Beam Therapeutics Reports Progress Across Base Editing Portfolio and Outlines Key Anticipated Milestones
January 09 2023 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported progress across the company’s hematology,
immunology-oncology and genetic disease portfolios and provided
updates on anticipated upcoming milestones.
“Beam enters 2023 with significant momentum across all of our
core pipeline areas and an expanding leadership position in the
next generation of gene editing,” said John Evans, chief executive
officer of Beam. “We have multiple clinical-stage candidates with
BEAM-101 and BEAM-201, another two candidates – BEAM-301 and
BEAM-302 – moving toward clinical trials, and an integrated
platform of editing technologies, scalable manufacturing
capabilities and diverse delivery modalities. We are also making
key investments in long-term platform opportunities that may
dramatically expand the reach and impact of base editing and create
a sustainable pipeline of highly differentiated programs, including
improved conditioning for transplant, allogeneic cell therapies and
in vivo delivery. I’m so proud of the accomplishments this
organization has achieved in our first five years of operations. We
believe we now have a unique opportunity to deliver on the
potential of our science and achieve our mission of bringing
life-changing treatments to patients suffering from serious
diseases.”
Hematology Portfolio
- BEAM-101: In November 2022, Beam enrolled the
first patient in its BEACON clinical trial evaluating BEAM-101 as a
treatment for sickle cell disease (SCD). Beam expects to complete
enrollment in the sentinel cohort and initiate enrollment in the
expansion cohort of BEACON in 2023, with plans to report data from
multiple patients from one or both cohorts in 2024. BEAM-101 is a
patient-specific, autologous hematopoietic stem cell (HSC)
investigational therapy designed to offer a potentially
best-in-class profile, incorporating base edits that are intended
to mimic single nucleotide polymorphisms seen in individuals with
hereditary persistence of fetal hemoglobin. BEAM-101 aims to
potentially alleviate the effects of SCD or beta-thalassemia by
leading to increases in fetal hemoglobin, which is expected to
restore the formation of a functional hemoglobin tetramer and, in
the case of SCD, inhibit hemoglobin S polymerization.
- Platform Opportunity: Beam is advancing its
Engineered Stem Cell Antibody Paired Evasion (ESCAPE) conditioning
strategy in an effort to bring base editing treatments to more
patients. ESCAPE aims to avoid toxicity challenges associated with
currently available conditioning regimens for patients with SCD and
beta-thalassemia ahead of autologous transplant. ESCAPE may also
have applications in other diseases of the blood and immune system
where transplant could deliver potential benefits but has been
limited by toxicities associated with standard conditioning
regimens. In December 2022, Beam presented in vivo proof-of-concept
data at the American Society of Hematology Annual Meeting and
Exposition (ASH) highlighting its potential. Beam has made
significant investments in its ESCAPE platform and plans to
continue its advancement in 2023.
Immunology-oncology Portfolio
- BEAM-201: In December 2022, Beam received
clearance from the FDA for its Investigational New Drug (IND)
application for BEAM-201. The company has initiated a
first-in-human Phase 1/2 clinical trial to evaluate the safety and
efficacy of BEAM-201 in patients with relapsed/refractory T-cell
acute lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma
(T-LL) and expects to dose the first patient by mid-2023. The Phase
1 portion of the trial is expected to include up to 48 patients
between the ages of 18 and 50, followed by a Phase 2 portion with
approximately 48 patients. 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 HSC
transplant, and proportion achieving minimal residual disease
negative status. Beam believes that BEAM-201 is the first
quadruple-edited, allogeneic CAR-T cell investigational therapy in
clinical-stage development. BEAM-201 is designed to target CD7 to
treat relapsed/refractory T-ALL/T-LL, a severe disease affecting
children and adults.
- Platform Opportunity: Beyond BEAM-201, Beam
continues to research potential next-generation allogeneic
strategies that could dramatically expand the utility and
accessibility of cell therapies in cancer and other diseases. Beam
anticipates that multiple edits will be required to enable
allogeneic cells to successfully avoid immune rejection and provide
the cells with other desirable properties. Beam believes that
multiplex base editing, with its high potency, efficiency in
editing and lack of double-strand breaks, is well suited for making
such highly engineered cells, and anticipates providing additional
updates on this research in 2023.
Genetic Disease Portfolio
- BEAM-301: IND-enabling studies for BEAM-301
continue, and by late 2023 or early 2024, the company plans to
submit a regulatory application for authorization to initiate
clinical trials for the program. BEAM-301 is a liver-targeting
lipid nanoparticle (LNP) formulation of base editing reagents
designed to correct the R83C mutation, the most common
disease-causing mutation which results in the most severe form of
glycogen storage disease 1a (GSDIa). GSDIa is an autosomal
recessive disorder caused by mutations in the G6PC gene that
disrupt a key enzyme, glucose-6-phosphatase, critical for
maintaining glucose homeostasis. Patients with this mutation
typically require ongoing corn starch administration, without
which, they may enter into hypoglycemic shock within one to three
hours.
- BEAM-302: Beam also continues to advance its
second liver-targeted in vivo program, BEAM-302, and in early 2024,
plans to submit a regulatory application for authorization to
initiate clinical trials for the program. BEAM-302 is designed to
offer a one-time treatment to genetically correct the E342K point
mutation (PiZZ genotype), which is most commonly responsible for
severe alpha-1 antitrypsin deficiency (AATD). AATD is an inherited
genetic disorder that can cause early onset emphysema and liver
disease.
- Platform Opportunity: Beam continues to
advance its LNP delivery technologies using its barcode screening
technology, which is designed to enable delivery of base editing
treatments to the liver and tissues beyond, potentially expanding
the number of diseases and patients that could benefit from base
editing medicines.
Manufacturing Facility
- Beam continues to expect operations at its North Carolina
manufacturing facility to commence in the first quarter of 2023 and
expects to initiate current good manufacturing practice compliant
operations in late 2023.
Cash Position and Runway
- Cash Position: Beam estimates that it had
cash, cash equivalents and marketable securities of approximately
$1.0 billion as of December 31, 2022. 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, 2022, and its results of operations
for the three months and year ended December 31, 2022. Accordingly,
undue reliance should not be placed on this preliminary
estimate.
- Cash Runway: Beam expects that its cash, cash
equivalents and marketable securities as of December 31, 2022, will
enable the company to fund its anticipated operating expenses and
capital expenditure requirements at least into 2025. This
expectation includes funding directed toward reaching each of the
key milestones for BEAM-101, BEAM-201, BEAM-301 and BEAM-302
described above, as well as continued investments in platform
advancements and manufacturing capabilities.
J.P. Morgan Healthcare ConferenceBeam
management will present and discuss Beam’s pipeline and business
updates during a presentation at the 41st Annual J.P. Morgan
Healthcare Conference today, Monday, January 9, 2023, at 11:15 a.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 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: our upcoming presentations at
the 41st Annual J.P. Morgan Healthcare Conference; our expectations
for transitioning to a multi-program clinical stage company; the
therapeutic applications and potential of our technology, including
with respect to SCD, GSDIa, T-ALL/TLL, AATD and our conditioning
regimens; the clinical trial design for BEAM-201; our plans, and
anticipated timing, to advance our programs; our estimated cash,
cash equivalents and marketable securities as of December 31, 2022
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 potential impact of the COVID-19 pandemic,
including its 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
enrollment and initiation 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; whether our actual audited results will be
consistent with our estimated cash, cash equivalents and marketable
securities as of December 31, 2022; 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, our Quarterly Report on Form 10-Q for the
quarter ended September 30, 2022, 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 ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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