- Data From Company’s PKU and Hunter Syndrome Gene Therapy Programs Featured in Presentations -
BEDFORD, Mass., April 14, 2021 – 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.
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.