Market Overview

Genentech's HEMLIBRA (emicizumab-kxwh) Every Four Weeks Controlled Bleeds in Phase III Study in Hemophilia A


– Less frequent administration of HEMLIBRA demonstrated clinically
meaningful bleed control –

Genentech, a member of the Roche Group ((SIX: RO, ROG, OTCQX:RHHBY),
announced today positive interim results from the Phase III HAVEN 4
study evaluating HEMLIBRA® (emicizumab-kxwh) prophylaxis
dosed once every four weeks in adults and adolescents (12 years of age
or older) with hemophilia A with and without inhibitors to factor VIII.
At this interim analysis after a median of 17 weeks of treatment,
HEMLIBRA prophylaxis showed a clinically meaningful control of bleeding.
These results are consistent with previous studies of HEMLIBRA dosed
once weekly or every two weeks, including the pivotal studies in
hemophilia A with inhibitors, HAVEN 1 in adults and adolescents and
HAVEN 2 in children, as well as the Phase III HAVEN 3 study in adults
and adolescents with hemophilia A without inhibitors. 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.

"Current treatment regimens for hemophilia A can require frequent
intravenous infusions. We are encouraged that HEMLIBRA prophylaxis
administered by injection under the skin once every four weeks showed
clinically meaningful bleed control in people with hemophilia A," said
Sandra Horning, M.D., chief medical officer and head of Global Product
Development. "Together with the findings from other Phase III studies,
these interim results support the potential for HEMLIBRA to be dosed at
different schedules, allowing patients to choose the option that's right
for them."

Data from the HAVEN 4 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 supporting that HEMLIBRA may benefit all people with hemophilia
A regardless of inhibitor status, while providing flexible and less
burdensome administration options.

About HAVEN 4 (NCT03020160)

HAVEN 4 is a single-arm, multicenter, open-label, Phase III study
evaluating the efficacy, safety and pharmacokinetics (PK) of
subcutaneous administration of HEMLIBRA dosed every four weeks. The
study included 48 patients (12 years of age or older) with hemophilia A
with or without inhibitors to factor VIII who were previously treated
with either factor VIII or bypassing agents, on-demand or as
prophylaxis. The study was conducted in two parts: a PK run-in; and an
expansion cohort. All patients in the PK run-in (n=7) were previously
treated on-demand, and received subcutaneous HEMLIBRA at 6 mg/kg to
fully characterize the PK profile after a single dose during four weeks,
followed by 6 mg/kg every four weeks for at least 24 weeks. Patients in
the expansion cohort (n=41) received subcutaneous HEMLIBRA prophylaxis
at 3 mg/kg/wk for four weeks, followed by 6 mg/kg every four weeks for
at least 24 weeks. Episodic treatment of breakthrough bleeds with factor
VIII therapy or bypassing agents, depending on a patient's inhibitor
status, was allowed per study 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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