We work to save lives.
Our story begins with science.
The founders of Inozyme Pharma observed the role of the biological pathways involved in mineralization, which is an underlying cause of various rare, debilitating, and life-threatening metabolic disorders. These diseases can lead to over-calcification of soft tissues and under-mineralization of bone, with devastating consequences for patients.
Based on a deep understanding of the biology of mineralization, the company is developing new medicines with the potential to drastically improve the standard of care and change treatment paradigms.
We Focus on Mineralization Disorders
We are a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue and skeleton.
Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 Deficiencies.
ENPP1 Deficiency is a rare, inherited, genetic inborn error of metabolism caused by mutations in the ENPP1 gene and we believe there are between 11,000 and 12,000 individuals worldwide with ENPP1 Deficiency. The condition is inherited as a recessive trait in which the genetic mutations result in decreased or absent activity of the ENPP1 enzyme. ENPP1 Deficiency results in low plasma levels of pyrophosphate, or PPi and AMP, a precursor of adenosine, which can lead to neointimal proliferation, and is associated with high early mortality and long-term morbidity. The spectrum of manifestations for ENPP1 Deficiency includes an acute infantile phase, a progressive pediatric phase, and an adult phase.
ABCC6 Deficiency is a rare, inherited, genetic inborn error of metabolism caused by mutations in the ABCC6 gene. The systemic and progressively debilitating condition is believed to affect more than 67,000 individuals worldwide. ABCC6 Deficiency is inherited as a recessive trait in which the genetic mutations result in decreased or absent activity of the ABCC6 protein. The Deficiency leads to low plasma levels of PPi and is associated with pathological mineralization in blood vessels and soft tissues throughout the body, resulting in significant morbidity, including blindness, potentially life-threatening cardiovascular complications, and skin calcification.
INZ-701 is designed to replace the lost enzymatic function of genetically deficient ENPP1 by restoring the normal balance in PPi and adenosine for ENPP1 Deficiency and providing therapeutic effect to treat other diseases, like ABCC6 Deficiency, involving low PPi levels.
Based on its mechanism of action, we believe that INZ-701 has the potential to normalize plasma PPi levels and provide therapeutic benefit to patients beyond those with monogenic defects in the ENPP1 or ABCC6 gene. Abnormal mineralization and neointimal proliferation may also manifest in non-genetic diseases, such as calciphylaxis. Calciphylaxis, a manifestation of chronic kidney disease, is associated with low levels of PPi and is characterized by pathological calcification of the vasculature in the skin and fat leading to blood clots and skin ulcers. Neointimal proliferation in the vasculature is a hallmark of a number of non-genetic diseases in which arteries have been damaged or disrupted by insertion of a stent, bypass graft occlusion, transplant vasculopathy or inflammation known as arteritis.
We are just getting started. We combine all the tools necessary for success:
- Solid science
- Strong business acumen
- Well-defined regulatory strategy
- Robust financial backing