Brelgometon

Brelgometon
Clinical data
Other namesATH-1105; ATH1105
Drug classHepatocyte growth factor (HGF)–c-MET signaling positive modulator; Neuroprotective agent; Anti-inflammatory drug
Identifiers
  • (6S)-6-methyl-8-[(2S)-2-methylbutyl]-1-[[4-(trifluoromethyl)phenyl]methyl]-3,6,9,9a-tetrahydro-2H-pyrazino[1,2-a]pyrimidine-4,7-dione
PubChem CID
ChemSpider
Chemical and physical data
FormulaC21H28F3N3O2
Molar mass411.469 g·mol−1
3D model (JSmol)
  • CC[C@H](C)CN1CC2N(CCC(=O)N2[C@H](C1=O)C)CC3=CC=C(C=C3)C(F)(F)F
  • InChI=1S/C21H28F3N3O2/c1-4-14(2)11-26-13-18-25(10-9-19(28)27(18)15(3)20(26)29)12-16-5-7-17(8-6-16)21(22,23)24/h5-8,14-15,18H,4,9-13H2,1-3H3/t14-,15-,18?/m0/s1
  • Key:DWLDFDZUDSQLPP-INHRHCAVSA-N

Brelgometon (INNTooltip International Nonproprietary Name; developmental code name ATH-1105) is a positive modulator of hepatocyte growth factor (HGF)–c-MET signaling which is under development for the treatment of amyotrophic lateral sclerosis (ALS) and frontotemporal dementia (FTD). It is a small-molecule and has favorable drug-like properties, such as oral bioavailability and blood–brain barrier permeabiliy. The drug shows potent neuroprotective and anti-inflammatory effects in preclinical research, including in models of ALS. It was originated by Athira Pharma and is under development by LeonaBio. As of April 2026, the drug is in phase 1 clinical trials for ALS and is in the preclinical research stage of development for FTD. A phase 2 trial for ALS is being planned for the second half of 2026.

See also

References

  1. 1 2 3 4 "Brelgometon - LeonaBio". adisinsight.springer.com. Retrieved 27 October 2026.
  2. 1 2 3 "Delving into the Latest Updates on ATH-1105 with Synapse". Synapse. 24 October 2026. Retrieved 27 October 2026.
  3. 1 2 3 Berthiaume AA, Reda SM, Kleist KN, Setti SE, Wu W, Johnston JL, Taylor RW, Stein LR, Moebius HJ, Church KJ (2024). "ATH-1105, a small-molecule positive modulator of the neurotrophic HGF system, is neuroprotective, preserves neuromotor function, and extends survival in preclinical models of ALS". Front Neurosci. 18 1348157. doi:10.3389/fnins.2024.1348157. PMC 10881713. PMID 38389786.
  4. 1 2 3 Berthiaume AA, Kleist KN, Reda SM, Setti SE, Wu W, Johnston JL, Taylor RW, Stein LR, Church KJ (2025). "ATH-1105 mitigates multiple pathologies in ALS models both alone and in combination with riluzole". Front Neurol. 16 1582765. doi:10.3389/fneur.2025.1582765. PMC 12825224. PMID 41586107.