While disappointing clinical trial results are not only common, they're what most investors should ultimately expect (only about 10 to 15% of new drugs make it to market). Even so, last week saw three highly-anticipated biotech events all go against long investors. While the prospects for these three stories do vary quite a bit, at a minimum they ought to serve as a reminder that there are no sure things in this space.
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Last week, Oncothyreon and its partner Merck KGaA announced that the company's Stimuvax (a cancer immunotherapy) failed in a pivotal trial (the START) trial. The study was powered to show a six-month improvement in overall survival (90% power) in non-small cell lung cancer (NSCLC), but Stimuvax failed to deliver the anticipated benefit, as the therapy likewise failed the secondary endpoint of progression-free survival.
Merck and Oncothyreon reported "positive trends in subgroups," suggesting that Stimuvax may not be dead. Unfortunately, it's almost certainly going to take new pivotal studies to explore these sub-groups; Merck has tried and failed (twice) to get Erbitux approved in the European Union for NSCLC on the basis of sub-group analysis, and the FDA likewise seldom accepts such analysis.
What happens now? Although institutional investors had pretty modest expectations regarding Stimuvax's success, retail investors bought the Stimuvax story and the stock was crushed on the news.
In any case, Oncothyreon still has a potentially promising drug in PX-866 (a PI-3K inhibitor), with Phase 2 data coming in the first half of 2013.
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Amicus Therapeutics (Nasdaq:FOLD)
While the Stimuvax trial failure was no great surprise, the failure of Amicus Therapeutics' migalastat in a Phase 3 study in Fabry disease was arguably a bigger disappointment. In a study designed to show a nine patient improvement from a placebo, the actual result was just a four patient improvement (13/32 of in the drug group, or 41%, and nine of 32 in the placebo group, or 21%). That difference is not statistically significant, making the trial a failure.
While the study failed to show the hoped for improvement in kidney GL-3 levels, there were several strange things about the study. For starters, the drug didn't work as well in this study as in earlier studies, and though that's not uncommon in cancer drug trials, it's more surprising here. What's more, the placebo response was quite a bit higher than normal - whether that was a product of patient enrollment or other factors, I do not know.
There's still a chance for this drug to work out for Amicus, and its partner GlaxoSmithKline (NYSE:GSK). This was a six-month study, but the company will be continuing on to a 12-month analysis. While good 12-month data would certainly create confusion after this negative six-month data, the FDA is usually more accommodating and flexible on submissions for rare diseases (as has been seen with Sanofi (NYSE:SNY) and BioMarin (Nasdaq:BMRN) in other lysosomal storage disorder drugs). Keep in mind, though, that Amicus has had other trial disappointments and the risk on this name is now considerably higher.
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Hemispherx Biopharma (AMEX:HEB)
We do not normally address sub-$1 stocks at Investopedia, but the case of Hemispherx is strange enough to be worth an exception. Hemispherx has spent the last 16 years casting around for success, but what the company has never done, though, is demonstrate solid and unequivocal efficacy for any of its compounds in clinical trials.
On Thursday, the company faced a FDA panel meeting for its Ampligen drug for chronic fatigue syndrome. At a minimum, investors should read the FDA's pre-panel comments, as it is about as close to a public dressing-down as the FDA ever gives. In short, the agency found that the company didn't follow the FDA's suggestions in regards to running new clinical trials and attempted to get by with post-hoc data analysis.
Not surprisingly, the panel meeting was not an easy or comfortable one for the company, even though it made a lengthy presentation arguing for the drug. Ultimately, the panel had serious doubts about the quality and reproducibility of the data, and voted against approval. While the FDA could, theoretically, overrule the panel and approve the drug that looks like a major long shot given the tenor of the FDA's pre-panel comments.
The Bottom Line
Perhaps there are some CFS patients who could benefit from Ampligen. Unfortunately, I believe that the poor quality of the trials run by Hemispherx (the resulting data) make it a moot point, and I believe this company and stock will continue to serve as clear warnings regarding biotechs that linger on for years at a time and cannot generate interest from proven biotech institutional investors.
At the time of writing, Stephen D. Simpson did not own any shares in any company mentioned in this article.