Ann: Successful MPSVI Safety Review and PARA OA 008 Update, page-35

At 22mins Donna confirms "The results from this study (008)....will support a Provisional Approval application with the TGA."

https://www.tga.gov.au/provisional-approval-pathway-prescription-medicines





PAR's use of Zilosul for OA already meets 4 out of 5 of these criteria imo (Phase 3 first patients dosed takes care of "Evidence of a plan to submit comprehensive clinical data"). The one that still needs to be proven is "Major therapeutic advance". 008 is attempting to show Zilosul's impact on biomarkers associated with the disease progression of OA, and also concomitant impact on pain and function in the same cohort at 56 days, 6 months, and 12 months.

The bar for current OA treatment is low. Doctors tell people to lose weight, or else prescribe NSAIDS, or even opioids. We know the problems these bring, and they only act for a short time.

My question is, what will PAR determine - in terms of how much the TGA will need to see - to be the right time to apply for this Provisional Approval?

Apparently, we only get one shot at Provisional Approval. If it is granted.....bang....we are selling Zilosul to OA sufferers in Australia, and driving market growth and revenues which turns up the heat dramatically on potential BP partners with an eye on US/Europe approval. If it is NOT granted....we've missed our chance, and will only be able to sell in Australia once we get a full label (years away).

PAR management are therefore faced with a very delicate scenario - one which could be quite punishing (in terms of shareholder unrest, SP dip, market disappointment, management reputation etc) in the near term if they get it wrong. Finding the right time, and the right way, to utilise 008 results to show that Zilosul is a major therapeutic advance on current treatments - is their next big challenge in my opinion. A cautious approach would say we should wait for the full 12-months results before applying. However, it is worth remembering that when Donna spoke in the preso above - there was no 12-month secondary endpoint to 008. The primary endpoint of the study is change to biomarkers from baseline to Day 56. We are going to find out imminently if that endpoint has been met. Which begs the question - is this enough?

What would you do in their shoes? Go for TGA provisional approval upon successfully meeting the primary endpoint of 008? Or wait another 10 months so we can furnish our application with 6 and 12 month data too? If biomarkers are down, and people are feeling pain and function relief after 56 days - isn't this already a serious therapeutic advance on current treatments?