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Phase 2 Results on AKCEA-APO(a)-LRx Presented in Late-Breaking Clinical Trial Presentation at AHA Scientific SessionsApproximately 98% of patients receiving highest dose achieved reductions in Lp(a) levels below the established threshold of risk for CVD events
Favorable safety and tolerability profile observed in largest study ever conducted in patients with cardiovascular disease and elevated Lp(a) BOSTON and CARLSBAD, Calif., Nov. 12, 2018 (GLOBE NEWSWIRE) -- Akcea Therapeutics, Inc. (NASDAQ: AKCA), an affiliate of Ionis Pharmaceuticals, Inc., and Ionis Pharmaceuticals, Inc. (NASDAQ: IONS), today announced that data from the Phase 2 clinical study of AKCEA-APO(a)-LRx in patients with established cardiovascular disease (CVD) and elevated levels of lipoprotein(a), or Lp(a), were presented in a late-breaking clinical trial presentation at the American Heart Association Scientific Sessions in Chicago November 10, 2018. Elevated Lp(a) is an independent, hereditary risk factor for CVD that cannot be well controlled with lifestyle modifications such as diet or exercise or with treatment using existing cholesterol-lowering therapies. It is estimated that there are 8 to 10 million treatable patients living with cardiovascular disease and elevated levels of Lp(a). “Lp(a) is a hereditary cardiovascular disease risk factor that impacts millions of families and cannot be addressed by diet and exercise. A drug that reduces Lp(a) could be a paradigm shift for the cardiovascular community. Physicians are looking for a tool to treat their patients who today have no pharmacological option to sustainably reduce their Lp(a) levels below 50mg/dL, the threshold for a Lp(a)-driven cardiac event such as a heart attack or stroke,” said Dr. Sotirios Tsimikas, vice president of global cardiovascular development at Ionis Pharmaceuticals and professor of medicine and director of vascular medicine at the University of California San Diego and an international expert on Lp(a). “The data from this study represent an important step forward. These results demonstrate that AKCEA-APO(a)-LRx can greatly lower Lp(a) levels with a favorable safety and tolerability profile. These results warrant advancement of this program to Phase 3 and I look forward to participating in its future development.” Patients were treated with AKCEA-APO(a)-LRx or placebo for at least six months, with some patients treated up to one year. The study met all primary and secondary efficacy endpoints analyzed at 6 months. Results from the study show statistically significant and dose dependent reductions from baseline in Lp(a) levels:
*LSMean: Least squares mean
AKCEA-APO(a)-LRx is an antisense drug developed using Ionis’ proprietary LICA technology, which has potential to produce new drugs that can be used at lower doses and with less frequent administration than non-LICA antisense drugs. Results from separate Phase 1 studies of eight LICA drugs in development, including three at Akcea, have shown that doses up to 30-fold lower than non-LICA drugs can result in consistent target reductions with a favorable safety and tolerability profile. “The AKCEA-APO(a)-LRx Phase 2 study is the largest and longest study to evaluate the Ionis LICA technology platform to date. We are encouraged by the robust reductions in Lp(a) levels and the favorable safety and tolerability profile,” said Brett Monia, Ph.D., chief operating officer at Ionis. “Because Lp(a) cannot be easily targeted with small molecules or antibodies, inhibition of apolipoprotein(a) is a great example of how antisense technology can have an impact where no other therapeutic approaches have proven effective. We look forward to further assessing our LICA platform with the 13 programs now in development.” Development of AKCEA-APO(a)-LRx is part of a strategic collaboration between Akcea and Novartis. If Novartis exercises its option to license AKCEA-APO(a)-LRx after an end of Phase 2 meeting with the FDA, Akcea will receive a $150 million milestone payment of which 50% will be paid to Ionis. After exercise of its option, Novartis will be responsible 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. As part of the collaboration, Akcea has the rights to co-commercialize AKCEA-APO(a)-LRx in selected markets, on mutually agreed terms and conditions. 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 dosing 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 approximately 100mg/dL [250 nmol/L]- more than three times the upper limit of normal). The trial had five cohorts: 20 mg (every 4 weeks), 40 mg (every 4 weeks), 60 (every 4 weeks), 20 mg (every 2 weeks), and 20 mg (every week). The primary efficacy endpoint was the percent change in Lp(a) from baseline at the primary analysis time point (6 months) compared to placebo. The secondary efficacy endpoints were mean percent change in LDL-C, apoB, OxPL-apoB, OxPL-apo(a), and the number of patients reaching pre-specific thresholds of <125 nmol/L (<50 mg/dL) or <75 nmol/L (<30 mg/dL). All patients were treated for at least six months, with some patients treated up to one year. ABOUT Lp(a) For additional information about Lp(a), please see the Lipoprotein(a) Foundation at http://www.lipoproteinafoundation.org/. About Akcea Therapeutics, Inc. About Ionis Pharmaceuticals Our sights are set on all the patients we have yet to reach with a pipeline of more than 40 drugs with the potential to treat patients with cardiovascular disease, rare diseases, neurological diseases, infectious diseases and cancer. We created TEGSEDI™ (inotersen) the world’s first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adult patients with hereditary transthyretin (TTR) amyloidosis (ATTR) that our affiliate Akcea Therapeutics is commercializing. Together with Akcea, we are also bringing new medicines to patients with cardiometabolic lipid disorders. To learn more about Ionis follow us on twitter @ionispharma or visit http://ir.ionispharma.com/. *Spinraza is marketed by Biogen. 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. Akcea Investor Contact: Akcea Media Contact: Lynn Granito Ionis Investor Contact: Ionis Media Contact: |