PGEN (2019 - Q3)

Good morning, and welcome to the Precigen Business and Clinical Update Conference Call. [Operator Instructions] Please note, this event is being recorded. I would now like to turn the conference over to Steve Harasym, Vice President of Investor Relations. Please go ahead. Steven H

Welcome to the Precigen Business and Pipeline Update Call. I'm Steve Harasym, Vice President of Investor Relations for Intrexon, and I'm pleased to be joined today by Dr. Helen Sabzevari, President of Precigen. 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 materially from those indicated by our forward-looking statements. Please read the safe harbor statement contained in this presentation as well as Intrexon's most recent SEC filings for a more complete discussion of these risks and uncertainties. As indicated last quarter, we will spend today's call providing a business and pipeline update on Precigen. R.J. Kirk, Intrexon's Chairman and CEO, will join Helen at the conclusion for a Q&A session. I would like to now turn the call over to Dr. Helen Sabzevari.

Thank you, Steve. I'm extremely pleased to be here today to highlight the significant progress Precigen has made since our official company debut during the JPMorgan Healthcare Conference in January. As we have communicated previously, we believe we hold a unique position in the biotech landscape, having all of the required technology platforms for advancing precision medicine. This technology platform are designed to enable us to construct powerful multi-gene program for optimized gene expression, deliver these multi-gene programs using viral and nonviral vectors with high-payload capacity and controller gene expression and regulate therapeutic performance, utilizing clinically advanced switches. The breadth and the versatility of these platforms, coupled with our expertise in immunology, has positioned us to have an unparalleled holistic approach with the potential to deliver the core promise of precision medicine. Next slide, please. These platforms have allowed us to lay the foundation to build a comprehensive pipeline in immuno-oncology, infectious diseases and autoimmune disorders. In 2019, we have successfully progressed 2 first-in-class UltraCAR-T programs into the clinic; advanced PRGN-2009 towards potential IND; advanced 2 discovery programs into preclinical testing; optimized our UltraCAR-T manufacturing process and built an in-house GMP manufacturing facility to support our off-the-shelf AdenoVerse immunotherapy program. Precigen now sits at an inflection point, as our achievements during this year positioned Precigen to deliver on important data readouts in the coming quarters. Given the breadth of our pipeline, business development and product prioritization remain key components of Precigen's business model. We remain disciplined in our R&D spending and while we plan to develop select programs on our own, we will also seek a strategic partnership to advance other pipeline programs in an efficient and cost-effective manner. On today's call, I will highlight the significant strides we have made since the beginning of the year, particularly in regard to our UltraCAR-T therapeutic platform and associated ongoing trials. Next slide, please. Our UltraCAR-T program is fundamentally differentiated from the competition and addresses major challenges associated with the current generation CAR-T therapy. One key differentiator with UltraCAR-T platform is the use of our nonviral sleeping beauty gene delivery system. We have optimized the sleeping beauty system using our UltraVector DNA construction platform to produce multi-genic UltraCAR-T cells. This optimization produces a homogeneous population of UltraCAR-T cells, where multiple genes are expressed [ on all ] modified T cells. Our UltraCAR-T co-express an antigen-specific CAR, a kill switch and a membrane-bound IL-15 simultaneously. Precigen's proprietary membrane-bound IL-15 enhances persistence, in-vivo expansion and maintains a stem like memory phenotype of UltraCAR-T cell. This leads to longer-lasting antitumor response compared to conventional CAR-T cells, which is essential for successful targeting of solid tumors. Another key differentiator with the UltraCAR-T therapeutic platform is our decentralized, rapid manufacturing process, which allows us to manufacture overnight at the medical center cGMP facility and reinfuse the patients the next day. We have evolved our manufacturing platform to advance rapidly through the clinic towards commercialization. We are the first company to implement nonviral, decentralized, rapid manufacturing of CAR-T cells in the clinic. We have demonstrated our ability to infuse patients the day after gene transfer at 2 different sites in our ongoing clinical trials. And I'm pleased to announce today that we have experienced 100% success in manufacturing of UltraCAR-T cells for both the PRGN-3005 and PRGN-3006 clinical trials to date. We consider this a very significant milestone in demonstrating the potential of our UltraCAR-T platform. We believe that Precigen is poised to disrupt the current CAR-T treatment landscape. Next slide, please. Now moving to the first program, I'll detail PRGN-3005 UltraCAR-T. This is an investigational therapy using autologous CAR-T cells targeting the Mucin 16 or MUC16 protein. MUC16 is an attractive CAR-T target for ovarian cancer since it is overexpressed on more than 80% of ovarian tumors with limited expression in normal tissue and that is important. This UltraCAR-T is multigenic and designed to co-express membrane-bound IL-15, a kill switch and antigen-specific CAR that targets MUC16. We have designed our MUC16 CAR to preferentially target PRGN-3005 to tumor cells. Furthermore, our team has optimized the affinity of our anti-MUC16 binding domain for optimal signaling by our CAR. These genes are delivered via our nonviral system, which enables homogeneous expression of these 3 genes in all modified T cells. These CAR-T cells homogeneity is an important factor for future commercialization. We choose ovarian cancer as one of our first tumor targets as these patients still have significant unmet need. There are few treatment options and survival rates remain low. It is also a large patient population, with approximately 300,000 patients diagnosed worldwide annually, including 22,000 in the U.S. alone. Next slide, please. We are currently testing PRGN-3005 in a Phase I study in collaboration with the University of Washington and Fred Hutchinson Cancer Center, leaders in immunotherapy and CAR treatment. This is an investigator-initiated dose escalation study to evaluate the safety and maximum tolerated dose of PRGN-3005, delivered by either intraperitoneal or intravenous infusion. This study population includes advanced stage 3 or 4 recurrent ovarian, fallopian tube and primary peritoneal cancer patients who are platinum resistant and have progressed after receiving a standard of care therapy. For both routes of administration, PRGN-3005 will follow a 3 x 3 dose escalation pattern. We expect to enroll up to 41 patients total in this study. In August, we communicated that we had dosed the first patient in this Phase I trial, and I am very pleased to announce that we recently completed dosing the first cohort in the intraperitoneal, or IP arm of this trial. We are incredibly pleased with the progress of this trial. Its execution to date demonstrates ability to enroll patients and rapidly manufacture UltraCAR-T. While the Phase I trial is obviously focused on safety, we are also evaluating the maximum tolerated dose and the ability of PRGN-3005 to expand in-vivo, a key factor for future success. We are excited to continue the dose escalation and look forward to updating you on our progress. We expect to provide an initial data readout from IP arm of this trial in the second half of 2020. Next slide, please. I'll now move to PRGN-3006 UltraCAR-T. PRGN-3006 is an autologous CAR-T therapy, targeting patients with relapsed or refractory acute myeloid leukemia, or AML, and higher-risk myelodysplastic syndrome, or MDS. There are approximately 20,000 AML patients diagnosed in the U.S. annually. This UltraCAR-T is multigenic and designed to co-express membrane-bound IL-15, a kill switch, an antigen-specific CAR that targets CD33. CD33 is an attractive target for immunotherapy because it is overexpressed on AML blast and leukemic stem cells, but is not expressed on normal hematopoietic stem cells. 85% to 90% of AML patients express CD33 under tumor cell. AML is a heterogeneous disease with very high relapse rates and rapid progression. Time is of the essence for these patients, and long manufacturing delays of viral-based CAR-T therapies can be an obstacle for successful treatment intervention. Our UltraCAR-T approach represents a significant advantage for the time-critical treatment of these patients. Next slide, please. PRGN-3006 is being evaluated in a Phase I/Ib study of the treatment of patients with relapsed or refractory AML and higher-risk MDS. This is a nonrandomized investigator-initiated safety and tolerability study of the PRGN-3006 UltraCAR-T following intravenous administration of escalating dose. The trial is being run in collaboration with the Moffitt Cancer Center, a pioneer in CAR-T clinical development. In the 3x3 dose escalation phase, patients will be treated in 1 of 2 arms. Arm 1 will receive CAR-T cell infusion without prior lymphodepletion and arm 2 will receive lymphodepleting chemotherapy. The dose escalation phase of each arm will be followed by a dose expansion phase at a maximum tolerated dose. Since our UltraCAR-T cells have potential for enhanced in-vivo expansion and persistence without additional cytokine requirement, we are very excited to evaluate PRGN-3006 in patients without prior lymphodepletion. In July, we announced that we had completed dosing of the first patient in the PRGN-3006 trial. I'm happy to announce that we recently completed treatment of patients at the first dose level in arm 1 without prior lymphodepletion. As with PRGN-3005, we are incredibly pleased with the progress of PRGN-3006 and see it as another validation of our ability to execute. For comparison, I would like to discuss PRGN-3006 versus INXN-3004, both targeting CD33, but with very different manufacturing process. INXN-3004 is a discontinued legacy investigational product that was in a Phase I safety study of autologous T-cell, transfused with lentivirus to express CD33 specific CAR in patients with relapsed or refractory AML. This study was conducted in collaboration with MD Anderson Cancer Center. Unfortunately, after nearly 2 years, only 3 patients were infused with the product. This was due to failures of viral-based ex vivo manufacturing that did not generate therapy fast enough for some patients whose disease progressed before the therapeutic CAR-T cells were available. This is why we spent so much energy developing a state-of-the-art nonviral manufacturing process to deliver UltraCAR-T therapy to patients overnight. We continue to believe that CD33 is an attractive target for treatment of AML and PRGN-3006 represents a viable treatment option for this patient population. We are extremely excited about the PRGN-3006 clinical trial and evaluation of our platform with or without lymphodepletion in AML patients. We expect to provide an initial data readout from the ongoing trial in the second half of 2020. Additionally, we will present preclinical data at the upcoming American Society of Hematology Annual Meeting and Exposition in December. Next slide, please. I'll now move to PRGN-2009. PRGN-2009 is an off-the-shelf immunotherapy product candidate utilizing AdenoVerse platform, designed to activate immune system to recognize and target HPV positive solid tumors. HPV positive cancers represent a significant health burden in indications such as head and neck, cervical, vaginal and anal cancer. Multiple approaches to targeting HPV positive cancers have recently been of great interest in immuno-oncology, of which TCR-T cell therapies are applicable to only a small subset of HPV positive cancer patients due to the HLA polymorphism and are further restricted by manufacturing and high cost. And [ critical ] cancer vaccines approaches have lacked efficacy due to the combination of a limited immune response and narrow antigen coverage. PRGN-2009 leverages our UltraVector platform to optimize HPV antigen design and coverage and our Gorilla adenovector, which has low to no seroprevalence in humans. This allows for durable immune response and the ability for repeat administration. Preclinical testing of PRGN-2009 has demonstrated robust antigen-specific immune response and potent antitumor activity in humanized mouse model. Based on results from in-vivo mouse model, PRGN-2009 also represents a target opportunity for combination with treatment, both within and outside of our pipeline. This program is currently under development through CRADA with Dr. Jeffrey Schlom, a world-renowned investigator in immuno-oncology at the NCI. This CRADA has been valuable to Precigen as it allowed us to complete the preclinical work and the forthcoming Phase I clinical trial in a cost-effective way and at a very rapid pace. It also provides for the potential for expansion to other targets and combinations. We are working very closely with the NCI team on an IND submission for PRGN-2009. We expect NCI to begin dosing patients in 2020, and are looking forward to providing additional details on this program in the near future. Furthermore, the opening of our manufacturing facility in Germantown, Maryland, which we announced in April is designed to support our AdenoVerse platform-based therapeutics, including PRGN-2009. The opening of this facility is a strategic long-term decision that will put Precigen firmly in control of our early phase clinical manufacturing needs for gene therapies, and future product development. Next slide, please. Lastly, I'll move to our multifunctional therapeutic platform, targeting solid tumors. While checkpoint inhibitors have had a significant impact on oncology treatment, a large percentage of patients fail to respond and among those that do, approximately 1/3 relapse. Our multifunctional therapeutics platform is designed to address multiple immunosuppressive pathways in the tumor microenvironment. We have taken a strategic approach to developing this platform with 2 preclinical assets, PRGN-5001 and PRGN-5002 with significant market opportunity. Our first multifunctional therapeutic candidate PRGN-5001 has demonstrated the ability to enhance T-cell activation and antitumor effect in mouse models of tumors that do not respond well to anti-PD-1 checkpoint inhibitor antibodies. As shown in the last section of this slide, we highlight that in a humanized mouse model of head and neck cancer, PRGN-5001 showed an ability to overcome tumor microenvironment immunosuppression and significantly improved T-cell function as compared to a stand-alone anti-PD-1 treatment, leading to superior antitumor response. Our team has generated a strong preclinical data package, demonstrating the effectiveness of PRGN-5001 in multiple cancer models where anti-PD-1 treatment is not very effective. Our second multifunctional therapeutic candidate, PRGN-5002, is advancing rapidly through preclinical development, targeting a different pathway to address tumor microenvironment driven immunosuppression, to enhance efficacy over checkpoint inhibitor. In a preclinical study, PRGN-5002 exhibited enhanced infiltration of cytotoxic T-cells into tumor and superior antitumor effect in humanized mouse model of cervical cancer compared to anti-PD-1 therapy. Our multifunctional therapeutics platform, including PRGN-5001 and PRGN-5002, has tremendous potential in multiple cancer indications, and we continue to evaluate the optimal path forward for their development, including partnership opportunities. Next slide, please. To conclude, I like to again highlight that Precigen is highly differentiated from other immuno-oncology companies. We have made significant progress in advancing our pipeline, and we expect several data readouts in the coming quarters. The current slide shows the objectives we outlined during the JPMorgan Conference in January. We have already achieved these objectives as we have initiated a Phase I/Ib trial in AML and MDS for PRGN-3006 UltraCAR-T and completed dosing of the first cohort; initiated a Phase I trial for ovarian cancer for PRGN-3005 UltraCAR-T and completed dosing of the first cohort; we have advanced the PRGN-2009 for solid tumors; we have advanced one infectious disease candidate; and finally, we have also advanced multiple preclinical candidates. We look forward to providing our key objectives for 2020 at the JPMorgan conference in January. We will now open the call up for questions.

[Operator Instructions] And our first question comes from Jason Butler of JMP Securities.

Helen, congrats on all the progress achieved this year. Helen, I just want to start off by following up on the comments that you had about success in manufacturing, 100% success rate for the UltraCAR-T clinical programs. Can you maybe just give us a little bit more color on what defines manufacturing success? And if the clinical experience you already have gives you -- or the manufacturing experience you already have gives you confidence that you will succeed at the higher doses also?

Thank you, Jason. Nice talking to you. Excellent question. In regard to our manufacturing success, as you know, we are -- and I believe we are the only company currently that have a nonviral overnight-based CAR-T -- UltraCAR-T platform in the clinic. And we do this under 2 days -- overnight basically -- and the patients the next day are receiving this. As you can imagine, we had to put a lot of resources and effort in making this happen. And from the FDA perspective, this was considered first in mankind. And what we have achieved currently is in every cohort, both in solid tumors and hematological that the GMP facilities of the hospital based on the SOPs that we have provided them and training that they have received, they have been 100% successful to actually manufacture the dose that they need under the 2 days or overnight, I should say, and infuse the patient without failure. And we have done this in hematological tumors, a very tough scenario; in AML, as you can see, some of these patients can have excess of 90% blast cells in their blood, which really make their T-cells number very, very low and also in ovarian cancer patient in the solid tumors. We are confident that we can now provide this higher doses as we move, and we are very excited about the cohort that we have already finished and as we are moving to the higher dose.

Great. And then just a quick follow-up on PRGN-3006. I think the advantage -- clinical advantages of not having to go through lymphoablation are clear. But can you maybe just walk us through the time line advantages that you would also have ahead of treatment by not having to go through lymphoablation?

I think this is very important because, first of all, as you know, the lymphodepletion is toxic. It's basically treatment with the chemotherapy and put the patients even further in danger, patients that are currently not in a very good stages. But most importantly, the process of lymphodepletion, especially in hematological, it takes somewhere between 5 days to more than a week, depending on what regimens they receive. And then in the previous setting, they have to go through the lentiviral CAR for manufacturing, which by itself takes another 3 to 4 weeks, if it's successful. And as you know, there is a 25% to 30% rate of failure there at least. So having said that, with this, we actually -- I think you should perceive it this way, we have turned the UltraCAR-T as close to as off-the-shelf you can make it because you really don't have that much preparation. The day before the patients get a [ free ] and then overnight we have our nonviral system transaction and the next day, the patient gets infused with their own autologous, basically CAR-T. This is a major achievement because, really now you don't have to go through the allogeneic, for instance, again, expansion and in the best case scenario, people can dose 100 patients with one preparation. Here, we take the patient CAR-T directly and expand it overnight -- or actually inspect it, we don't expand it at all -- and then the next day infuse and the expansion takes place in the patient. And this is one of the things we are following, and we are looking forward to reporting on it in the upcoming quarter.

Our next question comes from Tyler Van Buren of Piper Jaffray.

My first question was on 3005 or the ovarian program. Can you, I guess, tell us about what doses you're using or what range of doses you'll be exploring in the escalation? And the in-vivo expansion is obviously a unique component, so can you also discuss what magnitude of in-vivo expansion you expect? Or maybe you've already seen with this first cohort and how that compares to other therapies that don't have the membrane-bound IL-15?

Great. Tyler, good to have you on the call. Absolutely, great question. So number one, in regards to the doses that -- we have not disclosed the doses, but I can speak this way. We are [ long ] different in regard to our doses as we are infusing the patients currently. We are nowhere close to what Ultra -- where the lentiviral CARs are. The UltraCAR-T platform is designed in such a way that by having a membrane-bound IL-15, the CAR-T, we can actually expand them directly in patients and [ these cells, ] because they have the membrane-bound IL-15, they can persist even in the absence of antigen or when they are not seeing the antigen. And this is what it makes our UltraCAR really different than any other CAR-T that currently exists in the landscape. So from the perspective of doses, we are looking forward to show that in the upcoming quarters, as our investigators will report on this, and in regards to the expansion, we have not disclosed this. Obviously, we are moving to the other cohorts as well. We are very excited about this program. And we will be reporting on that in a very near future

Okay, that's helpful. And just as a follow-up on MUC16 as a target, you spoke about the expression, but can you remind us of what you've seen preclinically that gives you all confidence in using it as a target for the therapy?

Absolutely. So MUC16 is actually, as mentioned, it expressed in a large amount of ovarian cancers and tumors. What we have done, we have done various humanized ovarian cancer tumor models and we have put our overnight manufacturing process with our UltraCARs that they contain the MUC16 and membrane-bound IL-15 and the kill switch, which by the way, all 3 simultaneously are expressed in our UltraCAR, which is another differentiator factor because now you have a drug product homogeneity, which is extremely important for commercialization. And what we have seen is our UltraCAR is much superior to even having a regular CAR with just MUC16. And this we have done in various tumor models, in humanized mouse models and also because of having our membrane-bound IL-15 in there, we show that these cells directly expand in-vivo, they persist and they completely eradicate the tumor. But it's also very exciting for us and based on the preclinical data that we presented to FDA, we could actually see that [ these cells stay ] functional even after eradication of tumor for a long period of time. And this, again, speaks to the persistence in-vivo. And we are looking forward to our clinical trials and investigating a similar kind of observation.

Our next question comes from Tycho Peterson of JPMorgan.

This is Tejas on for Tycho. Helen, you spoke about some preclinical data coming out at ASH and then some initial readouts for 3005 and 3006 in the back half of next year. Just thinking about sort of the next 12 months, can you help us understand what are the 2 or 3 like most important readouts that investors should be focused on here? And perhaps, if you can lay out, like what conferences those are likely to happen at? That would be super helpful.

Absolutely. So currently, as mentioned, our PRGN-3006 will be -- the preclinical data will be presented at ASH. Very similarly, we have done extensive preclinical analysis of our UltraCAR with the CD33 and membrane-bound IL-15. And in this, actually, presentation for the first time, the audience would get to see how effective our UltraCAR is, how they persist over a long period of time and how they can eradicate the tumor. And that is compared to, for instance, having the CAR alone in there versus having a membrane-bound IL-15. I will be presenting also as a keynote at the World Vaccine Conference in the beginning of December, which I will allude to some of this work. And we have planned for 2020, which currently I cannot disclose, but there will be presentations, and we look forward to that, both at the scientific arenas as well as obviously, updating our investors and shareholders.

Got it. And then in terms of your broader portfolio, can you just walk us through your thought process in choosing where to partner and where to sort of deploy a go-it-alone approach in terms of your commercialization effort?

Absolutely. I think that's an excellent question, Tycho (sic) [ Tejas ]. I think when you look at our portfolio, this portfolio has been designed, actually very specific concepts in mind. It's not a random portfolio. The areas that we have picked has been picked because we wanted to maximize our portfolio, basically utilization of the technology that we have and the target that we have at hand. And therefore, having autoimmunity, for instance, which is the flip side of immuno-oncology, if you know how to activate the immune system tremendously, you also know how to suppress it somewhat or vice-versa. So we have used all of the knowledge, the technology and the knowledge that we have in immunology and focus on the 3 areas. One other aspect that I have to say, especially in the field of immuno-oncology, which is currently is a deficit in the field, people are approaching targets on a random fashion and the combinations are random. And therefore, billions of dollars and billions of dollars worth of failures, unfortunately, in the clinic for the patient. What we have done, for instance, in regard to our multifunctional and multigenic platform is based on indication we have identified, based on technologies that we have, the immunosuppressive mechanism that are specific to that indication and we have targeted multigenic platform around that. So it's not random. It's very specific. And that's why, for instance, in 5002, you can see that it's very specifically, for instance, in the cervical where anti-PD-1s are not working, it clearly had shown a significant eradication of tumors in preclinical setting. Having said that, what -- also we are very disciplined in our R&D. I don't want to give an impression that we basically go after every molecule. We prioritize our portfolio continuously to benefit from the technologies that we have, and we are fully aware no one company can develop everything on their own, and that's not our plan. We prioritize on where we are best to develop or we have the collaboration and partners that they can do that. And also, we look at our partnership and interest that we are receiving to see what would be the best route for partnering with certain assets and the value of that for patients because we want to get to clinical trials rapidly and it's very, very important to have the right partners that they can do that, not only financially but from a perspective of speed, to get there for the patients and, obviously, for our shareholders and investors. So we take all of that into consideration. And let me just say it this way, we don't take this light, we -- it's very well thought around, and we approach it in a logical fashion that what would be best for this portfolio for the company, for the patient and for the shareholders.

Got it. And Helen, if I can ask one final one here, more of an Intrexon question than a Precigen question. Can -- Steve, maybe you can take this. I mean, can you help us sort of think through the bridge from the $90 million or so in cash and equivalents on the balance sheet today and your goal of $175 million by year-end. And perhaps, if you can update us on your plans for the sale of Exemplar and Trans Ova? Is this still expected to occur by year-end?

Tejas, this is R.J. I think we provided that bridge in our press release issued this morning. So as you know, I've made an offer personally, and we're also in discussions with numerous parties, numerous assets. Based on all of that, we feel that we can give the guidance, reiterate the guidance we've given in the past, which is that we expect to be able to conclude the year with committed funds of $175 million in cash, $175 million. If we didn't believe it, we wouldn't have said it, and we certainly wouldn't have said it multiple times. So beyond that, obviously, we can't discuss individual transactions, individual -- if [indiscernible] that the offer made I've made will be considered by independent members of the company's Board of Directors, with the Board of their legal and financial adviser. So we're not going to provide further specifics at this time.

Our next question comes from Swayampakula Ramakanth of H.C. Wainwright.

Helen, congratulations on the progress you have been achieving at Precigen. Talking about the manufacturing success that you'll had so far in the clinical program, just trying to understand, would there be anything different when you're going to doing this real-time from a clinical study, especially going through the manufacturing process, the QA/QC, still would you think you can get the drug back to the patient within 48 hours?

It's good to have you on the call. And actually, we have done that already. So when I spoke to the first cohort of both PRGN-3005 and PRGN-3006 clinical trials, our manufacturing happened at both Fred Hutchinson site as well as Moffitt Cancer Center, and everything, including QC and infusion, it all went overnight and the next day infusion. And I think, this is why we are so excited because, not only we have met all the criterias that FDA had asked us for viability. But also, as you've mentioned, you brought a great point about the QC and having the drug product ready, but also because of UltraVectors that we have, the multigenic UltraVectors that we have, we are capable of now producing a very homogeneous drug product, which means the UltraCAR-Ts are either they all express all the genes, the CAR, the membrane-bound IL-15 and a kill switch or they express nothing. In the case that they don't express anything, the T-cells of the patient is returned to the patient. There's nothing wrong with that, and FDA accepts that. And in the other case, we have [ to cede ] everything, the UltraCARs, and the doses and we -- by the way, we have been able to do all the assays that need to be done under the time line and infuse the patient. And that's why we are so excited about the 100% success rate, both in solid tumors and hematological ones in the clinic up to date. And we are looking forward to the rest of the arms and dose escalation, as we are currently moving through rapidly.

And then on the 3005 program, once you get through the Phase I program, do you -- is there a way for you to accelerate into approvals or do you think you have -- think this is kind of a novel program, you might have to be going through the full length of a Phase II and Phase III development?

Yes. Excellent question. Our -- basically, obviously, we are currently going through the safety and dose escalation as well as what we would present is the expansion potential of our UltraCAR directly in patients. But one of the most important thing that we are quite aware of, because of the differentiated platform that we have, which we can do a very rapid manufacturing with very low cost compared to the basically lentivirus and all the other CAR and including TCRs. What we are looking forward to, as we basically finish our Phase I, we have dialogues with the FDA in regard to what would be the best way to advance basically our platforms and in occasion, especially in the 2 disease areas that we just started, both of them are unmet need for the patient, and in a way they consider it orphan diseases. So we have very high hopes that we can have great discussion with the FDA and discuss various path for approval as rapidly as possible for the patients.

Last question from me, Helen. Outside of the CAR program, obviously, you have a big pipeline. So what's the program that kind of excites you the most outside of the CAR-T one?

So I have to say, the way I approach programs is based on the science and how fast we can get them to the clinic for the patients and what we are seeing preclinically. I think the portfolio of what we are holding and continuously evaluating is based on the data that we receive for go/no-go decision. In the immuno-oncology currently, besides our UltraCAR-T, our AdenoVerse platform and off-the-shelf and we are moving in 2009. And as I mentioned, we are looking forward to dosing patients in 2020 with off-the-shelf AdenoVerse platform that we have for HPV. As you know, currently, there is a lot of effort is going on in manufacturing [ CARs ] for instance, for HPV, for some they have put vaccines in there. What we have done is actually used our technology and leapfrogged in a sense of developing a platform that is off-the-shelf, you can [ get it ] repeatedly and it costs much lower. And you basically create HPV-specific T-cell with a high affinity because we use our platform of UltraVector, the antigens that we have picked is very different and the delivery, that mechanism that we have is unlike what other companies have, and that differentiates us. So I'm looking forward to this platform, definitely. But at the same token, I have to say, we are quite excited about our multifunctional platform because now this is not one molecule anymore. We have made a platform that based on different indications we can take the immunosuppression away from the tumor microenvironment, especially in all the indications that the checkpoint inhibitors are not functioning. So those immuno-oncology are exciting to me. And what I would say in regard to our autoimmunity and especially infectious disease, I would like to say, you should wait and we will update you in the upcoming future because, I think it will be a very exciting program that currently in a preclinically, they are moving forward very, very rapidly as well.

This concludes our question-and-answer session. I would like to turn the conference back over to Dr. Helen Sabzevari for any closing remarks.

Thank you for taking the time to join us for our business and pipeline update. As you can see, we are very excited with the progress we've made and look forward to providing you with the updates in the coming months. Have a wonderful day, and thank you for attending.

The conference has now concluded. Thank you for attending today's presentation. You may now disconnect.