13th Jul 2020 18:36
(Alliance News) - Syncona Ltd on Monday said its portfolio company, Freeline, presented further data from the ongoing B-AMAZE phase 1/2 clinical trial investigating its gene therapy product candidate, FLT180a, for the treatment of Haemophilia B.
The presentation is part of a late-breaking abstract session and is in addition to five other posters from Freeline featuring throughout the virtual event.
The FTSE 250-listed healthcare company said a novel capsid is used to deliver this gene therapy and has the potential to drive higher protein expression than other programs have demonstrated in the clinic to date.
Freeline is seeking to identify the optimal dosing regimen for FLT180a with the goal of consistently delivering to patients Factor IX activity levels in the normal range of between 50% to 150%, which no other treatment has been able to achieve to date.
Reportable data is available for 10 patients who have been treated across four dose cohorts with FLT180a. Three patients saw reductions in Factor IX expression following a rise in alanine aminotransferases, an enzyme that is predominantly found in the liver.
Syncona said Freeline has continued to optimise its prophylactic immune management regimen throughout the trial, working to identify a regimen that consistently controls alanine aminotransferases to prevent loss of FIX expression.
"We are pleased to see additional encouraging data generated from this Haemophilia B trial. Patients in the first cohort have now shown durable expression of FIX activity for two years after dosing and the latest cohort, which has been expanded, is showing promising levels of expression in the normal range," said Syncona Chief Investment Officer Chris Hollowood.
"We look forward to seeing Freeline progressing its lead programme in Haemophilia B to a pivotal study," added Hollowood.
Syncona shares closed 3.9% higher in London on Monday at 269.50 pence each.
By Evelina Grecenko; [email protected]
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