A Study of VRG50635 in Healthy Volunteers

Summary

The primary objectives of this study are to investigate the safety and tolerability of VRG50635 and to determine how VRG50635 is absorbed by the body.

Timeline

Start

2024-02-29

Primary completion

2024-03-25

Completion

2024-04-08

Publications

• Background Hung ST, Linares GR, Chang WH, Eoh Y, Krishnan G, Mendonca S, Hong S, Shi Y, Santana M, Kueth C, Macklin-Isquierdo S, Perry S, Duhaime S, Maios C, Chang J, Perez J, Couto A, Lai J, Li Y, Alworth SV, Hendricks E, Wang Y, Zlokovic BV, Dickman DK, Parker JA, Zarnescu DC, Gao FB, Ichida JK. PIKFYVE inhibition mitigates disease in models of diverse forms of ALS. Cell. 2023 Feb 16;186(4):786-802.e28. doi: 10.1016/j.cell.2023.01.005. Epub 2023 Feb 7.
• Background Shi Y, Lin S, Staats KA, Li Y, Chang WH, Hung ST, Hendricks E, Linares GR, Wang Y, Son EY, Wen X, Kisler K, Wilkinson B, Menendez L, Sugawara T, Woolwine P, Huang M, Cowan MJ, Ge B, Koutsodendris N, Sandor KP, Komberg J, Vangoor VR, Senthilkumar K, Hennes V, Seah C, Nelson AR, Cheng TY, Lee SJ, August PR, Chen JA, Wisniewski N, Hanson-Smith V, Belgard TG, Zhang A, Coba M, Grunseich C, Ward ME, van den Berg LH, Pasterkamp RJ, Trotti D, Zlokovic BV, Ichida JK. Haploinsufficiency leads to neurodegeneration in C9ORF72 ALS/FTD human induced motor neurons. Nat Med. 2018 Mar;24(3):313-325. doi: 10.1038/nm.4490. Epub 2018 Feb 5.
• Gontier G, Kim G, Wang C, Zhu K, Gau R, Zhang N, Naphade S, Stewart A, Schmidt MJ, Galemmo R, Shook B, Tarachandani A, Choi I, Raines S, Scannevin RH, Grievink HW, Smits LMG, Kremer PHC, Cadavid D. Glycoprotein Non-Metastatic Melanoma Protein B (GPNMB): A Translational Pharmacodynamic Biomarker for PIKfyve Inhibition With VRG50635. Clin Transl Sci. 2026 Feb;19(2):e70489. doi: 10.1111/cts.70489.

Drugs

Indications

No indication recorded.