Homology Medicines Announces FDA Lifted Clinical Hold on pheNIX Gene Therapy Trial for PKU
June 13 2022 - 8:00AM
Homology Medicines, Inc. (Nasdaq: FIXX), a genetic medicines
company, announced today that the U.S. Food and Drug Administration
(FDA) lifted the clinical hold previously placed on the pheNIX gene
therapy clinical trial with investigational HMI-102 for adults with
phenylketonuria (PKU). The FDA noted in its response that Homology
satisfactorily addressed all clinical hold issues identified in its
letter of March 17, 2022. Homology plans to share the changes to
the protocol, which include a new steroid-sparing immunosuppression
regimen that incorporates a T-cell inhibitor and a shorter course
of steroids, with the clinical trial sites.
“We believe our ability to quickly and successfully respond to
the FDA is a testament to our experienced clinical and regulatory
teams who also applied key learnings from the gene therapy field
and our clinical program to update the pheNIX trial protocol,” said
Albert Seymour, Ph.D., President and Chief Scientific Officer of
Homology Medicines. “We appreciate the FDA’s active collaboration
during this process, and we remain committed to bringing forward
potential one-time treatments for people with PKU. Our plan is to
provide an update on the program this fall.” As previously
disclosed, the clinical hold on the pheNIX trial pertained to
elevated liver function tests observed in the trial, which were all
resolved with no hospitalizations required. Homology’s response to
the FDA included changes to the protocol intended to enhance
risk-mitigation measures and a new immunosuppression regimen with
the T-cell inhibitor tacrolimus, which will be used in combination
with a reduced duration of prophylactic steroids. The same approach
is being used in Homology’s ongoing pheEDIT gene editing trial for
PKU and juMPStart gene therapy trial for Hunter syndrome. Similar
regimens have been shown to dampen the immune response to AAVs in
the clinical setting.
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-102, an investigational
gene therapy for adults with phenylketonuria (PKU); HMI-103, a gene
editing candidate for PKU; and HMI-203, an investigational gene
therapy for Hunter syndrome. 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 protocol
amendments and the additional risk-mitigation measures in place for
our pheNIX trial, as discussed above; our plans and timing for the
release of additional preclinical and clinical data; our
expectations surrounding the potential, safety, and efficacy of our
product candidates; the potential of our gene therapy and gene
editing platforms; 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; risks associated with international
operations, such as political and economic instability, including
in light of the conflict between Russia and Ukraine; 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 March 31, 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 Contacts:Theresa McNeelyChief
Communications Officer and Patient
Advocatetmcneely@homologymedicines.com781-301-7277Media
Contact:Cara Mayfield Vice President, Patient Advocacy and
Corporate Communications cmayfield@homologymedicines.com
781-691-3510
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