Join HDSA at 3:00 PM EST on January 20th for an update from Roche on new findings from the data collected in the GENERATION HD-1 study. This 90-minute, community-focused webinar will be an opportunity to hear members of the Roche/Genentech team discuss new data from the study regarding tominersen, ask questions, and participate in a conversation with Roche & HDSA leadership. Register for the webinar here

Annexon Biosciences reports promising Phase II data for ANX005  

Earlier this week, Annexon Biosciences announced positive results from interim data analysis for its phase II study of ANX005 for treatment of HD. ANX005 is an antibody administered via IV to preserve synapses, the connections between brain cells that are damaged by the progression of HD. It does this by blocking C1q, a protein that activates the classical complements pathway, a part of the immune system that becomes overactive in HD, leading to neurodegeneration. Thus far, the study results have shown that ANX005 successfully engages the C1q target, and 50% of study participants have shown improvements in clinical outcomes after 6 months of treatment. The phase 2 trial is ongoing, and more results can be expected in the second quarter of 2022. 

PTC Therapeutics announces innovative platform for discovery of disease-modifying therapies 

An article recently published in Nature Communications detailed how PTC Therapeutics developed compounds that are currently being investigated to treat HD. Many experimental huntingtin (HTT)-lowering therapies that have been explored thus far require more invasive delivery methods to reach the areas of the brain that are most affected by the disease. With the innovative drug discovery platform described in this article, PTC has been able to identify small molecules that specifically target HTT protein. Through a process called splicing, these molecules can insert a message into the code for HTT protein that prematurely stops its production, and have been shown to lower levels of active HTT protein in cell and animal models.  Excitingly, these small molecules can be delivered by mouth for broad distribution through the brain and its surrounding tissues. One of these molecules, PTC 518, is currently being investigated as a potential treatment for HD; a Phase 2 trial is expected to begin recruitment in the near future. Read more about the platform in a press release from PTC Therapeutics here