MedKoo Cat#: 599108 | Name: Branaplam HCl
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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 HCl
Branaplam HCl
CAS#1562338-39-9 (HCl)

Theoretical Analysis

MedKoo Cat#: 599108

Name: Branaplam HCl

CAS#: 1562338-39-9 (HCl)

Chemical Formula: C22H28ClN5O2

Exact Mass: 429.1932

Molecular Weight: 429.94

Elemental Analysis: C, 61.46; H, 6.56; Cl, 8.25; N, 16.29; O, 7.44

Price and Availability

Size Price Availability Quantity
10mg USD 400.00 2 Weeks
50mg USD 950.00 2 Weeks
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Synonym
Branaplam hydrochloride; Branaplam HCl; LMI-070 hydrochloride; LMI-070 HCl; LMI 070 HCl; LMI070 HCl;
IUPAC/Chemical Name
5-(1H-pyrazol-4-yl)-2-(6-((2,2,6,6-tetramethylpiperidin-4-yl)oxy)pyridazin-3-yl)phenol hydrochloride
InChi Key
XJIMIVJABPKGIY-UHFFFAOYSA-N
InChi Code
InChI=1S/C22H27N5O2.ClH/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;/h5-9,12-13,16,27-28H,10-11H2,1-4H3,(H,23,24);1H
SMILES Code
OC1=CC(C2=CNN=C2)=CC=C1C3=NN=C(OC4CC(C)(C)NC(C)(C)C4)C=C3.[H]Cl
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
>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
More Info

Preparing Stock Solutions

The following data is based on the product molecular weight 429.94 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.