Viridian Therapeutics, Inc. · NASDAQ:VRDN
Viridian Therapeutics — Miragen Therapeutics — Jefferies Global Healthcare Conference 2017
30 slides from Viridian Therapeutics's conference presentation, Jun 8, 2017
Slide 1: miRagen Therapeutics
miRagen Therapeutics NASDAQ: MGEN Jefferies Global Healthcare Conference June 8, 2017
Slide 2: Cautionary Note Regarding Forward-Looking Statements
Cautionary Note Regarding Forward-Looking Statements This contains for ird-looki srl to Miragen Briscoe Inc., including statements about our plans to obtain funding, develop ani our planned clinical trials, the timing of and our ability to pan and maintain regulatory approvals for our therapeutic ca
Slide 3: miRagen Therapeutics Highlights
miRagen Therapeutics Highlights ¢ Aclinical stage biopharmaceutical company with programs in Oncology and Fibrosis ¢ MRG-106 in CTCL > miR-155 elevated lymphoma / leukemia ¢ MRG-201 cutaneous fibrosis & tissue repair > pathological fibrosis & connective tissue disorders * Expertise in nucleic acid d
Slide 4: Experienced Executive Leadership Team
Experienced Executive Leadership Team William S. Marshall, Ph.D. ThermoFisher )onaruacon ANIGEN ‘Adam Levy Chief Business Officer WEDBUSH {Swerittyncn Jefferies Jason A. Leverone, C.P.A. Chief Financial Officer Y Replidyne creat. Pseriunanoensen Paul Rubin, M.D. Executive Vice President, R&D XOM. 1A
Slide 5: microRNA Therapeutics - Regulate Systems Biology to Modify Disease
microRNA Therapeutics - Regulate Systems Biology to Modify Disease > The objective of microRNA-targeted therapy is to achieve disease modification by restoring system homeostasis. microRNAs regulate complex biological systems microRNA-targeted therapies are intrinsically focused on disease-relevant
Slide 6: Foothold Clinical Development Strategy
Foothold Clinical Development Strategy ¢ Biomarker driven early clinical trials * Progressive de-risking * May improve probability of success ¢ Accelerate proof of concept in humans + Initial rare disease indication may allow more rapid commercialization : mitRagen > g 6
Slide 7: Pipeline of Therapeutic Candidates
Pipeline of Therapeutic Candidates IND Pi dl ate | Disease Area Phase | Partner Enabling Internal Hematological MRG-106 - A a mtRagen Malignancies & miR-155 El NHL A MRG-201 Pathologic Fibrosis | [elem Wielp Nene Bag eo mtRagerr } < Other Fibrotic Indications miRagen MRG-110 Ischemia 7 ——
Slide 8: CTCL miR-155 Leukemia
CTCL miR-155 Leukemia Mycosis Fungoides High NHL CLL ALL [— a ee Le | € Nodular Diffuse oo -_ ~~“ MRG-106 ea (miR-155 inhibitor) a > & “4 Hematological Malignancies mitRagen g 8 ra g
Slide 9: Regulating Systems Biology to Modify Disease
Regulating Systems Biology to Modify Disease miR-155 is an OncomiR and a Pro-inflammatory microRNA T miR-155 ee Inflammation Leukemic M1-M2 transformation mtRagen . . é Inflammation / Immunity
Slide 10: MRG-106 Initial Indication: Mycosis Fungoides
MRG-106 Initial Indication: Mycosis Fungoides Mycosis Fungoides (MF) Most common form of CTCL United States MF prevalence of 16,000-20,000 cases Initially indolent but with serious quality of life detriment 5-year survival of approximately 90.6% in newly diagnosed CTCL patients Average age at onset
Slide 11: MRG-106: Two-Part Phase 1 CTCL Study
MRG-106: Two-Part Phase 1 CTCL Study PartA Intra-tumoral delivery of inhibitor of miR-155. 75 mg dose Pretreatment - 2. reveamer biopsy \ ae } ms Placebo J | MRG-108 biopsy 7) Part B Systemic SC or IV delivery to determine optimal potential dose. 300, 600 and 900 mg+ dose MRG-108_, /) Sub-cut Y) Obj
Slide 12: Exploratory Efficacy Measurements in Part A
Exploratory Efficacy Measurements in Part A: Intra-tumoral Injection CAILS Maximal % Hof jose Score Reduction in Patient Doses Schedule (Max/Min) — CAILS 100 © 107-001 3 7,4,2 18> 12 33% 80 102-001 5 -7,1,3,5,8 266 17% we so1001 6 7,1,3,5,8 1234 67% 80 F 105-001 4 1,358 168 50% . 102-003 4 1,3,5,8 1
Slide 13: Exploratory Efficacy Measurements in Part B
Exploratory Efficacy Measurements in Part B: Systemic Administration 20 ‘Change in mSWAT Score (%) 100 mtRagen g s oo 2 S$ > <a FESESEPSE PPE SEES E © aS [HM 300 mg sc HB 300 mg iv HB 600 mgsc E600 mg v HB 900 mg sc * treatment is on-going Note: Numbers in the bars are the number of doses administer
Slide 14: Potential Clinical Benefit of MRG-106 on Disease
Potential Clinical Benefit of MRG-106 on Disease Observed as Early as Study Day 19 112-001 107-003 27. 100 6. 100 3 n 80 & 5 g Bie 5 | fe = 5 é 15 a 0s o 3 & 3 g 3 S12 Ey 2, s — 40 3 zo. o Be p a g Ee 8 e 23 3 3 7m 1 3 & 2 1 7 43 19 25.31 37 43 49 55 61 67 10 20 30 40 50 60 70 80 90 100 Study Day St
Slide 15: Patient with Extensive Skin Disease Showed mSWAT Score Improvement
Patient with Extensive Skin Disease (Baseline mSWAT of 180) Showed mSWAT Score Improvement 102-007 an e < S80 Nba0 @ Ss 8 (aujaseg Jo %) LYMSu 20: Grey shading = drug administration 1 9 17 25 33 41 49 57 65 73 81 89 Study Day
Slide 16: Improvement in Total Skin Disease Score Correlates with MRG-106 Treatment
Improvement in Total Skin Disease Score Correlates with MRG-106 Treatment 102-005 Day 27 Day 57 be 100 CAILS: 8 CAILS: 5 18 wo & 0 2 16- > 8 14. a 212 og < 10: 2 3 : a 40 g 7 4 2 & 2. 3 Day 103 Day 131 Day 159 Day 186 $060 90 120 150 180 210 CAILS: 10 CAILS: 8 CAILS:7 _CAILS:6 Study Day a Grey shadi
Slide 17: Alterations to Gene Expression Pathways
Alterations to Gene Expression Pathways Consistent with Intended Mechanism of Action MRG-106 Saline Lesions Lesions Activated nei8 Groen Inactivated MRG-106 treatment believed to decrease CTCL associated disease miRagen: pathways including STAT and NFkB Pathways g 7
Slide 18: MRG-106 Potential Clinical Development Plan
MRG-106 Potential Clinical Development Plan Dose and Schedule Optimization in CTCL A A / mPoC] cPoC* Interim Analysis| DLBCL/ CLL Ph 2 in NHL/ Leukemia** Parallel Indication Expansion in Ph1 mtRager }
Slide 19: MRG-201 Pathological Fibrosis & Tissue Repair
MRG-201 (miR-29 replacement) Pathological Fibrosis & Tissue Répaid miRagen g
Slide 20: miR-29 is a Regulator of Biological Pathways Implicated in Fibrosis
miR-29 is a Regulator of Biological Pathways Implicated in Fibrosis mldated target in vivo Validat _in vivo Validated Targets _ is ss POSER PRGEC oT OTe IGFBPS, PDGFA, PDGFC . COL1A1, 1A2, 3A1, 5A1, 5A2, 5A3, TGF-B + Matrix ( cotagen vanscupiontansition | transcription/translation '6A4, 6AS, 6A6, 8A
Slide 21: MRG-201 First-In-Human Phase 1 Study in Induced Cutaneous Fibrosis
MRG-201 First-In-Human Phase 1 Study in Induced Cutaneous Fibrosis ¢ Normal healthy volunteers at a single trial site (Montreal) * 4 cohorts (n=3-10 per cohort): + A-—establish PD marker kinetics in skin incision / * B-single ascending dose in intact skin / * C-single ascending dose around skin inci
Slide 22: MRG-201 Mechanistic Proof of Concept: PD Biomarkers
MRG-201 Mechanistic Proof of Concept: PD Biomarkers Are Regulated in Human Incised Skin Log2 Fold Change Evidence of PD activity after single administration of MRG-201 PD bic that are up: in incised skin are down-regulated by MRG-201 PD biomarkers that are down-regulated in incised skin are up-regul
Slide 23: Blinded Histology Analysis Shows Statistically Significant Reduction of Fibroplasia
Blinded Histology Analysis Shows Statistically Significant Reduction of Fibroplasia with MRG-201 vs Saline Depth/Width (mm) or Area (mm?) mtRager g 44 Saline MRG-201 p=0.0464 Saline MRG-201 p=0.0078 Saline MRG-201 Width Depth Fibroplasia Area 23
Slide 24: Keloids — Cutaneous Pathologic Fibrosis
Keloids — Cutaneous Pathologic Fibrosis * Benign scar at the site of minor or major skin injuries (acne, trauma, surgery, burns) ¢ Results from an overgrowth of scar tissue + Excessive collagen | and III deposition * TGFB has been implicated in the pathogenesis ¢ Available treatments: steroids, radi
Slide 25: MRG-201 in IPF
29 in IPF miR mitRagen g
Slide 26: Nebulized MRG-201 Attenuates Fibrosis Induced by Bleomycin
Nebulized MRG-201 Attenuates Fibrosis Induced by Bleomycin in Preclinical Model 250 a 100 50 (0) Hydroxyproline yg/right lung a yu 3 * Control MRG-201 Saline Control MRG-201 Saline Saline Bleomycin — sp<0.05 MRG-201 or control dosing started 10 days after bleomycin administration 7 - administered da
Slide 27: MRG-201 Potential Clinical Development Plan
MRG-201 Potential Clinical Development Plan IND Keloids Ph 1 Healthy Vol. Ph 2 Keloids mPoC Interim Interim Analysis Analysis IND IND Ph 1 Hepatic ar muRager r
Slide 28: Upcoming Events and Milestones
Upcoming Events and Milestones Interim Phase 1 CTCL data Q Phase 1 trial expansion to include presentation at ASCO (Q2) 34 indication (H1) Hematological Phase 1 trial expansion to include Q Initiation of Phase 2 trial in CTCL / Malignancies 2" indication (H2) NHL (H2) (MRG-106) Interim Phase 1 CTCL
Slide 29: Intellectual Property Portfolio
Intellectual Property Portfolio * Owner / exclusive licensee of 113 issued patents over 100 pending applications * Composition of matter patents on all compounds - Exclusive licensee to LNA technology for multiple targets ° Freedom to operate with targeted 2 miRNAs based on current claims je and lik
Slide 30: miRagen Therapeutics
, miRagen Therapeutics miKager NASDAQ: MGEN Z \ June 2017
Miragen Therapeutics — Jefferies Global Healthcare Conference 2017 on 6ix