Homology Medicines Initiates Clinical Trial for HMI-203, a One-Time Investigational Gene Therapy Candidate for Adults with MPS II (Hunter Syndrome)
October 18 2021 - 8:30AM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today that it has initiated the Phase 1 trial
for HMI-203, a one-time, in vivo gene therapy candidate for the
treatment of adults with mucopolysaccharidosis type II (MPS II), or
Hunter syndrome. The juMPStart trial is an open-label,
dose-escalation study evaluating the safety and efficacy of a
single intravenous (I.V.) administration of HMI-203. Hunter
syndrome is a lysosomal storage disorder caused by mutations in the
iduronate 2-sulfatase (IDS) gene leading to absent or deficient I2S
enzymatic activity, which causes toxic lysosomal accumulation of
glycosaminoglycans (GAGs). There are no treatments currently
available that address both the peripheral organ and cognitive
manifestations of the disease, and there remains a high unmet
medical need for patients despite the availability of enzyme
replacement therapy (ERT).
“We are excited to start the Phase 1 trial for HMI-203, our
investigational gene therapy for Hunter syndrome, as our team
remains dedicated to improving outcomes for the patient community
since our prior work developing ERT,” said Arthur Tzianabos, Ph.D.,
President and Chief Executive Officer of Homology Medicines. “With
today’s milestone, we have also accomplished our goal of having
three clinical programs by the end of 2021, with our gene therapy
and gene editing clinical trials for PKU and the initiation of the
juMPStart trial for Hunter syndrome. We are executing on this and
our other programs, and we look forward to our continued
collaboration with the MPS II community.”
The juMPStart trial is expected to enroll up to 9 male patients
ages 18-30 years old who have been diagnosed with Hunter syndrome
and are currently receiving ERT. In addition to safety endpoints,
the trial plans to measure plasma I2S activity, urinary GAG levels
and other peripheral disease manifestations. The Phase 1
dose-escalation portion of the trial is designed to evaluate three
doses of HMI-203 to potentially determine the optimal dose(s) for a
future trial.
“I am pleased to work with the Homology team, as we both have a
long-held and shared commitment to the MPS II community and a focus
on bringing forward a one-time gene therapy to address the
underlying cause of this disease,” said Joseph Muenzer, M.D.,
Ph.D., Bryson Distinguished Professor in Pediatric Genetics at the
University of North Carolina at Chapel Hill. “While weekly
intravenous ERT has made a huge difference for the MPS II
community, it is very time-consuming and there remains the need to
address the brain disease in the severe or neuronopathic form of
MPS II. I am optimistic that Homology’s gene therapy approach for
MPS II could make a significant impact on all aspects of the
disease, including the peripheral manifestations, as well as the
day-to-day lives of patients and their families, and I look forward
to working with the Homology team and all of the investigators in
the HMI-203 clinical trial to evaluate this approach.”
Albert Seymour, Ph.D., Chief Scientific Officer of Homology
Medicines, added, “We are pleased to be transitioning HMI-203 into
the clinic for patients, building on the positive preclinical data
we presented at scientific conferences, including the upcoming ASHG
meeting. These data showed long-term expression of I2S in multiple
organs, sustained secretion in the serum, reduced GAGs in all
tissues tested as well as the cerebrospinal fluid, and phenotypic
correction in the Hunter disease murine model following a one-time
administration. We appreciated the insights from the Hunter
syndrome community as we designed our clinical program, and we look
forward to continuing our work together as we advance
juMPStart.”
In October 2021, the U.S. Food and Drug Administration (FDA)
cleared the Investigational New Drug (IND) application for HMI-203
for the treatment of Hunter syndrome to proceed.
About HMI-203HMI-203 is a one-time in vivo gene
therapy candidate in development for the treatment of Hunter
syndrome and designed to use one of Homology’s human hematopoietic
stem cell-derived adeno-associated virus vectors (AAVHSCs) to
deliver functional copies of the IDS gene to multiple organs where
there are missing or mutated copies of the gene. HMI-203 is
developed to enable the production of the I2S enzyme that is
responsible for breaking down glycosaminoglycans (GAGs), which
accumulate and cause progressive debilitation and shortened life
expectancy in people with Hunter syndrome.
About Mucopolysaccharidosis Type II (MPS II), Hunter
SyndromeMPS II, or Hunter syndrome, is a rare, X-linked
lysosomal storage disorder caused by mutations in the
iduronate-2-sulfatase (IDS) gene, which is responsible for
producing the I2S enzyme that breaks down large sugar molecules, or
cellular waste, called glycosaminoglycans (GAGs). Severe Hunter
syndrome results in toxic lysosomal accumulation of GAGs that
causes progressive debilitation and decline in intellectual
function. Hunter syndrome occurs in approximately 1 in 100,000 to 1
in 170,000 males, and the severe form leads to life expectancy of
10 to 20 years.
About Homology Medicines, Inc. Homology
Medicines, Inc. is a clinical-stage genetic medicines company
dedicated to transforming the lives of patients suffering from rare
diseases by addressing the underlying cause of the disease. The
Company’s lead clinical program, HMI-102, is an investigational
gene therapy for adults with phenylketonuria (PKU) and additional
programs focus on lysosomal storage disorders including Hunter
syndrome, paroxysmal nocturnal hemoglobinuria (PNH) and other
diseases. Homology’s proprietary platform is designed to utilize
its family of 15 human hematopoietic stem cell-derived
adeno-associated virus vectors (AAVHSCs) to precisely and
efficiently deliver genetic medicines in vivo through a gene
therapy or nuclease-free gene editing modality, as well as to
deliver one-time gene therapy to produce antibodies throughout the
body through the GTx-mAb platform. Homology has a management team
with a successful track record of discovering, developing and
commercializing therapeutics with a focus on rare diseases.
Homology believes its initial clinical data and compelling
preclinical data, scientific and product development expertise,
internal manufacturing capabilities and broad intellectual property
position the Company as a leader in genetic medicines. For more
information, visit www.homologymedicines.com.
Forward-Looking Statements This press release
contains forward-looking statements within the meaning of the
Private Securities Litigation Reform Act of 1995. All statements
contained in this press release that do not relate to matters of
historical fact should be considered forward-looking statements,
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surrounding the potential, safety, efficacy, and regulatory and
clinical progress of our product candidates; the potential of our
gene therapy and gene editing platforms, including our GTx-mAb
platform; our plans to name a development candidate and potential
thereof; our plans and timing for the release of additional
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Company ContactsTheresa McNeelyChief
Communications Officer and Patient
Advocatetmcneely@homologymedicines.com781-301-7277
Media Contact:Cara Mayfield Vice President,
Patient Advocacy and Corporate Communications
cmayfield@homologymedicines.com 781-691-3510
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