Bayer Initiates Phase III Trial of an Investigational Recombinant Factor VIII Compound in Children with Hemophilia A

Bayer Initiates Phase III Trial of an Investigational Recombinant Factor VIII
                    Compound in Children with Hemophilia A

Phase III PROTECT VIII Kids Trial will Evaluate the Pharmacokinetics, Safety
and Efficacy of BAY94-9027 for Prophylaxis and Treatment of Bleeding in
Previously Treated Children with Severe Hemophilia A

PR Newswire

WAYNE, N.J., July 8, 2013

WAYNE, N.J., July 8, 2013 /PRNewswire/ --Bayer HealthCare today announced the
company has started to enroll patients in an international Phase II/III trial
to evaluate its investigational compound BAY94-9027 for the treatment of
hemophilia A in children. The PROTECT VIII (PROphylaxis in hemophilia A
patienTs via directly pEgylated long-aCTing rFVIII) Kids trial is designed to
determine the effects of BAY94-9027, a recombinant human factor VIII (rFVIII),
when used as prophylaxis, dosed at least once weekly and as on-demand for
acute bleeding events.

BAY94-9027 has been designed to extend the circulating half-life of rFVIII
through site specific attachment of a polyethylene glycol (PEG) polymer to the
light chain of the rFVIII molecule, while preserving its full biologic
activity.

"We are happy that prophylaxis with this long-acting compound is now being
studied in children younger than twelve years of age to complement our ongoing
adult study. We look forward to sharing clinical trial results on BAY94-9027
when they are available," said Dr. Georg Lemm, Vice President, Global
Clinical Development Hematology, Bayer HealthCare.

Bayer is committed to developing treatment solutions that meet the needs of
the hemophilia community. Whether through research, support or clinical
management, Bayer is dedicated to helping persons with hemophilia and their
families.

About the Phase III Study
PROTECT VIII Kids is a multicenter, multinational, non-controlled, open-label
trial evaluating the pharmacokinetics, safety and efficacy of BAY 94-9027,
used as prophylaxis at 25-60 IU/kg at least 1x/week and as on-demand treatment
of bleeding in male children with severe hemophilia A. The study will enroll
50 previously treated patients (PTP) worldwide. Subjects will be males, up to
12 years of age, who have severe hemophilia A (<1% FVIIIc) and a documented
history of at least 50 exposure days (ED) with any FVIII product. Outcome
measures to be evaluated include pharmacokinetics, annualized bleeding
episodes, response of acute bleeding episodes to treatment and adverse events,
including inhibitor formation. The total duration of the individual treatment
with the study drug is the amount of time required to achieve at least 50
exposure days.

Subjects will also be offered participation in an optional extension study,
during which observations will be collected for at least 50 additional
exposure days.

About Hemophilia A
Hemophilia A, also known as factor VIII deficiency or classic hemophilia, is a
largely inherited bleeding disorder in which one of the proteins needed to
form blood clots in the body is missing or reduced. Hemophilia A, the most
common type of hemophilia, is caused by deficient or defective blood
coagulation proteins, known as factor VIII. Hemophilia A is characterized by
prolonged or spontaneous bleeding, especially into the muscles, joints or
internal organs.

About Bayer HealthCare Pharmaceuticals Inc.
Bayer HealthCare Pharmaceuticals Inc. is the U.S.-based pharmaceuticals
business of Bayer HealthCare LLC, a subsidiary of Bayer AG. Bayer HealthCare
is one of the world's leading, innovative companies in the healthcare and
medical products industry, and combines the activities of the Animal Health,
Consumer Care, Medical Care, and Pharmaceuticals divisions. As a specialty
pharmaceutical company, Bayer HealthCare provides products for General
Medicine, Hematology, Neurology, Oncology and Women's Healthcare. The
company's aim is to discover and manufacture products that will improve human
health worldwide by diagnosing, preventing and treating diseases.

BAYER and the Bayer Cross are registered trademarks of Bayer.

Intended for U.S. Media Only

Forward-Looking Statements
This news release may contain forward-looking statements based on current
assumptions and forecasts made by Bayer Group or subgroup management. Various
known and unknown risks, uncertainties and other factors could lead to
material differences between the actual future results, financial situation,
development or performance of the company and the estimates given here. These
factors include those discussed in Bayer's public reports which are available
on the Bayer website at www.bayer.com. The company assumes no liability
whatsoever to update these forward-looking statements or to conform them to
future events or developments.

SOURCE Bayer HealthCare Pharmaceuticals Inc.

Contact: Jane Ward, Bayer HealthCare, +1 973 420 2876, jane.ward@bayer.com
 
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