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Blu-263 Phosphate

Elenestinib (BLU-263) is an investigational drug being studied in clinical trials for the treatment of various forms of systemic mastocytosis, a rare blood disorder characterized by the abnormal accumulation of mast cells in different organs. These trials aim to evaluate the safety, efficacy, and optimal dosing of elenestinib in patients with advanced systemic mastocytosis (AdvSM) and indolent systemic mastocytosis (ISM), as well as its potential use in combination with other therapies.

Table of Contents

What is BLU-263 (Elenestinib)?

BLU-263, also known as Elenestinib, is an investigational drug being developed by Blueprint Medicines for the treatment of various blood disorders[1]. It is a selective KIT inhibitor, which means it targets a specific protein called KIT that is often mutated in certain blood cancers[2].

What Conditions Does BLU-263 Treat?

BLU-263 is being studied for the treatment of several conditions, including:

  • Advanced Systemic Mastocytosis (AdvSM): A rare blood disorder characterized by the accumulation of abnormal mast cells in various organs[1].
  • Indolent Systemic Mastocytosis (ISM): A less aggressive form of systemic mastocytosis[2].
  • Smoldering Systemic Mastocytosis (SSM): An intermediate form of systemic mastocytosis[2].
  • Other KIT-altered hematologic malignancies: Blood cancers that have mutations in the KIT gene[1].

How Does BLU-263 Work?

BLU-263 works by targeting and inhibiting the KIT protein, particularly when it has a specific mutation called D816V. This mutation is commonly found in systemic mastocytosis and some other blood cancers. By blocking the activity of the mutated KIT protein, BLU-263 aims to stop the growth and spread of abnormal cells[1][2].

Clinical Trials and Research

BLU-263 is currently being studied in several clinical trials:

  1. AZURE Study (BLU-263-2101): This is a Phase 1/2 study evaluating BLU-263 as both a monotherapy and in combination with azacitidine for patients with advanced systemic mastocytosis and other KIT-altered blood cancers[1].
  2. HARBOR Study: This is a Phase 2/3 study investigating BLU-263 for patients with indolent systemic mastocytosis[2].

These studies aim to determine the safety, efficacy, and optimal dosing of BLU-263 for different patient populations.

Dosage and Administration

BLU-263 is administered orally as a film-coated tablet. The exact dosage is still being determined through clinical trials, but current studies are exploring doses ranging from 25 mg to 100 mg daily[2]. The medication is typically taken once daily, but the optimal dosing schedule may vary depending on the specific condition being treated and individual patient factors.

Potential Side Effects

As BLU-263 is still in clinical trials, the full range of potential side effects is not yet known. However, common side effects of targeted therapies like BLU-263 may include:

  • Nausea and vomiting
  • Fatigue
  • Diarrhea
  • Changes in blood cell counts
  • Skin reactions

The ongoing clinical trials are closely monitoring patients for any adverse events to better understand the safety profile of BLU-263[1][2].

Patient Eligibility

Eligibility for BLU-263 treatment or clinical trials may include:

  • Confirmed diagnosis of advanced systemic mastocytosis, indolent systemic mastocytosis, or other KIT-altered blood cancers
  • Presence of the KIT D816V mutation
  • Age 16 or older (18 or older in some countries)
  • Adequate organ function
  • No recent treatment with certain other medications

Specific eligibility criteria may vary depending on the particular clinical trial or treatment protocol[1][2].

Future Prospects

BLU-263 (Elenestinib) shows promise as a potential treatment for various forms of systemic mastocytosis and other KIT-altered blood cancers. If the ongoing clinical trials demonstrate favorable results, it could provide a new targeted therapy option for patients who may not have responded well to existing treatments. As research continues, more information about the efficacy, safety, and optimal use of BLU-263 will become available, potentially leading to its approval as a new treatment for these challenging blood disorders[1][2].

Aspect Details
Drug Name Elenestinib (BLU-263)
Conditions Studied Advanced Systemic Mastocytosis (AdvSM), Indolent Systemic Mastocytosis (ISM), Smoldering Systemic Mastocytosis (SSM)
Trial Phases Phase 1/2 and Phase 2/3
Administration Oral, film-coated tablets
Treatment Arms Monotherapy and combination therapy with azacitidine
Primary Objectives Determine recommended dose, assess safety and tolerability, evaluate efficacy
Secondary Objectives Assess overall response rate, pharmacokinetics, quality of life measures
Key Inclusion Criteria Confirmed diagnosis of SM, presence of KIT D816V mutation, moderate to severe symptoms
Key Exclusion Criteria Other myeloproliferative disorders, significant cardiovascular disease, prior treatment with selective KIT inhibitors
Trial Duration Up to 4 years, including long-term follow-up

FAQ

  • What is elenestinib (BLU-263) and how does it work? Elenestinib, also known as BLU-263, is an investigational drug designed to target and inhibit the KIT D816V mutation, which is commonly found in systemic mastocytosis. By targeting this mutation, elenestinib aims to reduce the abnormal growth and accumulation of mast cells in various organs.
  • What types of systemic mastocytosis are being studied in these clinical trials? The clinical trials are investigating elenestinib in different forms of systemic mastocytosis, including advanced systemic mastocytosis (AdvSM), indolent systemic mastocytosis (ISM), and smoldering systemic mastocytosis (SSM). Some trials also include patients with other KIT-altered blood cancers.
  • How is elenestinib administered in these clinical trials? Elenestinib is administered orally in the form of film-coated tablets. The dosing regimens vary depending on the specific trial and study arm, with some patients receiving elenestinib alone and others receiving it in combination with other medications like azacitidine.
  • What are the main goals of these clinical trials? The main goals of these trials include determining the recommended dose (RD) of elenestinib, assessing its safety and tolerability, evaluating its efficacy in reducing symptoms and mast cell burden, and studying its pharmacokinetics (how the drug moves through the body) in different patient populations.
  • How long do these clinical trials last? The duration of the trials varies, with some studies lasting up to 4 years. They typically include different phases, such as screening, treatment periods, and long-term follow-up. Some trials also have extension periods to assess the long-term safety and efficacy of elenestinib.

Ongoing Clinical Trials on Blu-263 Phosphate

  • Study of BLU-263 and Azacitidine for Patients with Advanced Systemic Mastocytosis and Other KIT Altered Blood Cancers

    Not recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Belgium France Germany The Netherlands Norway Spain

Glossary

  • Systemic Mastocytosis (SM): A rare blood disorder characterized by the abnormal accumulation of mast cells in various organs throughout the body.
  • Advanced Systemic Mastocytosis (AdvSM): A more severe form of systemic mastocytosis that includes aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated hematologic neoplasm (SM-AHN), and mast cell leukemia (MCL).
  • Indolent Systemic Mastocytosis (ISM): A less aggressive form of systemic mastocytosis that typically has a slower progression and less severe symptoms compared to AdvSM.
  • KIT D816V Mutation: A specific genetic mutation commonly found in patients with systemic mastocytosis that leads to abnormal mast cell growth and accumulation.
  • Mast Cells: A type of white blood cell that plays a role in the body's immune response and allergic reactions.
  • Pharmacokinetics (PK): The study of how a drug moves through the body, including its absorption, distribution, metabolism, and excretion.
  • Recommended Dose (RD): The optimal dose of a drug determined through clinical trials that balances efficacy and safety.
  • Dose-Limiting Toxicity (DLT): Side effects that occur at a specific dose level that may limit the use of higher doses of a drug in clinical trials.
  • Overall Response Rate (ORR): The proportion of patients whose cancer shrinks or disappears after treatment.
  • Pure Pathological Response (PPR): A measure of treatment effectiveness based on the reduction of abnormal cells or tissue in biopsy samples.

References

  1. http://clinicaltrials.eu/trial/study-of-blu-263-and-azacitidine-for-patients-with-advanced-systemic-mastocytosis-and-other-kit-altered-blood-cancers/
  2. http://clinicaltrials.eu/trial/study-of-elenestinib-for-patients-with-indolent-systemic-mastocytosis/