This week's biggest % winners & losers: Oct 18 - 22, 21 (wk 42)

The following are this week's top percentage gainers and losers, categorized by sectors (over $300 mln market cap and 100K average daily volume).

This week's top % gainers

• Healthcare: RDUS (19.86 +31.52%)
• Materials: IPI (48.71 +17.59%)
• Industrials: HEES (45.82 +14.78%), KNX (56.41 +14.05%), MLI (49.42 +13.98%), DY (79.72 +13.46%)
• Consumer Discretionary: GRBK (25.51 +19.54%)
• Information Technology: EPAY (46.87 +14.91%)
• Financials: TSC (31 +41.42%), QD (1.86 +19.4%), ATH (90.08 +18.92%), APO (79.88 +18.48%), TRUP (106.85 +17.21%), MBI (15.25 +13.89%), AMBC (16.87 +13.45%)
• Energy: BRY (9.9 +19.71%), GLOP (5.18 +12.99%)

This week's top % losers

• Healthcare: RVNC (15.28 -32.72%), AERI (10.57 -20.11%), LXRX (5.05 -18.02%), OMER (6.36 -17.72%), NVAX (133.49 -17.57%), IONS (30.26 -13.79%), ACOR (4.12 -12.53%)
• Materials: KALU (100.77 -13.87%)
• Industrials: AZUL (15.55 -20.97%), GOL (6.07 -19.92%), DNOW (7.64 -16.74%)
• Consumer Discretionary: MMYT (26.61 -13.62%)
• Energy: BTU (13.94 -12.69%)
• Consumer Staples: BRFS (3.94 -18.02%), CBD (4.71 -17.51%)
• Utilities: CIG (2.34 -12.9%)

Ionis Pharmaceuticals (IONS) : Biogen-partnered ALS drug trial fails

 

Biogen (BIIB) and Ionis Pharmaceuticals' (IONS) approach to Lou Gehrig's disease flopped over the weekend, leading IONS stock to collapse to a five-year low Monday.

The duo tested their drug, tofersen, in patients with amyotrophic lateral sclerosis, also known as ALS. Patients with this disease lose muscle control due to dysfunction in the nerve cells of the brain and spinal cord.

Tofersen-treated patients didn't improve enough to deem the study a success, and IONS stock tumbled. SVB Leerink analyst Mani Foroohar says the data release marks "another misstep" for one of Ionis' partnered programs in central nervous system disorders. Previously, Roche (RHHBY) discontinued a study of Ionis' Huntington's disease treatment.

IONS Stock Dives On Misstep

Patients showed improvement in function — as measured by a scale for ALS assessment — but the results weren't strong enough to be statistically significant.

Biomarker data also suggested the drug was, in fact, engaging the mutated gene responsible for patients' disease. Further, patients who received tofersen showed greater declines in a marker of neurodegeneration, known as neurofilament, than those who received the placebo.

But the drug also carried some side effects. Serious adverse events occurred in almost 6% of patients who received tofersen vs. none in the placebo group, Needham analyst Joseph Stringer said in a report. Those included inflammation in the spinal cord, an infection called meningitis, a disease of the lumbar spinal nerve root and other nervous system disorders.

=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,"

-=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

=Long trade : IONS +13% (10/17)

a.  Oct. 2:  #6, 37;  vol. 1.2M   +13%

b.  Oct. 4:  #37;  vol. 860K  +10%

Mon, Oct. 16, 2017:  Ionis Pharma presents new data from NEURO-TTR Study; treatment resulted in a 20-point benefit in mNIS+7 compared to placebo 

• Ionis Pharmaceuticals announced that new data from the Phase 3 NEURO-TTR study with inotersen in patients with hereditary TTR amyloidosis with polyneuropathy
• In the study, inotersen-treated patients achieved a mean 19.73-point benefit in the mNIS+7 co-primary endpoint after 15 months of treatment, compared to placebo-treated patients, further demonstrating the clinically meaningful benefit of inotersen treatment.
• A statistically significant benefit in mNIS+7 was also observed at eight months.
• Key safety findings of thrombocytopenia and renal events identified during the study were shown to be monitorable and manageable with routine blood and urine testing.
• Multiple other innovative drugs from Ionis' portfolio of drugs for patients with serious neurological and neurodegenerative diseases will be highlighted as part of the Antisense Oligonucleotide Treatment of Genetic Neurological Diseases plenary session on Tuesday, October 17.

=Biogen (BIIB) reported earnings on Tue 25 July 2017 (b/o)

• Ionis Pharmaceuticals (IONS) -- Biogen's partner on the spinal drug

Biogen (BIIB) clung to gains early Tuesday after its Ionis Pharmaceuticals (IONS)-partnered spinal drug nearly tripled expectations in the second quarter, leading to a 4% guidance boost.

Sales of Spinraza, the Biogen-Ionis drug used to treat spinal muscular atrophy, came in at $203 million, obliterating analyst views for $70 million, Evercore analyst Umer Raffat wrote in a note to clients. Revenue from that drug grew 328% quarter over quarter.

U.S. sales comprised $195 million. Biogen has also built up $30 million in inventory. Raffat cautioned not to read through too far on Spinraza sales which are front-loaded. The first eight weeks cost $500,000 per patient with $250,000 over the next 32 weeks.

For the second quarter ended June 30, Biogen reported adjusted income of $5.04 per share on revenue of $3.1 billion, topping consensus views for $4.41 a share on $2.81 billion in sales. On a year-over-year basis, revenue grew 6% though profits sank 3.3%.

In afternoon trading on the stock market today, Biogen ticked up 10 cents to 284.80, after gaining as much as 4.3% before the open. Shares hit their highest point since April 25 while biotech stocks were generally flat early Tuesday.

=Ionis Pharma (IONS) reported earnings on Tue 28 Feb 17 (b/o)

• #97 on Mon 2/27

Ionis Pharma beats by $0.51, beats on revs; sees FY17 break-even or profitability on the operating line on a pro forma basis :

• Reports Q4 (Dec) earnings of $0.33 per share, excluding non-recurring items, $0.51 better than the Capital IQ Consensus of ($0.18); revenues rose 210.7% year/year to $160.3 mln vs the $97.47 mln Capital IQ Consensus. 
• Preannounced Jan 6: More than $150 mln in Q4 rev vs. $87 mln consensus; FY16 adj. operating income low to mid $50 mln.
• In 2017, Ionis plans to achieve the following objectives:
  • Support Biogen in expanding access to SPINRAZA
    • Assist Biogen with regulatory approval in the EU and filings in multiple countries outside the U.S., including Japan, Australia and Canada
  • Advance volanesorsen and IONIS-TTRRx toward the market
    • File regulatory submissions for volanesorsen with Akcea, if Phase 3 study is positive
    • File regulatory submissions for IONIS-TTRRx with GSK, if Phase 3 study is positive
    • Build out U.S. and EU commercial infrastructure and prepare for commercial launch of volanesorsen through Akcea
    • Assist GSK in preparing for commercial launch of IONIS-TTRRx
  • Advance the pipeline
    • Report clinical data on multiple drugs, including: Phase 3 data for volanesorsen in patients with familial chylomicronemia syndrome (FCS) Phase 3 data for IONIS-TTRRx in patients with transthyretin (:TTR) familial amyloid polyneuropathy (:FAP)
    • Advance volanesorsen Phase 3 study in patients with familial partial lipodystrophy (FPL)
    • Initiate multiple clinical studies
    • Advance multiple LICA and Generation 2.5 drugs in development
  • Broaden the pipeline by adding three to five new drugs into development
  • Continue to advance antisense technology
  • Achieve break-even or profitability on the operating line on a pro forma basis (FY17 EPS consensus $0.78).
• "In 2017, we expect to continue to have a strong R&D revenue base that funds most of our pro forma operating expenses. To that revenue base, we are adding commercial revenue from SPINRAZA royalties. This revenue is nearly all profit to us, in other words we have only a nominal amount of corresponding expense associated with it. For 2017, we expect our operating expenses to be essentially flat compared to 2016; however, the composition of our expenses will change to reflect the evolution of our business. We plan to continue to increase our commercial spending for volanesorsen as our subsidiary, Akcea, prepares to launch volanesorsen globally in 2018. These increases will be offset by a decrease in our R&D expenses, reflecting the fact that our current Phase 3 programs are coming to a close."

This week's biggest % winners & losers: Nov 7 - 11, 16 (wk 45)

The following are this week's top 20 percentage gainers and top 20 percentage losers, categorized by sectors (over $300 mln market cap and 100K average daily volume).

This week's top 20 % gainers

• Healthcare: IONS (42.72 +57.46%),MNKD (0.65 +52.16%),FOLD (9.21 +50%),TRVN (6.08 +49.29%),RARE (82.44 +49.21%),HALO (12.61 +49%),PRTA(63.83 +45.53%),AIMT (25.56 +42.55%),MGNX (29.63 +42.04%),FGEN (22.6 +40.81%),ARIA (12.49 +40.68%),CLDX (4.33 +40.13%),PBYI (50.4 +39.23%),KERX(6.4 +38.72%),ALNY (46.78 +38.61%)
• Materials:LXU (8.48 +42.76%),CENX (9.57 +38.82%)
• Industrials:TPC (26.25 +39.63%)
• Information Technology:TUBE (13.95 +90.82%)
• Financials:FNBC (8.25 +50%)

This week's top 20 % losers

• Healthcare:BIOS (1.35 -48.28%),ARWR (4.22 -25.7%),HMSY (16.55 -21.59%)
• Materials:PPP (0.81 -40.26%),DRD (3.93 -25.85%),HMY (2.54 -25.73%),MUX(2.67 -24.79%),SLW (17.92 -24.71%),EXK (3.74 -22.89%),AU (11.21 -20.94%),KGC(3.25 -20.92%)
• Industrials:GOL (16.52 -23.59%)
• Consumer Discretionary:RGR (47.5 -25.67%),WBAI (13.09 -23.09%),SWHC(21.31 -21.77%),LTRPA (17 -20.93%)
• Information Technology:WUBA (32.5 -21.72%)
• Financials:OMF (19.4 -28.83%),WMIH (1.43 -28.75%)
• Utilities:ELP (8.42 -21.16%)

Great trade : IONS +17% (11/16)

a. 11/3/16:  #97

b. 11/1/16:  #6, 8, 10, 22, 23, 52, 75, 85, 95

IONS — is it a buy?

• 9/7/16:  #37  is  IONS a buy?

• No, a buy was not until a week later, i.e. 9/14 and 9/15