Patient Enrollment into BEACON Phase 1/2 Trial of BEAM-101 On-track for Second Half of 2022

BEAM-201 IND Submitted to FDA; Currently on Clinical Hold

BEAM-102 IND Submission and BEAM-301 IND-enabling Studies On-track for Second Half of 2022

John Lo, Ph.D., Appointed as Chief Commercial Officer

Ended Second Quarter 2022 with $1.2 Billion in Cash, Cash Equivalents and Marketable Securities to Support Advancement of Broad Precision Genetic Medicines Portfolio

CAMBRIDGE, Mass., Aug. 09, 2022 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today provided pipeline and business updates and reported financial results for the second quarter ended June 30, 2022.

2022 is a critical year for Beams transition to becoming a multi-program clinical-stage company, as we prepare for the near-term initiation of patient enrollment in our BEACON Phase 1/2 trial, the first clinical trial evaluating BEAM-101 in patients with sickle cell disease, said John Evans, chief executive officer of Beam. In June, we submitted our IND for BEAM-201 for CD7-positive T-cell malignancies and recently received notification from the FDA of a clinical hold on the IND. We look forward to receiving more detail from the FDA and working with them in an effort to advance BEAM-201 for these difficult-to-treat cancer indications. We are on track to further expand our portfolio with a steady cadence of clinical and preclinical milestones expected in the quarters ahead, including the IND submission for BEAM-102, our second program in sickle cell disease, and the initiation of IND-enabling studies for BEAM-301, our first liver-directed base editing program in glycogen storage disease, both targeted in the second half of this year.

Mr. Evans added, As pioneers and leaders in the field of base editing, weve continued to extend the potential reach of our base editing technology and applications with the development of new base editors, as well as novel base editing-enabled therapeutic strategies, such as our work on non-genotoxic conditioning to improve transplant regimens. Weve also continued to enhance our team, and Im thrilled to welcome John Lo as chief commercial officer. John has a deep science background and an extensive track record in the strategic development and commercialization of novel medicines, including cell therapy products, at leading companies. I cant wait to work with him to advance our portfolio and our vision of providing a new class of precision genetic medicines to patients.

Pipeline Updates & Anticipated Milestones Ex Vivo HSC Programs

Ex Vivo T Cell Programs

In Vivo LNP Liver-targeting Programs

Recent Research Highlights

Business Updates

Second Quarter 2022 Financial Results

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 that includes a suite of gene editing and delivery technologies and is in the process of building internal manufacturing capabilities. Beams 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 presentation at the 2022 International HBV Meeting; our plans, and anticipated timing, to nominate additional development candidates, initiate IND-enabling studies, submit IND applications, and initiate clinical trials; our expectation that we are on-track to further expand our portfolio with a steady cadence of clinical and preclinical milestones expected in the quarters ahead; our expectations for transitioning to a multi-program clinical stage company; the potential economic benefits that may be achieved under our amended collaboration agreement with Verve Therapeutics; the therapeutic applications and potential of our technology, including with respect to SCD and our conditioning regimens, T-ALL/T-LL, GSDIa, Alpha-1, HBV, and CAR-T cells; the expected timing of enrolling the first subject in our BEACON Phase 1/2 clinical trial of BEAM-101; the clinical hold on our BEAM-201 IND, including the FDAs communication plans related to, and our plans and expectations for interactions with the FDA and the outcomes in connection therewith; the sufficiency of our capital resources to fund operating expenses and capital expenditure requirements; 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 potential impact of the COVID-19 pandemic, including its impact on the global supply chain; the uncertainty that our product candidates, including BEAM-201, will receive regulatory approval necessary to initiate human clinical studies; uncertainty in the FDAs plans to communicate and discuss the clinical hold on the BEAM-201 IND with us and the risk that those discussions may be delayed; the uncertainty in the outcome of our interactions with the FDA regarding the clinical hold on the BEAM-201 IND; 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 enrollment of our clinical trials may take longer than expected; that our product candidates may experience manufacturing or supply interruptions or failures; risks related to competitive products; 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, 2021, 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.

This press release contains hyperlinks to information that is not deemed to be incorporated by reference in this press release.

Contacts:

Investors: Chelcie Lister THRUST Strategic Communications chelcie@thrustsc.com

Media: Dan Budwick 1AB dan@1abmedia.com

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Beam Therapeutics Announces Pipeline and Business Highlights and Reports Second Quarter 2022 Financial Results - GlobeNewswire