Beam Therapeutics set out to demonstrate how precise base editing can produce new genetic medicines to treat a variety of diseases. That goal may not be achieved yet, but the company is currently narrowing its scope and reducing its headcount to ensure it has enough cash to support key projects that demonstrate the value of the technology and, in turn, create value for the company.
In addition to shelving some projects, Beam said Thursday it would lay off about 100 employees, or 20% of the biotech company’s workforce. Cambridge, Mass.-based Beam said the cuts will be completed during the current quarter.
Beam’s therapies are developed by using base-editing technology, which can make precise changes to individual bases in the genome without causing double-strand breaks in the DNA, which can lead to unwanted genetic modifications. The company’s product line has grown to 11 programs covering a variety of diseases. Beam will now focus on three signs. Sickle cell disease will be addressed through two programs: BEAM-101 and ESCAPE (short for Engineered Stem Cell Antibody Pairing Evasion). ESCAPE aims to improve conditioning regimens to prepare patients with pathological cell disorders and patients with beta thalassemia for stem cell transplantation.
ESCAPE is in the preclinical stage. BEAM-101 has entered the clinic. While initial studies of BEAM-101 used ex vivo delivery to hematopoietic stem cells, the company is also exploring in vivo base editing approaches, in which infusion therapies would use lipid nanoparticles to target stem cells, eliminating the need for a transplant step. body treatment.
In sick cell diseases, Beam could compete with Vertex Pharmaceuticals’ CRISPR gene-editing therapies and Bluebird Bio gene therapies, both of which are expected to receive FDA decisions in December. William Blair analyst Sami Corwin wrote in a note to investors that investor attention is focused on BEAM-101, which is expected to produce preliminary data in 2024.
“Two (sickle cell disease) gene therapies may be approved before the end of the year, and we continue to believe that developing targeted pretreatment or in vivo approaches will be critical for Beam differentiation itself,” she said.
Other projects Beam will continue to pursue include preclinical-stage BEAM-302, an in vivo gene-editing therapy for alpha-1 antitrypsin deficiency, a disease that causes liver and lung damage of liver protein deficiency. Beam also plans to develop BEAM-301 to treat glycogen storage disease 1a (GSD1a), a rare inherited enzyme deficiency. Beam management told Corwin that manufacturing is a strategic capability the company wants to keep in-house, with a cell and lipid nanoparticle manufacturing facility in Research Triangle Park, North Carolina expected to open by the end of the year.
Beam aims to find partners to continue developing some of its other projects. The company investigates the use of base editing to develop allogeneic or off-the-shelf cancer therapies by editing T cells from healthy donors. This research resulted in BEAM-201, which recently dosed its first patient in a Phase 1/2 study. Beam now says it plans to develop a focused data set with the goal of finding partners for this project and other potential in vitro CAR T therapies. Beam has also paused its hepatitis B program and intends to work with partners.
Beam said the restructuring is expected to generate $6.6 in earnings. The one-time cost amounted to $1 million, mainly related to severity payments. As of the end of the second quarter, Beam had a cash position of $1.1 billion, which the company said is enough to support the company’s growth for at least the next year. Beam said the restructuring should allow that cash to last until 2026.
“Beam’s strategy from the beginning has been to develop base editing technology broadly across a diverse portfolio of projects and delivery methods, and we have “The science and pipeline continue to advance across the board.” “However, in this challenging market environment, we need to make the difficult decision to focus our resources on the clinical programs and research areas that we believe have the greatest potential for near-term value creation, while continuing to build a strong company for the future.”
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