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Roche Unit Spark Therapeutics' Hemophilia A Gene Therapy Shows Durable Benefit In Early-Stage Study

· 02/05/2021 12:37
  • Spark Therapeutics, a unit of Roche Holdings (OTC: RHHBY), has announced preliminary data from part one of the ongoing Phase 1/2 trial evaluating SPK-8016 in hemophilia A, a genetic deficiency in clotting factor VIII (a necessary part of the blood-clotting cascade), resulting in increased bleeding. Data were at the European Association for Haemophilia and Allied Disorders 2021 Virtual Congress.
  • As of the October 26, 2020 data cutoff, all four treated participants have stable and durable factor VIII activity at greater than 52 weeks ranging from 5.9% to 21.8%, with no serious adverse events. Data showed a 98% reduction in annualized infusion rate and 85% reduction in annualized bleed rate after a follow-up of between 15 and 18 months.
  • One of four participants did not receive immunomodulatory agents and demonstrated the highest level of FVIII activity (21.8% at greater than 52 weeks). Three of four participants received oral corticosteroid therapy, two participants received azathioprine and/or tacrolimus as steroid-sparing immune-modulating co-therapy to limit total exposure to prednisone.
  • No participant demonstrated persistent liver enzyme elevations, except transaminitis associated with azathioprine toxicity in one participant resolved after azathioprine was discontinued.
  • Three participants who received immunomodulatory agents experienced mild-to-moderate, non-serious steroid-associated adverse events.
  • Price Action: Both RHHBY and RHHVF slipped 1.9% at $42.92 and 1.6% at $343.4 during market hours.