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Lead Product(s): Onis-Httrx
Therapeutic Area: Genetic Disease Product Name: RG6042
Highest Development Status: Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Ionis Pharmaceuticals
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 22, 2021
Details:
Tominersen, previously IONIS-HTTRx or RG6042, is an investigational antisense therapy designed to reduce the production of all forms of the huntingtin protein (HTT), including its mutated variant, mHTT.
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Lead Product(s): Rifampin
Therapeutic Area: Genetic Disease Product Name: Zolgensma
Highest Development Status: Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 15, 2021
Details:
Real-world data indicate older children (aged ≥6 months) achieved clinically meaningful benefit with Zolgensma alone, after or in combination with another SMA therapy, with events consistent with the previously described safety profile.
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Lead Product(s): Idebenone
Therapeutic Area: Genetic Disease Product Name: Puldysa
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 06, 2020
Details:
Data from an interim analysis conducted by the independent Data and Safety Monitoring Board concluded that the Phase 3 SIDEROS study with Puldysa® (idebenone) was unlikely to meet its primary endpoint.
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Lead Product(s): Idebenone
Therapeutic Area: Genetic Disease Product Name: Puldysa
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 06, 2020
Details:
Data from an interim analysis conducted by the (DSMB) concluded that the study was unlikely to meet its primary endpoint. Santhera will discontinue the study, withdraw the European marketing authorization application and end the global development program for Puldysa.
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Lead Product(s): Idebenone
Therapeutic Area: Genetic Disease Product Name: Puldysa
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 23, 2020
Details:
Uk's Medicines and Healthcare products Regulatory Agency has renewed for a further year the Early Access to Medicines Scheme scientific opinion for idebenone for patients with Duchenne muscular dystrophy in respiratory function decline who are not taking glucocorticoids.
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Lead Product(s): Idebenone
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Highbridge Capital Management
Deal Size: $21.1 million Upfront Cash: Undisclosed
Deal Type: Financing June 04, 2020
Details:
In combination with Santhera's existing cash and cash equivalents, this financing will provide the Company with sufficient funding to complete regulatory work for Puldysa®, including the CHMP review, and with the achievement of Company milestones.
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Lead Product(s): Idebenone
Therapeutic Area: Genetic Disease Product Name: Undisclosed
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 May 20, 2020
Details:
The large Phase 3 SIDEROS study was designed to confirm the efficacy of idebenone in patients with respiratory function decline who are concurrently taking glucocorticoids.
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