Ampio Pharmaceuticals, welcome to Groundhog Day. Four years ago, the FDA crushed the company’s stock by ruling one of its pivotal osteoarthritis studies fell short of the required standard. Ampio duly ran another study—only for the FDA to deem the COVID-19-ravaged trial to be unacceptable, sinking the stock once again and leading the biotech to consider embarking on yet another phase 3.
Colorado-based Ampio began the AP-013 trial in 2019 to meet the FDA’s request for another study. Last month, the biotech set out the limitations of the evidence it generated, revealing that COVID-19 resulted in “a large amount of missing data.” Even so, Ampio felt that it had followed the FDA’s advice on studies affected by COVID-19 and emerged with data that confirmed the result of its earlier study.
The FDA sees things differently. According to Ampio, the FDA disagreed with the proposed change to a modified intent-to-treat population for the primary endpoint, adding that the switch is a material change and the company should have sought its agreement before analyzing and unblinding the data.
After talking to its regulatory experts, Ampio thinks there may be ways to rescue the situation. The plan is to follow up with the FDA in the near term to discuss the potential options. However, Ampio has low expectations.
“Given the points in FDA's answer, it will be very difficult to salvage AP-013 itself as a pivotal trial," Ampio CEO Mike Martino said in an April 20 statement. "I want to be clear. At this point, I believe the best path forward for Ampio and [lead drug candidate] Ampion is likely conducting a new phase 3 trial.”
Martino, who took over as CEO late last year, put a positive slant on the situation, stating that the team has “learned a great deal from conducting and analyzing the prior trials” to inform the next steps.
One question is whether investors will support Ampio through those steps. The biotech ended last year with $33.9 million in the bank, a sum it expected to see through to the second half of next year. Shares in Ampio fell more than 30% in after-hours trading, driving the stock down toward 20 cents.