Market Overview

Genentech to Present New Phase III Data for HEMLIBRA (emicizumab-kxwh) in People With Hemophilia A at the World Federation of Hemophilia 2018 World Congress

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– Data include results from HAVEN 3 study in people with hemophilia A
without factor VIII inhibitors and HAVEN 4 study in people with
hemophilia A with or without factor VIII inhibitors –

– Ongoing HEMLIBRA clinical development program demonstrates
commitment to advancing care for all people with hemophilia A –

Genentech, a member of the Roche Group ((SIX: RO, ROG, OTCQX:RHHBY),
announced today that Phase III results for HEMLIBRA®
(emicizumab-kxwh) will be presented for the first time during the World
Federation of Hemophilia (WFH) 2018 World Congress from May 20-24 in
Glasgow, Scotland. The late-breaking presentations include positive
results from the pivotal HAVEN 3 study of HEMLIBRA dosed every week or
every two weeks in people with hemophilia A without factor VIII
inhibitors and the pivotal HAVEN 4 study of HEMLIBRA dosed every four
weeks in people with hemophilia A with or without factor VIII
inhibitors. These data support the promising potential of HEMLIBRA for
all people with hemophilia A.

"We look forward to sharing these new results from the HAVEN 3 and HAVEN
4 studies, which demonstrate the potential of HEMLIBRA to redefine
treatment expectations for people with hemophilia A with and without
inhibitors to factor VIII," said Sandra Horning, M.D., chief medical
officer and head of Global Product Development. "We will also be sharing
real-world data that provides new insight into the impact of hemophilia
A treatment on daily life, as part of our ongoing commitment to
advancing management and care for the global hemophilia community."

Data from the HAVEN 3 and HAVEN 4 studies will be presented for the
first time in late-breaking oral presentations on Monday, May 21. The
HAVEN 3 presentation will highlight new data on HEMLIBRA prophylaxis
administered every week or every two weeks in people 12 years of age or
older with hemophilia A without factor VIII inhibitors compared to no
prophylaxis. The presentation will also include results from an
intra-patient analysis comparing HEMLIBRA prophylaxis to prior treatment
with factor VIII prophylaxis. The U.S. Food and Drug Administration
(FDA) recently granted Breakthrough Therapy Designation for HEMLIBRA in
people with hemophilia A without factor VIII inhibitors based on data
from this study. The HAVEN 4 presentation will highlight primary data in
people 12 years of age or older with hemophilia A with or without factor
VIII inhibitors receiving HEMLIBRA prophylaxis every four weeks.

These presentations at WFH follow the announcement of positive top-line
results from the HAVEN 3 study in November 2017 and positive top-line
interim results from the HAVEN 4 study in December 2017. Data from both
studies are being submitted to health authorities around the world for
approval consideration.

Genentech will also present real-world data from a non-interventional
study in adults with hemophilia A without factor VIII inhibitors and
children with hemophilia A with factor VIII inhibitors. These data on
health-related quality of life and health status will provide insights
into challenges of living with and managing hemophilia A for patients
and caregivers.

Follow Genentech on Twitter via @Genentech and keep up to date with WFH
2018 World Congress news and updates by using the hashtag #WFH2018.

Overview of Genentech data at the WFH 2018 World Congress

         
Abstract title       Abstract number/Presentation details

Emicizumab prophylaxis administered once-weekly or every two weeks
provides effective bleed prevention in persons with hemophilia A
(PwHA) without inhibitors – Results from the Phase III HAVEN 3
study

     

#854
Free Papers: Late-Breaking
Oral
presentation

Monday, May 21
11:15–11:30
a.m. BST

Emicizumab subcutaneous dosing every 4 weeks is safe and efficacious
in the control of bleeding in persons with hemophilia A (PwHA) with
and without inhibitors: Results from Phase 3 HAVEN 4 study
     

#861
Free Papers: Late-Breaking
Oral
presentation

Monday, May 21
11:30–11:45
a.m. BST

New option of hemostatic treatment for acquired hemophilia A by
single injection of emicizumab, FVIIIa mimicking bispecific
antibody, irrespective of epitope specificity
     

#2
Poster presentation
Monday, May 21
9:45–10:15
a.m. BST and 3:45–4:30 p.m. BST

Impaired thrombus formation of von Willebrand Disease under high
shear flow condition is improved by Factor VIIIa mimetic bispecific
antibody (emicizumab)
     

#4
Free Papers: Young Researchers
Oral
presentation

Wednesday, May 23
5:15–5:30
p.m. BST

Changes in bleeding and daily life with emicizumab prophylaxis: a
questionnaire in patients with hemophilia A with inhibitors
(PwHAwI) and their families in a long-term Phase 1/2 study

     

#82
Poster presentation
Monday, May 21
9:45–10:15
a.m. BST and 3:45–4:30 p.m. BST

Health-related quality of life and health status in persons with
hemophilia A (PwHA) without inhibitors: prospective,
non-interventional study (NIS) from a real-world setting
     

#165
Poster presentation
Monday, May 21
9:45–10:15
a.m. BST and 3:45–4:30 p.m. BST

Health-related outcomes and caregiver burden in pediatric persons
with hemophilia A (PwHA) with inhibitors: prospective,
non-interventional study (NIS) in a real-world setting
     

#171
Poster presentation
Monday, May 21
9:45–10:15
a.m. BST and 3:45–4:30 p.m. BST

Assessment of depressive disorders among people with hemophilia A
with inhibitors
     

#159
Poster presentation
Wednesday, May
23

3:45–4:30 p.m. BST

Agreement between a chromogenic modified Nijmegen-Bethesda Assay and
a qualitative ELISA test in detection of Factor VIII inhibitors in
plasma from Persons with Hemophilia A (PwHA)
     

#59
Poster presentation
Monday, May 21
9:45–10:15
a.m. BST and 3:45–4:30 p.m. BST

     

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 factor VIII inhibitors.
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 for at least 24 weeks
(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.

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 factor VIII inhibitors 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 factor VIII inhibitor status,
was allowed per study protocol.

About HEMLIBRA

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

What is the most important safety information to know about HEMLIBRA?
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 prophylaxis.

HEMLIBRA may cause the following serious side effects when used with
aPCC (FEIBA
®), 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.

Before using HEMLIBRA, patients should tell their healthcare provider
about all of their medical conditions, including if they:

  • are pregnant or plan to become pregnant. It is not known if HEMLIBRA
    may harm an unborn baby. Females who are able to become pregnant
    should use birth control (contraception) during treatment with
    HEMLIBRA.
  • are breastfeeding or plan to breastfeed. It is not known if HEMLIBRA
    passes into breast milk.

What should patients know about lab monitoring?

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.

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 http://www.fda.gov/medwatch.
Side effects may also be reported to Genentech at (888) 835-2555.

Please see the HEMLIBRA full Prescribing
Information
and the Medication
Guide
, 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.

A serious complication of treatment is the development of inhibitors to
factor VIII replacement therapies. Inhibitors are antibodies developed
by the body's immune system that bind to and block the efficacy of
replacement factor VIII, making it difficult, if not impossible, to
obtain a level of factor VIII sufficient to control bleeding.

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 http://www.gene.com/hemophilia.

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 and 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 http://www.gene.com.

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