Synonym
AT-007; AT 007; AT007; Govorestat
IUPAC/Chemical Name
2-(4-oxo-3-((5-(trifluoromethyl)benzo[d]thiazol-2-yl)methyl)-3,4-dihydrothieno[3,4-d]pyridazin-1-yl)acetic acid
InChi Key
ORQGHAJIWGGFJK-UHFFFAOYSA-N
InChi Code
InChI=1S/C17H10F3N3O3S2/c18-17(19,20)8-1-2-13-12(3-8)21-14(28-13)5-23-16(26)10-7-27-6-9(10)11(22-23)4-15(24)25/h1-3,6-7H,4-5H2,(H,24,25)
SMILES Code
O=C(O)CC(C1=CSC=C12)=NN(CC3=NC4=CC(C(F)(F)F)=CC=C4S3)C2=O
Purity
>98% (or refer to the Certificate of Analysis)
Shipping Condition
Shipped under ambient temperature as non-hazardous chemical. This product is stable enough for a few weeks during ordinary shipping and time spent in Customs.
Storage Condition
Dry, dark and at 0 - 4 C for short term (days to weeks) or -20 C for long term (months to years).
Solubility
Soluble in DMSO
Shelf Life
>3 years if stored properly
Drug Formulation
This drug may be formulated in DMSO
Stock Solution Storage
0 - 4 C for short term (days to weeks), or -20 C for long term (months).
HS Tariff Code
2934.99.9001
Preparing Stock Solutions
The following data is based on the
product
molecular weight
425.40
Batch specific molecular weights may vary
from batch to batch
due to the degree of hydration, which will
affect the solvent
volumes required to prepare stock solutions.
| Concentration / Solvent Volume / Mass |
1 mg |
5 mg |
10 mg |
| 1 mM |
1.15 mL |
5.76 mL |
11.51 mL |
| 5 mM |
0.23 mL |
1.15 mL |
2.3 mL |
| 10 mM |
0.12 mL |
0.58 mL |
1.15 mL |
| 50 mM |
0.02 mL |
0.12 mL |
0.23 mL |
1: Zhu Y, Lobato AG, Rebelo AP, Canic T, Ortiz-Vega N, Tao X, Syed S, Yanick C,
Saporta M, Shy M, Perfetti R, Shendelman S, Züchner S, Zhai RG. Sorbitol
reduction via govorestat ameliorates synaptic dysfunction and neurodegeneration
in sorbitol dehydrogenase deficiency. JCI Insight. 2023 May 22;8(10):e164954.
doi: 10.1172/jci.insight.164954. PMID: 37014713; PMCID: PMC10322690.
2: Perfetti R, Bailey E, Wang S, Mills R, Mohanlal R, Shendelman S. Safety,
Pharmacokinetics, and Pharmacodynamics of the New Aldose Reductase Inhibitor
Govorestat (AT-007) After a Single and Multiple Doses in Participants in a Phase
1/2 Study. J Clin Pharmacol. 2024 Nov;64(11):1397-1406. doi: 10.1002/jcph.2495.
Epub 2024 Jul 10. PMID: 38988185.
3: Bailey E, Phan H, Ahmad A, Thomas J, Ames EG, Pritchard AB, Quinonez SC, Wang
S, Dayley C, Salt A, Pick C, Durrant A, Johnson S, Nicodemus-Johnson J, Dickson
SP, Perfetti R, Hendrix SB, Shendelman S. Results of the ACTION-Galactosemia
Kids Study to Evaluate the Effects of Govorestat in Pediatric Patients with
Classic Galactosemia. J Clin Pharmacol. 2024 Nov 21. doi: 10.1002/jcph.6170.
Epub ahead of print. PMID: 39569553.
4: Randall JA, Sutter C, Wang S, Bailey E, Raither L, Perfetti R, Shendelman S,
Burbridge C. Qualitative interviews with adults with Classic Galactosemia and
their caregivers: disease burden and challenges with daily living. Orphanet J
Rare Dis. 2022 Mar 28;17(1):138. doi: 10.1186/s13023-022-02287-9. PMID:
35346295; PMCID: PMC8959560.
