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

Rhea-AI Summary

Genentech announced positive results from the Phase III HAVEN 6 study of Hemlibra (emicizumab-kxwh), showing 66.7% of participants with moderate or mild hemophilia A experienced zero treated bleeds over a median follow-up of 55.6 weeks. The study demonstrated Hemlibra's favorable safety profile, with no new safety signals reported. Hemlibra is approved for treating hemophilia A in over 110 countries. The findings will support a submission to the European Medicines Agency for expanding the label to include non-severe hemophilia A patients.

Positive

• 66.7% of study participants experienced zero treated bleeds at 55.6 weeks follow-up.
• No new safety signals reported for Hemlibra.
• Hemlibra is approved in over 110 countries for treating hemophilia A.

Negative

• Limited treatment guidance may lead to delayed diagnoses of bleeding episodes.
• More than 30% of adults and 40% of children with moderate hemophilia A did not receive preventative treatments.

– Hemlibra continues to demonstrate clinically meaningful bleed control, with 66.7% of participants with moderate or mild hemophilia A experiencing zero treated bleeds at 55.6 weeks median follow-up –

– New data also reinforce Hemlibra’s favorable safety profile, with no new safety signals observed –

– There is limited information and treatment guidance on moderate and mild hemophilia A, which can lead to delayed or missed diagnoses of bleeding episodes –

– Hemlibra is approved to treat people of all ages with hemophilia A with factor VIII inhibitors in more than 110 countries and for people of all ages without factor VIII inhibitors in more than 95 countries –

SOUTH SAN FRANCISCO, Calif.--(BUSINESS WIRE)-- Genentech, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), today announced results from the primary analysis of the Phase III HAVEN 6 study, which show that Hemlibra^® (emicizumab-kxwh) continued to demonstrate a favorable safety profile and effective bleed control in people with moderate or mild hemophilia A, without factor VIII inhibitors. The data will be presented at the 30th International Society on Thrombosis and Haemostasis (ISTH) Annual Congress, on July 11, 2022, in London, United Kingdom, and are planned to support a submission to the European Medicines Agency to update the label for Hemlibra to include non-severe hemophilia A patients.

“We are proud that Hemlibra continues to redefine the standard of care for more people living with hemophilia A,” said Levi Garraway, M.D., Ph.D., chief medical officer and head of Global Product Development. “The data presented at ISTH this year underscore Genentech’s commitment to addressing gaps in care for hemophilia A, thereby ensuring that broader populations can potentially benefit from Hemlibra.”

In addition to HAVEN 6, data from the CHESS II (Cost of Haemophilia across Europe: a Socioeconomic Survey-II) and CHESS PAEDs studies will also be presented at ISTH 2022. These data show most adults with moderate or mild hemophilia A and more than half of children with moderate hemophilia A may not receive preventative treatments. This can result in worsened clinical burden, as more than 30% of adults and approximately 40% of children with moderate hemophilia A who were not taking preventative treatment in the study experienced at least three bleeds a year.

HAVEN 6 is a Phase III, multicenter, open-label, single-arm study evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of Hemlibra in people with moderate or mild hamophilia A without factor VIII inhibitors. The primary analysis included data from 72 participants (69 men and three women) who warranted prophylaxis; 21 had mild hemophilia A without factor VIII inhibitors and 51 had moderate hemophilia A without factor VIII inhibitors at a median follow-up of 55.6 weeks. At baseline, 37 participants were receiving factor VIII prophylactic treatment and 24 had target joints.

The data show that Hemlibra maintained low treated bleed rates across the study period, with 66.7% of participants experiencing no bleeds that required treatment, 81.9% experiencing no spontaneous bleeds that required treatment, and 88.9% experiencing no joint bleeds that required treatment. Model-based annualized bleed rates (ABR) remained low throughout the evaluation period at 0.9 (95% CI: 0.55-1.52).

The results also show that Hemlibra’s safety profile was consistent with findings across various subpopulations of people with hemophilia A, from previous HAVEN and STASEY studies, with no new safety signals observed. The most common adverse event (AE) related to treatment occurring in 10% or more people in the HAVEN 6 study was local injection site reactions (ISRs) (16.7%). Fifteen people (20.8%) reported a Hemlibra-related AE, of which the majority were local ISRs. One participant experienced a grade one thromboembolic event unrelated to Hemlibra. There were no deaths or cases of thrombotic microangiopathy, reinforcing Hemlibra’s favorable safety profile.

Hemlibra is approved to treat people with hemophilia A with factor VIII inhibitors in more than 110 countries worldwide and for people without factor VIII inhibitors in more than 95 countries worldwide, including the U.S. and Japan for all severities of hemophilia A, and the EU for only severe hemophilia A. 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 (eg, 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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Source: Genentech

FAQ

What were the results of the Phase III HAVEN 6 study for Hemlibra (RHHBY)?

The Phase III HAVEN 6 study showed that 66.7% of participants with moderate or mild hemophilia A experienced no treated bleeds over 55.6 weeks.

What safety profile does Hemlibra demonstrate in the HAVEN 6 study (RHHBY)?

Hemlibra maintained a favorable safety profile with no new safety signals reported in the HAVEN 6 study.

In how many countries is Hemlibra approved for treatment (RHHBY, CHGCY)?

Hemlibra is approved in over 110 countries for treating hemophilia A with factor VIII inhibitors.

What implications do the HAVEN 6 study findings have for hemophilia A treatment (RHHBY)?

The findings will support a submission to the European Medicines Agency to expand Hemlibra’s label to include patients with non-severe hemophilia A.