND) application-enabling activities for our undisclosed rare disease program as well as for DCR-HBVS. More recently, during the 12th International Workshop on Primary Hyperoxaluria held this past July, we presented new preclinical data for DCR-PHXC demonstrating how inhibition of the lactate dehydrogenase A (LDHA) gene reduced oxalate production in multiple animal models of PH. These data highlight the role of LDHA as an optimal therapeutic target and the potential utility of DCR-PHXC to treat all forms of the disease. These findings indicate that this novel target may offer the ability to treat an expanded population of patients who currently have no other effective options."

GalXC™ Program Update

Research from multiple animal models of PH demonstrated how DCR-PHXC inhibits LDHA, which the Company has identified as potentially being an optimal therapeutic target in patients with the disease. The data highlights included:

During the workshop, Dicerna also reported data from its Primary HYperoxaluria Observational Study (PHYOS), an international, multicenter, observational study in patients with a genetically confirmed diagnosis of PH1. PHYOS is collecting data on key biochemical parameters, including changes in oxalate, glycolate, and other metabolites, implicated in the pathogenesis of the disease. Dicerna continues to advance PHYOS to facilitate DCR-PHXC development and hopes to use the data to better understand the baseline PH1 disease state, which will help guide long-term drug development plans.

Dicerna is on track to file a clinical trial application (CTA) in Europe for DCR-PHXC in late 2017 and to commence human clinical trials in the first quarter of 2018. During the workshop, Dicerna disclosed that the DCR-PHXC clinical trial will be conducted at multiple sites in Europe and will include both healthy volunteer and patient cohorts. The Company anticipates that study participants will receive a single ascending dose of DCR-PHXC via subcutaneous injection, transitioning, as appropriate, to multiple ascending doses. The primary endpoints will include safety and tolerability, urine and plasma biomarkers, and pharmacokinetics.

Financing Update

Corporate 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 June 30, 2017, which was filed with the Securities and Exchange Commission (SEC) on August 10, 2017.


With the closing of its Preferred Stock transaction, 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 and a fourth program into formal preclinical development. This estimate assumes no additional funding from new collaboration agreements or from additional financing events.

Conference Call

Management will host a conference call at 4:30 p.m. ET today to review Dicerna's second 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 38183667 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 August 10, 2017. To access the replay, please dial (855) 859-2056 or (404) 537-3406, and refer to conference ID 38183667. 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, our expected timeline and plans for development of DCR-PHXC and other pipeline programs, 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)
June 30, December 31,
2017     2016
Cash and cash equivalents $ 38,777 $ 20,865
Held-to-maturity investments $ 49,953 $ 25,009
Total assets $ 94,723 $ 51,252
Total liabilities $ 9,583 $ 10,044
Redeemable convertible preferred stock $ 71,872 $ -
Total stockholders' equity $ 13,268 $ 41,208
Dicerna Pharmaceuticals, Inc.
Consolidated Statements of Operations Information
(In thousands, except share and per share data)
For the Three Months Ended For the Six Months Ended
June 30, June 30,
2017 2016 2017 2016
Revenue $ 252 - $ 385 -
Operating expenses:
Research and development 9,320 11,032 18,196 22,296
General and administrative 6,300   4,656   11,796   9,140  
Total operating expenses 15,620 15,688 29,992 31,436
Loss from operations (15,368 ) (15,688 ) (29,607 ) (31,436 )
Interest income 143   66   181   121  
Net loss $ (15,225 ) $ (15,622 ) $ (29,426 ) $ (31,315 )
Dividends on redeemable convertible preferred stock (2,622 ) - (2,622 ) -
Deemed dividend related to beneficial conversion feature of redeemable convertible preferred stock (6,144 ) -   (6,144 ) -  
Net loss attributable to common stockholders $ (23,991 ) $ (15,622 ) $ (38,192 ) $ (31,315 )
Net loss per share - basic and diluted $ (1.15 ) $ (0.75 ) $ (1.84 ) $ (1.51 )
Weighted average shares outstanding - basic and diluted 20,794,193 20,726,108 20,792,925 20,706,388

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

Source: Dicerna Pharmaceuticals, Inc.

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