Summary
- POWER-HD: Community advocate shares her experience
- The HDSA Podcast: Phyllis Foxworth & Dr. Victor Sung
- New PT Guidelines from the EHDN
PTC Therapeutics shares update on PTC-518
PTC Therapeutics announced today results from PIVOT-HD, the Phase 2 study of PTC518 for HD. This experimental huntingtin-lowering therapy is a pill taken by mouth and is a splicing modulator, meaning that PTC518 interferes with and prevents huntingtin protein production by inserting a premature stop sign into the body’s message to make huntingtin protein. Results from 32 study participants who have completed 12 months of treatment with PTC518 showed dose-dependent lowering of expanded huntingtin protein in both blood and cerebrospinal fluid. This means the more drug a participant received (which in the case of this study was 5 mg or 10 mg), the greater the extent of the huntingtin-lowering observed. Additionally, the results showed benefit in clinical measures such as motor symptoms and a good safety and tolerability profile for PTC518 at the 12-month timepoint.
You may remember that in October of 2022, the FDA paused recruitment for US sites in the PIVOT-HD study while they awaited more data to assess changes in the study protocol. Based on the results shared today, the FDA has lifted the partial clinical hold it placed on the program. There is no specific information at this time about what that means for continued study of PTC518 in the United States, but we do know that PTC Therapeutics intends to begin planning a larger, Phase 3 clinical trial for the future. Read the full press release from PTC Therapeutics here.
POWER-HD: Community advocate shares her experience
At HDSA’s 39th Annual Convention in Spokane, WA, HD community advocate and HDSA board member, Jenne Coler-Dark, shared her experience participating in POWER-HD. This online HD data collection platform seeks to collect experiences of people with HD, people at risk for HD, and HD caregivers. There is power in numbers, and your participation could accelerate research and improve clinical support for people with HD. Watch Jenne’s presentation here, or learn more about the POWER-HD platform here.
The HDSA Podcast: Phyllis Foxworth & Dr. Victor Sung
This month on the HDSA Podcast, President & CEO, Louise Vetter, talks about HD with HD neurologist and Director of the HDSA Center of Excellence at the University of Alabama, Dr. Victor Sung, and Senior Manager of Advocacy at HDSA, Phyllis Foxworth. The trio discuss the importance of the voices of people with HD to understand the complexities of the disease and the changing landscape of HD science. Listen to the full episode here.
