MedKoo Cat#: 526829 | Name: Branaplam mesylate

Description:

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

Branaplam, also known as NVS-SM1 and LMI-070, is a highly potent, selective and orally active small molecule SMN2 splicing modulator as a potential treatment for SMA Type 1 (Spinal Muscular Atrophy) .NVS-SM1 works by increasing the amount of functional SMN protein produced by the “back-up” gene, SMN2, through modifying its splicing.

Chemical Structure

Branaplam mesylate
Branaplam mesylate
CAS#Branaplam mesylate

Theoretical Analysis

MedKoo Cat#: 526829

Name: Branaplam mesylate

CAS#: Branaplam mesylate

Chemical Formula: C23H31N5O5S

Exact Mass:

Molecular Weight: 489.59

Elemental Analysis: C, 56.43; H, 6.38; N, 14.30; O, 16.34; S, 6.55

Price and Availability

This product is currently not in stock but may be available through custom synthesis. To ensure cost efficiency, the minimum order quantity is 1 gram. The estimated lead time is 2 to 4 months, with pricing dependent on the complexity of the synthesis (typically high for intricate chemistries). Quotes for quantities below 1 gram will not be provided. To request a quote, please click the button below. Note: If this product becomes available in stock in the future, pricing will be listed accordingly.
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Synonym
LMI070; LMI-070; LMI 070; Branaplam mesylate
IUPAC/Chemical Name
(6Z)-3-(1H-pyrazol-4-yl)-6-[3-(2,2,6,6-tetramethylpiperidin-4-yl)oxy-1H-pyridazin-6-ylidene]cyclohexa-2,4-dien-1-one mesylate
InChi Key
YIDPCWNMJPTXAZ-YBFBCAGJSA-N
InChi Code
InChI=1S/C22H27N5O2.CH4O3S/c1-21(2)10-16(11-22(3,4)27-21)29-20-8-7-18(25-26-20)17-6-5-14(9-19(17)28)15-12-23-24-13-15;1-5(2,3)4/h5-9,12-13,16,25,27H,10-11H2,1-4H3,(H,23,24);1H3,(H,2,3,4)/b18-17-;
SMILES Code
O=C1C=C(C2=CNN=C2)C=C/C1=C3C=CC(OC4CC(C)(C)NC(C)(C)C4)=NN\3.OS(=O)(C)=O
Appearance
Solid powder
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
>2 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
More Info

Preparing Stock Solutions

The following data is based on the product molecular weight 489.59 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.

Recalculate based on batch purity %
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: Cheung AK, Hurley B, Kerrigan R, Shu L, Chin DN, Shen Y, O'Brien G, Sung MJ, Hou Y, Axford J, Cody E, Sun R, Fazal A, Tomlinson RC, Jain M, Deng L, Hoffmaster K, Song C, Van Hoosear M, Shin Y, Servais R, Towler CS, Hild M, Curtis D, Dietrich WF, Hamann LG, Briner K, Chen KS, Kobayashi D, Sivasankaran R, Dales NA. Discovery of small molecule splicing modulators of survival motor neuron-2 (SMN2) for the treatment of Spinal Muscular Atrophy (SMA). J Med Chem. 2018 Nov 8. doi: 10.1021/acs.jmedchem.8b01291. [Epub ahead of print] PubMed PMID: 30407821. 2: Stam M, Wadman RI, Wijngaarde CA, Bartels B, Asselman FL, Otto LAM, Goedee HS, Habets LE, de Groot JF, Schoenmakers MAGC, Cuppen I, van den Berg LH, van der Pol WL. Protocol for a phase II, monocentre, double-blind, placebo-controlled, cross-over trial to assess efficacy of pyridostigmine in patients with spinal muscular atrophy types 2-4 (SPACE trial). BMJ Open. 2018 Jul 30;8(7):e019932. doi: 10.1136/bmjopen-2017-019932. PubMed PMID: 30061431; PubMed Central PMCID: PMC6067401. 3: Calder AN, Androphy EJ, Hodgetts KJ. Small Molecules in Development for the Treatment of Spinal Muscular Atrophy. J Med Chem. 2016 Nov 23;59(22):10067-10083. Epub 2016 Aug 16. Review. PubMed PMID: 27490705; PubMed Central PMCID: PMC5744254.