Generation Bio Announces Recent Business Highlights and First Quarter 2024 Financial Results
May 13 2024 - 4:05PM
Generation Bio Co. (Nasdaq: GBIO), a biotechnology company
innovating genetic medicines for people living with rare and
prevalent diseases, reported additional data on its cell-targeted
lipid nanoparticle (ctLNP) platform presented at the American
Society of Gene and Cell Therapy (ASGCT) 27th Annual Meeting as
well as first quarter 2024 financial results.“The data presented on
our ctLNP and iqDNA platforms highlight their maturity and
differentiation as the foundation for building programs in
hematology,” said Geoff McDonough, M.D., chief executive officer of
Generation Bio. “Our unique non-viral delivery and cargo may unlock
a series of non-viral in vivo therapeutic candidates, starting with
sickle cell disease and hemophilia A. We believe our medicines
could have a drug-like, redosable in vivo profile, meaning they can
surpass what is possible for current viral, biologic and ex vivo
approaches to reach people on a global scale.”
Recent Highlights:
- Presented New Data
Demonstrating In Vivo Delivery of
a Therapeutic Transgene to T cells with ctLNP: During an
oral presentation at ASGCT, Generation Bio discussed in vivo data
demonstrating selective delivery of a T cell-targeted ctLNP
carrying mRNA cargo encoding a CAR. In these studies, CAR
expression was efficient and dose-dependent with robust surface
presentation on T cells. Transduced CAR T cells demonstrated
preliminary efficacy in an in vitro tumor cell killing assay. The
data were generated as part of the company’s collaboration with
Moderna, Inc.Generation Bio has engineered its ctLNPs to avoid
clearance by the liver and spleen, allowing selective targeting of
tissues and cell types with the addition of a receptor-specific
targeting ligand. The next steps for the platform include assessing
efficacy in mouse disease models and evaluating the delivery of
immune-quiet DNA (iqDNA) with T cell ctLNPs.The company is also
developing a ctLNP targeting hematopoietic stem cells (HSCs) as
part of its sickle cell disease and beta-thalassemia program. At
ASGCT, the company showed preliminary data demonstrating selective
uptake and expression in human HSCs delivered by an HSC-targeted
ctLNP.“The demonstration of selective delivery with a therapeutic
cargo outside the liver is an important proof point for the ability
of our ctLNPs to reach previously unreachable cell types and
tissues,” said Phillip Samayoa, Ph.D., chief strategy officer of
Generation Bio. “We look forward to advancing our ctLNP platform in
T cells and continuing to expand the application into other cell
types, beginning with a wholly-owned program in sickle cell disease
and beta-thalassemia.”
- Disclosed New Information
About iqDNA Platform and RES: Also at
ASGCT, Generation Bio described its iqDNA as a modified,
structured, partially single-stranded DNA that is non-immunogenic
while remaining transcriptionally active. The company invented
iqDNA using its proprietary rapid enzymatic synthesis (RES) method.
RES is a cell-free process that allows for precise chemical and
structural changes to DNA. This high degree of control enables the
enhancement of DNA functionality by engineering molecular design
and components.Generation Bio is advancing its iqDNA platform with
the application of RES. The findings presented at ASGCT
demonstrated that a second generation of iqDNA achieved greater
luciferase expression than a first-generation iqDNA. The company is
currently testing formulations of iqDNA encoding Factor VIII
delivered by LNP.
First Quarter 2024 Financial Results
- Cash
Position: Cash, cash equivalents, and marketable
securities were $233.9 million as of March 31, 2024,
compared to $264.4 million in cash, cash equivalents, and
marketable securities as of December 31, 2023. The company
continues to believe that its cash, cash equivalents, and
marketable securities will fund its operating plan into the second
half of 2027.
- R&D
Expenses: Research and development (R&D) expenses
were $14.3 million for the quarter ended March 31,
2024, compared to $22.0 million for the quarter ended March 31,
2023.
- G&A
Expenses: General and administrative (G&A)
expenses were $10.4 million for the quarter ended March
31, 2024, compared to $12.9 million for the quarter ended March 31,
2023.
- Loss on lease
termination: During the three months ended March 31,
2024, we recognized a non-cash charge of $56.9 million in
connection with the termination of the Seyon Lease.
- Net Loss: Net loss was $74.5
million, or $1.12 basic and diluted net loss per share, for
the quarter ended March 31, 2024, compared to a net loss of $ 32.1
million, or $0.53 basic and diluted net loss per share, for the
quarter ended March 31, 2023.
About Generation Bio
Generation Bio is innovating non-viral genetic medicines to
provide durable and redosable treatments for hundreds of millions
of patients living with rare and prevalent diseases. The company is
developing two distinct and complementary platforms: a potent,
highly selective cell-targeted lipid nanoparticle (ctLNP) delivery
system and a novel immune-quiet DNA (iqDNA) cargo produced by a
scalable capsid-free manufacturing process that uses proprietary
cell-free rapid enzymatic synthesis (RES). With these platforms,
Generation Bio aims to develop the next wave of non-viral genetic
medicines to support its mission to extend the reach of genetic
medicine to more people living with more diseases, around the
world.
For more information, please visit www.generationbio.com.
Forward-Looking Statements
Any statements in this press release about future expectations,
plans and prospects for the company, including statements about the
company’s strategic plans or objectives, cash resources, technology
platforms, research and clinical development plans, and preclinical
data, including those relating to immune-quiet DNA, and other
statements containing the words “believes,” “anticipates,” “plans,”
“expects,” and similar expressions, constitute forward-looking
statements within the meaning of The Private Securities Litigation
Reform Act of 1995. Actual results may differ materially from those
indicated by such forward-looking statements as a result of various
important factors, including: uncertainties inherent in the
identification and development of product candidates, including the
conduct of research activities, the initiation and completion of
preclinical studies and clinical trials and clinical development of
the company’s product candidates; uncertainties as to the
availability and timing of results from preclinical studies and
clinical trials; uncertainties regarding our novel platforms and
related technologies; whether results from preclinical studies will
be predictive of the results of later preclinical studies and
clinical trials; challenges in the manufacture of genetic medicine
products; whether the company’s cash resources are sufficient to
fund the company’s operating expenses and capital expenditure
requirements for the period anticipated; as well as the other risks
and uncertainties set forth in the “Risk Factors” section of the
company’s most recent annual report on Form 10-K, which is on file
with the Securities and Exchange Commission, and in subsequent
filings the company may make with the Securities and Exchange
Commission. In addition, the forward-looking statements included in
this press release represent the company’s views as of the date
hereof. The company anticipates that subsequent events and
developments will cause the company’s views to change. However,
while the company may elect to update these forward-looking
statements at some point in the future, the company specifically
disclaims any obligation to do so. These forward-looking statements
should not be relied upon as representing the company’s views as of
any date subsequent to the date on which they were made.
Investors and Media ContactMaren
KillackeyGeneration Biomkillackey@generationbio.com857-371-4638
|
GENERATION BIO CO.CONSOLIDATED BALANCE
SHEET DATA(Unaudited)(In
thousands) |
|
|
|
|
|
|
|
|
|
|
|
|
|
Earnings Release Balance Sheet |
|
March 31, 2024 |
|
December 31, 2023 |
| Cash, cash
equivalents and marketable securities |
|
$ |
233,937 |
|
$ |
264,364 |
| Working
capital |
|
|
188,851 |
|
|
232,704 |
| Total
assets |
|
|
285,879 |
|
|
374,758 |
| Total
stockholders’ equity |
|
|
131,991 |
|
|
203,128 |
|
GENERATION BIO CO.CONSOLIDATED STATEMENTS
OF OPERATIONS(Unaudited)(in
thousands, except share and per share data) |
| |
|
|
|
|
|
|
|
| |
|
Three Months Ended March 31, |
| |
|
2024 |
|
|
|
2023 |
|
| Revenues: |
|
|
|
|
|
|
|
Collaboration Revenue |
|
$ |
4,059 |
|
|
$ |
— |
|
| Operating
expenses: |
|
|
|
|
|
|
|
Research and development |
|
|
14,335 |
|
|
|
22,000 |
|
|
General and administrative |
|
|
10,428 |
|
|
|
12,866 |
|
|
Loss on lease termination |
|
|
56,930 |
|
|
|
— |
|
|
Total operating expenses |
|
|
81,693 |
|
|
|
34,866 |
|
| Loss from
operations |
|
|
(77,634 |
) |
|
|
(34,866 |
) |
| Other income: |
|
|
|
|
|
|
|
Other income and interest income, net |
|
|
3,093 |
|
|
|
2,772 |
|
| Net loss |
|
$ |
(74,541 |
) |
|
$ |
(32,094 |
) |
| Net loss per
share, basic and diluted |
|
$ |
(1.12 |
) |
|
$ |
(0.53 |
) |
| Weighted average
common shares outstanding, basic and diluted |
|
|
66,433,640 |
|
|
|
60,230,077 |
|
| |
|
|
|
|
|
|
| Comprehensive
loss: |
|
|
|
|
|
|
| Net loss |
|
$ |
(74,541 |
) |
|
$ |
(32,094 |
) |
| Other
comprehensive (loss) income: |
|
|
|
|
|
|
|
Unrealized (losses) gains on marketable securities |
|
|
(471 |
) |
|
|
117 |
|
| Comprehensive
loss |
|
$ |
(75,012 |
) |
|
$ |
(31,977 |
) |
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