In the trial, the therapy will be assessed in those with moderately severe to severe haemophilia B, a bleeding disorder. Credit: Andrey_Popov/Shutterstock.com.

Be Biopharma has treated the first subject in the two-part BeCoMe-9 Phase I/II trial of BE-101, a treatment for haemophilia B, a bleeding disorder.

This marks a milestone in the development of B Cell Medicines (BCMs), as the therapy is administered without the need for preconditioning or immunosuppression.

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The first-in-human dose escalation, multi-centre trial aims to evaluate the preliminary efficacy and safety of the therapy in those with moderately severe to severe haemophilia B.

Part I of the trial focuses on the therapy’s dose escalation to determine the necessary dose for meeting target Factor IX (FIX) activity levels 28 days post-infusion.

Part II will expand on these findings to evaluate the therapy’s activity and safety at a chosen dose further.

In both parts of the trial, up to 24 subjects will be enrolled, encompassing 18 and six subjects in Parts I and II, respectively.

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Subjects in the trial will undergo monitoring for clinical activity and safety for around a 52-week period after receiving BE-101.

Be Biopharma CEO and president Joanne Smith-Farrell said: “Dosing the first participant in our first-in-human study of BE-101 marks a significant milestone for Be Bio and for people with haemophilia B.

“BE-101 is our first BCM to enter the clinic, and it holds the promise of transforming the treatment landscape for haemophilia B while demonstrating the power of BCMs to deliver groundbreaking in vivo biologics.”

According to the company, the therapy is tailored to insert the human FIX gene into human B cells for treating this condition.

It is designed to be re-dosable and titratable, offering a new therapeutic alternative for haemophilia B.

BE-101 has secured fast-track and orphan drug designations from the US Food and Drug Administration.

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