Generation Bio Reports Business Highlights and Fourth Quarter and Full Year 2023 Financial Results
07 March 2024 - 8:20AM
Generation Bio Co. (Nasdaq:GBIO), a biotechnology company
innovating genetic medicines for people living with rare and
prevalent diseases, reported business highlights and fourth quarter
and full year 2023 financial results.
“Late last year we announced important advances in our
cell-targeted lipid nanoparticle and immune-quiet DNA platforms
that we are now translating to our portfolio strategy,” said Geoff
McDonough, M.D., chief executive officer of Generation Bio. “The
potency and selectivity of our ctLNP platform open up significant
opportunities for in vivo delivery of T cell and hematopoietic stem
cell therapies, and this year we are laying the foundation for a
wholly-owned program in sickle cell disease and beta-thalassemia.
We are also continuing to optimize iqDNA to progress our hemophilia
A program and to explore therapeutic applications in cell types
beyond hepatocytes. With our current cash balance, we believe we
have sufficient runway to reach meaningful development milestones
for our platforms and programs.”
Business Highlights
- Developing ctLNP for in vivo
T cell and hematopoeitc stem cell (HSC) programs:
Generation Bio’s ctLNP platform has demonstrated a set of key
characteristics that could make possible in vivo genetic medicine
approaches to treating diseases for which only ex vivo therapies
are currently available. The company has developed ctLNPs that
selectively target and transduce 75% of circulating and splenic T
cells and maintain high levels of delivery at doses as low as 0.005
mg/kg in humanized mice. In addition to continuing this work in T
cells in collaboration with Moderna, Generation Bio is developing a
wholly-owned in vivo program targeting HSCs to address sickle cell
disease and beta-thalassemia. In vivo approaches could offer
important advantages over current ex vivo approaches, such as the
lack of pre-treatment chemotherapy, redosing, on-demand
availability and lower cost. In 2024, the company intends to show
highly selective in vivo delivery to T cells with therapeutic
transgenes, and proof of concept for specific and potent in vivo
HSC targeting in humanized mouse models.
- Developing iqDNA for
programs in hepatocytes and other cell types: In 2023,
Generation Bio announced that it had developed a novel DNA cargo
that was shown to avoid innate immune detection in both mice and
NHPs. In addition to these immune quiet characteristics, iqDNA also
exhibited robust and durable expression in mice. This profile could
lead to differentiated therapeutics utilizing hepatocytes to treat
a wide range of genetic diseases, including hemophilia A, as well
extrahepatic tissues and cell types, such as T cells and HSCs. In
2024, the company intends to optimize iqDNA for application in
hepatocytes and T cells with a focus on improving potency.
Fourth Quarter and Full Year 2023 Financial
Results
- Cash Position: Cash, cash equivalents and
marketable securities were $264.4 million as
of December 31, 2023, compared with $279.1 million in cash,
cash equivalents, and marketable securities as of December 31,
2022. The company continues to believe that its cash, cash
equivalents and marketable securities will fund its operating
expenditures and capital expenditure requirements into the second
half of 2027.
- R&D Expenses: Research and
development (R&D) expenses
were $27.9 million for the quarter ended December
31, 2023, including a one-time $5.1 million charge related to the
company’s reduction in force, and $93.6 million for the year ended
December 31, 2023, compared to $21.6 million for the quarter ended
December 31, 2022, and $96.7 million for the year ended December
31, 2022.
- G&A Expenses: General and
administrative (G&A) expenses
were $13.4 million for the quarter ended December
31, 2023, including a one-time $1.9 million charge related to the
company’s reduction in force, and $50.9 million for the year ended
December 31, 2023, compared to $13.1 million for the quarter ended
December 31, 2022, and $44.5 million for the year ended December
31, 2022.
- Net Loss: Net loss was $35.2 million, or $0.53
basic and diluted net loss per share, for the quarter ended
December 31, 2023, and $126.6 million, or $1.96 basic and diluted
net loss per share, for the year ended December 31, 2023, compared
to a net loss of $32.4 million, or $0.55 basic and diluted net loss
per share, for the quarter ended December 31, 2022, and $136.6
million, or $2.35 basic and diluted net loss per share, for the
year ended December 31, 2022.
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 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 and quarterly report on Form 10-Q, which are 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 |
December 31, 2023 |
|
December 31, 2022 |
Cash,
cash equivalents and marketable securities |
$ |
264,364 |
|
|
$ |
279,091 |
|
Working capital |
|
232,704 |
|
|
|
267,866 |
|
Total
assets |
|
374,758 |
|
|
|
376,264 |
|
Total
stockholders’ equity |
|
203,128 |
|
|
|
282,493 |
|
|
|
|
|
|
|
|
|
GENERATION BIO CO.CONSOLIDATED STATEMENTS
OF OPERATIONS (Unaudited)(in
thousands, except share and per share data) |
|
|
Three Months Ended December 31, |
|
Year Ended December 31, |
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Revenues: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration Revenue |
$ |
2,878 |
|
|
$ |
— |
|
|
$ |
5,904 |
|
|
$ |
— |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Operating expenses: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and development |
|
27,923 |
|
|
|
21,607 |
|
|
|
93,617 |
|
|
|
96,718 |
|
General and administrative |
|
13,376 |
|
|
|
13,081 |
|
|
|
50,850 |
|
|
|
44,464 |
|
Total operating expenses |
|
41,299 |
|
|
|
34,688 |
|
|
|
144,467 |
|
|
|
141,182 |
|
Loss from operations |
|
(38,421 |
) |
|
|
(34,688 |
) |
|
|
(138,563 |
) |
|
|
(141,182 |
) |
Other income: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Other income and interest income, net |
|
3,235 |
|
|
|
2,283 |
|
|
|
11,951 |
|
|
|
4,543 |
|
Net loss |
$ |
(35,186 |
) |
|
$ |
(32,405 |
) |
|
$ |
(126,612 |
) |
|
$ |
(136,639 |
) |
Net loss per share, basic and
diluted |
$ |
(0.53 |
) |
|
$ |
(0.55 |
) |
|
$ |
(1.96 |
) |
|
$ |
(2.35 |
) |
Weighted average common shares
outstanding, basic and diluted |
|
66,062,208 |
|
|
|
59,407,296 |
|
|
|
64,483,520 |
|
|
|
58,114,893 |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Comprehensive loss: |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss |
$ |
(35,186 |
) |
|
$ |
(32,405 |
) |
|
$ |
(126,612 |
) |
|
$ |
(136,639 |
) |
Other comprehensive income
(loss): |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Unrealized gains (losses) on marketable securities |
|
278 |
|
|
|
219 |
|
|
|
357 |
|
|
|
(83 |
) |
Comprehensive loss |
$ |
(34,908 |
) |
|
$ |
(32,186 |
) |
|
$ |
(126,255 |
) |
|
$ |
(136,722 |
) |
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
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