Homology Medicines Presents Preclinical Data Supporting Immunosuppression Regimen in Ongoing PKU and Hunter Syndrome Clinical Trials, and Details Optimized MLD Gene Therapy Candidate at the 19th Annual WORLDSymposium™ Meeting
February 22 2023 - 04:01PM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today the presentation of preclinical data that
supports the targeted, prophylactic immunosuppression regimen in
the ongoing pheEDIT gene editing clinical trial in adults with
phenylketonuria (PKU) and juMPStart gene therapy trial in adults
with Hunter syndrome (MPS II). Homology also shared additional
details of the optimized, in vivo gene therapy candidate HMI-204
for metachromatic leukodystrophy (MLD) during the 19th Annual
WORLDSymposium™ Meeting.
“We are pleased to share our work outlining the impact
immunosuppression regimens had on outcomes following AAVHSC dosing
in NHPs, as it contributes to a key area of research in the gene
therapy and gene editing field,” stated Albert Seymour, Ph.D.,
President and Chief Executive Officer of Homology Medicines. “In
this study, we evaluated multiple regimens, including the
combination of a T-cell inhibitor and steroid, and reported that
the combination was the most effective in reducing B- and T-cell
activity, reducing neutralizing antibody formation and improving
gene expression. This targeted, prophylactic approach is part of
our ongoing pheEDIT and juMPStart trials, from which we expect to
share initial clinical data mid-year and in the second half of the
year, respectively.”
Dr. Seymour continued, “Also at WORLD, we presented the details
of our optimized gene therapy development candidate for MLD, which
showed the ability to cross the blood-brain-barrier following a
single I.V. administration in the murine disease model and resulted
in levels of enzyme activity predicted to lead to efficacy in vivo.
The data also included HMI-204’s optimized tissue expression
profile and improvements in productivity packaging. We continue to
seek a partner for this candidate, which is ready to enter
IND-enabling studies.”
Details of Homology’s Presentations at
WORLDSymposium™In the
poster titled, “Tacrolimus Administration in Combination with
Dexamethasone Reduces Neutralizing Antibody Formation Against AAV
Vector and Increases Transgene Expression in Cynomolgus Macaques,”
data showed that administration of the T-cell inhibitor tacrolimus
with dexamethasone in AAVHSC-PAH-treated non-human primates (NHPs)
resulted in:
- Lowered secretion of inflammatory cytokines and activation
state of CD8+ T cells compared to the no immunosuppression (IS)
group;
- Reduced AAVHSC neutralizing antibody (nAb) formation compared
to NHPs treated with each agent alone and by 4.8-fold compared to
the no IS group; and
- Increased PAH gene expression, as measured by mRNA, compared to
each agent alone and by two-fold compared to the no IS group.
In the poster titled, “Gene Therapy for Metachromatic
Leukodystrophy: Lead Candidate Optimization,” a single I.V.
administration of optimized HMI-204 in the murine model of MLD
showed:
- Systemic and central nervous system (CNS) biodistribution,
including robust expression in the CNS;
- Human ARSA cellular expression patterns in the brain that were
nearly identical to that of murine Arsa distribution in wildtype
age-matched controls; and
- Sustained levels of ARSA activity reaching normal adult ARSA
brain activity levels.
Additionally, packaging productivity of HMI-204 demonstrated a
substantial improvement in vector genome yields, compared to the
earlier MLD gene therapy candidate.
For more information, please visit the Publications and
Presentations page on Homology’s website.
About Metachromatic Leukodystrophy (MLD)MLD is
a rare lysosomal storage disorder primarily caused by a mutation in
the ARSA gene. ARSA is responsible for the creation of the
arylsulfatase A (ARSA) protein, which is required for the breakdown
of sulfatides in cells. In MLD, sulfatides accumulate and destroy
myelin-producing cells in the peripheral and central nervous
systems leading to progressive and serious neurological
deterioration. The late infantile form of the disorder is estimated
to affect 1 in 40,000 people, and it is fatal within 5-10 years
after onset.
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 clinical programs include HMI-103, a gene editing
candidate for phenylketonuria (PKU); HMI-203, an investigational
gene therapy for Hunter syndrome; and HMI-102, an investigational
gene therapy for adults with PKU. Additional programs focus on
metachromatic leukodystrophy (MLD), 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 (AAVHSCs)
vectors to precisely and efficiently deliver genetic
medicines in vivo through a nuclease-free gene editing
modality, gene therapy, or GTx-mAb, which is designed to produce
antibodies throughout the body. Homology established an AAV
manufacturing and innovation business in partnership with Oxford
Biomedica, which was based on Homology’s internal process
development and manufacturing 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 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. We intend such
forward-looking statements to be covered by the safe harbor
provisions for forward-looking statements contained in Section 27A
of the Securities Act of 1933, as amended, and Section 21E of the
Securities Exchange Act of 1934, as amended. 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 plans to
engage in future collaborations and strategic partnerships; 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 plans to progress our
pipeline of genetic medicine candidates and the anticipated timing
for these milestones; and our position as a leader in the
development of genetic medicines. The words “believe,” “may,”
“will,” “estimate,” “potential,” “continue,” “anticipate,”
“intend,” “expect,” “could,” “would,” “project,” “plan,” “target,”
and similar expressions are intended to identify forward-looking
statements, though not all forward-looking statements use these
words or expressions. 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: 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 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, including for the
manufacture of materials for our research programs, preclinical and
clinical studies; 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; securities class action litigation; the impact
of the COVID-19 pandemic on our business and operations, including
our preclinical studies and clinical trials, and on general
economic conditions; failure to attract, retain and motivate
qualified personnel; the possibility of system failures or security
breaches; risks relating to intellectual property and significant
costs incurred 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
September 30, 2022 and our other filings with the Securities and
Exchange Commission 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 Contacts:Cara MayfieldVice President,
Patient Advocacyand Corporate
Communicationscmayfield@homologymedicines.com781-691-3510Investor
Contact:Brad Smith Chief Financial and Business Officer
bsmith@homologymedicines.com 781-301-7277
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