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Tovorafenib

Tovorafenib, also known as DAY101, is an oral type II RAF kinase inhibitor being investigated in several clinical trials for the treatment of pediatric low-grade gliomas and other solid tumors with alterations in the MAPK pathway. This article summarizes key information about ongoing clinical trials evaluating tovorafenib’s safety and efficacy in various patient populations.

Table of Contents

What is Tovorafenib?

Tovorafenib, also known as DAY101, Ojemda, TAK580, or AMG-2112819, is a new medication being studied for the treatment of various types of cancer[1][2][3][4][5]. It belongs to a class of drugs called RAF inhibitors, which target specific proteins involved in cancer growth[1].

How Does Tovorafenib Work?

Tovorafenib works by blocking enzymes called RAF kinases, which are part of a pathway in cells known as the MAPK pathway[4]. This pathway is often overactive in cancer cells, causing them to grow and divide uncontrollably. By inhibiting these enzymes, Tovorafenib may help slow down or stop the growth of cancer cells[1][4].

What Conditions Does Tovorafenib Treat?

Tovorafenib is being studied for the treatment of several types of cancer, including:

  • Low-grade gliomas: These are slow-growing brain tumors that typically occur in children and young adults[1][5].
  • Melanoma: A type of skin cancer[4].
  • Langerhans cell histiocytosis (LCH): A rare disease where certain white blood cells build up in various organs, causing damage[5].
  • Craniopharyngioma: A rare type of brain tumor that typically affects children and young adults[3].
  • Other solid tumors with specific genetic alterations in the MAPK pathway[4].

How is Tovorafenib Administered?

Tovorafenib is taken orally, usually as a tablet or a powder that is mixed with water to form a liquid[1][2]. The exact dosing schedule can vary depending on the specific clinical trial, but it is often given once weekly[1][5].

Current Clinical Trials

Tovorafenib is currently being studied in several clinical trials:

  • A study comparing Tovorafenib to standard chemotherapy for children with low-grade gliomas[5].
  • A trial testing Tovorafenib in combination with other drugs for various solid tumors[4].
  • A study of Tovorafenib for patients with Langerhans cell histiocytosis[5].
  • A trial combining Tovorafenib with another drug called nivolumab for craniopharyngioma[3].

Potential Side Effects

As Tovorafenib is still in clinical trials, all of its potential side effects are not yet known. The ongoing studies are closely monitoring patients for any adverse events[1][3][5]. It’s important to note that side effects can vary from person to person, and not everyone will experience the same effects. If you’re participating in a clinical trial, your healthcare team will closely monitor you for any side effects and provide appropriate care.

Aspect Details
Drug Name Tovorafenib (DAY101)
Drug Class Oral type II RAF kinase inhibitor
Primary Indications Pediatric low-grade gliomas, solid tumors with MAPK pathway alterations
Administration Oral, typically once weekly
Key Clinical Trials NCT05566795, NCT04985604, NCT05828069
Primary Outcomes Overall response rate (ORR), Progression-free survival (PFS)
Secondary Outcomes Duration of response, Overall survival, Safety and tolerability
Patient Populations Children and young adults with various solid tumors, focus on RAF-altered tumors
Combination Therapies Being studied alone and in combination with other therapies (e.g., vinblastine, pimasertib)

FAQ

  • What is tovorafenib and how does it work? Tovorafenib (DAY101) is an oral type II RAF kinase inhibitor. It works by blocking enzymes called RAF kinases that are involved in cell growth and division. By inhibiting these enzymes, tovorafenib may help stop the growth of cancer cells with certain genetic alterations.
  • What types of cancers is tovorafenib being studied for? Tovorafenib is primarily being studied for pediatric low-grade gliomas, but it's also being investigated for other solid tumors with MAPK pathway alterations, including melanoma, thyroid cancer, and various other cancers with specific genetic mutations.
  • How is tovorafenib administered? Tovorafenib is given orally, typically once weekly. It's available as tablets or as a powder for reconstitution into a liquid form.
  • What are some of the potential side effects of tovorafenib? While specific side effects are still being studied, common side effects of cancer treatments may include fatigue, nausea, and changes in blood cell counts. The clinical trials are closely monitoring patients for any adverse events to determine the safety profile of tovorafenib.
  • How long do patients typically receive tovorafenib in clinical trials? The duration of treatment varies between trials, but many studies are evaluating tovorafenib for extended periods, such as up to 24 cycles (where each cycle is 28 days) or until disease progression or unacceptable toxicity occurs.

Ongoing Clinical Trials on Tovorafenib

  • A study of ulixertinib, tovorafenib, and vinblastine sulfate for children with progressive, relapsed, or refractory low-grade glioma

    Recruiting

    1 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Czechia Denmark Germany Sweden

  • Study of tovorafenib (DAY101) in children and young adults aged 6 months to 25 years with brain tumors and other solid tumors that have RAF gene changes

    Recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    Denmark Germany The Netherlands

  • A study of tovorafenib for children and young adults with newly diagnosed or recurrent craniopharyngioma

    Not yet recruiting

    2 1 1
    Investigated diseases:
    Investigated drugs:
    France Germany The Netherlands

  • Study of tovorafenib versus standard chemotherapy as first treatment for children with low-grade glioma with RAF gene changes

    Not recruiting

    3 1 1 1
    Investigated diseases:
    Investigated drugs:
    Austria Belgium Czechia Denmark Finland France +10

Glossary

  • RAF kinase: An enzyme involved in cell signaling pathways that regulate cell growth and division. Mutations in RAF kinases can lead to uncontrolled cell growth in some cancers.
  • Low-grade glioma: A slow-growing type of brain tumor that develops from glial cells, which support and protect nerve cells in the brain and spinal cord.
  • MAPK pathway: A chain of proteins in the cell that communicates a signal from a receptor on the surface of the cell to the DNA in the nucleus. This pathway is important in some types of cancer.
  • Progression-free survival (PFS): The length of time during and after treatment that a patient lives with the disease but it does not get worse.
  • Overall response rate (ORR): The percentage of patients whose cancer shrinks or disappears after treatment.
  • RANO-LGG criteria: Response Assessment in Neuro-Oncology criteria for Low-Grade Gliomas, a standardized method for evaluating the response of brain tumors to treatment.
  • Pharmacokinetics (PK): The study of how a drug is absorbed, distributed, metabolized, and eliminated by the body.
  • Maximum tolerated dose (MTD): The highest dose of a drug that does not cause unacceptable side effects.
  • Recommended Phase 2 Dose (RP2D): The dose of a drug recommended for further testing in Phase 2 clinical trials, based on results from earlier studies.

References