Aspartyl-alanyl-diketopiperazine (Ampion), an intra-articular injection, works to regulate immune responses that are connected to pain, inflammation, and joint damage in patients with osteoarthritis of the knee.
Ampio Pharmaceuticals, Inc. announced the positive data of their Phase 3 clinical trial, AP-013, which analyzed the efficacy of aspartyl-alanyl-diketopiperazine (AmpionTM) on patients experiencing severe osteoarthritis of the knee (OAK), which currently affects more than 200 million people worldwide.
Ampion, an intra-articular injection, works to regulate immune responses that are connected to pain, inflammation, and joint damage in this patient population. Recent results have shown that the drug reduces inflammation by regulating pathways responsible for overproducing certain inflammatory proteins while stimulating anti-inflammatory proteins that are linked to tissue growth and healing.
AP-013, initiated in June 2019, was impacted by the COVID-19 pandemic, resulting in massive quantities of missing data. Ampio followed the FDA’s recommendation of conducting a sensitivity analysis to evaluate the influence of COVID-19 on the study.
“This sensitivity analysis found a statistically significant impact from COVID-19, and as specified in our study plan, we proposed a modified Intent-to-Treat (mITT) population to assess efficacy,” stated Holli Cherevka, Ampio’s President and Chief Operating Officer. This meant that Ampio would present 3 efficacy analysis populations as opposed to the standard 2 populations to the FDA for consideration.
In the study, which expanded upon the first trial, AP-003-A, patients received either Ampion or a saline control. Primary endpoints included improvements in pain as well as function.
A total of 618 patients were included in the mITT cohort, which showed that Ampion significantly reduced pain (p=0.042) and had favorable improvements in functions when compared with the control. Additionally, results from the Per Protocol efficacy population (n = 580) reported a significant reduction in pain (p=0.020) and significant improvements in function (p=0.027) when compared with the placebo.
Earlier this year, Ampio submitted a Type C meeting request to the FDA as well as a detailed briefing package. In addition to improving pain and function, investigators theorize that Ampion may work to stop or reverse disease progression. This will be evaluated in future trials.
“I believe we are on track to provide clarity on AP-013 by the end of the first half of this year, as previously guided,” stated Mike Martino, Ampio’s Chief Executive Officer and Chairman.