Pioneering Innovative Therapies for Liver Disease

Transcription

1 Pioneering Innovative Therapies for Liver Disease Investor Presentation January 2017

2 Forward-Looking Statements This document contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 that involve risks, uncertainties and assumptions that could cause BioVie s actual results and experience to differ materially from anticipated results and expectations expressed in these forward looking statements. BioVie has in some cases identified forward-looking statements by using words such as "anticipates," "believes," "hopes," "estimates," "looks," "expects," "plans," "intends," "goal," "potential," "may," "suggest," and similar expressions. Among other factors that could cause actual results to differ materially from those expressed in forward-looking statements are BioVie s need for, and the availability of, substantial capital in the future to fund its operations and research and development. Other risks are that BioVie s compounds may not successfully complete pre-clinical or clinical testing, or be granted regulatory approval to be sold and marketed in the United States or elsewhere. A more complete description of these risk factors is included in BioVie s filings with the Securities and Exchange Commission. You should not place undue reliance on any forwardlooking statements. BioVie undertakes no obligation to release publicly the results of any revisions to any such forward-looking statements that may be made to reflect events or circumstances after the date of that these slides are posted to BioVie s website or to reflect the occurrence of unanticipated events, except as required by applicable law or regulation. 2

3 BioVie Company Highlights Lead drug candidate BIV201 has Orphan Drug designation Submitted IND; currently addressing FDA comments Expect to begin mid-stage (Phase 1b) clinical trial in early 2017 Initial target is ascites due to advanced liver cirrhosis ~100,000 US patients and ~$500M US sales potential Developing a global franchise with $1 billion+ sales potential targeting multiple liver cirrhosis complications (HRS, EVB, other) Active agent in BIV201, terlipressin, is available in ~40 countries (not in US) Proof-of-concept as continuous infusion in ascites 2 independent studies overseas with 11/11 responders, no serious AES Proven management, directors, medical advisors 3

4 Management Team and Advisors MANAGEMENT Jonathan Adams, CEO, 27 years in pharma/biotech finance, commercialization & marketing; key role at Searle Pharma in global commercialization of Celebrex. Amrit Shahzad, President, 25 years in pharma/biotech business development spanning multiple therapeutic areas, technologies, and platforms; variety of leadership roles at Roche, Amgen and Ikaria. SCIENTIFIC ADVISORS Guadalupe Garcia-Tsao, MD, Yale School of Medicine, 2012 President of AASLD Paolo Angeli, MD, University of Padova, Italy, Current Secretary of International Ascites Club Roberto Groszmann, MD, Professor Emeritus Yale School of Medicine BOARD OF DIRECTORS Jonathan Adams, Amrit Shahzad Cuong Do, Executive VP, Samsung Global Strategy; former Chief Strategy Officer at Merck; former partner at McKinsey, where he helped build the healthcare practice. Jim Lang, former CEO of healthcare analytics firm Decision Resources Group; former CEO of Strategic Decisions Group; active investor and advisor to several healthcare companies. ADVISORY & CLINICAL TEAM Barrett Ehrlich: Capital Markets & Finance Penny Markham, PhD: Chief Scientist Patrick Yeramian, MD: Medical Director Stacy Suberg, PhD: Regulatory Lead Denise Smith, MS: CMC/Manufacturing Lead 4

5 Initial Target: Ascites Due to Liver Cirrhosis Ascites is the accumulation of excessive fluid in the abdomen, usually caused by liver cirrhosis ~40% mortality rate within 2 years of ascites diagnosis 1 Per year direct treatment costs of cirrhosis exceed $4B, with ~326,000 hospitalizations annually Current recommended therapy: Salt-restricted diet and diuretics (off-label) Paracentesis (withdrawal of fluid by large needle) The FDA has never approved any drugs specifically for treating ascites 1 D Amico

6 BIV201 Pipeline Clinical Program Pre-IND IND* Phase 1/2 Phase 3 Care Setting Size of Patient Pop. Refractory or Intractable Ascites Ambulatory Type 1 Hepatorenal Syndrome (HRS)** Type 2 HRS Treatment of Esophageal variceal bleed (EVB)** Prevention of EVB In hospital Ambulatory In hospital Ambulatory * IND for lead indication refractory ascites submitted but not yet open. ** Approved in Europe (and for EVB in Asia)

7 BIV201 Opportunities with P1/2 Trial Success Clinical Program Pre-IND IND* Phase 1/2 Phase 3 Care Setting Target NDA Filing Refractory or Intractable Ascites Ambulatory 2020 Type 1 Hepatorenal Syndrome (HRS)** In hospital 2021 Type 2 HRS Ambulatory TBD Treatment of Esophageal variceal bleed (EVB)** In hospital 2021 Prevention of EVB Ambulatory TBD 7 * IND for lead indication refractory ascites submitted but not yet open. ** Approved in Europe (and EVB in Asia). Other potential BIV201 disease targets: Pediatric/septic shock, hyponatremia, pre-liver/kidney transplant

8 BIV201 Clinical Development Plan BIV201 is designed to achieve continuous low concentrations of the active vasoconstrictive agent (terlipressin) in the bloodstream Clinical development program in refractory/intractable ascites: Submitted IND application in November 2016 Written FDA comments received January 2017 Projected clinical development timeframes: o Early Year 1: Commence P1b clinical trial (6 patients open-label) o Late Year 1: Commence P2 study with control arm (possibility of qualifying as a P3 study) o Early Year 3: Commence pivotal P3 study o Year 4: NDA filing 8

9 BIV201 IP Status 3-tier intellectual property strategy: 1. Orphan Drug exclusivity o o 7 years of US market exclusivity (if first to market) Orphan designation for ascites granted; will apply for others 2. Patent protection o o Filed for refractory ascites in US and Japan; PCT in Europe Applications published in January 2017: US 2017/ A1 and WO 2017/ A1 3. NCE protection and follow-on indications o o Targeting first approval of terlipressin NCE in US, which would provide 5 years protection against generics Add 6 months for each new indication (HRS, EVB, hyponatremia, etc.) 9

10 Mechanism of Action for Terlipressin The active agent in BIV201 is terlipressin, a receptor agonist acting on receptor V 1 a Terlipressin MOA: Vasoconstriction of the splanchnic bed Reduces portal hypertension Improves effective arterial pressure Improves renal function Terlipressin is well understood with safety/efficacy profile established in ex-us studies BIV201 employs novel continuous infusion (CI) delivery of terlipressin Anticipated advantages vs. traditional IV bolus dosing: Sustained therapeutic levels of active agent Lower Cmax to reduce side effects 10

11 MOA and Rationale for Ascites Liver Cirrhosis Hepatic Resistance Portal Hypertension Splanchnic Blood Pooling BIV201 Low Arterial Blood Pressure Activation of RAAS etc. Excess Retention of Salt + Water Ascites Expected to decrease splanchnic vasodilation and portal hypertension 11

12 Clinical Design Based on Overseas Experience European study conducted by Dr. Paolo Angeli using continuous IV infusion in HRS patients (Cavallin, M: Hepatology, Dec. 2015) 78 HRS patients randomized to IV infusion or bolus dosing Infusion dose started at 2 mg/day (mean daily dose of 2.23 mg/day vs. 3.5 mg/day for bolus dosing) Side-effects were reduced by ~50% with continuous IV infusion, with similar efficacy Gow study in refractory ascites patients (published July 2016) All 5 patients responded to therapy with no serious adverse events 48% reduction in ascites fluid drained Significant improvements in renal function (sodium excretion, etc.) 12

13 Similar Results for Angeli 2 and Gow 3 Studies 2 Angeli patent application published Jan 2017 on USPTO website. 3 Gow et al. Letter to Ed. AJ Gastroenterology Jul 2016.

14 U.S. Disease Incidence Hospitalizations ~138K patients were hospitalized in the US for liver cirrhosis complications in

15 Upcoming Milestones Targeting open IND in early 2017 Planning to initiate a mid-stage (Phase 1b) clinical trial in early 2017 Will apply for US Orphan Drug designation for additional condition(s) Seeking issuance of patent protection in US and Japan; leverage PCT placeholder in Europe at a later date 15

16 Stock Information (as of Dec. 9 th 2016) Symbol:...BIVI Stock Price:...$ Week Range:...$ $0.45 Average Daily Volume:...7,565 Shares Outstanding:...87M Inside Ownership:...46% Market Cap:...$26M Reverse Merger:...April 11,

17 Select Liver Disease Company Valuations Public Companies in Liver Disease Lead Drug Indication(s) Phase(s) Market Cap as of 12/9/16 Tobira Therapeutics (TBRA) NASH Phase 3 enabling studies Acquired by Allergan for $1.7B Vital Therapies (VTL) Acute Alcoholic Hepatitis (AAH) Phase 3 $163M Arbutus BioPharma (ABUS) Hepatitis B Phase 2 $178M 17 Arrowhead Pharma (ARWR) Hepatitis B & A1 Antitrypsin Deficiency 2x Phase 2 $97M

18 Pioneering Innovative Therapies for Liver Disease Investor Presentation January 2017