This conference call transcript was computer generated and almost certianly contains errors. This transcript is provided for information purposes only.EarningsCall, LLC makes no representation about the accuracy of the aforementioned transcript, and you are cautioned not to place undue reliance on the information provided by the transcript.
spk05: Good morning and welcome to the Precision First Quarter 2023 Financial Results and Business Update Call. All participants will be in the listen-only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press star, then one on your telephone keypad. To withdraw your question, please press star, then two. Please note, this event is being recorded. I would now like to turn the conference over to Steve Harrison, Vice President of Investor Relations. Please go ahead.
spk03: Thank you, Ryan, and thank you to everyone joining us this morning. With me today are Dr. Helen Sabzabari, President and CEO of Precision, as well as Harry Tomasi, our CFO. Helen will provide an update on the progress we have made across our pipeline programs and highlight our upcoming milestones, after which Harry will review our first quarter 2023 financial results. Following our prepared remarks, we will open the call to Q&A. Before we begin, I'd like to briefly review our forward-looking statements. During today's call, we will make various forward-looking statements. Investors are cautioned that our forward-looking statements are based on current expectations and are subject to risks and uncertainties that could cause actual results or outcomes to differ from those indicated by our forward-looking statements. Please read the Safe Harbor Statement, as well as risk factors contained in most recent SEC filings for a more complete discussion of the risks and uncertainties. I would now like to turn the call over to Dr. Savzavari. Helen?
spk08: Thanks, Steve, and thanks to all of you for joining us today. Before I provide an update on our lead clinical programs, I want to emphasize the four clinical principles that are the foundation of our approach to innovating therapies that have the potential to transform patient care and outcomes. First, we focus on indications that have high unmet need and for which new therapeutic approaches are urgently needed. Second, we strive to develop therapies that are differentiated not only by the clinical benefit they provide, but also by their potentially disruptive prices. A key limitation of many recent breakthrough therapies, especially those in the cell and gene therapy spaces, is pricing that can create significant barriers to access and impose substantial financial burdens on health systems and payers. Thus, we are committed to innovating not only new treatment modalities but also novel modes of manufacture and delivery design to enable more cost-effective pricing. Third, we pursue programs that we believe have potentially rapid paths to commercialization. This approach allows us to address unmet patient needs as rapidly as possible and moves us more quickly to generating product revenues that will build long-term value for our shareholders and sustain our ability to advance and expand our pipeline. And finally, we are committed to executing a cost-effective R&D model and maintaining a fiscal discipline. As Harry will review later in the call, we currently have capital runway into late 2024. which we anticipate would allow us to continue demonstrating important progress across our clinical development portfolio. Now, I'd like to provide you with highlights of our portfolio and capture what we have accomplished in the first quarter of this year. The first platform that I will be discussing is our Ad Universe platform. And I'm sure many of our audience are familiar with this platform. This is a unique and differentiated platform that uses gorilla adenoviruses. We have a full IP around this. And basically, these unique gorilla vectors allow, first of all, that you... introduce a large payload capacity, meaning you can put many genes up to 12 kB, many epitopes that can be combined, and these vectors can deliver. Secondly, and very importantly as far as differentiation is concerned with other platforms such as AT5, retroviruses, or lentiviruses, is the ability of complete redosing And as a result of that, enhancing the immune system. I highly recommend if you have not seen the video of this platform, please go to our website and take a look at this. The way this platform works is actually educate your own immune system directly from within And by giving these therapeutic drugs, you can enhance the T cell responses while you are keeping the neutralizing antibodies at bay, which makes this platform very unique and differentiated from all others. By utilization of this platform, what we have done is brought two drug products. The first one that I will be addressing is our PRGN 2012. In the January of this year, we showed a complete phase one and the expansion data for our PRG in 2012. PRG in 2012, it's a gorilla adenovirus that contains epitope, educating the immune system to HPV 6 and 11, which is the cause of basically generation of papilloma in a patient population which we refer to as recurrent respiratory papilloma disease in this patient. These patients, they continuously have the regeneration of this papilloma in their vocal cords and also in trachea. There is currently no therapies for this rare disease. And the only thing that can be done for this patient population is repeated surgery. And some of these patients acquired hundreds of surgeries over their lifetime. The way that PRGN 2012 works is a vaccination with the course of four vaccination that with the data that we presented, first of all, is a very favorable safety data We have not seen any kind of DLTs and nothing more than grade one and two, very similar to flu-like symptoms when they receive it subcutaneously. But at the same token, what we have shown in the data presentation in our R&D day on January was in the severe patient population, 50% complete responses after 12 months of follow-up. These patients did not require any surgery in 12 months follow-up, whereas some of them had up to seven or eight surgeries per year. At the same token, a very favorable safety profile, and in conjunction, we showed a mechanism of action data that after the vaccinations, how you have enhanced the T cells that corresponds exactly to this response rate. So, we are very excited about this. As we have communicated, we are in very productive discussions with the FDA. And this is continuing, and we are committed to, in the upcoming months, share the results. But at the same token, I like to highlight, in the recent months, I'm sure our shareholders have seen the communications that is coming from FDA, especially in regard to the rare diseases, which RRP is one. and the new guidances that are given and the attention of FDA for coming for fast solutions and approvals for these drugs. So we are very, very excited, and we are looking forward to communications in the upcoming months with our shareholders in regard to PRGEN 2012. Another drug product that is in the clinical trials currently using the same Adenovir platform is our PRGN-2009. The beginning of this year, we communicated that we would be sharing the data on the full phase one and combination cohorts with the checkpoint inhibitors with PRGN-2009. in HPV-related cancer patients. PR Gen 2009 targets HPV 16 and 18, which is the major cause of HPV-related cancer indications such as cervical cancer, head and neck, anal, and others. The totally 5% of all cancers in the world is actually HPV-related. Huge patient population in need and a huge market for addressing this. And if you look at what currently is available to this patient at cervical cancer, 15% response rate on the secondary lines is the best that checkpoint inhibitors have achieved on a head and neck is 18%. So you can understand the need for coming up with the innovative therapies, which we believe PRGN 2009 is one. And we are really excited about the data that will be presented in a stage four patient population. This is all comers, both cervical cancer, head and neck cancers, in the data that will be presented. And one thing that I should stress, these are patients that they have failed all other therapies, including the checkpoint inhibitors. And now the data that will be presented at ASCO will not only cover the safety and the dose response, but also the preliminary clinical efficacy that we are excited and our investigators will be sharing that at ASCO. So I would encourage you to look for that At the same token, what we have done is position PRG in 2009 and move it into frontline therapy in a neoadjuvant setting. We all know how important it is for these therapies to move to the frontlines. And in our phase two study that is currently ongoing, we have two arms in a neoadjuvant head and neck therapy that PRGN 2009 is actually positioned in front of the standard of care, and this is very exciting. We have finished the monotherapy arm of this, and I'm excited to tell you that the combination therapy with Keturda will be starting in the upcoming months. and that will address the patient population that basically will receive our drug products prior to their standard of care, and then they will be followed up, and especially for the enhancement of immune responses, which is very, very important in this setting. So we will be reporting at the interim data by the end of this year, as we have promised. on PRGN 2009, and I think this is quite an exciting program that addresses not only the latest stages, but also it addresses the early onset of the disease. So with that, I'd like to move to our Ultracar platform and give you an overview of what we have done. As you all know, Our Ultracar platform is unique and differentiated from all other classical cars, TCRs, in the fact that you actually can modify autologous T cells of the patient overnight. And this is, we believe, it's the only truly overnight platform that you modify the T cells of the patient and the next day you infuse them back. We currently have actually three clinical trials that are ongoing. The first one is our PRGN-3006, which this is the ultracard that addresses in the AML patient population, targets CD33. It includes membrane-bound IL-15, as well as the kill switch. And this is in a patient population that truly there is not anything left. These patients have failed all other therapies with a very, very limited time, which conventional CAR-Ts or other cell and gene therapy will not even have enough time for the manufacturing for these patients. At ASH, we reported not only on the safety, a very favorable safety of our ultra-CAR-T therapy, but also the preliminary data from our phase one and the expansion of that arm that it almost showed 30% objective responses in patients that they have failed all the other lines prior. And we showed not only are the T-Ulfacar can expand and persist in this patient, but can be effective against the tumors in the patients. Currently, on the multiple sites, we are expanding to a phase 1b. The FDA has not only given us the unmet, the orphan drug disease designation, but also a fast track designation, and also the ability to re-dose now in this patient. And why is that important? Because we believe this is the only UltraCard platform that in a cost-effective manner, overnight manufacturing, you can re-dose the patients as they needed over the period of time with keeping the cost at bay. And this is very, very important for the field of cell and gene therapy. As we have promised, we will be presenting data in 2024 on our Phase 1B expansion cohort, and we look forward to that. In regard to our next ultracar platform is our PR Gen 3005, which directly targets March 16 on ovarian cancer. It also includes membrane-bound IL-15 and a KEL switch. And as you recall, we had two arms in this trial, both intraperitoneal and IV, and FDA also allowed us to open the lymphodepletion arm. We have finished the dosing and all. The data, as promised, in early January will be presented at ASCO, and we are excited about this because not only we show The mechanism of action of these, that they can, these ultracars can expand, persist in these patients, but also a preliminary efficacy data. And with that, we also have moved to Phase 1B expansion, which that data will be presented in 2024. But also, one other aspect that is very important, based on the data that will be presented at ASCO, we have added an additional arm that would be a split dosing in expansion cohort, that it will Patients will receive both IV and IP based on the safety and the efficacy that you will see at ASCO. And this is going to be very, very exciting. And in the upcoming months, we will start this arm as well in our expansion phase. And we will be reporting on that in 2024. So please make sure that you go to the ASCO poster and take a look at the data that will be presented by our investigators. The third UltraCar T that I would like to touch base on is our PRGN 3007. And PRGN 3007, it's what we refer to as the next generation of our cars. What do we mean by that? It's simple. It has become evident that CARs in general, and now a lot of people are moving in conjunction with combination with other therapies, especially checkpoint inhibitor. And you can imagine, from that perspective, you're adding the systemic toxicity of a checkpoint inhibitor that has to be given, but also the cost of that, which already is quite tremendous when you use a classical CAR-T. In our next generation setup, what we have done is design our ultracard that not only expresses the card of interest, in this case, role one, which is expressed on both hematological and solid tumors, but also it has membrane-bound IL-15, heel switch, and an intrinsic mechanism that expresses or down-regulates the checkpoint inhibitor. So, it basically makes it irrelevant for the use of systemic checkpoint inhibitors and obviously the cost of that. We are excited and we have, as you have seen with the press release, the first patient was dosed in this quarter and the patients are being enrolled as we speak in this trial. We will be having an interim data presented by the end of this year as we have put that as part of our goals. And one of the most important parts of this with PRGN-3007, as I mentioned, not only it will be addressing hematological diseases such as CLL, BCL, as well as the solid tumors such as triple negative breast cancer. And this is an umbrella trial. And this has brought us one step closer to our vision of having a library of ultracars that can be basically made for various indications overnight as it's needed for the patient. And finally, what I like to stress after our PRGN-3007 is regaining the rights for CD19 and BCMA. We are very excited about that for various reasons. Number one, these are validated targets that already have shown that they can be very effective in these indications. At the same token, now we have shown that our platform of ultra-car, ultra-parader platform, now we have validated this across various indications, both in hematological and solid tumors. And the combination of these two, we believe, will offer the change, the paradigm for patients, for treatment, for the cost, for the ability to be redosed. And this is quite exciting with having both under the same roof. And our teams at Precision has been moving forward rapidly, and our aim is to be phase one ready by the end of this year for CD19, and then we will address also BCMA. So, from that perspective, we are excited that we can take these drug products to the market. The most important thing is we are aware that there are approved drugs currently and classical CAR-Ts out there. These are the first in the class, but sometimes being the best in the class, that's what is important not only for the patient, but also as far as taking the portion of the market. And we believe between our platform and the validity of the targets, we have a very good path forward to be best in the class. And with that, this is why we are excited and moving these programs very rapidly. At the same token, part of regaining the rights for our students CD19 and BCMA, we also regained the rights for IL-12 and especially our gorilla IL-12 back with a control switch. And why is that important? In the past decades, the role of IL-12 has always been considered as one of the best molecules for enhancement of immune responses and in combination. However, the biggest issue has always been the control for IL-12, and we are, we believe, the only company who has now the highest amount of clinical data with the control switch with IL-12. This makes it very unique and absolutely very appropriate for our portfolio, especially in combination with our current molecules for the future. Why I'm saying that? In the recent data that have been shown across various indications, but specifically head and neck, the role of IL-12 has become up and center. And now we have one of the most potent molecules under the same roof with our portfolio. We are excited about that, and we will be discussing this in an upcoming webinar. months and half of the year. So with that, I would like now to transfer to Harry to address our financial reports. Harry?
spk00: Thanks, Helen, and good morning to all of you on the call. I appreciate your participation in our quarterly update. During the first quarter, we've continued to make progress on strengthening our financial footing while containing costs to support our business objectives. I want to spend a few minutes updating you on the progress we continue to make from a financial perspective. I'll start with an update on our convertible notes. During the first quarter, we repurchased an additional $29.5 million face value of convertible notes at a discount to PAR. As of March 31st, the remaining balance of our convertible notes was $13.8 million, which will be paid at or before maturity on July 1st of this year. We will continue to utilize our restricted cash balance in the retirement of these securities. To date, we've retired $186.2 million face value of our original $200 million of convertible notes prior to maturity. which has saved the company close to $7 million. Secondly, when I started at Precision approximately a year and a half ago, we set a goal to reduce our G&A spend. Since that time, through a focus on efficiencies as well as settlement or progress towards settlement of older litigation matters, we believe that we have right-sized our G&A function costs. I am pleased to announce that our G&A expense has decreased by 15% in the first quarter of this year compared to the same period last year.
spk01: We believe that our first quarter G&A spend approximately the same as we anticipate for the remaining quarters this year.
spk00: You'll also see in our reported financial information our research and development costs have increased in comparison to the prior year quarter. With the reduction in interest in G&A costs, we have been able to redirect our capital towards our mission of drug discovery. We expect this trend to continue with further advancement of our product candidates. Turning to our cash burn, our net cash used in operations for the current quarter was $18.4 million versus $18.8 million in the year-ago quarter. Our cash burn was positively impacted by our reduced G&A and interest costs. These costs more than offset the positive cash flow in last year's first quarter of approximately $2 million from our previously owned Transova business prior to its sale in the third quarter of 2022. In addition, we were able to increase our R&D spend while still reducing our burn for the current quarter compared to the first quarter of 2022. In summary, our program of financial discipline, combined with our public equity offering in January and the early retirement of our debt, has provided a solid cash runway to support our priorities into late 2024. This is consistent with our previously provided guidance on our cash runway. I thank you for your support of Precision. We'll now be happy to take your questions. Ryan, if you could let questions come in from the queue, that'd be great.
spk05: Thank you. We will now begin the question and answer session. To ask a question, you may press star, then 1 on your telephone keypad. If you are using a speakerphone, please pick up your handset before pressing the keys. To withdraw your question, please press star, then two. At this time, we will pause momentarily to assemble our roster. Our first question comes from Jason Butler with JMP Securities. Please go ahead.
spk02: All right. Thanks for taking the questions and congrats on the progress. Two for me. The first one on PRGN 2009 is, Can you give us some more details on the data you're going to present at ASCO? Will we see data from all enrolled patients here or a subset, and how should we think about the duration of follow-up? And then secondly, for PRGN 3007, Helen, can you maybe just give us some color on the mechanistic rationale for role one in solid tumors? Thank you.
spk08: Thank you, Jason, for the questions. Absolutely. In regard to PR Gen 2009, there will be a full data presentation on the Phase 1 and also the expansion cohort with the checkpoint inhibitor that not only on the safety The mechanism of action and some of the scientific mechanism of action will be also presented and the efficacy. And to the point, I'm glad you brought that up. I think what is very exciting, not only there will be presentation on objective responses, which is unique. As I mentioned, this patient population in the best case scenarios, we have 15% response rate up to 18 in head and neck. And this is, by the way, there will be patients both cervical as well as head and neck in that cohort that will be presented. But also, we showed that in majority, a combination arm in patient population that they have failed the checkpoint inhibitor, and then when we add our PRGN-2009 to the checkpoint inhibitor in the same patient population that they have stopped responding to checkpoint inhibitors, then we will show their responses. And to the point that you raised, I think part of the other part of the excitement here is the durability of the responses which our investigators will be addressing and presenting on a patient basis. In regard to PRGN 3007, I think this is really an exciting target because role one is expressed on hematological as well as solid tumors. There is actually a range. that it covers and currently the way the trial is designed, you can have number of patients from various hematological settings such as CLL, BCL, ALL, and then at the same token, the triple negative. And especially the triple negative because this is an area of need for this patient. As you know, there are really truly no therapies out there for patients that they have failed everything. And no CAR, to my knowledge, really classical CAR T programs or any other therapies at this point for this patient. So it makes it unique. And the reason for the design is we know CARs, TCRs, T's, all of these, at the end of the day, they are T cells. Therefore, they will be subjected to the tumor microenvironment. And part of the issues of the tumor microenvironment is the inhibition of the T cell through the checkpoint inhibition. And as a result, that needs to be addressed. A lot of the off-the-shelf conventional cars, they are now entering and addressing that they need to combine with the checkpoint inhibitors. for a number of reasons, because first of all, they only have one shot. They cannot re-dose, so that's the issue. And secondly, they have to overcome these tumor microenvironment challenges. So with us, we have design and our ultra-vectors allows us to incorporate mechanisms that we can directly inhibit the checkpoint inhibitor Just within our ultracar T. Why is that important? Because there is no systemic toxicity or there is no signal to others. So it's just your own ultracar T that will stay active and You don't need now to add, for instance, Keturda any further. You get away from the systemic toxicity, but also, more importantly, from $150,000 price tag that has to be added to another $400,000 price tag of a conventional CAR T. So as a result of that, we think this is very unique, and the platform that we have allows the addition of this at the same token it makes the combination irrelevant. And that's why it's very exciting what is happening right now.
spk01: Great. Appreciate all the details there, Helen. Thank you. Thank you.
spk05: Our next question comes from Jennifer Kim from Canto Fitzgerald. Please go ahead.
spk04: Hey, everyone. Good morning and congrats on the quarter. I have a few questions here. Maybe to start with 3007, I think I heard in your comments that you plan to present some interim data this year. And I'm wondering, since it is an umbrella trial, is there anything in terms of what you'd flag and what you'd want to see in that initial look? Are there certain tumor types that you're interested in, solid tumor versus heme, et cetera? And then my second question is, I think you also mentioned for 3005, something on this, on pursuing split dosing. Could you go more into detail on what that entails and sort of the implications of that? Thanks.
spk08: Yes, absolutely. Thank you. So, in regards to the PRGM-3007, maybe I should just sort of make that more clear. Currently we are in a phase one and what we said that we will be perhaps by the end of the year have some view on the doses that will be completed, the full databases For a phase one and expansion cohort, we will be addressing in 2024. But the end of this year, we will give some color from perspective of how the phase one is going and some of maybe a sort of a preliminary database. So that's in regard to the PRGN 3007 as far as the data is going. Because we just started, and of course, we want to give more of a complete set of the data. But definitely we will be discussing views and how the trial is going on and some preliminary. We will not be finishing the phase one completely this year. This will be all presented by next year. In regard to the PRGN 3005 and the split dosing, This is very interesting because the data that will be presented at ASCO, and because of the embargoes, we cannot be going to the details right now that we have from ASCO, but this will be presented by our investigators. You will see a very unique mechanism for that both exist in when you give the intraperitoneal drug versus when you give the IV, and also from the perspective of when you add the lymphodepletion. And now, after observing, and this is why it was so important that we do two arms, and by the way, it really points out in regard to our Ultracar how expansion persistent works with and without lymphodepletion. Also, the route of infusion makes a difference, but also on top of that, what you see on the preliminary efficacy data. And as a result of that, Not only we have gone to the phase 1B already and we have the arms with the IV and lymphodepletion open, as you know, we also received the permission from FDA to redose. And that is going to be exciting data will be presented at ASCO in regard to that. But also... Now, based on the totality of the data, we can see that what else can be done. And the split dosing is actually, these are all stage four, by the way, patient population, ovarian cancer. So ovarian cancer, 10% response rate, and nothing has moved that needle for this patient population. Nothing in the upcoming, in the years. And with that, What we are doing is the same patient, because of the ability of our ultracar platform that you can make number of doses and even make a large enough dose that you can split, and a patient can receive a dose that is infused intraperitoneally because many of these patients, as you can imagine, the surgeons cannot remove the totality of the tumor that is within an intraperitoneal cavity. So you need to address that. But at the same token, you need to address the metastasis that is around the body. And so the IV dose, the intraperitoneal dose, the patient would receive this split dosing. And that's the design. And we are looking forward to this as this will be starting in the next few months. And we will be discussing that, which we are very excited about.
spk04: Okay, great. Thanks, Helen. Maybe one more question on the 2012 asset. Any updates on the timing of FDA discussions? And can you remind us on the possible outcomes and strategies that you're pursuing for 2012? Thanks.
spk08: Yeah, absolutely. So one thing, our PR Gen 2012, what I should mention is Really, the work that has been done has been outstanding because I just want to remind everyone, the phase one of this started in April of 2021. Not only we reported on the full phase one data and the expansion of those cohorts in January of 2023, but we had started the phase two basically clinical trial in 2022. And I want to stress, if by any chance I forgot, and it's in the press release today, that we have finished the Phase 2 enrollment, and now we are in a 12-month follow-up of those patients, which is very exciting. So you can see how rapidly our teams have moved, not only to get the Phase 1, and now have the 23 patients in a Phase 2, so the total 35 patients that have received the dose level two in a phase two, and we are really excited about that. In regard to the interactions with the FDA, we are in a continuous discussion, a very productive discussion. Of course, our communication with our shareholders and investors and outside has to be respected by the fact that finalization of the FDA giving us the complete answer. So we are in discussions. It's very productive and especially in a view that what Dr. Marks has been mentioning in the past few months in regard to new guidances and changes at OTAD and how the rare diseases especially are being treated for a faster, and more innovative guidances that allows safe and efficacious drug products to basically be advanced for the unmet needs of the patient. We are very encouraged by this. And it's exactly where our discussions are with the FDA. So as we have mentioned, we will be communicating As soon as we have the finalization of the strategy by the FDA, however, we have to respect the timelines of the FDA at this point and confidentiality of the discussion. So what I say is please stay tuned. I think it's going to be an exciting year for us. Awesome.
spk04: Thanks, Helen, and congrats again.
spk05: Thank you. Our next question comes from Ben Burnett from Steifel. Please go ahead.
spk07: Hello, good morning. This is Carolina Ibanez Ventoso for Ben Burnett. Congratulations on all the progress and thank you for taking our questions. Follow up on the PRGen 2012 program. I think, Marlon, you said in your prepared remarks that you plan to present additional results in a few months, if you could confirm that, and then what can we expect in terms of number of patients out of the 35 that you already have involved, and follow-up time in this update, and if you are going to wait to present results until you get a clear FDA feedback, or if we may see some data before that.
spk08: Thanks, Catalina, for the question. First of all, in regard to the PRG in 2012, there would be no further presentation of the data. We presented the full Phase I and the expansion cohort of the Phase I in January, in the totality of it, actually patient by patient. It's very solid safety, as we have shown, as well as a complete response, a 50% response. So the next, basically, data presentation, of course, will be in the context of the Phase 2 and when they have finished the full follow-up of that. As I mentioned, we are excited. We have finished the enrollment of the 23 patients in our phase two. And obviously, they are being followed from the first patient to the last patient. There will be a follow-up of 12 months complete. And the discussions that we have with the FDA obviously is... In the view of safety data, which as I mentioned, and our investigators have pointed out, this is the safety that has been shown is extremely favorable. It's almost like a flu shot, just some rash reaction at the side of the injection sub-Q and maybe a little bit of fever and aches for a day or two, but nothing more than that. and in view of efficacy data, which is very unique when you talk about not just reducing the number of surgeries, which in more than 83% of our patients that happened, but actually eliminating a requirement for a surgery in 50% of patients. And by the way, the uniqueness of the patient population that we have enrolled is that we have gone after the most severe patient population. These are patients that they have required, minimum required, three surgeries per year, and majority of them have had much more. So with that, from the perspective of the FDA, of course there is discussions that what does the pivotal role trial looks like or if what we are addressing currently can be considered that. The endpoints, which is, of course, ours, and we have made them extremely robust. We didn't go just after the number of reduction in the surgery, but we have gone to the endpoint of complete response with no requirement for surgery and for 12 months. So the durability is also very important. And, of course, a rapid path for the approval. These are the discussions that we are having with the FDA. And in a view that this is a rare disease, a patient population that they have no other options in front of them except continuous surgery, and also in view of current guidances that is coming out and changes that have been communicated by FDA leadership. We are excited and we are continuing these discussions. And of course, as I mentioned, again, the confidentiality, we need to respect the confidentiality and discussions that we have with the FDA. And as soon as we have a complete discussion A strategy approved, we will be communicating that to the outside.
spk07: Yeah, understood. Very helpful. If I may ask another follow-up question related to this program. So in light of all the results that you have accrued so far, what is the expectation for the need of a periodic, for example, an annual booster with this vaccine?
spk08: Yeah. So, excellent question. Currently, the way our trial has been is one course, which is for vaccination, and that's all these patients have received. Definitely, there will be a follow-on and trials that will address boosting because with the view of the safety that this platform and the PRGN 2012 has, definitely it can be imagined that in the patients that they didn't reduce completely the requirement for surgery, that will be an option for this patient population, definitely. And of course, another part of the strategy and path toward the commercialization will be also that we have to address the pediatric patient population, which currently That's not part of this trial, and that will be also in the future as we will be addressing that patient population and market. So for sure, those are within the works.
spk07: Okay, excellent. Thank you very much for all the callers.
spk01: Sure, of course.
spk05: Thank you. Our next question comes from the line of Otto Hee. from AC Wainwright. Please go ahead.
spk06: Good morning, Helen and Harry. This is Arthur Ong for RK. I have two questions. One is for the 305 and 306, the repeating dosing. Could you remind us how the repeating dosing can be done in terms of the judgment by the the physician or the patient needs. And I had a follow-up on that part as well.
spk08: Sure. Thanks, Arthur. So in regard to the repeat dosing, currently for the expansion phase is at the discretion of the physician. And why is that? First of all, in regard to 3006, we are dealing with a patient population that they have a few months, unfortunately, to live. And I think this is definitely we are getting a mixed patient population with a different requirement. We have had patients that they have came out of the hospice to receive this treatment. So you can imagine how sick the patients have been. So based on that and the need, the extent of the disease or the stability of the disease, the best... judge at this moment are their clinicians and their oncologists, that they can make that call instead of having a regimented that we say, well, every four months. Because a patient might not have four months in general and they might need a faster or a much more aggressive upfront. Then on the other hand, there might be patients that their diseases are stable. and then they might not require immediately, and this is why the patient. Or if the clinicians, they see early signs of progression, they can intervene accordingly. So that's why currently the Phase 1b, which is an investigational design, is to understand the course of the disease and the need, and also the amount of for instance, in various patients with this level of disease, what it requires. And the same thing goes for PRGN 3005 because, again, I'm going to stress one thing. Of course, the goal of all of the therapeutic drugs is eventually to move from a stage four, and we will be able to bring it in to the front lines that In reality, we don't allow the patients to get to a stage four. We need to stop the disease much earlier. But because these are investigational drugs, of course, we have to start for a safety perspective in a stage four. And where we are going with all of this in also our PRGN-3005, the ovarian cancer, is the same thing. You have And as it will be presented at ASCO, you will see we have patients that they have failed seven, eight line of therapies. So with that in mind, you can imagine that the requirement of those patients are different than a patient that might have failed two lines of therapy. And therefore... What we have done is design it this way. As we get the data from Phase 1b, then we can narrow down further according to the stage of the disease, according to the course of the disease, and then we can have basically a different design for various stages or dosing. But right now what is important is Number one, we can re-dose. We are the only platform that can do that without tremendous costs. And we can directly expand these cells in the patients. And I think the data for PRGen 3005 in ASCO will show that.
spk06: Sure, yeah. Just a follow-up on that part. So for the product to be used for the repeating dosing, on average, how many doses you can get from one batch of the production for repeat doses?
spk08: Yeah, I think this, of course, this is part of our manufacturing process, but I can tell you that we can, from the material that originally we can get, we can produce number of doses, as well as Obviously, you can always a-freeze again, and then if later on it's needed to be used for more than number that we have. So currently, basically, with the original material, we have been able to read those.
spk06: Okay, I see. And my last question is on the regaining rights to the CD19 and BCMA. target. Could you give us a broader picture of what your strategy is for those two targets?
spk08: Yeah, absolutely. So our strategy from the beginning has been really we know CD19 works. I mean, everyone has seen that. Kite, Novartis, they have shown that. BCMA, similarly, we know it works. The issue at hand for the field is How do you manufacture this? What platform do you use? And the cost. And we see that this is a struggle, even for the biggest pharma of the world. It's a struggle in order to get this to a patient with the price tag that not only few patients can afford this, but everybody can have access to this. First thing for us was very important, and a lot of people asked the question in regard to CD19, was to show our platform. First of all, scale our platform, put it in the clinic, make sure that it's safe, and make sure that it does what we ask the platform to do. And now, across multiple targets, hematological, solid tumors, with many patients we have shown this so this is the platform has shown its capability and by the way when we originally said that we have changed the Sleeping Beauty platform and we have not only modified it to fit what a commercially viable scalable platform should be that's exactly what we have done and I am Again, to my knowledge, no one else has been able to bring a non-viral platform overnight to this patient. So we did that. And now, after doing that across AML, ovarian, and DROR1 positive indication, what we can do is to bring the CD19 and BCMA. And why was it important for us to regain these assets? Because we know with application of our platform plus the validity of these targets, we can change the paradigm now. And as I said, it's not about just being first in a class or first to market. It's about the best in the class and then how you can affect the market. And we believe we can do this now. And even though, yes, there are drug products out there, but you have seen the availability of this and the costs and we think we can do better and that's why we are very rapidly moving it after showing our platform also has the ability to be scaled up and commercially viable.
spk06: Thanks for taking my question.
spk01: Congrats on the progress.
spk08: Thank you very much.
spk05: Thank you. Ladies and gentlemen, this concludes our question and answer session. At this time, I would now like to turn the conference back to Dr. Sabzavari for closing remarks.
spk08: Thank you. As I mentioned, this is really a very exciting time for Precision, and we are making significant progress in advancing multiple clinical programs. Each of the five clinical programs discussed today meet the foundational clinical principles that I outlined at the start of the call. Thus, while our goal pipeline provides multiple shots on goals in the diverse oncology indications and rare diseases, we've demonstrated unmet need. It is also aligned with our business strategy and organized to meet our financial discipline objectives. At this point, I would like to sincerely thank all of our patients for putting the trust in us and their faith, and everyone at Precision for their focus on dedication to our clinical and strategic objectives, which I'm honored to stand next to them, and to once again acknowledge our shareholders whose support is essential to our ongoing progress. I look forward to updating you in the months ahead as we make progress toward realizing our vision of developing transformative therapies with disruptive pricing that can improve outcomes for patients and the economics of safe and effective cancer therapy. Thank you again.
spk05: Thank you. The Conference of Precision has now concluded. Thank you for your participation, and you may now disconnect your lines.
Disclaimer