Palbociclib Expanded Access Program Now Open To Eligible U.S. Patients With HR+, HER2- Advanced Breast Cancer

  Palbociclib Expanded Access Program Now Open To Eligible U.S. Patients With
  HR+, HER2- Advanced Breast Cancer

Business Wire

NEW YORK -- August 21, 2014

Pfizer Inc. today announced that the company has initiated a multi-center,
open-label expanded access program (EAP) in the United States for the
investigational CDK 4/6 inhibitor, palbociclib. Through the program,
palbociclib is being made available for use in combination with letrozole for
post-menopausal women with hormone receptor positive (HR+), human epidermal
growth factor receptor 2 negative (HER2-) advanced breast cancer for whom
letrozole is considered appropriate therapy. Healthcare professionals and
patients can learn more about the palbociclib EAP by visiting (trial number: NCT02142868).

Under its expanded access programs, the U.S. Food and Drug Administration
(FDA) works with companies to allow access to investigational therapies to
patients with serious or life-threatening illnesses who do not otherwise
qualify for participation in a clinical trial and for whom there are no
comparable or satisfactory alternate therapies.

“Palbociclib is being evaluated as a potential new treatment for women with
HR+, HER2- advanced breast cancer, who represent the largest subgroup of women
with this disease,” said Dr. Mace Rothenberg, senior vice president, Clinical
Development and Medical Affairs, and chief medical officer, Pfizer Oncology.
“As announced this week, we have completed the submission of a New Drug
Application for palbociclib in the U.S. based on the results of our Phase 2,
PALOMA-1 study. With recruitment of new patients to our Phase 3 PALOMA-2 and
PALOMA-3 trials now complete, Pfizer is initiating the Palbociclib Expanded
Access Program. This program will provide a mechanism by which eligible women
who may benefit from treatment with palbociclib can gain access to this
investigational therapy at this time.”

About the Palbociclib EAP

The palbociclib EAP is a U.S.-only, single-arm, open label study for
post-menopausal women with HR+, HER2- advanced breast cancer. Women enrolled
to the study will receive palbociclib for use in combination with letrozole,
and therefore must be deemed appropriate for letrozole therapy. Additional
enrollment criteria are available at (trial number:

U.S.-based health care professionals seeking more information about the
palbociclib EAP can call 1-800-420-6755 or e-mail
for further details.

Patients who are interested in enrolling to the palbociclib EAP should speak
with their physician to understand if palbociclib is an appropriate treatment

Palbociclib is an investigational therapy and is not approved for any
indication in any markets.

About Palbociclib

Palbociclib is an investigational oral targeted agent that selectively
inhibits cyclin-dependent kinases (CDKs) 4 and 6 to regain cell cycle control
and block tumor cell proliferation.^1

Loss of cell cycle control is a hallmark of cancer and CDK 4/6 are
overactivated in numerous cancers, leading to loss of proliferative
control.^2,3 CDK 4/6 are key regulators of the cell cycle that trigger
cellular progression from growth phase (G1) into phases associated with DNA
replication (S).^4,5 CDK 4/6, whose increased activity is frequent in estrogen
receptor-positive (ER+) breast cancer (BC), are key downstream targets of ER
signaling in ER+ BC.^6,7 Preclinical data suggest that dual inhibition of CDK
4/6 and ER signaling is synergistic and has been shown to stop growth of ER+
BC cell lines in the G1 phase.

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

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 health care products. Our global portfolio includes medicines
and vaccines as well as many of the world's best-known consumer health care
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. To learn more, please visit us at

DISCLOSURE NOTICE: The information contained in this release is as of August
21, 2014. Pfizer assumes no obligation to update forward-looking statements
contained in this release as the result of new information or future events or

This release contains forward-looking information about palbociclib, an
investigational therapy, that involves substantial risks and uncertainties
that could cause actual results to differ materially from those expressed or
implied by such statements. Forward-looking statements include those about
palbociclib’s potential benefits and the potential indication for the
treatment of postmenopausal women with ER+, HER2- advanced breast cancer who
have not received previous systemic treatment for their advanced disease (the
“Potential Indication”). Risks and uncertainties include, among other things,
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 the FDA will accept the
New Drug Application (the “NDA”) submitted for palbociclib; whether the
PALOMA-2 Phase 3 trial of palbociclib for the Potential Indication will
demonstrate a statistically significant improvement in progression-free
survival and whether the other Phase 3 trials of palbociclib will meet their
primary endpoints; whether regulatory authorities will be satisfied with the
design of and results from our clinical studies; whether and when drug
applications may be filed in any other jurisdictions for the Potential
Indication or in any jurisdictions for any other potential indications for
palbociclib; whether and when the NDA or any such other applications may be
approved by the FDA or other 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 the FDA and other regulatory authorities regarding labeling and
other matters that could affect the availability or commercial potential of
the Potential Indication or any other such indications; and competitive

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, 2013 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 SEC and available at and

^1 Study of Letrozole with or without PD 0332991 for the
first-line treatment of hormone-receptor positive advanced breast cancer.
Available here:
Accessed August 19, 2014.
^2 Shapiro GI. Cyclin-dependent kinase pathways as targets for cancer
treatment. J Clin Oncol. 2006;24(11):1770-1783.
^3 Weinberg RA. The Biology of Cancer. New York, NY. Garland Science; 2013.
^4 Hirama T and H. Phillip Koeffler. Role of the Cyclin-Dependent Kinase
Inhibitors in the Development of Cancer. Blood. 1995; 86: 841-854.
^5 Fry D et al. Specific Inhibition of cyclin-dependent kinase 4/6 by PD
0332991 and associated antitumor activity in human tumor xenografts. Molecular
Cancer Therapeutics. 2004; 3: 1427-1437.
^6 Finn RS et al. PD 0332991, a selective cyclin D kinase 4/6 inhibitor,
preferentially inhibits proliferation of luminal estrogen receptor-positive
human breast cancer cell lines in vitro. Breast Cancer Res. 2009;11(5):R77.
^7 Lamb R, Lehn S, Rogerson L, Clarke RB, Landberg G. Cell cycle regulators
cyclin D1 and CDK4/6 have estrogen receptor-dependent divergent functions in
breast cancer migration and stem cell-like activity. Cell Cycle.


Pfizer Inc.
Sally Beatty, 212-733-6566
Ryan Crowe, 212-733-8160
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