Syncona's Freeline Therapeutics upbeat on haemophilia trial data
By Josh White
Date: Monday 08 Jul 2019
(Sharecast News) - Healthcare company Syncona announced on Monday that its portfolio company, Freeline Therapeutics, has presented 52-week data from the first cohort of two patients in the ongoing 'B-AMAZE FLT180a' phase 1 and 2 trial in haemophilia B.
The FTSE 250 company said the Freeline haemophilia B programme, 'FLT180a', used the 'AAVS3' capsid and a gain of function variant of human factor IX (FIX).
Freeline said the therapy was in a phase 1 and 2 trial known as 'B-AMAZE', with the goal of normalising FIX activity in patients with moderate and severe haemophilia B.
It said it presented data from the first cohort of two patients who were treated with the lowest study dose.
Over the 12 months since infusion, both patients' liver enzymes remained within the normal range at all time points and the patients' transgene expression was stable, with Factor IX levels of 40.5±4.5% at week 52, assessed by the one stage clotting assay using the 'SynthASil' reagent.
Following treatment, Freeline said both patients were free of spontaneous bleeding episodes and did not require any Factor IX supplementation.
It said it as continuing to enrol patients in the B-AMAZE trial, with the goal of optimising the dose to express Factor IX in the normal range of 50-150%, which would reportedly free patients from the symptoms of the disease.
"We are pleased to report updated data on the first two patients in our haemophilia B trial," said Freeline executive chairman Chris Hollowood.
"The results provide early validation for the potential durability and stability of our therapy at the lowest dose cohort.
"We continue to progress dose optimisation in additional patients and look forward to providing a further update as we seek to develop a functional cure for people with Haemophilia B."
Hollowood said the company believed there was a "significant opportunity" for Freeline's "highly efficient" capsid and manufacturing platform to offer gene therapies that substantially improve patients' lives in a wide range of chronic systemic diseases.
"We are seeking to leverage this same platform across Haemophilia A and B, Fabry and Gaucher Diseases, and ultimately into non-monogenic disorders."
Email this article to a friend
or share it with one of these popular networks:
You are here: news
10 Lower Thames St,
London EC3R 6EN