Beam Therapeutics Inc. Stock Price, News & Analysis

Beam Therapeutics (Nasdaq: BEAM) announced FDA clearance of its investigational new drug (IND) application for BEAM-302, targeting alpha-1 antitrypsin deficiency (AATD) treatment. This marks the company's second in vivo base editing program with an open IND in the U.S.

BEAM-302, a liver-targeting lipid-nanoparticle formulation, is designed to correct the PiZ mutation in AATD patients. The ongoing Phase 1/2 trial includes Part A for patients with lung disease and Part B for those with mild to moderate liver disease. Initial safety and efficacy data reported in March 2025 demonstrated proof of concept as a potential one-time treatment.

The treatment has received clinical trial authorization across six countries. Beam plans to continue dose-escalation in Part A, including a fourth dose cohort, and expects to begin dosing Part B patients in H2 2025, with updated data presentation scheduled for the same period.

Beam Therapeutics (Nasdaq: BEAM) has announced the pricing of an underwritten offering of 16,151,686 shares of common stock at $28.48 per share, along with pre-funded warrants for 1,404,988 shares at $28.47 per warrant. The offering is expected to generate approximately $500.0 million in gross proceeds.

The company plans to use the proceeds for advancing its platform technology, research and development activities, and pre-commercialization efforts. Key focus areas include the biologics license application for BEAM-101, advancement of a potential pivotal trial for BEAM-302 targeting alpha-1 antitrypsin deficiency, and development of a Phase 1/2 clinical trial for the ESCAPE conditioning platform in sickle cell disease patients.

The offering is expected to close around March 11, 2025, with J.P. Morgan, Jefferies, Cantor, Citigroup, and Wells Fargo Securities serving as joint book-running managers.

Beam Therapeutics (NASDAQ: BEAM) announced breakthrough initial data from its Phase 1/2 trial of BEAM-302 for alpha-1 antitrypsin deficiency (AATD). The trial demonstrated the first-ever clinical genetic correction of a disease-causing mutation through base editing.

Key findings from the first three single-ascending dose cohorts (15mg, 30mg, and 60mg) showed:

• The third dose level (60mg) achieved mean total AAT of 12.4µM at Day 28, exceeding the protective therapeutic threshold
• Reduction of mutant Z-AAT up to 78%
• Durable, dose-dependent increases in total and functional Alpha-1 Antitrypsin (AAT)
• Favorable safety profile with no serious adverse events or dose-limiting toxicities

The treatment demonstrated potential as a one-time therapy addressing both liver and lung disease in AATD patients. The company plans to continue dose escalation and expects to present updated data from Part A of the trial at a medical conference in H2 2025.

Beam Therapeutics reported its Q4 and full year 2024 financial results and reiterated key milestones for 2025. The company achieved its adult enrollment target in the BEACON trial for BEAM-101 in sickle cell disease, with dosing and updated data expected by mid-2025. Initial data from the Phase 1/2 trial of BEAM-302 in alpha-1 antitrypsin deficiency is anticipated in the first half of 2025, and dosing in the Phase 1/2 trial of BEAM-301 in glycogen storage disease type 1a is expected to start in early 2025. Beam ended Q4 2024 with $850.7 million in cash and equivalents, projecting a cash runway into 2027.

Financial highlights include:

• Q4 2024 R&D expenses: $101.4 million
• Full year 2024 R&D expenses: $367.6 million
• Q4 2024 G&A expenses: $28.7 million
• Full year 2024 G&A expenses: $111.5 million
• Q4 2024 net loss: $90.4 million, or $1.09 per share
• Full year 2024 net loss: $376.7 million, or $4.58 per share

Beam's strong financial position supports its ongoing and upcoming clinical trials and regulatory activities. The company's focus remains on delivering life-long cures for serious diseases through precision genetic medicines.

Beam Therapeutics (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines through base editing, has announced its management's upcoming participation in three major investor conferences in March 2025:

• The TD Cowen 45th Annual Health Care Conference on March 3 at 1:50 p.m. ET
• The Leerink Partners Global Biopharma Conference on March 10 at 1:40 p.m. ET
• The Barclays Global Healthcare Conference on March 11 at 3:30 p.m. ET

The company will participate in fireside chats at each event. Live webcasts will be accessible through Beam's investor website section at www.beamtx.com and will remain archived for 60 days after the presentations.

Beam Therapeutics (Nasdaq: BEAM), a biotechnology company focused on developing precision genetic medicines through base editing, has announced its participation in the upcoming Guggenheim SMID Cap Biotech Conference. John Evans, the company's CEO, will engage in a fireside chat scheduled for Thursday, February 6, 2025, at 2:30 p.m. ET in New York.

The event will be accessible to interested parties through a live webcast on the investor section of Beam's website (www.beamtx.com). The presentation recording will remain available on the website for 60 days following the event.

Beam Therapeutics (Nasdaq: BEAM) announced it will present data from the BEACON Phase 1/2 clinical trial of BEAM-101 for sickle cell disease at the 2025 Tandem Meetings in Hawaii. The presentation will feature results previously shown at the ASH Annual Meeting in December 2024, covering seven treated patients. Key findings include robust and durable increases in fetal hemoglobin (HbF), reductions in sickle hemoglobin (HbS), rapid neutrophil and platelet engraftment, and improved markers of hemolysis. The safety profile aligned with expected outcomes for busulfan conditioning and autologous hematopoietic stem cell transplantation. The company plans to present updated trial data in mid-2025.

Beam Therapeutics (NASDAQ: BEAM) announced significant progress in its hematology and genetic disease programs, highlighting key milestones for 2025. The company's BEACON trial for BEAM-101 in sickle cell disease has enrolled over 40 adult patients, with 13 already dosed. Beam expects to dose 30 patients and present updated data by mid-2025.

The company is advancing multiple programs: BEAM-302 for alpha-1 antitrypsin deficiency with initial Phase 1/2 data expected in first half 2025; BEAM-301 for glycogen storage disease type 1a with dosing anticipated in early 2025; and ESCAPE nongenotoxic conditioning program with BEAM-103 antibody study expected by year-end.

Financially, Beam reports estimated $850.7 million in cash, cash equivalents, and marketable securities as of December 31, 2024, expecting to fund operations into 2027, including commercial readiness activities for BEAM-101.

Beam Therapeutics (Nasdaq: BEAM), a biotechnology company specializing in precision genetic medicines through base editing, has announced its participation in the 43rd Annual J.P. Morgan Healthcare Conference. CEO John Evans will deliver a presentation on January 13, 2025, at 1:30 p.m. PT in San Francisco. The presentation will be accessible via live webcast on the company's website investor section at www.beamtx.com and will remain available for 60 days after the event.