Vamorolone | VBP-15 | CAS#13209-41-1 | Muscular Dystrophy Anti-Inflammatory

MedKoo Cat#: 327030 | Name: Vamorolone

Description:

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

Vamorolone, also known as VBP-15, is an anti-inflammatory compound used in the treatment of muscular dystrophy. Demonstrates potent inhibition of pro-inflammatory cytokines, including IL-6 and TNF-α, in macrophage cell lines.Vamorolone demonstrates potent inhibition of pro-inflammatory cytokines, including IL-6 and TNF-α, in macrophage cell lines. Vamorolone stabilizes cell membranes and reduces oxidative stress markers in skeletal muscle cells. Vamorolone preserves muscle function and reduces inflammation without the severe bone toxicity observed with classical corticosteroids.

Chemical Structure

Vamorolone

CAS#13209-41-1 (free base)

Theoretical Analysis

MedKoo Cat#: 327030

Name: Vamorolone

CAS#: 13209-41-1 (free base)

Chemical Formula: C22H28O4

Exact Mass: 356.1988

Molecular Weight: 356.46

Elemental Analysis: C, 74.13; H, 7.92; O, 17.95

Price and Availability

Size	Price	Availability
10mg	USD 150.00	Ready to ship
25mg	USD 250.00	Ready to ship
50mg	USD 450.00	Ready to ship
100mg	USD 750.00	Ready to ship
200mg	USD 1,250.00	Ready to ship
500mg	USD 2,850.00	Ready to ship
1g	USD 4,250.00	Ready to ship
2g	USD 7,250.00	Ready to ship

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Related CAS #

10106-41-9 (acetate) 13209-41-1 (free base)

Synonym

Vamorolone, VBP-15, VBP15, VBP 15

IUPAC/Chemical Name

(8S,10S,13S,14S,16R,17R)-17-hydroxy-17-(2-hydroxyacetyl)-10,13,16-trimethyl-6,7,8,10,12,13,14,15,16,17-decahydro-3H-cyclopenta[a]phenanthren-3-one

InChi Key

ZYTXTXAMMDTYDQ-DGEXFFLYSA-N

InChi Code

InChI=1S/C22H28O4/c1-13-10-18-16-5-4-14-11-15(24)6-8-20(14,2)17(16)7-9-21(18,3)22(13,26)19(25)12-23/h6-8,11,13,16,18,23,26H,4-5,9-10,12H2,1-3H3/t13-,16-,18+,20+,21+,22+/m1/s1

SMILES Code

C[C@@]12[C@](C(CO)=O)(O)[C@H](C)C[C@@]1([H])[C@]3([H])CCC4=CC(C=C[C@]4(C)C3=CC2)=O

Appearance

White to off-white 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

Product Data

Product Data

Certificate of Analysis

View CoA: current batch, Lot#BBC70224

QC Data

View QC data: current batch, Lot#BBC70224

Safety Data Sheet (SDS)

View Safety Data Sheet (SDS)

Instruction

View handling instruction

Biological target:

Vamorolone (VBP15) is a first-in-class, orally active dissociative steroidal anti-inflammatory drug and membrane-stabilizer.

In vitro activity:

VBP15 has protective physicochemical effects on the plasma membrane, protecting cells from injury and promoting membrane repair. This sub-activity is likely to be particularly important in duchenne muscular dystrophy (DMD) where disease pathogenesis is clearly linked to membrane instability and myofibre injury. A key anti-inflammatory activity, the inhibition of TNFα-induced NF-κB, is retained by VBP15. This mechanism occurs through protein–protein interactions of the VBP15 ligand-activated GR, independently of DNA binding, GRE activation, or upregulation of inhibitory transcripts. NF-κB activation is among the earliest histological features of DMD neonates (Chen et al, 2005; Porter et al, 2002, 2003), years before symptoms appear. Reference: EMBO Mol Med. 2013 Oct;5(10):1569-85. https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3799580/

In vivo activity:

The EAE model shows that VBP15 does, indeed, reduce CNS inflammation to a similar degree to that of prednisolone. VBP15 was able to reduce both the incidence and severity of disease which was assessed based on the degree of paralysis. This anti-inflammatory effect was confirmed via histological studies which showed a significant reduction of inflammatory foci. It was found that that a 5-week treatment regimen of VBP15 did not decrease trabecular thickness in mdx mouse model of Duchenne Muscular Dystrophy (Heier et al. 2013). With regard to muscle atrophy, it was determined that a 5 week treatment with prednisolone significantly upregulated expression of MurF1 and FBXO32, two genes known to play a key role in GC-mediated muscle catabolism, whereas VBP15 did not. Additionally, the weight of the diaphragms in prednisolone-treated mice was significantly less than that of VBP15-treated mice, providing further evidence that VBP15 may not induce the muscle-wasting processes associated with long-term glucocorticoid use. Reference: Cell Mol Neurobiol. 2015 Apr;35(3):377-387. https://link-springer-com.libproxy.lib.unc.edu/article/10.1007/s10571-014-0133-y

	Solvent	mg/mL	mM
Solubility
	Soluble in DMSO	62.5	175.34

Note: There can be variations in solubility for the same chemical from different vendors or different batches from the same vendor. The following factors can affect the solubility of the same chemical: solvent used for crystallization, residual solvent content, polymorphism, salt versus free form, degree of hydration, solvent temperature. Please use the solubility data as a reference only. Warming and sonication will facilitate dissolving. Still have questions? Please contact our Technical Support scientists.

Preparing Stock Solutions

The following data is based on the product molecular weight 356.46 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

Formulation protocol:

1. Heier CR, Damsker JM, Yu Q, Dillingham BC, Huynh T, Van der Meulen JH, Sali A, Miller BK, Phadke A, Scheffer L, Quinn J, Tatem K, Jordan S, Dadgar S, Rodriguez OC, Albanese C, Calhoun M, Gordish-Dressman H, Jaiswal JK, Connor EM, McCall JM, Hoffman EP, Reeves EK, Nagaraju K. VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects. EMBO Mol Med. 2013 Oct;5(10):1569-85. doi: 10.1002/emmm.201302621. Epub 2013 Sep 9. PMID: 24014378; PMCID: PMC3799580. 2. Heier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, Uaesoontrachoon K, Srinivassane S, Damsker JM, Hoffman EP, Nagaraju K, Spurney CF. Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Sci Alliance. 2019 Feb 11;2(1):e201800186. doi: 10.26508/lsa.201800186. PMID: 30745312; PMCID: PMC6371196. 3. Dillingham BC, Knoblach SM, Many GM, Harmon BT, Mullen AM, Heier CR, Bello L, McCall JM, Hoffman EP, Connor EM, Nagaraju K, Reeves EKM, Damsker JM. VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis. Cell Mol Neurobiol. 2015 Apr;35(3):377-387. doi: 10.1007/s10571-014-0133-y. Epub 2014 Nov 13. PMID: 25392236. 4. Heier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, Uaesoontrachoon K, Srinivassane S, Damsker JM, Hoffman EP, Nagaraju K, Spurney CF. Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Sci Alliance. 2019 Feb 11;2(1):e201800186. doi: 10.26508/lsa.201800186. PMID: 30745312; PMCID: PMC6371196.

In vitro protocol:

In vivo protocol:

1. Dillingham BC, Knoblach SM, Many GM, Harmon BT, Mullen AM, Heier CR, Bello L, McCall JM, Hoffman EP, Connor EM, Nagaraju K, Reeves EKM, Damsker JM. VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis. Cell Mol Neurobiol. 2015 Apr;35(3):377-387. doi: 10.1007/s10571-014-0133-y. Epub 2014 Nov 13. PMID: 25392236. 2. Heier CR, Yu Q, Fiorillo AA, Tully CB, Tucker A, Mazala DA, Uaesoontrachoon K, Srinivassane S, Damsker JM, Hoffman EP, Nagaraju K, Spurney CF. Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy. Life Sci Alliance. 2019 Feb 11;2(1):e201800186. doi: 10.26508/lsa.201800186. PMID: 30745312; PMCID: PMC6371196.

1: Givinostat (Duvyzat) for Duchenne muscular dystrophy. Med Lett Drugs Ther. 2024 Dec 23;66(1718):204-205. doi: 10.58347/tml.2024.1718c. PMID: 39762189. 2: Ibrahim MS, Abdelwahab OA, Elawfi B, Ali FY, Amro S, Mohammed SF, Shaheen N, Negida A, Arndt M, Hijazi MM, Schaefer J, Siepmann T. Meta-analysis of the efficacy and safety of vamorolone in Duchenne muscular dystrophy. Neurol Sci. 2024 Dec 24. doi: 10.1007/s10072-024-07939-1. Epub ahead of print. PMID: 39715964. 3: Li Z, Liu B, Wang Y, Yu C, Xu X, Chen P. Metabolic Characterization of Vamorolone in Human Liver Microsomes: Implications for Anti-Doping. Drug Test Anal. 2024 Oct 30. doi: 10.1002/dta.3819. Epub ahead of print. PMID: 39478335. 4: Pascual-Morena C, Lucerón-Lucas-Torres M, Martínez-García I, Rodríguez- Gutiérrez E, Patiño-Cardona S, Sequí-Domínguez I. Efficacy and Safety of Vamorolone in Duchenne Muscular Dystrophy: A Systematic Review. Paediatr Drugs. 2024 Nov;26(6):695-707. doi: 10.1007/s40272-024-00655-5. Epub 2024 Sep 27. PMID: 39331339. 5: Wang Q, Zeng Y, Jiao L, He J, Li B, Guo Y, Song Z. Efficacy and safety of different doses of vamorolone in boys with Duchenne muscular dystrophy: a systematic review and network meta-analysis. Front Neurol. 2024 Aug 21;15:1456559. doi: 10.3389/fneur.2024.1456559. PMID: 39233679; PMCID: PMC11371629. 6: Czifrus E, Berlau DJ. Corticosteroids for the treatment of Duchenne muscular dystrophy: a safety review. Expert Opin Drug Saf. 2024 Oct;23(10):1237-1247. doi: 10.1080/14740338.2024.2394578. Epub 2024 Aug 27. PMID: 39152782. 7: Ahmet A, Tobin R, Dang UJ, Rooman R, Guglieri M, Clemens PR, Hoffman EP, Ward LM. Adrenal suppression from vamorolone and prednisone in Duchenne muscular dystrophy: results from the phase 2b clinical trial. J Clin Endocrinol Metab. 2024 Aug 4:dgae521. doi: 10.1210/clinem/dgae521. Epub ahead of print. PMID: 39097643. 8: Schneidereit D, Bauer J, Mnuskina S, Nübler S, Cacciani N, Mühlberg A, Kreiss L, Ritter P, Schürmann S, Larsson L, Friedrich O. CAS3D: 3D quantitative morphometry on Second Harmonic Generation image volumes from single skeletal muscle fibers. Comput Biol Med. 2024 Aug;178:108618. doi: 10.1016/j.compbiomed.2024.108618. Epub 2024 Jun 25. PMID: 38925088. 9: Khodabukus A, Prabhu NK, Roberts T, Buldo M, Detwiler A, Fralish ZD, Kondash ME, Truskey GA, Koves TR, Bursac N. Bioengineered Model of Human LGMD2B Skeletal Muscle Reveals Roles of Intracellular Calcium Overload in Contractile and Metabolic Dysfunction in Dysferlinopathy. Adv Sci (Weinh). 2024 Aug;11(31):e2400188. doi: 10.1002/advs.202400188. Epub 2024 Jun 17. PMID: 38887849; PMCID: PMC11336985. 10: Li X, Sabbatini D, Pegoraro E, Bello L, Clemens P, Guglieri M, van den Anker J, Damsker J, McCall J, Dang UJ, Hoffman EP, Jusko WJ. Assessing Pharmacogenomic loci Associated with the Pharmacokinetics of Vamorolone in Boys with Duchenne Muscular Dystrophy. J Clin Pharmacol. 2024 Sep;64(9):1130-1140. doi: 10.1002/jcph.2446. Epub 2024 Apr 29. PMID: 38682893; PMCID: PMC11357888. 11: Hoffman E, Gaglianone S, Ketema R, Tu W, Peay H, Clemens P, Dang U, Conklin L. Return of participant-level clinical trial results to participants: pilot of a simplified centralised approach. BMJ Open. 2024 Mar 23;14(3):e080097. doi: 10.1136/bmjopen-2023-080097. PMID: 38521535; PMCID: PMC10961551. 12: Mercuri E, Vilchez JJ, Boespflug-Tanguy O, Zaidman CM, Mah JK, Goemans N, Müller-Felber W, Niks EH, Schara-Schmidt U, Bertini E, Comi GP, Mathews KD, Servais L, Vandenborne K, Johannsen J, Messina S, Spinty S, McAdam L, Selby K, Byrne B, Laverty CG, Carroll K, Zardi G, Cazzaniga S, Coceani N, Bettica P, McDonald CM; EPIDYS Study Group. Safety and efficacy of givinostat in boys with Duchenne muscular dystrophy (EPIDYS): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial. Lancet Neurol. 2024 Apr;23(4):393-403. doi: 10.1016/S1474-4422(24)00036-X. Erratum in: Lancet Neurol. 2024 Jun;23(6):e10. doi: 10.1016/S1474-4422(24)00172-8. Erratum in: Lancet Neurol. 2024 Aug;23(8):e12. doi: 10.1016/S1474-4422(24)00257-6. PMID: 38508835. 13: Dang UJ, Damsker JM, Guglieri M, Clemens PR, Perlman SJ, Smith EC, Horrocks I, Finkel RS, Mah JK, Deconinck N, Goemans NM, Haberlová J, Straub V, Mengle-Gaw L, Schwartz BD, Harper A, Shieh PB, De Waele L, Castro D, Yang ML, Ryan MM, McDonald CM, Tulinius M, Webster RI, Mcmillan HJ, Kuntz N, Rao VK, Baranello G, Spinty S, Childs AM, Sbrocchi AM, Selby KA, Monduy M, Nevo Y, Vilchez JJ, Nascimento-Osorio A, Niks EH, De Groot IJM, Katsalouli M, Van Den Anker JN, Ward LM, Leinonen M, D'Alessandro AL, Hoffman EP. Efficacy and Safety of Vamorolone Over 48 Weeks in Boys With Duchenne Muscular Dystrophy: A Randomized Controlled Trial. Neurology. 2024 Mar 12;102(5):e208112. doi: 10.1212/WNL.0000000000208112. Epub 2024 Feb 9. PMID: 38335499; PMCID: PMC11067696. 14: Armstrong N, Apkon S, Berggren KN, Braun C, Ciafaloni E, Connolly A, Kennedy A, Kuntz N, Mathews K, McGuire M, Parad R, Scavina M, Scharf RJ, Waldrop M. The Early Care (0-3 Years) In Duchenne Muscular Dystrophy Meeting Report. J Neuromuscul Dis. 2024;11(2):525-533. doi: 10.3233/JND-230180. PMID: 38189762; PMCID: PMC10977386. 15: Vamorolone. Am J Health Syst Pharm. 2024 Apr 3;81(8):256-260. doi: 10.1093/ajhp/zxad303. PMID: 38153112. 16: Keam SJ. Vamorolone: First Approval. Drugs. 2024 Jan;84(1):111-117. doi: 10.1007/s40265-023-01986-2. PMID: 38103149. 17: Grounds MD, Lloyd EM. Considering the Promise of Vamorolone for Treating Duchenne Muscular Dystrophy. J Neuromuscul Dis. 2023;10(6):1013-1030. doi: 10.3233/JND-230161. PMID: 37927274; PMCID: PMC10657680. 18: Liu G, Lipari P, Mollin A, Jung S, Teplova I, Li W, Ying L, More V, Lennox W, Yeh S, McGann E, Moon YC, Rice C, Huarte E, Gruszka B, Ray B, Goodwin E, Buckendahl P, Yurkow E, Braughton B, Narasimhan J, Welch E, Voronin G, Weetall M. Comparison of pharmaceutical properties and biological activities of prednisolone, deflazacort, and vamorolone in DMD disease models. Hum Mol Genet. 2024 Jan 20;33(3):211-223. doi: 10.1093/hmg/ddad173. PMID: 37819629; PMCID: PMC10800023. 19: Saad FA, Siciliano G, Angelini C. Advances in Dystrophinopathy Diagnosis and Therapy. Biomolecules. 2023 Aug 28;13(9):1319. doi: 10.3390/biom13091319. PMID: 37759719; PMCID: PMC10526396. 20: McCormack NM, Nguyen NY, Tully CB, Oliver T, Fiorillo AA, Heier CR. Vamorolone improves Becker muscular dystrophy and increases dystrophin protein in bmx model mice. iScience. 2023 Jun 16;26(7):107161. doi: 10.1016/j.isci.2023.107161. PMID: 37534133; PMCID: PMC10391915.