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Sunday, Jul 12, 2020

New Data From Phase IIIb Study Reinforces Safety Profile of Genentech’s Hemlibra (emicizumab-kxwh) in People With Hemophilia A

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

South San Francisco, CA -- July 12, 2020 --

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.

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