In our 30-year history, we have made several recommendations that comprised companies with revolutionary platform technologies, suggests John McCamant, biotech sector specialist and editor of The Medical technology Stock Letter.
New technologies are the backbone of innovation and in many cases it is where next-generation therapies are developed that treat previously difficult to treat diseases.
A classic example is Ionis (IONS) — a pioneer of antisense technology that went through a decade or two of the trials and tribulations of novel science and clinical trials before eventually figuring it out. The blockbuster drug Spinraza for spinal muscular atrophy came out of IONS and in 2021 global sales exceeded $2 billion with marketing partner Biogen (BIIB).
In fact, IONS owns the majority of antisense patents and even receives royalties from Alnylam Pharmaceuticals (ALNY) — another antisense leader. Over the years, IONS has licensed its technology and compounds to a long list of global biopharmaceutical players and now sits on over $2 billion in cash with no debt.
Ionis and its partner AstraZeneca (AZN) recently announced positive data from the ETESIAN Phase IIb trial of ION449 (AZD8233), an investigational antisense medicine designed to reduce blood cholesterol levels in patients with hypercholesterolemia by targeting PCSK9. The data were presented at the American College of Cardiology’s 71st Annual Scientific Session & Expo.
The ETESIAN Phase 2b study’s objective was to assess the efficacy, safety and tolerability of ION449 in patients with high-risk hypercholesterolemia. The study met its primary endpoint in reducing serum LDL-C levels by up to 79%. Reducing PCSK9 levels leads to lower LDL-C levels in the bloodstream, which is known to reduce the risk of developing coronary heart disease.
ETESIAN Phase IIb data for ‘449 demonstrated a clear dose-response for both PCSK9 and LDL-C levels. The results underscore the drug development candidate’s potential with a best-in-class efficacy profile and supports further development as a next-generation PCSK9 inhibitor that is self-administered monthly.
In our view, the ability to lower LDL by as much as 79% along with monthly dosing give ‘449 best-in-class potential which would allow it to capture significant market share from the pioneers.
The shares continue to act better than most biotechs these days, and the LDL data above is yet another example of the success with their leading antisense technology. IONS is a buy under $50 a share.