Beam Therapeutics Reports First Quarter 2025 Financial Results and Recent Business Highlights

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First Patient Dosed in the Phase 1/2 Study of BEAM-301 in Glycogen Storage Disease Type Ia, Beam’s Second Clinical Stage In Vivo Editing Program

Updated Data from BEACON Phase 1/2 Clinical Trial of BEAM-101 Accepted for Presentation at the European Hematology Association 2025 Congress in June

Following Positive Initial Data for BEAM-302 in Alpha-1 Antitrypsin Deficiency, Company Initiated Dosing of Fourth Cohort in Part A of Phase 1/2 Trial and Received Clearance of U.S. IND; Updated Data Expected to be Presented in Second Half of 2025

Ended First Quarter 2025 with $1.2 Billion in Cash, Cash Equivalents and Marketable Securities, Including Net Proceeds from $500 Million Financing; Cash Runway Expected to Support Operating Plans into 2028

CAMBRIDGE, Mass., May 06, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today reported first quarter 2025 financial results and provided an update on corporate and pipeline progress across the company’s hematology and genetic disease franchises.

"Beam has had a tremendous start to what we anticipate will be a transformative year. In March, we achieved a historic milestone with BEAM-302, delivering the first-ever clinical genetic correction of a disease-causing mutation for alpha-1 antitrypsin deficiency. Building on this momentum, we have swiftly advanced the program—initiating the fourth cohort of Part A of the Phase 1/2 BEAM-302 study and securing U.S. FDA clearance for our investigational new drug application, positioning us for continued rapid progress," said John Evans, chief executive officer of Beam. "Additionally, we recently dosed the first patient in our second in vivo program, BEAM-301, a potential treatment for glycogen storage disease type Ia. In our hematology franchise, we are preparing to share updated clinical data from the BEACON Phase 1/2 trial of BEAM-101 in sickle cell disease at EHA in June and remain on track to complete dosing for 30 patients by mid-year. This significant clinical progress is supported by our strong financial foundation, further reinforced by our recent $500 million financing, which extends our projected cash runway into 2028.”

First Quarter 2025 and Recent Progress

  • Recently, the first patient was dosed in the U.S.-based Phase 1/2 clinical trial evaluating BEAM-301 as a potential treatment for patients with glycogen storage disease type Ia (GSDIa), an autosomal recessive disorder that disrupts glucose homeostasis. BEAM-301 is an investigational in vivo base editing treatment designed to correct the R83C mutation, the most common disease-causing mutation that results in the most severe form of GSDIa.

  • Clinical data from the BEACON Phase 1/2 clinical trial of BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD), have been accepted for presentation at the European Hematology Association (EHA) 2025 Congress, taking place in Milan from June 12-15, 2025.

  • In March, the United States (U.S.) Food and Drug Administration (FDA) cleared the investigational new drug (IND) application for BEAM-302 for the treatment of alpha-1 antitrypsin deficiency (AATD), enabling the company to begin activating sites in the U.S. for its ongoing Phase 1/2 trial.

  • Also in March, the company announced positive initial safety and efficacy data from the Phase 1/2 trial of BEAM-302, establishing clinical proof of concept for a potential treatment addressing AATD and for in vivo base editing. Preliminary results from the first three single-ascending dose cohorts in Part A of the trial demonstrated that BEAM-302 was well tolerated, with single doses of BEAM-302 leading to durable, dose-dependent correction of the disease-causing mutation. In April, an encore of the data was presented at the 2025 Alpha-1 Foundation 7th Global Research Conference and 10th Patient Congress, alongside updated biomarker data from the 60 mg cohort that provided further evidence of BEAM-302’s clinical profile. Since reporting the data, Beam has initiated dosing in the fourth cohort of Part A, evaluating 75 mg of BEAM-302.

  • In conjunction with the BEAM-302 initial data, Beam completed a $500 million oversubscribed, registered direct financing, enabling the company to fund its anticipated operating expenses and capital expenditure requirements into 2028.