On $QURE: I wrote this in my Sept. 25 newsletter:
"Bear in mind: What we have seen so far of the AMT-130 data are mentioned in a Uniqure press release. Fairly detailed, yes, but it will be important to see the data fully presented at a future medical meeting and published in a peer-reviewed journal.
The validity of external control arms like the one used by Uniqure is still hotly debated, even as regulators, including the Food and Drug Administration, 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."
To be clear: I was also very positive on the data and its regulatory stance at the time. I thought this was a gene therapy that would be approved, even with Vinay in charge.
That looks wrong now, unless we see another pivot a la $REPL.
