Novartis Acquires License for Akcea-Ionis' Cardiac-Targeted Drug TQJ230 in $150M Deal to Address Unmet Need in Cardiovascular Therapy

VCBeat (WeChat Official Account: vcbeat) has learned that on February 25, 2019, Novartis paid $150 million to secure the licensing rights for the antisense oligonucleotide TQJ230. Akcea Therapeutics, the developer of TQJ230, will share this funding with Ionis Pharmaceuticals. This decision enables Novartis to conduct Phase III cardiovascular outcomes trials for this RNA-targeted therapy.

TQJ230, formerly known as AKCEA-APO(a)-Lrx, is designed to lower lipoprotein(a) [Lp(a)] levels by inhibiting the production of apolipoprotein(a), thereby preventing the accumulation of a hazardous form of low-density lipoprotein in the arteries (such accumulation poses a risk of obstructing blood supply). Lp(a) is of particular concern because it is a key driver of plaque and thrombus formation and cannot be controlled with existing therapies.

Given that TQJ230 has the potential to fill a gap in the treatment of heart disease, Novartis Pharmaceuticals struck a deal with another combination product under Akcea-Ionis, with a potential value exceeding $1 billion. Novartis has now provided $150 million to exercise its option for TQJ230.

Akcea and Ionis will share this cost equally, a decision made based on the results of Phase II trials, which demonstrated an association between TQJ230 and reduced Lp(a) levels. According to Novartis’ evaluation of TQJ230, mean lipoprotein(a) levels decreased by 35% to 80%. In a subgroup analysis, nearly all participants achieved Lp(a) levels below the threshold associated with cardiovascular disease risk.

Novartis Pharmaceuticals plans to conduct a Phase III cardiovascular outcomes trial for a drug based on these findings. John Tsai, the company’s Chief Marketing Officer, stated that this drug could offer a “game-changing” approach for individuals with elevated lipoprotein(a) [Lp(a)] levels. Novartis has previously heavily promoted cardiovascular drugs, including canakinumab and serelaxin, only to find that the clinical data failed to meet the thresholds required for market approval. Nevertheless, Novartis continues its efforts to expand the scope of its cardiometabolic division beyond heart failure.

Regarding the licensing agreement, Akcea modified its arrangement with Novartis “to establish a clearer framework” under which the partners can negotiate commercialization terms in certain markets. This framework provides Novartis with the flexibility to independently handle drug commercialization and pay higher fees to Akcea based on sales of TQJ230.

Novartis is a multinational pharmaceutical and biotechnology company headquartered in Basel, Switzerland. Its core businesses span various areas, including patented medicines, consumer health, generic drugs, eye care, and animal health. Founded in 1996, Novartis was formed through the merger of two Basel-based chemical and pharmaceutical companies, Ciba-Geigy and Sandoz.

Akcea Therapeutics is a biopharmaceutical company dedicated to helping patients with serious and rare diseases. As a subsidiary of Ionis Pharmaceuticals, Akcea possesses robust capabilities in developing and advancing drug portfolios through the registration stage. The company’s product pipeline covers multiple therapeutic targets and disease areas.

Ionis Pharmaceuticals is a publicly traded pharmaceutical company headquartered in Carlsbad, California, USA. Founded in 1989 by Stanley Crooke, former head of research at GlaxoSmithKline, the company aims to commercialize antisense therapy. Ionis Pharmaceuticals boasts industry-leading drug distribution channels, designed to provide essential medication access for patients with serious diseases.

(Compiled by Ning Chen)