The first patient has been dosed in the Phase 1/2 B-LIEVE trial of FLT180a, an investigational treatment for hemophilia B. Freeline Therapeutics, the drug developer, announced the news in a recent press release.

About the Phase 1/2 B-LIEVE Trial

This dose-confirmation trial will evaluate FLT180a at a starting dose of 7.7e11 vg/kg alongside a quick course of prophylactic immune management. Medical professionals will focus on the gene therapy’s ability to normalize human factor IX (FIX) levels. In the end, the goal is to confirm the best dosage for the next step in clinical development.

Future Clinical Development of FLT180a

If all goes well in the Phase 1/2 portion of the trial, Freeline will affirm what dosage will be used in their Phase 3 trial. This study is intended to evaluate FLT180a’s ability to regulate FIX expression so that it falls into the normal range.

In terms of the existing trial, it is expected to report initial data from the first dosing cohort in the first half of this year. Additional dosing will occur if necessary. If it does, that data will not be released until the third quarter of this year. Updates are expected to come in the second half of 2022 into the first half of 2023. After all of this data has been released, Freeline plans to initiate the Phase 3 trial, also in the first half of 2023.

About FLT180a

FLT180a employs a potent capsid (AAVS3) that holds an expression cassette that codes for a functioning variant of FIX. It has already been evaluated in the Phase 1/2 B-AMAZE trial, and the participants are continuing on to a long-term, follow-up study.

About Hemophilia B

Hemophilia B falls under the larger category of hemophilia, and it is characterized by a deficiency of the factor IX (FIX) gene. This stops blood from clotting properly, leading to prolonged bleeding. Affected individuals experience symptoms like unprovoked and spontaneous bleeds, easy bruising, nosebleeds, extending bleeding, blood in the stool, and bleeding in the urinary and/or gastrointestinal tract. It’s important to be aware of this condition if one is undergoing surgery or procedures, as they could experience prolonged bleeding. In fact, it is necessary to seek treatment in general, as untreated hemophilia B could lead to serious complications and even fatality.