Homology Medicines Presents on Design of pheEDIT Trial Evaluating One-Time Nuclease-Free Gene Editing Candidate HMI-103 for PKU at American Society of Human Genetics Meeting
October 27 2022 - 9:00AM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today a presentation on the design of pheEDIT, a
Phase 1, open-label, dose-escalation study evaluating one-time gene
editing candidate HMI-103 in adults with phenylketonuria (PKU). The
presentation included preclinical data demonstrating efficacy in a
PKU murine model and precision of editing in a humanized murine
model. During the American Society of Human Genetics (ASHG) Annual
Meeting, the Company also presented data that demonstrated
single-molecule, modified base sequencing can aid in the
characterization, design and optimization of AAV vectors.
“The pheEDIT dose-escalation clinical trial is the first gene
editing study for PKU, and investigational HMI-103 has the
potential to treat adult and pediatric PKU with its unique dual
mechanism of action designed to integrate the PAH gene and
liver-specific promoter into the genome and to maximize PAH
expression in all transduced liver cells,” said Albert Seymour,
Ph.D., President and Chief Executive Officer of Homology Medicines.
“The program’s preclinical data showed long-term Phe normalization
in the PKU model following a single I.V. administration, and a
genome-wide integration assay demonstrated the precision of
homologous recombination with no off-target integration in a
humanized liver model. Together, these data supported the
initiation of pheEDIT and highlight our commitment to leading the
field with an unbiased approach to evaluating on-and off-target
integrations.”
The poster presentation titled, “A Phase 1, Open-Label,
Dose-Escalation Study to Evaluate the Safety and Efficacy of
HMI-103, a One-Time Gene-Editing Vector in Adult Participants with
Classical PKU Due to PAH Deficiency,” outlined the design of the
pheEDIT trial, which is:
- A Phase 1, open label, sequential dose-escalation trial
evaluating safety and efficacy of a single I.V. administration of
HMI-103, including tolerability and Phe levels;
- Designed to enroll three dose cohorts with up to three patients
with uncontrolled PKU in each cohort; and
- Incorporating a steroid-sparing prophylactic immunosuppression
regimen, including a T-cell inhibitor, to temporarily dampen
potential immune response to the capsid.
A second presentation titled, “Single-Molecule, Modified Base
Sequencing to Identify Frequency and Cause of rAAV Vector
Breakpoints,” demonstrated that use of next-generation sequencing
can lead to improved vector design by identifying the locations and
frequency of potential breakpoints that can be addressed by
optimizing the vectors.
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 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 phenylketonuria (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 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 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,
including, without limitation, statements regarding: 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;
and our position as a leader in the development of genetic
medicines. 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 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; 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 June 30, 2022 and our other
filings with the Securities and Exchange Commission (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
Homology Medicines (NASDAQ:FIXX)
Historical Stock Chart
From Aug 2024 to Sep 2024
Homology Medicines (NASDAQ:FIXX)
Historical Stock Chart
From Sep 2023 to Sep 2024