– Second interim analysis of the STASEY study,
including data from 193 patients, consistent with results from
Phase III HAVEN studies, with no new safety signals identified
–
– STASEY is the largest open-label study
primarily assessing safety and tolerability of a medicine for
hemophilia A with factor VIII inhibitors –
– A separate analysis also suggests people on
Hemlibra may be able to undergo certain minor surgeries without
additional preventative coagulation treatment and major surgeries
could be managed with additional prophylactic coagulation factor
–
Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX:
RHHBY), today announced results from the second interim analysis of
the Phase IIIb STASEY study, which reinforce the safety profile of
Hemlibra® (emicizumab-kxwh) characterized in the Phase III HAVEN
clinical program. In the STASEY study, Hemlibra was effective with
no new safety signals identified in adults and adolescents with
hemophilia A with factor VIII inhibitors, which was consistent with
previous safety observations. Further new interim data suggest that
people on Hemlibra may be able to undergo certain minor surgeries
without additional preventative (prophylactic) coagulation factor.
These data were presented at the International Society on
Thrombosis and Haemostasis (ISTH) 2020 Virtual Congress, July
12-14, 2020.
“These important safety data continue to add to the extensive
clinical evidence reinforcing Hemlibra’s potential to redefine the
standard of care for people with hemophilia A,” said Levi Garraway,
M.D., Ph.D., chief medical officer and head of Global Product
Development. “The STASEY study reflects our continued focus on
providing valuable insights that meet the needs of the hemophilia
community and enhance our understanding of Hemlibra in clinical
practice.”
The second interim analysis of the STASEY study included data
from 193 patients with hemophilia A with factor VIII inhibitors,
who received Hemlibra prophylaxis once-weekly. No cases of
thrombotic microangiopathy or serious thrombotic events (TEs)
related to Hemlibra were reported, and no new safety signals were
observed. Thirty-three (17.1%) people reported a Hemlibra-related
adverse event (AE). The most common AEs, occurring in 10% or more
of people in the STASEY study, were common cold symptoms
nasopharyngitis; (12.4%), headache (11.9%) and injection site
reactions (ISRs) (11.4%). The ISRs reported were either mild or
moderate in severity and no patients discontinued due to ISR.
Annualized bleeding rates (ABR) were also consistent with
previously reported observations from the Phase III HAVEN
studies.
A separate analysis described management and outcomes of minor
and unplanned major surgeries in patients receiving Hemlibra,
although there was not a formal surgery endpoint in STASEY. Results
suggest people with hemophilia A with factor VIII inhibitors who
undergo certain minor surgeries while receiving Hemlibra may not
need additional preventative (prophylactic) coagulation factor. The
majority of minor surgeries (n=20/31) were performed without the
use of prophylactic coagulation factor (64.5%) and, of these, 85%
(n=17/20) did not result in treated post-operative bleeds. Of the
unplanned major surgeries (n=9), eight were managed with
prophylactic coagulation factor, four of which resulted in bleeds
managed successfully with recombinant factor VIIa. These findings
are consistent with results observed in a previous analysis of
surgeries in the pivotal HAVEN studies.
STASEY is a single-arm, multicenter, open-label, Phase IIIb
clinical study where patients received Hemlibra for an average of
50.9 weeks. The ABR of all bleeds, including treated bleeds,
treated spontaneous bleeds, treated joint bleeds and treated target
joint bleeds were low, with 167 patients (85.6%) experiencing zero
treated bleeds. In the STASEY study there were two TEs unrelated to
Hemlibra reported. One was a ST-elevation myocardial infarction in
a person with pre-existing risk factors, which the treating
physician assessed as unrelated to Hemlibra. The second was a
hypertrophic clot at the site of a tooth extraction, a known
complication of the procedure.
Hemlibra is approved to treat people with hemophilia A with
factor VIII inhibitors in more than 90 countries worldwide and for
people without factor VIII inhibitors in more than 70 countries
worldwide, including the U.S., EU and Japan. Hemlibra has been
studied in one of the largest clinical trial programs in people
with hemophilia A with and without factor VIII inhibitors,
including five completed Phase III studies. The STASEY study is the
largest open-label study primarily assessing the safety and
tolerability of a medicine for people with hemophilia A with factor
VIII inhibitors.
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, every two weeks or every four
weeks. Hemlibra was created by Chugai Pharmaceutical Co., Ltd. and
is being co-developed globally 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, ages newborn and older, with hemophilia A with or
without factor VIII inhibitors.
Important Safety Information
What is the most important information to know about
Hemlibra?
Hemlibra increases the potential for blood to clot. Patients
should carefully follow their healthcare provider’s instructions
regarding when to use an on-demand bypassing agent or factor VIII,
and the dose and schedule to use for breakthrough bleed treatment.
Hemlibra may cause the following serious side effects when used
with activated prothrombin complex concentrate (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 the 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.
Patients should tell their healthcare provider about all the
medicines they take, including prescription medicines,
over-the-counter medicines, vitamins, or herbal supplements.
Patients should keep a list of them to show their healthcare
provider and pharmacist when they get a new medicine.
How should patients use Hemlibra?
Patients should see the detailed “Instructions for Use” that
comes with Hemlibra for information on how to prepare and inject a
dose of Hemlibra, and how to properly throw away (dispose of) used
needles and syringes.
- Stop (discontinue) prophylactic use of bypassing agents the
day before starting Hemlibra prophylaxis.
- Patients may continue prophylactic use of factor VIII for
the first week of Hemlibra prophylaxis.
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 speak to their healthcare provider for medical
advice about side effects.
Medicines are sometimes prescribed for purposes other than those
listed in a Medication Guide. Patients should not use Hemlibra for
a condition for which it was not prescribed. Patients should not
give Hemlibra to other people, even if they have the same symptoms
that they have. It may harm them. Patients can ask their pharmacist
or healthcare provider for information about Hemlibra that is
written for health professionals.
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
Medication Guide for more important safety information including
Serious Side Effects.
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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20200712005037/en/
Media Contact: Adam Pryor (650) 467-6800
Advocacy Contact: Sonali Chopra (650) 467-0842
Investor Contacts: Loren Kalm (650) 225-3217 Karl Mahler 011 41
61 687 8503