Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company
developing precision genetic medicines through base editing, today
announced pipeline and business highlights and financial results
for the first quarter ended March 31, 2022. In addition, as part of
a long-term effort to better understand sickle cell disease (SCD),
a genetic disease in which individuals carry two copies of the
sickle cell mutation, and sickle cell trait, in which individuals
carry only one copy of the mutation, Beam will fund and collaborate
with the National Alliance of Sickle Cell Centers (NASCC) to
initiate the AUNT (Achieving Understanding of the Natural History
of Sickle Trait) Study, a natural history study of sickle cell
trait.“Base editing has the potential to offer life-changing
medicines for a broad range of diseases, and we are committed to
better understanding the pathophysiology of the diseases in our
pipeline, and their impacts on the lives of patients and their
families,” said John Evans, chief executive officer of Beam. “We
are excited to be collaborating with the Globin Research Network
for Data and Discovery (GRNDad) on the AUNT natural history study
in people with sickle cell trait. We are on track and expect to
commence SCD patient enrollment in our Phase 1/2 BEACON-101
clinical trial to evaluate the safety and efficacy of BEAM-101 in
patients with SCD, as well as make our planned IND submission for
BEAM-102, which is also in development for the treatment of SCD.
Our immunology and liver-directed pipelines are also progressing,
with plans for additional regulatory submissions, research studies
and program nominations throughout the year, and we continue to
build upon our delivery capabilities, including our novel LNP
technology platform, potentially allowing us to expand the future
reach of our programs. We believe the parallel advancement of these
diverse programs creates a broad foundation for our future growth
and is evidence of our commitment to developing new and better
treatments for multiple patient populations and to unlocking the
full potential of precision genetic medicine.”
Pipeline Highlights & Anticipated
MilestonesEx Vivo HSC Programs
- Beam continues to advance its BEAM-101 program for the
treatment of SCD and expects to enroll the first patient in its
Phase 1/2 clinical trial evaluating the safety and efficacy of
BEAM-101 for the treatment of SCD, referred to as the BEACON-101
trial, in the second half of 2022.
- Beam’s second SCD-focused program, 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’s BEAM-201 program for the treatment of
relapsed/refractory T cell acute lymphoblastic leukemia/T cell
lymphoblastic lymphoma is progressing with submission of an IND
application anticipated in the second half of 2022.
- Beam continues to make progress towards its planned nomination
of a second CAR-T development candidate in 2022.
In Vivo LNP Liver-targeting Programs
- Beam plans to present updated preclinical data from its
BEAM-301 program at the American Society of Cell and Gene Therapy
(ASGCT) meeting, demonstrating high and durable editing efficiency
in a mouse model of glycogen storage disease 1a (GSDIa) out to 35
weeks. Beam plans to initiate IND-enabling studies in 2022 for
BEAM-301, a liver-targeting LNP formulation of base editing
reagents designed to correct the R83C mutation, the most common
disease-causing mutation of GSDIa.
- Also at ASGCT, Beam will present 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 continues to anticipate the nomination of a second
liver-targeted development candidate in 2022.
Initiation of AUNT Natural History StudySCD is
a severe, inherited blood disorder that alters the structure and
function of oxygen-carrying hemoglobin in red blood cells and is
caused by a single point mutation in the beta globin gene. Carriers
of the disease, or individuals with sickle cell trait, have only
one copy of the sickle mutation and produce variable amounts of the
abnormal sickle hemoglobin (25-45% of total hemoglobin). Despite
the high prevalence of sickle cell trait (estimated to affect 300
million individuals worldwide), research to understand its full
biology and clinical features has been limited.
Beam is developing two programs for SCD, BEAM-101 and BEAM-102.
BEAM-101 is designed to raise fetal hemoglobin while lowering
abnormal sickle hemoglobin to <40% of total, which is similar to
levels seen in individuals with sickle cell trait. BEAM-102 is
designed to replace the sickle mutation with a normal human variant
of hemoglobin, HbG-Makassar, potentially reducing even further the
abnormal sickle hemoglobin in patient cells. A better understanding
of sickle cell trait, along with an in-depth understanding of SCD,
will help better establish the relationship between levels of the
abnormal sickle hemoglobin and long-term clinical outcomes. The
AUNT Study will create a first of its kind multi-center,
prospective, longitudinal cohort of individuals with sickle trait,
targeting a large enrollment of approximately 1,000 participants.
This research is designed to establish an understanding of the
hematologic and clinical phenotype of people with sickle cell
trait, including blood rheology, potential complications, and
genetic modifiers, in an effort to better understand the
hematologic phenotype that is associated with good health and lack
of organ dysfunction, as well as provide increased counseling to
people with sickle cell trait.
Business Updates
- Manmohan Singh, Ph.D., senior vice president, pharmaceutical
sciences & delivery technologies, was appointed to the
company’s executive leadership team. Dr. Singh joined Beam in 2018,
bringing more than 24 years of drug discovery and development
experience from Takeda Pharmaceuticals, Novartis and Chiron
Corporation.
- Anne Marie Woodland joined Beam as senior vice president,
regulatory affairs, bringing more than 20 years of experience to
Beam, previously serving in regulatory focused roles at Replimune,
UniQure and BioVex.
Upcoming ASGCT Presentation
DetailsTitle: Single, systemic
administration of BEAM-301 mitigates fasting hypoglycemia and
restores metabolic function in a transgenic mouse model of glycogen
storage disease type IaDate & Time: Monday,
May 16, 2022, from 4:15-4:30 p.m. ET
Title: Optimized base editing reagents yield
more potent genetic correction in a mouse model of alpha-1
antitrypsin deficiency (poster M-123)Date &
Time: Monday May 16, 2022, from 5:30-6:30 p.m. ET
Title: Efficient LNP delivery of mRNA in vivo
and in vitro to T and NK cells (poster Tu-107)Date &
Time: Tuesday May 17, 2022, from 5:30-6:30 p.m. ET
First Quarter 2022 Financial Results
- Cash Position: Cash, cash equivalents and
marketable securities were $1.2 billion as of March 31, 2022, as
compared to $965.6 million as of December 31, 2021.
- Research & Development (R&D) Expenses:
R&D expenses were $65.4 million for the first quarter of 2022,
compared to $190.1 million for the first quarter of 2021. R&D
expenses for the first quarter of 2021 includes $155.0 million of
expense related to in-process research and development acquired
from Guide Therapeutics, Inc.
- General & Administrative (G&A)
Expenses: G&A expenses were $19.2 million for the
first quarter of 2022, compared to $10.3 million for the first
quarter of 2021.
- Net Loss: Net loss was $69.2 million for the
first quarter of 2022, or $1.01 per share, compared to $201.6
million for the first quarter of 2021, or $3.35 per share.
About Beam Therapeutics
Beam 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
Statements
This 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 intended design and planned initiation
of the AUNT Study; our presentations at ASGCT, our plans, and
anticipated timing, to nominate additional development candidates,
initiate IND-enabling studies, and submit IND applications; the
therapeutic applications and potential of our technology, including
with respect to SCD, T-ALL/T-LL, GSDIa, Alpha-1, CAR-T cells and
LNPs, including our ability to deliver base editors to target
organs in and beyond the liver; the planned initiation and design
of our BEACON-101 clinical trial, including the timing of enrolling
the first subject in the trial; 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; 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.
Contacts:
Investors:Chelcie ListerTHRUST Strategic
Communicationschelcie@thrustsc.com
Media:Dan Budwick1ABdan@1abmedia.com
Condensed
Consolidated Balance Sheet Data (unaudited) |
(in
thousands) |
|
|
|
|
|
|
|
March 31, 2022 |
|
December 31, 2021 |
Cash, cash
equivalents, and marketable securities |
|
$ |
1,222,600 |
|
|
$ |
965,647 |
|
Total
assets |
|
|
1,453,402 |
|
|
|
1,474,453 |
|
Total
liabilities |
|
|
624,334 |
|
|
|
647,715 |
|
Total
stockholders’ equity |
|
|
829,068 |
|
|
|
826,738 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Condensed
Consolidated Statement of Operations (unaudited) |
(in
thousands, except share and per share data) |
|
|
|
|
|
|
|
Three Months Ended March 31, |
|
|
|
2022 |
|
|
|
2021 |
|
License and
collaboration revenue |
|
$ |
8,432 |
|
|
$ |
6 |
|
Operating
expenses: |
|
|
|
|
Research and development |
|
|
65,410 |
|
|
|
190,106 |
|
General and administrative |
|
|
19,247 |
|
|
|
10,273 |
|
Total operating expenses |
|
|
84,657 |
|
|
|
200,379 |
|
Loss from
operations |
|
|
(76,225 |
) |
|
|
(200,373 |
) |
Other income
(expense): |
|
|
|
|
Change in fair value of derivative liabilities |
|
|
13,600 |
|
|
|
(1,900 |
) |
Change in fair value of non-controlling equity investments |
|
|
(7,685 |
) |
|
|
1,039 |
|
Change in fair value of contingent consideration liabilities |
|
|
452 |
|
|
|
(305 |
) |
Interest and other income (expense), net |
|
|
644 |
|
|
|
(21 |
) |
Total other income (expense) |
|
|
7,011 |
|
|
|
(1,187 |
) |
Net
loss |
|
$ |
(69,214 |
) |
|
$ |
(201,560 |
) |
Net loss per
common share, basic and diluted |
|
$ |
(1.01 |
) |
|
$ |
(3.35 |
) |
Weighted-average common shares outstanding, basic and diluted |
|
|
68,703,864 |
|
|
|
60,210,120 |
|
|
|
|
|
|
Beam Therapeutics (NASDAQ:BEAM)
과거 데이터 주식 차트
부터 6월(6) 2024 으로 7월(7) 2024
Beam Therapeutics (NASDAQ:BEAM)
과거 데이터 주식 차트
부터 7월(7) 2023 으로 7월(7) 2024