Beam Therapeutics Announces First Patient Dosed in Phase 1/2 Trial of BEAM-201 in Relapsed, Refractory T-ALL/T-LL
September 05 2023 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced that in August, the first patient was treated with
BEAM-201, a quadruplex-edited allogeneic CAR-T cell investigational
therapy. BEAM-201 is being evaluated in a Phase 1/2 clinical study
for the treatment of relapsed/refractory T-cell acute lymphoblastic
leukemia/T-cell lymphoblastic lymphoma (T-ALL/T-LL), a severe
disease affecting children and adults.
“As the first patient dosed with a Beam therapeutic candidate
and the first patient in the U.S. to receive a base editing
therapeutic, this represents a major milestone for the company, the
scientists that made this possible, and the patients we hope to
serve,” said John Evans, chief executive officer of Beam. “We
believe that the full therapeutic potential of CAR-T therapies,
including the ability to utilize an allogeneic source of T cells,
will only be unlocked through higher levels of cellular engineering
enabled by multiple simultaneous genetic edits. Base editing is
especially well-suited to this challenge, as it is designed to
deliver highly efficient multiplex edits in cells without the
double stranded breaks that can lead to frequent chromosomal
rearrangements and loss of cell viability. BEAM-201, to our
knowledge the first quadruplex-edited cell therapy candidate in
clinical development, is an allogeneic CAR-T cell investigational
therapy with the potential to make a substantial impact for
patients diagnosed with challenging T-cell cancers who have limited
treatment options.”
About the BEAM-201 Phase 1/2 Clinical TrialThe
ongoing Phase 1/2 trial of BEAM-201 is a multicenter, open-label
study evaluating safety and efficacy in patients with
relapsed/refractory T-ALL/T-LL. T-ALL/T-LL is a highly aggressive
blood cancer arising from malignant transformation of T cell
precursors that has few treatment options.
The primary objectives of the Phase 1 portion of the trial are
the assessment of safety, tolerability, and identification of the
recommended Phase 2 dose and lymphodepletion regimen. Key safety
endpoints for the trial include treatment-emergent and
treatment-related adverse events, and key efficacy endpoints
include proportion of patients with complete or partial responses,
proportion eligible for hematopoietic stem cell transplant, and
proportion achieving minimal residual disease negative status.
Multiple sites for the Phase 1/2 clinical trial of BEAM-201 are now
open for enrollment. For more information, visit clinicaltrials.gov
(NCT05885464).
About BEAM-201BEAM-201 is a quadruplex
base-edited, anti-CD7 allogeneic chimeric antigen receptor T cell
(CAR-T) under clinical investigation for the treatment of CD7+
relapsed/refractory T-cell acute lymphoblastic leukemia/T-cell
lymphoblastic lymphoma (T-ALL/T-LL). Multiplexed base editing is
designed to eliminate expression of the CD7, TRAC, PDCD1 and CD52
genes. This approach has the potential to reduce fratricide,
graft-versus-host disease and CAR-T cell exhaustion and to enable
BEAM-201 cells to evade anti-CD52 lymphodepleting agents and enable
use of an allogeneic cell source.
About Beam TherapeuticsBeam 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.
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
StatementsThis 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: the therapeutic applications and
potential of our technology, including with respect to T-ALL/TLL;
our plans, and anticipated timing, to advance our clinical trials
and programs, including our trial of BEAM-201; 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
pandemics and other health emergencies, including their impact on
the global supply chain; the uncertainty that our product
candidates will receive regulatory approval necessary to initiate
human clinical studies; 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 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, 2022, our Quarterly Report on Form 10-Q for the
quarter ended June 30, 2023, 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:Chelcie ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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