Roche (OTCQX:RHHBY +0.6%) unit Spark Therapeutics reports updated data from three dose cohorts in a Phase 1/2 clinical trial evaluating gene therapy candidate SPK-8011 in hemophilia A patients. The results were presented virtually at the International Society of Thrombosis and Hemostasis Congress.
14 participants have been dosed, two at the lowest level, three at the middle level and nine at the highest level. The safety profile to date has been “acceptable.” On the efficacy front, there was a 91% reduction in annualized bleed rate and 96% reduction in factor VIII (FVIII) infusions. FVIII expression has been stable and durable between 2.0 and 3.3 years of follow-up.
As reported in August 2018, two patients in the highest dose cohort lost FVIII expression likely due to capsid-based immune responses.
Dosing in a Phase 3 trial should start in 2021.
Related ticker: BioMarin Pharmaceutical (BMRN +1.2%)
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