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[{"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"},{"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"},{"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"},{"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"},{"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"},{"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"}]

Poxel

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Poxel Announces the Publication of Two Preclinical Articles on X-Linked Adrenoleukodystrophy for PXL065 and PXL770

Details:

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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Pierre Fabre

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Pierre Fabre and the EspeRare Foundation Administer Investigational Treatment to First Patient in EDELIFE Clinical Trial for Rare Genetic Disease, XLHED

Details:

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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Poxel

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Poxel Announces New Solid Form Patent for PXL065 that Provides Additional Protection through 2041

Details:

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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Poxel

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Poxel Announces PXL065 and PXL770 Granted Orphan Drug Designation from the U.S. FDA for X-Linked Adrenoleukodystrophy

Details:

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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Inventiva Pharma

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Inventiva’s Latest Research on Odiparcil’s Mechanism of Action Published in Leading Peer-Reviewed Scientific Journal PLOS ONE

Details:

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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Inventiva Pharma

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Inventiva to Present Results from its Phase IIa Odiparcil Study

Details:

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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