Fabry disease program to be deprioritized,
shifting focus to other clinical-stage programs in lysosomal
disorder pipeline
Data updates for cystinosis and Gaucher disease
type 1 programs planned for 1H 2022, with regulatory interactions
anticipated across multiple programs in 2022
Cash runway to be extended into first quarter
of 2024
AVROBIO, Inc. (Nasdaq: AVRO), a leading clinical-stage gene
therapy company with a shared purpose to free people from a
lifetime of genetic disease, today announced that it is shifting
its portfolio priorities to focus on other clinical-stage programs
and extending its cash runway into the first quarter of 2024. The
company is deprioritizing its Fabry disease program due to several
factors, including new clinical data showing variable engraftment
patterns from the five most recently dosed Phase 2 FAB-GT patients
which would significantly extend the program’s development
timeline, as well as an increasingly challenging market and
regulatory environment for Fabry disease.
"Following steady progress in 2021, we have reset our corporate
priorities and will extend our cash runway to strengthen our
ability to deliver on the promise of our gene therapy programs,”
said Geoff MacKay, president and CEO of AVROBIO. “Powered by our
proprietary plato® gene therapy platform, we will focus our efforts
on moving value driving clinical-stage programs forward in 2022,
with data updates expected for our cystinosis and Gaucher disease
type 1 programs, as well as regulatory interactions anticipated
across multiple programs in our pipeline.
“Previously reported data from 13 patients treated across our
three clinical-stage programs have shown durable engraftment out 9
to 54 months. It is the new data from the five most recently dosed
Phase 2 FAB-GT patients that are discordant with these other data
and show variable engraftment. In addition, the last 12 months have
presented multiple challenging market and regulatory dynamics for
our Fabry disease program, which would now be exacerbated by a
meaningfully extended development timeline,” said MacKay. “We’re
fully aware of the impact this difficult decision has on the
patients and families whom we have had the privilege to get to know
over the years, but we believe deprioritizing and halting
enrollment in our Fabry disease program is the right step forward
for AVROBIO and preserves our ability to continue developing
therapies with the potential to address urgent unmet needs in the
lysosomal disorder community.”
New data from Phase 2 FAB-GT clinical trial show variable
engraftment The aggregated data from the five most recently
dosed FAB-GT patients showed variable engraftment patterns. Data
from three of the five patients showed both a reduction to near
baseline levels in alpha-galactosidase A (AGA) enzyme activity in
leukocytes and plasma, and a reduction in vector copy number (VCN)
in whole blood, potentially suggesting resistance to persistent
engraftment of the genetically modified cells observed at three to
nine months post infusion of AVR-RD-01. (See data slides here)
Based on its investigation, the company believes, due to the
large degree of heterogeneity in Fabry disease, that in some cases
there may be intrinsic resistance to engraftment related to the
unique underlying pathophysiology of untreated Fabry disease,
potentially caused by the persistently stressed vascular
endothelium. The company also has reviewed potential
procedure-related factors and conditioning parameters, including
the possible impact, in the context of untreated Fabry disease, of
a previous clinical trial protocol amendment for the five recently
dosed patients which prolonged the conditioning agent washout
period by up to 48 hours.
“Importantly, the drug product specifications for these five
patients met all release criteria,” said MacKay. “Additionally,
these variable engraftment patterns have not been observed to date
in data from the other nine Fabry disease patients previously dosed
in the Phase 1 trial and under the prior protocol amendments in the
FAB-GT trial, or in data from any patients in our other ongoing
clinical trials.”
Safety data from all nine adult patients dosed in the Phase 2
FAB-GT trial and the five adult patients dosed in the
investigator-sponsored Phase 1 trial show no adverse events (AEs)
or serious adverse events (SAEs) related to drug product AVR-RD-01,
as of the most recent data cut-off date.
The company will stop enrollment for the FAB-GT clinical trial
and continue monitoring the previously dosed patients for a total
of 15 years as required by regulators.
Updated 2022 program guidance Anticipated pipeline
milestones include:
- AVR-RD-04 for cystinosis: Provide an update at the
WORLDSymposium™ 2022 on collaborator-sponsored Phase 1/2 clinical
trial of AVR-RD-04 (CTNS-RD-04),i and plan to engage with
regulatory agencies on a planned Phase 2 company-sponsored clinical
trial
- AVROBIO’s Gaucher disease programs:
- AVR-RD-02 for Gaucher disease type 1: Provide a clinical update
in the first half of 2022
- AVR-RD-06 for Gaucher disease type 3: Engage with regulatory
agencies on a planned Phase 2/3 clinical development strategy for
AVR-RD-06; planning to initiate a clinical trial in 2023
- AVR-RD-05 for Hunter syndrome: Collaborators at the University
of Manchester plan to initiate a collaborator-sponsored Phase 1/2
clinical trial in 2023
- AVR-RD-03 for Pompe disease: Engage with regulatory agencies on
the clinical development strategy for AVR-RD-03; planning to
initiate a clinical trial in 2023
- plato® platform: Continue research collaborations to evaluate
the potential use of monoclonal antibody conditioning agents in
Gaucher disease type 1 trial
As of Sept. 30, 2021, the company had $201 million in cash and
cash equivalents. As a result of the pipeline reprioritization, the
company expects to extend its cash runway into the first quarter of
2024.
About AVROBIO Our vision is to bring personalized gene
therapy to the world. We aim to prevent, halt or reverse disease
throughout the body with a single dose of gene therapy designed to
drive durable expression of therapeutic protein, even in
hard-to-reach tissues and organs including brain, muscle and bone.
AVROBIO’s pipeline is powered by our industry-leading plato® gene
therapy platform, our foundation designed to deliver gene therapy
worldwide. It includes clinical programs in cystinosis and Gaucher
disease type 1, as well as preclinical programs in Gaucher disease
type 3, Hunter syndrome and Pompe disease. We are headquartered in
Cambridge, Mass., with an office in Toronto, Ontario. 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 plans and expectations for
reprioritizing our program pipeline, including the deprioritization
of our Fabry disease clinical program, our business strategy for
and the potential therapeutic benefits of our prospective product
candidates, results of preclinical studies, the design,
commencement, enrollment and timing of ongoing or planned clinical
trials, clinical trial results, product approvals and regulatory
pathways, anticipated regulatory interactions, 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, the
potential use of monoclonal antibody conditioning agents, and our
financial position and cash runway expectations. 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, risks related to regulatory interactions and
intended development pathways 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 on Form 10-Q, 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.
i 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).
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version on businesswire.com: https://www.businesswire.com/news/home/20220104005408/en/
Investor Contact: Christopher F. Brinzey Westwicke, an
ICR Company 339-970-2843 chris.brinzey@westwicke.com
Media Contact: Krystle Gibbs Ten Bridge Communications
krystle@tenbridgecommunications.com 508-479-6358
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