Pfizer Announces Final Results from Inotuzumab Ozogamicin Pivotal Phase 3 Study in Adults with Relapsed/Refractory Acute Lymphoblastic Leukemia

NEW YORK–(BUSINESS WIRE)– Pfizer Inc. (NYSE:PFE) today announced the publication of findings from
the Phase 3 INO-VATE ALL study in the online issue of The New England
Journal of Medicine. The study, also known as Study 1022, is an
open-label, randomized, Phase 3 study evaluating the safety and efficacy
of inotuzumab ozogamicin as compared with investigator-choice
chemotherapy in 326 adult patients with relapsed or refractory
CD22-positive acute lymphoblastic leukemia (ALL). Results showed
improvement over chemotherapy on a number of measures including complete
hematologic remission and progression-free survival (PFS). Updated
results and newly available overall survival (OS) data were also
presented as a late-breaking oral presentation (#LB2233) today at the 21^st
Congress of the European Hematology Association (EHA) 2016 Annual
Meeting in Copenhagen, Denmark.

“Relapsed or refractory ALL is an aggressive leukemia in urgent need of
new treatment options as about half of adult patients will not respond
to chemotherapy or will see their disease return,” said Hagop M.
Kantarjian, M. D., lead study investigator and professor, The University
of Texas MD Anderson Cancer Center. “The efficacy results seen in
patients treated with inotuzumab ozogamicin in this study are
impressive, particularly median progression-free survival, high rates of
hematological remission and absence of minimal residual disease. These
results suggest inotuzumab ozogamicin, if approved, could be a valuable
new addition to currently available treatment options for ALL patients,
including as a bridge to stem cell transplantation, which is the best
chance for a cure at this stage of the disease.”

The INO-VATE ALL study had two independent primary endpoints, complete
response with or without hematologic remission and OS. INO-VATE ALL met
its first primary endpoint of complete response, which was significantly
better with inotuzumab ozogamicin compared to chemotherapy (80.7% [95%
CI, 72%-88%] vs. 29.4% [95% CI, 21%-39%], P<0.001). Inotuzumab
ozogamicin also significantly extended PFS compared to chemotherapy (HR:
0.45 [97.5% CI, 0.34-0.61], P<0.001; median PFS, 5.0 vs. 1.8 months, in
their respective arms). The second primary endpoint of OS showed a
strong trend toward longer OS for patients treated with inotuzumab
ozogamicin compared to chemotherapy, but did not reach the level of
statistical significance (p < 0.0104) for the trial (HR: 0.77 [97.5% CI,
0.58-1.03], one-sided P=0.0203; median OS, 7.7 months [95% CI, 6.0-9.2] vs. 6.7 months [95% CI, 4.9-8.3]). The two-year OS rate for inotuzumab
ozogamicin was 23 percent (95% CI, 16%‒30%) compared to chemotherapy at
10 percent (95% CI, 5%‒16%).

“Adult patients with relapsed or refractory ALL have a five-year
survival rate of less than 10 percent, making these patients
particularly difficult to treat. To see remission rates and two-year
survival rates that are more than doubled compared to standard of care
chemotherapy is very gratifying. We believe these data add to the
growing body of evidence that supports inotuzumab ozogamicin as an
important potential treatment option in adults with relapsed or
refractory ALL,” said Mace Rothenberg, MD, Chief Development Officer,
Oncology, Pfizer Global Product Development.

Results from INO-VATE ALL also showed patients treated with inotuzumab
ozogamicin achieved high rates of minimal residual disease (MRD)
negativity (78.4% [95% CI, 68%-87%; P<0.001]), and experienced a
duration of response (DOR) of 4.6 months (95% CI, 3.9-5.4; HR: 0.55;
P<0.034). In comparison, 28.1 percent (95% CI, 14%-47%; P<0.001) of
patients treated with chemotherapy achieved MRD negativity and median
DOR was 3.1 months (95% CI, 1.4-4.9; HR: 0.55; P<0.034). More patients
also proceeded to stem-cell transplant with inotuzumab ozogamicin
compared to standard chemotherapy (41% vs. 11%, P<0.001).

The most common adverse events (AEs) observed for both inotuzumab
ozogamicin and chemotherapy were cytopenias, including febrile
neutropenia (16% vs. 22%). Common nonhematologic treatment-emergent AEs
with inotuzumab ozogamicin included nausea (32%), headache (28%) and
pyrexia (27%). Patients in the chemotherapy arm experienced nausea
(47%), pyrexia (43%) and diarrhea (40%).

Additionally, any-grade veno-occlusive liver disease (VOD) occurred more
frequently in patients treated with inotuzumab ozogamicin compared to
chemotherapy (11% vs. 1%). Five patients taking inotuzumab ozogamicin
developed VOD during treatment and 10 patients developed VOD after
subsequent stem cell transplant. Among those taking chemotherapy, one
patient developed VOD after transplant; no cases of VOD occurred during
treatment with chemotherapy.

Inotuzumab ozogamicin received Breakthrough Therapy designation from the
U.S. Food and Drug Administration (FDA) for ALL in October 2015. Pfizer
is working closely with the FDA and other regulatory authorities with
the aim of making inotuzumab ozogamicin available for adult patients
with relapsed or refractory CD22-positive ALL.

About Acute Lymphoblastic Leukemia (ALL)

Acute lymphoblastic leukemia (ALL) is an aggressive type of leukemia
with a poor prognosis in adults.¹ The current foundational
treatment is intensive, long-term chemotherapy.² In 2016, it
is estimated that 6,590 cases of ALL will be diagnosed in the United
States, with about 2 in 5 cases in adults.³ Approximately 20
to 40 percent of newly diagnosed adults with ALL are cured with current
treatment regimens.⁴ For patients with relapsed or refractory
adult ALL, the five-year overall survival rate is less than 10 percent.⁵

About Inotuzumab Ozogamicin

Inotuzumab ozogamicin 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. ^1,6 When
inotuzumab ozogamicin binds to the CD22 antigen on malignant B-cells, it
is thought to be internalized into the cell, where the cytotoxic agent
calicheamicin is released to destroy the cell.⁷

Inotuzumab ozogamicin 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 the discovery, investigation and
development of innovative treatment options to improve the outlook for
cancer patients worldwide. Our strong pipeline of biologics and small
molecules, 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 breakthrough medicines, to deliver the right drug for each patient
at the right time. For more information, please visit www.pfizer.com.

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. For more information, please visit us at www.pfizer.com.
In addition, to learn more, follow us on Twitter at @Pfizer and @Pfizer_News, LinkedIn,
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and like us on Facebook at Facebook.com/Pfizer.

DISCLOSURE NOTICE: The information contained in this release
is as of June 12, 2016. 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 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 the ability to meet anticipated 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 new drug applications may be
filed in any jurisdictions for inotuzumab ozogamicin; whether and when
any such applications may be approved by regulatory authorities, 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 inotuzumab ozogamicin; 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, 2015 and in its subsequent reports on Form 10-Q, including in the
sections thereof captioned “Risk Factors” and “Forward-Looking
Information 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 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.

² 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.

³ American Cancer Society: What are the key statistics about
acute lymphocytic leukemia? Available at: http://www.cancer.org/cancer/leukemia-acutelymphocyticallinadults/detailedguide/leukemia-acute-lymphocytic-key-statistics
(link is external). Accessed March 21, 2016.

⁴ Manal Basyouni A. et al. Prognostic significance of
survivin and tumor necrosis factor-alpha in adult acute lymphoblastic
leukemia. doi:10.1016/j.clinbiochem.2011.08.1147.

⁵ 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.

⁶ 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.

⁷ 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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