Aug. 20 at 11:38 AM
BofA🏁
$PRQR and said -- We initiate coverage of ProQR with a Buy rating and
$8 PO.
$MIRM $IPSEY IONS
BofA added—AX-0810 (NTCP, the liver's bile acid transporter) provides the first human validation of Axiomer, ProQR's technology for making precise, temporary RNA changes.
ProQR initially targets biliary atresia, a childhood disease where damaged bile ducts can lead to liver failure, affecting roughly 20k patients globally with no approved disease-modifying therapy.
'0810 and next-generation '0811 aim to reduce toxic bile acid exposure inside liver cells while preserving NTCP's other functions.
This differs from competitors Bylvay (Ipsen) and Livmarli (Mirum), which block intestinal bile acid recycling. Bylvay's recent Phase 3 failure on native liver survival removes a competitor and, in our view, reinforces the need for a liver-directed approach such as ProQR's.
