Prilenia, a clinical stage biotechnology company focused on the urgent mission to develop novel therapeutics to slow the progression of neurodegenerative diseases and neurodevelopmental disorders, has filed a European Marketing Authorization Application (MAA) with the European Medicines Agency (EMA) for pridopidine as a treatment for Huntington’s disease (HD). An MAA Read more…
“We are very pleased with these new data demonstrating a statistically significant, dose-dependent slowing of the progression of Huntington’s disease and lowering of NfL in the CSF at 24 months,” stated Walid Abi-Saab, M.D., chief medical officer of uniQure. “We believe this is the first clinical trial of any investigational medicine Read more…
Today, Wave Life Sciences announced positive results from SELECT-HD, our Phase 1b/2a placebo-controlled trial evaluating the investigational therapy WVE-003. These results demonstrate that WVE-003 selectively lowers toxic, mutant huntingtin (mHTT) protein and preserves healthy, wild-type huntingtin (wtHTT) protein for individuals with Huntington’s disease. Trial ResultsIn the analysis, it was compared Read more…
At Month 12, mutant Huntingtin (mHTT) in the blood was lowered by 22% and 43% respectively for 5mg and 10mg doses. A similar result was seen in cerebrospinal fluid, where mHTT was lowered by 21% and 43% respectively, for 5mg and 10mg doses. In addition PTC518 treatment resulted in a Read more…
Earlier this week Sage Therapeutics announced the results of their SURVEYOR study which quantified the cognitive impact of Huntington’s Disease. Using the HD-Cognitive Assessment Battery (HD-CAB) they measured the difference between 29 healthy volunteers and 40 participants with HD. As a secondary outcome the study measured the safety and tolerability Read more…
Today we are very happy in the Huntington community. The announcement from Uniqure that the company has received the Regenerative Medicine Advanced Therapy (RMAT) “Designation for Investigational Gene Therapy AMT-130 in Huntington’s Disease is more than promising”, says President of IHA, Svein Olaf Olsen. The RMAT ‘licence’ is based on Read more…
SOM Biotech, a clinical-stage drug discovery and development company based on a unique proprietary Artificial Intelligence platform (SOMAIPRO®), is pleased to announce that the recruitment of the Phase IIb clinical study with SOM3355 as a treatment for Huntington’s chorea has been completed. After a a Phase IIa study where the Read more…
Prilenia announced today that they plan to submit a Marketing Authorization Application in the EU for the use of Pridopidine in the treatment of Huntington’s Disease. According to Dr. Michael Hayden, CEO of Prilenia “Pridopidine demonstrates consistent treatment benefits across independent measures that are important to patients and families. These Read more…
Scientists from the Weizmann Institute in Israel have published the results of an animal study which holds tremendous promise for the treatment of Huntington’s Disease. They have discovered that two small molecules, called SPI-24 and SPI-77, can effectively reduce mutant huntingtin expression in the brain while leaving the normal gene Read more…
~ Patients treated with AMT-130 continue to show evidence of preserved neurological function with potential dose-dependent clinical benefits relative to an inclusion criteria-matched natural history of the disease ~ ~ Mean CSF NfL continue to demonstrate favorable trends with low-dose patients below baseline at 30 months and high-dose patients near baseline Read more…
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