BOSTON, Oct. 10, 2023 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“Inozyme” or the “Company”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, highlighted the inclusion of Generalized Arterial Calcification of Infancy (GACI), which is caused by mutations in the ENPP1 or ABCC6 genes, in the Genomic England’s Generation Study. This research initiative, embedded within the UK’s National Health Service (NHS), was developed to advance early detection and treatment of rare genetic conditions, with the goal of setting a new standard for newborn screening programs.
“We commend Genomics England for recognizing the crucial importance of early detection of GACI as we advance INZ-701 into clinical trials in infants. Including ENPP1 and ABCC6 in the initial list of genes selected for this groundbreaking study represents the hard work and dedication of the advocates, families, and healthcare professionals who work tirelessly for those living with this disease,” said Catherine Nester, R.N., Inozyme’s senior vice president of healthcare professionals and patient engagement. “The Generation Study has the potential to make a significant impact on the creation of more comprehensive newborn screening programs that save lives by empowering families to find appropriate care for their babies as quickly as possible.”
The Generation Study is scheduled to begin in late 2023, with the goal of sequencing the genomes of more than 100,000 infants and paving the way for potential widespread implementation of whole-genome sequencing in newborn screening. Genomics England collaborated with NHS experts as well as scientists, healthcare professionals (HCPs), and people living with rare conditions to select 223 individual conditions for its current screening program.
GACI is a rare genetic disorder that is fatal in 50 percent of affected infants by six months of age due to severe and pathological vascular calcification and neointimal proliferation (overgrowth of smooth muscle cells inside blood vessels). The disease can lead to the potential failure of major organs, such as the heart, lungs, and kidneys, and it is caused by ENPP1 and ABCC6 Deficiencies, rare disorders for which there are currently no approved treatments. Inozyme is developing INZ-701, an enzyme replacement therapy, for the treatment of rare disorders like GACI that impact the vasculature, soft tissue, or skeleton.
To learn more about Genomics England and its Generation Study, read the full announcement here.
INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. In preclinical studies, the experimental therapy has shown potential to prevent pathologic mineralization and intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels), which can drive morbidity and mortality in devastating genetic disorders such as ENPP1 Deficiency and ABCC6 Deficiency. INZ-701 is currently in clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.
About Inozyme Pharma
Inozyme Pharma, Inc. is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases impacting the vasculature, soft tissue, and skeleton. Inozyme is developing INZ-701, an enzyme replacement therapy, to address pathologic mineralization and intimal proliferation which can drive morbidity and mortality in these severe diseases. INZ-701 is currently in clinical trials for the treatment of ENPP1 Deficiency and ABCC6 Deficiency.
Cautionary Note Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the availability of data from clinical trials, and the potential benefits of INZ-701. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to conduct its ongoing clinical trials of INZ-701 for ENPP1 Deficiency and ABCC6 Deficiency; enroll patients in ongoing and planned trials; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain, and protect intellectual property rights related to its product candidates; manage expenses; comply with covenants under its outstanding loan agreement; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.
Stefan Riley, Director of IR and Corporate Communications