Corporate Presentation

Transcription

1 Corporate Presentation January 2019 Kadmon Holdings, Inc. 1

2 Disclaimers This presentation contains forward looking statements that are based on the beliefs and assumptions and on information currently available to management of Kadmon Holdings, Inc. (the Company ). All statements other than statements of historical fact contained in this presentation are forward-looking statements. Forward looking statements include information concerning the initiation, timing, progress and results of clinical trials of the Company s product candidates, the timing or likelihood of regulatory filings and approvals for any of its product candidates, and estimates regarding the Company s expenses, future revenues and future capital requirements. In some cases, you can identify forward-looking statements by terminology such as may, will, should, expects, plans, anticipates, believes, estimates, predicts, potential or continue or the negative of these terms or other comparable terminology. There are important factors that could cause the Company s actual results to differ materially from those expressed or implied by the forward-looking statements, including those factors discussed under the caption entitled Risk Factors in the Company s filings with the U.S. Securities and Exchange Commission ( SEC ), including the Company's Annual Report on Form 10-K for the fiscal year ended December 31, 2017 and Quarterly Report on Form 10-Q for the quarter ended September 30, 2018, filed pursuant to Section 13 of the Securities Exchange Act of 1934, as amended, with the SEC. Forward-looking statements involve known and unknown risks, uncertainties and other factors that may cause the Company s actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. Forward-looking statements represent the Company s beliefs and assumptions only as of the date of this presentation. Although the Company believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee future results, levels of activity, performance or achievements. Except as required by law, the Company assumes no obligation to publicly update any forward looking statements for any reason after the date of this presentation to conform any of the forward-looking statements to actual results or to changes in its expectations. 2

3 Kadmon Holdings, Inc. Fully Integrated Biopharmaceutical Company Late-stage biotech company headquartered in New York, NY Therapeutic focus areas: Autoimmunity Inflammation Research Fibrosis Immuno-oncology (I-O) Clinical Development Commercial Operation Two product candidates developed in-house expected to enter the clinic 2H 2019: KD045: ROCK inhibitor for fibrosis KD033: Anti-PD-L1/IL-15 fusion protein for I-O Most advanced candidate: KD025, a ROCK2 inhibitor FDA Breakthrough Therapy Designation and ongoing registration trial in cgvhd Ph2 trial in scleroderma initiating 1H 2019 Wholly-owned subsidiary Generic product candidate for Wilson s disease under FDA review Supports development and future commercialization of Kadmon s clinical pipeline 3

4 Kadmon 2019 Milestones Product Indication 2019 Milestone KD025 (ROCK2 inhibitor) Chronic Graft-Versus-Host Disease (cgvhd) Scleroderma (systemic sclerosis) Complete enrollment in ongoing pivotal trial (2H 2019) Initiate Phase 2 clinical trial (1H 2019) KD045 (pan-rock inhibitor) KD033 (anti-pd-l1/il-15 fusion protein) Fibrotic Diseases Investigational New Drug (IND) submission (2H 2019) Immuno-oncology IND submission (2H 2019) KD034 (generic trientine hydrochloride) Wilson s Disease Continue dialogue with FDA regarding regulatory approval 4

5 ROCK Inhibition and KD025 5

6 KD025: Most Advanced Product Candidate KD025, an Oral, Selective ROCK2 Inhibitor Rho-associated coiled-coil kinase (ROCK) is a serine/threonine kinase Mediates a wide range of functions, including cell movement, shape, differentiation and function 1 Two isoforms exist: ROCK1 and ROCK2 1 Kadmon research has helped define the role of ROCK in key areas, including: Autoimmune and inflammatory diseases Fibrotic diseases KD025 is Kadmon s oral, ROCK2-selective inhibitor More than 450 individuals have been treated with KD025 in ongoing and completed studies Well tolerated Demonstrated clinical activity at 200 mg KD025 has been granted FDA Breakthrough Therapy Designation and Orphan Drug Designation for the treatment of chronic graft-versus-host disease (cgvhd) 1 Small GTPases. 2014; 5: e

7 ROCK2 Plays Key Role in Autoimmune and Inflammatory Disease ROCK2 Inhibition Rebalances Immune Response to Treat Immune Dysfunction 1,2 ROCK2 inhibition downregulates pro-inflammatory Th17 responses and increases Treg function, helping to resolve immune dysregulation Reduces STAT3 phosphorylation and increases STAT5 phosphorylation ROCK2 inhibition re-establishes immune homeostasis ROCK2 Activation ROCK2 Inhibition STAT3 (Th17) Inflammation RORgt IRF4 STAT5 (Treg) STAT3 (Th17) STAT5 (Treg) Resolution Inflammation Resolution 1 Proc Natl Acad Sci, 2014; 2 Blood,

8 ROCK is an Intercellular Integrator of Pro-fibrotic Signals ROCK Regulates Multiple Pro-fibrotic Processes, Including Myofibroblast Activation ROCK is downstream of major pro-fibrotic mediators ROCK regulates fibroblast differentiation to myofibroblasts, a pathological cell type in fibrosis ROCK mediates stress fiber formation Matrix stiffness ROCK Myofibroblast Cell Stress fiber formation ROCK regulates transcription of pro-fibrotic genes MKL1 MKL1 MKL1 CTGF Am J Pathol Apr;185(4):

9 %CD4 + PD1Hi CXCR5+Foxp3- %CD19 + GL7+ FasHi %CD4 + PD1Hi CXCR5+Foxp3+ KD025 Rebalances ith17 cultures: Th17/Tregs %IL-17+ and Down-regulates ith17 cultures: %FOXP3+CD25+ B Cells p = ** p = * p = *** KD025 Reduces Pro-Inflammatory p = * Th17 Cells (IL-17) and Increases p = Regulatory ** T Cells (Tregs) p = *** p = *** 6 IL-17 p (Th17) < **** 60 Foxp3+ (Tregs) p = * % IL % FOXP3+CD Research conducted by Jeff Bluestone, Ph.D., UCSF 0 Control DMSO KD025 KD mm 1 mm Control DMSO Control Control DMSO KD025 KD mm 1 mm KD025 Decreases Tfh and B Cells and Up-regulates Tregs, Helping to Resolve Inflammation Tfh cells B cells Control DMSO Control Treg cells Flynn, Blazar et al, Blood 2016 BM BM + T cells (cgvhd) KD025 BM BM + T cells (cgvhd) KD025 BM BM + T cells (cgvhd) KD025 9

10 KD025 Reduced Lung Fibrosis in Bleomycin Model KD025 Treatment Administered on Day 8, When Fibrosis is Already Established Intratracheal Bleomycin KD025 treatment administered (orally, 50, 100 or 150 mg/kg QD) Tissue Harvest Day: Normal Lung Pre-Treatment Lung Intratracheal Bleomycin Day 0 Vehicle or KD025 Day 8 Treatment: Day 21 Control KD mg/kg QD KD mg/kg QD KD mg/kg QD 10

11 KD025 in cgvhd 11

12 Pathophysiology of Chronic GVHD (cgvhd) cgvhd is Driven by Immune Cells and Pro-inflammatory Cytokines cgvhd involves both T cells and B cells Overproduction of pro-inflammatory cytokines IL-21 and IL-17 Over-activation of T follicular helper (Tfh) cells and B cells, leading to over-production of antibodies Deficiency of regulatory T (Treg) cells, leading to a lack of appropriate regulation of immune response Blood,

13 cgvhd: Major Complication Post-Allogeneic HCT cgvhd: Leading Cause of Non-Relapse Mortality in Transplant Survivors Acute GVHD GVHD Classification Transplant GVHD Chronic GVHD Key cgvhd Risk Factors Prior history of acute GVHD Transplantation from an HLAmismatched related donor or from and HLA-matched unrelated donor Classic Acute Typically 100 days post-hct Late-onset Acute Typically >100 days post-hct Overlap Syndrome Presence of agvhd features Classic Chronic Typically >100 days post-hct Sex mismatched between recipient and donor Transplanted immune cells (graft) attack healthy cells (host), leading to inflammation and fibrosis in multiple organs Adverse effects include physical, functional, and psychosocial deficits, inability to return to work, and poor quality of life Older age of donor and recipient Use of a non-t-cell-depleted graft Sources: cgvhd KoL and Treater Research (N=15); Omer et al (2016); Lee (2005) 13

14 cgvhd: ~14K Patients with Significant Unmet Need Estimated 14K Patients and Growing, with ~50% Progressing Through 3+ Lines of Therapy cgvhd prevalence: ~14,000 (U.S., 2017) CAGR of 4% cgvhd incidence: ~5,200 (U.S., 2017) Growth consistent with that of HSCT transplants cgvhd patients are living longer, with nonrelapse mortality decreasing year over year One-year survival post cgvhd: 71% Four-year survival post cgvhd: 45% Unmet need remains, as 75% of patients progress to 2 lines of therapy, and ~50% progress to 3+ lines In first line, primary goal is to cure disease In subsequent lines, goals are to stabilize disease and limit adverse events while maintaining quality of life Patients cycle through lines, each line lasting ~3-4 months, reinforcing need for effective, well-tolerated therapy 1 Sources: June 2018 IQVIA Pharmetrics commercial medical claims; CMS Medicare Fee-for-Service medical claims (5% Non-Institutional Sample); Trinity KoL and Treater Interviews N=15 interviews; N=93 cgvhd Patient Charts; Chart study focused on patients with 3+ lines of treatment for cgvhd; Arai (2015) KD025 expected tx duration of >1 year, based on clinical efficacy and safety data as well as KOL feedback 14

15 Phase 2a Trial of KD025 in cgvhd: Design and Key Endpoints Key Eligibility Criteria: Adults who have had allogeneic HCT with steroid-dependent or steroid-refractory cgvhd Have persistent active cgvhd after at least 2 months of steroid therapy Receiving glucocorticoid therapy +/- calcineurin inhibitor therapy for cgvhd No more than 3 prior lines of treatment for cgvhd KD : Phase 2a Study of KD025 for Patients with cgvhd Oral Presentation at ASH 2018 Cohort 1: 200mg QD (n=17) Cohort 2: 200mg BID (n=16) Cohort 3: 400mg QD (n=21) Three cohorts enrolled sequentially, following safety assessment of previous cohort Key Endpoints: ORR, per 2014 NIH criteria Safety and tolerability of KD025 in patients with cgvhd Duration of response (DOR) Response by organ system Changes in corticosteroid and calcineurin inhibitor dose All data as of 13 September,

16 KD : Baseline Characteristics 48% of all patients had 4 organs affected Included both inflammatory and fibrotic manifestations 67% of all patients had received 2 prior lines of cgvhd therapy Baseline Characteristics Cohort 1 (n=17) Cohort 2 (n=16) Cohort 3 (n=21) Median age (years (range)) 50 (20-63) 55 (30-75) 46 (25-75) Male/Female (%/%) 76/24 56/44 57/43 Median time cgvhd diagnosis to study (months) Organ Involvement 4 organs involved 8 (47) 10 (63) 8 (38) Eyes 14 (82) 11 (69) 17 (81) Skin 13 (76) 12 (75) 15 (71) Mouth 13 (76) 11 (69) 11 (52) Joints and fascia 12 (71) 11 (69) 10 (48) Lungs 3 (18) 3 (19) 11 (52) Upper GI 2 (12) 4 (25) 2 (10) Esophagus 2 (12) 0 (0) 5 (24) Lower GI 1 (6) 2 (13) 1 (5) Liver 0 (0) 2 (13) 0 (0) Severe cgvhd 1 12 (71) 14 (88) 16 (76) Prior Therapies 2 Cohort 1 (n=17) Cohort 2 (n=16) Cohort 3 (n=21) Median prednisone dose at BL (mg/kg/day) Prior Lines of Therapy Median prior lines of therapy (n (%)) 15(88) 9(56) 12(57) Prior Therapies Corticosteroids 17(100) 16(100) 21(100) Sirolimus 10(59) 9(56) 7(33) Tacrolimus 8(47) 6(38) 11(52) Rituximab 8(47) 3(19) 5(24) MMF 4(24) 4(25) 4(19) Cyclosporin 3(18) 0(0) 2(10) Methotrexate 1(6) 2(13) 0(0) Ibrutinib 1(6) 0(0) 2(10) Ixazomib 1(6) 1(6) 0(0) Others for 1 patient each: Anti Thymocyte Globulin, Imatinib, Ofatumumab, Ruxolitinib, Ibrutinib/Placebo 1 Defined as at least 1 organ with NIH Activity Assessment score of 3, or lung score of 2 or 3, at baseline 2 ECP was not counted as a prior systemic therapy 16

17 KD : Safety and Tolerability No treatment-related SAEs No apparent increased risk of infection AEs were overall consistent with those expected in cgvhd patients receiving corticosteroids Safety Overview, n (%) Cohort 1 (n=17) Cohort 2 (n=16) Cohort 3 (n=21) ITT (n=54) Median weeks of treatment Any Adverse Event (AE) 16 (94) 16 (100) 19 (90) 51 (94) Grade 3 / 4 AE 10 (59) 10 (63) 9 (43) 29 (54) SAE 5 (29) 6 (38) 9 (43) 20 (37) Drug related AE Any related AE 6 (35) 9 (56) 9 (43) 24 (44) Related AE leading to discontinuation 2 (12) (4) Related Grade 3 / 4 event 2 (12) 4 (25) 2 (10) 8 (15) Related SAE On Study Deaths (14) 2 3 (6) 1 Headache; Diarrhea 2 Relapse of Leukemia; Lung infection; Cardiac arrest. All considered not related to KD025 Commonly Reported AEs, n (%) All Grade, in 15% Cohort 1 (n=17) Cohort 2 (n=16) Cohort 3 (n=21) ITT (n=54) Upper respiratory tract infection 5 (29) 6 (38) 3 (14) 14 (26) ALT / AST increased 6 (35) 4 (25) 3 (14) 13 (24) Fatigue 5 (29) 3 (19) 5 (24) 13 (24) Nausea 6 (35) 2 (13) 5 (24) 13 (24) Diarrhea 6 (35) 2 (13) 4 (19) 12 (22) Anemia 5 (29) 4 (25) 1 (5) 10 (19) GGT increased 4 (24) 5 (31) 1 (5) 10 (19) Cough 1 (6) 3 (19) 5 (24) 9 (17) Dyspnea 2 (12) 5 (31) 2 (10) 9 (17) Hypertension 5 (29) 2 (13) 2 (10) 9 (17) Hyperuricemia 3 (18) 1 (6) 5 (24) 9 (17) Grade 3 / 4, in 5% GGT increased 3 (18) 3 (19) 0 6 (11) Hyperglycemia 2 (12) 0 2 (12) 4 (7) Anemia 2 (12) 1 (6) 0 3 (6) Dyspnea 1 (6) 1 (6) 1 (5) 3 (6) Infection, All Grade, in 5% Upper respiratory tract infection 5 ( 29) 6 ( 38) 3 ( 14) 14 (26) Pneumonia 2 ( 12) 2 ( 13) 0 4 (7) Influenza 2 ( 12) 1 ( 6) 0 3 (6) Oral candidiasis 2 ( 12) 1 ( 6) 0 3 (6) Sinusitis 1 ( 6) 1 ( 6) 1 ( 5) 3 (6) 17

18 Overall Response Rate KD : Overall Response Rate (ORR) ORR (ITT Population) Responses Achieved Across Key Patient Subgroups ORR Cohort 1 (n=17) 65% (11/17) Cohort 2 (n=16) 63% (10/16) Cohort 3 (n=21) 52% 1 (11/21) 80% 70% 60% 59% 58% 62% 55% 50% 95% CI (41, 83) (39, 82) (32, 72) 40% 1 Three patients in Cohort 3 did not reach first response assessment. On a response-evaluable basis, Cohort 3 ORR = 61% (11/18) 30% 20% 10% 0% ITT 2 Prior prior lines of of therapy 4 Organs organs Involved involved Severe cgvhd (n=54) (n=36) (n=26) (n=42) 18

19 KD : Duration of Response (DOR) Responses Achieved with KD025 Are Durable Responses were rapid: 75% of responders have achieved a response at the first assessment (8 weeks) Amongst responders: Kaplan-Meier median DOR of 28 weeks in ITT responder population Responders with sustained responses for 20 weeks: Cohort 1: 9/11 (82%) Cohort 2: 5/10 (50%) Cohort 3: 4/11 (36%) Durability data continue to mature Duration of Response ITT Responders (n=32) Weeks Number at risk

20 KD : Corticosteroid and Tacrolimus Dose Reductions 7 Patients Have Completely Discontinued Steroids Cohort 1 (%) Cohort 2 (%) Cohort 3 (%) % reduction in median corticosteroid dose 44% 23% 10% Patients with corticosteroid dose reduction 13/17 (76) 9/16 (56) 15/21 (71) Responders with corticosteroid dose reduction 8/11 (73) 5/10 (50) 10/11 (91) Non-Responders with corticosteroid dose reduction 5/6 (83) 4/6 (67) 5/10 (50) Corticosteroid discontinuations 4/17 (24) 1/16 (6) 2/21 (10) Patients with tacrolimus dose reductions 4/6 (67) 5/6 (83) 5/11 (45) 20

21 KD : Pharmacodynamics With KD025 treatment: KD025 May Modulate Immune Homeostasis by Restoring the TH17/Treg Balance Treg cells increased TH17 decreased Consistent with KD025 mechanism of action 21

22 KD : Conclusions KD025 Achieved Clinically Meaningful Responses Exploratory Pharmacodynamic Data Support KD025 Mechanism of Action KD025 was well tolerated: No treatment-related SAEs No apparent increased risk of infection Patients achieved NIH-defined clinical responses with KD025 ORRs of 65%, 63% and 52% in Cohorts 1, 2 and 3 respectively CRs in multiple organs, including in organs with fibrotic disease Responses are clinically meaningful: Durable Patients were able to reduce or discontinue corticosteroids and other immunosuppressants Patients experienced clinically meaningful improvement in symptoms (LSS score) Exploratory PD data show a decrease in TH17 and an increase in Treg cells during treatment with KD025 22

23 KD : Ongoing Open-Label Registration Trial in cgvhd Registration trial in adults who have had allogeneic HCT with active cgvhd Objective: Demonstrate clinically meaningful responses with KD025 in cgvhd patients Primary endpoint: ORR (95% CI to exclude <30%) Open-label, two-arm trial with two doses of KD025 (200 mg QD and 200 mg BID) Either dose may be considered for registration Enrollment expected to complete in 2H 2019 Adults with 2 prior lines of systemic therapy for cgvhd R KD mg QD (n=63) KD mg BID (n=63) Treat to progression Primary Endpoint: ORR Key Secondary Endpoint: Duration of response 23

24 KD025: Additional Clinical Development Scleroderma: Chronic multi-system disease characterized by skin thickening and organ fibrosis Positive clinical data of KD025 in cgvhd and IPF support investigation of KD025 in scleroderma Affects 75,000 to 100,000 people in the United States 1 Unmet need: No FDA-approved targeted therapies Phase 2 trial of KD025 in scleroderma initiating 1H American College of Rheumatology, KD025: Planned Phase 2 Clinical Trial in Systemic Sclerosis (Scleroderma) KD025: Demonstrated Proof of Concept in Idiopathic Pulmonary Fibrosis (IPF) KD025 demonstrated clinical activity and tolerability in ongoing Ph2 proof-of-concept clinical trial Trial expanded to include an additional 40 patients, increasing total enrollment to ~81 patients Data to support the development of Kadmon s novel ROCK inhibitors for fibrotic diseases Lead candidate from this platform: KD045 IND anticipated 2H

25 KD045: ROCK Inhibitor for Fibrosis 25

26 KD045: ROCK Inhibitor Candidate for Fibrosis Kadmon is Developing Next-Generation ROCK Inhibitors for Fibrosis Kadmon research has validated the role of ROCK signaling in fibrosis KD045 Kadmon has identified and developed proprietary next-generation ROCK inhibitors for fibrotic diseases G-loop Developing candidates with enhanced potency and AGC-kinase selectivity hinge KD045: Lead candidate selected for clinical development Inhibited key fibrotic processes in multiple in vivo pharmacology models, including in models of bleomycin-induced lung fibrosis, renal fibrosis and liver fibrosis Selectively targets ROCK, exhibiting favorable safety profile Entering the clinic 2H 2019 Activation loop 26

27 KD045: Significant Activity in STAM NASH Model Pathological process of STAM TM mice Histology H & E Staining (x200) Naive 3 mg/kg - BID KD045 Drug Treatment Groups: KD045 3 mg/kg BID KD045 6 mg/kg BID KD mg/kg QD Telmisartan 10 mg/kg QD Drug Administration: Week 6-9 Vehicle Telmisartan 6 mg/kg - BID 10 mg/kg - QD N A F L D a c t i v i t y s c o r e Mean ± SEM s i r i u s r e d - p o s i t i v e a r e a ( % ) N A F L D a c t i v i t y s c o r e, m e a n S E M NAFLD Activity score D u n n e t t 's m u l t i p le c o m p a r is o n s v s v e h ic le Normal Vh 3 6 BID BID N o r m a l Fibrosis Area V e h i c l e K D 0 4 5, 3 m g / k g B I D K D 0 4 5, 1 0 m g / k g Q D K D 0 4 5, 6 m g / m g B I D *** **** ** * * * * * * * * * 10 QD Telm T e l m i s a r t a n, 1 0 m g / k g Q D 1. 5 Dunnett's multiple comparisons test Significant? * * Summary * * * Adjusted * * P Value Vehicle vs. Normal Yes **** Vehicle vs. KD045, mg/kg BID No ns Vehicle vs. KD045, 6mg/mg BID Yes ** Vehicle vs. KD045, 10mg/kg QD Yes *** Vehicle vs. Telmisartan, 10mg/kg QD Yes **** N o r m a l F i b r o s i s a r e a, m e a n S E M D u n n e t t 's m u l t i p le c o m p a r is o n s v s v e h ic le V e h i c l e K D 0 4 5, 3 m g / k g B I D ** Normal Vh 3 6 BID BID K D 0 4 5, 1 0 m g / k g Q D K D 0 4 5, 6 m g / m g B I D T e l m i s a r t a n, 1 0 m g / k g Q D ** p < 0.01 *** p < **** p < vs Vehicle Dunnett s multiple comparison test Dunnett's multiple comparisons test Significant? Summary Adjusted P Value ** 10 QD *** Telm * Plasma ALT and liver triglyceride levels also trended toward improvement in this model 27

28 Biologics Platform 28

29 Kadmon is Developing Next-Generation I-O Therapies Although anti-pd-l1/pd-1 therapies have been game changing, there is room to improve I-O therapies 60% of patients are refractory to or relapse following approved anti-pd-l1/pd-1 therapies 1,2 Limited durability of response for anti-pd-l1/pd-1 therapies (1-2 years) 1,2 Current industry focus: Administer two I-Os or I-O/chemo therapies at approved therapeutic dosages to enhance durability of response Potential additional safety concerns (narrower therapeutic window) Kadmon s approach: Develop IL-15-containing fusion proteins to target I-O non-responders and enhance durability of response Localized IL-15 stimulation of NK, NKT and CD8 + memory T cells should overcome resistance, promote efficacy and induce long-lasting responses compared to existing combination therapies Targeted IL-15 activity to tumor microenvironment to mitigate safety concerns Lead candidate: KD033, an anti-pd-l1/il-15 fusion protein, entering the clinic 2H Keytruda (pembrolizumab) [package insert]. Merck & Co, Inc., Opdivo (nivolumab) [package insert]. Bristol-Myers Squibb Company,

30 Percent Survival Lead I-O Candidate KD033 Entering the Clinic 2H 2019 KD033 (anti-pd-l1/il-15 Fusion Protein) Targets IL-15 Activity to Tumor Microenvironment Demonstrated efficacy in multiple syngeneic mouse models Significantly inhibited tumor growth in PD-L1-expressing murine models resistant to approved I-O therapies Induced immune system memory KD033 surrogate-treated MC38 colon adenocarcinoma mouse model survived tumor rechallenges Vehicle D7A8 10 mg/kg IP QWx4 1 KD033 surrogate 3.3 mg/kg IP QWx4 2 n = 12 per group 100% tumor free post rechallenge Dosing once a week *** Day 77 Day 142 tumor ~80 mm 3 Rechallenge Days Post Inoculation Rechallenge 1 D7A8: Anti-PD-L1 antibody in KD033 surrogate, without IL-15 2 KD033 surrogate recognizes human, NHP, rat and mouse PD-L1 30

31 Commercial Operation 31

32 Kadmon Pharmaceuticals: Commercial Operation Specialty-Focused Commercial Operation Kadmon Pharmaceuticals is a wholly-owned subsidiary focused on specialty pharmaceuticals Experience in specialty pharmacy distribution channel Currently markets and distributes products for a variety of indications Supports development and future commercialization of Kadmon s clinical product candidates Capabilities include logistics, regulatory, quality assurance, compliance and pharmacovigilance Kadmon commercial infrastructure based in Warrendale, PA Kadmon will leverage its commercial organization to market its products, if approved 32

33 KD034 for Wilson s Disease KD034: Generic Product Candidate for Wilson s Disease Wilson s Disease: Genetic disorder characterized by excess copper accumulation 9,000 in the United States are affected by WD 1 KD034: generic formulation of trientine hydrochloride for WD Two ANDAs submitted to the FDA for KD034 (bottle and blister pack) Blister pack offers room temperature stability 2 Kadmon is in dialogue with the FDA regarding regulatory approval for KD034 Kadmon will leverage its commercial operation to market KD034, if approved 1 National Organization for Rare Disorders, 2018; 2 For up to 30 days 33

34 Financial Profile 34

35 Financial Profile KDMN Financial Summary Cash and cash equivalents of $113.4 million as of September 30, % ownership 1 of MeiraGTx (Nasdaq: MGTX), a clinical-stage gene therapy company 113,130,817 common shares outstanding as of September 30, 2018 ATM offering initiated December 2017; gross proceeds received in January 2019: ~$30mm Trades on the NYSE under the ticker symbol KDMN 1 As of September 30,