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I think that everything hangs on the US Phase III results. Everything. That's what I think the EMA is waiting for. I know of at least one case where the EMA approved a drug for an orphan indication where it almost became a problem for the EMA (icatibant for acute attacks of hereditary angioedema). they approved icatibant on the basis of a positive Phase III trial. Subsequently, the US Phase III for that drug failed. The company was then asked by the FDA to perform a third Phase III trial, which was positive. The drug was then approved by the FDA. It wouldn't have looked good for the EMA if they had approved a drug on the basis of a positive Phase III, only to have two subsequent Phase III trials contradict that evidence. That is why I think they are waiting.
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Clinuvel has applied for Orphan Status and was granted! Now I sencerly doubt if the EMEA is aware of what the substance of this status content!


In my believe you will be completly supported by the EMEA during your whole research. They should give you just extra support?


..and now at the last day of the race the EMEA, suddenly, out of nowhere, has some certain questions that has to be answered!


What interest do they have by waiting so long with these questions?

In my believe the EMEA could have informed Clinuvel in the early or mid stage of their research!


They are through the years that much experienced that they no by the start already which questions at the end can arise!


I'm starting to doubt their credibility and sincere intentions.





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Based on the review of the data, at the time of the withdrawal, the CHMP had given a negative opinion recommending that the marketing authorisation be refused for Raxone for the treatment of LeberÃÆâ€â„¢ÃƒÆ’ƒâہ¡ÃƒÆ’‚¢ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¡Ãƒâہ¡ÃƒÆ’‚¬ÃƒÆ’¢Ã¢Ã¢Ã¢Ã¢â€š¬Ã…¡Ãƒâ€šÃ‚¬ÃƒÆ’…¾Ãƒâہ¡ÃƒÆ’‚¢s hereditary optic neuropathy (LHON).


At the time of the negative opinion, the CHMP was concerned that in patients with LHON whose symptoms started in the previous five years, taking Raxone for six months did not lead to a significant improvement in vision compared with placebo (patients taking Raxone were able to distinguish three more letters on the letter chart compared with patients taking placebo). The CHMP did not consider this benefit to be significant.


Based on the same study, the company later proposed to restrict the use of Raxone to patients with LHON whose symptoms started in the previous year. These patients showed an improvement of 17 letters on the letter chart compared with placebo. However, the CHMP concluded that the new sub-group of patients proposed for treatment was not well represented in the study (28 patients) and the reliability of the results is questionable. Given the small size of this sub-group, the CHMP considered that a spontaneous improvement could not be ruled out.


In addition, the CHMP considered that the data supporting the mode of action for idebenone in LHON are not sufficient.


Therefore, at the time of the withdrawal, the CHMP was of the opinion that the benefits of Raxone did not outweigh its risks.



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Eculizumab for paroxysmal nocturnal hemoglobinuria (PNH)


first in class

approved for orphan disease treatment by FDA since 2007


Now a lot of publicity in Belgium about a small boy that needs the treatment but parents can't pay the 9000 euro per 2 weeks treatment any longer while the government refuses to pay. The government needs to apply austerity measures for reimbursements and asked the company (Soliris) to reduce the price with 3.69 pct) and the company refused.


It just shows that (small) companies can make a lot of money without having a blockbuster in volume sales as long as you find just a few patients that are really helped, a monopoly and a monopolist price. Their business model is about big net profits on small sales volumes and slow growth. (second orphan indication FDA approved in 2011) The company website is built on patient testimonies.

A true orphan drug strategy.


Compared to the Scenesse 32.000 usd annual treatment costs we would need a few extra patients to cover the gap but we are doing well on the patient support side for EPP. I would think it would be better if Clinuvel would find another orphan drug indication rather then pursuing the vitiligo story with very expensive phase III trial and very slim chances of approval. Also they will never to go alone on the trials or the drug delivery and production. With another orphan indication all of that is possible, the risk is less and the growth is more steady and realistic. EPP income could easily pay for a new phase II/III on another orphan drug indication or submission of a dossier etc.


They would keep integrity towards ema and chmp as an ethical company that delivers drugs to under served patient communities with real needs in dermatology in a sustainable business model.

This entire vitiligo story is very close to blockbuster, cosmetics and worldwide use and distribution of Scenesse. The fact that vitiligo patients are so enthusiastic in the US should not defer clinuvel from focus on the core strategy to develop Scenesse for orphan disease EPP and possibly other orphan indications in the future. If EPP can generate 100 million ausd per year then it is realistic to add slowly another 100 or so with every orphan indication. It would be highly profitable and less risky but of course not so good for the ego of the CEO and his football sponsorship plans.

For the regulators that cosmetic route is a clear nono for years already and for the company this is a area where they could not go alone. Not even for Vitiligo.


Last but not least the eculizumab story shows the difficult environment for clinuvel to negotiate reimbursements with payors.

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Nice little tease at the beginning of the year when the sp shot up. But talk about an illiquid investement. It's something when trading of 100 shares (less than $200) can drop the sp 4% in one day. There could be another 60+ days of dead silence before any news is released. Hate to guess where the sp will be then. To paraphrase a comment from Benjamin recently this merry-go-round is getting pretty old; would like to get off someday.


Do expect a US EPP PH III announcement prior to the end of June. It may in fact be one reason, along with a desire to meet with actual EPP patients and clinicians, for the request for more time.

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Nothing surprising.


In june 2012 3M raised @1.75 now 10 months closer to a result it will be interesting to see just how different this raising is. Not much is my guess.


In the last 10 months, this company has been in a holding pattern the first Vitiligo results have been released and CUV9900 has been shown to exist( by Uhoh) but management have done nothing else to support the sp.


I can't believe that there is nothing more that they can say. Tell us about the vitiligo program, the EU study, the OTR AK trial, make an announcement about CUV9900.


PW do something. How can the company be worth the same when vitiligo results were so positive and this is a potential multimillion dollar indication ?

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It was surprising that the last capital raising was priced at $.15 higher (if I recall correctly) than the previous closing price at the halt. I'm hoping for something similar. At the last round, shares had been on a long slide and we approval was a way off still. Now it could be in three months. And most investors can look at the last few months and see how quickly this investment could pay off.


5 million shares at $2.10 would fund us for a year!

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Frankly, if some sophisticated investor(s) is willing to pony up $10 million or so within 60 days of a decision we should be encouraged. No doubt they have access to more information than we do even if it's just to read PW's body language in a conference room discussion.
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