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Statement from FDA commissioner Beneficial for CTSO and also for those who were doubting spectral months ago;)
Statement from FDA Commissioner Scott Gottlieb, M.D., on new FDA efforts to support more efficient development of targeted therapies
For Immediate Release
December 15, 2017
Statement
In recent years, the medical community has experienced a shift in the way health care is practiced. Rather than focusing solely on how to treat an overall disease type, medical innovators are now exploring how to tailor treatments that target unique characteristics of an individual’s disease, such as the genetic profile of a person's tumor. Innovation in this modern, targeted approach to medicine has already led to new more targeted medicines and, in some cases, therapies that are tailored to individual patients.
The FDA has an important role to play in advancing this targeted approach to treating disease by building a modern framework that ensures we’re providing the guidance and resources needed to efficiently develop these novel products using new technology. In particular, the FDA needs to clarify and expand an existing pathway that allows innovators to develop products based on the molecular markers that the drug targets, rather than the more traditional approach to drug development, where new medicines were developed based on the disease phenotype that they targeted. In many cases, science is revealing that the driver of disease is really a molecular change in the body. New drugs are being developed based solely on their ability to target these underlying molecular subtypes. Moreover, this same molecular change may be present as the driving factor of many different disease phenotypes. When drugs successfully target these molecular mistakes to reverse the effects of different diseases, we need a development pathway that allows the new drug to pursue approval in each of these novel settings on the basis of the molecular marker that the drug targets. In the setting of oncology, this is often referred to as tissue agnostic drug development.
By providing clear guidance on the regulatory and scientific frameworks for product developers, safe and effective targeted treatments can be identified with scientifically valid tests and ultimately, made available to patients faster. That’s why today we are issuing two draft guidances that will provide medical product developers with greater clarity on the FDA’s recommendations for researching and developing the next generation of individualized therapies.
The first draft guidance, “Developing Targeted Therapies in Low-Frequency Molecular Subsets of a Disease,” addresses the important topic of finding treatments that address the underlying molecular changes (e.g., genetic mutations) that often cause or contribute to diseases, including uncommon molecular changes that are present in a small subset of patients. This draft guidance (which is also in a more concise, streamlined format) proposes an approach for drug developers to enroll patients based on the identification of rare mutations into clinical trials for targeted therapies when reasonable scientific evidence suggests the drug could be effective in patients with these genomic findings. The guidance discusses the evidence needed to demonstrate effectiveness for a variety of molecular subsets within a particular disease, which could lead to more consistent development and approval of targeted therapies for patients who are likely to benefit from them. These scientific principles described in the guidance could also be applied to supporting “tissue agnostic” drug development. This relates to how drugs may be able to gain regulatory approval on the basis of targeting a molecular subtype that is common across different phenotypes, rather than solely on the individual disease states. We think both approaches have the potential to increase the likelihood of finding viable treatment options for those with less common mutations. When finalized, this draft guidance will represent the FDA’s current thinking. The FDA also plans to publish a manuscript early next year that provides a detailed, comprehensive look at this issue.
The second draft guidance, “Investigational IVD Devices Used in Clinical Investigations of Therapeutic Products,” seeks to provide those running clinical trials with a clear framework to reference when determining if an in vitro diagnostic (IVD) device used in a therapeutic product study must undergo its own FDA review, distinct from the drug being studied. To develop new targeted therapies that are safe and effective, clinical trials often use investigational, or unapproved, IVDs to assess biomarkers and guide the selection of therapeutic products or care strategies that are applied to study participants.
When final, this draft guidance will clarify the appropriate regulatory pathway for investigational IVDs used in clinical trials for therapeutic products, which is significant so that trial results for a novel targeted therapy are not undermined just because the diagnostic test to determine a specific biomarker did not meet appropriate regulatory criteria. The aim is to make the process for developing more targeted “drug and diagnostics systems” more efficient and to simplify the proper development of these approaches.
Building on the IVD draft guidance issued today, we are also considering ways to streamline the review of oncology therapeutic products and the IVDs used with these products, and plan to issue draft guidance on this in the near future. The goal is to reduce the burden on sponsors for the development of certain cancer drugs, and on FDA staff as well.
These draft guidances are just a few examples of the FDA’s ongoing efforts to advance the development of innovative, targeted drugs and foster the availability of individualized treatment approaches. For example, earlier this month, the FDAissued draft guidance that describes a potential new approach for companies to collaborate and test multiple drug products in the same clinical trials for rare pediatric diseases, thereby reducing the number of patients treated with placebo. We also outlined how modeling and simulation can be used to reduce the reliance on placebo arms in these rare pediatric settings. Finally, we specifically addressed a more efficient pathway for developing drugs targeted to the rare pediatric disorder Gaucher Disease. We also recently authorized three, novel next generation sequencing-based devices for the detection of multiple cancer markers with the run of a single test – enabling the development of evidence to drive more individualized care management decisions.
By proposing streamlined approaches for our colleagues in the research and development communities, the FDA hopes to enable more efficient access to safe and effective, novel targeted therapies for the patients who need them. We look forward to receiving feedback on the draft guidances issued today and remain committed to assisting the medical community as it further modernizes and individualizes approaches to care, to increase the public health benefit offered by new medical technologies.
The FDA, an agency within the U.S. Department of Health and Human Services, protects the public health by assuring the safety, effectiveness, and security of human and veterinary drugs, vaccines and other biological products for human use, and medical devices. The agency also is responsible for the safety and security of our nation’s food supply, cosmetics, dietary supplements, products that give off electronic radiation, and for regulating tobacco products.
Read more at http://www.stockhouse.com/companies/bullboard?symbol=t.edt&postid=27184357#itedq8356qXAe8h7.99
Boris, for the last time, the pump is not required! It's simply a supplementary product to their polymyxin B endotoxin removal. It's just reinventing the current dialysis pump where PMX works already. More efficient and economical, that's it.
You are correct on that I'm not sure why I said venture as I own almost 200k shares on the tsx. My mistake though on that however, every single piece of information I have given is factual. You can argue about the 150-170k treatments but all the information I gave in regards to the trial and subgroups along with personalized medicine where the FDA is headed, all completely factual.
You're looking at old information, you guys are seriously hopeless, it's unbelievable. And about the 5%, see if you're actually capable of reading through the first post I made about the STATISTICAL AND CLINICAL EFFICACY achieved in those subgroups, it's far better than 5%, also look at the underpowered part of the post and how I said it shows personalized medicine is needed. You know the part where I mentioned the 21st century cures act? You're hopeless, I don't have a need to sell anything, I was simply informing people but you're all too ignorant to open your eyes so I won't waste my time.
They have enough cash to get them to FDA decision along with 4.5MM to spend on marketing until then. After FDA decision no matter the result there will likely be a buyout regardless so raising cash is not an issue for them. They have stated this multiple times.
2 of my posts have been removed for being "off topic and spam". Not once have I said anything incorrect or misleading about CTSO or Spectral. All information has been factual and it's about educating people on here about Cytosorbs DIRECT COMPETITION. That is completely ON TOPIC as it has a direct effect on the perceived success of cytosorb. Anybody who doesn't want to see this is just being a very novice investor and a child just seeing or listening to what they want to hear.
5% was not the result of the trial, it was an overall result as there was a too far gone group which skewed those results due to only being able to remove less than 50% of the endotoxin in the blood. With more treatments (exactly what CTSO does by varying the amount of treatments based on the condition you experience better results. THIS IS PERSONALIZED MEDECINE this is what the world is coming to. The fact is that CTSO is doing the exact thing Spectral is by varying dosing based on the patient. Imagine a trial where you could only use 2 treatments on patients which had a varying level of illness, some of which were much sicker than others and you gave them all the same treatment. There will be a skewed portion of those results as well effecting the overall results making it LOOK like a failed trial. the subgroup numbers achieved were statistically and clinically significant, that's what the FDA will look at. The Euphrates Trial was about using EAA to pinpoint where a treatment with a long standing safety record works and where it doesn't. The Taiwanese study will only confirm this as Euphas did.
Also don't forget SOC right now does not include EAA. In the trial EAA was included so nurses could see levels rising and respond more effectively along with the fact this treatment was done at some of the top hospitals in the US where the SOC is arguably better. This directly indicates a skewed sham group mortality rate. Change the sham mortality alone and you have a successful trial nevermind fixing the underdosing of the sicker patients.
I agree with you there but at the end of the day Spectral is doing the same via locking down distribution rights in other countries already so they are positioned well incase of a no from the FDA. The issue I have with cytokines as does the FDA is the fact that there are so many variations of them. Some of which are naturally occurring and some that should not. This creates a lot of complexity as to the proper dosing, effective removal of the proper cytokines and potentially adverse safety effects which go along with that. This will come to light during larger blinded studies.
The way I see it, Spectral is 5 years ahead of Cytosorb. One must ask why cytosorb has moved towards endotoxin removal if Spectral really wasn't on to anything? Worst case scenario which again I highly doubt as there is nothing in the pipeline sooner than 5 years is that the FDA outright says no and let's countless more die versus an approval or at least a conditional approval monitoring the effectiveness of PMX in different subgroups. If some other treatment was already available and hundreds of thousands weren't dying each year I would have to agree with you but the fact is, there isn't, many are dying and no other FDA approved treatment is coming anytime soon.
Even if Spectral does not achieve approval, which again, I highly doubt. Their EAA is extremely valuable along with their SAM pump and I suspect they still get bought out regardless so the EAA can be effectively sold and distributed by a larger company with an inside sales force. Make no mistake, there are large, well known companies interested in purchasing Spectral but this won't happen until we see the FDAs verdict. When this happens at worst I suspect a buyout in the range of the current share price plus or minus 25% and if an approval is granted we could see a $5-7 USD/share buyout overnight. With a conditional approval I suspect $2-3 USD. This is based on a newly analyzed target market of about 80% to what it was after the subgroup analysis. Everything depends on where the FDA approves its use so if it's greater than the 80% initially suspected then obviously it warrants a larger buyout. Vice versa with a reduced market.
With return potentials at 700-2400% from the current share price and downside risk protection with the Value of EAA along with the Sam pump, it's a good play.
Everyone can think what they like, but to say this is a dead stock, you really need to educate yourself and like some of you have said "know and understand your competition".
I'll post this here as well for all the misinformed CTSO investors on their board. They deleted the post, no reply no nothing? Hmm, I wonder why?
Oh where do I start here...
First off, I do like Cytosorbs technology and do believe it will be instrumental in the fight against sepsis and other disorders. You all are very misinformed as to what Spectral has on the table as are many investors hence their share price at the moment. This is 100% managements fault but I don't believe they are concerned as they are aware of what they have and that there are big name pharma companies looking at purchasing as they have 22 employees where as large pharma companies have large inside sales forces inside hospitals already to effectively market their product; the exact thing spectral needs. This is also the reason PMX hasn't been used in Canada, they have a spineless government which relies on US FDA approval as a baseline for its drugs and medical devices. Without US FDA approval the Canadian health care system being paid for by the government will not cover such a treatment.
To start, when you compare CTSO to spectral you're comparing a company with one very small Trial to another company who is 5 years ahead which has completed a very large, double blinded FDA PMA study. Spectral looked great at the stage CTSO is at currently and there was evidence of extreme efficacy with little to no known adverse safety effects.
This study (Euphrates) was underpowered from the start, it is believed that the study was largely done this way to see where EXACTLY PMX was effective because like any drug or device treatment there are cases where people are simply too far gone to respond to treatment. Why do I say this? Simple, look at the 21st century cures act that was brought in which mandates the FDA to look differently at devices or drugs in which no treatment exists or there is an extreme need for where we all know Sepsis falls in that category. It also mandates them to take a different approach when looking at data including looking at real world data in other studies as well as subgroup analysis which if you look into the details on Spectral's trial they achieved extreme efficacy in the subgroups where EAA was above 0.6 and below 0.9. Furthermore for the group which was above 0.9 the endotoxin levels were above 50 iu and the absorption capacity of each cartridge of PMX was 12.5 iu meaning that in the most sick patients we only removed less than 50% of their endotoxin which indicates the study was underpowered as spectral did achieve up to a 42% risk reduction at 28 days (21% mortality versus 42% mortality) in a subgroup where arterial pressure was maintained in both arms and where there was no culterable bacteria present (antibiotics not gonna help you there). One needs to ask, if the trial allowed for 3 or 4 columns to be used on these sicker patients, could we have had the same results as the subgroup?? This is exactly the type of data the FDA is going to start looking at. Precision medicine is the future and the 21st century cures act clearly outlines it's happening now.
In respect to the SAM pump, spectral does not need this for the PMX treatment, a dialysis pump works fine but they have refined the average dialysis pump making it more economical, more effective, less bulky and easier to use.
As far as Spectral's EAA diagnostic which is approved in Canada and the US, this is the holy grail and it will be used in many different cases other than sepsis. The market for this diagnostic alone is huge! Imagine being a doctor with an approved treatment available and you now have the data to show you when exactly it is effective and also the instrument/test to show you where exactly you are in that spectrum and if it's working.
Also to note is the fact that there is a Taiwanese study going on right now starting treatment sooner with EAA levels at 0.5 vs 0.6. This doesn't seem like much of a difference but when you look at it in detail, you are 3 times as likely to die with EAA levels of 0.6 than 0.5.
Someone on this board said that PMX cannot treat patients who actually have sepsis. I'm sorry but you are sadly mistaken. With the Euphrates trial look at what MODS 9 means. (Multiple organ disfunction score) multiple organs were required to be not operating as they should or failing to receive treatment, this includes the subgroup where there was success listed above. These organs were failing as a direct result of sepsis and these patients were going into septic shock (multiple organs failing, health drastically deteriorating).
Finally, the Euphrates Trial was done at some of the top hospitals in the US where the SOC (standard of care) is above average compared to your national average across the USA. This also impacted Spectral's absolute reduction and primary end point in the Euphrates Trial with a baseline mortality rate of 42%. They also were using the sham cartridge PLUS EAA which is not the current standard of care. One must ask, without EAA being used and in an average hospital would that baseline mortality be much higher than 42% as it has been in previous studies. Food for thought.
Toray and Birch hill own over 70 million shares and have not sold anything. Now approval isn't a certainty but with the EAA, Sam Pump, 21st century cures act, the fact there is no cure and the closest one is 5 years away, also the "smart money" not going anywhere. You have to ask yourself the following question:
If you were the FDA and you have hundreds of thousands dying each year from a disease which there is no cure or viable approved treatment for do you wait 5 years and let countless more die or do you approve or at the very least conditionally approve a treatment which is approved in many countries around the globe, has been used over 170,000 times effectively and also has minimal adverse safety effects through the countless number of trials conducted?
I'm very confident it will go the right direction but that's just my opinion.
Something seemed to have gotten lost... no rebuttal??
Oh where do I start here...
First off, I do like Cytosorbs technology and do believe it will be instrumental in the fight against sepsis and other disorders. You all are very misinformed as to what Spectral has on the table as are many investors hence their share price at the moment. This is 100% managements fault but I don't believe they are concerned as they are aware of what they have and that there are big name pharma companies looking at purchasing as they have 22 employees where as large pharma companies have large inside sales forces inside hospitals already to effectively market their product; the exact thing spectral needs. This is also the reason PMX hasn't been used in Canada, they have a spineless government which relies on US FDA approval as a baseline for its drugs and medical devices. Without US FDA approval the Canadian health care system being paid for by the government will not cover such a treatment.
To start, when you compare CTSO to spectral you're comparing a company with one very small Trial to another company who is 5 years ahead which has completed a very large, double blinded FDA PMA study. Spectral looked great at the stage CTSO is at currently and there was evidence of extreme efficacy with little to no known adverse safety effects.
This study was underpowered from the start, it is believed that the study was largely done this way to see where EXACTLY PMX was effective because like any drug or device treatment there are cases where people are simply too far gone to respond to treatment. Why do I say this? Simple, look at the 21st century cures act that was brought in which mandates the FDA to look differently at devices or drugs in which no treatment exists or there is an extreme need for where we all know Sepsis falls in that category. It also mandates them to take a different approach when looking at data including looking at real world data in other studies as well as subgroup analysis which if you look into the details on Spectral's trial they achieved extreme efficacy in the subgroups where EAA was above 0.6 and below 0.9. Furthermore for the group which was above 0.9 the endotoxin levels were above 50 iu and the absorption capacity of each cartridge of PMX was 12.5 iu meaning that in the most sick patients we only removed less than 50% of their endotoxin which indicates the study was underpowered as spectral did achieve up to a 42% risk reduction at 28 days (21% mortality versus 42% mortality) in a subgroup where arterial pressure was maintained in both arms and where there was no culterable bacteria present (antibiotics not gonna help you there). One needs to ask, if the trial allowed for 3 or 4 columns to be used on these sicker patients, could we have had the same results as the subgroup?? This is exactly the type of data the FDA is going to start looking at. Precision medicine is the future and the 21st century cures act clearly outlines it's happening now.
In respect to the SAM pump, spectral does not need this for the PMX treatment, a dialysis pump works fine but they have refined the average dialysis pump making it more economical, more effective, less bulky and easier to use.
As far as Spectral's EAA diagnostic which is approved in Canada and the US, this is the holy grail and it will be used in many different cases other than sepsis. The market for this diagnostic alone is huge! Imagine being a doctor with an approved treatment available and you now have the data to show you when exactly it is effective and also the instrument/test to show you where exactly you are in that spectrum and if it's working.
Also to note is the fact that there is a Taiwanese study going on right now starting treatment sooner with EAA levels at 0.5 vs 0.6. This doesn't seem like much of a difference but when you look at it in detail, you are 3 times as likely to die with EAA levels of 0.6 than 0.5.
Someone on this board said that PMX cannot treat patients who actually have sepsis. I'm sorry but you are sadly mistaken. With the Euphrates trial look at what MODS 9 means. (Multiple organ disfunction score) multiple organs were required to be not operating as they should or failing to receive treatment, this includes the subgroup where there was success listed above. These organs were failing as a direct result of sepsis and these patients were going into septic shock (multiple organs failing, health drastically deteriorating).
Finally, the Euphrates Trial was done at some of the top hospitals in the US where the SOC (standard of care) is above average compared to your national average across the USA. This also impacted Spectral's absolute reduction and primary end point in the Euphrates Trial with a baseline mortality rate of 42%. They also were using the sham cartridge PLUS EAA which is not the current standard of care. One must ask, without EAA being used and in an average hospital would that baseline mortality be much higher than 42% as it has been in previous studies. Food for thought.
Toray and Birch hill own over 70 million shares and have not sold anything. Now approval isn't a certainty but with the EAA, Sam Pump, 21st century cures act, the fact there is no cure and the closest one is 5 years away, also the "smart money" not going anywhere. You have to ask yourself the following question:
If you were the FDA and you have hundreds of thousands dying each year from a disease which there is no cure or viable approved treatment for do you wait 5 years and let countless more die or do you approve or at the very least conditionally approve a treatment which is approved in many countries around the globe, has been used over 170,000 times effectively and also has minimal adverse safety effects through the countless number of trials conducted?
I'm very confident it will go the right direction but that's just my opinion.