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Spark Therapeutics Reports Second Quarter 2016 Financial Results and Recent Business Highlights

Earlier today, released additional positive data from the pivotal Phase 3 trial of voretigene neparvovec

Reported positive preliminary safety and efficacy from the Phase 1/2 clinical trial of SPK-9001 in hemophilia

PHILADELPHIA, Aug. 10, 2016 (GLOBE NEWSWIRE) -- Spark Therapeutics (ONCE) today announced financial results for the quarter ended June 30, 2016 and updated investors on its progress.

“During the last several months, we have made significant progress advancing our clinical portfolio of AAV gene therapy candidates and showcasing the strengths of our overall platform capabilities,” said Jeffrey D. Marrazzo, chief executive officer of Spark. “Notably, we released additional positive data from the continuation of the Phase 3 trial of voretigene neparvovec and remarkable, preliminary safety and efficacy data from the Phase 1/2 clinical trial of SPK-9001 in hemophilia B, which also was granted breakthrough therapy designation.”

“We also continued to make significant progress in preparing the potentially first-ever BLA submission of a gene therapy for a genetic disease, and we are on track to submit the clinical modules and finalize the vast majority of the chemistry, manufacturing and controls (CMC) components before the end of the year,” continued Mr. Marrazzo. “However, based on a recent reassessment of the timelines for completion of the CMC modules, we now expect the generation of certain data using validated quality control methods that are part of the CMC modules to occur in early 2017.”

“We closed the quarter with a healthy balance sheet and we remain focused on finalizing our regulatory submissions of voretigene neparvovec, initiating a clinical trial of SPK-8011 for hemophilia A and further scaling-up of our manufacturing process for future applications.”

Recent highlights

Voretigene neparvovec for RPE65-mediated inherited retinal disease:

  • Reported positive one-year safety and efficacy data for the nine subjects in the Phase 3 crossover group:
    • The eight responders demonstrated the maximum improvement measurable on the bilateral mobility test, the primary endpoint
    • The safety profile was largely consistent with prior studies and there were no product candidate-related serious adverse events, or SAEs, however, one procedure-related SAE was reported in one subject
  • The eight responders demonstrated the maximum improvement measurable on the bilateral mobility test, the primary endpoint
  • The safety profile was largely consistent with prior studies and there were no product candidate-related serious adverse events, or SAEs, however, one procedure-related SAE was reported in one subject
  • Overall, 27 of 29, or 93%, of Phase 3 subjects responded, demonstrating a gain of functional vision as measured by the primary endpoint at one year
  • Reported positive follow-up two-year durability of benefit data for the 20 subjects in the Phase 3 intervention group, as measured by both bilateral mobility and full-field light sensitivity threshold testing
  • Positive follow-up data from a Phase 1 trial in 11 subjects were published in The Lancet, providing insight into the long-term safety and the durability of contralateral-eye administration for at least 3 years

SPK-9001 for hemophilia B:

  • Released initial Phase 1/2 safety and efficacy data, as of July 12, with a combined 76 weeks of observation across the first four subjects enrolled in the study:
    • Consistent and sustained average steady-state factor IX activity levels of 31.8% ±6.9% (range 20% to 44%) of normal determined by averaging levels beginning at 8 weeks post administration through follow up over 12 to 31 weeks following a single administration of SPK-9001 at the initial dose level of 5 x 1011 vg/kg
    • SPK-9001 has been well-tolerated, with no sustained elevation in liver enzyme levels above 1.5x the upper limit of normal
    • No subjects have needed, or received, immunosuppression
    • No subjects have received regular infusions of factor IX concentrates to prevent bleeding events
  • Consistent and sustained average steady-state factor IX activity levels of 31.8% ±6.9% (range 20% to 44%) of normal determined by averaging levels beginning at 8 weeks post administration through follow up over 12 to 31 weeks following a single administration of SPK-9001 at the initial dose level of 5 x 1011 vg/kg
  • SPK-9001 has been well-tolerated, with no sustained elevation in liver enzyme levels above 1.5x the upper limit of normal
  • No subjects have needed, or received, immunosuppression
  • No subjects have received regular infusions of factor IX concentrates to prevent bleeding events
  • Expanded the Phase 1/2 trial, in collaboration with Pfizer, at the initial dose of 5 x 1011 vg/kg with a target of up to 10 subjects
  • Granted breakthrough therapy designation by FDA

SPK-8011 for hemophilia A:

  • Presented strong preclinical data showing factor VIII expression in the 15% to 35% range in preclinical models at clinically relevant doses of 2 x 1012 vg/kg
  • Presented additional preclinical data, utilizing an in vitro assay, of the Spark200 capsid, a novel bio-engineered adeno-associated virus (AAV) capsid optimized for more efficient transduction of human hepatocytes, demonstrating high efficiency in transducing human hepatocytes compared to Spark100, the capsid used in...

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