RARE Daily

Inozyme Pharma Acquires Assets from Alexion for ENPP1 Deficiency Program

July 23, 2020

Rare Daily Staff

A few days before a planned initial public offering, Inozyme Pharma said it acquired Alexion Pharmaceuticals’ intellectual property and assets focusing on ENPP1 gene deficiencies to complement the ongoing development of its lead candidate for the treatment of ENPP1 and ABCC6 deficiencies—rare diseases of abnormal mineralization.

Genetic mutations resulting in the deficiency of the enzyme ENPP1 lead to severe disorders of calcification, including Generalized Arterial Calcification of Infancy (GACI) and autosomal recessive hypophosphatemic rickets type 2.

Mutations in the human ABCC6/MRP6 gene is a known cause of pseudoxanthoma elasticum, a heritable recessive disorder characterized by calcification of elastin fibers in connective tissue, including the heart, vasculature, skin and eyes.

The acquisition includes Alexion’s patent estate, preclinical data, and manufacturing research relating to Alexion’s prior ENPP1 deficiency program. As consideration for these assets, Alexion received shares of Inozyme’s preferred stock representing a single-digit ownership percentage on a fully diluted basis.

Axel Bolte, co-founder, president, and CEO of Inozyme Pharma, said Alexion’s intellectual property and scientific data complement its own research and development programs for ENPP1 deficiency and related genetic 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.

INZ-701 is an enzyme replacement therapy in development for the treatment of mineralization disorders of the circulatory system, bones, and kidneys.

In preclinical studies, the experimental therapy has shown potential to generate plasma pyrophosphate and to restore it to appropriate physiological levels, thereby preventing calcification in the vasculature and kidneys, while at the same time normalizing bone mineralization.

“Given the development programs underway at Inozyme and their focus on ultra rare diseases of abnormal mineralization, this agreement represents an innovative opportunity to advance the development of therapies for ENPP1 gene deficiencies,” said Rajinder Khunkhun of Alexion Business Development.

Photo: Axel Bolte, co-founder, president, and CEO of Inozyme Pharma


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