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Myalepta (metreleptin) is a USFDA approved leptin receptor activator, which is now approved by Health Canada for the treatment of congenital or acquired generalized lipodystrophy.
Lead Product(s): Metreleptin
Therapeutic Area: Genetic Disease Product Name: Myalepta
Highest Development Status: ApprovedProduct Type: Peptide
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable February 05, 2024
Details:
The agreement aims to develop, manufacture, and commercialise OHB-607 (mecasermin rinfabate), a recombinant version of insulin-like growth factor-1 (IGF-1), being developed to treat complications of extremely premature birth.
Lead Product(s): Mecasermin Rinfabate
Therapeutic Area: Pulmonary/Respiratory Diseases Product Name: OHB-607
Highest Development Status: Phase IIProduct Type: Large molecule
Recipient: Oak Hill Bio
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement January 09, 2024
Details:
Filsuvez (birch triterpenes) is a topical gel which is approved for the treatment of wounds associated with dystrophic and junctional epidermolysis bullosa.
Lead Product(s): Birch Triterpenes
Therapeutic Area: Genetic Disease Product Name: Filsuvez
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 19, 2023
Details:
The agreement aims to develop, manufacture, and commercialise outside China and adjacent territories, HSK31858, an oral, potent, and highly selective small molecule DPP1 inhibitor with the potential to be an anti-inflammatory agent in bronchiectasis.
Lead Product(s): HSK31858
Therapeutic Area: Pulmonary/Respiratory Diseases Product Name: HSK31858
Highest Development Status: Phase IIProduct Type: Small molecule
Recipient: Haisco Pharmaceutical
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement November 20, 2023
Details:
Under the terms of the agreement, Chiesi Group acquired all assets and certain liabilities related to Raxone (idebenone) in all indications worldwide, including Raxone in leber hereditary optic neuropathy (LHON).
Lead Product(s): Idebenone
Therapeutic Area: Genetic Disease Product Name: Raxone
Highest Development Status: ApprovedProduct Type: Small molecule
Recipient: Santhera Pharmaceuticals
Deal Size: $104.0 million Upfront Cash: $49.2 million
Deal Type: Divestment July 31, 2023
Details:
Elfabrio (pegunigalsidase alfa-iwxj) is a PEGylated enzyme replacement therapy (ERT) for the treatment of adult patients with Fabry disease. It is a recombinant human α–Galactosidase–A enzyme expressed in plant-cell culture that is designed to provide a long half-life.
Lead Product(s): Pegunigalsidase alfa-iwxj
Therapeutic Area: Genetic Disease Product Name: Elfabrio
Highest Development Status: ApprovedProduct Type: Large molecule
Recipient: Protalix BioTherapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 10, 2023
Details:
PRX-102 (pegunigalsidase alfa), EC authorized, is 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: ApprovedProduct Type: Large molecule
Recipient: Protalix BioTherapeutics
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable May 05, 2023
Details:
The acquisition expands chiesi’s rare disease medicine portfolio by adding Amryt's pipeline including, Filsuvez (oleogel-S10), a topical therapeutic gel, is a potential treatment for the cutaneous manifestations of Epidermolysis Bullosa.
Lead Product(s): Oleogel-S10
Therapeutic Area: Rare Diseases and Disorders Product Name: Filsuvez
Highest Development Status: ApprovedProduct Type: Small molecule
Recipient: Amryt Pharma
Deal Size: $1,480.0 million Upfront Cash: $1,250.0 million
Deal Type: Acquisition April 12, 2023
Details:
Ferriprox™ (deferiprone) is a chelating agent with an affinity for ferric ion (iron III), being developed for the treatment of patients with transfusional iron overload due to thalassemia syndromes.
Lead Product(s): Deferiprone
Therapeutic Area: Genetic Disease Product Name: Ferriprox
Highest Development Status: ApprovedProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 31, 2023
Details:
Under the terms of the collaboration, Affibody and Chiesi Group will collaborate closely to progress up to three programs based on Affibody® molecules against undisclosed targets for respiratory diseases.
Lead Product(s): Undisclosed
Therapeutic Area: Pulmonary/Respiratory Diseases Product Name: Undisclosed
Highest Development Status: DiscoveryProduct Type: Large molecule
Recipient: Affibody
Deal Size: $214.0 million Upfront Cash: Undisclosed
Deal Type: Collaboration March 07, 2023
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