On track to initiate registrational global
Phase 2/3 clinical trial for Gaucher disease type 3 (GD3) in second
half 2023, subject to regulatory alignment
Plan to provide clinical and regulatory updates
on cystinosis program in conjunction with ASGCT annual meeting in
May 2023; plan to initiate late-stage cystinosis clinical trial
activities in second half 2023, subject to regulatory alignment
Collaborator-sponsored Phase 1/2 clinical trial
for mucopolysaccharidosis type II (MPS-II), or Hunter syndrome,
initiated
Announced appointment of current CFO Erik
Ostrowski as interim CEO
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 first quarter
ended March 31, 2023, and provided a business update.
“We look forward to building upon last year’s positive data and
regulatory updates for our lead programs and demonstrating the
potential of our HSC gene therapy approach in the year ahead,” said
Erik Ostrowski, interim CEO and current CFO of AVROBIO. “We plan to
initiate a global, registrational Phase 2/3 trial of our Gaucher
disease type 3 (GD3) program later this year and are happy to
report that our collaborator-sponsored Phase 1/2 trial for Hunter
syndrome has recently commenced enrollment. Additionally, we plan
to provide an update on our cystinosis program in mid-May at the
American Society of Gene and Cell Therapy (ASGCT) Annual
Meeting.”
Program Highlights and Milestones
AVR-RD-02 for Gaucher disease:
- “The Guard1 clinical trial – A first in-human, Phase 1/2 study
evaluating AVR-RD-02, an HSC gene therapy for Gaucher disease:
Preliminary safety, pharmacodynamic and clinical efficacy results
from the subjects observed for up to 24 months post-infusion” --
AVROBIO presented safety and efficacy data at the 19th annual
WORLDSymposium™, Feb. 22-26, 2023.
- Guard1 is recruiting individuals between the ages of 16 and 50
with Gaucher disease type 1 (GD1), including those who are
treatment-naïve and who are stable on enzyme replacement therapy,
with sites in Canada and the U.S.
- “Sustained improvement of clinical CNS and somatic features of
GD3 after HSC gene therapy: A first-in-world report” -- Clinical
data from the first pediatric GD3 patient, dosed with
investigational AVR-RD-02, was presented by one of the patient’s
physicians from the University of Manchester (UoM), U.K. at
WORLDSymposium™. New data included longer time points for
peripheral blood glucocerebrosidase, chitotriosidase and albumin
levels, all trending consistently with previously presented data.
The 11-year-old GD3 patient was dosed at UoM on a named patient
basis.
- Plan to initiate Guard3, a global registrational Phase 2/3
trial for GD3, in the second half of 2023, subject to regulatory
alignment.
- AVR-RD-02 has been granted Rare Pediatric Disease Designation
and Fast Track Designation by FDA, Orphan Drug Designation in the
U.S. and U.K., and an Innovation Passport by MHRA under the
Innovative Licensing and Access Pathway (ILAP).
AVR-RD-04 for cystinosis:
- “Phase 1/2 clinical trial of autologous hematopoietic stem and
progenitor cell (HSPC) gene therapy for cystinosis” --
Collaborators at the University of California, San Diego,1
presented updated data on the six patients dosed in the fully
enrolled Phase 1/2 clinical trial at WORLDSymposium™, including
additional vector copy number (VCN) data, as well as longer time
points for leukocyte cystine levels and skin and GI mucosa cystine
crystal data, for some patients. All clinical and safety data
updates are trending consistently with the prior reported data as
of the most recent safety data cut-off date of Jan. 9, 2023.
- Plan to provide a clinical and regulatory update on the
cystinosis program at the American Society of Gene & Cell
Therapy (ASGCT) Annual Meeting in mid-May 2023.
- Plan to initiate activities for the Phase 1/2 clinical trial in
the second half of 2023 subject to regulatory alignment.
- AVR-RD-04 has been granted Rare Pediatric Disease Designation
and Fast Track Designation by FDA and Orphan Drug Designation in
the U.S. and U.K.
AVR-RD-05 for neuronopathic mucopolysaccharidosis type II
(MPS-II), or Hunter syndrome:
- Collaborator-sponsored Phase 1/2 clinical trial for
neuronopathic mucopolysaccharidosis type II (MPS-II), or Hunter
syndrome, initiated.
- “Validation of a GMP stem cell gene therapy manufacturing
process for mucopolysaccharidosis type II (MPS II) in preparation
for an approved Phase 1/2 clinical trial” -- Collaborators at UoM
highlighted data validating their manufacturing process in
preparation for a Phase 1/2 clinical trial for Hunter syndrome
anticipated to start later this year at WORLDSymposium.
- AVR-RD-05 has been granted Rare Pediatric Disease Designation
and Orphan Drug Designation by FDA.
AVR-RD-03 for Pompe disease:
- AVR-RD-03 is currently being evaluated in a pre-clinical
research program and the data to-date have shown significantly
reduced toxic accumulation of glycogen in a mouse model of Pompe
disease, including in cardiac and skeletal muscle as well as the
central nervous system (CNS).
Organizational Update
On May 1, 2023, AVROBIO announced the appointment of Erik
Ostrowski as interim CEO. Mr. Ostrowski, who will continue as
AVROBIO’s CFO, succeeds founding president and CEO Geoff MacKay,
who left to join an emerging early-stage company. The Board of
Directors has begun a search to identify a permanent CEO.
First Quarter 2023 Financial Results
AVROBIO reported a net loss of $25.0 million for the first
quarter of 2023 as compared to a net loss of $29.8 million for the
comparable period in 2022.
Research and development expenses were $17.3 million for the
first quarter of 2023 as compared to $19.3 million for the
comparable period in 2022. This decrease was driven by a reduction
in personnel-related costs, including non-cash stock-based
compensation, and partially offset by an increase in program
development expenses.
General and administrative expenses were $7.9 million for the
first quarter of 2023 as compared to $10.2 million for the
comparable period in 2022. This decrease was driven by a decrease
in personnel-related costs, including non-cash stock-based
compensation.
Other income (expense), net was $0.3 million for the first
quarter of 2023 as compared to other (expense) income, net of
($0.4) million for the comparable period in 2022. This increase in
other income is due to an increase in interest income which was
partially offset by interest expense related to our term loan.
As of March 31, 2023, AVROBIO had $72.3 million in cash and cash
equivalents, as compared to $92.6 million in cash and cash
equivalents as of Dec. 31, 2022. Based on AVROBIO’s current
operating plan, AVROBIO expects its cash and cash equivalents as of
March 31, 2023, 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 of durably
expressing the therapeutic protein throughout the body, including
the central nervous system. Our first-in-class pipeline includes
clinical programs for Gaucher disease, cystinosis and Hunter
syndrome, as well as a preclinical program for Pompe disease. Our
proprietary plato® gene therapy platform is scalable for planned
global commercialization. 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 forward-looking terminology such as
“aims,” “anticipates,” “believes,” “continue,” “could,” “designed
to,” “estimates,” “expects,” “forecasts,” “goal,” “intends,” “may,”
“plans,” “possible,” “potential,” “predicts,” “projects,” “seeks,”
“strives,” “should,” “will,” and similar expressions or the
negative of these terms. These forward-looking statements include,
without limitation, statements regarding our business strategy for
and the potential therapeutic benefits of our current and
prospective preclinical and clinical 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,
preclinical, compassionate use or clinical trial results, product
approvals and regulatory pathways, the timing of patient
recruitment and enrollment activities, our expectations with
respect to our plans with collaborators, 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, statements
regarding a leadership transition including the appointment of an
interim CEO and our search to identify a permanent CEO, 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, needs for additional financing, and ability to
continue as a going concern including the risk that additional
funding may not be available on acceptable terms or at all and that
failure to obtain capital when needed may force us to delay, limit
or terminate our product development efforts or other operations,
risks relating to our identification and pursuit of any strategic
opportunities with respect to one or more of our programs, our
technology or our plato® platform, 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)
March 31,
December 31,
2023
2022
Cash and cash equivalents
$
72,326
$
92,563
Prepaid expenses and other current
assets
4,925
7,112
Property and equipment, net
2,574
2,894
Operating lease assets
2,857
1,057
Other assets
323
323
Total assets
$
83,005
$
103,949
Accounts payable
$
591
$
384
Accrued expenses and other current
liabilities
11,081
11,732
Note payable, net of discount
15,356
15,276
Operating lease liabilities
2,979
1,187
Total liabilities
30,007
28,579
Total stockholders’ equity
52,998
75,370
Total liabilities and stockholders’
equity
$
83,005
$
103,949
CONDENSED CONSOLIDATED
STATEMENTS OF OPERATIONS
(In thousands, except per
share data)
(Unaudited)
Three Months Ended
March 31,
2023
2022
Operating expenses:
Research and development
$
17,333
$
19,253
General and administrative
7,887
10,165
Total operating expenses
25,220
29,418
Loss from operations
(25,220)
(29,418)
Other income (expense), net
263
(415)
Net loss
$
(24,957)
$
(29,833)
Net loss per share — basic and diluted
$
(0.57)
$
(0.68)
Weighted-average number of common shares
outstanding — basic and diluted
44,037
43,695
____________________________ 1 Collaborator-sponsored Phase 1/2
clinical trial of AVR-RD-04 is funded in part by grants to UCSD
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/20230511005197/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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