Ariad Pharmaceuticals (Nasdaq: ARIA ) and Merck (NYSE: MRK ) challenged Murphy's law and beat it. Tempting fate, they named the clinical trial testing their sarcoma drug, ridaforolimus, SUCCEED. And it did.
The phase 3 trial extended the time it took for the cancer to proceed by 3.1 weeks. While that may not sound like much, it only took the placebo group 14.6 weeks to proceed, so that's a solid 21% improvement.
While the data is highly statistically significant -- p-values of 0.0001 don't just happen by themselves -- there are a couple of issues that investors should keep an eye on when the full dataset is presented at medical meetings later this year.
First, the median progression free survival (PFS) was considerably better for the ridaforolimus-treated patients when the investigators measured the data compared with the decision by the independent review committee. PFS is somewhat subjective, and both sets of reviewers were blinded as to whether the patient was taking ridaforolimus or placebo, but it is still a strange result that could use an explanation.
The placebo group also progressed a bit faster than the company had expected -- 3.5 months versus an expected four months to six months -- so investors will want to make sure the trial groups were balanced well when the full data set is presented.
Both issues could be -- probably are -- nothing, but they're worth investigating further.
While getting ridaforolimus approved is most important for Ariad -- Merck plans to submit the data for Food and Drug Administration by the end of the year -- the positive data should give Ariad's investors some hope that ridaforolimus will work in other tumor types.
Pfizer's (NYSE: PFE ) Torisel and Novartis' (NYSE: NVS ) Afinitor both work in the same way as ridaforolimus by targeting a protein called mTOR. Torisel is approved for treating kidney cancer, and Afinitor is approved for fighting a type of brain cancer and as a second-line treatment for kidney cancer patients that have failed Pfizer's Sutent or Bayer and Onyx Pharmaceuticals' (Nasdaq: ONXX ) Nexavar.
If Ariad and Merck can get ridaforolimus approved in additional larger cancer types, they could have a blockbuster on their hands. Might I suggest THRIVE as the name of the next clinical trial?
Matt Koppenheffer wonders how he could be so stupid.