Interim Data From Phase III Study Presented at ASH 2022 Show Hemlibra (emicizumab-kxwh) Achieved Meaningful Bleed Control in Infants From Birth

– The HAVEN 7 study was designed to further confirm the benefit of preventative treatment (prophylaxis) with Hemlibra from birth in previously untreated or minimally treated infants with severe hemophilia A without inhibitors –

– 77.8% of participants had no bleeding episodes that required treatment –

– In addition, real-world efficacy and safety data from the EUHASS database and ATHN 7 study were also presented –

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced interim results from the Phase III HAVEN 7 study. The study shows Hemlibra® (emicizumab-kxwh) achieved meaningful bleed control with a favorable safety profile in infants (up to 12 months) with severe hemophilia A without factor VIII inhibitors: 77.8% of participants did not have any bleeds that required treatment and 42.6% did not have any treated or untreated bleeds at all. These results help support the use of Hemlibra in this population, in which it is already approved in many countries around the world. The new data were presented at the 64th American Society of Hematology (ASH) Annual Meeting and Exposition taking place in New Orleans from December 10-13, 2022.

The burden of severe hemophilia A in infants and on their parents and caregivers is significant. The World Federation of Hemophilia treatment guidelines consider the standard of care in hemophilia to be regular prophylaxis initiated at a young age, as studies have shown that early prophylaxis improves long-term outcomes, while reducing the risk of intracranial hemorrhage. However, for many infants with hemophilia A, prophylaxis is not started until after the first year of life because of the high treatment burden. Hemlibra provides a flexible treatment option that can be administered subcutaneously from birth at different dosing frequencies.

“These initial results support the benefit of starting Hemlibra from birth given that early preventative treatment is essential in infants,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “Hemophilia can substantially reduce quality of life for those affected, starting at infancy, which is especially distressing for parents and caregivers. We continue to explore Hemlibra's potential benefits to a broad range of people with hemophilia A.”

HAVEN 7 is a Phase III, multi-center, open-label study evaluating the efficacy, safety, pharmacokinetics and pharmacodynamics of Hemlibra in infants with severe hemophilia A without factor VIII inhibitors. The results of this interim analysis, which included data from 54 participants, showed that 77.8% of participants (n=42) did not have any bleeds which required treatment, while 42.6% (n=23) did not have any treated or untreated bleeds at all. There were no treated spontaneous bleeds in any participants, and all treated bleeds were traumatic. A total of 77 bleeds occurred in 31 participants (57.4%); 88.3% were traumatic. Mean model-based annualized bleeding rate (ABR) at the time of interim analysis was 0.4 (95% CI: 0.23–0.65) for treated bleeds.

Hemlibra’s safety profile was consistent with previous studies, with no new safety signals observed. Nine people (16.7%) reported a Hemlibra-related adverse event (AE), all of which were local injection site reactions. Eight participants (14.8%) reported 12 serious AEs, unrelated to Hemlibra. There were no deaths, thromboembolic events or cases of thrombotic microangiopathy, reinforcing Hemlibra’s favorable safety profile. No intracranial hemorrhages occurred.

Primary analysis will be conducted at 52 weeks. The study also has an additional seven-year follow-up period to collect long-term data such as safety and joint health outcomes, further building upon our understanding of the benefit of Hemlibra in this population.

EUHASS Database and ATHN 7 Study

Genentech also presented data from the European Haemophilia Safety Surveillance (EUHASS) database and the prospective observational ATHN 7 study at ASH 2022. Data from EUHASS, which collects real-world safety data on treatments for inherited disorders, showed the safety profile of Hemlibra in people with hemophilia A was favorable and consistent with clinical trial data. Data from ATHN 7, exploring the efficacy of Hemlibra in women with hemophilia A, showed two of the three female participants had no bleeds; the third had one treated bleed associated with a dental procedure and one untreated bleed associated with menses. Ongoing evaluation is vital to further understand the safety and efficacy profile of Hemlibra in this rare and under-represented population.

Hemlibra is approved as a treatment for people with hemophilia A with factor VIII inhibitors in more than 110 countries worldwide, and for people without factor VIII inhibitors in more than 100 countries worldwide. It has been studied in one of the largest clinical trial programs in people with hemophilia A with and without factor VIII inhibitors, including eight Phase III studies.

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. People who use activated prothrombin complex concentrate (aPCC; Feiba^®) to treat breakthrough bleeds while taking Hemlibra may be at risk of serious side effects related to blood clots.

These serious side effects include:

• Thrombotic microangiopathy (TMA), a condition involving blood clots and injury to small blood vessels that may cause harm to one’s kidneys, brain, and other organs
• Blood clots (thrombotic events), which may form in blood vessels in the arm, leg, lung, or head

Patients should talk to their doctor about the signs and symptoms of these serious side effects, which can include

• Confusion
• Stomach, chest, or back pain
• Weakness
• Nausea or vomiting
• Swelling, pain, or redness
• Feeling sick or faint
• Decreased urination
• Swelling of arms and legs
• Yellowing of skin and eyes
• Eye pain, swelling, or trouble seeing
• Fast heart rate
• Numbness in your face
• Headache
• Shortness of breath
• Coughing up blood

If patients experience any of these symptoms during or after treatment with Hemlibra, they should get medical help right away.

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. 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.

Patients’ bodies may make antibodies against Hemlibra, which may stop Hemlibra from working properly. Patients should contact their healthcare provider immediately if they notice that Hemlibra has stopped working for them (e.g., increase in bleeds).

The most common side effects of Hemlibra include: injection site reactions (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 can speak with their healthcare provider for more information.

What else should patients know about 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.

• Patients should stop taking their prophylactic bypassing therapy the day before they start Hemlibra
• Patients may continue taking their prophylactic factor VIII for the first week of Hemlibra

Hemlibra may interfere with laboratory tests that measure how well blood is clotting and create an inaccurate result. Patients should speak with their healthcare provider about how this may affect their care.

Medicines are sometimes prescribed for purposes other than those listed in a Medication Guide. Patients should only use Hemlibra for the condition it was prescribed. Patients should not give Hemlibra to other people, even if they have the same symptoms that they have. It may harm them.

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.

Before using Hemlibra, patients should tell their healthcare provider about all of their medical conditions, including if they are pregnant, plan to become pregnant, are breastfeeding, or plan to breastfeed.

Since Hemlibra was tested in males, there is no information on whether Hemlibra may impact an unborn baby or breast milk. Females who are able to become pregnant should use birth control during treatment.

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

Please see Important Safety Information, including Serious Side Effects, as well as the Hemlibra full Prescribing Information and Medication Guide.

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% 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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