Phio Pharmaceuticals Corp. (PHIO) September 2026 Conference Transcript

for joining the next session at the 2026 H.C. Wainwright Annual Global Investment Conference. My name is Matt Keller. I'm a VP in the Equity Research Department. At this point, I'd like to welcome our next presenting company, Fiio Pharmaceuticals, and presenting for the company is Robert Bitterman, President and CEO. Robert, please go ahead.

Thank you, Matt. It's a pleasure, and thank you for giving me the opportunity to present today. So Fiio is an immuno-oncology company. It's focused specifically on a certain type of skin cancer called cutaneous squamous cell carcinoma. We address this with our Intucil technology. It is a broadly patented technology platform that effectively allows us to make our immune cells more effective at killing cancer cells. So as I quickly flash up our forward-looking statement, let me suggest that what I'd like to accomplish over the next few minutes is to introduce you to this disease state, also tell you about how our technology is addressing it, and then talk about the comments or the results that we have received from our recently completed Phase 1b clinical trial and how we're going to move forward from that point going forward. So, with that, let me introduce you to cutaneous—oops, wrong slide okay here we go don't tell me that this is not going to click here we go so let me introduce you to cutaneous squamous cell carcinoma it is the second largest incidence of solid tumors in the human body and the incidence is increasing because the population is aging and as we age our immune systems become a little bit weaker as well it is a condition that is result of prolonged exposure to UV radiation. And it is also a condition that is recurring. If you have one squamous cell, it is likely that you're going to have more. And the greater the immune burden that you have, the more likelihood that these will occur more frequently and at an earlier age. Typically, if left untreated at an early stage, they will advance to later stages and lead to death. And also, it's known that the mortality rate on cutaneous squamous cell is almost twice that of melanoma. So, when one is considering the surgical intervention for this condition, one has to look at the size of the lesion, the location of the lesion on the body, the patient's general health. All of these come into play. And it's not just merely the lesion itself but there is also the requirement to excise around the excision to reach the surgical margins and that can actually increase the surgical incision area by two to three fold with that we also have to look at where the lesion is that determines the risk profile you can have a one centimeter lesion that's near the eye it's much higher risk than a three centimeter lesion on the back. And also another factor that comes into play here are comorbidities associated with the patient. For example, does the patient have a previous compromise in their immune system, or are they diabetic, or are they on certain types of drugs? All of these things will factor the decision. And so as we look at this, and this is probably about a two centimeter lesion near the ear that you're looking at, if you think of anybody who has actually gone through this process, think of what happens if they have to go through this repeatedly year after year. And in so doing, we basically find ourselves with patients who are saying, how many times can I go through this? And what we're doing is looking for an alternative to surgery, if not a neoadjuvant tissue sparing therapy, something that will mitigate the size and the frequency of the excisions with the goal to basically reduce if not eliminate the tumor to minimize the surgical excision size and the related disfigurement because you're going to have hypopigmentation as well as scarring and to hopefully reduce the frequency that will be required we're looking to preserve the integrity of the skin to promote faster healing and recovery time and also even to decrease the need for reconstructive or plastic surgery and in the end all this really comes down to the importance of alleviating surgical fatigue, a condition which those who have to go through this annually or sometimes even more frequently are confronted with the agony of having to have another hole cut into their skin and the recovery time that goes along with it. So having said that, what is our approach to treating this? And our approach deals with our intercell technology. It's a short interfering RNA. It's a drug. And how does it work? It selectively silences the signal from a designated gene, a gene that is ultimately producing a protein that in the presence of these squamous cells causes our immune cells to become inert. So they become ineffective. And by suppressing this gene, by cutting down that signal, we allow the body's immune cells to reactivate. And when they reactivate, they basically go about their job of attacking the tumors and killing the tumor cells in the process. This is only a temporary shutdown. It's not a permanent one when it comes to turning off the signal. This is a picture of how this looks like, what it looks like in the tumor microenvironment. We're injecting 7-6-2 from the upper left-hand side of the screen into the tumor through the cell membrane, and it goes directly and taken up into the T cell, where it basically takes about five minutes in order for that to do that. Once in the T cell, it starts to knock down or down regulate the PD-1 protein that's in there. And as that gets knocked down, the T cell regains its health. It invigorates. It attacks the tumor cell, which you see at the lower end of the bottom of the screen. But it does one other thing as well that's important. It increases the release of interferon gamma, a cytokine, which does two things. Interferon gamma also attacks tumor cells. And also importantly, it attracts new healthy T cells, T cells of different varieties, including memory stem cell T cells. And these memory cells implant themselves into the tissue and they stay there for years. And it's believed, theoretically, that the presence of these memory cells can prevent the recurrence of future lesions in the spot where the first one was removed. That's basically the flow of the activity. So what does it look like? What is our drug? It really, it is, the chemistry structure is an asymmetric duplex. It consists of two strands of synthetic RNA fragments that are very carefully sequenced or designed to target only the gene that we want. There's 20,000 working genes in the body. We're only going to basically turn off the signal of one of them, which is producing this PD-1 protein. So the programming and the engineering is critical. it basically eliminates the risk of off-target complications. The other thing that's special about this technology is that in order to cross the cell membrane, we basically are able to do so with nothing other than sterile saline. Most drugs have a difficult time crossing the barrier, and they have delivery enhancements that are required, such as lipid nanoparticles or viral vectors. We don't need those. We can basically get the drug into any cell or tissue where it needs to go through the concept of endocytosis. And finally, the drug also has phosphothyroids, which are basically stability protectants that allow for the drug to do what it has to do in terms of turning off that signal until the job is done. So it is through these different characteristics that the drug works in people, and also it leads to very extensive and deep and broad patent protection. The core in the center of that bullseye represents the picture that I just showed you, the sequence of the nucleotides. But we also are able to patent the sequences of the various nucleotides, which in turn form compounds, which are also patented. And then finally, we also have method and use application patents around that as well. So you can see in the terms of the patent world, we have a number of picket fences, or in this case is concentric circles protecting the core, and also going out, and even in combination use when we have patents, and a few other elements of this as well. What this all leads to is 54 issued patents layered out through 2044, and with uh additional pending patents in certain key countries around the world so what is our oops what is our strategy with regard to uh the the development programs that we have we have two tiers to our strategy one is to basically develop internally our lead program ph762 and the other is a backup program which we'll talk about briefly later in the presentation But beyond the development strategy, we also look to monetize our non-strategic portions of the portfolio via outlicense. And we'll talk about that briefly in a few minutes. But the two focused development programs, first is the PH762, which silences PD-1. That completed its phase 1b clinical stage back in March of this year. And we're now in the final stages of preparing for the phase 2 study, which will start sometime after the first of the year. Our second program is PH894, which silences a different gene, the BRD4 gene. So let's talk about intratumoral therapy and the results of the phase 1b clinical summary, because this really has been a very effective guide in helping us to consider how to move forward into the next study. This study, which was completed in March, was a dose escalating trial. The purpose of which, as most phase one studies are, is to determine safety and tolerability of 7.62 as a neoadjuvant therapy prior to surgery. The phase one design included five dose escalating concentrations. There were four intratumoral injections given to the patient over three weeks with a resection of the residual lesion at week five. And the endpoints included safety, which were looking for adverse events, tumor pathology to determine if the levels of cancer were still present in the tissue, and the pharmacokinetics to determine what activity the drug had on the patient's plasma and blood levels. 22 patients completed the trial. 20 of them were cutaneous squamous cell, one Merkel cell, and one melanoma patient as well. The summary on the safety was as follows. There were no immune or treatment limiting toxicities in any patient. Those five-dose escalations revealed a safe 20-fold increase in drug concentration without any concerns in terms of the data. And there was no recurrence seen at the end of the study at day 113 for the CSCC patients. To what do we attribute that? the safety probably is attributable to the direct injection into the tumor and the precision targeting that essentially eliminates off-target serious adverse events, which is in contrast to the antibody therapy, which is systemically infused and centers throughout the entire body, often causing autoimmune response. The other factor is that given the absence of formulation enhancers that are typically needed for other drugs to get across the cell membrane, we can deliver pH762 into the desired target through sterile buffered saline. This further mitigates the risk of any types of other local toxicities as well. On the efficacy front, in the fifth cohort, we had an 85% pathological response in that first study. The six of seven patients responded, four of the six had 100% pathologic clearance, one had greater than 90 percent, one had greater than 50 percent. And if we look across all five cohorts that include those which had varying degrees of concentration of active drug, there was still a 65 percent weighted average across the entire cohorts. So the response to this was quite impressive. And with that, gave us the encouragement to move forward into our next study. A couple of other factors that are relevant about this particular drug in the program. There's a lot of convenience associated with this. First, the drug is administered in the doctor's office, so it avoids the logistics of systemic infusion centers. It also minimizes post-surgical recovery. If we're shrinking the size of the tumor or possibly eliminating it totally, the recovery time becomes much more attractive for the patient and for the managing physician as well. The drug is refrigeration-stable for two years, and it has flexible dosing to accommodate various lesion sizes depending on what is presented with a given patient. There's also some interesting economic factors associated with the program. first office visits in the form of four injections and follow-ups they drive the physician practice economics so it's good for the doctor with regard to capital outlays unlike radiation therapy there's really no capital outlay for the physician to uh to put out to do this therapy in fact the therapy is pretty simple the injection can be done within one to two minutes for the lesion and lastly from the standpoint of the active pharmaceutical ingredient as well as the drug product they are sourced in the u.s so that eliminates any flexible not flexible but any adverse changes in the tariff regulations that seem to change periodically so we have a pretty much a fixed cost on that factor but what are our next steps at this point well we're currently in the final stages of the design of the protocol for the Phase IIb study, which is to undertake itself or to start in the first quarter of 27. FDA feedback and responses are targeted now for the third quarter of this year. Meanwhile, we have recently commenced the commercial scale production of API, which will put us in a position to meet any type of robust standards for active pharmaceutical ingredient that the FDA might impose on us. And that should be completed, that first batch, by the end of September. That batch, in turn, will be sent to our drug product manufacturer, who will produce drug product that is scheduled for release in the fourth quarter to support the clinical trial that will take place in Q1. And finally, in the midst of all this, we're in the process of completing a non-clinical toxicology study of 90-day duration in order to support any types of further lengthy trials that might be considered. And while that's not probably going to be a factor for us, in the world of our out-licensing and looking at this for other potential indications, it would be certainly useful and support further studies with the FDA. So a quick comment on the other program, the 894. 894 silences the BRD4 gene. It has implicated numerous different potential cancers, such as melanoma, breast, cervical, lung, liver, etc., head and neck. It has a dual mode of action. It works a little differently than 762. It activates immune cells, but it also directly kills tumor cells at the same time. program. 894 is precisely selective just for the BRD4 gene, only that. So it eliminates any toxicities associated with previously studied non-selective development compounds. It has a clean profile as evidenced in a non-human primate study. And 894 has completed most all of the IND enabling studies. So we will undertake to move that to an IND stage once we launch the 7-6-2 program sometime in the first quarter of next year. So we talked about maximizing or monetizing the value of our non-strategic portfolio. The intercell portfolio is extremely versatile, and we have a number of compounds and ways of applying it. Different therapeutic applications as well as different geographies throughout license. And one of the ways of monetizing it is just through 762 alone, looking at other cancers as well as other geographies. The PD-1 protein is present in a number of other cancers such as head and neck, breast, liver, and Merkel cell. And so it has the potential for application in those diseases. We've also shown that it can be useful in adoptive cell therapy for someone who might want to lock the 7-6-2 campaign into a special form of TILS, which we have done in the past, years ago. And also, there's the opportunity for potential combination with antibody therapy as well. On PH894, there's also other cancers and other geographies that are covered with our patent protection and with that we can look at advanced cutaneous squamous cell melanoma non-small cell lung cancer ovarian cervical and breast so in addition to the two main compounds there are other intercell sequences and there's a number of them and on this slide from the right you can see these are the various sequences which we have confirmed silencing on and which are patented And they target solid tumors and oncology, hematologic cancers, as well as different types of proliferative infectious diseases, such as HPV, HSV, and HCV. So, all of these are far beyond the bandwidth of what FIO is capable of handling, both in terms of our capital sources, as well as our personnel. But many of these may be of interest to other parties who have of interest in these Southern specific cancers and diseases. So with that, just to conclude here with some financial metrics, we have cash on hand that will extend through the first half of 2027 and beyond. Our liquidity in terms of cash at the end of June, as we just reported in our 10Q was $13 million. We have an ATM capacity of 6.3 million, which has not been tapped. and we also have the exercisable value of common warrants that are fixed at a price of two dollars and five cents that's worth about 25 million dollars in terms of our cap structure there are 11.6 million shares outstanding the fixed strike price warrants are expiring in december of 27 there's 13.4 million of those and we carry no debt on our balance sheet and so with that But I will conclude by saying that we believe that we're very strategically positioned with Intersil and this precision silencing technology. We've established a phase 1b study that demonstrates safety and efficacy for the 7.6.2 compound. we're looking at a market size which is very significant in cutaneous squamous cell carcinoma we have extensive broad and deep intellectual property to protect the the technology out through the years 2044 in a very experienced and a lead lean management infrastructure which means that we're directing all of our monies all that we can back into development and into additional patent protection. And so with that, I leave you with Fiopharmaceuticals, a company that is finding out how to make immune cells more effective in killing cancer cells and doing so effectively in our first study. So thank you very much.

Great. Thank you, Robert. Fantastic presentation. On behalf of HCU Reinhardt, I want to thank FiO and all of our presenters. They'll be participating at our conference this year. thank you again we appreciate the time and effort it takes in participating appreciate it thank you again Robert thank you Matt