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Details:

ALXN1840 (WTX101) is a potential new once-daily, oral medicine in development for the treatment of Wilson disease. This investigational, novel molecule is designed to selectively and tightly bind to and remove copper from the body’s tissues and blood.


Lead Product(s): Tiomolibdate Choline

Therapeutic Area: Genetic Disease Product Name: ALXN1840

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Alexion Pharmaceuticals

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 27, 2022

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Results from positive FoCus trial in Wilson disease showed that WTX101 (ALXN1840), met its primary endpoint demonstrating three-times greater copper mobilisation from tissues compared to SoC including in patients who had been treated previously for an average of 10 years.


Lead Product(s): Tiomolibdate Choline

Therapeutic Area: Genetic Disease Product Name: WTX101

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable June 23, 2022

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ALXN1840, a potential new once-daily, oral medicine, demonstrated approximately three times greater copper mobilisation than SoC. ALXN1840 demonstrated approximately three times greater copper mobilisation from tissues than standard-of-care treatments.


Lead Product(s): Tiomolibdate Choline

Therapeutic Area: Genetic Disease Product Name: WTX101

Highest Development Status: Phase IIIProduct Type: Small molecule

Partner/Sponsor/Collaborator: Not Applicable

Deal Size: Not Applicable Upfront Cash: Not Applicable

Deal Type: Not Applicable August 26, 2021

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