Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy
leader, today announced additional interim data from an ongoing
proof-of-concept clinical trial evaluating the safety and efficacy
of OTL-203, an investigational ex vivo autologous
hematopoietic stem cell (HSC) gene therapy in development for the
treatment of mucopolysaccharidosis type I (MPS-I) at the San
Raffaele Telethon Institute for Gene Therapy (SR-Tiget)
in Milan, Italy. The readout from the primary endpoint at one
year of follow-up is expected in 2021. Today's results are being
shared virtually in an invited oral presentation at the 46th Annual
Meeting of the European Society for Blood and Bone Marrow
Transplantation (EBMT).
“We continue to see encouraging data from the ongoing clinical
trial in MPS-I, including promising preliminary clinical effects on
motor development, acquisition of cognitive skilIs and growth in
the first two patients that were treated now 1.5 and 2 years ago,
respectively. Additionally, new preliminary analyses of
radiological outcome measures suggest that treatment with OTL-203
leads to stabilization or improvement in disease-related
neurological abnormalities, as measured by brain and spine MRI,
which we look to confirm with longer follow-up,” said Maria
Ester Bernardo, M.D., Ph.D., principal investigator at SR-Tiget.
"These data, taken together with those from clinical studies of HSC
gene therapy for other metabolic disorders and leukodystrophies,
support the potential for this therapeutic approach to correct a
wide spectrum of multisystemic manifestations of the disease,
bringing clinically meaningful benefits for patients.”
Interim Study Results Eight patients with the
severe Hurler subtype of MPS-I had been treated with OTL-203 in the
ongoing proof-of-concept study, which completed enrollment in
December 2019. As of July 2020, all patients had been followed for
a minimum of six months, with the longest follow-up extending out
to 24 months. Treatment with OTL-203 was generally well-tolerated
with a safety profile consistent with the selected conditioning
regimen. Consistent with previous analyses, treatment across all
eight patients continued to demonstrate:
- Rapid hematologic reconstitution, with neutrophil and platelet
engraftment within 21 days following treatment.
- Biological efficacy established by sustained supranormal
alpha-L-iduronidase (IDUA) enzyme expression in peripheral blood
for all patients within 3 months post-gene therapy and up to 18
months in the first treated patient.
- Metabolic correction as measured by reduction in
glycosaminoglycan (GAG) levels in urine achieved in all patients by
6 months post-gene therapy, with sustained correction out to 18
months in the first treated patient.
- Further results for the first two treated patients
demonstrated:
- Rapid metabolic correction of GAG levels in the cerebral spinal
fluid (CSF), reflecting restoration of IDUA enzyme expression in
the central nervous system.
- Improved motor function and acquisition of cognitive and
language skills.
- Stabilization or improvement in white matter abnormalities,
cervical canal stenosis, kyphosis and vertebra deformity, as
measured by brain and spine MRI scores.
- Continued growth progression above the 50th percentile of
normal.
- Improved range of motion (an indicator of joint
stiffness).
“We continue to see positive trends in all biomarker and
clinical measures as we follow patients in the OTL-203 proof of
concept study for longer periods of time,” said Bobby Gaspar,
M.D., Ph.D., chief executive officer of Orchard. “With a growing
amount of data to support advancing this program, we have recently
convened a panel of disease experts to develop a design for a
registrational trial that we intend to take to the regulators in
advance of initiating the study in 2021 and ultimately progressing
towards commercialization.”
About OTL-203 and MPS-I
Mucopolysaccharidosis type I (MPS-I) is a rare, inherited
neurometabolic disease caused by a deficiency of the
alpha-L-iduronidase (IDUA) lysosomal enzyme, which is required to
break down sugar molecules called glycosaminoglycans (also known as
GAGs). The accumulation of GAGs across multiple organ systems
results in symptoms including neurocognitive impairment, skeletal
deformity, loss of vision and hearing, and cardiovascular and
pulmonary complications. MPS-I occurs at an overall estimated
frequency of one in every 100,000 live births. There are three
subtypes of MPS-I; approximately 60 percent of children born with
MPS-I have the most severe subtype, called Hurler syndrome, and
rarely live past the age of 10 when untreated.
Treatment options for MPS-I include hematopoietic stem cell
transplant and chronic enzyme replacement therapy, both of which
have significant limitations. Though early intervention with enzyme
replacement therapy has been shown to delay or prevent some
clinical features of the condition, it has only limited efficacy on
neurological symptoms. OTL-203 is an investigational ex
vivo autologous hematopoietic stem cell gene therapy being
studied for the treatment of MPS-I. Orchard was granted an
exclusive worldwide license to intellectual property rights to
research, develop, manufacture and commercialize the gene therapy
program for the treatment of MPS-I developed by the San
Raffaele Telethon Institute for Gene Therapy in Milan,
Italy.
About Orchard
Orchard Therapeutics is a global gene therapy leader
dedicated to transforming the lives of people affected by rare
diseases through the development of innovative, potentially
curative gene therapies. Our ex vivo autologous gene
therapy approach harnesses the power of genetically modified blood
stem cells and seeks to correct the underlying cause of disease in
a single administration. In 2018, Orchard acquired GSK’s rare
disease gene therapy portfolio, which originated from a pioneering
collaboration between GSK and the San Raffaele Telethon
Institute for Gene Therapy in Milan, Italy. Orchard now has
one of the deepest and most advanced gene therapy product candidate
pipelines in the industry spanning multiple therapeutic areas where
the disease burden on children, families and caregivers is immense
and current treatment options are limited or do not exist.
Orchard has its global headquarters
in London and U.S. headquarters in Boston.
For more information, please visit www.orchard-tx.com, and
follow us on Twitter and LinkedIn.
Availability of Other Information About
Orchard
Investors and others should note that Orchard communicates with
its investors and the public using the company website
(www.orchard-tx.com), the investor relations website
(ir.orchard-tx.com), and on social media
(Twitter and LinkedIn), including but not limited to
investor presentations and investor fact sheets, U.S.
Securities and Exchange Commission filings, press releases,
public conference calls and webcasts. The information that Orchard
posts on these channels and websites could be deemed to be material
information. As a result, Orchard encourages investors, the media,
and others interested in Orchard to review the information that is
posted on these channels, including the investor relations website,
on a regular basis. This list of channels may be updated from time
to time on Orchard’s investor relations website and may include
additional social media channels. The contents of Orchard’s website
or these channels, or any other website that may be accessed from
its website or these channels, shall not be deemed incorporated by
reference in any filing under the Securities Act of 1933.
Forward-Looking Statements
This press release contains certain forward-looking statements
about Orchard’s strategy, future plans and prospects, which are
made pursuant to the safe harbor provisions of the Private
Securities Litigation Reform Act of 1995. Such forward-looking
statements may be identified by words such as “anticipates,”
“believes,” “expects,” “plans,” “intends,” “projects,” and “future”
or similar expressions that are intended to identify
forward-looking statements. Forward-looking statements
include express or implied statements relating to, among other
things, Orchard’s business strategy and goals, the therapeutic
potential of Orchard’s product candidates, including the product
candidates referred to in this release, Orchard’s expectations
regarding the timing of clinical trials for its product candidates,
including the product candidates referred to in this release, the
timing of interactions with regulators and regulatory submissions
related to ongoing and new clinical trials for its product
candidates, the timing of announcement of clinical data for its
product candidates, and the likelihood that such data will be
positive and support further clinical development and regulatory
approval of these product candidates. These statements are
neither promises nor guarantees and are subject to a variety of
risks and uncertainties, many of which are beyond Orchard’s
control, which could cause actual results to differ materially from
those contemplated in these forward-looking statements. In
particular, these risks and uncertainties include, without
limitation: the severity of the impact of the COVID-19 pandemic on
Orchard’s business, including on clinical development, its supply
chain and commercial programs; the risk that Orchard will not
realize the anticipated benefits of its new strategic plan or the
expected cash savings associated with such plan; the risk that any
one or more of Orchard’s product candidates, including the product
candidates referred to in this release, will not be successfully
developed, approved or commercialized; the risk of cessation or
delay of any of Orchard’s ongoing or planned clinical trials; the
risk that Orchard may not successfully recruit or enroll a
sufficient number of patients for its clinical trials; the risk
that prior results, such as signals of safety, activity or
durability of effect, observed from preclinical studies or clinical
trials will not be replicated or will not continue in ongoing or
future studies or trials involving Orchard’s product candidates or
that long-term adverse safety findings may be discovered; the delay
of any of Orchard’s regulatory submissions; the failure to obtain
marketing approval from the applicable regulatory authorities for
any of Orchard’s product candidates or the receipt of restricted
marketing approvals; and the risk of delays in Orchard’s ability to
commercialize its product candidates, if approved. Given these
uncertainties, the reader is advised not to place any undue
reliance on such forward-looking statements.
Other risks and uncertainties faced by Orchard include those
identified under the heading "Risk Factors" in Orchard’s quarterly
report on Form 10-Q for the quarter ended June 30, 2020, as
filed with the U.S. Securities and Exchange
Commission (SEC), as well as subsequent filings and reports
filed with the SEC. The forward-looking statements contained
in this press release reflect Orchard’s views as of the date
hereof, and Orchard does not assume and specifically disclaims any
obligation to publicly update or revise any forward-looking
statements, whether as a result of new information, future events
or otherwise, except as may be required by law.
Contacts
InvestorsRenee LeckDirector, Investor Relations
+1 862-242-0764Renee.Leck@orchard-tx.com
Media Molly Cameron Manager, Corporate
Communications +1 978-339-3378 media@orchard-tx.com
Orchard Therapeutics (NASDAQ:ORTX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Orchard Therapeutics (NASDAQ:ORTX)
Historical Stock Chart
From Apr 2023 to Apr 2024