Lead Product(s) : Pegunigalsidase Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Protalix BioTherapeutics
Deal Size : Inapplicable
Deal Type : Inapplicable
Chiesi & Protalix Announce EMA Validation for Pegunigalsidase Alfa Submission
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
Lead Product(s) : Dexamethasone Sodium Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Quince Therapeutics
Deal Size : $485.0 million
Deal Type : Acquisition
Quince Therapeutics Completes Acquisition of EryDel S.p.A.
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
Lead Product(s) : Dexamethasone Sodium Phosphate
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Quince Therapeutics
Deal Size : $485.0 million
Deal Type : Acquisition
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
Lead Product(s) : Pegunigalsidase Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Protalix BioTherapeutics
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Pegunigalsidase Alpha
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Protalix BioTherapeutics
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Givinostat HCl
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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
Lead Product(s) : Lixivaptan
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Samsara BioCapital
Deal Size : $20.0 million
Deal Type : Series B Financing
Palladio Completes $20 Million Series B Financing
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
Lead Product(s) : Givinostat HCl
Therapeutic Area : Genetic Disease
Study Phase : Phase III
Sponsor : Inapplicable
Deal Size : Inapplicable
Deal Type : Inapplicable
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