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  3. Branaplam

Branaplam (Synonyms: LMI070; NVS-SM1)

Cat. No.: HY-19620 Purity: 99.67%
Handling Instructions

Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model.

For research use only. We do not sell to patients.

Branaplam Chemical Structure

Branaplam Chemical Structure

CAS No. : 1562338-42-4

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Solution
10 mM * 1 mL in DMSO USD 132 In-stock
Estimated Time of Arrival: December 31
Solid + Solvent
10 mM * 1 mL
ready for reconstitution
USD 132 In-stock
Estimated Time of Arrival: December 31
Solid
5 mg USD 120 In-stock
Estimated Time of Arrival: December 31
10 mg USD 190 In-stock
Estimated Time of Arrival: December 31
50 mg USD 490 In-stock
Estimated Time of Arrival: December 31
100 mg USD 690 In-stock
Estimated Time of Arrival: December 31
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Based on 4 publication(s) in Google Scholar

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Description

Branaplam (LMI070; NVS-SM1) is a highly potent, selective and orally active survival motor neuron-2 (SMN2) splicing modulator with an EC50 of 20 nM for SMN. Branaplam inhibits human-ether-a-go-go-related gene (hERG) with an IC50 of 6.3 μM. Branaplam elevates full-length SMN protein and extends survival in a severe spinal muscular atrophy (SMA) mouse model[1][2].

IC50 & Target

IC50: 20 nM (SMN)[1]
EC50: 6.3 μM (hERG)[2]

In Vitro

Branaplam (LMI070; NVS-SM1) treatment induces changes in the levels of 175 genes in human fibroblasts[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

In Vivo

Branaplam (LMI070; NVS-SM1; 3, 10, 30 mg/kg; oral) produces dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord[1].
Branaplam (1 mg/kg of IV; 3 mg/kg of PO) has a CL of 25 mL/min/kg and an AUC of 3.03 μM•h[2].
A single Branaplam (oral; 30 mg/kg) results in significant and durable SMN protein elevation in brain for up to 160 hours in C/+ mice[1].
Branaplam (oral; 0.03, 0.1, 0.3, 1, 3 mg/kg) improves body weight and extendes lifespan in n SMNΔ7 mice[1].

MCE has not independently confirmed the accuracy of these methods. They are for reference only.

Animal Model: C/+ SMA mouse model[1]
Dosage: 3, 10, 30 mg/kg
Administration: Oral
Result: Produced dose-dependent elevations of SMN2-FL transcript and SMN protein in brain and spinal cord.
Animal Model: Male Sprague-Dawley rat[2]
Dosage: 1 mg/kg (IV);3 mg/kg (PO) (Pharmacokinetic Analysis)
Administration: IV or PO
Result: Had a CL of 25 mL/min/kg and an AUC of 3.03 μM•h.
Clinical Trial
Molecular Weight

393.48

Formula

C₂₂H₂₇N₅O₂

CAS No.
Shipping

Room temperature in continental US; may vary elsewhere.

Storage

4°C, sealed storage, away from moisture

*In solvent : -80°C, 6 months; -20°C, 1 month (sealed storage, away from moisture)

Solvent & Solubility
In Vitro: 

DMSO : 6.12 mg/mL (15.55 mM; Need ultrasonic and warming)

Preparing
Stock Solutions
Concentration Solvent Mass 1 mg 5 mg 10 mg
1 mM 2.5414 mL 12.7071 mL 25.4143 mL
5 mM 0.5083 mL 2.5414 mL 5.0829 mL
10 mM 0.2541 mL 1.2707 mL 2.5414 mL
*Please refer to the solubility information to select the appropriate solvent.
In Vivo:
  • 1.

    Add each solvent one by one:  10% DMSO    40% PEG300    5% Tween-80    45% saline

    Solubility: ≥ 1 mg/mL (2.54 mM); Clear solution

  • 2.

    Add each solvent one by one:  10% DMSO    90% (20% SBE-β-CD in saline)

    Solubility: ≥ 0.71 mg/mL (1.80 mM); Clear solution

  • 3.

    Add each solvent one by one:  10% DMSO    90% corn oil

    Solubility: ≥ 0.71 mg/mL (1.80 mM); Clear solution

  • 4.

    Add each solvent one by one:  5% DMSO    40% PEG300    5% Tween-80    50% saline

    Solubility: ≥ 0.57 mg/mL (1.45 mM); Clear solution

  • 5.

    Add each solvent one by one:  1% DMSO    99% saline

    Solubility: 0.12 mg/mL (0.30 mM); Suspended solution; Need ultrasonic

*All of the co-solvents are provided by MCE.
References
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Product Name:
Branaplam
Cat. No.:
HY-19620
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