8-K

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

 

 

FORM 8-K

 

 

CURRENT REPORT

PURSUANT TO SECTION 13 OR 15(d)

OF THE SECURITIES EXCHANGE ACT OF 1934

Date of Report (Date of earliest event reported): January 8, 2020

 

 

DICERNA PHARMACEUTICALS, INC.

(Exact name of registrant as specified in its charter)

 

 

 

Delaware   001-36281   20-5993609

(State or other jurisdiction

of incorporation)

  (Commission
File Number)
 

(IRS Employer

Identification Number)

 

 

33 Hayden Avenue

Lexington, Massachusetts

   02421  
  (Address of registrant’s principal executive office)    (Zip code)  

(617) 621-8097

(Registrant’s telephone number, including area code)

 

(Former name or former address, if changed since last report)

 

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

 

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class

 

Trading

symbol(s)

 

Name of each exchange

on which registered

Common Stock, par value $0.0001 per share   DRNA   The Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 or Rule 12b-2 of the Securities Exchange Act of 1934.

Emerging growth company  ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  ☐

 

 

 


Item 2.02

Results of Operations and Financial Condition

The information contained in Exhibit 99.1 and described in Item 7.01 of this Form 8-K, as it relates to the cash balance of Dicerna Pharmaceuticals, Inc. (the “Company”) for the year ended December 31, 2019, is incorporated herein by reference.

 

Item 5.02

Departure of Directors or Certain Officers; Election of Directors; Appointment of Certain Officers; Compensatory Arrangements of Certain Officers.

Approval of Form of Restricted Stock Unit Award Agreement and Form of Restricted Stock Unit Grant Notice

On January 8, 2020, the Compensation Committee (the “Committee”) of the Board of Directors of the Company adopted a form of Restricted Stock Unit Award Agreement (the “RSU Agreement”) and a form of Restricted Stock Unit Grant Notice ( the “RSU Grant Notice”) under the Company’s 2014 Amended and Restated Performance Incentive Plan (the “Plan”).

The RSU Agreement provides for the grant of restricted stock unit awards (“RSUs”) consisting of the right to receive, upon the vesting date of such RSUs, one share of common stock of the Company for each vested RSU. RSUs generally will vest in equal annual installments over the period specified for each award. The vesting of the RSUs is further subject to the grantee’s continued employment or service through the vesting date.

A copy of the RSU Agreement and the RSU Grant Notice are filed as Exhibit 10.1 and Exhibit 10.2, respectively, and are incorporated herein by reference. The foregoing description of the RSU Agreement is qualified in its entirety by reference to the full text of the RSU Agreement.

Promotional Grants

On January 8, 2020, the Committee approved a grant to Bob Brown of 21,300 stock options and 5,700 RSUs for his promotion on September 17, 2019 to Executive Vice President, Research and Development. The Committee further approved a grant to James Weissman of 21,300 stock options and 5,700 RSUs for his promotion on September 17, 2019 to Executive Vice President and Chief Operating Officer.

 

Item 7.01.

Regulation FD Disclosure

The Company from time to time presents and/or distributes to the investment community at various industry and other conferences slide presentations to provide updates and summaries of its business. The Company is posting to the “Investors” portion of its website at http://investors.dicerna.com/ a copy of its current corporate slide presentation. These slides are attached to this Current Report on Form 8-K as Exhibit 99.1. The Company undertakes no obligation to update, supplement or amend the materials attached hereto as Exhibit 99.1.

The information in Items 2.02 and 7.01 of this Form 8-K, including Exhibit 99.1 attached hereto, shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”) or otherwise subject to the liabilities of that section, nor shall it be deemed incorporated by reference in any filing under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such a filing.

 

Item 9.01

Financial Statements and Exhibits.

(d) Exhibits

 

Exhibit
No.

  

Description

10.1    Form of Restricted Stock Unit Award Agreement
10.2    Form of Restricted Stock Unit Grant Notice
99.1    Dicerna Pharmaceuticals, Inc. corporate presentation.


SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, as amended, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

Date: January 13, 2020     DICERNA PHARMACEUTICALS, INC.
    By:  

/s/ Douglas M. Fambrough, III

      Douglas M. Fambrough, III, Ph.D.
      Chief Executive Officer
EX-10.1

Exhibit 10.1

DICERNA PHARMACEUTICALS, INC.

AMENDED AND RESTATED 2014 PERFORMANCE INCENTIVE PLAN

RESTRICTED STOCK UNIT AWARD AGREEMENT

THIS STOCK UNIT AWARD AGREEMENT (this “Agreement”) is dated as of [            , 2020] by and between Dicerna Pharmaceuticals, Inc., a Delaware corporation (the “Corporation”), and [                    ] (the “Participant”).

W I T N E S S E T H

WHEREAS, pursuant to the Dicerna Pharmaceuticals, Inc. Amended and Restated 2014 Performance Incentive Plan (the “Plan”), the Corporation has granted to the Participant effective as of the date hereof (the “Award Date”), a credit of stock units under the Plan (the “Award”), upon the terms and conditions set forth herein and in the Plan.

NOW THEREFORE, in consideration of services rendered and to be rendered by the Participant, and the mutual promises made herein and the mutual benefits to be derived therefrom, the parties agree as follows:

1.    Defined Terms. Capitalized terms used herein and not otherwise defined herein shall have the meaning assigned to such terms in the Plan.

2.    Grant. Subject to the terms of this Agreement, the Corporation hereby grants to the Participant an Award with respect to an aggregate of [                    ] restricted stock units (subject to adjustment as provided in Section 7.1 of the Plan) (the “Stock Units”). As used herein, the term “stock unit” shall mean a non-voting unit of measurement which is deemed for bookkeeping purposes to be equivalent to one outstanding share of the Corporation’s Common Stock (subject to adjustment as provided in Section 7.1 of the Plan) solely for purposes of the Plan and this Agreement. The Stock Units shall be used solely as a device for the determination of the payment to eventually be made to the Participant if such Stock Units vest pursuant to Section 3. The Stock Units shall not be treated as property or as a trust fund of any kind.

3.    Vesting. Subject to Section 8 below, the Award shall vest and become nonforfeitable with respect to [twenty-five percent (25%) of the total number of Stock Units (subject to adjustment under Section 7.1 of the Plan) on each of the first, second, third and fourth anniversaries of the Award Date]. [Modify as needed for vesting terms of the particular grant.]

4.    Continuance of Employment/Service. The vesting schedule requires continued employment or service through each applicable vesting date as a condition to the vesting of the applicable installment of the Award and the rights and benefits under this Agreement. Employment or service for only a portion of the vesting period, even if a substantial portion, will not entitle the Participant to any proportionate vesting or avoid or mitigate a termination of rights and benefits upon or following a termination of employment or services as provided in Section 8 below or under the Plan.

Nothing contained in this Agreement or the Plan constitutes an employment or service commitment by the Corporation, affects the Participant’s status as an employee at will who is subject to termination without cause, confers upon the Participant any right to remain employed

 

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by or in service to the Corporation or any Subsidiary, interferes in any way with the right of the Corporation or any Subsidiary at any time to terminate such employment or services, or affects the right of the Corporation or any Subsidiary to increase or decrease the Participant’s other compensation or benefits. Nothing in this Agreement, however, is intended to adversely affect any independent contractual right of the Participant without his or her consent thereto.

5.     Rights as a Stockholder. The Participant shall have no rights as a stockholder of the Corporation, no dividend rights and no voting rights, with respect to the Stock Units and any shares of Common Stock underlying or issuable in respect of such Stock Units until such shares of Common Stock are actually issued to and held of record by the Participant. No adjustments will be made for dividends or other rights of a holder for which the record date is prior to the date of issuance of such shares.

6.    Restrictions on Transfer. Neither the Award, nor any interest therein or amount or shares payable in respect thereof may be sold, assigned, transferred, pledged or otherwise disposed of, alienated or encumbered, either voluntarily or involuntarily. The transfer restrictions in the preceding sentence shall not apply to (a) transfers to the Corporation, or (b) transfers by will or the laws of descent and distribution.

7.    Timing and Manner of Payment of Stock Units. On or as soon as administratively practical following each vesting of the applicable portion of the total Award pursuant to Section 3 hereof or Section 7 of the Plan (and in all events not later than two and one-half months after the end of the year in which the applicable vesting date occurs), the Corporation shall deliver to the Participant a number of shares of Common Stock (either by delivering one or more certificates for such shares or by entering such shares in book entry form, as determined by the Corporation in its discretion) equal to the number of Stock Units subject to this Award that vest on the applicable vesting date, unless such Stock Units terminate prior to the given vesting date pursuant to Section 8. The Corporation’s obligation to deliver shares of Common Stock or otherwise make payment with respect to vested Stock Units is subject to the condition precedent that the Participant or other person entitled under the Plan to receive any shares with respect to the vested Stock Units deliver to the Corporation any representations or other documents or assurances required pursuant to Section 8.1 of the Plan. The Participant shall have no further rights with respect to any Stock Units that are paid or that terminate pursuant to Section 8.

8.    Effect of Termination of Employment or Service. The Participant’s Stock Units shall terminate to the extent such units have not become vested prior to the first date the Participant is no longer employed by or in service to the Corporation or one of its Subsidiaries, regardless of the reason for the termination of the Participant’s employment or service with the Corporation or a Subsidiary, whether with or without cause, voluntarily or involuntarily; provided, however, that if the termination of the Participant’s employment or services is due to the Participant’s death or “permanent and total disability” (within the meaning of Section 22(e)(3) of the Code or as otherwise determined by the Administrator), the Award shall become vested and payable upon the date of such death or disability as to fifty percent (50%) of the then-outstanding and unvested portion of the Award (and any portion of the Award that is not vested after giving effect to such accelerated vesting shall terminate upon such death or disability). If any unvested Stock Units are terminated hereunder, such Stock Units shall automatically terminate and be cancelled as of the applicable termination date without payment of any consideration by the Corporation and without any other action by the Participant, or the Participant’s beneficiary or personal representative, as the case may be.

 

2


9.    Adjustments Upon Specified Events. Upon the occurrence of certain events relating to the Corporation’s stock contemplated by Section 7.1 of the Plan (including, without limitation, an extraordinary cash dividend on such stock), the Administrator shall make adjustments in accordance with such section in the number of Stock Units then outstanding and the number and kind of securities that may be issued in respect of the Award. No such adjustment shall be made with respect to any ordinary cash dividend for which dividend equivalents are credited pursuant to Section 5(b).

10.    Tax Withholding. Subject to Section 8.1 of the Plan, upon any distribution of shares of Common Stock in respect of the Stock Units, the Corporation shall automatically reduce the number of shares to be delivered by (or otherwise reacquire) the appropriate number of whole shares, valued at their then fair market value (with the “fair market value” of such shares determined in accordance with the applicable provisions of the Plan), to satisfy any withholding obligations of the Corporation or its Subsidiaries with respect to such distribution of shares at the minimum applicable withholding rates. In the event that the Corporation cannot legally satisfy such withholding obligations by such reduction of shares, or in the event of a cash payment or any other withholding event in respect of the Stock Units, the Corporation (or a Subsidiary) shall be entitled to require a cash payment by or on behalf of the Participant and/or to deduct from other compensation payable to the Participant any sums required by federal, state or local tax law to be withheld with respect to such distribution or payment.

11.    Notices. Any notice to be given under the terms of this Agreement shall be in writing and addressed to the Corporation at its principal office to the attention of the Secretary, and to the Participant at the Participant’s last address reflected on the Corporation’s records, or at such other address as either party may hereafter designate in writing to the other. Any such notice shall be given only when received, but if the Participant is no longer an employee of or in service to the Corporation, shall be deemed to have been duly given by the Corporation when enclosed in a properly sealed envelope addressed as aforesaid, registered or certified, and deposited (postage and registry or certification fee prepaid) in a post office or branch post office regularly maintained by the United States Government.

12.    Plan. The Award and all rights of the Participant under this Agreement are subject to the terms and conditions of the provisions of the Plan, incorporated herein by reference. The Participant agrees to be bound by the terms of the Plan and this Agreement. The Participant acknowledges having read and understanding the Plan, the Prospectus for the Plan, and this Agreement. Unless otherwise expressly provided in other sections of this Agreement, provisions of the Plan that confer discretionary authority on the Board or the Administrator do not (and shall not be deemed to) create any rights in the Participant unless such rights are expressly set forth herein or are otherwise in the sole discretion of the Board or the Administrator so conferred by appropriate action of the Board or the Administrator under the Plan after the date hereof.

13.    Entire Agreement. This Agreement and the Plan together constitute the entire agreement and supersede all prior understandings and agreements, written or oral, of the parties hereto with respect to the subject matter hereof. The Plan and this Agreement may be amended

 

3


pursuant to Section 8.6 of the Plan. Such amendment must be in writing and signed by the Corporation. The Corporation may, however, unilaterally waive any provision hereof in writing to the extent such waiver does not adversely affect the interests of the Participant hereunder, but no such waiver shall operate as or be construed to be a subsequent waiver of the same provision or a waiver of any other provision hereof.

14.    Limitation on Participant’s Rights. Participation in the Plan confers no rights or interests other than as herein provided. This Agreement creates only a contractual obligation on the part of the Corporation as to amounts payable and shall not be construed as creating a trust. Neither the Plan nor any underlying program, in and of itself, has any assets. The Participant shall have only the rights of a general unsecured creditor of the Corporation with respect to amounts credited and benefits payable, if any, with respect to the Stock Units, and rights no greater than the right to receive the Common Stock as a general unsecured creditor with respect to Stock Units, as and when payable hereunder.

15.    Counterparts. This Agreement may be executed simultaneously in any number of counterparts, each of which shall be deemed an original but all of which together shall constitute one and the same instrument.

16.    Section Headings. The section headings of this Agreement are for convenience of reference only and shall not be deemed to alter or affect any provision hereof.

17.    Governing Law. This Agreement shall be governed by and construed and enforced in accordance with the laws of the State of Delaware without regard to conflict of law principles thereunder.

18.    Construction. It is intended that the terms of the Award will not result in the imposition of any tax liability pursuant to Section 409A of the Code. This Agreement shall be construed and interpreted consistent with that intent.

19.    Clawback Policy. The Stock Units are subject to the terms of the Corporation’s recoupment, clawback or similar policy as it may be in effect from time to time, as well as any similar provisions of applicable law, any of which could in certain circumstances require repayment or forfeiture of the Stock Units or any shares of Common Stock or other cash or property received with respect to the Stock Units (including any value received from a disposition of the shares acquired upon payment of the Stock Units).

20.    No Advice Regarding Grant. The Participant is hereby advised to consult with his or her own tax, legal and/or investment advisors with respect to any advice the Participant may determine is needed or appropriate with respect to the Stock Units (including, without limitation, to determine the foreign, state, local, estate and/or gift tax consequences with respect to the Award). Neither the Corporation nor any of its officers, directors, affiliates or advisors makes any representation (except for the terms and conditions expressly set forth in this Award Agreement) or recommendation with respect to the Award. Except for the withholding rights set forth in Section 10 above, the Participant is solely responsible for any and all tax liability that may arise with respect to the Award.

[Remainder of page intentionally left blank]

 

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IN WITNESS WHEREOF, the Corporation has caused this Agreement to be executed on its behalf by a duly authorized officer and the Participant has hereunto set his or her hand as of the date and year first above written.

 

DICERNA PHARMACEUTICALS, INC.,

a Delaware corporation

    PARTICIPANT
By:  

     

   

 

      Signature
Print Name:  

     

                                   
Its:  

     

   

 

      Print Name

 

5

EX-10.2

Exhibit 10.2

DICERNA PHARMACEUTICALS, INC.

AMENDED AND RESTATED 2014 PERFORMANCE INCENTIVE PLAN

RESTRICTED STOCK UNIT GRANT NOTICE

Dicerna Pharmaceuticals, Inc. (the “Company”), pursuant to its Amended and Restated 2014 Performance Incentive Plan (the “Plan”), hereby awards to you, the Participant, the number of Restricted Stock Units (“RSUs”) set forth below (“Award”). This Award is subject to all of the terms and conditions as set forth herein and in the Restricted Stock Unit Award Agreement and the Plan, both of which are attached hereto and incorporated herein in their entirety.

 

Name and Address of Participant:   
Date of Grant:   
Total Number of RSUs Awarded1:   
Fair Market Value per RSU on Date of Grant:   
Total Fair Market Value of RSUs Awarded on Date of Grant:   
Vesting Commencement Date:   
Vesting Schedule:    Subject to the limitations contained herein and in the Restricted Stock Unit Award Agreement, your Award will vest with respect to twenty-five percent (25%) of the total number of RSUs (subject to adjustment under Section 7.1 of the Plan) on each of the first, second, third and fourth anniversaries of the Date of Grant. Your Award is also subject to continued employment or service through each applicable vesting date as set forth in the accompanying Restricted Stock Unit Award Agreement.

Additional Terms/Acknowledgements: You acknowledge receipt of, and understand and agree to, this Restricted Stock Unit Grant Notice, the Restricted Stock Unit Award Agreement and the Plan. You further acknowledge that as of the Date of Grant, this Restricted Stock Unit Grant Notice, the Restricted Stock Unit Award Agreement and the Plan set forth the entire understanding between you and the Company regarding the acquisition of RSUs of the Company and supersede all prior oral and written agreements on that subject with the exception of (i) Awards previously granted and delivered to you under the Plan, and (ii) the following agreements only:

 

OTHER AGREEMENTS:   

 

  
  

 

  

 

1 

The amount of shares to be issued upon settlement is subject to an automatic reduction to satisfy any withholding obligations as described in Section 10 of the Restricted Stock Unit Award Agreement.


DICERNA PHARMACEUTICALS, INC.:     PARTICIPANT:
By:  

 

   

 

  Signature       Signature
Name:  

 

    Date:  

 

  Print      
Title:  

 

     
Date:  

 

     

ATTACHMENTS: Restricted Stock Unit Award Agreement, Amended and Restated 2014 Performance Incentive Plan


ATTACHMENT I

RESTRICTED STOCK UNIT AWARD AGREEMENT


ATTACHMENT II

AMENDED AND RESTATED 2014 PERFORMANCE INCENTIVE PLAN

EX-99.1

Slide 1

January 2020 Corporate Overview Exhibit 99.1


Slide 2

This presentation has been prepared by Dicerna Pharmaceuticals, Inc. (“we,” “us,” “our,” “Dicerna,” or the “Company”) and includes forward-looking statements. 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. Examples of forward-looking statements include, among others, statements we make regarding: (i) the therapeutic and commercial potential of nedosiran (DCR-PHXC), DCR-HBVS, DCR-A1AT and the GalXC™ platform; (ii) research and development plans and timelines, as well as regulatory pathways and plans, related to nedosiran, DCR-HBVS, DCR-A1AT and GalXC; (iii) the potential of Dicerna’s technology and drug candidates in the Company’s research and development pipeline; (iv) the Company’s collaborations with Novo Nordisk A/S; Roche; Eli Lilly and Company; Alexion Pharmaceuticals, Inc.; Boehringer Ingelheim International GmbH; and (v) the Company’s strategy, business plans and focus. The process by which an early-stage investigational therapy such as nedosiran and an early-stage platform such as GalXC could potentially lead to an approved product is long and subject to significant risks. Applicable risks and uncertainties include those relating to Dicerna’s clinical research and other risks identified under the heading "Risk Factors" included in the Company’s most recent Form 10-K filing and in other subsequent filings with the Securities and Exchange Commission. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities; the likelihood of Dicerna’s clinical programs being executed within timelines provided and reliance on the Company’s contract research organizations and predictability of timely enrollment of subjects and patients to advance Dicerna’s clinical trials; the potential for future data to alter initial and preliminary results of early-stage clinical trials; the unpredictability of the duration and results of the regulatory review of Investigational New Drug (IND) applications and Clinical Trial Applications that are necessary to continue to advance and progress the Company’s clinical programs and the regulatory review of submissions relevant to regulatory agencies for marketing approvals, including New Drug Applications (NDAs); market acceptance for approved products and innovative therapeutic treatments; competition; the possible impairment of, inability to obtain and costs of obtaining needed intellectual property rights; possible safety or efficacy concerns that could emerge as new data are generated in R&D; that the Company may not realize the intended benefits of its collaborations; general business, financial and accounting risks; and the risks and potential outcomes from litigation.   Dicerna is providing this information as of this date and does not undertake any obligation to update or revise it, whether as a result of new information, future events or circumstances or otherwise. Additional information concerning Dicerna and its business may be available in press releases or other public announcements and public filings made after the date of this information. Dicerna™, GalXC™, and PHYOX™ are trademarks of Dicerna Pharmaceuticals, Inc. Forward-looking Statements


Slide 3

Dicerna Vision and Strategy VISION Develop and commercialize our core high-probability-of-success programs either alone or in collaboration with partners Broadly enable the use of our GalXC™ technology by collaborating with therapeutic area leaders on non-core opportunities STRATEGY Maximize the impact of RNAi on medicine


Slide 4

Dicerna Pipeline of Core and Collaborative Programs *Under its agreement with Roche, Dicerna has the option to co-fund pivotal development for heightened royalties and co-promotion rights in U.S. ORPHAN PREVALENT CANDIDATE TARGET INDICATION DISCOVERY/ RESEARCH PRECLINICAL CLINICAL PROOF-OF-CONCEPT TRIALS REGISTRATION TRIALS DICERNA’S PRODUCT RIGHTS Core Programs Nedosiran (DCR-PHXC) Primary Hyperoxaluria 100% DCR-HBVS* Hepatitis B Virus U.S. opt-in DCR-A1AT A1AT Liver Disease 100% DCR-undisclosed Undisclosed 100% NASH 1 target Discovery/Research Cardiometabolic (CM; liver and non-liver), neurodegeneration and pain Up to 8 CM targets DRNA retains rights to certain neuro orphan indications First IND/CTA expected late 2020 (LY3561774) HBV infection DCR-HBVS and multiple potential targets DRNA option to co-fund dev. and co-promote in U.S. Anticipated 2020 start Complement-mediated 4 targets Initially two targets; ALXN exercised option for two more targets in Dec. 2019 Liver-related cardiometabolic diseases 30+ potential targets 2 Novo programs: DRNA opt-in DRNA retains rights to 2 new orphan programs (Novo retains opt-in rights) Initial targets selected Currently active discovery or preclinical-stage liver program Currently active discovery or preclinical-stage non-liver program


Slide 5

Delivery agents Proprietary, patented RNA interference (RNAi) technology with potential to extend to diverse tissues beyond the liver Clinically compelling pharmaceutical properties The Foundation of Our Value GalXC RNAi trigger Subcutaneously delivered ® convenient administration Long duration of action ® infrequent dosing High target specificity ® predictable activity High therapeutic index ® broad applicability Established manufacturing ® scalable


Slide 6

Multiple Milestones Throughout 2020 Nedosiran: Multi-dose data from PHYOX™3 open-label clinical trial – 1H 2020 Nedosiran: PHYOX2 pivotal clinical trial enrollment completion – by the end of 1H 2020 DCR-HBVS: Phase 1 proof-of-concept data from all planned cohorts – mid-2020 DCR-A1AT: First patient dosing in Phase 1/2 trial – 2H 2020 Collaborative Program: IND or CTA filing for LY3561774 – late 2020 Nedosiran: PHYOX2 last patient out – by YE 2020 GalXC: Present data for extending GalXC technology to additional tissues – 2020


Slide 7

Primary Hyperoxaluria


Slide 8

A family of ultra-rare, life-threatening genetic disorders resulting in renal complications Primary Hyperoxaluria (PH) Oxalate Calcium oxalate crystallization Kidney stones Nedosiran Kidney function preservation Known Types of Primary Hyperoxaluria PH1 PH2 PH3 Genetic mutation AGXT Genetic mutation GRHPR Genetic mutation HOGA1 LDHA GO GO = glycolate oxidase LDHA = lactate dehydrogenase A Three known types of PH, each resulting from a mutation in one of three different genes, cause enzyme deficiencies manifesting in overproduction of oxalate Abnormal production and accumulation of oxalate leads to: Recurrent kidney stones Nephrocalcinosis (deposition of calcium in the kidney) Chronic kidney disease that may progress to end-stage renal disease, requiring regular dialysis and transplant (dual liver-kidney or kidney) Systemic oxalosis, which also impacts the heart, skin, eyes, bones Dicerna’s nedosiran silences LDHA, the ultimate step in the oxalate production pathway Impacted by systemic oxalosis


Slide 9

14/18 participants achieved normalization or near-normalization PHYOX1: Phase 1 Single-Dose Study of Nedosiran in PH1 and PH2 Participants Days After Single Dose Nedosiran Day One 3.0 mg/kg 6.0 mg/kg ≥0.46 & <0.60 near-normal <0.46 normal PHYOX1: Mean Urinary Oxalate Levels by Dose* PHYOX1 open-label study included: 18 participants dosed: PH1 (n=15) and PH2 (n=3) Genetically confirmed diagnosis Uox ≥0.7mmol/24hr eGFR ≥30mL/min/1.73m2 1.5, 3.0, 6.0 mg/kg doses delivered subcutaneously Normalization or near-normalization in 60%, 83% and 100% of patients at doses of 1.5, 3.0 and 6.0 mg/kg, respectively 2 of 3 PH2 patients with normalization or near-normalization The only drug-related AEs were mild to moderate injection-site reactions No drug-related SAEs were observed PH Type Dose (mg/kg) Pts. Reaching Normalization or Near-Normalization (%) Max Reduction Uox (%) Mean (range) PH1 1.5 (n=5) 3 (60) 51 (28-72) 3.0 (n=6) 5 (83) 72 (62-80) 6.0 (n=4) 4 (100) 72 (35-100) PH2 1.5 (n=1) 0 (0) 39 3.0 (n=2) 2 (100) 54 (42-66) *Days with at least two values ClinicalTrials.gov: NCT03392896 Uox Content (mmol/24 hr) Mean ±SEM


Slide 10

Coordinated program of clinical trials to support a broad label in PH Trial Description/Details Status PH Type Study 201: Pivotal, double-blind, randomized, placebo-controlled trial (2:1 randomization) Monthly fixed-dose, enabling prefilled syringes at launch Enrolling n=~36 1,2 Study 301: Long-term, multi-dose, open-label extension; rollover study open to all patients in PHYOX trials Enrolling 1,2,3 Study 104: Single-dose open-label study in patients with primary hyperoxaluria type 3 (PH3) 3 Study 204: Multi-dose trial in patients (birth to adult) with PH and end-stage renal disease (ESRD) 1,2 Study 203: Open-label study in children <6 yrs 1,2 Nedosiran Clinical Trial Program to NDA Filing Pivotal Study Package Additional Supportive Studies


Slide 11

High unmet medical need across all PH types can be addressed Nedosiran: The Only RNAi Drug Candidate in Development for All PH Types Genetics of PH PH1 PH2 PH3 All Known PH Types Genetic prevalence (per million) 8.23 5.08 12.58 25.89 US 2,681 1,655 4,098 8,434 EU 2,607 1,609 3,986 8,202 Estimated Prevalent Population (U.S. + EU) 5,288 3,264 8,084 16,636 J Am Soc Nephrol. 2015 Oct;26(10):supp, and applied to population sizes Dicerna estimates nedosiran peak sales between $500M and $1B Pre- and post-launch activities will improve diagnosis across all PH types Patient & Caregiver Support Availability of Nedosiran Physician Education


Slide 12

Chronic Hepatitis B Virus Infection


Slide 13

Significant worldwide prevalence: ~292 million infected Causes more than 887,000 deaths per year Current treatments are rarely effective in achieving functional cures Dicerna is collaborating with Roche to develop DCR-HBVS and potentially other agents for the treatment of HBV the treatment Hepatitis B: A Severe, Global Unmet Medical Need Electron micrograph of HBV showing infectious viral particles (~42 nm) and non-infectious sub-viral “decoy” particles (~22 nm) and filaments Sources: Global prevalence, treatment, and prevention of hepatitis B virus infection in 2016: a modelling study. The Lancet Gastroenterology and Hepatology. Volume 3, Issue 6, June 2018, Pages 383-403. World Health Organization. Finding a cure for hepatitis B: are we close? https://www.who.int/hepatitis/news-events/hbv-cure-overview/en/. Accessed Dec. 30, 2019.


Slide 14

Organization of the HBV genome enables effective RNAi targeting of multiple viral functions GalXC RNAi May Play a Key Role in Establishing a Functional HBV Cure The Promise of RNAi for HBV RNAi can simultaneously inhibit multiple viral activities due to overlapping transcripts RNAi can target all viral transcripts from cccDNA and integrated genomes Current HBV Therapies Are Inadequate Functional cure of chronic HBV would be the best treatment outcome Defined by the lack of detectable HBsAg in serum (often associated with seroconversion to anti-HBsAg+) Interferons and NUCs are the only approved therapies, but offer very low functional cure rates AAAAA P gene: Polymerase. Viral genome production S gene (HBsAg): Surface protein. Hepatocyte entry; Immune decoy X gene: Epigenetic maintenance of viral genome C gene: Core protein. Capsid assembly; E antigen secretion DCR-HBVS target site Overlapping mRNAs and protein-coding regions enable targeting multiple HBV genes and proteins with a single GalXC trigger AAAAA AAAAA AAAAA viral genome viral mRNA transcript


Slide 15

Time (weeks) 3mg/kg qWx3 Striking pharmacodynamic differences between targeting in the S alone vs. S and X ORFs in preclinical model Single-Dose GalXC-HBVS Reduced HBsAg to Below Lower Level of Detection GalXC-HBVS: ≥3.9 log reduction, long duration of activity X gene targeted: 3.0 log reduction, shorter duration of activity Immunohistochemical staining of mouse liver sections for HBV Core Protein reveals differential subcellular localization in the HDI-HBV plasmid model Silencing of X gene leads to nuclear localized Core Protein likely driving additional S expression These results have been reproduced using alternative guide strand sequences (i.e., different mRNA binding sites) for both GalXC-HBVS and GalXC-HBVX 2/3 BLOQ 3/3 BLOQ Vehicle control GalXC-HBVS HDI-HBV Plasmid Model (cccDNA-dependent) HDI-HBV Vehicle control GalXC-HBVS X targeted Only S gene targeted -1 0 1 2 3 4 5 6 7 8 9 0.001 0.01 0.1 1 10 100 10 11 12 %HBsAg +/- SEM (Normalized to d0) S and X genes targeted


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Includes placebo-controlled studies in both NUC-naïve and NUC-experienced patients Group A Placebo-controlled, single- ascending-dose study Completed n=30 DCR-HBVS dose cohorts: 0.1, 1.5, 3.0, 6.0, 12.0 mg/kg Conducted in healthy volunteers Group B Placebo-controlled, single-dose study in NUC-naïve patients with chronic HBV infection Dosing n=8 DCR-HBVS dose cohort: 3.0 mg/kg (NUCs initiated after 12 wks) Enrolls patients with no prior use of nucleoside or nucleotide analogue (NUC) therapy Group C Placebo-controlled, multiple- ascending-dose study in NUC-experienced patients with chronic HBV infection Dosing n=18 Enrolling 6.0 mg/kg cohort DCR-HBVS dose cohorts: 1.5, 3.0, 6.0 mg/kg; 4 monthly doses Patients with ≥1 log HBsAg suppression continue on study after 4 months. Multiple patients have continued on study. DCR-HBVS Clinical Program for Proof of Concept Three-Part Study in Healthy Volunteers and Patients With Chronic HBV Infection Data expected to be presented in mid-2020


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Alpha-1 Antitrypsin Deficiency-Associated Liver Disease


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Most disease is caused by a single missense allele that forms aggregates in hepatocytes Alpha-1 Antitrypsin Deficiency-Associated Liver Disease Alpha-1 antitrypsin (A1AT): predominantly produced in liver and secreted into blood Pi*ZZ genotype of the SERPINA1 gene affects 95% of patients with A1AT deficiency The Z-allele of the SERPINA1 gene produces an abnormal form of the protein, which can form pathological aggregates in hepatocytes Abnormal A1AT protein aggregates accumulate in liver, triggering injury cascade that can lead to liver disease Lack of normal A1AT protein can lead to lung disease, especially in smokers Janciauskiene et al. 2013: Acute Phase Proteins Loss of function deficiency Toxic gain of function a1-Antitrypsin Deficiency Z mutation Glu342®Lys342 Polymerization of a1-antitrypsin Intracellular accumulation neonatal hepatitis, liver cirrhosis Plasma deficiency early-onset emphysema


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Activity of DCR-A1AT in Mice and Non-Human Primates Potency and duration of action Relative Z-AAT Protein Concentration Mean ± SEM Relative A1AT Protein Concentration Mean ± SEM Weeks Post-Dose Single Subcutaneous Dose PBS 3.0 mg/kg 10.0 mg/kg Weeks Post-Dose 3.0 mg/kg 10.0 mg/kg Z-AAT Protein Knockdown in PiZ Mice A1AT Protein Knockdown in Monkeys Sustained, dose-dependent knockdown of A1AT protein in both mice and monkeys Single Subcutaneous Dose


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Part of larger clinical plan to achieve a rapid path to approval Two-part study: SAD in healthy volunteers (HVs) and MAD in patients with A1AT deficiency-associated liver disease HVs: Single-ascending-dose study, placebo-controlled, up to 36 patients 5 dose cohorts: 0.1, 1.0, 3.0, 6.0, 12.0 mg/kg, with a potential additional cohort Overlapping cohorts PK/PD data will be used to determine MAD regimen Patients with A1AT deficiency-associated liver disease: Multiple-ascending-dose study, up to 24 patients 2-3 dose cohorts: dose levels dependent on PK/PD data from HVs 2-4 doses to be administered within a 13-week period Currently dosing healthy volunteers Expect to dose first patient in 2H 2020 We believe the Phase 1/2 program will enable a pivotal trial without additional studies DCR-A1AT Clinical Program for Proof of Concept


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Corporate Collaborations


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Up to 8 cardiometabolic (CM) targets, liver and non-liver Neurodegeneration, pain targets DRNA retains rights to certain neuro orphan indications Successive collaborations increasing in scope and value Enhancing Value Through GalXC Collaboration Strategy Scope, Responsibility, Value 1 NASH target $5M option exercised 4 complement-mediated targets $20M option exercised $100M upfront $100M equity $200M upfront $175M upfront $75M total annuals $50M equity DCR-HBVS and multiple potential HBV-related targets DRNA option to co-fund development and co-promote in U.S. 30+ potential targets for liver-related CM diseases 2 Novo programs: DRNA opt-in DRNA retains rights to 2 new orphan programs (Novo retains opt-in rights) $10M upfront $22M upfront $15M equity Dicerna retains opt-in rights 2017 2018 2019


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Summary


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Primary Hyperoxaluria HBV (U.S. opt-in) Alpha-1 Antitrypsin Deficiency-Associated Liver Disease Undisclosed liver program Novo Nordisk program (U.S. opt-ins) Future programs beyond the liver We seek to generate value across the full spectrum of GalXC clinical applications Elements of Portfolio Strategy Highest Resource Investment Lower Resource Investment Complement-mediated Diseases Cardiometabolic Neurodegeneration Pain Value Retention Value-Add Core Programs Collaborative Programs Cardiometabolic and Liver-related Diseases Chronic HBV Infection NASH


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Strong Cash Position Provides Runway Through Commercialization Company well capitalized with approximately $350 million in cash (and short-term investments) as of January 1, 2020* including the following amounts received in December 2019: $20 million option exercise payment from Alexion $50 equity investment from Novo Nordisk Pro forma cash of approximately $725 million as of January 1, 2020, which also includes: $200 million upfront payment from Roche, received on January 7, 2020 $175 million upfront payment from Novo Nordisk expected in early Q1 2020 We expect our current cash and estimated future proceeds from existing collaborations will fund operations beyond 2022 *Unaudited estimated amount of cash, cash equivalents and marketable securities. Dicerna expects to announce audited full-year 2019 financial results, including year-end cash, cash equivalents and marketable securities, during Q1 2020. Additional cash upside expected from collaboration milestones and potential royalties on product sales


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Multiple Milestones Throughout 2020 Nedosiran: Multi-dose data from PHYOX™3 open-label clinical trial – 1H 2020 Nedosiran: PHYOX2 pivotal clinical trial enrollment completion – by the end of 1H 2020 DCR-HBVS: Phase 1 proof-of-concept data from all planned cohorts – mid-2020 DCR-A1AT: First patient dosing in Phase 1/2 trial – 2H 2020 Collaborative Program: IND or CTA filing for LY3561774 – late 2020 Nedosiran: PHYOX2 last patient out – by YE 2020 GalXC: Present data for extending GalXC technology to additional tissues – 2020


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