Akcea Therapeutics’ TQJ230 targets a lipoprotein associated with higher cardiovascular risk.

 Novartis paid Akcea Therapeutics an up-front license fee of $150 million for rights to TQJ230, formerly AKCEA-APO(a)-L_Rx, which uses an RNA platform to target Lp(a), a lipoprotein, that when present at elevated levels, is associated with higher cardiovascular risk. Novartis had previously paid Akcea $225 million in cash and equity investments for TQJ230 and another candidate. Akcea also has the potential to receive potential royalties, licensing fees and milestones of more than $1.6 billion. Novartis will take full responsibility for worldwide development and commercialization of TQJ230.

The drug, if approved, would be the first for the treatment of elevated Lp(a) and therefore is a potential first-in-class therapy that could help millions of patients, according to Novartis. Akcea estimates that more than eight million people worldwide have cardiovascular disease (CVD) and elevated Lp(a) levels. Having high levels of Lp(a) is an independent genetic risk factor for cardiovascular disease that cannot be managed by lifestyle modifications.

Unlike many other big pharma companies that have exited the cardiovascular space due to the limited number of obvious targets still available, Novartis remains committed to developing novel therapies for cardiovascular disease. It did recently have to withdraw one of its lead candidates –– canakinumab –– from regulatory submission after the FDA and EMA raised questions about its efficacy. TQJ230 could fill that hole.

Results of a phase II study of TQJ230 showed that it significantly reduced Lp(a) in patients with high Lp(a) and pre-existing CVD. Novartis will conduct a phase III cardiovascular outcomes trial to provide further evidence of efficacy and safety in a larger number of patients.