Updates about Roche GENERATION-HD1 and Tominersen
It’s not over until it’s over
Written by the Moving Forward Team on February 3rd 2022
The pharmaceutical company Roche has recently shared with the HD community further details about the phase III clinical trial GENERATION HD-1. This clinical trial involved nearly 800 HD patients from around the world and stopped dosing in March 2021 following the advice from an Independent Data Monitoring Committee.
This recommendation to stop the trial was based on the potential benefit/risk profile of Tominersen. This means that the benefits were not clear nor significant when the patients that received the experimental therapy were compared to patients who did not receive the experimental therapy. Furthermore, some patients seemed to have worsen their clinical condition.
While doing a more detailed analysis of the data collected during GENERATION-HD1, the Roche team found that there was a sub-group of patients who appeared to benefit from the effects of Tominersen. These were younger patients (people under 48 years of age) with less severe disease and who were exposed to a less frequent dose of Tominersen (120 mg every 16 weeks)
For this specific group, there were some positive trends in the results of the tests selected to assess the impact of Tominersen after participants were treated for 69 weeks. The results are only indications and do not provide us any certainty. But the results are seen as interesting, and Roche has consequently decided to investigate more whether treatment with Tominersen is beneficial for this subgroup of HD patients.
Therefore, the company is designing a more targeted phase II clinical trial for this group of patients. The trial is going to be launched soon, and will evaluate the safety, tolerability and efficacy associated with different dosages of Tominersen. The Moving Forward team believes that bringing Tominersen’s updates to patients in a simple and clear language through press releases and webinars are very important steps towards understanding the work behind each clinical trial and each company involved in the search for effective therapies for Huntington’s disease.
While there is still a lot of data that the Huntington’s community has yet to learn, we are very grateful for the extensive analytical work that Roche has done over the past months. It is because of this commitment, and the resources invested, that Roche has been able to determine whether or not to launch a new clinical trial with this same drug, but this time with a very specific subgroup of patients.
Every time a clinical trial starts, it is a time of great excitement and hope, with high expectations. The development of new drugs and clinical trials are experiments and, as all experiments, we don’t know if they will succeed or not. But from every trial we learn valuable lessons. We, as a community, need to stay informed and keep educating and encouraging each other to cope with all these experiments. We need to be prepared for setbacks and failures, but still not give up. This is the natural course of drug development. And we will never quit before we have found good solutions to treat HD.
Thus, while we must always remain cautious and careful while researchers proceed to ensure the safety, tolerability and efficacy of the drugs, we think it’s good news that Roche is continuing to explore potential HD therapies. HD families are vulnerable, but they are also extremely enduring and willing to make incredible efforts to ensure HD clinical progress. They are heroes delivering information to advance science. That is an amazing contribution and should be honoured by the community.
All the information and data that can be obtained from this new Phase II trial will help us increase and improve our knowledge about HD. It will also be an excellent opportunity to start a fresh and early dialogue with the HD families, so that they can have realistic hopes about the trial impact on their lives. We understand that these will be big achievements, as they will undoubtedly help us in our collective quest for an effective therapy to modify the course of the disease.