Novartis to pursue transformational therapy to reduce risk of cardiovascular disease in people living with elevated levels of inherited lipoprotein(a)

  • Novartis announced today that it is exercising its option to license the rights to develop and commercialize TQJ230, an investigational agent previously known as AKCEA-APO(a)-LRx, from Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, for targeted cardiovascular therapy 
  • Novartis is now responsible for worldwide development and commercialization of TQJ230
  • Millions of people have elevated lipoprotein(a) (Lp(a)), an independent inherited cardiovascular disease (CVD) risk factor that cannot be effectively addressed by diet and other lifestyle changes
  • If approved TQJ230 could be first in class treatment specifically targeting elevated Lp(a) 

Basel, February 25, 2019 - Novartis announced today that it is exercising its option to license the rights to develop and commercialize TQJ230, an investigational agent previously known as AKCEA-APO(a)-LRx, from Akcea Therapeutics, an affiliate of Ionis Pharmaceuticals, for targeted cardiovascular therapy. TQJ230 was discovered by Ionis and has been co-developed to date by Akcea and Ionis. Novartis is now responsible for worldwide development and commercialization of this asset.

Millions of people have elevated Lp(a), an independent inherited cardiovascular disease (CVD) risk factor. It is estimated that 20-30% of people who suffer from CVD have elevated Lp(a).[1],[2] Currently no treatment exists that specifically targets elevated Lp(a), and diet and other lifestyle changes are also not effective at reducing elevated levels.[3] Results of a Phase 2 study presented at AHA in November 2018 showed that TQJ230 significantly reduced Lp(a) in patients with high Lp(a) and pre-existing CVD.[4] Novartis plans to conduct a Phase 3 cardiovascular outcomes trial with the potential of addressing the Lp(a) patient community's unmet need for effective treatment.

"No treatments are currently available to substantially lower Lp(a). People with this inherited risk factor are facing cardiovascular risks that cannot be addressed effectively with lifestyle changes," said John Tsai, Head of Global Drug Development and Chief Medical Officer at Novartis. "We're excited about the novel, RNA-targeting approach that could be a game-changer for people with elevated Lp(a). If our Phase 3 trial succeeds, we expect that TQJ230 will become the leading treatment option and another pillar of our longstanding commitment to re-imagining cardiovascular medicine."

About Lipoprotein(a) (Lp(a))
Lp(a) is a lipoprotein that travels through the blood. Elevated levels of Lp(a) collect in the arteries, gradually narrowing the arteries and limiting blood supply to the heart, brain, kidneys and legs. This can lead to increased risk of coronary heart disease, atherosclerosis, thrombosis and stroke. Additional information is available through Lipoprotein Foundation at

This press release contains forward-looking statements within the meaning of the United States Private Securities Litigation Reform Act of 1995. Forward-looking statements can generally be identified by words such as "evaluating", "plan", "possibilities", "potential," "can," "will," "expect," "committed," or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for TQJ230, or regarding potential future revenues from TQJ230. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that TQJ230 will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that TQJ230 will be commercially successful in the future. In particular, our expectations regarding TQJ230 could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG's current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About Novartis
Novartis is reimagining medicine to improve and extend people's lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world's top companies investing in research and development. Novartis products reach more than 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 130 000 people of nearly 150 nationalities work at Novartis around the world. Find out more at

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[1]   Buuren, F. V., Horstkotte, D., Knabbe, C., Hinse, D., & Mellwig, K. P. (2017). Incidence of elevated lipoprotein (a) levels in a large cohort of patients with cardiovascular disease. Clinical Research in Cardiology Supplements, 12(S1), 55-59. doi:10.1007/s11789-017-0087-y
[2]   Varvel, S., Mcconnell, J. P., & Tsimikas, S. (2016). Prevalence of Elevated Lp(a) Mass Levels and Patient Thresholds in 532 359 Patients in the United States. Arteriosclerosis, Thrombosis, and Vascular Biology, 36(11), 2239-2245. doi:10.1161/atvbaha.116.308011
[3]   Tsimikas, S. (2017). A Test in Context: Lipoprotein(a). Journal of The American Cardiology, 69(6), 692-711.
[4]   Tsimikas et al. (2018). Late-breaking clinical trial presentation at the American Heart Association Scientific Sessions.

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