Agamree (vamorolone)

To treat Duchenne muscular dystrophy Drug Trials Snapshot

FDA Approval: 10/26/2023

Research Synopsis

  • Agamree (vamorolone) is a novel glucocorticoid analogue designed to offer anti-inflammatory benefits while minimizing the side effects commonly associated with traditional steroids.
  • Recent studies demonstrate that vamorolone effectively reduces muscle inflammation and promotes repair in conditions like Duchenne muscular dystrophy (DMD) without triggering adverse hormonal or immune responses.
  • Clinical trials have shown that vamorolone is well-tolerated, with favorable pharmacokinetics and a safety profile that includes reduced insulin resistance and lower risk of adrenal suppression compared to conventional glucocorticoids.
  • Vamorolone has been found to improve muscle function in clinical trials, showing significant benefits in boys with DMD compared to placebo groups.
  • In preclinical studies, vamorolone has exhibited superior efficacy in reducing inflammatory markers and symptoms in models of autoimmune diseases and arthritis, suggesting a broader therapeutic potential.
  • Unique to vamorolone is its dissociative steroid properties, which allow it to engage with glucocorticoid receptors in a way that provides anti-inflammatory effects without the negative consequences typical of traditional steroid treatment.
  • Research indicates that vamorolone targets signaling pathways associated with muscle regeneration and inflammation and may serve as a safer alternative for various inflammatory disorders, potentially including asthma and critical illness myopathy.
  • Overall, the findings from multiple studies position vamorolone as a promising new therapy that can maintain the beneficial anti-inflammatory effects of glucocorticoids while reducing the significant risks associated with their long-term use.

Related articles

Research articles about Agamree (vamorolone)

Agamree (vamorolone)

VBP15, a novel anti-inflammatory and membrane-stabilizer, improves muscular dystrophy without side effects.

London, UK

2 hours ago

1 Received

  • The absence of dystrophin in Duchenne muscular dystrophy (DMD) makes muscles vulnerable to injury, leading to cell membrane damage and ongoing inflammation, while current glucocorticoid treatments pose significant side effects and unclear benefits.
  • Researchers discovered a new oral drug, VBP15, that provides muscle protection and promotes repair through anti-inflammatory and membrane-stabilizing actions, without triggering hormonal or immune suppression.
  • In DMD model mice, VBP15 demonstrated improved muscle strength and pathology, suggesting it could be a promising option for clinical use in DMD and other chronic inflammatory conditions.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Asynchronous remodeling is a driver of failed regeneration in Duchenne muscular dystrophy.

London, UK

2 hours ago

1 Received

  • The study investigates why muscle regeneration fails in dystrophic muscle by using data from human dystrophy and mouse models.
  • It reveals that transforming growth factor β networks, which are linked to fibrosis and poor regeneration, are activated during normal muscle repair but at different times.
  • By creating an experimental model that mimics differing regeneration rates, the researchers found that specific treatments improved muscle recovery, suggesting the model could help understand tissue failure in other chronic conditions.

Figma Sketch HTML5

$100 - $150

Hourly Rate

VBP15, a novel anti-inflammatory, is effective at reducing the severity of murine experimental autoimmune encephalomyelitis.

London, UK

2 hours ago

1 Received

  • - Multiple sclerosis is a chronic autoimmune disease that damages the central nervous system through inflammation and demyelination, often treated with glucocorticoids like prednisolone, which have significant side effects.
  • - A new compound called VBP15 has shown promise in treating multiple sclerosis with fewer side effects while effectively reducing disease severity and inflammation, particularly in mouse models.
  • - VBP15 was found to inhibit harmful pro-inflammatory gene expression in human macrophages without the bone loss and muscle atrophy related to prednisolone, indicating its potential as a safer treatment option.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Pharmacologically-induced mitotic synchrony in airway epithelial cells as a mechanism of action of anti-inflammatory drugs.

London, UK

2 hours ago

1 Received

  • Mitotic synchrony is the normal, synchronized division of cells, but asthmatic airway epithelial cells show mitotic asynchrony, which contributes to inflammation.
  • A study compared the effects of traditional glucocorticoids with a new drug, VBP15, on mitotic synchrony and inflammatory responses in these asthmatic cells after mechanical injury.
  • VBP15 not only improved mitotic synchrony but also significantly reduced key inflammatory and fibrogenic cytokines, suggesting it may be more effective and safer for treating asthma than traditional glucocorticoids.

Figma Sketch HTML5

$100 - $150

Hourly Rate

A novel dissociative steroid VBP15 reduces MUC5AC gene expression in airway epithelial cells but lacks the GRE mediated transcriptional properties of dexamethasone.

London, UK

2 hours ago

1 Received

  • Overproduction of mucins in inflammatory lung diseases worsens health outcomes, with a focus on the mucin gene MUC5AC being regulated by inflammatory mediators like IL-1β and glucocorticoid drugs such as Dexamethasone (Dex).
  • VBP15, a compound from ReveraGen BioPharma, shows promise as an anti-mucin agent by suppressing MUC5AC gene expression without some of the side effects associated with long-term steroid use.
  • Experimental results indicate that VBP15 effectively reduces MUC5AC levels in human airway epithelial cells through mechanisms involving the glucocorticoid receptor (GR) and inhibition of NFκB, differing from the action of Dex.

Figma Sketch HTML5

$100 - $150

Hourly Rate

VBP15, a novel dissociative steroid compound, reduces NFκB-induced expression of inflammatory cytokines in vitro and symptoms of murine trinitrobenzene sulfonic acid-induced colitis.

London, UK

2 hours ago

1 Received

  • - The study aimed to evaluate the effects of VBP15, a steroidal compound, on reducing inflammation and symptoms of colitis in mice, as well as its impact on growth in juvenile mice.
  • - Experiments involved treating human intestinal epithelial cells with VBP15 and administering it to mice with induced colitis over specific periods to compare its effects against prednisolone.
  • - Results showed that VBP15 significantly reduced inflammatory markers and colitis symptoms, while causing less growth stunting compared to prednisolone, suggesting it could be a safer treatment for inflammatory bowel disease.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Vamorolone, a dissociative steroidal compound, reduces pro-inflammatory cytokine expression in glioma cells and increases activity and survival in a murine model of cortical tumor.

London, UK

2 hours ago

1 Received

  • Corticosteroids like dexamethasone are commonly used in neuro-oncology for their anti-inflammatory effects, but many patients suffer from negative side effects.
  • This study explored vamorolone, a new steroid alternative, in a mouse model to see if it could reduce harmful side effects while maintaining anti-inflammatory benefits.
  • Results showed that vamorolone effectively reduced pro-inflammatory signals like dexamethasone, but had a better safety profile, improving activity and survival in mice compared to those treated with dexamethasone.

Figma Sketch HTML5

$100 - $150

Hourly Rate

The corticosteroid compounds prednisolone and vamorolone do not alter the nociception phenotype and exacerbate liver injury in sickle cell mice.

London, UK

2 hours ago

1 Received

  • * A preclinical trial evaluated the effects of two corticosteroids, vamorolone (dissociative) and prednisolone (conventional), on inflammation and organ dysfunction in sickle cell disease mice.
  • * While both corticosteroids reduced inflammation and white blood cell counts, they also caused significant liver damage, highlighting possible risks of hepatic toxicity despite reduced inflammation in sickle cell disease patients.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Phase 1 trial of vamorolone, a first-in-class steroid, shows improvements in side effects via biomarkers bridged to clinical outcomes.

London, UK

2 hours ago

1 Received

  • - Vamorolone (VBP15) is a new type of glucocorticoid drug that effectively reduces inflammation without many side effects commonly seen with traditional glucocorticoids, like prednisone.
  • - In clinical trials, Vamorolone was shown to be well-tolerated by healthy adults, with a similar metabolism to prednisone but significantly less impact on adrenal function and side effects like bone fragility or immune suppression.
  • - The unique structure of Vamorolone allows it to bind to glucocorticoid and mineralocorticoid receptors differently than existing drugs, which may explain its reduced side effects while retaining anti-inflammatory properties.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Muscle miRNAome shows suppression of chronic inflammatory miRNAs with both prednisone and vamorolone.

London, UK

2 hours ago

1 Received

  • Corticosteroids are effective anti-inflammatory drugs, but their chronic use leads to harmful side effects, especially in children, prompting research for safer alternatives like vamorolone (VBP15).
  • Vamorolone, a dissociative glucocorticoid receptor ligand, shows similar anti-inflammatory effects as traditional steroids without the negative side effects, as demonstrated in the mdx mouse model of Duchenne muscular dystrophy (DMD).
  • The study found that vamorolone primarily reduces pro-inflammatory miRNAs, whereas prednisolone activates miRNAs linked to steroid side effects; both treatments influence the inflammatory transcription factor NF-κB, highlighting potential new biomarkers and drug targets.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Membrane Stabilization by Modified Steroid Offers a Potential Therapy for Muscular Dystrophy Due to Dysferlin Deficit.

London, UK

2 hours ago

1 Received

  • Mutations in the DYSF gene reduce dysferlin protein levels, causing limb girdle muscular dystrophy type 2B (LGMD2B), which impairs muscle fiber repair and causes inflammation.
  • A study compared the effects of the dissociative steroid vamorolone and the glucocorticoid prednisolone on muscle repair in dysferlin-deficient mice.
  • Vamorolone improved myofiber membrane stability and repair, increased muscle strength, and was more effective than prednisolone, which actually worsened muscle condition, suggesting vamorolone's potential for clinical use in LGMD2B.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Phase IIa trial in Duchenne muscular dystrophy shows vamorolone is a first-in-class dissociative steroidal anti-inflammatory drug.

London, UK

2 hours ago

1 Received

  • The study investigates vamorolone, a new type of steroidal anti-inflammatory drug, in boys aged 4 to under 7 with Duchenne muscular dystrophy over a 2-week period.
  • The results show that vamorolone was safe and well-tolerated, with improved safety profiles such as reduced insulin resistance and adrenal suppression compared to traditional glucocorticoids.
  • The research also indicated that vamorolone has potential anti-inflammatory effects, suggesting it could preserve the benefits of steroids while minimizing their negative side effects, with further studies planned to explore clinical outcomes.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Vamorolone treatment improves skeletal muscle outcome in a critical illness myopathy rat model.

London, UK

2 hours ago

1 Received

  • Critical illness myopathy (CIM) is a condition resulting from critical care that leads to muscle atrophy and can increase mortality and healthcare costs; glucocorticoids (GCs) like prednisolone may contribute to CIM.
  • In an experiment, rats were subjected to ICU conditions for five days while receiving either prednisolone, the new drug vamorolone, or no treatment, to assess outcomes like muscle strength and weight.
  • Results indicated that both GCs reduced muscle atrophy compared to the untreated group, but vamorolone was more effective than prednisolone, particularly in protecting fast-twitch muscle fibers, suggesting that vamorolone is a better alternative for treating CIM.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Vamorolone targets dual nuclear receptors to treat inflammation and dystrophic cardiomyopathy.

London, UK

2 hours ago

1 Received

  • Cardiomyopathy is a major concern in Duchenne muscular dystrophy, with mineralocorticoid and glucocorticoid receptors playing distinct roles in heart and muscle issues.
  • The drug vamorolone can act as an MR antagonist and a unique GR ligand, reducing inflammation and offering better safety compared to prednisone.
  • In studies on mice, vamorolone effectively prevents harmful effects linked to MR activation, unlike prednisolone, which worsens symptoms of cardiomyopathy.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Population Pharmacokinetics of Vamorolone (VBP15) in Healthy Men and Boys With Duchenne Muscular Dystrophy.

London, UK

2 hours ago

1 Received

  • Duchenne muscular dystrophy (DMD) is a genetic disorder affecting boys, caused by mutations in the dystrophin gene, for which vamorolone is being studied as a treatment.
  • Vamorolone shows favorable pharmacokinetics (PK) with a half-life of about 2 hours and a maximum plasma concentration occurring 2-4 hours after dosing, with consistent results in both healthy men and boys with DMD.
  • The drug is absorbed better when taken with food, exhibits dose-linear pharmacokinetics without drug accumulation during daily dosing, and has similar clearance rates in both populations.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Vamorolone trial in Duchenne muscular dystrophy shows dose-related improvement of muscle function.

London, UK

2 hours ago

1 Received

  • The study evaluated vamorolone, a novel anti-inflammatory drug, in 48 boys aged 4-7 with Duchenne muscular dystrophy (DMD) to determine the optimal dosage and effectiveness.
  • Conducting a 24-week trial with varying doses (0.25, 0.75, 2.0, and 6.0 mg/kg/d), researchers found that the 2.0 mg/kg/d dose significantly improved muscle function without the common side effects associated with glucocorticoids.
  • Results indicated that vamorolone was safe and well-tolerated, showing potential benefits in bone health and lower risk of adrenal suppression and insulin resistance compared to traditional glucocorticoid treatments.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Vamorolone, a dissociative steroidal compound, reduces collagen antibody-induced joint damage and inflammation when administered after disease onset.

London, UK

2 hours ago

1 Received

  • - This study aimed to evaluate the effects of vamorolone, a novel steroid, on inflammation in a mouse model of arthritis, specifically after the disease has begun rather than before.
  • - The research used 84 mice, with treatments (vamorolone or prednisolone) given orally for 7 days post-disease onset, assessing disease severity, joint inflammation, and inflammation markers.
  • - Results showed that vamorolone significantly reduced inflammation and joint damage compared to prednisolone, suggesting it could be a safer and effective treatment option for rheumatoid arthritis and similar conditions.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Serum biomarkers associated with baseline clinical severity in young steroid-naïve Duchenne muscular dystrophy boys.

London, UK

2 hours ago

1 Received

  • * A study on 39 young boys with DMD used serum protein biomarkers to create models that correlate with their physical performance over time, utilizing methods like weighted correlation network analysis.
  • * Key proteins were identified that linked serum levels to both clinical and muscle tissue severity, highlighting connections between muscle strength, endurance, and underlying biological pathways involved in muscle health.

Figma Sketch HTML5

$100 - $150

Hourly Rate

Disruption of a key ligand-H-bond network drives dissociative properties in vamorolone for Duchenne muscular dystrophy treatment.

London, UK

2 hours ago

1 Received

  • Duchenne muscular dystrophy is a genetic disorder that causes progressive damage to muscles, leading to early death, and current steroid treatments have significant side effects, particularly bone loss.
  • A new drug called vamorolone has been developed that modifies the steroid structure to maintain effectiveness while significantly reducing these negative side effects.
  • Research reveals that vamorolone's unique properties are due to its partial agonism, which disrupts specific interactions within the glucocorticoid receptor, resulting in targeted effects on muscle regulation without compromising other important functions.

Figma Sketch HTML5

$100 - $150

Hourly Rate