Market Overview

Genentech's HEMLIBRA (emicizumab-kxwh) Significantly Reduced Bleeds in Phase III Study in Hemophilia A


– HAVEN 3 study met primary endpoint and key secondary endpoints –

– Intra-patient comparison demonstrated superiority of HEMLIBRA
prophylaxis compared to prior factor VIII prophylaxis –

Genentech, a member of the Roche Group ((SIX: RO, ROG, OTCQX:RHHBY),
announced today positive results from the Phase III HAVEN 3 study
evaluating HEMLIBRA® (emicizumab-kxwh) in adults and
adolescents (aged 12 years or older) with hemophilia A without
inhibitors to factor VIII. The study met its primary endpoint, showing a
statistically significant and clinically meaningful reduction in the
number of treated bleeds over time in people receiving HEMLIBRA
prophylaxis every week compared to those receiving no prophylaxis. The
study also met key secondary endpoints, including a statistically
significant and clinically meaningful reduction in the number of treated
bleeds over time with HEMLIBRA prophylaxis dosed every two weeks
compared to no prophylaxis. Importantly, once-weekly HEMLIBRA
prophylaxis was superior to factor VIII prophylaxis, as demonstrated by
a statistically significant and clinically meaningful reduction in
treated bleeds in an intra-patient comparison of patients receiving
HEMLIBRA prophylaxis compared to their prior factor VIII prophylaxis.
The most common adverse events with HEMLIBRA were injection site
reactions, with no new safety signals observed. No thrombotic
microangiopathy or thrombotic events occurred in this study.

"HEMLIBRA is the first product to show superior efficacy to factor VIII
prophylaxis. These results in people with hemophilia A without
inhibitors represent the next step forward in our clinical trial
program, which includes the positive HAVEN 1 and interim HAVEN 2 data in
people with inhibitors," said Sandra Horning, M.D., chief medical
officer and head of Global Product Development. "We look forward to
working with health authorities to make this treatment available for all
people with hemophilia A as soon as possible."

"It is well established that prophylaxis is the preferred approach for
treatment of hemophilia A, but this can require frequent intravenous
infusions, and some patients on prophylaxis can still experience bleeds,
while others prefer on-demand treatment," said Johnny Mahlangu, Faculty
of Health Sciences, University of the Witwatersrand and NHLS,
Johannesburg, South Africa. "Given its potential to be dosed through
subcutaneous injection only once weekly or every other week, HEMLIBRA
may provide a further effective prophylactic treatment option for more
people with hemophilia A and help alleviate some of the administration
burden associated with current treatment."

Data from the HAVEN 3 study will be presented at an upcoming medical
meeting and submitted to health authorities around the world for
approval consideration. These results add to the growing body of
evidence shown in the inhibitor population and support that HEMLIBRA may
benefit all people with hemophilia A regardless of inhibitor status.
Data from the HAVEN 1 and HAVEN 2 studies supported the recent U.S. Food
and Drug Administration (FDA) approval of HEMLIBRA for adults and
children with hemophilia A with inhibitors, and are being reviewed under
accelerated assessment by the European Medicines Agency.

About HAVEN 3 (NCT02847637)

HAVEN 3 is a randomized, multicenter, open-label, Phase III study
evaluating the efficacy, safety and pharmacokinetics of HEMLIBRA
prophylaxis versus no prophylaxis (episodic/on-demand factor VIII
treatment) in people with hemophilia A without inhibitors to factor
VIII. The study included 152 patients with hemophilia A (12 years of age
or older) who were previously treated with factor VIII therapy either
on-demand or for prophylaxis. Patients previously treated with on-demand
factor VIII were randomized in a 2:2:1 fashion to receive subcutaneous
HEMLIBRA prophylaxis at 3 mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk
until the end of study (Arm A), subcutaneous HEMLIBRA prophylaxis at 3
mg/kg/wk for 4 weeks, followed by 3 mg/kg/2wks until the end of study
(Arm B), or no prophylaxis (Arm C). Patients previously treated with
factor VIII prophylaxis received subcutaneous HEMLIBRA prophylaxis at 3
mg/kg/wk for 4 weeks, followed by 1.5 mg/kg/wk until the end of study
(Arm D). Episodic treatment of breakthrough bleeds with factor VIII
therapy was allowed per protocol.


HEMLIBRA is a bispecific factor IXa- and factor X-directed antibody. It
is designed to bring together factor IXa and factor X, proteins required
to activate the natural coagulation cascade and restore the blood
clotting process for hemophilia A patients. HEMLIBRA is a prophylactic
(preventative) treatment that can be administered by an injection of a
ready-to-use solution under the skin (subcutaneously) once weekly.
HEMLIBRA was created by Chugai Pharmaceutical Co., Ltd. and is being
co-developed by Chugai, Roche and Genentech.

HEMLIBRA U.S. Indication

HEMLIBRA is a prescription medicine used for routine prophylaxis to
prevent or reduce the frequency of bleeding episodes in adults and
children with hemophilia A with factor VIII inhibitors.

Important Safety Information

HEMLIBRA increases the potential for blood to clot. Discontinue
prophylactic use of bypassing agents the day before starting HEMLIBRA
prophylaxis. Carefully follow the healthcare provider's instructions
regarding when to use an on-demand bypassing agent, and the dose and
schedule one should use. Cases of thrombotic microangiopathy and
thrombotic events were reported when on average a cumulative amount of
>100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC)
was administered for 24 hours or more to patients receiving HEMLIBRA

HEMLIBRA may cause the following serious side effects when used with
®), including:

  • Thrombotic microangiopathy (TMA). This is a condition involving
    blood clots and injury to small blood vessels that may cause harm to
    one's kidneys, brain, and other organs. Patients should get medical
    help right away if they have any of the following signs or symptoms
    during or after treatment with HEMLIBRA:
    • confusion
    • weakness
    • swelling of arms and legs
    • yellowing of skin and eyes
    • stomach (abdomen) or back pain
    • nausea or vomiting
    • feeling sick
    • decreased urination
  • Blood clots (thrombotic events). Blood clots may form in blood
    vessels in one's arm, leg, lung or head. Patients should get medical
    help right away if they have any of these signs or symptoms of blood
    clots during or after treatment with HEMLIBRA:
    • swelling in arms or legs
    • pain or redness in the arms or legs
    • shortness of breath
    • chest pain or tightness
    • fast heart rate
    • cough up blood
    • feel faint
    • headache
    • numbness in the face
    • eye pain or swelling
    • trouble seeing

If aPCC (FEIBA®) is needed, patients should
talk to their healthcare provider in case they feel they need more than
100 U/kg of aPCC (FEIBA
®) total.

How should patients use HEMLIBRA?

HEMLIBRA may interfere with laboratory tests that measure how well blood
is clotting and may cause a false reading. Patients should talk to their
healthcare provider about how this may affect their care.

What are the other possible side effects of HEMLIBRA?

The most common side effects of HEMLIBRA include: redness,
tenderness, warmth, or itching at the site of injection; headache; and
joint pain.

These are not all of the possible side effects of HEMLIBRA. Patients
should call their doctor for medical advice about side effects.

Side effects may be reported to the FDA at (800) FDA-1088 or
Side effects may also be reported to Genentech at (888) 835-2555.

Please see the HEMLIBRA full Prescribing
and the Medication
, including Serious Side Effects, for more important
safety information.

About hemophilia A

Hemophilia A is an inherited, serious disorder in which a person's blood
does not clot properly, leading to uncontrolled and often spontaneous
bleeding. Hemophilia affects around 20,000 people in the United States,
with hemophilia A being the most common form and approximately 50-60
percent of people living with a severe form of the disorder.

People with hemophilia A either lack or do not have enough of a clotting
protein called factor VIII. In a healthy person, when a bleed occurs,
factor VIII brings together the clotting factors IXa and X, which is a
critical step in the formation of a blood clot to help stop bleeding.
Depending on the severity of their disorder, people with hemophilia A
can bleed frequently, especially into their joints or muscles. These
bleeds can present a significant health concern as they often cause pain
and can lead to chronic swelling, deformity, reduced mobility and
long-term joint damage.

About Genentech in hemophilia

In 1984, Genentech scientists were the first to clone recombinant factor
VIII in response to the contaminated hemophilia blood supply crisis of
the early 1980s. For more than 20 years, Genentech has been developing
medicines to bring innovative treatment options to people with diseases
of the blood within oncology, and in hemophilia A. Genentech is
committed to improving treatment and care in the hemophilia community by
delivering meaningful science and clinical expertise. For more
information visit

About Genentech

Founded more than 40 years ago, Genentech is a leading biotechnology
company that discovers, develops, manufactures and commercializes
medicines to treat patients with serious or life-threatening medical
conditions. The company, a member of the Roche Group, has headquarters
in South San Francisco, California. For additional information about the
company, please visit

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