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TOPIC: Scleroderma - trial expanding to multiple sites?

Scleroderma - trial expanding to multiple sites? 10 May 2013 19:52 #449

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As per the latest quarterly press release, "In Europe, Cytori has been informed by an investigator in France that an ongoing pilot study in patients with hand complications from scleroderma is being considered for a multi-center trial based on encouraging preliminary results."

Very positive news that opens up Celution derived ADRCs for treating this autoimmune disease based on "encouraging preliminary results". MS, Parkinsons to follow? :yep:

What is truly amazing is that the trial only started enrolling in November 2012 as per the info on:

www.clinicaltrials.gov/ct2/show/NCT01813279?term=scleroderma+adipose&rank=1

To exhibit positive results in such a short period of time is very encouraging. :happy: The question is, if they expand the trial will it be sufficient to cover reimbursement or just the application for CE mark? So much going on ........ so little disclosed.

I am begining to think that apart from the Celution system CYTX has developed some sort of stealth technology that hides their progess from investors radar. The above sort of news would normally be more than enough to elicit a strong upward move in most biotechs....... certainly if a bit of info was provided. Still, the shorts appear to be reducing exposure and although I would love to see a short squeeze (unlikely), I am happy to see them assessing the changing risk reward environment and slowly going away. :bye:

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Scleroderma - trial expanding to multiple sites? 10 May 2013 23:37 #450

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Encouraging in a difficult disease...but with something 7 patients in the total trial and far less the PRECISE that failed to gain approval its wait and see it appears. Besides...how many really know about what was mentioned in the call that would be willing to move the PPS on the news today. First thing first...and solving the cash problem is #1.

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Scleroderma - trial expanding to multiple sites? 11 May 2013 09:33 #451

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Hedge, I agree that the financing issue is paramount in investors minds. However, the time it takes to conduct trials that produce tangible evidence of efficacy is also very important. Quick tangible results means less expensive trials need to be conducted.

The fact that the investigators are contemplating expanding the trial size and sites in such a short time frame from the initiation of the trial is strong evidence of the treatments efficacy. If the initial data is sufficiently strong to warrant the trial expansion, then the question becomes how big an increase and how quick an enrollment ? As there is no effective treatment, this trial can be run without a placebo arm. It is a very serious disease which warrants orphan designation. Patient advocates for this disease would no doubt be very interested in knowing more ....... as would we. If there are no other treatment options it stands to reason that enrolment size should be such to at least warrant CE approval. This disease kills and severely restricts quality of life, I do not see EU authorities procrastinating in reviewing this indication.

I never saw mention of a particular number of patients treated (7) to which you refer. Can you provide a link of where you found this number?

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Scleroderma - trial expanding to multiple sites? 11 May 2013 11:57 #452

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rongside
I re-read your original post and my response and I guess I was tired when I posted.
Please let me clarify a bit.
Again, very encouraging and I would love to see the trial expanded. What I was trying to say was that it was a very small trial...much smaller than PRECISE even ...so I would expect it would need to be bigger than that at least even for CE mark approval....your point on no control group is a interesting one...something like that would need to be discussed and agreed upon before starting as I am sure you know...but would limit the time and cost a bit. It is a terrible disease and I am very aware it kills.

Somehow I also confused your post with another discussion on valuation and what I was trying to say was the good news on this topic will unlikely add any valuation to CYTX at this point in time. You never went there in your original post and that was completely my mix up...sorry for the confusion.

As for the trial size of 7 patients....I believe it was mentioned somewhere...if I find it I will get back to you.

Orphan drug status ???...excellent idea...possible I would think.

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Scleroderma - trial expanding to multiple sites? 13 May 2013 07:27 #453

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I am begining to think that apart from the Celution system CYTX has developed some sort of stealth technology that hides their progess from investors radar. The above sort of news would normally be more than enough to elicit a strong upward move in most biotechs....... certainly if a bit of info was provided


Yes- I agree. This has been very frustrating to me. If you read all those announcements from MesoBlast and Osiris on their accomplishments, one recognizes that there is a lot of "overstatement" (or worse). Cytori hides its "good news" in minuscule lines in the quarterly reports. :cry: Anyway- our time will come.

maybe its a good thing to re-iterate the statement by Tom- that they are working very close together and also that in order to get the funding from the French- the scientists in France had to INDEPENDENTLY provide a lot of scientific evidence on the viability of the cells.

6.Assuming the initial French scleroderma trial on hands and skin proves to be successful, it will be expanded to more serious cases of scleroderma, possibly to cover reimbursement? Is there a framework for pan European trials sponsored by the european state health care authorities (eg european version of NIH) to fund and approve the Celution device for an indication such as scleroderma? If not, is CYTX able to use the data for its own use or in partnership discussions?

a. The current study to be clear is addressing serious forms of the disease. There are various paths to market should the data warrant it be advanced further, including government funds. We can’t speculate at this time to what extent we’ll have access to the data, but this is a site we are close with so we’re optimistic. Let’s see how the data looks and all parties including Cytori will learn more at that time about the next steps.


No surprise therefore that the next Cellsociety meeting gets an European version also in Marseille, the city of the present trial- I think I will visit that one-

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Board moderator and Site-owner. I still regret the day I started analysing the prospects of MacroPore (now Cytori) back in 2004- a left-over from the tech-bubble at that time from the century change in my portfolio- and became addicted to Cytori´s fat cell technology. :cry:

Scleroderma - trial expanding to multiple sites? 22 Jul 2013 14:50 #681

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Now that we have the exclusive license on Thomas Ichim´s patent on auto immune, I thought it was time to publish my article on Scleroderma, which was really something I had on the cell-treatment.net site for ages (before I dumped everything and started new)

I do expect some deals soon- one of them could be to move this application to the US - with ORPHAN status of course. :grin:

see front page for the article.
The following user(s) said Thank You: joeycav11, rongside, d9dozrman

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Board moderator and Site-owner. I still regret the day I started analysing the prospects of MacroPore (now Cytori) back in 2004- a left-over from the tech-bubble at that time from the century change in my portfolio- and became addicted to Cytori´s fat cell technology. :cry:

Scleroderma - trial expanding to multiple sites? 23 Jul 2013 05:22 #682

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Fas, loved the article on Scleroderma.

This is a favorite indication of mine as I believe that patients experience relief very quickly. I believe that it is for this reason that the French trial was/is being expanded so quickly after initiation. As you mentioned, the fact that they had done additional independent characterization trials of the Celution output is also very important in giving researchers and regulators confidence in moving forward quickly. :nice:

I am looking forward to learning the new trial size. A large size trial will provide potential validation of the significance the regulatory authorities ascribe to ADRC - SVF treatment.

CYTX's exclusive autoimmune licensing from Medistem is enlightened / huge, and has no doubt been in the works for a very long time.
The fact that we had the patents in ADRC processing is what made it possible. We were the only game in town. Management deserves some credit for this. :joy:

Orphan drug status .......... ????? ............ is it a drug ???????. Whatever, it is amazing to see the valuation of some drug companies treating small patient populations while having Orphan drug exclusivity. With the whole autoimmune space opened to us, and the huge range of diseases potentially being treated by ADRC ............ skies the limit. :vegas:

Auto-Immune Diseases

Diabetes Type I (Juvenile)
Systemic Lupus
Sjogren's Syndrome
Myasthenia
Autoimmune Cytopenia
Scleromyxedema
Scleroderma
Crohn's Disease
Behcet's Disease
Rheumatoid Arthritis
Juvenile Arthritis
Multiple Sclerosis
Polychondritis
Systemic Vasculitis
Alopecia Universalis
Buerger's Disease

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Scleroderma - trial expanding to multiple sites? 23 Jul 2013 12:49 #683

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Yes- auto-immune is pretty gigantic- I believe I read at Wikipedia a while ago that there are over 60 disorders classified in the "group", mind you- in some cases the medical profession is not sure of themselves.

Orphan status- is provided to the disorder I believe. From that perspective I would think it is irrelevant whether you "combat" the disorder with a drug or device therapy. If I recall right-the benefits to a developer lies in expedited review by the FDA etc and monopoly status for a certain period of time in view of the "limited size market" i.e. no competitive drug or device therapy can get approval for a certain period of time.

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Board moderator and Site-owner. I still regret the day I started analysing the prospects of MacroPore (now Cytori) back in 2004- a left-over from the tech-bubble at that time from the century change in my portfolio- and became addicted to Cytori´s fat cell technology. :cry:

Scleroderma - trial expanding to multiple sites? 23 Jul 2013 13:05 #684

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For those interested in FDA orphan status.

www.fda.gov/RegulatoryInformation/Legislation/FederalFoodDrugandCosmeticActFDCAct/SignificantAmendmentstotheFDCAct/OrphanDrugAct/default.htm

Scroll to orphan products board for devices mention

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