Beam Therapeutics Provides Business and Pipeline Updates and Reports Third Quarter 2021 Financial Results
08 11월 2021 - 8:00PM
Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
reported recent business and pipeline updates, as well as third
quarter 2021 financial results.
As part of today’s update, Beam announced that its
Investigational New Drug (IND) application for BEAM-101 for the
treatment of sickle cell disease was cleared by the U.S. Food and
Drug Administration (FDA). BEAM-101, the company’s lead ex vivo
base editing product candidate, is a patient-specific, autologous
hematopoietic investigational cell therapy which incorporates base
edits that mimic single nucleotide polymorphisms seen in
individuals with hereditary persistence of fetal hemoglobin (HPFH)
to potentially alleviate the effects of mutations causing sickle
cell disease or beta-thalassemia. This is the first open IND for
base editing technology, a next-generation form of CRISPR capable
of making single base changes without creating double strand breaks
in the DNA. Beam is preparing to initiate a Phase 1/2 clinical
trial designed to assess the safety and efficacy of BEAM-101 for
the treatment of sickle cell disease, which Beam refers to as the
BEACON-101 trial.
“We are thrilled to share that the FDA has cleared our first
IND. BEAM-101 has the potential to offer a one-time treatment for
patients with sickle cell disease, and this clearance enables the
important transition from a preclinical to a clinical-stage
company, bringing us closer to our ultimate goal of helping
patients,” said John Evans, chief executive officer of Beam. “As
leaders in the field of base editing, this milestone underscores
the expertise of our team and the significant potential of our
technology. We are grateful to the team members who have dedicated
countless hours to this effort, and we look forward to this
program’s continued evaluation in the clinic. As we look toward
2022, we believe we are well-positioned both financially and
organizationally to execute on our vision.”
“In addition to the clearance of our IND for BEAM-101, we are
pleased to announce that we have initiated IND-enabling studies for
BEAM-102, our Makassar base editing approach for the treatment of
sickle cell disease,” said Giuseppe Ciaramella, Ph.D., president
and chief scientific officer of Beam. “We have also made continued
progress on BEAM-201, our multiplexed base editing approach for the
treatment of relapsed/refractory T-cell acute lymphoblastic
leukemia and T-cell lymphoblastic leukemia as well as continued
advancement across our robust portfolio, as showcased by a number
of recent and upcoming data presentations demonstrating our
leadership in base editing as well as optimized delivery
technologies for our programs. Importantly, we also expect to
nominate our first development candidate for in vivo base editing
in the liver using LNP delivery for the treatment of patients with
glycogen storage disease type Ia (R83C mutation) by the end of the
year. We’ve made extraordinary progress as a company this year and
remain committed to advancing novel science so that we may reach as
many patients as possible.”
Upcoming Base Editing Data Presentations
- Preclinical data highlighting
potential of CAR T multiplex base editing approach targeting CD5 at
2021 SITC Annual Meeting. At the Society for Immunotherapy
of Cancer’s (SITC) 36th Annual Meeting, Beam will present
preclinical data from its multiplex edited allogeneic CAR T
research targeting CD5-positive hematologic malignancies in a
poster titled “CD5 knockout enhances the potency of multiplex
base-edited allogeneic anti-CD5 CAR T-cell therapy for the
treatment of T-cell malignancies.”
- Multiple abstracts accepted for
presentation at the 63rd American
Society of Hematology Annual Meeting & Exposition
(ASH). At this year’s ASH meeting, Beam will present
preclinical data highlighting its base editing approach to address
sickle cell disease, including new preclinical data for
BEAM-102, and data highlighting Beam’s in vivo
high-throughput lipid nanoparticle (LNP) screening approach to
identify novel LNPs that can deliver base editors to tissues beyond
the liver, such as hematopoietic stem cells. In addition, Beam will
present an overview on the application of base editing for the
treatment of beta-hemoglobinopathies and other genetic blood
disorders during a scientific program session. Details of the
presentations can be found here.
Recent Base Editing Progress & Data
Updates
- BEAM-102 IND-enabling studies
initiated. Beam has initiated IND-enabling studies for
BEAM-102 for the treatment of sickle cell disease. BEAM-102 aims to
directly correct the causative mutation in sickle cell disease by
recreating a naturally-occurring normal human hemoglobin variant,
HbG Makassar.
- Preclinical data highlighting
base editing approach to address GSDIa presented at European
Society of Gene & Cell Therapy. Beam recently
presented preclinical data demonstrating the ability of its
liver-targeted base editing approach to directly correct R83C, one
of the primary disease-causing mutations of glycogen storage
disease type Ia (GSDIa), and the elimination of the disease
phenotype in a novel in vivo model.
- Optimized LNP delivery
approaches for in vivo base
editing to the liver and other tissues presented at TIDES
2021. Beam announced new preclinical data highlighting
advancements with its approach to developing novel LNP formulations
for increased in vivo liver editing potency, with high levels of
editing at what Beam believes could be a clinically-relevant dose
level of 1.0 mg/kg. Beam remains on track to nominate its first
Development Candidate for in vivo base editing using LNP delivery
by the end of 2021. Also at TIDES, Beam reported an update on its
proprietary approach to developing LNPs to deliver base editors to
tissues beyond the liver, including hematopoietic stem and
progenitor cells.
- Preclinical data highlighting
potential of base editors to target disease drivers of chronic
hepatitis B infection presented at 2021 International HBV
Meeting. Beam recently presented data demonstrating the
potential of its cytosine base editors to reduce viral markers,
including hepatitis B surface antigen (HBsAg) expression, and
prevent viral rebound of hepatitis B virus (HBV) in in vitro
models.
Business Highlights
- Executed non-exclusive option and license agreement
with Sana Biotechnology for Cas12b. Under the agreement,
Beam granted Sana non-exclusive commercial rights to utilize its
CRISPR Cas12b system with certain allogeneic T-cell and stem
cell-derived programs. The agreement excludes any rights to base
editing using Beam’s Cas12b system, which rights remain exclusively
with Beam. Sana agreed to pay Beam an upfront payment of $50
million, and Beam may also receive certain target option exercise
fees, certain milestone payments upon the achievement of certain
development and commercial sale milestones, and certain royalties
on net sales of royalty-bearing products by Sana.
Third Quarter 2021 Financial Results
- Cash
Position: Cash, cash equivalents and marketable
securities were $933.4 million as of September 30, 2021,
compared to $299.7 million as of December 31, 2020.
- Research & Development
(R&D) Expenses: R&D expenses were $54.6
million for the third quarter of 2021, compared to $29.8
million for the third quarter of 2020.
- General & Administrative
(G&A) Expenses: G&A expenses were $15.8
million for the third quarter of 2021, compared to $7.5
million for the third quarter of 2020.
- Net Loss: Net loss
attributable to common stockholders was $28.1 million, or $0.42 per
share, for the third quarter of 2021, compared to $34.5 million, or
$0.69 per share, for the third quarter of 2020.
About Sickle Cell Disease and
Beta-Thalassemia
Sickle cell disease, a severe inherited blood disease, is caused
by a single point mutation, E6V, in the beta globin gene. This
mutation causes the mutated form of sickle hemoglobin (HbS) to
aggregate into long, rigid molecules that bend red blood cells into
a sickle shape under conditions of low oxygen. Sickled cells
obstruct blood vessels and die prematurely, ultimately resulting in
anemia, severe pain (crises), infections, stroke, organ failure,
and early death. Sickle cell disease is the most common inherited
blood disorder in the United States, affecting an estimated 100,000
individuals, of which a significant proportion are of
African-American descent. Beta-thalassemia is another inherited
blood disorder characterized by severe anemia caused by reduced
production of functional hemoglobin due to insufficient expression
of the beta globin protein. Transfusion-dependent beta-thalassemia
(TDBT) is the most severe form of this disease, often requiring
multiple transfusions per year. Patients with TDBT suffer from
failure to thrive, persistent infections, and life-threatening
anemia.
About BEAM-101BEAM-101 is a patient-specific,
autologous hematopoietic cell therapy under investigation for the
treatment of sickle cell disease. BEAM-101 incorporates ex vivo
base edits that mimic single nucleotide polymorphisms seen in
individuals with hereditary persistence of fetal hemoglobin (HPFH)
to potentially alleviate the effects of mutations causing sickle
cell disease or beta-thalassemia by increasing the levels of fetal
hemoglobin (HbF).
About Beam TherapeuticsBeam Therapeutics Inc.
(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 enables precise, predictable
and efficient single base changes, at targeted genomic sequences,
without making double-stranded breaks in the DNA. This enables 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 potentially
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 commencement of clinical
trials for BEAM-101, including our BEACON-101 trial; our
expectation that we will nominate our first development candidate
for in vivo base editing in the liver using LNP delivery for the
treatment of patients with GSDIa disease (R83C mutation) by the end
of the year; any future payments we may receive under our agreement
with Sana; our planned base editing data presentations at upcoming
scientific conferences; and the therapeutic applications and
potential of our technology, including 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;
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, 2020, our Quarterly Report on Form 10-Q for the
quarter ended March 31, 2021, our Quarterly Report on Form 10-Q for
the quarter ended June 30, 2021, our Quarterly Report on Form 10-Q
that we will file for the quarter ended September 30, 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.
Contacts: Investors:Chelcie ListerTHRUST
Strategic Communications+chelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
Condensed Consolidated Balance Sheet Data
(unaudited) |
|
|
|
|
(in thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
September 30, 2021 |
|
December 31, 2020 |
|
|
|
|
Cash, cash equivalents, and marketable securities |
|
$ |
933,405 |
|
|
$ |
299,671 |
|
|
|
|
|
Total assets |
|
|
1,156,555 |
|
|
|
451,677 |
|
|
|
|
|
Total liabilities |
|
|
302,741 |
|
|
|
206,116 |
|
|
|
|
|
Total stockholders’ equity |
|
|
853,814 |
|
|
|
245,561 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Condensed Consolidated Statement of Operations
(unaudited) |
|
|
|
|
(in thousands, except share and per share
data) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Six Months Ended September 30, |
|
|
|
2021 |
|
|
|
2020 |
|
|
|
2021 |
|
|
|
2020 |
|
License revenue |
|
$ |
763 |
|
|
$ |
6 |
|
|
$ |
775 |
|
|
$ |
18 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
Research and development |
|
|
54,623 |
|
|
|
29,825 |
|
|
|
290,306 |
|
|
|
70,728 |
|
General and administrative |
|
|
15,774 |
|
|
|
7,502 |
|
|
|
39,450 |
|
|
|
21,251 |
|
Total operating expenses |
|
|
70,397 |
|
|
|
37,327 |
|
|
|
329,756 |
|
|
|
91,979 |
|
Loss from operations |
|
|
(69,634 |
) |
|
|
(37,321 |
) |
|
|
(328,981 |
) |
|
|
(91,961 |
) |
Other income (expense): |
|
|
|
|
|
|
|
|
Change in fair value of derivative liabilities |
|
|
35,800 |
|
|
|
2,700 |
|
|
|
(8,400 |
) |
|
|
(8,700 |
) |
Change in fair value of long-term investments |
|
|
(4,892 |
) |
|
|
- |
|
|
|
21,960 |
|
|
|
517 |
|
Change in fair value of contingent consideration liabilities |
|
|
10,599 |
|
|
|
- |
|
|
|
9,553 |
|
|
|
- |
|
Interest and other income (expense), net |
|
|
9 |
|
|
|
169 |
|
|
|
(63 |
) |
|
|
1,016 |
|
Total other income (expense) |
|
|
41,516 |
|
|
|
2,869 |
|
|
|
23,050 |
|
|
|
(7,167 |
) |
Net loss |
|
$ |
(28,118 |
) |
|
$ |
(34,452 |
) |
|
$ |
(305,931 |
) |
|
$ |
(99,128 |
) |
Accretion of redeemable convertible preferred stock to redemption
value, including dividends on preferred stock |
|
|
- |
|
|
|
- |
|
|
|
- |
|
|
|
(1,277 |
) |
Net loss attributable to common stockholders |
|
$ |
(28,118 |
) |
|
$ |
(34,452 |
) |
|
$ |
(305,931 |
) |
|
$ |
(100,405 |
) |
Net loss per common share attributable to common stockholders,
basic and diluted |
|
$ |
(0.42 |
) |
|
$ |
(0.69 |
) |
|
$ |
(4.86 |
) |
|
$ |
(2.31 |
) |
Weighted-average common shares used in net loss per share
attributable to common stockholders, basic and diluted |
|
|
66,377,611 |
|
|
|
50,087,747 |
|
|
|
62,960,219 |
|
|
|
43,438,919 |
|
|
|
|
|
|
|
|
|
|
Beam Therapeutics (NASDAQ:BEAM)
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부터 9월(9) 2024 으로 10월(10) 2024
Beam Therapeutics (NASDAQ:BEAM)
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부터 10월(10) 2023 으로 10월(10) 2024