Research news

Disclaimer: I have written this piece from a position of privilege - as an HD family member that has been fortunate to receive an education that allows me to deeply understand the nuances of Huntington’s disease. I know what it means not only at the biological level, but also at the family level. I am profoundly aware of the desire for a disease-modifying drug. But my hopes are tempered through the privileged lens of understanding complex scientific data and interpretation. Here, I report facts and my opinion of those facts with no vested interest in any specific therapeutic approach. If Prilenia feels errors have been made, they are invited to reach out and any factual corrections will gladly be made.

Recently, there have been a few press releases from Prilenia Therapeutics about their advancement of the drug pridopidine toward regulatory approval for the treatment of Huntington’s disease (HD). There’s also been mixed messaging about findings from pridopidine clinical trials and statements made by the company. Let’s break down what the research really says and what the recent press releases mean in the bigger picture.

MermaiHD Trial

We’ve written about the long and storied path of pridopidine before. It’s been tested in a number of different clinical trials, by a few different companies. Over the years, the use of pridopidine has shifted. Once thought to have utility for helping to regulate movements associated with HD, it’s now being tested for potentially slowing the disease course.

In 2008, pridopidine (previously called Huntexil) was tested in a European Phase 2 trial called MermaiHD. Back then, the company running the trial, NeuroSearch, thought the drug could be used to help with changes in movement control caused by HD.

Specifically, they thought the drug could help people control their voluntary movements that get more stiff and rigid as HD progresses. While people on pridopidine had slight improvements in movement control, the effect wasn’t large enough to determine that it was caused by pridopidine, and the trial failed to meet its endpoints.

HART Trial

Around the same time, NeuroSearch also carried out the HART study in America. Again in this study, there wasn’t a conclusive improvement in voluntary movements associated with stiffness and rigidity.

After the HART study, they pooled that data with the MermaidHD study to find that with this combined dataset, there did seem to be an improvement on voluntary movements. However, both the US FDA and European EMA determined that a larger trial was needed to conclude what effect pridopidine was truly having on HD-associated movement changes.

PRIDE-HD Trial

After this, the rights and ownership for the drug changed hands. The new owners, Teva Pharmaceuticals, wanted to test if a higher dose of pridopidine was needed to see a positive effect on HD movements. Enter the 2013 trial called PRIDE-HD.

In PRIDE-HD, a key goal was to see if pridopidine could improve total motor score - a robust collection of tests that assign a numerical value to movement symptoms associated with HD. Unfortunately, again, pridopidine failed to meet its primary endpoint and did not show an improvement in total motor score.

A change in direction

At this point, scientists went back to the drawing board to try and better understand the drug. They did more experiments and when they emerged, they had a new model for how pridopidine might improve brain health.

Now, the idea was that pridopidine might not just control HD-associated movements, but might modify disease course. This would mean pridopidine isn’t just treating the symptoms of HD, but is treating the disease itself - a huge difference.

New goal for PRIDE-HD

With this new theory, the drug developers added a few new goals mid-way through the PRIDE-HD study. The primary endpoint of testing movement was the same, and wasn’t met, but they also added a second test - total functional capacity, or TFC.

TFC is measured through a collection of tests that determine how well someone functions day-to-day. Things like the ability to hold a job, manage finances, and perform domestic chores. These abilities decline as HD progresses.

So did pridopidine improve TFC in the PRIDE-HD study? Kind of. Out of the 4 doses tested, only the lowest dose showed improvement on the TFC scale. These results were inconsistent, TFC wasn’t a primary endpoint of the trial, and there wasn’t enough data to draw a firm conclusion – so for the drug to have a chance of getting licensed, yet another trial would be needed.

Big conclusions were drawn - too big

Despite these inconsistent findings, Teva interpreted the results from PRIDE-HD to mean that the drug was slowing the progression of HD. This was a big message for the data that was generated. Quite frankly, too big of a message. This conclusion cannot be substantiated with the results from this trial.

Why not? It’s important to understand that improving or stabilizing TFC does not necessarily mean that something has slowed HD progression. For example, successful treatment of depression in a person with HD could enable them to resume work, improving that person’s TFC by 1 or 2 points – but not because the underlying progression of the disease has been slowed.

To be clear, a drug that improves the ability of people with HD to function would be fantastic. And a drug that slows the progression of HD would be super fantastic! But there is a clear distinction between those two.

Improvements in function cannot directly be interpreted as slowing HD progression – that requires much more solid evidence, from direct measures like MRI scans or biomarkers that can tell us whether something has rescued brain cells, or from much longer-term improvements in symptoms.

PROOF-HD Trial

Following the results from PRIDE-HD, pridopidine changed hands again, this time to Prilenia Therapeutics. Another trial was begun to test the ability of pridopidine to modify disease course of HD. This new Phase 3 trial began in 2020 under the name PROOF-HD.

This time, the primary endpoint was TFC, which it did not meet. People who took pridopidine in the PROOF-HD trial did not show any improvement in day-to-day functioning as measured by TFC. Again, this was a negative pridopidine trial. The trial also failed to meet its secondary endpoint, an overall assessment of HD severity called the composite Unified Huntington's Disease Rating Scale (cUHDRS).

However, one would be hard-pressed to understand this, based on the headlines about the PROOF-HD trial. Again, there were controversial interpretations that have touted PROOF-HD as a success.

Drug interactions

Previous results had suggested an interaction between pridopidine and drugs that reduce dopamine activity in the brain. Dopamine is a chemical that helps control movement, memory, and mood. The interaction between pridopidine and these dopamine-altering drugs is perhaps unsurprising, given that pridopidine was originally designed to alter dopamine activity to help with HD movements.

Drugs that reduce dopamine activity in HD include commonly-used medications like tetrabenazine for chorea, or ‘neuroleptic’ drugs like olanzapine and risperidone, which are used to help control some of the most difficult symptoms of HD like aggression, impulsivity, paranoia, delusions, or suicidality. Physicians don’t take prescribing these medications lightly, and often do so to reduce the risk that a person with HD will harm themselves or others.

Prilenia decided, in advance, to do a ‘subgroup analysis’ of the PROOF-HD trial results in people who were not taking any of these dopamine-altering drugs. This group had only 79 people with HD compared with the full trial population of 499 people with HD. They reported that this subgroup had benefited from treatment on cUHDRS and one measure of cognition, but not the TFC.

A troubling narrative

Of the drug, Prilenia CEO Dr. Michael Hayden is quoted as saying, “Pridopidine has delivered consistent efficacy benefits across multiple key measures of HD”. We at HDBuzz do not agree that the data collected to date support this interpretation.

Pridopidine has been tested in more trials than any other drug in the HD landscape, and has consistently failed to deliver the benefits anticipated by the companies running the trials. Apparent improvements seen in one aspect of HD have not been replicated when tested in subsequent trials.

What if it’s true that pridopidine works in people not taking neuroleptics? That would be good news! But to make this claim and have the regulatory agencies believe it, another clinical trial would generally be needed, focusing exclusively on this group and enrolling enough people to test this robustly. This is a valid scientific hypothesis, and it’s reasonable to test it with such a trial.

However, that’s not what Prilenia are planning.

We were troubled by an announcement from a recent, non-peer-reviewed poster summary supported by Prilenia where the authors claimed to show links between neuroleptic treatment and the progression of HD. This is a very difficult thing to prove with existing data and statistics, since neuroleptics are generally given to people whose HD symptoms are worse, or whose HD is progressing more rapidly.

It is certainly important to study how different medications might impact HD progression. But when a company whose unapproved drug may rely on people not taking neuroleptics, starts supporting research into whether neuroleptics may be bad for people with HD, we worry about what conclusions HD families might draw, and we worry especially about people stopping medicines that are protecting themselves and others from harm.

Hope vs. hype

At HDBuzz, we’re less worried about the science around pridopidine – in every trial, the results are what you get, and continuing to test theories based on those results is reasonable, if the sponsor thinks there is a real effect to be found in a particular group of people. This process is what needs to be done until we get better drugs for HD.

What troubles us is the message being put out about pridopidine, downplaying the big things it has failed to do, and emphasizing less compelling findings in subgroups or individual endpoints. We worry that people from HD families – families like mine – could end up with a much more favorable impression than is warranted of pridopidine being the first disease-slowing drug for HD. Unfortunately, all the evidence so far does not support that hope.

Hope that a drug will work is useful – but only when that hope is grounded in truth.

We want people from HD families to participate in trials for drugs with a good chance of working. Drugs that have strong scientific reasoning and solid evidence to support asking people and families to make the sacrifice of time, effort, and risk.

We have the right to expect that trial results will be presented plainly and understandably. Trials that fail to meet their outcomes should not be presented as positive, and companies with vested interests should be extremely careful about commenting on how HD clinicians and their patients choose from currently available treatment options.

Getting an application accepted for review

Most recently, we’ve heard news from Prilenia about advancing pridopidine through the European regulatory agency, the EMA. Their recent press release titled, “Prilenia’s Pridopidine for Huntington’s Disease Accepted for European Marketing Authorisation Review” is notable for its use of the word “Accepted” considerably earlier than the word “Review”.

What the press release actually says is that Prilenia has put together the paperwork to ask the EMA to consider the results from pridopidine to date, and the EMA has accepted the submission of their application.

Getting applications accepted for consideration is a process that every approved drug goes through. But it’s also a process that every refused drug goes through. Applying to get into a university is very different from being accepted. We’re reliably informed by an expert familiar with EMA procedures that this step is not that big of a deal.

Most of the time, we don’t hear about when companies go through these minor steps. Press releases generally aren’t issued for these commonplace steps throughout the regulatory process. While it’s broadly good that Prilenia are ensuring the HD community gets regular updates on where they are in the regulatory chain, we want to make sure that the news isn’t causing undue hype. We’re more keen on seeing the full, peer-reviewed results of the PROOF-HD trial in a scientific journal.

What we know

Pridopidine has been tested in various clinical trials with various primary endpoints, all of which have sadly failed to be met. Regardless of the messaging being put out from Prilenia, thus far pridopidine has produced negative results from every major endpoint in every trial. Period.

We’re not even remotely happy about this: we love drugs that work, and we love trials that prove it. We even love negative trials - when the results are presented clearly, without spin, and give a scientifically-grounded path ahead, whether that’s planning another trial or calling it quits.

The goalpost for the intended use of pridopidine continues to shift: to control movements, to modify disease course, to modify disease course for those not on neuroleptics. It’s fantastic that Prilenia continue to study pridopidine in the lab! It’s in everyone’s best interest that researchers understand medicines from as many angles as possible. However, we need to make sure the intended use of a drug is shown through positive clinical trial results.

An application has been submitted to the EMA for consideration. So far, this doesn’t mean much. An application was submitted. It will be reviewed.

Seeking truth

Scientists are truth seekers. At HDBuzz, we believe researchers have a duty to accurately relay scientific findings to the patient communities seeking answers for a cure. For truth.

If hope that a drug will work becomes so blind that it turns to hype, something has gone wrong. Results from clinical trials, by design, are not subjective. Reporting results should also not be subjective. Messaging matters. Headlines matter. Ensuring that the patient community receives and understands the full, balanced truth matters.

The team at HDBuzz honestly does hope that pridopidine delivers on everything Prilenia says it does, and more! We all want a drug that makes a positive difference to people with HD, but right now there is only data from 79 people to support moving this drug forward. In our opinion, that isn't sufficient for regulatory approval – time will tell whether the regulators have the same view or are persuaded otherwise.

Until we get there, HDBuzz will be here to report the truth and tease out the hope from the hype. We’re sorry if our take is disappointing, but we make no apology for keeping openness, frankness, and science at the heart of our reporting.

Stem cells grown in 3D in a research lab can mimic some features of Huntington’s disease (HD). They also hold promise for transplantation studies to potentially add back cells that are lost in HD. But what would happen to those new cells? Would they get along with the cells still in the brain that have the HD gene? And what can this system teach us about ongoing clinical trials aimed at lowering the HD-causing message in only parts of the brain? Read on to find out!

The power of stem cells

Stem cells hold a certain mystique. They can either retain their “stemness”, remaining a stem cell, or to turn into something else altogether. Contained within each one is the ability to become almost any cell type in the human body. Scientists can coax them into becoming a heart cell, or a muscle cell, or even a brain cell, providing scientists with a powerful research tool that can be used to answer questions about people’s brains in health and disease.

For brain diseases like Huntington’s disease (HD), there’s a second powerful potential application for stem cells – transplantation. As a neurodegenerative disease, HD causes the gradual loss of brain cells. This primarily happens in a central portion of the brain, called the striatum, and in the outer wrinkly bit of the brain, called the cortex.

Several groups of researchers are exploring approaches that would allow them to harness the power of stem cells to replace cells that are lost over the course of HD. We recently wrote about the work Dr. Leslie Thomspon is advancing for stem cell transplants from our coverage of the Hereditary Disease Foundation conference. But what would happen to the new cells? Would they adopt features of HD?

Dr. Elena Cattaneo and her team from the University of Milan, in Italy, recently published a study aiming to answer some of these questions. Elena’s lab are world leaders in using stem cells to research HD. In this new paper, they sought to better understand the effect that cells with the gene for HD have on cells without the HD gene. This might help inform future cell transplantation studies and trials aimed at lowering the disease-causing message since those drugs are unlikely to hit every cell in the brain equally.

Mini brain in a dish

Typically, when cells are used in lab experiments, they’re grown flat on the back of a dish. But if you’ve ever seen another person, you know that people aren’t 2D! So more sophisticated technologies allow researchers to grow cells in 3D.

The fancy term for these 3D cells is “organoids”, aka “mini brains”. We’ve previously written about these lab-grown brains and what researchers have learned from them. While mini brains can adopt some of the cellular features of a brain, such as connections between different cells, they don’t actually have the ability to transmit thoughts and feelings.

While these mini brains look deceivingly unsophisticated on the outside (like a little whitish, pinkish snot to be honest!), they’re elegantly complex on the inside. The cells form intricate networks between brain cells that can be seen communicating with one another under the microscope. These mini brains give researchers a way of understanding in 3D how HD affects connections and communication between different cells.

Scientists know that in a human brain, HD reduces the ability of cells in the outer cortex to communicate with the inner striatum. This communication breakdown leads to a loss in those connections over time. When those connections go unused for extended periods of time, it can create an unhealthy environment for the brain cells, and they may eventually die.

A positive influence

Elena and her team see something similar in their mini brains that have the HD gene. At the molecular level, brain cells communicate across a very small gap called the synapse. This is where the tips of brain cells meet to send bubbles of information back and forth to one another. In HD, the number of bubbles is reduced over time. In this new paper, the team sees the same thing in HD mini brains – there is less communication at the synapse than in mini brains without the gene for HD.

A key experiment in the new paper from Elena’s lab asked what happens to cells in mini brains when cells without the gene for HD are combined with cells that have the gene for HD.

The team performed a very detailed analysis of the genetic messages contained in the mixed population mini brains, seeing that they more closely resemble the mini brains without the HD gene rather than the ones with the HD gene. This suggests that the cells without the HD gene have a positive influence on those with the HD gene. Good friends to have around!

They also looked at the synapses in these mixed population mini brains. They found the communication being sent from the synapse was greatly improved! It more closely matched the mini brains without the gene for HD. This suggests the cells without HD might be helping the cells with HD to communicate better.

The team also identified some features that weren’t totally rescued by the presence of the cells without the gene for HD. In the mixed population mini brains there were still some changes at the genetic message level. Additionally, the number of cells that died in the mixed population mini brains wasn’t totally rescued. This suggests that while cells without HD help the mixed population mini brains, they can’t overcome every feature caused by the HD gene.

Informing ongoing and future trials

Overall, this type of research can help determine the therapeutic potential for using stem cells to slow progression and treat HD. It is also informative for ongoing trials that lower levels of the disease-causing genetic message.

While the goal for some of those trials is to lower the message by about 50%, that won’t occur in every cell in the brain. Because of that, those cells with reduced HD genetic message will exist in a mixed population with cells that have more of the HD genetic message. Data from studies like those highlighted here help researchers understand exactly what may happen at the molecular level when such mixed populations of cells with and without the gene for HD exist.

An important point the research team was able to tease out in this paper is that the cells without HD have a positive influence on the cells with the HD gene. But the opposite is not true. The cells that have the HD gene don’t seem to alter programs in the cells without HD. This is important for future transplantation studies because it suggests cells without HD that are added may have a positive effect, but the cells already in the brain with HD possibly won’t have a negative effect on the new cells. A win, win!

Moving treatments forward

While stem cells and mini brains are super cool, there are some limitations to their use. Firstly, they don’t truly mimic what’s happening inside a human brain in a living person. Nothing in a lab dish can. This is why it’s important to study potential treatments in a functioning brain, like in a mouse, and eventually run clinical trials in people.

Additionally, the mini brains that contained cells with and without the gene for HD were mixed before they were made. Meaning the mixed population was there from “birth”. In the case of a person with HD, the cells or treatment would be added after the person had a fully formed brain.

Despite the caveats, this work represents a cool approach for better understanding how cells without the gene for HD may act if they were added to a brain with HD. It also sheds light on what may happen in a brain when some cells have the gene for HD while others have less of that message.

The human brain, both inside a lab dish and out, is incredibly complex, so knowing as much as possible about how HD affects cellular and molecular features will help move treatments forward.

New data from uniQure, who developed a one-and-done gene therapy for Huntington’s disease (HD) called AMT-130, indicates that the drug is relatively safe and might be able to slow down signs and symptoms of HD. AMT-130 is currently under investigation in Phase I/II clinical trials in Europe and the US which are mainly focused on drug safety. These hot new data are very encouraging, so let’s dive into what it all means!

What is AMT-130?

Developed by uniQure, AMT-130 is the first gene therapy for HD. Like many of the other therapies being tested in the clinic right now, it aims to reduce the levels of the HD protein, huntingtin, in the brain. What makes it a bit different, however, is that AMT-130 is a one-and-done gene therapy; you are only given one dose of the drug ever in the course of your life.

AMT-130 is made up of a harmless virus packaged with genetic material that contains the instructions to reduce the amount of huntingtin in each cell that the virus infects in the brain. The drug is given to people with HD by a very specialised type of brain surgery.

All of this was obviously rather daunting back when AMT-130 was first developed and we didn’t know how safe the drug might be. The one-and-done nature of the drug means that effects of the drug, good or bad, can not be undone.

uniQure did a huge number of studies before they tested AMT-130 in people, which took place over years using many different types of HD animal models. Even when uniQure began testing AMT-130 in people in 2019, they did so very slowly, starting with just a few brave folks who selflessly signed up to test this innovative therapy. Only when things looked ok following these first surgeries did they begin giving the drug to more people.

HD-GeneTRX-1 and HD-GeneTRX-2 - two trials for AMT-130 on two continents

There are in fact two clinical trials testing AMT-130 in people with HD; HD-GeneTRX-1 in the US and HD-GeneTRX-2 in Europe. Together, 39 participants of the trials were given either a high dose of AMT-130, a low dose of AMT-130, or a sham surgery, which means that participants underwent surgery but no drug was given. All people in the trial are then tracked for 4 years after their surgery, where all sorts of clinical, biomarker, brain imaging, and other measurements are taken.

The key aim of both trials is to investigate whether AMT-130 is safe in people. In addition to this, lots of other data are collected along the way which might hint at how well AMT-130 is working and how it might impact signs and symptoms of HD.

Since the trials began, AMT-130 has had a bit of a bumpy road. In the first people treated, everything seemed to be going ok but in August 2022, serious side effects were reported for some people who received the high dose of AMT-130. Fortunately, things got back on track after a 3 month pause in enrollment into the trial, and uniQure shared the good news that their trial will continue as planned, with new safety measures in place.

Since the brief trial pause, uniQure has reported steady progress with signs that this drug appears safe. There were also some hints of trends in the data they collected from all of the study participants that seemed to suggest that the drug might be having an effect on some symptoms of HD, although this was just a signal and is not conclusive.

Some things to keep in mind with this latest update

It’s important to note that the two trials are not over, the most recent data is an interim update. There are still 2+ more years of data to be collected for most folks. In fact, only 12 people who received the low dose (out of 13 in this group) and 9 people who received the high dose (out of 20 in this group) are at the 24 month mark.

Given the arduous way this drug is delivered, it takes a long time for everyone to get their surgery, even after they are enrolled. This means that the numbers of people from which the data comes from in this release are very tiny, so we should be very cautious in how we interpret the findings - we don’t yet know how this will play out in a bigger pool of people over a longer period of time.

Another important thing to note is that all comparisons in this data release are against natural history data, not placebo controls. Natural history data tracks people with HD over the course of their lives to see how their symptoms, brain imaging, biomarkers, and other clinical measurements change over time. This is very different to a placebo group who undergo the same procedures as the folks receiving the drugs, the only difference being they don’t actually receive the drug. The placebo effect can be very powerful so if we are using natural history data as our baseline, we should be cautious in the direct comparisons we draw. This decision was taken as there is only complete data for people in the sham surgery group up until 12 months.

Keeping all that in mind, this update is still rather exciting, so buckle in!

What’s the latest news about AMT-130?

Safety

The good news is that AMT-130, at both the low and high dose, appears to be relatively safe. There are manageable effects which we would expect to see following brain surgery, like headaches and pain associated with the procedure. However, the important part is that no new serious side effects were reported since the trial was paused back in August 2022, which is good news.

NfL - insights to brain health

An important measurement for tracking general brain health is the biomarker neurofilament light, often called NfL. Because HD has a detrimental effect on brain health, NfL levels go up over time as HD progresses. Therefore, NfL measurements can tell us two things: Firstly, whether the therapy might be causing harm, and secondly, whether the therapy might be slowing down disease progression, and therefore slowing the rate at which NfL levels go up over time in someone with HD.

We learned in previous updates from uniQure that there’s an initial spike in NfL levels. This is to be expected for any treatment requiring brain surgery, since the surgery itself will temporarily reduce overall brain health. What’s important is that this is short-lived - the initial spike is followed by a rapid decline in NfL levels over the next 6-8 months after surgery. Looking at NfL levels after the initial spike is where the juicy details are - this is what will tell us if AMT-130 is improving brain health and slowing HD progression.

In the last data release in December of 2023, only 6 people in the low-dose group and 2 people in the high-dose group had made it to the 24 month time point. Now, there are 12 people from the low-dose group and 9 people from the high-dose group that have reached the 24 month mark. Having data from more people helps give us a clearer picture of the effect AMT-130 is having on NfL 2 years after treatment.

Excitingly, the new data show that people treated with both the low- and high-dose of AMT-130 have NfL levels significantly below what would be expected, suggesting their decline in brain health is slowed compared to folks who have not been treated with AMT-130. While this sounds incredibly exciting, this is still a very small dataset so we shouldn’t get our hopes up too high.

Clinical measures

uniQure also looked at clinical measures to get an idea of the effect that AMT-130 might have on slowing or stopping symptoms of HD. Specifically, they looked at the Composite Unified Huntington's Disease Rating Scale, or cUHDRS. This is a collection of tests that measures the ability of someone with HD to carry out daily tasks, movement control, capacity to pay attention, and memory. Overall, the cUHDRS is known to be the most sensitive way to measure clinical progression of HD.

At the end of the day, clinical measures will be the most important readout. Having a drug that is effective at slowing or stopping progression of clinical signs and symptoms of HD is what we all want. Compared to a natural history study, disease progression was slowed by around 80% in people on the high dose of AMT-130. This suggests that AMT-130 may be having an effect in slowing progression of HD as measured by cUHDRS. Again, this is only data from 9 people, so it must be interpreted with caution.

cUHDRS is actually made up of many different clinical measures including Total Function Capacity (TFC) and Total Motor Score (TMS). Looking at these individual measures, the effect of AMT-130 is less obvious although there is a suggestion of a trend of things heading in the direction of slowing HD symptom progression. Not to be a broken record, but again, the tiny number of folks whose data is being analysed at this stage means we have to be careful in drawing too strong conclusions.

Other measures uniQure didn’t report this time

Interestingly, this update included no new information about whether huntingtin protein levels are being lowered by the drug, the effect we expect this drug to have in the brain. We also didn’t learn any new information about what brain imaging might tell us about how AMT-130 is working. Hopefully, uniQure gives us updates on both of these measures the next time they share data.

What does this all mean?

Overall, this update is exciting, positive and certainly cause for very cautious optimism. That said, this does not mean that AMT-130 is a cure for HD, there is still a long road ahead. We need more data from more people over longer timeframes to be sure of the effect this drug is really having on slowing down symptoms of HD. Nonetheless, the fact that the drug appears relatively safe and there are positive signs in how it might be helping slow down symptoms is good news for the HD community.

What’s next for AMT-130?

Recently, the FDA granted AMT-130 Regenerative Medicine Advanced Therapy (RMAT) designation - the very first time this has happened for an HD therapeutic. This gives them more frequent interactions with the FDA and priority review of their data, so that if the time comes that they’re ready to file for regulatory approval, they can hit the ground running to get accelerated approval.

uniQure have disclosed that they expect to meet with the FDA in the second half of 2024 to continue their discussions about the development of AMT-130. In those conversations, they hope to define a path for getting approval of AMT-130 for HD.

Lot’s of things to be thankful for

Sometimes when it rains, it pours! We have had what feels like a deluge of positive and encouraging news about HD clinical trials lately, and certainly at HDBuzz, we are feeling thankful. It was not so long ago that the news deluge was delivering a very different and much more difficult message, that many drugs just weren’t working as we had hoped.

So, what’s changed? Well it’s important to remember that even when clinical trials don’t give us the results we had hoped for, there is still so much to be learnt from the wealth of data that is collected. All of the selfless hours in the clinic from the folks with HD who sign up for these trials count for a huge amount. The rich datasets they help generate have a huge impact in how scientists understand how different therapies might work in people, and what they can change and improve to give us the best chance of success. Their contributions have gotten us to this exciting point where we still have lots of irons in the fire and are edging closer to disease-modifying therapies.

The future of HD clinical trials is bright, thanks to the resiliency, fortitude, and sacrifice of so many people with HD who bravely stepped up to test these experimental drugs. We are forever thankful to them and are buckled in for the ride to see what comes next.