Jan. 28 at 1:11 PM
$RGNX Clinical Hold Analysis: The Good, The bad
The News: FDA holds RGX-111/121 after a CNS tumor was found in one patient (dosed 4 years ago).
The Risk :
Finding AAV integration with PLAG1 overexpression is concerning. It raises the "Insertional Oncogenesis" flag (virus caused cancer). This implies a scenario closer to the
$BLUE (Lenti-D) safety scare rather than the benign
$QURE (Hemophilia B) event. Resolving this will take months of forensic molecular analysis.
The Silver Lining (The Bull Case):
Crucially, RGX-314 (Wet AMD) is NOT on hold. This program (partnered with
$ABBV) is the company's main value driver. The eye is a separate compartment; the FDA sees the risk as specific to the brain/CNS delivery.
Verdict:
Expect high volatility. If the market prices RGNX solely on the MPS failure, it may be discounting the cash & AbbVie partnership too heavily. However, until the investigation clears the vector, this is likely "dead money" for the short term. Holding for now.
