AMT-130 - initial surgical inflammation confounded a tracked biomarker, greater than expected patient stability 24 months later reversed sentiment.
Huntington's Disease - A prospective target of CLPT
Uniqure just reported long-term follow-up data on a greater number of patients. For Huntington’s patients, a lot of them in the high dose cohort are more stable than you’d expect them to be at 2 years. It’s very promising data today, but if this is still true in another 2-4 years, everyone in medicine will know. And hopefully the FDA won’t be stupid enough to gate keep the product behind a large P3 and this goes directly to market should patient stability remain above what’s expected from Huntington’s Disease.
I wrote about this on June 26th here a little bit. There are better programs in scope with wider indications.
Beyond just the scope of Uniqure this matters a lot for ClearPoint. It’s another case of “hey, this worked, and we helped pull off something no one else ever has— come work with us and pay more money upfront now. — Joe Burnett & Jacqueline (Jordan) Keller” (NOT A REAL QUOTE).