BBP-671
/ BridgeBio
- LARVOL DELTA
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April 27, 2025
Relief of CoA Sequestration by PANKs Agonist As a Potential Treatment for Acidemias
(ENDO 2025)
- "Moreover GSC003896 could further reduce C3:C2 ratio, comparing to BBP-671. GenSci compounds exhibit a favorable ADME profiles and are under active IND-enabling study."
Genetic Disorders • Metabolic Disorders • Ophthalmology
August 13, 2024
Development of Brain Penetrant Pyridazine Pantothenate Kinase Activators.
(PubMed, J Med Chem)
- "These studies led to the prioritization of three late-stage preclinical lead PANK modulators with improved pharmacokinetic profiles and the ability to substantially increase brain CoA levels. Compound 22 (BBP-671) eventually advanced into clinical testing for the treatment of PKAN and propionic acidemia."
Journal • CNS Disorders
December 19, 2023
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
(clinicaltrials.gov)
- P1 | N=79 | Terminated | Sponsor: CoA Therapeutics, Inc., a BridgeBio company | N=128 ➔ 79 | Recruiting ➔ Terminated; Slow recruitment
Enrollment change • Trial termination • Genetic Disorders • Metabolic Disorders • Ophthalmology
October 25, 2023
Pantothenate kinase activation restores brain coenzyme A in a mouse model of pantothenate kinase associated neurodegeneration.
(PubMed, J Pharmacol Exp Ther)
- "BBP-671 crosses the blood brain barrier to correct the neuron-specific CoA deficiency and improve motor function in a mouse model of pantothenate kinase associated neurodegeneration. The central role of CoA and acetyl-CoA in intermediary metabolism suggests that pantothenate kinase activators may be useful in modifying neurological metabolic disorders."
Journal • Preclinical • CNS Disorders • Dystonia • Metabolic Disorders • Movement Disorders • Parkinson's Disease • Solid Tumor • ANK2
January 13, 2023
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
(clinicaltrials.gov)
- P1 | N=128 | Recruiting | Sponsor: CoA Therapeutics, Inc., a BridgeBio company | Trial completion date: May 2023 ➔ Dec 2023 | Trial primary completion date: May 2023 ➔ Dec 2023
Trial completion date • Trial primary completion date • Genetic Disorders • Metabolic Disorders • Ophthalmology
October 18, 2022
Relief of CoA sequestration and restoration of mitochondrial function in a mouse model of propionic acidemia.
(PubMed, J Inherit Metab Dis)
- "Here, we use a hypomorphic PA mouse model to test the effectiveness of BBP-671 in correcting the metabolic imbalances in PA...Furthermore, the low survival of PA mice is restored to normal by BBP-671. These data show that BBP-671 relieves CoA sequestration, improves mitochondrial function, reduces plasma PA biomarkers and extends the lifespan of PA mice, providing the preclinical foundation for the therapeutic potential of BBP-671."
Journal • Preclinical
October 11, 2022
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
(clinicaltrials.gov)
- P1 | N=128 | Recruiting | Sponsor: CoA Therapeutics, Inc., a BridgeBio company | Trial completion date: Jan 2023 ➔ May 2023 | Trial primary completion date: Jan 2023 ➔ May 2023
Trial completion date • Trial primary completion date • Genetic Disorders • Metabolic Disorders • Ophthalmology
September 22, 2022
BBP-671, AN INVESTIGATIONAL MODULATOR OF PANTOTHENATE KINASES, DEMONSTRATES PROOF OF CONCEPT IN A PKAN MOUSE MODEL AND TARGET ENGAGEMENT IN HUMANS
(MDS Congress 2022)
- P1 | "In a PKAN mouse model, BBP-671 improved both survival and movement. In healthy human subjects, BBP-671 was generally well-tolerated and detected in plasma and CSF at concentrations at which efficacy was observed in dKO mice. Increases in WB acetyl-CoA observed with BBP-671 demonstrated target engagement and proof of mechanism."
Preclinical • CNS Disorders
June 24, 2022
BBP-671, A Potential First-in-Class, Potent, Allosteric Modulator of Human Pantothenate Kinases, is Well-Tolerated and Deomonstrates Target Engagement in Healthy Volunteers
(MDS-PAS 2022)
- P1 | "BBP-671 is an oral, potentially first-in-class, novel, potent, allosteric modulator of human PanKs specifically developed for the treatment of diseases associated with CoA deficiency. BBP-671 was safe and well-tolerated following oral dosing in healthy volunteers. BBP-671 was detected in healthy volunteer plasma and CSF."
Clinical • CNS Disorders
April 15, 2022
A First in Human, Dose Escalation Study to Evaluate the Safety and Tolerability of BBP-671 in Healthy Volunteers and Patients With Propionic Acidemia or Methylmalonic Acidemia
(clinicaltrials.gov)
- P1 | N=128 | Recruiting | Sponsor: CoA Therapeutics, Inc., a BridgeBio company | Trial completion date: Jul 2022 ➔ Jan 2023 | Trial primary completion date: Jul 2022 ➔ Jan 2023
Trial completion date • Trial primary completion date • Genetic Disorders • Metabolic Disorders • Ophthalmology
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