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Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"CytRx Corporation \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"CytRx Corporation \/ Inapplicable"},{"orgOrder":0,"company":"CytRx Corporation","sponsor":"Inapplicable","pharmaFlowCategory":"DU","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2021","type":"Inapplicable","leadProduct":"Arimoclomol Citrate","moa":"HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"CytRx Corporation","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"CytRx Corporation \/ Inapplicable","highestDevelopmentStatusID":"9","companyTruncated":"CytRx Corporation \/ Inapplicable"},{"orgOrder":0,"company":"LadRx","sponsor":"XOMA","pharmaFlowCategory":"D","therapeuticArea":"Rare Diseases and Disorders","country":"U.S.A","productType":"Other Small Molecule","year":"2024","type":"Agreement","leadProduct":"Arimoclomol Citrate","moa":"HSF-1 pathway","graph1":"Rare Diseases and Disorders","graph2":"Phase II\/ Phase III","graph3":"LadRx","amount2":0,"highestDevelopmentShortName":"Ph II\/III","therapeuticAreaShortName":"Rare Diseases","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"LadRx \/ XOMA","highestDevelopmentStatusID":"9","companyTruncated":"LadRx \/ XOMA"}]

Find Clinical Drug Development Pipelines & Deals | PipelineProspector

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                          Top Deals by Deal Size (USD bn)

                          01

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Investigational data from long-term, follow-up studies showed that olipudase alfa provided sustained improvement in lung function (as measured by DLco) and reduction of spleen and liver volumes over time in patients with ASMD.

                          Product Name : Xenpozyme

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          September 02, 2022

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          02

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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

                          Product Name : Xenpozyme

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          June 12, 2021

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          03

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Study Phase : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Details : Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.

                          Product Name : Xenpozyme

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          January 30, 2020

                          Lead Product(s) : Olipudase Alpha

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

                          Sanofi Company Banner

                          04

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : QRX003

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 27, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : QRX003

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 23, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : QRX003

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 14, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : QRX003

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          January 06, 2025

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          08

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          Not Confirmed

                          Details : The net proceeds from the offering will be used for the development of QRX003, a serine protease inhibitor, small molecule drug candidate for treating Netherton Syndrome.

                          Product Name : QRX003

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          December 20, 2024

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Maxim Group LLC

                          Deal Size : $6.8 million

                          Deal Type : Public Offering

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                          09

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : QRX003

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          December 19, 2024

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          10

                          German Wound Congress
                          Not Confirmed
                          German Wound Congress
                          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.

                          Product Name : QRX003

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          December 18, 2024

                          Lead Product(s) : QRX003

                          Therapeutic Area : Rare Diseases and Disorders

                          Highest Development Status : Phase II/ Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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