RECODE READY TO 37th Annual J.P. Morgan Healthcare Conference - bluebird bio, Inc.
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READY TO
RECODE
37th Annual J.P. Morgan
Healthcare Conference
January 8, 2019 NASDAQ: BLUE
NASDAQ: BLUE 1
Forward-Looking Statements
These slides and the accompanying oral presentation contain forward-looking statements and information. The use
of words such as “may,” “might,” “will,” “should,” “expect,” “plan,” “anticipate,” “believe,” “estimate,” “project,” “intend,”
“future,” “potential,” or “continue,” and other similar expressions are intended to identify forward-looking
statements. For example, all statements we make regarding the initiation, timing, progress and results of our
preclinical and clinical studies and our research and development programs, our ability to advance product
candidates into, and successfully complete, clinical studies, and the timing or likelihood of regulatory filings and
approvals are forward looking. All forward-looking statements are based on estimates and assumptions by our
management that, although we believe to be reasonable, are inherently uncertain. All forward-looking statements are
subject to risks and uncertainties that may cause actual results to differ materially from those that we expected.
These statements are also subject to a number of material risks and uncertainties that are described in our most
recent quarterly report on Form 10-Q, as well as our subsequent filings with the Securities and Exchange
Commission. Any forward-looking statement speaks only as of the date on which it was made. We undertake no
obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future
events or otherwise, except as required by law.
NASDAQ: BLUE 2
WE RECODE FOR LIFE
RADICAL THIS IS PIONEERS
CARE PERSONAL WITH PURPOSE
We care in a way that’s intense Gene therapy is about saving lives We’re exploring new frontiers for
and truly sets us apart. one person at a time. And we are, the sake of patients.
each of us, personally all in.
NASDAQ: BLUE 3
We LIVE By Our Non-negotiables NASDAQ: BLUE 4
Our 2022 Vision -- Just Got BOLDER
LentiGlobin TDT Lenti-D CALD
2019 EU Potential Approval 2020 Potential Approval
2020 U.S. Potential Approval
THE GENE THERAPY
PRODUCTS COMPANY
LentiGlobin SCD ∞
Patient Impact
bb2121 Multiple Myeloma
2022 Potential Filing/Approval 2020 Potential Approval
4 Products
on the Market 5+ Clinical Programs 1-2 INDs per year starting in 2020
NASDAQ: BLUE 5
UNPRECEDENTED OPPORTUNITY
Anticipated research, development, regulatory and commercial milestones 2023+
2022 SCD:
• Potential Global
LAUNCH
2021
SCD: MM:
• U.S. & EU • Earlier Line
FILING/APPROVALS APPROVALS &
LAUNCHES
2020
TDT: MM:
• Label EXPANSION • Earlier Line Studies
TDT: CALD:
• EU Country EXPANSION • Global EXPANSION
• U.S. APPROVAL &
LAUNCH MM: 5+ New Clinical Programs
• Global EXPANSION in
2019
• Label EXPANSION
Relapsed/Refractory
CALD: • Earlier Line Studies
• U.S./EU APPROVAL 1-2 INDs
TDT:
• EU APPROVAL & MM:
1st LAUNCH
• APPROVAL in 1-2 INDs
Relapsed/Refractory
1-2 Clinical Starts 1-2 INDs
NASDAQ: BLUE 6
RECODE FOR LIFE
RECODE RECODE RECODE
THE SCIENCE THE SYSTEMS T H E S TAT U S Q U O
NASDAQ: BLUE 7
RECODE THE SCIENCE: R&D with SOUL
W H AT Y O U S E E W H AT Y O U D O N ’ T S E E
PRODUCT CANDIDATES PROGRAM AREA PRECLINICAL PHASE 1/2 PHASE 2/3 RIGHTS/PARTNER
Severe Genetic Diseases TGF-B
DARIC Flip
Lenti-D™
Drug Product
Cerebral Adrenoleukodystrophy Worldwide Controls
CBL-B
Transfusion-Dependent β-Thalassemia Non-β0/β0 Edit
P13K Inhibition
LentiGlobin™ Internal Research Enhanced Mftg
Transfusion-Dependent β-Thalassemia β0/β0 Worldwide
Drug Product
Sickle Cell Disease **
BCL11a
Sickle Cell Disease Worldwide
shRNA (miR)* GAMMA DELTA
Tools & Technology megaTALs
Cancer
T Cell
Multiple Myeloma Fourth Line
Multiple Myeloma Third Line**
bb2121 Celgene
Multiple Myeloma Second Line** Partners for Success
Multiple Myeloma First Line**
bb21217 Multiple Myeloma Fourth Line Celgene
*Development is led by Dana-Farber/Boston Children's Cancer and Blood Disorders Center
**Planned studies
NASDAQ: BLUE 8
Anti-Pure Play Principles - What Do We Mean?
RECODING TRADITIONAL R&D
INTEGRATE
INTERNAL Platform Tools MM - bb21217
& Technology 1:Many SCD - BCL11a
TDT – LentiGlobin
SCD – LentiGlobin
R&D Enablers Target Liquid Tumor Programs
MM – bb2121
Strategy Solid Tumor Programs
CALD – Lenti-D
EXTERNAL Clinical Insights SGD Programs
ITERATE
NASDAQ: BLUE 9Our Philosophy Applied in a Tumor Microenvironment NASDAQ: BLUE 10
Oncology Pipeline Enabled by Our Partners and Our Core Technologies
20+ 15+ 2+
Two Academic Clinical Trial
Starts Planned in 2019
NASDAQ: BLUE 11Research Strategy Yielding Emerging Oncology Pipeline
TA R G E T PRECLINICAL
Internal Program E A R LY C L I N I C A L L AT E C L I N I C A L
VA L I D AT I O N DEVELOPMENT
• Dual-targeted CAR UNC Collaboration
• Liquid tumor • CAR target
• Next gen + megaTAL edit • Solid tumors
Binders, Potency • Next gen + Synbio
Binders & Potency
bb2121 MM
bb21217 MM
Regeneron Collaboration
LentiGlobin TDT
• CAR target BCL11A SCD
• Solid tumor LentiGlobin SCD
• Next gen + Synbio
Target, Binders, Potency Lenti-D CALD
Medigene Collaboration Fred Hutch Collaboration
• TCR target • TCR target (viral antigen)
• Solid tumors • Solid tumors
• Next gen + Synbio • Next gen + megaTAL edit
Potency Potency
2020 & BEYOND: 1-2 INDs PER YEAR
NASDAQ: BLUE 12RECODE FOR LIFE
RECODE RECODE RECODE
THE SCIENCE THE SYSTEMS T H E S TAT U S Q U O
NASDAQ: BLUE 13RECODE THE SYSTEMS:
ANYTHING BUT TRADITIONAL
Novel Drug Product Bold & Balanced
Science/Medicine Production Access Model
Vector Supply Chain &
Production Patient Management
ONE-TIME
UNKNOWN
POTENTIALLY
IMPACT
O U R G O A L - M A K E I T S E A M L E S S T O A L L S TA K E H O L D E R S CURATIVE
NASDAQ: BLUE 14Platform Is Gearing Up for Launch
Virus Supply Chain
Manufacturing
D E L I V E R Y
P L A T F O M
S C A L A B L E
Collecting Cells,
Preparing,
Drug Product Shipping,
Receiving Cells
BLUE Style:
Field Team
MAKE IT SEAMLESS
Clinical Data
T D T
Real World
Evidence to
Support
NASDAQ: BLUE 15Preparing to Serve Patients in Europe in 2019
Apheresis Apheresis Cell Lab Transplant Transplant Transplant Transplant bbb Manufacturing
Coordinator Operator Operator Coordinator Nurse Physician Administrator Logistics
Apheresis Personnel Cell Lab Personnel Transplant Personnel (ATC) Manufacturer
Launch Expectations:
1 Drug Product Manufacturing
1. Optimal patient experience
through a seamless delivery Munich, Germany
network
&
2. Steady country by country
launch with progressive build 9 Qualified Treatment Centers
at 2019 Launch
3. Get the model right for long 3 - Germany
term success 4 - Italy
2 - UK
4. Advance value-based payment 4 - France (in 2020)*
over time reimbursement
NASDAQ: BLUE *Will support future launches in 2020+ 16RECODE FOR LIFE
RECODE RECODE RECODE
THE SCIENCE THE SYSTEMS T H E S TAT U S Q U O
NASDAQ: BLUE 17Business of Cures – Complex with Lots of Questions, Perspectives, etc.
Value? Cost Effectiveness?
“How much would
Price?
“Can Pay-For-
you pay for the
Results Drug
miracle of gene
Affordability?
Pricing Work?”
therapy?”
One-Time?
Value-based Payments?
“Value-Based Pricing
COGs?
“Gene therapy
And Reimbursement: could be cost
Price Increases?
Still More Promise effective in SMA,
Than Reality”
Revenue?
but not at $4M”
NASDAQ: BLUE 18bluebird Access Strategy – Proactive and Transparent Approach
“Biotech Proposes Paying for Pricey
Drugs by Installment”
NASDAQ: BLUE 19Access and Reimbursement Model – A Major Opportunity Across Stakeholders
System NOT setup for one-time potentially curative treatments BLUEBIRD
OPPORTUNITY
Focus is on price, NOT on value • The Will To Recode
• One-Time Treatment
Focus is on difficulties and barriers, NOT solutions • Potentially Curative
• Product Engine
Growing alignment on value & outcome based payment models • Unconstrained
NASDAQ: BLUE 20Keep It Simple, Keep It Focused on the Patient & the Long Term
Unapologetically fund & reward
B L U E VA L U E P R I N C I P L E S
innovation that matters
Focus on patient innovation and access
&
Focus on real value delivered
to the patient & system
Creative and disruptive
Flexible and share risk
Don’t truncate value because
it’s a one-time potentially
Transparent, proud and proactive curative treatment
Don’t price at what you
Don’t do silly short sighted stuff can get away with or what the
market can bear
NASDAQ: BLUE 21Our Goal Is to Simultaneously Achieve Five Key Objectives
BLUE has CONVICTION in the VALUE of the LIFELONG transformative benefit that our
1 therapies may bring to patients, physicians, caregivers, healthcare systems and society at large
2 BLUE is willing to SHARE the RISK of uncertainty to PROVE the life long value of its therapies
3
BLUE wants to make its treatments AFFORDABLE so PATIENTS (and SYSTEM) can actually
REALIZE the VALUE of its therapies
4
BLUE wants to be a CATALYST for CHANGE to establish a SUSTAINABLE MODEL for
5 pricing & reimbursement of gene therapies
NASDAQ: BLUE 22Approach – VALUE-BASED PAYMENT Over Time Based on OUTCOME
OBJECTIVE S T R AT E G I C A P P R O A C H
1
FAIR VALUE Lifetime cost-time effectiveness timeframe
RECOGNITION Base value only on patient QOL and Life Extension
2
SHARED Pay ONLY IF the treatment works
RISK Put UP TO 80% of the price at risk based on success
3
PER PATIENT Spread payments over UP TO A FIVE YEAR period
AFFORDABILITY NO PRICE INCREASES above CPI
4
HEALTH SYSTEM
AFFORDABILITY
NO COST after payment period (vs. for life)
NASDAQ: BLUE 23Payment Model – Patient and System Friendly
T R A D I T I O N A L F O R L I F E M O D E L *
Years 1 2 3 4 5 6 7 8 9 10
$300 - $300 - $300 - $300 - $300 - $300 - $300 - $300 - $300 - $300 -
$600k $600k $600k $600k $600k $600k $600k $600k $600k $600k
Lifetime (50 years)
Treatment Costs
$1.5M - $3M $3M - $5M $13M - $29.5M
Over Time
P O T E N T I A L B L U E B I R D A P P R O A C H
Years 1 2 3 4 5
X X X X X NO COST
Only with Success
No price increase beyond CPI
*Ranges based on 2018 U.S. list prices from a sampling of seven chronic rare disease therapies.
NASDAQ: BLUE 24Payment Model – Sharing Risk & Value with the System
Spread payments over UP TO FIVE years No payments after payment period
P O T E N T I A L B L U E A P P R O A C H
Years 1 2 3 4 5
X X X X X NO COST
Only with Success
No price increase beyond CPI
UP TO 80% of payment only made Proactively commit to
upon treatment success NO PRICE INCREASES over CPI
NASDAQ: BLUE 25Not the Easy Path but the Challenges Are Solvable
• Challenge: How do you define and track
outcomes?
TRACKABILITY?
• Possible Solutions: Payer to use claims
and/or registry data
Spread payments over UP TO FIVE Years No payments after payment period
P O T E N T I A L B L U E A P P R O A C H • Challenge: What happens when patient
Yea
rs
1 2 3 4 5 changes payer?
X X X X X NO COST
PORTABILITY? • Possible Solutions: Address in contract
Only with Success
negotiation, mutual recognition strategy across
payers, etc.
No price increase beyond CPI
UP TO 80% of payment only made Proactively commit to NO PRICE
upon treatment success INCREASES over CPI
• Challenge: How do you handle Medicaid “Best
Price” rules?
BEST PRICE?
• Possible Solutions: Innovative federal or state
pilot programs or waivers
NASDAQ: BLUE 26VALUE-BASED PAYMENT Over Time Based on OUTCOMES
OBJECTIVE S T R AT E G I C A P P R O A C H
1
FAIR VALUE Lifetime cost-time effectiveness timeframe
RECOGNITION Base value only on patient QOL and Life Extension
What is the value that LentiGlobin can bring to
TDT patients, payers, system and society?
AND how can it be fairly measured?
NASDAQ: BLUE 27What Value Can LentiGlobin Bring to TDT Patients, Payers and System?
Disease and treatment-related burden builds up over LentiGlobin, if successful, has the potential to relieve
time and can even lead to early death patients of their daily disease and treatment-related
burden, and patients may live a longer life
Pain
Iron
Fatigue
Overload
Organ
Depression QUALITY OF LIFE LIFE
Failure
IMPROVEMENTS EXTENSION
Impact of One-Time
Time Lost Potentially Curative
Cost of
due to
Transfusion
Chelation Treatment?
SOCIETAL COST OF CARE
BENEFITS REDUCTIONS
Inability to Cost & Time
Work for Care
Tethered to Impact on
Healthcare the Blood
System Supply
NASDAQ: BLUE 28Cost Effective Analysis Focused on Actual Patient Value:
QOL and Life Extension
T H E VA L U E AT W H I C H T R E AT M E N T I S C O S T E F F E C T I V E * ( N O T P R I C E )
Traditional LentiGlobin
All Inclusive Calculation Intrinsic Value
$4M Societal Value
Cost Offsets (Returned To
• Clinical Trial Data $3M The System)
(U.S./EU Vary)
QUALITY OF LIFE LIFE
• Outcomes Research
IMPROVEMENTS EXTENSION
$2M $2M
Life Extension Life Extension
• Real World Evidence $1M $1M
$2.1M
SOCIETAL COST OF CARE
BENEFITS REDUCTIONS
Quality of Life Quality of Life
• Economic Modeling
$0 $0
The actual LentiGlobin price is TBD, but will not exceed the intrinsic value
(total value minus cost offsets).
*We have quantified the impact on patient quality of life, survival,
NASDAQ: BLUE treatment cost and society using established modeling techniques 29Catalyst for Change - Recode the Status Quo for the Better
Value-based payment over time tied to outcomes
Base value only on actual patient value (no offsets)
CATALYST FOR
Payments over ~5 years with up to 80% at risk 5 CHANGE
No price increases beyond CPI
Actual price TBD, but will not exceed intrinsic value
NASDAQ: BLUE 30WE RECODE FOR LIFE Our ambition is to recode science, systems and the status quo, so lives can be lived fully. NASDAQ: BLUE 31
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