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Vertex Kalydeco Cystic Fibrosis mutations of CFTR gene: G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N, or S549R $294,000 $1.2B** * Figures from company press releases ** Leerink analyst report Market Cap of CF and Fibrosis Focused Companies 24 $286 $685 $1,347 $5,335 $26,000 $77 30 300 3000 30000 Corbus ProQR Insmed PTC Intercept Vertex Market Phase 3 PC Phase 2a Approved Products Conclusions Lead Product Resunab is a novel, safe and promisingly potent clinical stage anti inflammatory/anti fibrotic drug which acts to resolve inflammation Targets multiple rare inflammatory indications Proven safe in two Phase 1 trials Promising potency in multiple pre clinical models Launch two Phase 2 trials in 2015 (Cystic Fibrosis and Scleroderma) Completion of studies in 2016 Strong patent portfolio until 2033 25 26 100 River Ridge Drive Norwood, MA 02062 www.CorbusPharma.com
UMass Medical School Michael Knowles, M.D., Ph.D. UNC Chapel Hill James Chmiel, M..D. Case Western Reserve Medical School Robert Simms, M.D. Boston University School of Medicine Daniel Furst, M.D. UCLA School of Medicine Robert Zurier, M..D. UMass Medical School 22 Financial Profile 22 Stock Ticker: CRBP:OTCQB $77,400,000 Market capitalization as of November 5, 2014 $10,300,000 Raise from successful private placement (Q2 2014) from institutional and retail base 25,800,000 Common shares outstanding 41,500,000 Fully diluted shares outstanding (including warrants and stock options) $11,400,000 Available from exercise of callable warrants NASDAQ Up listing to NASDAQ planned by Q 1 2015 Q1 2015 File IND with FDA Q1+Q2 2015 Launch clinical trials Q1 2015 Plan to up list to NASDAQ H2 2016 Data from clinical trials Q4 2014 Q4 2016 23 Corbus Poised for Significant Upside Recent Deals Date Company Partner Type Drug Indication Stage Up Front Deal Total 11/14 Galecto BMS Option to acquire TD139 Idiopathic pulmonary fibrosis Phase 1 NA $444M* 8/14 InterMune Roche Acquisition Esbriet Idiopathic pulmonary fibrosis Approved NA $8.3B* 9/2013 Galapagos AbbVie License GLPG1837 Mutations in CF patients, including F508del and G551D Pre clinical $45M* $405M* 7/2011 Amira BMS Acquisition AM152 Idiopathic pulmonary fibrosis and systemic sclerosis Phase 1 $325M* $475M* * Figures from company press releases $405 $444 $450 $8,300 $100 $1,000 $10,000 Galapagos (License AbbVie) Galecto (M A BMS) Amira (M A BMS) Intermune (M A Roche) Recent Deals in CF and Fibrosis Pre Clinical Phase 1 Phase 3 Potential Value Indicators Recent IPO Date Company Lead Compound Indication Stage Market Cap 9/2014 ProQR QR 010 Cystic Fibrosis RNA repair Pre clinical $284.11M Company Drug Indication Cost per Year 2018 Sales Est.
Chief Executive Officer Amb. Alan Holmer Chairman of the Board David Hochmann Renu Gupta, MD Avery W. (Chip ) Caitlin World Class Scientific Advisors Professor of Biochemistry and Pharmacology; inventor of Resunab Professor of Pulmonary and Critical Care Medicine Professor Medicine, National PI on largest ever anti inflammatory CF study Chairman of International Clinical Scleroderma Consortium Director of UCLA Scleroderma Program Ex Chair of Rheumatology 21 Sumner Burstein, Ph.D.
Chapter of the Cystic Fibrosis Foundation Managing Partner of Orchestra Medical Ventures Over 17 years of venture capital and investment banking experience Former Managing Director of Spencer Trask Ventures, Inc. securing over $420 million in equity capital 25 years of development, regulatory and senior management experience in the biopharm industry Former CMO of Insmed, a specialty CF company and current advisor to the CEO Former Vice President and Head of US Clinical Research and Development at Novartis (2003 2006) CFO Celldex Therapeutics (CLDX) since 2000 Raised over $415MM financing 20 years experience in industry: Repligen (CFO) and Endogen (CFO) 20 Yuval Cohen, Ph.D.
Expert rheumatologist in scleroderma with decades of experience in clinical trial development. 19 Board of Directors Former CEO of PhRMA (1996 2005) Over two decades of public service in Washington, D.C. including Special Envoy to China (2007 2009) Former board member Inspire Pharma (sold to Merck for $430m in 2011) Chairman of the Board of the Metropolitan Washington, D.C.
Chief Executive Officer Mark Tepper, Ph.D. President Chief Scientific Officer Sean Moran, C.P.A. M.B.A. Chief Financial Officer Barbara White, M.D. Chief Medical Officer Co founder and former President of Celsus Therapeutics (CLTX) Expertise in developing anti inflammatory drugs including for CF Former VP USA Research Operations, EMD Serono; Sr. Investigator, Bristol Myers Squibb Key member of project teams which developed the following marketed drugs: Taxol (Ovarian Cancer, 2000 peak sales of $1.6B), Orencia (RA, 2013 sales of $1.4B), Rebif (MS, 2013 sales of $2.59B), Gonal F (Fertility, 2013 sales of $815MM) Former CFO: InVivo (NVIV), Celsion (CLSN), Transport Pharma, Echo Therapeutics (ECTE) Anika Therapeutics (ANIK) Former VP Clinical Medical Director at Amgen, UCB and Medimmune.
Kalydeco by Vertex priced at $320,000/yr) CFTR gene defect Infection Fibrosis Underlying Inflammation 11 Antibiotics Kalydeco (for 6%) Resunab Resunab Resunab targets key CF inflammatory players 11 TGF Lipoxin A4 Absent in CF lungs Genetically linked to disease Associated with worsening symptoms Replacement therapy effective in animal models CFTR defect Infection Fibrosis Underlying Inflammation Fibrosis inducing agent (Bleomycin) administered to lungs day 1 followed by daily oral Resunab for 21 days P .004 Bleomycin+ Resunab therapy Bleomycin Control Gonzales et.al., Annals of Rheumatic Diseases, 2012. 71:1545 51 * Measured by hydroxyproline Alveoli air sacs Resunab Reduces Pulmonary Fibrosis In Animal Models Control Bleomycin Bleomycin + Resunab 12 0 50 100 150 200 250 300 350 Resunab Planned Cystic Fibrosis Phase 2 Trial 13 Q1 2015 Q2 2015 Q3 2015 Q4 2015 Q1 2016 Q2 2016 Q3 2016 Q4 2016 Protocol filed with FDA X Study launches X First patient dosed X Study duration X X X X X X Last patient dosed X Study data released X Double blind placebo control study in the USA under IND from FDA Primary end points: Safety/tolerability + PK Secondary end points: Inflammatory biomarkers+ MOA + change in clinical outcome measures Patient number: 70 adults with CF in 15 20 sites Treatment duration: 3 months + 1 month follow up Dose response: 1mg/day, 5mg/day, 20mg/day and 20mg/2Xday 14 Diffuse Systemic Sclerosis ( Scleroderma ) Relief for a disease with no effective long term therapy Overview: Diffuse Systemic Sclerosis (Scleroderma) 15 Chronic inflammatory disease causing fibrosis of skin, joints and internal organs Orphan disease (50,000 patients in USA) 80% of patients are women in their 30 s and 40 s Common cause of death: lung fibrosis (50% mortality in 10 years) Early stage of disease responds to steroids/methotrexate but with serious side effects No effective and safe long term therapy available Pipelines often target Idiopathic Pulmonary Fibrosis (IPF) in conjunction to SSC 16 Resunab Inhibits Key Factors in SSC TGF beta plays key role in SSC progression (same in CF and IPF) Elevated TGF beta levels associated with disease progression Strong Resunab efficacy data in animal models Resunab reduces TGF beta and collagen in skin fibroblasts from SSC patients TGF beta Collagen Concentration of Resunab ( M) Concentration of Resunab ( M) Healthy skin fibroblasts Scleroderma patients skin fibroblasts 0 5 10 15 20 25 30 35 40 45 0 0.1 1 5 10 0 50 100 150 200 250 300 350 400 0 0.1 1 5 10 Resunab Inhibits Skin Thickening In Mouse SSC Model Gonzales et.al., Annals of Rheumatic Diseases, April 4, 2012 17 Control Bleomycin Bleomycin + Resunab Thick skin induced by Bleomycin Near normal skin after oral Resunab taken once daily for four weeks Healthy skin Resunab : Planned SSC Phase 2 Clinical Trial 18 Q1 2015 Q2 2015 Q3 2015 Q4 2015 Q1 2016 Q2 2016 Q3 2016 Q4 2016 Protocol filed with FDA X Study launches X First patient dosed X Study duration X X X X X X Last patient dosed X Study data released X Double blind placebo control study in USA under IND from FDA Primary end points: Safety/tolerability + PK+ Change in clinical outcomes Secondary end points: Inflammatory biomarkers+ quality of life (QOL) Patient number: 35 adults with SSC with 8 10 sites Treatment duration: 3 months + 1 month follow up Dose response: 5mg/day, 20mg/day and 20mg/2Xday Management Team Yuval Cohen, Ph.D.
Aspirin , ibuprofen, Celebrex ) Lacks metabolic side effects of corticosteroids Resuna b : Only CB2 Agonist Targeting Inflammation Company Indication Brain penetration Status Affects CNS Corbus Pharma Inflammation Minimal Entering Phase 2 No AbbVie Pain Full Phase 1 Yes Glenmark Pain Full Phase 1 Yes Eli Lilly Knee pain Full Phase 2 Yes AstraZeneca Post operative pain Full Phase 2 Yes Resunab is the only CB2 drug that can be used to treat inflammation because it does not target the brain 8 9 Cystic Fibrosis Targeting inflammation at the core of the disease 9 10 Antibiotics Kalydeco (for 6%) of patients Very limited treatment No treatment Overview: Cystic Fibrosis 10 Inflammatory orphan disease (30,000 patients in USA, 75,000 WW) Average life expectancy of CF patients is approximately 40 years Inflammation at core of disease s morbidity and mortality (pulmonary fibrosis) Very high doses of steroids/ibuprofen effective but rarely used due to toxicity Need for safe, chronic anti inflammatory drug is unmet and universally recognized Pharmaco economics support premium pricing (e.g.
The Company undertakes no obligation to publicly update any forward looking statement, whether as a result of new information, future events or otherwise. 3 Overview Corbus Pharma is focusing on rare, life threatening, chronic inflammatory diseases Lead drug Resunab : a first in class oral anti inflammatory/fibrosis small molecule Acts to trigger inflammatory resolution: the off switch for chronic inflammation Proven safe in Phase 1 + promising pre clinical potency in multiple animal models Phase 2 clinical trials to commence 2015: Successful $10.3m private financing round (May 2014) Obtained $1.3m in NIH grants IP protection until 2033 and potentially longer Commenced trading on OTC.QB in October 2014 3 Cystic Fibrosis (CF) Diffuse Systemic Sclerosis (SSC) also known as Scleroderma Our Target Indications: Current Future Indication Patient numbers (USA) Estimated Market size Current therapies for inflammation Drawbacks to current therapies Current lead indications: Cystic Fibrosis 30,000 $3B Steroids, ibuprofen Considerable side effects Diffuse Systemic Sclerosis (Scleroderma) 50,000 $2B Steroids, methotrexate Side effects, poor efficacy Potential future indications: Dermatomyositis 13,000 $1B Steroids, mAbs Side effects, poor efficacy Marfan Syndrome 5,000 $1B N/A Lupus (SLE) 500,000 1.5MM $3B Steroids, mAbs Side effects, poor efficacy Idiopathic Pulmonary Fibrosis (IPF) 70,000 $1B Pirfenidone Limited efficacy InterMune bought by Roche for $8.5B (2014) 4 5 Inflammation ON Pro inflammatory Mediators ( IL6, IL8, PGE2, LTB4) Immune cell TGF CB2 receptor Inflammation OFF Fibrosis ON Fibrosis OFF CB2 receptor is present on immune cells and activated by endogenous lipid mediators Activation of CB2 turns inflammation off ( inflammatory resolution ) Resunab expected to be first CB2 binding anti inflammatory drug to reach market Upstream of other approaches: potential for better safety and potency CB2 Receptor: Turns inflammation off Immune cell 5 COX LOX LXA Anti inflammatory Mediators (Lipoxins, Resolvins, etc) 4 6 7 Resunab 7 Resunab: synthetic oral CB2 agonist small molecule Designed to trigger the resolution of chronic inflammation Full manufacturing, drug supply, non clinical safety pharmacology package for Phase 2 programs Excellent clinical safety profile to date: two prior Phase 1 clinical trials (n=121) Preparing to launch two Phase 2 clinical studies in 2015 Lacks CNS side effects of other CB2 binding class members Lacks GI side effects of NSAID s (e.g.
These statements involve known and unknown risks, uncertainties, and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward looking statements. Prospective investors are cautioned not to place undue reliance on such forward looking statements, which speak only as of the date of this presentation.
Additional written and oral forward looking statements may be made by the Company from time to time in filings with the Securities and Exchange Commission (SEC) or otherwise. The Private Securities Litigation Reform Act of 1995 provides a safe harbor for forward looking statements. These statements may be identified by the use of forward looking expressions, including, but not limited to, expect, anticipate, intend, plan, believe, estimate, potential, predict, project, should, would, will and similar expressions and the negatives of those terms.
Developing Breakthrough Therapies for Rare Inflammatory Diseases www.CorbusPharma.com OTCQB: CRBP Exhibit 99.1 2 Forward Looking Statement 2 This presentation contains certain forward looking statements, including those relating to the Company s product development, clinical and regulatory timelines, market opportunity, competitive position, possible or assumed future results of operations, business strategies, potential growth opportunities and other statement that are predictive in nature.