The editors of HD Buzz recently sat down with leaders from Roche to ask questions on behalf of the HD community about the GENERATION-HD1 clinical trial of tominersen. This interview was conducted right around the time that Roche presented at the EHDN conference. From lessons taken from this trial to future endeavors in HD research, Roche shared information on the community-wide questions that have lingered since dosing was halted in March of this year. While data analysis is still in progress, this in-depth conversation shed light on what we know so far, and laid out a tentative timeline for more answers down the road. To read the full article, visit HD Buzz here.
PTC Therapeutics: Developing an oral Huntingtin-lowering therapy
PTC Therapeutics is developing an oral genetic drug to lower huntingtin, which was tested in healthy adults over the course of the past several months. On September 21st they hosted a conference call to discuss these results and to lay out plans for beginning a Phase 2 clinical trial for HD patients by the end of 2021. The goal of this trial is to test safety and huntingtin lowering in around 100-150 HD gene carriers ages 25 and up, with a CAG repeat of 42-50. Eligibility will also depend on a new score called PINHD that will help to select participants with very early symptoms. There is not yet information on where the global trial will take place, but one exciting announcement from this conference is that PTC will be applying to the FDA for an accelerated regulatory pathway. This could potentially enable approval following a successful Phase 2 trial.
HDSA & Me Webinar NEXT WEEK on October 7th
HDSA & Me returns just one week from today for the next session in the fall installment of this webinar series. Join HDSA at 2 PM (ET) on Thursday, October 7th, for a conversation on psychiatric symptom management in HD with Kristin A. Rapp, LMSW & Michael Schrift, DO. Click here to register.