PER (ASX) Open House 09/SEP/2024

Originally posted by schueym99: ↑

hi all. Sorry have been off the charts busy at work to the extent I nearly missed the session!

a great summary outlined above by a number of attendees.

some other thoughts
CSL is not in the dmd space so I got the feeling that this would be a real outlier but we will see. No on their strategy roadmap imo so I would be surprised despite the number of ex CSL management and staff we have
tox study a non issue - more of a gate opener rather than a massive positive. I’m not expecting much of a pop on this news which sounds likely by end of September. I thought the discussion re getting the tox hold lifted before the readout was interesting but was thought not to be a positive or negative to the ability to move forward with other indications. A fail in dmd would not limited the ability of the hold to be lifted after the event so MS could be explore for example.
deals deals deals. Regional model a positive. More bites of the cherry and some ability to get early deals to underscore the potential. The parters are very unlikely to be Johnnys back yard pharma. Most likely ( I took that as virtually certain) to be big name pharma. These will therefore all be on a global footprint but several deals will be better than one. And may enable a pre readout derisk without selling the farm.
manufacturing is an often overlooked area and it is a key one for fda. Can the product when approved get to market in a controlled way? Is the quality at scale assured. Sounded positive around this.
No boys are known to have gotten off the drug. No one knows who’s had what but any placebo boys that have continued have presumably not gotten sick / worse than the general decline of dmd patients. Hopefully those that have transitioned will have seen improvement. None have stopped which in a double blind should be taken as the most visible good news
options are contingent on a deal. IMO would b need to be well in the money to inspire conversion. I’m counting them in the ‘bonus’ column now. A real bonus if it happens - despite itsas $1 by Christmas which I am on board with in hope and at some stage if all goes well.
re CR sounds unlikely but 24 hours is a long time…….
it was clear that the management is aligned with shareholder interests, commercial, pragmatic not just chasing science. I also got a strong sense that heaps has been happening in the background. Visible results will only be visible once results or inked deals can be announced. I got a strong sense of confidence that real things are happening behind the scenes.
I know there has been a lot of debate about bonuses etc but will give the benefit of the doubt at this stage. I didn’t get a sense of milking the shareholder but more of a board that knows people are focused and busting their guts on really tangible things that cannot be announced but deserve some reward.
Anthony’s thoughts on results being good were on the basis that the trial set up was much the same as the ph2a study and hence unless we got 9 boys of unique stature then the results should track similarly. I think the 25/50g mix might shed some new light on path forward as has been discussed before.
he also was very focused on the right deal rather than what might look like the right deal. A deal at lower royalties for example with a mega partner with a desire to aggressively drive sales will be better than a high percentage with a desire to mothball the product to prevent competition.
with the 16 placebo boys they will end up in a partially placebo , partially 25/50g cohort. This will be interesting data imo. A 6 month period of decline and then a period of improvement would be very telling imo. Same boy, different results over time. That cannot be a coincidence ! We might get 32 full time on avicursen boys and 16 mixed. 48 total results. The analysis of this will be very insightful imo.
they are working with the trial to try to get results in the first part of December.

all in all, I continue to be pleased with the focus on commercial success and focus on one end game whilst modestly thinking about other indications etc.

count down is now well on as we head to December. I’m tipping results between 1-10 December.

good luck all.

Expand

another thing to add- whist the pul 2 metrics are important to the primary end point, all other approvals have been based on the secondary endpoints.

also pul 2 is import at but parters and patients are more interested with whether the drug stabilises and stops or slows disease progression.

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another thing to add- whist the pul 2 metrics are important to...

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