This Current Report on Form 8-K (this Current Report) updates the Annual
Report on Form 10-K of Dicerna Pharmaceuticals, Inc. (the Company or Dicerna) for the year ended December 31, 2015 (the 2015 Annual Report) and Dicernas most recent Quarterly Report on Form 10-Q
for the quarter ended June 30, 2016 (the 2016 2
nd
Quarter Report) to reflect, among other things, the following:
Effective in the third quarter of 2016, Dicerna will focus its resources on its GalXC
TM
RNAi
platform, for the treatment of rare diseases involving the liver and for other therapeutic areas involving the liver, such as chronic liver diseases, cardiovascular diseases and viral infectious diseases.
Under this plan, Dicerna will transition its primary hyperoxaluria (PH) development program to focus on DCR-PHXC, a subcutaneously delivered GalXC clinical
candidate, which was announced earlier this year. The Company will discontinue its development program for DCR-PH1, an investigational therapy formulated in an EnCore
TM
lipid nanoparticle
(LNP) delivery system obtained through a licensing agreement with Arbutus Biopharma Corporation (formerly known as Tekmira Pharmaceuticals Corporation). Based on the DCR-PH1 proof-of-concept data in humans, the utility of the
GalXC platform, and the DCR-PHXC preclinical data, the Company believes DCR-PHXC has the potential to be a better therapeutic candidate for patients with PH.
The Company will also discontinue clinical development of DCR-MYC, a DsiRNA-based therapeutic formulated as an LNP for delivery to solid tumors, because
preliminary results do not meet the Companys expectations for further development. In addition to DCR-MYC, Dicerna has a second oncology program, DCR-BCAT, which targets the WNT-beta-catenin pathway. Given the Companys focus on
advancing its GalXC-based programs, Dicerna will seek strategic alternatives to further develop DCR-BCAT, which employs an improved and enhanced EnCore LNP delivery capability, compared to earlier versions of the technology.
The preceding and certain other information is filed hereunder as Exhibit 99.1 and Exhibit 99.2 which are incorporated herein by reference. Item 9.01 of
this Current Report updates the information contained in Part I, Item 1, Business in the 2015 Annual Report and in Part II, Item 1A, Risk Factors in the 2016 2
nd
Quarter Report.
By virtue of this Current Report, the Company will be able to incorporate the updated
information by reference into future registration statements or post-effective amendments to existing registration statements. This Current Report does not update for any other changes since the filing of the 2015 Annual Report and the 2016 2
nd
Quarter Report.
Cautionary Note on Forward-Looking Statements
This Current Report (including the exhibits) includes forward-looking statements, including, for example, potential therapeutic and drug discovery
capabilities, the Companys expected timeline of development and licensing plans. 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 relating to the Companys clinical and preclinical research and other risks identified under the heading Risk Factors included in Exhibit 99.2 to this Current Report
and in other future filings with the SEC. The forward-looking statements contained in this Current Report reflect Dicernas current views with respect to future events, and Dicerna does not undertake and specifically disclaims any obligation to
update any forward-looking statements.