[{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"CytRx Corporation","sponsor":"Orphazyme","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"U.S.A","productType":"Small molecule","year":"2020","type":"Licensing Agreement","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Phase III","graph3":"CytRx Corporation","amount2":0.12,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0.12,"dosageForm":"Capsule","sponsorNew":"CytRx Corporation \/ Orphazyme A\/S","highestDevelopmentStatusID":"10","companyTruncated":"CytRx Corporation \/ Orphazyme A\/S"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Michael J. Fox Foundation","pharmaFlowCategory":"D","therapeuticArea":"Neurology","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Collaboration","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Preclinical","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Preclinical","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Michael J. Fox Foundation","highestDevelopmentStatusID":"4","companyTruncated":"Orphazyme \/ Michael J. Fox Foundation"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"CytRx Corporation","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Public Offering","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0.080000000000000002,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0.080000000000000002,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ CytRx","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ CytRx"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2020","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Immunology","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Immunology","graph2":"Phase II","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II","therapeuticAreaShortName":"Immunology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"8","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Neurology","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Neurology","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Neurology","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"CytRx Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSF-1","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CytRx Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CytRx Corporation \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"CytRx Corporation \/ Not Applicable"},{"orgOrder":0,"company":"CytRx Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CytRx Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CytRx Corporation \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"CytRx Corporation \/ Not Applicable"},{"orgOrder":0,"company":"CytRx Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSP","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CytRx Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CytRx Corporation \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"CytRx Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2021","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"CytRx Corporation","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"U.S.A","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"Molecular chaperones","graph1":"Genetic Disease","graph2":"Phase II\/ Phase III","graph3":"CytRx Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"CytRx Corporation \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"CytRx Corporation \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"DENMARK","productType":"Small molecule","year":"2022","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"HSP","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Orphazyme \/ Not Applicable"},{"orgOrder":0,"company":"Orphazyme","sponsor":"KemPharm","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"DENMARK","productType":"Small molecule","year":"2022","type":"Acquisition","leadProduct":"Arimoclomol","moa":"HSP","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Orphazyme","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Orphazyme \/ KemPharm","highestDevelopmentStatusID":"9","companyTruncated":"Orphazyme \/ KemPharm"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Capsule","sponsorNew":"Zevra Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"10","companyTruncated":"Zevra Therapeutics \/ Not Applicable"},{"orgOrder":0,"company":"Zevra Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Arimoclomol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase III","graph3":"Zevra Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph 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                          01

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Miplyffa (arimoclomol) is a first in class oral HSF1 activator approved for Niemann-Pick disease type C in combination with miglustat.

                          Brand Name : Miplyffa

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 21, 2024

                          Lead Product(s) : Arimoclomol,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          02

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Miplyffa (Arimoclomol) is indicated for use in combination with miglustat for the treatment of Niemann-Pick disease type C in adult and pediatric patients 2 years of age and older.

                          Brand Name : Miplyffa

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          September 20, 2024

                          Lead Product(s) : Arimoclomol,Miglustat

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Approved

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          03

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : The net proceeds from this offering will used to support the pre-commercial launch activities for BRX-345 (arimoclomol) for the treatment of Niemann-Pick Disease Type C.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Undisclosed

                          August 08, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Cantor

                          Deal Size : Undisclosed

                          Deal Type : Public Offering

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                          04

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          August 02, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          05

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : BRX-345 (arimoclomol) is a first in class oral HSF1 activator being investigated for Niemann-Pick disease type C. The FDA has accepted the resubmitted NDA and set a PDUFA date of September 21, 2024.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          July 09, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          06

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : BRX-345 (arimoclomol) is a first in class investigational HSF1 activator, which is being evaluated in phase 2/3 clinical trials for the treatment of Niemann-Pick disease type C.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          March 04, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          07

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : XOMA acquired royalty and milestone rights for LadRx Corporation’s BRX-345 (arimoclomol) for Niemann-Pick Disease Type C, and aldoxorubicin, an albumin-linked formulation of doxorubicin.

                          Brand Name : BRX-345

                          Molecule Type : Small molecule

                          Upfront Cash : $5.0 million

                          January 11, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : XOMA

                          Deal Size : $58.6 million

                          Deal Type : Agreement

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                          08

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          January 08, 2024

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          09

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Arimoclomol is a first in class investigational HSF1 activator, which is under phase 2/3 clinical development for the treatment of Niemann-Pick disease type C.

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          December 27, 2023

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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                          10

                          Pharma, Lab & Chemical Expo
                          Not Confirmed
                          Pharma, Lab & Chemical Expo
                          Not Confirmed

                          Details : Arimoclomol is believed to function by stimulating a normal cellular protein repair pathway through the activation of molecular chaperones. It is being developed as a treatment for Niemann-Pick disease type C (NPC).

                          Brand Name : Undisclosed

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          February 23, 2023

                          Lead Product(s) : Arimoclomol

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

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