Interim Data From Phase III HAVEN 6 Study Demonstrate Favorable Safety and Efficacy Profile of Genentech’s Hemlibra (emicizumab-kxwh) in People With Moderate or Mild Hemophilia A

– People with moderate or mild hemophilia A have significant unmet clinical needs, as this population may not use preventative treatments due to missed or delayed diagnoses of bleeding episodes and a lack of treatment guidelines –

– New data indicate that Hemlibra has a favorable safety profile in people with moderate or mild hemophilia A without factor VIII inhibitors, with no new safety signals identified –

– Hemlibra also achieved clinically meaningful bleed control, with 80.3% of participants experiencing no bleeding episodes that required treatment and 90.1% experiencing no joint bleeds that required treatment –

– A separate analysis of thrombosis-related events in people taking Hemlibra, including real-world data, further confirmed the safety profile of Hemlibra –

Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced results from an interim analysis of the Phase III HAVEN 6 study, which show Hemlibra^® (emicizumab-kxwh) demonstrated a favorable safety profile and effective bleed control in people with moderate or mild hemophilia A without factor VIII inhibitors. The data were presented at the 63rd American Society of Hematology (ASH) Annual Meeting and Exposition as an oral presentation on December 12, 2021.

While the treatment and management of severe hemophilia A are well established, there is less information and treatment guidance on moderate and mild hemophilia A, which can lead to delayed or missed diagnoses of bleeding episodes. Considering this population may not use preventative treatments, these patients may experience worsened clinical burden, with less than 30% of people with moderate or mild hemophilia A living a bleed-free life.

“We are pleased to see that Hemlibra continues to show benefit in additional hemophilia A populations, regardless of severity,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “The clinical evidence for Hemlibra derives from one of the largest pivotal clinical trial programs in hemophilia A, with and without factor VIII inhibitors. We remain committed to working together with the hemophilia community as we further explore the efficacy and safety of Hemlibra in broader populations.”

HAVEN 6 is a Phase III study evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with moderate or mild hemophilia A without factor VIII inhibitors. This interim analysis included data from 71 participants (69 men and two women), 20 of whom had mild hemophilia A without factor VIII inhibitors and 51 of whom had moderate hemophilia A without factor VIII inhibitors. Thirty-seven participants were on factor VIII prophylaxis at baseline.

This interim analysis was conducted after 50 participants with moderate hemophilia A completed at least 24 weeks in the study or withdrew. Data cutoff was on April 16, 2021. These data show Hemlibra demonstrated a favorable safety profile and effective bleed control in the HAVEN 6 study, with 80.3% of participants experiencing no bleeding episodes that required treatment and 90.1% experiencing no joint bleeds that required treatment. Annualized bleeding rates (ABR) remained low, consistent with previously reported observations from the HAVEN 1-4 studies. In addition, of the 50 participants aged 12 years or older who responded to the EmiPref questionnaire, 48 (96.0%) preferred Hemlibra to their previous treatment, one preferred their old treatment, and one expressed no preference.

The most common adverse events (AEs) occurring in 10% or more people in the HAVEN 6 study were headache (14.1%) and local injection site reactions (ISRs) (12.7%). Eleven people (15.5%) reported a Hemlibra-related AE, with ISRs being the most common (12.7%). There were no deaths, or cases of thrombotic microangiopathy (TMA) or serious thrombotic events (TEs) in the study as of the data cutoff, reinforcing Hemlibra’s favorable safety profile.

A separate analysis of TE and TMA events in people taking Hemlibra, including real-world data, will also be presented as a poster at ASH. These results showed that the evaluation of reported events without concomitant activated prothrombin complex concentrate (aPCC) remains similar to previous analyses as exposure increases, and the benefit/risk profile of Hemlibra remains unchanged. These data further confirm the favorable safety profile of Hemlibra, consistent with results from previous HAVEN and STASEY studies.

Hemlibra is approved to treat people with hemophilia A with factor VIII inhibitors in more than 100 countries worldwide and people without factor VIII inhibitors in more than 90 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 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. 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% 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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