This week's Biotech scorecard newsletter: (Almost) 100% positive vibes
1. $QURE, of course.
The 75% slowing of disease progression reported by Uniqure with its Huntington’s disease gene therapy AMT-130 was the kind of stop-reading-and-gasp moment that crystalizes the profound impact these types of one-time treatments can have for patients dealing with rare diseases.
But, bear in mind...
The validity of external control arms like the one used by Uniqure is still hotly debated, even as regulators, including the FDA, allow them under certain circumstances. It’s been lost to memory, but recall that the AMT-130 study started with a true, sham-surgery control arm that showed no benefit for the gene therapy. It was only after the company reconfigured the study and started using natural history comparators was a benefit observed.
Also:
-- Why $STOK 's quiet move higher makes sense. The stock is up almost four-fold from its April low and has doubled year to date. Why? I asked around...
-- $ACAD was a "clearing event" for $SLNO. Next up, earnings. What's the whisper number for Vykat sales?
Newsletter link below...
