Vaxart, Inc. · OTC:VXRT
Vaxart — Technical Presentation (March 2023)
45 slides from Vaxart's technical presentation, Mar 28, 2023
Slide 1: Norovirus KOL Event
NOROVIRUS KOL EVENT March 28, 2023
Slide 2: Forward-Looking Statements
FORWARD-LOOKING STATEMENTS This presentation contains forward-looking statements that involve substantial risks and uncertainties. All statements, other than statements of historical facts, included in this presentation regarding Vaxart’s strategy, prospects, plans and objectives, results from precl
Slide 3: Vaxart's Oral Vaccine Platform
Vaxart’s Oral Vaccine Platform Sean Tucker, PhD Vaxart Founder and Chief Scientific
Slide 4: Vaxart Solution: Intestinal Delivery + Targeted Immune Activation
Vaxart Solution: Intestinal Delivery + Targeted Immune Activation: Non-replicating Vector With A Molecular Adjuvant • Key Issues we solve: 1. Replicating oral vaccines don’t work well in the developing world 2. Protein delivered to the intestine is treated like food 3. Pre-existing adenovirus immuni
Slide 5: Benefits of Vaxart Vaccine Platform
Benefits of Vaxart Vaccine Platform Distribution • Temperature stable tablets • Self administered • Rapidly deployed in a pandemic or stockpiled Safety • Well-tolerated in studies to date • No injection site pain Immunogenicity • Vaccine antigens delivered to a mucosal surface creating mucosal IgA •
Slide 6: The Importance of Mucosal IgA and IgA Antibody Secreting Cells
The Importance of Mucosal IgA and IgA Antibody Secreting Cells • Mucosal Immunity Important For Protection Against Norovirus o Correlates of protection from human challenge studies: rapid induction of mucosal IgA, serum IgA • Correlate of protection in oral polio vaccine challenge o a4b7 positive Ig
Slide 7: Oral Tablet Vaccine Protects As Well As An Injected Vaccine Against A Respiratory Pathogen
Oral Tablet Vaccine Protects As Well As An Injected Vaccine Against A Respiratory Pathogen Oral Vaccine Candidate protected against influenza infection as well as market leading injected vaccine after influenza challenge Reduced Infection Rates for both vaccines • Both vaccines protected against ill
Slide 8: Working Model: Oral rAd5 Tablet Vaccine for the Norovirus Indication
Working Model: Oral rAd5 Tablet Vaccine for the Norovirus Indication 8
Slide 9: VP1 Capsid Protein Is the Transgene Antigen in Vaxart's Oral Norovirus Vaccine
VP1 Capsid Protein Is the Transgene Antigen in Vaxart’s Oral Norovirus Vaccine • VP1 mediates attachment to epithelial cells through interactions with carbohydrates 9
Slide 10: Vaxart Oral Norovirus G1.1 Vaccine Elicits Mucosal Homing B Cells
Vaxart Oral Norovirus G1.1 Vaccine Elicits Mucosal Homing B Cells That Are Enriched for IgA Production % of IgA+ 55.3% Day 1 Day 7 3.99% b7 CD19 IgA 19.9% Kim et al. JCI, 2018 IgG Clinical trial # NCT02868073 10
Slide 11: Summary
Summary Vaxart is developing an oral vaccine platform • There are major logistical advantages of giving out pills for vaccines • A key technical benefit of oral vaccination is the ability to make a mucosal response • Oral vaccine platforms have been tricky to develop, but Vaxart has solved the major
Slide 12: Norovirus Clinical Trials
Norovirus Clinical Trials James F. Cummings, MD Vaxart Chief Medical Officer
Slide 13: Norovirus Clinical Trials - Completed
Norovirus Clinical Trials - Completed Trial ID Phase/Design # subjects Age Vaccine & Dose (IU) # doses Top line data Completed VXA-G11-101 Phase 1 66 18-49 GI.1 monovalent: 2 • Favorable safety and tolerability profile ✓ double blinded 1.7x1010 • Higher immunogenicity responses observed in high dose
Slide 14: VXA-NVV-101 Study
VXA-NVV-101 Study Phase 1 Double-Blinded Placebo Controlled • GI.1, monovalent, two dose levels • monovalent in 66 healthy adult volunteers Vaccine Group Designation Dose (I.U.) Subject number Monovalent GI.1 VXA-G1.1-NN 1.7x1010 23 Monovalent GI.1 VXA-G1.1-NN 1x1011 23 Placebo -- -- 20 14
Slide 15: Dose Dependent Antigen-Specific IgG and IgA ASC Responses
Vaxart Oral Norovirus Vaccine: Dose Dependent Antigen-Specific IgG and IgA ASC Responses • This study tested 1 tablet (1.7e10) vs 7 tablets (1e11) generating immune responses • IgA ASC have correlated to protection in other enteric pathogen studies ASC - Day 7 10000 1000 ASC per 1e6 cells 100 10 1 P
Slide 16: Fecal IgA Responses Durable to 180 Days
Vaxart Oral Norovirus Vaccine: Fecal IgA Responses Durable to 180 Days No significant differences in fecal IgA responses between 1 tablet vs 7 tablets Fecal IgA response 10000 Fold Increase in VP1 IgA 1000 D28 D180 100 10 1 0.1 e e o os os eb D D ac w h Pl ig Lo H Group Kim et al. JCI, 2018 Fecal Ig
Slide 17: Immunophenotyping After G1.1 Viral Challenge Compared to rAd5 Vaccination
Immunophenotyping After G1.1 Viral Challenge Compared to rAd5 Vaccination Mucosal Homing IgA & IgG Plasmablasts B Cells Plasmablasts 7 days post- Norovirus Vaccination VP1 - G1.1 8 days post- Norovirus Infection G1.1 NVV-101 and Titration study 17
Slide 18: Plasmablast Responses Following Oral Immunization Comparable to GI.1 Infection
Plasmablast Responses Following Oral Immunization Is Comparable to GI.1 Infection NVV-101 and Titration study 18
Slide 19: VXA-NVV-103 Study
VXA-NVV-103 Study • Phase 1B Double-Blinded Placebo Controlled o GI.1, GII.4 or GI.1/GII.4 o monovalent or bivalent dosing in 80 healthy adult volunteers o Clinical trial # NCT03897309 Vaccine Group Designation Dose (I.U.) Subject number Monovalent GII.4 VXA-G2.4-NS 5x1010 5 sentinels + 15 Monovalen
Slide 20: Monovalent and Bivalent Norovirus Vaccine Generates Robust IgA ASC
Both Monovalent and Bivalent Norovirus Vaccine Generates Robust IgA ASC PBMCs Responder definition: • 3 standard deviations above the mean pre- vaccination count ASC assay • IgA > 23 spots/ 106 PBMCs Antigen Vaccine G1.1 G11.4 GI.1 85.70% 14.30% G11.4 68.40% 89.50% GI.1/ GII.4 77.80% 92.60% 20
Slide 21: VXA-NVV-104 Study – Elderly Subjects
VXA-NVV-104 Study – Elderly Subjects • Phase 1 Double-Blinded Placebo Controlled o GI.1, Low, medium and high doses in elderly subjects 55-80 years of age o Each dose cohort randomized with placebo o Two placebo subjects dropped out leaving 63 subjects to finish the trial Group Study Drug Dose (IU)
Slide 22: No Difference in ASC IgA Response by Age
No Difference in ASC IgA Response Is Detected When Stratifying by Age or When Comparing to Previous G1.1 Monovalent Clinical Trials IgA ASC by Age 10000 VP1 IgA ASC/ 106 PBMCs 55-65 1000 66-80 100 10 1 0.1 Low Med High Cut-off for responder (dotted line) is 23 spots 22
Slide 23: Sustained Serum IgA Antibody Responses After 200+ Days
Sustained Serum IgA Antibody Responses After 200+ Days Fold Change AU/ml 60 Placebo Placebo Low Low Medium Medium Anti-VP1 G1.1 IgA Anti-VP1 G1.1 IgA High High 40 Fold Change AU/ml 107 20 0 106 D0 D29 D57 D210 D0 D29 D57 D210 23
Slide 24: Sustained Serum IgG Antibody Responses After 200+ Days
Sustained Serum IgG Antibody Responses After 200+ Days Fold Change AU/ml 25 107 Placebo Placebo Low Low 20 Medium Medium Anti-VP1 G1.1 IgG Anti-VP1 G1.1 IgG High High Fold Change 15 AU/ml 10 5 106 0 D0 D29 D57 D210 D0 D29 D57 D210 24
Slide 25: BT50 Is Maintained for >210 Days
BT50 Is Maintained for >210 Days VXA-NVV-104 – BT50 BT50 (GI.1) 1 5 0 Placebo Low Medium BT50 (GMT) 1 0 0 High 5 0 0 1 29 57 210 25
Slide 26: Distal Mucosal IgA Is Detected in Subjects Given Norovirus Vaccine
Distal Mucosal IgA Is Detected in Subjects Given Norovirus Vaccine Fold Change MSD RLU per µg of Total IgA Nasal IgA G1.1 Nasal IgA G1.1 10 1500 Placebo Placebo Low Low 8 Medium Medium Anti-VP1 G1.1 IgA Anti-VP1 G1.1 IgA RLU/µg total IgA High 1000 High Fold Change 6 4 500 2 0 0 D0 D29 D57 D210 D0 D2
Slide 27: Norovirus Clinical Trials – In Progress and Upcoming
Norovirus Clinical Trials – In Progress and Upcoming Trial ID Phase/Design # subjects Ages Vaccine & Dose # doses Objectives Completed VXA-NVV-201 Phase 2 140 18-49 GI.1 monovalent: 1 Primary: double blinded vaccinated 1x1011 • Clinical efficacy of VXA-G1.1 NN to protect against Infectious virus 120
Slide 28: VXA-NVV-201 Study – Norovirus Challenge Study
VXA-NVV-201 Study – Norovirus Challenge Study • Phase 2 Double-Blinded Placebo Controlled Study o GI.1 or placebo, given to healthy subjects o Given norovirus infection 29+ days after vaccination o Determine infection and illness rates (AGE) in placebo and vaccinated subjects o Measure immune parame
Slide 29: VXA-NVV-202: Bivalent NVV-GI.1-NN & NVV-GII.4 – In Progress
VXA-NVV-202: Bivalent NVV-GI.1-NN & NVV-GII.4 – In Progress Study Design: Phase 2, multicenter, randomized, double-blind, placebo-controlled, single dose regimen, dose confirmation study Population: Healthy adults ages 55-80; 125 randomized + 10 open label high dose sentinels Product: Bivalent GI.1
Slide 30: VXA-NVV-108 Study
VXA-NVV-108 Study • Phase 1 Double-Blinded Placebo Controlled o GI.1, G2.4 bivalent vaccine, two dose levels o 76 healthy breast-feeding volunteers Vaccine Group Designation Dose (I.U.) Subject number VXA-G2.4-NS Bivalent 5x1010 + 5x1010 25 + VXA-G1.1-NN VXA-G2.4-NS Bivalent 1x1011 + 1x1011 25 + VXA
Slide 31: Vaxart Norovirus Vaccine Clinical Highlights
Vaxart Norovirus Vaccine Clinical Highlights • Induces broad immune responses – mucosal and serum • Immune response rates above 90% for high dose • Immune responses were durable, lasting over 200 days • Vaccine responses can be boosted after a year • Bivalent vaccine showed no interference between s
Slide 32: Norovirus Market Opportunity and Disease Burden
Norovirus Market Opportunity and Disease Burden Andrei Floroiu Vaxart Chief Executive Officer
Slide 33: Norovirus Program: Addressing A Very Large Opportunity
Norovirus Program: Addressing A Very Large Opportunity With Compelling Clinical Data Generated So Far • Enterically delivered mucosal vaccine addressing enteric mucosal infection • Compelling data based on completing 6 norovirus clinical trials enrolling almost 350 subjects – immune responses have b
Slide 34: Norovirus: $10 billion+ Disease Burden
Norovirus: $10 billion+ Disease Burden Presents significant threat to children and seniors 34
Slide 35: U.S. Opportunity Is Driven By Pre-school Children and Seniors
U.S. Opportunity Is Driven By Pre-school Children and Seniors Estimated community incidence of norovirus AGE among outpatients in Kaiser Permanente Northwest and Mid-Atlantic Health Plans, by age group* Age 0-4 5 – 64 65+ Population US 20M 260M 55M Price Target $5005 $501 $755 Prospect of ACIP High
Slide 36: Annual Delivery Matches Flu Seasonality
Annual Delivery of Norovirus Vaccine Matches Flu Seasonality: “Winter Vomiting Disease” Acute gastroenteritis outbreaks in the U.S.A. Commercial Impact: • The seasonality of disease allows for vaccination of norovirus vaccine at the same time people get flu vaccine • Potential customers already gett
Slide 37: ACIP Recommendation Would Insure Federal Government Coverage
ACIP Recommendation for A Norovirus Vaccine Would Insure Federal Government Coverage for Key Segments Population Size (M) Payer Pediatrics 20 VFC (~60%), ACIP recommendations likely for <5 year olds and ≥65 year olds Insurance Elderly 55 CMS This means that the federal government would pays for most
Slide 38: Norovirus and Pneumococcal Vaccine Markets
ACIP Recommended Norovirus and Pneumococcal Vaccine Markets Category A Norovirus Pneumococcal Population Size (M) Payer Population Size (M) Payer Pediatrics 20 VFC, Insurance Pediatrics 4 VFC, Insurance Elderly 55 CMS, Elderly 55 CMS First responders Employer, 2.7 Insurance Immunocompromised 7.4 Mil
Slide 39: Norovirus and Rotavirus Vaccine Markets
ACIP Recommended Norovirus and Rotavirus Vaccine Markets Category A Norovirus Rotavirus Population Size (M) Payer Population Size (M) Payer Pediatrics 20 VFC, Insurance Pediatrics 4 VFC, Insurance Elderly 55 CMS, First responders Employer, 2.7 Insurance Immunocompromised 7.4 Military 1.4 Rotavirus V
Slide 40: Norovirus and Zoster Vaccine Markets
ACIP Recommended Norovirus and Zoster Vaccine Markets Category A Norovirus Zoster Population Size (M) Payer Population Size (M) Payer Pediatrics 20 VFC, Insurance CMS, Elderly 65+ 55 Insurance Elderly 55 CMS, Employer, Elderly 50 - 64 60 Insurance First responders 2.7 Insurance Immunocompromised 7.4
Slide 41: Addressing the Pediatric Population Through Breast Milk Antibodies and Minitablets
Addressing the Pediatric Population Through Breast Milk Antibodies and Minitablets - Nursing infants (Gates Foundation co-funded study) • Utilize the current 250mg adult formulation to vaccinate lactating mothers - ~7/8 to 18 year old population • Utilize the current 250mg adult formulation - > 6/12
Slide 42: Different Formulations for Pediatric and Adult Markets Enable Optimal Pricing
Vaxart’s Different Formulations for the Pediatric and Adult Markets Enable Optimal Pricing for Each Segment Cost-effective prices vary greatly between the pediatric and adult markets US Norovirus Pediatric Older Adults Vaccine Market (6m – 5y) (≥65y) Population Size (M) 20 56 Cost-Effective Price $5
Slide 43: Additional Market Opportunity: ~90M People
Additional Market Opportunity: ~90M People Population Size (M) Payer ACIP Pediatrics 20 VFC, Insurance Recommended Permissive Category A Category B Elderly 55 CMS, First responders Employer, 2.7 Insurance Immunocompromised 7.4 Military 1.4 Childcare 1.8 Employer, Health Care Workers Self Pay 12.6 Fo
Slide 44: Norovirus Program: Addressing A Very Large Opportunity
Norovirus Program: Addressing A Very Large Opportunity With Compelling Clinical Data Generated So Far • Enterically delivered mucosal vaccine addressing enteric mucosal infection • Compelling data based on completing 6 norovirus clinical trials enrolling almost 350 subjects – immune responses have b
Slide 45: Questions
df} VAXART Questions ir@vaxart.com