Exclusive Q&A: Ionis CEO Sees Watershed 2017 On ‘Rapid’ Drug OKs

Ionis is based in Carlsbad, Calif., just north of San Diego, a biotech hotbed.

Ionis is based in Carlsbad, Calif., just north of San Diego, a biotech hotbed.

Ionis Pharmaceuticals (IONS) expects its 28th year in business to be a watershed year, with potentially three new drugs entering the market in 2017, CEO Stanley Crooke told IBD.

For now, Ionis has a pair of drugs on the market — one approved in 1998 and another in 2013 — but over the past four years the $6 billion-cap biotech has prepped to release the trio of new drugs, in what Crooke sees as potentially the “most rapid approvals ever.”

Wall Street is noticing, Crooke says. Though Ionis stock is down 14% for the year, it was up more than 6% in premarket trading in the stock market today, after the company late Friday announced that the Food and Drug Administration has approved its spinal muscular atrophy treatment Spinraza. (Biogen (BIIB), which licensed the global rights to develop and commercialize the drug, was up 3% in premarket trading Tuesday.)

Ionis also rose 3.5% Friday after the company late Thursday announced it had received a $5 million milestone payment from Janssen Biotech in their collaboration on treatments for patients with gastrointestinal autoimmune disease. Shares have traded well above their 50-day moving average since early November. And unlike other biotechs, Ionis stock hasn’t given back any of its gains since Donald Trump’s election.


Crooke won’t speculate on what effect the real estate mogul and reality-TV star will have on the White House and America. But he sees Ionis as well positioned with its antisense tech, a method of making more targeted drugs by using DNA- and RNA-like compounds.

So even as the drug-pricing debate unfolds in 2017, Ionis will be a big winner, says Crooke.

IBD: What catalysts do you see for the biotech industry in 2017?

Crooke: I think the catalysts in our industry that really matter are almost always the performances of individual drugs. And there are, as one of would expect, a meaningful number of drugs that will report important results from advanced clinical trials and also registration activities.

Pricing is a big concern now and it’s a concern that has several components. The first is pricing as it’s been practiced with generics and some of the generic companies, price increases that were considered inappropriate by some, and a broader concern about the ability of health care systems around the world to afford the cost of therapy. The cost of therapy is higher than anyone would like because drug discovery and development is time consuming, still very costly and in most cases somewhat inefficient because it’s limited by technology.

IBD: Any thoughts on legislation that would speed up FDA approvals?

Crooke: I support strong regulations of our industry. I support it because we’re an industry that, to do our work, we expose other humans to sometimes unquantifiable risks. Over the last probably 10 to 15 years … there have been substantial advances in regulatory processes in the U.S. that have made the process more accountable, more transparent and more flexible. I think that all began with AIDS activists who introduced the notion that patients really must have a say in the regulatory process and encouraged other patient advocacy groups to exercise more influence. As a longtime participant in this industry, I’ve been encouraged by many of the reforms.

IBD: How do you see the drug-pricing debate unfolding in 2017?

Crooke: I think the trends are, to a large extent, independent of any interventions. They are driven by much more fundamental concerns. That is, we have an aging population who need to be cared for, especially (for) chronic illnesses. We have a costly and difficult process we must engage in to develop new medicines for patients in need. The prices are, by definition, going to be significant just to pay the bills. And there are very limited dollars to manage all of this, not just in America but around the world.

Our focus from day one has been to invent a new platform for drug discovery technology, (our) antisense technology (which is RNA-targeted, a nucleic acid, as is DNA, that constitute molecules necessary for all forms of life). It is capable of producing better drugs and is also dramatically more efficient than traditional drug discovery processes. We believe all of this pricing pressure is going to have a profound influence on the industry. We believe we’re positioned optimally to be a leader for years to come in what will turn out to be the new drug development and discovery industry.

IBD: What are the biggest upcoming events for Ionis?

Crooke: We have in registration today a drug to treat spinal muscular atrophy (Spinraza, in partnership with Biogen, which got FDA approval late Friday). It’s a catastrophic disease caused by the loss of the function of both of the genes you have to produce a protein called SMN, which is necessary to make and then keep functional the junction between nerves and muscles. So if you don’t have that, the body is essentially denervated chemically. … We’ve completed two phase-three studies, one in infants and one in children. Both were stopped early because of profound efficacy that was demonstrated in every measure we used. The NDA (new drug application) was filed in September, and we’re optimistic it will be granted approval in the U.S. before the end of this year or very early next year, which will certainly make it one of the most rapid approvals ever.

IBD’S TAKE: Good news for Ionis and Biogen’s SMA drug could lead to a major breakout, analysts say. Nusinersen, under the brand name Spinraza, showed early efficacy in phase-three trials. Get the deeper dive in IBD’s Industry Themes.

IBD: Can you go into Ionis’ phase-three drugs?

Crooke: Volanesorsen is a drug to treat severely high triglycerides. These people have such high triglycerides that their blood is thick and milky. And that leads to many health problems that include recurrent pancreatitis, which is potentially fatal; numerous other issues with fat deposits in the body; and substantially increased risk of diabetes. Volanesorsen is focused on these patients with the greatest need. One group is called SES patients and the other group is called FBL patients. We are completing two phase-three trials. This drug has been shown to work effectively in all populations irrespective of level of triglycerides they have.

The next drug is Ionis TTRX. It’s being developed in collaboration with GSK (GlaxoSmithKline). It’s designed to treat a disorder people have called TTRM amyloidosis. What happens in this disease is there’s a protein all of us have in our blood called TTR. And unfortunately if one has mutations — or even without mutations in some older patients — the TTR misfolds. As a consequence, it precipitates in various tissues — sometimes in nerves, sometimes in the heart, generally all over. That forms aggregates in these tissues called amyloid. That amyloid then leads to failure of these organs. The phase-three study we have is focused on patients who have the peripheral neuropathy form of the disease.

IBD: So if all goes well, Ionis will have five drugs through the approval process by the end of 2017. Is this the watershed year for Ionis?

CrookeYes, it is. And that’s exactly the way it should be.

I’m fond of telling the story that when I started Ionis, I told investors that nothing was known about the technology, that almost certainly it would fail and it would be at least 20 years and $2 billion before I knew. It’s turned out to take a little longer and little more money, as it should be. This is a fundamental process of inventing new chemistry, new biology, sorting it all out, building a whole bunch of examples, taking them into clinic, figuring out what works and what fails.

Most drugs with traditional technologies fail, so we fully expected to have some failures along the way and we certainly did. So all of that took perseverance and good science and a long time and a lot of money. So this is, we think, the beginning of reaping the benefit of those many years of investments and we look forward to seeing that happen.


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