1800 244 735

Huntexil hits the headlines again

The results of the MermaiHD trial of Huntexil for movement problems in HD have been published in the scientific journal Lancet Neurology. Despite conflicting media reports, this doesn’t change the need for a further trial before Huntexil can be approved for use in patients.

Huntexil and movement problems

Huntexil is the brand name of ACR16, also known as pridopidine. Developed by Neurosearch, a Danish pharmaceutical company, Huntexil is a new possible treatment aimed at improving symptoms of Huntington’s disease.

Huntexil’s target is the movement, or ‘motor’ symptoms of HD. Unlike existing drugs, Huntexil isn’t just aimed at damping down the involuntary movements (‘chorea’ and ‘dystonia’) but at improving overall motor function including balance and voluntary control.

Neurosearch conducted two clinical trials of Huntexil – the MermaiHD trial in Europe and the HART trial in the USA. The results of those trials have been presented in press releases and at several scientific conferences, and we’ve written about them before on HDBuzz.

It’s worth highlighting that Huntexil is a symptom-control drug, and we’ve no reason to believe it’s disease-modifying for HD – there’s nothing to suggest it can prevent or slow down the progression of the disease.

Why the headlines?

Huntexil was back in the news this week, with a confusing array of headlines from “Pridopidine shows promise in trial” to “Drug disappoints in Huntington’s”. So what’s actually happened?

The simple answer is: not a lot. These headlines don’t relate to a new trial, or any new data. They’ve been triggered by the formal publication of the results of the MermaiHD study in the peer-reviewed scientific journal The Lancet Neurology.

What does the article say?

The Lancet Neurology article says pretty much what we expected from Neurosearch’s previous presentations of the MermaiHD data. The bottom line is that the drug failed to meet the ‘primary endpoint’ – that’s the benchmark of success – that had been set in advance.

This failure was the reason why the FDA in America, and the EMA in Europe, both declined to approve Huntexil for use in patients, insisting that a further trial that met its primary endpoint was required before they’d consider it.

In among the many different things that were measured, there were a couple of hints that pridopidine might still have some benefits. At the higher of two doses tested, some movement scores did appear to be slightly better in the treated patients. And the drug was found to be pretty safe without many side effects. Because of these hints, Neurosearch is pressing on with a further trial.

Why the confusion?

The confusion in the headlines about this article highlights a common problem in science – the tendency of news sources to want to put forward a very simple message, when science is seldom straightforward.

As ever, we encourage readers to look behind the headlines and not to rely on a single source for news. Our ‘ten golden rules’ article gives tips for finding the truth among the hype.

What now?

So fundamentally, the situation hasn’t really changed for Huntexil. If anything, the Lancet Neurology article provides reassurance that the MermaiHD trial was well run, the drug remains interesting and is well tolerated.

But a further trial – currently being planned by Neurosearch – will still be needed if Huntexil is to become an approved treatment for Huntington’s disease. We’ll update you on the trial once the details have been announced.

In the meantime, there are drugs already available which are widely used to help with movement symptoms – things like tetrabenazine, olanzapine, risperidone and sulpiride. And physiotherapy and exercise can make big differences, too. So if you have concerns about your movement control, speak to your HD physician.

Latest Research Articles

How many is too many? Exploring the toxic CAG threshold in the Huntington’s disease brain

Published date: 21 April, 2024

Drug hunters have been particularly interested in the repeating C-A-G letters of genetic code that lead to Huntington’s disease (HD). The number of CAG repeats gets bigger in vulnerable brain cells over time and may hold the key for slowing or stopping HD. Many scientists have been asking what happens to HD symptoms if we ... Read more

Cry your eyes out: detecting huntingtin in tears

Published date: 10 April, 2024

A recently published collaboration between academic researchers and pharmaceutical companies was successful at detecting huntingtin in tears. The scientists were looking for a new, easy way to track Huntington’s disease (HD). If you don’t mind shedding a tear or two, they found it! Biomarkers – biological metrics in tune with disease progression Tracking disease progression ... Read more

The director’s cut: how CAG repeats change the editing of genetic messages

Published date: 26 March, 2024

Long repetitive sequences of C-A-G letters in the DNA code are associated with at least 12 genetic diseases, including Huntington’s disease (HD). A group of scientists in Massachusetts, USA, have recently developed a new genetic strategy to study how CAG repeats can lead to harmful proteins being made in cells, causing cells to become unhealthy. ... Read more

Understanding expansions at the single cell level

Published date: 12 March, 2024

In two recent studies, researchers looked at how different parts of the brain are affected by CAG expansions in Huntington’s disease (HD) at the level of individual brain cells. The scientists looked at post-mortem brains from people with and without HD to track molecular changes in different brain regions called the cortex and striatum. These ... Read more

Huntington's Disease Therapeutics Conference 2024 – Day 3

Published date: 7 March, 2024

HDBuzz is back for the last day of the CHDI HD Therapeutics Conference: Thursday February 29th in Palm Springs, California. This article summarizes our real-time updates of the conference in community-friendly language. From genes to medicines The morning session will focus on how human genetics is driving the development of therapeutics. “Genetic modifiers” are genes ... Read more

Huntington's Disease Therapeutics Conference 2024 – Day 2

Published date: 6 March, 2024

HDBuzz is back for Day 2 of the CHDI HD Therapeutics Conference: Wednesday February 28th in Palm Springs, California. This article summarizes our real-time updates of the conference in community-friendly language. It’s a brain disease This morning’s session is titled “It’s a brain disease” and will feature talks about BRAINSSSS! HD scientists are a bit ... Read more