Pfizer Announces Top-Line Results Of Phase 3B/4 Study Of RAPAMUNE® (sirolimus) In Renal Transplant Recipients

  Pfizer Announces Top-Line Results Of Phase 3B/4 Study Of RAPAMUNE®
  (sirolimus) In Renal Transplant Recipients

Business Wire

NEW YORK -- November 8, 2013

Pfizer Inc. (NYSE:PFE) announced today top-line results from a Phase 3B/4
study of RAPAMUNE® (sirolimus) evaluating kidney transplant patients who
transitioned from tacrolimus-based therapy (TAC) to RAPAMUNE® 3 to 5 months
after transplant. The primary endpoint of the study was not achieved as there
was not a statistically significant difference in renal function improvement
between patients who continued receiving TAC and those who switched to

For patients who were switched to RAPAMUNE®, 33.7% of patients achieved the
primary endpoint of a ≥ 5 ml/min/1.73m2 renal function improvement based on
glomerular filtration rate (GFR) from randomization to 24 months
post-transplantation and 42.3% of patients continuing to take TAC achieved the
primary endpoint (p=0.239).

The study was an open-label, randomized, comparative, multi-center,
multi-national study conducted in Europe, Latin America, North America, and
the Pacific Region. There were 256 subjects randomized at 3 to 5 months after
transplant, with two subjects not receiving any medication. Group I included
131 subjects receiving RAPAMUNE®; the dose was adjusted to sirolimus blood
levels of 7-15 ng/mL during the first year post-transplantation and 5-15 ng/mL
thereafter. Group II, the control group, included 123 subjects who continued
receiving the same TAC-based treatment that was being administered prior to
randomization. Subjects in both groups were evaluated at a pre-randomization
visit, at the day of randomization, 4-weeks after randomization, and 6-, 12-,
18- and 24-months post transplantation.

The study also evaluated the safety of RAPAMUNE® and the adverse events
observed in the study were consistent with the known safety profile of
RAPAMUNE®. The results of this study are expected to be submitted for
presentation at upcoming scientific congresses and for publication in
peer-reviewed medical journals.

In the United States, RAPAMUNE® is indicated for the prevention of organ
transplant rejection in kidney transplant patients aged 13 years and older.

Important RAPAMUNE® Safety Information

There is an increased risk of developing infections or certain cancers,
especially lymphoma and skin cancers. RAPAMUNE® has not been shown to be safe
and effective in people who have had liver or lung transplants. Serious
complications and death may happen in people who take RAPAMUNE® after a liver
or lung transplant. You should not take RAPAMUNE® if you have had a liver or
lung transplant without talking with your doctor.

Do not take RAPAMUNE® if you know you are allergic to sirolimus or any of the
other ingredients in RAPAMUNE®. Symptoms of an allergic reaction include
swelling of your face, eyes, or mouth; trouble breathing or wheezing; throat
tightness; chest pain or tightness; feeling dizzy or faint; and rash or
peeling of your skin.

Before taking RAPAMUNE®, tell your doctor if you have liver problems, skin
cancer or it runs in your family, high cholesterol or triglycerides, are
breastfeeding or plan to breastfeed, and are pregnant or plan to become
pregnant. Women of childbearing potential should use effective birth control
before therapy, during therapy, and for 12 weeks after RAPAMUNE® therapy has
been stopped. RAPAMUNE® may interact with other medicines. Make sure that your
doctor is aware of all prescription and over-the-counter drugs that you are
taking, including vitamins, herbs, and nutritional supplements.

RAPAMUNE® may cause swelling in your hands, feet, and in various tissues of
your body. Call your doctor if you have trouble breathing.

RAPAMUNE® may cause your wounds to heal slowly or not heal well resulting in
redness, drainage, or opening of the wound.

RAPAMUNE® may increase the levels of cholesterol and triglycerides (lipids or
fat) in your blood. Your doctor should do blood tests to check your lipids
during treatment with RAPAMUNE®. Your doctor may recommend treatment if your
lipid levels become too high. Your lipid levels may remain high even if you
follow your prescribed treatment plan.

In patients taking RAPAMUNE® with cyclosporine, decreased kidney function has
been observed. Your doctor will regularly check your kidney function.

RAPAMUNE® may increase protein in your urine. Your doctor may monitor you for
abnormal protein in your urine from time to time.

RAPAMUNE® may increase your risk for viral infections. Certain viruses can
live in your body and cause active infections when your immune system is weak.
One of these viruses, BK virus, can affect how your kidney works and cause
your transplanted kidney to fail. A certain virus can cause a rare serious
brain infection called Progressive Multifocal Leukoencephalopathy causing
death or severe disability.

RAPAMUNE® may cause potentially life-threatening lung or breathing problems.
Symptoms may include coughing, shortness of breath, or difficulty breathing.

When RAPAMUNE® is taken with cyclosporine or tacrolimus, you may develop a
blood clotting problem resulting in unexplained bleeding or bruising.

Common side effects associated with RAPAMUNE® include high blood pressure,
pain (including stomach and joint pain), diarrhea, headache, fever, urinary
tract infection, low red blood cell count (anemia), nausea, and low platelet
count (cells that help blood to clot). If you experience any side effects,
contact your doctor.

RAPAMUNE® Indications and Usage

RAPAMUNE® (sirolimus) is indicated for the prevention of organ rejection in
kidney transplant patients aged 13 years or older. Blood levels of sirolimus
should be checked in all patients taking RAPAMUNE®.

In patients at low to moderate risk of acute rejection, it is recommended that
RAPAMUNE® be used initially in combination with cyclosporine and
corticosteroids; cyclosporine should be withdrawn approximately 3 months after
transplantation. Cyclosporine withdrawal has not been studied in patients who
have had severe acute rejection prior to cyclosporine withdrawal, those who
require dialysis or have a high serum creatinine, Black patients, patients
receiving a repeat kidney transplant, patients receiving other transplanted
organs besides the kidney transplant, or patients with antibodies that may be
directed against the kidney transplant.

In patients at high risk of acute rejection (defined as Black patients and/or
patients receiving a repeat kidney transplant who lost a previous kidney
transplant from rejection and/or patients with high levels of antibodies that
may be directed against the kidney transplant), it is recommended that
RAPAMUNE® be used in combination with cyclosporine and corticosteroids for the
first year following transplantation. The safety and efficacy of this
combination in high-risk patients have not been studied beyond one year;
therefore, after the first year, adjustments to the immunosuppressive regimen
may be considered by your doctor.

In pediatric patients, the safety and efficacy of RAPAMUNE® have not been
established in kidney transplant patients less than 13 years old, or in
patients less than 18 years old who are considered at high risk of acute

The safety and efficacy of RAPAMUNE® without cyclosporine in newly
transplanted kidney patients have not been established.

The safety and efficacy of changing from either cyclosporine or tacrolimus to
RAPAMUNE® in maintenance kidney transplant patients have not been established.

Please see full Prescribing Information, including Medication Guide and Boxed
Warning, available at

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Kim Bencker
M: 610-329-1340
Suzanne Harnett
O: 212-733-8009
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