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Details:
BBP-418, an investigational oral substrate supplementation therapy, upstream of the mutant FKRP enzyme to help drive residual activity of the enzyme to glycosylate αDG in patients with limb-girdle muscular dystrophy type 2I (LGMD2I/R9).
Lead Product(s): Ribitol
Therapeutic Area: Genetic Disease Product Name: BBP-418
Highest Development Status: Phase IIIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable July 31, 2023
Details:
43% increase in ratio of glycosylated alpha-dystroglycan to total αDG from baseline were measured for BBP-418, across all three dosing cohorts, signifying the oral therapy has potential to address both the root cause of LGMD2i and drive functional improvements for patients.
Lead Product(s): Ribitol
Therapeutic Area: Genetic Disease Product Name: BBP-418
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 14, 2022
Details:
BBP-418 is being investigated to function as a substrate supplementation therapy that is hypothesized to drive glycosylation of αDG and target the root cause of the disease.
Lead Product(s): Ribitol
Therapeutic Area: Genetic Disease Product Name: BBP-418
Highest Development Status: Phase IIProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable March 11, 2022
Details:
The Phase 1 clinical trial is designed to assess safety, tolerability, pharmacokinetics and food effect of BBP-418 in healthy volunteers.
Lead Product(s): Ribitol
Therapeutic Area: Genetic Disease Product Name: Undisclosed
Highest Development Status: Phase IProduct Type: Small molecule
Partner/Sponsor/Collaborator: Not Applicable
Deal Size: Not Applicable Upfront Cash: Not Applicable
Deal Type: Not Applicable June 11, 2020
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