AKCEA-APO(a)-LRx Advances as Leading Pharmaceutical Company Exercises Option to License
BOSTON and CARLSBAD, Calif., Feb. 25, 2019 (GLOBE NEWSWIRE) --Akcea Therapeutics Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. Inc. (NASDAQ: IONS), today announced that Novartis has exercised its option to license AKCEA-APO(a)-LRx, a drug to treat patients with elevated levels of lipoprotein(a), or Lp(a), and established cardiovascular disease (CVD). AKCEA-APO(a)-LRx, referred to by Novartis as TQJ230, was discovered by Ionis and co-developed by Akcea and Ionis.
Elevated Lp(a) is an independent genetic risk factor for CVD that cannot be managed by lifestyle modifications, including diet or exercise, or with treatment using existing cholesterol-lowering therapies1,2. It is estimated that there are more than eight million patients living with CVD and elevated levels of Lp(a).
“We are very pleased that Novartis, an established global leader in drug development and commercialization, will now shepherd this landmark therapy through late-stage clinical development and toward the market,” said Paula Soteropoulos, chief executive officer at Akcea. “The Phase 2 study results presented last year at AHA showed that AKCEA-APO(a)-LRx significantly reduced Lp(a) levels below the recognized threshold for cardiovascular risk in patients living with cardiovascular disease and elevated Lp(a) levels with a favorable safety and tolerability profile. AKCEA-APO(a)-LRx is the first and only medicine to do this. We believe that this therapy has the potential to be a significant advance for millions of people affected by cardiovascular disease around the world.”
Novartis will assume responsibility for all future development activities for AKCEA-APO(a)-LRx, including a planned global Phase 3 cardiovascular outcomes study and, pending regulatory approval, global commercialization activities.
AKCEA-APO(a)-LRx is an antisense drug developed based on Ionis’ proprietary LIgand Conjugated
“The Ionis LICA platform is a game-changing breakthrough for our antisense technology. AKCEA-APO(a)-LRx, our most advanced LICA medicine, is a particularly exciting and important new medicine because of its potential to treat millions of patients with cardiovascular disease. This treatment represents one of many important new medicines within our LICA pipeline and illustrates our continued commitment to innovation and in bringing transformative medicines to patients in great need,” said Brett Monia, chief operating officer at Ionis.
Based on the strategic collaboration agreement between Akcea and Novartis, Akcea will receive a $150 million license fee that will be split equally with Ionis. Akcea will settle its $75 million obligation to Ionis in Akcea common stock.
ABOUT AKCEA-APO(a)-LRx AND THE PHASE 2 STUDY
The Phase 2 study was designed to evaluate the safety and tolerability of AKCEA-APO(a)-LRx and to determine the appropriate dose and dose regimen for a planned Phase 3 cardiovascular outcomes study. The randomized, double-blind, placebo-controlled, dose-ranging Phase 2 study included 286 patients with established CVD and high Lp(a) levels (baseline mean of 100mg/dL [250 nmol/L]- more than three times the upper limit of normal). Results from the study showed statistically significant dose-dependent reductions of Lp(a) compared to placebo at all dose levels, including low monthly doses of AKCEA-APO(a)-LRx. In the phase 2 study, 98% of patients in the 20mg weekly cohort and 81% of patients in the 60mg every 4 week cohort achieved clinically significant reductions in Lp(a) levels bringing them below the recommended threshold of risk for CVD events (<50 mg/dL). Treatment with AKCEA-APO(a)-LRx was associated with decreases in LDL-C, apoB, OxPL-apoB, OxPL-apo(a). Most adverse events were mild. The most frequent adverse events were injection site reactions (ISRs). ISRs occurred in 26% of patients and were mostly mild and one patient discontinued due to an ISR. There were no safety concerns related to platelet counts, liver function or renal function. Approximately 90% of patients completed treatment and the rate of discontinuation was comparable between the active and placebo groups. All patients were treated for at least six months, with some patients treated up to one year.
For additional information about Lp(a), please see the Lipoprotein(a) Foundation at http://www.
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AKCEA AND IONIS FORWARD-LOOKING STATEMENT
In this press release, unless the context requires otherwise, “Ionis”, “Akcea,” “Company,” “Companies” “we,” “our,” and “us” refers to Ionis Pharmaceuticals and/or Akcea Therapeutics.
Ionis Pharmaceuticals™ is a trademark of Ionis Pharmaceuticals, Inc. Akcea Therapeutics™, TEGSEDI™ and WAYLIVRA™ are trademarks of Akcea Therapeutics, Inc.
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1. Viney N et al. Antisense oligonucleotides targeting apolipoprotein(a) in people with raised lipoprotein(a): two randomised, double-blind, placebo-controlled, dose-ranging trials. The Lancet 2016. 388: 2239-2253.
2. Kronenberg , Utermann G; J Intern Med. 2013 Jan;273(1):6-30. doi: 10.1111/j.1365-2796.2012.