March 2025
2
Investment Highlights
CMV Vaccine with Positive
Phase 2 Data
1
Potential Peak Sales of $1BN+
Robust IP, Biologic Exclusivity
7
Phase 2 Trial in Liver
Transplant to Start in 2024,
Other Trials (HSCT, SOT, HIV)
Ongoing & Planned
2
Large Potential Market with
100K+ Organ Transplants
Performed Globally Each Year
6
Development Pipeline Covers
Entire Spectrum of CMV
Prevention and Control in
SOT and HSCT
5
Significant Unmet Medical
Need, No Approved
Immunotherapy
3
Several Key Advantages vs.
Standard of Care (SoC)
Antivirals
4
3
Company Overview
2024
Initiation of
Phase 2 in Liver
Transplant
Clinically
Advanced
CMV Vaccine
Phase 2 Study
Met Primary Endpoint
in difficult-to-treat
allogeneic HSCT
2025
Initiation of
Phase 2 in Kidney
Transplant
2027
Topline Data in
Liver Transplant
$1BN+
Potential Peak US/EU
Sales in SOT alone
▪ Helocyte (or the “Company”) is a private, late-stage biotech company that addresses the unmet need in CMV prevention and control
— CDC estimates 50-80% of individuals are infected with CMV by age 40
o Asymptomatic in healthy individuals
o Life-threating disease in those undergoing allogeneic stem cell and solid organ transplantation
▪ Current Standard of Care (SoC): antivirals with significant limitations
— Includes severe toxicity (Black Box), delayed immune reconstitution, late CMV, drug resistance, and inconvenient dosing
▪ Our lead therapy, Triplex, for CMV control in Transplant
— Allogeneic hematopoietic stem cell transplant (HSCT): met primary endpoint in first-in patient Phase 2 study
o Results: Triplex safe, well-tolerated, highly immunogenic and efficacious
o End-of-Phase 2 Meeting with FDA completed (1Q2020)
— Solid Organ Transplant (SOT): kidney and liver; significantly larger markets, less competition, lower bar than HSCT
— Potential for significantly better safety, enhanced immune recovery, and limited dosing versus SoC
Helocyte is developing novel immunotherapies for the prevention and treatment of cytomegalovirus (“CMV”)
4
Triplex Phase 1
Development
Evolution Since Inception: Groundwork Completed
Helocyte has consistently met critical developmental milestones, paving the way for pivotal studies and approval…
2015
Company Inception
& Initial Growth
2015 -
2016
2020 -
Future
Clear Anticipated
Development Pathway
Mar. 2015
Executed an exclusive
worldwide license and option
agreement with City of Hope
Apr. 2015
Began
operations as
DiaVax
Biosciences
through the
collaboration of
City of Hope
and Fortress
Dec. 2015
Presented
Phase 1 data
at ASH
Nov. 2016
Phase 1 data is
published in Blood
2016-
2019
Triplex Phase 2
Development
June 2016
Opened
primary site
for Phase 2
Study at City
of Hope (PI,
Nakamura)
Nov. 2016
Opened second
site for Phase 2
Study at Dana
Farber (PI,
Baden)
Mar. 2019
Oral presentation of
Topline Phase 2 data
at the EMBT
Conference
Feb. 2017
Opened third site
for Phase 2 Study
at MD Anderson
(PI, Ariza Heredia)
2008
Earliest
development of
Triplex
commenced at
City of Hope
2024
Initiate Phase 2 Study
of Triplex in Liver Transplant
2020
End of Phase 2
Meeting; Publication
of Phase 2 data
2021
Phase 2 initiated for Triplex
in HIV/CMV co-infection
5
Triplex: Addresses Significant Unmet Medical Need in CMV Control
(1) Not approved for use in SOT patients
Four of five approved CMV antivirals carry Black Box
warnings for severe toxicity; newly-approved
Prevymis
(1)
caused diarrhea, nausea, fever and/or rash
in >20%
Antivirals delay reconstitution of immunity to CMV
Prophylactic antivirals must be administered daily for
up to 200 days (Prevymis for 180 days in SOT)
CMV events often occur after Day 100 post-transplant
and/or following use of preemptive or prophylactic
antivirals
All antivirals are susceptible to CMV resistance,
particularly following extended use; recent data
suggests Prevymis at greater risk (see Helou 2019)
Triplex (Immunotherapy)
Safe and well-tolerated in Phase 2
Rapid, robust and durable CD4 and CD8 virus-specific
global T Cell reconstitution in Phase 2 (key objective
post-transplant)
More convenient, less frequent dosing
Reduced rate of late CMV in Phase 2
Immunotherapy, thus not susceptible to resistance
(or anti-vector immunity)
Current Standard of Care (Antivirals)
Prophylactic antivirals (like newly-approved Prevymis
approved for HSCT) have demonstrated efficacy in the
range of ~40% (see Prevymis Phase 3 primary endpoint)
Equal or better efficacy than antivirals (50% reduction
in CMV events versus placebo in Phase 2)
Safety
Immune
Response
Efficacy
Late CMV
Dosing
Resistance
Prior to approval of Merck’s Letermovir (Prevymis) in HSCT
(1)
($370MM in 2021 sales), last CMV antiviral approved 15+ years ago
6
Triplex: Market & Commercial Opportunity
▪ Standard of Care: Antivirals
— Similar to Solid Organ Transplant, but greater preemptive use
o Preemptive: used in matched related/unrelated donors
— Merck’s Prevymis approved for prophylactic use in HSCT
o 2019 Sales: ~$165M
o 2020 Sales: ~$281M
o 2021 Sales: ~$370M
o 2022 Sales: ~$428M
o 2023 Sales: ~$605M
o Trend towards prophylactic use in haploidentical donors,
mismatches, cord blood
Triplex targets CMV control in SOT and HSCT; well-defined markets with significant unmet need and no approved immunotherapy
Hematopoietic Stem Cell Transplant (HSCT) Solid Organ Transplant (SOT)
▪ Standard of Care: Antivirals
— Preemptive: initiated upon evidence of CMV
o Used in low-to-moderate risk patients
— Prophylactic Antivirals: initiated prior to transplant, >180 days
o Used in higher risk patients
o Merck's Prevymis approved in SOT as of 06/2023
▪ Prophylaxis in high-risk kidney transplant (D+R-)
▪ Phase 3 (n=589): randomized (1:1), non-inferiority to
Valganciclovir (10% vs. 12%)
▪ Substantially larger market, treating both CMV(+) and (-) recipients
Procedure US Incidence EU Incidence Peak Sales
Allogeneic Stem
Cell Transplant
~9,000 ~15,000 ~$340MM
Procedure US Incidence EU Incidence Peak Sales
Kidney
Transplant
~25,000 ~25,000
$1BN+
Liver Transplant ~9,000 ~9,000
7
Triplex Overview:
• Mechanism
• Indications
• Administration
• Safety / Side
Effects
Triplex: Universal Multi-Antigen MVA-based CMV Vaccine
Triplex’s unique clinical profile positions it to potentially be a leading treatment for CMV control in SOT and HSCT recipients
Mechanism: Cellular Immunity to Primary CMV Proteins
▪ CD4 & CD8 T Cell Response to: pp65 + IE1 + IE2
— CD8 T Cell Responses: exert rapid antiviral effects against CMV (Feuchtinger, 2010)
o Triplex induced robust, durable CD8 T cells in HSCTs early post transplant in Phase 2
— CD4 T Cell Responses: provide physiological, sustained immune response to CMV (Feuchtinger, 2010);
o Triplex induced robust, durable CD4 T cells in HSCTs early post transplant in Phase 2
o HOOKIPA’s HB101 failed to show any appreciable CD4 T cell responses
▪ Vector: Modified Vaccinia Ankara (MVA), dosed safely in over 120,000 (elderly, children included), demonstrated
safety and immunogenicity in HSCTs with rapid, robust and durable CD4 & CD8 T cell responses observed
Indication
▪ Prophylactic control of CMV in solid organ transplantation (SOT) and allogeneic hematopoietic stem cell
transplant (HSCT)
Administration
▪ Intramuscular administration prophylactically as a single agent to recipient
▪ If CMV exceeding threshold detected after Triplex vaccination, antivirals given per institutional guidelines
Safety / Side Effects
▪ Triplex has proven to be safe and well tolerated in clinical trials with no Grade 3 or 4 side effects probably or
definitely related to the vaccine and no anti-vector immunity observed
▪ Most common Grade 1 and 2 side effects include fatigue, myalgia, transient headaches at the time of injection
▪ No cases of secondary transmission of MVA have occurred and MVA not integrated into host DNA
▪ Triplex can be used alone and following discontinuation of prophylactic antivirals
8
Pipeline
TRIPLEX
Pre
-Clinical
Phase 1
Phase
2
Phase 3
Liver Transplant
(Recipient Vaccination)
Kidney Transplant
(Recipient Vaccination)
CMV
-HIV Co-Infection on ART
Allogeneic (MRD) Stem Cell
Transplant (Donor Vaccination)
Allogeneic (
Haplo) Stem Cell
Transplant (D+R Vaccination)
Allogeneic (
Haplo, Mismatch)
Stem Cell Transplant in Peds
Combo with CD19 CAR for NHL
CMV
-HIV CAR (+/- Triplex)
9
Completed Studies of Triplex (4)
10
Completed Studies of Triplex
▪ Design: open-label, single-arm, single-center, dose-escalation (three levels)
▪ Patient Population: CMV+ and CMV- healthy volunteers
▪ Enrollment: 24 adult volunteers, eight in each dose cohort
▪ Data
— Safety: well-tolerated, no SAE or dose-limiting toxicities
— Immunogenicity: rapid, robust, durable CD4 and CD8 T-cell responses to
each of the three antigens
— Presented at ASH (12/2015) and published in Blood (11/2016)
Four Completed Trials Demonstrate Triplex Safety, Immunogenicity and Efficacy
(1) Data published in Blood
Phase 1 (Completed) Phase 2 in HSCT (Completed)
▪ Design: multicenter, double-blind, randomized (1:1), placebo-control
— Top Transplant Centers: City of Hope, Dana Farber, MD Anderson
▪ Patient Population: R+ matched related, unrelated allogeneic HSCT
▪ Enrollment: 102 adult subjects
▪ Safety
— Safe, well-tolerated; no grade 3-4 adverse events related to vaccine
— No adverse impact on transplant (GvHD, relapse, or survival)
▪ Immunogenicity
— Rapid, robust, durable CD4 and CD8 T-cell responses
▪ Efficacy
— Comparable to Prevymis and other SoC antivirals
— Met Primary Endpoint: 50% reduction in CMV events through Day 100
o 5 CMV Events in Vaccine Arm (9.8%) vs 10 in Placebo (19.6%) (p=0.08)
▪ Presented at EBMT (03/2019), Published in Annals of Internal Medicine (02/2020)
Representative
(1)
CD4 & CD8 T-cell responses of two healthy volunteers to each of three CMV
proteins targeted by Triplex, durable to 360 days
11
Completed Studies of Triplex (cont.)
Phase 1 in Auto-HSCT (Completed)
▪ Design: open-label, single-center (University of Minnesota)
▪ Patient Population: 20 autologous HSCT recipients (10 CMV+, 10 CMV-)
▪ Rationale: increase adaptive NK cells after transplant, reduce relapse in MM
▪ Data
— Safety: safe, well-tolerated, no SAE or dose-limiting toxicities
— Immunogenicity: CD4, CD8 T-cell responses in both R+ and R-
— Adaptive NK Cells: significant increase (p=0.02 versus unvaccinated control)
▪ Published in Transplantation and Cellular Therapy (03/2022)
Phase 1: Donor Vaccination in Allo-HSCT (Completed)
▪ Design: open-label, single-center (City of Hope)
▪ Patient Population: R+ with matched related adult allogeneic HSCT donors
▪ Enrollment: 17 adult donor-recipient pairs; 16 with CMV+ donors
▪ Data
— On Day 28 Post HSCT, significantly higher CD137+CD8 T Cells (p=0.017 and
for pp65 alone, p=0.0001)
— Reduced number of CMV events (18%) vs cohorts receiving prophylactic
antiviral Prevymis (37%)
— Strong immunity in recipient derived from vaccination of donor
— CMV Events (requiring antiviral intervention): 18% vs. 37% in cohort
treated prophylactically with Prevymis
▪ Presented at 2023 Tandem Meetings, Published in American J. Hematology
▪ Funded by City of Hope, other non-dilutive sources
Four Completed Trials Demonstrate Triplex Safety, Immunogenicity and Efficacy (cont.)
12
Ongoing Studies of Triplex (4)
13
Ongoing Studies of Triplex
▪ Design: open-label, single-center (City of Hope)
▪ Patient Population: adults with intermediate or high Grade B-lineage NHL
indicated for autologous HSCT in first relapse after complete remission
▪ Enrollment: 12-15 adults
▪ Administration: single infusion of CD19 CAR in combo with Triplex
▪ Primary Endpoints
— Safety of CD19-CAR T Cells alone and in combo with Triplex
▪ Secondary Endpoints
— Feasibility of autologous cell manufacturing
— Short- and longer-term CMV specific CD19 CAR T cell in vivo expansion
and persistence
— Assessment as to whether CMV specific CAR T cells respond to Triplex
— Rate of CMV reactivation after CAR T cell infusion
— Rate of progression free survival (PFS) and median overall survival (OS)
at 12 months after autologous HSCT
▪ Follow-Up: 1 year (primary endpoint), 3 year (secondary endpoints)
▪ Funded by City of Hope, other non-dilutive sources
Pilot Study: Triplex + CD19 CAR T for NHL (ongoing)
14
Ongoing Studies of Triplex (cont.)
▪ Design: randomized, multicenter, placebo-controlled trial
▪ Patient Population: adults aged 18-65 co-infected with HIV and CMV
▪ Enrollment: 90 subjects (fully enrolled as of 10/2023)
— 60 participants randomized to receive Triplex
— 30 participants randomized to receive placebo
▪ Administration: Days 0 and 28 after enrollment
▪ Primary Endpoints (through Week 48)
— Safety
— Change in pp65-secific CD137+ CD8+ T Cells
▪ Secondary Endpoints (through Week 96)
— Reduction in viral shedding (CMV replication)
— Change in IL-6, sCD163, IP-10, sTNFRII, D-Dimers
— Change in IE1-, IE2-specific CD137+ CD8 T Cells
▪ Follow-Up: 96 weeks following enrollment
▪ Funded by National Institutes of Health ($3.22M)
Phase 2: Adults Co-Infected with CMV & HIV (Ongoing)
Expanding Dataset in Other Indications
▪ Design: open-label, Phase 1 dose-finding trial at single-center (City of Hope)
▪ Patient Population: adults living with HIV-1 on stable ART who have
maintained viral suppression
▪ Enrollment: ~12-18 adults (TBD)
▪ Administration: single infusion of Bi-Specific CMV/HIV CAR, optionally
followed by administration of Triplex to drive proliferation of CAR
▪ Primary Endpoints
— Safety of Bi-Specific CMV/HIV CAR T cells (+/- Triplex)
— Dose Limiting toxicities
▪ Secondary Endpoints
— CD4+ T cell count and HIV RNA levels in peripheral blood
— Number of EGFR+ CD3+ T cells in peripheral blood
— Time to viral rebound (HIV RNA > 1,000 copies/mL for 4 weeks
— HIV reservoir analysis (total HIV DNA in CD4+ T cells)
▪ Follow-Up: 1 year (TBD)
▪ Preclinical data published in Molecular Therapy: long-term durability,
eradication of latent viral T cell reservoirs in immune cells
▪ Phase 1 Funded by $11.3M grant from California Institute of Regenerative
Medicine (CIRM), other non-dilutive sources
Phase 1: Triplex + Bispecific CMV-HIV CAR (ongoing)
)
15
Planned Studies of Triplex (4)
16
Planned Studies of Triplex
Targeting Largest Segments of Solid Organ Transplant: Liver and Kidney
Phase 2: Liver Transplant (2024) Phase 2: Kidney Transplant (2025)
▪ Design: randomized, double-blind, placebo-control
▪ Patient Population: seronegative kidney transplant candidates receiving a
transplant from a CMV positive donor within 2-12 months
▪ Enrollment: 176 adult subjects (at least 88 to receive transplant)
▪ Randomization: 1:1 between vaccine and placebo
▪ Administration: Days 0 and 28 after enrollment
▪ Sites: ~10 U.S.-based
▪ Primary Endpoints
— Safety
o Solicited AEs within seven days of each dose
o Unsolicited AEs within 28 days of each dose
o Anti-vector immunity (none in Phase 2)
— Immunogenicity
o Proportion of Responders
o Robustness & Duration of vaccine-induced CMV-specific immunity
— Efficacy
o Vaccine impact on CMV infection, reactivation 12-mos post-transplant
o CMV viremia requiring treatment (duration of treatment, etc.)
▪ Design: randomized, double-blind, placebo-control
▪ Patient Population: seronegative liver transplant candidates receiving a
transplant from a CMV positive donor within 2-12 months (D+R-) with
preemptive antiviral therapy strategy
▪ Enrollment: ~420 adult subjects (D+R-)
▪ Randomization: 2:1 between vaccine and placebo
▪ Administration: Days 0 and 28 after enrollment
▪ Sites: 15 U.S.-based (CAPSIL consortium)
▪ Primary Endpoints
— Safety
o Solicited AEs within seven days of each dose
o Unsolicited AEs within 28 days of each dose
o Anti-vector immunity (none in Phase 2)
— Immunogenicity
o Proportion of Responders
o Robustness & Duration of vaccine-induced CMV-specific immunity
— Efficacy
o Impact on days of CMV antiviral therapy within 100 days post-Tx
o CMV infection, reactivation 12-mos post-transplant
o CMV viremia requiring treatment (duration of treatment, etc.)
o Funded by up to $20M from National Institutes of Health
17
Planned Studies of Triplex (cont.)
Expanding Dataset in HSCT via Vaccination of HSC Donor
Phase 2: Donor Vaccination in
Matched Related Donor (MRD) Allo-HSCT (2023)
Phase 1b: Donor + Recipient Vaccination in High-Risk
Haploidentical Allo-HSCT (2024)
▪ Design: single arm, open label trial
▪ Patient Population: haploidentical transplant from CMV+
▪ Enrollment: ~18 adult subjects
▪ Randomization: 1:1 between vaccine and placebo
▪ Administration
— Haploidentical Donor vaccinated: single dose, -60- to -10 days
— Haploidentical Recipient vaccinated: three doses, D28, D56, D100
— Three Cohorts (+/- Prevymis): no prophy, D7-D28 prophy, D7-D100 prophy
▪ Sites: TBD
▪ Primary Endpoints
— Safety
o Solicited AEs within seven days of each dose
o Unsolicited AEs within 28 days of each dose
o Anti-vector immunity (none in Phase 2)
— Immunogenicity
o Proportion of Responders
o Robustness & Duration of vaccine-induced CMV-specific immunity
— Efficacy
o Vaccine impact on CMV infection, reactivation
o CMV viremia requiring treatment (duration of treatment, etc.)
▪ Funded by City of Hope, National Institutes of Health
▪ Design: randomized, double-blind, placebo-control
▪ Patient Population: haploidentical transplant recipients from CMV+ donor
▪ Enrollment: ~108 adult subjects
▪ Randomization: 1:1 between vaccine and placebo
▪ Administration
— MRD Donor vaccinated -60 to -10 days
— MRD Recipient not vaccinated
— No antiviral prophylaxis (preemptive therapy)
▪ Sites: ~3 clinical sites
▪ Primary Endpoints
— Safety
o Solicited AEs within seven days of each dose
o Unsolicited AEs within 28 days of each dose
o Anti-vector immunity (none in Phase 2)
— Immunogenicity
o Proportion of Responders
o Robustness & Duration of vaccine-induced CMV-specific immunity
— Efficacy
o Vaccine impact on CMV infection, reactivation
o CMV viremia requiring treatment (duration of treatment, etc.)
▪ Funded by $3.22M grant from National Institutes of Health
18
➢ HCMV Antigens Expressed in MVA, Methods (7,163,685), expires
2024
➢ rMVA Vaccines and Methods of Prep Thereof (8,580,276), expires
2031
➢ rMVA Vaccines and Methods of Prep Thereof (9,675,689), expires
2033
➢ Patent Term Adjustment, Patent Term Extension possible
(1)
➢ De Facto market exclusivity may extend well beyond patent life as
Triplex is a complex biologic associated with significant
manufacturing know-how
Robust IP Protection & Orphan Market Exclusivity
(1) Up to 5 years for Patent Term Extension (based on any regulatory delays), but in no event more than 14 years from product's approval date
➢ Orphan Exclusivity: 7
Years (US), 10 Years
(EU)
➢ Biologic Exclusivity: 12
Years (US), 11 Years
(EU)
Regulatory
Triplex IP
Biologic exclusivity for 12 years from initial launch in the U.S. provides high barrier to
entry and minimizes risk of generic or biosimilar competition
19
Experienced Management Team with Proven Track Record
Key Management
Lindsay A. Rosenwald, M.D.
Co-Founder and Executive Chairman
20+ years
experience
Life Sciences Entrepreneur
and Investor
Frank Taffy, J.D.
Co-Founder & Strategic Advisor
20+ years
experience
Board of Directors
Lindsay A. Rosenwald, M.D.
Executive Chairman
Michael S. Weiss, J.D.
Director
Frank Taffy, J.D.
Director
Tenor Overview Prior Experience
20
World-Class Key Opinion Leaders
Source: Company websites
Helocyte has partnered with a distinguished group of key opinion leaders
Don J. Diamond, PhD
Scientific Founder & Chair of SAB
35+ years of
experience
▪ Professor in the Department of Hematology & Hematopoietic Cell Transplantation at City
of Hope
▪ Research includes developing vaccines to combat hematologic malignancies, solid tumors,
and infectious pathogens such as the herpesvirus, cytomegalovirus (CMV) and HIV
Ajit Limaye, MD
CMV in SOT Expert
25+ years of
experience
▪ Board certified physician at the Infectious Diseases & Tropical Medicine Clinic and Kidney
Care and Transplantation Services at University of Washington (UW) Medical Center
▪ UW professor of Medicine and Allergy and Infectious Diseases
Michael Boeckh, MD, PhD
CMV in HSCT Expert
30+ years of
experience
▪ Head of the Infectious Disease Sciences Program within the Vaccine and Infectious Disease
Division at Fred Hutch
▪ Clinical expertise focuses on infections in the immunocompromised host, especially
diagnosis, prevention and treatment of CMV, VZV, BK virus, and respiratory virus infections
Krishna Komanduri, MD
HSCT Clinical Expert
20+ years of
experience
▪ Professor of Medicine, Transplantation and Cellular Therapy at University of Miami
▪ Research interest includes immune reconstitution after stem cell transplantation (SCT);
human T cell immunity to pathogenic viruses and fungi; graft-versus-host disease (GvHD)
and graft engineering
Ryo Nakamura, MD
PI at City of Hope for Phase 2 Trial
15+ years of
experience
▪ Professor in the Department of Hematology & Hematopoietic Cell Transplantation at City
of Hope
▪ Research is focused on stem cell transplantation and development of cancer vaccines
Lindsey Baden, MD
PI at Dana Farber for Phase 2 Trial
30+ years of
experience
▪ Associate Professor of Medicine at Harvard Medical School
▪ Research is focused on transplant / oncology infectious diseases, HIV vaccines, and novel
diagnostics for invasive fungal disease
Ella Ariza Heredia, MD
PI at MD Anderson for Phase 2
Trial
15+ years of
experience
▪ Associate Professor in the Department of Infectious Diseases, Division of Internal
Medicine, Baylor College of Medicine, Houston, TX
▪ Research is focused on stem cell transplant
Key Opinion Leaders Tenor Overview
21
CMV Market Overview and Unmet Need
22
CMV Overview
▪ CMV is a member of the herpes virus family and the
largest among known human viruses
▪ Once infected, an individual carries the virus for life,
typically in a latent state
— Prevalence of infection is strongly correlated with age
— CDC estimates 50-80% infected with CMV by age 40
▪ CMV can lead to severe disease and increased mortality
in immunocompromised individuals
— High risk individuals include HSCT and SOT recipients,
as well as developing fetus or newborn children
— CMV infection can predispose patients to transplant
rejection/failure, CMV-related infections, other
opportunistic infections, and increased mortality risk
— Most common infectious complication in HSCT & SOT
▪ Current Standard of Care: moderately effective antivirals,
often associated with toxicity, resistance, delayed
immune reconstitution, late CMV and/or extended dosing
— No approved vaccine for CMV prevention or control
▪ Helocyte currently progressing novel biologic (Triplex)
— Induces CMV-specific T cell immunity to control
reactivation in post-transplant (HSCT, SOT)
▪ Triplex Advantages vs. SoC Antivirals: safety, immune
reconstitution, dosing, and no likelihood of resistance
CMV infection is ubiquitous and usually benign, but is a major cause of morbidity and mortality in immunosuppressed patients
CMV Overview Effects of CMV
CMV is easily
transmitted and is
associated with a life-
long latency…
…with the ability to
become active
(reactivation) in those
with weakened
immune systems…
…and progress to CMV
disease and create
significant risk for
morbidity and
mortality
Direct
Effects
▪ Direct clinical effects include CMV viral
syndrome and end-organ disease
▪ CMV disease: pneumonitis,
gastrointestinal disease, hepatitis,
pancreatitis, nephritis, cystitis,
myocarditis, retinitis, CNS diseases,
thrombocytopenia, hemolytic anemia,
adrenalitis, disseminated disease
▪ Indirect effects include opportunistic
infections:
— Caused by bacteria, fungi or other
virus
— More commonly, it is these indirect
effects that contribute to mortality
with CMV infection
Indirect
Effects
CMV in Hematopoietic Stem Cell & Solid Organ Transplantation
▪ CMV syndrome
▪ Tissue-invasive CMV/end-organ disease
(GI tract most common)
▪ Include graft rejection, graft failure,
opportunistic infections, atherosclerosis
and heart disease, obliterative
bronchiolitis, new onset diabetes, lung
transplantation, higher mortality
23
Market Overview & Opportunity: Solid Organ Transplant (SOT)
Source: Transplant Observatory
(1) Market research indicated KOLs strongly supported vaccination in D+R- and R+ groups
The number of solid organ transplants, specifically kidney and liver, has risen year-over-year
Observations
Kidney & Liver Transplant Patient Stratification
2019 Transplants by Organ & Region
Organ U.S. Europe Globally
Kidney 25,490 23,593 92,532
Liver 9,236 9,333 34,694
Heart 3,863 2,350 8,409
Lung 2,569 1,964 6,470
Pancreas 963 609 2,025
Small Bowel 96 46 172
Total Organ Transplants 42,217 37,895 144,302
Represents Triplex’s target patient population for SOT
37K+ Solid Organ Transplants
in the EU per year
CMV occurs in ~50% of SOT
and Represents the Most
Common Infectious
Complication
Recent Executive Order
Aimed at Doubling the
Number of Kidneys available
in the US by 2030
Prevymis Approved in Kidney
Transplant as of 06/2023,
Currently No Approved
Immunotherapy or Vaccine
in SOT
D+R-
20-25%
R+
60-70%
D-R-
5-10%
(~50/50 D+R+/D‐R+)
Planned Phase 2
studies of Triplex in
Kidney and Liver
Transplant To Target
D+R- and R+ groups
(80%+ of market)
(1)
D+R- (Donor Seropositive,
Recipient Seronegative)
R+ (Recipient Seropositive)
D-R- (Donor Seronegative,
Recipient Seronegative)
40K+ Solid Organ Transplants
in the US per year
24
Low to Moderate Risk
~4,500 Patients in US
~30%+ Rate of CMV Events
Market Overview & Opportunity: Hematopoietic Stem Cell Transplant (HSCT)
Source: Center for International Blood & Marrow Transplant Research
(1) Extended dosing not only creates compliance issues, but gives rise to resistance
The number of stem cell transplants worldwide has grown steadily year-over-year, and correlates with an increased CMV burden
Observations
10K+ Allogeneic Stem Cell
Transplants in US per year
17K+ Allogeneic Stem Cell
Transplants in EU per year
CMV occurs in 30%-60%+ of
CMV(+) HSCT Patients and is
the most common infectious
complication
Recently Approved Antiviral
Prevymis with Better Toxicity
Profile, but Extended Dosing
(up to 180 days)
(1)
Estimated HSCT Patient Stratification & Rate of Infection
Stability in Number of Allo-HSCT Transplants in the U.S.
Moderate to High Risk
~2,250 patients in US
~40%+ Rate of CMV Events
High Risk
~1,350 patients in US
~50%+ Rate of CMV Events
High Risk
~900 patients in US
~50%+ Rate of CMV Events
9,400
10,200
10,800
11,000
10,700
10,700
10,800
10,300
~11,000
2010 2011 2012 2013 2014 2015 2016 2017 2018E
CMV occurs early after HSCT
(Day ~40) or after Day 100
(“Late CMV”)
25
Limitations of Current Standard of Care…
Source: Company websites
(1) Prevymis sales are based on actual growth from Q1 to Q2 2019 and annualized estimated Q3 2019
(2) Not approved for use in SOT
Preemptive and Prophylactic Antivirals (most with Black Box Warnings)
Prophylaxis:
▪ Commence treatment at the time of or
immediately following transplant (regardless
of viremia) and continue for 100 – 200 days
▪ Monitoring may still be recommended
▪ Historically, toxicity and/or low efficacy of
antivirals has limited use of this strategy
Preemptive:
▪ Commence treatment when CMV viremia
exceeds institutional threshold (monitored
weekly as part of standard of care)
▪ Therapy given two weeks or until viremia
reduced (discontinued with negative qPCR)
▪ Shorter duration of use has enabled use of
this strategy despite severe toxicities
associated with most approved antivirals
Prophylactic vs. Preemptive
Drug Label Admin / Dosing Black Box Warning
Ganciclovir
Prophylactic; preemptive
(1L or 2L)
IV, Oral (up to 200
daily doses)
Hematologic Toxicity, Impairment of
Fertility, Teratogenicity, and
Carcinogenicity
Foscarnet Preemptive (1L or 2L) IV Renal Impairment
Cidofovir Preemptive (2L or 3L) IV
Hematologic Toxicity, Impairment of
Fertility, Fetal toxicity, Mutagenesis, and
Carcinogenesis
Valganciclovir Prophylactic & Preemptive
Oral (up to 200
daily doses)
Hematologic Toxicity, Carcinogenicity,
Teratogenicity, and Impairment of Fertility
Letermovir
(2)
Prophylactic (R+ all-HSCT)
IV, Oral (up to 100
daily doses)
No Black Box, but Diarrhea, Nausea Fever,
Rash observed in 20%+ (Phase 3)
FDA Approved Antivirals for CMV
▪ Prevymis (Letermovir) - newest antiviral approved for CMV
Prophylaxis in Allo-HSCT (November 2017)
— 1st new agent for CMV infection in 15 years
— Efficacy: reduced rate of CMV to 38% vs. 61% in placebo
— Late CMV still occurs (nearly 20% in Phase 3)
— Daily administration through Day 100 post-transplant
— WAC price of $19,500 oral or $27,000 injectable for 14-
week (100-day) course of therapy (partially subsidized)
— 2023 Annual Sales (Projected) >$500M
26
Triplex: Addresses Significant Unmet Medical Need in CMV Control
(1) Not approved for use in SOT patients
Four of five approved CMV antivirals carry Black Box
warnings for severe toxicity; newly-approved
Prevymis
(1)
caused diarrhea, nausea, fever and/or rash
in >20%
Antivirals delay reconstitution of immunity to CMV
Prophylactic antivirals must be administered daily for
up to 200 days (Prevymis for 180 days in SOT)
CMV events often occur after Day 100 post-transplant
and/or following use of preemptive or prophylactic
antivirals
All antivirals are susceptible to CMV resistance,
particularly following extended use; recent data
suggests Prevymis at greater risk (see Helou 2019)
Triplex (Immunotherapy)
Safe and well-tolerated in Phase 2
Rapid, robust and durable CD4 and CD8 virus-specific
global T Cell reconstitution in Phase 2 (key objective
post-transplant)
More convenient, less frequent dosing
Reduced rate of late CMV in Phase 2
Immunotherapy, thus not susceptible to resistance
(or anti-vector immunity)
Current Standard of Care (Antivirals)
Prophylactic antivirals (like newly-approved Prevymis
approved for HSCT) have demonstrated efficacy in the
range of ~40% (see Prevymis Phase 3 primary endpoint)
Equal or better efficacy than antivirals (50% reduction
in CMV events versus placebo in Phase 2)
Safety
Immune
Response
Efficacy
Late CMV
Dosing
Resistance
Prior to approval of Merck’s Letermovir (Prevymis) in HSCT
(1)
($370MM in 2021 sales), last CMV antiviral approved 15+ years ago
27
Triplex Clinical Trials (Detailed)
28
Triplex: CMV Immunotherapy with Best-in-Class Potential
(1) Protocol still in process of being finalized and subject to review by FDA during EOP2
Indication
▪ Indicated for prophylactic control of CMV in post allogeneic hematopoietic stem cell transplant (HSCT) in oncology
Description /
Mechanism of
Action
▪ Universal (off-the-shelf, broadly-recognized) vaccine engineered to induce a rapid, robust and durable virus-specific T-
cell response to control CMV in recipients of allogeneic HSCT
▪ Consists of a recombinant Modified Vaccinia Ankara (MVA) vector incorporating genes expressing three immuno-
dominant proteins linked to CMV events in the post-transplant setting, UL83 (pp65), UL123 (IE1), and UL122 (IE2)
Administration
▪ Two Intramuscular injections administered prophylactically 28 days apart (optional third injection in HSCT)
Efficacy
Summary
Safety / Side
Effects
▪ MVA dosed safely in over 120,000 individuals in Germany (adults, children, high risk subjects)
▪ Triplex has proven to be safe and well tolerated in clinical trials with no Grade 3 or 4 side effects related to vaccine
▪ Most common Grade 1 and 2 side effects include fatigue, myalgia, and transient headaches at the time of injection
▪ No cases of secondary transmission of MVA have occurred, and MVA not integrated into host DNA
▪ Triplex can be used in conjunction with (and after discontinuation of) antivirals
▪ Immunogenicity: Triplex demonstrated rapid, robust and durable CD4 and CD8 T Cell responses in both healthy subjects
(Phase 1, n=24) and immuno-compromised allogeneic HSCT recipients (Phase 2, n=102)
o Initial data from donor vaccination and autologous HSCT trials further demonstrate robust, durable immunity
▪ Efficacy: Triplex demonstrated efficacy comparable to Prevymis and other SoC antivirals (Phase 2)
o Primary Endpoint (Met): pre-specified, one-sided 0.10 test (appropriate for Phase 2 trial)
o 50% reduction in CMV events versus placebo through Day 100
o 5 CMV events in Vaccine Arm (9.8%) vs 10 in Placebo Arm (19.6%) (p=0.08)
Differentiated Product Profile Addresses Significant Limitations of Standard of Care Antivirals
29
Triplex: Phase 1 Safety and Tolerability Study
Phase 1 Trial (Completed)
Design Method
▪ Open-label, single arm, dose-escalating trial to assess safety and
immunogenicity of Triplex
▪ Sample size of 24 healthy volunteers
▪ 3 Dose Levels (DL) / cohorts with 8 subjects per cohort
— DL1: 10Xe7 plaque forming units (PFU)
— DL2: 5X10e7 PFU
— DL3: 5X10e8 PFU
▪ Eligible Subjects: 18-60 years old, CMV(+) and CMV(-) healthy volunteers
▪ Administration: 1mL IM injection with identical booster 28 days later
▪ Primary Endpoint: safety and immunogenicity of Triplex for one year
after first injection
▪ CMV-specific and MVA vector-specific immune responses in PBMC by
longitudinally measuring T-cell levels through Day 360
Phase 1 Trial Results
Triplex was safe and well tolerated in all subjects and demonstrated robust and durable CD4 and CD8 T cell responses
Safety Immunogenicity
▪ Well-tolerated in most subjects at all DLs
▪ Single Grade 3 injection site AE (erythema)
resolved in a day reported in one DL3 subject
▪ Three mild to moderate cutaneous reactions
▪ Most common systemic reaction: mild fatigue,
myalgia, headache
▪ Rapid, robust, durable CD4 and CD8 T-cell
responses to each of the three antigens
▪ Responses observed in both seropositive and
seronegative recipients, including those who
were previously vaccinated for smallpox
▪ Responses to pp65 portion of vaccine recorded
in >80% and highly significant (P<0.00001)
▪ Responses to IE1 and IE2 Less Substantial: likely
due to nonviremic status of healthy population
as IE1 & IE2 among first proteins to be expressed
in CMV infection and reactivation
Phase 1 data confirms safety, tolerability and immunogenicity of Triplex
30
Triplex: Phase 2 Trial in Allogeneic HSCT
(1) To be included in the analysis for Phase 2, subjects must receive at least the first of two planned injections
Phase 2 Trial (Completed)
Design Method
▪ Multi-Center
— City of Hope (PI, Nakamura)
— Dana Farber (PI, Baden)
— MD Anderson (PI, Ariza-Heredia)
▪ Double-blind, randomized (1:1) vaccine / placebo
▪ Sample size of 102 patients
▪ Eligible Subjects: CMV-seropositive undergoing allogeneic HSCT from
matched related and unrelated donors
▪ Patients enrolled pre-transplant
▪ Received two post-transplant IM vaccinations (Day 28 and 56)
(1)
▪ Patients received Triplex or placebo injections on Day 28 and Day 56
post-transplant and followed for one year
▪ Primary endpoint: reduction in CMV Events through Day 100, Day 365
Phase 2 Trial Results
Top-Line Data Presented at European Society for Blood and Marrow Transplantation (EBMT) Conference (March 2019)
Full Dataset Published in Peer-reviewed Annals of Internal Medicine (February 2020)
Safety
Immunogenicity
Efficacy
▪ Independently monitored
▪ Safe, well-tolerated; no significant difference in grade 3-
4 adverse events (AEs) probably or definitely related to
vaccine, or serious adverse events (SAEs) between arms
▪ Balanced patient characteristics
▪ No adverse impact on any transplant-related outcome
(GvHD, relapse, or survival)
▪ Recipients with both CMV(-) and CMV(+) donors showed
strong reconstitution of CD4 and CD8 CMV-specific
immunity
▪ Immune response initiated soon after first injection and
elevated for 365 days post-HSCT
▪ Within the range of antivirals
▪ Primary endpoint met: 50% reduction in CMV
Events versus placebo through Day 100
— 5 CMV Events in Vaccine Arm (9.8%)
versus 10 in Placebo Arm (19.6%) (one-
sided 0.10 p=0.08)
▪ Powered by at least 90% at one-sided 0.10
level of significance to detect decrease in
events from 30% to 10% or from 40% to 15%
— Number of CMV Events Anticipated (30 to
40 total) versus Observed (15 total)
Phase 2 data demonstrates Triplex safety, immunogenicity and efficacy
31
Triplex for CMV Control in Liver Transplant
Phase 2 Trial (Planned)
Phase 2 Study Anticipated to Commence in 2024
▪ Induce the expansion of CMV-pp65, IE1, and IE-2-specific functional T-cells in CMV
seronegative and CMV seropositive patients with advanced liver disease who are
awaiting liver transplant from a CMV seropositive donor
— Triplex has the potential to decrease CMV-related complications and need for
toxic antiviral therapy in the post-transplant setting
Overview
Primary
▪ Safety
— Solicited AEs within seven days of each dose
— Unsolicited AEs within 28 days of each dose
— SAEs
— Development of anti-vector immunity (none observed in Phase 2)
▪ Immunogenicity
— Determine proportion of responders
— Determine robustness and duration of CMV-specific immunity
▪ Efficacy
— CMV infection or reactivation up to 12-mos post-transplant
— CMV viremia requiring treatment (duration of treatment, subjects with viremia at any
level, time to development of viremia, subjects with recurrent viremia)
Secondary
▪ Other clinical outcomes up to 12 months post-transplant
▪ CMV disease
▪ Acute allograft rejection
▪ Mortality
▪ Re-transplant
▪ Non-CMV infections
Objectives
▪ Prospective, randomized, double-blind, placebo-controlled trial
— Patient Population: CMV seronegative liver transplant candidates expected to
receive transplant from CMV seropositive donor within 2-12 months (D+R-) with
preemptive antiviral therapy strategy (no prophylaxis)
▪ Enrollment of approximately 420 patients
▪ Randomized 2:1 between vaccine and placebo
▪ Two planned vaccine administrations on Day 0 and Day 28
▪ Study will consist of approximately 15 U.S. sites (CAPSIL consortium, others)
▪ Funded by National Institutes of Health (~$20M)
Design
32
Triplex for CMV Control in Kidney Transplant
Phase 2 Trial (Planned)
Phase 2 Study Anticipated to Commence in 2025
▪ Induce the expansion of CMV-pp65, IE1, and IE-2-specific functional T-cells in CMV
seronegative and CMV seropositive patients with advanced kidney disease who are
awaiting kidney transplant from a CMV seropositive donor
— Triplex has the potential to decrease CMV-related complications and need for
toxic antiviral therapy in the post-transplant setting
Overview
Primary
▪ Safety
— Solicited AEs within seven days of each dose
— Unsolicited AEs within 28 days of each dose
— SAEs
— Development of anti-vector immunity (none observed in Phase 2)
▪ Immunogenicity
— Determine proportion of responders
— Determine robustness and duration of CMV-specific immunity
▪ Efficacy
— CMV infection or reactivation up to 12-mos post-transplant
— CMV viremia requiring treatment (duration of treatment, subjects with viremia at any
level, time to development of viremia, subjects with recurrent viremia)
Secondary
▪ Other clinical outcomes up to 12 months post-transplant
▪ CMV disease
▪ Acute allograft rejection
▪ Mortality
▪ Re-transplant
▪ Non-CMV infections
Objectives
▪ Prospective, randomized, double-blind, placebo-controlled trial
— Patient Population: CMV seronegative kidney transplant candidates expected to
receive transplant from CMV seropositive donor within 2-12 months (D+R-) with
preemptive antiviral strategy (no prophylaxis)
▪ Enrollment of approximately 176 patients
— Continues until target of 88 subjects receive kidney transplant from a CMV
seropositive donor within 12 months after first dose
▪ Randomized 1:1 between vaccine and placebo
▪ Two planned vaccine administrations on Day 0 and Day 28
▪ Study will consist of approximately 10 sites in the U.S.
▪ Option to Include Interim Analysis and/or Deploy Adaptive Phase 2/3 Design
Design
33
Pathway to Approval in HSCT
Generating Additional Data in HSCT to Support Broad Phase 3 Inclusion
Phase 3 Trial Overview
▪ Phase 3 study for Triplex will be an expanded version of the Phase 2 study with the same
endpoint and enrollment criteria, but a larger sample size and interim analysis
▪ Michael Boeckh, MD, PhD serves as a key internal resource for HSCT
— World-renowned expert on CMV in HSCT, advised large pharma on antiviral development
▪ End of Phase 2 Meeting Completed 1Q2020
▪ FDA confirms proposed Phase 3 trial will be sufficient for registration in lead indication
▪ Follow post-transplant patients for CMV events from Day 100 to day 365, while also tracking
patients’ relapse and survival from underlying cancer
▪ Ongoing studies in HSCT to facilitate integration of donor-vaccination paradigm and/or
expansion of patient population to include pediatrics and haploidentical donors
— Consistent with recent market assessment and survey (Cello Health)
Phase 3 Trial Highlights
Design: Randomized 2:1 between vaccine
and placebo
Endpoint: Reduction in CMV events through
day 100 (same as in Phase 2)
Total Participants: ~585 patients
Total Sites: ~50, likely to include the EU
Projected Total Cost: ~$30MM
34
Triplex HB-101
Clinical Stage
Met Primary Endpoint in Multi-Center Phase 2 in difficult-
to-treat allogeneic stem cell transplant (HSCT)
Failed Phase 2 in less difficult-to-
treat kidney transplant recipients
(more immuno-competent than HSCTs)
Vector
Modified Vaccinia Ankara (MVA), dosed safely in over
120,000 (elderly, children included), demonstrated safety
and immunogenicity in HSCTs with rapid, robust and
durable CD4 & CD8 T cell responses observed
Lymphocytic choriomeningitis (LCM), rodent-borne
viral infectious disease; deaths reported from LCM-
infected organ
donor (see CDC MMWR 05/2005); LCM vector primarily
generates CD8 T cells, but reduced CD4 T cell memory (Flatz,
2010)
Immune Targets pp65 + IE1 + IE2 pp65 + gB
Relevance of Targets
Prevention of CMV viremia critically dependent on
reconstitution of pp65-specific T cells (Walter, 1995); IE
gene with decisive role in CMV reactivation, IE2
indispensable to CMV replication (Paulus, 2009)
CMV humoral immunity to gB is not required for control of CMV
viremia after HSCT (Bowden, 1991); gB failed to prevent viremia
in kidney transplant (Astellas, ASP0113)
CD8 T Cell Responses
(1)
CD8 T cells exert rapid antiviral effects against CMV
(Feuchtinger, 2010); Triplex induced robust, durable CD8
T cells in HSCTs early post-transplant in Phase 2
In CMV(-) healthy volunteers, two injections of Triplex induced
rapid, robust pp65-specific CD8 response (max ~2.3%) compared
to three injections of HB101 (max ~0.3%)
CD4 T Cell Responses
(1)
CD4 T cells provide physiological, sustained immune
response to CMV (Feuchtinger, 2010); Triplex induced
robust, durable CD4 T cells in HSCTs early post-transplant
in Phase 2
In CMV(-) healthy volunteers, two injections of Triplex induced
rapid, robust pp65-specific CD4 response (max ~2%) compared to
three injections of HB-
101 (max ~0.02%). Recent immune analysis
of HOOK data makes no mention of CD4 or humoral responses,
consistent with previous data
Valuation Privately-Held (No Equity Financing To Date)
Public (NASDAQ: HOOK); Leerink 5/13/19 analyst report values
HB101 alone at ~$200M (prior to Phase 2 data)
Triplex vs. HOOKIPA’s HB101
HOOKIPA’s HB101 is a key comparable to Triplex for CMV control in solid organ transplant
35
Helocyte Summary
36
Commercial Opportunity
(1) United Network for Organ Sharing
SOT/HSCT Market Opportunity
Well-defined, Orphan disease markets with modest sales and marketing requirement
Current Transplant Center Landscape Commercial Infrastructure Required
SOT
▪ ~200 major transplant centers in US
(1)
performing 35,000+ solid transplants each
year
▪ ~400 major transplant centers across the
US and EU conduct over 75,000 solid
organ procedures on an annual basis
HSCT
▪ 25,000+ total allo-HSCT transplants are
performed in the US and EU annually
▪ Merck’s Prevymis >$500M in 2023 Sales
Projected (based on 1H2023 Sales)
— Robust financial performance to date
demonstrates need for innovative and
efficacious therapies for CMV
Number of SOT by Organ & Region
Only 20 sales reps with the ability to
target growing SOT and HSCT
patient populations
✓
Targeting an EU market with higher
incidence of CMV and greater
number of HSCT and SOT procedures
✓
Matched
Related,
Unrelated
Donor
50%
Haplo-identical
Donors
25%
Mismatched
Donors
15%
Cord
Blood
10%
~4,500
Patients in US
~2,250
patients in US
~1,350
patients in US
~900 patients
in US
Number of HSCT Patients in US Alone
Organ U.S. Europe Globall
y
Kidney 25,490 23,593 92,532
Liver 9,236 9,333 34,694
Heart 3,863 2,350 8,409
Lung 2,569 1,964 6,470
Pancreas 963 609 2,025
Small Bowel 96 46 172
Total Organ
Transplants
42,217 37,895 144,302
37
Investment Highlights Overview
Key Investment Highlights
Significant Unmet
Need for Safe &
Effective CMV
Immunotherapy
2
Two Novel Biologics
with Robust Phase 2
Data and Proof of
Concept
1
▪ Triplex: vaccine engineered to prophylactically reduce the occurrence of early and late CMV after Allogeneic Hematopoietic
Stem Cell Transplant (HSCT) and Solid Organ Transplant (SOT)
▪ Recent positive Ph. 2 data for HSCT; met primary endpoint with 50% reduction in CMV events through day 100 (p value
based on pre-specified, one-sided 0.10 test = 0.8); no safety issues, no adverse impact on GvHD, relapse, survival
— Strong Biomarker data suggest clinical outcomes T cell-mediated and vaccine-driven
▪ Multiple programs ongoing to evaluate further use in HSCT involving haploidentical donors, donor vaccination and pediatric
patients, in addition to kidney and liver transplant
▪ ~50-80% of population infected with CMV by age 40, life-threatening for immuno-compromised patients
▪ Limitations of current standard of care:
— 4 approved antivirals used preemptively, and 3 approved antivirals used prophylactically
— Generic antivirals are associated with high toxicity with Black Box Warnings for myelotoxicity / nephrotoxicity, as well as
late CMV after discontinuation, delayed immune reconstitution, and potential for resistance
— Newly-approved Prevymis for prophylactic use requires prolonged dosing regimen of up to 100 daily oral or IV doses
— No approved vaccine or immunotherapy
Clear Development
and Approval Strategy
3
▪ Phase 2 Trial of Triplex in Liver Transplant to commence in 2024
▪ Phase 2 Trial of Triplex in Kidney Transplant to commence in 2025
▪ Registrational Study of Triplex in HSCT to commence after donor, pediatric, haploidentical trials
— Feedback from End of Phase 2 Meeting supports proposed design and endpoints
38
Investment Highlights Overview (cont’d)
Key Investment Highlights
Attractive Niche
Target Market
5
Triplex: Differentiated
Clinical Profile
4
Robust IP Protection
and Orphan / Biologic
Market Exclusivity
6
▪ Robust patent portfolio and BLA status provides high barriers to entry and minimizes risk of generic / biosimilar competition
▪ Orphan Exclusivity provides 7 years and 10 years of protection in the US and EU, respectively
▪ Biologic Exclusivity provides 12 years and 11 years of protection after first commercial sale in US and EU, respectively
▪ BLA development pathway provides robust protection from future generic biosimilar threat
▪ Portfolio of three issued patents protecting Triplex through 2033, Biologic Exclusivity for 12 years from First Licensure
▪ Safe and well-tolerated in transplant recipients with no adverse impact on GvHD, relapse, survival
▪ Efficacy: equivalent or better than standard of care antivirals in Phase 2
▪ Convenient dosing of 2-3 IM injections with efficacy in the range of approved antivirals
▪ Drives immune reconstitution: key objective after transplant (delayed by antivirals)
▪ Durable effect following limited administration with potential to address late CMV (after day 100)
▪ No drug resistance: major issue with antivirals, especially with longer duration of use (Prevymis)
▪ ~25k annual allogeneic stem cell transplants in US/EU, ~50k annual kidney transplants in US/EU, and ~20k annual liver
transplants in US/EU; Executive Order for kidney health (July 2019) aims to increase kidney transplants per year by 17k
▪ Highly specialized market with favorable reimbursement and pricing dynamics; covered under Medicare
▪ Ability to effectively detail ~200 transplant centers in US with a cost effective salesforce of 20+ representatives
▪ Potential Triplex US & EU peak sales of $1BN+