Patient dosing completed in
collaborator-sponsored Phase 1/2 clinical trial for cystinosis
Rare pediatric disease designations received
for Gaucher disease and cystinosis investigational gene
therapies
Comprehensive Gaucher disease program update
planned for Wednesday, Dec. 7
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 third quarter
ended Sept. 30, 2022 and provided a business update.
“We had a steady cadence of news this quarter highlighting our
steadfast efforts to bring our gene therapies to patients,” said
Geoff MacKay, president and CEO of AVROBIO. “We look forward to
providing a comprehensive update next month on our HSC gene therapy
targeting Gaucher disease, the most common lysosomal disorder,
impacting an estimated 30,000 patients worldwide. This update will
contain interim clinical data from our Gaucher disease type 1 Phase
1/2 trial, including data out over two years. Notably, we plan to
provide an update on our regulatory interactions for a planned
Gaucher disease type 3 clinical study, as well as our overall
Gaucher clinical development strategy.”
Program Updates
AVR-RD-02 in Gaucher disease:
- Received rare pediatric disease designation (RPDD) from the
U.S. Food and Drug Administration (FDA). Read full press release
here
- Received Innovation Passport under the Innovative Licensing and
Access Pathway (ILAP) from the U.K. Medicines and Healthcare
products Regulatory Agency (MHRA). Read full press release
here
- AVR-RD-02 previously has been granted Fast Track status from
FDA and orphan drug designation (ODD) in the U.S. and EU
AVR-RD-04 in cystinosis:
- Patient dosing completed in collaborator-sponsored Phase 1/2
trial. Interim efficacy data from first five patients show systemic
gene therapy effect across multiple tissues evaluated, including
peripheral blood leukocytes, eyes, skin, gastrointestinal mucosa
and neurocognitive system. Read full press release here
- Received RPDD for AVR-RD-04 from FDA. Read full press release
here
- AVR-RD-04 previously has been granted ODD in the U.S. and
EU
- Submitted a meeting request to the MHRA to discuss a
company-sponsored clinical trial for AVR-RD-04 and expect a
scientific advice meeting with MHRA in Q1 2023; planning to engage
with other regulatory authorities as we expand this anticipated
global trial
AVR-RD-05 in Hunter syndrome:
- Clinical trial application (CTA) approved by the MHRA, Research
Ethics Committee and Health Research Authority for a Phase 1/2
clinical trial in infants diagnosed with neuronopathic
mucopolysaccharidosis type II (nMPS-II) or Hunter syndrome.
AVROBIO’s collaborators at the University of Manchester, U.K.,
expect to dose the first patient in the trial in 1H 2023. Read full
press release here
- AVR-RD-05 previously has been granted RPDD and ODD in the
U.S.
Presented positive data on the combined
use of two state-of-the-art assays to evaluate the genotoxicity
risk of integrating vectors during preclinical development, at the
29th Annual Congress of the European Society of Gene & Cell
Therapy (ESGCT), Oct. 11-14, 2022 in Edinburgh, Scotland
- Read the full press release here
Gaucher disease program update planned for Dec. 7,
2022
The company will provide an update to investors on its Gaucher
disease program, including new and updated data from the Guard1
Phase 1/2 clinical trial of AVR-RD-02 for Gaucher disease type 1
and an update on regulatory interactions for a planned Gaucher
disease type 3 clinical study, as well as the overall Gaucher
clinical development strategy. More information on this virtual
event will follow in the next few weeks and will be posted on the
corporate website ahead of the event.
Third Quarter 2022 Financial Results
AVROBIO reported a net loss of $23.0 million for the third
quarter of 2022 as compared to a net loss of $32.6 million for the
comparable period in 2021. This decrease was driven by a reduction
in research and development expenses.
Research and development expenses were $15.9 million for the
third quarter of 2022 as compared to $23.0 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 $7.1 million for the
third quarter of 2022 as compared to $9.6 million for the
comparable period in 2021. This decrease was attributable to a
decrease in personnel-related costs, including non-cash stock-based
compensation, and a decrease in other expenses, primarily related
to facilities costs and professional fees.
Other income (expense), net was less than $0.1 million in income
for the third quarter of 2022 as well as for the comparable period
in 2021.
As of September 30, 2022, AVROBIO had $116.0 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
September 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 we may be unable to realize the
potential benefits associated with rare pediatric disease
designation, the Innovative Licensing and Access Pathway, or any
other regulatory strategy, 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)
September 30,
December 31,
2022
2021
Cash and cash equivalents
$
115,968
$
189,567
Prepaid expenses and other current
assets
9,773
9,578
Property and equipment, net
3,223
4,126
Other assets
533
566
Total assets
$
129,497
$
203,837
Accounts payable
$
1,814
$
3,486
Accrued expenses and other current
liabilities
14,378
15,900
Note payable, net of discount
15,205
14,945
Deferred rent, net of current portion
10
30
Total liabilities
$
31,407
$
34,361
Total stockholders’ equity
98,090
169,476
Total liabilities and stockholders’
equity
$
129,497
$
203,837
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS (In thousands, except per share
data) (Unaudited)
Three Months Ended September
30,
Nine Months Ended September
30,
2022
2021
2022
2021
Operating expenses:
Research and development
$
15,919
$
23,043
$
54,049
$
64,114
General and administrative
7,066
9,577
26,128
26,765
Total operating expenses
22,985
32,620
80,177
90,879
Loss from operations
(22,985
)
(32,620
)
(80,177
)
(90,879
)
Other income (expense), net
16
7
(679
)
(20
)
Net loss
($
22,969
)
($
32,613
)
($
80,856
)
($
90,899
)
Net loss per share — basic and diluted
($
0.52
)
($
0.75
)
($
1.85
)
($
2.13
)
Weighted-average number of common shares
outstanding — basic and diluted
43,773
43,623
43,722
42,588
View source
version on businesswire.com: https://www.businesswire.com/news/home/20221108005465/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
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