January 17, 2019
BIOINVEST NEWS: Ionis (IONS)
Next generation LICA Technology In Focus At JPM
IONS focused on their next generation LICA technology during its recent JPM presentation. IONS’ partner Novartis plans to initiate an outcomes study for AKCEAAPO(a)-LRx following the recent positive end of Phase II meeting with the FDA. This LICA drug candidate delivered excellent Phase II data at AHA demonstrating very good tolerability with no platelet concerns (no patient in the study experienced a platelet count below 100K/mm3, and incidence of platelet levels below normal was comparable between active and placebo groups). During the presentation, IONS said that following the end of their recent positive Phase II meeting, they have reached an agreement with the FDA on the pivotal trial design. The next steps for the program include a likely opt-in by Novartis, potentially as soon as this quarter. That catalyst would trigger a $150 million upfront payment, and the start of Phase III shortly thereafter.
IONS’ management has done an excellent job transitioning their pipeline towards LICA technology. With 13 pipeline drug development candidates now using the LICA technology, including AKCEA-TTR-LRx, we see IONS’ pipeline rapidly transitioning towards this safer and more potent ASO platform.
Based upon the past issues of platelet drops, we believe this is very important and rather positive. The Phase I/II trial for AKCEA-TTRLRx is underway (monthly dosing) and with data expected in mid-2019, we expect the Phase III program to begin later in H2:19. Beyond AKCEA-TTR-LRx, we expect at least four additional Phase II LICA readouts in 2019 including IONIS-GHR-LRx in acromegaly, IONIS-FXI-LRx in clotting disorders, AKCEA-APOCIII-LRx in CVD and AKCEA-ANGPTL3-LRx in cardiometabolic disorders. The LICA safety database of >600 subjects (>200 subjects over 6 mos) is growing rapidly, and importantly there have been no safety issues related to platelets nor liver or kidney function reported.