Beam Therapeutics Announces Pipeline and Business Highlights and Reports Second Quarter 2022 Financial Results
09 August 2022 - 8:30PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
provided pipeline and business updates and reported financial
results for the second quarter ended June 30, 2022.
“2022 is a critical year for Beam’s transition to becoming a
multi-program clinical-stage company, as we prepare for the
near-term initiation of patient enrollment in our BEACON Phase 1/2
trial, the first clinical trial evaluating BEAM-101 in patients
with sickle cell disease,” said John Evans, chief executive officer
of Beam. “In June, we submitted our IND for BEAM-201 for
CD7-positive T-cell malignancies and recently received notification
from the FDA of a clinical hold on the IND. We look forward to
receiving more detail from the FDA and working with them in an
effort to advance BEAM-201 for these difficult-to-treat cancer
indications. We are on track to further expand our portfolio with a
steady cadence of clinical and preclinical milestones expected in
the quarters ahead, including the IND submission for BEAM-102, our
second program in sickle cell disease, and the initiation of
IND-enabling studies for BEAM-301, our first liver-directed base
editing program in glycogen storage disease, both targeted in the
second half of this year.”
Mr. Evans added, “As pioneers and leaders in the field of base
editing, we’ve continued to extend the potential reach of our base
editing technology and applications with the development of new
base editors, as well as novel base editing-enabled therapeutic
strategies, such as our work on non-genotoxic conditioning to
improve transplant regimens. We’ve also continued to enhance our
team, and I’m thrilled to welcome John Lo as chief commercial
officer. John has a deep science background and an extensive track
record in the strategic development and commercialization of novel
medicines, including cell therapy products, at leading companies. I
can’t wait to work with him to advance our portfolio and our vision
of providing a new class of precision genetic medicines to
patients.”
Pipeline Updates & Anticipated Milestones
Ex Vivo HSC Programs
- Beam remains on track to begin patient enrollment in its BEACON
trial, an open-label, single-arm, multicenter, Phase 1/2 clinical
trial evaluating the safety and efficacy of BEAM-101 in adult
patients with severe sickle cell disease (SCD) in the second half
of 2022.
- BEAM-102 continues to progress, and the company plans to submit
an investigational new drug (IND) application for BEAM-102 for the
treatment of SCD in the second half of 2022.
Ex Vivo T Cell Programs
- Beam submitted its IND for BEAM-201 to the U.S. Food and Drug
Administration (FDA) in June 2022, and on July 29, 2022, was
notified via e-mail that the IND was placed on clinical hold. The
FDA indicated it will provide an official clinical hold letter to
Beam within 30 days. Beam plans to provide additional updates
pending interaction with the FDA.
- Beyond BEAM-201, Beam is focused on identifying the collection
of multiplex base edits required to make cells fully allogeneic,
with internal and external data suggesting a higher number of edits
will be required to meet this goal. As a result, Beam does not
expect to nominate a second CAR-T development candidate in 2022 and
anticipates providing further updates in 2023.
In Vivo LNP Liver-targeting Programs
- Beam presented updated preclinical data from its BEAM-301
program at the American Society of Cell and Gene Therapy (ASGCT)
meeting, highlighting that BEAM-301 demonstrated high and durable
editing efficiency in a mouse model of glycogen storage disease 1a
(GSDIa) out to 35 weeks. BEAM-301, a liver-targeting lipid
nanoparticle (LNP) formulation of base editing reagents designed to
correct the R83C mutation, the most common disease-causing mutation
of GSDIa, is on track for initiation of IND-enabling studies in the
second half of 2022.
- At ASGCT, Beam also presented new preclinical data from its
base editing program targeting the treatment of alpha-1 antitrypsin
deficiency, highlighting optimizations made to the editor and the
guide RNA that have led to two-fold increases in observed editing
potency in mice, leading to potentially clinically relevant
increases in circulating alpha-1 antitrypsin at doses below 1
mg/kg.
- Beam plans to present new in vivo preclinical data from its
multiplex base editing program for the potential treatment of
hepatitis B virus (HBV) in a poster titled, “Cytosine base editing
inhibits Hepatitis B Virus replication and reduces HBsAg expression
in vitro and in vivo,” at the 2022 International HBV Meeting from
Sept. 18-22. The data will build on initial in vitro data presented
in September 2021, which showed that base editing can introduce
permanent mutations in covalently closed circular DNA (cccDNA) and
prevent HBV rebound in relevant models.
- Beam continues to anticipate the nomination of a second
liver-targeted development candidate in 2022.
Recent Research Highlights
- At the Federation of American Societies for Experimental
Biology (FASEB) Genome Engineering Conference in June, Beam
presented the first research highlighting the company’s internal
efforts to develop improved transplant conditioning regimens for
patients with SCD undergoing hematopoietic stem cell
transplantation (HSCT). With a goal of overcoming limitations of
today’s conditioning regimens, Beam leveraged its base editing
capabilities to develop a potentially non-genotoxic approach that
combines antibody-based conditioning with multiplex gene-edited
hematopoietic stem cells (HSCs) called ESCAPE, or Engineered Stem
Cell Antibody Paired Evasion. These improved conditioning regimens
could potentially be paired with BEAM-101 and BEAM-102, as well as
other future programs.
- At the CRISPR 2.0 conference in June, Beam highlighted research
that led to the creation of an improved class of cytosine base
editors (CBEs), leveraging a TadA enzyme-based CBE (CBE-T), that
are capable of editing at levels comparable to traditional CBEs but
with lower off-target editing potential. Further, Beam disclosed an
additional editor, CABE-T, that can conduct both C-to-T and A-to-G
edits with a single TadA deaminase.
Business Updates
- Beam recently appointed John Lo, Ph.D., as chief commercial
officer, where he will be responsible for commercial readiness, as
well as leading product and portfolio strategy. Dr. Lo joins Beam
after serving as an advisor to multiple private- and public-stage
biotechnology companies, including Beam. Dr. Lo has held a number
of global strategic and operating roles of increasing
responsibility within the biopharmaceutical industry, including as
senior vice president, worldwide hematology at Bristol Myers
Squibb; head of global marketing and market access at Astra Zeneca;
corporate vice president, hematology and oncology at Celgene; and
multiple P&L roles at Novartis. While at these companies, Dr.
Lo successfully helped launch numerous drugs in the U.S. and
globally, including Tagrisso in lung cancer and two cell therapies.
Dr. Lo has also helped build strategies and grow R&D pipelines
as co-chair of the Development Committee at Celgene, leading to
multiple pivotal study investments. Dr. Lo also spent several years
as an associate principal at McKinsey & Company and holds a
Ph.D. in molecular biology from MIT.
- In July, Beam and Verve Therapeutics amended their
collaboration and license agreement, originally executed in April
2019. The amended agreement returned two targets to Beam, while
adding a new, third target toward an additional liver-mediated,
cardiovascular disease target. Beam has the right to opt in on this
target after Phase 1 to share 35% of worldwide costs and profits
from the program. The two lead targets in the collaboration, PCSK9
and ANGPTL3, are unchanged. Verve also granted to Beam licenses and
options to certain delivery technologies.
Second Quarter 2022 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $1.2 billion as of June 30, 2022, as
compared to $965.6 million as of December 31, 2021.
- Research & Development (R&D) Expenses:
R&D expenses were $74.6 million for the second quarter of 2022,
compared to $45.6 million for the second quarter of 2021.
- General & Administrative (G&A)
Expenses: G&A expenses were $24.1 million for the
second quarter of 2022, compared to $13.4 million for the second
quarter of 2021.
- Net Loss: Net loss was $72.0 million for the
second quarter of 2022, or $1.02 per share, compared to $76.3
million for the second quarter of 2021, or $1.23 per share.
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: our upcoming presentation at the
2022 International HBV Meeting; our plans, and anticipated timing,
to nominate additional development candidates, initiate
IND-enabling studies, submit IND applications, and initiate
clinical trials; our expectation that we are on-track to further
expand our portfolio with a steady cadence of clinical and
preclinical milestones expected in the quarters ahead; our
expectations for transitioning to a multi-program clinical stage
company; the potential economic benefits that may be achieved under
our amended collaboration agreement with Verve Therapeutics; the
therapeutic applications and potential of our technology, including
with respect to SCD and our conditioning regimens, T-ALL/T-LL,
GSDIa, Alpha-1, HBV, and CAR-T cells; the expected timing of
enrolling the first subject in our BEACON Phase 1/2 clinical trial
of BEAM-101; the clinical hold on our BEAM-201 IND, including the
FDA’s communication plans related to, and our plans and
expectations for interactions with the FDA and the outcomes in
connection therewith; the sufficiency of our capital resources to
fund operating expenses and capital expenditure requirements; 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 the COVID-19 pandemic, including its impact
on the global supply chain; the uncertainty that our product
candidates, including BEAM-201, will receive regulatory approval
necessary to initiate human clinical studies; uncertainty in the
FDA’s plans to communicate and discuss the clinical hold on the
BEAM-201 IND with us and the risk that those discussions may be
delayed; the uncertainty in the outcome of our interactions with
the FDA regarding the clinical hold on the BEAM-201 IND; 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 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, 2021, 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 30,2022 |
|
|
December 31,2021 |
|
Cash, cash equivalents, and marketable securities |
|
$ |
1,166,115 |
|
|
$ |
965,647 |
|
Total
assets |
|
|
1,418,375 |
|
|
|
1,474,453 |
|
Total
liabilities |
|
|
618,214 |
|
|
|
647,715 |
|
Total
stockholders’ equity |
|
|
800,161 |
|
|
|
826,738 |
|
Condensed Consolidated Statement of Operations
(unaudited) |
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended June 30, |
|
|
Six Months Ended June 30, |
|
|
|
2022 |
|
|
2021 |
|
|
2022 |
|
|
2021 |
|
License
and collaboration revenue |
|
$ |
16,652 |
|
|
$ |
6 |
|
|
$ |
25,084 |
|
|
$ |
12 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
|
74,556 |
|
|
|
45,577 |
|
|
|
139,966 |
|
|
|
235,683 |
|
General and administrative |
|
|
24,062 |
|
|
|
13,403 |
|
|
|
43,309 |
|
|
|
23,676 |
|
Total operating expenses |
|
|
98,618 |
|
|
|
58,980 |
|
|
|
183,275 |
|
|
|
259,359 |
|
Loss
from operations |
|
|
(81,966 |
) |
|
|
(58,974 |
) |
|
|
(158,191 |
) |
|
|
(259,347 |
) |
Other
income (expense): |
|
|
|
|
|
|
|
|
|
|
|
|
Change in fair value of derivative liabilities |
|
|
12,200 |
|
|
|
(42,300 |
) |
|
|
25,800 |
|
|
|
(44,200 |
) |
Change in fair value of non-controlling equity investments |
|
|
(4,124 |
) |
|
|
25,814 |
|
|
|
(11,809 |
) |
|
|
26,852 |
|
Change in fair value of contingent consideration liabilities |
|
|
(120 |
) |
|
|
(741 |
) |
|
|
332 |
|
|
|
(1,046 |
) |
Interest and other income (expense), net |
|
|
2,060 |
|
|
|
(52 |
) |
|
|
2,704 |
|
|
|
(72 |
) |
Total other income (expense) |
|
|
10,016 |
|
|
|
(17,279 |
) |
|
|
17,027 |
|
|
|
(18,466 |
) |
Net
loss |
|
$ |
(71,950 |
) |
|
$ |
(76,253 |
) |
|
$ |
(141,164 |
) |
|
$ |
(277,813 |
) |
Net loss
per common share, basic and diluted |
|
$ |
(1.02 |
) |
|
$ |
(1.23 |
) |
|
$ |
(2.03 |
) |
|
$ |
(4.54 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
70,210,227 |
|
|
|
62,210,239 |
|
|
|
69,461,207 |
|
|
|
61,215,705 |
|
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