In short
Miransertib, also known as MK-7075 or ARQ 092, is an investigational drug being studied in clinical trials for the treatment of PIK3CA-Related Overgrowth Spectrum (PROS) and Proteus Syndrome (PS). These rare genetic disorders are characterized by overgrowth of various body tissues. The clinical trials aim to evaluate the safety, tolerability, and effectiveness of miransertib in managing these conditions, offering hope for patients with limited treatment options.
At a glance
- Drug Name
- Miransertib (MK-7075, ARQ 092)
- Conditions Studied
- PIK3CA-Related Overgrowth Spectrum (PROS), Proteus Syndrome (PS)
- Administration
- Oral, once daily
- Dosage Range
- 5-35 mg/m² (varies by trial and patient)
- Trial Phases
- Phase 1/2
- Primary Outcomes
- Safety, tolerability, adverse events, treatment discontinuation
- Secondary Outcomes
- Changes in pain, physical functioning, quality of life, lesion growth
- Duration
- Varies by trial, up to 96 cycles (approximately 7-8 years)
- Age Groups
- 2 years and older (varies by trial)
- Key Assessments
- Imaging (MRI, CT, ultrasound), photography, blood tests, quality of life surveys
What is Miransertib?
Miransertib, also known as MK-7075 or ARQ 092, is an experimental drug being developed to treat rare overgrowth disorders. It is a small molecule drug, which means it's a tiny compound that can easily enter cells in the body. Miransertib is taken orally as a capsule, making it convenient for patients to use.
What Conditions Does Miransertib Treat?
Miransertib is being studied primarily for two rare conditions:
- PIK3CA-Related Overgrowth Spectrum (PROS): This is a group of rare disorders characterized by overgrowth of various body tissues. The overgrowth can affect different parts of the body and can vary in severity.
- Proteus Syndrome (PS): This is an extremely rare condition that causes overgrowth of bones, skin, and other tissues. It usually begins in infancy and becomes more noticeable as a child grows.
Both of these conditions are caused by genetic mutations that lead to excessive cell growth in certain parts of the body.
How Does Miransertib Work?
Miransertib works by targeting a specific protein in the body called AKT1. In patients with PROS and Proteus Syndrome, the AKT1 protein is overactive, which leads to excessive cell growth and tissue overgrowth.
Miransertib is an AKT inhibitor, meaning it blocks the activity of the AKT1 protein. By doing this, it aims to slow down or stop the overgrowth of tissues in patients with these rare disorders.
Current Clinical Trials
Several clinical trials are currently underway to study the effectiveness and safety of miransertib:
- The MOSAIC study (NCT03094832) is a Phase 1/2 trial studying miransertib in patients with PROS and Proteus Syndrome.
- Another study (NCT04980872) is looking at the long-term safety of miransertib in patients who have been treated with the drug in other studies.
- A Phase 2 trial (NCT04316546) is specifically studying miransertib in patients with Proteus Syndrome.
Dosage and Administration
In the clinical trials, miransertib is typically given as follows:
- It is taken orally once daily, either 1 hour before or 2 hours after a meal.
- The dosage is often based on body surface area, starting at 15 mg/m² and potentially increasing to 25 or 35 mg/m² depending on how well the patient tolerates the drug.
- Treatment is given in cycles, with each cycle typically lasting 28 days.
Safety and Side Effects
As with any medication, miransertib can cause side effects. The clinical trials are closely monitoring patients for any adverse events (unwanted or harmful effects). Common ways of assessing safety include:
- Tracking the number of patients who experience adverse events.
- Monitoring for serious adverse events, which are more severe or life-threatening effects.
- Keeping track of how many patients need to stop treatment due to side effects.
The specific side effects of miransertib are still being studied. Patients in clinical trials are closely monitored with regular check-ups, blood tests, and other assessments to ensure their safety.
Future Prospects
Miransertib represents a promising approach to treating rare overgrowth disorders like PROS and Proteus Syndrome. If the clinical trials show positive results, it could become the first targeted treatment for these conditions.
Researchers are not only looking at whether miransertib can slow or stop overgrowth, but also whether it can improve patients' quality of life, reduce pain, and improve physical functioning.
While the results are still pending, miransertib offers hope for patients with these rare and challenging disorders. As always, patients should discuss with their healthcare providers about the potential benefits and risks of participating in clinical trials or using experimental treatments.