Beam Therapeutics advances multiple gene editing trials, expects significant patient dosing, and plans data presentations through mid-2025.
Quiver AI Summary
Beam Therapeutics has announced significant progress in its clinical trials and portfolio regarding precision genetic medicines, particularly in the area of sickle cell disease (SCD). Over 40 adult patients have enrolled in the BEACON trial for BEAM-101, a gene therapy expected to dose 30 patients by mid-2025. Additional trials for conditions like alpha-1 antitrypsin deficiency and glycogen storage disease type 1a are also underway, with initial data expected in early to mid-2025. Beam is financially positioned to support its operations into 2027 and is preparing for the potential commercial launch of BEAM-101. The company is also developing the ESCAPE platform for improved gene editing without traditional toxic conditioning methods. Beam's CEO emphasized the importance of these advancements as the company aims to offer life-long cures through its innovative gene editing technologies.
Potential Positives
- More than 40 adult patients with sickle cell disease (SCD) have been enrolled in the BEACON trial, demonstrating significant progress in patient recruitment for the clinical study of BEAM-101.
- Beam expects to present updated data from the BEACON trial by mid-2025, indicating ongoing commitment to transparency and updating stakeholders on clinical progress.
- Cash run rate is projected to support operations into 2027, which includes plans for commercial readiness activities, showcasing a strong financial position for upcoming product launches.
- Beam's diverse pipeline includes multiple ongoing clinical trials, with anticipated significant data releases in 2025 for programs targeting SCD, alpha-1 antitrypsin deficiency, and glycogen storage disease type 1a.
Potential Negatives
- Company's cash position is based on preliminary estimates and subject to completion of financial statement procedures, which could lead to potential undisclosed financial issues.
- The reliance on forward-looking statements carries significant risks, including the possibility of delayed development timelines and uncertain regulatory approval for product candidates.
- The press release does not provide detailed information on any potential adverse effects from ongoing clinical trials, raising concerns about transparency on patient safety and trial outcomes.
FAQ
What is the BEACON trial for BEAM-101?
The BEACON trial is a clinical study evaluating the safety and efficacy of BEAM-101 for adult patients with sickle cell disease.
When will Beam present updated data from the BEACON trial?
Beam expects to present updated data from the BEACON trial by mid-2025.
What are the key upcoming milestones for Beam Therapeutics in 2025?
Key milestones include updated data presentation for BEAM-101 and initial data from the BEAM-302 trial in alpha-1 antitrypsin deficiency.
What is Beam's cash position and runway for operations?
As of December 31, 2024, Beam estimates having $850.7 million, expected to support operations into 2027.
What are the primary diseases targeted by Beam's genetic therapies?
Beam’s therapies target sickle cell disease, alpha-1 antitrypsin deficiency, and glycogen storage disease type 1a among others.
Disclaimer: This is an AI-generated summary of a press release distributed by GlobeNewswire. The model used to summarize this release may make mistakes. See the full release here.
$BEAM Insider Trading Activity
$BEAM insiders have traded $BEAM stock on the open market 7 times in the past 6 months. Of those trades, 0 have been purchases and 7 have been sales.
Here’s a breakdown of recent trading of $BEAM stock by insiders over the last 6 months:
- CHRISTINE BELLON (Chief Legal Officer) has traded it 2 times. They made 0 purchases and 2 sales, selling 1,626 shares.
- BETHANY J CAVANAGH (SVP, Finance and Treasurer) has traded it 2 times. They made 0 purchases and 2 sales, selling 1,406 shares.
- GIUSEPPE CIARAMELLA (President) has traded it 2 times. They made 0 purchases and 2 sales, selling 102,220 shares.
- JOHN M. EVANS (CEO) sold 60,000 shares.
To track insider transactions, check out Quiver Quantitative's insider trading dashboard.
$BEAM Hedge Fund Activity
We have seen 95 institutional investors add shares of $BEAM stock to their portfolio, and 84 decrease their positions in their most recent quarter.
Here are some of the largest recent moves:
- FIRST TRUST ADVISORS LP removed 1,464,299 shares (-98.8%) from their portfolio in Q3 2024
- NIKKO ASSET MANAGEMENT AMERICAS, INC. added 1,328,414 shares (+59.7%) to their portfolio in Q3 2024
- KYNAM CAPITAL MANAGEMENT, LP added 1,302,085 shares (+inf%) to their portfolio in Q3 2024
- ARK INVESTMENT MANAGEMENT LLC removed 1,095,040 shares (-14.4%) from their portfolio in Q3 2024
- DEUTSCHE BANK AG\ removed 818,356 shares (-94.9%) from their portfolio in Q3 2024
- CITADEL ADVISORS LLC added 727,041 shares (+94.5%) to their portfolio in Q3 2024
- ARCH VENTURE MANAGEMENT, LLC removed 608,386 shares (-11.2%) from their portfolio in Q3 2024
To track hedge funds' stock portfolios, check out Quiver Quantitative's institutional holdings dashboard.
Full Release
More Than 40 Adult Sickle Cell Disease Patients Now Enrolled in BEACON Trial of BEAM-101; Beam Expects to Dose 30 Patients and Present Updated Data by Mid-2025
Initial Data from Phase 1/2 Trial of BEAM-302 in Alpha-1 Antitrypsin Deficiency Expected in First Half 2025
Dosing Anticipated to Commence in Phase 1/2 Trial of BEAM-301 in Glycogen Storage Disease Type 1a in Early 2025
IND-enabling Studies of ESCAPE Nongenotoxic Conditioning Approach Underway, with Healthy Volunteer Study of BEAM-103 Antibody Expected to Initiate by Year-end
Cash Runway Expected to Support Operating Plans into 2027, Now Inclusive of Commercial Readiness Activities for BEAM-101
Cambridge, Mass., Jan. 13, 2025 (GLOBE NEWSWIRE) --
Beam Therapeutics Inc.
(Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced progress across the company’s hematology and genetic disease franchises and provided updates on anticipated upcoming milestones.
“We're entering 2025 at an important inflection point in the evolution of Beam, having advanced four programs into the clinic, established clinical differentiation for base editing with our lead sickle cell disease program, and prioritized two high-value core franchises with best-in-class potential – all of the key attributes needed to create a long-term leading company in gene editing,” said John Evans, chief executive officer of Beam. “Importantly, we remain in a strong financial position, with our core manufacturing, regulatory and clinical capabilities now in place. This year, we are poised to deliver critical data and achieve key milestones across our pipeline, which we expect will bring us closer to our mission of offering life-long cures for patients in need.”
Pipeline Updates and 2025 Anticipated Milestones
Hematology Franchise
Beam is pursuing a long-term, staged development strategy for sickle cell disease (SCD) that includes three “waves” of innovation intended to progressively expand the reach of the company’s base editing approach to broader subsets of patients.
BEAM-101
: Wave 1 gene editing treatments aim to deliver a genetically modified cell product through stem cell transplant, enabled by chemotherapy conditioning, for the most severe SCD patients. Beam’s wave 1 approach is BEAM-101, an autologous investigational cell therapy designed to efficiently and uniformly increase fetal hemoglobin (HbF) in red blood cells without relying on double-stranded breaks, offering a potentially best-in-class profile. BEAM-101 is being evaluated in the BEACON Phase 1/2 clinical trial, and initial results were
presented
at the 66th American Society of Hematology (ASH) Annual Meeting and Exposition in December 2024.
To date, more than 40 adult patients with SCD have cleared screening and enrolled in BEACON, and, of these, 13 have been dosed. Beam expects to dose 30 patients by mid-2025.
The U.S. Food and Drug Administration (FDA) and the BEACON data monitoring committee approved enrollment of adolescent SCD patients ages 12-17 years in the study, and screening has commenced.
Beam expects to present updated data from the BEACON trial in mid-2025.
ESCAPE:
Beam’s wave 2 approach is its Engineered Stem Cell Antibody Evasion (ESCAPE) platform, which aims to provide the same
ex vivo
-manufactured cell product deployed in wave 1, but now using a non-genotoxic alternative to traditional transplant myeloablative conditioning. Proof-of-concept data in non-human primates (NHPs) demonstrating engraftment of base-edited cells using antibody conditioning were
presented
at ASH. Beam plans to develop the ESCAPE technology initially in SCD and beta-thalassemia as well as potential future hematology indications.
In December, Beam initiated Phase 1-enabling preclinical toxicology studies for ESCAPE.
The company expects to initiate a Phase 1 healthy volunteer clinical trial of BEAM-103, an anti-CD117 monoclonal antibody (mAb) designed to suppress hematopoietic stem and progenitor cells that express CD117, by the end of 2025.
In vivo
: In wave 3, Beam is exploring the potential for
in vivo
base editing programs for SCD, in which base editors would be delivered to the patient through intravenous infusion of lipid nanoparticles (LNPs) targeted to hematopoietic stem cells, eliminating the need for transplantation altogether.
Genetic Disease Franchise
Beam’s second core area of focus seeks to create single-course gene editing therapies for genetic diseases by delivering base editors through intravenous infusion of LNPs, which are a clinically validated technology for delivery of nucleic acid payloads to the liver.
BEAM-302:
Beam’s lead genetic disease program is BEAM-302, a potentially best-in-class liver-targeting LNP formulation of base editing reagents designed to correct the PiZ allele, the most common gene variant associated with severe alpha-1 antitrypsin deficiency (AATD). BEAM-302 has the potential to simultaneously reduce the aggregation of mutant, misfolded AAT protein that causes toxicity to the liver and increase circulating levels of corrected and functional AAT protein, thus addressing the underlying pathophysiology of both the liver and lung disease. BEAM-302 is being evaluated in a Phase 1/2 dose-escalation clinical trial.
The company continues to advance global regulatory and site activation activities with sites now open in the United Kingdom, New Zealand, Australia and Netherlands.
Beam expects to report initial data from multiple cohorts from the Phase 1/2 study in the first half of 2025.
BEAM-301:
BEAM-301 is a liver-targeting LNP formulation of base editing reagents designed to correct the R83C mutation, the most common disease-causing mutation that results in the most severe form of glycogen storage disease type 1a (GSD1a). GSD1a is an autosomal recessive disorder caused by mutations involved in maintaining glucose homeostasis and is associated with life-threatening fasting hypoglycemia as well as long-term complications impacting the liver and kidney. BEAM-301 has the potential to normalize blood glucose without continuous supplementation and improve metabolic parameters. BEAM-301 is being evaluated in a Phase 1/2 dose-escalation clinical trial.
The first clinical trial site for the Phase 1/2 clinical trial of BEAM-301 is now active, with patient dosing expected to commence in early 2025.
Partnered Programs:
Beam continues to progress its research collaborations with Pfizer and Apellis. Under the Apellis collaboration, which is focused on multiple base editing programs that target specific genes within the complement system, the companies are advancing preclinical studies for a one-time treatment targeting the neonatal Fc receptor (FcRn) using gene editing technology from Beam.
Cash Position and Updated Operating Runway
As of December 31, 2024, Beam estimates that it had $850.7 million in cash, cash equivalents and marketable securities. This estimate is preliminary, unaudited and is subject to completion of Beam’s financial statement closing procedures. This estimate also does not present all information necessary for an understanding of Beam’s financial condition as of December 31, 2024, and its results of operations for the three months and year ended December 31, 2024. Accordingly, undue reliance should not be placed on this preliminary estimate.
Beam now expects that its estimated cash, cash equivalents and marketable securities as of December 31, 2024, will enable the company to fund its anticipated operating expenses and capital expenditure requirements into 2027, inclusive of commercial spend related to the potential launch of BEAM-101.
J.P. Morgan Healthcare Conference
Beam management will present and discuss Beam’s pipeline and business updates during a presentation at the 43rd Annual J.P. Morgan Healthcare Conference today, Monday, January 13, 2025, at 1:30 p.m. PT. A live webcast will be available in the investor section of the company’s website at www.beamtx.com and will be archived for 60 days following the presentation.
About Beam Therapeutics
Beam Therapeutics (Nasdaq: BEAM) is a biotechnology company committed to establishing the leading, fully integrated platform for precision genetic medicines. To achieve this vision, Beam has assembled a platform with integrated gene editing, delivery and internal manufacturing capabilities. Beam’s suite of gene editing technologies is anchored by base editing, a proprietary technology that is designed to enable precise, predictable and efficient single base changes, at targeted genomic sequences, without making double-stranded breaks in the DNA. This has the potential to enable a wide range of potential therapeutic editing strategies that Beam is using to advance a diversified portfolio of base editing programs. Beam is a values-driven organization committed to its people, cutting-edge science, and a vision of providing life-long cures to patients suffering from serious diseases.
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Investors are cautioned not to place undue reliance on these forward-looking statements, including, but not limited to, statements related to: our upcoming presentations at the 43rd Annual J.P. Morgan Healthcare Conference; the therapeutic applications and potential of our technology, including with respect to SCD, AATD, GSD1a and beta thalassemia; our plans, and anticipated timing, to advance our programs; the clinical trial designs and expectations for BEAM-101, BEAM-103, BEAM-301 and BEAM-302; our estimated cash, cash equivalents and marketable securities as of December 31, 2024 and our expectations related thereto; the sufficiency of our capital resources to fund operating expenses and capital expenditure requirements and the period in which such resources are expected to be available; and our ability to develop life-long, curative, precision genetic medicines for patients through base editing. Each forward-looking statement is subject to important risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statement, including, without limitation, risks and uncertainties related to: our ability to develop, obtain regulatory approval for, and commercialize our product candidates, which may take longer or cost more than planned; our ability to raise additional funding, which may not be available; our ability to obtain, maintain and enforce patent and other intellectual property protection for our product candidates; the uncertainty that our product candidates will receive regulatory approval necessary to initiate or continue human clinical trials; that preclinical testing of our product candidates and preliminary or interim data from preclinical studies and clinical trials may not be predictive of the results or success of ongoing or later clinical trials; that initiation and enrollment of, and anticipated timing to advance, our clinical trials may take longer than expected; that our product candidates, including the delivery modalities we rely on to administer them, may cause serious adverse events; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; whether our actual audited results will be consistent with our estimated cash, cash equivalents and marketable securities as of December 31, 2024; and the other risks and uncertainties identified under the headings “Risk Factors Summary” and “Risk Factors” in our Annual Report on Form 10-K for the year ended December 31, 2023, our Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, and in any subsequent filings with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We undertake no obligation to update any forward-looking statement, whether as a result of new information, future developments or otherwise, except as may be required by applicable law.
Contacts:
Investors:
Holly Manning
Beam Therapeutics
hmanning@beamtx.com
Media:
Josie Butler
1AB
josie@1abmedia.com
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