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 other financial and business matters, including expectations regarding clinical development, product sales, operating 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,” “estim ate ,” “continue,” “ongoing,” “consider,” “anticipate,” “intend,” “seek,” “plan,” “project,” “expect,” “should,” “would,” or “assume” o r 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 our 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. 2
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 3
Product Pipeline: Current Program Funding Support Preclinical Phase 1 Phase 2 Phase 3 RSV Infants (Maternal Immunization) Elderly Pediatrics (6 mos – 6 yrs) Influenza Seasonal Pandemic (H7N9+Matrix-M) Pandemic (H5N1) New Vaccines Combination Respiratory Ebola w Matrix-M Rabies (@ CPLB JV) 4
Recombinant Nanoparticle Vaccines • 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 Infect Sf9 insect cells Sf9 cells express Clone genes into with baculovirus protein antigens baculovirus 5
Novavax Nanoparticle Portfolio Virus-Like Particles (VLP) Recombinant Protein Micelles Seasonal & Pandemic Influenza RSV, Rabies Hydrophilic head of HA, NA protein Protein particle Empty - No genetic material Hydrophobic tail of protein particle M1 Matrix Protein Protein particles form micelles for efficient Configuration antigen and size of presentation: the virus • Single without RNA antigen genome • Repeating unit 6
RSV F Nanoparticle Vaccine Update and Clinical Plans
Respiratory Syncytial Virus (RSV): Important Facts • Most common cause of infant LRTI globally ‒ Mortality exceeded only by Pneumo, HiB, and highest in LDCs 1 • Most frequent cause of infant hospitalization in the U.S. ‒ 132,000 – 172,000 U.S. hospitalizations annually 2 • Severe disease often leads to ongoing wheezing ‒ Associated with recurrent wheezing for years 3 • 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 8
RSV Vaccine Landscape 9
Target Populations for an RSV Vaccine Young Infants Via Pediatric Elderly Maternal Immunization Mitigate RSV disease Provide protection for Decrease respiratory burden that results from infants younger than six disease burden in waning immunity and months and most at risk children, prevent medical immunosenescence, of serious RSV disease, care, wheezing prevent hospitalization prevent hospitalization, and death medical care, wheezing 10
RSV Hospitalization Rates Peak in Infancy • RSV is largely a disease of healthy, term infants • Up to 80% of hospitalizations occur 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 11
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 Antibody Mother’s Blood to infants against RSV Novavax’ RSV -F vaccine induces palivizumab-competing antibodies (PCA) that will be transferred to the baby. Baby’s Blood 12
Maternal Immunization Induces Palivizumab-like Antibodies M202 in women of childbearing age • PCA levels at 400 μ g/ml, potential for placental concentration effect • Palivizumab ‘protective’ at 30μ g/ml in CR • PCA “Near absence” suggests that the site is immunologically cryptic, important to reinfection 500 Two-dose, 60 μ g One-dose, 120 μ g Palivizumab-Competitive ELISA ~400 μ g/ml 400 Responses 300 Day 0 Day 14 200 Day 28 100 Near-Absence 0 of PCA Placebo Group B Group C Group D Group E Group F Group G Group A N=77 N=75 N=76 N=79 N=76 N=81 N=85 N=80 13
RSV F-protein Nanoparticle Vaccine Candidate Clinical Development Plans - Maternal • M203 in Pregnant Women, 3 rd 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, 3 rd Trimester (2015) ‒ Efficacy: Prevention of severe RSV + LRTI in infants • RSV present by RT-PCR ‒ Global RSV trial in Northern and Southern Hemisphere 14
Target Populations for an RSV Vaccine Young Infants Via Pediatric Elderly Maternal Immunization Mitigate RSV disease Provide protection for Decrease respiratory burden that results from infants younger than six disease burden in waning immunity and months and most at risk children, prevent medical immunosenescence, of serious RSV disease, care, wheezing prevent hospitalization prevent hospitalization, and death medical care, wheezing 15
Elderly Immunization • E101: Immunogenicity in Elderly Adults: PCA • 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 Duration of Needed Immunity Palivizumab Competing Antibody 16
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