Recruitment for Phase 1/2 CEDAR clinical trial
of GPH101 for sickle cell disease underway at multiple sites
Details about CEDAR trial to be presented at
63rd ASH Annual Meeting and Exposition in December
$395.0 million in cash, cash equivalents and
restricted cash as of September 30, 2021
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage,
next-generation gene editing company focused on therapies that
harness targeted gene integration to treat or cure serious
diseases, today reported recent business progress and third quarter
2021 financial results.
“We continue to remain focused on execution as we work to carry
out our vision to develop potential one-time cures for patients
with a wide range of serious and life-threatening diseases,
starting with sickle cell disease,” said Josh Lehrer, M.Phil.,
M.D., chief executive officer of Graphite Bio. “We were pleased to
present preclinical data that are foundational to our sickle cell
disease program at the Sickle Cell Disease Association of America’s
National Convention last month, and we are excited to share details
about our Phase 1/2 CEDAR trial of GPH101 during a poster
presentation at the ASH Annual Meeting next month. Importantly,
recruitment for our CEDAR trial is underway at multiple clinical
trial sites, keeping us on track to enroll our first patient before
the end of the year.”
Recent Highlights and Upcoming Milestones
- Activated multiple clinical trial sites for Graphite Bio’s
Phase 1/2 CEDAR trial of GPH101, an investigational therapy
designed to directly correct the genetic mutation responsible for
sickle cell disease (SCD). With patient recruitment and activation
of additional clinical trial sites underway, the company remains on
track to enroll the first patient in the trial before the end of
the year and expects to report initial proof-of-concept data by the
end of 2022.
- Received acceptance of an abstract at the 63rd American Society
of Hematology (ASH) Annual Meeting and Exposition, which will take
place December 11-14 virtually and in Atlanta. Graphite Bio will
present details about the company's Phase 1/2 CEDAR trial of
GPH101.
- Presented preclinical data for GPH101 at the 49th Annual SCDAA
National Convention. The data presented support the ability of
Graphite Bio’s gene editing platform to precisely and efficiently
correct the underlying SCD-causing mutation to reduce sickle
hemoglobin production and restore adult hemoglobin expression to
levels that are considered potentially curative. These data are
foundational to the company’s SCD program and support the
evaluation of GPH101 in the Phase 1/2 CEDAR trial.
Third Quarter Financial Highlights
- Cash, Cash Equivalents and Restricted Cash: As of
September 30, 2021, cash, cash equivalents and restricted cash
totaled $395.0 million, which includes approximately $33.2 million
in net proceeds received on July 2 from the full exercise of the
underwriters’ option to purchase additional shares from the
company’s upsized IPO.
- R&D Expenses: Research and development expenses were
$8.7 million for the third quarter of 2021, which includes $0.8
million in stock-based compensation expense.
- G&A Expenses: General and administrative expenses
were $5.9 million for the third quarter of 2021, which includes
$1.6 million in stock-based compensation expense.
- Net Loss: Net loss was $14.6 million, or $0.28 per basic
and diluted share, for the three months ended September 30,
2021.
About Sickle Cell Disease (SCD)
SCD is a serious, life-threatening inherited blood disorder that
affects approximately 100,000 people in the United States and
millions of people around the world, making it the most prevalent
monogenic disease worldwide. SCD is caused by a single mutation in
the beta-globin gene that leads red blood cells to become
misshapen, resulting in anemia, blood flow blockages, intense pain,
increased risk of stroke and organ damage, and reduced life span by
approximately 20-30 years. Despite advancements in treatment and
care, progressive organ damage continues to cause early mortality
and severe morbidity, highlighting the need for curative
therapies.
About GPH101
GPH101 is an investigational next-generation gene-edited
autologous hematopoietic stem cell (HSC) therapy designed to
directly correct the genetic mutation that causes sickle cell
disease (SCD). GPH101 is the first investigational therapy to use a
highly differentiated gene correction approach that seeks to
efficiently and precisely correct the mutation in the beta-globin
gene to decrease sickle hemoglobin (HbS) production and restore
normal adult hemoglobin (HbA) expression, thereby potentially
curing SCD.
Graphite Bio is evaluating GPH101 in the CEDAR trial, an
open-label, multi-center Phase 1/2 clinical trial designed to
assess the safety, engraftment success, gene correction rates,
total hemoglobin, as well as other clinical and exploratory
endpoints and pharmacodynamics in patients with severe SCD.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing
company harnessing high efficiency targeted gene integration to
develop a new class of therapies to potentially cure a wide range
of serious and life-threatening diseases. Graphite Bio is
pioneering a precision gene editing approach that could enable a
variety of applications to transform human health through its
potential to achieve one of medicine’s most elusive goals: to
precisely “find & replace” any gene in the genome. Graphite
Bio’s platform allows it to precisely correct mutations, replace
entire disease-causing genes with normal genes or insert new genes
into predetermined, safe locations. The company was co-founded by
academic pioneers in the fields of gene editing and gene therapy,
including Maria Grazia Roncarolo, M.D. and Matthew Porteus, M.D.,
Ph.D.
Learn more about Graphite Bio by visiting www.graphitebio.com
and following the company on LinkedIn.
Forward-Looking Statements
Statements we make in this press release may include statements
which are not historical facts and are considered forward-looking
statements within the meaning of Section 27A of the Securities Act
of 1933, as amended (the “Securities Act”), and Section 21E of the
Securities Exchange Act of 1934, as amended (the “Exchange Act”).
These statements may be identified by words such as “aims,”
“anticipates,” “believes,” “could,” “estimates,” “expects,”
“forecasts,” “goal,” “intends,” “may,” “plans,” “possible,”
“potential,” “seeks,” “will,” and variations of these words or
similar expressions that are intended to identify forward-looking
statements. Any such statements in this press release that are not
statements of historical fact, including statements regarding the
clinical and therapeutic potential of our gene editing platform and
our product candidates, and the timing for enrollment of the first
patient in the Phase 1/2 CEDAR trial of GPH101 and the availability
of initial proof-of-concept data, may be deemed to be
forward-looking statements. We intend these forward-looking
statements to be covered by the safe harbor provisions for
forward-looking statements contained in Section 27A of the
Securities Act and Section 21E of the Exchange Act and are making
this statement for purposes of complying with those safe harbor
provisions.
Any forward-looking statements in this press release are based
on Graphite Bio’s current expectations, estimates and projections
only as of the date of this release and are subject to a number of
risks and uncertainties that could cause actual results to differ
materially and adversely from those set forth in or implied by such
forward-looking statements, including the risk that we may
encounter delays in patient enrollment and in the initiation,
conduct and completion of our planned clinical trials. These risks
concerning Graphite Bio’s programs and operations are described in
additional detail in its periodic filings with the U.S. Securities
and Exchange Commission, including but not limited to the Company’s
most recently filed periodic report. Graphite Bio is providing the
information in this press release as of this date and explicitly
disclaims any obligation to update any forward-looking statements
except to the extent required by law.
GRAPHITE BIO, INC.
Condensed Statements of
Operations
(Unaudited)
(In thousands, except share
and per share data)
Three Months Ended
Nine months ended
September 30,
September 30,
2021
2020
2021
2020
Operating expenses*:
Research and development
$
8,683
$
2,310
$
26,727
$
2,733
General and administrative
5,919
1,454
14,776
2,444
Total operating expenses
14,602
3,764
41,503
5,177
Loss from operations
(14,602
)
(3,764
)
(41,503
)
(5,177
)
Other income (expense), net:
Other income (expense), net:
10
—
14
—
Change in fair value of the Series A
redeemable
convertible preferred stock tranche
liability
—
380
(10,341
)
380
Related party convertible note interest
expense
—
—
—
(40
)
Total other income (expense), net
10
380
(10,327
)
340
Net loss and comprehensive loss
$
(14,592
)
$
(3,384
)
$
(51,830
)
$
(4,837
)
Net loss per share attributable to common
stockholders - basic and diluted
$
(0.28
)
$
(1.00
)
$
(2.51
)
$
(2.53
)
Weighted-average shares used in computing
net loss
per share attributable to common
stockholders - basic and diluted
52,769,916
3,382,118
20,668,560
1,908,759
* Includes stock-based compensation as
follows:
Research and development
$
824
$
6
$
1,635
$
6
General and administrative
1,550
30
3,790
30
Total stock-based compensation
expense
$
2,374
$
36
$
5,425
$
36
GRAPHITE BIO, INC.
Condensed Balance
Sheets
(In thousands)
September 30,
December 31,
2021
2020
(unaudited)
Assets
Current assets:
Cash and cash equivalents
$
394,804
$
19,782
Restricted cash
149
35
Prepaid expenses and other current
assets
6,276
1,286
Total current assets
401,229
21,103
Property, plant, and equipment, net
5,834
1,461
Operating lease right-of-use assets
9,077
—
Other assets
12
—
Total assets
$
416,152
$
22,564
Liabilities, redeemable convertible
preferred stock, and stockholders’ equity (deficit)
Current liabilities:
Accounts payable
$
1,763
$
630
Accrued compensation
1,659
466
Accrued research costs
1,730
1,764
Accrued expenses and other current
liabilities
1,314
126
Current portion of operating lease
liabilities
4,806
—
Series A redeemable convertible preferred
stock tranche liability
—
29,062
Total current liabilities
11,272
32,048
Non-current operating lease
liabilities
4,684
—
Other liabilities
64
316
Total liabilities
16,020
32,364
Series A redeemable convertible preferred
stock
—
55,608
Stockholders’ equity (deficit):
Common stock
1
—
Additional paid-in capital
522,552
5,183
Accumulated deficit
(122,421
)
(70,591
)
Total stockholders’ equity (deficit)
400,132
(65,408
)
Total liabilities, redeemable convertible
preferred stock, and stockholders’ equity (deficit)
$
416,152
$
22,564
View source
version on businesswire.com: https://www.businesswire.com/news/home/20211110006152/en/
Company: Stephanie Yao VP, Communications and Investor
Relations 443-739-1423 syao@graphitebio.com
Investors: Stephanie Ascher Stern IR, Inc. 212-362-1200
ir@graphitebio.com
Media: Christy Curran Sam Brown, Inc. 615-414-8668
media@graphitebio.com
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