Branaplam free base
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MedKoo CAT#: 526827

CAS#: 1562338-42-4 (free base)

Description: 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.


Price and Availability

Size
Price

25mg
USD 250
200mg
USD 950
2g
USD 4650
Size
Price

50mg
USD 450
500mg
USD 1650
5g
USD 7650
Size
Price

100mg
USD 650
1g
USD 2650

Branaplam, known as NVS-SM1 and LMI-070, purity > 98%, is in stock. The same day shipping out after order is received.


Chemical Structure

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Theoretical Analysis

MedKoo Cat#: 526827
Name: Branaplam free base
CAS#: 1562338-42-4 (free base)
Chemical Formula: C22H27N5O2
Exact Mass: 393.2165
Molecular Weight: 393.491
Elemental Analysis: C, 67.15; H, 6.92; N, 17.80; O, 8.13


Related CAS #: 1562338-42-4 (free base)   1562338-39-9 (HCl)    

Synonym: NVS-SM1; LMI-070; NVSSM1; LMI070; NVS SM1; LMI 070; Branaplam

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

InChi Key: YIFFDXMJVNKGBL-ZCXUNETKSA-N

InChi Code: InChI=1S/C22H27N5O2/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/h5-9,12-13,16,25,27H,10-11H2,1-4H3,(H,23,24)/b18-17-

SMILES Code: O=C1C=C(C2=CNN=C2)C=C/C1=C3C=CC(OC4CC(C)(C)NC(C)(C)C4)=NN\3


Technical Data

Appearance:
Solid powder

Purity:
>98% (or refer to the Certificate of Analysis)

Certificate of Analysis:

Safety Data Sheet (MSDS):

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).

Harmonized System Code:
2934990300


References

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.