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Details:
Disc Medicine (bitopertin) is an oral, selective inhibitor of glycine transporter 1. is developing bitopertin as the potentially first disease-modifying therapy to treat erythropoietic porphyrias – a family of rare, genetic disorders caused by dysregulated heme synthesis.
Lead Product(s): Bitopertin
Therapeutic Area: Genetic Disease Product Name: DISC-1459
Highest Development Status: Phase II Product Type: Small molecule
Recipient: Disc medicine
Deal Size: $175.0 million Upfront Cash: Undisclosed
Deal Type: Merger December 29, 2022
Details:
The team’s ability to overenroll the study highlights the unmet need and the potential for GEM103 to address these suboptimal outcomes in the setting of regular anti-VEGF therapy and its subsequent depletion in CFH levels causing complement dysfunction.
Lead Product(s): GEM103,Aflibercept
Therapeutic Area: Ophthalmology Product Name: GEM103
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 May 13, 2021
Details:
ReGAtta is a dose escalation trial of GEM103, a recombinant human complement factor H (CFH), in dry AMD patients with CFH loss-of-function gene variants.
Lead Product(s): GEM103
Therapeutic Area: Ophthalmology Product Name: GEM103
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 February 10, 2021
Details:
ReGAtta is a dose escalation trial of GEM103, in dry AMD patients with CFH loss-of-function gene variants. GEM103 targets a genetically-defined subset of age-related macular degeneration (AMD) patients with complement dysregulation.
Lead Product(s): GEM103
Therapeutic Area: Ophthalmology Product Name: GEM103
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 February 01, 2021
Details:
Results showed that, in the four ascending single IVT doses tested (50, 100, 250 and 500 µg of GEM103 in a 50µL dose volume), there were no inflammation, anti-drug antibody, or treatment-related adverse events. A single intravitreal injection of GEM103 met safety endpoint.
Lead Product(s): Recombinant human complement Factor H
Therapeutic Area: Ophthalmology Product Name: GEM103
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 November 13, 2020
Details:
Proceeds from the transaction are expected to provide Gemini with the capital needed to further develop its clinical programs and preclinical portfolio, including GEM103, Further clinical programs in selected wet AMD populations, Future programs to treat intermediate AMD.
Lead Product(s): GEM103
Therapeutic Area: Ophthalmology Product Name: GEM103
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator: FS Development Corp
Deal Size: $216.0 million Upfront Cash: Undisclosed
Deal Type: Merger October 15, 2020
Details:
The company looking forward to topline data from this study and the potential to initiate a Phase 2 study supported by the CLARITY natural history study data.
Lead Product(s): GEM103
Therapeutic Area: Ophthalmology Product Name: Undisclosed
Highest Development Status: Phase I Product Type: Large molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable January 09, 2020