QUESTION – Why do you consider the FDA response to our OAK proposal favorable?
ANSWER – The FDA expressed their willingness to work with Ampio on a path forward toward an OAK BLA, even during the COVID Pandemic. We felt that any solution, that did not include re-running the entire trial would be favorable. The options that FDA provided us to consider are all fair, expected and include reasonable options to only use existing data and/or add additional patients to increase our odds of success. Now that we have the FDA response, we will thoroughly review / consider their comments and provide a thoughtful response. Remember, everyone is flying in the dark here during COVID, there are no historical precedents to guide us.
QUESTION – If the SPA included over 1,000 patients to be analyzed, and now will be evaluated with less, why do you feel that you can show significance with less than 1,000 patients?
ANSWER – The KL4 population covering 417 patients and the related treatment protocol that occurred in all of our previous trials were identical:
- All patients, covering all clinical trials, received a single 4 cc injection
- All patients were randomized 1 to 1 (Ampion vs. Saline)
- All patients were included, no data mining, every KL4 single injection patient was evaluated
- All patients evaluated had identical inclusion and exclusion requirements in comparison to the current AP-13 trial.
The summary of the analysis of these 417 KL4 patients, demonstrated statistically significant and clinically meaningful effects of Ampion, compared to saline for pain and function in knee OAK. These results have been previously presented to the FDA and published in a peer reviewed scientific journal (Orthopedics. 2018 Jan 1;41 (1) :e77-e83).
Although the AP-13 study was scheduled to enroll over 1,000 patients, a number of patients had fully satisfied the conditions of the SPA at the time the trial was put on COVID hold. Consequently, given the historic integrated analysis of our previous 417 KL4 patients, we believe that a comparable number of patients, who already fully satisfied the conditions of the AP-13 SPA, may be able to demonstrate safety and efficacy.
QUESTION – Does the FDA have any concerns about adding patients during the pandemic?
ANSWER – No, actually one of the options presented is adding patients, but only if it can be managed safely.
QUESTION – If the Company concludes that it is best to add patients, do we have to wait until the pandemic is under control to begin the trial?
ANSWER – We do not have to wait until the pandemic is over, but we must ensure the pandemic will not negatively influence the collection, or analysis, of the data in the trial. A small add on trial, could be managed without our high-risk patients leaving their homes, a home doctor visit could work and is possible. Remember, this is one option, we would like to not run additional patients.
QUESTION – What happens if you decide to go with the existing data, you unblind it, and then realize you need more patients to achieve significance?
ANSWER – Then we would have to re-run the trial, so the decision is not an easy one to make. We can play it safe, and simply add patients, or become comfortable that the combined statistical data from our previous KL4 patients and AP-13 KL4 patients who completed the SPA conditions would be sufficient to satisfy the FDA, and then choose to unblind the study. We have to do a fair amount of statistical analysis to help us make that decision. The FDA will provide guidance, the final decision will be ours.
QUESTION – Do you have enough money to manage the Covid trials, including the upcoming International trial, and an OAK add-on trial if that is the decision.
ANSWER – Yes, as consistently disclosed we operate the Company with 8-12 months of cash on hand and, if needed we will have sufficient time to access additional capital if / when needed.
QUESTION – What is your opinion of the final outcome?
ANSWER – We continue to believe in the anti-inflammatory therapeutic power of Ampion, which has been demonstrated and supported by (i) pre-clinical / invitro data , (ii) by peer reviewed publications in scientific journals, (iii) previous clinical trials with over 2,000 patients receiving interarticular injections in the knee, (iv) intravenous delivery into the vascular system of COVID patients, and (v) currently testing COVID patients via delivery of inhaled Ampio directly into the lungs.