clinical-trial
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Prilenia shares HD trial data with CHDI to help future research
Clinical trial data can drive discovery long after a study ends. Prilenia donated placebo-arm data from 2 HD clinical trials to CHDI, a valuable resource for researchers to better understand placebo effects, HD progression and future trial design.
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Looking back and moving forward: Questions and lessons 4 weeks out from Rocheās disappointing news
ā±ļø10min read | Roche’s decision to stop development of 2 HTT-lowering programmes is disappointing, but the story doesn’t end there. Years of research answered some key questions, raised many new ones, and will inform the next generation of HD trials.
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Putting it in print: a clinical trial trying to rein in runaway pruning
ā±ļø10 min read | Four years after announcing positive results in their trial of ANX005 (tanruprubart) for HD, Annexon has formally published the results. Itās safe, hits its target, and may have helped some participants ā so whatās the holdup?
By Dr Leora Fox -
Roche ends two Huntington’s disease drug programmes after disappointing results
ā±ļø 10 min read | Roche announced it is stopping development of two of its huntingtin-lowering ASO drugs, tominersen and RG6496. This marks a disappointing end to an important chapter in HD clinical research.Ā
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Skyhawk Shares A Closer Look at SKY-0515 Data, Plus A Peek At Expanding US Trial Access
ā±ļø 6 min read | New 12-month data from Skyhawk’s SKY-0515 is in and all four measures that make up the cUHDRS are at or above baseline, beating expected natural decline. Plus, a first look at expanding US trial sites.
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The Other Shoe Drops: uniQure Shares Plans To Submit Licensing Application With The FDA for AMT-130
ā±ļø 9 min read | After a turbulent past year, uniQure have shared encouraging news that the FDA has agreed their Phase I/II study data can serve as the basis for an application that could lead to the approval of AMT-130 in the US.
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First participant dosed in pioneering neural stem cell trial for Huntingtonās disease
A new clinical trial for HD is underway. The first participant has been dosed in REGEN4HD, a study testing whether transplanting neural stem cells is safe as a potential treatment for HD.
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Skyhawk reports encouraging 12-month results for oral Huntington’s disease drug SKY-0515
One year into treatment, SKY-0515 continues to lower huntingtin and shows encouraging trends with signs and symptoms of HD. A larger trial is enrolling to determine whether the drug truly slows HD progression
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A Second Path: uniQure Plans Regulatory Filing for AMT-130 in the UK
ā±ļø 6 min read | While the US regulatory path for AMT-130 remains complicated, uniQure announced plans to seek approval for its HD gene therapy in the UK. Here’s what that means, and what might be to come.
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Two Years In: New Long-Term Extension Data from PIVOT-HD for Votoplam
ā±ļø10 min read | PTC Therapeutics shared 2-year data for votoplam, a daily HTT-lowering pill. Stage 2 participants showed up to 52% slowing of disease progression. Here’s what the data show, what’s still missing, and the roll out of the Phase 3 trial.