JPMorgan Healthcare Conference Highlights: Ariad Pharmaceuticals

Earlier this week we kicked off the start of the 2014 JPMorgan Healthcare Conference, which is arguably the most important health-care conference of the year, bringing together pharmaceutical, biotechnology, and medical device makers under one roof.

Just like the recently concluded Consumer Electronics Show in Las Vegas, Nev., this annual event gives health care companies a chance to demonstrate to investors and Wall Street where they've been and where they're headed next. Because earnings guidance can be somewhat irrelevant for clinical-stage biotech and medical device companies, consider this event your chance to gain guidance from some 300 top health-care companies.

Today, we're going to take a closer look at Ariad Pharmaceuticals' presentation, which was given on Tuesday by CEO Harvey Berger.

Some background on AriadPharmaceuticals' past year
I know those of you who are familiar with the Chinese Zodiac calendar are going to call me out for this, but I'd contend 2013 was the year of the ostrich for Ariad shareholders, because I can't think of anything I'd have rather done in the second-half of the year than bury my head in the sand.

Source: Ariad Pharmaceuticals.

The reason was a follow-up safety study released in October which showed that its leukemia drug, Iclusig, had caused an increasing number of arterial thrombotic events (i.e., blood clots) at the two-year mark (11% of patients) than at the 11-month mark (8%). This increase in serious vaso-occlusive events led to a temporary sales suspension of Iclusig in the U.S., a review of Iclusig in Europe, and the discontinuation of Ariad's treatment-naïve chronic myeloid leukemia trial known as Epic. About the only thing Epic was the greater-than-80% drop in share price witnessed over a matter of weeks.

Since then, however, a few things have worked in Ariad's favor. Iclusig has returned to market with more stringent warning labels in the U.S. as of last month, and it's been given the OK to continue selling its drug and filing for marketing applications in Europe.

Now that we have a better understanding of where Ariad Pharmaceuticals has been over the past year, let's have a closer look at its presentation at the JPMorgan Healthcare Conference to decipher where it may head next.

What Ariad Pharmaceuticals had to say
For those of you who aren't familiar with Ariad, keep in mind that while it has eight ongoing clinical studies, there is only one study currently ongoing that does not involve Iclusig. In other words, Ariad's value is based on Iclusig's success, so it really shouldn't be too surprising when CEO Harvey Berger spends 24 of his 27 minutes discussing Iclusig's efficacy, safety, and the many ways Ariad plans to squeeze value for shareholders out of this drug.

As you might suspect, Berger did his best to walk the line on admitting that damage has been done to the Iclusig brand, but also alluding to the bright future that Iclusig will hold for Ariad. One way he really tried to hammer this home is by focusing on Iclusig's efficacy in relation to other approved third-line or greater treatments in chronic myeloid leukemia (CML) patients.

Source: Ariad Pharmaceuticals, Lipton et al, ASH 2013.

The slide pictured above, which was also presented at the American Society of Hematology's annual meeting, shows that Iclusig delivered a complete cytogenic response in close to 60% of treated patients, compared to Pfizer's Bosulif or Novartis' Tasigna, which delivered cytogenic response rates in about half as many patients as Iclusig.

Furthermore, this efficacy data led to comments from Berger that 89% of patients who exhibited a complete cytogenic response maintained that response after two years. The implication, based on data extrapolation, would presume a median treatment life of 10 years -- potentially fantastic news for disease sufferers and investors looking for recurring cash flow. In addition, Iclusig also works quickly, with a response often witnessed within three to four months.

Berger also shared some exciting, and disappointing, figures. At the time of Iclusig's temporary sales suspension in the U.S., Ariad had 640 commercial U.S. patients. Looking ahead, in just the two months that Iclusig was off the market, the FDA approved 370 single-patient INDs (emergency or compassionate use), a majority of which the company believes will become commercial customers. Ariad views its current U.S. market potential as roughly 1,300 people at an annual price point of $125,000. Therefore, optimally, Ariad's top-line revenue figure is around $162.5 million, which is likely not enough to make this company profitable without additional indications.

Finally, Berger spent a short time discussing the potential of API26113, its refractory ALK-positive non-small cell lung cancer drug, which has shown surprising efficacy in cases where brain metastases are involved and has demonstrated good tolerability in early studies.

Making sense of it all
As you might imagine, like most presentations at the JPMorgan Healthcare Conference this one was jam-packed with information, but not a lot that we weren't already aware of.

Despite the select efficacy charts and discussion, which favor Ariad's Iclusig in third-line Philadelphia chromosome-positive leukemias, I really didn't hear how the company planned to change its marketing strategy to rebuild the brand. In fact, we really didn't hear much at all about the relaunch other than that it's ongoing.

To some extent I won't fault Ariad too much for passing over this key point because it's marketable audience is only 1,300 patients in the U.S., and prior to its partial sales suspension it was already treating about half of them. Then again, in the back of my mind I have concerns that Ariad's toxicity warnings could come into play when physicians decide whether to prescribe Iclusig or a competing therapy.

What I'm most pleased to see from Ariad's presentation is that it's exploring a multitude of ways to mitigate the possibility of an adverse cardiovascular event while using Iclusig through multiple dosing level trials beginning in the second-half of this year, as well as potentially administering Iclusig with anti-coagulants which would theoretically help reduce thrombosis.

Ultimately, though, we're looking at a work in progress at best. Ariad has enough money, according to Berger, to make it through the midpoint of next year and perhaps even longer. However, it looks as if it could be years before any additional indications are approved for Iclusig, meaning the potential for ongoing losses is quite real. Everything is going to depend on how quickly Ariad can put Iclusig's issues in the rearview mirror, and frankly the timeline on that is an unknown to everyone.

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The article JPMorgan Healthcare Conference Highlights: Ariad Pharmaceuticals originally appeared on Fool.com.

Fool contributor Sean Williams has no material interest in any companies mentioned in this article. You can follow him on CAPS under the screen name TMFUltraLong, track every pick he makes under the screen name TrackUltraLong, and check him out on Twitter, where he goes by the handle @TMFUltraLong.Try any of our Foolish newsletter services free for 30 days. We Fools may not all hold the same opinions, but we all believe that considering a diverse range of insights makes us better investors. The Motley Fool has a disclosure policy.

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