INTROPaul started with a brief introduction and his thoughts:
From 6 Questions down to 1 question.
We are "very confident that we can respond within one month"
Dr SKERRETT PresentationPresentation by Dr Donna Skerrett
- Global Harmonisation : timetable
- History of events leading up to today
- 26 non clinical studies, addressing toxicity from animal studies
- FDA further clarification required on one question out of the 6.
Pathway forwardThere was one outstanding question
Relates to one of the GLP studies performed in rats. Response within one month and mitigation plan.
The process back and forth is something that we expect in a program like OA where the indication will impact a very large population. Thus it is common practice to experience this slower pace and it is due to changes with communication with the FDA mostly due to Covid. All interactions have been written response with the 30 day period. Despite this we are confident we will be able resolve this outstanding issue so we can move forward with the program.
Following slides were presented: (Mozz note- apologies it is a bit unclear but thats how it appeared on my screen, see PAR website later where this will no doubt be posted later)
QUESTION AND ANSWERSHow does the FDA response impact clinical timeline?
Q 1. Although longer than we have expected, it will pose a slight delay. We are looking forward to addressing this to reduce the overall program time. Eg additional site initiation. Bring forward Europe and Aus site recruitments
Q 2. Finance question - what impact does FDA have on our cash position
(Justin - CFO)
We currently have 71 mil cash, this is sufficient capital to commence p3 and 008 and mps studies and pipeline studies (ARDS, CHF). Once we have finalised the process with FDA, the company will have a clearer view and will update the market as reqd.
Q 3. Is focus on rat glp study a normal process or is there some other reason fda focusing on it for some other reason?
(Donna)- We looked at rodents, I'd like to stress that the Question outstanding with FDA regards a tissue finding in one organ, without affected side effects there were no other findings in other species/clinical human studies.
In our clinical experience which consist of approx 600, there are no suggestions of the impact of this finding. While we need to provide this data we don't expect this to be of clinical significance.
The best evidence to see if it is of clinical significance is to monitor it in the upcoming program.
This will be the topic of response we will be provided to the agency in the next 30 days
Q 4 Why is company confident that this time round the response will be received positively?
The previous responses were received positively. Only one q has come back. We are consulting with the experts to thoroughly mitagte such a finding within the clinical process. 6 down to 1 questions -we feel confident we provided all the info the agency and how pps works from a toxicology point of view and also in humans based on PK study conducted this year in humans.
This will be included in our response. We believe this will produce the clearance to proceed.
Q 5. Review process
Has PAR considered getting advice from other consulting experts?
Yes we have expanded the panel to draw on other experts with current and former FDA experts.
Q 6 Is there a risk that FDA could request more testing if they are not satisfied?
(Donna) I think the risk of this is extremely unlikely. We have done a large no. of studies in two species looking at everything they have asked. We believe the q's will be answered by the data that already exists.
Q7 Have agreements been put in place for CRO in Europe and Aus?
Yes a lot of activity has been going on since the IND submission and before then. We have selected and are in contract with some of them. Site feasibility
Bio matching group and statistical
Work has been done DB and randomisation
This background work has been done. So when we get clearance we will be able to quickly move into the next phase.
This prep work has been going on simultaneously across the three areas.
Q8 Why did it take 5 days to say to makrker that there is 1 remaining question?
(Paul) We need to understand and decipher what the FDA is asking us, need to determine this first. Then we need to consult with our experts who are generally in USA so we need to work with them in their time zones. Need to thoroughly understand and investigate the question.
Q9 When is the company likely to partner for OA?
(Paul) Our immediate goal is to get IND open, then the number of co's we have had thus far will turn to more intense partnering discussions. (We have one later this week). Our position is let's get the IND open. We have been open and transparent with potential partners to date. Once it is open then the potential partners will be more willing to talk to us in more specific detail. Some of these companies have stated that PAR have done an amazing job in this amount of time thus far.
Our asset will increase with the IND being opened.
Q10 At any point is there a fast track designation available to PAR?
(Donna) Yes I think fast track will be valuable at time of NDA (not IND) to shorten the time for NDA application.
(Paul) - We are fast track at time of IND but it is not appropriate to discuss this here.
Q11 What is time line for commencement of trials in Europe in Aus?
Aus - by the end of this year
Timeline for Europe we have not yet disclosed as we are still working on some areas around some areas of ethics and that varies by country
Q12 Why did it take FDA to get to this stage in terms of Non clinical study/question?
Why wasn't this data prepared at Pre IND time?
(Donna)
FDA has approved Elmiron (pill format) but FDA has not approved injectable form (though this is approved in other countries) so for IND and NDA the FDA needed drug info and plan for this new format. This did not exist for iPPS. At Pre Ind historical data was discussed and data was not in GLP format.
(Paul) When we had our pre ind, along with consultants, tox sdata we assumed it could be cross referenced but agency wanted to see GLP studies across wide range of different study types.
Q13 How does response from FDA in terms if iPPS compare with Oral PPS?
Is there any relationship between this Q and the experience between oral PPS?
(Donna) It appears unrelated that appears to be reported with oral formulation. Tissue finding in one species only. we have looked at rats and dogs, we have not seen this in dogs or in clinic (Human). Elmiron has its own tox profile. Unlikely to be of any clinical sig. on our end,
Q14 Why do FDA respond in 30 days
(Donna) Responses from the FDA are within 3o days. Our experience to date means at 30 days. Due to covid it appears that written resonses and response cycle is within 30 days.
Q15
DISCLAIMERMy interpretations, best is to hear it for yourself. Have tried to maintain the sanctity/spirit of what was said.
DYOR
(20min delay)