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Dicerna Pharmaceuticals Initiates Phase 1 Study of DCR-MYC in Patients with Solid Tumors and Hematological Malignancies
[April 16, 2014]

Dicerna Pharmaceuticals Initiates Phase 1 Study of DCR-MYC in Patients with Solid Tumors and Hematological Malignancies


WATERTOWN, Mass. --(Business Wire)--

Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA), a leader in the development of RNAi-based therapeutics, today announced the initiation of a Phase 1 dose-escalating clinical study of DCR-MYC, (also known as DCR-M1711), in patients with solid tumors, multiple myeloma, or lymphoma. DCR-MYC, Dicerna's first drug candidate to enter clinical testing, is a Dicer Substrate siRNA (DsiRNA) that targets the driver oncogene MYC, which is central to the growth of many hematologic and solid tumor malignancies. Dicerna is investigating DCR-MYC in a variety of tumor types with the initial focus on hepatocellular carcinoma.

"The MYC oncogene has been a therapeutic target of great interest for many years because of its expression in a variety of tumor types and the demonstration that increased MYC expression is related to the progression of these cancers," commented Anthony W. Tolcher, M.D., Director of Clinical Research at Southern Texas Accelerated Research Therapeutics, a principal investigator in the study. "The DsiRNA approach holds the possibility to overcome the challenges faced by previous drug development efforts to modulate this important target by directly interfering with the expression of MYC."

"In preclinical studies, DCR-MYC demonstrated impressive knockdown of the MYC oncogene," stated Pankaj Bhargava, M.D., Chief Medical Officer of Dicerna. "The Phase 1 trial of DCR-MYC now provides u the opportunity to test these promising data in clinical trials, furthering our efforts to realize the significant therapeutic potential of Dicer Substrate RNA interference."



About the Phase 1 Study of DCR-MYC

The multi-center, dose-escalating Phase 1 study will assess the safety and tolerability of DCR-MYC in patients with solid tumors, multiple myeloma, or lymphoma who are refractory or unresponsive to standard therapies. DCR-MYC will be administered by one hour intravenous (IV) infusion, once weekly for three weeks followed by a rest week. The goals of this study are to identify the maximum tolerated dose and study the pharmacokinetic profile, potential pharmacodynamic effects, and antitumor activity of DCR-MYC.


About DCR-MYC

DCR-MYC (DCR-M1711) is Dicerna's novel Dicer Substrate siRNA, a synthetic double-stranded RNA in a stable lipid particle designed to serve as a potent and specific inhibitor of the MYC oncogene. MYC is a key target in oncology because it has been demonstrated to cause or promote cancer when abnormally expressed or activated. Abundant genetic data implicates the MYC oncogene in promoting tumors and inhibition of MYC has exhibited strong anti-tumor effects in numerous animal models of human cancers. Dicerna is investigating DCR-MYC in a variety of tumor types with the initial focus on hepatocellular carcinoma, which is the third leading cause of cancer death worldwide.

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 (News - Alert) 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.


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