pioneering innovative therapies for liver disease · o 7 years of us market exclusivity in ascites*...
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Non-confidential Investor PresentationJanuary 2018
Pioneering Innovative Therapies for Liver Disease
Forward-Looking Statements
This document contains forward-looking statements made pursuant to the safe harbor provisionsof the Private Securities Litigation Reform Act of 1995 that involve risks, uncertainties andassumptions that could cause BioVie’s actual results and experience to differ materially fromanticipated results and expectations expressed in these forward looking statements. BioVie has insome 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 differmaterially from those expressed in forward-looking statements are BioVie’s need for, and theavailability of, substantial capital in the future to fund its operations and research anddevelopment. Other risks are that BioVie’s compounds may not successfully complete pre-clinicalor clinical testing, or be granted regulatory approval to be sold and marketed in the United Statesor elsewhere. A more complete description of these risk factors is included in BioVie’s filings withthe Securities and Exchange Commission. You should not place undue reliance on any forward-looking statements. BioVie undertakes no obligation to release publicly the results of anyrevisions to any such forward-looking statements that may be made to reflect events orcircumstances after the date of that these slides are posted to BioVie’s website or to reflect theoccurrence of unanticipated events, except as required by applicable law or regulation.
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Company & BIV201 Highlights
• Lead drug candidate BIV201 has Orphan Drug designation, US patent protection, and FDA Fast Track status– Active agent in BIV201, terlipressin, is available in ~40 countries (not in
US or Japan)
• 2 of 6 patients have been dosed with BIV201 in ongoing Phase 2a clinical trial
• Initial target is ascites due to advanced liver cirrhosis– ~20,000 US patients and ~$500M US sales potential
• “Proof-of-concept” as continuous infusion in ascites– 2 independent studies overseas with 11/11 responders, no serious AES
• Developing a global franchise with $1 billion+ sales potential targeting multiple liver cirrhosis complications (HRS, BEV, other)
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Company History
• LAT Pharma LLC founded by J. Adams in 2007
• In 2016 LAT Pharma merged into NanoAntibiotics, Inc. (NNAB) and was renamed BioVie Inc. (BIVI)
• BIV201 in development for ascites due to liver cirrhosis:
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Key Milestones
Sep-16 Orphan Drug designation for ascites
Mar-17 FDA IND approval
May-17 Core US patent issues covering ascites
Jun-17 McGuire Research Institute IRB approval
Sep-17 Dosed 1st of 6 refractory ascites patient in Phase 2a clinical trial; 2nd patient in Nov
Dec-17 BIV201 receives FDA Fast Track status
Jan-18 DSMB interim review
MANAGEMENT
• Jonathan Adams, MBA, CEO, 28 years in
pharma/biotech finance, commercialization, and
marketing; key role at Searle Pharma in global
commercialization of Celebrex.
• Julie Anderson, MBA, COO, former marketing director
for Durata Therapeutics and Searle, where she led
Celebrex global marketing.
• Rich Wieland, CFO, former chief financial officer for
Lyphomed, Fujisawa USA, Advanced Life Sciences.
CLINICAL TEAM
• Penny Markham, PhD: Chief Scientist
• Patrick Yeramian, MD: Medical Director
• Leslie Koehler, RAC: Regulatory Lead
• Denise Smith, MS: CMC/Manufacturing Lead
PRIMARY MEDICAL 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
Experienced and Effective Management
BOARD OF DIRECTORS
• J. Adams, J. Anderson
• Cuong Do, MBA, Executive VP, Samsung Global
Strategy; former Chief Strategy Officer at Merck;
former partner at McKinsey, where he helped
build the healthcare practice.
• Hari Kumar, PhD, former CEO of Amira
Pharmaceuticals and Adheron Therapeutics; he
sold both companies very successfully.
• Jim Lang, MBA, former CEO of healthcare
analytics firm Decision Resources Group; former
CEO of Strategic Decisions Group; active investor
and advisor to several healthcare companies.
• Michael Sherman, JD, former managing director
at Barclays and Lehman Brothers; previously a
securities attorney.
• Mina Sooch, MBA, Former CEO of Gemphire
Therapeutics and ProNAi both went public on
NASDAQ raising >$100 M; venture capitalist
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Initial Disease Target: Ascites
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• Ascites is the accumulation of excessive fluid in the abdomen, usually caused by liver cirrhosis
• ~40% mortality rate within 2 years of ascites diagnosis*
– Refractory ascites: 50% mortality at 6-12 months
• Annual 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
* D’Amico 2014.
Scientific Rationale for Ascites
Liver Cirrhosis
Hepatic Resistance
Portal Hypertension
Splanchnic Blood Pooling
Ascites
Low Arterial Blood Pressure
Activation of RAAS etc.
Excess Retention of Salt + Water
BIV
201
Expected to decrease
splanchnic vasodilation
and portal hypertension
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Ex-US “Proof-of-Concept” Supports BIV201 Use in Ascites
Angeli et al. patent assigned to BioVie and published June 2017Gow et al. Letter to Ed. AJ Gastroenterology Jul 2016.
Continuous Infusion Terlipressin Dosing Offers Improved Safety
• BIV201 is designed to achieve a continuous low concentration of the vasoconstrictive agent terlipressin in the bloodstream
• European study of continuous IV infusion in HRS found improved safety (Cavallin M, Angeli P, et. al: 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)
– Serious adverse events (SAEs) were reduced by 51% (20% vs. 43%) with continuous IV infusion, with similar efficacy
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Liver Cirrhosis: A Substantial Burden of Care
• ~138K patient hospitalizations annually for liver cirrhosis complications*
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* 2010 US Hospital Discharge Data
BIV201 Pipeline & Support Studies
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ClinicalProgram Pre-IND Phase 2 Phase 3 Support
Estimated USPatient Pop.
Refractory/Intractable/ Advanced ascites
Gow 2016, Angeli 2016, Fimiani 2011
Type 2 HRS*Jindal 2016,use in Italy and Australia
Bleeding esophagealvarices (BEV)*
Approved ex-US (~40 countries)
Catecholamine-resistanthypotension
Morelli 2009Serpa Neto 2012
Prevention of BEV Conceptual
20,000 Amb
5,000 Amb
6,000 Hosp
125,000 Hosp
20,000 Amb
* HRS-1 and BEV hospital use approved in Europe; BEV approved in Asia.
Opportunity to Expand BIV201 Franchise Relatively Quickly and Inexpensively
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ClinicalProgram Pre-IND Phase 2 Phase 3
Estimated P3 Trial Cost
Target NDA Filing
Refractory/Intractable/ Advanced ascites
$6 - $8 M
Type 2 HRS $3 - $4 M
Bleeding esophagealvarices (BEV)
$4 - 5 M
Catecholamine-resistanthypotension*
$8 - $10 M
Prevention of BEV $6 – $8 M
2020
2020
2021
2021
2022
* Exploring a related opportunity in sepsis due to cirrhosis.
Addressing Critical Unmet Needs Creates Substantial Revenue Potential
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ClinicalProgram Estimated Mortality
Orphan Drug Potential?
Peak Revenue Estimates (US only)
Refractory/intractable/ advanced ascites
• 50% survival over 6-12 months• 75% mortality rate within 2
years
BIV201 hasOrphan
designation$500 M
Type 2 HRS
• HRS-2 – median survival 6-12 months
• HRS-1 (hospitalized) – median survival of 1.7 to 4 weeks
Yes $150 M
Bleeding esophagealvarices (BEV)
• 15 – 25% mortality at 6 weeks after bleeding episode Yes $100 M
Catecholamine-resistanthypotension
• 28-day mortality exceeds 50%
No $500 M
Prevention of BEV ? $200 M
BIV201 Clinical Development Plan
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• 2 patients have been treated with BIV201 in 6-patient open label Phase 2a trial
• Current Phase 2b trial plan: 24 patients (randomized placebo-controlled trial)
• UPSIDE SCENARIO: Skip P2b and proceed to pivotal Phase 3 trial– Commence Phase 3 in 60-80 subjects in 2018
– File NDA in 2020
– Possible composite endpoint: change in ascites fluid volume + serum creatinine
• Fast Track status secured; will seek Breakthrough Therapy designation
BIV201 IP Strategy
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• 3-tier intellectual property strategy:1. Orphan exclusivity:
o Orphan designation for BIV201 for ascites granted
o 7 years of US market exclusivity in ascites*
o Applying for additional Orphan drug designations
2. Patent protection:
o US patent covering BIV201 for ascites issued May 2017
➢ Patent expires in 2036
o Filed for ascites patent in Japan and China; PCT in Europe
3. NCE protection and follow-on indications:
o First approval of terlipressin NCE would provide 5 years’ protection against generics in US
o Add 6 months for each new indication (BEV, HRS, etc.)
* If first-to-market
Upcoming Catalysts
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• Targeting the following milestones in 2018:
1. Present PK study results on 6 patients Q2
2. Complete Phase 2a trial in Q2
3. Secure Breakthrough Therapy designation
4. Reach agreement with FDA for ascites trial pivotal endpoints
5. Request FDA clearance to commence Phase 3 trial
6. Establish commercial quality manufacturing by Q4
• Submitted $2 M+ NIH grant application in January
• Applying for additional US Orphan Drug designations
• Seeking patent protection in Japan and China
• Seeking a corporate strategic partner
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