Phase III failure a death knell for Ampio?
This article was originally published in Scrip
Small Colorado biotech Ampio Pharmaceuticals tanks after its lead product fails in a late-stage trial, with cash waning, the biotech might not survive until next year.
Ampio shares fell more than 65% to linger near $2.72 on 20 April after the company announced the results of the STRIDE study.
The Phase III study compared Ampion, a low molecular weight filtrate of an FDA approved biologic, with placebo in the treatment of osteoarthritis of the knee. Patients were given three 4 mL intra-articular injections of either Ampion or saline over the course of four weeks.
Ampion failed to reach its primary endpoint versus the saline, but Ampion showed a statistically significant reduction in pain compared with baseline, said the company.
Ampio is trying to blame the failure on several different factors, including an abnormally high placebo rate. According to the company, saline is the standard placebo used in osteoarthritis trials, even though patients are known to show a therapeutic benefit with saline – typically about 30% to 35%. Ampion claims that the placebo arm showed a benefit of 12% to 60%.
The biotech also tried to use demographics of the trial as an excuse – previous clinical trials of Ampion were conducted in patients whose condition was not as severe. Patients in the STRIDE trial also weighed more.
Analysts have been skeptical of the STRIDE trial since its initiation last year. "This MI study would be the first multi-injection trial and therefore we're unclear of the placebo response, and we lack confidence in its success," wrote Jefferies analyst Biren Amin in a November note.
The firm says it will discuss the results with the FDA and hopes to file a Biologics License Application (BLA) based on the results of the previous two late-stage clinical trials. Yet, BioMedTracker rates the likelihood for approval of Ampion to be about 47%, about 15% below average.
Ampio claims it has a long enough cash runway to get it through 2016 and allow it to conduct clinical trials if necessary.
This isn't the first time Ampio has had issues with Ampion clinical trials – about 70% of the product being used during the STEP trial was exposed to cold temperatures, compromising the quality of the drug.
Results from the STEP trial could not be used to support a filing with the FDA.