Pfizer and Spark Therapeutics present early data on bio-engineered haemophilia drug

pharmafile | May 23, 2016 | News story | Research and Development, Sales and Marketing Pfizer, Spark Therapeutics, gene therapy, haemophilia B 

Pfizer (NYSE: PFE) and Spark Therapeutics (NASDAQ: ONCE) have announced that they will present data showing encouraging signs in their early stage trials for a novel bio-engineered candidate in the treatment of haemophilia B.

Haemophilia B is caused by a deficiency in the level of factor IX, and lead to frequent, uncontrolled bleeding.

The compound, SPK-9001, is a bio-engineered adeno-associated virus (AAV) capsid expressing a codon-optimised high activity human Factor IX variant. It was developed using Spark Therapeutics’s proprietary technology platform for selecting, designing, manufacturing and formulating highly optimised gene therapies.

In the initial test subjects, factor IX levels rose consistently through the first four weeks post-administration of SPK-9001. Following just one administration of the drug, the first subjects achieved levels of factor IX above 12% of normal, which is considered sufficient to reduce the risk of joint bleeds and the need for prophylactic clotting factor infusions.

Katherine A. High, chief scientific officer at Spark, says: “We are highly encouraged by these initial data, which are supportive of the target profile of a potential gene therapy product capable of eliminating the need for regular infusions to control and prevent bleeding episodes through a one-time, intravenous administration.”

Greg LaRose, chief scientific officer of the rare disease research unit at Pfizer, comments: “Although these results are early, we believe that the initial SPK-9001 data are promising. We look forward to our continued collaboration with Spark as we work toward our shared goal of bringing to market a novel potential therapy for haemophilia patients around the world.”

Pfizer announced its step into gene therapy with this partnership with Spark Therapeutics in 2014. The data from these initial trials are set to be presented on June 11 at the European Haematology Association meeting.

Sean Murray

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