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Elenestinib Phosphate

Elenestinib Phosphate, also known as BLU-263, is an investigational drug currently being studied in clinical trials for the treatment of Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM). This article explores the ongoing research into Elenestinib Phosphate’s potential to improve outcomes for patients with these rare mast cell disorders.

Table of Contents

What is ELENESTINIB PHOSPHATE?

ELENESTINIB PHOSPHATE, also known as BLU-263 phosphate, is an investigational drug being developed for the treatment of certain blood disorders[1]. It is a selective KIT inhibitor, which means it targets a specific protein involved in the growth and survival of certain cells in the body[1].

Medical Conditions Treated

The primary focus of the current clinical trial is on treating Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM)[1]. These are rare disorders characterized by the abnormal accumulation of mast cells (a type of white blood cell) in various tissues of the body.

Symptoms of ISM and SSM can include:

  • Skin rashes or flushing
  • Stomach pain and diarrhea
  • Bone pain
  • Fatigue
  • Anaphylaxis (severe allergic reactions)

How ELENESTINIB Works

ELENESTINIB PHOSPHATE works by targeting the KIT protein, which is often mutated in patients with systemic mastocytosis. By inhibiting this protein, the drug aims to reduce the number of abnormal mast cells and alleviate symptoms associated with the disease[1].

Current Clinical Trial

A large-scale clinical trial called “HARBOR” is currently underway to evaluate the effectiveness and safety of ELENESTINIB (also referred to as BLU-263) in patients with Indolent Systemic Mastocytosis[1]. This trial is divided into several parts:

  1. Part 1: A 12-week period testing different doses of ELENESTINIB
  2. Part 2: A longer-term study comparing ELENESTINIB to a placebo
  3. Part 3: An extension period for long-term safety and efficacy assessment
  4. Part S: An exploratory study for patients with Smoldering Systemic Mastocytosis

Dosage and Administration

In the clinical trial, ELENESTINIB is being tested at various doses, including 25 mg, 50 mg, and 100 mg. It is administered orally in the form of a film-coated tablet[1]. The maximum daily dose being studied is 100 mg.

Who Can Participate in the Trial?

The trial is primarily looking for patients who:

  • Are 16 years of age or older (18 or older in some countries)
  • Have been diagnosed with Indolent Systemic Mastocytosis or Smoldering Systemic Mastocytosis
  • Have moderate to severe symptoms that haven’t been adequately controlled by other treatments
  • Have a specific genetic mutation called KIT D816V

Patients with certain other conditions, such as aggressive systemic mastocytosis or mast cell leukemia, are not eligible for this trial[1].

Potential Benefits

The researchers hope that ELENESTINIB will provide several benefits for patients with ISM and SSM, including:

  • Reduction in mast cell burden (the number of abnormal mast cells in the body)
  • Improvement in symptoms such as skin flushing, gastrointestinal issues, and fatigue
  • Better quality of life
  • Reduction in the need for other medications to manage symptoms

Safety Considerations

As with any investigational drug, there may be unknown risks and side effects. The clinical trial is designed to carefully monitor patients for any adverse events. Some safety measures include:

  • Regular check-ups and blood tests
  • Monitoring of heart function through ECG evaluations
  • Careful tracking of any side effects or unexpected reactions

It’s important to note that ELENESTINIB is still being studied, and its full safety profile is not yet known. Patients considering participation in the clinical trial should discuss potential risks and benefits with their healthcare provider[1].

Aspect Details
Drug Name Elenestinib Phosphate (BLU-263)
Conditions Studied Indolent Systemic Mastocytosis (ISM), Smoldering Systemic Mastocytosis (SSM)
Trial Design Randomized, Double-Blind, Placebo-Controlled Phase 2/3 Study
Primary Objectives Determine recommended dose, assess safety and efficacy
Key Endpoints Change in ISM-SAF Total Symptom Score, serum tryptase levels, KIT D816V allele fraction
Treatment Duration Up to approximately 4 years
Dosing 25 mg, 50 mg, 100 mg doses tested
Patient Population Adults (≥16 or ≥18 years, depending on country) with confirmed ISM or SSM

FAQ

  • What is Elenestinib Phosphate? Elenestinib Phosphate is an investigational drug being studied for the treatment of Indolent Systemic Mastocytosis (ISM) and Smoldering Systemic Mastocytosis (SSM). It is a selective KIT inhibitor designed to target the underlying cause of these mast cell disorders.
  • What is the main goal of the clinical trial studying Elenestinib Phosphate? The main goal of the clinical trial is to determine the recommended dose (RD) of Elenestinib Phosphate and assess its safety, tolerability, and effectiveness in improving symptoms and reducing mast cell burden in patients with ISM and SSM.
  • How is the clinical trial structured? The trial is structured in multiple parts: Part 1 is a 12-week double-blind treatment period testing different doses, Part 2 is an extension period using the recommended dose, and Part 3 is a long-term open-label extension. There are also exploratory parts for SSM and mast cell activation syndrome (MCAS) patients.
  • What are the main symptoms being evaluated in the trial? The trial is evaluating various symptoms associated with ISM using the ISM Symptom Assessment Form (ISM-SAF). This includes assessing changes in total symptom score, individual symptom scores, and specific symptom clusters such as neurocognitive symptoms.
  • How long will patients be treated in the clinical trial? Patients may receive treatment for up to approximately 4 years, depending on which part of the study they participate in. The trial includes long-term follow-up to assess the safety and efficacy of Elenestinib Phosphate over an extended period.

Ongoing Clinical Trials on Elenestinib Phosphate

  • Study of Elenestinib for Patients with Indolent Systemic Mastocytosis

    Recruiting

    4 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Denmark France Germany +9

Glossary

  • Indolent Systemic Mastocytosis (ISM): A rare disorder characterized by the abnormal growth and accumulation of mast cells (a type of white blood cell) in various tissues of the body, causing a range of symptoms but progressing slowly.
  • Smoldering Systemic Mastocytosis (SSM): A subtype of systemic mastocytosis that is more advanced than ISM but does not yet show organ damage. It is characterized by a higher mast cell burden and may progress to more aggressive forms of the disease.
  • KIT D816V: A specific genetic mutation commonly found in patients with systemic mastocytosis, which leads to abnormal mast cell growth and activation.
  • Serum Tryptase: An enzyme released by mast cells that can be measured in the blood. Elevated levels often indicate increased mast cell activity or burden in the body.
  • ISM-SAF (ISM Symptom Assessment Form): A tool used to measure the severity of symptoms in patients with Indolent Systemic Mastocytosis, including total symptom score (TSS) and individual symptom scores.
  • Best Supportive Care (BSC): The standard treatments used to manage symptoms of systemic mastocytosis, which may include antihistamines, antileukotrienes, and other medications to control mast cell-related symptoms.
  • QTcF: The QT interval on an electrocardiogram corrected for heart rate using Fridericia's formula. It is used to assess the risk of certain heart rhythm abnormalities.
  • Bone Marrow (BM) Biopsy: A procedure to examine the bone marrow, which is often used in diagnosing and monitoring systemic mastocytosis.
  • Allele Fraction: The proportion of a particular gene variant (such as KIT D816V) present in a sample, which can indicate the extent of mast cell involvement in the disease.

References

  1. http://clinicaltrials.eu/trial/study-of-elenestinib-for-patients-with-indolent-systemic-mastocytosis/