Mozzarc
Emerged
Rightio....Welcome to Mozz Posts from 2019...Yeah it dates back a bit but I will include some relevant ones here that still contain info about PAR. After all, some of the science and our discoveries are still relevant and will form a good background as to what PAR is about and what it has. You'll get a sense of potential of this company as you go through them. Its a labour of love for sure, it will take time to get them across but I'll drip feed them in and thus it will give you a chance to read and digest over time.
Generally I wont edit these, except for perhaps sometimes any references to any external forums for readability purposes.
Enjoy!
-----------------
PAR AND THE IND - Originally posted 25/01/2020
PAR friends and fellow shareholders, a very good morning to you on this long weekend (well in Aus anyway), today let's cover a topical subject....
PRE IND and we'll touch on the IND itself.
Note: I suspect there are a lot of new players and various members reading these posts, the numbers have swelled in just the last few weeks. If you are new to PAR a most warm welcome. You have a lot of catching up to do to learn about just what exactly we have and why is it just so compelling. DO enjoy the journey with some of us more 'veteran' players.
Paradigmers, in just three short weeks we approach the FDA for our first ever PRE IND. I have never personally been involved with a Bio Phrama at such an early stage, I've always come in way too late...I've dabbled in some options on large pharma companies but that was much further along the post FDA approval stages... nothing like this and never so early in the lifespan of such a great company with this much potential.
As we draw much closer to not one, but two separate IND meetings with the FDA, it's time to explore just what exactly IS a Pre IND meeting anyway?
DEFINITION
Investigational New Drug Application..Sure, ours isn't strictly a new drug, it's a repurposed drug but it still must go through this same procedure and pathway.
PURPOSE
Well how about we don't rely on me, let's go directly to the source itself, the FDA: "For pre-IND meetings, the purpose is to discuss CMC issues as they relate to the safety of an investigational new drug proposed for use in initial clinical studies ".(1)
CMC an acronym, simply stands for three aspects:
Hmm these three topics cover quite a bit, no wonder it takes so long to get all the docs together. I've heard for an IND meeting and a new drug (remember ours isn't new it's repurposed) it can be around 100,000 pages of docs! Not sure what ours would be but wouldn't be surprised if its still around half that?
In terms of the IND itself, this is the approval process that drugs such as our must go through. So much so that it is a violation of federal US law to even transport or handle unapproved drugs. This is one of the reasons why it took a while to get the ten American ex footballers going as its finally an unapproved drug at this early stage, special application under the expanded access program had to be made.
A very strong upcoming catalyst for us in terms of share price and word spreading of who we are and what we have once the results are out mid year...
WHAT ARE THE BENEFITS OF A MEETING BEFORE A MEETING/SUBMISSION (PRE IND)?
Well I liken it to a grocery shop. Huh? Well I make a list at home before I "meet" with the supermarket (do you like what I did there) ...I think of all my needs for the next week, I stocktake the pantry and write it on a list as a plan. In the very same way this is a process of planning a drug development program. We are essentially at high level running our high level plan by them and getting some feedback as far as if our high level plan makes sense.
Mozz pre planning the next grocery trip, likening this to what's happening in PAR's exciting life.
It's also a great opportunity to ask questions, yes there are pro forma docs to be filled but each company is different, each drug solution can have its own quirks and individuality's. The more efficient use of this valuable meeting and time with the appropriate resources (our time, the sponsor, as well as the FDA's time and resources) means less chance of any pesky 'clock stops' along the way. Other advantages include:2
Both parties will be in constant communication. In fact a PRE IND like what we are about to soon go through COULD be very advantageous to us indeed. Why? Because in the case of a "serious" or "life-threatening" disease there is a chance for us to achieve a higher priority type designation such as Fast Track or Priority or even Break Through. This may be discussed in the PE IND and if its not, it will be a consideration for the FDA between this meeting and the actual IND meeting later on, at least the FDA will have material enough to consider it amongst themselves.
WHAT'S ON OUR REQUIRED LIST OF THINGS TO DO?
So what do we bring into the IND Meeting? Well before this step, we have to submit a briefing package which generally contains summary info related to the product, a proposed clinical trial protocol and statistical analysis plan (3). The FDA will generally respond to this briefing during or after the review. The advantage is that this allows the FDA to ask further questions and help facilitate the Pre IND meeting so as to not waste time. Such a meeting is a two way street, questions and guidance from both the FDA as well as questions from the sponsor, PAR. Well here are some examples of what is taken into the meeting from our end.:
FOCUS OF THE MEETING
Addressing questions based on the pre submitted info package is one of the objectives of this meeting. Typically the agency will discuss relevant questions on safety and the various scientific and regulatory aspects of the drug. This is where Dr Felson will be invaluable. These questions and discussions are specific to the drug and sponsor in question.
Now here is an insight for you guys: This is what could be discussed at the Pre Ind (2) along with my thoughts:
1) Physical, chemical, and/or biological characteristics
Mozz view: Well we are in good stead here, PPS is well known, its been used for years, there has been no AE's in all that time. There is lots of info on this molecule.
2) Manufacturer
Mozz view: Again here we have an excellent track record with the one and only FDA approved manufacturer. 51Cap conducted an excellent coverage of their facilities, the lucky ducks got to go and check it out. State of the art facilities and pride in their work, sounds very German to me. Also very happy to work with us. German ingenuity since the 1940's...yes we are in good hands...and Paradigmers, think of the massive amount of time we are saving by ALREADY HAVING GOOD MANUFACTURING PROCESS through Bene already in motion and for the last how many decades?!
Note for new guys to PAR, if you haven't read 51Cap's articles this is required reading.
3) Source and method of preparation
Mozz view : Again see 2) above
4) Removal of toxic reagents
Mozz view: Huh? There are none that I know of, do you? Toxic? Not a chance. IPPS has a brilliant safety profile.
5) Quality controls (e.g., identity, assay, purity, impurities profile)
Mozz view - again this relates t the whole manufacturing process and adherence to the FDA rules not to mention random audits. See 2) above.
6) Formulation
Mozz view: Again refer to 2)
7) Sterility (e.g., sterilization process, release sterility and endotoxin testing, if applicable)
Mozz view: nah not applicable.
8) Linkage of pharmacological and/or toxicity batches to clinical trial batches
Mozz view: Kinda related to the manufacturing process again and we have mentioned toxicity...
9) Stability information
Mozz view: Very stable, inert compound.
WHO ATTENDS THIS PRE IND MEETING?
Well from our end its usually the CEO, CMO, CSO and the Babe Ruth/Donald Bradman of OA Analysis... Dr Felson. From the FDA side its can comprise of multidisciplinary personal such as representatives fromhttps://hotcrapper.com.au/styles/default/xenforo/clear.pngharmacologyPharmacokineticsChemistryMicrobiologyStatisticsand other such disciplines.2
SUMMARY
Ok I know some people get a lost in a sea of words, a graphic here might just clarify where we are and what's coming. I will post more about the next step later on perhaps next month and we can have an update on how we are tracking, here's a pic:
Typical flowchart. Where are we? Well I had to manually add in the red Pre IND step as not all companies go through this. In my view it does add some time to the process BUT it is invaluable as it helps with adequate and appropriate planning of that 'grocery list' and what we need to have done for the real IND meeting.
The key here is to come prepared, "These are not just “shoot the breeze” conversations with FDA and the outcome of the meeting is commensurate with the planning done prior to the meeting." (4) This is a major milestone in our company's life. If we get the go ahead for the next step it will be a great thing indeed. We as shareholders and then as a sub set of that, informed PAR investors know a LOT about our magnificent company and how the drug works. We have actual patients that contribute here and regularly give us detailed updates on their progress. We know the facts...we are on the inside. We know that we have a good chance of success. I believe we are well armed with a lot of unique talent from a company point of view with the likes of the great Dr Felson and the CMO as well as CSO. There is a lot of talent and experience here not to mention the board and CEO.
Very exciting next three weeks for us Paradigmers, let's see what transpires.
DISCLAIMERS
There may be forward looking statements here. There can be unforeseen circumstances that cause delays or hurdles....do you own....yeah, research. Don't rely on just me, I'm not a professional Financial Consultant or Advisor. We can only speculate on rough time milestones. This is going to be a fun trip, let me tell you.
REFERENCES
1) https://www.fda.gov/files/Guidance-...facturing--and-Controls-Information-(PDF).pdf
2) https://www.fda.gov/drugs/cder-smal...ed-questions-pre-investigational-new-drug-ind
3) https://www.fda.gov/files/Guidance-...facturing--and-Controls-Information-(PDF).pdf
4) https://facetlifesciences.com/fda-meetings-when-more-meetings-are-a-good-thing/
5) https://www.researchgate.net/figure...quire-an-IND-application-for-an_fig1_26671573
Generally I wont edit these, except for perhaps sometimes any references to any external forums for readability purposes.
Enjoy!
-----------------
PAR AND THE IND - Originally posted 25/01/2020
PAR friends and fellow shareholders, a very good morning to you on this long weekend (well in Aus anyway), today let's cover a topical subject....
PRE IND and we'll touch on the IND itself.
Note: I suspect there are a lot of new players and various members reading these posts, the numbers have swelled in just the last few weeks. If you are new to PAR a most warm welcome. You have a lot of catching up to do to learn about just what exactly we have and why is it just so compelling. DO enjoy the journey with some of us more 'veteran' players.
Paradigmers, in just three short weeks we approach the FDA for our first ever PRE IND. I have never personally been involved with a Bio Phrama at such an early stage, I've always come in way too late...I've dabbled in some options on large pharma companies but that was much further along the post FDA approval stages... nothing like this and never so early in the lifespan of such a great company with this much potential.
As we draw much closer to not one, but two separate IND meetings with the FDA, it's time to explore just what exactly IS a Pre IND meeting anyway?
DEFINITION
Investigational New Drug Application..Sure, ours isn't strictly a new drug, it's a repurposed drug but it still must go through this same procedure and pathway.
PURPOSE
Well how about we don't rely on me, let's go directly to the source itself, the FDA: "For pre-IND meetings, the purpose is to discuss CMC issues as they relate to the safety of an investigational new drug proposed for use in initial clinical studies ".(1)
CMC an acronym, simply stands for three aspects:
- Chemistry
- Manufacturing
- Controls
Hmm these three topics cover quite a bit, no wonder it takes so long to get all the docs together. I've heard for an IND meeting and a new drug (remember ours isn't new it's repurposed) it can be around 100,000 pages of docs! Not sure what ours would be but wouldn't be surprised if its still around half that?
In terms of the IND itself, this is the approval process that drugs such as our must go through. So much so that it is a violation of federal US law to even transport or handle unapproved drugs. This is one of the reasons why it took a while to get the ten American ex footballers going as its finally an unapproved drug at this early stage, special application under the expanded access program had to be made.
A very strong upcoming catalyst for us in terms of share price and word spreading of who we are and what we have once the results are out mid year...
WHAT ARE THE BENEFITS OF A MEETING BEFORE A MEETING/SUBMISSION (PRE IND)?
Well I liken it to a grocery shop. Huh? Well I make a list at home before I "meet" with the supermarket (do you like what I did there) ...I think of all my needs for the next week, I stocktake the pantry and write it on a list as a plan. In the very same way this is a process of planning a drug development program. We are essentially at high level running our high level plan by them and getting some feedback as far as if our high level plan makes sense.
Mozz pre planning the next grocery trip, likening this to what's happening in PAR's exciting life.
It's also a great opportunity to ask questions, yes there are pro forma docs to be filled but each company is different, each drug solution can have its own quirks and individuality's. The more efficient use of this valuable meeting and time with the appropriate resources (our time, the sponsor, as well as the FDA's time and resources) means less chance of any pesky 'clock stops' along the way. Other advantages include:2
- Identifying any unnecessary studies
- Clearly defining endpoints and the actual goals of the program
- Gaining FDA support for a proposed strategy.
- Increasing the probability of clinical trial success by getting the guidance at this earlier stage.
- The beginning of a relationship between the sponsor and the FDA, this is a vital step.
Both parties will be in constant communication. In fact a PRE IND like what we are about to soon go through COULD be very advantageous to us indeed. Why? Because in the case of a "serious" or "life-threatening" disease there is a chance for us to achieve a higher priority type designation such as Fast Track or Priority or even Break Through. This may be discussed in the PE IND and if its not, it will be a consideration for the FDA between this meeting and the actual IND meeting later on, at least the FDA will have material enough to consider it amongst themselves.
WHAT'S ON OUR REQUIRED LIST OF THINGS TO DO?
So what do we bring into the IND Meeting? Well before this step, we have to submit a briefing package which generally contains summary info related to the product, a proposed clinical trial protocol and statistical analysis plan (3). The FDA will generally respond to this briefing during or after the review. The advantage is that this allows the FDA to ask further questions and help facilitate the Pre IND meeting so as to not waste time. Such a meeting is a two way street, questions and guidance from both the FDA as well as questions from the sponsor, PAR. Well here are some examples of what is taken into the meeting from our end.:
- Brief summary of the CMC components.
- The development status of the drug in question.
- Summary of data (if available)
FOCUS OF THE MEETING
Addressing questions based on the pre submitted info package is one of the objectives of this meeting. Typically the agency will discuss relevant questions on safety and the various scientific and regulatory aspects of the drug. This is where Dr Felson will be invaluable. These questions and discussions are specific to the drug and sponsor in question.
Now here is an insight for you guys: This is what could be discussed at the Pre Ind (2) along with my thoughts:
1) Physical, chemical, and/or biological characteristics
Mozz view: Well we are in good stead here, PPS is well known, its been used for years, there has been no AE's in all that time. There is lots of info on this molecule.
2) Manufacturer
Mozz view: Again here we have an excellent track record with the one and only FDA approved manufacturer. 51Cap conducted an excellent coverage of their facilities, the lucky ducks got to go and check it out. State of the art facilities and pride in their work, sounds very German to me. Also very happy to work with us. German ingenuity since the 1940's...yes we are in good hands...and Paradigmers, think of the massive amount of time we are saving by ALREADY HAVING GOOD MANUFACTURING PROCESS through Bene already in motion and for the last how many decades?!
Note for new guys to PAR, if you haven't read 51Cap's articles this is required reading.
3) Source and method of preparation
Mozz view : Again see 2) above
4) Removal of toxic reagents
Mozz view: Huh? There are none that I know of, do you? Toxic? Not a chance. IPPS has a brilliant safety profile.
5) Quality controls (e.g., identity, assay, purity, impurities profile)
Mozz view - again this relates t the whole manufacturing process and adherence to the FDA rules not to mention random audits. See 2) above.
6) Formulation
Mozz view: Again refer to 2)
7) Sterility (e.g., sterilization process, release sterility and endotoxin testing, if applicable)
Mozz view: nah not applicable.
8) Linkage of pharmacological and/or toxicity batches to clinical trial batches
Mozz view: Kinda related to the manufacturing process again and we have mentioned toxicity...
9) Stability information
Mozz view: Very stable, inert compound.
WHO ATTENDS THIS PRE IND MEETING?
Well from our end its usually the CEO, CMO, CSO and the Babe Ruth/Donald Bradman of OA Analysis... Dr Felson. From the FDA side its can comprise of multidisciplinary personal such as representatives fromhttps://hotcrapper.com.au/styles/default/xenforo/clear.pngharmacologyPharmacokineticsChemistryMicrobiologyStatisticsand other such disciplines.2
SUMMARY
Ok I know some people get a lost in a sea of words, a graphic here might just clarify where we are and what's coming. I will post more about the next step later on perhaps next month and we can have an update on how we are tracking, here's a pic:
Typical flowchart. Where are we? Well I had to manually add in the red Pre IND step as not all companies go through this. In my view it does add some time to the process BUT it is invaluable as it helps with adequate and appropriate planning of that 'grocery list' and what we need to have done for the real IND meeting.
The key here is to come prepared, "These are not just “shoot the breeze” conversations with FDA and the outcome of the meeting is commensurate with the planning done prior to the meeting." (4) This is a major milestone in our company's life. If we get the go ahead for the next step it will be a great thing indeed. We as shareholders and then as a sub set of that, informed PAR investors know a LOT about our magnificent company and how the drug works. We have actual patients that contribute here and regularly give us detailed updates on their progress. We know the facts...we are on the inside. We know that we have a good chance of success. I believe we are well armed with a lot of unique talent from a company point of view with the likes of the great Dr Felson and the CMO as well as CSO. There is a lot of talent and experience here not to mention the board and CEO.
Very exciting next three weeks for us Paradigmers, let's see what transpires.
DISCLAIMERS
There may be forward looking statements here. There can be unforeseen circumstances that cause delays or hurdles....do you own....yeah, research. Don't rely on just me, I'm not a professional Financial Consultant or Advisor. We can only speculate on rough time milestones. This is going to be a fun trip, let me tell you.
REFERENCES
1) https://www.fda.gov/files/Guidance-...facturing--and-Controls-Information-(PDF).pdf
2) https://www.fda.gov/drugs/cder-smal...ed-questions-pre-investigational-new-drug-ind
3) https://www.fda.gov/files/Guidance-...facturing--and-Controls-Information-(PDF).pdf
4) https://facetlifesciences.com/fda-meetings-when-more-meetings-are-a-good-thing/
5) https://www.researchgate.net/figure...quire-an-IND-application-for-an_fig1_26671573