Sangamo and Pfizer give Spark more haemophilia worries

As if things weren’t bad enough for Spark Therapeutics right now. Another rival in haemophilia A gene therapy, Sangamo, has just taken a step forward with its project, SB-525. True, Sangamo did not release detailed phase I/II results yesterday, instead promising in-depth data at a haematology conference later this year, probably Ash. But the company claimed that it had seen therapeutic levels of factor VIII in one patient treated at the third – presumably highest – dose. And Pfizer obviously saw enough potential to partner the project a year ago. At the time this raised doubts about Spark’s haemophilia A gene therapy candidate, SPK-8011 – particularly as Pfizer was already collaborating with Spark in haemophilia B. And disappointing SPK-8011 data this week seemed to confirm these fears, making Biomarin the clear leader in the haemophilia A gene therapy chase. In haemophilia B Spark, which had once streaked ahead, could also be overtaken, this time by Uniqure. The latter has begun a phase III trial of its next-generation candidate, AMT-061 which, like Spark/Pfizer’s SPK-9001, uses the Padua gene variant thought to confer higher factor IX activity. Pfizer's phase III trial of SPK-9001 is also ongoing, but it has lost its head start. 

Selected haemophilia gene therapies in development
Project Company/ies Indication Status Trial name Trial ID
Valoctocogene roxaparvovec Biomarin Haemophilia A Phase III Gener8-1, Gener8-2 NCT03370913, NCT03392974
SPK-8011 Spark Haemophilia A Phase I/II - NCT03003533
SB-525  Sangamo/Pfizer Haemophilia A Phase I/II - NCT03061201
SPK-9001/fidanacogene elaparvovec Spark/Pfizer Haemophilia B Phase III - -
AMT-061 Uniqure Haemophilia B Phase III Hope-B NCT03569891
Source: EvaluatePharma, clinicaltrials.gov.

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