GPH101 for sickle cell disease granted U.S. FDA
Fast Track Designation; dosing of first patient in Phase 1/2 CEDAR
clinical trial on track for second half of 2022, with initial
proof-of-concept data anticipated in 2023
Oral presentation highlighting preclinical data
for GPH102 for beta-thalassemia at upcoming ASGCT 25th Annual
Meeting
$352.1 million in cash, cash equivalents and
investments in marketable securities as of March 31, 2022; cash
runway into fourth quarter of 2024
Graphite Bio, Inc. (Nasdaq: GRPH), a clinical-stage,
next-generation gene editing company harnessing the power of
high-efficiency precision gene repair to develop therapies with the
potential to treat or cure serious diseases, today reported recent
business progress and first quarter 2022 financial results.
“In the first quarter of 2022, we announced our updated research
and development priorities to focus on programs that maximize the
capabilities of our next-generation gene editing platform and can
significantly impact patient outcomes. We continue to advance our
research and development efforts across our pipeline, particularly
the execution of our Phase 1/2 CEDAR clinical trial of GPH101 for
sickle cell disease. We remain on track to dose our first patient
in the second half of this year, and we look forward to generating
data that demonstrates GPH101’s curative potential using our unique
gene correction approach,” said Josh Lehrer, M.D., M.Phil., chief
executive officer of Graphite Bio. “In addition, we are excited to
share at the ASGCT 25th Annual Meeting next week more information
about our GPH102 program for beta-thalassemia, which demonstrates
our platform’s gene replacement capabilities. Similar to our gene
correction approach in sickle cell disease, we believe our gene
replacement approach in beta-thalassemia could be the optimal way
to treat the disease and provide a definitive cure to
patients.”
Program Updates
GPH101 for Sickle Cell Disease
- Granted U.S. Food and Drug Administration (FDA) Fast Track
Designation, which facilitates the expedited development and review
of new drugs or biologics that are intended to treat serious or
life-threatening conditions and demonstrate the potential to
address unmet medical needs. In November 2021, GPH101, an
investigational therapy designed to directly correct the genetic
mutation responsible for sickle cell disease (SCD), was granted
orphan drug designation by the FDA.
- Continued patient enrollment in the Phase 1/2 CEDAR clinical
trial of GPH101 at multiple sites across the United States. The
company remains on track to dose its first patient in the second
half of 2022, with initial proof-of-concept data anticipated in
2023.
GPH102 for Beta-Thalassemia
- Received acceptance of an oral presentation highlighting the
discovery and preclinical development of GPH102 at the American
Society of Gene and Cell Therapy (ASGCT) 25th Annual Meeting. The
company will also present an encore trial-in-progress poster about
the Phase 1/2 CEDAR trial of GPH101 for SCD. The hybrid meeting
will take place virtually and at the Walter E. Washington
Convention Center in Washington, D.C., from May 16-19.
- Continued to advance the preclinical development of GPH102.
Using the company's gene replacement approach, GPH102 is designed
to directly replace and normalize the entire mutated beta-globin
gene with a functional gene and restore adult hemoglobin expression
to levels similar to individuals who do not have the disease. The
company expects to submit an IND for this program by mid-2024,
pending feedback from regulatory authorities
First Quarter Financial Highlights
- Cash Position: As of March 31, 2022, cash, cash
equivalents and investments in marketable securities totaled $352.1
million. The company continues to expect this will fund its planned
operations into the fourth quarter of 2024.
- R&D Expenses: Research and development expenses were
$18.2 million for the first quarter of 2022, which includes $1.4
million in stock-based compensation expense.
- G&A Expenses: General and administrative expenses
were $7.7 million for the first quarter of 2022, which includes
$2.0 million in stock-based compensation expense.
- Net Loss: Net loss was $25.8 million, or $0.48 per basic
and diluted share, for the quarter ended March 31, 2022.
About GPH101 for Sickle Cell Disease
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). 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 blood disease worldwide. 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 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 safety, engraftment success, gene correction rates, total
hemoglobin, as well as other clinical and exploratory endpoints and
pharmacodynamics in patients with severe SCD.
About GPH102 for Beta-Thalassemia
GPH102 is Graphite Bio’s research program for the treatment of
beta-thalassemia, one of the most common autosomal recessive
disorders with approximately 68,000 people worldwide born with the
disease each year. Beta-thalassemia is a genetic blood disorder
characterized by reduced production of beta-globin, a protein that
forms oxygen-carrying hemoglobin with alpha-globin. Individuals
with the most severe form of beta-thalassemia fail to produce
functional beta-globin, which results in severe anemia and
transfusion dependency. Using Graphite Bio’s gene replacement
approach, GPH102 is designed to replace the mutated beta-globin
gene with a functional gene and restore adult hemoglobin (HbA)
expression to levels similar to individuals who do not have the
disease.
About Graphite Bio
Graphite Bio is a clinical-stage, next-generation gene editing
company harnessing the power of high-efficiency precision gene
repair 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 UltraHDR™ gene editing platform is designed to precisely
correct genetic mutations, replace entire disease-causing genes
with functional 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,
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 views about our plans, intentions,
expectations, strategies, prospects, 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 regulatory hurdles or delays in patient enrollment and
dosing, and in the initiation, progress, conduct and completion of
our planned clinical trials, and that our operating expenses may
exceed our current estimates. These risks concerning Graphite Bio’s
programs and operations are described in additional detail in its
periodic filings with the SEC, including its most recently filed
periodic report, 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.
Condensed Statements of
Operations and Comprehensive Loss
(unaudited)
(in thousands, except share
and per share data)
Three Months Ended
March 31,
2022
2021
Operating expenses*:
Research and development
$
18,246
$
5,377
General and administrative
7,712
3,991
Total operating expenses
25,958
9,368
Loss from operations
(25,958
)
(9,368
)
Other income (expense), net:
Other income, net
123
—
Change in fair value of the Series A
redeemable convertible preferred
stock tranche liability
—
(10,341
)
Total other income (expense), net
123
(10,341
)
Net loss attributable to common
stockholders
$
(25,835
)
$
(19,709
)
Unrealized loss on investments
(309
)
—
Net loss and comprehensive loss
(26,144
)
(19,709
)
Net loss per common share – basic and
diluted
$
(0.48
)
$
(5.75
)
Weighted-average number of common shares
outstanding – basic and
diluted
54,005,299
3,425,089
* Includes stock-based compensation as
follows:
Research and development
$
1,373
$
196
General and administrative
1,969
837
Total stock-based compensation
expense
$
3,342
$
1,033
GRAPHITE BIO, INC.
Condensed Balance
Sheets
(in thousands)
March 31,
December 31,
2022
2021
Assets
(unaudited)
Current assets:
Cash and cash equivalents
$
185,070
$
376,976
Investments in marketable securities,
current
154,112
—
Prepaid expenses and other current
assets
4,574
4,760
Total current assets
343,756
381,736
Restricted cash, non-current
1,716
1,716
Investments in marketable securities,
non-current
12,927
—
Property and equipment, net
9,374
6,507
Operating lease right-of-use assets
10,122
11,574
Other assets
711
454
Total assets
$
378,606
$
401,987
Liabilities and stockholders’
equity
Current liabilities:
Accounts payable
$
3,069
$
2,453
Accrued compensation
1,023
2,689
Accrued research costs
1,879
633
Accrued expenses and other current
liabilities
1,158
886
Operating lease liabilities, current
5,636
5,482
Total current liabilities
12,765
12,143
Operating lease liabilities,
non-current
4,542
5,794
Total liabilities
17,307
17,937
Stockholders’ equity:
Common stock
1
1
Additional paid-in capital
528,793
525,400
Accumulated other comprehensive loss
(309
)
—
Accumulated deficit
(167,186
)
(141,351
)
Total stockholders’ equity
361,299
384,050
Total liabilities and stockholders’
equity
$
378,606
$
401,987
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220512005981/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: Sheryl Seapy Real
Chemistry 949-903-4750 media@graphitebio.com
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