When a Clinical Trial Ends, Hope Doesn't

Understanding Roche’s Decision to Discontinue the Phase II GENERATION HD2 and Phase I POINT-HD —and Why Hope Continues

Today, we learned that Roche has made the decision to end two of its Huntington’s disease trials. Both GENERATION HD2, and the POINT-HD Phase I study, will not be continued.

The Generation HD2 study was recently completed, however the results showed that Tominersen is safe, but it doesnt show any significant benefit for patients. It simply doesnt work like we hoped for. In POINT-HD, a trial in early phase I stage, the animals given multiple dosages of the compound called RG6496, showed a negative effect. Its important to note that for the few patients that has been given one dosage of RG6496, there are no safety concerns.

Many families have followed Roche’s research journey from the very beginning. Some have participated in the trials themselves. Others have watched each milestone with hope, believing that one day these studies could make a real difference for people living with HD.

Receiving news about negative results is harsh and many may feel disappointed, frustrated or emotionally exhausted. Those feelings are real, and they deserve to be acknowledged.

More than 1,500 people and families from the HD community around the world chose to take part in these studies, often without knowing whether they will personally benefit, but because they believe their participation can help build a better future for the generations that follow.

Every participant should know, that no matter how disappointed you are today, your contribution matters, and it will continue to make a difference!

Because medical research is rarely a straight path. Every potential treatment is carefully studied to ensure that it is both safe and capable of making a meaningful difference in people’s lives. Sometimes, that means stopping a programme because it has not shown the hoped-for clinical benefit or because safety concerns have emerged. While this is never the news our community hopes to hear, it is an essential part of developing treatments that are both effective and safe.

Scientific progress is built step by step, and every study—whether it reaches its intended goal or not—helps us learn more about Huntington’s disease, adds valuable knowledge.

Although today's news is disappointing, it is also worth taking a step back and recognising just how far Huntington's disease research has come over the past decade.

The progress has been extraordinary. Between 2014 and 2025, 165 clinical studies—including 111 interventional trials and 54 observational studies—have been launched, evaluating 50 different therapeutic candidates, with 21 programmes still actively moving forward today, reflecting a research pipeline that is broader and more diverse than ever before.

Perhaps the biggest change is not only the number of studies, but what they are trying to achieve. While many studies a decade ago aimed primarily to manage symptoms, advances in genetics, biomarkers, imaging and our understanding of Huntington’s disease biology, have opened the door to entirely new therapeutic strategies, from huntingtin-lowering approaches and gene therapies to RNA-based medicines, DNA repair targets and other innovative technologies, focused on disease-modifying therapies that seek to slow or alter the course of Huntington’s disease.

This progress did not happen overnight. It is the result of years of scientific collaboration, investment and, above all, dedication and generosity of the HD community.

The programme ending today represents much more than an individual clinical trial.

When Roche chose to invest in Huntington’s disease more than a decade ago, treatment options were extremely limited and disease-modifying therapies were still a distant goal. Their long-term commitment helped change the research landscape, demonstrating that Huntington’s disease was a field worth pursuing and encouraging many other biotechnology and pharmaceutical companies to invest in HD research.

Today, more than 50 companies became involved in Huntington’s disease research, bringing new therapies, innovative technologies and new clinical programmes.

Just ten years ago, such a broad range of therapeutic approaches would have seemed almost unimaginable.

While today’s announcement marks the end of two programmes, GENERATION HD2 because it did not demonstrate the hoped-for clinical benefit, and POINT-HD due to safety concerns in animals models, it does not erase the impact this work has had on the wider Huntington’s disease community or on the future of research.

Every clinical trial teaches us something. Negative or inconclusive results are not failures—they are part of the scientific process.

Every study helps researchers better understand the disease, improve future trial designs and identify new paths towards effective treatments.

Since 2014, around 55% of HD clinical trials have been completed, about 11% have been discontinued , and almost 40% are still ongoing. These numbers remind us that setbacks are part of medical research, but they are not the end of the story.

To everyone who participated in these studies.

To every person living with Huntington's disease who chose to participate.

To every family who travelled to study visits.

To every caregiver who offered encouragement and support.

To every researcher, study coordinator and healthcare professional who dedicated years of work and expertise to these programmes.

Thank you.

Your contribution has made a lasting difference. Together, you have helped deepen our understanding of Huntington’s disease, and the knowledge gained through these studies will continue to shape future research for years to come.

It is also important to remember that Huntington’s disease research has never been more active than it is today.

The HD research community has shown remarkable resilience over the years. While some programmes come to an end, many others continue to move forward. Across the world, researchers, clinicians, biotechnology and pharmaceutical companies continue to develop new therapeutic approaches whilst new technologies are emerging thanks to an expanding pipeline of clinical research.

Each programme is different. Each brings new lessons. Together, they represent a research ecosystem that is broader, stronger and more diverse than ever before.

Today’s news is disappointing. But it is not the end of Huntington’s disease research, and it is certainly not the end of hope. While today’s announcement marks the end of two clinical programmes, it is important to note that the Roche’s commitment to Huntington’s disease research continues.

Its Phase I/II clinical trial of the investigational gene therapy RG6662 (formerly from Spark Therapeutics; study SPK-101, NCT06826612) is continuing as planned.

The company has also reaffirmed its commitment to exploring multiple therapeutic approaches for Huntington’s disease and will continue to pursue promising scientific opportunities. We will continue to follow the progress of this and other research programmes closely and keep our community informed as new developments emerge.

As a community, we know that progress is rarely linear. We have experienced setbacks before, learned from them, and continued moving forward—together.

We also know that research is powered not only by science, but by people. By every person who takes part in a study, every family who offers their support, and every professional dedicated to improving the lives of those affected by Huntington’s disease.

That shared commitment, resilience and hope will continue to guide us as we work together towards better treatments and a brighter future for everyone affected by Huntington’s disease.

For full details of the announcement, we encourage you to read the Roche’s community letter.

– Article written by Ruth Blanco and Astri Arnesen

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