Investor Presentation

Transcription

1 Investor Presentation James Sapirstein, CEO NASDAQ: CTRV

2 Safe Harbor Statement This presentation may contain forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of Such forwardlooking statements are characterized by future or conditional verbs such as may, will, expect, intend, anticipate, believe, estimate and continue or similar words. You should read statements that contain these words carefully because they discuss future expectations and plans, which contain projections of future results of operations or financial condition or state other forwardlooking information. Such statements are only predictions and our actual results may differ materially from those anticipated in these forward-looking statements. We believe that it is important to communicate future expectations to investors. However, there may be events in the future that we are not able to accurately predict or control. Factors that may cause such differences include, but are not limited to, those discussed under Risk Factors in our periodic reports filed with the Securities and Exchange Commission, including the uncertainties associated with product development, the risk that products that appeared promising in early clinical trials do not demonstrate safety and efficacy in larger-scale clinical trials, the risk that we will not obtain approval to market our products, the risks associated with dependence upon key personnel and the need for additional financing. We do not assume any obligation to update forward-looking statements as circumstances change. This presentation does not constitute an offer or invitation for the sale or purchase of securities or to engage in any other transaction with ContraVir or its affiliates. The information in this presentation is not targeted at the residents of any particular country or jurisdiction and is not intended for distribution to, or use by, any person in any jurisdiction or country where such distribution or use would be contrary to local law or regulation. 1

3 Corporate Overview Company Profile Advanced clinical stage drug development company with a Virology focus Value Drivers CMX157: Novel, high potency lipid conjugate of tenofovir for treating Hepatitis B Phase 1b/2a trials ongoing CRV431: Novel, high potency cyclophilin inhibitor for treating Hepatitis B Preparing for pre-ind studies FV-100: Reduced shingles-associated, post-herpetic neuralgia (PHN) in Phase 2 trials Phase 3 trial ongoing Seeking to create a diversified and complementary virology pipeline Preclinical Phase 1 Phase 2 Phase 3 CMX157 CRV431 FV-100 Hepatitis B Hepatitis B Shingles and PHN 2

4 Experienced Leadership Leadership History James Sapirstein, R.Ph. Chief Executive Officer 30+ years bio/pharma experience, including leading six product launches Key in the development and launch of tenofovir John Cavan Chief Financial Officer 20+ years financial management experience at both public and private companies Financial/operational positions at large multinational companies John Sullivan-Bólyai, M.D., MPH Chief Medical Officer 25+ years antiviral development experience in all four clinical phases, focused on HCV and HBV Key in the filing of four successful NDAs Robert Foster, Pharm.D., Ph.D. Chief Scientific Officer 30+ years of pharmaceutical discovery and development Track record of successful biotechnology entrepreneurship Theresa Matkovits, Ph.D. Senior Vice President, Drug Development 20+ years of proven leadership in global drug development Development Team Leader for four globally marketed products 3

5 Experienced Leadership Scientific Advisory Board Nathaniel Katz, M.D. (Chair) Global PHN Expert Renu Gupta, M.D. Renowned Antiviral Academic Carol L. Brosgart, M.D. Tenofovir Developer Richard Whitley, M.D. Herpes Virus Expert Board of Directors Gary Jacob, Ph.D. (Chair) CEO, Synergy Pharmaceuticals John P. Brancaccio, CPA CFO, Accelerated Technologies Timothy M. Block, Ph.D. Hepatitis B Foundation, Co-Founder and President Arnold S. Lippa, Ph.D. Chair and CSO, RespireRX Pharmaceuticals 4

6 Global Antiviral Market $38 billion (2013) Projected to grow to $44 billion by 2018 Well-Served Segments Human Immunodeficiency Virus (HIV) Hepatitis C (HCV) Seasonal Flu Focused on high-need segments Potential to capture expanding markets Poorly-Served Segments Hepatitis B (HBV) Herpes Zoster Virus (HZV) Respiratory Syncytial Virus (RSV) Herpes Simplex Virus (HSV) 5

7 HBV Market Opportunity 350 million chronic HBV patients worldwide 15.4%* growth in chronic HBV patients in the U.S. through thousand Ref. 1 Ref. 2 Ref. 3 HBV-related deaths in World Health Organization (WHO) 2 DataMonitor Lavancy 2004 HBV-related ESLD or HCC are responsible for > million deaths per year and currently represent 5% to 10% of cases of liver transplantation US is the largest individual market w/ highest absolute growth Limited efficacy of current antiviral treatments highlights need for new therapeutic tools for chronic HBV treatment Ref. 4 *European Growth in Chronic HBV Cases UK France Spain 10.3% 9.2% 7.5% 4 DataMonitor

8 Factors Driving Selection of Direct- Acting HBV Therapies and US Sales Relative Importance of Factors Efficacy (potency) Barrier to resistance (durability) Safety & tolerability 2014 U.S. Sales of Current HBV Drugs Ref. 4 Nucleos(t)ide Immunomodulator Baraclude Epivir Hepsera Tyzeka Viread Total Pegasys $303.1M $3.6M $31.2M $96.8M $392.2M $929.9M $103M *Nucleos(t)ide Analogs 4 BioMed Tracker 7

9 Hepatitis B Portfolio Highlights Two Candidates with Best-in-class Potency Complementary Mechanisms of Action Derived from Proven Antiviral Compounds CMX157 Differentiated Profile Lipid prodrug of successful antiviral drug tenofovir (TFV) 97-fold more active than TFV against HBV in in vitro studies At least as potent as tenofovir alafenamide (TAF) Phase 1b/2a study ongoing Drug detected in blood at 5mg dose Phase 2 is head-to-head vs. Viread Composition of matter coverage to 2031 CRV431 Differentiated Profile Next-gen, non-immunosuppressive cyclophilin inhibitor Best-in-class potency, optimized selective index Interrupts HBV at multiple points Strong Preclinical Profile Blocks HBV entry into liver cells in vitro Reduces HBV DNA without toxicity; prevents liver fibrosis in vivo Suppresses HBsAg and HBeAg in vitro Composition of matter coverage to

10 CMX157 Novel and Highly Potent Prodrug of Tenofovir Enhanced Efficacy and Safety Increased bioavailability by harnessing lipid uptake mechanisms Enhanced target tissue penetration Decreased renal and bone toxicity by reducing circulating TFV Enhanced Potency 97-fold more potent in HBV than TFV in vitro Outperforms Gilead s TAF 200-fold more potent in HIV than TFV in vitro 9

11 CMX157 Best-in-Class in vitro Potency and Safety Test Article EC 50 (nm)* EC 90 (nm)* CMX ± ± 53 TDF (Gilead s Viread ) 52.7 ± ± 427 TAF (Gilead s new HIV backbone drug) 32.4 ± ± 261 High Potency and Antiviral Activity * Mean ± standard deviation Favorable in vitro safety profile Best-in-class anti-hbv activity in vitro Equal, possibly better efficacy at low dose vs. Gilead s Viread At least as potent as Gilead s TAF (numerically more active) Results are consistent across these and other experiments performed in different laboratories Potential for improved safety profile due to less circulating TFV Low potential for QT interval cardiac arrhythmias No genotoxicity in standard assays Low potential for cytotoxicity across a large panel of cell lines from different tissues Low potential for mitochondrial toxicity 10

12 CMX157 Delays Viral Rebound in vitro All Antivirals Tested at IC 90 All Antivirals Tested at 5 nm CMX157 showed two- to three-fold reduced viral rebound compared to TAF Longer duration of action supports once-daily dosing as well as potential for increased potency and safety at lower doses Results are consistent across two different experimental conditions 11

13 CMX157 Highly Targeted to the Liver Drug Concentration in Hepatic Portal Vein vs. Systemic Circulation (+ SD) Drug Concentration in Liver (+ SD) Rapid, efficient extraction and conversion to high liver levels of the active antiviral, TFV-PP after single dose in rat Portal vein levels of CMX157 peaked at 1-3 hours and declined rapidly 86% first pass extraction Same pattern observed in liver Low systemic levels of TFV Highly targeted to liver, with accumulation in kidney and small intestine. No substantial accumulation or retention in the heart. 12

14 Ongoing CMX157 Phase 1b/2a Clinical Study Week Healthy Volunteers 2wk dose + 2wk follow-up DSMB Review CMX157 25mg qd (n=10) TDF 300mg qd (n=2) CMX157 50mg qd (n=10) TDF 300mg qd (n=2) CMX mg (n=10) TDF 300mg qd (n=2) 50mg qd (n=8) Placebo (n=2) 100mg qd (n=8) Placebo (n=2) CMX157 10mg qd (n=10) TDF 300mg qd (n=2) DSMB Review 10mg qd (n=8) Placebo (n=2) 25mg qd (n=8) Placebo (n=2) CMX157 5mg qd (n=10) TDF 300mg qd (n=2) HBV Patients 4wk dose + 4wk follow-up 5mg qd (n=8) Placebo (n=2) 13

15 505 (b)2 Pathway and CMX157 Opportunity Offers an opportunity to streamline the CMX157 Development Program by referencing Viread s select preclinical and clinical safety data Can be leveraged for New Molecular Entities (NMEs) that are based upon changes to prior drugs CMX157 and Viread share identical chemistry of their active moiety, Tenofovir Allows reliance on investigations not conducted by or for the applicant and for which the applicant has not obtained a right of reference. Potential reference of Safety Database created over last decade as well as completed preclinical toxicity and carcinogenicity studies 14

16 CMX157 Development Strategy FDA Discussions Negotiate appropriate clinical plan TFV Safety Database Access Database created over last decade Dosing studies Study prospective dosing regimens and combination therapies CMX157 Development Develop for global use 15

17 CRV431 Novel and Highly Potent Cyclophilin Inhibitor HBV-Optimized Derivative Cyclosporine A (CsA) Positioned to be leading host-targeting cyclophilin inhibitor antiviral for treatment of HBV Mechanism of Action Interrupts viral life cycle at multiple points by blocking host factor interactions Blocks HBV entry into liver cells in vitro Highlights Best-in-class potency against HBV Well-characterized safety profile for drug class Issued composition of matter and use patents through 2032 Pre-clincial Findings Good oral bioavailability; significant first pass effect Non-immunosuppressive and safe in two animal models Evidence that it may prevent liver fibrosis Reduced or eliminated production/secretion of HBsAg and HBeAg Expected to be effective against all HBV genotypes 16

18 Goal of HBV Therapy Reduction of viremia below threshold of detection of % with virtually no emergence of resistance Current: Reduction in Viremia HBV Cure Eradication of the intrahepatic replication of the virus Future: Functional Cure Suppression of HBsAg cccdna eradication 17

19 HBV Targets: Combinations Toward a Functional Cure Indirect Acting Antivirals Direct Acting Antivirals (DAAs) In use Polymerase CMX157 Potential Rnase H RNAi Capsid Inhibitors rcdna-cccdna conversion cccdna inhibitors In use Immuno- Modulatory Interferons Potential Therapeutic Vaccines Toll R agonists STING, other innate defense Interleukins, other cytokines In use CRV431 Essential Host Functions None for HBV Potential Entry Inhibitors CRV431 Exit Inhibitors ContraVir is Open to Potential Combination Partnerships With CMX157 18

20 CMX 157 and CRV431 as Prime Candidates for Combination Therapy De-risked approach to a functional cure Improvements to current treatments by combining the complementary mechanisms of action of CMX157 and CRV431 Complementary mechanisms of action against hepatitis B virus (HBV) CMX157 is adirect-acting antiviral that inhibits HBV replication CRV431 blocks HBV from entering liver cells; interrupts viral life cycle at multiple points through interactions with host molecules Both are designed specifically to improve safety and substantially enhance clinical utility of the parent molecules from which they re derived Highly potent, best-in-class antivirals 19

21 FV-100 Poised to Capture Growing Demand in PHN FV-100 Global market expansion driven by aging population Millions Number of Shingles Cases Treatable Population is Derived From US Population Minus Those Effectively Vaccinated Times The Incidence Rate % Diagnosed = 91% % Treated = 74% PHN Disease Mild to excruciating pain long after shingles rash resolves >65-70% of shingles patients suffer from PHN for 30 days or more Can last for 2-3 years Disrupts sleep, mood, work, and activities of daily living FV-100 Opportunity No FDA approved antiviral therapies for PHN No other trials ongoing with PHN endpoints Generic antivirals not labeled for PHN pain Vaccines are unreliable 20

22 FV-100: Potential to be First Approved Antiviral Therapy for PHN Pain Targeted Mechanism of Action Highly specific nucleoside analogue designed for rapid and potent inhibition of HZV replication within dorsal root ganglion Differentiated Profile Oral prodrug formulation more readily absorbed Potential improvement in dosing convenience vs. current standard of care No dose adjustment required for patients with renal insufficiency Clinically Risk-Mitigated Well tolerated with clinically meaningful reduction in PHN pain Strong IP position Composition of matter protection to

23 Herpes Zoster (Shingles) Treatments Acyclovir Valacyclovir Famciclovir FV-100 Formulation(s) IV, PO (capsule, tablet, suspension), topical PO (tablet, caplet) PO (tablets, which contain lactose) PO Dose 800mg PO 5x/day for 7-10 days 1000mg PO 3x/day for 7 days 500mg PO 3x/day for 7 days 400mg PO 1x/day for 7 days Specificity for HZV Dose adjustment for renal disease? Yes Yes Yes No Current antivirals can speed lesion healing and resolution of pain but they do not reliably prevent the onset of PHN 22

24 FV-100 is Market-Differentiated Annual Prescriptions & Market Share Valacyclovir 11.6M 57% Acyclovir 8.0M 39% Famciclovir 0.8 M 4% 20 million prescriptions annually (U.S.) 5.8% Rx growth since 2010 (annualized average) Majority of prescriptions are generic Highly Competitive Profile: Opportunity for QD dosing Efficacy profile superior to valacyclovir Acute pain: 25-30% reduction Sub-acute pain: significant reduction PHN: significant risk reduction Safety profile comparable to valacyclovir Source: NPA Data - 12 months ending 12/31/2012 Drug 2014 Global Sales Acyclovir $1.113B Valacyclovir $802M Famciclovir $207M Drug 2014 U.S. Sales Acyclovir $471M Valacyclovir $359M Famciclovir $27M 23

25 FV-100 Clinical Highlights Demonstrated Safety and Efficacy >350 patients treated with FV-100 Phase 1 (N=3) and Phase 2 (N=1) studies completed Clinically meaningful reduction in PHN occurrence vs. valacyclovir (Valtrex ) Meaningful reduction in time to resolution of clinically significant pain 8-10% reduction in narcotic use for pain control Safety similar to other antivirals Proprietary & Differentiated Rapid onset of action for quick pain relief Higher potency vs. approved agents against HZV Potential for QD dosing vs. 3-5x/day for Valtrex No dose adjustments needed for patients with renal insufficiency Proceeding to Phase 3 Pivotal Phase 3 (Study 007) 3-arm study with PHN reduction endpoint (vs. Valtrex ) 24

26 PHN Endpoint Favors FV-100 FV-100 Key Results (Study 005) % PHN at 90 days % Reduction vs. valacyclovir % Reduction vs. valacyclovir Both FV-100 arms performed better than valacyclovir at reducing the percent of patients reporting PHN pain at 90 days Secondary pain endpoints consistently favor FV-100 Pain endpoints are FV-100 dose dependent Study 007 (Phase 3) will further examine this endpoint mg 400 mg Val 25

27 FV-100 Pivotal Trial vs. Valacyclovir (Study 007) Basic Design Multi-center, randomized, double-blind, parallel-group, comparative study (N=825) Three-arms, 275 patients/arm: 1. FV mg QD 2. FV mg BID 3. Valacyclovir 1000mg TID 7-day treatment with follow up through day 120 Up to 200 US centers Goals Optimize FV-100 dose and regimen Confirm efficacy using registerable primary endpoint with commercial feasibility Focus on clinically significant PHN Primary Endpoint Proportion of patients with PHN at day 120 Key Secondary Endpoints Time to loss of acute PHN pain Other pain and quality of life assessments Safety Pharmacokinetics 26

28 Financial Metrics Share Statistics Trading Symbol NASDAQ:CTRV Stock Price (6/27/16) $1.00 Capital Structure (Current) 32.2M shares of common Common Shares O/s 32.2M 4.1M options for common 52wk Range $ $ M warrants for common 3 Month Avg. Volume 383, million shares of preferred series A Cash $6M* 120k shares of preferred series B *Does not include April 2016 closing of $7 million offering of common stock and warrants 27

29 ContraVir Value Drivers Strategic Focus Develop and commercialize novel drug treatments to improve the lives of patients suffering from chronic viral infections Experienced Leadership Significant expertise in drug development and commercialization of targeted antivirals HBV Program: Phase 1b/2a studies; advancing CRV431 into clinical Combination therapy has a greater chance of eradicating chronic infection Strong composition of matter patents for both compounds Leveraging the TFV database could open up multiple clinical options, including a 505(b)2 pathway for streamlined approval and commercialization PHN Program: Ongoing Phase 3 Trial No FDA approved antiviral therapies for PHN If drug shows 60% prevention, high chance FDA will grant 2 indicators 28

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