Syncona's Freeline makes progress in dose-finding trials

The FTSE 250 firm said the patient remained off enzyme replacement therapy more than 16 weeks post-dosing, while the first patient showed durable expression of the key enzyme, which was absent or “markedly deficient” in patients, over two years with “promising” efficacy.

It said the third patient in the trial for Fabry's disease would be dosed at the same level as the second patient, with additional safety monitoring after a case of “mild and transient” myocarditis was observed.

Freeline was evaluating whether additional studies would be required to enable the business to progress Haemophilia A into the clinic, and the necessity of conducting the additional studies.

The company said its outlook was for three clinical studies by the end of the calendar year, and multiple data read-outs in 2022, with Freeline on track to report long-term durability data from the phase 1 and 2 dose-finding trial for Haemophilia B by the end of this year.

It said the Haemophilia B phase 1 and 2 dose-confirmation study would now start in the first quarter of 2022, instead of by the end of 2021, as the business engaged with regulatory authorities to update study protocols.

In the phase 1 and 2 trial for Fabry's disease, the company was expecting to continue patient dosing in the first quarter of 2022, with further interim data outs from the study expected next year.

Syncona said Freeline was on target for trial site initiation for the phase 1 and 2 study for Gaucher Type 1 by the end of 2021 as well, with data read-outs from the trial expected in 2022.

“We are pleased that Freeline has been able to re-initiate clinical studies and are encouraged by the data generated by the second patient dosed in the phase 1 and 2 Fabry's study,” said Chris Hollowood, chief investment officer at Syncona Investment Management and chair of Freeline.

“We are fully supportive of the safety protocols recommended by the data monitoring committee and look forward to seeing further progress from this study.”

Hollowood said that by the end of the calendar year, the business would have three live clinical studies with the expected site initiation of its phase 1 and 2 programme for Gaucher's disease and multiple data read-outs expected across those programmes in 2022, with the potential to drive value for the business.

“We remain excited by the potential of the therapies the company is developing to have a meaningful impact on the lives of patients.”

At 0909 GMT, shares in Syncona were up 0.24% at 201.48p.