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    [{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros\u2122 Phase 2 RAINBOW study, Evaluating Nizubaglustat in GM2 and NPC Patients, is Now Fully Enrolled","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"December 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Announces Completion of 12-week Phase 2 RAINBOW Study Evaluating Lead Asset Nizubaglustat in Rare Disease Patients","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Azafaros","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Azafaros Announces Positive Phase 2 Data with Nizubaglustat in GM2 and Niemann-Pick","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"NETHERLANDS","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2024","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"}]

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              Azafaros

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              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.

              Lead Product(s): Nizubaglustat

              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 July 16, 2024

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              Azafaros

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              Azafaros Announces Completion of 12-week Phase 2 RAINBOW Study Evaluating Lead Asset Nizubaglustat in Rare Disease Patients

              Details:

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

              Lead Product(s): Nizubaglustat

              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 March 12, 2024

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              Azafaros

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              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 disease type C (NPC).

              Lead Product(s): Nizubaglustat

              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 December 11, 2023

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              Biophore is a research-driven global pharmaceutical company focused on niche APIs for the generic industry.
              Athena Pharmaceutiques is a Partner of Choice for Drug Delivery & Life Cycle Management.
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