Pfizer Receives Positive CHMP Opinion for BESPONSA® (Inotuzumab Ozogamicin) for the Treatment of Relapsed or Refractory Acute Lymphoblastic Leukemia

NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE:PFE) today announced that the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines Agency (EMA) has
adopted a positive opinion recommending approval of BESPONSA^®
(inotuzumab ozogamicin) in the European Union (EU) as monotherapy
for the treatment of adults with relapsed or refractory CD22-positive
B-cell precursor Philadelphia chromosome negative (Ph-) acute
lymphoblastic leukemia (ALL) and Philadelphia chromosome positive (Ph+)
ALL, who have previously failed treatment with at least one tyrosine
kinase inhibitor (TKI). The CHMP’s opinion will now be reviewed by the
European Commission (EC). If approved, BESPONSA will be the first
antibody drug conjugate available for patients with this type of
leukemia.

“Relapsed or refractory ALL is a rapidly progressive and often fatal
disease. BESPONSA is an antibody-drug conjugate that has been designed
to bind to a receptor – CD22 – that is present on the leukemia cells of
most patients with ALL and deliver a potent toxin – calicheamicin – into
those cells,” said Mace Rothenberg, MD, chief development officer,
Oncology, Pfizer Global Product Development. “The positive results of
the INO-VATE 1022 Phase 3 trial provide strong evidence of the important
role BESPONSA may have versus commonly used chemotherapy regimens used
in this situation, and we believe BESPONSA could provide patients with
relapsed or refractory ALL with a much needed treatment option.”

“Approximately 10,000 new adult cases of ALL are diagnosed in Europe
each year, and there is an urgent unmet need for patients with relapsed
or refractory adult ALL, as the reported long term survival rates range
from between less than 10 percent to approximately 20 percent¹,”
said Professor Matthias Stelljes, Department of Medicine A/Hematology
and Oncology, University of Münster, Germany. “Today’s decision by the
CHMP to recommend marketing authorization of BESPONSA is an important
step forward for patients in Europe, and the community looks forward to
potentially having a new treatment option available.”

ALL is an aggressive type of leukemia with a poor prognosis in adults.² The
current foundational treatment is intensive, long-term chemotherapy.³
ALL is uncommon in adults, representing about 15 percent of leukemias,
with about 10,000 new adult cases diagnosed in Europe each year.⁴
Approximately 20 to 40 percent of newly diagnosed adults with ALL are
cured with current treatment regimens,⁵ however about 20
percent of adult patients will be refractory, or resistant, to
treatment, and an additional 40 to 50 percent will relapse within months
or years. For patients with relapsed or refractory adult ALL, the
five-year overall survival rate is less than 10 percent.⁶

The Marketing Authorization Application (MAA) for BESPONSA was based on
results from the Phase 3 INO-VATE 1022 trial, which enrolled 326 adult
patients with relapsed or refractory B-cell ALL, and compared BESPONSA
to standard of care chemotherapy. The INO-VATE 1022 study had two
primary endpoints, complete response with or without hematologic
remission (CR/CRi) and overall survival (OS). Results from the trial
were published in The New England Journal of Medicine in June
2016.

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

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, including acute myeloid leukemia (AML), acute lymphoblastic
leukemia (ALL), chronic myeloid leukemia (CML) and mantle cell lymphoma
(MCL).

About BESPONSA^® (Inotuzumab Ozogamicin)

BESPONSA is an investigational antibody-drug conjugate (ADC) comprised
of a monoclonal antibody (mAb) targeting CD22, a cell surface antigen
found on cancer cells in almost all B-ALL patients, linked to a
cytotoxic agent.⁷ 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.⁸ The most
common adverse events (AEs) observed in clinical trials for BESPONSA
were cytopenias, including febrile neutropenia. Common nonhematologic
treatment-emergent AEs with BESPONSA included nausea, headache and
pyrexia. Additionally, veno-occlusive liver disease (VOD) was observed
more frequently in patients treated with BESPONSA, especially those who
went on to receive hematopoietic stem cell transplantation.

BESPONSA originates from a collaboration between Pfizer and Celltech,
now UCB. Pfizer has sole responsibility for all manufacturing and
clinical development activities for this molecule.

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.

Pfizer Inc.: Working together for a healthier world^®

At Pfizer, we apply science and our global resources to bring therapies
to people that extend and significantly improve their lives. We strive
to set the standard for quality, safety and value in the discovery,
development and manufacture of healthcare products. Our global portfolio
includes medicines and vaccines as well as many of the world’s
best-known consumer healthcare products. Every day, Pfizer colleagues
work across developed and emerging markets to advance wellness,
prevention, treatments and cures that challenge the most feared diseases
of our time. Consistent with our responsibility as one of the world’s
premier innovative biopharmaceutical companies, we collaborate with
health care providers, governments and local communities to support and
expand access to reliable, affordable health care around the world. For
more than 150 years, Pfizer has worked to make a difference for all who
rely on us. We routinely post information that may be important to
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In addition, to learn more, please visit us on www.pfizer.com
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DISCLOSURE NOTICE: The information contained in this release
is as of April 21, 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), an investigational oncology therapy, 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, the uncertainties inherent in research and
development, including, without limitation, 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 MAA, the BLA and any such other applications for
BESPONSA may be approved by the European Commission, 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.

________________________________
¹ Gokbuget N. et al.
Outcome of relapsed adult lymphoblastic leukemia depends on response to
salvage chemotherapy, prognostic factors, and performance of stem cell
transplantation. Blood. 2012; 120(10): 2032-2041.
²
National Cancer Institute: Adult Acute Lymphoblastic Leukemia Treatment
(PDQ®) – General Information About Adult Acute Lymphoblastic Leukemia
(ALL). Available at: http://www.cancer.gov/cancertopics/pdq/treatment/adultALL/HealthProfessional/page1.
Accessed March 21, 2016.
³ American Cancer Society:
Typical treatment of acute lymphocytic leukemia. Available at: http://www.cancer.org/cancer/leukemia-acutelymphocyticallinadults/detailedguide/leukemia-acute-lymphocytic-treating-typical-treatment.
Accessed March 21, 2016.
⁴ Orphan Europe. Acute
lymphoblastic leukaemia. Available at: http://www.orphan-europe.com/patients-and-families/acute-lymphoblastic-leukaemia.
Accessed March 1, 2017.
⁵ Manal Basyouni A. et al.
Prognostic significance of surviving and tumor necrosis factor-alpha in
adult acute lymphoblastic leukemia.
doi:10.1016/j.clinbiochem.2011.08.1147.
⁶ Fielding A. et
al. Outcome of 609 adults after relapse of acute lymphoblastic leukemia
(ALL); an MRC UKALL12/ECOG 2993 study. Blood. 2006; 944-950.
⁷
Leonard J et al. Epratuzumab, a Humanized Anti-CD22 Antibody, in
Aggressive Non-Hodgkin’s Lymphoma: a Phase I/II Clinical Trial Results. Clinical
Cancer Research. 2004; 10: 5327-5334.
⁸ DiJoseph JF.
Antitumor Efficacy of a Combination of CMC-544 (Inotuzumab Ozogamicin),
a CD22-Targeted Cytotoxic Immunoconjugate of Calicheamicin, and
Rituximab against Non-Hodgkin’s B-Cell Lymphoma. Clin Cancer Res.
2006; 12: 242-250.

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