Neuren and Darwinism : Neurenism?

When Benjamin Franklin penned a letter to J-B Leroy, noting that nothing can be said to be certain, except death and taxes, he memorialised words that apply to natural persons and juridic persons equally. Just like people, all companies are supposed to – and do – eventually die. Some are meant to die quickly (like Cynata), others have a long and fruitful life before sort of dissipating into the ether. Some firms change so dramatically over an extended period of time that they are hardly recognisable as the original corporate “persona.” Eventually, if a company becomes too big and bloated, it gets torn apart like a rotting carcass in shark-infested waters, returning nourishment back to the sea of intellectual capital.

A few companies, as much as we might wish it, just don’t seem to die. Neuren (ASX: NEU) is one of them.

One of the things I despise about ASX life sciences is the number of zombie companies that keep re-wrapping the same old turd, and seem to defy the corporate Darwinism that should be part-and-parcel of failure. I got plenty of scathing emails about my last slightly dove-ish post about Circadian, yet at least it is a company that may have one more shot at delivering on the promise, even if its market cap has been significantly decimated (lots of emotional shareholders out there … wow!). Neuren simply does not have this potential. There is no robust clinical evidence to date that a Glypromate analogue (a.k.a. NNZ-2566) is going to be useful or efficacious as a therapeutic. The indications that Neuren is now pursuing following a disastrous Phase III study are the sort of ultra-orphan indications that get investors all aroused and make for great bedtime reading, but it’s hard to really see the clinical case for Neuren’s “pipeline” (to use the term generously) – or the financial focus in running so many concurrent indications.

Indeed most of Neuren’s proposed indications are so tough to clinically validate that most proper pharma companies wont touch them with someone else’s barge pole. Certainly the majority of current drug studies for NNZ-2566’s proposed neurological indications are about symptom management, not “cure.”

I mean Fragile-X, seriously?

This condition has such a broad continuum of symptoms and disability that it, quite frankly, mystifies me how you would even recruit and run an effective clinical trial. Plus current fetal screening tests are 99% accurate so this isn’t going to be a rapidly growing patient pool.

Traumatic brain injury?

Over what period of time do you have to see improvement and how do you control for it in a drug setting, vs the “standard of care”? Don’t even get me started on concussion. How long and arduous are the outcome studies going to have to be to show that a neuroprotectant used in the concussion setting confers a lifetime benefit over rest and not thinking too hard for a few weeks? How do you control for it? Who the hell is going to pay for it?

One gets the impression that Neuren has not learned any real lessons from a failed Phase III. I think it’s pretty much generally agreed that Neuren’s Phase III trial design was poorly implemented and even questionable whether treatment of cognitive impairment in patients undergoing cardiac surgery with bypass was an intelligent strategy to understand the purported neuroprotective characteristics of GPE. The current strategy doesn’t seem a whole lot smarter.

Now it seems we have the same old Neuren behavior re-surfacing. By this I mean ploughing ahead with clinical development, without a data-driven basis for doing so. In the last set of announcements I saw in relation to the Phase II Rett’s study from November last year, I frankly scratched my head. How can you call this a success in any way except confirming that in a short-term dose regimen the toxicity / AE profile is not problematic? Very small number of difficult late-stage patients that undergo a huge amount of temporal variability, and a very short duration of clinical evaluation. Even Neuren’s clinical collaborators wouldn’t hang their hat on the data in the public domain – not really a proper Phase II study, more like a Phase I expansion cohort for dose escalation. Apparently the FDA felt similarly because had the data really shown statistically defensible improvement, I am sure they would have been successful in obtaining Breakthrough status.

Moreover, Neuren is the typical over-hyping company that uses the drama of trading halts and (in my opinion) excessive use of forward-looking announcements (like the announcement of submission of an FDA application package for Breakthrough designation just before the JP Morgan conference). I hate that crap. Neuren also has, in my opinion, a fairly murky divide between board and executive team and it needs to properly address this if it is to give shareholders confidence in decision making and objective evaluation of program development progress (i.e. one independent NED – that’s it).

In short, Treagus needs to show that he hasn’t simply transplanted the (purported) autocratic and toxic board dynamic of Acrux into a new entity. He’s deservedly earned his reputation for being a sharp guy, now he needs to show that he can give Neuren a last-ditch chance by cutting back the “pipeline”, showing some plausible clinical strategy and demonstrating a commitment to corporate governance.

Nobody in this picture has Rett's or Fragile-X.

Nobody in this picture has Rett’s or Fragile-X.

23 thoughts on “Neuren and Darwinism : Neurenism?

  1. Time will tell whether Neuren deserves to be consigned to the Darwinian scrap-heap , and in this respect the company is running about 6 months behind schedule in obtaining conclusive outcomes, Meanwhile it may be noted in relation to Fragile X that indeed some ” proper companies ” have pursued this indication , Roche and Novartis among them. In exploring 4 groups of efficacy measures including global function , cognitive and physiological , Neuren seems to be taking a realistic and useful approach to what resembles a spectrum disorder , with severity and range of symptoms linked to numbers of repeats , whether mosaic , and so on . Given limited treatment options currently available for both FX and Rett , they appear to be worth pursuing so that patients and carers can lead happier healthier lives .

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    • It’s always worth pursuing better patient outcomes, but there has to be a scientific basis for it, not just a repackaging of an asset that failed for other indications and selling it to shareholders. I DO actually think that there is a science story behind Neuren, I just struggle to see the chain of decision-making that links data to action and I think the company has a sloppy track-record in this regard. It’s not just behind, it hasn’t justified itself.

      You are correct, some proper companies have pursued Fragile X, including Roche and Novartis – both of whom discontinued their programs last year. In fact, anyone who shares my concern about Neuren’s decision-making ethos should look no further than the hard lessons learned from Novartis’ experience with AFQ056. Drug had a solid scientific thesis, had extensive clinical experience and was widely expected to have impact. Quite the contrary.

      Who knows, maybe if they are successful, they will be a highly desired acquisition target because almost nobody else has an active program. In which case I will be happy to eat my words and join everyone in crowing over another successful Australian/NZ biotech success story!

      Thanks for engaging. I appreciate it.

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    • You are, of course, the long term partner/wife of Bruce Hancox–who is on the Neuren board? If I recall correctly you were both involved in QRXPharma as well. This does not preclude your having a valid opinion, but your connection to the company is not purely about patients, carers and happier lives. You’ve worked in investment advisory and likely have “skin in the game” on this. Even if it is primarily Lang Walker’s “skin” that is exposed to Neuren.

      I, for one, appreciate the debate the good doctor has opened here. As a person with–what is considered by some, a “pathological” cognitive style–I live a highly functional life that I enjoy. Over time, I have become the carer of my carers. We are not a pathology, or a set of symptoms, we are people. We deserve protection; especially where it is not us, but our carers, who place us in clinical trials. When it is our carers, and clinicians, and not us who get to determine whether we “improved”.

      I cannot foresee whether Neuren will succeed or fail; either in terms of clinical significance or making lots of money for investors and their advisers. But I applaud open debate, and I applaud the sensible approach being taken by the FDA. Especially where medications are likely to be delivered to the very young, or even pre-birth; we should take care that we do not deliver a life sentence far more debilitating. The brain is a complex and plastic instrument; remaining astute to unanticipated consequences is vital.

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      • Sorry, yes – I saw that. Apologies.

        I elected not to personalise my response to that extent on the basis that I don’t mind people sharing their opinions. But your point is well made.

        I have incensed a lot of people with this post but my basic point is that Neuren’s public marketing claims don’t fit the data. Given the emotion that is present around something like Rett syndrome, Neuren owes it to patients (and shareholders) to get this right.

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      • i am not ” of course ” the partner/ wife of bruce hancox. i do know a bit about neuren and have a minute shareholding but no other financial stake. as a director of the fragile X Assoc of Australia , i also know a bit about fragile X

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      • Ms Lenart. Perhaps you prevaricate, or perhaps I have unintentionally embarrassed you. At mimimum, you are the former defacto of Mr Hancox and still appear to work in close association. I recall your participation on the QRXPharma conference call together. Your appointment to the Fragile X association is recent, October last year, I believe. This is a governance position and does not require in-depth scientific knowledge. Your qualifications are, as I understand it, in property valuation. Earned some 40 years ago. As far as I am aware, the two (adult) children you share with Mr Hancox are not affected by Retts or Fragile X. Your interest appears to have begun with investment in this company, rather than a neuroscience qualification.

        That being said, I acknowledge your role as an investor in this company, and as noted your opinions are as valid as anyone else’s. They are, of course, opinions. Even those with cognitive “disabilities” such as myself, are entitled to them.

        Do let me know if anything I have stated is incorrect. I am comfortable with correction where valid.

        Liked by 1 person

  2. I don’t see your point about the previous GPE trial, ok it failed, but there’s a new management, different product, and different conditions. People and companies have failures and if they learn from them, which i think they have, then ok.

    TBI – this trial seems well constructed and at lease has good logistic and financial support from the U.S. That for me will be the pivotal one.

    As you say, Treagus is smart and I’m reassured that he has confidence in this company.

    I have one worry. After the Retts results were published, it was clear that they had used a sophisticated statistical analysis which firstly is completely beyond me, and secondly could possibly be forcing a positive result. The FDA, in its response to BD application, didn’t accept this analysis. Neuren then stated that it’s only the neuro division of the FDA that doesn’t accept this approach. Seems odd to me but hopefully we’ll get clarity after Neuren and FDA meet.

    Liked by 1 person

    • Failures are fine, if you can afford them.

      What’s not ok is to gloss over data and proceed just because there is no other option for the company. Neuren has done that once, we need to make sure they don’t do it again.

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  3. I meant to comment on Board independence. You clearly have a strong view about corporate governance and the need for a strong and independent Board structure, which is absolutely appropriate.

    I’ve served at Board level in several UK organisations and been around the block a few times on this question. I’ve seen strong independent Boards; and I’ve seen strong and effective Boards that you couldn’t really call independent (in that there were few NEDs). I’ve also seen a common pattern with Boards that you’d call independent but which morphed into just another part of the corporate structure.

    My point is that’s what really important is the calibre and perspective of the individuals serving on the Board. I’d rather see high calibre, questioning individuals in any structure than individuals in a supposedly independent Board who don’t fulfill that role.

    At face value, and I don’t know the individuals concerned apart from their brief cv’s, the Neuren board seems to (mostly) be an impressive group.

    I fully agree with your general analysis about corporate structure, my rambling point is that there are models which don’t fit the ideal structure, but which can still produce results.

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    • I disagree on this topic of corporate governance. Now, I will say from the outset, it’s always easier to give advice than it is to do it yourself. But I stand by fairly firmly on the premise that one of the BIGGEST reasons why Australian life sciences is weak is because of this issue. Things happen and people get away with stuff that would never fly in the US, either legally or in terms of credibility.

      I think it matters less when a company is private. In fact, in some ways, having a slightly captive board can be very useful because everyone is incentivised to work together for an exit. I do agree that in the private setting, an effective board is more important than an independent board. Frankly, in the private setting – especially for venture backed boards – there IS no independence. Investors take board seats and their Director’s hats come off in a heartbeat.

      Not so for a public company. In decision making and critical review of key data points, there is an absolute obligation to shareholders to have an impartial and objective negotiation between a management team and board. Neuren absolutely doesn’t have this.

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  4. “Certainly the majority of current drug studies for NNZ-2566’s proposed neurological indications are about symptom management, not “cure.””

    I don’t really understand this point. The majority of drug sales are generated by drugs that are not cures – eg every cancer drug. Surely reducing symptoms of a disease provides a fundamental – and therefore commercially valuable – benefit to the patient?

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    • Hi James,

      Absolutely – and I will admit that this is badly written, especially when “cut” out of the context. An orally-available GPE isn’t going to fundamentally address the underlying biological basis of Fragile-X, but what it may do is abate/moderate at specific moments of neurobiological development (though I note that to understand if this works, we don’t need Neuren’s technology, we just need to inject GPE). The challenge has been to determine where best to tackle the problem and what outcome measures to really use given that this genetic syndrome has hugely wide symptomatic range. Most other drug development approaches are about dealing with the psychiatric/behavioural issues, not the developmental neurobiology.

      I suppose where I was going with this, is that for Fragile-X to be really tackled effectively, it needs to be cured as a genetic disease. There is a growing body of evidence that gene therapies may have efficacy as a therapeutic strategy (https://sfari.org/news-and-opinion/conference-news/2012/society-for-neuroscience-2012/fragile-x-syndrome-can-be-treated-in-adulthood). Plus new screening tests for Fragile-X are 99% accurate so this is going to be a declining patient population in the future anyhow.

      And yes, your comment about about commercial value and cancer drugs is spot-on. Though to be fair, we generally don’t aspire to only manage the “symptoms” of cancer.

      Thanks for engaging.

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      • Hi Christian, thanks for all the hard work you’ve been injecting into this debate

        We may be talking at cross purposes but I did not mean to suggest the Retts Phase II was only to establish safety. Proof-of-concept is concerned with among other things : “preliminary evidence of efficacy in a clinically relevant endpoint.” from PubMed.

        My bigger concern is this issue:

        You originally said “But what it raises red flags for is that the study design and outcomes did not offer statistical confidence and this is very problematic for the future of this drug”;
        but in your latest response to me you have changed what is “problematic for the future of this drug” to be the whole BT debacle.

        This is different to what is implied by the assertion that the FDA’s issue with lack of statistical confidence is problematic for the future of the drug. This latter assertion will serve to reinforce the misperception among many that the Phase II trial failed on statistical grounds. It didn’t. The statistical failure was purely in relation to the BT application. Such failure does not imply anything about the likelihood of the drug working or not working. This is an extremely important point, which many do not appreciate. So I continue to believe that your original comment was incorrect, misleading and therefore potentially damaging to the company.

        Regards

        PS – haven’t been at Morningstar for quite some time

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      • I guess we are talking at cross-purposes, I don’t see them as unrelated.

        I understand your point, but I don’t agree. In my view there is nothing complex or contentious about Neuren’s Orphan or Fast Track designation – I think I have been clear on that. Those designations have relatively little to do with the drug’s data and a lot to do with the patient and business case.

        In the case of Breakthrough designation (to the point of your comment), I don’t claim that it is a low bar, but for something like Rett Syndrome, I am confident that the Agency would take a careful look at it given the recent failures in the space and the huge unmet need. In my view, the Phase II study was not really designed to prove efficacy, it was designed to prove safety but capture an efficacy signal. There is a difference.

        As such, I consider the primary outcome of the study to be a success but until Neuren publishes their clinical results I can’t ascertain whether the efficacy signal was meaningful or not – all I have a press release that is factually correct but has no detail.

        Therefore, the only data point that I have right now is that the FDA looked at the efficacy data from the study and concluded that it wasn’t sufficient. Breakthrough status is just once lens but it is an extremely important one and a very objective one.

        And this is why I am annoyed with Neuren. Not only did they show a lack of restraint by “announcing” they had applied for Breakthrough, but they actually unnecessarily damaged the perception of the Phase II study by having to endure a process of retraction. What it does is it now further pressure Neuren to subject themselves to a process of peer review and I really hope it gets done soon so that the damage can be reversed.

        As a final comment – don’t you have any doubt about what the Agency thinks about those kinds of forward-looking statements from drug companies. There is plenty of politics there and they don’t suffer fools AT ALL. Not only was the preemptive announcement crass and unnecessary, it would have been a red flag to a bull as far as the FDA is concerned.

        So, I’m flattered that you think my comments are potentially damaging. I don’t think anyone really cares enough about my opinions to view them as damaging. If they are damaging, I regret that, but they are not unsubstantiated. Anyhow, as you commented in your previous post, I lack credibility so why do you care anyhow?

        Regards,

        PS: You need to update your online profile then

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  5. Let me blunt, this diatribe of nonsense isn’t worth the time you took to write it. Speaking as a parent whose child went through the Phase II Rett trial, I can assure you, that it was in no way a failure. Breakthrough Status is given to only 30% of applications, so it is not surprising that it was not received on the first try, disappointing, though. While TROFINETIDE, (NNZ-2566) is not a cure at the cellular level, and it appears only you find this astonishing, it is a possible “virtual” cure by addressing multiple life threatening and life shortening symptoms, not unlike “Lorenzo’s Oil”.

    Neuren and Trofinitide represent the best and ONLY hope right now that children with Rett syndrome and Fragile X may have a chance, how dare you scoff at it. I can personally defend the improvements seen with Trofinitide. Also, Rett doctor after Rett doctor HAVE come out and publically spoken about their support of that trial, Neuren and Trofinitide. In addition, the US Army, which has funded Trofinitide and it’s use in TBI would not continue to fund it if they personally were not seeing positive results. What one sees with their eyes does not always translate well into statistics, but you should be more careful before you wish for the only hope Fragile X and Rett syndrome suffers have to die and fade away, especially when it works.

    And, in reference to Darwinism, I think that “survival of the fittest and natural selection” will come into play soon enough, with Neuren on top and you proven so wrong as to become obsolete and made to fade a way.

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    • I am sorry if I upset you – I think you mistake the purpose of my commentary. Your response actually makes me wonder if you read a different blog!

      Although I am very skeptical of the benefit of GPE, there is no doubt that at a higher-level IGF-1 is an interesting pathway for possibly addressing Rett Syndrome and there is some growing scientific momentum around this idea as you probably know – likely better than I if you are dealing with this condition in your daily life. You may note that I have not actually “scoffed” at the science, and I certainly did not scoff at the Rett Syndrome study. What I scoffed at was the company, it’s decision-making behavior, it’s clinical strategy/claims and its lack of corporate governance.

      But, for the record, yes – I am skeptical of GPE/Trofinide. The science is fairly old and it’s neuroprotective/neurodevelopmental function unproven, though I am glad you found benefit. That is encouraging.

      Also, before I continue, Fragile X and Rett, though they have some of the same developmental biology issues, should not even be remotely “clumped” together in assessment as to whether or not a drug has potential to help patients. As you have done. That is precisely the the type of irresponsible, evidence-devoid behavior that this company uses. As a patient stakeholder you should not be sucked in.

      Now, to get to the nub of your comment – which I appreciated by the way – I’d like to offer you two thoughts.

      1) You should be an incensed as I am – if not more – that this company can’t get its act together. If you think that Trofinitide is something special, then you should own a share or two of the company and be present at AGM meetings and ask why the company can’t design robust clinical trials, or use statistical methods that the FDA understands. I agree with you about the Breakthrough designation, incidentally, it’s not the end of the world at all. But what it raises red flags for is that the study design and outcomes did not offer statistical confidence and this is very problematic for the future of this drug. It is YOU who should be asking why, not me, because it will ultimately impact you.

      2) I have children and I couldn’t imagine the heartache of living with some of these conditions. I appreciate how few real options there are. However when companies do a very underwhelming job of taking capital from investors and conducting themselves in a way that doesn’t ultimately result in the best outcome for patients, all it does it make it harder to concentrate resources on those rare diseases, not easier. If Trofinitide is your salvation, then I can tell you that it is currently residing in a commercial vehicle that is unworthy of your hopes, dreams and expectations and it needs to pull its socks up.

      So, to summarise, I appreciate your emotion. I hope that when you reblogged your scathing reply, that you will also – in the interest of balanced dialog – also reblog my response. Ultimately, I write these posts because I want to see quality clinical development benefit patients. I can’t do this as a patient stakeholder, as you can, but I can certainly call on mediocre performance when I see it. And contrary to your assertions, I will not feel remorse when I am proven wrong – I will feel joy for the patients who will benefit.

      For something like this, I am delighted to be proven wrong. Meanwhile, I continue to do what I do – which is attempt to hold companies to a higher standard of performance.

      Thanks for engaging.

      Liked by 1 person

      • Whilst not suggesting incontrovertibly positive evidence from the Phase II, these eminent experts were prepared to be very positive about how promising the results were, and note the use of the term “proof of concept”, very much a Phase II goal and in contrast with your “expanded Phase I ” assertion:

        Walter Kaufmann MD, Professor of Neurology at Harvard Medical School and Director of the Rett
        Syndrome Program at the Boston Children’s Hospital, who was not involved in the trial, commented:
        “The outcome of this trial is very promising in terms of both safety and clinical improvement. It was a challenging study since the older age of the cohort and the short duration of the trial made it less likely to show a positive effect. It opens not only the possibility of successful treatment of adults with Rett syndrome, but also of early interventions modifying the course of the disease.”

        Alan Percy MD, Professor of Pediatric Neurology at the University of Alabama was one of the trial
        investigators. He commented: “The results of this trial suggest a very promising proof of concept as we continue on the pathway to develop a disease-altering treatment for girls and women with Rett
        syndrome. Not only was this short-term trial managed successfully, but also the data analyses were
        conducted in a very robust fashion.”

        Regarding your assertion: “But what it raises red flags for is that the study design and outcomes did not offer statistical confidence and this is very problematic for the future of this drug”:
        The FDA said “that data from Neuren’s exploratory Phase 2 trial provide insufficient evidence to demonstrate substantial improvement over existing therapies using conventional statistical methods.”

        FDA’s judgment that the data was not statistical confidence is specifically in relation to the Breakthrough Therapy application for which the trial was not in fact designed. This does not imply or suggest that the trial results were not statistically significant in relation to the trial’s specified endpoints – they were. Very rarely does a Phase II prove efficacy – that is the purpose of a Phase III. With respect, your above assertion is therefore fundamentally wrong and misleading. In no sense is the FDA’s response “very problematic for the future of the drug”.

        Positioning yourself as an immensely knowledgeable authority who exposes the sins of ASX biotechs (which I entirely agree are legion) for the benefit of the unsophisticated Aussie biotech investors (again a valid generalisation) but then making egregious such mistakes to support an (inexplicably) excessively dismissive and damning article, damages your credibility.

        By the way do you really think Treagus would have come to Neuren if he intended merely to preside over the “rewrapping of an old turd.”?

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      • Dear James,

        I trust all is well at Morningstar…

        I agree with much of what you have written – I have also read the press releases so although I do occasionally get things wrong (I am human) I think we have access to the same (very limited) information.

        Here is what I agree with:

        – That the safety profile doesn’t seem to be problematic. Anyhow, this was always a fundamental tenant of the technology and I take this reasonably at face value. Results haven’t, as far as I am aware, been published but I accept it. By the way, for a CNS drug, that is a huge point of faith.

        – That any drug in this patient population where the safety/AE profile is “good” and where there is some evidence of improvement (i.e. caregiver assessment) is exactly consistent with both of the academic opinions you have quoted.

        – I do agree that the PI has reason to be satisfied that the study yielded its primary outcome measure – which is safety / acceptable AE profile (please have a look at the clinicaltrials.gov entry).

        Things I don’t agree with / or don’t have enough information to assert as you have:

        – A Phase II study (indeed all stages of clinical development of a drug) has safety as a fundamental tenant. However safety usually isn’t the primary OUTCOME measure of a Phase II study, especially in the CNS setting when data has been established in healthy volunteers (and, additionally, in NNZ-2566’s instance, female-specific group in a relevant age range). So your statement about “very rarely does Phase II prove efficacy” is simply incorrect unless you are actually talking about failure rates (yes, plenty of drugs fail in Phase II for lack of efficacy). It may take several Phase II studies to ultimately establish the correct dose range/interval, patient population, etc. to feel confident about proceeding to Phase III but the VERY POINT of a Phase II study is to determine safety AND efficacy. In the completed Rett study, efficacy measures are a secondary end-point.

        – To be clear, the purpose of the study was : (and I quote) “to determine whether NNZ-2566 is safe and well tolerated in the treatment of Rett Syndrome in adolescent and adult females.” This is not an efficacy statement. In my opinion, and I have stated that I am not a CNS drug developer or a specialist in Rett Syndrome, all of the secondary endpoints/measures in the study are as important for determining tolerabilty/AE profile as they are about hinting at any efficacy. I have never stated or implied that this study was not VALUABLE. It is.

        – I don’t have the knowledge of whether or not breakthrough status was a prior goal of the Neuren management team or not. Given the timing of the launch of the FDA’s breakthrough program in 2012, it is quite possible that it wasn’t a goal at the outset, but then consultation with the FDA about the requirements for breakthrough status can happen any time and I am sure for a patient population like Rett that would be a fairly welcome discussion, notwithstanding the usual capacity limitations of the Agency.

        – I also stand by the assertion that a priori announcing application for breakthrough status was a huge tactical error for Neuren (as well as being essentially just crass hype) when the substance of the Rett study doesn’t actually fit well with the industry guidance on breakthrough designation (which is based on efficacy), notwithstanding that the overall guidance is less robust or established than Orphan or Fast Track. However, I am not an insider to the company and so I can only speculate on the decision-making process. Irrespective, however, the decision to announce a potential FDA outcome on the basis of an application, was the the incorrect one by any “standard” of behavior in the pharmaceutical industry.

        I suppose I have a few final comments. The whole idea of the FDA’s expedited designations is to give drugs that are already “fragile” (no pun intended) in their chance of commercial success, a boost. When those avenues are incorrectly exploited, it does harm. There was no need for Neuren to put itself into the position where shareholder and patient expectation of breakthrough status was set, and then have to retract it. I’m sorry, but it is problematic for the drug and it’s future. Of course, Neuren can get more data and go back to the FDA but it is my (reasonably informed) opinion that follow-on approaches will have to be cautious based on the way the current situation was handled.

        I don’t, in fact, position myself as an “immensely knowledgeable authority”. I am a generalist and I certainly lack depth in many areas. I also think I am fairly direct and front-facing about my limitations. What I am prepared to do is ask the questions, with a reasonably informed scientific and experiential toolbox, that you are clearly not prepared to ask – even if I (occasionally) find the responses uncomfortable. I don’t think my article is damning, I think it asks some reasonable questions and makes some reasonable analysis about a public company. Unfortunately, when a company is public, such discourse is part-and-parcel, it is the price to pay for taking money from the public purse.

        As for your last question, I don’t have an answer for that. Although I often try to look at companies through the lens of a CEO to understand culture and motivation for behavior, I don’t – in this instance – have an appropriate or fair answer to your question. There are many reasons for doing things in life and I am sure given the rational and, frankly, serious intelligence of Dr. Treagus, that his reasons are solid, whether personal, professional, financial or patient-centric.

        Unfortunately a turd is a turd until it is anaerobically digested and turned into something proven to be useful. This is, in fact, Dr. Treagus’ leadership challenge. It has yet to be demonstrated and not all behavior of the company (not Dr. Treagus as an individual, but the company as a whole) is indicative that this transformation is happening in a robust way.

        Thanks for your engagement.

        Like

  6. Reblogged this on Trail To A Texas Trial and commented:
    Well, this will make Rett syndrome parents’ blood boil-
    A blog post about the “failed” Phase II Rett study with NNZ-2566 by someone who cannot grasp the significance of the findings. It’s like someone saying the world is flat and all those round world map believers are wrong because they (the author) can’t comprehend the data.
    Here’s the response I wrote-
    Let me blunt, this diatribe of nonsense isn’t worth the time you took to write it. Speaking as a parent whose child went through the Phase II Rett trial, I can assure you, that it was in no way a failure. Breakthrough Status is given to only 30% of applications, so it is not surprising that it was not received on the first try, disappointing, though. While TROFINETIDE, (NNZ-2566) is not a cure at the cellular level, and it appears only you find this astonishing, it is a possible “virtual” cure by addressing multiple life threatening and life shortening symptoms, not unlike “Lorenzo’s Oil”.

    Neuren and Trofinitide represent the best and ONLY hope right now that children with Rett syndrome and Fragile X may have a chance, how dare you scoff at it. I can personally defend the improvements seen with Trofinitide. Also, Rett doctor after Rett doctor HAVE come out and publically spoken about their support of that trial, Neuren and Trofinitide. In addition, the US Army, which has funded Trofinitide and it’s use in TBI would not continue to fund it if they personally were not seeing positive results. What one sees with their eyes does not always translate well into statistics, but you should be more careful before you wish for the only hope Fragile X and Rett syndrome suffers have to die and fade away, especially when it works.

    And, in reference to Darwinism, I think that “survival of the fittest and natural selection” will come into play soon enough, with Neuren on top and you proven so wrong as to become obsolete and made to fade a way.

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  7. Pingback: No Delirium in Adherium | The Long Tail

  8. Pingback: My Fragile Excitement | The Long Tail

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