=Akcea Therapeutics (AKCA) & Ionis Pharmaceuticals (IONS)

Akcea Therapeutics & Ionis Pharmaceuticals (IONS) announce 'positive' topline results from a Phase 2 clinical study of AKCEA-APO(a)-LRx in patients with established cardiovascular disease and elevated levels of lipoprotein 

Results from the study show:

• Statistically significant dose-dependent reductions of Lp(a) compared to placebo at all dose levels, including low monthly doses of AKCEA-APO(a)-LRx.
• Most patients in the active group achieved Lp(a) reductions below the established threshold of risk for CVD events.
• Treatment emergent adverse events were balanced between the active and placebo groups.
• Most common adverse event was injection site reactions (ISRs). ISRs were mostly mild and occurred in a minority of patients.
• No patient in the study experienced a confirmed platelet level below 100,000/mm3. The incidence of platelet levels below normal (140,000/mm3) was comparable between the active (10.5%) and placebo (14.9%) groups.
• Approximately 90% of patients completed treatment and the rate of treatment discontinuation was comparable between the active and placebo groups.

=Akcea Therapeutics (AKCA) and Ionis (IONS) : FDA rejects rare disease drug

The FDA rejected a drug developed by Massachusetts-based biotech Akcea Therapeutics Inc. in partnership with California-based Ionis Pharmaceuticals that's intended to treat a rare lipid disorder, halting hopes for a treatment for the currently unserved, debilitating disease.


Akcea Therapeutics and Ionis (IONS) receive Complete Response Letter for WAYLIVRA from FDA  

• Akcea Tx (AKCA) an affiliate of Ionis Pharma (IONS) received a Complete Response Letter (CRL) from the Division of Metabolism and Endocrinology Products of the U.S. FDA regarding the New Drug Application (NDA) for WAYLIVRA (volanesorsen).
• "We are extremely disappointed with the FDA's decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them. We continue to feel strongly that WAYLIVRA demonstrates a favorable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward,"

-=Long trade : AKCA +10% (5/18)

Therapeutics confirms FDA Advisory Committee votes in favor of WAYLIVRA for treatment of familial chylomicronemia syndrome  

• Akcea Therapeutics, Inc. (AKCA), an affiliate of Ionis (IONS), announced that the FDA Division of Metabolism and Endocrinology Products Advisory Committee voted 12-8 to support approval of WAYLIVRATM (volanesorsen) for the treatment of people with familial chylomicronemia syndrome. The committee's non-binding recommendation will be considered by the FDA in its review of Akcea's New Drug Application for WAYLIVRA. The PDUFA date for completion of the review of WAYLIVRA is August 30, 2018.
• The Advisory Committee reviewed data from two Phase 3 clinical trials, APPROACH and COMPASS, as well as the ongoing APPROACH Open Label study for WAYLIVRA. Results from the phase 3 APPROACH trial, the largest study ever conducted in patients with FCS, show that patients with FCS treated with WAYLIVRA achieved a statistically significant mean reduction in triglycerides of 77% from baseline and decreased risk of pancreatitis. The most common adverse events in the APPROACH study were injection site reactions and platelet declines. The Committee's input will be considered by the FDA in its review of the New Drug Application for WAYLIVRA. The FDA is not bound by the Committee's guidance, but takes its advice into consideration when reviewing investigational medicines. WAYLIVRA is also under regulatory review in the European Union and Canada.

-=Ionis Pharma (IONS) and Akcea Therapeutics (AKCA) to commercialize inotersen and AKCEA-TTR-LRx

Ionis Pharma and Akcea Therapeutics (AKCA) announce exclusive, worldwide license for inotersen and AKCEA-TTR-LRx; Akcea will pay Ionis an upfront licensing fee of $150 mln, Ionis will purchase $200 mln of Akcea common stock  

The co's announced an exclusive, worldwide license by Ionis to Akcea for inotersen and AKCEA-TTR-LRx, formerly IONIS-TTR-LRx, in a transaction potentially worth up to approximately $1.7 billion to Ionis plus profit sharing payments. The companies are also developing AKCEA-TTR-LRx for hereditary and wild-type forms of ATTR. AKCEA-TTR-LRx is planned to enter clinical development in 2018.

• Under the agreement, Akcea will pay Ionis an upfront licensing fee of $150 million, payable in shares of common stock priced by reference to a recent trading average. Akcea will have rights to commercialize inotersen and AKCEA-TTR-LRx globally. To support commercialization of inotersen, Ionis will purchase $200 million of Akcea common stock priced by reference to a recent trading average. Upon closing this transaction, Ionis' ownership in Akcea will increase by 7%, from 68% to 75%, totaling 64,114,545 shares. Regulatory approval of inotersen and AKCEA-TTR-LRx in the U.S. and EU will trigger milestone payments to Ionis of $50 million and $40 million, respectively, with additional milestone payments due upon approval of both programs in various other geographies.
• The license fee and initial milestone payments may be payable in Akcea common stock at fair market value. Commercial profits and losses from inotersen will be split 60% to Ionis and 40% to Akcea until the first commercial sales of AKCEA-TTR-LRx, after which the profits and losses will be shared 50/50. The costs of the development of AKCEA-TTR-LRx and the profits from its commercialization will be shared 50/50. The license for the two drugs also includes various sales milestone payments of up to nearly $1.3 billion. For this transaction, Ionis was advised by Stifel, Nicolaus & Company, Incorporated and Akcea was advised by Cowen and Company, LLC.

Read more:http://hosting.briefing.com/cschwab/InDepth/InPlay.htm#ixzz59pUZAiUR

=Akcea Therapeutics (AKCA) reported earnings on Mon 26 Feb 2018 (a/h)

Akcea Therapeutics misses by $0.20, beats on revs 

• Reports Q4 (Dec) loss of $0.35 per share, $0.20 worse than the two analyst estimate of ($0.15). Revenue totaled $18.04 mln, up 100% y/y
  • Co expects approval and launch of volanesorsen in mid-2018 in patients with FCS in US, EU and Canada.
  • Co will report top line results in the second half of 2018 from a Phase 2b trial of AKCEA-APO(a)-LRx in patients with high lipoprotein(a).
  • Co expects to report top line results in 2018 from an exploratory Phase 2 study of AKCEA-ANGPTL3-LRx in patients with rare hyperlipidemias.