Beam Therapeutics Reports Pipeline Updates and First Quarter 2024 Financial Results
07 Mayo 2024 - 5:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported first quarter 2024 financial results and provided updates
across the company’s hematology and genetic disease franchises.
“We’re pleased to share progress across our high-priority
programs that exemplify our commitment to rapid, focused execution
and a dedication to developing differentiated, one-time medicines
for serious genetic diseases,” said John Evans, chief executive
officer of Beam. “We have successfully completed dosing and
engraftment for three sickle cell disease patients in the sentinel
cohort of the BEAM-101 BEACON trial allowing us to now move forward
with expansion phase dosing. We look forward to sharing data for
multiple patients treated with BEAM-101 later this year. In
addition, our team has done an incredible job executing our ex-U.S.
clinical strategy for BEAM-302, securing CTA clearance in the UK
and rapidly working toward the initiation of our Phase 1/2 trial in
patients with alpha-1 antitrypsin deficiency. This study is
designed to demonstrate proof-of-concept for correction of the
disease-causing mutation that could potentially help patients with
both lung and liver disease manifestations. These updates,
supported by our robust balance sheet, mark a significant stride
toward our goal of establishing base editing as a potentially
transformative and differentiated therapeutic option for patients
in need.”
First Quarter 2024 and Recent Progress
- Sequential dosing and engraftment have been successfully
completed for the three patients in the sentinel cohort of the
BEACON Phase 1/2 clinical trial of BEAM-101 in severe sickle cell
disease.
- Following clearance by the data monitoring committee, the
expansion cohort of the BEACON trial of BEAM-101, in which patients
can be dosed in parallel, is now open with dosing expected to begin
imminently.
- In March 2024, Beam announced the clearance of its clinical
trial authorisation (CTA) application by the United Kingdom (UK)
Medicines and Healthcare Products Regulatory Agency for BEAM-302,
the company’s priority genetic disease program for the treatment of
alpha-1 antitrypsin deficiency (AATD).
Key 2024 Anticipated Milestones
Hematology Franchise
- In the BEACON Phase 1/2 clinical trial of BEAM-101 in adults
with severe sickle cell disease, Beam anticipates continuing to
enroll and dose patients in the expansion cohort of the trial, with
a total target of up to 45 treated patients.
- The company expects to report data from multiple patients in
the BEACON trial in the second half of 2024.
- Beam continues to advance and invest in its Engineered Stem
Cell Antibody Paired Evasion (ESCAPE) conditioning platform and
anticipates initiating Phase 1-enabling preclinical studies for the
program in 2024.
Genetic Disease Franchise
- Beam expects to initiate the Phase 1/2 clinical trial for
BEAM-302 in AATD in the first half of 2024.
- Beam expects to submit an investigational new drug (IND)
application in the U.S. for BEAM-301 for the potential treatment of
glycogen storage disease type 1a (GSD1a) in the first half of
2024.
First Quarter 2024 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $1.1 billion as of March 31, 2024,
compared to $1.2 billion as of December 31, 2023.
- Research & Development (R&D) Expenses:
R&D expenses were $84.8 million for the first quarter of 2024,
compared to $99.6 million for the first quarter of 2023.
- General & Administrative (G&A)
Expenses: G&A expenses were $26.7 million for the
first quarter of 2024, compared to $23.5 million for the first
quarter of 2023.
- Net Loss: Net loss was $98.7 million for the
first quarter of 2024, or $1.21 per share, compared to $96.5
million for the first quarter of 2023, or $1.33 per share.
Cash RunwayBeam expects that its cash, cash
equivalents and marketable securities as of March 31, 2024, will
enable the company to fund its anticipated operating expenses and
capital expenditure requirements into 2027. This expectation
includes funding directed toward reaching each of the key
anticipated milestones for BEAM-101, ESCAPE, 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 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 sickle cell
disease, AATD, GSD1a, and ESCAPE; our plans, and anticipated
timing, to advance our programs; the clinical trial designs and
expectations for BEAM-101, BEAM-301, BEAM-302 and ESCAPE; our
estimated cash, cash equivalents and marketable securities as of
March 31, 2024 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 successfully achieve the benefits of our
portfolio prioritization and strategic restructuring; 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 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, 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
Condensed Consolidated Balance Sheet Data
(unaudited) |
|
(in thousands) |
|
|
|
|
|
|
|
|
|
|
March 31,2024 |
|
|
December 31,2023 |
|
Cash, cash equivalents, and marketable securities |
|
$ |
1,094,554 |
|
|
$ |
1,189,876 |
|
Total
assets |
|
|
1,359,796 |
|
|
|
1,459,714 |
|
Total
liabilities |
|
|
446,306 |
|
|
|
478,385 |
|
Total
stockholders’ equity |
|
|
913,490 |
|
|
|
981,329 |
|
Condensed Consolidated Statement of Operations
(unaudited) |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2024 |
|
|
2023 |
|
License and collaboration revenue |
|
$ |
7,410 |
|
|
$ |
24,208 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
|
84,818 |
|
|
|
99,646 |
|
General and administrative |
|
|
26,724 |
|
|
|
23,490 |
|
Total operating expenses |
|
|
111,542 |
|
|
|
123,136 |
|
Loss
from operations |
|
|
(104,132 |
) |
|
|
(98,928 |
) |
Other
income (expense): |
|
|
|
|
|
|
Change in fair value of derivative liabilities |
|
|
(2,900 |
) |
|
|
5,600 |
|
Change in fair value of non-controlling equity investments |
|
|
(3,353 |
) |
|
|
(12,797 |
) |
Change in fair value of contingent consideration liabilities |
|
|
(133 |
) |
|
|
(296 |
) |
Interest and other income (expense), net |
|
|
11,849 |
|
|
|
9,961 |
|
Total other income (expense) |
|
|
5,463 |
|
|
|
2,468 |
|
Net
loss |
|
$ |
(98,669 |
) |
|
$ |
(96,460 |
) |
Unrealized gain (loss) on marketable securities |
|
|
(1,525 |
) |
|
|
1,665 |
|
Comprehensive loss |
|
$ |
(100,194 |
) |
|
$ |
(94,795 |
) |
Net loss per common share, basic and diluted |
|
$ |
(1.21 |
) |
|
$ |
(1.33 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
81,698,633 |
|
|
|
72,273,829 |
|
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