Roche presents data for hemophilia A therapy ACE910
Posted: 9 December 2014 |
Roche presented data from a first-in-patient study of the novel humanised bispecific antibody ACE910 (RG6013) in hemophilia A patients during the 56th American Society of Hematology (ASH) Annual Meeting…
Roche (SIX: RO, ROG; OTCQX: RHHBY) presented data from a first-in-patient study of the novel humanised bispecific antibody ACE910 (RG6013) in hemophilia A patients during the 56th American Society of Hematology (ASH) Annual Meeting. Results from the open-label phase I study, conducted by Chugai, showed once-weekly subcutaneous administration of ACE910 was well tolerated and possessed a promising efficacy profile in hemophilia A patients. Results showed that ACE910 significantly reduced the annualised bleeding rate (ABR) during treatment, compared to the rate seen in the six-month period prior to study enrolment.
Importantly, ACE910 was effective irrespective of the presence of inhibitors to blood clotting factor VIII (FVIII). FVIII is an essential blood clotting protein required for the normal functioning of the coagulation system.1 People with hemophilia A lack FVIII and therefore need to get it injected into their bloodstream. Approximately 30% of severe hemophilia A patients develop inhibitors to injected FVIII, rendering current FVIII replacement therapies ineffective.2
As detailed in the table below, all patients with prior bleeding events experienced reductions in their ABR, with the rate of reduction reaching 100% for patients in each of the three cohorts.
“ACE910, designed to overcome the limitations of intravenous FVIII replacement therapy, may offer an effective, convenient prophylactic treatment option for hemophilia A patients with or without inhibitors to factor VIII,” said Sandra Horning, MD, Roche’s Chief Medical Officer and Head of Global Product Development. “This exciting expansion into hemophilia A represents Roche’s commitment to benign as well as malignant hematology, and based on the study outcome, we plan to move into more advanced clinical trials of this promising compound in 2015.”
The objectives of the phase I investigations were to assess the safety and efficacy of ACE910 in this patient group, as well as the pharmacokinetics and pharmacodynamics of the drug. Full results of the data set were presented at the American Society of Hematology (ASH) Annual Meeting 2014 in San Francisco (Abstract #691).
Abstract #691 Safety and Prophylactic Efficacy Profiles of ACE910, a Humanised Bispecific Antibody Mimicking the FVIII Cofactor Function, in Japanese Hemophilia A Patients Both without and with FVIII inhibitors: First-in-Patient Phase 1 Study
(Shima et al.) | Patient profile
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Safety data
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Efficacy data | |||
Treatment Arm | N | Annualized Bleeding Rate (ABR) | |
Cohort-1 0.3mg/kg ACE910 | 2/6 without inhibitors 4/6 with FVIII inhibitors | 22.8%-100% reduction 64.7%-100% reduction | |
Cohort-2 1mg/kg ACE910 | 2/6 without inhibitors 4/6 with FVIII inhibitors | 100% reduction 88.9%-100% reduction | |
Cohort-3 3mg/kg ACE910 | 3/6 without inhibitors 3/6 with FVIII inhibitors | 0%*-100% reduction 100% reduction | |
*One patient did not report bleeding episodes at baseline nor during the conduct of this study.
References
- The National Center for Biotechnology Information, 2014. F8 coagulation factor VIII, procoagulant component. Available at: http://www.ncbi.nlm.nih.gov/gene/2157 (last accessed 12.11.14).
- Berntorp E, Shapiro AD. Modern haemophilia care. Lancet 2012; 379: 1447–56.
