Transcript: Key data and insights from ReNeu trial
Supported by SpringWorks Therapeutics
Dusica Babovic-Vuksanovic, MD
All transcripts are created from interview footage and directly reflect the content of the interview at the time. The content is that of the speaker and is not adjusted by Medthority.
- The ReNeu study is a very important study, which is evaluating drug, mirdametinib, in the patients with plexiform neurofibromas, due to condition NF1 or neurofibromatosis type 1. So, this is very important study because it is bringing evidence that MEK inhibitor, mirdametinib, can be used in management of these complex tumours. So, obviously there is an unmet need for patients with neurofibromatosis because currently we only have a surgical treatment. We have option of observation only. And the only other systemic therapy, that is currently on the market is another MEK inhibitor, which is called selumetinib. And mirdametinib, is a second drug, which is bringing hope to these patients.
What is known about this drug so far, it is standing out as a drug which does have effect on treatment of plexiform neurofibromas by decreasing their size. It can also improve quality of life, and help with the pain control in patients with NF1 plexiform neurofibroma. Side effects of these drugs are also relatively tolerable, which is standing out compared to some other oncological drugs. Mirdametinib showed efficacy in about half of adult and paediatric patients with a tumour shrinkage 20% or more. 20% is considered partial tumour response. And about roughly 50% of individuals treated with drug for at least 24 months did experience improvement, meaning that they had experienced shrinkage of the tumour.
So, the deep response means that the patient had at least 50% in a tumour reduction, and a majority of treated patients who experienced tumour regression in a mirdametinib trial actually had a deep response, which is very remarkable. They did not know which patients are going to respond better based on a tumour size, or based on a tumour allocation, or their age or gender. So, there is really no way that we can know which patient is going to respond positively or a patient who will not respond. Well, what is really exciting is that in a long term follow up, we did observe additional responders. So, patients who were treated after 24 cycles of therapy, in a long-term follow-up, a few of them experienced a shrinkage of the tumour, and they continued to experience improvement in pain control and quality of life. So, patients treated with mirdametinib did experience improvement in the pain, in overall quality of life, and decreased in the pain mostly. So, that was a really remarkable response. We did not observe any additional, concerning side effects during the follow-up phase.
Patients who were treated for longer than 24 cycles continued to experience similar toxicity like diarrhoea and nausea and skin rash, but no additional, concerning side effects. There was a very small difference in a safety profile of the drug between children and adults. Children appear to tolerate the drug a little bit better than adults, because we had a higher rate of dropouts from the study in adult cohort. But, the toxicity was similar in both groups. I think this is very exciting news for clinicians, because for a long time they did not have any treatment available. Now we have additional drugs. This is second drug which is showing efficacy in plexiform neurofibroma, and this time we have a drug which is marketed also for adult population, which was completely missing in the past.
- [Dusica] This is possible. This drug still does not have effect in a 100% of patients, so additional drugs probably will be developed over time. Perhaps some combination treatments will become available, but this drug certainly has potential to be long-term treatment for many patients with NF. I just wish we continue our efforts to develop new drugs, and overall, to increase awareness of neurofibromatosis type one, because improved awareness and engagement may result in better outcomes for these patients.
I think that most of specialties should know about neurofibromatosis type one because it's a multi systemic condition, which can involve pretty much any part of the human body. So, we need dermatologists, ophthalmologists, neurologists, the surgeons of any sort, endocrinologists. And, we also have to make sure that adult providers are very well aware of neurofibromatosis, because currently we have most of these providers effect of being involved in treatment of children. But, in adulthood, this condition is not so very well recognised. So we just need to increase awareness among adult medical providers.
And, also remember, this is hereditary condition, so you have entire families affected with neurofibromatosis. So, it can be very emotional, and very, very special for patients who have treatment, and who have providers who are taking care of them.
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Development of this education content has been supported by a grant/funding from SpringWorks. The content has been independently developed by EPG Health with no input or influence from SpringWorks.