Beam Therapeutics Presents Preclinical Data Highlighting Utility and Durability of BEAM-301 to Correct a Glycogen Storage Disease Type I Deficiency Disease-Causing Mutation at ESGCT
October 25 2023 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported new preclinical data demonstrating the ability of its in
vivo drug candidate, BEAM-301, to directly correct the R83C
mutation, one of the primary disease-causing mutations of glycogen
storage disease type Ia (GSDIa). The data were presented today in
an oral presentation titled “A Single, Systemic Administration of
BEAM-301 Mitigated Fasting Hypoglycemia One Year after Dosing in a
Transgenic Mouse Model of Glycogen Storage Disease Type-Ia” at the
30th Annual European Society of Gene & Cell Therapy (ESGCT)
Congress in Brussels.
“GSDIa is a devastating disease that significantly impacts an
individual’s quality of life and puts them at consistent risk of
hypoglycemia, coma and potentially death. Today, there are limited
options, requiring patients to adhere to burdensome dietary
regimens in order to maintain blood glucose at an appropriate
level,” said Giuseppe Ciaramella, Ph.D., president of Beam. “We
designed BEAM-301 with these patients in mind, aiming to create a
one-time treatment that could correct the disease-causing mutation
and improve their glucose control and other metabolic parameters.
Today’s data continue to show that treatment with BEAM-301 yielded
potent and durable liver editing that translated into normalization
of blood glucose without continuous supplementation and improved
metabolic parameters and survival in a mouse model of homozygous
GSDIa.”
Dr. Ciaramella continued, “These compelling and durable data
support the continued development of BEAM-301 as a potential
treatment to address the unmet need for these patients. We’re
encouraged by the recent approvals of IND applications for clinical
trials investigating both nuclease editing and base editing
therapeutics by the U.S. FDA, and believe it indicates the agency’s
support of the benefit risk profile of gene editing to address
diseases with unmet medical needs. We remain focused on the
submission of our IND application in the first half of next year
and plan to conduct an initial BEAM-301 clinical trial for the
treatment of GSDIa at a select number of sites in the United
States."
GSDIa is a genetic disease caused by mutations in the G6PC gene
encoding glucose-6-phosphatase (G6Pase), a predominantly
liver-expressed enzyme vital to glucose metabolism. The prevalent
pathogenic variant, G6PC-p.R83C, completely abolishes G6Pase
activity and is associated with life-threatening fasting
hypoglycemia as well as long-term complications impacting the liver
and kidney. BEAM-301 is a liver-targeting lipid-nanoparticle (LNP)
formulation containing base editing reagents optimized to correct
the R83C mutation.
Beam evaluated the ability of its novel base-editing candidate,
BEAM-301, to correct the R83C mutation in a transgenic GSDIa mouse
model that is homozygous for human G6PC-p.R83C (huR83C) and
deficient of G6Pase activity. Beam previously demonstrated that
treatment with a single dose of BEAM-301 yielded up to ~60%
base-editing efficiency to correct the R83C mutation and was
associated with restored G6Pase activity in the livers of young
huR83C mice. Today’s data build on those findings and show that a
single dose of BEAM-301 yielded:
- Long-term survival of treated mice out to at least one year
post treatment, compared to untreated GSDIa mice that exhibit poor
survival of only a few weeks;
- Sustained editing of G6PC in liver, confirmed by the
normalization of glucose homeostasis and glycogen accumulation at
one year (the longest time point assessed);
- Normal growth and liver size throughout one year post treatment
relative to untreated homozygous mutant mice that developed
three-fold larger liver size by three weeks of age;
- Normalization of circulating glucose and metabolites, including
cholesterol, triglycerides, lactic and uric acid; and,
- Prevention of hypoglycemia during several, intermittent 24-hour
fasts up through one year post dosing.
These findings support the potential of BEAM-301 to directly
correct the disease-causing R83C mutation with a single dose. Beam
plans to submit a U.S. IND application in the first half of 2024
for authorization to initiate clinical trials of BEAM-301.
About Glycogen Storage Disease Type IaGSDIa is
an autosomal recessive disorder caused by mutations in the G6PC
gene that disrupt a key enzyme, glucose-6-phosphatase (G6Pase),
involved in maintaining glucose homeostasis. Inhibition of G6Pase
activity results in low fasting blood glucose levels that can be
fatal. Beam is advancing BEAM-301, composed of a guide RNA and an
mRNA encoding an adenine base editor (ABE) delivered via LNP, which
aims to directly correct the R83C mutation, one of the primary
disease-causing mutations of GSDIa.
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 GSDIa; our
plans, and anticipated timing, to submit a regulatory application
for authorization to initiate clinical trials of BEAM-301; 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 March 31, 2023, 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:Holly ManningTHRUST
Strategic Communicationsholly@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
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