Beam Therapeutics Reports Pipeline and Business Updates and Third Quarter 2023 Financial Results
08 November 2023 - 12:30PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported third quarter 2023 financial results and provided an
update on the business and its clinical and pipeline progress.
“This year, Beam has established an exceptional foundation for
future growth, with significant financial strength, a broad
portfolio of differentiated base editing programs in large
addressable markets driving toward clinical milestones, and a fully
operational suite of research, development, and manufacturing
capabilities for precision genetic medicines,” said John Evans,
chief executive officer of Beam. “We have made critical decisions
on where to focus and how to prioritize our investments over the
coming years, creating significant momentum in our business and
portfolio. In the BEACON trial, patient enrollment, mobilization,
and manufacturing are progressing well, and we remain on track to
treat the first trial patient with BEAM-101 this year and report
clinical trial data on multiple patients next year. We’re also
advancing our first in vivo program, BEAM-302, for patients with
alpha-1 anti-trypsin deficiency with a regulatory filing expected
in the first quarter of next year, followed by our planned IND
submission for BEAM-301 shortly thereafter. External interest in
our base editing technology continues to grow, illustrated most
recently by our transaction with Lilly. With a strong balance
sheet, a focused team, and leading capabilities, we are well
positioned to advance our base editing programs and platform, which
we believe have transformative potential for patients.”
Pipeline Updates and Key 2023-2024 Anticipated
Milestones
Beam recently announced portfolio priorities and plans to
streamline its business operations to support potential near-term
value drivers and long-term growth and, as such, is now focusing
its efforts as follows:
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). 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.
- Beam has continued to consent additional patients in the BEACON
trial, all of whom are now moving in parallel through the
screening, mobilization, and manufacturing activities required to
enable treatment with BEAM-101. Beam anticipates that currently
consented patients are sufficient to both fill the sentinel cohort
(n=3) and to initiate the expansion cohort.
- The first patient in the trial is expected to be treated with
BEAM-101 by year-end 2023.
- 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.
- The company anticipates 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. ESCAPE
aims to avoid toxicity challenges associated with currently
available conditioning regimens for patients with SCD and
beta-thalassemia ahead of autologous transplant.
- In addition, the company is exploring the potential for in vivo
base editing programs for SCD, in which base editors would be
delivered to the patient through an infusion of lipid nanoparticles
(LNPs) targeted to hematopoietic stem cells (HSCs), eliminating the
need for transplantation altogether.
Genetic Disease (in vivo) Portfolio
- Beam continues to advance its two lead in vivo base editing
product candidates, BEAM-302 for the treatment of alpha-1
antitrypsin deficiency (AATD) and BEAM-301 for the treatment of
glycogen storage disease Ia (GSDIa). BEAM-302 and BEAM-301 have the
potential to be the first clinical programs to directly correct a
genetic mutation back to a normal functional gene sequence.
- BEAM-302 is a liver-targeting LNP formulation of base editing
reagents designed to correct the PiZ allele, the most common gene
variant associated with severe AATD. The company expects to submit
a regulatory application in the first quarter of 2024 for
authorization to initiate clinical trials initially outside of the
U.S., with an Investigational New Drug (IND) expected to be filed
with the U.S. Food and Drug Administration (FDA) subsequently
during early development.
- In September 2023, Beam reported the first preclinical
data demonstrating the ability of BEAM-302 to significantly
increase levels of corrected and functional alpha-1 antitrypsin
(AAT) and reduce mutant PiZ AAT in multiple in vivo rodent disease
models at clinically relevant doses in an oral presentation at the
Alpha-1 Antitrypsin Deficiency 2023 Meeting in Naples, Italy. 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.
- An encore presentation at the ESGCT/SFTCG/NVGCT Collaborative
Congress in October 2023 showed additional preclinical data
supporting the durable effects of BEAM-302.
- 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
GSDIa. Given its rare nature and geographic distribution of disease
burden, the company will focus development of BEAM-301 in the U.S.
and expects to submit an IND application in the first half of 2024.
- In October 2023, Beam presented new preclinical data
demonstrating the ability of a single administration of BEAM-301 to
directly and durably correct the R83C mutation in vivo, with
results lasting more than one year, in an oral presentation at the
30th Annual European Society of Gene & Cell Therapy (ESGCT)
Congress in Brussels.
Immunology/Oncology Portfolio
- In September 2023, Beam announced dosing of the first patient
in the Phase 1/2 clinical trial of 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). The Phase 1/2 trial
continues to enroll, and the company expects to report initial data
in 2024.
- Beam plans to generate a focused clinical dataset for BEAM-201
and seek potential partnership for this and other potential ex vivo
CAR-T programs, including Beam’s ongoing research into creating
next-generation allogeneic cell therapies with multiplex base
editing.
Research Portfolio
- Beam plans to focus near-term research and platform investments
on specific applications leveraging Beam’s in vivo editing
capabilities in the liver targeting both rare genetic and common
disorders, as well as select opportunities in hematology and
immunology/oncology.
Corporate Updates
- Manufacturing: Beam has initiated current good
manufacturing practice (GMP) operations at its North Carolina
manufacturing facility, where capabilities include both CD34 cell
manufacturing and LNP production, with additional capabilities
expected to be added in the future.
- Business Development: In October 2023, Beam
announced that Eli Lilly and Company (Lilly) acquired certain
rights under Beam’s amended collaboration and license agreement
with Verve Therapeutics, Inc. (Verve), including Beam’s opt-in
rights to co-develop and co-commercialize Verve’s base editing
programs for cardiovascular disease, which includes programs
targeting PCSK9, ANGPTL3, and an undisclosed liver-mediated,
cardiovascular target. Beam received a $200 million upfront payment
and a $50 million equity investment from Lilly. Beam is also
eligible to receive up to $350 million in potential future
development-stage payments from Lilly upon the completion of
certain clinical, regulatory and alliance events for a total of up
to $600 million in potential total deal consideration.
Third Quarter 2023 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities, excluding upfront proceeds from Lilly, were
$1.0 billion as of September 30, 2023, as compared to $1.1
billion as of December 31, 2022.
- Research & Development (R&D) Expenses:
R&D expenses were $100.0 million for the third quarter of 2023,
compared to $85.3 million for the third quarter of 2022.
- General & Administrative (G&A)
Expenses: G&A expenses were $25.4 million for the
third quarter of 2023, compared to $21.8 million for the third
quarter of 2022.
- Net Loss: Net loss was $96.1 million for the
third quarter of 2023, or $1.22 per share, compared to $109.6
million for the third quarter of 2022, or $1.56 per share.
Cash Runway
Beam expects that its cash, cash equivalents and marketable
securities as of September 30, 2023, together with upfront and
equity investment proceeds received from Lilly, will enable the
company to fund its anticipated operating expenses and capital
expenditure requirements into the second half of 2026. This
expectation assumes anticipated cost savings related to the
company’s 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.
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 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 preclinical development plans; the
therapeutic applications and potential of our technology, including
with respect to SCD, beta-thalassemia, GSDIa, T-ALL/T-LL, 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
September 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; any future payments we may
receive under our agreement with Lilly; the potential impact of the
portfolio prioritization and strategic restructuring on our
operations and development timelines; our ability to seek,
establish and maintain a collaboration or partnership to develop
our programs with a collaborator or partner; 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 the portfolio prioritization and strategic
restructuring, including our ability to seek, establish and
maintain partners for certain of our programs; 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, our Quarterly
Report on Form 10-Q that we will file 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.
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 30,2023 |
|
|
December 31,2022 |
|
Cash, cash equivalents, and marketable securities |
|
$ |
1,015,457 |
|
|
$ |
1,078,134 |
|
Total assets |
|
|
1,290,534 |
|
|
|
1,341,714 |
|
Total liabilities |
|
|
511,593 |
|
|
|
608,240 |
|
Total stockholders’
equity |
|
|
778,941 |
|
|
|
733,474 |
|
|
|
|
|
|
|
|
|
|
Condensed Consolidated Statement of Operations
(unaudited) |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
|
Nine Months Ended September 30, |
|
|
2023 |
|
|
2022 |
|
|
2023 |
|
|
2022 |
|
License and collaboration revenue |
$ |
17,193 |
|
|
$ |
15,799 |
|
|
$ |
61,517 |
|
|
$ |
40,883 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
0 |
|
Research and development |
|
100,050 |
|
|
|
85,287 |
|
|
|
297,304 |
|
|
|
225,253 |
|
General and administrative |
|
25,410 |
|
|
|
21,815 |
|
|
|
73,556 |
|
|
|
65,124 |
|
Total operating expenses |
|
125,460 |
|
|
|
107,102 |
|
|
|
370,860 |
|
|
|
290,377 |
|
Loss from operations |
|
(108,267 |
) |
|
|
(91,303 |
) |
|
|
(309,343 |
) |
|
|
(249,494 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
|
0 |
|
Change in fair value of derivative liabilities |
|
4,700 |
|
|
|
(4,900 |
) |
|
|
9,400 |
|
|
|
20,900 |
|
Change in fair value of non-controlling equity investments |
|
(11,221 |
) |
|
|
10,431 |
|
|
|
(17,870 |
) |
|
|
(1,378 |
) |
Change in fair value of contingent consideration liabilities |
|
6,002 |
|
|
|
(875 |
) |
|
|
7,877 |
|
|
|
(543 |
) |
Interest and other income (expense), net |
|
12,698 |
|
|
|
4,982 |
|
|
|
34,612 |
|
|
|
7,686 |
|
Total other income (expense) |
|
12,179 |
|
|
|
9,638 |
|
|
|
34,019 |
|
|
|
26,665 |
|
Net loss before income
taxes |
|
(96,088 |
) |
|
|
(81,665 |
) |
|
|
(275,324 |
) |
|
|
(222,829 |
) |
Provision for income taxes |
|
— |
|
|
|
(2,410 |
) |
|
|
— |
|
|
|
(2,410 |
) |
Loss from equity method investment |
|
— |
|
|
|
(25,500 |
) |
|
|
— |
|
|
|
(25,500 |
) |
Net loss |
$ |
(96,088 |
) |
|
$ |
(109,575 |
) |
|
$ |
(275,324 |
) |
|
$ |
(250,739 |
) |
Unrealized gain (loss) on
marketable securities |
|
(9 |
) |
|
|
(484 |
) |
|
|
406 |
|
|
|
(4,624 |
) |
Comprehensive loss |
$ |
(96,097 |
) |
|
$ |
(110,059 |
) |
|
$ |
(274,918 |
) |
|
$ |
(255,363 |
) |
Net loss per common share, basic and diluted |
$ |
(1.22 |
) |
|
$ |
(1.56 |
) |
|
$ |
(3.63 |
) |
|
$ |
(3.59 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
79,024,647 |
|
|
|
70,343,196 |
|
|
|
75,902,612 |
|
|
|
69,758,434 |
|
|
|
|
|
|
|
|
|
|
|
|
|
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