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Can you elaborate on why you think the merger was poorly planned and at a ridiculous capitalization? Also, everyone likes to point to "relentless" short selling but the short interest has never been very high here. The hold obviously threw a wrench into some things but was beyond their control. I know everyone complains about their lack of communications but the reality is they have released all necessary information. Like I've said before what people should be annoyed with, if anything, is the fact that they haven't had many newsworthy events. Obviously this is changing. The reality of all of this though is that if SP were 5 to 10 times higher literally no one would be complaining about their "lack of communication."
Let's be real. Retail investors never matter but that's not why the SP has tanked.
Very insulted that you don't think I have the clout to influence share price, lol.
Always a chance but it is HIGHLY unlikely. 30 days after SP closes under $1 the company will be given a notice saying hey are not compliant with minimum listing rules and have 180 to get the SP back over $1. If at the end of that 180 days SP is not over $1 then the company can request an extension for another 180 days if they can provide a plan on how they will regain listing requirements. So from the point the SP drops below $1 they potentially have 390 days to get SP over $1 for 10 days which would regain compliance with the minimum listing requirements. If SP isn't over $1 a year from now we have a lot worse problems then delisting.
Besides Seeking Alpha being 90% garbage I just can't get over the fact that this particular author is stuck on the AML PII trial design and from what I can tell does not fully understand it. He states there are three group, active, adjuvant, and SOC and goes on to say that each intervention should have a control arm. How the hell does the SOC have a control arm. It is the control arm. This aside, there are not three groups as we all know. There are two. Active and adjuvant. The adjuvant group has two arms, those treated with MT401 and then then the control, or SOC, arm. SOC is just observation which is fine here because these patients are already in complete response. Anyone in the SOC arm that relapses can be moved to the active group. Now to the active group. I stated this when his last "article" was posted and the author suggested a control arm for the active group. What does he want them to do? Hi patients who have relapsed and are actively dying. We are going to go ahead and not treat you so we can see how much faster you die than the patients we are going treat. Sound good? The company is not going to let patients who have active disease, failed multiple prior therapies, and are knocking on death's door to go untreated and it's ridiculous to think otherwise. That being said, there is technically a control for the active group that this author has failed to mention both times. They are using a synthetic arm of 200 patients. So rather than enrolling an active group control arm they will be looking at historical data from patients that have gone untreated in the past.
This author is pointing to faults that quite frankly do not exist.
There is no IP issue. This keeps getting brought up by one person who I have on ignore so I don't see their posts but am assuming they brought it up again since you are asking this question. I think we are approaching a year now of that question being asked multiple times with said individual refusing to do any DD or elaborating on the issue even though links to the licensing agreements and patents have been posted.
Mana Therapeutics will have a big problem on their hand if they ever try to market anything.
Catherine Bollard used to work at BCM. She helped Ann Leen and Juan Vera although she did not have an integral part in developing the MultiTAA therapy. When she left for Texas Children's Hospital? she wanted to continue the work she was helping with at BCM and Dr. Leen allowed her to do so in a clinical capacity. She chose to effectively steal the idea and start Mana. They are not the only company to have infringed on Marker's IP but Marker is aware of it. If they try to take anything to market they will be dealt with.
That being said Marker's plan right now is to beat them to market which is obviously likely as Marker is way ahead of Mana. At this point this is the only thing they can really do that will have much effect since I don't believe any lawsuits can be brought against Mana as the work is all clinical.
Gotta blame someone. The fact that that bio market has been in a downtrend for a while, with smaller bios being hit harder, certainly has nothing to do with any of this.
The AML trial is specifically for post stem cell transplant. Initial treatment is still chemotherapy. MT401 is not being given as a stand alone therapy. It will be given only to patients after they have had a stem cell transplant. Prior to the transplant they more than likely will have gone through chemotherapy already. In the adjuvant arm they can compare to a standard of care (SOC) because the SOC post transplant is just observation.
It looks like they are trying to somewhat mirror the BCM pancreatic trial in that those patients were not given MultiTAA until after chemo. I would imagine they feel this gives them the best shot. Keep in mind they are only running this trial to get a data set that will be large enough to appeal to a BP partner. The future PII trial design will be the one that really matters.
There's your answer. They did slightly modify the antigen mix for pancreatic.
I wish I could. I can only speculate like anyone else and my guess is that they are just waiting to announce everything on the Q4 conference call which should be taking place sometime between now and February 15th. This orphan designation just confirms, at least for me, that they will indeed announce a new pancreatic trial like I mentioned recently. With the share price so depressed as it is I honestly don't think it really matters if they announce orphan designation now or within the next couple of weeks.
Based on a previous conversation I had with the company I believe they will continue to be keep most news under wraps until it is absolutely necessary to release it. They acknowledged that in the past they were burned by having news released prior to when they were ready and don't want to have that happen again. They lean heavily on their external business advisors (Solebury Trout) so I'm sure that is playing a part in their decision to release news now or later.
I definitely understand the frustration with the lack of news. I personally am comfortable ignoring it as I believe that as long as the company executes on their plans in the end it won't matter if they announce orphan designation now or in a couple weeks. The other thing is that there isn't much news to really be had. For the most part they have released everything that is necessary to release. If you are going to be frustrated with anything it should be the fact that they don't have anything going on that is worthy of a news release. Not the fact that they aren't releasing news. Again, all of this should become moot in the coming months.
We'll get interim data on active group of the PII AML trial end of Q1. It's reasonable to then assume we get full data readout on the active group sometime mid year. I would assume we get some update on enrollment for the adjuvant arm as that one will take longer to enroll. Per Dr. Koneru's comments during her last presentation could see full enrollment by the end of this year. I would think a more conservative estimate would be early next year but depending on how it goes it's possible we see some interim data on the adjuvant arm end of this year or early next. If the active group data is sufficient we could see a BLA filing as early as the end of this year with a decision on approval middle of next year. It really looks like they are initiating another trial in pancreatic. If that begins sometime in Q1 it's possible to get interim data on that prior to year's end with full readout the following year. This is the point I expect to see a partnership for a larger pivotal trial in pancreatic. These are definitely not hard timelines but there should be plenty of data and news worth events coming in the next couple of years.
My point here is that up until now there really hasn't been many news worthy events. The company chose to focus on getting the PII AML trial up and running before progressing with anything else. We can argue all day about whether or not this was a good decision or not but what's done is done. The AML trial is fully up and running and they are clearly getting ready to move on to more trials. This just means that at some point in the near future news will begin rolling in as they should have multiple news worthy events coming up in the next couple of years. Unlike prior years. What this will do for the share price remains to be seen.
Most likely a bunch of retail catching wind of the pancreatic orphan designation.
https://www.accessdata.fda.gov/scripts/opdlisting/oopd/detailedIndex.cfm?cfgridkey=775620
Orphan designation for MultiTAA in pancreatic. New trial announcement should be incoming
I'm just fortunate enough to have been able to open up a direct line of communication with the company. Back in 2018 prior to the merger I had reached out to the company's IR department which was internal at the time. I'm pretty sure it was just one person though. I asked a bunch of questions regarding PolyStart and it's progress as it had been hyped up quite a bit by prior management and at the time seemed to be the company's best bet to actually make something of themselves. The IR person could not answer my questions as they were more focusing on the science. He forwarded my email to Dr. Florkiewiczs, who created PolyStart, so he could provide answers to my questions and then asked if I would like to set up a phone call with the CEO. Apparently when Peter Hoang came on as CEO toward the end of 2017 he had been making a point of trying to communicate with shareholders who were contacting IR. I forget the frequency but IIRC they were trying to do two calls a month with individuals who were reaching out to IR. I can't imagine there were many people making valid inquiries though.
So, I had my phone call with Peter and asked my PolyStart questions and some financing questions as at the time I was irritated with all the warrants that prior management kept issuing. He was straight up with me. Said that prior management hyped up PolyStart a little too much and it was nowhere near where they had been saying it was and still had a long way to go before it would be of much benefit to the company. Side note, I've fairly recently brought PolyStart up again and was told that their lab techs were working with it and found some "interesting applications" for it. We'll see if anything ever comes of it. He understood my frustration with the warrants and said their goal moving forward was to keep the warrant situation in check. Throughout the conversation I focused on asking my questions about the science and financing but still let it be known my frustration with how the company seemed to be operating and the slide in share price. I’m always going to remember this part of the conversation because he said, paraphrased, “I understand your frustrations and that it has been a long wait, but you won’t have to wait much longer.” At the end of this conversation, he said that these calls don’t usually go like this. I’m assuming that the other calls they set up were people just complaining about the share price but not actually trying to learn anything about the company and their plans for moving forward. He said if I ever had any other questions, I could email him directly. I’m assuming this is because I showed a genuine interest in what the company was doing. Anyways, I kid you not, he emailed me right after the merger was announced to say, “see, I said you wouldn’t have to wait much longer.” We set up another call and I got some information on Marker, MultiTAA, and some insight into what the plans for the future were going to be.
Since then, I’ve emailed periodically with questions and/or concerns. I am generally able to set up phone calls around “eventful” news or if I have questions that might need a little more detail than one would want to type in an email. I would say I send an email off once every couple of months and have phone calls maybe three times a year on average. I don’t get any direct information that wouldn’t be available to anyone else who asked but during my communications things are implied and I can read between the lines to make certain assumptions. As an example, I’ve stated I expect to see new trials announced by February 15th next year. I was not directly told this, but I inquired as to why they did not do a third quarter conference call as they generally do a call for Q1 and Q3 results. During this inquiry I also expressed my concerns that the fate of the company was resting on this upcoming interim data and with no other trials to fall back on it could spell disaster should the results not be received well. For the record, I still think the results will be good, but it is only for the active group right now which is a lot more difficult to treat. During this conversation, and based on previous communication, it was implied that they are indeed working on initiating new trials and that the reason they delayed the Q3 conference call is that they feel the call would be better suited for Q4 results, which are due by February 15th, as they seem to want to announce something that wasn’t quite ready. This is just a brief rundown of that conversation and doesn't include every detail, but I decided to share my expectations as I do believe them to be highly probable of happening. Otherwise, I would have just kept my assumptions to myself because if new trials aren’t announced in Q1 of next year I am going to look like a dummy. Regardless, I try to share as much information as I can obtain as I feel it will help others.
All of this to say that I am not on the “inside” but have been lucky enough to be able to communicate directly with the company which does provide me a little more insight that what comes out via PRs but nothing that one couldn’t figure out on their own. I would be lying if I said I wasn’t biased though. Everyone is biased and almost all opinions are based on some bias in one way or another. Most of what I post can be verified and if not, I will indicate as my opinion. It’s all about presenting enough evidence to back up those opinions as much as possible whenever possible. Whether I’ve done a good enough job of that or not is up to whoever is reading.
The last I followed up on the antigen mix for future solid tumor trials it sounded like the plan would be to just continue on with the current antigen mix given that they did see some success with it. That was a while ago so I guess now is as good a time as any to follow up on it. I just sent an email off and will let you know if I get a response.
I figured since it’s the end of the year I would put together my thoughts on how this year went and what we could potentially see in the future. Notwithstanding the current share price, the company has made some decent progress this year which hopefully they can carry forward.
Manufacturing: The company opened their in-house manufacturing facility. This gives them complete oversight of the full manufacturing process which, given how complex it is, should help ensure the highest quality of product moving forward. The facility seems to be designed in a way that is cost conscious and allows them quite a bit of flexibility. From what I was told it cost them ~$6M to get it up and running to the point that it is right now, supporting the PII AML trial, and if they fully build out their current space it would be under $20M. I personally don’t think that’s too bad all things considered. I got to tour it and it is literally just a giant warehouse with closed off rooms built inside where they manufacture and store the product. When they need to expand, they just build more rooms and if they ever need to leave their current space, they just disassemble the rooms and move them to a new location. I would imagine having this infrastructure in place would only help to attract BP when the time comes. In addition to the new facility, they also announced improvements to the manufacturing process earlier this year. They were able to reduce the original manufacturing time by 50% and are currently working to bring that down another 50% for a total reduction in time of 75% from the original time taken at BCM. This will help them dose patients a lot sooner. They were also able to reduce the number of operator interventions by over 90%. The more people handling the product the more chances there are for mistakes to be made and potential product degradation so reducing that by 90% is pretty big. These improvements, along with others, helped increase the overall number of T cells available at the time they are administered to the patients which should in turn produce improved trial results moving forward. This new process is being used for all patients in the PII AML trial so hopefully we see an improvement over the PI results.
Financing: They closed an offering of ~32.2M shares at $1.75. Total proceeds after offering expenses were ~$56.5M. This offering was highlighted by purchases of ~5.7M shares ($10M) by NEA, ~1.7M shares ($3M) by John Wilson, ~1.7M shares ($2M) by Aisling Capital, ~570K shares ($1M) by Juan Vera, & ~142K shares ($250K) by Peter Hoang. As of the end of Q3 2021 they have $48.7M which they anticipate should fund operations into Q1 of 2023. It has become apparent, and probably isn’t a bad idea, that the company does not want to have less than a year’s worth of operating capital in the bank so I would expect some sort of financing to come shortly after the Q1 2022 data release. They still have ~$24M available under their purchase agreement with Aspire Capital which expires sometime around August or September of 2023 so they don’t necessarily need to do a full separate offering to extend their cash runway. I don’t think they go much further than mid 2022 before they look to do some sort of offering. They do have outstanding warrants that could provide future capital but most of them are exercisable at prices far higher than we are now. ~5M are held by the original Marker holders, most of them held by John Wilson, Juan Vera & Ann Leen, are exercisable at $2.99. ~13M warrants are held by the participants of the original financing done concurrently with the merger. These are exercisable at $5 with the major holders being NEA, Aisling, Perceptive, and Baker Brothers. All the above referenced warrants will expire in October of 2024. I fully expect most of the 13M warrants issued in the original financing to be exercised at some point but it probably won’t be anytime soon.
Grant Funding: They received a $13M grant from CPRIT to fund their AML PII trial. Given that this trial was estimated to cost around $25M in total this grant covers just over half of the entire trial. It basically frees up $13M for the company to allocate elsewhere which for a small biotech company with zero revenue is a big deal.
Trials: Obviously the pivotal PII AML trial is the main focus with data on the horizon. My best guess based on prior communication w/ the company, and this is the optimistic timeline, would be interim data on the active group end of Q1 2022, full data readout mid-year 2022 (sometime end of Q2 or beginning of Q3) and a BLA filing by end of year. If the BLA filing is done end of 2022 I would expect a decision on approval to then come sometime mid-year 2023. MT401 does have orphan drug designation for AML so FDA review should be quicker than normal. Again, this is only for the active group. The adjuvant group is a bit more difficult to nail down as it is larger and will take longer to enroll. Dr. Koneru did mention in her last presentation she thinks full enrollment of the adjuvant group could happen by end of 2022. Once full enrollment of the adjuvant group is complete patients need to be monitored for at least 12 months. If this timeline holds, I would expect interim data from the adjuvant group in the middle of 2023 & full data, BLA, and decision on approval sometime in 2024.
In addition to the AML trial, I expect them to announce the initiation of new trials in Q1. As I've said before I am 99% sure they will announce a pancreatic trial next month or at the latest by February 15th. I hope I’m not eating my words come that time. This trial shouldn't take nearly as long to enroll as the AML trial and patients also won't need to be monitored for as long. I fully expect this pancreatic trial to be done with the intention of partnering it out for a pivotal PII. If they start it in the beginning of the year and can reach full enrollment by mid-year, which could be possible as I expect this to be a smaller trial, maybe around 25 patients, they could have primary outcomes reached as early as end of year 2022 or early 2023. I think it would look similar to the PI done at BCM. If data is half decent, I don't think they will have any problem finding a BP partner sometime in 2023 to move the pancreatic trials forward.
It would not surprise me to see the initiation of even another trial in Q1 2022. If I had to take a guess, I would assume lymphoma but I’m not as confident in this assumption as I am with pancreatic. This one I would guess would somewhat mirror the AML trial in that they will design it to be pivotal and fully intend on running it on their own. No guess on timeline of this trial, if it’s even announced, as it will be new territory. It is possible they hold off on this one too and just run with two active company sponsored trials but the more shots on goal the better so if it won’t run them too thin, I’d like to see at least these three trials running at the same time.
If they can get these trials up and running, we should see plenty of data in the next few years as well as the potential for some FDA reviews. This is the stuff that moves share prices. Most of us on this board have been drowning for quite some time now but if they can deliver on half of what I’ve detailed above it just might be the life preserver we've been waiting for. Even without the current market situation for small bios it’s tough to sustain any kind of growth without producing the goods. Up until this point we’ve been running on faith in the technology and hope that the market would see what we all believe we see. Now we are about to get some actual new data rather than the rehashed PI data. The goods are coming and I’m in too deep to back out now. I’m either sailing off to riches and glory or going down with the ship.
Good luck to all you crazy SOBs.
The BLA is only filed after they have data to support a review. If the optimistic timeline holds, and while early it looks like it will, then we should see interim AML data on the active group end of Q1 2022 and potentially the full readout mid 2022 (sometime end of Q2 or beginning of Q3). If data is good enough to support a review then I would expect a BLA filing by the end of 2022. Since MT401 does have orphan designation I would expect a decision from the FDA no later than mid 2023. This is not an absolute timeline but just based of my best guess from the DD I have done.
A lot of the same but this time there really is data on the horizon. Interim data from the active group of the PII AML trial expected end of Q1 next year. I expect sometime before the end of January, and would all but guarantee by February 15th, we get news of the initiation of one or more new trials. I would be shocked if one of those isn't pancreatic and if they initiate more than one I expect lymphoma to be the the other. I believe I remember being told that full data from the active group could be announced sometime mid year next year. If that timeline holds true we could see a BLA by end of 2022. I would say that is being very optimistic on the timeline and more realistic timing would be BLA filing early 2023 if data is good.
Seems to have been brushed off but they received a grant from CPRIT for ~$13M to be used for the AML trial. This covers just over half the total cost of the entire trial.
Their manufacturing facility is fully up and running with capacity to expand upon new trial initiations and commercialization should that be granted. They continue to improve the manufacturing process which could bode well for the PII trial as well as the others they look to initiate.
This is obviously all great for the company but the stock keeps getting hammered. Our only saving grace is that it isn't just MRKR. Most smaller bio companies aren't doing so hot either. Sucks that when we finally have some news on the the market as a whole is a little under the weather. Would much rather the launching off point be somewhere around $5/share but right now it's looking like we'll be lucky to see $5/share even after the date release.
All this being said there is no way the current MC conveys actual value of the company. At $5/share that would put MRKR at a MC of ~400M based on current outstanding share count and ~$500M based on fully diluted share count. While I think ultimately the company should be worth more than that there is no reason MC should be any lower with the current state of the company.
It's a non issue. I wouldn't bother worrying about it. Clearly no one else is.
There is no issue with IP. This person keeps pushing that narrative and has been since the beginning of the year even when presented with supporting evidence. They can never point to any actual legitimate reasons to even believe there might be an issue with it. Previously they said they would "do some research and come back with a response." I put them on ignore so I don't know if they ever came back with any findings but I put the odds at practically zero.
Again, there is no issue with the IP. You can read the licensing agreements between MRKR and Baylor and it clearly shows who owns what and how everything will be dealt with in the future.
I've touched on how I think the pancreatic trial will go in the past and my thoughts stay the same. They will ultimately partner it out to BP. Issue is the BCM PI is only 13 patients and they have their own number they usually like to see in PI data. Usually somewhere around 25. With all the manufacturing process updates MRKR has made, and continue to make, I expect they will initiate a company sponsored PI with roughly 25 patients all dosed with product using the new process. If results mirror or are better than the BCM PI, and they should be, then the PII will be pivotal and with a BP partner. On her last presentation Dr. Koneru illuded to news coming on more trials in the near future. They usually do a conference call for Q3 results but deliberately pushed it back to Q4. This call needs to happen by February 15th. I strongly believe that we will get an announcement on the initiation of a new pancreatic trial and possibly another indication, my vote is for lymphoma, and any other plans for the pipeline moving forward. This should all come prior to the interim data from the AML trial which should be toward the end of Q1 to allow them to collect as much data as they can.
Obviously none of this is concrete but is my opinion based on the DD that I have done. Take it as you will.
I believe they are only mentioning the 20 patients enrolled by the end of the year because that will be the data set they will be reporting the interim results on in Q1 next year. Officially, it makes sense that they would not give a timeline on when they expect full enrollment as there are many things that can come up that cause delays. It's one thing for them to announce next year that there was a delay in enrollment but another for them to announce next year that there was a delay in enrollment when they already stated enrollment would be complete by the end of this year. This statement of 20 patients enrolled by the end of this year is not new. They have been stating this for the past few months. Per prior communication with the company and reading a bit between the lines I believe that enrollment of the active group should be complete around the end of Q1 next year with a full readout on the active group sometime mid year. If this timeline holds the BLA filing could then come by the end of next year for the active group. The adjuvant group is larger and they have not provided official guidance on enrollment timelines but I believe Dr. Koneru said in her last presentation that full enrollment in that group could be reached by the end of next year. They've been opening up new trial sites over the past few months and have a few more to get open so enrollment should only pick up as new sites are added.
Are Doctors NOT recommending Marker trials for AML and Pancreatic and if so, why?
You've been talking about the IP since March yet haven't done any looking into it? Can't believe you actually will now.
And your reasoning for the manufacturing is that they aren't going to be a CMO and they should? Or that they don't own the building? Neither of those make any sense at all and no one is learning anything from listening to opinions. You need to provide evidence of your claims if you want people to learn.
This will be my last response to you.
Good luck.
Lol. If you know so much why don't you actually provide reasoning for your viewpoints? It's easy enough to say the IP might not be strong enough or management is paying themselves a bunch of cash bonuses but if any of this is accurate how come you can't provide evidence to your statements? You saying you know a lot doesn't mean anything if you can't prove it.
Surprisingly I think learningcurve is pretty close. If I remember correctly it was stated the trial would cost roughly $25M or so.
Ah yes. If there is one thing this company is known for its doling out millions in cash bonuses.
Per the PR the grant is going to support the adjuvant arm of the AML trial. What it does though is free up $13M of their own funds that they can now allocate elsewhere. The logical thought would be that that frees them up to initiate another trial. I believe we should be hearing updates regarding new trials by the end of this year or early next year.
Per data from Yahoo Finance today was the 6th highest volume day since the merger. Below are the top six days for share volume with their estimated dollar volume:
3/19/2021 - 64,414,800 ($150,730,632)
3/22/2021 - 43,000,600 ($130,076,815)
3/12/2021 - 12,932,600 ($24,313,288)
3/18/2021 - 9,309,700 ($16,990,202)
3/23/2021 - 8,917,300 ($22,739,115)
8/20/2021 - 6,108,146 ($13,652,880)
It is strictly for their use. They have no intention to offer services to anyone else. It's pretty black and white.
You are literally the only person bringing the IP up. I've responded to you before. I see no issues with the IP. If you can point to specific reasons you have to question this I would be more than happy to discuss further but as of right now there is nothing more to discuss on this topic.
I honestly have no clue where this goes on approval. Until I see something that brings me to doubt myself, and I haven't yet, I will continue to stick with my opinion that the MC here should be somewhere between $1B - $2B. Current outstanding share count is ~83M. This would equate to a SP between $12.05 - $24.10. I like to look at the fully diluted numbers and last I checked I think the fully diluted share count was around ~115M. I think that was after the last offering so it should be the current number. Someone please correct me if I'm wrong. This would equate to a SP between $8.70 - $17.40.
I get that these are large ranges but I don't like picking a singular number due to the large fluctuations generally seen in these types of stocks. I personally see no reason MRKR should be under a MC of $500M (~$6/shr) right now but clearly the market doesn't agree. I am still expecting a rise into the Q1 2022 data to start sometime before the end of this year.
Where are you getting your numbers from?
THIS release directly from CPRIT states they just awarded ~$142M in grants. Not a number into the billions. This program is funded with bonds that are currently capped at $300M per year. HERE is the ballot initiative if you care to read into it. You talk about how pathetic the grant is but it seems you have zero understanding of how this all works.
Very promising 2 yrs ago to now just staying afloat above the quicksand
For all of you who have stuck around all these years it about to crater big time if this on gong trial doesn’t show significant results to get an approval from the FDA
The IP does not need to be addressed. It's a non issue.
Has Marker set up the manufacturing operation so common shareholders benefit directly?
is all the IP solidly in place for success for Marker common should the biological show promise?
Lol. I'll never say zero but in this case it's as close to zero as can be. They have no money to be buying other companies. AlloVir has a MC of over $1B. No reason to even entertain the thought.
There is damn near zero chance they acquire anybody. Especially not AlloVir.
I'm not going to defend the filing because it is horsesh*t but they did already have an active shelf. They could have been actively diluting this whole time and they weren't. Just because they filed a new shelf doesn't mean it will be used right away or at all. At this point the only thing we know for sure is they have an active plan to use up to $75M of it in the near to mid future. They better have a plan for that.
They should be releasing earnings this week sometime and more than likely there won't be a call this quarter. This is all based on previous years.
I'll give two answers due to the trial being split into two groups (active and adjuvant) and my understanding that they will be submitting two BLAs. The only caveat I want to put on this is my answers are going to assume that the improvements to the manufacturing process result in better trial results as I personally believe they should.
With the adjuvant group I would go with a 9. If the PII results can just mirror that PI results I see no way approval isn't granted. That being said I am expecting the results to be better than the PI due to the previously mentioned manufacturing improvements. The only thing with the adjuvant group is that it is the larger of the two so will take longer to enroll and longer to complete as patients need to be monitored for at least a year. We should be getting interim data along the way but I wouldn't expect a BLA filing for this group until late 2023 or even sometime in 2024. This is my conservative guess based on an assumption of the adjuvant arm reaching full enrollment by the end of next year which certainly isn't a guarantee.
The active arm I would put at a 6 or 7. This one has a lot more to consider and I believe it is all going to depend on how it is ultimately presented. The primary endpoint is based on CRs and their duration. Tumor reduction will also be taken into account. The PI only had eight patients with one achieving CR. That's about a 12% CR rate but the sample size is too small to really assume much. If they see a 12% complete response rate in the 40 patient sample I think that should bode well for them. A comparison should be made between MultiTAA and Donor Lymphocyte Infusions (DLI). DLIs are given when a patient relapses post stem cell transplant (SCT). Unfortunately they are generally reserved for patients that are in relatively good shape, all things considered, as they come with some serious side effects. Essentially if someone relapses post SCT and aren't eligible for a DLI there are almost no more options. If you are eligible for DLI it comes at a high cost and serious adverse effects. The main issue with a DLI is Graft vs Host Disease (GvHD) where the donor cells attack the recipient’s healthy cells. The incidence of GvHD in these patients ranges from 40%-60% with 20%-35% being grade 3 or 4. The ORR for DLIs is anywhere between 5%-10%. So all this said, if MultiTAA can just match the response rates of DLIs, ~10% on the high end, it should be approvable based on the extremely favorable safety profile. If approved it could potentially replace DLIs and also be administrable to patients who previously did not qualify for a DLI. I would assume the FDA will make this connection but I really have no faith in them so we'll see what happens. The interim data Q1 of next year will be from the active group. IIRC the full data readout should be available sometime mid next year but it is dependent on how quick they are able to enroll this group of 40 patients. If everything runs to these timelines or better I would expect a BLA at the end of next year at the earliest.
As a whole I have high expectations for this trial. It's become very apparent we need data to move the SP higher and hopefully hold gains so hopefully that first data release will be promising.
Can do, lol.
That makes sense.