Beam Therapeutics Highlights Progress Across Base Editing Portfolio and Outlines 2024 Anticipated Milestones

First Patient Dosed and Successfully Engrafted in BEACON Phase 1/2 Trial of BEAM-101 in Patients with Severe Sickle Cell Disease; Significant Enrollment Progress Supports First Expected Clinical Data Readout in Second Half of 2024

European Clinical Trial Application (CTA) Submitted for BEAM-302; Trial Initiation in Alpha-1 Antitrypsin Deficiency Planned for First Half of 2024

Investigational New Drug (IND) Application for BEAM-301 On-track for First Half of 2024

Cash Runway Expected to Support Operating Plans into 2027

CAMBRIDGE, Mass., Jan. 08, 2024 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported progress across the company’s hematology and genetic disease portfolios and provided updates on anticipated upcoming milestones.

“Our vision is to establish Beam as a sustainable, fully integrated company pioneering a new class of genetic medicines with base editing. We made tremendous progress toward this goal in 2023, including opening our own GMP manufacturing facility, dosing the first patients in multiple ex vivo clinical programs, including BEAM-101 for sickle cell disease (SCD), and accelerating our in vivo program for alpha-1 antitrypsin deficiency (AATD), as exemplified by the filing of our CTA for BEAM-302,” said John Evans, chief executive officer of Beam Therapeutics. “Building on this momentum and benefiting from the significant clinical validation, regulatory clarity, and scientific breakthroughs occurring in the broader gene editing field, we expect 2024 to be a year of significant catalysts for Beam. Our highly differentiated SCD and AATD programs have the potential to provide best-in-class therapies for significant patient populations with high unmet need, while also establishing a platform for sustainable long-term growth across multiple therapeutic areas.”

Pipeline Updates and 2024 Anticipated Milestones

Sickle Cell Disease (SCD) Franchise

Beam is pursuing a long-term, staged development strategy for SCD that has three Waves of innovation intended to progressively expand the reach of our base editing approach to broader subsets of patients.

• Wave 1: BEAM-101 is an autologous investigational cell therapy designed to efficiently and uniformly increase fetal hemoglobin (HbF) in red blood cells without relying on double stranded breaks, offering a potentially best-in-class profile. Preclinical models suggest base editing could lead to improved HbF induction and lower residual disease-causing hemoglobin S compared to existing gene therapy options.
  • The first patient was dosed in the fourth quarter of 2023 and successfully achieved engraftment in the BEACON Phase 1/2 clinical trial, an open-label, single-arm, multicenter study evaluating the safety and efficacy of BEAM-101 in adult patients with severe SCD. Treatment with BEAM-101, in which the edited cell product is delivered in an autologous bone marrow transplant, will occur on a sequential basis for the first three patients treated in the trial, and then will be given in parallel for all subsequent patients.
  • Patients have continued to be consented in the BEACON trial, and Beam anticipates dosing the remaining two patients in the sentinel cohort and initiating dosing in patients in the expansion cohort in the first half of 2024.
  • The company is on-track to report initial data on multiple patients from the BEACON trial in the second half of 2024.
• Wave 2: Beam continues to advance and invest in its Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning platform and anticipates initiating Phase 1-enabling preclinical studies for the program in 2024. ESCAPE aims to avoid the toxicities associated with currently available conditioning regimens for patients with SCD required prior to autologous transplant.
• Wave 3: The company is also exploring the potential for in vivo base editing programs for SCD, in which base editors would be delivered to the patient through intravenous infusion of lipid nanoparticles (LNPs) targeted to hematopoietic stem cells, eliminating the need for transplantation altogether.

Genetic Disease Franchise