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 fourth quarter and full-year ended December 31, 2017.
“2017 was a transformative year for Dicerna, as we successfully transitioned to a clinical stage company advancing our growing pipeline of GalXC™-based RNAi therapeutics, and signed our first GalXC platform collaboration agreement with Boehringer Ingelheim,” said Douglas M. Fambrough, Ph.D., president and chief executive officer of Dicerna. “With our lead candidate, DCR-PHXC for the treatment of primary hyperoxaluria (PH), now in a Phase 1 clinical trial, and our other priority programs, including DCR-HBVS for chronic hepatitis B and an undisclosed program for a rare disease of the liver, following quickly behind, we plan to have three GalXC product candidates in clinical development by early 2019.
“We entered this year with a significantly stronger balance sheet and simplified capital structure, which we anticipate permits the funding of our development activities through 2019. Subsequent to our recent $46.0 million follow-on offering of common stock, at December 31, 2017, we had $113.7 million in cash and cash equivalents, which we expect will be sufficient to advance our DCR-PHXC development program through proof-of-concept trials and into advanced clinical development and advance our DCR-HBVS development program into clinical proof-of-concept studies in HBV patients, and provides a runway for us to further develop our earlier stage pipeline programs. As we advance through 2018, we are focused on seeking to achieve the key clinical milestones ahead of us, including dosing the first PH patient in our Phase 1 trial, the submission of clinical trial applications (CTAs) and/or investigational new drug (IND) applications for our other priority programs, and sharing clinical proof-of-concept data from our DCR-PHXC Phase 1 study in the second half of 2018.”
GalXC™ Research Collaboration
GalXC™ Pipeline Program Update
Financial Condition and Operating Results
For more detailed information and analysis, see Dicerna’s Annual Report on Form 10-K for the year ended December 31, 2017, which was filed with the Securities and Exchange Commission (SEC) on March 8, 2018.
Dicerna believes that it has sufficient cash to fund the execution of its current clinical and operating plan through 2019, which includes focusing its resources on advancing its DCR-PHXC development program through proof-of-concept trials and into advanced clinical development, and advancing its DCR-HBVS development program into proof-of-concept studies in HBV patients. This estimate assumes no new funding from additional collaboration agreements or from external financing events.
Management will host a conference call at 4:30 p.m. ET today to review Dicerna's fourth quarter and year end 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 9997829 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 9997829. 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, viral infectious diseases, chronic liver diseases, and cardiovascular 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 GalXC™ RNAi Technology Platform
GalXC™ is a proprietary technology platform invented by Dicerna to discover and develop 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 the Company’s proprietary RNAi 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 duration and usage of current cash and cash equivalents. 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-K 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|
|December 31,||December 31,|
|Cash and cash equivalents||$||68,789||$||20,865|
|Total stockholders’ equity||$||101,238||$||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 Year Ended|
|December 31,||December 31,|
|Deemed dividend on|
|Net loss per share|
|- basic and|
|- basic and|
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