Spark Therapeutics (ONCE), Pfizer (PFE) Announce Presentation of Updated SPK-9001 Phase 1/2 data in Hemophilia B

November 3, 2016 9:06 AM EDT

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Spark Therapeutics (Nasdaq: ONCE) and Pfizer Inc. (NYSE: PFE) announced that Lindsey A. George, M.D., a hematologist and attending physician at the Children’s Hospital of Philadelphia and lead investigator in the Phase 1/2 clinical trial of SPK-9001 for treatment of hemophilia B, will present an updated dataset from the ongoing Phase 1/2 clinical trial of SPK-9001 at the 58th American Society of Hematology (ASH) Annual Meeting, to be held Dec. 3-6, 2016, in San Diego. The conference abstract, including a plot of factor IX activity levels (as a % of normal) expressed over time by the first seven participants as of Aug. 4, 2016, was made available today: ASH Abstract.

The results are from the first seven participants who received a single administration of investigational SPK-9001 at a dose of 5 x 1011 vector genomes (vg)/kg body weight. Four of the seven participants had reached greater than 12 weeks post-vector administration at the time of abstract submission; those four participants experienced consistent and sustained factor IX activity levels, with a mean greater than 30% of normal, with no sustained elevation in liver enzyme levels.

Spark Therapeutics and Pfizer report that one participant, who had not reached 12 weeks post-vector administration, manifested an immune response to the adeno-associated virus (AAV) capsid, accompanied by a drop in factor IX activity level, and was put on a tapering course of corticosteroids. Despite the immune response and decline in factor IX activity level, this participant has not had any bleeds or required replacement factor. No other participant has required the use of corticosteroids. The companies expect to announce updated trial results at the upcoming ASH Annual Meeting in December.

None of the participants received infusions of factor IX concentrates to prevent bleeding events. One precautionary infusion took place in one participant two days after administration of SPK-9001 due to a suspected ankle bleed.

"These initial observations are encouraging, underscoring the potential of investigational SPK-9001 to deliver a potentially consistent, sustained and therapeutically meaningful level of factor IX activity through one administration," said Dr. George. “Participants in the trial did not require prophylactic factor IX infusions to prevent bleeding, including one participant who we have followed for more than eight months as of the data cutoff. We look forward to reporting additional data as we continue to document the longer-term experience with SPK-9001.”

As of Aug. 4, 2016, total consumption of clotting factor in all seven trial participants during a cumulative 724 days following vector administration was reduced by more than 540,000 international units, based on their pre-trial usage levels. Additionally, to date six of seven participants reported increased physical activity and improved quality of life, based on the Haemophilia Quality of Life Questionnaire for Adults, a validated instrument that measures health-related quality of life in adults with hemophilia.

Presentation Details:

SPK-9001: Adeno-Associated Virus Mediated Gene Transfer for Hemophilia B Achieves Sustained Mean Factor IX Activity Levels of >30% without Immunosuppression (Abstract # 91358)

Presenter: Lindsey George, M.D., Children’s Hospital of PhiladelphiaDate: Sunday, Dec. 4, 2016 
Session time: 2-4 p.m. PSTLocation: San Diego Convention Center, Hall AB



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