Trial of gene-editing therapy for SCD now enrolling at 5 sites

BEAM-101 study seeking young adults with 4 or more pain crises

Marisa Wexler, MS avatar

by Marisa Wexler, MS |

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A Phase 1/2 clinical trial testing Beam Therapeutics’ gene-editing therapy BEAM-101 is continuing to enroll young adults with sickle cell disease (SCD).

The study, called BEACON (NCT05456880), is active at five sites, with a waiting list for enrollment, according to Beam, which is sponsoring the trial. Enrollment is ongoing at Boston Children’s Hospital, the Medical College of Wisconsin, and the University of Minnesota. The company did not specify the other two sites that are now active.

“As we work through the sentinel cohort process with our investigators, we are encouraged by the overall momentum of the BEACON trial, with multiple new sites activated, a growing number of patients on the wait list for future enrollment, and continuing progress preparing for … manufacturing in our North Carolina facility,” John Evans, Beam’s CEO, said in a company press release announcing first-quarter 2023 financial results and a drug pipeline update.

The BEACON trial is open to adults ages 18 to 35 with sickle cell who have experienced at least four vaso-occlusive crises (VOCs), or bouts of substantial pain due to a disease flare. Such crises can affect any part of the body and often require emergency treatment.

The VOCs must have occurred in the two years prior to entering the trial, despite the patient receiving treatment with hydroxyurea or other supportive medications.

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Participants in BEAM-101 will first undergo a procedure to collect hematopoietic stem cells or HSCs from their bone marrow. HSCs are precursor cells that can give rise to all types of blood cells in the body.

These cells will then be modified in a laboratory with the BEAM-101 gene-editing therapy, which specifically aims to increase the production of fetal hemoglobin (HbF).

Sickle cell is caused by mutations that affect the adult version of hemoglobin, the protein red blood cells use to carry oxygen through the bloodstream. HbF is a version of hemoglobin that is usually only made during early fetal development and is more effective at transporting oxygen than its adult counterpart. Increasing HbF levels in adulthood may help compensate for the faulty adult protein in people with sickle cell.

Patients then will undergo a conditioning regimen, in which the chemotherapy agent busulfan will be administered to eliminate HSCs in their bone marrow. This process will make room for the new edited cells. Finally, the modified HSCs will be transplanted back into the patient via a one-time into-the-vein (intravenous) infusion.

The first group of patients in the trial, referred to as the sentinel cohort, is slated to include three participants who will be treated one after the other. The plan is to carefully evaluate these individuals for safety and efficacy outcomes.

The first patient enrolled late last year, but withdrew from the study due to “personal, non-medical reasons” before beginning treatment, according to Beam.

Two other patients have since been enrolled in the sentinel cohort, and are now undergoing screening procedures needed to permit the HSC collection step. Beam is expecting that the sentinel cohort will be fully enrolled by the end of this year.

An expansion cohort also is expected to start enrolling patients this year, and interim data is anticipated in 2024, according to the company.

“We plan to continue to follow the science, maintain a disciplined investment strategy, and preserve optionality in our portfolio, so that we can maximize our potential to create medicines that provide life-long cures for patients in need,” Evans said.