Roche Unit Spark Therapeutics' Hemophilia A Gene Therapy Shows Durable Benefit In Early-Stage Study - Roche Holding (OTC:RHHBY), Roche Holding (OTC:RHHBF), Roche Holding (OTC:RHHVF)

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.

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