Inozyme Pharma Acquires ENPP1 Deficiency Program Assets from Alexion Pharmaceuticals

BOSTON, July 17, 2020 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. today announced the acquisition of Alexion Pharmaceuticals Inc.’ intellectual property and assets focusing on ENPP1 gene deficiencies. The acquisition complements the ongoing development of Inozyme’s lead product candidate, INZ-701, which Inozyme is investigating for the treatment of ENPP1 and ABCC6 deficiencies, which are rare diseases of abnormal mineralization.

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.

“We are pleased to acquire this intellectual property and scientific data to complement our own research and development programs for ENPP1 deficiency and related genetic diseases,” said Axel Bolte, MSc, MBA, co-founder, president and chief executive officer of Inozyme Pharma. “The acquisition expands our intellectual property portfolio, and we welcome Alexion as a shareholder of Inozyme. We look forward to building on the work of both companies to develop potential new therapies for patients who have limited choices today.”

“Alexion and Inozyme share a commitment to advancing the treatment of rare diseases,” said Rajinder Khunkhun of Alexion Business Development. “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.”

About INZ-701

INZ-701 is a soluble, recombinant, or genetically engineered, fusion protein that is designed to correct a defect in the mineralization pathway caused by ENPP1 and ABCC6 deficiencies. In preclinical studies conducted in ENPP1-deficient mouse models, dosing with INZ-701 resulted in increased plasma pyrophosphate (PPi) levels, reduction in ectopic calcium deposits in a variety of tissues, prevention of calcification in the heart and aorta, and improvements in overall health and survival.  In ABCC6-deficient mouse models, dosing with INZ-701 also increased plasma PPi levels. In addition to normalizing levels of PPi, in preclinical studies, INZ-701 prevented neointimal proliferation in both wild-type and ENPP1-deficient mice. The nonclinical INZ-701 toxicology studies that Inozyme has conducted in two animal species showed no systemic adverse effects at doses that significantly exceeded potential human doses.

About Inozyme Pharma
Inozyme Pharma is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization. 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.

Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit

Inozyme Pharma
Axel Bolte, Co-founder, President and CEO 
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