Presented positive data from first five
patients in Phase 1/2 cystinosis trial1 showing systemic gene
therapy effect at the American Society of Gene and Cell Therapy
Annual Meeting; all five patients remain off oral cysteamine
Comprehensive Gaucher disease franchise update
planned for Q4 2022
Regulatory interactions planned in 2H 2022 to
inform clinical development strategies for cystinosis and Gaucher
disease type 3
Hunter syndrome program received FDA Orphan
Drug Designation (ODD)
Strong balance sheet with cash runway into Q1
2024
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene
therapy company working to free people from a lifetime of genetic
disease, today reported financial results for the second quarter
ended June 30, 2022 and provided a business update.
“We’ve made significant progress advancing our pipeline this
quarter,” said Geoff MacKay, president and CEO of AVROBIO. “We’re
thrilled with the ongoing clinical data generated by the first and
only gene therapy trial for cystinosis, showing hematopoietic stem
cell (HSC) gene therapy’s potential to stabilize or reduce the
impact of cystinosis on different tissues throughout the body.
We’re preparing for regulatory agency interactions this fall to
discuss our clinical development and regulatory strategy for this
program, with the intent of initiating a company-sponsored clinical
trial in 2023.
“We have multiple milestones planned for the second half of the
year that will further inform the advancement of our pipeline. We
plan to provide a comprehensive Gaucher disease franchise update in
Q4 2022, including new clinical data from our Guard1 Phase 1/2
clinical trial for Gaucher disease type 1, and updates on the
clinical development and regulatory strategy for our Gaucher
disease type 3 program. In addition, our collaborators continue to
make progress toward the initiation of the planned Phase 1/2
clinical trial for Hunter syndrome,” said MacKay.
“More broadly in the gene therapy sector, the Food and Drug
Administration (FDA) Advisory Committee unanimously voted in June
to recommend approval of two new HSC gene therapies for two
distinct genetic disorders, a potentially important milestone for
patients and families. We believe the Advisory Committee’s
discussions may provide a window into how future HSC gene therapies
will be evaluated by the agency with the recognition that each
vector-promoter-gene combination has its own benefit/risk profile,”
MacKay added.
Program Updates
Presented clinical data on the first five
patients dosed in the fully enrolled Phase 1/2 clinical trial for
AVR-RD-04 in cystinosis, including new, early data showing key
visual motor integration, visual perception and motor coordination
measures impacted by cystinosis stabilized or improved post gene
therapy, at the American Society of Gene and Cell Therapy (ASGCT)
Annual Meeting
- Early data indicate that post HSC gene therapy, patients have
been able to produce functional cystinosin protein throughout the
body, as evidenced by measurements of blood, eye, skin and
gastrointestinal mucosa, which indicate a reduction in the
accumulation of cystine crystals or prevention of further
pathological accumulation
- Sustained engraftment demonstrated by stable vector copy number
(VCN) in patients beyond 12 months with all five dosed patients
remaining off oral cysteamine post gene therapy
- Data from the five patients dosed to date indicate no adverse
events (AEs) related to drug product. All AEs were related to
myeloablative conditioning, stem cell mobilization, underlying
disease or pre-existing conditions
- Collaborator-sponsored Phase 1/2 clinical trial is funded in
part by grants to University of California San Diego from the
California Institute for Regenerative Medicine (CIRM), Cystinosis
Research Foundation (CRF) and National Institutes of Health
(NIH)
- Read full press release here
Received Orphan Drug Designation (ODD)
from FDA for AVR-RD-05 for Hunter syndrome (Mucopolysaccharidosis
Type II (MPSII))
- Read full press release here
Presented preclinical data demonstrating
that HSC gene therapy significantly reduced accumulation of
glycogen in a mouse model of infantile-onset Pompe disease,
including in cardiac and skeletal muscle as well as the central
nervous system at ASGCT
- Eight months post infusion, substrate levels were reduced
across multiple tissues to levels nearly indistinguishable from
healthy mice
- Read full press release here
Upcoming 2H 2022 Milestones
- AVR-RD-04 in cystinosis: Plan to engage with regulatory
agencies in 2H 2022 to discuss our clinical development and
regulatory strategy with the intent of initiating a
company-sponsored clinical trial in 2023, subject to regulatory
alignment
- AVR-RD-02 in Gaucher disease:
- Gaucher disease type 1: Plan to provide interim clinical data
update in 2H 2022
- Gaucher disease type 3: Plan to engage with regulatory agencies
on a Phase 2/3 clinical development strategy with aim to initiate a
trial in 2023, subject to regulatory alignment
- AVR-RD-05 in Hunter syndrome: Clinical Trial Application (CTA)
authorization expected 2H 2022 with plans to initiate a
collaborator-sponsored Phase 1/2 clinical trial in 2023, subject to
regulatory alignment
Second Quarter 2022 Financial Results
AVROBIO reported a net loss of $28.1 million for the second
quarter of 2022 as compared to a net loss of $31.4 million for the
comparable period in 2021. This decrease was driven by a reduction
in research and development expenses.
Research and development expenses were $18.9 million for the
second quarter of 2022 as compared to $22.5 million for the
comparable period in 2021. This decrease was driven by a reduction
in program development expenses and personnel-related costs,
including non-cash stock-based compensation.
General and administrative expenses were $8.9 million for the
second quarter of 2022 as compared to $8.8 million for the
comparable period in 2021.
Other (expense) income, net was $0.3 million in expense for the
second quarter of 2022 as compared to other (expense) income, net
of less than $0.1 million in expense for the comparable period in
2021. This increase was driven by interest expense related to our
term loan with Silicon Valley Bank which we entered into during the
fourth quarter of 2021.
As of June 30, 2022, AVROBIO had $132.4 million in cash and cash
equivalents, as compared to $189.6 million in cash and cash
equivalents as of December 31, 2021. Based on AVROBIO’s current
operating plan, AVROBIO expects its cash and cash equivalents as of
June 30, 2022, will enable AVROBIO to fund its operating expenses
and capital expenditure requirements into the first quarter of
2024.
About AVROBIO
Our vision is to bring personalized gene therapy to the world.
We target the root cause of genetic disease by introducing a
functional copy of the affected gene into patients’ own
hematopoietic stem cells (HSCs), with the goal to durably express
the therapeutic protein throughout the body, including the central
nervous system. Our first-in-class pipeline includes clinical
programs for cystinosis and Gaucher disease type 1, as well as
preclinical programs for Gaucher disease type 3, Hunter syndrome
and Pompe disease. Our proprietary plato® gene therapy platform is
designed to be scaled to support late-stage clinical development
and commercialization globally. We are headquartered in Cambridge,
Mass. For additional information, visit avrobio.com and follow us
on Twitter and LinkedIn.
Forward-Looking Statement
This press release contains forward-looking statements,
including statements made pursuant to the safe harbor provisions of
the Private Securities Litigation Reform Act of 1995. These
statements may be identified by words and phrases such as “aims,”
“anticipates,” “believes,” “could,” “designed to,” “estimates,”
“expects,” “forecasts,” “goal,” “intends,” “may,” “plans,”
“possible,” “potential,” “seeks,” “will,” and variations of these
words and phrases or similar expressions that are intended to
identify forward-looking statements. These forward-looking
statements include, without limitation, statements regarding our
business strategy for and the potential therapeutic benefits of our
current and prospective product candidates, the expected safety
profile of our investigational gene therapies, results of
preclinical studies, the design, commencement, enrollment and
timing of ongoing or planned clinical trials, clinical trial
results, product approvals and regulatory pathways, the timing of
patient recruitment and enrollment activities, our plans and
expectations with respect to interactions with regulatory agencies
and the timing and likelihood of success thereof, the expected
benefits and results of our implementation of the plato® platform
in our clinical trials and gene therapy programs and its potential
impact on our manufacturing and commercialization activities, and
statements regarding our financial and cash position and expected
cash runway, including impact on anticipated milestones. Any such
statements in this press release that are not statements of
historical fact may be deemed to be forward-looking statements.
Results in preclinical or early-stage clinical trials may not be
indicative of results from later stage or larger scale clinical
trials and do not ensure regulatory approval. You should not place
undue reliance on these statements, or the scientific data
presented.
Any forward-looking statements in this press release are based
on AVROBIO’s current expectations, estimates and projections about
our industry as well as management’s current beliefs and
expectations of future events only as of today 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. These risks and
uncertainties include, but are not limited to, the risk that any
one or more of AVROBIO’s product candidates will not be
successfully developed or commercialized, the risk of cessation or
delay of any ongoing or planned clinical trials of AVROBIO or our
collaborators, the risk that AVROBIO may not successfully recruit
or enroll a sufficient number of patients for our clinical trials,
the risk that AVROBIO may not realize the intended benefits of our
gene therapy platform, including the features of our plato®
platform, the risk that our product candidates or procedures in
connection with the administration thereof will not have the safety
or efficacy profile that we anticipate, the risk that prior
results, such as signals of safety, activity or durability of
effect, observed from preclinical or clinical trials, will not be
replicated or will not continue in ongoing or future studies or
trials involving AVROBIO’s product candidates, the risk that we
will be unable to obtain and maintain regulatory approval for our
product candidates, the risk that the size and growth potential of
the market for our product candidates will not materialize as
expected, risks associated with our dependence on third-party
suppliers and manufacturers, including sole source suppliers, risks
regarding the accuracy of our estimates of expenses and future
revenue, risks relating to our capital requirements and needs for
additional financing, risks relating to clinical trial and business
interruptions resulting from the COVID-19 outbreak or similar
public health crises, including that such interruptions may
materially delay our enrollment and development timelines and/or
increase our development costs or that data collection efforts may
be impaired or otherwise impacted by such crises, and risks
relating to our ability to obtain and maintain intellectual
property protection for our product candidates. For a discussion of
these and other risks and uncertainties, and other important
factors, any of which could cause AVROBIO’s actual results to
differ materially and adversely from those contained in the
forward-looking statements, see the section entitled “Risk Factors”
in AVROBIO’s most recent Annual or Quarterly Report, as well as
discussions of potential risks, uncertainties and other important
factors in AVROBIO’s subsequent filings with the Securities and
Exchange Commission. AVROBIO explicitly disclaims any obligation to
update any forward-looking statements except to the extent required
by law.
CONDENSED CONSOLIDATED BALANCE
SHEETS (In thousands) (Unaudited)
June 30,
December 31,
2022
2021
Cash and cash equivalents
$
132,409
$
189,567
Prepaid expenses and other current
assets
9,672
9,578
Property and equipment, net
3,618
4,126
Other assets
545
566
Total assets
$
146,244
$
203,837
Accounts payable
$
28
$
3,486
Accrued expenses and other current
liabilities
12,757
15,900
Note payable, net of discount
15,104
14,945
Deferred rent, net of current portion
12
30
Total liabilities
$
27,901
$
34,361
Total stockholders’ equity
118,343
169,476
Total liabilities and stockholders’
equity
$
146,244
$
203,837
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS (In thousands, except per share data)
(Unaudited)
Three Months Ended June 30,
Six Months Ended June 30,
2022
2021
2022
2021
Operating expenses:
Research and development
$
18,877
$
22,544
$
38,130
$
41,024
General and administrative
8,897
8,831
19,062
17,235
Total operating expenses
27,774
31,375
57,192
58,259
Loss from operations
(27,774
)
(31,375
)
(57,192
)
(58,259
)
Other (expense) income, net
(280
)
(12
)
(695
)
(27
)
Net loss
($
28,054
)
($
31,387
)
($
57,887
)
($
58,286
)
Net loss per share — basic and diluted
($
0.64
)
($
0.74
)
($
1.32
)
($
1.39
)
Weighted-average number of common shares
outstanding — basic and diluted
43,696
42,510
43,696
42,067
1 Collaborator-sponsored Phase 1/2 clinical trial of AVR-RD-04
is funded in part by grants to University of California San Diego
from the California Institute for Regenerative Medicine (CIRM),
Cystinosis Research Foundation (CRF) and National Institutes of
Health (NIH)
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220809005420/en/
Investor Contact: Christopher F. Brinzey Westwicke, an
ICR Company 339-970-2843 chris.brinzey@westwicke.com Media
Contact: Kit Rodophele Ten Bridge Communications 617-999-9620
krodophele@tenbridgecommunications.com
AVROBIO (NASDAQ:AVRO)
Historical Stock Chart
From Mar 2024 to Apr 2024
AVROBIO (NASDAQ:AVRO)
Historical Stock Chart
From Apr 2023 to Apr 2024