PBT2

PBT2

Names
IUPAC name 5,7-Dichloro-2-[(dimethylamino)methyl]quinolin-8-ol
Identifiers
3D model (Jmol)	Interactive image
ChemSpider	8191585
PubChem	10016012
UNII	Q7K6GJQ4O4
InChI InChI=1S/C12H12Cl2N2O/c1-16(2)6-7-3-4-8-9(13)5-10(14)12(17)11(8)15-7/h3-5,17H,6H2,1-2H3 Key: YZPOQCQXOSEMAZ-UHFFFAOYSA-N
SMILES CN(C)CC1=NC2=C(C=C1)C(=CC(=C2O)Cl)Cl
Properties
Chemical formula	C12H12Cl2N2O
Molar mass	271.14 g·mol^−1
Except where otherwise noted, data are given for materials in their standard state (at 25 °C [77 °F], 100 kPa).
Infobox references

PBT2 is an experimental drug candidate. It is a second-generation 8-hydroxyquinoline analog^[1] intended to be a successor to clioquinol and a potential treatment of Alzheimer's disease.^[2]

Clinical trials

PBT2 was the subject of phase II clinical trials for Alzheimer's disease and Huntington's disease. The results for Alzheimer's disease failed to meet its goals,^[3][4] and there is no evidence that PBT2 is of any benefit in Alzheimer's dementia.^[5] The drug also missed efficacy goals for Huntington's disease.^[6][7]

References

1. Adlard, Paul A.; Cherny, Robert A.; Finkelstein, David I.; Gautier, Elisabeth; Robb, Elysia; Cortes, Mikhalina; Volitakis, Irene; Liu, Xiang; et al. (2008). "Rapid Restoration of Cognition in Alzheimer's Transgenic Mice with 8-Hydroxy Quinoline Analogs is Associated with Decreased Interstitial Aβ". Neuron. 59 (1): 43–55. doi:10.1016/j.neuron.2008.06.018. PMID 18614028.
2. Duce, James A.; Tsatsanis, Andrew; Cater, Michael A.; James, Simon A.; Robb, Elysia; Wikhe, Krutika; Leong, Su Ling; Perez, Keyla; et al. (2010). "Iron-Export Ferroxidase Activity of β-Amyloid Precursor Protein is Inhibited by Zinc in Alzheimer's Disease". Cell. 142 (6): 857–67. doi:10.1016/j.cell.2010.08.014. PMC 2943017. PMID 20817278. Lay summary – ScienceDaily (September 10, 2010).
3. "Prana Biotech Plunges 76%; Drug Fails in Alzheimer Study". Bloomberg News. Mar 31, 2014.
4. "PBT2 Takes a Dive in Phase 2 Alzheimer's Trial". April 2014.
5. "There is no evidence that MPACs (PBT1 or PBT2) are of benefit in Alzheimer's dementia". Cochrane Database of Systematic Reviews: Plain Language Summaries. PubMed Health.
6. "Prana Bio Huntington's Disease Drug Fails Key Efficacy Hurdles". thestreet.com.
7. Huntington Study Group Reach2HD Investigators (2015). "Safety, tolerability, and efficacy of PBT2 in Huntington's disease: A phase 2, randomised, double-blind, placebo-controlled trial". The Lancet Neurology. 14 (1): 39–47. doi:10.1016/S1474-4422(14)70262-5. PMID 25467848.