Beam Therapeutics Reports Pipeline Updates and Second Quarter 2023 Financial Results
August 08 2023 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported second quarter 2023 financial results and provided an
update on its clinical and pipeline progress.
“The first half of 2023 has been marked by
focused execution across the business, with the singular goal of
making an impact on the lives of people suffering from serious
diseases,” said John Evans, chief executive officer of Beam. “We
are very pleased with the continued enrollment progress in the
BEACON trial, having now consented enough patients projected to
both fill the sentinel cohort and initiate the expansion cohort. In
addition, the BEAM-201 trial is now open for enrollment at multiple
clinical sites, with the first patient having been consented and
dosing expected this quarter. We have also continued to accelerate
development of BEAM-302, a potential best-in-class product
candidate for patients with alpha-1 anti-trypsin deficiency, and
are now prioritizing a BEAM-302 regulatory filing in the first
quarter of 2024 as our first in vivo program, with a regulatory
filing for BEAM-301 expected to follow shortly thereafter. Our
critical manufacturing capability in North Carolina is anticipated
to be cGMP ready for both cell manufacturing and LNP manufacturing
this year. Finally, Beam is well capitalized to pursue the next
wave of growth in its innovative research platform, from
non-genotoxic conditioning with ESCAPE in hematology, to
next-generation allogeneic cell therapies in cancer and immunology,
to a growing number of wholly owned and partnered base editing
programs targeting the liver. We believe we are well positioned to
establish an industry-leading platform in precision genetic
medicine.”
Second Quarter 2023 Business Updates and Key 2023-2024
Anticipated Milestones
Hematology Portfolio
- Beam continues to advance its 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 sickle cell
disease (SCD).
- Beam has continued to consent
additional patients in the BEACON trial, all of whom are now moving
in parallel through the screening, transfusion and mobilization
activities required to enable treatment with BEAM-101.
- Beam now anticipates that currently
consented patients are sufficient to both fill the sentinel cohort
(n=3) and to initiate the expansion cohort. Beam will continue
adding additional patients to the BEACON trial through the end of
year and beyond, with a total target of 45 treated patients.
- 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.
- The company continues to anticipate
reporting initial data on multiple patients from the BEACON trial
in 2024.
- Beam continues to advance and
invest in its Engineered Stem Cell Antibody Paired Evasion (ESCAPE)
conditioning platform.
Immunology/Oncology Portfolio
- Beam continues to advance BEAM-201,
a multiplex-edited allogeneic CAR-T product candidate, for the
treatment of relapsed/refractory T-cell acute lymphoblastic
leukemia (T-ALL)/T-cell lymphoblastic lymphoma (T-LL). Multiple
sites for the Phase 1/2 clinical trial of BEAM-201 are now open for
enrollment.
- The first patient has consented and
is expected to be dosed in the third quarter of 2023.
- Beam also continues to invest in
and advance its next-generation allogeneic strategies designed to
improve cell persistence and expand the utility and accessibility
of cell therapies in cancer and other diseases. The company plans
to share updates on these efforts by year-end 2023.
Genetic Disease (in vivo) Portfolio
- Beam continues to advance its two
in vivo base editing product candidates, BEAM-301 for the treatment
of glycogen storage disease 1a (GSD1a) and BEAM-302 for the
treatment of alpha-1 antitrypsin deficiency (AATD), leveraging
lipid nanoparticles (LNPs) for delivery to the liver.
- To promote speed to the clinic of a
top priority program, the company has leveraged the learnings and
capability build from BEAM-301 to accelerate development of
BEAM-302, which is now expected to be its first in vivo liver
regulatory filing, followed by BEAM-301.
- The company expects to:
- Submit a regulatory application for authorization to initiate
clinical trials for BEAM-302 in the first quarter of 2024; and
- Submit a regulatory application for authorization to initiate
clinical trials for BEAM-301 in the first half of 2024.
- Beam continues to advance multiple
additional in vivo editing programs targeting the liver, including
both its wholly owned and collaboration programs, through lead
optimization, and advance its LNP delivery technologies for
delivery of base editing medicines to the liver and other
tissues.
Manufacturing Updates
- Beam continues to expect initiation
of current good manufacturing practice compliant operations at its
North Carolina manufacturing facility in late 2023.
- Beam is now planning to enable cGMP
manufacturing of both autologous cell products in support of its
sickle cell programs as well as LNP products in support of its
liver programs BEAM-302 and BEAM-301 in its North Carolina
facility.
Recent Nature Genetics
Preclinical Publication Suggests Base Editing Enables More
Uniform HbF Upregulation than Nuclease Editing
- In July, Beam co-founder David Liu,
Ph.D., and St. Jude Children’s Research Hospital collaborators
Jonathan Yen, Ph.D., and Mitchell Weiss, M.D., Ph.D., published
preclinical research comparing five gene editing strategies in
CD34+ hematopoietic stem and progenitor cells using either Cas9
nuclease or adenine base editors to induce fetal hemoglobin (HbF)
red blood cells. Notably, the data suggest that base editing can
provide a strategy for potent, uniform induction of HbF, yielding a
consistent, predictable, and precise editing
outcome. Conversely, nuclease editing of either the fetal
hemoglobin gene or the BCL11A enhancer created a complex,
uncontrolled distribution of alleles with a wide range of outcomes
for induction of fetal hemoglobin, including numerous cells with
minimal or no induction detected. These data illustrate the
potential advantages of base editing’s mechanism of action,
including the creation of predictable and consistent gene
modifications with well-characterized, uniform biological effects
across edited cells, as compared to the uncontrolled mixture of
allele outcomes that result from nuclease-based knockout through
double-stranded breaks.
Second Quarter 2023 Financial Results
- Cash Position:
Cash, cash equivalents and marketable securities were $1.1 billion
as of June 30, 2023, as compared to $1.1 billion as of December 31,
2022.
- Research & Development
(R&D) Expenses: R&D expenses were $97.6 million
for the second quarter of 2023, compared to $74.6 million for the
second quarter of 2022.
- General &
Administrative (G&A) Expenses: G&A expenses were
$24.7 million for the second quarter of 2023, compared to $24.1
million for the second quarter of 2022.
- Net Loss: Net loss
was $82.8 million for the second quarter of 2023, or $1.08 per
share, compared to $72.0 million for the second quarter of 2022, or
$1.02 per share.
Cash Runway
Beam expects that its cash, cash equivalents and
marketable securities as of June 30, 2023, 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 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.
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 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: the therapeutic applications and
potential of our technology, including with respect to SCD, GSDIa,
T-ALL/TLL, and AATD; our plans, and anticipated timing, to advance
our clinical trials and programs, including our 2023-2024
anticipated milestones; our estimated cash, cash equivalents and
marketable securities as of June 30, 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; our
anticipated timing for initiating current good manufacturing
practice compliant operations at our North Carolina manufacturing
facility; 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 that we will file 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.
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) |
|
|
|
|
|
|
|
|
|
|
June 30,2023 |
|
|
December 31,2022 |
|
Cash, cash equivalents, and marketable securities |
|
$ |
1,073,016 |
|
|
$ |
1,078,134 |
|
Total assets |
|
|
1,353,887 |
|
|
|
1,341,714 |
|
Total liabilities |
|
|
542,244 |
|
|
|
608,240 |
|
Total stockholders’ equity |
|
|
811,643 |
|
|
|
733,474 |
|
Condensed Consolidated Statement of Operations
(unaudited) |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
|
Six Months Ended June 30, |
|
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
License and collaboration revenue |
|
$ |
20,116 |
|
|
$ |
16,652 |
|
|
$ |
44,324 |
|
|
$ |
25,084 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
97,608 |
|
|
|
74,556 |
|
|
|
197,254 |
|
|
|
139,966 |
|
General and administrative |
|
|
24,656 |
|
|
|
24,062 |
|
|
|
48,146 |
|
|
|
43,309 |
|
Total operating expenses |
|
|
122,264 |
|
|
|
98,618 |
|
|
|
245,400 |
|
|
|
183,275 |
|
Loss from operations |
|
|
(102,148 |
) |
|
|
(81,966 |
) |
|
|
(201,076 |
) |
|
|
(158,191 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
Change in fair value of derivative liabilities |
|
|
(900 |
) |
|
|
12,200 |
|
|
|
4,700 |
|
|
|
25,800 |
|
Change in fair value of non-controlling equity investments |
|
|
6,148 |
|
|
|
(4,124 |
) |
|
|
(6,649 |
) |
|
|
(11,809 |
) |
Change in fair value of contingent consideration liabilities |
|
|
2,171 |
|
|
|
(120 |
) |
|
|
1,875 |
|
|
|
332 |
|
Interest and other income (expense), net |
|
|
11,953 |
|
|
|
2,060 |
|
|
|
21,914 |
|
|
|
2,704 |
|
Total other income (expense) |
|
|
19,372 |
|
|
|
10,016 |
|
|
|
21,840 |
|
|
|
17,027 |
|
Net loss |
|
$ |
(82,776 |
) |
|
$ |
(71,950 |
) |
|
$ |
(179,236 |
) |
|
$ |
(141,164 |
) |
Unrealized gain (loss) on
marketable securities |
|
|
(1,250 |
) |
|
|
(1,481 |
) |
|
|
415 |
|
|
|
(4,140 |
) |
Comprehensive loss |
|
$ |
(84,026 |
) |
|
$ |
(73,431 |
) |
|
$ |
(178,821 |
) |
|
$ |
(145,304 |
) |
Net loss per common share, basic and diluted |
|
$ |
(1.08 |
) |
|
$ |
(1.02 |
) |
|
$ |
(2.41 |
) |
|
$ |
(2.03 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
76,335,175 |
|
|
|
70,210,227 |
|
|
|
74,315,721 |
|
|
|
69,461,207 |
|
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