Company Announcements

Genentech Presents a Broad Range of Data for Hemlibra (Emicizumab-kxwh) Demonstrating Continued Benefits for People With Hemophilia A at the ISTH 2019 Congress

– New analyses from Phase III HAVEN studies support Hemlibra’s sustained efficacy, safety and quality of life benefit in people with hemophilia A, with and without factor VIII inhibitors –

– First data of Phase IIIb STASEY study reinforces safety profile of Hemlibra seen in pivotal HAVEN 1 clinical trial –

– New analysis of pivotal data suggests additional factor treatment may not be needed for people on Hemlibra undergoing certain minor surgeries –

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)--Jul. 9, 2019-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced today new data for Hemlibra® (emicizumab-kxwh) across multiple pivotal studies in people with hemophilia A with and without factor VIII inhibitors at the International Society on Thrombosis and Haemostasis (ISTH) 2019 Congress on July 6-10 in Melbourne, Australia. In total, Genentech presented 21 abstracts from its hemophilia program, including five oral presentations. Further data from the four pivotal HAVEN clinical trials were presented, demonstrating the long-term safety, efficacy and quality of life benefit of Hemlibra in people with hemophilia A with and without factor VIII inhibitors. Genentech also presented the first interim analysis from the Phase IIIb STASEY study, reinforcing the safety profile of Hemlibra in adults and adolescents (aged 12 years or older) with hemophilia A with factor VIII inhibitors seen in the HAVEN 1 clinical trial.

“Data presented at ISTH continues to reinforce Hemlibra’s potential to redefine the standard of care for people living with hemophilia A,” said Sandra Horning, M.D., chief medical officer and head of Global Product Development. “We are particularly excited to present the first interim analysis of safety data from the STASEY study, which adds to the growing body of evidence supporting Hemlibra as an important treatment option for people with hemophilia A.”

Long-term efficacy, safety and quality of life data show sustained benefit of Hemlibra

Updated data from the pooled HAVEN studies (HAVEN 1, HAVEN 2, HAVEN 3 and HAVEN 4; n=400), in people with hemophilia A of all ages with and without factor VIII inhibitors, showed that a high proportion of patients experienced zero treated bleeds on Hemlibra, and that this was maintained over a median of 83 weeks. Across all four HAVEN studies, over 87 percent of participants had no treated joint bleeds (either spontaneous or due to injury/trauma) and over 92 percent of participants experienced no spontaneous bleeds in each interval from week 25. Hemlibra’s established safety and tolerability profile was maintained.

Additionally, updated data from the HAVEN 3 and HAVEN 4 studies demonstrate that Hemlibra prophylaxis offers a clinically meaningful improvement in long-term health-related quality of life, versus previous episodic or prophylactic factor VIII treatment, for people with hemophilia A with and without factor VIII inhibitors as measured by the Haem-A-QoL questionnaire. In the 28 days prior to starting treatment with Hemlibra, 76 percent and 79 percent of employed patients from HAVEN 3 and HAVEN 4 studies, respectively, reported no missed days of work. At week 25 of HAVEN 3 and HAVEN 4, 91 percent and 93 percent of participants reported no missed workdays, respectively, with these figures remaining stable thereafter.

Interim data from the STASEY study reinforce Hemlibra’s safety profile

Results from the first interim analysis of the Phase IIIb STASEY study, including data from 88 patients, reinforce the safety profile of Hemlibra characterized in the pivotal HAVEN 1 study. HAVEN 1 has formed the basis of Hemlibra’s approval in people with hemophilia A with factor VIII inhibitors in over 70 countries worldwide to date. In the STASEY study, in people with hemophilia A with factor VIII inhibitors, no cases of thrombotic microangiopathy or thrombotic events were reported and no new safety signals were identified. Eighteen (20.5 percent) patients reported a Hemlibra-related adverse event (AE), of which one was a serious AE (catheter site abscess). The most common AEs, occurring in 10 percent or more of people in the STASEY study were injection site reactions (14.8 percent), joint pain (arthralgia; 13.6 percent), headache (11.4 percent) and common cold symptoms (nasopharyngitis; 11.4 percent). Bleeding rates in people with hemophilia A with factor VIII inhibitors receiving Hemlibra in the STASEY study were also in line with previously reported observations from the HAVEN 1 study.

Additional factor treatment may not be needed for people on Hemlibra undergoing certain minor surgeries

A retrospective analysis of pooled data across the HAVEN studies suggests that people with hemophilia A with and without factor VIII inhibitors may not need additional preventative (prophylactic) coagulation factor (factor VIII replacement therapy or bypassing agents) when undergoing certain minor surgeries. The majority of minor surgeries (n=215) were performed without prophylactic coagulation factor (n=141; 65.6 percent), and of these, 90.8 percent did not result in a treated post-operative bleed. Of the 18 major surgeries, three were managed without prophylactic coagulation factor, with no post-operative bleeds. The remaining 15 major surgeries were managed with prophylactic coagulation factor, only one of which resulted in a treated post-operative bleed.

Hemlibra regulatory status

Hemlibra is approved for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in people with hemophilia A with factor VIII inhibitors in over 70 countries worldwide, based on the results of the pivotal HAVEN 1 and HAVEN 2 studies. This includes the U.S. in November 2017, EU member states in February 2018 and Japan in March 2018. Hemlibra has also been approved in people with hemophilia A without factor VIII inhibitors in over 40 countries worldwide, based on results from the pivotal HAVEN 3 and HAVEN 4 studies. This includes the U.S. in October 2018, EU member states in March 2019 and Japan in December 2018. Submissions to other regulatory authorities around the world are ongoing.

About the HAVEN clinical trial program

The HAVEN clinical trial program is one of the largest pivotal clinical trial programs in hemophilia A, designed to assess the efficacy and safety of Hemlibra in people with and without factor VIII inhibitors, and its potential to help overcome current clinical challenges: the short-lasting effects of existing treatments, the development of factor VIII inhibitors and the need for frequent venous access.

  • HAVEN 1 is a randomized, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of once-weekly subcutaneous administration of Hemlibra prophylaxis compared to no prophylaxis in adults and adolescents (12 years of age and older) with hemophilia A with inhibitors to factor VIII, who were previously treated with bypassing agents on-demand or as prophylaxis.
  • HAVEN 2 is a multicenter, open-label, clinical study in children younger than 12 years of age with hemophilia A with factor VIII inhibitors. The study is evaluating the efficacy, safety and pharmacokinetics of once-weekly, every two weeks or every four weeks subcutaneous administration of Hemlibra prophylaxis.
  • HAVEN 3 is a randomized, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of Hemlibra prophylaxis once weekly or every two weeks versus no prophylaxis (episodic/on-demand factor VIII treatment) in people (12 years of age or older) with hemophilia A without factor VIII inhibitors, who were previously treated with factor VIII therapy either on-demand or as prophylaxis.
  • HAVEN 4 is a single-arm, multicenter, open-label, Phase III study evaluating the efficacy, safety and pharmacokinetics of subcutaneous administration of Hemlibra dosed every four weeks. The study included adults and adolescents (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.

About STASEY

STASEY is a single-arm, multicenter, open-label, Phase IIIb clinical trial to evaluate the safety and tolerability of Hemlibra prophylaxis in people with hemophilia A with factor VIII inhibitors. The study included 88 patients (12 years of age or older) who had completed 24 weeks on study or discontinued, receiving subcutaneous Hemlibra 3 mg/kg/week for four weeks, followed by 1.5 mg/kg/week for the remainder of the treatment period. In the STASEY study:

  • No cases of thrombotic microangiopathy or thrombotic events were reported.
  • Eighteen (20.5 percent) patients reported a Hemlibra-related adverse event, of which one was a serious AE (catheter site abscess). The most common adverse events (AE) occurring in 10 percent or more of people in the STASEY study were injection site reactions (14.8 percent), joint pain (arthralgia; 13.6 percent), headache (11.4 percent) and common cold symptoms (nasopharyngitis; 11.4 percent).
  • The rates of treated, all, spontaneous, joint and target joint bleeds were low, with 71 patients experiencing zero treated bleeds (80.7 percent).
  • Of 17 patients who received treatment for a spontaneous or traumatic bleed, 16 received recombinant factor VIIa and one received factor VIII. No thrombotic events or thrombotic microangiopathy were seen with concomitant bypassing agents or factor VIII.

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.

Source: Genentech

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