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    [{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2025","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2024","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2023","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Genetic Disease","graph2":"Phase I","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph I","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"6","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Forbion","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2020","type":"Series A Financing","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Rare Diseases and Disorders","graph2":"Undisclosed","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Undisclosed","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral","sponsorNew":"Azafaros \/ Forbion","highestDevelopmentStatusID":"1","companyTruncated":"Azafaros \/ Forbion"},{"orgOrder":0,"company":"Azafaros","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2022","type":"Inapplicable","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Azafaros","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Undisclosed","sponsorNew":"Azafaros \/ Inapplicable","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Inapplicable"},{"orgOrder":0,"company":"Azafaros","sponsor":"Jeito Capital","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"NETHERLANDS","productType":"Other Small Molecule","year":"2025","type":"Series B Financing","leadProduct":"Nizubaglustat","moa":"GBA2 protein","graph1":"Rare Diseases and Disorders","graph2":"Phase II","graph3":"Azafaros","amount2":0.14999999999999999,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Rare Diseases","amount2New":0.14999999999999999,"dosageForm":"Oral Capsule","sponsorNew":"Azafaros \/ Jeito Capital","highestDevelopmentStatusID":"8","companyTruncated":"Azafaros \/ Jeito Capital"}]

    Find Clinical Drug Pipeline Developments & Deals by Azafaros

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                            01

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Phase II

                            Sponsor : Jeito Capital

                            Deal Size : $147.0 million

                            Deal Type : Series B Financing

                            DEALS_DEV arrow-down

                            Azafaros Raises €132M Series B for Phase 3 Lysosomal Disorder Programs

                            Details : The financing aims to advance the clinical development of company's mid-stage product AZ-3102 (nizubaglustat), which is being evaluated for Niemann-Pick disease.

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Undisclosed

                            May 13, 2025

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Phase II

                            Sponsor : Jeito Capital

                            Deal Size : $147.0 million

                            Deal Type : Series B Financing

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                            02

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Receives Regulatory Clearance for Phase 3 Studies in 2025

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor, being evaluated for the treatment of GM1 Gangliosidosis and Type C Niemann-Pick Disease.

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 08, 2025

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            03

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Completes Phase 2 RAINBOW Study for Nizubaglustat in Rare Diseases

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor. Currently, it has completed the ongoing Phase II clinical trial studies for GM2 Gangliosidosis and Type C Niemann-Pick Disease.

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            December 03, 2024

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            04

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Announces Positive Phase 2 Data with Nizubaglustat in GM2 and Niemann-Pick

                            Details : AZ-3102 (nizubaglustat) is a small molecule, GBA2 protein inhibitor, being evaluated for the treatment of GM2 Gangliosidosis and Type C Niemann-Pick Disease.

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            July 16, 2024

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            05

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros™ Phase 2 RAINBOW study, Evaluating Nizubaglustat in GM2 and NPC Patients, is Now Fully Enrolled

                            Details : AZ-3102 (nizubaglustat) 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 diseas...

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            November 12, 2023

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            06

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Announces Enrollment of First Patient in Phase 2 RAINBOW Study Evaluating AZ-3102 in GM2 and NP-C Pat...

                            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).

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            August 06, 2023

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase II

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            07

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Receives FDA’s IND Clearance and Fast Track Designation

                            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...

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            May 01, 2023

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : IND Enabling

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            08

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Study Phase : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Announces FDA Grant of Orphan Drug Designation for AZ-3102 in the Treatment of Niemann-Pick Disease

                            Details : 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 ...

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            March 24, 2022

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Rare Diseases and Disorders

                            Highest Development Status : Preclinical

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            09

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Presents Positive Clinical and Preclinical Data Supporting Development of Lead Compound AZ-3102 in Ly...

                            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 metabolis...

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            February 14, 2022

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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                            10

                            Azafaros

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                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Study Phase : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

                            DEALS_DEV arrow-down

                            Azafaros Receives FDA Orphan Drug Designation for AZ-3102 in GM2 Gangliosidosis

                            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 survi...

                            Product Name : AZ-3102

                            Product Type : Other Small Molecule

                            Upfront Cash : Inapplicable

                            January 02, 2022

                            Lead Product(s) : Nizubaglustat

                            Therapeutic Area : Genetic Disease

                            Highest Development Status : Phase I

                            Sponsor : Inapplicable

                            Deal Size : Inapplicable

                            Deal Type : Inapplicable

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