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[{"orgOrder":0,"company":"Azafaros","sponsor":"Forbion","pharmaFlowCategory":"D","amount":"$28.0 million","upfrontCash":"Undisclosed","newsHeadline":"Azafaros Completes EUR 25 Million Series A Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Undisclosed","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Undisclosed"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Enters Clinical Stage with AZ-3102, An Oral Small Molecule Being Developed for Rare Neurogenetic Disorders","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2021","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Lysosomal Storage Disorders at the 18th Annual WORLDSymposium\u2122 Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease","therapeuticArea":"Rare Diseases and Disorders","highestDevelopmentStatus":"Preclinical","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Rare Diseases and Disorders","graph2":"Preclinical"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Receives FDA\u2019s IND Clearance and Fast Track Designation","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"IND Enabling","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"IND Enabling"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"}]

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            Details:

            AZ-3102 is an orally available azasugar with a unique dual mode of action, developed as a potential treatment for rare lysosomal storage disorders with neurological involvement, including GM1 and GM2 gangliosidoses and Niemann-Pick disease type C (NP-C).

            Lead Product(s): AZ-3102

            Therapeutic Area: Genetic Disease Product Name: AZ-3102

            Highest Development Status: Phase II Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable June 08, 2023

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            Details:

            AZ-3102 is a, novel small molecule, candidate developed for Lysosomal Storage Disorders with neurological involvement. AZ-3102 is an oral, brain penetrant azasugar, having dual mode of action by inhibiting two key enzymes which modulate the metabolism of glycosphingolipids.

            Lead Product(s): AZ-3102

            Therapeutic Area: Genetic Disease Product Name: AZ-3102

            Highest Development Status: IND Enabling Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable January 05, 2023

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            AZ‑3102, an orally available, small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism, is designed to selectively inhibit two enzymes involved in glycolipid metabolism aims to reduce toxic glycolipid accumulation.

            Lead Product(s): AZ-3102

            Therapeutic Area: Rare Diseases and Disorders Product Name: AZ-3102

            Highest Development Status: Preclinical Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable March 24, 2022

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            Details:

            Positive safety, tolerability and pharmacodynamics data from multiple dose levels in healthy volunteers support evaluation of AZ-3102, a small molecule designed to be a potent and selective inhibitor of two target enzymes involved in glycolipid metabolism in Gangliosidoses.

            Lead Product(s): AZ-3102

            Therapeutic Area: Genetic Disease Product Name: AZ-3102

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 14, 2022

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            Details:

            The ODD for AZ-3102, selectively inhibit two enzymes involved in glycolipid metabolism GCS and GbA2 for the treatment ofGM2 gangliosidosis has been granted based on efficacy demonstrated in a Sandhoff mouse model, including a clear effect on animal survival.

            Lead Product(s): AZ-3102

            Therapeutic Area: Genetic Disease Product Name: AZ-3102

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable February 01, 2022

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            Details:

            Phase 1 clinical study investigates safety and tolerability, pharmacokinetics and pharmacodynamic effects of first-in-class azasugar AZ-3102 in healthy subjects. AZ-3102 is being initially developed for the treatment of patients suffering from GM1 and GM2 gangliosidoses.

            Lead Product(s): AZ-3102

            Therapeutic Area: Genetic Disease Product Name: AZ-3102

            Highest Development Status: Phase I Product Type: Small molecule

            Partner/Sponsor/Collaborator: Not Applicable

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable April 06, 2021

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            Details:

            The financing helps Azafaros to build its organization and expand its executive team with passionate and experienced people and to advance its rare metabolic disorders pipeline.

            Lead Product(s): AZ-3102

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            Highest Development Status: Undisclosed Product Type: Small molecule

            Partner/Sponsor/Collaborator: Forbion

            Deal Size: $28.0 million Upfront Cash: Undisclosed

            Deal Type: Series A Financing February 06, 2020

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