Ionis Pharmaceuticals: Ph2 Alzheimer's data support pivotal development – positive vs low expectations

Research evidence excerpt

Ionis Pharmaceuticals: Ph2 Alzheimer's data support pivotal development – positive vs low expectations

Equity Research

U.S. Small & Mid Cap Biotechnology

14 May 2026

Ionis Pharmaceuticals

Ph2 Alzheimer's data support

pivotal development – positive vs

IONS OVERWEIGHTlow expectations

U.S. Small & Mid Cap POSITIVE

IONS/BIIB announced the Ph2 CELIA trial showed robust Biotechnology Price Target USD 115.00

reductions in tau pathology and slowing of cognitive decline. Price (13-May-26) USD 77.60

Primary dose response endpoint missed, but BIIB plans to Potential Upside/Downside +48.2% Source: Bloomberg, Barclays Research

move forward with registrational development. We think a

positive for IONS; SHTG launch a major focus for the rest of U.S. Small & Mid Cap Biotechnology

this year Eliana Merle, CFA +1 212 526 9445

eliana.merle@barclays.com

IONS/BIIB (BIIB covered by Emily Field) announced topline data from the Ph2 CELIA study of BCI, US

diranersen/BIIB080/IONS-MAPT-Rx in early Alzheimer's Disease. Diranersen demonstrated Jasmine Fels, PhD

robust reductions in tau pathology (CSF tau and tau-PET) and slowing of clinical decline on +1 212 526 0865

cognitive endpoints across all doses studied. IONS/BIIB reported cognitive benefit was jasmine.fels@barclays.com

particularly pronounced at the lowest dose (60mg Q24W). Tolerability was consistent with Ph1b BCI, US

results and BIIB noted it does not expect to see ARIA (unlike the amyloid-targeting antibodies). Tejas Wein

CELIA did not meet its primary endpoint specifically assessing dose response on change from +1 212 526 3806

baseline on CDR-SB at Week 76, but based on the strength of the biomarker and efficacy data, tejas.wein@barclays.com

BIIB plans to advance diranersen to registrational development.