The 3 Most Intriguing Stories From the JPMorgan Healthcare Conference

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Last week saw what was undoubtedly one of the most exciting events of the year for the health care industry, the JPMorgan Healthcare Conference. Rarely do you have the opportunity to get some 300 industry leaders in the pharmaceutical, biotechnology, and medical device sectors all under one roof talking about what's going on with their pipelines and where they're headed.

As you might imagine, the conference itself created quite a few double-digit moves (good and bad) in the underlying share price of some presenting companies. Here, I've picked out three stories that really stood out from the rest and should help define this year's conference.

Obeticholic acid
There is absolutely no question in my mind that Intercept Pharmaceuticals' (NASDAQ: ICPT  )  obeticholic acid, or OCA, for nonalcoholic steatohepatitis, or NASH, stole the show. The evidence is right there in Intercept's 545% two-day stock gain.

Intercept's drug could help some 6 million people in the U.S. with an advanced form of NASH, a disease characterized by fatty deposits in the liver that can lead to liver cirrhosis and cancer. Midstage studies of OCA were stopped early after the data safety monitoring board determined that the treatment was exhibiting statistically significant efficacy.

Days later, though, it came out that this nearly perfect data wasn't so perfect after all, with Intercept reporting that lipid levels, specifically relating to bad cholesterol levels, rose in the OCA intent-to-treat arm. In other words, Intercept now has to spend time studying its results to determine if OCA caused this lipid abnormality.

Intercept's JPMorgan Healthcare Conference presentation helped outline the process by which it expects OCA to move forward. First, investors should keep an eye out for full trial data from its midstage OCA study in the fourth quarter of 2014. We know that the data clearly shows OCA was effective, but the overall incidence of lipid abnormalities remains a mystery that I believe everyone wants to know more about. Assuming OCA proves safe, then a phase 3 trial would be expected to begin about a year from now.

In the rare disease department, few stories continue to be more exciting than the evolution of Sarepta Therapeutics' (NASDAQ: SRPT  ) exon-skipping drug eteplirsen, which is designed to treat Duchenne muscular dystrophy, or DMD.

On the same day as its presentation, Sarepta released follow-up data from its ongoing midstage study of eteplirsen as a treatment for DMD at the 120-week mark. Not surprisingly, the data continues to demonstrate a remarkable stabilization in walking ability based on the six-minute walk test, or 6MWT. According to the results, the eteplirsen intent-to-treat arm has witnessed just a 13.9-meter decline in the 6MWT since baseline (more than two years prior), while the placebo arm that was switched to eteplirsen at week 24 has seen a stabilization in the 6MWT, with a reduction of just 9 meters since week 36.

Source: Sarepta Therapeutics.

Better yet, Sarepta's data appears to show that both ends of the spectrum -- those with high 6MWT and low 6MWT measurements -- are benefiting equally from eteplirsen, implying that it could be helpful at multiple stages of disease progression.

We now know that the next step for Sarepta this quarter is to finalize the clinical end point of its phase 3 confirmatory study of eteplirsen, and that it plans to begin enrollment in its much larger study in the second quarter. Given that its midstage study didn't show marked dystrophin production until at least week 24, it's probably going to be close to a year before we have conclusive data on whether this trial will meet its end point. This means the second quarter of 2015 will be a crucial time frame for Sarepta, its shareholders, and exon 51-based DMD patients (about 13% of all DMD) cases.

HiSeq X Ten
The JPMorgan Healthcare Conference is more than just a platform for biopharmaceutical companies to present their latest findings. It's also an opportunity for medical device and diagnostic companies to show off their latest gadgets. Last week there was clearly nothing more exhilarating in this segment than the unveiling of the HiSeq X Ten by Illumina (NASDAQ: ILMN  ) .

Source: Illumina.

Illumina says the HiSeq X Ten, a genetic analysis machine that can easily fit onto a tabletop, crossed the all-important $1,000 sequencing barrier for the human genome. Not only can this machine sequence a human genome for a fraction of the cost seen just two decades ago, but it can do this 10 times as fast as its predecessor, the HiSeq X. This means that a single machine can now sequence 18,000 or more human genomes per year, compared to the HiSeq X's 1,800-per-year capability.

This product unveiling couldn't have come at a more opportune time, with personalized treatment, especially as it relates to cancer diagnoses, expected to be a primary driver of growth in genome sequencing, as well as patient quality of life, over the coming decade. With genome sequencing now a quick and affordable option we can expect Illumina to get its fair share of what analysts predict will be a $20 billion genome-sequencing market.

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Read/Post Comments (4) | Recommend This Article (3)

Comments from our Foolish Readers

Help us keep this a respectfully Foolish area! This is a place for our readers to discuss, debate, and learn more about the Foolish investing topic you read about above. Help us keep it clean and safe. If you believe a comment is abusive or otherwise violates our Fool's Rules, please report it via the Report this Comment Report this Comment icon found on every comment.

  • Report this Comment On January 21, 2014, at 4:29 PM, ScottAtlanta wrote:

    Your suggestion to sell ILMN at it's recent 52 week high around 80....causing a miss of 60 point run up to 140...was a....

    Bad call dude.

  • Report this Comment On January 21, 2014, at 5:07 PM, TMFUltraLong wrote:


    Illumina's valuation - along with much of the market - appears well ahead of where it should be. In retrospect it was a bad call, but I'm not a huge fan of Illumina here despite what analysts have said. Something like 90 times forward earnings is an insane price to pay...


  • Report this Comment On January 24, 2014, at 12:15 PM, ScottAtlanta wrote:

    Just wanted to call you out on that call. But....I did sell ILMN around 117 due to valuation being too high.

    Even tho ILMN is clear leader and will continue to be I agree that at some point the price cannot be sustained and its better to reduce risk with other less valued growth opps.

    I hope to buy back into ILMN when it comes down. Also, I have an ETF with 6% stake in ILMN so I cont. to own it there....

  • Report this Comment On January 31, 2014, at 12:45 PM, DanielB wrote:


    P/E is only a valuable metric when comparing like-companies in a specific industry. Well it happens to be that ILMN is the only company in the industry, having 100% of market share of what the scientific community believes to be the future of medical treatment making unique on-of-a-kind products. It is not farfetched to believe that is the next PFE. Its not just forward 2015 or 2016 earnings we should be looking at, but a company that is positioned perfectly for a 20% YOY growth for the next 20 years. This time horizon is beginning to be priced-in.

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Sean Williams

A Fool since 2010, and a graduate from UC San Diego with a B.A. in Economics, Sean specializes in the healthcare sector and in investment planning topics. You'll usually find him writing about Obamacare, marijuana, developing drugs, diagnostics, and medical devices, Social Security, taxes, or any number of other macroeconomic issues.

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