A few weeks ago in a tread regarding the Kite acquisition members of this board (MONYMAN3 and TREEPAK) raised the topic of IONS. Read everything I could find on the company and their pipeline. I took a small position and have been closely following. The following is the most recent news on their progress.

29% Objective Response Rate and 57% Disease Control Rate in Combination with Immune Checkpoint Inhibitor Treatment in Patients with Head and Neck Cancer CARLSBAD, Calif., Sept. 11, 2017 /PRNewswire/ – Ionis Pharmaceuticals, Inc. (NASDAQ: IONS) today reported that new data from the clinical program for IONIS-STAT3-2.5(Rx) (AZD9150) were presented at the 2017 European Society for Medical Oncology (ESMO) Annual Congress. The presentation included clinical and safety results from the Phase 1b/2 study evaluating the activity of IONIS-STAT3-2.5(Rx) in combination with Imfinzi (durvalumab), AstraZeneca’s programmed death ligand (PD-L1) blocking antibody, in patients with advanced solid tumors and recurrent metastatic head and neck cancer. In the study, the combination treatment resulted in encouraging antitumor activity with a safety and tolerability profile supportive of continued development.

The stock is up about 15% over the past month.


Hyde marsh,
I am trying to do more DD too. Just very hard w two little ones at home and my wife and me working full time.

A few questions that I am trying to answer now.

  1. Does each drug that the bring through the pipeline have essentially the same safety concern (platelet (sp?) reduction)?
  2. If so, does IONS really have a handle on it through monitoring as they claim?
  3. How safe is Spiranza?
  4. Are the 2.5 and 3.0 w LICA really safer and more effective.

As I said originally, there does not seem to me to be a question about the efficacy of most of their drugs. It is and always has been a safety concern. This is obviously a big problem. But, most diseases the company targets, kill. So the risk/reward measurement skews in their favor. Now, if low platelet is really the only major concern, drug to drug, and IONS can demonstrate to FDA that w monitoring it can be treated effectively, does that essentially solve this problem going forward? I do know from research that there have been some other issues-liver, injection sight reactions, etc. But, not sure how major. Finally, I was under the impression that had a clean safety profile. It is administered to infants after all. I read one Seeking Alpha article from last year that does indicate safety. But, Spiranza was since approved, sales have been amazing, and I have not yet read of further issues. If Spiranza really is safer than the rest of IONS’ pipe, why? What makes it different? I don’t believe it is a 2.5 compound. Meaning, I believe it is the older technology.

My position on IONS today is that at $7bn+ it is not the no brained that it was at sub $4bn 3 months ago. It is more of a prove it now. If they get past the toxicity issues, just on drug performance and the platform, this should be $35bn plus company in 5 years. They will have “proved” it by then.

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I should also mention that I am no longer crazy about biotech in general and am very far from expert on the subject. But, I have been intrigued by the ISIS/IONS story for awhile.

Yes IONS 38 shots on goal (38 drugs in trials). Nine read outs on trials the 2nd half of this year (1 already reported positively). Another “platform” type company that if successful can attempt many extensions. I like it and have been in it as low as $23.45.


I have held IONS (formerly ISIS) since November, 2014. Compared with my other stocks, I have not had a great return (+40%), but I remain very hopeful. With RNA-targeted therapeutics (aka antisense), there are manageable safety concerns with platelet counts. The lower the dose, the less this seems to be a concern.

Why continue to hold IONS? It has a HUGE pipeline:

Rare Disease: 6 drugs in Phase 3 or later, 4 in Phase 2, and 3 in Phase 1.

Cardiovascular Treatment: 3 in Phase 2, one in Phase 1.

Cancer: 3 in phase 2 and one in Phase 1.

Miscellaneous Drugs: Phase 1 (Macular Degeneration), Phase 3 (antibiotic resistant bacteria) treatment), and Phase 2 with MS.

Spinraza is doing very well in the market and may be at an inflection point. I am particularly interested in IONIS-FXIRx, an antisense drug for the treatment of thrombosis. Admittedly, I bought in WAY to early, but I think the next two years will bring the results I have been waiting for.