Novel Approaches to Hemophilia and Venous Thromboembolism Care

Due to the unwise choice to held the 59th ASH Annual Meeting in Atlanta this delicate time of the year and due to the possibly foreseeable and now prevailing harsh weather conditions Oncoletter as well as many other attendees of the meeting couldn't make it timely to reach Atlanta.

At this time Oncoletter can instead of a webcast only provide the abstracts of the following studies which were presented at the press conference this morning:

Abstracts: 

85 Guy Young, Robert F. Sidonio, Ri Liesner, Johannes Oldenburg, et al. HAVEN 2 Updated Analysis: Multicenter, Open-Label, Phase 3 Study to Evaluate Efficacy, Safety and Pharmacokinetics of Subcutaneous Administration of Emicizumab Prophylaxis in Pediatric Patients with Hemophilia A with Inhibitors. Slides (by courtesy of Dr. Guy Young)

The authors of this study conclude that:

HAVEN 2 is the largest study in pediatric PwHA with inhibitors to date, and demonstrates that emicizumab prophylaxis prevented or substantially reduced bleeds and was well tolerated in this patient population. PK remained consistent with that seen in adolescent/adult PwHA. Weekly subcutaneous emicizumab has the potential to reduce overall treatment and disease burden and may provide a new standard of care for hemophilia management by providing an effective, safe and convenient option for pediatric PwHA with inhibitors.

603 K. John Pasi, Savita Rangarajan, Benjamin Kim, Will Lester, et al. Achievement of Normal Circulating Factor VIII Activity Following Bmn 270 AAV5-FVIII Gene Transfer: Interim, Long-Term Efficacy and Safety Results from a Phase 1/2 Study in Patients with Severe Hemophilia a

The authors of this study conclude that:

AAV5-FVIII gene transfer in subjects with severe HA has resulted in sustained, clinically relevant FVIII activity that profoundly reduced self-reported bleeding and exogenous FVIII use >1.2 years post-gene transfer in the 6E13 vg/kg cohort. FVIII activity levels in the 4E13 vg/kg cohort continue to steadily rise, reaching the lower end of the normal range >0.6 years post-gene transfer. BMN 270 was well-tolerated. Efficacy and safety data up to 1.5- and 1-year follow-up for all subjects in the 6E13 and 4E13 cohorts, respectively, will be presented. Both doses appear to enable achievement of long-term normalization of FVIII activity in severe HA patients and complete protection against hemophilia-related bleeding, with a favorable safety profile. Optimal dosing of BMN 270 will be further evaluated in upcoming Phase 3 clinical trials.

625 Annie Young, Andrea Marshall, Jennifer Thirlwall, Catherine Hill, et al. Anticoagulation Therapy in Selected Cancer Patients at Risk of Recurrence of Venous Thromboembolism: Results of the Select-D Pilot Trial

The authors of this study conclude that:

Select-d is a large pilot randomised trial for the treatment of VTE, investigating a DOAC versus a LMWH in patients with cancer. Treating with rivaroxaban resulted in a very low VTE recurrence rate at 6 months with a similar number of major bleeds reported across trial arms but more CRNMBs were seen with rivaroxaban. A large phase III trial will confirm the use of rivaroxaban for the treatment of VTE in cancer patients.

See also late breaking abstract press conference with presentation of the Hokusai VTE-Cancer Study (Embargo lifts Tuesday 13:30)