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FDA rejects Ampio’s proposed changes to a phase III study of a pain medication for osteoarthritis

Ampio Pharmaceuticals, Inc., a biopharmaceutical company focused on the advancement of immunomodulatory therapies for the treatment of pain resulting from osteoarthritis in the knee and potentially other articular joints, announced the receipt of written responses from the United States Food and Drug Administration (FDA) pursuant to a Type C meeting request submitted by the Company earlier in the year regarding the AP-013 trial results.

Osteoarthritis is the most common form of arthritis, affecting millions of people worldwide. It occurs when the protective cartilage that cushions the ends of the bones wears down over time. Although osteoarthritis can damage any joint, the disorder most commonly affects joints in your hands, knees, hips and spine.

The Company submitted its Type C meeting request to gain clarity from the FDA regarding the acceptability of the proposed modified intent-to-treat (mITT) population as the primary analysis population for evaluating efficacy in the AP-013 trial, and the acceptability of AP-013 as a second pivotal trial to support a biologics license application (BLA) for Ampion.

FDA responded that it did not agree with the Company’s proposed change from the ITT population to the mITT population for the primary endpoint analysis, that mITT is a substantive and material change to the Protocol and Statistical Analysis Plan that is not in accordance with the Special Protocol Assessment agreement, and that despite the COVID related impact on patients and trial centers, the Company should have sought FDA’s agreement on these changes prior to analyzing and unblinding the data.

Mike Martino Chief Executive Officer and Chairman of Ampio, said:

We are very disappointed in FDA’s answer. Ampion has been in development for several years, and many shareholders have remained loyal to the company throughout the ups and downs of that development history. Severe osteoarthritis of the knee is an unmet medical need that affects nearly 17 million people in the United States, and we continue to believe that Ampion is a drug which can provide a safe and efficacious treatment for many of those patients.

FDA further stated that it did not agree that AP-013 could serve as a second pivotal trial for Ampion based on both the change in the analysis population and the analysis of pain only instead of the original prespecified co-primary endpoints.

Mike Martino, added:

However, given the points in FDA’s answer, it will be very difficult to salvage AP-013 itself as a pivotal trial. Nonetheless, we and our regulatory experts believe there may be ways to do that, and we will follow-up with the FDA in the near term to discuss those options. However, I want to be clear. At this point, I believe the best path forward for Ampio and Ampion is likely conducting a new Phase 3 trial. This management team has learned a great deal from conducting and analyzing the prior trials, including AP-013, and believe we are positioned to design and execute a trial that can lead to BLA approval.


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