PROOF-HD: understanding the preliminary results and balancing our expectations
– June 12th, 2023
Prilenia presented the preliminary topline results of the PROOF-HD clinical trial (PRidopidine Outcome On Function in Huntington Disease) at the end of April. Last year, the Moving Forward team wrote a news piece about this trial.
Now, we look at the first findings of PROOF-HD and share our expectations for the near future. PROOF-HD enrolled 499 participants with early manifest Huntington’s disease (HD) in the U.S., Canada, Austria, Czech Republic, France, Germany, Italy, the Netherlands, Poland, Spain and the United Kingdom.
Enroll-HD: 10 Years Observing HD
– March 15, 2023
Enroll-HD is the world’s largest observational study being conducted in HD. This study is promoted by the CHDI Foundation, Inc. to speed up the development of effective treatments for Huntington’s Disease. Enroll-HD aims to observe and monitor how HD affects people and how it changes over time. It is focused on collecting a huge amount of demographic, clinical and biological information from the worldwide HD community with three specific goals: enhance the understanding of HD, support clinical trials and improve clinical care.
JOIN-HD: A platform for Juvenile HD
– March 9th, 2023
JOIN-HD is a worldwide registry for families affected by Juvenile onset Huntington’s Disease (JoHD), which aims to shed light on this diagnosis, bring together those impacted by JoHD, increase awareness and knowledge about this condition, facilitate research and help advocate for better care.
Juvenile onset Huntington’s Disease is diagnosed when a 20-year-old person or younger develops unequivocal HD symptoms.
Roche GENERATION HD2: giving Tominersen a second chance
– February 14th, 2023
After a hard setback in March 2021, when Roche stopped the GENERATION HD1 trial because of potential safety issues, the company is now fully prepared to give its huntingtin-lowering drug, Tominersen, a second chance.In fact, at the end of 2021, a comprehensive data analysis led Roche to conclude that the drug seemed to have benefited younger people who began the trial in earlier stages of HD.
Wave SELECT HD: when one is better than two
– February 14th, 2023
Wave is a strong contender in the Huntingtin lowering drug race. Their approach is a bit different from other companies, as their ASO is only trying to silence the expanded copy of the HD gene, while leaving the other copy of the gene alone. In other words, the company wants to selectively target the mutated Huntingtin protein and preserve the unaffected Huntingtin protein, based on the hypothesis that keeping the healthy protein might be beneficial for the person affected by HD
Prilenia Phase 3 Trial with Pridopidine – Why is it Different?
– September 13th, 2022
For all those who have been following Huntington’s disease research for some time, Pridopidine is an “old acquaintance”.
To date, several studies have been conducted with this drug, looking closely to its effect on dopamine. Although they have helped us to confirm the safety and good tolerability of pridopidine, none of these trials has been completely successful.
UniQure: the Highs and Lows of AMT-130
– September 9th, 2022
UniQure is a company with an expertise in gene therapy that has long been involved in the search for HD treatment options. Their preclinical studies in animal models showed that AMT-130, the gene therapy product candidate for HD, reduced the mutant huntingtin protein and was associated with a decrease in the progression of Huntington’s disease signs.
PIVOT-HD, PTC Therapeutics Phase 2 Trial to Lower Huntingtin
– September 8th, 2022
PTC Therapeutics is a US biopharmaceutical company specialized in the development of gene therapies for rare diseases. The team at PTC Therapeutics has screened more than 300,000 molecules in the search for an effective therapeutical approach to HD, one that can lower huntingtin, can be administered orally and can have an impact inside and outside of the brain.
Suspending is not Ending: Updates about the Novartis VIBRANT-HD trial
– September 8th, 2022
At the end of August, Novartis has written a letter to the HD community to explain the reasons behind their decision to temporarily suspend dosing of Branaplam in the Phase 2b VIBRANT-HD study with adults with HD.
While we know that this kind of news always comes as a shock to all families living with HD, we must remember that we are protecting and safeguarding the health of the brave and generous patients who have voluntarily signed up for this research.
Another Vibrant Possibility for Huntington’s Disease Treatment
– March 17th, 2022
Novartis Pharmaceuticals is recruiting participants for their phase IIb clinical trial called VIBRANT-HD. This study will investigate the huntingtin lowering drug Branaplam, which was originally developed for Spinal Muscular Atrophy, a devastating paediatric genetic disease.
Novartis will test Branaplam in patients with early manifest HD to check the safety of the drug in HD and learn what is the right dose to lower huntingtin, the harmful protein.
Updates about Roche GENERATION-HD1 and Tominersen: it’s not over until it’s over
– 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.