Beam Therapeutics Inc. Stock Price, News & Analysis

The FDA has granted orphan drug designation to Beam Therapeutics' (NASDAQ: BEAM) BEAM-302 for treating Alpha-1 Antitrypsin Deficiency (AATD). BEAM-302 is a liver-targeting treatment designed to correct the disease-causing mutation in AATD patients. This follows the recent RMAT designation received in May 2025.

Initial Phase 1/2 trial results showed positive safety and efficacy data, with single doses leading to durable, dose-dependent mutation correction. The 60 mg dose cohort achieved AAT protein levels above therapeutic threshold. The company has begun dosing the fourth cohort at 75 mg and plans to start Part B trials with AATD patients with mild to moderate liver disease in H2 2025.

The orphan drug designation provides benefits including tax credits for clinical trials, user fee exemptions, and potential seven-year market exclusivity post-approval.

Beam Therapeutics (NASDAQ: BEAM) will present new data from its BEACON Phase 1/2 clinical trial of BEAM-101, a base-edited cell therapy for sickle cell disease (SCD), at the European Hematology Association 2025 Congress in Milan. The presentation will include updated safety and efficacy data from 17 patients treated with BEAM-101, a one-time treatment for SCD patients with severe vaso-occlusive crises (VOCs).

The company will showcase four presentations at EHA2025, focusing on BEAM-101's clinical results, red blood cell health post-treatment, manufacturing process, and biomarker findings. Beam will host an investor webcast on June 13, 2025, at 4:00 p.m. ET to review the key presentations.

Beam Therapeutics (Nasdaq: BEAM), a biotechnology company focused on developing precision genetic medicines through base editing, has announced its upcoming participation in the 2025 RBC Capital Markets Global Healthcare Conference. The company's management will engage in a fireside chat on Tuesday, May 20, 2025, at 11:00 a.m. ET in New York. Investors and interested parties can access the live webcast through Beam's website investor section at www.beamtx.com, where it will remain archived for 60 days after the presentation.

Beam Therapeutics (NASDAQ: BEAM) has received Regenerative Medicine Advanced Therapy (RMAT) designation from the FDA for BEAM-302, a genetic medicine designed to treat Alpha-1 Antitrypsin Deficiency (AATD). This follows the recent clearance of their U.S. IND application in March 2025.

The Phase 1/2 trial has shown promising initial results, with BEAM-302 demonstrating good tolerability and dose-dependent mutation correction. In the 60 mg dose cohort, total AAT protein levels exceeded therapeutic threshold. The company has begun dosing in the fourth cohort (75 mg) and plans to initiate Part B of the trial in H2 2025, targeting AATD patients with mild to moderate liver disease.

The RMAT designation provides benefits including accelerated approval pathways, increased FDA interaction, and potential priority review, recognizing BEAM-302's potential as a transformative, one-time treatment for AATD patients.

Beam Therapeutics (NASDAQ: BEAM) reported significant progress in Q1 2025 and key financial results. The company achieved several clinical milestones, including: dosing the first patient in the Phase 1/2 study of BEAM-301 for Glycogen Storage Disease Type Ia, advancing BEAM-302 to its fourth cohort for Alpha-1 Antitrypsin Deficiency after positive initial data, and receiving FDA IND clearance. The BEACON Phase 1/2 trial of BEAM-101 for sickle cell disease is progressing, with updated data to be presented at EHA 2025.

Financially, Beam ended Q1 2025 with $1.2 billion in cash, cash equivalents, and marketable securities, bolstered by a $500 million financing. Q1 2025 showed a net loss of $109.3 million ($1.24 per share), with R&D expenses at $98.8 million and G&A expenses at $27.9 million. The company's cash runway is expected to extend into 2028.

Beam Therapeutics (BEAM) presented additional data from its Phase 1/2 clinical trial of BEAM-302 for alpha-1 antitrypsin deficiency (AATD) at the 2025 Alpha-1 Foundation Conference in Lisbon. Key findings from the 60 mg cohort (n=3) showed:

- Corrected M-AAT reached 91% of total AAT in circulation at Day 28
- 79% mean decrease in mutant Z-AAT observed at Day 28
- Single doses were well-tolerated with durable dose-dependent correction

The company has initiated a fourth cohort evaluating 75 mg of BEAM-302 and plans to report additional data in H2 2025. Beam also received FDA clearance for BEAM-302's IND, allowing U.S. site activation. The company plans to begin Part B of the trial, including AATD patients with mild to moderate liver disease, in H2 2025.

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.