Orchard Therapeutics, recently acquired by Kyowa Kirin with the
goal of accelerating the delivery of new gene therapies to patients
around the globe, today announced four oral and four poster
presentations from across its hematopoietic stem cell (HSC) gene
therapy platform will be featured at the 27th Annual Meeting of the
American Society of Gene and Cell Therapy (ASGCT) taking place May
7-11, 2024, in Baltimore.
Featured data include several accepted abstracts
and an Oral Presidential Symposium supporting the safety and
efficacy of atidarsagene autotemcel (formerly OTL-200 which was
recently approved as Lenmeldy™ in the U.S. and is marketed as
Libmeldy® in Europe), as well as three presentations detailing
neurological, skeletal, and other clinical outcomes from a
proof-of-concept (PoC) study of investigational OTL-203 in the
Hurler subtype of mucopolysaccharidosis type I (MPS-IH). In
addition, Orchard Therapeutics will give an invited oral
presentation highlighting the potential of OTL-104, a pre-clinical
HSC gene therapy developed by its in-house research team, to
address a severe and treatment refractory form of Crohn’s
disease.
“Our presentations at ASGCT add to the
compendium of evidence supporting the transformative impact and
broad applicability of our approach,” said Leslie Meltzer, Ph.D.,
chief medical officer of Orchard Therapeutics. “In particular, the
Presidential Symposium highlighting the long-term follow-up data in
MLD and the invited oral presentation spotlighting our early
pre-clinical pipeline continue to demonstrate the scientific
interest generated by our platform and underscore our commitment to
leveraging insights gleaned from our late-stage portfolio to inform
the development strategy and indication prioritization of our
earlier-stage programs.”
Details of the oral presentations are follows
(all times in EDT):
- Title: Hematopoietic Stem Cell Gene
Therapy for Hurler Syndrome: Interim Skeletal Outcome and Skeletal
Cross-correction MechanismsDate/Time: Tuesday, May 7 at 1:30
p.m.Presenter: Maria Ester Bernardo
- Title: Atidarsagene autotemcel
(Hematopoietic Stem Cell Gene Therapy) Preserves Cognitive and
Motor Development in Metachromatic Leukodystrophy with up to 12
Years Follow-up (Oral Presidential Symposium)Date/Time: Wednesday,
May 8 at 11:15 a.m.Presenter: Alessandro Aiuti
- Title: Restoring Macrophage Immune
Functions by Transplantation of Gene-modified HSCs: a Therapeutic
Approach to NOD2 Crohn’s Disease (Invited Oral
Presentation)Date/Time: Thursday, May 9 at 9:18 a.m.Presenter:
Pervinder Sagoo
- Title: Somatic Mutation Tracking in
Hematopoietic Stem Cell Gene Therapy Reveals Absence of Clonal
HematopoiesisDate/Time: Saturday, May 11 at 11:45 a.m.Presenter:
Francesco Gazzo
Details of the poster presentations are as
follows (all times in EDT):
- Title: Development of an Ex Vivo
Hematopoietic Stem Cell Gene Therapy for Frontotemporal Dementia
(FTD)Date/Time: Thursday, May 9 from noon to 1:30 p.m. and from
5:30 to 7:00 p.m.Presenter: Yuri CiervoPoster #1136
- Title: Lentiviral Hematopoietic
Stem Cell Gene Therapy for Late Juvenile Metachromatic
leukodystrophyDate/Time: Friday, May 10 from noon to 1:30 p.m. and
from 5:30 to 7:00 p.m.Presenter: Valeria CalbiPoster #1905
- Title: Non-neurological,
Non-Skeletal Outcomes After Autologous Hematopoietic Stem Cell Gene
therapy in Hurler Patients: Retrospective Comparison with
Allogeneic Hematopoietic Stem Cell TransplantationDate/Time:
Friday, May 10 from noon to 1:30 p.m. and from 5:30 to 7:00
p.m.Presenter: Maria Ester BernardoPoster #1904
- Title: Interim Analysis on
Neurological Outcomes in Hurler Syndrome Patients Treated with
Autologous Ex Vivo Hematopoietic Stem Cell Gene TherapyDate/Time:
Friday, May 10 from noon to 1:30 p.m. and from 5:30 to 7:00
p.m.Presenter: Maria Ester BernardoPoster #1903
Early skeletal outcomes from OTL-203 PoC
study in MPS-IH published in Science Translational
Medicine
In addition to the data presented at ASGCT,
Orchard’s collaborators at the San Raffaele-Telethon Institute for
Gene Therapy (SR-Tiget) in Milan, Italy, recently published a
detailed analysis of early skeletal measures in eight children with
MPS-IH treated with OTL-203 in the PoC study which continue to show
improved clinical, functional, and radiological outcomes with a
median follow-up of 3.78 years.
The manuscript, titled, “Early skeletal outcomes
after hematopoietic stem and progenitor cell gene therapy for
Hurler syndrome,” was published in the May 1, 2024 issue of Science
Translational Medicine.
About LenmeldyLenmeldy™
(atidarsagene autotemcel), formerly known as OTL-200, is the only
approved therapy in the U.S. for the treatment of children
with pre-symptomatic late infantile (PSLI), pre-symptomatic early
juvenile (PSEJ) or early-symptomatic early juvenile (ESEJ)
metachromatic leukodystrophy (MLD).
For additional details about Lenmeldy, please
refer to the full Prescribing Information.
In Europe, Lenmeldy is known as Libmeldy®, where
it has been approved by the European Commission (EC), UK Medicines
and Healthcare products Regulatory Agency (MHRA), and Swiss Agency
for Therapeutic Products (Swissmedic). For more information about
Libmeldy, please see the Summary of Product Characteristics
(SmPC) available on the EMA website.
The program was originated by and developed in
partnership with the San Raffaele-Telethon Institute for Gene
Therapy (SR-Tiget) in Milan, Italy.
INDICATION
LENMELDYTM (atidarsagene autotemcel) is an autologous
hematopoietic stem cell-based gene therapy indicated for the
treatment of children with pre-symptomatic late infantile (PSLI),
pre-symptomatic early juvenile (PSEJ), or early symptomatic early
juvenile (ESEJ) metachromatic leukodystrophy (MLD).
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS
Thrombosis and Thromboembolic Events:
Treatment with LENMELDY may increase the risk of
thrombosis and thromboembolic events. A child with PSEJ MLD died
after experiencing a left hemisphere cerebral infarction secondary
to a thrombotic event in a large blood vessel approximately 1 year
after treatment with LENMELDY. Evaluate the risk factors for
thrombosis prior to and after LENMELDY infusion according to best
clinical practice.
Encephalitis:Treatment with LENMELDY may
increase the risk of encephalitis. A child with ESEJ developed a
serious event of encephalitis after treatment with LENMELDY. The
etiology of this event is unclear but attribution to LENMELDY
cannot be ruled out. Treatment with LENMELDY may trigger a
relapsing-remitting pattern of disease progression. No other events
related to encephalitis have been reported during the clinical
development of LENMELDY. Monitor children for signs or symptoms of
encephalitis after LENMELDY treatment.
Serious Infection:In the period between start
of conditioning and within 1 year after LENMELDY treatment, severe
Grade 3 infections occurred in 39% of all children (21% bacterial,
5% viral, 5% bacterial and viral or bacterial and fungal, and 8%
unspecified). Grade 3 febrile neutropenia developed within 1
month after LENMELDY infusion in 82% of children. In the event of
febrile neutropenia, monitor for signs and symptoms of infection
and manage with broad-spectrum antibiotics, fluids, and other
supportive care as medically indicated. Monitor children for signs
and symptoms of infection after myeloablative conditioning and
LENMELDY infusion and treat appropriately. Administer prophylactic
antimicrobials according to best clinical practice.
Veno-Occlusive Disease:
Three children (8%) treated in clinical trials
of LENMELDY developed veno-occlusive disease (VOD) with one Grade 4
SAE and two Grade 3 AEs. None of these three events met Hy’s Law
criteria. Monitor children for signs and symptoms of VOD including
liver function tests in all children during the first month after
LENMELDY infusion. Consider prophylaxis for VOD with
anti-thrombotic agents based on risk factors for VOD and best
clinical practice.
Delayed Platelet Engraftment (DPE):DPE has been
observed with LENMELDY treatment. Bleeding risk is increased prior
to platelet engraftment and may continue after engraftment in
children with prolonged thrombocytopenia. In clinical trials of
LENMELDY, 4 (10%) children had delayed platelet engraftment after
day 60 (range day 67-109), with 3 children requiring platelet
transfusions until engraftment occurred. Patients should be
informed of the risk of bleeding until platelet recovery has been
achieved. Monitor patients for thrombocytopenia and bleeding until
platelet engraftment and recovery are achieved.
Neutrophil Engraftment Failure: There is a
potential risk of neutrophil engraftment failure after treatment
with LENMELDY. Monitor neutrophil counts until engraftment has been
achieved. If neutrophil engraftment failure occurs in a child
treated with LENMELDY, provide rescue treatment with the
unmanipulated back-up collection of CD34+ cells.
Insertional Oncogenesis: There is a potential
risk of LVV-mediated insertional oncogenesis after treatment with
LENMELDY. Children treated with LENMELDY may develop hematologic
malignancies and should be monitored life-long. Monitor for
hematologic malignancies with a complete blood count (with
differential) annually and integration site analysis as warranted
for at least 15 years after treatment with LENMELDY. In the event
that a malignancy occurs, contact Orchard Therapeutics at
1-888-878-0185 for reporting and to obtain instructions on
collection of samples for testing.
Hypersensitivity Reactions: The dimethyl
sulfoxide (DMSO) in LENMELDY may cause hypersensitivity reactions,
including anaphylaxis which is potentially life-threatening and
requires immediate intervention. Hypersensitivity including
anaphylaxis can occur in children with and without prior exposure
to DSMO. Monitor for hypersensitivity reactions during infusion and
after infusion.
Anti-Retroviral Use: Children should not
take prophylactic HIV anti-retroviral medications for at least one
month prior to mobilization, or for the expected duration of time
needed for the elimination of the medications. Anti-retroviral
medications may interfere with the manufacturing of LENMELDY. If a
child requires antiretrovirals for HIV prophylaxis, initiation of
LENMELDY treatment should be delayed until confirmation of a
negative test for HIV.
Interference With Serology Testing:
Due to the likelihood of a false-positive test for HIV, children
who have received LENMELDY should not be screened for HIV infection
using a PCR-based assay.
USE IN SPECIFIC POPULATIONS
Females and Males of Reproductive Potential
Pregnancy TestingAs a precautionary measure, a negative serum
pregnancy test must be confirmed prior to the start of
mobilization, and reconfirmed prior to conditioning procedures, and
before administration of LENMELDY in females of childbearing
potential.
Contraception
Consult the Prescribing Information of the mobilization and
conditioning agents for information on the need for effective
contraception. Males capable of fathering a child and females of
childbearing age should use an effective method of contraception
from start of mobilization through at least 6 months after
administration of LENMELDY.
InfertilityThere are no data on the effects of LENMELDY on
fertility.
Data are available on the risk of infertility with myeloablative
conditioning. In clinical trials of LENMELDY, seven children (50%
of females) developed ovarian failure. Advise children of the
option to cryopreserve semen or ova before treatment, if
appropriate.
For additional safety information, please see the full
Prescribing Information.
About Orchard
TherapeuticsOrchard Therapeutics, a Kyowa Kirin company,
is a global gene therapy leader focused on ending the devastation
caused by genetic and other severe diseases by discovering,
developing, and commercializing new treatments that tap into the
curative potential of hematopoietic stem cell (HSC) gene therapy.
In this approach, a patient’s own blood stem cells are genetically
modified outside of the body and then reinserted, with the goal of
correcting the underlying cause of disease with a single
treatment.
Founded in 2015, Orchard’s roots go back to some
of the first research and clinical developments involving HSC gene
therapy. Our team has played a central role in the evolution of
this technology from a promising scientific idea to a potentially
life-transforming reality. Today, Orchard is advancing a pipeline
of HSC gene therapies designed to address serious diseases where
the burden is immense for patients, families and society and
current treatment options are limited or do not exist.
For more information, please
visit www.orchard-tx.com.
About Kyowa KirinKyowa Kirin
aims to discover novel medicines with life-changing value. As a
Japan-based Global Specialty Pharmaceutical Company, we have
invested in drug discovery and biotechnology innovation for more
than 70 years and are currently working to engineer the next
generation of antibodies and cell and gene therapies with
the potential to help patients affected by a severe or
rare disease. A shared commitment to our values, to sustainable
growth, and to making people smile unites us across our four
regions – Japan, Asia Pacific, North America, and
EMEA/International. You can learn more about the business of Kyowa
Kirin at www.kyowakirin.com.
Contact
Benjamin Navon
+1 857-248-9454
Benjamin.Navon@orchard-tx.com
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