clinical-trial
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Australia Opens The Door for SKY-0515: Skyhawk Seeks Provisional Approval for Its Oral HD Drug
⏱️ 8 min read | An oral Huntington’s disease drug is eligible for accelerated approval in Australia. This isn’t full approval yet, but it opens a faster path to potentially getting this once-daily pill to people with HD sooner.
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The road ahead for uniQure: FDA says more data needed for AMT-130 gene therapy
⏱️ 10 min read | The FDA wants more data before approving AMT-130 for Huntington’s disease in the U.S. On March 2026, uniQure shared in an update that current Phase 1/2 data weren’t enough for the agency. A new randomized, sham-controlled trial may be required.
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Vico’s Trial Adjusts With Twice-Yearly Dosing, And The U.S. Is Next
⏱️ 6 min read | Vico Therapeutics’ Huntington’s disease drug VO659 is now being tested just twice a year, and the FDA has cleared the way for US trials to begin later this year.
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Putting it in print: oral drug branaplam lowered huntingtin, but safety concerns halted development
⏱️10 min read | Results from the VIBRANT-HD trial are now published in a peer reviewed journal. This trial tested the oral drug, branaplam, which lowered HTT but had serious safety problems, ultimately halting the trial.
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Phase 1 results announced for Skyhawk’s drug SKY-0515
Skyhawk Therapeutics shared results for SKY-0515, a drug designed to lower huntingtin. The drug appeared generally safe, with 60% reduction of huntingtin in blood at the highest dose tested. This data is an encouraging step as larger trials continue.
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Keeping up with HD research just got easier: Introducing the HDBuzz Trial Tracker
⏱️6 min read | Ever wonder when to expect the next update from a clinical trial for Huntington’s disease? The HDBuzz Trial Tracker puts future updates for HD trials on a clear timeline, with deeper explanations in our articles when you want them.
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UniQure Secures Type A Meeting with FDA: What This Means for AMT-130
⏱️6 min read | UniQure secured a Type A FDA meeting, a high-priority discussion for urgent issues. Within 30 days, both sides will discuss what kind of data package might support the advancement of AMT-130 in the US.
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Putting Huntington’s disease medications to the test: results from the Neuro-HD trial
⏱️6 min read | A clinical trial, Neuro-HD, compared 3 common drugs for HD symptoms. The 1 year study found no “best” medication, but clear differences between treatments. The results support a personalised, symptom-by-symptom approach to HD treatment.
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First participants dosed in new POINT-HD huntingtin-lowering trial
POINT-HD has begun dosing its first participants with the drug RG6496, marking an early but important step for a new selective huntingtin-lowering approach.
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SOM3355 Moves Toward Phase 3 as Both EMA and FDA Signal Support
SOM3355, a drug to help manage HD symptoms, has received a positive opinion from the EMA for orphan drug designation, and the company is aligned with the FDA after its End-of-Phase-2 meeting. A global Phase 3 trial is expected to start in 2026.