2021 for PCI Biotech. My name is Per Walde. I'm the CEO of PCI Biotech and I have with me also Ronen Skogedahl, who is the CFO of PCI Biotech. First, please pay attention to this important notice. We have in this conference now a webcast, but also a phone line.
And you have all the information on this slide for how you can post questions through the webcast or through the phone live after the presentation. I will start the presentation with going through PCI Biotech, the company in general and the technology specifically, and then review each of the 3 different programs a bit more in detail before we go through the financial numbers and the outlook at the end. And then we take questions. I think we start with live questions and then we will go over to questions from the webcast at the end. So first, PCI Biotech.
PCI is an oncology focused pipeline company. We are developing a specific technology for the chemical internalization. We have 3 different programs. We call them FIMAChem, FIMAVAC and FIMAC. And FemaChem is our lead program.
A product called Amphenex is developed for bile duct cancer treatment in combination with Gemcitabine. And this is currently in global pivotal development in a study with registration intent. The second program is FIMA WACC. It's really utilizing the same platform to enhance immunotherapeutic effects of different kind of vaccines and other immunotherapies. That has been through a Phase 1 and with some very good results showing Good immune responses with our technology and we are now looking at taking this to the next step.
And the 3rd program is a preclinical program. It's called FIMA NAC. NAC stands for nucleic acid. And nucleic acid therapeutics is a new class of drugs that are being developed, which this platform can help to really realize the full potential for certain applications of these called the so called nucleic acid therapeutics or gene therapies as they are called. So all of this is done through a platform called photochemical internalization.
I'll talk a little bit about what PCI or photochemical internalization is before I move on to the highlights for the first in Q2. So all cells in the body have a cell membrane around the cell. And this cell membrane is sort of the wall that protects the inside of the cell from whatever is outside of it. And a number of Compounds will have problems passing through this cell membrane getting into the cell. And then if the therapeutic target where the medicine needs to reach to actually be active is within the cell.
It actually needs to pass the cell membrane. And therefore this limits the efficacy of a number of drugs. And some drugs are completely non efficient without being taken up by special means into cells. There is also an eating mechanism in all cells called endocytosis. This is where they invaginate a certain part of the cell membrane and create a bubble where they take in what has been outside of the cell.
And this is like a digestive system afterwards in the cell. Now a number of drugs that can't pass through this cell membrane will be taken up through this endocytosis, but it's then trapped in these small bubbles inside the cell. What we have is a technology that can open up these bubbles. We have a photosensitizer that accumulates inside these so called endosomes, these bubbles. And when we illuminate, you can rupture the membrane, destabilize the membrane of these small bubbles and whatever has been taken up can leak into the cell.
And thereby, the therapeutic target being in this capsule can leak out and be effective. So that's the platform technology, the basis for everything we do. As mentioned, we have 3 different programs. One is then PimaChem where we enable already approved drugs to become more effective by enhancing the local effect of them where it's most needed. FIMA WACC is to enhance the immune responses that are important with vaccines for example for a therapeutic effect.
And Feimanak is to enable big nucleic acid therapeutics. These are big molecules that can't pass into sales without some kind of help and they need to get into cells to work, we provide the delivery solution for this. So this is in a nutshell what PCI Biotech is and what we do. So let's have a look then at the highlights for the first half of twenty '21. So to start with, going back a bit, when COVID-nineteen hits us in 2020, We started immediately and we saw that this really had was a problem for the capacity of hospitals.
We understood that this would affect the clinical trial. We started to implement a number of different initiatives and also to expand the study at that time. And these implementations was done during the autumn last year. And we saw effect of this at the beginning of this year. We saw that the opening of Asian sites and the amended protocol increased the screening and increased the enrollment to release in Q1.
And we reported then that we were cautiously optimistic. We saw a good trend. Now unfortunately activity declined significantly in Q2. We had only 3 patients included during that quarter. And this now with the delta virus really having a large effect in certain regions, We can't really keep what we've said has been the time range for this.
And now the expected interim analysis for a potential accelerated approval is revised to second half twenty twenty three. We currently have a financial runway to well into the second half of twenty twenty two, but not covering this interim read then. Now on the positive side, recruitment started stronger again in Q3. We had 4 patients enrolled in July, which is normally a relatively difficult period. And then we have August after that, which is the most difficult month in the month in the year.
On the positive side also, we had our 1st U. S. Patient enrolled in the release study last year, the first half, I'm sorry. And we also had orphan drug designation granted in South Korea for bile duct cancer in treatment in combination with gemcitabine. And we've had good activity in South Korea.
So that's a key market for us in Asia. So it was really good to have this orphan drug designation granted. Now what we are continuing to focus on is, of course, enrollment of patients into the release study. We're emphasizing really regular trial management, Overall performance evaluation, replacement of underperforming sites with sites we think can perform better And also to manage and proactively manage all the risks that we see in a study like this, so that we ensure that we can recruit and make this and complete this study successfully as quickly as possible. For FEMAWAC, we had a successful Phase 1 vaccination proof of concept study that I mentioned published in a high impact immunology journal early in the year, Frontiers in Immunology, where we saw that immune responses to peptide and protein based vaccines are really enhanced by our technology in healthy volunteers.
We also had a very important patent for FIMA work granted in the U. S. This is in combination with immune checkpoint inhibitors. And these are normally what you today talk about as immune therapy, very important drugs for cancer treatment. Unfortunately, a number of patients do not respond to them.
And this is by means of enhancing The immune response through different technologies you may be able to increase the rate of responses to this immune checkpoint inhibitors. That is what we think we can do with FIMA WACC. And in that perspective, this U. S. Patent is very important.
And then the highlights for FEMA NAC. We had encouraging data on enhanced delivery of mRNA for various medical applications presented at the conference early in the year for RNA therapeutics. This was collaborative research that we've had in our collaborations as for Manak is primarily a collaborative program. We also established a new research collaboration with a South Korean company, a quite extensive collaboration with Olix Pharmaceuticals, who are a leading developer of Interference RNA Interference Therapeutics. In addition to this, We have also now we are also now significantly strengthening the organization.
We have employed 3 highly skilled individuals, one experienced operational leader for release that will drive this study taking forward and 2 key employees within clinical science and business development who will be focusing more on FIMA WACC and FIMA NAK. As most of our resources have had to go into the release study and ensure that we get all these initiatives implemented and which we have seen a good effect of, which but unfortunately, As the pandemic has not resolved as quickly as we hoped, we are still seeing fluctuating recruitment this year. Those were the highlights for the first half twenty twenty one. I will now go into a deeper dive into the 3 different programs and start with FIMA Chem, which is in development for first line treatment of the orphan indication bile duct cancer. We have some very positive early clinical results from a Phase 1 study, both with tumor response and with survival data.
We have had discussions with the regulatory authorities both in Europe and in the U. S. And we have a pathway to market settled by these interactions. We can do a single pivotal study with a potential accelerated approval based on an interim analysis, which is now expected to be second half twenty twenty three. So what we're doing now is what we call the release study.
And the release study is a global pivotal registration intent study. Excuse me. And recruitment is ongoing at approximately 50 hospitals across the 3 continents. And the number of hospitals will Vary a bit going forward as we replace those who are underperforming and put in place new sites which we think can perform better. So there is an excellent fit between the medical need.
I'm sorry, I just need to adjust this microphone. So there is an excellent fit between the medical need and existing treatments with PCI. We really can enhance the effect of gemcitabine, which is a drug that is being used as a standard drug in cholangiocarcinoma. It's also easy to illuminate cholangiocarcinoma, bile duct cancer because you can go in with an endoscope and put a fiber into the bile duct and illuminate the tumor from the inside. And it's also a disease where you need better local treatment.
Most of these patients die from the local effects. So local regional control of the tumor is very important. We did a Phase 1 study here as I mentioned. We saw a median overall survival of almost 23 months at the selected dose for the release study in the dose escalation part of this. And the normal Overall median overall survival in the standard treatment today is little less than a year.
So some very encouraging but early results. I mentioned easy to use. Also this means that since this is the first recommended first line therapy, gemcitabine in combination with cisplatin. And we enhanced the first line treatment. Our treatment also is the first line, which is not very common when you come with a new technology within oncology.
We have, as I mentioned, also orphan drug designation, not only in South Korea, but also in EU and the U. S, which offers market exclusivity. And then when you look at the competition, it's not that much in development. Most of it is precision or gene or small molecules that are mainly second line or That are mainly second line or towards mutations or targets that are present primarily in intrahepatic bile blood cancer. I think the main competitor that is targeting a broader population containing our population is Nucana.
So but that's really the main only main competitor that we see in the development pipeline. Of course a premium price potential is expected with an orphan disease like this. It's a rare disease. So It's a good market potential for that. As I mentioned, South Korea, in May, We had the Ministry of Food and Drug Safety in South Korea granting us orphan drug designation for fimaporphin.
And this is then in combination with gemcitabine in patients with an operable locally advanced or metastatic bylaw cancer, which is our target population. And this may provide several benefits, including conditional approval, extended market exclusivity and exemption from required data. So we have now with this, as I said, orphan designation granted in 3 major markets across all the continents where we are doing currently the release study. So what is the release study then? It is a study in 186 patients with a 1 to 1 randomization between standard of treatment today, which is gemcitabine plus cisplatin and experimental arm where we enhanced gemcitabine in up to 2 different cycles with our PCI technology with FIMAChem.
And you can see on this slide the treatment regimen at the end at the bottom and our treatment part of it in the little box on the right hand corner down in the right hand corner. We have 11 European countries, 2 Asian countries that is South Korea and Taiwan and the U. S. That are open in this study. So the status of it, 47 sites currently open.
We have 9 sites in Asia. We have 6 sites in the U. S. And the rest of the sites are in Europe. We had the 1st Asian patient enrolled in October 20 and the 1st U.
S. Patient enrolled in April 21. Our first patient in Europe was enrolled in May 19. We have, as I said at the beginning, implemented several initiatives in the autumn of 2020 with the aim to recoup what we saw as the COVID-nineteen caused delays. And the most important there is, of course, Increased number of sites, we expanded the study and also a protocol amendment where we went through the screening log that we had to see What was the screen failures here and how can we adjust this to expand the eligible patient population.
The expansion to Asia has contributed significantly to patient recruitment, specifically South Korea is a very active country, which is a quite positive sign. And they seem to have good control of the pandemic situation there for the hospitals. So this initiative, the initiatives that we did resulted in increased screening and enrollment in the Q1. And we were positive and thought that this would increase further as the pandemic resolved more and more. Now then the delta variant of this virus came and a number of companies reported that both sales and clinical trial enrollment decreased in Q2.
It was a difficult quarter. It was really a difficult quarter for us as well, May June specifically. We only had 3 patients including included during that quarter. Now Q3 again started stronger with 4 patients included in And as mentioned, August is always a difficult month because it's the holiday season in many of the countries. So the focus now going forward with regard to the progress of this and timelines is on regular trial management as mentioned.
We look at the overall performance evaluation with replacement of underperforming sites And also proactive management of study specific risk, such as retention of randomized panor patients that always a risk, especially in open label studies like this, that we lose patients from the control arm and we need to follow that and proactively manage that. So we are looking at strategies for how to best do that. And then also adherence to study procedures and eligibility criteria, which is always important for the quality of the study. Now as I said, the previously communicated time range for interim readout, which was a year, it was from Second half 'twenty two to first half 'twenty three required had a recruitment plan that from now on required 14 to 18 patients per quarter. Our best quarter to date is 10 patients.
So it's a bit above our best quarter. And this is the average that is needed. This was an ambitious plan. And in this, we looked at what could we do with the expanded sites with the eligibility criteria that had been modified. And also when we put this plan in place, we assume that after the summer, the COVID-nineteen impact would be clear and close to gone on the study.
Now where we are today, we consider that unlikely. So therefore, on this basis, we are revising the expected timing of the planned interim analysis to second half twenty twenty three. Having said that, we now have full focus on release. We have further strengthened the team with an experienced operational leader. And we are identifying all opportunities to optimize the overall performance of this study from the operational part to the design part of the study.
The study today looks like this. We have an interim analysis for potential accelerated approval, which looks at objective response rate and is a secondary overall survival. The final analysis is progression free survival. The secondary endpoint there of course important is overall survival. We had our first patients enrolled as I said in 2019, 2020 and 2021 respectively in Europe, Asia and the U.
S. U. S. Has been underperforming and it has There is a difficult situation also now in the U. S.
With the Delta variant. We have done a small extension of the Phase 1 study to look at the ability to do 2 treatments. The data I showed you with 22.8 The months for median overall survival was based on one treatment, but there's no reason to think that we can't do 2 treatments of this of the PCI treatment during the cycles. And Therefore, we did an extension to the Phase I study to look at safety in a few patients. We want to extend that to have IDMC, our independent data monitoring committee to confirm the safety of 2 treatments after we've treated 8 patients with 2 treatments and they've completed the safety window.
In this study, it is either 1 or 2 treatment depending on a number of factors and decided by the patient and the investigator. But we now think with the patient populations we have that we will have the safety review this half the second half of this year. And then the interim analysis, second half twenty twenty three. And we have approximately second half twenty twenty four for the final analysis, depending a bit on what happens at the interim analysis. So that is where we are currently with the release study.
We have some very strong results. We have good faith in this study. We have been struggling with recruitment during COVID-nineteen. But we really implemented in the organization and worked hard to make sure that we have the best possible study going forward. And we have good faith in the treatment and the potential of FemaChem for this patient population.
Then let's move over to our 2nd program, which is FIMA WACC. FIMA WACC is then using our technology enhance immunotherapeutics. We have some really compelling preclinical results where we see particularly strong CD8 T cell immune responses which are important for the therapeutic effect of vaccines. We did a Phase 1 study in healthy volunteers with peptide and protein based vaccines. And we saw some very good results that these were published earlier this year.
And this is really a versatile vaccination platform because it can potentially be used with several modalities, not just protein peptide based vaccines, but also nucleic acid based technologies. And to that end, for example, intratumoral immunotherapy with naked mRNA is an interesting area. And we have some encouraging preclinical results when it comes to the expressing the fold the increase of expression of mRNA in cells using FINAVAK in TC1 tumor cells and in MC38 tumor cells. And you can see the fold increase in this figure, which is quite high and also comparing in one of the tumors here versus LNP's lipid nanoparticles, which is the normally used technology today to deliver mRNA. So with this, you can also get systemic therapeutic effects.
And you can have mRNA that either in antigens or other immunostimulating factors. Important thing is here also that the expression is really confined to tumor. That may be important to avoid potential side effects of some of these technologies. And the positive thing that we saw clearly better effect than LMPs with FIMA WACC. And we also have with this photochemical treatment with FIMA WACC a modulation or a potential inherent adjuvant effect, a modulation of the microenvironment when we go in and do something directly in the tumor.
So we think this is an interesting area to look further into. We Have as I said also these were preclinical results the previous slide. This is clinical results in Health Volunteers. Where we saw successful clinical proof of concept for FIMA WACC in healthy volunteers with regard to both the tolerability and safety and immune responses. And we saw in that study what we provided with our technology was an increased number of responders, enhanced T cell responses.
And also those T cells that were enhanced had an improved T cell functionality, which is important for them to work as they should in combating tumor. So the full Phase 1 study were published early January 2021 in Frontiers in Immunology, which is a very good immunology journal. In June 2021 we also had this new U. S. Patent that was granted, which is covering the use with checkpoint inhibitors, a very important class of immunotherapeutics.
We have had during this period, as I said, had to focus during the pandemic, we've had to focus a lot of our resources into release, into the initiatives and into ensuring that we have the best possible study. And it has unfortunately reduced available resources for FIMA WACC. We have strong confidence in the commercial potential of this and also of Hima NAC, which is the next program I'll talk about. So we have strengthened the organization with 2 highly skilled people within Clinical Science and Business Development. And they are here to drive the preparation for a potential clinical proof of concept study for a therapeutic vaccination in relevant cancer, not just in healthy volunteers, but actually in the disease.
So I hope to be able to report more progress in this going forward from now. FIRA NAK, the 3rd program, it's a preclinical program. We have some really compelling preclinical Results, strong data for nucleic acid therapeutics delivery, intracellular delivery. And it works in synergy with a number of vehicles that are being used to try to enhance the effect of nucleic acid therapeutics. It addresses really the major hurdle for this class of drugs, which is getting sufficient payload of the nucleic acid therapeutics into the cells.
And we have then had this as a collaborative strategy where we work with companies with nucleic acid therapeutics in their pipeline to see whether there's a synergy between their technologies and our FIMA NAK technology that can help them enhance the effect of what they're doing. One of these collaborations produced some very promising results that were presented at an international RNA therapeutics conference early in 2021, where we show that this is local delivery technology where you can just mix mRNAs and Fema Porphyrin, our photosensitizer administer that as one injection and also illuminate in the same procedure. And we can get this nice expression and transfection of mRNA in the cells. And it's also then spatially restricted to where you illuminate. It doesn't happen outside of that area.
The beauty about this being a preclinical program is that it's already in clinic as a platform technology in the 2 other areas in FIMA Chem. So we have ample safety data in humans already with our platform. And there are a number of applications where a local effect is decided, a number of different diseases and organs. And we see in this what we've done a substantial enhancement when we do this into tumor, when we do it into muscle and when we do it into skin. And what we're now doing is we will increase our internal research efforts and actively center those to where we think the most attractive applications are to try to develop these applications to the next step and reach out in a more targeted way to where we have the biggest benefit.
We have established one collaboration in the spring with Olix Pharmaceuticals. This was done in May 2021. We entered into an extensive research collaboration with this company who is in South Korea And he's a leading developer of interference products in RNA, RNAi therapeutics. And we will combine our technology and know how and explore synergies and further partnership based on this. So we have a research collaboration and we do this collaboratively really.
It's a really true collaborative approach on this. And we look at look for synergies in these studies. It's all in preclinical with the current agreement. And then when we have the results, we will come back to see whether there is a potential to further enhance this. And it is within areas that are really Interesting because there is a need and medical unmet medical need for better treatments in specific local disease areas where the PCI technology fits quite well in this collaboration.
So January Research Collaborations, we have that within FIMA ANAK and FIMA WACC. We have currently 5 collaborations spanning across different classes drugs and different therapeutic applications. They, as I've shown you with the previous slides with some data, provide not just the collaborative effort in itself, but also a lot of scientific know how and encouraging results and also for us intellectual property. So it's important even those that may not lead to commercial agreement sometimes provide a lot of value to the company. And as I said the most recently with Olex, we are continuing to pursue new and value added collaborative opportunities with this technology.
And with that, I finished the FEMA NAC Kueh and Wack Parts as well. And the next is to go through the finances. And I have Ronniskogedal, the CFO here, who will spend some minutes to review where we are on the finances.
Thank you, Per. And first of all, it's good to see you fit and healthy here again. So I'm not alone at these presentations. So regarding the key financial figures. As you know, parts of our cash is placed in euros, meaning that we face fluctuations in the exchange rates affecting the net financial results.
And these variations you clearly see here in the figures in the net financial result. The BEA grant for FEMAWAC ended now in the Q2 of 2021 With SEK 13,400,000 accumulated funding over 4 years from the Norwegian Research Foundation. So a big thank you to them for this valuable support. We have a solid cash Position here of SEK146,000,000 at the end of the quarter compared to SEK 187 €7,000,000 at year end, meaning that we have spent around €40,000,000 during first half 'twenty one, partly placed in euros. And we expect this to give us a financial runway well into second half of twenty twenty two.
And I draw your attention to the revised Time lines for release. On the operational side, We have a fairly stable result around SEK 20,000,000 per quarter and SEK 40,000,000 for the half first half
year. I
think that was it, Per. Okay. Very good. Thank you.
So To close this presentation then, what we're doing is really progressing the PCI Technology pipeline, we have 3 different assets here. The most important one for us is FIMACEM because that's the lead project that is currently in global clinical pivotal study for registration intent. And there is an interim read in here for potential accelerated approval, which is important in this orphan indication. We have implemented improved trial design And we've done several initiatives to optimize the execution of this study during the pandemic as the pandemic has really affected the progress of the study. We saw recruitment increasing in 2021, also then especially with good contributions from Asia.
But it is still fluctuating, as we saw in the decline in the second quarter. The IDMC safety review of 2 treatments is expected this second half and Time lines for the expected interim read is now revised to second half twenty twenty three. For FINMAWAK, we have done a successful study in health volunteers with enhanced immune responses that has been published in a high impact journal earlier this year. We have also now a new patent granted in the U. S.
Which is very important for combination with immune checkpoint inhibitors, the most important immunotherapy on the market today. And this technology is today available for licensing. And we are planning also for clinical proof of concept in a disease setting with FIMA WACC. And we have strengthened the organization significantly to drive this forward. We are also then with the 3rd program in the preclinical setting, providing an intracellular delivery solution to nucleic acid therapeutics.
We had some really encouraging mRNA results presented at the conference early in the year. And we have also a new extensive research collaboration established within the interference area of RNA. And we are now actually center our internal research towards the most attractive applications for local treatment with an illuminated technology like the PCI technology. So I think that was the end of the presentation. So now we have we are opening up for questions to start with from callers.
And then After we've had those, we'll go through the webcast and see what we have questions from the webcast.
The first question comes from the
line of Svein Erik Sivensson. Please go ahead. Your line will now be unmuted.
Good morning. Good morning. My name is Svein Rick Sjevansson. First, thank you for a good and interesting presentation. I think as I said several times before that Ideally, you would like to have a partner for the release study in Asia.
We have been looking for a partner for quite some time here now. Does lack of success mean that there was no interest for a partnership in Asia?
No. And I wouldn't call it lack of success. These are processes that takes time. So we are still working on seeing whether we are able to establish a partner for Asia and we want to do that as quickly as possible.
So do you have does that mean that you have discussion with any companies currently?
We are not talking about we're not guiding around partnerships in timing or talking about discussions around things. We are actively working on this. That's what I can tell you.
So does that mean that you are optimistic with regard to getting a partner, let's say, by the end
That would sound like giving some kind of guidance. And I don't want to give guidance with regard to partners because we can't really control that process.
You have said a couple of times that The PimaVax study could be, not necessarily will be, but could be finished next year. There could even be some result next year. Do you still think that's possible?
I think with the Focus that we've had to do to take to the release study, I don't know. It's getting tougher to get that done. But now we have new people in the organization who will drive this forward. So we'll see what we can We have strengthened the organization really. So we'll see what we can manage to do.
I haven't really guided on that either. I've just asked someone asked hypothetically about when the results could potentially be available for this. And I said in a couple of years I think at that time.
Thank you. Okay. The agreement with Oleg Was announced, I guess, early May here. Can you say a little bit about the progression of that corporation so far?
It's progressing well. Research is being done and that's a very good interaction between the companies.
Have you started to test your technology together with OLX OLX 104C product to grow Back here on mice, have you actually started to do that preclinical?
I am not Updated on what has been done and not has been done in that research program, to be honest. So this is under Sorgset who is managing that program. But it goes according to plan. But exactly what has been done so far, I don't know, to be honest.
Alex have said that they would like to move on with this technology and may go to Clinical next year, do you think that's possible that, that will be together with your technology?
Well, it's possible. Whether that will happen, I don't know. But it is possible.
The CEO of Pollak have also said that they would like to extend the cooperation Into cancer and MMR vaccines, are you discussing this Possible extensive with all ex C phase?
This is something that we are We will announce when and if that extension is done. But there is a clear wish from Oleg's to also broaden this into further areas. That is correct. And that was I think his quote in the in our common in our joint press release that we had when we established this collaboration.
You had some very good results with AstraZeneca and the cooperation we had together with Demner. For the time being, you have 5 other corporations. Do you have encouraging results with any of these 5 companies that you can say a little bit about?
I can't talk too much about the different collaborations here because it's under confidentiality all of these research collaborations. But I can But I can tell you that we have encouraging results in some of these collaborations clearly.
Good. Okay. You have worked the return now for I think it's more than 5 years now. I would think that you have worked that long that You have some interesting results with them. Otherwise, I would think you have terminated that Corporations, can you confirm that you have some encouraging results with Eterna?
I'm not going into specific results on any collaboration. But I can tell you that we've had a long collaboration with Aethurna, but it's been a roller coaster thing because of management changes and Things happening strategy changes happening within Atherna during this period. Been an effective progressed collaboration through all of those years.
You took over the my guest, Corporation Derberas was saying earlier this year here. Can you say a little bit about how you are promoting this technology here now?
Sorry, I missed the start of your question.
Okay. You took over the right, I guess, for the work you had together with us for Henry, January 1st this year. Can you say a little bit about how you promote, I guess, this technology towards other companies?
Yes, this is done through the regular channels that are being used for discussions around business Development and Research. So it's partly sharing the research and doing it at scientific conferences as we did at R and I conference. It's partly at partnering and business development conferences where we meet with companies who have these kind of nucleic acid therapeutics that might be have a benefit from our technology. We are Also actively looking at where we best can use our technology and trying to shape up the applications within this area to have an even better concept to sell when we go out.
The results that we know with our opinion was very good. Can you say a little bit about now the response So that you have got for the interest you have got for this technology from other companies. Are there clearly interest among other companies to cooperate with you?
Yes, there is a clear interest among companies to collaborate with us. But it needs to be an application that fit what they're doing. But when there is, there is clearly they see the strong results that we have. And we've had very positive effects out of the results that we had with the AstraZeneca collaboration?
A couple of bold statement that you have done in the past. I guess in the annual report from 2020, it stated that FEMA market is well positioned to Capture a significant part of the nucleic acid therapeutic delivery market. And you have also said a couple of times that PimaMab could be the new gold standard Within parts of the nucleic acid therapies, do you still believe that this is likely to That, you know, Fibonacci is going to be a significant player in the future?
In applications, this is absolutely a potential because it is a very efficient way of delivering and spiritually restricted delivery in certain applications. So I do absolutely think that it can play a very important role to cover some unmet medical need with regards to gene therapies or nucleic acid therapeutics in specific areas.
The stock price has been quite disappointed lately. A lot of people are quite frustrated and angry about how the So perhaps actually well off. We have talked a lot about the technology and answered some questions so far today here. Is there anything else you would like to add to those People are feeling a little bit on the fence. They're not sure about what to do based on, I guess, what you have said today And what has happened in the past?
I just would like to reiterate that I think we have some very good results for FEMA Chem, Early phase results, we've been unfortunate with the pandemic that has really made an impact on the progress of our study. But we have strong faith in that these results that we saw that were quite encouraging brings the potential to the further development of FemaChem for bile duct cancer. There's no doubt about that in my mind. Now it will take some time. As you know, pivotal studies are not something you do overnight.
It is a long endeavor. But we have an interim read here and we have a possibility to do it on just the single study with an interim read. So I think that's a great opportunity. In addition to that, FIMA WACC is some very strong results in Phase 1, as you've seen, and it's been published now. And we have been a bit restricted lately due to in the progression of that program due to the all the focus the organization needed to do on our lead program.
But we have now strengthened the organization and I think we can hopefully provide more progress in that area as well. And for FIMA NAK clearly just repeating what you said. There are Very strong results with our technology and for certain applications within the delivery of nucleic acid therapeutics, it has some real advantages. And this is what we need to try to exploit.
Okay. Thank you, Per.
Thank you, Sveinrich.
Thank you. And as there are no further questions, I
will hand the word back to the speakers.
Thank you. Do we have some questions from the web that has not been covered through all the questions that Svein Erik Ask I think we need a microphone here.
Okay, okay. Yes. We have some questions from the web and I will start here with the release. Can you please explain a bit around early signs suggest a somewhat lower retention in the release control arm And the company is proactively pursuing strategies to address this during second half 'twenty one.
Yeah. So One of the risks that we have mentioned and the reason why we went to the authorities to start with to ask to do a single arm study was that this is not a study that can be blinded. It has to be an open label study. Open label studies means that the patient knows So whether they come into the control arm or into the experimental arm, excuse me. And There is a risk that is in all studies, in all clinical trial studies, you need to watch your attention to see that you don't lose too many patients.
Now there's a higher risk of course, it's in an open label study when patients are randomized to control arm, they know that they are randomized to control arm. They might go on to look for other therapies. It's also sometimes that continuing in the study requires travel because they go to a central Hospital. And they live maybe they have to travel a couple of hours to get to that central hospital. And they might have to have a local hospital that can provide the same treatment that they get in the control arm.
Now specifically under a pandemic, When you are under this kind of treatment, you don't want to travel. So it even heightens sort of the risk for the retention during a pandemic. Now we have seen a slightly higher level of discontinuation from the control arm. And this is something that it's far too early to say whether this is how big this risk is. But we want to proactively manage this risk.
So we are looking at All sorts of things from doing operationally into making sure that you can be compensated for travel, that are there opportunities to decentralize? Is there something with the design of the study we can do? And all of these things are we have now proactively looking into to see what's the best strategy to ensure that we mitigate this retention risk as early and as good as possible.
Good. Thank you. And regarding recruitment, How many must be recruited per month to reach interim read in second half 'twenty three?
12, On average?
Per quarter, you mentioned.
Per quarter. You asked per month.
Yes. I didn't ask it.
Oh, yeah. Okay. I thought they asked the question was per quarter. So divide by 3 and you get per month 4. On average, it's a fluctuating I mean, it's an orphan disease, So it fluctuates, but a bit but on average 4 per month or 12 per quarter.
Are there any plans to open sites in Thailand since Asia is clearly a positive Market.
Yes. We are looking into Thailand as well. It's not that easy actually in that country. And it's a slightly different kind of disease because it's highly infected areas there due to this liver fluke that can give bile duct cancer. And the etiology might be slightly different.
So you have to be careful so you don't sort of tend to study in a way that it's not valid any longer for the normal chronic carcinoma that we have in the U. S. And Europe. But we are looking into opportunities into Thailand as well. But it's not as exciting as it might have as it might sound in when you hear the high numbers because the high numbers there in incidence is really related to a slightly different disease.
And I received a message here that there is one more question from the call. Is it possible To hear if there are any more questions on the call.
Yes. Thank you. We do have a question from the line of Adam Carlson from ABG Sundercoglje.
Please go ahead. Your line will now be unmuted.
Hi. Thank you for taking my questions. I I wanted to ask a bit more around the signs of poor retention and the control arm that you were just mentioning. And you obviously described what the potential cause of that could be. I was wondering if you could say anything more about Kind of the implications for the interoperability of the study and whether at the rates that you're seeing now, If there's concerns that that could have kind of a significant impact or whether things would need to get quite a bit worse for that to be a potential issue.
So I think if the rate that we've seen during the pandemic is the rate that we will see going forward then it is a concern and we need to proactively manage it. But there are so there are ways of managing this of course. And there are a number of different actions you can put in place to try to manage it. The sort of the if you don't manage to increase retention or sort of keep it down to a level where you need it to be. Then the other thing you could do is increase the sample size, of course.
But that's not the way we want to go, of course.
Sure. Okay. And can you say anything about whether patients are Leaving the control arm in order to become eligible for kind of just standard photodynamic Therapy, is that a potential pull factor for patients leaving the trial?
Not that I've heard of. Not that I've heard of. I know that there are some patients leaving the control arm, but I don't really have the reason for why they've done this. So not sure.
Okay. And it's I don't know if the numbers are enough to break it down in any way, but is there a geographical component To the for retention, is it being seen more in certain geographies?
I don't have the figures of that in I had actually so I can't answer that question. But I don't think there is Well, I'll let's not answer that question because I don't know. So I'll have to come back to that, Adam, if that's something you want to hear more about.
Sure. Sure. That's fine. And I wanted to ask whether there was whether you've communicated this previously, if there's a specific number of U. S.
Patients That you're aiming for that or that you need for regulatory reasons to recruit to the trial?
There isn't a number of Patients you need to recruit from regulatory reasons. I think the more patients you have or that you have a substantial cohort within is always a positive. It's not a negative when you come to the FDA. But the There's not a specific number. And there are instances of course when FDA has approved drugs without a single American patients.
And As long as you can justify that there is no difference in ethnicity or etiology or therapeutic way of handling the The seas between the regions where you've done the study and the U. S.
Okay, great. And I was wondering if you could give any more information on what the kind of specific activities for the FIMA vac and In that program that you would have hoped to pursue if resources that they need to be focused primarily or entirely on the release study, Are there specific activities that you kind of have on the agenda as such? Or is it a more general sense?
Now there are, of course, plans here. And for Fimavak, it's really to move this into a disease setting. And we have internal plans for how this can be done. We have also discussions with external partners for whether we can do this in some kind of a partnership. So moving it into a proof of concept in a disease setting is a high priority for FIMA WACC.
And preferably of course in an area where we think it's really commercially interesting to move it to the next level. And then for FIMA NAK, it is what I mentioned. It's really to develop the applications Our own applications for having a more readily sellable concept to the companies. This is the application you do for this type of organ or this type of treatment and this is the application you do for that. It's been really for a long period more of an opportunistic approach where we have this technology.
We've presented it to companies. And then they if they're interested, they can start testing it. Now we're looking at whether we can develop this a bit further ourselves internally through internal disease and actively focus it to the where we think from the results partly that we had with the AstraZeneca collaboration actually which is very interesting where we have the most potential to bring value with our technology.
Okay, great. And just a final question maybe to understand the dynamics Of the poor recruitment during the pandemic, obviously, that's not unique to yourself, affecting Most trials or many trials anyway. But given the kind of the severity of the disease and so on, patients are obviously Still receiving treatment one way or another, whether that's in the trial or not. And obviously, you were mentioning travel inconvenience to go to a central Hospital as a potential reasons why patients wouldn't want to enroll and so on. But Is it your kind of assessment that patients that a significant proportion of patients Who would have enrolled but didn't because of the pandemic.
Are they receiving photodynamic therapy of some kind? Or is this kind of the route for them to potentially receive that? That would be interesting to understand.
I don't have I mean, it's sort of a version of the same question you had, another version of the same question you had before. It's an interesting question. I think it depends on which region you look at. If you look in certain Regions where photodynamic treatments of other kinds might be used by physicians, this might be an effect in certain regions. Now generally I don't think that is the case.
So I do not think that this is a big factor that they will go to a specific other photodynamic treatment.
Okay. No, because I'm just trying to understand If these patients would be receiving similar treatment, whether in a trial or outside of a trial, Whether the pandemic is then a primary reason for them not to enroll, if they're still going to go to hospital to receive photodynamic therapy, And why wouldn't they just as well do that as part of the trial, notwithstanding the traveling convenience and other things? But I understand what you're saying in terms of that being a geographical variance.
Yes.
Okay?
Okay. Great. Thank you very much.
Thanks, Adam. I think with this, we say
There is one
question. One last question.
One last question here is, Are you now changing the communication policy for release?
No. We're not changing the communication policy for release. The communication policy for release is that we talk about timelines and special events and countries and sites where we enroll patients. We talk about the milestones, whether we are on plan or not to reach certain milestones. And the reason that we're going out with some numbers right now is to give you an understanding of the challenges that we've had and the challenges that we see going forward.
It's to provide a more transparent scene on where we are and how the pandemic have affected us. But we will keep on the same kind of communication policy for release study. That is the normal industry standard going forward. Okay. With that, many thanks to all of you listening in for your attention.