Jefferies Global Healthcare Conference, Nov. 20, 2014 1 - - PowerPoint PPT Presentation

jefferies global healthcare conference nov 20 2014
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Jefferies Global Healthcare Conference, Nov. 20, 2014 1 - - PowerPoint PPT Presentation

Jefferies Global Healthcare Conference, Nov. 20, 2014 1 www.novavax.com Safe Harbor Statement Certain information contained herein, particularly information relating to future financial or business performance, conditions or strategies and


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www.novavax.com

Jefferies Global Healthcare Conference, Nov. 20, 2014

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Safe Harbor Statement

Certain information contained herein, particularly information relating to future financial or business performance, conditions

  • r strategies and other financial and business matters, including expectations regarding clinical development, product sales,
  • perating expenses, and anticipated milestones constitute forward-looking statements within the meaning of the Private

Securities Litigation Reform Act. Forward-looking statements can be identified by the fact that they do not relate strictly to historical or current facts and generally contains words such as “believe,” “may,” “could,” “will,” “possible,” “can,” “estimate,” “continue,” “ongoing,” “consider,” “anticipate,” “intend,” “seek,” “plan,” “project,” “expect,” “should,” “would,” or “assume” or any variations of such words or other words with similar meanings, although all forward-looking statements do not contain these identifying words. These Novavax cautions that these forward-looking statements are subject to numerous assumptions, risks and uncertainties, which change over time and could cause actual results to differ materially from the results discussed in the forward-looking statements. These risks and uncertainties include, but are not limited to, risks relating to the early stage of Novavax’s product candidates under development; current results may not be predictive of future pandemic results, results of

  • ur seasonal influenza vaccine or any other vaccine that we may develop; further testing is required before regulatory

approval can be applied for and the FDA may not approve a vaccine even if further trial results are similar to those disclosed previously by the company; uncertainties relating to clinical trials, including possible delays in initiating or completing the trials and safety and efficacy results; dependence on the efforts of third parties; competition for clinical resources and patient enrollment from drug candidates in development by other companies with greater resources and visibility; and risks that we may lack the financial resources and access to capital to fund our operations including further clinical trials. Further information on the factors and risks that could affect Novavax’s business, financial conditions and results of operations, is contained in Novavax’s filings with the U.S. Securities and Exchange Commission, including our Annual Report on Form 10- K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which are available at http://www.sec.gov. Forward-looking statements are based on current expectations and assumptions and currently available data and are neither predictions nor guarantees of future events or performance. You should not place undue reliance on forward-looking statements which speak only as of the date hereof. The Company does not undertake to update or revise any forward-looking statements after they are made, whether as a result of new information, future events, or otherwise, except as required by applicable law.

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Company Overview:

  • Recombinant Nanoparticle Vaccine Technology:

‒ Virus-Like Particles and Nanoparticles induce robust and functional immunity

  • Late-Stage Development Programs:

‒ RSV Vaccine - Only late-stage RSV vaccine candidate

  • Infants via Maternal Immunization, Pediatrics and the Elderly

‒ Seasonal & Pandemic Flu – Novel recombinant vaccine

  • Breakthrough Clinical Data

‒ RSV Vaccine: induces immune response activity similar to blockbuster mAb ‒ H7N9 flu vaccine: first positive clinical data, published in NEJM

  • Strong Vaccine Development Infrastructure

‒ Experienced management team ‒ Strong balance sheet ‒ Commercial and clinical GMP manufacturing capacity

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Seasonal Ebola w Matrix-M Pandemic (H5N1) Pandemic (H7N9+Matrix-M) Combination Respiratory Infants (Maternal Immunization) Pediatrics (6 mos – 6 yrs) Elderly

Product Pipeline: Current

Preclinical Phase 1 Phase 2 Phase 3

RSV Influenza New Vaccines

Program

Funding Support

Rabies (@ CPLB JV)

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  • Select genetic sequences encoding vaccine antigens
  • Genes cloned into baculovirus
  • Baculovirus infects insect (Sf9) cells
  • Antigens expressed and purified as multimeric nanoparticles
  • Immunogenic particle with antigens in native configuration
  • Safety database with over 7,000 subjects

Recombinant Nanoparticle Vaccines

Clone genes into baculovirus Infect Sf9 insect cells with baculovirus Sf9 cells express protein antigens

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Novavax Nanoparticle Portfolio

HA, NA Protein M1 Matrix Protein Empty - No genetic material

Virus-Like Particles (VLP) Seasonal & Pandemic Influenza Recombinant Protein Micelles

RSV, Rabies

Hydrophilic head of protein particle Hydrophobic tail of protein particle Protein particles form micelles for efficient antigen presentation:

  • Single

antigen

  • Repeating

unit Configuration and size of the virus without RNA genome

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RSV F Nanoparticle Vaccine

Update and Clinical Plans

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Respiratory Syncytial Virus (RSV): Important Facts

  • Most common cause of infant LRTI globally

‒ Mortality exceeded only by Pneumo, HiB, and highest in LDCs1

  • Most frequent cause of infant hospitalization in the U.S.

‒ 132,000–172,000 U.S. hospitalizations annually2

  • Severe disease often leads to ongoing wheezing

‒ Associated with recurrent wheezing for years3

  • Complex pathophysiology = challenging vaccine puzzle

1. Nair, et al. Lancet 2010, 375:1545 2. MMWR , 2013, 263:141 3. Escobar et al. BMC Pediatrics 2013, 13:97

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RSV Vaccine Landscape

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Young Infants Via Maternal Immunization Pediatric Elderly

Target Populations for an RSV Vaccine

Provide protection for infants younger than six months and most at risk

  • f serious RSV disease,

prevent hospitalization, medical care, wheezing Decrease respiratory disease burden in children, prevent medical care, wheezing Mitigate RSV disease burden that results from waning immunity and immunosenescence, prevent hospitalization and death

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RSV Hospitalization Rates Peak in Infancy

  • RSV is largely a disease of

healthy, term infants

  • Up to 80% of hospitalizations
  • ccur in the first 6 months of

life

  • Infants have small airways,

immature immune systems

  • Natural immunity, derived from

the mother, is ineffective

  • No effective therapeutic

licensed for full-term infants

  • Palivizumab (Synagis ™)

licensed for prevention of RSV in pre-term infants only

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Maternal Immunization

Active transport of mother’s antibodies into baby’s blood

  • Mother’s antibodies from past

infections and vaccines

  • At full term baby has >100% of

mother’s antibody levels.

Natural RSV infection of mothers over decades provides incomplete protection to infants against RSV Novavax’ RSV-F vaccine induces palivizumab-competing antibodies (PCA) that will be transferred to the baby.

Mother’s Blood Baby’s Blood

Antibody

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Maternal Immunization Induces Palivizumab-like Antibodies

M202 in women of childbearing age

  • PCA levels at 400μg/ml, potential for placental concentration effect
  • Palivizumab ‘protective’ at 30μg/ml in CR
  • PCA “Near absence” suggests that the site is immunologically cryptic, important to reinfection

100 200 300 400 500 Placebo Group B Group C Group D Group E Group F Group G Group A Palivizumab-Competitive ELISA Responses Day 0 Day 14 Day 28

Two-dose, 60μg

N=77 N=75 N=76 N=79 N=76 N=81 N=85 N=80

One-dose, 120μg

~400μg/ml

Near-Absence

  • f PCA
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  • M203 in Pregnant Women, 3rd Trimester, Enrolling

‒ Randomized, observer-blinded, placebo-controlled ‒ Intramuscular with vaccine in third trimester women ‒ Safety of mothers and their infants through an RSV season, U.S. IND ‒ Maternal antibody transfer to infants ‒ Antibody half-life in infants up to 6 months ‒ All infant subjects followed for 1 year

  • M301 Efficacy in Pregnant Women, 3rd Trimester (2015)

‒ Efficacy: Prevention of severe RSV+ LRTI in infants

  • RSV present by RT-PCR

‒ Global RSV trial in Northern and Southern Hemisphere

RSV F-protein Nanoparticle Vaccine Candidate Clinical Development Plans - Maternal

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Young Infants Via Maternal Immunization Pediatric Elderly

Target Populations for an RSV Vaccine

Provide protection for infants younger than six months and most at risk

  • f serious RSV disease,

prevent hospitalization, medical care, wheezing Decrease respiratory disease burden in children, prevent medical care, wheezing Mitigate RSV disease burden that results from waning immunity and immunosenescence, prevent hospitalization and death

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  • 220 Elderly Adults, >60 yrs, single Dose RSV-F vaccine administered w/Flu vaccine.
  • The vaccine was well-tolerated and the PCA competing antibody response was

robust, and durable

  • Non-alum adjuvanted vaccination likely to be protective in the elderly as annual

seasonal vaccine

Elderly Immunization

  • E101: Immunogenicity in Elderly Adults: PCA

Duration of Needed Immunity

Palivizumab Competing Antibody

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  • E201 in Elderly, FSI in October 2014

‒ Epidemiology as primary endpoint, efficacy as exploratory endpoint ‒ Randomized, observer-blinded, placebo-controlled 1:1 in 1,600 subjects ‒ Primary endpoint: incidence of RSV+ Medically Attended Illness ‒ Exploratory, vaccine effect on a RSV+ Medically Attended Illness ‒ Will determine the attack rate and vaccine efficacy to power Phase 3 ‒ Safety of the vaccine

  • E301 Pivotal Efficacy in Elderly and High Risk Adults,

2015

‒ Primary endpoint, size and location determined by E201 ‒ Basis for licensure

RSV F-Protein Nanoparticle Vaccine Candidate Clinical Development Plans

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Young Infants Via Maternal Immunization Pediatric Elderly

Target Populations for an RSV Vaccine

Provide protection for infants younger than six months and most at risk

  • f serious RSV disease,

prevent hospitalization, medical care, wheezing Decrease respiratory disease burden in children, prevent medical care, wheezing Mitigate RSV disease burden that results from waning immunity and immunosenescence, prevent hospitalization and death

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Maternal Immunization

  • M203 in Pregnant women, 3rd trimester (FSI Sept 16, 2014)

‒ POC for transfer of maternal antibodies to infants

  • M204 Pivotal Efficacy in Pregnant women, 3rd trimester (FSI: ~2015)

‒ Efficacy: Prevention of medically attended RSV+ illness

Elderly

  • E201 in Elderly, Enrolling

‒ Epidemiology as primary endpoint, efficacy as exploratory endpoint

  • E301 Pivotal Efficacy in Elderly and High Risk Adults

‒ Basis for licensure

Pediatric Immunization

  • P101 in seropositive 2-5 year olds, Enrolling

RSV F-Protein Nanoparticle Vaccine Candidate Clinical Development Plans

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Summary of Clinical Studies to Date

  • Study 101: Safety and immunogenicity in healthy adults (n=120)

‒ Stimulated robust immune responses ‒ Induced production of palivizumab-competing antibodies

  • Study E101: Safety, immunogenicity, and dose finding in Elderly Adults (n=220)

‒ Safety, dose selection

  • Study E201: Safety, epidemiology and efficacy in Elderly Adults (n=1600)

‒ Define attack rate, Vaccine efficacy ‒ Enrolling

  • Study M201: Safety and immunogenicity, WOCBA (n=330)

‒ Confirmation of safety and immunogenicity in target population

  • Study M202: Safety dose finding in women of childbearing age (n=720)

‒ Selection of dose and schedule for pregnant women

  • Study M203: Safety and immunogenicity in 3rd trimester women infants

‒ Active immunization of mothers, safety, transplacental antibody transfer and half life ‒ Enrolling

  • Study P101: Safety and immunogenicity in pediatrics

‒ Seropositive children 2-5 years of age ‒ Enrolling

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Influenza VLP Vaccine Program

Seasonal and Pandemic

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Seasonal Influenza Vaccine Market

  • Approximately $3B in 2010/2011 across major markets
  • Strong growth since 2005/2006

‒ 2010 recommendation for universal vaccination in U.S. ‒ Improved supply

  • US remains the largest market with ~40% of sales
  • Medical need remains high

‒ Need for improved vaccines for children and elderly ‒ More rapid, scalable, and reliable production technologies needed

  • Novavax Seasonal and pandemic Influenza program

funded by $179 million BARDA contract

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Immunologic Targets of the Novavax Influenza Vaccines:

  • Three Shots on Goal

Hemagglutinin:

Receptor binding domain (RBD) and Viral entry (stem)

‒ NVAX VLP vaccine induces antibodies to both ‒ Khurana, J Virol. 2011

Neuraminidase:

Release of virus from infected cells

‒ Neuraminidase inhibiting antibodies measured in H7N9 and H5N1 NVAX vaccine trials ‒ Fries, NEJM, 2013

VLP

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Seasonal Influenza Clinical Development Plan

  • 2012 Phase 2 Seasonal influenza Vaccine Trial

‒ Transitioned to quadrivalent vaccine ‒ Demonstrated immunogenicity against all four viral strains ‒ Well-tolerated/No SAEs ‒ FDA seroprotection criterion for all four vaccine strains FDA, seroconversion criterion for three of four vaccine strains

  • Phase 2 dose confirmation (FSI November 17, 2014)

‒ Finalize manufacturing process ‒ Data for end-of-phase 2 meeting with FDA

  • Plan for initiation of Phase 3 trial

‒ Efficacy study in healthy adults

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Combination of RSV & Flu

1st Pentavalent Respiratory Vaccine Candidate

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Combination Respiratory Vaccine:

  • RSV F + Seasonal Influenza VLP, Preclinical Data
  • Combination Vaccine Could Replace Seasonal Flu Vaccines

RSV Flu Combo

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Emerging Viruses

Pandemic Flu – H7N9

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Pandemic Flu Clinical Trials

  • A/H7N9 Anhui VLP alone or with Saponin Adjuvant (2013)
  • Achieved dose-sparing goals with dose of 5 μg
  • Seroconversion and seroprotection rates, 81% HAI, 97% NAI
  • Data Published in NEJM
  • A/H5N1 Indonesia VLP alone or with Saponin Adjuvant (2012)
  • Achieved dose-sparing goals with dose of 3.75 μg
  • Seroconversion and seroprotection rates, 86% to 100% at all doses
  • Observed strong cross-reactive antibody responses for a second strain of

A/H5N1 Vietnam

Novavax avian influenza vaccines are more immunogenic than any similar vaccines described in published results to date.

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Pandemic Flu Clinical Trial: H7N9 VLP with Matrix-M™

P201 A(H7N9) VLP Antigen With Matrix-M1™ Adjuvant

  • Novavax proprietary saponin adjuvant, H7N9 VLP
  • Safety and Immunogenicity in 610 subjects, 18 to 64 years old
  • Data Announced on September 23, 2014

‒ Vaccine and adjuvant were well-tolerated, consistent with the company’s prior studies with saponin-based adjuvants ‒ Hemagglutination-inhibiting antibody responses similar to prior Novavax studies of A(H7N9) with another saponin adjuvant ‒ Strong neuraminidase inhibiting antibody responses in adjuvanted vaccine groups ‒ Matrix-M demonstrated a clear dose response

  • BARDA funded trial
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Upcoming Development Milestones

Clinical Trials Q1 Q2 Q3 Q4 Q1 Q2 Q3

RSV Maternal Ph 2 (M202) Data RSV Elderly Ph1 (E101) Data H7N9+Matrix Ph2 (P201) Data RSV Maternal Ph 2 (M203) RSV Pediatric Ph1 (RP101) RSV Elderly Ph2 (RE201) Seasonal Flu Ph2 (S206) Ebola GP w Matrix-M Ph 1 Combo RSV+Flu (RIC101) FSI FSI Data FSI Data

2014 2015

FSI Data FSI Data FSI Data FSI Data

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Experienced Management Team

Stan Erck, President and CEO

‒ Iomai, Procept, Integrated Genetics (Genzyme), Baxter

Sven Andréasson, SVP, Corporate Development

‒ Isconova, Beta-Cell, Active Biotech, Pharmacia

Greg Glenn, M.D., SVP, Research & Development

‒ Iomai, Walter Reed Army Institute of Research

Timothy Hahn, Ph.D., SVP, Manufacturing

‒ MedImmune, Merck

John Herrmann, J.D., SVP, General Counsel and Corporate Secretary

‒ Gene Logic, Celera Genomics, Baxter

Cindy Oliver, Ph.D., SVP Process Development

‒ MedImmune, Merck, NIH

Barclay “Buck” Phillips, SVP, CFO & Treasurer

‒ Micromet, Vector Fund Management, INVESCO Funds Group

John Trizzino, SVP, Commercial Operations

‒ Immunovaccine, MedImmune, ID Biomedical, Henry Schein

Russell “Rip” Wilson, SVP, Business Development

‒ Supernus, Iomai, North American Vaccines

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Financial and Corporate Highlights

Novavax by the Numbers: Cash and Investments as of September 30, 2014 $190.3 M Debt (not including equipment leases): None BARDA contract for seasonal and pandemic flu: $179 M Cash runway into: 2016 Number of employees (approx.): ~280 Shares Outstanding: September 30, 2014 238.5 M Fully-Diluted: September 30, 2014 255.5 M

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Company Overview:

  • Recombinant Nanoparticle Vaccine Technology:

‒ Virus-Like Particles and Nanoparticles induce robust and functional immunity

  • Late-Stage Development Programs:

‒ RSV Vaccine - Only late-stage RSV vaccine candidate

  • Infants via Maternal Immunization, Pediatrics and the Elderly

‒ Seasonal & Pandemic Flu – Novel recombinant vaccine

  • Breakthrough Clinical Data

‒ RSV Vaccine: induces immune response activity similar to blockbuster mAb ‒ H7N9 flu vaccine: first positive clinical data, published in NEJM

  • Strong Vaccine Development Infrastructure

‒ Experienced management team ‒ Strong balance sheet ‒ Commercial and clinical GMP manufacturing capacity