Download J.P. Morgan Healthcare Conference 2017

Nasdaq: ATRA
J.P. Morgan Healthcare Conference 2017
Forward-Looking Statements
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the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. All statements other than
statements of historical facts contained in this presentation, including statements regarding our future results of
operations and financial position, business strategy, product candidates, regulatory approvals, ability to sell,
manufacture or otherwise commercialize our product candidates, research and development costs, timing and
likelihood of success, plans and objectives of management for future operations, any royalty payments, and our
ability to obtain and maintain intellectual property protection for our product candidates, are forward-looking
statements. These statements involve known and unknown risks, uncertainties and other important factors that
may cause our actual results, performance or achievements to be materially different from any future results,
performance or achievements expressed or implied by the forward-looking statements. These and other
important risk factors are described more fully under the heading “Risk Factors” in Atara Bio's quarterly report on
Form 10-Q filed with the Securities and Exchange Commission (SEC) on November 4, 2016, including the
documents incorporated by reference therein and subsequent filings with the SEC. Because forward-looking
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Leader in Allogeneic T cell Therapy
• Potential first-to-market allogeneic T cell therapy
Lead Product Candidate Entering Phase 3 Registration Trials
• Phase 3 designs agreed with FDA; Submitting for conditional approval in EU
Well-Capitalized; Highly Experienced Management Team
• Cash and short term investments of ~$278.1 million at 9/30/2016
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Poised to Become a Registration Stage Company
ATA129 Entering Phase 3 in US, Registration in EU
2016
• Phase 3 designs agreed with FDA
for lead product candidate
• PRIME designation granted and
Scientific Advice received from
EMA and HTAs
• Transferred manufacturing
process from MSK to CMO
2017
• Commence Phase 3 trials in PTLD
• Preparing conditional marketing
authorization application in EU
• Develop commercial capabilities
• Advance
pipeline of allogeneic
4
cellular therapies
• Opened multicenter Expanded
Access Protocol (EAP) trial
4
Leading Allogeneic Cellular Therapy Platform
Breadth
Multiple validated
targets with strong
rationale for an
antigen’s role in
driving disease
Robustness
Utilizing healthy
donor derived cells
with complete
immune function
Discipline
Cells persist
untilhealthy
Utilizing
durabledonor
response
derived cells
achieved
and
with
complete
eventually
recede
immune
function
Precision
Product candidates
identify and
selectively kill
diseased cells
Efficiency, Safety and Convenience
•
•
•
•
•
Broad library of cell lines manufactured ahead of time and held as inventory
Proprietary algorithm matches cell lines to a patient’s unique immune profile
Cells are available in 3-5 days and ready-to-administer
Short IV infusion, no need for pre-treatment
Mechanism of action limits off target activity and potential for serious safety events
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Broad and Diverse Cellular Therapy Pipeline
Multiple Product Candidates in Development
Product Candidate (Indication)
Target
Phase 1
Phase 2
Phase 3
Registration
HEMATOLOGIC MALIGNANCIES
ATA129
(PTLD following HCT a)
EBV
MATCH trial
ATA129
(PTLD following SOT)
EBV
ALLELE trial
ATA129
(Expanded Access: PTLD, EBV+ cancers b)
EBV
ATA520
(Multiple Myeloma, Plasma Cell Leukemia)
WT1
EUEU
SOLID TUMORS
ATA129
(Nasopharyngeal carcinoma)
EBV
AUTOIMMUNE DISEASES
Autologous EBV-CTL
(Multiple Sclerosis c)
EBV
(Select Targeting)
INFECTIOUS DISEASES
ATA230
(Refractory CMV infection after HCT/SOT)
CMV
CMV = Cytomegalovirus
PTLD = Post transplant lymphoproliferative disorder
EBV = Epstein-Barr virus
SOT = Solid organ transplant
HCT = Allogeneic hematopoietic cell transplant WT1 = Wilms Tumor 1 antigen
a
Registration stage in EU, Phase 3 in US
Includes solid tumors
c In collaboration with QIMR Berghofer; Atara retains option to license
b
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EBV-SPECIFIC T CELL THERAPY FOR PTLD
REGISTRATION STAGE OPPORTUNITY
7
EBV-PTLD is an Aggressive Disease and Frequently Progresses
Despite Standard Therapy
Patient History
Progression Following Treatment with Rituximab
Week -10
Week 0
Week -4
Disease
progression
after 2 ritux
cycles
• 36 year old woman with
Fanconi anemia who
received an HCT
Disease
progression
after 3 ritux
cycles
New
• PTLD develops and
progresses despite
rituximab therapy
New
Complete Response (CR) Following Treatment with ATA129
After 2 Cycles
PR after 3 Cycles
CR after 4 Cycles
• Administered ATA129
• Complete response after
receipt of ATA129
• No treatment related
serious adverse events
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ATA129 has shown Durable Survival vs. Historical Data to Date
EBV-associated PTLD – Disease Overview
Indication
Standard First Line
Systemic Therapy
Expected Survival after
Rituximab Failure
EBV-PTLD after HCT
Rituximab monotherapy
16-56 Days
EBV-PTLD after SOT
Rituximab monotherapy; or
Rituximab + Chemotherapy
36% at 1-yr and 0% at 2-yr in
patients with high-risk disease
ATA 129 Phase 2 Efficacy and Safety Data: All Patients Failed Prior Rituximab
OS, EBV-PTLD after HCT
OS, EBV-PTLD after SOT
Safety Data
•
1-yr OS ~ 68% (N = 23)
Response rate > 60%
1-yr OS ~ 61% (N = 13)
Response rate > 50%
•
•
•
Few treatment-related
serious adverse events
9 ≥ grade 3 treatment
related SAE’s (n = 126)
No infusion related
toxicities, no CRS
One grade 1 GvHD
Prockop, S et al., Proc ASCO 2015; Choquet et al., 2007; Atara data on file; GvHD = graft vs. host disease; CRS = cytokine release syndrome; OS = Overall Survival
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Phase 3 Trial in EBV-PTLD after HCT (MATCH Trial)
Agreement Reached with FDA on Protocol Design
Enrollment
Treatment
• Primary Endpoint:
Objective Response Rate
15 + Sites in US and ex-US
Rituximab Refractory
EBV-PTLD after HCT
(N = ~35)
Follow Up
ATA 129 Cycles
2x106 cells / kg weekly
for three weeks followed
by 2 weeks rest
• Secondary Endpoints:
- Response duration
- Overall survival
- QOL
- Safety
- HEOR data
Expect to File BLA in Ritux-Refractory EBV-PTLD Following Trial Completion
QOL = Quality of Life; HEOR = Health economic outcomes research
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Phase 3 Trial in EBV-PTLD after SOT (ALLELE Trial)
Agreement Reached with FDA on Protocol Design
Enrollment
Treatment
Follow Up
15 + Sites in US and ex-US
Cohort 1
• Primary Endpoint:
Objective Response Rate
EBV-PTLD after SOT
after Failure of
Rituximab Monotherapy
(N = ~35)
Cohorts Enroll Concurrently
Not Comparative
Cohort 2
EBV-PTLD after SOT
after Failure of
Rituximab + Chemo
ATA 129 Cycles
2x106 cells / kg weekly
for three weeks followed
by 2 weeks rest
• Secondary Endpoints:
- Response duration
- Overall survival
- QOL
- Safety
- HEOR data
(N = ~35)
Expect to File BLA Based on First Cohort to Complete the Trial
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Plan to Submit Conditional Marketing Authorization
Application (CMA) in EU for ATA129
• Based on EMA Scientific Advice (SA), conducted under PRIME, expect to
file CMA in rituximab-refractory EBV-PTLD after HCT
• CMA will be based on data from prior Phase 1, 2 trials conducted at MSK
• Supported by available data from Phase 3 trials in EBV-PTLD after HCT
and SOT, which will be ongoing at the time of filing
• Participating in the SA meeting were Health Technology Assessment
agencies (HTA) representing England and Wales, Germany and France
• Submission activities underway with filing expected in 2018
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EBV-PTLD Presents an Attractive Market Opportunity in a
Condition with High Unmet Medical Need
2~# of Cases in US/EU Per Year
1,700
1.5-
1,400
• Initial addressable market:
Rituximab refractory PTLD
1-
• ~60-70% of rituximab treated
patients fail to respond or relapse
.5-
0-
• Occurs in ~ 2-20% of patients with
greatest rates occurring in settings
of highest immunosuppression
• Toxicity of chemotherapy can limit
its utility
EBV-PTLD after HCT
EBV-PTLD after SOT
Approximately $400-600 Million Opportunity in Ritux-Refractory EBV-PTLD
Department of Health and Human Services; European Society for Blood and Marrow Transplantation; Choquet et al., Blood 2006; Choquet et al., Ann Hematol 2007; Oertel
et al., Am J Transplant 2005; Uhlin et al. Haematologica 2014; OPTN/SRTR Annual Report; Ibrahim et al., Adv Hem 2012Redbook, Drug Prescribing Information; Based on
comparable novel oncology drug pricing assumptions
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EBV-SPECIFIC T CELL THERAPY FOR MS
EXPANSION OPPORTUNITY
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The Biologic Connection Between EBV and MS is Strong
Elimination of EBV+ B Cells has Potential to Treat MS
Growing Evidence Linking EBV and MS
•
•
•
•
EBV is present in nearly all multiple sclerosis (MS) patients
Clinical history of infectious mononucleosis increases the risk of MS several fold
EBV infected B cells are found at sites of MS lesions in the brain
High level of anti-EBV antibodies prior to onset increases risk of MS
EBV+ B cells in the Brain Catalyze Autoimmune Response and MS pathophysiology
Antigen Presentation
T-cell
Autoimmune Attack: Release
of Inflammatory Cytokines
Loss of Myelin
Latent EBV+
Memory B Cell
In CNS
Auto-Reactive
Antibody Production
Antibody Attack
© 2017, Atara Biotherapeutics, Inc. All rights reserved.
Ascherio and Munch 2000; Pakpoor et al 2013; Serafini et al 2013; Thacker et al. 2006; Sundström et al. 2004; Ascherio et. al, 2012; Levin et al.2005; Pender MP 2014
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Clinical POC for Autologous EBV-CTL in Progressive MS
A Durable Response Lasting More Than Three Years
Gd-Enhanced MRI
Baseline
CSF Analysis
Clinical Findings
• Single patient who
received 4 ascending
doses
• Reduction in fatigue,
painful lower limb
spasms
After EBV-CTL Therapy
• Improvement in
cognition, hand
function, and work
productivity
• No significant
adverse effects
Pender, M et al., Multiple Sclerosis Journal 2014; CA Neurology Assoc., 2016;
Gd = Gadolinium; CSF = Cerebrospinal fluid; IgG = Immunoglobulin G
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OTHER PIPELINE DEVELOPMENT
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Our Pipeline of Product Candidates Targeting Other Viral
and Cancer Antigens Will Continue to Advance in 2017
Product
Candidate
Target
Antigen
Indication
Stage
Next Steps
ATA 129
EBV
EBV+
cancer
Phase 2
Expanding EAP to other EBV + cancers
ATA 129
EBV
NPC
Phase 2
Ph 1/2 trial with checkpoint inhibitor
ATA 230
CMV
Refractory
CMV
Infection
Phase 2
Engage EMA
Finalize Phase 3 trial design
ATA 520
WT1
MM/PCL
Phase 1
Finalize clinical design
STM 434
Activin A
Ovarian
Cancer
Phase 1
Dose escalation complete
Evaluate results
Establish appropriate clinical next steps
ATA 621
BKV
BKV
Infection
Preclin
IND / CTN submission
ATA 368
HPV
Solid
Tumors
Preclin
IND / CTN submission
BKV = BK virus; CTN = Clinical Trial Notification; EAP = Expanded access protocol; HPV = Human papilloma virus; MM = multiple myeloma; PCL = plasma cell leukemia;
NPC = nasopharyngeal carcinoma
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Antiviral Resistant or Refractory CMV has High Mortality
ATA 230 has Shown Durable Survival vs. Historical Data to Date
Antiviral Resistant or Refractory CMV Infection after HCT – Disease Overview
Indication
Standard Antiviral Therapy
Survival after Antiviral
Resistance
Antiviral Refractory,
Resistant CMV after HCT
Ganciclovir, Foscarnet, Cidofovir
59-80% mortality
ATA 230 Efficacy, Safety Data: Antiviral Resistance Mutations, Median of 4 Prior Antivirals
Safety Data
•
•
•
Few treatment-related serious
adverse events;
16 ≥ grade 3 treatment related
SAEs among 66 patients
1 patient with de-novo GvHD
Prockop, S et al., Proc ASH 2016
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Chemotherapy Refractory NPC has a Poor Prognosis
Encouraging Signs of Clinical Activity with ATA129
EBV-associated Nasopharyngeal Carcinoma – Disease Overview
Indication
Standard First Line
Systemic Therapy
Expected Survival after
Failure of Chemotherapy
EBV+ NPC
Radiation Therapy;
Platinum-based chemotherapy
5-11 months
ATA 129 Efficacy, Safety Data: All Patients Failed 1-3 lines of Chemo for Metastatic Disease
OS, EBV+ NPC
1.0
Efficacy Data
0.6
•
0.4
1-yr & 2-yr OS
~ 84% (N = 14)
•
0.0
0.2
Probability of Survival
0.8
•
0
10
20
30
40
50
Response rates of ~20%
(2 PR + 1 CR)
11 of 14 patients alive with
median 18 month follow-up
EBV-CTLs expanded after
administration without
concomitant
lymphodepleting
chemotherapy
Safety Data
•
•
•
•
Few treatment-related
serious adverse events
9 ≥ grade 3 treatment
related SAE’s (n = 126)
No infusion related
toxicities, no CRS
One grade 1 GvHD
Months from Start of Treatment
Hsu et al., 2015; Ma, et al. 2008.; Prockop, S et al., Proc ASCO 2016
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2017: A PIVOTAL YEAR FOR ATARA
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Atara Bio: Multiple Milestones Expected in 2017
• Initiate two Phase 3 trials of ATA129 in rituximab refractory EBV-PTLD
• Broaden EAP trial for ATA129 to other EBV+ malignancies
• Diffuse large B-cell lymphoma, Hodgkin lymphoma, Burkitt lymphoma, NPC
• Present initial MS data from Phase 1 trial of autologous EBV-CTLs
• Initiate ATA188 Phase 1 trial in MS (allogeneic EBV-CTL)
• Initiate Phase 1/2 trial of ATA129 + checkpoint inhibitor in NPC
• Finalize ATA230 Phase 3 trial design in refractory CMV infection
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Leader in Allogeneic T cell Therapy
• Potential first-to-market allogeneic T cell therapy
Lead Product Candidate Entering Phase 3 Registration Trials
• Phase 3 designs agreed with FDA; Submitting for conditional approval in EU
Well-Capitalized; Highly Experienced Management Team
• Cash and short term investments of ~$278.1 million at 9/30/2016
23
Nasdaq: ATRA
Thank you. Q&A