Syncona eyes buyout of gene therapy biotech Freeline after early data in two Gaucher patients

The investment trust co-founded Freeline alongside University College London in 2015, with Syncona currently owning 57.9% of company shares, according to an SEC filing. But the trust has now placed an offer to buy the rest for $5 apiece, according to another SEC filing.

The bid represents a 20% premium on Freeline’s weighted average share price of $4.16 since the data release on Oct. 4 through Oct. 16. Syncona said it plans to further discuss the offer with the UK biotech. Freeline’s stock $FRLN surged by as much as 12% to $5 on Wednesday in response to the news.

Freeline’s Gaucher therapy, dubbed FLT201, is under investigation in the Phase I/II GALILEO-1 trial. FLT201 uses an AAVS3 capsid to deliver a transgene to the liver to produce an engineered version of the glucocerebrosidase (GCase) enzyme, which is missing in Gaucher disease patients.

Results from two patients in GALILEO-1 shared earlier this month hinted at favorable tolerability, with no cases of liver transaminase elevations or infusion reactions observed at the data cutoff. One patient was 13 weeks post-treatment administration, while the second patient was six weeks in.

Both patients also saw an increase in plasma GCase levels. The first patient had an almost 700-fold increase from baseline to more than 70 μmol/L/h GCase at 12 weeks. The second patient had more than a 300-fold increase from baseline to around 30 μmol/L/h at four weeks.

These recent developments mark a welcome change for Freeline, which was forced to hit the brakes on its early-stage gene therapy program for hemophilia B in 2021 due to CMC concerns raised by the FDA.

In July last year, the company published long-term data from a Phase I/II dose-finding trial of the bleeding disorder treatment named FLT180a. But, as part of a broader strategic reprioritization, Freeline stopped investing in the FLT180a program in November 2022, per an earnings report.

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