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[{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma\u2019s ML Bio Solutions Dosed First Subject in Phase 1 Clinical Trial of BBP-418 For Limb Girdle Muscular Dystrophy Type 2i","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase I","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"June 2020","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase I"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma, Inc. to Host Investor Call to Discuss Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"Bridge Biotherapeutics","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Announces Positive Phase 2 Data for Limb-girdle Muscular Dystrophy Type 2i (LGMD2i)","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase II","country":"SOUTH KOREA","productType":"Small molecule","productStatus":"New Molecular Entity","date":"March 2022","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase II"},{"orgOrder":0,"company":"BridgeBio Pharma","sponsor":"Not Applicable","pharmaFlowCategory":"DU","amount":"Not Applicable","upfrontCash":"Not Applicable","newsHeadline":"BridgeBio Pharma Announces Opportunity for Accelerated Approval Pathway in Limb-girdle Muscular Dystrophy Type 2I (LGMD2I\/R9) Based on Glycosylated Alpha-dystroglycan (lgmd2i\/r9) Levels and Announces First Patient Dosed in FORTIFY Phase 3 Study","therapeuticArea":"Genetic Disease","highestDevelopmentStatus":"Phase III","country":"U.S.A","productType":"Small molecule","productStatus":"New Molecular Entity","date":"July 2023","url1":"","url2":"","graph1":"Genetic Disease","graph2":"Phase III"}]
Find Clinical Drug Pipeline Developments & Deals for Ribitol
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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 III
Product 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 II
Product 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 II
Product 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 I
Product Type: Small molecule
Partner/Sponsor/Collaborator:
Not Applicable
Deal Size: Not Applicable
Upfront Cash: Not Applicable
Deal Type: Not Applicable
June 11, 2020