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TOPIC: FDA Orphan Drug Status

FDA Orphan Drug Status 17 Sep 2018 08:35 #12253

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CYTX has received FDA orphan drug status for its ATI-1123 chemotherapy drug product for the treatment of small cell lung cancer - see news release.

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FDA Orphan Drug Status 17 Sep 2018 09:59 #12254

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rodney.strongg wrote: CYTX has received FDA orphan drug status for its ATA-1123 chemotherapy drug product for the treatment of small cell lung cancer - see news release.


So- now the job is to find a development partner to be able to start that Phase 2. Glass half empty says- no way they are capable to do so- Glass half full would say- a potential partner could have waited for this designation. Time will tell.

The full announcement:

Sep 17, 2018
SAN DIEGO, Sept. 17, 2018 (GLOBE NEWSWIRE) -- Cytori Therapeutics (NASDAQ: CYTX) (“Cytori” or the “Company”) today announced that it received FDA orphan drug designation for its ATI-1123 chemotherapy drug product candidate, an albumin-stabilized pegylated liposomal docetaxel, for the treatment of small cell lung cancer.

The FDA’s Orphan Drug Designation Program provides orphan status to drugs, such as ATI-1123, which are intended for the safe and effective treatment of rare diseases/disorders that affect fewer than 200,000 people in the U.S. Sponsors of a drug receiving orphan designation qualify for various incentives including tax credits for clinical testing, 7 years of marketing exclusivity following FDA approval, and a waiver of prescription drug user fees.

Small cell lung cancer (SCLC), which accounts for approximately 15% of bronchogenic carcinomas with 33,375 new cases and 23,380 deaths estimated for 2017, has been identified by Cytori as a compelling target for ATI-1123. While SCLC is responsive to chemotherapy and radiation therapy, cure occurs only in ~20% of patients, generally restricted to those with limited stage disease. The remaining 80% of patients, including all patients with extensive disease, relapse within months of completing their initial therapy. Availability of 2nd line therapy options are limited, toxic, and provide little benefit in terms of extending survival. Topotecan is the only FDA-approved agent for 2nd line treatment of SCLC and is associated with an overall response rate of 24%, median response duration or time to progression of 14 weeks, and median overall survival of 25 weeks. Treatment usually involves a consecutive 5 day regimen of either IV or oral administration of drug, both which have black box warnings for severe myelosuppression as their use is associated with substantial morbidity including bone marrow suppression leading to neutropenia, thrombocytopenia and anemia requiring interventions of transfusion and growth factor support.

No major treatment advances for SCLC have occurred over the past 30 years. Hence, there remains a significant unmet need for novel agents with better safety profiles both for patients who cannot tolerate the adverse effects of 1st line chemo-radiotherapy and for relapsed/refractory patients who receive Topotecan. Cytori’s ATI-1123 has been designed to fill this need. ATI-1123’s combination of improved liposome stability, reduced toxicity, and superior delivery are expected to provide a therapeutic for SCLC that offers comparable or better efficacy to currently-available standards while having a less intensive administration routine and improved side effect profile.

Cytori is also exploring the development of ATI-1123 to address the shortcomings of docetaxel, a workhorse chemotherapy drug which generated $2.7B in worldwide sales at its peak. Compared to docetaxel, ATI-1123 may have potential to improve safety by removing the need for unwanted solvents, reduce morbidity by eliminating the requirement for standard pretreatment medications, provide better patient convenience and comfort via less time spent in the treatment center, decrease the cost of therapy, and enhance systemic docetaxel exposure.

A U.S. Phase 1 clinical study of ATI-1123 has been completed and published. Of the 29 patients in the study with cervical, gastric, melanoma, non-small cell lung, ovarian, pancreatic, prostate, thyroid, urachal and uterine cancers, 82% demonstrated a clinical benefit with ATI-1123. ATI-1123 exhibited an improved safety profile versus the Taxotere label with a 31% reduction in neutropenia and anemia. Further, ATI-1123 showed a 20% increase in maximum tolerated dose versus standard docetaxel and signs of efficacy with 1 partial responder.

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FDA Orphan Drug Status 18 Sep 2018 02:02 #12255

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Oh I think you left a lot out on the glass half empty side Fas but todays news certainly is a check in the positive column if just from the standpoint management got something of a positive nature done.

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FDA Orphan Drug Status 18 Sep 2018 11:05 #12256

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myownhedgefund wrote: Oh I think you left a lot out on the glass half empty side Fas but todays news certainly is a check in the positive column if just from the standpoint management got something of a positive nature done.

/

Whether the glass is half full or half empty on this matter is just speculation - what is real is that most of our original investment on this stock is more than 95% empty,:bash: :puke: :puke: :puke: :puke: :puke:

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FDA Orphan Drug Status 18 Sep 2018 15:36 #12257

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Very true RS.
The cost basis of the few shares left is quite high for me. Mentally written off last year...LOL
Still back to the news and some possible deal.
This product is quite early so up front money would be small....$5 to $10 million area with $5 to $7 most likely....now 75% of that has to go to Oxford (up to $7 million) Cytori has usually also placed shares with deals they make...so we get the dilution and Oxford gets the cash. Yep, I smell another winner management move coming...LOLOLOL !!! Note, they will have their usual bargaining position of ZERO leverage.
Somehow, even the laughter doesn't hide the pain anymore :(...
:cry:

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FDA Orphan Drug Status 19 Sep 2018 04:05 #12258

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myownhedgefund wrote: Very true RS.
The cost basis of the few shares left is quite high for me. Mentally written off last year...LOL
Still back to the news and some possible deal.
This product is quite early so up front money would be small....$5 to $10 million area with $5 to $7 most likely....now 75% of that has to go to Oxford (up to $7 million) Cytori has usually also placed shares with deals they make...so we get the dilution and Oxford gets the cash. Yep, I smell another winner management move coming...LOLOLOL !!! Note, they will have their usual bargaining position of ZERO leverage.
Somehow, even the laughter doesn't hide the pain anymore :(...
:cry:


Hedge- I find that statement on upfront cash for ATI-1123 very optimistic, I personally do not expect something there. I do see some potential for escaping the rope around the Cytori-neck with European partners for either ATI-0918 and/or scleroderma, since that is not development but commercial. A longer shot might be the Chinese opportunity for ATI-0918- my guess is still that Franshei might have his arms around that one. An even longer one- but also by far the major reason why I still support Cytori would be a development partner for CCT on nano basis. As we heard recently from Neil Riordan- the IP around cell technology is not very rigid. A patented delivery method, (which is difficult to copy) with the basic science already performed (it does not make a difference whether cells or vesicles are used in the micro-infrastructure of tissues) might become a highly attractive opportunity for BP which have done their homework- like Astellas or Saichi.
Anyway- we will soon find out.

To give new- and old readers some idea of the disaster that Cytori has been-

In the early days, say from 2000 to 2005, folks who invested in MacroPore had on average an average cost base of 4$ per share. Actually history repeated itself back in 2009-2010, when Calhoun/Hedrick started with their "smelling the inflection point" humbug and promised partner deals are on their way. Large groups of investors, including DOV´s, had a similar cost base of 4$ in those days.

That 4$ is post-split now 600$ of course and with a current 44 cents PPS, owners of those shares presently have less than one-tenth of a percent left of their money. To be exact- 0,0073%. This is a lot worse than the doomsday apostels like Cytxer and others have predicted. They were obviously quite right, but the game is not over yet - I do recall Hedrick had ownership (besides options) of about 650.000 shares. He lost that money too, so I expect him to put up a decent fight.

The present share price must be an attractive game for gamblers too of course, yes but not for investors. I guess in essence, I am a gambler now too. :grin:

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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:

FDA Orphan Drug Status 19 Sep 2018 08:40 #12259

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***
***Hedge- I find that statement on upfront cash for ATI-1123***

Probably right Fas. I knew it was very early stage but forget what work Azaya had completed on it.
Also considered that didn't they get $5 million for the hair growth application ? Which I didn't remember Cytori doing much work on either.
Doesn't matter imho....What ever the deal, my main point is it wont solve the cash issue alone.

I also wonder if it doesn't make the most sense to partner both 1123 and 0918 together.

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FDA Orphan Drug Status 19 Sep 2018 10:42 #12260

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fas wrote:

myownhedgefund wrote: Very true RS.
The cost basis of the few shares left is quite high for me. Mentally written off last year...LOL
Still back to the news and some possible deal.
This product is quite early so up front money would be small....$5 to $10 million area with $5 to $7 most likely....now 75% of that has to go to Oxford (up to $7 million) Cytori has usually also placed shares with deals they make...so we get the dilution and Oxford gets the cash. Yep, I smell another winner management move coming...LOLOLOL !!! Note, they will have their usual bargaining position of ZERO leverage.
Somehow, even the laughter doesn't hide the pain anymore :(...
:cry:


Hedge- I find that statement on upfront cash for ATI-1123 very optimistic, I personally do not expect something there. I do see some potential for escaping the rope around the Cytori-neck with European partners for either ATI-0918 and/or scleroderma, since that is not development but commercial. A longer shot might be the Chinese opportunity for ATI-0918- my guess is still that Franshei might have his arms around that one. An even longer one- but also by far the major reason why I still support Cytori would be a development partner for CCT on nano basis. As we heard recently from Neil Riordan- the IP around cell technology is not very rigid. A patented delivery method, (which is difficult to copy) with the basic science already performed (it does not make a difference whether cells or vesicles are used in the micro-infrastructure of tissues) might become a highly attractive opportunity for BP which have done their homework- like Astellas or Saichi.
Anyway- we will soon find out.

To give new- and old readers some idea of the disaster that Cytori has been-

In the early days, say from 2000 to 2005, folks who invested in MacroPore had on average an average cost base of 4$ per share. Actually history repeated itself back in 2009-2010, when Calhoun/Hedrick started with their "smelling the inflection point" humbug and promised partner deals are on their way. Large groups of investors, including DOV´s, had a similar cost base of 4$ in those days.

That 4$ is post-split now 600$ of course and with a current 44 cents PPS, owners of those shares presently have less than one-tenth of a percent left of their money. To be exact- 0,0073%. This is a lot worse than the doomsday apostels like Cytxer and others have predicted. They were obviously quite right, but the game is not over yet - I do recall Hedrick had ownership (besides options) of about 650.000 shares. He lost that money too, so I expect him to put up a decent fight.

The present share price must be an attractive game for gamblers too of course, yes but not for investors. I guess in essence, I am a gambler now too. :grin:


Fas, an investment now in CYTX makes a lot more sense than -99% ago (back then, we were all foolish to put it mildly) - however, anything is possible from this price level and triple digit gains would not surprise me - unfortunately, those gains would hardly make a dent in the original investment(s).

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FDA Orphan Drug Status 19 Sep 2018 10:46 #12261

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myownhedgefund wrote: ***
***Hedge- I find that statement on upfront cash for ATI-1123***

Probably right Fas. I knew it was very early stage but forget what work Azaya had completed on it.
Also considered that didn't they get $5 million for the hair growth application ? Which I didn't remember Cytori doing much work on either.
Doesn't matter imho....What ever the deal, my main point is it wont solve the cash issue alone.

I also wonder if it doesn't make the most sense to partner both 1123 and 0918 together.


What they did was described in detail in the announcement. Its phase II ready (which is normally also quite some work) and had a decent Phase 1 of 29 patients. I do recall Cytori received 5 Mio for the exclusive rights from KeraStem for the fat-cleaning app. I however never heard that a Phase 3 was going to follow the Phase 2 on alopecia, which they did,and looked pretty decent too. So- royalties are only an issue for the private pay stuff abroad.

Yeah- on cash- you either believe they can raise some or you dont. Everybody - inclusive the shorts- believe they cannot -see the market cap. But basically, we are talking here of products -close to market- with hundreds of Mios annual revenue potential. A goldmine if one guesses right.

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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:
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