All Data
Sanofi Active Ingredient Solutions cover both APIs sales & CDMO.
Lead Product(s): Olipudase alfa
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 30, 2020
Details:
Olipudase alfa is the first and only investigational enzyme replacement therapy in late-stage development for the treatment of ASMD.
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Lead Product(s): Sepofarsen
Therapeutic Area: Genetic Disease Product Name: QR-110
Highest Development Status: Phase II/ 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 January 07, 2021
Details:
Illuminate is a Phase 2/3 trial, which randomized 36 patients aged eight years or older to receive either sepofarsen at the target registration dose, a low dose, or sham treatment.
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Lead Product(s): Arimoclomol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 27, 2020
Details:
U.S. Food and Drug Administration (FDA) has extended the review period of the New Drug Application (NDA) for arimoclomol for the treatment of Niemann-Pick Disease Type C (NPC) by a standard extension period of three months.
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Lead Product(s): REN001
Therapeutic Area: Genetic Disease Product Name: REN001
Highest Development Status: Phase II/ Phase III Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Novo Ventures
Deal Size: $95.0 million Upfront Cash: Undisclosed
Deal Type: Series B Financing December 09, 2020
Details:
Funds will support developments of REN001 which is an oral, once-daily investigational drug known to control several genes involved in mitochondrial activity.
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Lead Product(s): FT-4202
Therapeutic Area: Genetic Disease Product Name: FT-4202
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Jefferies
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Public Offering December 08, 2020
Details:
Funds to be used in the development of its lead program FT-4202 in sickle cell disease and completion of Phase 1 clinical trial, advancement of FT-7051 in metastatic castration-resistant prostate cancer, pre-approval activities for FT-2102 in AML.
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Lead Product(s): FT-4202
Therapeutic Area: Genetic Disease Product Name: FT-4202
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable December 07, 2020
Details:
FT-4202 is a novel, investigational, selective red blood cell pyruvate kinase-R activator in development as a potential disease-modifying therapy for SCD. 6 of 7 (86%) patients on 300 mg of FT-4202 for 14 days achieved a hemoglobin increase > 1 g/dL from baseline.
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Lead Product(s): ARO-AAT
Therapeutic Area: Genetic Disease Product Name: ARO-AAT
Highest Development Status: Phase II/ Phase III Product Type: Large molecule
Partner/Sponsor/Collaborator: Takeda Pharmaceutical
Deal Size: $1,040.0 million Upfront Cash: $300.0 million
Deal Type: Licensing Agreement November 24, 2020
Details:
Under the terms of the agreement, Takeda and Arrowhead will co-develop ARO-AAT which, if approved, will be co-commercialized in the United States under a 50/50 profit-sharing structure.
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Lead Product(s): Tideglusib
Therapeutic Area: Genetic Disease Product Name: AMO-02
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 09, 2020
Details:
Designation qualifies AMO-02 for fast track review and AMO Pharma to receive a priority review voucher pending marketing approval. AMO-02, AMO-01 and AMO-04 are investigational medicines that have not yet been approved for the treatment of patients anywhere in the world.
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Lead Product(s): RTA 402
Therapeutic Area: Genetic Disease Product Name: RTA 402
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 09, 2020
Details:
Bardoxolone achieved the year 2 primary and key secondary endpoints with statistically significant improvements in EGFR as compared to placebo at week 100 and week 104 .
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Lead Product(s): FT-4202
Therapeutic Area: Genetic Disease Product Name: FT-4202
Highest Development Status: Phase II/ Phase III Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable November 04, 2020
Details:
The oral presentation will feature clinical data from the multiple ascending dose cohort of a randomized, multi-center, placebo-controlled Phase 1 trial of FT-4202 in people living with sickle cell disease (SCD).
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