Jefferies 2015 Healthcare Conference

Harlan W. Waksal, M.D.

President and CEO

March 27, 2015

June 2, 2015

Forward-Looking Statement

This presentation contains forward-looking statements. These forward-looking statements are based

on management’s expectations and are subject to certain factors, risks and uncertainties that may

cause actual results, outcome of events, timing and performance to differ materially from those

expressed or implied by such statements. The information contained in this presentation is believed to

be current as of the date of original issue. Kadmon expressly disclaims any obligation or undertaking

to release publicly any updates or revisions to any forward-looking statements contained herein to

reflect any change in our expectations with regard thereto or any change in events, conditions or

circumstances on which any such statements are based.

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Company Snapshot

Developing innovative medicines for serious unmet medical needs

Privately held, New York-based biopharmaceutical company

– Founded in 2009; approximately 170 employees

Specialty pharmacy-focused commercial operation

– Profitable commercial platform with near-term 505(b)(2) candidates

Value-generating technology and drug discovery platforms

– Gene therapy subsidiary

– Biologics license agreements (monoclonal antibodies)

Diverse portfolio of novel drug candidates

– Four drugs currently in Phase 2 or higher clinical development

Experienced management team

– Successful history in biotechnology and drug development

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Robust Pipeline with Multiple Shots on Goal

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Clinical Stage Compounds Indication Preclinical Phase 1 Phase 2 Status

Autoimmune

and Fibrotic

Diseases

KD025

Psoriasis Ph 2 ongoing;

Data expected Q3 2015

Systemic Lupus Erythematosus Ph 2 planned Q1 2016

Chronic Graft-Versus-Host Disease Ph 2 planned Q1 2016

Scleroderma Ph 2 planned Q1 2016

Idiopathic Pulmonary Fibrosis Ph 2 planned Q1 2016

Oncology KD019

HER2+ Breast Cancer with Brain

Metastases

Ph 1b ongoing;

Ph 2a planned Q4 2015

NSCLCPh 2 study planned

Q4 2015

GlioblastomaClinical study planned

Q4 2015

Monogenic

Diseases

KD019

Autosomal Dominant Polycystic Kidney

Disease (ADPKD)

Ph 2a ongoing; Ph 2/3 reg. study

planned Q1 2016

Autosomal Recessive Polycystic

Kidney Disease (ARPKD) Phase 1b/2a planned Q1 2016

KD034 Wilson’s DiseaseBioequivalence registration

study

505(b)(2) approval anticipated

Q1 2017

Metabolic

DiseaseKD026 Type 2 Diabetes

First Ph 2 study completed;

dose-ranging Ph 2 study

initiated Q2 2015

Multi-Targeted Tyrosine Kinase Inhibitor (KD019)

Oncology

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KD019: A Multi-Target Tyrosine Kinase Inhibitor

Unique inhibitor of specific tyrosine kinases that are established therapeutic targets

– EGFR: molecular target in cancer

– HER2: molecular target in cancer

– VEGFR2/3: molecular vascular target in cancer

– Src Kinase Family: molecular target in cancer (resistance and metastases)

KD019 Characteristics

– Orally available, reversible and highly potent small molecule kinase inhibitor

– Blood brain barrier penetrant

Opportunity to treat brain metastases and brain tumors

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KD019: Demonstrated Clinical Activity in Non-Small Cell Lung Cancer (NSCLC)

55 patients with treatment-naïve NSCLC (adenocarcinoma)

Dosed daily 300 mg or intermittent 350 mg

ITT population – 26% ORR (major responses: > 50% tumor reduction)

EGFR mutations – 57% ORR; PFS 9.3 months; OS 22.5 months

Phase 2 Study – NSCLC

More than 200 patients treated

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Four Phase 1 clinical trials with 104 patients

Two open-label Phase 2 clinical trials with 96 patients

Well tolerated with clear evidence of clinical activity

KD019: Clinical Plan, Future Clinical Considerations

Planned Clinical Studies, Planned Clinical Indications

KD019 and trastuzumab in subjects with HER2+ metastatic breast cancer with or without brain

metastases

KD019 dose escalation cohorts; MTD to be determined (no dose-limiting toxicities to date)

– Phase 1b data found KD019 + trastuzumab combination to be well tolerated; study ongoing

Ongoing Clinical Study

HER2+ metastatic breast cancer with brain metastases: Phase 2a study planned Q4 2015

NSCLC: Phase 2 study planned Q4 2015 (leptomeningeal disease)

Glioblastoma: Clinical study planned Q4 2015

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Polycystic Kidney Disease (PKD)

Monogenic Diseases

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KD019: Polycystic Kidney Disease Pathophysiology

Most prevalent monogenic disease

– 600,000 PKD patients in U.S.

– Autosomal dominant (AD) presents in adulthood

– Autosomal recessive (AR) form affects newborns

– No approved therapies in U.S. or EU

PKD is driven by EGFR and Src

– Results in cyst formation and renal tissue disruption

KD019 is ideal potential treatment for PKD

– Down-regulates both EGFR and Src

KD019 is uniquely positioned to be a first-in-class drug to treat both ADPKD and ARPKD

Normal Vs. PKD Human Kidney

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KD019: Effective in Rat and Mouse Models of ARPKD

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Increasing Doses of KD019 Reduces Kidney Volume and Number and Size of Cysts

Kidney Histology: Rat Model

KD019: 7.5 mg/kg/dayTreatment: Vehicle KD019: 15 mg/kg/day

PCK Rat Model BPK Mouse Model

Normal Untreated

Diseased

Diseased

KD019

7.5 mg/kg/day

Diseased

KD019

15 mg/kg/day

PCK

Vehicle

PCK

7.5 mg/kg/dayPCK

15 mg/kg/dayKD019

7.5 mg/kg/day

KD019

15 mg/kg/day

KD019: Development Status in PKD

Autosomal Dominant (AD) PKD

– Phase 1b completed; Phase 2a fully enrolled (24 patients)

– KD019 appears safe, well-tolerated and optimized at 50 mg QD

– Potential ADPKD registration study: randomized Phase 2/3 study initiating Q1 2016

Autosomal Recessive (AR) PKD

– KD019 reformulated for liquid dosing

– Developmental toxicology ongoing

– Phase 1b/2a trial initiating Q1 2016

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KD026 in Type 2 Diabetes

Metabolic Disease

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KD026: A Targeted Inhibitor of Lipid Transport

A unique, selective inhibitor of microsomal triglyceride transfer protein (MTP) targeted to cells of

the intestine

Essentially no systemic distribution in humans, minimizing side effects

– Less than 1% absorbed into system

Potential clinical targets include metabolic disease: diabetes, dyslipidemia, obesity

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Kadmon is developing KD026, an enterically targeted MTP inhibitor

First-generation MTP

inhibitors were designed to

target MTP in the gut and liver

(multiple safety issues)

KD026 is an MTP inhibitor

designed to only target

enterocytic MTP

(no systemic toxicity)

Adverse

Effect on

Liver

Liver

Functions

Normally

KD026: Phase 2 Clinical Trial in Type 2 Diabetes

82 patients

Inclusion criteria:

HbA1c 7%-11% Placebo & metformin

100 mg KD026 BID & metformin

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KD026: Clinically Significant Reduction in HbA1c

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Patients with HbA1c > 7.0

Mean Baseline HbA1c = 8.6%

Overall treatment effect; ANOVA; p = 0.02

* p < 0.05 to corresponding placebo visit

Overall treatment effect; ANOVA; p = 0.03

* p < 0.05 to corresponding placebo visit

** p < 0.01 repeated measures to Visit 2 baseline

Patients with HbA1c > 8.0

Mean Baseline HbA1c = 9.4%

Per Protocol ITT Population Segmented Population (subgroup analysis)

KD026 in Diabetes: Phase 2 Clinical Summary

Phase 2 Clinical Summary

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Well tolerated; minimal adverse events (diarrhea, grades 1-2, self-limiting)

Statistically significant decrease in HbA1c

– 0.8% from baseline (0.6% placebo adjusted)

Additional benefits:

– Lowers plasma glucose

– Lowers LDL-C

– Lowers post-prandial lipids

– Weight loss

Second Phase 2 Clinical Study (Ongoing)

Placebo-controlled, dose-ranging three-month study in Type 2 diabetes

Approximately 120 patients, HbA1C ≥ 7

Initiated Q2 2015

Broad Program Developing

Selective Rho-Associated Protein Kinase 2 (ROCK2) Inhibitors

Autoimmune, Fibrotic and Neurological Diseases

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Most Advanced ROCK2 Inhibitor: KD025

Kadmon’s Target: Rho-associated Protein Kinase 2 (ROCK2)

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What is ROCK2?

– ROCK2 is a multi-function intracellular

kinase

– ROCK2 is up-regulated in multiple

diseases including autoimmune, fibrotic

and neurologic diseases

Kadmon has generated a large portfolio of

selective ROCK2 inhibitors, including blood-

brain barrier penetrant molecules

Lead compound: KD025

– Phase 1 safety data complete; no dose-

limiting toxicities up to 1,000 mg QD

– Phase 2 study underway in psoriasis

(including dose optimization)

Kadmon has crystalized and solved the structure of the

human ROCK2 kinase domain dimer bound to KD025

and is using this information to generate the next

generation of ROCK inhibitors

Immune system distinguishes foreign invaders

and prevents anti-self responses

Autoimmunity is caused by imbalance of Th17

activity and Tregs, leading to uncontrolled

inflammation

KD025: ROCK2 Inhibition in Autoimmunity

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Autoimmune Disease

STAT3(Th17)

STAT5 (Treg)INFLAMMATION

RESOLUTION

ROCK2 inhibition reestablishes

immunologic homeostasis:

– Reduces Th17 activity, decreasing pro-inflammatory cytokines IL-17, IL-21, IL-22

– Increases number and function of Tregs

ROCK2 Inhibition (KD025 Treatment)

INFLAMMATION RESOLUTION

Kadmon has defined a crucial role for ROCK2: affects key transcription factors that control

immune system

STAT5 (Treg)

STAT3(Th17)

KD025 Selectively Inhibits Th17 Cell Response in Human Peripheral Blood

Lymphocytes in vitro (healthy human volunteers)

Th17 Response

General T Cell Responses

IL-17

0

0.2

0.4

0.6

0.8

0 1.2 2.5 5 10

KD025 (mM)n

g/m

l

0

0.1

0.2

0.3

0.4

0.5

0 1.2 2.5 5 10

KD025 (mM)

ng

/ml

0

1

2

3

4

5

6

0 1.2 2.5 5 10

ng

/ml

KD025 (mM)

0

1

2

3

0 1.2 2.5 5 10

KD025 (mM)

ng

/ml

0

1

2

3

4

5

6

7

0 1.2 2.5 5 10

KD025 (mM)

ng

/ml

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IL-2 IFN-g IL-10

IL-22IL-21

* 500 mg correlates to mM – 10 mM

*

0

1

2

3

4

5

0 1.2 2.5 5 10

KD025 (mM)

ng

/ml

KD025 Phase 2a Trial in Autoimmune Disease: Psoriasis

Screening (Day -28 to -1)

On-Study KD025 200 mg QD x 28 days30 Day

Follow-up

Seven of eight patients evaluable

– Endpoints: T cell cytokine secretion (IL-17), PK, tissue biopsy markers, PGA and PASI

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Outcome

Design

Open-label, single-arm, Phase 2a study

Eight subjects (18-65 years of age) with moderately severe psoriasis vulgaris

Failed at least one line of systemic therapy

PASI of ≥ 12; no requirement around PGA score

KD025: Specific Inhibition of Th17 Cytokines in Cells from Evaluable Patients

Th17 Response

-100

-80

-60

-40

-20

0

% c

ha

nge

-100

-80

-60

-40

-20

0

% c

ha

nge

-100

-80

-60

-40

-20

0

20

% c

ha

nge

-100

-80

-60

-40

-20

0

20

% c

ha

nge

-100

-80

-60

-40

-20

0

20

% c

ha

nge

-100

-50

0

50

100

% c

ha

nge

IL-17 IL-21 IL-22

IL-2 IFN-Ɣ IL-10

General T Cell Response

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KD025 in Psoriasis: Phase 2a Conclusion, Phase 2 Ongoing

Phase 2a Study Conclusion

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KD025 at 200 mg QD for one month demonstrated encouraging pharmacodynamic activity

– All evaluable patients showed specific decrease in Th17 activity

– Correlation between PASI and PK

– Tissue biopsies indicate biological response

Reduction of PASI in 3 of 8 patients (up to 66%) after only one month of treatment

Dose-escalating three-month study in moderate to severe psoriasis vulgaris ongoing; data expected Q3 2015

Additional planned autoimmune clinical studies:

– Systemic lupus erythematosus

– Chronic graft-versus-host disease (cGVHD)

– Scleroderma

Phase 2 Study, Additional Planned Autoimmune Studies

ROCK2 in Fibrotic Disease

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Fibrosis and resultant organ failure account for more than one-third of deaths worldwide1

Fibrosis is a consequence of aberrant wound-healing

– Prolonged activation of myofibroblasts can result in permanent scarring, organ malfunctionanddeath

ROCK2 is key common pathway and therapeutic target for pathologic fibrosis

– ROCK2 inhibition reduces collagen deposition (Type 1 collagen) and stellate cell formation, improving organ function

ROCK Signaling Plays Key Role in Fibrosis

Based on image from

New England Journal of Medicine

1 Zeisberg M, Kalluri R. Cellular mechanisms of tissue fibrosis: 1. Common and organ-specific

mechanisms associated with tissue fibrosis. Am J Physiol Cell Physiol 2013; 304: C216-C225.

KD025 Attenuates Pulmonary Fibrosis in Bleomycin Induced Mouse Model

0 8 21

KD025 treatment

administered when fibrosis

is already established

(orally, 50, 100 or 150 mg QD)

Day:

Tissue Harvest

Intratracheal

Bleomycin

Intratracheal

Bleomycin

Day 0

Vehicle or

KD025

Day 8

Normal Lung Pre-Treatment Lung

Treatment – Day 21

Control

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KD025 in Idiopathic Pulmonary Fibrosis, Future Indications

Phase 2 study in idiopathic pulmonary fibrosis

– Dose selection dependent on PK results from psoriasis study

– 24 patients

Results in bleomycin lung disease model suggest that clinical benefit is likely in IPF

Endpoint: Forced vital capacity (FVC)

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Planned Clinical Study in Idiopathic Pulmonary Fibrosis – Q1 2016

Future Clinical Indications in Fibrosis

Liver fibrosis

Kidney fibrosis

Wilson’s Disease

Monogenic Diseases

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KD034: Reformulated Trientine for Wilson’s Disease

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Kadmon already controls large portion of Wilson’s Disease market

Orphan genetic liver disease

– Causes impairment in liver’s ability to excrete copper

– Excess copper leads to hepatic, neurologic, psychiatric and ophthalmologic abnormalities

– 10,000 with Wilson’s Disease in the United States

Requires lifelong treatment

– Disease progression can be halted and disability reversed

– Therapies chelate excessive copper, which is then excreted in urine

Kadmon co-promotes Syprine® (trientine) for Wilson’s Disease with Valeant Pharmaceuticals

– Current Syprine U.S. market is approximately $100 million

– Provides Kadmon with entry into orphan liver disease market, leveraging our current

commercial infrastructure

KD034: Kadmon Developing Improved Formulations of Trientine

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Syprine (current formulation):

– Capsule, 3-4x per day, requires refrigeration, generic

Kadmon is developing four next-generation trientine formulations:

– KD034 (proprietary formulation): Effervescent powder that dissolves upon contact with water

(ease of administration for children); room temperature stable

– Tablet: room temperature stable

– Capsule: generic Syprine®

– BID and QD formulation: First-line and maintenance label

Benefits of Kadmon Formulations over Trientine

KD034 Development Status

Reformulation has been completed; conducting stability and bioequivalent studies

505(b)(2) approval anticipated Q1 2017

Kadmon Pharmaceuticals: Commercial Operation

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Fully integrated pharmaceutical company

– Comprehensive, scalable infrastructure

with a specialty pharmacy-focused sales

platform

– Headquartered in Warrendale, PA;

70 employees

Distributes and markets branded and

generic medicines; focused in liver disease

– Ribavirin for chronic hepatitis C:

35% market share

– Co-promote Syprine® (trientine) for

Wilson’s Disease with Valeant

Pharmaceuticals

Multiple potential co-promote agreements

– Near-term opportunities leveraging

specialty pharmacy focus

Kadmon Technology Subsidiary: Meira Gene Therapy (MeiraGTx)

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Independent subsidiary of Kadmon

Corporation, LLC

Pipeline of therapies targeting a range of

inherited and acquired disorders

– Initial focus on treatments for xerostomia

and ocular diseases

Established partnerships with world-leading

institutions

Expected to go public within 12 months with

Kadmon owning a meaningful interest

post-IPO

Kadmon Preclinical Pipeline

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Monoclonal Antibodies

– VEGFR2

– PD-L1

– PD-1

– TIM-3

Fusion / Bi-functional Biologic Proteins

– Anti-PD-L1/IL-15 fusion protein

– VEGFR2/PDGFRβ

Preclinical Product Candidates

Kadmon Preclinical Pipeline: Future Anticipated INDs (2016 / 2017)

Glucose transporter 1 (GLUT1) inhibitor

Anti-PD-L1/IL-15 fusion protein

ROCK2 blood-brain penetrant molecule

ROCK2 soluble inhibitor

Kadmon Summary

Robust Clinical Pipeline With Multiple Shots on Goal

Newly Established Gene Therapy Subsidiary

Multiple Value-Enhancing Near-Term Inflection Points

Attractive Market Dynamics Across Multiple Therapeutic Indications

Profitable Commercial Business

Innovative Drug Discovery Platform

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