News Column

Dicerna Provides Operational Update and Reports Second Quarter 2014 Financial Results

August 18, 2014

By a News Reporter-Staff News Editor at Clinical Trials Week -- Dicerna Pharmaceuticals, Inc. (NASDAQ: DRNA), a leader in the development of RNAi-based therapeutics targeting genetically defined oncology and inherited rare diseases, announced operational and financial results for the quarter ending June 30, 2014 (see also Dicerna Pharmaceuticals, Inc.).

"During Q2, Dicerna took the exciting step of launching a Phase 1 clinical trial with DCR-MYC for patients with solid tumors and hematological malignancies," stated Douglas M. Fambrough, Ph.D., Dicerna's President and CEO. "This study marks the first clinical trial of a Dicer Substrate RNA interference (DsiRNA) drug candidate and highlights the potential for RNAi to modulate drug targets that have been previously considered undruggable. We are utilizing this technology to develop a pipeline of novel DsiRNA therapeutics, including DCR-MYC and DCR-PH1 for the treatment of Primary Hyperoxaluria 1 (PH1)." Oncology Program Updates DCR-MYC is a potent and specific inhibitor of MYC, an oncogene that is frequently amplified or otherwise upregulated in a wide variety of cancer tumor types, including hepatocellular carcinoma (HCC). The MYC oncogene encodes for a small intracellular protein that lacks a good binding site, making it a challenging target for monoclonal antibody therapies or traditional small molecules. Historically, this challenge has led to MYC being deemed an undruggable target. However, DsiRNA-based therapeutics may be able to overcome these challenges - Dicerna has shown that DCR-MYC knocked down gene transcript activity and reduced tumor volume in multiple mouse tumor models, involving a variety of tumor types. Phase 1 DCR-MYC Trials in Solid Tumors and Hepatocellular Carcinoma (HCC) In April 2014, Dicerna initiated a Phase 1 dose-escalating clinical study of DCR-MYC in patients with solid tumors, multiple myeloma, or lymphoma.

Dicerna expects top-line data from this Phase 1 trial in 2015.

Dicerna plans to initiate a second Phase 1 trial in hepatocellular carcinoma (HCC) in U.S. and Asia in the second-half of 2014. Rare Disease Program Update DCR-PH1 is a therapeutic candidate for Primary Hyperoxaluria 1 (PH1), a rare, inherited autosomal recessive disorder of metabolism in the liver that usually results in life-threatening damage to the kidneys. In the genetic mouse model of PH1, Dicerna has shown that DCR-PH1 inactivates the gene encoding glycolate oxidase, significantly reducing the production of oxalate, the key disease pathology of PH1.

In June 2014, preclinical data were presented at the 11th International Primary Hyperoxaluria Workshop by Eduardo Salido, Ph.D., Professor of Pathology at the University of La Laguna in Santa Cruz de Tenerife, Spain showing that DCR-PH1 provides potent and long-term inhibition of HAO1, a gene implicated in the pathogenesis of PH1.

Dicerna expects to initiate a Phase 1 trial of DCR-PH1 in 2015. Financial Results Cash Position - As of June 30, 2014, the Company had $120.3 million in cash and cash equivalents and held-to-maturity investments as compared to $46.6 million in cash and cash equivalents as of December 31, 2013. In February 2014, the Company completed its initial public offering (IPO) of common stock, raising net proceeds of approximately $92.7 million. Non-Operating Uses of Cash & Cash Equivalents - On April 7, 2014, we repaid the remaining amount of a secured term loan from Hercules Technology Growth Capital of approximately $3.6 million. In addition, for accounting purposes, we classified $1.1 million as restricted cash to reflect the collateral securing a letter of credit established in conjunction with the lease agreement for our new facility signed on July 11, 2014. R&D Expenses - Research and development expenses for the second quarter were $6.8 million, compared to $2.5 million for the same period in 2013, due primarily to the initiation of clinical development activities and advancement of our pre-clinical programs and increased employee-related expenses, including stock-based compensation of $0.9 million. G&A Expenses - General and administrative expenses for the second quarter were $4.4 million, compared to $1.1 million for the same period in 2013. The increase was primarily from the increased costs of operating as a public company and increased employee-related costs, including stock-based compensation of $1.5 million. Net Loss - Net loss for the second quarter was $11.4 million compared to a net loss of $3.8 million for the same period in 2013.

More detailed financial information and analysis may be found in the Company's Quarterly Report on Form 10-Q, which was filed with the Securities and Exchange Commission (SEC) on August 7, 2014. Guidance Based on our current cash position and operating plan, the Company re-iterates its expectation that it has sufficient cash to fund operations through 2016. 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 second-quarter financial results. The call can be accessed by dialing (855) 453-3834 or (484) 756-4306, and referencing conference ID 75498270. 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,, prior to the even. The webcast will also be archived on the Company's website. About Dicerna Pharmaceuticals, Inc.

Keywords for this news article include: Dicerna Pharmaceuticals Inc., Genetics, Oncology, Pathology, Technology, Investment and Finance, Hepatocellular Carcinoma, Clinical Trials and Studies.

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Source: Clinical Trials Week

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