(Alliance News) - Syncona Ltd said Monday said portfolio company, Freeline Therapeutics, has modified the clinical development plan for its FLT180a program for Hemophilia B.
Shares in the midcap life sciences investor were down 4.4% on Monday afternoon in London at 258.25 pence each.
Freeline - a clinical-stage biotechnology company developing gene therapies for patients suffering from inherited systemic debilitating diseases - has modified the trial following feedback from the US Food & Drug Administration.
The feedback, Freeline noted, relates to the "characterisation and comparability" of the investigational drug product used in its Phase 1/2 B-AMAZE trial that was "produced at smaller scale, as compared to its current investigational drug product that is produced at commercial scale".
"Under the modified clinical development plan, Freeline now plans to conduct dose confirmation in a FLT180a Phase 1/2 trial instead of in the Phase 2b part of the previously planned Phase 2b/3 pivotal trial," it explained.
Freeline expects that the Phase 1/2 dose confirmation trial will be a six-month single dose safety and efficacy trial designed to confirm the dose and immune management regimen for the planned Phase 3 pivotal trial.
The company continued: "Freeline believes this modified clinical development plan should enable it to meet its objectives of initiating the clinical trial sites for the dose confirmation trial by the end of 2021, while in parallel, working to meet FDA's Chemistry, Manufacturing and Control requirements in advance of commencing the planned Phase 3 pivotal trial."
Freeline expects that the design of the Phase 3 pivotal trial will be "nearly identical" to the Phase 3 part of the previously planned Phase 2b/3 pivotal trial.
Freeline, Syncona said, is targeting a data readout for the Phase 1/2 dose confirmation trial by the end of 2022, initiation of the Phase 3 trial by the middle of 2023 and continues to target a filing of the Biologic License Application with the FDA by the end of 2024.
In the first six months of the 2020, Freeline posted a pretax loss of USD33.3 million, widened from a USD24.6 million loss the year prior.
This was as administrative expenses rose 88% to USD11.3 million and research and development costs increased 25% to USD29.4 million. Total other income was USD7.8 million, up from USD5.1 million the year before.
As at September 30, Freeline had cash of USD252.6 million, up from USD106.6 million at the end of June.
By Paul McGowan; paulmcgowan@alliancenews.com
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Syncona (SYNC)