Patient enrollment in Phase 1/2 CEDAR clinical
trial of GPH101 for sickle cell disease ongoing at multiple sites;
dosing of first patient now planned for second half of 2022, with
initial proof-of-concept data anticipated in 2023
Prioritized R&D to maximize capabilities of
company’s next-generation gene editing platform and significantly
impact patient outcomes
Bolstered company leadership with hiring of
chief financial officer and chief of staff, and promotion of chief
people officer
$378.7 million in cash, cash equivalents and
restricted cash as of December 31, 2021; cash runway extended into
the fourth quarter of 2024
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage,
next-generation gene editing company focused on developing
therapies that harness targeted gene integration to treat or cure
serious diseases, today reported recent business progress and
fourth quarter and fiscal year 2021 financial results.
“2021 was a pivotal year in our company’s history as we
established ourselves as a public company and initiated the
clinical trial for our lead candidate, GPH101 for sickle cell
disease. In 2022, we remain focused on advancing our research and
development priorities, in particular the execution of our Phase
1/2 CEDAR clinical trial of GPH101. We continue to work toward
demonstrating that our unique gene correction approach to reduce
sickle hemoglobin production and restore adult hemoglobin
expression has the potential to achieve the ideal genetic outcome
and provide a definitive cure for patients with sickle cell
disease,” said Josh Lehrer, M.D., M.Phil., chief executive officer
of Graphite Bio.
“Over the course of this year, we look forward to sharing more
information about our research programs, including GPH102, our
differentiated gene replacement program for beta-thalassemia,”
Lehrer continued. “With our updated pipeline priorities, our
programs now even more closely align with our goal of transforming
the gene therapy treatment paradigm – from how we develop and
manufacture these individualized therapies to how we deliver them
to patients. Our mission is to make ‘one dose, one cure’ a reality
for as many patients as possible with serious diseases and
inadequate treatments.”
Program Updates
GPH101 for Sickle Cell Disease
- Continued patient enrollment in the Phase 1/2 CEDAR clinical
trial of GPH101, an investigational therapy designed to directly
correct the genetic mutation responsible for sickle cell disease
(SCD), at multiple sites across the United States. Due to impacts
of the recent COVID-19 Omicron variant surge on patients and site
resources and operations, the company now plans to dose its first
patient in the second half of 2022, with initial proof-of-concept
data anticipated in 2023.
- Presented a trial-in-progress poster about the CEDAR trial at
the 63rd American Society of Hematology (ASH) Annual Meeting &
Exposition and hosted an event featuring a discussion among key
opinion leaders about how the company’s differentiated gene
correction approach using GPH101 could lead to curative outcomes
for SCD patients.
- Presented preclinical data for GPH101 at the 49th Annual Sickle
Cell Disease Association of America’s National Convention. These
data, which support the ability of the company’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, are foundational to the company’s SCD program
and support the evaluation of GPH101 in the CEDAR trial.
Additional R&D Updates
Prioritized and advanced research programs to maximize the
capabilities of the company’s next-generation gene editing platform
and significantly impact patient outcomes, including:
- Announced a new research program, GPH102 for the treatment of
beta-thalassemia, a genetic blood disorder that reduces the
production of hemoglobin and leads to severe anemia and, in severe
cases, transfusion dependency. Using the company’s gene replacement
technology, GPH102 is designed to replace the entire mutated
beta-globin gene and restore adult hemoglobin expression to levels
similar to individuals who do not have disease or have
beta-thalassemia trait. The company expects to submit an IND for
this program by mid-2024, pending feedback from health
authorities.
- Disclosed an early-stage research program for the treatment of
alpha-1 antitrypsin (AAT) deficiency, a severe inherited genetic
disorder that can cause progressive lung and liver disease. The
program leverages the company’s targeted gene insertion technology
to permanently increase AAT protein production, offering a
potentially differentiated approach to treating the disease. The
company expects to provide updates about this program over the
course of 2022.
- Initiated discovery research efforts to develop non-genotoxic
hematopoietic stem cell (HSC) targeted conditioning (NGTC)
regimens, which could greatly broaden the number of diseases and
patients who can be treated with precision engineered, one-time
treatments and cures. The company also intends to leverage industry
advances to accelerate its efforts in this area.
- Announced intentions to develop GPH201 for X-linked combined
deficiency syndrome (XSCID) with an academic partner. The company
believes GPH201 has the potential to be an important treatment
option for this ultra-rare disease, and data from this program
could be informative to the company’s platform and pipeline.
- Reassessed the development strategy for GPH301 for Gaucher
disease. The company believes GPH301 can have the greatest impact
as a potential one-time curative treatment and alternative to
chronic enzyme replacement therapy in patients with Type 1 Gaucher
disease, when combined with a potential NGTC regimen. The company
is assessing a strategy and timeline for GPH301 to enter clinical
testing with an NGTC regimen.
Business Updates
- Appointed Alethia Young as chief financial officer. Ms. Young
has more than 20 years of experience in healthcare and
biotechnology equity research and investing. She joins the company
from Cantor Fitzgerald, where she served as senior biotechnology
analyst and head of research, managing the equity research
department covering companies across the biotechnology
industry.
- Welcomed Christine Garrett, Ph.D., as chief of staff and senior
vice president of operations. Dr. Garrett has more than 20 years of
industry experience leading cross-functional teams comprised of
research, development and commercial leaders across various phases
of drug development.
- Promoted Julia Tran to the role of chief people officer. Ms.
Tran has more than 20 years of experience building and growing
passionate, mission-driven organizations that are committed to
developing innovative solutions to tackle some of the biggest
global challenges in biotech, cleantech and cybersecurity.
Fourth Quarter Financial Highlights
- Cash, Cash Equivalents and Restricted Cash: As of
December 31, 2021, cash, cash equivalents and restricted cash
totaled $378.7 million. The company expects this will fund its
planned operations into the fourth quarter of 2024.
- R&D Expenses: Research and development expenses were
$11.2 million for the fourth quarter of 2021, which includes $1.1
million in stock-based compensation expense.
- G&A Expenses: General and administrative expenses
were $7.7 million for the fourth quarter of 2021, which includes
$1.4 million in stock-based compensation expense.
- Net Loss: Net loss was $18.9 million, or $0.35 per basic
and diluted share, for the fourth quarter of 2021.
Fiscal Year 2021 Financial Highlights
- R&D Expenses: Research and development expenses were
$37.9 million for fiscal year 2021, which includes $2.7 million in
stock-based compensation expense.
- G&A Expenses: General and administrative expenses
were $22.5 million for fiscal year 2021, which includes $5.2
million in stock-based compensation expense.
- Net Loss: Net loss was $70.8 million, or $2.45 per basic
and diluted share, for fiscal year 2021. This includes a change in
the fair value of our Series A redeemable convertible preferred
stock, which was fully converted upon our initial public offering
in June 2021.
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, the timing for treating the first patient
in our Phase 1/2 clinical trial of GPH101 and the availability of
initial proof-of-concept data, our research and development plans,
including our GPH102 research program for the treatment of
beta-thalassemia and our plans to submit an IND for this program,
our research program for the treatment of AAT and our plans to
provide updates regarding this program, our plans to develop GPH201
for XSCID and GPH301 for Gaucher disease with an NGTC regimen, and
the timing of these activities, and our anticipated cash runway,
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 SEC, including
its quarterly report on Form 10-Q filed with the SEC on November
10, 2021, and subsequent filings thereafter. Graphite Bio
explicitly disclaims any obligation to update any forward-looking
statements except to the extent required by law.
GRAPHITE BIO, INC.
Statements of Operations and
Comprehensive Loss
(In thousands, except share
and per share data)
Three Months Ended
Twelve Months Ended
December 31,
December 31,
2021
2020
2021
2020
Operating expenses*:
Research and development
$
11,205
$
6,390
$
37,932
$
9,123
General and administrative
7,736
1,933
22,511
4,377
Total operating expenses
18,941
8,323
60,443
13,500
Loss from operations
(18,941
)
(8,323
)
(60,443
)
(13,500
)
Other income (expense), net:
Other income, net
10
—
24
—
Related party convertible note interest
expense
—
—
—
(40
)
Change in fair value of the Series A
redeemable
convertible preferred stock tranche
liability
—
(55,213
)
(10,341
)
(54,833
)
Total other income (expense), net
10
(55,213
)
(10,317
)
(54,873
)
Net loss and comprehensive loss
$
(18,931
)
$
(63,536
)
$
(70,760
)
$
(68,373
)
Net loss per common share – basic and
diluted
$
(0.35
)
$
(18.68
)
$
(2.45
)
$
(29.93
)
Weighted-average number of common
shares
outstanding – basic and diluted
53,429,766
3,401,912
28,919,255
2,284,087
* Includes stock-based compensation as
follows:
Research and development
$
1,051
$
117
$
2,685
$
123
General and administrative
1,396
24
5,186
54
Total stock-based compensation
expense
$
2,447
$
141
$
7,871
$
177
GRAPHITE BIO, INC.
Balance Sheets
(In thousands)
December 31,
December 31,
2021
2020
Assets
Current assets:
Cash and cash equivalents
$
376,976
$
19,782
Restricted cash
-
35
Prepaid expenses and other current
assets
4,760
1,286
Total current assets
381,736
21,103
Restricted cash, non-current
1,716
—
Property, plant, and equipment, net
6,507
1,461
Operating lease right-of-use assets
11,574
—
Other assets
454
—
Total assets
$
401,987
$
22,564
Liabilities, redeemable convertible
preferred stock, and stockholders’ equity (deficit)
Current liabilities:
Accounts payable
$
2,453
$
630
Accrued compensation
2,689
466
Accrued research costs
633
1,764
Accrued expenses and other current
liabilities
886
126
Current portion of operating lease
liabilities
5,482
—
Series A redeemable convertible preferred
stock tranche liability
—
29,062
Total current liabilities
12,143
32,048
Non-current operating lease
liabilities
5,794
—
Other liabilities
—
316
Total liabilities
17,937
32,364
Series A redeemable convertible preferred
stock
—
55,608
Stockholders’ equity (deficit):
Common stock
1
—
Additional paid-in capital
525,400
5,183
Accumulated deficit
(141,351
)
(70,591
)
Total stockholders’ equity (deficit)
384,050
(65,408
)
Total liabilities, redeemable convertible
preferred stock, and stockholders’ equity (deficit)
$
401,987
$
22,564
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220321005380/en/
Company Contact: 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: Sheryl Seapy Real Chemistry 949-903-4750
media@graphitebio.com
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