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[{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Provides Update on Advanced-Stage Clinical Development Program with Givinostat in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"April 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Italfarmaco S.p.A"},{"orgOrder":0,"company":"Italfarmaco S.p.A","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Provides Update on Ongoing Clinical Programs with Givinostat in Oral Presentation at XVIII International Conference","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"February 2021","url1":"","url2":"","graph1":"Small molecule","graph2":"Italfarmaco S.p.A"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chiesi Global Rare Diseases Announces Presentations on Lysosomal Storage Diseases at the Society for the Study of Inborn Errors of Metabolism Annual Symposium","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"September 2022","url1":"","url2":"","graph1":"Large molecule","graph2":"Chiesi Group"},{"orgOrder":0,"company":"Chiesi Group","sponsor":"Protalix BioTherapeutics","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Chiesi Global Rare Diseases and Protalix BioTherapeutics Receive Positive CHMP Opinion for Pegunigalsidase Alfa for Treatment of Fabry Disease","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Large molecule","productStatus":"New Molecular Entity","date":"February 2023","url1":"","url2":"","graph1":"Large molecule","graph2":"Chiesi Group"},{"orgOrder":0,"company":"MolMed","sponsor":"Orchard Therapeutics","pharmaFlowCategory":"D","amount":"Undisclosed","upfrontCash":"Undisclosed","newsHeadline":"Orchard Therapeutics and MolMed Announce Extension of Gene Therapy Manufacturing Collaboration","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Cell and Gene therapy","productStatus":"New Molecular Entity","date":"July 2020","url1":"","url2":"","graph1":"Cell and Gene therapy","graph2":"MolMed"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Gets FDA Rare Pediatric Disease Designation for Givinostat in Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"October 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"The Italfarmaco Group"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Group Announces Positive Topline Data from Phase 3 Trial Showing Beneficial Effect of Givinostat in Patients with Duchenne Muscular Dystrophy","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2022","url1":"","url2":"","graph1":"Small molecule","graph2":"The Italfarmaco Group"},{"orgOrder":0,"company":"The Italfarmaco Group","sponsor":"Syneos Health","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"Italfarmaco Group Completes FDA Submission of New Drug Application for Givinostat in Duchenne Muscular Dystrophy and Receives Priority Review","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"The Italfarmaco Group"},{"orgOrder":0,"company":"EryDel","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","amount":"$485.0 million","upfrontCash":"$485.0 million","newsHeadline":"Quince Therapeutics to Acquire EryDel SpA and its Phase 3 Asset Targeting Ataxia-Telangiectasia with No Currently Approved Treatments and Estimated $1+ Billion Peak Sales Opportunity","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"Approved","date":"July 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"EryDel"},{"orgOrder":0,"company":"EryDel","sponsor":"Quince Therapeutics","pharmaFlowCategory":"D","amount":"$485.0 million","upfrontCash":"$485.0 million","newsHeadline":"Quince Therapeutics Completes Acquisition of EryDel S.p.A.","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"Approved","date":"October 2023","url1":"","url2":"","graph1":"Small molecule","graph2":"EryDel"},{"orgOrder":0,"company":"Palladio Zannini Industrie","sponsor":"Samsara BioCapital","pharmaFlowCategory":"D","amount":"$20.0 million","upfrontCash":"Undisclosed","newsHeadline":"Palladio Completes $20 Million Series B Financing","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"ITALY","productType":"Small molecule","productStatus":"New Molecular Entity","date":"September 2020","url1":"","url2":"","graph1":"Small molecule","graph2":"Palladio Zannini Industrie"}]
Find Clinical Drug Development Pipelines & Deals | PipelineProspector
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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.
Lead Product(s):
Dexamethasone Sodium Phosphate
Therapeutic Area: Genetic Disease
Product Name: EryDex
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Quince Therapeutics
Deal Size: $485.0 million
Upfront Cash: $485.0 million
Deal Type: Acquisition
October 23, 2023
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.
Lead Product(s):
Dexamethasone Sodium Phosphate
Therapeutic Area: Genetic Disease
Product Name: EryDex
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Quince Therapeutics
Deal Size: $485.0 million
Upfront Cash: $485.0 million
Deal Type: Acquisition
July 24, 2023
Details:
ITF2357 (givinostat) is an investigational drug act as a inhibits histone deacetylases (HDACs), which is investigated for the treatment of Duchenne Muscular Dystrophy.
Lead Product(s):
Givinostat
Therapeutic Area: Genetic Disease
Product Name: ITF2357
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Syneos Health
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 29, 2023
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 efficacy.
Lead Product(s):
Pegunigalsidase alfa
Therapeutic Area: Genetic Disease
Product Name: PRX–102
Highest Development Status: Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Protalix BioTherapeutics
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
February 24, 2023
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 Fabry disease.
Lead Product(s):
Pegunigalsidase alfa
Therapeutic Area: Genetic Disease
Product Name: PRX-102
Highest Development Status: Phase III
Product Type: Large molecule
Partner/Sponsor/Collaborator:
Protalix BioTherapeutics
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
September 02, 2022
Details:
The study compared ITF2357 (givinostat) to placebo and met the primary endpoint (change from baseline in the time to climb 4 stairs) following 18 months of treatment in the target population1 with key secondary endpoints consistent with the functional primary endpoint.
Lead Product(s):
Givinostat
Therapeutic Area: Genetic Disease
Product Name: ITF2357
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 25, 2022
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 treatment.
Lead Product(s):
Givinostat
Therapeutic Area: Genetic Disease
Product Name: ITF2357
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
February 22, 2021
Details:
Third regulatory designation Italfarmaco has received from the FDA for Givinostat after the Orphan Drug designation and Fast Track designation and reflects the agency’s recognition of Givinostat’s potential to treat DMD, a devastating genetic disease.
Lead Product(s):
Givinostat
Therapeutic Area: Genetic Disease
Product Name: ITF2357
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
October 09, 2020
Details:
Proceeds from the financing will fund Palladio’s Phase 3 ALERT Study and advance operations.
Lead Product(s):
Lixivaptan
Therapeutic Area: Genetic Disease
Product Name: VPA-985
Highest Development Status: Phase III
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Samsara BioCapital
Deal Size: $20.0 million
Upfront Cash: Undisclosed
Deal Type: Series B Financing
September 25, 2020
Details:
With the extension of the collaboration, MolMed will continue to support activities related to the development and manufacturing of vectors and drug products for several of Orchard’s investigational ex vivo hematopoietic stem cell (HSC) gene therapies in the upcoming years.
Lead Product(s):
OTL-200
Therapeutic Area: Genetic Disease
Product Name: OTL-200
Highest Development Status: Phase III
Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator:
Orchard Therapeutics
Deal Size: Undisclosed
Upfront Cash: Undisclosed
Deal Type: Collaboration
July 09, 2020