Simufilam 2HCl | CAS#2480226-06-8 | filamin A altered conformations binder

MedKoo Cat#: 126054 | Name: Simufilam 2HCl

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Description:

WARNING: This product is for research use only, not for human or veterinary use.

Simufilam, also known as PTI-125, is a filamin A altered conformations binder. PTI-125 is an oral small molecule drug candidate that binds and reverses an altered conformation of the scaffolding protein filamin A found in Alzheimer's disease brain. PTI-125 binds and reverses an altered conformation of filamin A to reduce Alzheimer's disease pathogenesis. PTI-125 also reduced tau hyperphosphorylation, aggregated Aβ42 deposition, neurofibrillary tangles, and neuroinflammation. Efficacy in postmortem AD and Aβ42-treated age-matched control hippocampal slices was concentration-dependent starting at 1 picomolar (pM) concentration. PTI-125 is the first therapeutic candidate to preferentially bind an altered protein conformation and reverse this proteopathy.

Chemical Structure

Simufilam 2HCl

CAS#2480226-06-8 (2HCl)

Theoretical Analysis

MedKoo Cat#: 126054

Name: Simufilam 2HCl

CAS#: 2480226-06-8 (2HCl)

Chemical Formula: C15H23Cl2N3O

Exact Mass: 0.0000

Molecular Weight: 332.27

Elemental Analysis: C, 54.22; H, 6.98; Cl, 21.34; N, 12.65; O, 4.82

Price and Availability

Size	Price	Availability	Quantity
5mg	USD 450.00	2 Weeks
10mg	USD 750.00	2 Weeks

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Related CAS #

2480226-07-9 (HCl) 1224591-33-6 (free base) 2375909-85-4 (HCl hydrate) 2480226-06-8 (2HCl)

Synonym

Simufilam 2HCl; Simufilam HCl; Simufilam (dihydrochloride); Simufilam hydrochloride anhydrous; simufilamum; simufilam; PTI125; PTI 125; PTI-125

IUPAC/Chemical Name

1-benzyl-8-methyl-1,4,8-triazaspiro[4.5]decan-2-one dihydrochloride

InChi Key

IZIPGYSLPYPJDQ-UHFFFAOYSA-N

InChi Code

InChI=1S/C15H21N3O.2ClH/c1-17-9-7-15(8-10-17)16-11-14(19)18(15)12-13-5-3-2-4-6-13;;/h2-6,16H,7-12H2,1H3;2*1H

SMILES Code

Cl.Cl.CN1CCC2(CC1)NCC(=O)N2CC3=CC=CC=C3

Appearance

To be determined

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

To be determined

Shelf Life

>2 years if stored properly

Drug Formulation

To be determined

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

More Info

Instruction

View handling instruction

Preparing Stock Solutions

The following data is based on the product molecular weight 332.27 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: Wang HY, Pei Z, Lee KC, Lopez-Brignoni E, Nikolov B, Crowley CA, Marsman MR, Barbier R, Friedmann N, Burns LH. PTI-125 Reduces Biomarkers of Alzheimer's Disease in Patients. J Prev Alzheimers Dis. 2020;7(4):256-264. doi: 10.14283/jpad.2020.6. PMID: 32920628. 2: Zhang L, Huang T, Teaw S, Nguyen LH, Hsieh LS, Gong X, Burns LH, Bordey A. Filamin A inhibition reduces seizure activity in a mouse model of focal cortical malformations. Sci Transl Med. 2020 Feb 19;12(531):eaay0289. doi: 10.1126/scitranslmed.aay0289. PMID: 32075941. 3: Wang HY, Lee KC, Pei Z, Khan A, Bakshi K, Burns LH. PTI-125 binds and reverses an altered conformation of filamin A to reduce Alzheimer's disease pathogenesis. Neurobiol Aging. 2017 Jul;55:99-114. doi: 10.1016/j.neurobiolaging.2017.03.016. Epub 2017 Mar 31. PMID: 28438486. 4: Burns LH, Wang HY. Altered filamin A enables amyloid beta-induced tau hyperphosphorylation and neuroinflammation in Alzheimer's disease. Neuroimmunol Neuroinflamm. 2017;4(12):263-271. doi: 10.20517/2347-8659.2017.50. Epub 2017 Dec 8. PMID: 34295950; PMCID: PMC8294116. 5: Wang HY, Bakshi K, Frankfurt M, Stucky A, Goberdhan M, Shah SM, Burns LH. Reducing amyloid-related Alzheimer's disease pathogenesis by a small molecule targeting filamin A. J Neurosci. 2012 Jul 18;32(29):9773-84. doi: 10.1523/JNEUROSCI.0354-12.2012. Erratum in: J Neurosci. 2021 Dec 15;41(50):10405. Erratum in: J Neurosci. 2022 Jan 19;42(3):529. PMID: 22815492; PMCID: PMC6621293.