Homology Medicines Announces Presentation of Data Supporting Clinical Programs in MPS II and PKU, including Nonclinical and Patient-Focused Research, at American Society of Human Genetics Meeting
October 20 2021 - 8:30AM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today presentations of nonclinical data and
patient and caregiver feedback that support the Company’s clinical
programs for mucopolysaccharidosis type II (MPS II), or Hunter
syndrome, and phenylketonuria (PKU). During the American Society of
Human Genetics (ASHG) 2021 Virtual Meeting, an oral presentation
included data from Homology’s IND-enabling studies with HMI-203, a
one-time, in vivo investigational gene therapy in development for
the treatment of MPS II in the recently initiated Phase 1 juMPStart
clinical trial. Patient and caregiver feedback on unmet medical
needs in MPS II that informed trial design were also presented.
Additionally, Homology shared results from a nonclinical study that
demonstrated the precision of its nuclease-free, homologous
recombination-based, in vivo gene editing candidate for PKU as the
Company starts its first gene editing trial.
“Our presentations at ASHG show the holistic approach we have
taken to prepare for our Hunter syndrome gene therapy and PKU gene
editing trials, demonstrating our commitment to thoroughly
evaluating the product candidates and the needs of the patient
community ahead of clinical studies,” stated Albert Seymour, Ph.D.,
Chief Scientific Officer of Homology Medicines. “Armed with patient
and caregiver feedback on the unmet medical needs that persist, we
recently initiated our juMPStart trial to evaluate a single-dose of
HMI-203 in adults with Hunter syndrome. Additionally, integration
assays from our in vivo PKU gene editing program demonstrated no
evidence of off-target integration in human hepatocytes in a
xenograft murine model and support our Phase 1 trial.”
In the oral presentation titled, “Long-Term Systemic Expression
and Cross-Correction Ability of HMI-203: Investigational Gene
Therapy Candidate for Mucopolysaccharidosis Type II or Hunter
Syndrome,” data showed that a single I.V. dose of HMI-203 in the
MPS II murine model resulted in the following, through 52 weeks
(end of study):
- Systemic expression of vector genomes, transcripts and
functional I2S enzyme;
- Secretion of active I2S into circulation demonstrating
cross-correction;
- Systemic reduction of disease-relevant biomarkers, including
positive and significant correlation between cerebral
glycosaminoglycan heparin sulfate (GAG-HS) and LAMP1 levels as well
as cerebrospinal fluid (CSF) and cerebral GAG-HS levels; and
- Prevention of progression of craniofacial and hindlimb
deformities.
Also related to HMI-203, “Patient-Focused Drug Development for a
Single Intravenous Dose of HMI-203 Gene Therapy in Adult
Mucopolysaccharidosis (MPS) II, or Hunter Syndrome, Patients”
included qualitative data on unmet medical needs from enzyme
replacement therapy (ERT)-treated adult MPS II patients and/or
their caregivers. They reported:
- Weekly ERT infusions, surgeries and supportive therapies
inadequately address range of motion and mobility, pain, and
hearing loss;
- Burdens associated with ERT and other therapies, including
frequency and duration of treatment, and painful and extended
recoveries;
- High degree of anxiety regarding prognosis, longevity, need for
more invasive surgeries, and financial challenges; and
- Expectations for a potential one-time gene therapy include
ability to maintain current quality of life with ERT
independence.
To support HMI-103, its gene editing candidate for PKU, Homology
also presented, “Genome-Wide Integration Assay For rAAV Mediated
Homologous Recombination (HR) in Human Hepatocytes Demonstrated
Precision of In Vivo Gene Editing Approach.” Using quantitative
molecular methods, including long-read sequencing, in a murine
liver model populated with human hepatocytes, a single I.V. dose of
HMI-103 demonstrated on-target integration into the desired locus
and no evidence of integration into any other genomic location.
For more information, please visit
www.homologymedicines.com/publications.
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 StatementsThis 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,
including without limitation statements regarding our expectations
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 and timing for the release of additional
preclinical and clinical data; our beliefs regarding our
manufacturing capabilities; our position as a leader in the
development of genetic medicines; and our participation in upcoming
presentations and conferences. These statements are neither
promises nor guarantees, but involve known and unknown risks,
uncertainties and other important factors that may cause our actual
results, performance or achievements to be materially different
from any future results, performance or achievements expressed or
implied by the forward-looking statements, including, but not
limited to, the following: the impact of the COVID-19 pandemic on
our business and operations, including our preclinical studies and
clinical trials, and on general economic conditions; we have and
expect to continue to incur significant losses; our need for
additional funding, which may not be available; failure to identify
additional product candidates and develop or commercialize
marketable products; the early stage of our development efforts;
potential unforeseen events during clinical trials could cause
delays or other adverse consequences; risks relating to the
capabilities of our manufacturing facility; risks relating to the
regulatory approval process; interim, topline and preliminary data
may change as more patient data become available, and are subject
to audit and verification procedures that could result in material
changes in the final data; our product candidates may cause serious
adverse side effects; inability to maintain our collaborations, or
the failure of these collaborations; our reliance on third parties;
failure to obtain U.S. or international marketing approval; ongoing
regulatory obligations; effects of significant competition;
unfavorable pricing regulations, third-party reimbursement
practices or healthcare reform initiatives; product liability
lawsuits; failure to attract, retain and motivate qualified
personnel; the possibility of system failures or security breaches;
risks relating to intellectual property and significant costs as a
result of operating as a public company. These and other important
factors discussed under the caption “Risk Factors” in our Quarterly
Report on Form 10-Q for the quarter ended June 30, 2021 and our
other filings with the SEC could cause actual results to differ
materially from those indicated by the forward-looking statements
made in this press release. Any such forward-looking statements
represent management’s estimates as of the date of this press
release. While we may elect to update such forward-looking
statements at some point in the future, we disclaim any obligation
to do so, even if subsequent events cause our views to change.
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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