Ampio Stock Craters After FDA Rejects Trial Changes

Shares of Ampio Pharmaceuticals continue to fall during premarket trading this morning following the company’s announcement Wednesday afternoon that the U.S. Food and Drug Administration rejected proposed changes to a Phase III study of its pain treatment for osteoarthritis of the knee and other joints.

Last month, California-based Ampio released what it called a positive data analysis from its Phase III AP-013 study of Ampion. However, the company noted in previous announcements that the study was found to have been impacted by COVID-19 using a blinded sensitivity analysis. The AP-013 study was initiated in June 2019, prior to the outbreak of COVID-19.

When COVID-19 hit and interrupted AP-013, as well as numerous other clinical studies, it resulted in a large amount of missing data due to the pandemic, Ampio said last month. To mitigate the negative impact, Ampio proposed using a modified-intent-to-treat (mITT) population to assess the efficacy.

Ampio met with the regulatory agency in a Type C meeting earlier this month to address that trial proposal, as well as a proposal for using AP-013 as a second pivotal trial to support a biologics license application for Ampion. On Wednesday, the company announced the FDA rejected these proposals. The regulatory agency said it did not agree with Ampio’s proposed changes to use the mITT population instead of the initial intent-to-treat population.

The FDA said the proposal should have been made prior to Ampio’s having unblinded the data from the AP-013 study. The agency said the switch to mITT is not acceptable due to the Special Protocol Assessment agreement for the trial. Also, the FDA said that P-o13 cannot serve as a second pivotal trial based on both the change in the analysis population and the analysis of pain only, instead of the original co-primary endpoints that were initially established. Those co-primary endpoints included improvement in function.

Because of COVID-19, the trial did not include as many people as initially expected. The company said the trial required a number of patients to evaluate those improvements compared to improvements in pain alone.

Ampio Chairman and CEO Mike Martino expressed disappointment in the FDA’s decision. He said osteoarthritis of the knee is an unmet need in the United States, with approximately 17 million people impacted by the medical issue.

"… we continue to believe that Ampion is a drug which can provide a safe and efficacious treatment for many of those patients. However, given the points in FDA's answer, it will be very difficult to salvage AP-013 itself as a pivotal trial,” Martino said in a statement.

With that in mind, Martino said they believe the best path forward for Ampio and its pain asset is to conduct a new Phase III study. He said the company has “learned a great deal” from the AP-013 study, as well as previous clinical trials of Ampion. That will allow Ampio to design a trial that can lead to regulatory approval, he said.

Ampio shares were down as much as 38% in premarket trading. The stock was trading at 21 cents per share, down from Wednesday’s close of 34 cents.