Forum Topics NEU NEU ASX Announcements

Pinned straw:

Added 3 months ago

$NEU have announced that their first site for the phase 3 trial of NNZ-2591 in Phelan-McDermid Syndrome (PMS) is underway.

ASX Announcement

Here's the short text:

"Neuren Pharmaceuticals (ASX: NEU) today announced initiation of the first investigational site in the United States for its Phase 3 clinical trial of NNZ-2591 in Phelan-McDermid syndrome (PMS), after receiving Institutional Review Board (IRB) approval. Other trial sites in the US are at various stages of the initiation process. This is the first ever Phase 3 trial in PMS, a serious neurodevelopmental disorder with no approved treatments. The randomised, double-blind, placebo-controlled trial will assess treatment for 13 weeks in approximately 160 children aged 3-12 with PMS. All participants may be eligible to continue treatment with NNZ-2591 for 12 months in an open-label extension trial. The trial program is fully funded by Neuren’s existing cash reserves. Neuren conducted an End of Phase 2 Type B meeting and a subsequent Type C meeting with the US Food and Drug Administration, at which alignment was reached on the design of the Phase 3 trial, including the primary efficacy endpoints."

My comments

So this is the Phase 3 trial for the first of several rareneurological conditions for NNZ-2951, and likely the most consequential for thbusiness. If success is achieved here, it increases the likelihood that treating the bioavailability of IGF-1 may hold the key to treatment of several conditions.

As a rare condition, recruitment will take a while, although Neuren will have maintained involvement with the relevant patient advocacy groups. A clear motivation for patients to join the trial, is the possibility of continuing on after the trial period is complete.

Over the coming months expect newsflow like: 1st patient in, 50% patients in, 100% patients in, trial complete, preliminary readout.

Disc: Held in RL and SM

wtsimis
Added 3 months ago

Positive news @mikebrisy and appreciate your insights.

Would be interested to get your thoughts ahead of the FY results this week ?

What are you looking out for in the FY results ?

We have seen a doubling of the SP since April 25 to $17 plus today with a MC of 2.2b?

Are you thinking the market is expecting something special or is it simply a case that the it was underpriced at $8-$9 and we are seeing brokers simply play catch up and appropriately price Neuren and the growth of Daybue via Acadia Pharmaceuticals and the positive momentum with phase 3 trials?

I will take my hat off to Jon Pilcher and Executive team whom recognised the opportunity to purchase shares at lower levels on the 14th Nov 24 . They have re-purchased 4,076,150 shares at a average price of $12.26 at fullfiled the $50million announced. At current SP of of $17+ it has been a sound use of capital delivering shareholders 40%+ returns on invested capital.

Cheers wtsimis

Disc : Held IRL & SM

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mikebrisy
Added 3 months ago

@wtsimis here are a few thoughts - I hadn't done my $NEU prep. yet, so your questions helped prompt me!

Revenue is totally determined by Acadia / DAYBUE, which is known and you can see Jon's charts for that. No big royalty milestones for the foreseeable future. (Maybe FY27). Acadia is de-risked in my view, because they have a good CEO in Catherine Owen, and the Commercial VP also seems to be good. They are also willing to spend more on sales and marketing to expand US-market penetration, which is good for $NEU which is on a revenue royalty. Also, Catherine Owen is very experienced in rare disease treatments in Europe, so I anticipate good decision-making by Acadia for DAYBUE in the EU.

$NEU costs will be driven by R&D and clinical trials, but there is a huge cash pile, to fund that.

In terms of tracking $NEU, I am focused on the following:

  1. DAYBUE - closely track the quarterly revenue trends and KPIs by Acadia Pharmaceuticals. I expect them to hit mid-to-upper end of 2025 revenue guidance.
  2. NNZ-2591 clinical development milestones. PMS phase 3 trial is the next biggie. Just kicked off. So, I will be listening to what Jon has to say about that.
  3. NNZ-2591 - new indication for SYNGAP-1 related disorder. Hopefully we'll hear more about that. But, commercially, it's some way in the future. 5+ years?


On valuation, check my SM valuation, from a year ago.

I had DAYBUE valued at $12 - $16 - $20. This, in retrospect, was probably a little bullish, and if I were to model it again, I'm GUESSING I'd be more like $12 -$14 - $16, i.e., upside muted but downside firmed up.

NNZ-2591, pick a number! I was $0 - $16 - $32, and the addition of the latest indication probably means I'll stick to that, but if all indications succeed (very unlikely, but possible) then the sky is the limit.

On a sum of parts, where I more heavily risk the development valuations, I'm happy to sit with my valuation of c. $24, but with a very wide range around it. I expect that when I redo my valuation the combination of less risk on DAYBUE, more cash, more excitement for NNZ-2591 will see my valuation nudge updwards.

(The analyst consensus is $24.86, which is an average of 8 TPs ranging from $18.60 to $30.90)

Why I like $NEU, is that I cannot see it being worth less than $12, so the upside both from DAYBUE and then from NNZ-2591 is highly assymetric. This is why I refer to my investment thesis for $NEU as "Two for the price of one". We are getting NNZ-2591 development exposure for the value of DAYBUE. Basically, a speculative upside is being almost entirely underwritten by an established commerical product in the market. In other words, buy $NEU and get DAYBUE plus NNZ-2591 for free and a cash pile.

The cash pile was $359m (pro forma) at end of FY24, As you say, they've spent c.$50m buying shares cheaply, but the cash is still flowing in from Acadia at A$62-67m for CY2025, which is probably more than they are spending.

In conclusion, I have no idea what the $NEU share price will do in the short term. Nor do I care that much. My horizon for $NEU is 3-to-5 years, and the thesis is well and truly on track.

Hope that helps. Personally, I would not hold $NEU if I was concerned about the SP in the next 1-2 years. Of course, I can conceive of negative outcomes that would make me act, e.g.,

Scenario 1: DAYBE starts to significantly underperform in the US and it fails to get reimbursement agreements in the UK/EU (and remains on "managed access" indefintely).

Scenario 2: NNZ-2591 PMS Phase 3 initial readout in 2026 is negative.


In those cases, I would probably reduce my holding to reduce the risk exposure. Currently, I am at a c. 5% of my RL ASX portfolio.

I'll have another look at my valuation for $NEU after reporting season, and after I've heard what Jon has to say.

In summary - I am very happy where this one is at.

Disc: Held in RL and SM

26

wtsimis
Added 3 months ago

Cheers and appreciate your perspective as your intimate knowledge of the evolution of Neuren and Acadia for that matter.

If the alignment with Acadia is as what you outline led by Catherine it will enable for smoother developments in Europe , Japan and remainder of the world are brought to market. I am sure it a considerable consideration for Jon and the executive team when they were awarding rights globally. The "managed access" you refer to is that in the UK or across Europe and is it time based ?

The 2-1 reference in your past analysis brought a smile to my face when i first read it as we all enjoy felling that there is value in our purchases.

Much to digest on how the thesis is playing out, but mostly positive and coming from a non medical perspective it has been a good learning curve for myself.

It is not a investment i would traditionally look into as it sits outside my circle of competence but having listened to Jon over the past 2-3 years and the dialogue on SM (led by yourself ) in combination with the positive outcomes being achieved for so many young children and their families it has been rewarding in more ways than simply financial.

Cheers

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