BESPONSA® Approved in the EU for Adult Patients with Relapsed or Refractory B-cell Precursor Acute Lymphoblastic Leukemia

NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE:PFE) today announced that the European Commission has
approved BESPONSA^® (inotuzumab ozogamicin) as
monotherapy for the treatment of adults with relapsed or refractory
CD22-positive B-cell precursor acute lymphoblastic leukemia (ALL). This
indication includes treatment of adults with Philadelphia chromosome
positive (Ph+) as well as Philadelphia chromosome negative (Ph-)
relapsed or refractory B-cell precursor ALL. Adults with Ph+ relapsed or
refractory CD22-positive B-cell precursor ALL should have failed
treatment with at least one tyrosine kinase inhibitor (TKI). With this
approval, BESPONSA becomes the first and only antibody drug conjugate
(ADC) available for patients with this type of leukemia in the European
Union (EU).

“The European Commission’s approval of BESPONSA represents an important
milestone for patients, the oncology community and Pfizer,” said Andreas
Penk, M.D., regional president, Pfizer Oncology. “This is the first
approval for BESPONSA and provides patients in the EU, who are battling
an especially hard-to-treat leukemia, with a new treatment option beyond
chemotherapy.”

ALL is an aggressive type of leukemia that can be fatal within a matter
of months if left untreated.¹ The goal of treatment in
relapsed or refractory (resistant) ALL is to achieve complete remission
without excessive toxicity so patients may proceed to additional
therapeutic intervention, particularly stem cell transplant, which is
the most recognized option to prolong patient survival, maintenance
therapy or other therapy.² In adult patients with relapsed or
refractory ALL, median overall survival is just three to six months.^3,4,5
The current standard of care is intensive chemotherapy⁶,
which is effective in less than 50 percent of relapsed or refractory
patients and associated with poor long-term survival, high toxicities,
lengthy inpatient stays and continuous infusions.⁷

“Acute lymphoblastic leukemia that has recurred or is refractory
following first-line therapy is a rare and rapidly progressive disease
with poor prognosis,” said Professor David Marks, Department of
Hematology, University Hospitals Bristol NHS Foundation Trust, Bristol,
United Kingdom. “The approval of BESPONSA (inotuzumab ozogamicin)
provides a much needed treatment option for physicians and patients
alike, that may help improve outcomes for some of the most vulnerable
leukemia patients in Europe.”

The European Commission’s approval of BESPONSA is supported by results
from the Phase 3 INO-VATE ALL trial, in which 326 adult patients with
relapsed or refractory B-cell precursor ALL were enrolled and which
compared BESPONSA to standard of care chemotherapy. The INO-VATE ALL
study had two primary endpoints, complete response with or without
hematologic recovery (CR/CRi) and overall survival (OS). Results from
the trial were published in The New England Journal of Medicine in
June 2016.

In the U.S., BESPONSA received Breakthrough Therapy designation from the
Food and Drug Administration (FDA) in October 2015 for ALL. A Biologics
License Application (BLA) for BESPONSA for the treatment of adult
patients with relapsed or refractory B-cell precursor ALL was accepted
for filing and granted Priority Review by the FDA in March 2017. The
Prescription Drug User Fee Act (PDUFA) goal date for a decision by the
FDA is August 2017.

With a growing hematology pipeline, Pfizer is committed to extending
therapeutic progress in acute and chronic leukemias that leverage select
pathways and mechanism of actions (MOAs). Specifically, our
investigational products aim to treat some of the hardest to treat
leukemias and lymphomas including, acute myeloid leukemia (AML), acute
lymphoblastic leukemia (ALL), chronic myeloid leukemia (CML) and mantle
cell lymphoma (MCL).

Indication for BESPONSA^® (Inotuzumab Ozogamicin) in
the EU

BESPONSA is approved as monotherapy for the treatment of adults with
relapsed or refractory CD22-positive B-cell precursor ALL in the EU.
Adult patients with Ph⁺ relapsed or refractory B-cell
precursor ALL should have failed treatment with at least one TKI.

Important Safety Information for BESPONSA^®
(Inotuzumab Ozogamicin) in the EU

The most common (≥ 20%) adverse reactions associated with BESPONSA were
thrombocytopenia (51%), neutropenia (49%), infection (48%), anaemia
(36%), leukopenia (35%), fatigue (35%), haemorrhage (33%), pyrexia
(32%), nausea (31%), headache (28%), febrile neutropenia (26%),
increased transaminases (26%), abdominal pain (23%), increased
gamma-glutamyltransferase (21%), and hyperbilirubinaemia (21%).

The most common (≥ 2%) serious adverse reactions associated with
BESPONSA were infection (23%), febrile neutropenia (11%), haemorrhage
(5%), abdominal pain (3%), pyrexia (3%), veno-occlusive
disease/sinusoidal obstruction syndrome (VOD/SOS) (2%), and fatigue (2%).

In the Phase 3 INO-VATE ALL trial (N=164 patients treated with
BESPONSA), VOD/SOS was reported in 22 (13%) patients including 5 (3%)
patients during study therapy or in follow-up without an intervening
hematopoietic stem cell transplant (HSCT). Among the 77 patients who
proceeded to a subsequent HSCT (6 of whom received additional salvage
therapy after treatment with BESPONSA before proceeding to HSCT),
VOD/SOS was reported in 17 (22%) patients. Five of the 17 VOD/SOS events
that occurred post-HSCT were fatal.

VOD/SOS was reported up to 56 days after the last dose of BESPONSA
without an intervening HSCT. The median time from HSCT to onset of
VOD/SOS was 15 days (range: 3-57 days). Of the 5 patients who
experienced VOD/SOS during treatment with BESPONSA but without an
intervening HSCT, 2 patients had also received an HSCT before BESPONSA
treatment.

Among patients who proceeded to HSCT after BESPONSA treatment, VOD/SOS
was reported in 5/11 (46%) patients who received an HSCT both prior to
and after BESPONSA treatment and 12/66 (18%) patients who only received
an HSCT after BESPONSA treatment.

The EU Summary of Product Characteristics (SmPC) is available at http://www.ema.europa.eu.

About BESPONSA^® (Inotuzumab Ozogamicin)

BESPONSA is an antibody-drug conjugate (ADC) comprised of a monoclonal
antibody (mAb) targeting CD22, a cell surface antigen expressed on
cancer cells in almost all B-ALL patients, linked to a cytotoxic agent.⁸
When BESPONSA binds to the CD22 antigen on B-cells, it is internalized
into the cell, where the cytotoxic agent calicheamicin is released to
destroy the cell.⁹

BESPONSA originates from a collaboration between Pfizer and Celltech,
now UCB. Under the terms of this agreement, Pfizer has sole
responsibility for all manufacturing and clinical development activities
for this molecule. Pfizer also collaborated with SFJ Pharmaceuticals
Group (SFJ) on the registrational program (INO-VATE ALL) for BESPONSA.

About Pfizer Oncology

Pfizer Oncology is committed to pursuing innovative treatments that have
a meaningful impact on those living with cancer. As a leader in oncology
speeding cures and accessible breakthrough medicines to patients, Pfizer
Oncology is helping to redefine life with cancer. Our strong pipeline of
biologics, small molecules and immunotherapies, one of the most robust
in the industry, is studied with precise focus on identifying and
translating the best scientific breakthroughs into clinical application
for patients across a wide range of cancers. By working collaboratively
with academic institutions, individual researchers, cooperative research
groups, governments and licensing partners, Pfizer Oncology strives to
cure or control cancer with its breakthrough medicines. Because Pfizer
Oncology knows that success in oncology is not measured solely by the
medicines you manufacture, but rather by the meaningful partnerships you
make to have a more positive impact on people’s lives.

DISCLOSURE NOTICE: The information contained in this release
is as of June 30, 2017. Pfizer assumes no obligation to update
forward-looking statements contained in this release as the result of
new information or future events or developments.

This release contains forward-looking information about BESPONSA
(inotuzumab ozogamicin), and an approval by the European Commission as
monotherapy for the treatment of adults with relapsed or refractory
CD22-positive B-cell precursor acute lymphoblastic leukemia, including
its potential benefits, that involves substantial risks and
uncertainties that could cause actual results to differ materially from
those expressed or implied by such statements. Risks and uncertainties
include, among other things, uncertainties regarding the commercial
success of BESPONSA; the uncertainties inherent in research and
development, including the ability to meet anticipated clinical trial
commencement and completion dates and regulatory submission dates, as
well as the possibility of unfavorable clinical trial results, including
unfavorable new clinical data and additional analyses of existing
clinical data; whether and when applications for BESPONSA may be filed
in any other jurisdictions; whether and when the BLA pending in the
United States and any other such applications that may be pending or
filed for BESPONSA may be approved by the FDA or other regulatory
authorities, respectively, which will depend on the assessment by such
regulatory authorities of the benefit-risk profile suggested by the
totality of the efficacy and safety information submitted; decisions by
regulatory authorities regarding labeling and other matters that could
affect the availability or commercial potential of BESPONSA; and
competitive developments.

A further description of risks and uncertainties can be found in
Pfizer’s Annual Report on Form 10-K for the fiscal year ended December
31, 2016 and in its subsequent reports on Form 10-Q, including in the
sections thereof captioned “Risk Factors” and “Forward-Looking
Information and Factors That May Affect Future Results”, as well as in
its subsequent reports on Form 8-K, all of which are filed with the U.S.
Securities and Exchange Commission and available at www.sec.gov
and www.pfizer.com.

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