Precigen Q1 2023 Earnings Call Transcript

Quartr
Provided by Quartr
May 10, 2023

There are 8 speakers on the call.

Operator

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. After today's presentation, there will be an opportunity to ask 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.

Speaker 1

Thank you, Ryan, and thank you to everyone joining us this morning. With me today are Doctor. Helen Sabzavari, President and CEO of Precigen as well as Harry Tamesian, 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 Q1 2023 financial results.

Speaker 1

Following our prepared remarks, we will open the call to Q and 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 Prestige's most recent SEC filings for a more complete discussion of the risks and uncertainties.

Speaker 1

I would now like to turn the call over to Doctor. Salisbury. Ellen?

Speaker 2

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 4 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. 2nd, we strive to develop therapies that are differentiated not only by the clinical benefit they provide, but also by the 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 a significant barriers to access and impose substantial financial burdens on health systems and payers.

Speaker 2

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. 3rd, we pursue programs that we believe have potentially rapid parts 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 and our pipeline. And finally, we are committed to executing a cost effective R and 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.

Speaker 2

Now I'd like to provide you with highlights of our portfolio and after what we have accomplished in the Q1 of this year. The first platform that I will be discussing is our adenovers 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.

Speaker 2

And basically, these unique Gorilla vectors allows, 1st of all, that you introduce a large Payload capacity, meaning you can put many genes up to 12 kilobytes 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 At 5 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 these platform very unique and differentiated from all others.

Speaker 2

By utilization of these platform, What we have done is brought 2 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 1 and the expansion data for our PRGN 2012. PRGN 2012, it's a gorilla adenovirus That contains epitope, educating the immune system to HPV 6 and 11, which is the cause are basically generation of papilloma in a patient population, which we refer to as recurrent respiratory papilloma and disease in this patient. These patients, they continuously have The regeneration of dyspabyloma in the vocal cords and also in trachea.

Speaker 2

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-twenty 12 works is A vaccination with the course of 4 vaccination that with the data that we presented, 1st of all, It's a very favorable safety data. We have not seen any kind of DLTs These are nothing more than Grade 1 and 2, 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 and 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 7 or 8 surgery 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.

Speaker 2

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 their 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 And the new guidances that are given and the attention of FDA for coming for a fast solutions and approvals for these drugs. So we are very, very excited and we are looking forward to communications in upcoming months with our shareholders in regard to PRGEM 2012. Another drug product that is in the clinical trials currently using the same adenoverse platform is our PRGN-two thousand and nine.

Speaker 2

The beginning of this year, we communicated that we will be sharing data On the full Phase 1 and combination cohorts of with the checkpoint inhibitors with PRGN-two thousand and nine in HPV related cancer patients. PRGENT 2,009 targets HPV1618, which is the major cause of HBV 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 to the 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 We're coming up with the innovative therapies, which we believe PRGN-two thousand and nine is 1.

Speaker 2

And we are really excited about The data that will be presented in a Stage IV patient population, this is all commerce, 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 we 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 TRGN-two thousand and nine and move it into frontline therapy in a new adjuvant setting. We all know how important it is for these therapies to move to the front lines.

Speaker 2

And in our Phase II study that is Currently ongoing, we have 2 arms in a neoadjuvant head and neck therapy that PRGN-two thousand and nine He 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 a combination therapy With Keytruda, we'll 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 We followed up and especially for the enhancement of immune responses, which is very, very important in this setting. So that will be we will be reporting at the interim data by the end of this year as we have promised on PRGN-two thousand and nine. 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 Ultra Car platform and give you an overview of what we have done.

Speaker 2

As you all know, Our UltraCAR platform is a 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 patients and the next day you infuse them back. We currently have Actually, 3 clinical trials that are ongoing. The first one is our PRGN-three thousand and six, which this is the Ultra card that addresses in the AML patient population, Target CD33, it includes membrane bound IL-fifteen 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.

Speaker 2

At ASH, we reported not only on the safety and favorable very favorable safety of our UltraCAR But also the preliminary data from our Phase 1 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 our the T ORFA CAR 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 redose now in this patient. And why is that important?

Speaker 2

Because we believe this is the only Ultra Card platform that in a cost effective manner, Overnight manufacturing, you can redose 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 Phase 1b expansion cohort and we look forward to that. In regard to our next UltraCAR platform is our PRGN-three 10005, which directly It targets MUC16 on ovarian cancer. It also includes membrane bound IL-fifteen and a KL switch.

Speaker 2

And as you recall, we had 2 arms in this trial, both intraperitoneal and IV And FDA also allowed us to open the lymphodepletion arm. We have finished the dosing in 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 But also a preliminary efficacy data. And with that, We also have moved to Phase 1b expansion, which that 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.

Speaker 2

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 3rd Olfracar T that I would like to touch base on is our PRGN-three 10007. And PRGN 3,007, it's what we refer to as the next generation of our cars.

Speaker 2

What do we mean by that? It's simple. It has become evident that CARs in general, 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 it's quite tremendous when you use a classical CAR T. In our next generation setup, What we have done is design our Ultra card that not only expresses the card of interest, in this case, BRO-one, which is expressed on both hematological and solid tumors, but also that has Member in bound dialysis heal 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.

Speaker 2

We are excited and we have as you have seen with the press 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-three thousand and seven, 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 And finally, what I'd like to stress after our PRGN-three 10007 is regaining the rights for CD19 and VCMA. We are very excited about that for various reasons. Number 1, these are validated targets that already has shown that they can be very effective in these indications.

Speaker 2

At the same token, now we have shown that our platform of UltraCAR, UltraPorator platform. Now we have validated this across Various indications, both in hematological and solid tumors and the combination of these 2, we believe will Teams at Precigen has been moving forward rapidly and our aim is to be Phase 1 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 1st in a 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.

Speaker 2

And with that, this is why we are excited and moving these programs very rapidly. At the same token, As part of regaining the rights for our CD19 and BCMA, We also regained the rights for IL-twelve and especially our gorilla IL-twelve back with a control switch. And why is that important? In the past decades, the role of IL-twelve has always been considered as one of the best for IL-twelve. And we are, we believe, the only company who has now the highest amount of clinical data With the control switch with IL-twelve, this makes it very unique and absolutely Very appropriate for our portfolio, especially in combination with our current molecules for the future.

Speaker 2

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-twelve has become up and center. And now we have one of the most potent molecule under the same roof with our portfolio. We are excited about that and we will be discussing this in the upcoming in months and half of the year. So with that, I would like now to transfer to Harry to address our financial reports.

Speaker 2

Harry?

Speaker 3

Thanks, Alan, and good morning to all of you on the call. I appreciate your participation in our quarterly update. During the Q1, We continue 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.

Speaker 3

During the Q1, we repurchased an additional $29,500,000 face value of convertible notes at a discount to par. As of March 31, the remaining balance of our convertible notes was 13,800,000 which will be paid at or before maturity on July 1 this year. We will continue to utilize our restricted cash balance in the retirement of these securities. To date, we've retired $186,200,000 face value of our original $200,000,000 of convertible notes prior to maturity, which has saved the company close to $7,000,000 Secondly, when I started at Precigen approximately 1.5 years ago, we set a goal to reduce our G and 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 and A function costs.

Speaker 3

I am pleased to announce that our G and A expense has decreased by 15% in the Q1 of this year compared to the same period last year. We believe that our Q1 G and A spend approximates what we anticipate for the remaining quarters this year. 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 and 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.

Speaker 3

Turning to our cash Vern, our net cash used in operations for the current quarter was $18,400,000 versus $18,800,000 in the year ago quarter. Our cash burn was positively impacted by a reduced G and A and interest costs. These costs more than offset the positive cash flow in last year's Q1 of approximately $2,000,000 from our previously owned Transova business prior to its sale in the Q3 of 2022. In addition, we were able to increase our R and D spend, while still reducing our burn for the current quarter compared to the Q1 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.

Speaker 3

This is consistent with our previously provided guidance on our cash runway. I thank you for your support of Precigen. We'll now be happy to take your questions. Ryan, if you could let questions come in from the queue, that'd be great.

Operator

Thank you. We will now begin the question and answer session. Our first question comes from Jason Butler with JMP Securities. Please go ahead.

Speaker 4

All right. Thanks for taking the questions and congrats on the progress. 2 for me. The first one on PRGN-two thousand and nine. Can you give us some more details on the data you're going to present at ASCO?

Speaker 4

Will we see data from all enrolled patients here or subset? And How should we think about the duration of follow-up? And then secondly, for PRGN-three thousand and seven, Helen, can you maybe just give us some color on the mechanistic rationale for ROREL-one in solid tumors? Thank you.

Speaker 2

Thank you, Jason, for the questions. Absolutely. In regard to PRGEM-two thousand and nine, there will be a full data Presentation on the Phase 1 and also the expansion cohort for with the checkpoint inhibitor That's 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.

Speaker 2

As I mentioned, this patient population in the best Case scenarios, we have 15% response rate up to 18% in the head and neck. And this is by the way, there will be patients both cervical as well as head and neck in that cohorts 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-two thousand and nine to the checkpoint inhibitor in the same patient population that they have a stop 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 which our investigators will be addressing and presenting on a patient basis. In regard to PRGN-three thousand and Devin, I think this is really an exciting target because LORE-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 as unmet need for this patient.

Speaker 2

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 us unique. And the reason for the design is we know CARs, TCR, T, 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.

Speaker 2

And as a result, that needs to be addressed. A lot of the off the shelf conventional parts, 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 redose, so that's the issue. And secondly, they have to overcome this tumor microenvironment challenges. So with us, We have designed and our UltraVacters allows us to incorporate mechanism that we can directly Inhibit the checkpoint inhibitor just within our UltraCAR T.

Speaker 2

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, Keytruda 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 party. 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.

Speaker 4

Great. Appreciate all the details there, Helen. Thank you.

Speaker 2

Sure.

Operator

Thank you. Our next question comes from Jennifer Kim from Cantor Ms. Sherrill, please go ahead.

Speaker 5

Hi, everyone. Good morning and congrats on the quarter. I have a few questions here. Maybe to start with 3,007. I think I heard in your comments that you plan to present some interim data this year.

Speaker 5

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 hem, etcetera. And then my second question is, I think you also mentioned for 3,005 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.

Speaker 2

Yes, absolutely. Thanks, Jennifer. So in regards to the PRGEM-three thousand segment, maybe I should just sort of make that more clear. Currently, we are in a Phase I. 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 Phase 1 and expansion cohorts, we will be addressing in 2024.

Speaker 2

But the end of this year, We will give some color from perspective of how the Phase I is going and some of maybe a sort of a preliminary database. So that's in regard to the PRGN-three thousand and seven as far as the data is 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 I completely this year. This will be by all presented by next year. In regard to the PRGN 3,005 and the split dosing.

Speaker 2

And this is very Interesting because the data that will be presented in the at ASCO and because of the Embargos, 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 mechanisms that both it exists And when you give the intraperitoneal, the 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 2 arms. And by the way, it really points out in regard to our Ultra Car, how Our infusion makes a difference, but also on top of that, how the 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 We and lymphodepletion open.

Speaker 2

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 4, 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 intraperitoneal cavity, so you need to address that.

Speaker 2

But at the same token, you need to address the metastases that it's 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.

Speaker 5

Okay, great. Thanks for your color. 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.

Speaker 2

Yes, absolutely. So one thing, our PRGENT 2012, what I should mention is Really, the work that has been done has been outstanding because I just want to remind everyone that Phase 1 of this started in April of 20 21. Not only we reported on the full Phase 1 data and the expansion dose cohort In January of 2023, but we had started the Phase 2 basically clinical trial In 2022, and I want to stress if I 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 I and now have the 23 patients in a Phase 2, so the total 35 patients that has received the dose level 2 in a Phase 2, And we are really excited about that. In regard to the interactions with the FDA, we are in a continuous discussions are very productive discussions.

Speaker 2

Of course, our communication with Our shareholders and investors and outside has to be respected by the fact that finalization of and FDA giving us the complete answer. So We are in discussions. It's very productive and especially in a view that what Doctor. Marks has been mentioning in the past A few months in regard the new guidances and changes at OTAD and how the rare diseases especially are being treated for a faster and more innovative guidances that allows a 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.

Speaker 2

So as we have mentioned, we will be communicating as soon as We have the finalization of this 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 exciting year for us.

Speaker 5

Awesome. Thanks, Helen, and congrats again.

Operator

Thank you. Our next question comes from Ben Burnett from Stifel. Please go ahead.

Speaker 6

Hello, good morning. This Carolina Ivan and Ventoso for Ben Burnett. Congratulations on all the progress and thank you for taking our questions. Follow-up on the PRGEM 2012 program. I think, Arlen, you said in the in your prepared remarks that you plan to present additional results in a few months.

Speaker 6

If you could confirm that and then what can we expect in terms of number 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?

Speaker 2

Yes. Thanks, Catalina, for the questions. First of all, in regard to the PRG in 2012, There would be no further presentation of the data. We presented the full Phase 1 and the expansion cohorts of the Phase 1 In January, in totality of it, actually patient by patient, it's Very solid safety as we have shown as well as a complete responses, 50% responses. So the next basically data presentation, of course, will be in the context of the Phase 2 and when they have finished their full follow-up of that.

Speaker 2

As I mentioned, we are excited. We have finished the enrollment of the 23 patients in our Phase 2. It's and obviously, they have been 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 subcu and maybe A little bit of fever and aches for a day or 2, but nothing more than that.

Speaker 2

And in view of efficacy data, which is very unique, when you talked about not just reducing the number of surgery, which in 80 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 3 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 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 We didn't go just after the number of reduction in the surgery, but we have gone to the endpoint of complete a response with no requirement for surgery and for 12 months. So the durability is also very important.

Speaker 2

And of course, 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 has 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.

Speaker 2

And as soon as we have a complete strategy approved, we will be communicating that to the outside.

Speaker 6

Yes, 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?

Speaker 2

Yes. 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 a surgery, That will be an option for this patient population definitely.

Speaker 2

And of course, another part of the strategy and path Towards 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.

Speaker 6

Okay, excellent. Thank you very much for all the color.

Speaker 2

Sure. Of course.

Operator

Thank you. Our next question comes from the line of Arthur He from H. C. Wainwright. Please go ahead.

Speaker 7

Hey, good morning, Helen and Harry. This is Arthur on 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 physician or the patient need.

Speaker 7

And

Operator

I had a

Speaker 7

follow-up on that part as well.

Speaker 2

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 3,006, we are dealing with the patient population That they have 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.

Speaker 2

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 the clinicians and their oncologists that they can make that call instead of having a regimented that we say, well, every 4 months because a patient might not have 4 months in general and they might need 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 signs Early signs of progression, they can intervene accordingly. So that's why currently the Phase Ib, which It 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 The level of disease what it's required. And the same thing goes for PRGN-three thousand and five because again, I'm going to stress one thing.

Speaker 2

Of course, the goal of all of the therapeutic drug is eventually to move from a Stage 4, and we will be able to bring it in to the front lines that in reality, we don't allowed that the patients to get to a stage 4. We need to stop the disease much earlier. But Because these are investigational drug, of course, we have to start for a safety perspective in the state force. And where we are going with all of this in also our PRGN-three thousand and five, 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 7, 8 line of therapies.

Speaker 2

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 a basically different design for various stages or dosing. But right now what is important is number 1, we can redose.

Speaker 2

We are the only platform that can do that without tremendous costs and we can directly expand these cells And I think the data in for PRGN-three thousand and five in ASCO will show that.

Speaker 7

Sure. Yes. Just a follow-up on that part. So for the product used to be used for the repeating dosing, On average, how many doses you can get from 1 batch of the production for repeat doses.

Speaker 2

Yes. I think this are the of course, this Part of our manufacturing process, but I can tell you that we can, from the material that originally we can get, we can And then as later on, is needed to be used for more than number that we have. So currently, basically, with the original material, we have been able to redose.

Speaker 7

Okay. Thanks, Vinh. And my last question is on the regaining rights to the CD19 and BCMA target.

Operator

Could you give us a

Speaker 7

broader picture of what your strategy to for those 2 targets?

Speaker 2

Yes, absolutely. So our strategy from the beginning has been really We know CD19 works and 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?

Speaker 2

What platform do you use? And The cost. And we see that this is a struggle even for the biggest pharmas of the world. They are It's a struggle in order to get this to a patient with the price tag that not only few patient can afford this, but everybody So first thing for us was very important and

Speaker 6

a lot of people asked the

Speaker 2

question in regard to CD19 was to show our platform, 1st 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 modify 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.

Speaker 2

So we did that. And now after doing that across AML, ovarian and Dror1 positive indication, What we are know we can do is to bring the CD19 and BCMA. And why was important for us to regain these assets? Because we know with application of our 1, plus the validity of these targets, we can change the paradigm now. And as I said, it's not About just being 1st in a class or 1st to market, it's about the best in the class and then how you can affect the market.

Speaker 2

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.

Speaker 7

Okay. Thanks, Kiara, and thanks for taking my question. Congrats on the program. Sure.

Speaker 2

Thank you very much.

Operator

Thank you. Ladies and gentlemen, this concludes our question and answer session. At this time, I would Now I'd like to turn the conference back to Doctor. Sabzavari for closing remarks.

Speaker 2

Thank you. As I mentioned, this is really a very exciting time for Precigen and we are making significant progress in advancing multiple clinical programs. Each of the 5 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 a diverse oncology indications and rare diseases With 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 their trust in us and their faith and everyone at Precigen 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.

Speaker 2

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.

Operator

Thank you. The conference of Precision has now concluded. Thank you for your participation, and you may now disconnect your lines.

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Earnings Conference Call
Precigen Q1 2023
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