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Disease","therapeuticArea":"Neurology","highestDevelopmentStatus":"Discovery","country":"U.S.A","productType":"Undisclosed","productStatus":"New Molecular Entity","date":"April 2023","url1":"","url2":"","graph1":"Neurology","graph2":"Discovery"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"AstraZeneca","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Danicopan as Add-on to Ultomiris or Soliris Improved Haemoglobin Levels and Maintained Disease Control in Patients with PNH Experiencing Signs or Symptoms of Clinically Significant Extravascular Haemolysis","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III"},{"orgOrder":0,"company":"Alexion Pharmaceuticals","sponsor":"BridgeBio 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Find Clinical Drug Pipeline Developments & Deals by Portola Pharmaceuticals
Ultomiris (ravulizumab) has been approved in the US as the first and only long-acting C5 complement inhibitor for the treatment of adult patients with anti-aquaporin-4 (AQP4) antibody-positive (Ab+) neuromyelitis optica spectrum disorder.
The collaboration aims to develop an undisclosed initial therapeutic molecule that applies JCR’s proprietary J-Brain Cargo®, blood-brain barrier penetration technology, for the treatment of a neurodegenerative disease.
AG-10 (acoramidis) is an investigational, next-generation, orally-administered, highly potent, small molecule stabiliser of transthyretin (TTR). It is being evaluated in phase 3 clinical trials for the treatment of Transthyretin Amyloid Cardiomyopathy.
Under the agreement, Alexion expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
The collaboration aims to identify novel drug targets for rare neurodegenerative and neuromuscular diseases by leveraging CONVERGE, a full-stack platform that combines highly predictive human tissue datasets with machine learning to find new targets.
Under the agreement, Alexion will expand its rare disease gene therapy portfolio by gaining Pfizer’s early-stage preclinical gene therapy programmes. The agreement furthers Alexion's commitment to advancing next-generation genomic medicines.
AG10 (acoramidis) is an investigational, orally-administered small molecule designed to potently stabilize tetrameric transthyretin (TTR), being investigated for the treatment of transthyretin amyloid cardiomyopathy (ATTR-CM).
ALXN2040 (danicopan) is an investigational oral medicine in development as an add-on to standard of care C5 inhibitor therapy Ultomiris (ravulizumab) or Soliris (eculizumab) for patients with PNH who experience clinically significant EVH.
Under the agreement, the two companies will begin research and development of new treatments using the J-Brain Cargo®, blood-brain barrier (“BBB”) penetration technology, in combination with an undisclosed effector molecule for the treatment of a neurodegenerative disease.
The acquisition creates an opportunity to accelerate Alexion’s growth in genomic medicines through unique technology, an experienced rare disease R&D team, and expertise in pre-clinical development.
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