Homology Medicines Announces Presentations at ACMG Annual Clinical Genetics Meeting
April 14 2021 - 8:30AM
Homology Medicines, Inc. (Nasdaq: FIXX), a clinical-stage genetic
medicines company, announced today two presentations highlighting
clinical and preclinical data from the Company’s phenylketonuria
(PKU) and MPS II (Hunter syndrome) in vivo gene therapy programs,
respectively, at the virtual American College of Medical Genetics
and Genomics (ACMG) Annual Clinical Genetics Meeting.
“We continue to demonstrate the breadth of our growing pipeline
with expanded focus on diseases of the central nervous system,”
stated Albert Seymour, Ph.D., Chief Scientific Officer of Homology
Medicines. “Our AAVHSC vectors have shown the ability to target
peripheral organs and the central and peripheral nervous systems,
and have crossed the blood-brain barrier in preclinical studies
with a single I.V. dose of HMI-203, our gene therapy candidate for
Hunter syndrome. We remain on track to initiate a Phase 1/2
dose-escalation clinical trial this year.”
In the poster titled, “The pheNIX Trial: First-In-Human Gene
Therapy Trial for PKU Due to Phenylalanine Hydroxylase (PAH)
Deficiency,” Olaf Bodamer, M.D., Ph.D., FACMG, FAAP, Park Gerald
Chair in Genetics & Genomics and Associate Chief of Genetics
& Genomics at Boston Children’s Hospital, and principal
investigator of the pheNIX trial, presented results from the
dose-escalation phase of the pheNIX trial, which is evaluating
Homology’s lead gene therapy candidate, HMI-102, in adults with
PKU. These data, previously presented at the New England Consortium
of Metabolic Programs annual meeting in November, showed that
HMI-102 was generally well-tolerated and resulted in marked
reductions in phenylalanine (Phe) and the Phe-to-tyrosine (Tyr)
ratio (Phe/Tyr ratio) at two doses. Recruitment is ongoing for the
Phase 2 randomized, concurrently controlled, dose expansion phase
of the pheNIX trial, with initial data expected this year.
In an additional poster titled, “HMI-203: Gene Therapy
Development Candidate for Mucopolysaccharidosis Type II (MPS II),
or Hunter Syndrome,” a single I.V. dose of HMI-203 in the adult
murine model led to robust biodistribution and sustained human I2S
(hI2S) enzyme expression, ameliorated paw deformities and also led
to uptake of hI2S from the serum of the HMI-203-treated model in
human cell lines. Importantly, these data, which were featured for
the first time at the WORLDSymposium™, demonstrated the potential
for cell cross-correction.
Homology’s e-poster presentations will be available to view each
day of the virtual meeting. For more information, 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
genetic diseases with significant unmet medical needs by curing the
underlying cause of the disease. Homology’s proprietary platform is
designed to utilize its human hematopoietic stem cell-derived
adeno-associated virus vectors (AAVHSCs) to precisely and
efficiently deliver genetic medicines in vivo either through a gene
therapy or nuclease-free gene editing modality across a broad range
of genetic disorders. Homology has a management team with a
successful track record of discovering, developing and
commercializing therapeutics with a particular focus on rare
diseases. The Company’s intellectual property covers its family of
15 AAVHSCs. Homology believes that its compelling preclinical data,
scientific expertise, product development strategy, manufacturing
capabilities, and intellectual property position it as a leader in
the development of genetic medicines. For more information, please
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,
including without limitation statements regarding our expectations
surrounding the potential, safety, efficacy, and regulatory and
clinical progress of our product candidates, including with respect
to the planned Phase 1/2 clinical trial for HMI-203 and the Phase 2
pheNIX trial and related timing; 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; the sufficiency of our
cash and cash equivalents to fund our operations; 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 Annual
Report on Form 10-K for the year ended December 31, 2020 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:Marisa CitranoSenior Corporate
Communications
Associatemcitrano@homologymedicines.com617-335-2841
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