Beam Therapeutics Announces Portfolio Progress and Reports Third Quarter 2022 Financial Results
November 07 2022 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
provided portfolio updates and reported financial results for the
third quarter, ended September 30, 2022.
“As pioneers in base editing, Beam has been at the forefront of
advancing a broad pipeline across a range of therapeutic areas and
delivery strategies, and today we report significant progress and
updates in each of these areas,” said John Evans, chief executive
officer of Beam. “Our top priority is executing the BEACON trial
for BEAM-101. Bringing a novel transplant-based medicine into
clinical development in the U.S. has been a tremendous effort, and
we are laser-focused on screening and site activation efforts to
enroll our first sickle cell patient by year-end. We are also
accelerating our overall BEAM-101 development program, where we see
an opportunity to potentially seek regulatory approval on data
generated from the BEACON trial. Given those priorities, we have
elected not to file an IND in 2022 for BEAM-102. Instead, we will
take the opportunity to streamline the BEACON trial while focusing
the next phase of our investment in sickle cell disease on our Wave
2 programs for non-genotoxic conditioning, as well as our Wave 3
strategy for in vivo lipid nanoparticle delivery of base editors,
continuing to leverage both HbF and Makassar editing
strategies.”
Evans added, “Beyond hematology, the Beam team has also executed
on the rest of our ambitious goals for advancing and expanding our
pipeline. In immune cell therapies, we have submitted our response
to the FDA clinical hold for BEAM-201, and we continue our research
toward a potential best-in-class platform for allogeneic cell
therapies leveraging the strength of multiplex base editing, which
may be the key to delivering deeper and more durable responses. In
the liver portfolio, we have initiated IND-enabling studies for
BEAM-301, to our knowledge the first gene editing program to target
direct in vivo correction of a genetic mutation. Finally, we are
very pleased to announce the selection of our second in vivo
development candidate, BEAM-302, for alpha-1 antitrypsin deficiency
(AATD). BEAM-302 is a first-in-kind program targeting direct in
vivo DNA correction of the disease-causing E342K mutation in
patients with severe AATD, which could address both the lung and
liver manifestations of the disease with a potentially one-time
treatment. I am so proud of the entire Beam team for advancing the
science and medicine of base editing across such a diverse
portfolio, and we are excited about the near-term opportunity to
bring each of these potential medicines to patients.”
Portfolio Updates & Anticipated
MilestonesEx Vivo Hematopoietic Stem Cell (HSC)
Pipeline
- Prioritization of BEAM-101 Clinical Execution:
Beam continues to focus on development of BEAM-101, which
upregulates the expression of fetal hemoglobin (HbF), as the lead
program in its Wave 1 sickle cell disease (SCD) strategy to bring
this medicine to patients as quickly as possible. Beam has
activated multiple U.S. clinical trial sites, which are now
recruiting patients for its BEACON trial, an open-label,
single-arm, multicenter, Phase 1/2 clinical trial evaluating the
safety and efficacy of BEAM-101 in adult patients with severe SCD.
Per the BEACON trial protocol, once the first patient is enrolled,
they will undergo a transfusion and mobilization process for HSC
retrieval. The cells are then edited, creating an autologous
BEAM-101 drug product. Following the drug product manufacturing,
the patient will receive pre-treatment conditioning using a
standard-of-care chemotherapy regimen, after which the edited cells
are transplanted back into the patient. Following successful
engraftment, treatment of a second patient can proceed. Beam
continues to expect enrollment of the first patient in the BEACON
trial by the end of 2022.To accelerate development of BEAM-101,
Beam is currently working to amend the BEACON trial protocol to
enable clinical trial modifications designed to streamline and
expedite patient enrollment and trial conduct based on the recent
regulatory landscape in SCD trials. The company also plans to make
investments required to finalize its commercial manufacturing
process for BEAM-101.
- Decision to Forgo BEAM-102 IND in 2022 and Optimize
Program for Future SCD Waves: Beam has continued efforts
in its Wave 2 strategy, which is designed to enable an improved,
reduced-toxicity conditioning regimen for patients undergoing HSC
transplantation, as well as its Wave 3 strategy, which is focused
on in vivo delivery of base editors directly to HSCs. In connection
with these efforts, Beam will not submit an investigational new
drug (IND) application for BEAM-102, its base editing program
designed to treat SCD by directly editing the causative HbS point
mutation to create the naturally occurring normal human hemoglobin
variant, HbG-Makassar, in 2022. Instead, the company plans to
optimize its Makassar approach, alongside its HbF upregulation
approach, as part of its future ex vivo and in vivo HSC candidates
in Wave 2 and Wave 3 development, respectively.
- Progress with Wave 2 Non-genotoxic Conditioning to be
Reported at ASH: Beam plans to present new data on its
improved conditioning technologies at the American Society of
Hematology (ASH) Annual Meeting in December. Beam has leveraged its
base editing capabilities to develop a potentially non-genotoxic
approach to SCD treatment that combines antibody-based conditioning
with multiplex gene-edited HSCs called ESCAPE (Engineered Stem Cell
Antibody Paired Evasion). ESCAPE-1 features the upregulation of
fetal hemoglobin, the mechanism for BEAM-101, and ESCAPE-2 features
HbG Makassar editing, the mechanism for BEAM-102.
Ex Vivo T Cell Pipeline
- BEAM-201 IND Response Submitted to FDA:
BEAM-201 is a first-in-kind, potent and specific anti-CD7,
multiplex-edited, allogeneic chimeric antigen receptor T cell
(CAR-T) development candidate. Beam submitted an IND for BEAM-201
to the FDA in June 2022, and in July 2022, was notified that the
IND was placed on clinical hold. Beam has submitted its response to
the FDA and will provide an update on next steps, as
available.
In Vivo LNP Liver-targeting Pipeline
- IND-enabling Studies Initiated for BEAM-301:
BEAM-301 is designed to treat glycogen storage disease 1a (GSDIa),
an autosomal recessive disorder caused by mutations in the G6PC
gene that disrupt a key enzyme, glucose-6-phosphatase (G6Pase),
critical for maintaining glucose homeostasis. Inhibition of G6Pase
activity results in low fasting blood glucose levels that can
result in seizures and be fatal. 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 of GSDIa.
- BEAM-302 Named Development Candidate for Alpha-1
Antitrypsin Deficiency (AATD): Beam has nominated its
second in vivo development candidate, BEAM-302, which it plans to
develop as a potential one-time treatment to genetically correct
the E342K point mutation (PiZZ genotype), which is most commonly
responsible for severe AATD. AATD is a rare, inherited genetic
disorder that can cause early onset emphysema and liver
disease.
- Patients with severe AATD (PiZZ) have accumulation of a
non-functional protein in the liver (toxic gain of function) that
causes liver damage and a decrease in circulating alpha-1
antitrypsin (AAT) (toxic loss of function) that results in lung
damage due to insufficient inhibition of neutrophil elastase.
Augmentation therapy with plasma-purified AAT is currently utilized
in patients with lung disease and may slow lung function decline,
but definitive data on long-term clinical outcomes, such as quality
of life, frequency of exacerbations or survival, are limited. There
are no FDA-approved therapies that can simultaneously treat
AATD-related lung and liver disease.
- In mouse models of AATD, LNP delivery of BEAM-302 was capable
of efficiently correcting the PiZ mutation at clinically relevant
doses of <1 mg/kg. Optimization of both the guide and editor for
BEAM-302 has resulted in a greater than two-fold improvement in
editing efficiency compared with previously published precursor
reagents.
- The increased level of mutation correction achieved with
BEAM-302 increased secretion of functional AAT protein to levels
that could be therapeutically relevant for protection of lung
function in patients with AATD.
- Prior precursor editors and guides have also demonstrated a
corresponding reduction of toxic liver aggregates, suggesting the
first-in-class potential of BEAM-302 to treat both lung and liver
manifestations of the disease.
- Beam has initiated preparations for preclinical manufacturing
and preclinical development of BEAM-302.
- First In Vivo Data
for Multiplex Base Editing Approach to Treat Chronic HBV
Infection: In September, Beam presented new preclinical
data demonstrating the potential of the company’s multiplex base
editing approach to both reduce viral markers – including hepatitis
B surface antigen (HBsAg) expression – and prevent viral rebound of
hepatitis B virus (HBV) in in vivo models.
Third Quarter 2022 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $1.1 billion as of September 30, 2022,
as compared to $965.6 million as of December 31, 2021.
- Research & Development (R&D) Expenses:
R&D expenses were $85.3 million for the third quarter of 2022,
compared to $54.6 million for the third quarter of 2021.
- General & Administrative (G&A)
Expenses: G&A expenses were $21.8 million for the
third quarter of 2022, compared to $15.8 million for the third
quarter of 2021.
- Net Loss: Net loss was $109.6 million for the
third quarter of 2022, or $1.56 per share, compared to $28.1
million for the third quarter of 2021, or $0.42 per share.
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 presentation at the
ASH annual meeting; our plans, and anticipated timing, to advance
our programs; our expectations for transitioning to a multi-program
clinical stage company; the therapeutic applications and potential
of our technology, including with respect to SCD and our
conditioning regimens, GSDIa, AATD, 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 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 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;
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 ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
Condensed
Consolidated Balance Sheet Data (unaudited) |
|
|
|
|
(in
thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30, 2022 |
|
December 31, 2021 |
|
|
|
|
Cash, cash
equivalents, and marketable securities |
|
$ |
1,094,554 |
|
|
$ |
965,647 |
|
|
|
|
|
Total
assets |
|
|
1,350,250 |
|
|
|
1,474,453 |
|
|
|
|
|
Total
liabilities |
|
|
635,773 |
|
|
|
647,715 |
|
|
|
|
|
Total
stockholders’ equity |
|
|
714,477 |
|
|
|
826,738 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Condensed
Consolidated Statement of Operations (unaudited) |
(in
thousands, except share and per share data) |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
|
|
2022 |
|
|
|
2021 |
|
|
|
2022 |
|
|
|
2021 |
|
License and
collaboration revenue |
|
$ |
15,799 |
|
|
$ |
763 |
|
|
$ |
40,883 |
|
|
$ |
775 |
|
Operating
expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
85,287 |
|
|
|
54,623 |
|
|
|
225,253 |
|
|
|
290,306 |
|
General and administrative |
|
|
21,815 |
|
|
|
15,774 |
|
|
|
65,124 |
|
|
|
39,450 |
|
Total operating expenses |
|
|
107,102 |
|
|
|
70,397 |
|
|
|
290,377 |
|
|
|
329,756 |
|
Loss from
operations |
|
|
(91,303 |
) |
|
|
(69,634 |
) |
|
|
(249,494 |
) |
|
|
(328,981 |
) |
Other income
(expense): |
|
|
|
|
|
|
|
|
Change in fair value of derivative liabilities |
|
|
(4,900 |
) |
|
|
35,800 |
|
|
|
20,900 |
|
|
|
(8,400 |
) |
Change in fair value of non-controlling equity investments |
|
|
10,431 |
|
|
|
(4,892 |
) |
|
|
(1,378 |
) |
|
|
21,960 |
|
Change in fair value of contingent consideration liabilities |
|
|
(875 |
) |
|
|
10,599 |
|
|
|
(543 |
) |
|
|
9,553 |
|
Interest and other income (expense), net |
|
|
4,982 |
|
|
|
9 |
|
|
|
7,686 |
|
|
|
(63 |
) |
Total other income (expense) |
|
|
9,638 |
|
|
|
41,516 |
|
|
|
26,665 |
|
|
|
23,050 |
|
Net loss
before income taxes |
|
|
(81,665 |
) |
|
|
(28,118 |
) |
|
|
(222,829 |
) |
|
|
(305,931 |
) |
Provision for income taxes |
|
|
(2,410 |
) |
|
|
— |
|
|
|
(2,410 |
) |
|
|
— |
|
Loss from equity method investment |
|
|
(25,500 |
) |
|
|
— |
|
|
|
(25,500 |
) |
|
|
— |
|
Net
loss |
|
$ |
(109,575 |
) |
|
$ |
(28,118 |
) |
|
$ |
(250,739 |
) |
|
$ |
(305,931 |
) |
Unrealized gain (loss) on marketable securities |
|
|
(484 |
) |
|
|
(12 |
) |
|
|
(4,624 |
) |
|
|
28 |
|
Comprehensive loss |
|
$ |
(110,059 |
) |
|
$ |
(28,130 |
) |
|
$ |
(255,363 |
) |
|
$ |
(305,903 |
) |
Net loss per
common share, basic and diluted |
|
$ |
(1.56 |
) |
|
$ |
(0.42 |
) |
|
$ |
(3.59 |
) |
|
$ |
(4.86 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
70,343,196 |
|
|
|
66,377,611 |
|
|
|
69,758,434 |
|
|
|
62,960,219 |
|
|
|
|
|
|
|
|
|
|
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