Corporate Overview. Douglas Fambrough, President and CEO Cowen and Company 39 th Annual Health Care Conference March 12, DICERNA

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1 Corporate Overview Douglas Fambrough, President and CEO Cowen and Company 39 th Annual Health Care Conference March 12, 2019

2 Forward-looking Statements This information may contain projections and other forward looking statements regarding future events, including statements regarding Dicerna s technology platform, product candidates, preclinical and clinical pipeline and milestones, regulatory objectives, market opportunities, and intellectual property. Such statements are predictions only and are subject to risks and uncertainties that could cause actual events or results to differ materially. These risks and uncertainties include, among others, the cost, timing and results of preclinical studies and clinical trials and other development activities; the unpredictability of the duration and results of regulatory review of New Drug Applications (NDAs) and Investigational NDAs; market acceptance for approved products and innovative therapeutic treatments; competition; the possible impairment of, inability to obtain and costs of obtaining intellectual property rights; and possible safety or efficacy concerns, general business, financial and accounting risks and litigation. More information concerning Dicerna and such risks and uncertainties is available on its website and in its press releases, and in its public filings with the U.S. Securities and Exchange Commission. 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. 2

3 Dicerna Vision and Strategy: Driving RNAi Therapies Forward for Patients Vision Build a fully integrated company by developing innovative RNAi-based therapies for high unmet medical needs and broadly capture the value of our GalXC platform Strategy Build Value in rare diseases by independently developing and commercializing programs with high value and high probability of success Capture Value in common diseases by developing high probability of success programs through clinical proof of concept before seeking development and commercialization partners Enhance Value by partnering with therapeutic field leaders on discovery stage programs 3

4 The Foundation of Our Value Dicer substrate (DsiRNA) RNAi trigger GalNAc targeting ligands Proprietary, patented RNA interference technology Objectively excellent pharmaceutical properties Subcutaneously delivered convenient administration Long duration of action convenient regimens High target specificity predictable activity High therapeutic index broad applicability Potential to extend the GalXC platform to diverse tissues beyond the liver 4

5 Dicerna Development Pipeline CANDIDATE INDICATION RESEARCH PRECLINICAL CLINICAL POC TRIALS REGISTRATION TRIALS PARTNER DCR-PHXC Primary Hyperoxalurias DCR-HBVS Hepatitis B Virus DCR-undisclosed Rare Disease DCR-undisclosed Undisclosed DCR-LIV1 NASH Boehringer Ingelheim DCR-LIV2 NASH Boehringer Ingelheim DCR-CM1 Cardiometabolic Lilly DCR-CM2 Cardiometabolic Lilly DCR-CM3 Cardiometabolic Lilly DCR-NEURO1 Neurodegeneration Lilly DCR-NEURO2 Neurodegeneration Lilly DCR-COMP1 Complement-mediated Alexion DCR-COMP2 Complement-mediated Alexion 5 ORPHAN PREVALANT

6 Select Dicerna GalXC Programs: Primary Hyperoxalurias

7 The Primary Hyperoxalurias (PH) A family of rare, inherited, liver metabolic disorders resulting in oxalate overproduction PH Type 1: PH Type 2: PH Type 3: Most serious form of PH Median age of kidney failure mid-20s Systemic oxalosis Very serious, chronic stones with significant risk of kidney failure Chronic stones, especially in youth Disease Progression of PH Type 1 (PH1) Abnormal liver metabolism produces excess oxalate Calcium oxalate crystals form in the kidneys Decline in kidney function results in systemic oxalosis Primary hyperoxaluria 1 Peroxisome Glycine AGT Glycolate Glyoxylate LDH GO HEPATOCYTE Oxalate Primary hyperoxaluria 2 & 3 Glycolate GRHPR Glyoxylate DCR-PHXC Pathway Inputs HOGA Mitochondria Median age of onset of kidney failure is mid-20s 7 Patients require intensive daily dialysis while awaiting a liver-kidney transplant DCR-PHXC silences LDHA, the final common pathway of oxalate production, for the treatment of all forms of PH

8 PHYOX : An Ongoing Phase 1 Study of DCR-PHXC in PH1 and PH2 A two-part, single-ascending dose study (ClinicalTrials.gov: NCT ) PHYOX: A Placebo-Controlled, Single-Blind, Single-Center Phase 1 Study in Healthy Volunteers and Open-Label Multi-Center Study in Patients With Primary Hyperoxaluria (PH) to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Single Ascending Doses of DCR-PHXC Solution for Injection (Subcutaneous Use) Group A: Healthy volunteers 25 healthy volunteers Randomized 3:2 DCR-PHXC to placebo 5 cohorts: 0.3, 1.5, 3.0, 6.0, 12.0 mg/kg 5 subjects per cohort Group A is complete Group B: PH Patients 18 PH1 and PH2 patients, open label Genetically confirmed diagnosis Uox 0.7mmol/24hr egfr 30mL/min/1.73m 2 PH1 cohorts: 1.5, 3.0, 6.0 mg/kg PH2 cohort: variable dosing Trial dosing is complete Cohort 1 (1.5 mg/kg): - 3/4 participants 24Hr Uox values reached near-normalization (<0.6 and 0.46 mmol/24hr) at one or more post-dose time points - Mean maximal 24Hr Uox reduction = 50% (range: 39%-59%) - Per protocol, 2 participants are still in follow-up as of post-dose Day 85, as their 24Hr Uox has not yet returned to within 80% of the lowest baseline 24Hr Uox measurement Cohort 2 (3.0 mg/kg): - 3/4 participants 24Hr Uox values have reached normalization (<0.46 mmol/24hr) at one or more post-dose time points - Mean maximal 24Hr Uox reduction = 65% (range: 56%-80%) - 3 participants are still in follow-up and may not yet have reached maximal 24Hr Uox reductions Cohort 3 (6.0 mg/kg): - As of October 1, 2018 data cut, only 1 participant has 24Hr Uox results - Maximum reduction for that 1 participant = 64% as of post-dose Day 43 8

9 9 Mean [± SEM] Uox Content (mmol/24hr) PHYOX: Uox Reduction in Patients After a Single Dose PD and safety/tolerability results lay groundwork for planned pivotal trial Mean 24Hr Oxalate Over Time, Following Single Administration of DCR-PHXC Single Dose DCR-PHXC Days After Single Dose * * * * Mean [± SEM] Uox:Cre (umol/mmol) 1.5 mg/kg 3.0 mg/kg Mean Oxalate-to-Creatinine Ratio Over Time, Following Single Administration of DCR-PHXC Single Dose DCR-PHXC Days After Single Dose At 1.5 mg/kg, n=5 at all time points, except where 057* denotes data point where only 3 participants are included. At 3.0 mg/kg, n=4 at all time points, except where * denotes data point where only 3 participants are included. Results based on availability of data as of October 1, * * * *

10 Screening to Maximum Observed Reduction in 24hr Urinary Oxalate 6/10 patients achieve normalization or near-normalization; 8/10 patients with >50% Uox reduction 2500 Observed Uox Values (µmol/l) Screening to Maximal Reduction 100 % Reduction in Uox Maximal Reduction vs. Screening Uox µmol/l and <600 near-normal % Change <460 normal Screening Maximal Reduction 0 Screening Maximal Reduction 10

11 DCR-PHXC: Moving into Approval Campaign in Q Coordinated program of Phase 2 pivotal trial and additional supportive trials PHYOX2 (PIVOTAL TRIAL): Double-blind, randomized, placebo-controlled trial (2:1 randomization) in approx. 36 patients with PH1 and PH2 (Q initiation). Convenient, monthly fixed-dose regimen, enabling pre-filled syringes or autoinjectors at product launch. PHYOX3 (ROLL-OVER STUDY): Patients from PHYOX may be re-enrolled into a long-term, multi-dose open label extension trial, allowing continuous readout of multi-dose data (Q initiation) PH3 Patient Trial: Open-label study in 10 patients with PH3 ESRD Patient Trial: Single dose trial in adults with end stage renal disease (ESRD) to determine PK Pediatric Trial: Open-label study in children (2-5 years) Additional trials exploring dosing paradigms are also anticipated These plans may be adjusted pending outcome of regulatory meetings in Q

12 DCR-PHXC is the Only RNAi Drug Candidate in Development for all PH Types DCR-PHXC significantly expands to the patient pool compared to PH1-specific therapy The Genetics of PH Current Diagnosis Rates Genetic prevalence (per million) PH1 PH2 PH US 2,681 1,655 4,098 EU 2,607 1,609 3,986 Total Potential Patients 5,288 3,264 8,084 PH1 PH2 PH3 Hoppe et al 2003 (US) 20.5% 3.8% Unknown Hoppe et al 2005 (Germany) 20.3% 2.0% Unknown OxalEUROPE 23.2% 4.0% Unknown We anticipate that diagnostic rates will increase with an available effective treatment, especially for PH2 Epidemiology, diagnostic and uptake models developed by the company predict peak sales between $500M and $1B 12 J Am Soc Nephrol Oct;26(10):supp, and applied to population sizes Pediatr Nephrol Oct;18(10): Am J Nephrol May-Jun;25(3): Am J Nephrol. 2005; 25:

13 Select Dicerna GalXC Programs: Chronic Hepatitis B Infection

14 Hepatitis B: A Severe, Global Unmet Medical Need Massive worldwide economic burden: ~257 million chronically infected (WHO) >10th leading cause of death worldwide: 887,000 in 2015 (WHO) Asymptomatic during the acute infection phase: just 9% of all HBV infections were diagnosed in 2015, and just 8% of those diagnosed were on treatment (WHO) Responsible for 80% of primary liver cancers Current treatments are rarely effective in achieving functional cures Electron micrograph of HBV showing infectious viral particles (~42 nm) and non-infectious sub viral decoy particles (~22 nm) and filaments 14

15 GalXC RNAi May Play a Key Role in Establishing a Functional HBV Cure Organization of the HBV genome enables effective RNAi targeting of multiple viral functions Current HBV Therapies Are Insufficient Functional Cure of chronic HBV is the best treatment outcome currently 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 The Promise of RNAi for HBV RNAi can simultaneously inhibit multiple viral activities due to overlapping transcripts Overlapping mrnas and protein-coding regions enable targeting multiple HBV genes and proteins with a single GalXC trigger P gene: Polymerase. Viral genome production Polymerase 2,458 2,856 2,309 3,221 EcoRI DCR-HBVS target site pres1, pres2 and S 834 S gene: Surface protein. HBsAg, hepatocyte entry and immune decoy Viral genome AAAAA Viral mrna transcript RNAi can target viral transcripts and pgrna from cccdna and integrated genomes DCR-HBVS Has Entered Clinical Development Healthy volunteer dosing underway First chronic HBV patient dosing expected Q C gene: Core protein. Capsid assembly; E antigen secretion precore, Core 1,873 1,622 1,816 X gene 1,376 X gene: Epigenetic maintenance of viral genome Region of viral genome conservation 15

16 Single Dose HBsAg Reduced to Below Lower Level of Detection Driven by striking pharmacodynamic differences between targeting in the S versus X ORFs %HBsAg +/- SEM (Normalized to d0) mg/kg qwx3 HDI-HBV Plasmid Model (cccdna-dependent) 0 1 2/3 BLOQ 2 3 Vehicle Control 4 3/3 BLOQ Time (weeks) (days) GalXC-HBVS: 3.9 log reduction, long duration of activity 5 X gene targeted: 3.0 log reduction, shorter duration of activity X Gene targeted treatment GalXC-HBVS Vehicle Ctl. X targeted GalXC-HBVS HDI-HBV Immunohistochemical staining of mouse liver sections for HBV Core Protein (HBc) reveals extreme differences in subcellular localization of HBc in the HDI-HBV plasmid model These results have been reproduced using alternative guide strand sequences (i.e., different mrna binding sites) for both GalXC-HBVS and GalXC-HBVX 16

17 DCR-HBVS Clinical Program for Proof of Concept Includes placebo-controlled studies in both NUC-naïve and NUC-experienced patients Three Part Study in Healthy Volunteers and Chronic HBV Patients Group A: Single dose ascending dose study, placebo controlled, in 30 healthy volunteers - 5 dose cohorts: 0.1, 1.5, 3.0, 6.0, 12.0 mg/kg Group B: Single-dose study, placebo controlled, in 8 NUC-naïve chronic hepatitis B patients - 1 dose cohort at 3 mg/kg - Introduction of NUCs after 12 weeks Group C: Multiple dose-ascending dose study, placebo controlled, in 18 NUC-experienced chronic hepatitis B patients - 3 dose cohorts: 1.5, 3.0, and 6.0 mg/kg, 4 monthly doses, in parallel to Group A after 2nd cohort complete - Patients with HBsAg seroclearance will be followed up for seroconversion (anti-hbsag) Study Launched in Q Filed Clinical Trial Application (CTA) with the New Zealand Medicines and Medical Devices Safety Authority (MedSafe) and the Health and Disability Ethics Committee (HDEC). Further CTA submissions in Asia-Pacific planned Dosed first healthy volunteer in January 2019 and expect to dose the first patient in Q Interim data expected in Q4 2019

18 Select Dicerna GalXC Programs: Collaborations with Lilly, Alexion and Boehringer Ingelheim

19 Realizing Value Through Strategic Collaborations Developing GalXC candidates with established pharmaceutical partners Lilly: cardiometabolic, neurodegenerative and pain targets Alexion: complement pathway targets Boehringer Ingelheim: NASH targets Expanding the range of the GalXC technology platform to go beyond liver tissue Neural tissues: exclusive collaboration with Lilly to develop RNAi neural delivery technology; Dicerna retains rights to select rare diseases for Dicerna development Cardiometabolic tissue: non-exclusive collaboration with Lilly Providing resources to drive our strategic programs $252 million in upfront and option payments and equity at a premium among the three collaborations 19

20 Elements of Portfolio Strategy We seek to generate value across the full spectrum of GalXC clinical applications Highest Resource Investment ORPHAN DISEASE Strategic Programs COMMON DISEASE Partnered Programs COMMON DISEASE ORPHAN DISEASE Lower Resource Investment Dicerna drives to market Dicerna drives to clinical POC Dicerna discovers Partner develops Dicerna discovers Partner develops Primary Hyperoxalurias Hepatitis B Virus Lilly & Boehringer Collaborations Alexion Collaboration Undisclosed indication Undisclosed indication DCR-LIV1 DCR-LIV2 DCR-NEURO1 DCR-NEURO2 DCR-CM1 DCR-CM2 DCR-CM3 DCR-COMP1 DCR-COMP2 Key Value Drivers Additive Value 20

21 2019 Milestones Building Value, Capturing Value, Enhancing Value Advance DCR-PHXC for the treatment of primary hyperoxaluria (PH) Initiate PHYOX2, a pivotal multi-dose, double-blind, randomized, placebo-controlled trial Initiate PHYOX3, a long-term, multi-dose, open-label, registration roll-over extension trial Report longer-term clinical data from PHYOX3 Advance DCR-HBVS for the treatment of patients with chronic hepatitis B virus (HBV) Dose first healthy volunteer in Phase 1 trial Dose first patient with non-cirrhotic chronic HBV in Phase 1 trial Report proof-of-concept data Advance investigational GalXC therapy for the treatment of an undisclosed rare disease File clinical trial application Achieve anticipated corporate milestones Evolve Board composition and expand leadership team to support continued growth 21

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