Member Info for ddubya

Agree with the positivity on Ezutromid thus far. The unfortunate truth is that Sarepta is raking in $millions from US healthcare providers for a product that might be of limited or no benefit to DMD boys. You really could not make it up but I guess without Sarepta money, SUMM would have been in financial difficulty already and utrophin regulation may never have reached P2. Eteplirsen and exon skipping variations will need to demonstrate efficacy at some point, otherwise the FDA will face a crisis of confidence and healthcare providers may refuse to meet the costs, so SUMM could yet gain gain an upper hand in the US market. In that scenario, my view is that Sarepta would be forced to bid for the DMD side of SUMM, hopefully using valuations currently set by Sarepta itself.

I understand the journalist drew a blank eventually and the FDA did not release more documents into the public domain. I cannot see the Europeans doing Sarepta a similar favour either. Personally, I have not seen reports of Eteplirsen failing to deliver benefit to patients and did see one that suggested the boy was able to walk further, so perhaps the exon skipping method does have some benefits? Then again I have not seen anything convincing from Sarepta on efficacy.

A couple of links from the Labiotech site. Not news but might be of interest. The first is a simple summary of SUMM's utrophin regulation approach and recent results. https://labiotech.eu/duchenne-muscular-dystrophy-summit/ The second snippets re emerging treatments and repeating doubt regarding efficacy of Exondys 51. https://labiotech.eu/genethon-sarepta-muscular-dystrophy/

SUMM has not fully funded Ridinilazole so certainly there is more fundraising to be done. However, for added context, if SUMM received exceptional 48 week data for ezutromid and raised hopes of accelerated commercialisation, $40 would relate to only a few months potential income on conservative measures. In this best case scenario, it is not inconceivable that SUMM could fund the cost of ridinazole P3 itself, particularly if an early green light was forthcoming from the FDA. The next few months will be critical for holders.

Thanks Sheephatch - just checked Nasdaq myself to confirm the uptick and see the index itself is up only just over 1%, so the US market is favouring Summit today for whatever reason. Hopefully, it will translate to a similar uplift in the UK next week!.

And they have gone out of their way to mention cadmium free QD specifically. The description fits at least and another great spot Eskers.

When 24 week results were issued, GE made mention that SUMM would keep us posted in terms of updated analysis and this RNS seems to be the first of that series. Welcome news that adds to a stream of positive updates for SUMM. A decrease in muscle inflammation might be an indicator that ezutromid is modifying the effects of DMD, and could lead to a crucial improvement in walk test results for DMD boys. I expect we shall hear nothing too committal on that point until the 48 weeks results but this latest analysis of 24 week data provides a little more confidence that ezutromid specifically is reducing muscle damage and improve muscle health. It also suggests that conduct of trials has been well planned and executed. All good IMO.

As a very LT holder myself SL, I feel your pain but if you have averaged down you may now be at or near break even or at least closer to it? News could come at any moment but don’t hold your breath. If news is as good (as many expect) it will all have been worthwhile. If not, we may feel hard done but what can we do nothing more than move on and stay positive.

As a shareholder it is reasonable to seek facts and knowledge and each has been in short supply with NANO. It is also true that Nanosys is open and Jason Hartlove is pretty slick with PR but perhaps it needs to talk itself up give that it has much to lose if NANO succeeds? I placed a lot of store on the body language in the CES video presentations. Keith Wiggins was overtly pleased and Jason Hartlove either plain exhausted or a little hacked off with life. I also know that NANO is exporting/selling now, although not to whom or how much. I still have faith in NANO and a strong sense it will come good soon but I stopped trying to prove it to myself some time ago. Absence of proof does not mean my investment decision is wrong (or right) but sometimes judgements must be formed on limited information, if you want to stay in the game.. However, I really appreciate the efforts of other posters who find quality information through perseverance and detailed research. For me, the circumstantial evidence is growing and compelling.

I spotted that article too but I recollect it dtermines value by reference to published earnings and why not - by that yardstick it is. If you are believer in the NANO story what surely matters are earnings we hope are In the pipeline but as yet not currently visible in public information.

There continue to be so many unknowns with NANO that speculation is understandable. We would prefer more certainty (and deserve it) and whilst we appear to be selling and exporting, I would like to see disclosure of values, if not customers, at the first practical opportunity. On the other hand, I suspect ME has faced and managed many more challenges than we know of and so I am inclined to think he is worthy of the benefit of any doubt for a few more months yet, by which time I hope we shall have been overtaken by more positive events.

FootieladBaz, EP and myself and perhaps others seem to have been sharing dreams of eventual �20 + share price. Delusion perhaps at least we are not alone! I have my shares (and those of my wife) in ISA and Pension just in case it does take off. There is still much more science to be undertaken and the nature of the beast is that efficacy is not established until that work is completed and analysed - but I am still pleased to be invested in SUMM over Sarepta. In fact, I have been contemplating whether a short position on Sarepta might be a shrewd move?

Or even Ezutromide ! Sorry for the slip up but at least I have reminded myself we have another potential blockbuster.

Thanks for that gmcc. In event of good 48 week results, Ridinilazole might well be best in class, provided that the FDA applies impartiality.

Thanks gmcc so not an Eteplirsen (E51) trial. Do you have a link to your source that you could post? Hopefully the patient will recover soon - sounds as thoroughly unpleasant as the illness itself.

According to Schaeffer Research, Sarepta has curiously paused a UK trial of DMD candidate Eteplirsen. There could be any number of reasons unconnected to the drug itself but it seems to be denting their share price. Not sure why SUMM has dropped even more with only good news behind it? https://www.schaeffersresearch.com/content/news/2018/02/09/3-volatile-drug-stocks

Good to see more coverage for SUMM from TMF all of which can surely only be positive.

You tickled me there AppleMacMan and I believe there to be more than a grain of truth to the Ratings theory. I am so hopeful the David v Goliath title fight might be renamed Real deal v Super placebo later this year.

AppleMacMan - I also suspect the FDA response to positive 48 week data will be pivotal to timescales. Approval of Sarepta�s drug was made despite their failure to meet FDA advisory committee criteria on multiple counts. However, I have a feeling the FDA will be tougher next time to avoid further criticism. I am no expert in either pharma or pharma compliance but if improvement in the walk test data is in evidence at 48 weeks and reduction in muscle damage /safety is maintained, it would surely be difficult for the FDA to insist on a full roll out. There might be other things they could do, such as offering restricted approval for use in boys currently using E51 but not responding to it. I suspect the role of the DMD community will be important too, so some PR there might benefit to all. On that point, I found it interesting that in the Jeremy Vine Radio 2 piece yesterday, the charity representative, herself a mother of a DMD boy, identified exciting new DMD treatment in the gene therapy space and alluded to gene skipping but did not seem to be aware of Utrophin regulation?

I see what you mean EP, and hopefully a Christmas party at your castle this year!