Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported progress across the company’s hematology and genetic
disease portfolios and provided updates on anticipated upcoming
milestones.
“Our vision is to establish Beam as a sustainable, fully
integrated company pioneering a new class of genetic medicines with
base editing. We made tremendous progress toward this goal in 2023,
including opening our own GMP manufacturing facility, dosing the
first patients in multiple ex vivo clinical programs, including
BEAM-101 for sickle cell disease (SCD), and accelerating our in
vivo program for alpha-1 antitrypsin deficiency (AATD), as
exemplified by the filing of our CTA for BEAM-302,” said John
Evans, chief executive officer of Beam Therapeutics. “Building on
this momentum and benefiting from the significant clinical
validation, regulatory clarity, and scientific breakthroughs
occurring in the broader gene editing field, we expect 2024 to be a
year of significant catalysts for Beam. Our highly differentiated
SCD and AATD programs have the potential to provide best-in-class
therapies for significant patient populations with high unmet need,
while also establishing a platform for sustainable long-term growth
across multiple therapeutic areas.”
Pipeline Updates and 2024 Anticipated
Milestones
Sickle Cell Disease (SCD) Franchise
Beam is pursuing a long-term, staged development strategy for
SCD that has three Waves of innovation intended to progressively
expand the reach of our base editing approach to broader subsets of
patients.
- Wave 1: BEAM-101 is 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. Preclinical models suggest base editing could lead to
improved HbF induction and lower residual disease-causing
hemoglobin S compared to existing gene therapy options.
- The first patient was dosed in the fourth quarter of 2023 and
successfully achieved engraftment in the BEACON Phase 1/2 clinical
trial, an open-label, single-arm, multicenter study evaluating the
safety and efficacy of BEAM-101 in adult patients with severe SCD.
Treatment with BEAM-101, in which the edited cell product is
delivered in an autologous bone marrow transplant, will occur on a
sequential basis for the first three patients treated in the trial,
and then will be given in parallel for all subsequent
patients.
- Patients have continued to be consented in the BEACON trial,
and Beam anticipates dosing the remaining two patients in the
sentinel cohort and initiating dosing in patients in the expansion
cohort in the first half of 2024.
- The company is on-track to report initial data on multiple
patients from the BEACON trial in the second half of 2024.
- Wave 2: Beam continues to advance and invest
in its Engineered Stem Cell Antibody Paired Evasion (ESCAPE)
conditioning platform and anticipates initiating Phase 1-enabling
preclinical studies for the program in 2024. ESCAPE aims to avoid
the toxicities associated with currently available conditioning
regimens for patients with SCD required prior to autologous
transplant.
- Wave 3: The company is also 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 seeks to treat genetic diseases using single course gene
editing therapies delivered through intravenous infusion of LNPs,
which are a clinically validated technology for delivery of nucleic
acid payloads to the liver.
- BEAM-302, the company’s priority genetic disease program, is a
potential treatment for AATD, which is characterized by early onset
emphysema, liver disease, and increased all-cause mortality
compared to the general population. There is a large unmet need for
novel therapies that can treat patients with AATD-associated lung
and liver disease.
- BEAM-302 is 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 AATD.
Approximately 100,000 patients in the U.S. are estimated to carry
the PiZZ genotype.
- Preclinical data to date demonstrated that treatment with
BEAM-302 led to significantly increased levels of corrected and
functional alpha-1 antitrypsin (AAT) and reduced mutant PiZ AAT in
multiple in vivo rodent disease models at clinically relevant
doses. These findings support the potential of BEAM-302 to
efficiently correct the disease-causal PiZ mutation after a single
dose and potentially address both the liver and lung disease
associated with AATD.
- Beam has filed a CTA for BEAM-302, and, assuming CTA
acceptance, plans to initiate a clinical trial for BEAM-302 in the
first half of 2024.
- In addition, Beam is also advancing BEAM-301 for the potential
treatment of glycogen storage disease type 1a (GSD1a), an autosomal
recessive disorder caused by mutations in the G6PC gene that
disrupt a key enzyme, glucose-6-phosphatase, involved in
maintaining glucose homeostasis.
- 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
GSD1a.
- Preclinical data have shown that a single administration of
BEAM-301 directly and durably corrected the R83C mutation in vivo,
with an ongoing significant survival benefit one year after the
initial dosing.
- Beam is focusing initial development of BEAM-301 in the U.S.
and expects to submit an investigational new drug (IND) application
in the first half of 2024.
- Beam is also advancing lead liver-targeted programs from its
collaborations with Pfizer and Apellis.
Sustainable Research Portfolio
- Beam’s near-term research and platform investments are focused
on specific applications leveraging Beam’s in vivo editing
capabilities in the liver targeting both rare and common genetic
disorders, as well as opportunities in hematology and
immunology/oncology.
- Enrollment in the company’s Phase 1/2 clinical trial of
BEAM-201, a multiplex-edited allogeneic CAR-T product candidate, is
ongoing for the treatment of relapsed/refractory T-cell acute
lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma
(T-LL). Beam expects to report an initial clinical dataset for
BEAM-201 in the second half of 2024.
Cash Position and Updated Operating Runway
As of December 31, 2023, Beam estimates that it had $1.2 billion
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, 2023, and its results
of operations for the three months and year ended December 31,
2023. 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, 2023 will enable the
company to fund its anticipated operating expenses and capital
expenditure requirements into 2027. This expectation assumes
anticipated cost savings related to the company’s previously
announced portfolio prioritization and streamlining of operations
and includes funding directed toward reaching each of the key
anticipated 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 Conference
Beam management will present and discuss Beam’s pipeline and
business updates during a presentation at the 42nd Annual J.P.
Morgan Healthcare Conference today, Monday, January 8, 2024,
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 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. 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 42nd
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, AATD, GSD1a, T-ALL/TLL, and our
conditioning regimens; our plans, and anticipated timing, to
advance our programs, the clinical trial designs and expectations
for BEAM-101, BEAM-201, BEAM-301 and BEAM-302; our estimated
cash, cash equivalents and marketable securities as of
December 31, 2023 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 successfully achieve the benefits of our
portfolio prioritization and strategic restructuring; 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; whether our actual
audited results will be consistent with our estimated cash, cash
equivalents and marketable securities as of December 31, 2023;
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 March 31, 2023, our
Quarterly Report on Form 10-Q for the quarter ended June 30, 2023,
our Quarterly Report on Form 10-Q for the quarter ended September
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:Holly ManningTHRUST
Strategic Communicationsholly@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
Beam Therapeutics (NASDAQ:BEAM)
Gráfico Histórico do Ativo
De Abr 2024 até Mai 2024
Beam Therapeutics (NASDAQ:BEAM)
Gráfico Histórico do Ativo
De Mai 2023 até Mai 2024