Neuren Pharmaceuticals Limited

ASX:NEU

Neuren Pharmaceuticals Limited

ASX:NEU
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$11.68 (+5.9%)
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#25
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Pinned straw:
mikebrisy
Added a month ago

$NEU gave an update on the engagement with the FDA on NNZ-2591 endpoints for the PMS Phase 3 trials.

ASX Announcement

"Neuren Pharmaceuticals (ASX: NEU) today announced that the US Food and Drug Administration (FDA) has granted a Type C Meeting to discuss the primary efficacy endpoints in Neuren’s planned pivotal Phase 3 clinical trial program for NNZ-2591 to treat Phelan-McDermid syndrome. Neuren previously announced the positive outcomes from a Type B End of Phase 2 Meeting, at which alignment with FDA was reached on the other key features of the Phase 3 clinical trial program. A Type C Meeting was considered by FDA as the best forum for completion of the remaining efficacy endpoints discussion. The meeting will take place face-to-face in early April. Neuren CEO Jon Pilcher commented: “We look forward to another constructive discussion with FDA. Having a confirmed Type C meeting now establishes a clear timetable and in parallel we are continuing all our preparations, planning for mid-year commencement of the first ever Phase 3 trial for children with Phelan-McDermid syndrome.”

My Assessment

We've been waiting for this news, and I think it confirms my puzzlement at the unfolding delay at getting agreement on endpoints. CEO Jon Pilcher had previously indicated that the endpoints would be agreed via email correspondence. Not so.

Clearly, this indicates that there hasn't been a ready agreement on endpoints for Phase 3, or perhaps that $NEU wishes to get greater clarity from the FDA than they have been able to get from the email correspondence.

As I understand it, a Type C meeting is one that is requested by the drug sponsor, and it is not a required part of the End of Phase 2 process.The release says that "A Type C Meeting was considered by FDA as the best forum for completion of the remaining efficacy endpoints discussion." In reading between the lines, this indicates to me that $NEU were perhaps looking for greater clarity on what the FDA was writing in their email responses, and the FDA said something along the lines of "we have written what we've written, if you want to discss further, then request a Type C meeting." I am speculating here, but I am trying to reconcile this latest news with Jon's previous statements.

I expect there will be quite a bit of discussion about this at the FY24 Results presentation, which should be towards the end of this month. Hopefully Jon will illuminate why they've gone down this path.

The other news is that we are getting a first indication on timing for Phase 3:TYPE C FDA meeting in early April, leading to the trial starting in mid year.

If, the endpoints are agreed!

The market doesn't seem to have read any additional uncertainty into this. So maybe, neither should I.

Disc: Held in RL and SM
Slideup
Added a month ago

@mikebrisy , it is a bit surprising as the impression i had previously was that they were just working through the fine print but everyone was more or less on the same level for the design endpoints. I don't see this development as a negative per se, more just part of the process.

I asked chatgpt about what a type C meeting was and it looks like its really just a more formal version of email correspondence. It looks like it is designed to work through any problem areas in person to save time and misunderstandings. I would think that NEU would have a pretty good understanding of what areas are currently ambiguous to them and this should give them the chance to move through these quicker than continued email correspondance.

From ChatGPT

Key Characteristics:

  • Flexibility: Type C meetings can cover a wide range of topics, providing flexibility in addressing specific challenges or concerns in the drug development process.
  • Less Formal than Type A and B: Type A meetings are typically for more urgent issues (e.g., critical development decisions), while Type B meetings are for discussions of major clinical trial development plans (like end-of-phase 2). Type C meetings are the most common type and can address a broad range of issues.
  • Outcome: The meeting will provide the drug sponsor with written feedback from the FDA on the discussed issues, helping to guide the development of the drug.

Examples of When a Type C Meeting Might Be Requested:

  • Determining the appropriate clinical trial design or endpoints for a Phase 3 study.
  • Clarifying statistical methodologies for analyzing clinical trial data.
  • Seeking input on clinical development strategies or preclinical data before advancing to human trials.

In general, Type C meetings are a tool that helps the drug sponsor navigate the complexities of regulatory requirements and ensures alignment with the FDA's expectations throughout the drug development process.

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mikebrisy
Added a month ago

@Slideup Yes, I think this is right.

Jon strikes me as being a pretty cautious guy, and as I think this is the first drug to go through Phase 3 for PMS, if there is any ambiguity around the endpoints, then it would make sense to have the face to face meeting to work through it.

The advancement into Phase 3 (or not) for PMS is hugely consequential for the company. So an abundance of caution is to be welcomed!

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