Beam Therapeutics Reports Pipeline Updates and First Quarter 2023 Financial Results
May 10 2023 - 5:30AM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported first quarter 2023 financial results and provided an
update on its BEACON clinical trial and pipeline progress.
“As we work through the sentinel cohort process with our
investigators, we are encouraged by the overall momentum of the
BEACON trial, with multiple new sites activated, a growing number
of patients on the wait list for future enrollment, and continuing
progress preparing for internal GMP manufacturing in our North
Carolina facility,” said John Evans, chief executive officer of
Beam. “In parallel to BEACON, we are preparing to initiate our
second clinical trial this year, evaluating BEAM-201 in patients
with a devastating form of blood cancer, while advancing our in
vivo programs, BEAM-301 and BEAM-302, all of which remain on track.
As leaders in the field of base editing, we have spent the last
several years building a deep platform and pipeline and securing
meaningful funding to support this ambitious set of goals. We plan
to continue to follow the science, maintain a disciplined
investment strategy, and preserve optionality in our portfolio, so
that we can maximize our potential to create medicines that provide
life-long cures for patients in need.”
Hematology Program Updates
- Beam continues to advance its BEACON Phase 1/2 clinical trial
evaluating BEAM-101 as a treatment for sickle cell disease (SCD),
with five sites now active and a wait list for
enrollment.
- In the sentinel cohort, which is designed to include three
patients treated on a sequential basis, the first patient enrolled
in the BEACON trial in late 2022 has been withdrawn from the study
by the investigator due to personal, non-medical reasons prior to
treatment with BEAM-101.
- Two additional patients have been enrolled in the sentinel
cohort and are undergoing the screening procedures required to
enable mobilization and ultimately treatment with BEAM-101.
- Beam expects to fully enroll the sentinel cohort in 2023.
- Enrollment of additional patients for the expansion cohort,
which can occur in parallel with the sentinel cohort, is also
anticipated to begin this year.
- The company continues to anticipate reporting initial data on
multiple patients from the BEACON trial in 2024.
Immunology/Oncology Program Updates
- Beam is advancing 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 company expects to begin enrollment and dose the first
patient in its Phase 1/2 clinical trial of BEAM-201 by
mid-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 in 2023.
Genetic Disease (in
vivo) Program Updates
- Beam is advancing two in vivo base editing product candidates,
BEAM-301 and BEAM-302, leveraging lipid nanoparticles (LNPs) for
delivery to the liver. The company expects to:
- Submit a regulatory application for authorization to initiate
clinical trials for BEAM-301 for the treatment of glycogen storage
disease 1a (GSD1a) by late 2023 or early 2024; and
- Submit a regulatory application for authorization to initiate
clinical trials for BEAM-302 for the treatment of alpha-1
antitrypsin deficiency (AATD) in early 2024.
- In 2023, Beam plans to continue advancement of multiple
additional in vivo liver editing programs, 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 expects to initiate current good manufacturing practice
compliant operations at its North Carolina manufacturing facility
in late 2023.
Leadership Addition
- In March 2023, Beam appointed Gopi Shanker, Ph.D., as chief
scientific officer. Dr. Shanker is a scientific leader with more
than 20 years of drug development experience as well as deep
expertise in novel genetic medicine modalities.
Upcoming Presentations at ASGCT Beam plans to
present new in vivo data from its Engineered Stem Cell Antibody
Paired Evasion (ESCAPE) platform, which it continues to advance in
an effort to enable utility of its base editing investigational
medicines in as many patients as possible, and from its base
editing program for the treatment of Stargardt disease at the
American Society of Gene and Cell Therapy 26th Annual Meeting.
The meeting is being held May 16-20, 2023, in Los Angeles.
Details of the presentations are as follows:
Title: Paired HSC Epitope Engineering of CD117
(Ckit) for Antibody-Mediated Autologous Hematopoietic Stem Cell
Therapy Conditioning for the Potential Treatment of
HemoglobinopathiesDate & Time: Friday, May 19,
2023 12:00 pm – 2:00 pm PTSession Title: Friday
Poster Session
Title: (308) In Vivo Genetic Eye Disease
Correction Using Split AAV-Mediated Adenine Base
EditingDate & Time: Friday May 19, 2023, 4:30
pm - 4:45 pm PTSession Title: Gene Targeting and
Gene Correction: Hemoglobin, Muscle, and Eye
First Quarter 2023 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $1.1 billion as of March 31, 2023, as
compared to $1.1 billion as of December 31, 2022.
- Research & Development (R&D) Expenses:
R&D expenses were $99.6 million for the first quarter of 2023,
compared to $65.4 million for the first quarter of 2022.
- General & Administrative (G&A)
Expenses: G&A expenses were $23.5 million for the
first quarter of 2023, compared to $19.2 million for the first
quarter of 2022.
- Net Loss: Net loss was $96.5 million for the
first quarter of 2023, or $1.33 per share, compared to $69.2
million for the first quarter of 2022, or $1.01 per share.
Cash RunwayBeam expects that its cash, cash
equivalents and marketable securities as of March 31, 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
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, 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 March 31, 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; our upcoming presentations; 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,
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) |
|
|
|
|
|
|
|
|
|
|
March 31,2023 |
|
|
December 31,2022 |
|
Cash, cash equivalents, and marketable securities |
|
$ |
1,059,471 |
|
|
$ |
1,078,134 |
|
Total assets |
|
|
1,332,352 |
|
|
|
1,341,714 |
|
Total liabilities |
|
|
570,846 |
|
|
|
608,240 |
|
Total stockholders’ equity |
|
|
761,506 |
|
|
|
733,474 |
|
Condensed Consolidated Statement of Operations
(unaudited) |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2023 |
|
|
2022 |
|
License and collaboration revenue |
|
$ |
24,208 |
|
|
$ |
8,432 |
|
Operating expenses: |
|
|
|
|
|
|
Research and development |
|
|
99,646 |
|
|
|
65,410 |
|
General and administrative |
|
|
23,490 |
|
|
|
19,247 |
|
Total operating expenses |
|
|
123,136 |
|
|
|
84,657 |
|
Loss from operations |
|
|
(98,928 |
) |
|
|
(76,225 |
) |
Other income (expense): |
|
|
|
|
|
|
Change in fair value of derivative liabilities |
|
|
5,600 |
|
|
|
13,600 |
|
Change in fair value of non-controlling equity investments |
|
|
(12,797 |
) |
|
|
(7,685 |
) |
Change in fair value of contingent consideration liabilities |
|
|
(296 |
) |
|
|
452 |
|
Interest and other income (expense), net |
|
|
9,961 |
|
|
|
644 |
|
Total other income (expense) |
|
|
2,468 |
|
|
|
7,011 |
|
Net loss |
|
$ |
(96,460 |
) |
|
$ |
(69,214 |
) |
Unrealized gain (loss) on
marketable securities |
|
|
1,665 |
|
|
|
(2,659 |
) |
Comprehensive loss |
|
$ |
(94,795 |
) |
|
$ |
(71,873 |
) |
Net loss per common share, basic and diluted |
|
$ |
(1.33 |
) |
|
$ |
(1.01 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
72,273,829 |
|
|
|
68,703,864 |
|
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