Pfizer Rare Disease here to listen, learn and make a difference
Michael
Duchenne Muscular Dystrophy
Amelia
Growth Hormone Deficiency Kyle
Sickle Cell Disease
Ian
Growth Hormone Deficiency Kyle
Friedreich’s Ataxia
Carolyn
Acromegaly
Curt
Hemophilia B
Amiya
Sickle Cell Disease
Gary
TTR-FAP
Emily
Cystic Fibrosis
Amy
Growth Hormone Deficiency Michael
Duchenne Muscular Dystrophy
Amelia
Growth Hormone Deficiency Kyle
Sickle Cell Disease
Susan
Acromegaly
Tiahna
Sickle Cell Disease
Ian
Growth Hormone Deficiency Kyle
Friedreich’s Ataxia
Cyndi
Gaucher Disease
Carolyn
Acromegaly
Matt
Friedreich’s Ataxia
Curt
Hemophilia B
Gabe
Hemophilia A Amiya
Sickle Cell Disease
Gary
TTR-FAP
David
TTR-FAP
Confidential. Internal Use Only.
Amy
Growth Hormone Deficiency Michael
Duchenne Muscular Dystrophy
Amelia
Growth Hormone Deficiency Kyle
Sickle Cell Disease
Susan
Acromegaly
Tiahna
Sickle Cell Disease
Ian
Growth Hormone Deficiency Kyle
Friedreich’s Ataxia
Cyndi
Gaucher Disease
Carolyn
Acromegaly
Matt
Friedreich’s Ataxia
Curt
Hemophilia B
Gabe
Hemophilia A Amiya
Sickle Cell Disease
Gary
TTR-FAP
David
TTR-FAP
1
Sonal Bhatia, MD
Vice President
North America Rare Disease Medical Affairs Lead
February 13, 2018
• Global footprint & investment
• Around 70 scientists working to find
treatments
• Over 20 compounds in pre-clinical and
clinical development
• Late-stage pipeline opportunities
• Investigational one time gene therapy
treatments
Our Commitment
Michael | DMD Amiya | SCD
Scientists | Pfizer
2
3
Pfizer’s Global Rare Disease Portfolio*
Approximately 300,000 infants worldwide are born each year with sickle cell disease
1. Piel FB, et al. NEJM. 2017;376:1561-1573. 2. Hassell KL. Am J Prev Med. 2010;38(4S):S512-S521. 3. SicklecellNA.org.2017
Sickle Cell Disease (SCD): The Global Presence
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~100,000 Americans have SCD and 2% to 10% are Hispanic (~2,500–10,000)
~2 million Americans are carriers of the sick cell trait
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SCD patient in VOC
presents in ED Subject receives supportive
care (IV hydration, opioids)
from ER assessment to
discharge
Pivotal Phase 3 study in patients with SCD experiencing Vaso-Occlusive Crisis (VOC)
• N=350, randomized, double blind, placebo controlled; approximately 70 centers US & Canada
• Study population: 6 years of age; VOC requiring hospitalization
• Primary Endpoint: Readiness for discharge
• Secondary Endpoints: Time to stopping IV opioids; cumulative IV opioid consumption
Open-label extension study
• N=150
• Study population: Subjects who complete the double-blind study and meet other qualification criteria
• Objective: Evaluate the safety of rivipansel when used to treat >1 episode of VOC.
Safety follow-
up 35-day
post last dose
Placebo
Rivipansel DIS
CH
AR
GE
DE
CIS
ION
TO
AD
MIT
1:1
RA
ND
OM
IZA
TIO
N
Open Label
Study
Phase 3 Program
Empowers Patients to Self Advocate thereby Elevating the Care Paradigm
rareCourage Social Wall
Integrating vetted disease content into members’ conversations
Curation Working Group Guides curation of credible content to address knowledge gaps
Community Managers Engages patients & advocates to utilize oneSCDvoice by providing tips & timely connections to content
rareCurate Knowledge
Access to vetted disease content
Engagement Working Group Drives awareness & connectivity to patients engaging local advocacy networks
Platform Developed With The Community and For The Community
rareClinical trials
Education for drug development and diversity in clinical trials
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Harmonizing and Empowering a Fragmented Patient Community
7
Partner Organizations
Pfizer collaborates with many groups and organizations both through and beyond
“Council For Change”
Pfizer Rare Disease here to listen, learn and make a difference
Michael
Duchenne Muscular Dystrophy
Amelia
Growth Hormone Deficiency Kyle
Sickle Cell Disease
Ian
Growth Hormone Deficiency Kyle
Friedreich’s Ataxia
Carolyn
Acromegaly
Curt
Hemophilia B
Amiya
Sickle Cell Disease
Gary
TTR-FAP
Emily
Cystic Fibrosis
Amy
Growth Hormone Deficiency Michael
Duchenne Muscular Dystrophy
Amelia
Growth Hormone Deficiency Kyle
Sickle Cell Disease
Susan
Acromegaly
Tiahna
Sickle Cell Disease
Ian
Growth Hormone Deficiency Kyle
Friedreich’s Ataxia
Cyndi
Gaucher Disease
Carolyn
Acromegaly
Matt
Friedreich’s Ataxia
Curt
Hemophilia B
Gabe
Hemophilia A Amiya
Sickle Cell Disease
Gary
TTR-FAP
David
TTR-FAP
Confidential. Internal Use Only.
Amy
Growth Hormone Deficiency Michael
Duchenne Muscular Dystrophy
Amelia
Growth Hormone Deficiency Kyle
Sickle Cell Disease
Susan
Acromegaly
Tiahna
Sickle Cell Disease
Ian
Growth Hormone Deficiency Kyle
Friedreich’s Ataxia
Cyndi
Gaucher Disease
Carolyn
Acromegaly
Matt
Friedreich’s Ataxia
Curt
Hemophilia B
Gabe
Hemophilia A Amiya
Sickle Cell Disease
Gary
TTR-FAP
David
TTR-FAP
Pfizer Rare Disease is committed to transforming the lives of the
rare disease community through life-changing innovations,
trusted partnerships, and relentless passion. 8
Pfizer Rare Disease Time to seize the moment! We have been here, we are
here and we will be here for
the Rare Disease community.