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.
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