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[{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inventiva to Present Results from its Phase IIa Odiparcil Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Inventiva Pharma"},{"orgOrder":0,"company":"Inventiva Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Inventiva\u2019s Latest Research on Odiparcil\u2019s Mechanism of Action Published in Leading Peer-Reviewed Scientific Journal PLOS ONE","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Inventiva Pharma"},{"orgOrder":0,"company":"Poxel","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"May 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Poxel"},{"orgOrder":0,"company":"Poxel","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Poxel Announces New Solid Form Patent for PXL065 that Provides Additional Protection through 2041","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Poxel"},{"orgOrder":0,"company":"Pierre Fabre","sponsor":"EspeRare","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Pierre Fabre and the EspeRare Foundation Administer Investigational Treatment to First Patient in EDELIFE Clinical Trial for Rare Genetic Disease, XLHED","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Large molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Pierre Fabre"},{"orgOrder":0,"company":"Poxel","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Poxel Announces the Publication of Two Preclinical Articles on X-Linked Adrenoleukodystrophy for PXL065 and PXL770","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"Poxel"},{"orgOrder":0,"company":"Poxel","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Poxel Receives Orphan Drug Designation from the European Commission for PXL770 and PXL065 for Treatment of Adrenoleukodystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"FRANCE","productType":"Small molecule","productStatus":"New Molecular Entity","date":"January 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"Poxel"}]

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            PXL770 is a novel, firstin-class direct adenosine monophosphate-activated protein kinase (AMPK) activator. PXL065 is a novel, proprietary deuterium-stabilized R-stereoisomer of pioglitazone which exerts effects via multiple non-genomic pathways engaged by thiazolidinedione.

            Lead Product(s): PXL770

            Therapeutic Area: Genetic Disease Product Name: PXL770

            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 January 25, 2023

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            PXL065 is a novel, proprietary deuteriumstabilized R-stereoisomer of pioglitazone which exerts effects via multiple non-genomic pathways engaged by thiazolidinedione molecules.

            Lead Product(s): Deuterated Pioglitazone

            Therapeutic Area: Genetic Disease Product Name: PXL065

            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 05, 2022

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            ER004 is a pioneering in-utero therapy designed to replace the function of endogenous Ectodysplasin A1 (EDA1), a protein key to the normal development of ectodermal structures in the foetus.

            Lead Product(s): ER004

            Therapeutic Area: Genetic Disease Product Name: ER004

            Highest Development Status: Phase II Product Type: Large molecule

            Partner/Sponsor/Collaborator: EspeRare

            Deal Size: Not Applicable Upfront Cash: Not Applicable

            Deal Type: Not Applicable July 01, 2022

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            PXL065 is the deuterated-stabilized R stereoisomer (single R-isomer) of pioglitazone, its parent molecule. It is currently being evaluated in a Phase 2 study (DESTINY-1) for NASH, with results expected in Q3 2022.

            Lead Product(s): Deuterated Pioglitazone

            Therapeutic Area: Genetic Disease Product Name: PXL065

            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 June 09, 2022

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            PXL065 (deuterium-stabilized R-pioglitazone) was observed to ameliorate key features of ALD in preclinical models, including both patient-derived cells and a classical rodent disease model.

            Lead Product(s): Deuterated Pioglitazone

            Therapeutic Area: Genetic Disease Product Name: PXL065

            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 May 17, 2022

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            The results show that odiparcil was associated with decreased glycosaminoglycan (GAG) accumulation as well as increased GAG excretion, and highlight its distribution in MPS VI disease-relevant tissues and organs.

            Lead Product(s): Odiparcil

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 May 18, 2020

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            The Phase IIa clinical study results in MPS VI adult patients showed the capacity of odiparcil to improve key clinical parameters such as corneal clouding and cardiac and respiratory functions.

            Lead Product(s): Odiparcil

            Therapeutic Area: Genetic Disease Product Name: Undisclosed

            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 February 03, 2020

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