BEAM-201 Represents First Quadruplex-edited, Allogeneic CAR-T Cell Therapy Candidate in Clinical-stage Development and First Treatment with a Base Editing Candidate in United States

CAMBRIDGE, MA, USA I September 05, 2023 IBeam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that in August, the first patient was treated with BEAM-201, a quadruplex-edited allogeneic CAR-T cell investigational therapy. BEAM-201 is being evaluated in a Phase 1/2 clinical study for the treatment of relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL), a severe disease affecting children and adults.

“As the first patient dosed with a Beam therapeutic candidate and the first patient in the U.S. to receive a base editing therapeutic, this represents a major milestone for the company, the scientists that made this possible, and the patients we hope to serve,” said John Evans, chief executive officer of Beam. “We believe that the full therapeutic potential of CAR-T therapies, including the ability to utilize an allogeneic source of T cells, will only be unlocked through higher levels of cellular engineering enabled by multiple simultaneous genetic edits. Base editing is especially well-suited to this challenge, as it is designed to deliver highly efficient multiplex edits in cells without the double stranded breaks that can lead to frequent chromosomal rearrangements and loss of cell viability. BEAM-201, to our knowledge the first quadruplex-edited cell therapy candidate in clinical development, is an allogeneic CAR-T cell investigational therapy with the potential to make a substantial impact for patients diagnosed with challenging T-cell cancers who have limited treatment options.”

About the BEAM-201 Phase 1/2 Clinical Trial
The ongoing Phase 1/2 trial of BEAM-201 is a multicenter, open-label study evaluating safety and efficacy in patients with relapsed/refractory T-ALL/T-LL. T-ALL/T-LL is a highly aggressive blood cancer arising from malignant transformation of T cell precursors that has few treatment options.

The primary objectives of the Phase 1 portion of the trial are the assessment of safety, tolerability, and identification of the recommended Phase 2 dose and lymphodepletion regimen. Key safety endpoints for the trial include treatment-emergent and treatment-related adverse events, and key efficacy endpoints include proportion of patients with complete or partial responses, proportion eligible for hematopoietic stem cell transplant, and proportion achieving minimal residual disease negative status. Multiple sites for the Phase 1/2 clinical trial of BEAM-201 are now open for enrollment. For more information, visit (NCT05885464).

About BEAM-201
BEAM-201 is a quadruplex base-edited, anti-CD7 allogeneic chimeric antigen receptor T cell (CAR-T) under clinical investigation for the treatment of CD7+ relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL). Multiplexed base editing is designed to eliminate expression of the CD7, TRAC, PDCD1 and CD52 genes. This approach has the potential to reduce fratricide, graft-versus-host disease and CAR-T cell exhaustion and to enable BEAM-201 cells to evade anti-CD52 lymphodepleting agents and enable use of an allogeneic cell source.

About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.

SOURCE: Beam Therapeutics