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XOMA"},{"orgOrder":0,"company":"Avicanna","sponsor":"Undisclosed","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Licensing Agreement","leadProduct":"Cannabidiol","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Avicanna","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Cream","sponsorNew":"Avicanna \/ Undisclosed","highestDevelopmentStatusID":"9","companyTruncated":"Avicanna \/ Undisclosed"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2023","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"SKINVISIBLE PHARMACEUTICALS INC","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"SKINVISIBLE PHARMACEUTICALS INC","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"SKINVISIBLE PHARMACEUTICALS INC \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"SKINVISIBLE PHARMACEUTICALS INC \/ Not Applicable"},{"orgOrder":0,"company":"Quoin Pharmaceuticals","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"QRX003","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Quoin Pharmaceuticals","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Lotion","sponsorNew":"Quoin Pharmaceuticals \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Quoin Pharmaceuticals \/ Not Applicable"},{"orgOrder":0,"company":"Galectin Therapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"","productType":"Small molecule","year":"2024","type":"Not Applicable","leadProduct":"Belapectin","moa":"","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"Galectin Therapeutics","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"","sponsorNew":"Galectin Therapeutics \/ Not Applicable","highestDevelopmentStatusID":"9","companyTruncated":"Galectin Therapeutics \/ Not Applicable"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          Therapeutic Area by Lead Product

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Details : Olipudase alfa is an investigational enzyme replacement therapy designed to replace deficient or defective ASM, allowing for the breakdown of sphingomyelin.

                          Brand Name : GZ402665

                          Molecule Type : Large molecule

                          Upfront Cash : Not Applicable

                          December 06, 2021

                          Lead Product(s) : Olipudase Alfa

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Not Applicable

                          Deal Size : Not Applicable

                          Deal Type : Not Applicable

                          Sanofi Company Banner

                          02

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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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                          03

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : QRX003 is a topical lotion formulated with Invisicare and contains a broad-spectrum serine protease inhibitor, being developed for Netherton Syndrome in 14 years and older.

                          Brand Name : QRX003

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          November 05, 2024

                          Lead Product(s) : QRX003

                          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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                          04

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : MTP-131 (elamipretide) is an investigational mitochondrial protective agent , which is currently being evaluated for the treatment of patients with Barth syndrome.

                          Brand Name : MTP-131

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          October 11, 2024

                          Lead Product(s) : Elamipretide

                          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

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : MTP-131 (elamipretide) is an investigational mitochondrial protective agent , which is currently being evaluated for the treatment of patients with Barth syndrome.

                          Brand Name : MTP-131

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          September 09, 2024

                          Lead Product(s) : Elamipretide

                          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

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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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                          07

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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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                          08

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : MTP-131 (elamipretide) is an investigational mitochondrial protective agent , which is currently being evaluated for the treatment of patients with Barth syndrome.

                          Brand Name : MTP-131

                          Molecule Type : Peptide

                          Upfront Cash : Not Applicable

                          July 29, 2024

                          Lead Product(s) : Elamipretide

                          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

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          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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                          10

                          Medlab Asia & Asia Health
                          Not Confirmed
                          Medlab Asia & Asia Health
                          Not Confirmed

                          Details : GR-MD-02 (belapectin) is a complex carbohydrate drug that targets galectin-3, which is being evaluated with the patients for the treatment of Cirrhotic Portal Hypertension.

                          Brand Name : GR-MD-02

                          Molecule Type : Small molecule

                          Upfront Cash : Not Applicable

                          April 09, 2024

                          Lead Product(s) : Belapectin

                          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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