drugset

Drugs / BAMOCAFTOR

Follow last change Sep 2020 re-read 9 days ago

BAMOCAFTOR

also known as VX-659 · BAMOCAFTOR POTASSIUM · VX-659 POTASSIUM · VX-659 potassium salt

Small molecule targets CFTR via restoration

Developed for: cystic fibrosis
Investigated by: Vertex Pharmaceuticals Incorporated

Trials 6

today NCT03633526 · Ph 3 NCT03447262 · Ph 3 NCT03460990 · Ph 3 NCT03447249 · Ph 3 NCT03224351 · Ph 2 NCT03029455 · Ph 1

20172018201920202021202220232024202520262027

Phase	Registry id	Dates	Indication	Sponsor	Status	Outcome
Phase 11 trial · 1 met primary
Phase 1	NCT03029455	Nov 2016 → Aug 2017	cystic fibrosis	Vertex Pharmaceuticals Incorporated	Completed	Met primary
Phase 21 trial · 1 met primary
Phase 2	NCT03224351	Aug 2017 → Feb 2018	cystic fibrosis	Vertex Pharmaceuticals Incorporated	Completed	Met primary
Phase 34 trials · 2 met primary
Phase 3	NCT03633526	Aug 2018 → Jan 2019	cystic fibrosis	Vertex Pharmaceuticals Incorporated	Terminated	No outcome recorded
Phase 3	NCT03447262	Jul 2018 → Sep 2020	cystic fibrosis	Vertex Pharmaceuticals Incorporated	Terminated	No outcome recorded
Phase 3	NCT03460990	May → Sep 2018	cystic fibrosis	Vertex Pharmaceuticals Incorporated	Completed	Met primary
Phase 3	NCT03447249	Mar 2018 → Feb 2019	cystic fibrosis	Vertex Pharmaceuticals Incorporated	Completed	Met primary

News releases announcing trial results or a regulatory action · 6

Date	Issuer	Release
2018-11-27	Vertex Pharmaceuticals Incorporated	Results Two Phase 3 Studies of the Triple Combination of VX-659, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function (ppFEV1) in People with Cystic Fibrosis vrtx.com ↗
2018-10-18	Vertex Pharmaceuticals Incorporated	Results Vertex Data Presented at North American Cystic Fibrosis Conference (NACFC) Demonstrate Rapid Progress Toward Expanding and Enhancing Options for Treating the Underlying Cause of Cystic Fibrosis vrtx.com ↗ Phase 2 results of VX-659 and VX-445 triple combination regimens concurrently published in The New England Journal of Medicine
2018-06-07	Vertex Pharmaceuticals Incorporated	Results Vertex Data Presented at European Cystic Fibrosis Society (ECFS) Conference Demonstrate Early and Long-Term Disease-Modifying Potential of Treating the Underlying Cause of CF vrtx.com ↗ Studies of investigational triple combination regimens highlight continued progress toward developing medicines to treat the underlying cause of the disease for up to 90 percent of people living with CF
2018-02-01	Vertex Pharmaceuticals Incorporated	Results Vertex Selects Two Next-Generation Correctors, VX-659 and VX-445, to Advance into Phase 3 Development as Part of Two Different Triple Combination Regimens for People with Cystic Fibrosis vrtx.com ↗ Initial data from the ongoing VX-659 and VX-445 studies were announced today and are below.
2017-11-03	Vertex Pharmaceuticals Incorporated	Results Vertex Announces Presentations of Data at North American Cystic Fibrosis Conference that Demonstrate Important Progress Toward Goal of Helping All People with CF vrtx.com ↗ All studies showed statistically significant improvements in lung function (ppFEV1) with a triple combination regimen in these patients.
2017-07-18	Vertex Pharmaceuticals Incorporated	Results Vertex Announces Positive Phase 1 & Phase 2 Data from Three Different Triple Combination Regimens in People with Cystic Fibrosis Who Have One F508del Mutation and One Minimal Function Mutation (F508del/Min) vrtx.com ↗

All press releases naming this drug 9 releases

Date	Issuer	Release
2018-11-27	Vertex Pharmaceuticals Incorporated	Two Phase 3 Studies of the Triple Combination of VX-659, Tezacaftor and Ivacaftor Met Primary Endpoint of Improvement in Lung Function (ppFEV1) in People with Cystic Fibrosis vrtx.com ↗
2018-10-18	Vertex Pharmaceuticals Incorporated	Vertex Data Presented at North American Cystic Fibrosis Conference (NACFC) Demonstrate Rapid Progress Toward Expanding and Enhancing Options for Treating the Underlying Cause of Cystic Fibrosis vrtx.com ↗
2018-09-06	Vertex Pharmaceuticals Incorporated	Vertex Completes Enrollment of Two Phase 3 Studies of VX-659 in Triple Combination with Tezacaftor and Ivacaftor for the Treatment of Cystic Fibrosis vrtx.com ↗
2018-06-07	Vertex Pharmaceuticals Incorporated	Vertex Data Presented at European Cystic Fibrosis Society (ECFS) Conference Demonstrate Early and Long-Term Disease-Modifying Potential of Treating the Underlying Cause of CF vrtx.com ↗
2018-03-01	Vertex Pharmaceuticals Incorporated	Vertex Initiates Phase 3 Study of VX-659, Tezacaftor and Ivacaftor as a Triple Combination Regimen for People with Cystic Fibrosis Who Have Two Copies of the F508del Mutation vrtx.com ↗
2018-02-21	Vertex Pharmaceuticals Incorporated	Vertex Initiates First Phase 3 Study of VX-659, Tezacaftor and Ivacaftor as a Triple Combination Regimen for People with Cystic Fibrosis vrtx.com ↗
2018-02-01	Vertex Pharmaceuticals Incorporated	Vertex Selects Two Next-Generation Correctors, VX-659 and VX-445, to Advance into Phase 3 Development as Part of Two Different Triple Combination Regimens for People with Cystic Fibrosis vrtx.com ↗
2017-11-03	Vertex Pharmaceuticals Incorporated	Vertex Announces Presentations of Data at North American Cystic Fibrosis Conference that Demonstrate Important Progress Toward Goal of Helping All People with CF vrtx.com ↗
2017-07-18	Vertex Pharmaceuticals Incorporated	Vertex Announces Positive Phase 1 & Phase 2 Data from Three Different Triple Combination Regimens in People with Cystic Fibrosis Who Have One F508del Mutation and One Minimal Function Mutation (F508del/Min) vrtx.com ↗

Evidence & citations 9 cited values

Every value below carries the sentence it was read from. 8 sources stand behind the page.

Field	Value	Cited text
Known as	BAMOCAFTOR	ChEMBL registry synonym — accepted as the source's own label CHEMBL4297849 ↗
Known as	BAMOCAFTOR POTASSIUM	ChEMBL registry synonym — accepted as the source's own label CHEMBL4297849 ↗
Known as	VX-659	ClinicalTrials.gov intervention name — accepted as the source's own label NCT03447262 ↗ 5 NCT03224351 ↗ NCT03029455 ↗ NCT03633526 ↗ NCT03447249 ↗ NCT03460990 ↗
Known as	VX-659 POTASSIUM	ChEMBL registry synonym — accepted as the source's own label CHEMBL4297849 ↗
Known as	VX-659 potassium salt	ChEMBL registry synonym — accepted as the source's own label CHEMBL4297849 ↗
Action	Restore	“was developed to restore the function of Phe508del CFTR protein” PMID 30334693 ↗ Oct 2018
Modality	Small molecule	— CHEMBL4297849 ↗
Route	Oral	“A range of oral VX-659-tezacaftor-ivacaftor doses in triple combination were then evaluated” PMID 30334693 ↗ Oct 2018
Target	CFTR	“was developed to restore the function of Phe508del CFTR protein” PMID 30334693 ↗ Oct 2018