Beam Therapeutics Announces Progress in Sickle Cell Disease and Alpha-1 Antitrypsin Deficiency Trials
Beam Therapeutics Inc. (BEAM) announced significant progress in its clinical trials for sickle cell disease (SCD) and alpha-1 antitrypsin deficiency (AATD).
35 patients have enrolled in the BEACON Phase 1/2 trial for BEAM-101, an investigational genetically modified cell therapy for SCD.
8 patients have received BEAM-101 treatment, with the remaining patients undergoing pre-transplant preparation.
Preliminary safety data showed no ≥ Grade 3 adverse events or serious AEs related to BEAM-101.
One patient died due to respiratory failure likely related to busulfan conditioning, but the death was deemed unrelated to BEAM-101.
All treated patients achieved over 60% fetal hemoglobin (HbF) and less than 40% HbS at Month 1, sustained through all time points.
Markers of hemolysis normalized or improved for all patients.
No vaso-occlusive crises were reported following BEAM-101 treatment.
Patient enrollment in the Phase 1/2 trial for BEAM-302 in AATD patients is progressing.
Site activation globally has continued, and dosing has been completed for the first cohort.
Beam has nominated a development candidate for its ESCAPE technology, consisting of BEAM-103 (anti-CD117 monoclonal antibody) and BEAM-104 (cell therapy with edits to HBG1/2 genes and CD117).
The company intends to advance BEAM-103 and BEAM-104 for development in SCD and beta-thalassemia.
Beam Therapeutics ended the third quarter of 2024 with $925.8 million in cash, cash equivalents, and marketable securities, providing a cash runway into 2027.
BEAM stock is down 2.40% at $23.78 at the last check on Tuesday.