Science defines our company and guides our work.
Our lead product candidate is INZ-701, an enzyme replacement therapy (ERT) in preclinical development for the potential treatment of patients with a variety of calcification disorders linked primarily to mutations in the ENPP1 and ABCC6 genes, or ENPP1 Deficiency and ABCC6 Deficiency respectively. We have demonstrated proof of concept with INZ-701 in both GACI and ARHR2, and we expect IND-enabling programs to be completed in the next 18 months.
Proof of Concept Demonstrated in GACI and ARHR2 with IND Enabling Programs to be Completed in Next 18 Months
|Proof of Concept||IND-Enabling||Clinical|
The disorders resulting from a mutation in the ENPP1 gene include:
Generalized arterial calcification of infancy (GACI) type 1, a devastating and often fatal disease caused by a deficiency in the ENPP1 enzyme, which regulates inorganic pyrophosphate (PPi) levels in plasma. GACI type 1 affects infants aged 0-12 months and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about 60-70% of patients within the first six months of life.
Autosomal recessive hypophosphatemic rickets type 2 (ARHR2) is another disease resulting from ENPP1 deficiency. It is a form of rickets that usually appears in the post-infancy stage, though it can occur in patients without prior GACI. ARHR2 affects children (aged 1-18 years) as well as adults (over 18 years). The disease is characterized by short stature, skeletal deformities, weakened bones and muscles, repeated bone fractures, fatigue, and bone pain.
The disorders resulting from a mutation in the ABCC6 gene include:
Pseudoxanthoma elasticum (PXE), an inherited disorder caused by mutations in the ABCC6 transporter gene, which regulates PPi levels in plasma. PXE affects older children and adults and is characterized by mineralization of elastic tissue in the body, whereby calcium deposits form in the tissue, often resulting in changes in the skin, eyes, cardiovascular system, and gastrointestinal system.
Generalized arterial calcification of infancy (GACI) type 2, a devastating and often fatal disease caused by a deficiency in the ABCC6 gene. GACI type 2 affects infants aged 0-12 months and is very similar to GACI type 1 in that it is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about 60-70% of patients within the first six months of life.
Given the broad therapeutic applicability of the INZ-701 clinical development program, we may embark on testing INZ-701 in other chronic diseases such as:
Chronic kidney disease (CKD), also known as chronic kidney failure, is a condition characterized by a gradual loss of kidney function. CKD may lead to a number of additional complications, including hypertension (high blood pressure), anemia (low blood count), weak bones, malnutrition, and nerve damage. Heart disease is the major cause of death for all people with CKD.
Calciphylaxis is a serious, uncommon disease in which calcium accumulates in small blood vessels of the fat and skin tissues. Calciphylaxis can cause blood clots, painful skin ulcers, and serious infections that can lead to death. People who have calciphylaxis usually experience kidney failure and are on dialysis or have had to undergo a kidney transplant procedure. The condition can also occur in people without kidney disease.
We have an ongoing collaboration with the laboratory of Demetrios Braddock at Yale University to further characterize disorders of aberrant calcification. This translational research program potentially opens a number of additional indications to drug therapy.
Building on the foundation of the research conducted at Yale, we have invested in our own internal lab capabilities with more than 3,500 square feet of research facilities located in the heart of Boston’s Longwood Medical Area.