Beam Therapeutics Reports Fourth Quarter and Year-End 2022 Financial Results and Reiterates Anticipated Milestones
February 28 2023 - 6:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reaffirmed its anticipated upcoming milestones across the company’s
hematology, immunology-oncology, and genetic disease portfolios and
reported fourth quarter and full year 2022 financial results.
“Just five years since our founding, we achieved significant
milestones in 2022, having advanced two ex vivo assets – BEAM-101
and BEAM-201 – into clinical-stage development, and two in vivo
liver programs – BEAM-301 and BEAM-302 – on a path to regulatory
filings,” said John Evans, chief executive officer of Beam. “As we
look ahead in 2023, we are focused on the execution of our clinical
trials and advancing preclinical work that we expect will support
regulatory filings for BEAM-301 and BEAM-302. Importantly, as our
confidence in the power and differentiation of our base editing
platform grows, we also continue to make important investments in
new programs across each of our strategic pillars. This includes
our ESCAPE platform in hematology, our next-generation of highly
engineered cell therapies in immunology-oncology, and numerous new
base editing programs targeting the liver on our own and with our
collaborators. I am incredibly excited about the year ahead as we
execute our clinical trials and focus on advancing an entirely new
class of potentially one-time transformative medicines that could
benefit so many patients in need.”
Key 2023-2024 Anticipated Milestones and
InvestmentsHematology Portfolio
- Continue enrollment in sentinel cohort of BEACON Phase 1/2
clinical trial evaluating BEAM-101 as a treatment for sickle cell
disease (SCD)
- Finish enrollment in the sentinel cohort in 2023
- Initiate enrollment in the expansion cohort in 2023
- Report initial data from multiple patients from one or both
cohorts in 2024
- Continue to advance and invest in Engineered Stem Cell Antibody
Paired Evasion (ESCAPE) conditioning platform throughout 2023
Immunology/Oncology Portfolio
- Enroll and dose first patient in Phase 1/2 clinical trial of
BEAM-201 in patients with relapsed/refractory T-cell acute
lymphoblastic leukemia (T-ALL)/T-cell lymphoblastic lymphoma (T-LL)
by mid-2023
- Continue to invest in and advance potential next-generation
allogeneic strategies designed to significantly improve cell
persistence to expand the utility and accessibility of cell
therapies in cancer and other diseases, with updates on these
efforts to be provided in 2023
Genetic Disease Portfolio
- Advance preclinical studies for BEAM-301 in glycogen storage
disease 1a (GSDIa) and, by late 2023 or early 2024, submit a
regulatory application for authorization to initiate clinical
trials for the program
- Advance preclinical studies for BEAM-302 for severe alpha-1
antitrypsin deficiency (AATD) and, in early 2024, submit a
regulatory application for authorization to initiate clinical
trials for the program
- Advance multiple additional in vivo liver editing programs
through lead optimization in 2023, including both Beam wholly owned
and collaboration programs
- Continue advancement of lipid nanoparticle delivery
technologies targeting the liver and other tissues throughout
2023
Manufacturing
- Initiate current good manufacturing practice compliant
operations at Beam’s internal North Carolina manufacturing facility
in late 2023
Recent Nature
Biotechnology Publication In
January 2023, Beam published preclinical research in Nature
Biotechnology highlighting work that led to the creation of an
improved class of cytosine base editors (CBEs), leveraging a TadA
enzyme-based CBE (CBE-T), that demonstrated edits at levels
comparable to traditional CBEs and that benefited from favorable
attributes of TadA for precise, specific, and flexible base
editing. In addition, the paper highlights research to identify
cytosine and adenine base editors (CABEs) that can conduct both
C-to-T and A-to-G edits with a single TadA deaminase—called
CABE-Ts. Together with ABEs, CBE-Ts and CABE-Ts demonstrated
programmable installation of all transition mutations using
laboratory-evolved TadA variants with improved properties relative
to previously reported CBEs.
Fourth Quarter and Full Year 2022 Financial
Results
- Cash Position: Cash, cash equivalents and
marketable securities were $1.1 billion as of December 31, 2022,
compared to $ 965.6 million as of December 31, 2021.
- Research & Development (R&D) Expenses:
R&D expenses were $86.3 million for the fourth quarter of 2022
and $311.6 million for the full year ended December 31, 2022,
compared to $96.8 million for the fourth quarter of 2021 and $387.1
million for the full year ended December 31, 2021.
- General & Administrative (G&A)
Expenses: G&A expenses were $22.7 million for the
fourth quarter of 2022 and $87.8 million for the full year ended
December 31, 2022, compared to $17.8 million for the fourth quarter
of 2021 and $57.2 million for the full year ended December 31,
2021.
- Net Loss: Net loss attributable to common
stockholders was $38.3 million, or $0.54 per share, for the fourth
quarter of 2022 and $289.1 million, or $4.13 per share, for the
year ended December 31, 2022, compared to $64.7 million, or $0.95
per share, for the fourth quarter of 2021 and $370.6 million, or
$5.77 per share, for the full year ended December 31, 2021.
Cash RunwayBeam expects that its cash, cash
equivalents and marketable securities as of December 31, 2022, 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 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
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 SCD, GSDIa,
T-ALL/TLL, AATD and our conditioning regimens; our plans, and
anticipated timing, to advance our programs, including our Key
2023-2024 Anticipated Milestones and Investments; our estimated
cash, cash equivalents and marketable securities as of December 31,
2022 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 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 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, 2022, 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) |
|
|
|
|
|
|
|
|
|
|
December 31,2022 |
|
|
December 31,2021 |
|
Cash, cash equivalents, and
marketable securities |
|
$ |
1,078,134 |
|
|
$ |
965,647 |
|
Total assets |
|
|
1,341,714 |
|
|
|
1,474,453 |
|
Total liabilities |
|
|
608,240 |
|
|
|
647,715 |
|
Total stockholders’ equity |
|
|
733,474 |
|
|
|
826,738 |
|
Condensed Consolidated Statement of Operations
(unaudited) |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended December 31, |
|
|
Year Ended December 31, |
|
|
|
2022 |
|
|
2021 |
|
|
2022 |
|
|
2021 |
|
License and collaboration
revenue |
|
$ |
20,037 |
|
|
$ |
51,069 |
|
|
$ |
60,920 |
|
|
$ |
51,844 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
86,341 |
|
|
|
96,781 |
|
|
|
311,594 |
|
|
|
387,087 |
|
General and administrative |
|
|
22,681 |
|
|
|
17,772 |
|
|
|
87,805 |
|
|
|
57,222 |
|
Total operating expenses |
|
|
109,022 |
|
|
|
114,553 |
|
|
|
399,399 |
|
|
|
444,309 |
|
Loss from operations |
|
|
(88,985 |
) |
|
|
(63,484 |
) |
|
|
(338,479 |
) |
|
|
(392,465 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
Change in fair value of derivative liabilities |
|
|
3,000 |
|
|
|
7,400 |
|
|
|
23,900 |
|
|
|
(1,000 |
) |
Change in fair value of non-controlling equity investments |
|
|
21,578 |
|
|
|
(4,270 |
) |
|
|
20,200 |
|
|
|
17,690 |
|
Change in fair value of contingent consideration liabilities |
|
|
19,447 |
|
|
|
(4,407 |
) |
|
|
18,904 |
|
|
|
5,146 |
|
Interest and other income (expense), net |
|
|
7,611 |
|
|
|
54 |
|
|
|
15,297 |
|
|
|
(9 |
) |
Total other income (expense) |
|
|
51,636 |
|
|
|
(1,223 |
) |
|
|
78,301 |
|
|
|
21,827 |
|
Net loss before income taxes |
|
|
(37,349 |
) |
|
|
(64,707 |
) |
|
|
(260,178 |
) |
|
|
(370,638 |
) |
Provision for income taxes |
|
|
(1,000 |
) |
|
|
— |
|
|
|
(3,410 |
) |
|
|
— |
|
Loss from equity method investment |
|
|
— |
|
|
|
— |
|
|
|
(25,500 |
) |
|
|
— |
|
Net loss |
|
$ |
(38,349 |
) |
|
$ |
(64,707 |
) |
|
$ |
(289,088 |
) |
|
$ |
(370,638 |
) |
Unrealized gain (loss) on
marketable securities |
|
|
2,244 |
|
|
|
(12 |
) |
|
|
(2,380 |
) |
|
|
(41 |
) |
Comprehensive loss |
|
$ |
(36,105 |
) |
|
$ |
(64,719 |
) |
|
$ |
(291,468 |
) |
|
$ |
(370,679 |
) |
Net loss per common share
attributable to common stockholders, basic and diluted |
|
$ |
(0.54 |
) |
|
$ |
(0.95 |
) |
|
$ |
(4.13 |
) |
|
$ |
(5.77 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
70,777,542 |
|
|
|
67,988,717 |
|
|
|
70,015,305 |
|
|
|
64,227,676 |
|
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