Precigen, Inc. (PGEN) Earnings Call Transcript & Summary

I think we'll get started here. My name is Turner Keith. I'm one of the biotech analysts at JPMorgan. I'm pleased to introduce our next presenting company, Precigen. And speaking on behalf of the company is CEO, Helen Sabzevari. And with that, I'll let Helen take it away, and we'll be doing a breakout session in Olympic Room just afterwards. So thank you.

Thank you very much. First of all, I'd like to thank the audience. I know you had a long day, and for being here. Also, the JPMorgan organizers for having us. And today, what I would like to do is basically discuss our -- Precigen and programs. But before I get to that, I will be making some forward-looking statements, which hopefully, you look at the slides directly yourself and look at the content. What we have been especially communicating in the past few weeks was really the restructuring of Intrexon into Precigen. And as you can see, there were multiple pillars in this decision. One of the reasons for the restructuring of Intrexon to Precigen and renaming it now Precigen was the increased focus on the health care based on the health care portfolio that we have been bringing forward in the past 2 years. And it has become very clear for us that where the value for patients and for our investor exists is in the technologies and the platform that we have also with the programs that we have moved forward. And similarly, in the -- in that spirit, what we have decided to do in the past year and has been announced this year was divestment of nonhealth care and we have done so by transferring these assets to Third Security, and therefore, creating a cash runway for us of upwards of $175 million, which is a healthy runway that can allow us to focus on our health programs. And in the process, I have to thank our previous CEO, RJ Kirk, and his vision and also our Board for allowing me to lead Precigen, and they have the confidence in my leadership, and I'm thankful for them. In view of what you see, one might ask what does Precigen contain. At this point, Precigen is really contains all of the subsidiaries that are health-oriented: the previous Precigen Therapeutics, Inc., which has all the portfolio in the next-generation of gene therapy and with the multiple clinical and preclinical programs in our portfolio. We have our microbial subsidiary, ActoBio, that it has very exciting programs. And I will highlight some of those. We also have multigenic Triple-Gene subsidiary, that it has the first Phase I of its kind, very innovative technology for heart failure patients, and we're very excited about that. And similarly, we have Exemplar Genetics under Precigen, which is a market leader in genetically engineered miniswine models. And this actually entity has the ability and the potential to really redefine the regenerative medicine and the use of these miniswines for that aspects. And we are very excited about actually this subsidiary. Well, how do we see now with having these subsidiaries under the same roof? And what would be the Precigen strategy? For us, what's important to focus, and we had done that originally for our health care assets under Precigen, execution is a major pillar for us, to focus on a rapid execution and move our portfolio with value to clinic. We also will be very fiscally responsible, and we have strengthened our cash runway with basically having the nonhealth subsidiaries now off of our portfolio and not only monetizing it, but also have created and take some of the cash burn off. Furthermore, we will be continuously manage our portfolio with go and no-go decision. This is extremely important for our execution and to be fiscally responsible. And as I always say, we are not married to a program. We are married to the science and the merit for the patient. And that's what we're going to be continuously execute. And finally, we will be seeking strategic partnerships that would allow for the right indications and right portfolio, basically, assets, and this would be part of our activity in the upcoming year. I am not going to spend tremendous amount of time on this slide, but one of the things that I have to stress, where Precigen is differentiated from the rest of the gene therapy companies is in the breadth of the technology platform that is required for precision medicine. And having all of this technology under the same roof has allowed actually Precigen to use sort of a jumping board and execute very rapidly, and I'm going to highlight some of the programs that currently moving in the clinic and with rapid speed here. And I think in this slide, for the first time, you see the comprehensive breadth of the portfolio, clinical portfolio, for Precigen. As you can see, we have a number of clinical assets that are directly run within Precigen, and they expand from Phase I to Phase IIs. And we have 4 programs that in partnerships with our partners, expand from Phase I all the way to Phase IIIs. And we are very excited that the majority of these programs, as you see, there is inflection points that will be repeated this year within 2020, and we have many readouts that are coming up, which speaks to the usage of these technologies in the right indication and its added value for our portfolio. Similarly, we have focused our preclinical portfolio. And as you can see, we have focused on 3 major area: oncology, immuno-oncology, autoimmunity, and infectious diseases. And we have a number of programs that are moving forward in various areas. One thing that I will stress over and over again. The portfolio that we have built, it will be managed very, very tightly for go/no-go decisions and bringing the most valuable and priority programs to clinic. As any good portfolio, there is never 100% and should not be 100% movement of programs to the clinic. And this, we are very committed to ensure that we will only bring the programs that are relevant for our patients with the highest probability of success in the clinic. In that spirit, what I want to highlight is some of the programs that we currently have in the clinic and give you a view of where we are and where we'll be in 2020. Obviously, our UltraCAR-T programs are one of the highest value for us and is one of the exciting programs. I think to this audience and to the rest, one thing that we have seen very clearly through the conventional CAR-T is the issues that has been coming up with manufacturing of these, which are extensive. They are expensive. There is a number of failures associated with it. And at the end, the type of cells that are put back in the patients are semi-exhausted, therefore, the failures that you see in solid tumors. What we have done in the past 1.5 years to advance the platform -- technology platforms to basically turn the CAR-T therapy to a therapy that is accessible at any hospital within their clean room overnight, requiring no manufacturing expansion outside, which cost less, cuts time. The time to the patient, we have reduced it to overnight. Basically, the patient walks in, is, a, freezed. T-cells are transfected with our nonviral platform and next day, QC-ed and back to the patients. And this is the first time in the history of mankind, as FDA quoted, that such technology has been introduced in the clinic. This is not our hope, and it's not our dream. This is what currently is happening. And we have started 2 clinical trials, one in solid tumors, ovarian, and one in hematological one. And as we speak, patients are receiving this overnight, manufacturing of their own T-cell. Basically, what we have done at this point, the allogeneic CAR-T becomes irrelevant for us because the concept of overnight, putting these T-cells back next day without any manufacturing, it's what it has allowed us to do that. And this has been as a result of extensive engineering and using our specific platforms that we have the IP around it, with membrane-bound IL-15 as well as our UltraVectors and our CMC to achieve this. As I have reported previously in our quarterly, the PRGN-3005 is an example of UltraVector that -- UltraCAR that we have in the clinic for ovarian cancer targeting MUC16 and it's -- the Phase I has started. We have recruited the first cohort, and currently, we are recruiting the second cohort. Up to date, we have had 100% manufacturing success, which is we are extremely excited about. But we also very encouraged with our preliminary findings of UltraCAR-T kinetics in the patients. And this, especially in a view that the doses that these patients have been receiving, its magnitude of order lower than conventional CAR-T, it makes us very excited about this platform, and we are looking forward to initial data readout from the IP arm of this in 2020, second half. And again, I want to stress that this program is currently enrolling their second cohort, no manufacturing failure. And very -- and we are very excited and encouraged with our preliminary data from the first cohort in the CAR-T cell kinetics. Similarly, last year, we had started our UltraCAR program for AML patients. This is a patient population with very limited time. They definitely cannot benefit from the conventional CAR therapy since they have 3 to 4 months to live. And definitely, the overnight manufacturing of CAR-T and infusion is the best way, as we believe. Currently, as we have reported, our phase -- in Phase I, the patients, the first cohort, they have been dosed. We have had 0 manufacturing failure. This is in a different center at Moffitt in Florida. And what we also have seen in this cohort, very encouraging preliminary findings of our UltraCAR-T cell kinetics. And again, what is of the most important here for us, not only we are seeing encouraging data here, but also in the position that the doses that we are giving, magnitudes of order lower than conventional CAR-T, this is very exciting for us. We -- another point of this trial. This is, again, especially this data in the context of this arm is a non-lymphodepleted, which makes a tremendous difference because at the current point in a hematological CAR-Ts, everyone does lymphodepletion. Our technology, we believe, allows us to bypass lymphodepletion. And currently, as you can see, in 2 arms of this study, we are interrogating this concept in lympho and non-lymphodepleted patients, and we are very excited about this. We expect that the initial data readout is in second half of 2020. And also, we are very encouraged by receiving the orphan drug designations from the FDA for this trial. And another one of our very exciting assets is the PRGN-2009, which, last year, I communicated. And as part of our goal was preclinically move this and advance it, and we have done that exactly. The PRGN-2009, it basically uses our specific AdenoVerse platform, the gorilla adenovectors, which with the specific antigen toward the HPV indications, such as head and neck, in cervical cancers, in all cancers. And we are very excited that we are doing this, I mean, collaboration with NCI, Dr. Jeffrey Schlom, and we expect that the Phase I clinical trial is initiated in 2020. And the advantage of these platforms over the current off-the-shelf cancer vaccine platform is that the gorilla AdenoVerse platform allows you a payload unlike anything else that exists. We can be up to 10 to 15 kb. We have number of antigens, which are very unique on the HPV. We can target the actually hot spots, and we have done so. And this asset, one other specific things is unlike other viruses and unlike other Ad5s that you can only give once because of the lack of previous immunity to these vectors, we can give this multiples of times. And we are very, very excited as we move this toward the clinic in 2020. And finally, from the Precigen portfolio, we had mentioned to you the platform that we built to address the immunosuppression and tumor microenvironment based on the tumor indications. And currently, we have 2 targets that addresses different indications and addressing the different pathways of immunosuppression currently. And what we have is in the upper-hand side, you see that, for instance, our PRGN-5001, when we have this up against the approved anti-PD-1, these are clinical-grade basically anti-PD-1 molecules, it basically does significantly better in keeping the tumors at check and reducing the tumor burden. Our 5002, which addresses and interrogates a different immunosuppressive mechanism in a tumor microenvironment, as you can see, for instance, in a cervical cancer, it, head-to-head against the checkpoint inhibitor, did actually eradicate the tumors completely. We are extremely excited about these assets. We are currently moving these to IND-enabling studies. And we are evaluating our strategic partnering options as a number of the companies have expressed their interest. And we would -- after evaluation, we will make a decision, what would be the best strategy to move these assets forward for us. In this slide, what I'd like to also highlight is some of the clinical trials that we currently have with our microbial platform at ActoBio. In partnership with Oragenics, we have been actually targeting the oral mucositis, which is the effect after the chemoradiation therapy, especially in patients with the head and neck. It's really a very difficult disease. These patients, as you can see in the left-hand picture, they end up with ulcerative basically lesions in the mouth. It's quite painful, and there is really no treatment for these patients currently. And what we have is now we have been able to use our microbial with the expression of a gene that actually repairs the lining of the mouth. And it has been formulated into a rinse. So it's a mouthwash. And what you see, we are expecting an interim data from a Phase II of this clinical trial in first half of 2020. And very excited that the FDA has fast-tracked this designation for us. In this slide, I'm not going to take you through all of the details, except telling you that on a Phase I, the safety is quite good. And we already, at the Phase I, we have seen that 29% of our responders report much fewer ulcerative lesions in the mouth than the placebo controls. And we are looking forward to seeing the Phase II data in the first half of 2020. On the similar platform of microbial platform from our ActoBio subsidiaries, we also have a very active program in diabetes, T1D. And I'm showing you on the right-hand side, the preclinical data which clearly showed 89% of the new onset of diabetes in mice were cured by our drug product, AG019. Currently, we have finished the Phase I, and we have moved to Phase I/II for this, and we are expecting a readout in the second half of 2020. And finally, one of the most exciting also assets that we have in our portfolio is INXN-4001 at -- within our Triple-Gene subsidiary. It's a novel gene therapy for heart failure patients, which are extremely sick, they have 0 options. And this uses our UltraVector platform by introducing 3 genes. And we have reported on interim data for a Phase Ib. As you can see, for the 6-minute walks, we have encouraging data on the -- this therapy. And tomorrow, Dr. Reed will be addressing this in a more comprehensive fashion at the 11:30 talk that he will be giving. But we also expect a full readout, 1 year readout, on this by the end of 2020, and we are very excited about this asset. Last year, when I was standing here, I'm presenting on behalf of Precigen as a subsidiary of Intrexon, we put up a set of goals that we wanted to achieve in 2019. And I am excited and happy to say that we not only achieved all those goals but actually, we beat some of the time lines that we had for the achievement of these goals. One thing that is extremely important for us is to keep our focus on our portfolio to maximize the value for the patient and investors; to execute, execute and execute against that; and ensure that we make the right decision for the movement of our assets within the cash runway that we have. In order to do that for 2020, these are the highlights of the goal and the high priority goals that we have set for Precigen, including all the subsidiaries. We will be reporting on initial data from IP arm of ovarian UltraCAR Phase I trials which, as I mentioned to you, we have very encouraging data from our first cohorts on the CAR-T kinetics. We will report on the initial data from PRGN-3006 similarly, by the end of the year in AML. And again, this is exciting because we have an arm that is non-lymphodepleted. We will report on our interim data from a Phase II with our partners at Oragenics on AG013 for oral mucositis in head and neck patients. We will have interim data from a Phase Ib and IIa trial for our T1D diabetes microbial platform. And we will have a Phase I data completion of our gene therapy for the cardiovascular for in INXN-4001. And to add further, as we had promised, we are moving the 2009 PRGN rapidly toward the clinic, and we will initiate the clinical trial Phase I in collaboration with NCI in 2020. Similarly, for our preclinical portfolio, we have a ambitious but focused goal, and we will be rapidly advancing these programs that are listed here toward go/no-go decision. But more importantly, I want to get your attention, we are advancing our next-generation of UltraCAR-T that it gives us a unique advantage over even further in the platforms for us toward the clinic. And we will be advancing the infectious disease candidate that we have promised last year going toward the IND-enabling studies. And with that, I hope that I have given you a view of what the new Precigen and its comprehensive portfolio, both clinically and preclinically, looks like. And if I may leave you with a message at the end of this talk. Our new Precigen, it will be focused on health care asset based on the innovative technology that we have, and we continuously advance. We are advancing a robust portfolio that will be managed extremely carefully to make sure that we bring the highest priority with best possibility of success to the clinic. We have a strong cash balance runway that we will use to basically move our portfolio forward; and we have multiple value-creating opportunities upcoming and various strategies for partnerships, which we'll clearly, we will take advantage depending on the programs and assets. With that, I'd like to thank you for your attention, and we will be moving, I think, to our question and answer. Thank you.