The FDA has requested additional preclinical data from Beam Therapeutics on BEAM-201.
This article was updated on September 7, 2022.
The FDA has placed a clinical hold on Beam Therapeutics’ investigational new drug application (IND) for BEAM-201, a chimeric antigen receptor (CAR) T-cell therapy for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia (T-ALL)/T cell lymphoblastic lymphoma (T-LL).1
The FDA has asked the company for information from preclinical experiments, including from genomic rearrangement assessments, data on off-target edits, and from a cytokine independent growth assay.2 The FDA has also asked Beam to update the brochure given to researchers with information on any new preclinical studies.
Beam Therapeutics submitted the IND for BEAM-201 in June 2022. BEAM-201 is an allogeneic, anti-CD7, CAR T-cell therapy electroporated and transduced ex vivo that uses multiplex editing for high-precision editing of the cell therapy.
Earlier this year, Pfizer and Beam announced a multi-target research collaboration to advance Beam’s novel, in-vivo base editing programs.2 The collaboration uses Beam’s mRNA and lipid nanoparticle in vivo delivery technologies to develop candidates for rare liver, muscle, and central nervous system genetic diseases. Beam will research and develop candidates which Pfizer will later develop and commercialize.
John Evans, chief executive officer, Beam, commented on the collaboration in a statement, saying, “Our leading platform for precision genetic medicine has greatly evolved over the last few years, and we are committed to ensuring the broadest reach of these potentially life-changing technologies. This collaboration will provide a unique opportunity to create potentially transformative base editing programs for indications with critical unmet needs, leveraging our proprietary base editing technology and expanding delivery capabilities. We look forward to working together with Pfizer to advance these technologies and potentially expand our impact for people suffering from serious diseases.”3
Beam’s lead program is in sickle cell disease (SCD) with their candidate BEAM-101, the IND for which was accepted in December 2021.4 BEAM-101 is an ex vivo base editing autologous cell therapy product designed to alleviate the effects of mutations in SCD or beta-thalassemia by mimicking single nucleotide polymorphisms seen in these diseases. The therapy uses next-generation CRISPR base editing technology designed to make single base changes without double strand DNA breakage. The safety and efficacy of BEAM-101 will be evaluated in the phase 1/2 BEACON-101 clinical trial.
BEAM-101 has the potential to offer a one-time treatment for patients with sickle cell disease, and this clearance enables the important transition from a preclinical to a clinical-stage company, bringing us closer to our ultimate goal of helping patients,” Evans said in a previous statement.4 “As leaders in the field of base editing, this milestone underscores the expertise of our team and the significant potential of our technology. We are grateful to the team members who have dedicated countless hours to this effort, and we look forward to this program’s continued evaluation in the clinic.