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Tafamidis Phase 3 Transthyretin Amyloid Cardiomyopathy (ATTR-ACT) Study Results Presented as Late-Breaking Data at the ESC Congress 2018

ATTR-ACT Showed that Tafamidis Significantly Reduced the Combination of All-cause Mortality and Cardiovascular-related Hospitalizations Data Showed a 30% Reduction in the Risk of Mortality and 32% Reduction in the Rate of Cardiovascular-related Hospitalizations with Tafamidis in People with Transthyretin Amyloid Cardiomyopathy versus Placebo

Monday, August 27, 2018 - 5:21am
EDT

NEW YORK--(BUSINESS WIRE)--Pfizer Inc. (NYSE:PFE) announced today the primary results from the
Tafamidis Phase 3 Transthyretin Cardiomyopathy (ATTR-ACT) study, which
showed tafamidis significantly reduced the hierarchical combination of
both all-cause mortality and frequency of cardiovascular-related
hospitalizations compared to placebo over a 30-month period (P=0.0006)
in patients with wild-type or variant (hereditary) transthyretin amyloid
cardiomyopathy (ATTR-CM).1 ATTR-CM is a rare, fatal, and
underdiagnosed condition associated with progressive heart failure for
which there are no approved pharmacologic treatments.2,3

The late-breaking findings were presented during Hot Line Session 3 at
the ESC Congress 2018 in Munich, Germany and simultaneously published
online
in the New England Journal of Medicine (NEJM).

“We believe the ATTR-ACT study findings bring us a significant step
closer to our goal of providing an urgently needed therapy for a serious
and often fatal disease,” said Brenda Cooperstone MD, Senior Vice
President and Chief Development Officer, Rare Disease, Pfizer Global
Product Development. “We look forward to continuing discussions with
global regulatory authorities about the potential of tafamidis as a
treatment option for people living with ATTR-CM.”

The ATTR-ACT study showed tafamidis significantly reduced all-cause
mortality (29.5% vs. 42.9%; hazard ratio = 0.70, 95% confidence interval
[CI] 0.51-0.96, P=0.0259) and cardiovascular-related hospitalizations
(0.48 vs 0.70 annualized rate; relative risk ratio = 0.68, 95% CI
0.56-0.81, P1 This
represents a 30% reduction in the risk of mortality and 32% reduction in
the rate of cardiovascular-related hospitalization. The findings also
showed a consistent directional mortality benefit of tafamidis across
all sub-groups.1

Secondary study endpoints also showed tafamidis reduced the decline in
the six minute walk test distance (P capacity, and reduced the decline in aspects of quality of life measured
by the Kansas City Cardiomyopathy Questionnaire – Overall Score
(P1 Tafamidis
was also well tolerated, with an observed safety profile comparable to
placebo.1

“ATTR-CM patients face a difficult diagnosis and treatment journey. By
examining tafamidis in the ATTR-ACT trial, we were hoping the data for
this oral agent would yield positive results for these patients and I am
very pleased by the findings,” said Claudio Rapezzi MD, Director,
Cardiology, School of Cardiovascular Diseases, University of Bologna,
and ATTR-ACT study presenter at the ESC Congress 2018. “The ATTR-ACT
data provide strong evidence that tafamidis improves survival, which
could mean a significant advance for patients living with ATTR-CM today.”

The NEJM manuscript, titled “Tafamidis Treatment for Patients
with Transthyretin Amyloid Cardiomyopathy,” will be published in the
September 13 printed issue of NEJM.

In light of the seriousness of the disease and the lack of pharmacologic
treatment options, Pfizer has established an expanded access treatment
protocol to make tafamidis available to ATTR-CM patients who may benefit
from treatment prior to regulatory approval. The expanded access
treatment protocol is posted on clinicaltrials.gov (NCT02791230), and
additional information about requesting access may be found at www.pfizercares.com.
Access to these programs may vary by country; physicians may contact
their local Pfizer Medical department for further information.
Interested ATTR-CM patients should contact their local physician to
discuss whether accessing tafamidis may be an appropriate option.

Tafamidis was granted Orphan Drug Designation for ATTR-CM in both the EU
and US in 2012 and in Japan in 2018. In June 2017 and May 2018,
respectively, the US Food and Drug Administration (FDA) granted
tafamidis Fast Track and Breakthrough Therapy designations for ATTR-CM.
Additionally, in March 2018, the Ministry of Labor Health and Welfare in
Japan granted SAKIGAKE designation to tafamidis for this indication.

About the ATTR-ACT Study
1

ATTR-ACT is a Phase 3 international, multicenter, double-blind,
placebo-controlled, randomized, 3-arm clinical study in 441 patients
with ATTR-CM that investigated the efficacy, safety, and tolerability of
an oral daily dose of 20 mg or 80 mg tafamidis meglumine capsules
compared to placebo. The study included both patients with variant
(ATTRm), or hereditary, form of the disease, and those with wild-type
(ATTRwt) form, which is not hereditary and may occur as people age. The
primary analysis of the study, which compared a pooled tafamidis (80 mg
and 20 mg) treatment group to placebo, was the hierarchical combination
of all-cause mortality and frequency of cardiovascular-related
hospitalizations over a 30-month period in patients with transthyretin
amyloid cardiomyopathy.

For more information on the ATTR-ACT study, go to www.clinicaltrials.gov.

Tafamidis is an investigational treatment for transthyretin amyloid
cardiomyopathy and is not approved for this indication.

About ATTR-CM

ATTR-CM is a rare, progressive, and underdiagnosed disease caused by
destabilization of a transport protein called transthyretin, which is
composed of 4 identical sub units (a tetramer).3 In ATTR-CM,
heart failure occurs when unstable tetramers dissociate, resulting in
misfolded proteins that aggregate into amyloid fibrils and deposit
predominantly in the heart.3

Pfizer Rare Disease

Rare disease includes some of the most serious of all illnesses and
impacts millions of patients worldwide,4 representing an
opportunity to apply our knowledge and expertise to help make a
significant impact on addressing unmet medical needs. The Pfizer focus
on rare disease builds on more than two decades of experience, a
dedicated research unit focusing on rare disease, and a global portfolio
of multiple medicines within a number of disease areas of focus,
including hematology, neuroscience, and inherited metabolic disorders.1

Pfizer Rare Disease combines pioneering science and deep understanding
of how diseases work with insights from innovative strategic
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to deliver transformative treatments and solutions. We innovate every
day leveraging our global footprint to accelerate the development and
delivery of groundbreaking medicines and the hope of cures.

Click here
to learn more about our Rare Disease portfolio and how we empower
patients, engage communities in our clinical development programs, and
support programs that heighten disease awareness.

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DISCLOSURE NOTICE: The information contained in this release is as of
5am EDT/11am CEST on August 27, 2018. 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 a potential
indication for tafamidis for the treatment of transthyretin amyloid
cardiomyopathy (the “Potential Indication”) and Pfizer’s rare disease
portfolio, including their 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
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; the risk that clinical trial data
are subject to differing interpretations, and, even when we view data as
sufficient to support the safety and/or effectiveness of a product
candidate, regulatory authorities may not share our views and may
require additional data or may deny approval altogether; whether
regulatory authorities will be satisfied with the design of and results
from our clinical studies; whether and when any new or supplemental drug
applications may be filed in any jurisdictions for tafamidis for the
Potential Indication; whether and when regulatory authorities in any
such jurisdictions where applications for tafamidis may be pending
(including the application pending with the FDA for the potential
treatment of transthyretin familial amyloid polyneuropathy, for which
the company received a complete response letter in 2012) or filed may
approve any such applications, which will depend on the assessment by
such regulatory authority of the benefit-risk profile suggested by the
totality of the efficacy and safety information submitted, and, if
approved, whether tafamidis will be commercially successful; decisions
by regulatory authorities regarding labeling and other matters that
could affect the availability or commercial potential of tafamidis,
including for the Potential Indication; 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, 2017 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 Data on file. Pfizer Inc. New York, NY.

2 Rapezzi C, Quarta CC, Riva L, et al. Transthyretin-related
amyloidoses and the heart: a clinical overview. Nat Rev Cardiol.
2010;7:398-408.

3 Ando Y, Coelho T, Berk JL, et al. Guideline of
transthyretin related hereditary amyloidosis for clinicians. Orphanet J
Rare Dis. 2013;8:31.

4 Pfizer Inc. Rare disease. https://www.pfizer.com/health-wellness/disease-conditions/rare-diseases/areas-of-focus.
Accessed August 9, 2018.



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Pfizer Inc.
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