KD025 in IPF: Topline Results
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1 KD025 in IPF: Topline Results Webcast Presentation February 13, 2018 Kadmon Holdings, Inc. 1
2 Forward-looking Statement 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 Quarterly Report on Form 10-Q for the period ended September 30, 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 ROCK Inhibition in Fibrosis 3
4 ROCK: Signaling Pathway with Major Therapeutic Potential ROCK Signaling Pathway Plays a Key Role in Multiple Tissue Types Rho-associated coiled-coil kinase (ROCK) is a serine/threonine kinase that mediates a wide range of functions, including cell movement, shape, differentiation and function 1 Two isoforms exist: ROCK1 and ROCK2, with both overlapping and non-redundant functions 1 ROCK is ubiquitously expressed, with high levels in smooth muscle tissues, brain and heart 2 Scientific literature has shown that ROCK is dysregulated in many chronic diseases, including IPF, chronic graft-versus-host disease (cgvhd), scleroderma, renal fibrosis, hypertension, glaucoma and others 3-6 ROCK inhibition has therapeutic potential in a wide range of diseases 1 Small GTPases. 2014; 5: e Post-Genomic Cardiology (Second Edition). 2014, Pages Pharmacol Rev Jan; 67(1): Blood Apr 28;127(17): Proc Natl Acad Sci USA Apr 25;114(17): Curr Drug Discov Technol Mar;6(1):
5 ROCK is the Common Pathway of Initiating Factors in IPF ROCK Regulates Multiple Pro-Fibrotic Processes, Including Myofibroblast Activation ROCK is downstream of major pro-fibrotic mediators, including: Transforming growth factor beta (TGF-β) Connective tissue growth factor (CTGF) CTGF Myofibroblast Cell Stress fiber formation Lysophosphatidic acid (LPA) ROCK regulates fibroblast differentiation to myofibroblasts, a defining feature of pathologic fibrosis ROCK ROCK MKL1 Pro-fibrotic genes ROCK mediates stress fiber formation ROCK regulates transcription of pro-fibrotic genes, including CTGF and alpha-smooth muscle actin (α-sma) Am J Pathol Apr;185(4): MKL1 MKL1 CTGF 5
6 ROCK Activity is Increased in Regions of Active Fibrosis RhoA/ROCK activity is increased in regions of active fibrosis in human IPF and in mice with bleomycin-induced lung injury Phosphorylation of myosin light chain (MLC 20 ) in the fibroblastic foci: evidence of RhoA/ROCK signaling in lung fibrosis ROCK activity analyzed in an ELISA-based assay IHC staining of IPF patients and mouse lung sections stained for phospho-mlc 20 (A F and H) or collagen (G, Masson s trichrome) J Clin Invest. 2013;123(3): Am J Pathol Dec; 179(6):
7 ROCK Inhibitor KD025 Reduced Lung Fibrosis in Bleomycin Model Reduced Fibrosis in Multiple Preclinical Models, Including Bleomycin and cgvhd Mouse Models Intratracheal Bleomycin KD025 treatment administered when fibrosis is already established (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 1 Blood Apr 28;127(17):
8 KD025: an Investigational, Oral ROCK2 Inhibitor for IPF KD025 Represents an Innovative Approach to IPF Therapy KD025 is an oral ROCK2 inhibitor in Phase 2 development for inflammatory and fibrotic diseases Demonstrated clinical activity of KD025 in an ongoing Phase 2 clinical trial in cgvhd 1 KD025 achieved durable clinical responses in approximately two-thirds of patients, including Complete Responses in organs with fibrotic disease KD025 was well tolerated, with no treatment-related SAEs KD025 demonstrates potential therapeutic utility in IPF by targeting key fibrotic processes mediated by ROCK 1 ASH 2017, Poster
9 KD : Trial Design 9
10 Eligibility Criteria, Trial Design and Key Endpoints IPF diagnosis within 5 years by HRCT/ lung biopsy Received pirfenidone and/or nintedanib or been offered both 18 years of age FVC 50% predicted DL CO 30% predicted FEV 1 / FVC ratio :1 KD mg QD (n=25) BSC* (n=14) Option to extend Crossover allowed at investigator s discretion Primary Endpoints: Safety and tolerability Change in FVC from baseline to 24 weeks Key Secondary Endpoints: Percentage of patients with a decline in FVC of 5% at 24 weeks Pulmonary Function Test (PFT) High-Resolution Computed Tomography (HRCT) 1 J Manag Care Spec Pharm Mar;23(3-b Suppl):S17-S weeks 48 weeks Diffusion capacity (DL CO) 6-Minute Walk Test (6MWT) Lung fibrosis as measured by quantitative HRCT *Best Supportive Care, as determined by investigator. BSC may include pulmonary rehabilitation, supplemental oxygen therapy, and/or other symptomatic treatments 1 ; excludes pirfenidone/nintedanib 10
11 KD : Trial Sites Site Name Location Principal Investigator University of Pittsburgh Pittsburgh, PA Kevin F. Gibson, MD Medical University of South Carolina Charleston, SC Timothy P. Whelan, MD University of California, Davis Sacramento, CA Timothy E. Albertson, MD, PhD, MPH University of Arizona Tucson, AZ Sachin Chaudhary, MD Pulmonary Associates Phoenix, AZ David M. Baratz, MD Central Florida Pulmonary Group Orlando, FL Syed I. Mobin, MD Pulmonary Disease Specialists Kissimmee, FL Thomas W. O'Brien, MD St. Francis Medical Center Clearwater, FL Francis Averill, MD 11
12 KD : Data As of February 1,
13 Demographics and Baseline Characteristics KD025 (N=25) BSC (N=14) Age, years 71.4 ± ± 6.8 Male, n (%) 18 (72.0%) 11 (78.6%) Time since IPF diagnosis, years 2.5 ± ± 2.2 Former smokers, n (%) 17 (68.0%) 10 (71.4%) Prior treatment: Pirfenidone or nintedanib, n (%) 13 (52.0%) 4 (28.6%) Lung function: FVC, % of predicted value 67.2 ± ± 18.2 FVC (L) 2.51 ± ± 0.87 DL CO, % of predicted 45.6 ± ± 10.8 FEV 1 /FVC ratio 0.83 ± ± 0.07 Data are presented as mean ± SD, unless otherwise specified 13
14 Patient Disposition KD025 arm: Patients have received 24 weeks of KD025 treatment (n=20) Includes crossover patients (n=3); excludes patients not evaluable at 24 weeks (n=10) BSC arm: Patients have received 24 weeks of follow-up (n=9) W24 Evaluable (n=20) Patients Randomized 2:1 (n=39) KD025 (n=25) n=30 Crossover to KD025 Prior W24 (n=5) Withdrawal / LFU/Not Evaluable (n=10) Withdrawal / LFU / Not Evaluable (n=10) AEs* (7) Not evaluable at W24 + (1) IPF misdiagnosed (1) Lost to follow-up (1) BSC (n=14) W24 Evaluable (n=9) 24 weeks (EOPT) *Each occurred in 1 patient: Gr3 LFTs (considered related); basal ganglia hemorrhage; carcinoma; congestive heart failure; dyspnea; hypotension; death due to unknown cause + Sternotomy; patient continues on KD025 14
15 KD025 Slows Change in FVC at 24 Weeks Median Change in FVC (ml) Absolute FVC difference was 127 ml, a relative difference of 73% Decline in FVC % predicted was 1% with KD025 versus 2% with BSC, a relative difference of 50% FVC%-Predicted Decline 5% 20% of KD025 patients experienced FVC decline 5%, compared to 44% BSC patients at Week 24, a relative difference of 55% Change in FVC (ml) at W KD ml (n=20) BSC -175 ml (n=9) % Patients with 5% FVC % Patients with 5% FVC Decline 50% 40% 30% 20% 10% 0% 20% (n=20) KD025 44% (n=9) BSC 15
16 Additional Analyses Demonstrate Benefit of KD025 KD025 Showed Benefit in Annualized FVC Decline Compared to 1 Year Prior to Randomization Evaluable patients randomized to KD025 for whom prior-year data were available (n=17): Annualized FVC decline of 126 ml one year prior to randomization, compared to an annualized FVC decline of 32 ml at 24 weeks of KD025 Patients with Prior Pirfenidone/Nintedanib Treatment Responded Better to KD025 Previously treated KD025 patients (n=10) showed an FVC decline of 2 ml at 24 weeks Previously treated BSC patients (n=2) showed an FVC decline of 273 ml at 24 weeks 16
17 KD025 Efficacy Compares Favorably to Approved IPF Therapies Patients who Continue on KD025 to Week 36 Maintain Slowed Rate of FVC Decline 0-50 KD ml (n=20) KD ml (n=12) Change in FVC (ml) BSC -175 ml (n=9) Range of ΔFVC in treatment arms of Phase 3 studies of approved IPF therapies nintedanib and pirfenidone 1,2 Range of ΔFVC in placebo arms of Phase 3 studies of approved IPF therapies 1, Time (weeks) 1 Supplement to: N Engl J Med 2014;370: Page 18, Table S5: Secondary lung function endpoints in INPULSIS -1 and INPULSIS -2 at week 52 (pooled data). 2 N Engl J Med 2014;370: DOI: /NEJMoa Page 2088, Fig 2B (ASCEND). 17
18 KD025 was Well Tolerated, with No Drug-Related SAEs 18/20 (90%) of KD025 Patients Evaluable at W24 Elected to Continue KD025 Beyond W24 KD025 1 (N=34) BSC (N=14) Mean duration of exposure (weeks) Patient-years of exposure All-grade adverse events 29 (85%) 11 (79%) Drug-related adverse events: All-grade adverse events 2 (6%) 0 Grade 3 or higher adverse events 1 (3%) 0 Serious adverse events 0 0 Resulting in study drug discontinuation 2 (6%) All patients treated with KD025 (includes 9 crossovers) 2 KD025 arm could receive treatment beyond 24 weeks; BSC arm received maximum 24 weeks of follow-up 3 Depression, Grade 3 LFT elevations 18
19 Conclusions Demonstrated Clinical Activity of KD025 in IPF KD025 slowed the decline in lung function in IPF patients over 24 weeks of treatment: Absolute change in FVC of -48 ml in KD025 patients, compared to -175 ml with BSC KD025 patients demonstrated 1.0% FVC decline, compared to 2.0% with BSC 20% of KD025 patients experienced an FVC % predicted decline 5%, compared to 44% of BSC patients KD025 was well tolerated, with no drug-related SAEs 90% of patients on KD025 elected to continue on treatment beyond 24 weeks Findings support continued development of KD025 in IPF Data to be presented at American Thoracic Society (ATS) International Conference in May
20 Q&A 20
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