Spark Therapeutics’ SPK-8011 Suggests Stable and Durable Factor VIII Expression

Spark Therapeutics

PHILADELPHIA, PA — Spark Therapeutics, a member of the Roche Group (SIX: RO, ROG; OTCQX: RHHBY), announced updated data from the ongoing Phase 1/2 clinical trial of investigational SPK-8011 in hemophilia A during the recent International Society of Thrombosis and Hemostasis (ISTH) 2021 Virtual Congress (July 17-21).

These data, presented by Principal Investigator Lindsey A. George, M.D., The Perelman School of Medicine, University of Pennsylvania and Children’s Hospital of Philadelphia, demonstrated that administration of SPK-8011 in patients with hemophilia A resulted in sustained factor VIII (FVIII) expression in 16 of 18 participants with up to 4 years of follow-up, as of the May 3, 2021 data cutoff.

“We are encouraged by the results from the phase 1/2 trial for investigational SPK-8011, which has been evaluated in the largest phase 1/2 gene therapy trial in this disease to date, and demonstrates continued response over time, a critical measure of a therapy’s potential to transform lives for people living with this chronic condition,” said Gallia Levy, M.D., Ph.D., Chief Medical Officer, Spark Therapeutics. “We remain focused on optimizing the dose and immunomodulatory regimen in the phase 1/2 study and look forward to continuing our evaluation of this therapy in a Phase 3 study.”

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Eighteen participants in the Phase 1/2 trial received a single administration of investigational SPK-8011 in four dose cohorts, ranging from 5×1011 vg/kg to 2×1012 vg/kg. In the 16 patients with sustained FVIII expression, there was a 91.2% reduction in annualized bleed rate (ABR) and 97% reduction in annualized FVIII infusion rate (AIR) after vector administration.

Administration of SPK-8011 in patients with hemophilia A resulted in an acceptable safety profile with no deaths and no FVIII inhibitor development with up to 4 years of follow-up. As previously disclosed, two of the 17 participants with over one year of data lost FVIII expression due to a presumed cellular immune response to the AAV capsid that was unresponsive to immunosuppression. Seven participants reported transient, asymptomatic liver function test (LFT) elevations. All LFT elevations were mild or moderate and have resolved. One participant experienced a mild to moderate acute infusion reaction, which presented as four nonserious adverse events (AEs) (pyrexia, myalgia, vomiting, and back pain) and were resolved. One participant experienced Grade 2 transaminitis, which resulted in elective hospitalization for IV steroid administration, qualifying as a serious AE. The event was subsequently resolved.

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These data reinforce the ability of AAV gene therapy targeting hepatocytes to achieve stable and durable FVIII expression with an acceptable safety profile.

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