[{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2024","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Injection","sponsorNew":"Chiesi Group \/ Protalix BioTherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Protalix BioTherapeutics"},{"orgOrder":0,"company":"EryDel","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Steroid","year":"2023","type":"Acquisition","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"EryDel","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-erythrocyte Infusion","sponsorNew":"EryDel \/ Quince Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"EryDel \/ Quince Therapeutics"},{"orgOrder":0,"company":"EryDel","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Steroid","year":"2023","type":"Acquisition","leadProduct":"Dexamethasone Sodium Phosphate","moa":"Glucocorticoid receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"EryDel","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intra-erythrocyte Infusion","sponsorNew":"EryDel \/ Quince Therapeutics","highestDevelopmentStatusID":"10","companyTruncated":"EryDel \/ Quince Therapeutics"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2023","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Chiesi Group \/ Protalix BioTherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Protalix BioTherapeutics"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Enzyme","year":"2022","type":"Inapplicable","leadProduct":"Pegunigalsidase Alpha","moa":"Alpha-galactosidase A","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Chiesi Group","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Intravenous Infusion","sponsorNew":"Chiesi Group \/ Protalix BioTherapeutics","highestDevelopmentStatusID":"10","companyTruncated":"Chiesi Group \/ Protalix BioTherapeutics"},{"orgOrder":0,"company":"Palladio Zannini Industrie","sponsor":"Samsara BioCapital","pharmaFlowCategory":"D","therapeuticArea":"Genetic Disease","country":"ITALY","productType":"Other Small Molecule","year":"2020","type":"Series B Financing","leadProduct":"Lixivaptan","moa":"V2 receptor","graph1":"Genetic Disease","graph2":"Phase III","graph3":"Palladio Zannini Industrie","amount2":0,"highestDevelopmentShortName":"Ph III","therapeuticAreaShortName":"Genetic Disease","amount2New":0,"dosageForm":"Oral Capsule","sponsorNew":"Palladio Zannini Industrie \/ Samsara BioCapital","highestDevelopmentStatusID":"10","companyTruncated":"Palladio Zannini Industrie \/ Samsara BioCapital"}]

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

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : Elfabrio (pegunigalsidase alfa) is a globotriaosylceramide inhibitor enzyme drug candidate, which is being evaluated to label a less frequent dosing regimen for treating fabry disease.

                          Product Name : Elfabrio

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          December 09, 2024

                          Lead Product(s) : Pegunigalsidase Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Protalix BioTherapeutics

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          02

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : Through the acquisition, Quince gains right for EryDel's Phase 3 lead asset, EryDex (dexamethasone sodium phosphate), targeting a rare fatal pediatric neurological disease, Ataxia-Telangiectasia (A-T), which currently has no approved treatments.

                          Product Name : EryDex

                          Product Type : Steroid

                          Upfront Cash : $485.0 million

                          October 23, 2023

                          Lead Product(s) : Dexamethasone Sodium Phosphate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Quince Therapeutics

                          Deal Size : $485.0 million

                          Deal Type : Acquisition

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                          03

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : Through the acquisition, Quince gains right for EryDel's Phase 3 lead asset, EryDex (dexamethasone sodium phosphate), targeting a rare fatal pediatric neurological disease, Ataxia-Telangiectasia (A-T), which currently has no approved treatments.

                          Product Name : EryDex

                          Product Type : Steroid

                          Upfront Cash : $485.0 million

                          July 24, 2023

                          Lead Product(s) : Dexamethasone Sodium Phosphate

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Quince Therapeutics

                          Deal Size : $485.0 million

                          Deal Type : Acquisition

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                          04

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : PRX–102 (pegunigalsidase alfa) a novel PEGylated, covalently crosslinked form of α-galactosidase A developed as enzyme replacement therapy for fabry disease, was designed to increase plasma half-life and reduce immunogenicity, thereby enhancing effica...

                          Product Name : PRX-102

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          February 24, 2023

                          Lead Product(s) : Pegunigalsidase Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Protalix BioTherapeutics

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          05

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : Results from the clinical development programme developed with Protalix BioTherapeutics evaluating pegunigalsidase alfa (PRX-102) an investigational enzyme replacement therapy (ERT), support its potential as a treatment option for adult patients with Fab...

                          Product Name : PRX-102

                          Product Type : Enzyme

                          Upfront Cash : Inapplicable

                          February 09, 2022

                          Lead Product(s) : Pegunigalsidase Alpha

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Protalix BioTherapeutics

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          06

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : Italfarmaco provided evidence which continues to show a delay in disease progression in DMD boys aged 7-11 years at treatment start, supported by data on the 7-year follow-up period from the long-term study with Givinostat in addition to steroid treatmen...

                          Product Name : Duvyzat

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          February 22, 2021

                          Lead Product(s) : Givinostat HCl

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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                          07

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : Proceeds from the financing will fund Palladio’s Phase 3 ALERT Study and advance operations.

                          Product Name : VPA-985

                          Product Type : Other Small Molecule

                          Upfront Cash : Undisclosed

                          September 25, 2020

                          Lead Product(s) : Lixivaptan

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Samsara BioCapital

                          Deal Size : $20.0 million

                          Deal Type : Series B Financing

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                          08

                          Indo Livestock Expo
                          Not Confirmed
                          Indo Livestock Expo
                          Not Confirmed

                          Details : The Interim data from EPYDIS Phase 3 trial shows that the trial is NOT FUTILE and the Independent Data Monitoring Committee recommends trial continuation.

                          Product Name : Duvyzat

                          Product Type : Other Small Molecule

                          Upfront Cash : Inapplicable

                          April 27, 2020

                          Lead Product(s) : Givinostat HCl

                          Therapeutic Area : Genetic Disease

                          Highest Development Status : Phase III

                          Sponsor : Inapplicable

                          Deal Size : Inapplicable

                          Deal Type : Inapplicable

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