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Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Product Name: BPS-804
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Ultragenyx Pharmaceutical
Deal Size: $304.0 million Upfront Cash: $50.0 million
Deal Type: Collaboration December 17, 2020
Details:
Ultragenyx will lead future global development of setrusumab in both pediatric and adult patients. Mereo granted Ultragenyx an exclusive license to develop and commercialize setrusumab in the US and rest of the world, excluding Europe where Mereo retains commercial rights.
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Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: IB1001
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable October 19, 2020
Details:
IB1001 demonstrated a statistically significant and clinically meaningful change in both the primary and secondary endpoints.
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Lead Product(s): N-Acetyl-L-Leucine
Therapeutic Area: Genetic Disease Product Name: IB1001
Highest Development Status: Phase II Product Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 30, 2020
Details:
IB1001 demonstrated a statistically significant and clinically meaningful improvement in symptoms, functioning, and quality of life in both primary and topline secondary endpoints for both pediatric and adult patients with NPC.
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Lead Product(s): Setrusumab
Therapeutic Area: Genetic Disease Product Name: BPS-804
Highest Development Status: Phase II Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable September 24, 2020
Details:
In Mereo’s Phase 2b ASTEROID study, setrusumab demonstrated a dose-dependent bone building effect and a trend of reduction in fractures in addition to being safe and well tolerated in adults with OI.
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Lead Product(s): OTL-103
Therapeutic Area: Genetic Disease Product Name: OTL-103
Highest Development Status: Phase II Product Type: Cell and Gene therapy
Partner/Sponsor/Collaborator: Orchard Therapeutics
Deal Size: Undisclosed Upfront Cash: Undisclosed
Deal Type: Licensing Agreement July 15, 2020
Details:
Orchard has entered into this license agreements with GSK for use of their lentiviral stable cell line technology for Orchard’s investigational hematopoietic stem cell gene therapies for Wiskott Aldrich syndrome and transfusion-dependent beta thalassemia.
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