In its Phase III trial, PROOF-HD, Prilenia is currently studying the effects of pridopidine on total functional capacity in people with HD. On Wednesday September 7th at 12 PM Eastern, HDSA will host a research webinar in which Dr. Michael Hayden, CEO of Prilenia, and clinical leads Drs. Andy Feigin and Sandra Kostyk will provide an overview and insights into the progress of PROOF-HD. Attendees will also have the opportunity to ask questions about Prilenia, pridopidine and PROOF-HD. To register for the webinar, click here.
Pridopidine works by increasing the activity of proteins that help brain cells survive under stress. Last week, at the Hereditary Disease Foundation 2022 Milton Wexler Biennial Symposium, Prilenia’s CEO, Dr. Michael Hayden, announced that we can expect results from PROOF-HD in the second quarter of 2023.
To read a community advocate’s take on pridopidine and PROOF-HD, read the most recent post from Gene Veritas here.
Updates from the 2022 Hereditary Disease Foundation Conference
Last week, HD experts from around the world gathered in Boston for the Hereditary Disease Foundation 2022 Milton Wexler Biennial Symposium. Over the course of the 4-day conference, HD scientists shared the latest from their cutting-edge labs, and industry representatives shared updates on clinical developments. The experts of HDBuzz were on the scene to hash out the highlights. Notable talks from day one included a discussion on early biomarkers of HD and the HD-ISS with Prof. Sarah Tabrizi, and a presentation of the latest update from Roche’s GENERATION-HD1 data analysis from Peter McColgan. To read the full day one update, click here.
Day two of the conference included implementing the HD-ISS in clinical trials with Dr. Jeff Long, using artificial intelligence to better understand HD with Dr. Steven Finkbeiner, and interactions between different cell types of the brain from Dr. Michelle Gray. The second day’s presentations also included updates from numerous pharmaceutical companies on their HD clinical pipelines. To read the full day two summary, click here.
HDBuzz reports on high-dose procedure pause in uniQure’s study of AMT-130
Earlier this summer, uniQure shared positive news: the low-dose of AMT-130, an experimental gene therapy for treatment of HD, seemed to be safe and well-tolerated in participants one year after administration. Newer data, however, revealed that three participants in the high-dose cohort experienced serious side-effects in the weeks after undergoing surgery to receive the drug. All have fully or substantially recovered. There is still no clear cause for this outcome, but HDBuzz laid out what we know about AMT-130 so far and what this means for the future of the trial. Read the full article here.