Providing Hope to the Underserved - March 16, 2021 - Global Blood Therapeutics, Inc.
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Providing Hope to the Underserved March 16, 2021
SAFE HARBOR STATEMENT
Statements we make in this presentation may include statements that are not historical facts and are considered forward-looking statements within the meaning of
Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended (collectively, the “Acts”). We intend these
forward-looking statements, including statements regarding our mission, vision, goals, plans, milestones, strategy, positioning and future activities, achievements and
impact, the safety, efficacy, mechanism of action, other product characteristics, availability, use, commercialization and commercial and therapeutic potential of Oxbryta®
(voxelotor), including the potential to reduce morbidity and mortality, to be a standard of care and disease-modifying therapy, to address top priorities, transforming the
treatment paradigm, and the significance of reducing hemolysis and increasing hemoglobin, Oxbryta awareness and education, the impact of the COVID-19 pandemic
and our related response and expectations, the commercial supply of Oxbryta, the availability, use and impact of GBT Source ®, payer coverage, implementing and
completing clinical development plans, generating and reporting data and analyses from past, ongoing and potential future studies, inferences drawn from studies and
related analyses, regulatory review, our manufacturing and commercial infrastructure, our R&D pipeline, the attributes, potential and future development of drug
candidates, actual and potential partnerships and distribution arrangements, expanding access to Oxbryta for patients in the U.S. and globally, our financial position,
guidance and expectations, and intellectual property rights, to be covered by the safe harbor provisions for forward-looking statements contained in the Acts and are
making this statement for purposes of complying with those safe harbor provisions. These forward-looking statements reflect our views as of the time made about our
plans, intentions, expectations, strategies and prospects, which are based on the information then available to us and on assumptions we have made. We can give no
assurance that the plans, intentions, expectations or strategies will be attained or achieved, and, furthermore, actual results may differ materially from those described in
the forward-looking statements and will be affected by a variety of risks and factors that are beyond our control, including, without limitation, risks and uncertainties
relating to the COVID-19 pandemic, including the extent and duration of the impact on our business, the risks that we are continuing to establish our commercialization
capabilities and may not be able to successfully commercialize Oxbryta, risks associated with our dependence on third parties for development, manufacture,
distribution and commercialization activities related to Oxbryta, government and third-party payer actions, including relating to reimbursement and pricing, risks and
uncertainties relating to competitive products and other changes that may limit demand for Oxbryta, the risks regulatory authorities may require additional studies or data
to support continued commercialization of Oxbryta, the risks that drug-related adverse events may be observed during commercialization or clinical development, and
that data and results may not meet regulatory requirements or otherwise be sufficient for further development, regulatory review or approval, compliance with obligations
under the Pharmakon loan, progress under our distribution agreement for select Middle East countries, and progress of our collaboration with Syros, along with those
risks set forth in our Annual Report on Form 10-K for the fiscal year ended December 31, 2020, filed with the U.S. Securities and Exchange Commission, as well as
discussions of potential risks, uncertainties and other important factors in our subsequent filings with the U.S. Securities and Exchange Commission. Except as required
by law, we assume no obligation to update publicly any forward-looking statements, whether as a result of new information, future events or otherwise.
© Global Blood Therapeutics, Inc. 2021
2
LIVING
OUR
MISSION
GBT discovers,
develops and delivers life-
changing treatments for
people living with grievous
blood-based disorders,
starting with sickle cell
disease (SCD).
© Global Blood Therapeutics, Inc. 2021
3
OUR LONG-TERM VISION
Leader in SCD and
Other Underserved
Orphan Disease
Communities
3
Leverage Capabilities
to Expand Beyond SCD
Value
2 Advance SCD Pipeline
1 Establish Oxbryta as
Standard of Care
Time
© Global Blood Therapeutics, Inc. 2021
4
FOCUS ON NEAR-TERM GROWTH
Where We Are Today Our Near-Term Goals
Successful Oxbryta launch, despite Build on Oxbryta momentum with
COVID-19 pandemic labeling and geographic expansion
Advancing pipeline of potential Initiate 2 inclacumab pivotal trials mid-2021
best-in-class SCD therapies Deliver GBT601 POC data by year end
Pursue investment opportunities
Solid balance sheet with $560.9M1
to drive growth
SCD, sickle cell disease; POC, proof of concept.
1. Cash, cash equivalents, and marketable securities as of December 31, 2020.
© Global Blood Therapeutics, Inc. 2021
5
SCD: AN UNDERSERVED ORPHAN CONDITION
SCD: AN URGENT UNMET NEED
Lifelong inherited blood disorder
Hb polymerization causes / leads to:
Multi-organ morbidity1
~30 year reduction in life expectancy2
Historically limited treatment options
Drug development was focused on acute pain crisis
(VOCs), which impact less than 50% of the patients3
Underserved patient population
>350K patients in U.S., Europe, Middle East and Latin America4
Millions worldwide, including low-resource countries
Deej, at age 13
Hb, hemoglobin; VOC, vaso-occlusive crisis.
1. Data on file. 2. Akinsheye, I. et al. Fetal hemoglobin in sickle cell anemia. Blood. 2011. 118:19-27. 3. Shah, N. et al. Sickle Cell Disease Complications: Prevalence and Resource Utilization. Plos One. 2019.
4. Population data: Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed February 24, 2021; European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed February 24, 2021. Data on file.
© Global Blood Therapeutics, Inc. 2021
7
HbS POLYMERIZATION IS THE ROOT PROBLEM IN SCD
HbS
Polymerization
Hemolytic Anemia Vaso-occlusion
Pain / Vaso-occlusive
Organ Damage Fatigue Organ Damage
crisis (VOC)
Stroke Osteonecrosis
Renal Failure Retinopathy
Pulmonary Hypertension
Priapism
Leg Ulcers
Mortality
HbS, sickle hemoglobin.
Adapted from Eaton, W. and Bunn, F. Targeting HbS Polymerization. Blood. 2017.
© Global Blood Therapeutics, Inc. 2021
8
MULTI-ORGAN DYSFUNCTION IN SCD IS LINKED TO
CHRONIC ANEMIA AND HEMOLYSIS
Chronic Organ Damage:
Brain Leading Cause of Death in Adults1
Stroke
Silent cerebral infarct
Neurocognitive impairment Other
Heart (13%)
Cardiomyopathy
Lungs
Pulmonary Unknown
hypertension (8%) Irreversible Organ
Liver/gallbladder Damage (42%)
Hepatopathy Trauma (Lung, Kidney,
Kidney Gallstones (8%) and/or Liver)
Renal insufficiency
Renal failure Infection
(5%)
Acute
Pulmonary
Skin GU Disorders Stroke
Leg ulcers Priapism (11%) (13%)
≥ 20 years of age, n=186
Image adapted from Kato GJ et al. Nat Rev Dis Primers. 2018;4:18010.
1. Powars, DR et al. Medicine. 2005;84:363–376.
© Global Blood Therapeutics, Inc. 2021
9
MAJOR BURDEN ON U.S. PATIENTS AND SOCIETY
Up to $286,000 ~$700,000 in lost
annually in cost of lifetime income
medical care1 per patient3
End-organ damage Major caregiver
drives major productivity impact;
healthcare utilization, often devastating
average of 30-54 financial burden
days/year2
1. Song, X, et al. Economic Burden of End Organ Damage Among Patients with Sickle Cell Disease in the US. 2019 American Society of Hematology Annual Meeting. Poster #3388. 2. GBT Internal Data.
3. Lubeck, D. et al. Estimated Life Expectancy and Income of Patients With Sickle Cell Disease Compared With Those Without Sickle Cell Disease. JAMA Netw. Open. 2019 Nov 1;2(11):e1915374.
© Global Blood Therapeutics, Inc. 2021
10OXBRYTA: FIRST-IN-CLASS SCD THERAPY
OXBRYTA ATTACKS THE ROOT CAUSE OF SCD
Increases oxygen
Once-daily, oral Binding to Hb stabilizes Inhibits HbS
affinity safely to create
treatment the oxyHb (R) state1 polymerization3
non-sickling Hb2
100 1.0
HbS polymerization
(Optical Density)
80 0.8
% O2 Saturation
60 0.6
40 0.4
20 0.2
0 0.0
0 20 40 60 80 100 0 10 20 30 40 50
pO2 (mm Hg) Time to Polymerization (min)
Oxbryta Oxbryta Healthy No drug, Oxbryta,
No drug Oxbryta
subject SCD subject SCD subject
oxyHb, oxygenated hemoglobin; Hb, hemoglobin; HbS, sickle hemoglobin.
1. Adapted from Bunn and Eaton, Blood. 2017. 2. Hutchaleelaha, A. et al., British Journal of Clinical Pharmacology. 2019. 3. Oksenberg , D. et al., Br J. Haematol. 2016.
© Global Blood Therapeutics, Inc. 2021
12OXBRYTA IMPACTS RBCs RAPIDLY
Pre-Treatment Day 21 of Treatment
Source: Patient Perception of Oxbryta Treatment Benefit, ASH 2020 Poster #1723. Typical peripheral blood smear. Before and after Oxbryta treatment.
© Global Blood Therapeutics, Inc. 2021
13HOPE STUDY: DURABLE IMPROVEMENTS AT 72 WEEKS
Nearly 90% of Patients Achieve Significant Hb Increase (>1 g/dl)
100.0
88.9
80.0
Percentage of patients, (95% CI)
58.9
60.0
40.0
25.0
20.0
20.0
3.3
0
0.0
>3 g/dL >2 g/dL >1 g/dL
Voxelotor 1500 mg Placebo
Source: Long-Term Efficacy and Safety of Voxelotor in Adolescents and Adults with Sickle Cell Disease: HOPE Trial 72-Week Analysis, ASH 2020 Poster #1716
© Global Blood Therapeutics, Inc. 2021
14PATIENTS AND HCPs REPORT PATIENT IMPROVEMENT –
AT VARYING LEVELS OF Hb INCREASE
Patient and Clinician Assessment Hemoglobin Change and
Improvement with Oxbryta Therapy Clinical Improvement
60% n=23 60% Patients Assessed as
Greatly Improved or Improved1
50% 50%
n=16
40% 40%
Percentage of Patients
30% 30%
20% 20%
10% 10%
0% 0%
Greatly Improved A Little No A Little Much < 0 g/dL 0-1 g/dL > 1 g/dL
Improved Improved Change Worse Worse
Patient (PGI-I) Clinician (CGI-I)
1.The remaining 19% of patients measured rated as a little improved or no change (one patent of 0-1 g/dL improvement and 2 patients with >1 g/dl improvement).
Source: Patient Perception of Oxbryta Treatment Benefit, ASH 2020 Poster #1723.
© Global Blood Therapeutics, Inc. 2021
15VOCs LOWEST IN PATIENTS ACHIEVING HIGHEST
Hb LEVELS
4
MCF of VOC incidence during treatment period
Placebo (n=91)
Hb 5.9 toDEMONSTRATING CLINICAL BENEFIT IN LEG ULCERS1
Post-Hoc Analysis of HOPE Study % Patients with Leg Ulcers that
Published in American Journal of Hematology Resolved or Improved by Week 24 & 72
>90% voxelotor patients had leg ulcer
improvement/resolution by week 72
100% voxelotor 1500 mg patients had leg ulcer
resolution by week 72
75% voxelotor 1500 mg patients had leg ulcer
resolution by week 24
1. The American Journal of Hematology post hoc analysis valuating the incidence of leg ulcers and outcomes in patients enrolled in the HOPE trial across the 72-week treatment period. Published January 2021.
© Global Blood Therapeutics, Inc. 2021
17OXBRYTA CAN IMPROVE SCD PATIENT LIVES
…I feel like I’m more able to be the …I feel like I’m more able to help …I feel like I’m able to do more
parent and the person that I want take care of my kids and my with my family and friends.
to be. family.
Lakesha D. Muyiwa S. Michelle P.
© Global Blood Therapeutics, Inc. 2021 Individual patient results may vary. 18DELIVERING FOR PATIENTS
First-in-class disease modifying therapy approved to treat SCD patients ages 12+
~5,000 ~1,365 ~90% $123.8M
new unique of covered lives, 2020 revenue
prescriptions1 prescribers1 broad payer
coverage
Launch progress despite headwinds from COVID-19
FDA approval on November 25, 2019. As of December 31, 2020 unless otherwise stated.
1. From launch through December 31, 2020.
© Global Blood Therapeutics, Inc. 2021
19TARGETING HCPs AND KOLs
17 states represent ~85% of
SCD patients
~60 sickle cell therapeutic specialists
targeting ~5,000 HCPs
12 medical science liaisons targeting
the top 500 KOLs
KOL, key opinion leader.
Symphony Health Claims Data, 2012-2018.
© Global Blood Therapeutics, Inc. 2021
20ADAPTING TO COVID-19 ENVIRONMENT
COVID-19 Impact is Real How GBT is Adapting
SCD patients at increased risk of severe Encouraging increased telemedicine adoption
illness and death from COVID-191
Enhancing capabilities for virtual field
HCP/patient interactions down significantly engagements
from pre-pandemic averages
Executing in-person field engagements,
Industry-wide, HCPs often less comfortable wherever appropriate
initiating new therapies virtually
Increasing HCP/patient education and
Significantly reduced in-person field real-world evidence
engagements
Direct correlation between COVID-19 cases Augmenting patient support services and
and new Rx’s of Oxbryta patient communication
Rx, prescription.
1. Centers for Disease Control and Prevention (CDC)
© Global Blood Therapeutics, Inc. 2021
21GBT SOURCE PROVIDES HIGH-TOUCH PATIENT SUPPORT
Home Delivery (office, school)
Product &
Services
Education
Disease Adherence
Rx Rx
Education & Refill
Support
Patient HCPs Specialty
Pharmacy
Network
Reimbursement Financial &
Assistance Copay
Support
Payers
© Global Blood Therapeutics, Inc. 2021
22ENHANCING STRATEGIES TO DRIVE INCREASED ADOPTION
Access Specialty Sales
Navigators Pharmacy Teams
Network
High-touch patient Reimbursement Resource for patients HCP engagement
engagement support
Schedule refills New education,
Nurse support on Patient follow-ups and marketing materials
adherence reminders Access to pharmacist
Weekly updates for HCP and patient
HCPs education
© Global Blood Therapeutics, Inc. 2021
23DEPLOYING NEW AND UPDATED EDUCATION MATERIALS
© Global Blood Therapeutics, Inc. 2021
24WE BELIEVE OXBRYTA WILL BECOME STANDARD OF CARE
Opportunity to Reach >350K Patients by 2022
U.S. Latin America Europe Middle East
Current Oxbryta label
86K Patients Age 12+ 100K Patients 52K Patients 100K Patients
17K Age 4-11 Seeking to partner with MAA under review to treat Partnered with distributor
distributor for Brazil hemolytic anemia in SCD for six GCC countries
patients 12+ years old
Planning to file regulatory
application to expand
label to ages 4 to 11
= 10K patients
Population data: Centers for Disease Control and Prevention website. Sickle Cell Disease (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed February 24, 2021; European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed February 24, 2021. Data on file.
© Global Blood Therapeutics, Inc. 2021
25ADVANCING THE
-
GBT PIPELINE
-
26GBT PIPELINE TARGETS SCD VIA MULTIPLE APPROACHES
Program Preclinical Phase 1 Phase 2 Phase 3 Commercial
Treatment of SCD
Oxbryta (voxelotor)
HbS Polymerization Inhibitor Ongoing studies: • HOPE-KIDS 1 study • ActIVe study
• HOPE-KIDS 2 TCD post-approval study
Inclacumab Chronic VOC prevention
Goal: 1H 2021 Phase 3 initiation (two studies)
P-Selectin Inhibitor
Acute VOC re-admission prevention
GBT601
Next-Generation HbS Treatment of SCD Goals: 1H 2021 enter the clinic, POC data by end of 2021
Polymerization Inhibitor
HbF Induction
(Syros Partnership)
Anti-sickling
(Sanofi In-license)
Inflammation & Oxidative
Stress Reduction
(Sanofi In-license)
POC, proof of concept; HbF, fetal hemoglobin.
© Global Blood Therapeutics, Inc. 2021
27OXBRYTA STUDIES: DEMONSTRATING EFFECT ON
MULTIPLE ORGANS
Planned Sponsored and Investigator-Initiated Organ Damage in SCD Patients Related
Studies of Oxbryta (voxelotor) to Hemolytic Anemia
Investigator Initiated:
Chronic kidney disease Brain
Stroke
Organ damage (brain/cardiac/kidney) Silent cerebral infarct
Voxelotor-MRI study Neurocognitive impairment
Lungs
GBT-Sponsored: Pulmonary
Heart
Cardiomyopathy
HOPE-KIDS 2: TCD confirmatory study hypertension
Physical activity (ActIVe Ph4 study)
Kidney Liver/gallbladder
Renal insufficiency Hepatopathy
Planned Investigator-Initiated Studies of Renal failure Gallstones
Neurological Complications of SCD
Stroke epidemiology in adults with SCD Skin GU
Leg ulcers Priapism
Prevalence and short-term incidence of neurological morbidity
MRI, magnetic resonance imaging; TCD, transcranial doppler.
Image adapted from Kato GJ et al. Nat Rev Dis Primers. 2018;4:18010.
© Global Blood Therapeutics, Inc. 2021
28INCLACUMAB: BEST-IN-CLASS POTENTIAL FOR VOCs
Patients with SCD
P-selectin
Binding site on P-selectin
PLA inhibition, %
Concentration, µg/mL
Goal: Quarterly infusion dosing
PSGL-1 Inclacumab Crizanlizumab
Encouraging safety
Inclacumab more closely mimics the natural binding site on P-selectin (>700 patients in non-SCD studies)
VOC, vaso-occlusive crisis.
Source: Inclacumab, a Fully Human Anti-P-selectin Antibody, Directly Binds to PSGL-1 Binding Region and Demonstrates Robust and Durable Inhibition of Cell Adhesion. ASH 2020 Poster #1707.
© Global Blood Therapeutics, Inc. 2021
29VISION IS TO OPTIMIZE PATIENT OPTIONS AND INCREASE
ADDRESSABLE MARKET
Home Physician Office Hospital (Re-)Admission
SCD is the leading
cause of 30-Day
re-admission rates1
Goals: Quarterly Dosing
• Improve health Lower VOC frequency
• Maintain daily activities
On-Demand Dosing
• Reduce healthcare
utilization & cost Reduce re-admissions
1. SCD readmissions / number of index stays: Elixhauser A and Steiner C, HCUP Statistical Brief #153, April 2013.
Source: Inclacumab, a Fully Human Anti-P-selectin Antibody, Directly Binds to PSGL-1 Binding Region and Demonstrates Robust and Durable Inhibition of Cell Adhesion. ASH 2020 Poster #1707.
© Global Blood Therapeutics, Inc. 2021
30PHASE 3 PROGRAM WILL STUDY THE VOC CONTINUUM
Chronic Prevention Protocol Acute Re-Admission Protocol
N = 280
N = 240
Primary Endpoint: Proportion of participants
Primary Endpoint: VOC rate during
with at least 1 re-admission for VOC within
48-week treatment period
90 days of hospitalization for VOC
Open-Label Extension Protocol
VOC, vaso-occlusive crisis.
Source: Inclacumab, a Fully Human Anti-P-selectin Antibody, Directly Binds to PSGL-1 Binding Region and Demonstrates Robust and Durable Inhibition of Cell Adhesion. ASH 2020 Poster #1707.
© Global Blood Therapeutics, Inc. 2021
31GBT601: POTENTIAL FUNCTIONAL CURE FOR SCD
Next-Generation HbS Polymerization Inhibitor Improved RBC Health & Survival
RBC Survival
10
Potential benefits include:
• Normalized Hb 8
RBC half-life (d)
• Improved RBC survival, health and organ 6
function
4
• One pill per day
2
• Functional cure as single agent
0
Goal to advance into the clinic with SCD vehicle 20 40 75 150
patients by 1H 2021 Dose (mg/kg)
HbS, sickle hemoglobin; RBC, red blood cell.
Source: GBT021601 Inhibits HbS Polymerization, Prevents RBC Sickling and Improves the Pathophysiology of Sickle Cell Disease in a Murine Model. ASH 2020 Poster #1704.
© Global Blood Therapeutics, Inc. 2021
32THOUGHTFUL & SUSTAINABLE APPROACH TO ACHIEVING
WORLDWIDE ACCESS OVER TIME
Execute Expand Advance Develop
on U.S. launch U.S. label and pipeline and distribution and
of Oxbryta secure ex-U.S. continue investing funding approaches
approvals in innovation in sub-Saharan
Africa and India
© Global Blood Therapeutics, Inc. 2021
33UPCOMING MILESTONES
20211 By Mid 2021 By Mid 2021 Late 2021 Q1-Q2 2022 2021-20232
First patients File regulatory Initiate Deliver GBT601 MAA approval Oxbryta Middle
enrolled in application with inclacumab POC data from EMA for East approvals
HOPE-KIDS 2 & FDA to expand pivotal studies & Oxbryta for ages
ActIVe Studies Oxbryta label to GBT601 clinical 12 and up
ages 4 to 11 trial in SCD
Oxbryta U.S. Commercialization
FDA, Food & Drug Administration; POC, proof of concept; MAA, marketing authorization application; EMA, European Medicines Agency.
1. Subject to Covid-19 pandemic. 2. Projected approvals.
© Global Blood Therapeutics, Inc. 2021
34OUR LONG-TERM VISION
Leader in SCD and
Other Underserved
Orphan Disease
Communities
3
Leverage Capabilities to
Expand Beyond SCD
• Benign hematology
Advance SCD Pipeline • Orphan diseases
Value
2
• Inclacumab
• GBT601
1 Establish Oxbryta as SOC • HbF inducers
• More-real world experience • Novel targets
• Label expansion
• Global launches
• Access in low resource countries
Time
SOC, standard of care.
© Global Blood Therapeutics, Inc. 2021
35Thank You
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