- Spark Therapeutics, a unit of Roche Holdings AG (OTC:RHHBY), has announced data from its Phase 1/2 trial of SPK-8011 in hemophilia A.
- At a median efficacy follow-up of 33.4 months, the study found that 16 of 18 study participants had sustained factor VIII (FVIII), which permitted prophylaxis cessation and reduction in bleeding episodes.
- The latest results were published online in the New England Journal of Medicine (NEJM).
- The updated analysis (cutoff May 3) of all 18 study participants demonstrated a 91.5% reduction in annualized bleed rate (ABR) and a 96.4% reduction in the annualized number of FVIII infusions.
- Related Link: Spark Therapeutics’ Hemophilia Gene Therapy Achieve Stable, Durable FVIII Expression.
- In the safety analysis, 33 treatment-related adverse events (AEs) occurred in 8 participants, of which 17 were vector-related, including one serious AE, and 16 were glucocorticoid-related.
- As previously reported, two participants lost all FVIII expression due to an anti-AAV capsid cellular immune response, unresponsive to immunosuppression.
- The remaining 16 participants maintained FVIII expression.
- Price Action: RHHBY shares closed at $49.86 on Wednesday.