All patients remain off oral cysteamine up to
36 months post gene therapy
Sustained engraftment and durable reduction in
leukocyte cystine levels across all patients
Received positive regulatory feedback from US
and UK agencies
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene
therapy company working to free people from a lifetime of genetic
disease, today announced follow-up data demonstrating a durable
treatment effect across key measures out to 36 months from a
collaborator-sponsored Phase 1/2 clinical trial1 evaluating an
investigational gene therapy for the treatment of cystinosis. These
data are being presented at the 26th Annual Meeting of the American
Society of Gene and Cell Therapy (ASGCT) in Los Angeles,
California, on May 18, 2023.
Cystinosis is a rare, progressive disease with a high treatment
burden and unmet need. The fully enrolled Phase 1/2 clinical trial
is monitoring long-term safety and efficacy in six adult patients
affected by the most severe and common form of cystinosis who
previously had been treated with the standard of care (SOC),
cysteamine. The patients’ own hematopoietic stem cells (HSCs) were
genetically modified to express a functional version of cystinosin,
the protein that is deficient in people living with cystinosis.
Preliminary data suggest that post-gene therapy, functional
cystinosin is produced throughout the body as evidenced by clinical
measures in multiple tissues, including the eyes, skin and
gastrointestinal mucosa, as well as by neurocognitive tests
suggestive of activity in the central nervous system. No adverse
events (AEs) related to the drug product or serious adverse events
have been reported to date.
“These data show that genetically modifying a patient’s own HSCs
has the potential to restore functional cystinosin and systemically
reduce the accumulation of cystine, laying the foundation for a
registration-enabling clinical trial,” said AVROBIO Chief Medical
Officer Essra Ridha, M.D., MRCP, FFPM. “We are excited about moving
this investigational gene therapy closer to patients.”
In addition to the data presented, the Company also announced
positive and productive meetings with the U.K. Medicines and
Healthcare products Regulatory Agency (MHRA) and U.S. Food and Drug
Administration (FDA) designed to align on regulatory paths and
obtain feedback on this program.
Data show investigational HSC gene therapy durably and
systemically impacts neurocognitive measures and reduces cystine
levels in the blood, and crystal accumulation in the skin and
gastrointestinal mucosa
Follow-up data suggest that after receiving HSC gene therapy,
patients can produce functional cystinosin protein throughout the
body. As a result, leukocyte cystine levels in the blood have
decreased below baseline in all six patients, and stabilized up to
36 months out from treatment. Skin and gastrointestinal mucosa
biopsies reveal a decline in tissue cystine crystals below baseline
in the first four patients, who have been observed for at least 12
months, with two patients observed up to 24 months.
Patients with cystinosis typically do not see an improvement in
visual spatial or visual motor function over time in standardized
tests evaluating the ability of the brain to interpret and
translate visual information into an exact motor response. The
first four patients treated with gene therapy have shown an
improvement or stabilization of scores on the Beery – Buktenica
Developmental Test of visual motor integration, up to 36 months
out, suggesting a potential impact on the neuropathology of the
disease.
These data represent an extension of trends that have previously
been measured, confirming the durability of the treatment effect up
to 36 months.
Safety and tolerability profile remains strong
Preliminary data from this trial suggest that this HSC gene
therapy is well tolerated, with no AEs related to the drug product
to date. All AEs were related to myeloablative conditioning, stem
cell mobilization, the underlying disease, co-morbid or
pre-existing conditions. The majority of AEs were mild or moderate
and resolved without clinical sequelae.
An oral presentation by Dr. Cherqui on these data, “Phase 1/2
Clinical Trial of Autologous Hematopoietic Stem and Progenitor Cell
Gene Therapy for Cystinosis,” will occur today at 3:45 PM PT in the
session Metabolic, Storage, Endocrine, Liver and Gastrointestinal
Disease II of the ASGCT Annual Meeting. Further details on the
Phase 1/2 trial (NCT03897361) are available on
clinicaltrials.gov.
About cystinosis
Cystinosis is a rare, progressive disease marked by the
accumulation of cystine in cellular organelles known as lysosomes.
This buildup causes progressive organ damage and debilitating
corneal damage, swallowing dysfunction, chronic kidney disease
leading to end-stage renal disease and muscle wasting leading to a
shortened lifespan. Currently, more than 90% of treated cystinosis
patients require a renal transplant in the second or third decade
of life. The current standard of care for cystinosis is cysteamine,
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 complications,
which may be difficult to tolerate.
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 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
product candidates, including our AVR-RD-04 investigational gene
therapy for the treatment of cystinosis, regulatory pathways,
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
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, including AVR-RD-04
for the treatment of cystinosis, 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, 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.
_____________________________ 1The collaborator-sponsored Phase
1/2 clinical trial for cystinosis 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).
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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
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