Roche announces new data at the ISTH 2020 Congress, demonstrating ongoing commitment to advancing care for people with haemophilia A – GlobeNewswire

June 30th, 2020 5:49 am

Basel, 29 June 2020 - Roche (SIX: RO, ROG; OTCQX: RHHBY) today announced that new data from its haemophilia A clinical programme will be presented at the International Society on Thrombosis and Haemostasis (ISTH) 2020 Congress on 12-14 July 2020. Data will include updated safety results from the phase IIIb STASEY study of Hemlibra (emicizumab) and new results from the phase III HAVEN 5 study of Hemlibra. Data will also include insights into the impact of living with haemophilia A. Spark Therapeutics (a member of the Roche Group) will also present data from the initial dose cohorts of its phase I/II SPK-8011 gene therapy study.

We are excited to share updated data from our combined haemophilia A programme at this years virtual ISTH 2020, said Levi Garraway, M.D., Ph.D., Chief Medical Officer and Head of Global Product Development. These data exemplify our efforts to increase our knowledge and capabilities in haemophilia A, including in the context of gene therapy, with the goal of advancing care and providing innovative treatment approaches for people living with this chronic condition.

SPK-8011 data presentationData from the initial dose cohorts of Sparks phase I/II SPK-8011 gene therapy study in haemophilia A will be presented at the congress. Updated data from five participants in the initial dose cohorts, who are up to 142 weeks post-vector infusion, show stable and durable factor VIII expression and a 91% reduction in annualised bleed rate (ABR). There is no evidence of decline in factor VIII expression after more than two years of follow up.1

These data indicate an acceptable safety profile, with no development of factor VIII inhibitors. Furthermore, they represent the longest stable expression of factor VIII following gene transfer and support the use of adeno-associated virus-mediated (AAV-mediated), liver directed gene therapy to achieve durable factor VIII expression for the treatment of haemophilia A.1

Key Hemlibra data presentationsData for Hemlibra will be featured in four poster presentations at the congress. This further supports the comprehensive body of clinical evidence available for Hemlibra, including from the HAVEN studies the most extensive clinical development programme in haemophilia A. This includes results from the second interim analysis of the phase IIIb STASEY study, evaluating the safety and tolerability of Hemlibra prophylaxis in people with haemophilia A with factor VIII inhibitors.

Further data from the STASEY study to be presented outline surgical experiences in the trial, as well as additional insights into the pharmacokinetics and pharmacodynamics profile of Hemlibra.

Roche will also share the first clinical data from the phase III HAVEN 5 study, evaluating the efficacy, safety and pharmacokinetics of Hemlibra in 70 people with haemophilia A with and without factor VIII inhibitors in the Asia-Pacific region. The study met its primary endpoint, demonstrating that Hemlibra prophylaxis dosed every week or every four weeks resulted in a statistically significant 96% (p<0.0001) reduction in the number of treated bleeds over time compared to those receiving no prophylaxis.5 In addition, all secondary bleed-related endpoints were met with clinically meaningful results. Overall, this study showed that Hemlibra was effective and well tolerated in this population.5 HAVEN 5 was conducted as part of the post-approval agreement with the Chinese health authorities to provide supportive data in people with haemophilia A in China, and was expanded to enrol patients from other Asia-Pacific countries.

Key data presentations on impact of haemophilia ARoche will also present two analyses providing insights into clinical outcomes from the CHESS II (Cost of Haemophilia in Europe: a Socioeconomic Survey-II) study evaluating disease burden in people with haemophilia A. The first analysis examines clinical outcomes in adults with mild, moderate and severe haemophilia A without factor VIII inhibitors, focusing on bleeding episodes and joint outcomes. Results show most people with mild and moderate haemophilia A (91% and 98% respectively) did not receive prophylaxis and the majority of these experienced one or more bleeds (74% and 85% respectively).6 These data demonstrate the potential treatment needs in these populations, and the clinical burden on those living with mild and moderate haemophilia A. Additional data from the CHESS II study explores the correlation between bleed frequency and physical activity levels in the same patient population, suggesting there is a correlation between the two.7

Key abstracts from Roche and Spark that will be presented at ISTH can be found in the table below.

Follow Roche and Spark on Twitter via @Roche and @Spark_tx respectively, and keep up to date with ISTH 2020 Congress news and updates by using the hashtag #ISTH2020.