Roche to Present Latest Advances in Hemophilia A Therapies at ISTH, Including Sustained Factor VIII Expression with Gene Therapy SPK-8011

Today, Roche announced that it will present the latest research progress on multiple therapies for hemophilia A at the annual meeting of the International Society on Thrombosis and Haemostasis (ISTH) to be held in July. This includes long-term follow-up data from gene therapy developed by its subsidiary Spark Therapeutics, as well as clinical trial data of Roche's bispecific antibody therapy Hemlibra in patients in the Asia-Pacific region.

Hemophilia A affects the lives of 320,000 patients worldwide, caused by genetic variations in the gene encoding coagulation Factor VIII, leading to insufficient levels of Factor VIII in the body. For these patients, even minor injuries can result in pain and potentially life-threatening bleeding. Patients with severe hemophilia often experience spontaneous bleeding into muscles and joints, which can cause permanent joint damage. Currently, the standard treatment for severe Hemophilia A is prophylactic intravenous infusion of coagulation Factor VIII. Patients not only require treatment 2–3 times per week, but breakthrough bleeding events may still occur despite therapy, significantly impacting their quality of life. Furthermore, some patients develop antibodies against exogenous Factor VIII (known as Factor VIII inhibitors) due to long-term infusions, thereby rendering Factor VIII replacement therapy ineffective.

SPK-8011, a gene therapy developed by Spark Therapeutics, a Roche company, is an adeno-associated virus (AAV) vector-based therapy that targets the liver to deliver a transgene expressing coagulation factor VIII. This marks the first update on this gene therapy since Roche’s acquisition of Spark Therapeutics. The press release noted that among the initial five patients treated with the gene therapy, the longest follow-up period has reached 142 weeks. Latest data show a 91% reduction in the annualized bleeding rate (ABR), with no evidence of declining factor VIII expression levels during follow-up periods exceeding two years. Furthermore, no patients developed inhibitors against coagulation factor VIII.

Meanwhile, Roche will announce for the first time the results of a Phase 3 clinical trial of its bispecific antibody therapy for hemophilia A, Hemlibra (emicizumab-kxwh), conducted in patients with hemophilia A in the Asia-Pacific region. Hemlibra was granted accelerated approval by China’s National Medical Products Administration (NMPA) in November 2018. This Phase 3 trial is a post-approval confirmatory study.

The trial results demonstrated that Hemlibra met the primary endpoint in 70 patients with hemophilia A and factor VIII inhibitors in the Asia-Pacific region. Prophylactic treatment with Hemlibra, administered either weekly or every four weeks, reduced the number of bleeding events requiring acute treatment by 96% compared to no prophylactic treatment (p<0.0001). The trial also met all key secondary endpoints. Overall, the trial data demonstrated that Hemlibra exhibited favorable efficacy and tolerability in this patient population.

“We are pleased to share the progress of our hemophilia A development program at the ISTH Annual Congress,” said Dr. Levi Garraway, Chief Medical Officer and Head of Global Product Development at Roche. “These data reflect our commitment to providing innovative treatment options for these patients.”

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