Skip to content

Ionis up after cardio-renal pipeline update

September 2, 2020

Ionis Pharmaceuticals (IONS +1.3%) is slightly ahead of the broad market advance following its update on its cardio-renal franchise. Highlights:

Company expects to file at least 10 marketing applications through 2025.

Candidates are based on ligand conjugated antisense (LICA) technology which enhances drug delivery with 20x – 30x greater potency while allowing less-frequent dosing (monthly or less) with an excellent safety profile.

Two Phase 3 programs: AKCEA-TTR-Lrx in transthyretin amyloidosis and pelacarsen in cardiovascular disease (CVD).

Seven additional programs in preclinical – Phase 2.

Phase 3 study of AKCEA-APOCIII-Lrx in patients with high triglycerides and established CVD to launch this year. More than 90% of treated patients in a Phase 2 trial achieved serum triglyceride levels less than 150 mg/dL (normal healthy range). Additional indications include high and very high triglycerides and multifactorial chylomicronemia syndrome.

Pfizer to conduct expanded Phase 2b study of vupanorsen (AKCEA-ANGPTL3-Lrx) in statin-treated patients with elevated non-HDL-C (high levels of “bad” cholesterol) and triglycerides this year. Results to inform on optimal dose for Phase 3 CV outcomes study.

Phase 3 HORIZON trial evaluating pelacarsen (AKCEA-APO(a)-Lrx) is recruiting CVD patients with elevated Lp(a) levels. Lp(a) is an inherited risk factor for CVD.

Licensee Bayer to launch Phase 2b study evaluating anti-thrombotic IONIS-FXI-Lrx in patients with end-stage renal disease on dialysis this year.

Phase 3 study, CARDIO-TTR, evaluating AKCEA-TTR-Lrx in patients with transthyretin-mediated amyloid cardiomyopathy is underway. Data expected in 2023.

Results from two Phase 2 studies assessing IONIS-AGT-Lrx in patients with treatment-resistant hypertension expected this year.

From → Uncategorized

Leave a Comment

Leave a Reply

Fill in your details below or click an icon to log in: Logo

You are commenting using your account. Log Out /  Change )

Twitter picture

You are commenting using your Twitter account. Log Out /  Change )

Facebook photo

You are commenting using your Facebook account. Log Out /  Change )

Connecting to %s

%d bloggers like this: