On Thursday, 08 May 2025, Inovio Pharmaceuticals (NASDAQ:INO) presented at The Citizens JMP Life Sciences Conference 2025. The company showcased its DNA medicines platform, emphasizing its stability and T-cell response capabilities. While optimistic about its lead program INO-3107 for recurrent respiratory papillomatosis, Inovio also addressed manufacturing challenges and financial sustainability.
Key Takeaways
- Inovio plans a rolling submission for INO-3107, targeting a mid-2025 PDUFA date.
- The company reported $94.1 million in cash, extending its runway into early 2026.
- Positive trial data for INO-3107 showed a significant reduction in surgeries for RRP patients.
- Manufacturing issues with a device component have been resolved.
- Inovio is expanding trial sites for INO-3107 to over 20 locations in the US.
Financial Results
- Cash Position: $94.1 million at the end of the year, with funds expected to last into Q1 2026.
- Cost of Goods: Varies with manufacturing scale; vaccines generally cheaper than therapeutics.
INO-3107 (RRP Program) Updates
- Regulatory: Rolling BLA submission to start mid-year; aims for acceptance by year-end.
- Manufacturing: Resolved issues with the single-use disposable array component.
- Phase 1/2 Trial Data: Significant reduction in surgeries, with 72% of patients seeing a 50% or greater decrease.
- Durability: Clinical improvement sustained over 2.8 years, with 50% needing no surgeries in the second year.
- Confirmatory Trial: Placebo-controlled, expanding to over 20 US sites.
Operational Updates
- DNA Medicines Platform: Highlights include stability and unique delivery methods.
- Manufacturing: Plasmid production at commercial scale; devices for IM and intradermal delivery.
- dMAb Technology: Demonstrated sustained in vivo production of COVID-19 antibodies.
Future Outlook
- INO-3107: Focus on confirmatory trials and regulatory approval.
- dMAb Program: Potential applications for other antibodies and diseases.
- INO-3112 (Oropharyngeal Cancer): Targeting high-risk HPV-driven cancer patients.
Q&A Highlights
- RRP Prevalence: Estimated patient numbers are likely underestimated.
- FDA Interactions: Consistent engagement, with guidance to file based on Phase 1/2 data.
Inovio’s strategic focus on its DNA platform and INO-3107 program was evident throughout the conference. For a deeper dive, refer to the full transcript below.
Full transcript - The Citizens JMP Life Sciences Conference 2025:
Moi, Conference Host: Alright. Thank you again everybody for joining us here at the Citizens Life Sciences Conference. So next company we have is exciting, Inovio Pharmaceuticals. I I think they’re still on track, to have the first DNA immunotherapy approved in The US. You know, mRNA gets a lot of attention or has gotten a lot of attention, but there’s some very differentiating aspects of DNA.
I think most recently demonstrated with their, dMAb monoclonal antibody durability data that we’ll hopefully get into. But we’re happy to have CEO, Doctor. Jackie Hsieh, and Doctor. Mike Sumner, Chief Medical Officer here to present the story. So maybe Jackie and Mike start Just briefly review the the platform, how it differs from these other platforms like mRNA or viral platforms may have broad you can go with the DNA.
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yeah. Great question, Moi, and thanks very much for the invitation. Nice to be here today. So when you think about DNA medicines and RNA medicines they’re both nucleotide based therapies, but there are actually quite a lot of differences between the two platforms. So the mRNA molecules are inherently
DNA is much more stable and they’re also delivered in different ways so mRNA requires lipid nanoparticles for delivery and to protect the mRNA from degradation whereas with DNA we use our proprietary delivery systems called CELLECTRA. So mRNA can be quite dependent on ultra cold chain for distribution, DNA being much more stable we don’t require any frozen shipping or storage. And then when you think about the kinds of immune responses that mRNA generates versus the DNA medicines platform, mRNA is very good at generating very high ultra ultra high levels of antibody especially neutralising antibody. DNA is very good at generating T cell responses particularly CD8 T cell responses and then when you contrast the DNA platform with viral vectors, obviously with the DNA platform we don’t need to worry about any pre existing anti vector immunity or generating anti vector immunity as part of repeated dosing, so that’s a key differentiator there. And when you look across both mRNA platforms and viral vectors, another key difference with the DNA platform is that we don’t have those kind of flu like adverse events that you can get with the mRNA and with viral vector administration.
So those are some of the key differences.
Moi, Conference Host: Okay, great. And based on the data that I’ve seen, I’d put the difference in durability between DNA and mRNA, know, a couple orders of magnitude hundreds. Is that is that fair?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: So so when you look at the durability of the protein expression, I mean, as we’ve recently shown with our most recent dMAb data, we can get much more sustained in vivo protein production of the different proteins that we’re encoding with our DNA medicines Whereas with mRNA, you’re getting much more rapid turnover.
Moi, Conference Host: Right. Okay. So it’s not just a 10% difference or 50% difference, multiples. So just sticking to the platform, couple more details. Do you know lipids are used in the injection of the DNA?
Is it just saline?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: That’s right. So when we’re administering our DNA medicines, it’s literally just the DNA in plasmids, which are circular molecules of DNA that we insert our optimized gene sequences into and then some salts and water. So no lipid nanoparticles associated.
Moi, Conference Host: Okay. Then how many patients total have received an injection? What’s the safety and tolerability profile been so far?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yeah. I’ll let Mike talk about that. Mike?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: So we’re now up to about 19,000 administrations and 6,000 subjects and we’ve actually been fortunate also to conduct some placebo controlled studies with our platform and really we see beyond some mild tolerability associated with the administration. We really do not see any differentiation between the placebo reported adverse events.
Moi, Conference Host: Okay, great. There’s a couple more platform and then we’ll get into 3,107. What are the general cogs for the manufacturing cost maybe excluding the device. And then device can be used multiple times. Right?
So if you had a flu clinic and you were doing flu vaccinations, they could use it all season and multiple seasons. Correct?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yes. So let’s talk about first of all, let’s talk about the drug components. So our DNA medicines platform, we have commercial scale manufacturing for our plasmids conducted at various CMOs. We’re manufacturing lots in the tens of thousands of doses So it really depends on the scale of manufacturing as to what the costs of goods are. If we’re going for a vaccine indication, clearly we need to get the cost of goods down much lower than we would for a therapeutic indication.
So cost of goods is really dependent on the scale of manufacture. And when it comes to the device, we have a suite of different devices. For our lead program 3,107 for recurrent respiratory papillomatosis, we’re using our I’m delivery device where we’re delivering the plasmids usually to the deltoid muscle. But for vaccine indications where we’re looking to generate more of an antibody response as opposed to a T cell response we tend to use intradermal delivery. Both devices are able to be used both our I’m devices and our intradermal devices are able to be used multiple times.
We use single use disposables which are the patient contacting piece and you’re just changing that out with each administration.
Moi, Conference Host: Okay, great. Alright, let’s get into INO 03/2007. You mentioned that you you said the full name, so say it once, don’t need to say it again. RRP, indication. So maybe let’s, Jackie and Mike, if you could discuss the RRP, is the indication, what causes it, the unmet need, prevalence in The US.
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Great, maybe I’ll kick off there. RRP is a rare disease of the respiratory tract. It’s caused by HPV six and eleven and it generates these kinds of wart like growths or papillomas, primarily in the larynx and the vocal cord. The way the sort of disease manifests is this affects patients’ ability to talk, it can affect breathing and in some cases you get dissemination of the RRP lesions down into the lungs as well. That unfortunately has a low rate of malignant transformation and can be pretty fatal.
Patients who are undergoing treatment for RRP, the standard of care is repeated surgery. So an RRP patient’s life is really problematic. You’re constantly wondering when these lesions are going to come back then you’re having to undergo surgery to remove them and then go on to voice rest and then you’re going into repeated cycles of this. And every surgery for an RRP patient comes with both risk and cost. There’s the risk of permanent damage to the vocal cords and by the time you’ve had 10 or more surgeries eighty to ninety percent of patients have had some form of permanent damage to their vocal cords.
Then obviously the cost in terms of quality of life, economic costs etc. So it’s a really difficult disease for patients to deal with.
Moi, Conference Host: Okay and then I think the data that we’ve seen on the prevalence suggests about fourteen thousand active patients. That data is a little bit old. Do you expect that number is higher, lower now? Has Gardasil had any real impact? Is it do you expect it to have impact going forward or is that kinda stabilized?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yes. So the the fourteen thousand came from a a survey that was done quite a while ago. It is relatively old data and we think it’s probably an underestimate of the instance of RRP within the population. In terms of HPV vaccination, what we’re actually seeing here in The US is levels of HPV vaccination holding pretty steady, certainly not increasing currently. The majority of adults in The US are still unvaccinated So unfortunately what that means for RRP according to HPV experts is in the adult population we’re not going to see a decrease in RRP cases for at least a generation to come.
And when you look at some other HPV related diseases such as HPV related throat cancer, the incidence there is actually going up. Currently about twenty thousand new cases of HPV sixteen and eighteen related throat cancer diagnosed each year in The US, expected to go up to thirty thousand cases by the end of the decade. So unfortunately RRP is going to be with us for a long time to come.
Moi, Conference Host: Okay. And then you you guys are almost ready to submit the the BLA. I think are you still on target to have that potentially accepted by year end? I guess, what’s gating for that? Can you request a rolling review?
Can you request that now or do you need to wait for these stability studies?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Mike?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: Yeah. So what we guided on our last call was that we will target commencing rolling submission in the middle of this year. The reason we are waiting till then is obviously we are currently completing a design verification testing for the device and when you’re dealing with the FDA they appreciate a very defined time table so that they can schedule their resources for the review. So as soon as we are certain of the timing of the testing, we will request rolling review. All the non device modules are ready to be filed and we are targeting to get the file accepted by the end of the year which would give us PDUFA date hopefully in the mid year, next year with priority review.
Moi, Conference Host: Okay, great. And maybe you can discuss the prior that you had a prior timeline delayed a little bit due to this manufacturing issue. Maybe just discuss what the issue is, what you need to do to address it and kind of where you are in that process.
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yes. Last year, we identified a manufacturing issue with the single use disposable array component of our device. That required us to to go back and the issue was in relation to an injection molded plastic component of that array. We had to go back and basically strengthen that plastic component within the array and then we had to conduct testing to show that that fix was appropriate and resolved the problem. We announced back in our last earnings call back in March that we believe we’ve resolved that issue.
We’re now completing the combination testing of the array and the device together, which is the data we’ll need to submit as part of our BLA submission.
Moi, Conference Host: Okay. Great. And that’s a third party doing that testing. Right?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yes, it’s a third party doing that BLA testing also, although some of the testing is performed in house.
Moi, Conference Host: Okay. Alright. Maybe let’s talk a bit about the so you have a phase one, two that’s been completed open label. Just what did that trial show you and how did it inform the confirmatory study that you need to do? And maybe let’s get into the, at some point, the confirmatory trial design too.
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: Mike? So yes, our phase one two study we recruited 32 patients across eight US sites and they actually had a median of four surgeries in the preceding fifty two years coming into the study with a
Jackie Hsieh, CEO, Inovio Pharmaceuticals: two weeks.
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: 50 two weeks, coming into the study and so with a range of two to eight, significant burden of illness coming into the study. We then gave four doses over a nine week period and we count every single surgery from day zero against efficacy because as you heard from Jackie, every single surgery is a meaningful event to these patients. And what we saw was a statistically significant reduction from a median of four down to a median of one and seventy two percent of those patients actually saw a fifty percent or greater reduction in the number surgeries that they had.
Moi, Conference Host: Okay, great. And you did not mandate a surgery before the first dose of 03/2007, is that correct?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: So they had a clinically warranted surgery so it was part of the normal disease management of these patients and that was counted again in the preceding fifty two week period.
Moi, Conference Host: Right, okay, got it. Okay, understood and then maybe discuss, so before we get into the confirmatory trial and what you’re thinking there, you have a pretty differentiated design. The durability recently showed some durability out through I think it was up to three years. So maybe discuss that. Maybe surprisingly, the data actually looked to get better.
So maybe just tell us what that showed.
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: Yeah, absolutely. So we were able to go back and look at, get data on twenty eight out of the original thirty two patients and as you said, we now have data out to a median of two point eight years in that patient population. And what we saw was continued clinical improvement and I think that links to the fact as Jackie talked about, we get that protein continued We have a very robust immune response that we documented at the fifty two week period. And so it’s not really surprising that these patients continue to improve.
And what we saw was that the patient numbers that saw a fifty percent or greater improvement now increased up to eighty six percent from seventy two percent and fifty percent of those patients required zero surgeries in that year two period.
Moi, Conference Host: Okay, great. Yeah, so complete response rate went from twenty eight percent to fifty percent, Okay, and I think there were four patients lost to follow-up. Why were those lost?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: So two were not back in contact with their physicians, which as you can imagine in a rare disease that hopefully means that they’re doing well and they didn’t want to come back, and two patients did not want to consent into the study.
Moi, Conference Host: Okay, great. Do we have enough natural history data out there? Was this a pretty representative population going into the trial and what are you planning maybe to discuss the confirmatory trial? So you and Precigen both need to start the confirmatory trial before submitting the BLA, correct? Maybe discuss your trial design and how it’s a bit different.
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: Yes, so just answering your first one, we actually have previous data on the patients into our trial from three years and so we’re now actually able to look at that three year period and compare it to the two point eight years of data. And we’re in our year two, we’re seeing a seventy five percent reduction in the number of surgeries. So now however we look at the data, we’re seeing a significant impact on the disease. And so when we started discussing our confirmatory study with the FDA, they really gave us two options. They said if you want to mirror the population that we had in the original study, I.
E. Two or more surgeries, then you need to run a placebo controlled study and so it’s clearly a benefit to us to have a broader population as we look to bring this product to the market. But also we were in discussions with the European regulators and they were very clear that we needed to bring to them placebo controlled data to get approval in the future.
Jackie Hsieh, CEO, Inovio Pharmaceuticals: And just to add to that, when we looked at the patients being enrolled in our phase onetwo study, we have patients who’d had between two and eight surgeries in the prior year with a median of four surgeries and that seems to align very well with the disease severity that you see across RRP. Also very importantly we had a mix of both HPB6 patients and HPB11 patients, even a couple who were co infected with both HPB6 and HPB11. Gender balance again very similar to the population which you see with RRP which tends to skew male. We believe our phase onetwo population was very representative. We conducted that phase onetwo study across eight clinical sites so again a good different representation of the different standards of care.
Moi, Conference Host: Do you plan to use the same sites for the confirmatory trial?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: You’re sorry, forgot. You also asked about our plans. So we actually plan to expand our clinical trial site base. We’re actually looking at 20 plus US sites. Over half of those are already under contract and several have IRB approval.
So when we’re in discussions with the FDA, mean the goal of them saying start a confirmatory study is really to demonstrate to them that we can meet the commitment of delivering the data that they need for confirming our efficacy in a timely manner. So I think we have a very robust plan and actions to demonstrate that we will do that.
Moi, Conference Host: Okay, great. And how is the, in the trials, how do you make the determination to do a surgery? Is that the investigator? Is there any kind of central review for this and is it going be the same for both trials of phase one, two and the confirmatory?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: So we obviously did document the evidence behind why surgery occurred in our phase one two and they were all linked with growth of papilloma. Taking that data we’ve obviously refined how we’re going to record the need for surgery, but there is always a personal element between the surgeon and the patient and it can be dependent on where that papilloma grows. I mean for me if I had RRP and I had a single papilloma on my vocal cords that was impacting my voice, I would want that operated on. If I was somebody who was at the end stage of my RRP and I already had that permanent vocal cord damage, your sensitivity around the impact on your voice is going to be less. So it really has to be patient and surgeon specific at the time based on the symptoms that that patient is having.
Moi, Conference Host: Okay, great. Yeah, I think the placebo controlled bit didn’t mention earlier, great with the new administration and their commentary. Have you had any interactions with the new people at FDA, if any are dealing with you and any changes in your interactions?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yes, so our interactions with the FDA have been pretty similar so we’re not seeing any changes currently.
Moi, Conference Host: Okay. Way back in the program, it basically sounded like FDA told you to file this on the data that you have, right? This was kind of surprisingly to me, guess, I don’t know what the right word is, not lenient but accommodating from the agency.
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Well we were very excited with our phase one, two data. In discussions with the agency they gave us breakthrough therapy designation and then they called us up as you said and said actually we think this stage is good enough to file under the accelerated approval program. So we’ve been working closely with the agency since then and we’re very excited about this path.
Moi, Conference Host: Okay. Great. I have more questions, but maybe people have to we’re running out of time, get to them in one on one. So I want to talk a bit about the dMAbs. I thought that data was pretty amazing actually.
Recently, it had a publication for some COVID antibodies that, you know, have already actually been used in their antibody form. So maybe just discuss that platform, what you can do and what the data showed us.
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yeah. So this is some of our really exciting next generation DNA medicine technology. And what we were doing in this proof of concept trial was we were expressing two different monoclonal antibodies against COVID-nineteen within the same patients and then we were following production of those monoclonal antibodies in the circulation showing that they were functional and showing that we weren’t generating any anti drug antibodies and we were able to show sustained levels of production of the monoclonals and presence in the circulation out to seventy two weeks. We saw a dose respondent level of production as well and we didn’t see evidence of any anti drug antibodies in more than a thousand blood samples across the participants. So very excited by that proof of concept, technology.
Clearly what it means is we can apply that technology to other monoclonal antibodies, but we can also apply the technology to protein replacement diseases because at the end of the day what we’re doing is producing proteins within the body. They can be therapeutic proteins such as monoclonal antibodies or they can be other kinds of proteins for instance to address protein replacement diseases. So we’re very excited by the prospects of that technology and what it could mean for patients.
Moi, Conference Host: What do you expect to advance next out of the dMAb or protein replacement programs?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yep, so we have another number of different candidates in development. We also are talking with some potential partners and clearly we’ll we’ll look to move those on as best we can. However, the vast majority of our resources are clearly focused on getting INO-three thousand one hundred seven across the finish line and that’s really where our focus is at the moment.
Moi, Conference Host: Yeah, makes sense. Okay, may I discuss quickly INO-three thousand one hundred twelve for oropharyngeal cancer partnered with Coherus Lacturzi. Yeah, just what’s next for that program? Where is that at?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: Yeah, so we’ve been in discussions with both the FDA and European regulators. We’re aligned on the patient population that we are going to be studying, is patients with local regionally HPV driven sixteen eighteen throat cancer and we’re focusing on the high risk population and these are the patients that the surgeons look at them and they say that they know they’re going to relapse it’s just a question of when and that’s based on their tumor size, nodal involvement, their extensive smoking history and so what we’re going to look to do with the combination of three thousand one hundred twelve and the anti PD-one Loptozzi is to try and prevent that relapse in those patients because once they do relapse they have very poor outcomes with sort of overall survival of around twelve months.
Moi, Conference Host: Okay, about how many patients is that in The US and when do you think you might start the phase three?
Mike Sumner, Chief Medical Officer, Inovio Pharmaceuticals: So I’ve talked to the patient size. HPV driven throat cancer is now the most frequently diagnosed HPV driven cancer. They’re estimating thirty thousand patients by the end of the by 2029. So we’re estimating that high risk population is currently somewhere around three thousand four hundred patients. Each year?
Each year. Okay.
Jackie Hsieh, CEO, Inovio Pharmaceuticals: And in terms of when we start the study as I said we’re putting most of our resources into getting thirty one zero seven across the finish line. Clearly, once we’ve accomplished that, then we’ll start moving the rest of the pipeline on.
Moi, Conference Host: Okay. Last question, the cash position and your runway?
Jackie Hsieh, CEO, Inovio Pharmaceuticals: Yep. So at the end of the year, we had 94,100,000.0 in cash. That cash runway takes us into the first quarter of twenty twenty six.
Moi, Conference Host: Okay. Great. Okay. Thank you, Jackie. Thank you, Mike.
Appreciate it. So Thank you.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.