Beam Therapeutics (NASDAQ:BEAM) said its sickle cell disease treatment risto-cel met the primary and secondary endpoints in the BEACON trial, and the company remains on track to submit its first biologics license application as early as the end of this year.
President Giuseppe Ciaramella said at a Guggenheim discussion that the ex vivo treatment achieved high levels of fetal hemoglobin and reduced hemoglobin S to less than 40%. He said treated patients’ hemoglobin levels returned to normal and cited faster neutrophil engraftment, which he said could reduce infection risks and hospital stays.
Beam expects to launch risto-cel during 2027, depending in part on whether the Food and Drug Administration grants priority review, Ciaramella said. The treatment has not yet been approved.
Manufacturing and pricing
Ciaramella said Beam has automated and optimized risto-cel’s manufacturing process to improve reliability. Patients in the trial underwent a median of one cell-mobilization procedure, he said, with the vast majority requiring two or fewer. Mobilization draws cells from bone marrow into circulation for collection.
He declined to discuss a potential price or how many patients Beam would need to treat to break even. He argued that one-time treatments can reduce lifetime health care costs despite high initial prices, citing existing sickle cell gene therapies CASGEVY and LYFGENIA as examples.
Liver disease programs advance
Beam’s next most advanced program, BEAM-302, is in a pivotal phase for alpha-1 antitrypsin deficiency, Ciaramella said. The inherited condition can cause liver damage and emphysema. He said Beam has agreed with the FDA on an accelerated approval pathway based on biomarkers and expects to complete the pivotal phase in the first half of next year.
At the 60-milligram dose, all patients reached or exceeded a blood alpha-1 antitrypsin level of 11 micromolar, which Ciaramella described as an accepted protective threshold. He said the FDA had seen those data and considered the dataset approvable, while noting that Beam was not disclosing all trial endpoints.
Another liver-directed candidate, BEAM-301, targets a mutation in the glucose-6-phosphatase enzyme. Ciaramella said two patients who received a 40-milligram dose were able to go without feeding for 15 hours, the maximum allowed by the study protocol, compared with two hours before treatment. A third patient responded less strongly; Ciaramella said it remained unclear whether the patient’s genetic profile, high body mass index or both contributed. Beam is testing a 60-milligram dose.
Regulatory approach and pipeline
Ciaramella said Beam can cross-reference certain data between programs that use its lipid nanoparticle RNA platform. For its phenylketonuria, or PKU, program, he said the FDA agreed to include three guide-editor combinations under one investigational new drug application and clinical protocol. Together, those combinations target mutations affecting approximately half of the patient population, he said.
Beam plans to begin dosing in PKU soon, Ciaramella said. He added that the company sees an opportunity for at least four programs to launch before the end of the decade, while also working on delivery to tissues beyond the liver.
About Beam Therapeutics (NASDAQ:BEAM)
Beam Therapeutics is a biotechnology company developing genetic medicines using base editing, a form of gene editing designed to make precise changes to DNA. Its platform includes base-editing technologies and delivery systems intended to address diseases by altering the genetic instructions involved in their development. The company’s research and development programs include potential treatments for genetic diseases, including sickle cell disease. Its work spans medicines made by editing a patient’s cells outside the body as well as approaches intended to deliver gene editing directly to cells in the body.
