Spark Therapeutics and Pfizer have amended their licence agreement for SPK-9001, an investigational gene therapy for haemophilia B, a rare genetic bleeding disorder.

Under the new agreement, Spark Therapeutics will enrol up to five additional participants in the current Phase 1/2 clinical trial.

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The participants will receive SPK-9001 manufactured using an enhanced process to test its comparability to the SPK-9001 taken by the first ten participants who were enrolled in the ongoing trial.

Spark Therapeutics Research & Development president and head Katherine High said: “Early data on the 11th participant suggests potential clinical comparability with the preliminary safety and efficacy results seen with the initial ten trial participants, who all have discontinued routine infusions of factor IX concentrates.

“We look forward to transitioning this programme to Pfizer and potentially bringing to market this one-time investigational gene therapy for patients with haemophilia B.”

“We look forward to transitioning this programme to Pfizer and potentially bringing to market this one-time investigational gene therapy for patients with haemophilia B, who otherwise rely on frequent infusions of factor IX to control and prevent bleeding episodes.”

Spark Therapeutics will transfer the improved SPK-9001 manufacturing process to Pfizer, which in turn plans to use material generated with this process in the Phase III clinical trial of SPK-9001.

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The activities outlined in the amended agreement are set to take place parallel to Pfizer’s ongoing preparation to assume responsibility for SPK-9001 after the transfer of the Investigational New Drug application to Pfizer.

Subject to the amendment, Spark Therapeutics will initially receive a $10m cash payment and up to an additional $15m in potential milestone payments from Pfizer.

People with haemophilia B, also known as congenital factor IX deficiency, have a deficiency in clotting factor IX, a specific protein in the blood.