BEDFORD, Mass., Feb. 18 10, 2022 (GLOBE NEWSWIRE) — Homology Medicines, Inc. (Nasdaq: FIXX), a genetic drug company, today announced that the U.S. Food and Drug Administration (FDA) has notified the company that its pheNIX gene therapy trial of HMI-102 in adults with phenylketonuria (PKU) has been placed on clinical hold due to the need to modify risk mitigation measures in the study in response to test observations from the elevated liver function. The Society expects to receive an official clinical waiting letter within 30 days. Homology plans to provide an update pending further clarification from the FDA.
“We look forward to working with the FDA to respond to the Agency’s questions and comments once we receive the letter,” said Arthur Tzianabos, Ph.D., President and CEO of Homology Medicines. . “This suspension of our PCU gene therapy trial is based on clinical observations in the pheNIX study and does not affect manufacturing/CMC capabilities or other Homology clinical programs. We plan to provide next steps once we will have more information following our interactions with the FDA.
Homology has two ongoing clinical programs, the pheEDIT gene-editing trial of HMI-103 for PKU and the juMPStart gene therapy trial of HMI-203 for Hunter syndrome, with program updates expected by the end of this year.
About Homology Medicines, Inc.
Homology Medicines, Inc. is a clinical-stage genetic medicine company dedicated to transforming the lives of patients with rare diseases by addressing the underlying cause of the disease. Homology’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 experimental gene therapy for Hunter syndrome. Other programs focus on metachromatic leukodystrophy (MLD), paroxysmal nocturnal hemoglobinuria (PNH) and other diseases. Homology’s proprietary platform is designed to use its family of 15 human hematopoietic stem cell-derived adeno-associated virus (AAVHSC) vectors to accurately and efficiently deliver genetic drugs live via gene therapy or nuclease-free gene editing modality, as well as to provide single gene therapy to produce antibodies throughout the body via the GTx-mAb platform. Homology has a leadership team with a proven track record in the discovery, development and commercialization of therapeutics focused on rare diseases. Homology believes its compelling initial clinical and preclinical data, scientific and product development expertise, in-house manufacturing capabilities, and broad intellectual property position Homology as a leader in genetic medicines. For more information, visit www.homologymedicines.com.
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. All statements in this press release that do not relate to historical facts should be considered forward-looking statements, including, without limitation, statements regarding our expectations regarding the potential, safety, efficacy, and regulatory and clinical progress of our product candidates, including timing and expectations regarding communications with the FDA regarding discontinuation clinical trial of the pheNIX trial and related updates from the Company; the potential of our gene therapy and gene editing platforms, including our GTx-mAb platform; our plans and timing for the publication of additional preclinical and clinical data; our beliefs regarding our manufacturing capabilities; and our leadership position in the development of genetic medicines. These statements are not promises or 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 future results, performance or achievements. expressed or implied by the forward-looking statements. 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; failure to identify additional product candidates and develop or commercialize marketable products; the first step in our development efforts; potential unforeseen events during clinical trials could cause delays or other adverse consequences; risks relating to the regulatory approval process; interim, primary and preliminary data may change as more patient data becomes available and is subject to audit and verification procedures which could result in material changes to the final data; our product candidates may cause serious adverse side effects; failure to obtain marketing authorization in the United States or abroad; and ongoing regulatory obligations. These and other important factors discussed under “Risk Factors” in our Quarterly Report on Form 10-Q for the three months ended September 30, 2021 and our other filings with the SEC could cause results actual differ significantly from those indicated by the future. – forward-looking statements made in this press release. These forward-looking statements represent management’s estimates as of the date of this press release. Although we may choose to update these forward-looking statements at some time in the future, we disclaim any obligation to do so, even if subsequent events change our views.
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