First five patients in Phase 1/2 trial show
systemic gene therapy effect across multiple tissues evaluated,
including eyes, skin, gastrointestinal mucosa and neurocognitive
system
AVROBIO planning to initiate company-sponsored
trial in 2023
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene
therapy company working to free people from a lifetime of genetic
disease, today announced that the sixth and final patient has been
dosed in the collaborator-sponsored, Phase 1/2 clinical trial of an
investigational gene therapy for the treatment of cystinosis.
Cystinosis is a life-threatening disease that causes progressive
multi-organ damage, including early, acute kidney disease
progressing to end-stage kidney disease.
The Phase 1/2 clinical trial for cystinosis is evaluating the
safety and efficacy of this hematopoietic stem cell (HSC) gene
therapy approach in adult patients affected by the most severe and
common form of cystinosis who previously had been treated with the
current standard of care, cysteamine. Pharmacodynamic and clinical
efficacy endpoints include evaluation of the effect of treatment on
leukocyte cystine levels, kidney function, corneal cystine crystal
accumulation, muscle strength, as well as measures of visual motor
integration, visual perception and motor coordination. The first
patient was dosed in 2019 in this University of California San
Diego (UCSD) trial, 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).
“Completing the dosing of the first and only gene therapy trial
for cystinosis represents a major milestone for a patient community
living with a devastating genetic disease. Unmet medical needs
impact the lives of patients and their family members every day,”
said Stephanie Cherqui, Ph.D., lead study investigator and
associate professor of Pediatrics at UCSD. “To date, the results
from the trial show the potential of this investigational gene
therapy to stabilize or reduce the impact of cystinosis on
different tissues throughout the body with a one-time dose.”
Preliminary data from this trial suggest that this approach is
well tolerated, with no adverse events (AEs) related to the drug
product reported to date. All AEs reported were related to
myeloablative conditioning, study procedures, the underlying
disease or pre-existing conditions. The majority of AEs were mild
or moderate and resolved without clinical sequelae. Clinical data
to date indicate this investigational gene therapy approach
provides therapeutic effect in multiple tissues evaluated,
including the eyes, skin, gastrointestinal mucosa and the
neurocognitive system.
“With proof-of-concept demonstrated, we continue to lay the
groundwork for an AVROBIO-sponsored clinical trial planned to begin
in 2023,” said AVROBIO Chief Medical Officer, Essra Ridha, M.D.,
MRCP, FFPM. “We look forward to our interactions with regulators on
our clinical and Chemistry Manufacturing and Controls (CMC)
strategy.”
About AVR-RD-04 AVR-RD-04 is designed to genetically
modify patients’ own HSCs to express the gene encoding cystinosin,
the protein that is critically deficient in people living with
cystinosis. AVR-RD-04 has received Rare Pediatric Disease
Designation and Fast Track Designation from the U.S. Food and Drug
Administration (FDA) and Orphan Drug Designation from FDA and the
European Medicines Agency. AVROBIO is planning for regulatory
agency interactions to discuss clinical development and regulatory
strategy, with the intent of initiating a company-sponsored
clinical trial in 2023.
About cystinosis Cystinosis, a rare, progressive disease
that is believed to impact thousands of patients worldwide,
including approximately 1,600 patients in the U.S., Europe and
Japan, is marked by the accumulation of cystine in cellular
organelles known as lysosomes. Untreated cystinosis is fatal at an
early age. The current standard of care for cystinosis, a treatment
regimen that can require dozens of pills per day, does not prevent
overall disease progression and carries side effects, such as
breath and body odor and gastrointestinal symptoms, which can
impede compliance. More than 90% of treated cystinosis patients
require a kidney transplant in the second or third decade of
life.
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 Statements 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 preclinical and clinical
product candidates, including AVR-RD-04 for the treatment of
cystinosis, the potential benefits and incentives provided by FDA’s
rare pediatric disease designation for AVR-RD-04, the design,
commencement, enrollment and timing of planned clinical trials,
preclinical or clinical trial results, product approvals and
regulatory pathways, our plans and expectations with respect to
interactions with regulatory agencies, anticipated benefits of our
gene therapy platform including potential impact on our
commercialization activities, timing and likelihood of success, the
expected benefits and results of our implementation of the plato®
platform in our clinical trials and gene therapy programs, and the
expected safety profile of our preclinical and investigational gene
therapies. 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, including in
collaborator-sponsored 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, 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 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.
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version on businesswire.com: https://www.businesswire.com/news/home/20221025005356/en/
Investors: Christopher F. Brinzey ICR Westwicke
339-970-2843 chris.brinzey@westwicke.com
Media: Kit Rodophele Ten Bridge Communications
617-999-9620 krodophele@tenbridgecommunications.com
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