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' (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.
Eteplirsen
In the rare disease department, few stories continue to be more exciting than the evolution of Sarepta Therapeutics' (SRPT 3.52%) 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 (ILMN -2.01%).
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.
