Try keeping the trains running on schedule. Good morning, everybody. My name is Jon Wolleben , analyst here at Citizens, and welcome to the first day of our Life Sciences Conference for 2025. We're pleased to have DBV Technologies joining us and CEO Daniel Tassé. So, Daniel, thanks so much for joining us today.
Happy to be here. Thanks for having me. Good morning, all.
I think this is, you know, a name people may be familiar with, but just in case someone's new to the story, tell us what you're working on at DBV Technologies.
DBV Technologies is a company working in food allergies in the pediatric population. The core, the magic of our technology, of the platform, is the fact that we're exploiting the very powerful immune properties of the human skin. We don't always think about the human skin as a powerful immune organ, but it is. It's possible by exposing Langerhans cells on the back of children to an allergen to, over time with a great safety profile, desensitize the child to that allergen if they happen to be allergic to it. The founders of the company focused on food allergies first, which made sense.
Of all the food allergies, picked peanut first, which also made sense, given not just the prevalence of peanut allergy, but the fact it's one of the most difficult ones to live with because your last reaction is not predictive of your next reaction. There's no such thing as kids having light or not serious peanut allergy. That does not exist. That's our lead product deep in phase 3. Very pleased with the progress we've made over the last few years.
We've been covering.
You have been the word.
Yeah, we've been covering the story. We've got to keep this kind of abridged.
Yeah.
We've been covering since 2016. You had initial phase 3 failure, I think, in 2019, 2020.
2019, yeah.
Can you give us, like, a high level of what's happened since then? Because there's also been wrinkles every couple of months in the last year or so, and all productive, but it's been a long road to get to where we are today. Maybe can you talk a little bit at a very high level kind of the history of the program?
Yeah. The original pivotal trial in children 4 - 11 with peanut allergies missed its primary endpoint by a hair. The FDA wants a 15% delta to the lower bound of confidence interval between active and placebo. The company that was the previous management, if I may say that, hit 12.4, had reason to believe the product would be approved anyways. That was not an unreasonable position. Submitted the product, and the agency gave the company a CRL and asked the company to redesign the patch, our product being a patch, obviously. It should say, obviously, since we're exporting the skin, it's a patch. And to redesign the patch so that it essentially would adhere better to the patient's skin.
That was the genesis of what's been the discussion in the last few years that has been resolved last year to our satisfaction, the agency's satisfaction, mostly to the great benefit of future patients. It's to understand that adhesion does not matter. It's wear time that matters. Be optimized to get the right clinical response. We understand that much better. We've agreed with the agency on a regulatory pathway in 1 - 3 and in 4 - 7, where all of this is quantifiable or quantified already, giving us a very clear regulatory pathway. There were many years in the working, coming from the original observation from the agency, that the company disagreed with. There was nothing wrong with the patch. That being said, we redesigned it to satisfy the agency.
We're now at a place where the product is well understood by the agency, well characterized, and the regulatory pathway is clear, straightforward, and we like to think as low risk as you can have for a phase 3 program.
The original.
Is that a good summary of those six years?
Perfect, because then we can jump into some of the details. Because the original study is 4 - 11.
Yeah.
In the meanwhile, there was a trial ongoing in 1 - 3 year olds. That data came out and looked phenomenal. That kind of changed the plan a little bit, right? Now you're going after 1 - 3 and 4- 7. How did that come about?
That was actually why we were confident. You're asking the most critical question about the regulatory history here and the clinical reality of the product. With all forms of allergenic immunotherapy, younger patients respond better for obvious reasons. They have younger, more plastic immune systems. So all forms of immunotherapy work better in younger kids. When on top of it, using the skin, the thickness of the skin makes a difference also. So younger kids respond better. When the agency asked us to redesign the product in 4 - 11, we analyzed 4- 7 versus 8- 11. We split the population into seeing the younger patients respond better, no surprise. On top of it, the trial in 1 - 3 year olds, which we expect was going to be a home run, was enrolling at that point in time.
We were quite confident that we would show success in 1 - 3, which we did. That put us in an interesting situation with the agency where, again, I think they were smart. We said, well, we started doing work on the modified patch in kids 4 and older, but we just showed you that the younger kids respond better. Moreover, of the 4 to 11 original population, the younger kids 4 - 7 are also better responders. Can we track two separate BLAs, one in 1- 3, one in 4 - 7, with two different patches? They're slightly different. The agency agreed to that because it was a very elegant way out of a situation that we had sort of co-created here. We have two BLAs, 1 - 3, 4 - 7, although the patches are quite similar.
That serves us well also when it comes to just risk management and obviously investor appetite here. If one program goes faster than the other, not having a BLA, supplemental BLA, sort of subordinate value creation situation when it gets spot here to file both BLAs next year.
Do you need a different brand name then for both products?
We will have different brand names. We have to. We'll have different NDC codes. So by law, we'll need different brand names.
Potentially different pricing, which is also important.
That's also important. Yeah. We have the flexibility of having two brand names and with that, obviously, two pricing strategies, two sets of pricing tactics. Once we unblind the data in 4 - 7 year olds, which, as you know, is going to be Q4 of this year, the data in 1 - 3 we have, it's been published in the New England Journal of Medicine, so that's well known. We can then assess the most attractive value proposition and then decide what's the right price point for both of them. We may choose the same price point, but it's good to have the luxury of diving deep into the data and making the best decision in the interests, obviously, of patients and shareholders.
You mentioned, you know, clear development path now in both programs. You know, where are we today?
Okay. In one to three, well, let me start with 4-7 . The first file to go in will be Viaskin Peanut for children with peanut allergies, age four to seven. 4-7 , to be clear, is four to seven at the age of treatment initiation. You do not have to come off the patch at the age of eight or nine. You can go on to three. We have 18 year olds who have been on the open label extension for many years now. That program, the pivotal trial VITESSE, is reading out in Q4 this year, 2025. The BLA will follow shortly after that. We are guiding the BLA filing in four to seven in the first half of 2026.
In children one to three, the pivotal trial showing efficacy and safety read out a few years ago was published in the New England Journal of Medicine. The FDA is very satisfied with the efficacy. It was a smaller trial, so the agency wants us to have a supplemental safety study. Again, there's no safety signals being chased here. Nothing's been observed that the agency wants us to investigate or validate. That's not the question. Their guidelines have been 600 patients on active for at least six months. We don't have that. We have 244 patients on active for one year. We are running a small 480 patient safety study. The enrollment will start this quarter. That should read out in time for us to file a BLA in one to three year olds towards the end of 2026.
A couple, I think, nuanced points here. The 4 - 7 year old VITESSE trial, you're going to have a double-blind placebo-controlled food challenge. Is there a requirement for efficacy? Because originally it was talking about adhesion and the interplay. You know, what do you have to show? Is there clinical?
In 4 - 7, we would.
4 - 7.
Yeah. We have to hit the primary endpoint, which is, again, the delta in eliciting dose baseline versus exit at 12 months. This is how we assess kids' ability to tolerate peanut protein as you give them peanut protein at the start of the study, see at what dose they react, makes them sick, just short of anaphylaxis. After one year of treatment, you do the same test and see how big the delta is. The delta, as you know, needs to be quite significant to be responders. No, only thing we have to show in VITESSE, we hit the primary endpoint and obviously have a decent safety profile. We got a lot of safety data with the technology. We do not expect that is going to be an issue. There are no other primary, co-primary endpoint or any other architecture.
No adhesion.
No. Adhesion would be assessed and form a label. It's not an approval criteria.
You've done a good job stacking the deck, so to speak, for VITESSE versus the original PEPITES trial. Can you talk a little bit about the changes you've made?
Yeah. The guy who would be sitting here.
Faris.
Faris is the one who did all that nice work. He's my Chief Medical Officer. We were smart about two things, and we got lucky on two things. Smart is four to seven year olds respond better. By digging into the 190 patients that were aged four to seven in our original trial that had 360 in four to eleven, we got a chance to really understand who responds best. We made a decision to target children who are particularly allergic, so have low EDs at baseline. It does not take much peanut protein for them to come into anaphylaxis, which also makes for a better label. If the sicker kids, the most at-risk kids are the best responders, why not enroll these children in your trial?
We did that by going to younger patients and going to younger patients and more sensitive patients, which is going to make for a better label. That's the engineering. It's probably better than stacking engineering part of that trial.
Sure.
Where we got good fortune coming our way is through the randomization, which was obviously just completely blinded. We have more 4 and 5 year olds than 6 or 7 year olds in the trial. That is good because, again, younger kids tend to respond better. Of the children who are sensitive, the ones with low IgE at baseline also tend to be the better responders. We happen to have a slightly lower median IgE in the population we had originally. It seems that the patients we have enrolled, we were targeting the more sensitive patients. Through the luck of the draw, we got even more of them than expected. The trial at 600 patients was powered, 90% powered to pick up the delta between active and placebo. We have enrolled to 654. Plus these good fortunes, we like to think that our probability of success is significant.
Again, we can't promise you that we hit primary endpoints. Obviously, we will have to wait for the unblinding in Q4.
Q4 this year.
Yeah.
Then the.
Does that answer your question?
Yeah.
Yeah.
Yeah. No, that nailed it. COMFORT Toddlers, the 1 - 3 year old safety study. What's interesting there, if I recall, is you're going to require a double-blind food challenge for entry, which we would think is hard. Like, I don't want to have my 1 year old go through a food challenge. Talk us through why that's not going to be a hindrance and how you're thinking about enrollment timing.
It's a small hindrance. It's not a critical hindrance. Obviously, without having your food challenge, the enrollment is easier. The food challenge, as I described to you, causes anxieties in families. You have your child who every day of your life you spend making sure they avoid eating peanut. You sit them there in a chair, you feed them a milligram of peanut protein, you wait 20 minutes, there's a reaction. You feed them 10, 3, and 10, and 30 until they're sick. It's not a fun study to run. The fact, though, that parents will bring their kids to participate in those trials tells you about the unmet need. There's a need for treatment out there because parents want to participate in all of this. Those food challenges are stressful.
If you have to do two at entry and exit, obviously even more so. Having only one at entry in 1- 3 year old, obviously, is less stressful than having entry and exit. Food challenges in younger kids are a bit easier to do, quite simply, than older kids, simply because older kids are more mobile, they're more likely to complain. They're just, some of them have learned to play the game also. Food challenges at entry in 1 - 3 year olds tend to be less difficult to do. The reason we do them is since we're trying to enrich the safety database, we want to mimic the very same population we had in the efficacy trial where there was a food challenge at entry.
Instead of using wheel size, history, and IgE as the markers of peanut allergy, as would be the case in most safety trials, in this one, we chose to have the food challenge at entry to ensure that the population in the supplemental safety trial is quite similar to the one we had in the efficacy trial.
To try and replicate the safety you saw previously.
That's exactly it, which was, as you know, superb.
Given the, you know, yes, a five-year follow-up now from some patients, would there be any safety concerns in broadening that 1 - 3 year old population, you think, outside of what you saw in EPITOPE?
There wouldn't be. There wouldn't be. I think the smarter part here of that on our part is, although there's been no safety signal with the product, kids have been on treatment for five years, as you know, the adverse events, there were no serious adverse events in years 2, 3, 4, 5. We've seen no anaphylaxis on treatment. Skin irritation, which is the most common adverse event, tends to be less frequent and less severe with time. The kid's skin obviously adapts to the therapy here. There's no safety issues. Still, since FDA prefers randomized clinical trial for the safety study, instead of putting a lot of weight on the open label extension, which is open label by definition, to run a placebo-controlled safety study in a similar population, stroke testing being the smartest way to go, although we were not chasing a safety signal.
How does accelerated approval factor in then?
Yeah. So in one to three year olds, accelerated approval. In four to seven, just straight BLA. Because the original efficacy trial, the one that was published in the New England Journal of Medicine, was originally supposed to be a supplemental BLA in four to eleven, so the study was smaller, the agency has asked us to do a confirmatory trial. We've also, along the way, changed the scale of our API manufacturing since it's a natural product, as you know, we're extracting peanut protein from Virginia peanuts. Since we changed the scale of manufacturing between the patch that was used in the development program versus the ones we will be commercializing, the FDA is asking us to do a confirmatory trial post-approval. So one to three is an accelerated approval pathway.
We're going to pull together the safety study with the commercial patch, the efficacy study with the development patch for the approval, and then the confirmatory study will be with the commercial patch in 1 to 3 year old.
Do you have to start that before approval?
It has to start before approval and have the regulation see some momentum by the time the product is approved to avoid companies essentially not pushing hard enough to get the results.
Usually when we hear accelerated approval, we're thinking about some confirmatory endpoint. What does the primary look like in the confirmatory trial?
It's the same, which is, again, why I want to recognize the FDA being quite constructive here in finding a solution. They knew the patient community wants the product. There's been a lot of demands made by patient advocacy group, by patients, by Congress, by experts to get the product approved. This was an elegant way for them to respond to that so that we don't have a higher bar for the confirmatory study because that's usually progression-free survival to survival is a higher bar to use the example from oncology. We have the very same bar. We have to replicate the study we did with the development patch, with the commercial patch. Be about the same size, same primary endpoint, no higher bar anywhere. It was an elegant way for the agency to deal with the dynamic of the product is needed.
It's been very long, but they need the data. They need on what would be the commercial grade of API manufacturing. I thought it was a very elegant solution.
I don't think I've heard. So you have peanuts from Virginia. You ship to Paris to put on patches?
No, we send them to the South of France first before we send them to Paris.
I didn't know that.
Yeah. It's Virginia peanuts, out of which we send them to Santa Fe, which has an API manufacturing facility in, name of the town will come back to me. It was in the South of France, I promised you, right on the water, where they turn that into our API. The API is sent to a contract manufacturing organization by the name of Fareva. It's a big, big European CMO. That plant is about two hours south of Paris.
Got it.
This is where they made the final patch.
The one other clinical thing I want to talk about is the label in, label out.
Yeah.
Dynamic because the data look compelling, but why was that important and what does it mean for approval and commercial use?
Yeah. Actually, it turned out to be a way to look at the product that allowed the agency to understand, data in hand, that their questions, which are very legitimate about wear time and patch falling off, because some kids, the patch will fall off because you're putting a patch on the skin of an active 4-year-old, on the skin of a 4-year-old who's allergic to peanuts, and on that patch is peanuts. So guess what? Sometimes it gets red and it gets irritated. The question was asked by the agency, which was a great progress question. This is about a year ago, was, what should the label say if the patch falls off a child sometime after 12 hours or 21 hours? What should the label say about that because it's going to happen?
The agency said, this is why you may want to collect more adhesion data in your safety trial. We said, well, that does not make a lot of sense because if you disconnect adhesion from efficacy, you're not answering an interesting question here. We can dive really deep with the data and wear time data we have from our efficacy trial to answer that question because we think the label should make it clear to patients. If you can achieve the following wear time experience, you're going to respond well. If you don't, may not respond quite as well. The doctor and the parent should know that. Which is how the label in, label out came to be. We look at the optimization function of clinical response as a function of wear time in children 1- 3 from the study published in the New England Journal of Medicine.
It showed that pretty much bimodal, 70% of kids wear the patch for pretty much 24 hours a day, day- after -day, every day. For them to wear the patch is a non-event when it comes to adhesion. For about 30% of kids, the daily wear time varies greatly. It could be 24 hours one day, 12 hours the next day. Those kids are high variability. What do they need to average to be good responders? Towards the end, it was 20.3 hours. That is what we told the FDA is there should be a discussion between the parents and the physician after 60 or 90 days of treatment. We recommend 90 days to say, if your child can wear the patch every day, you are going to be actually a super responder. You respond even better than your overall population.
If your child is struggling and cannot average 20 hours of wear time, they may not respond as well. You should have that conversation with them. You want to continue on therapy, except the clinical response may not be as robust, or maybe you want to move on to something else instead. The FDA loved that because we're using data from a randomized clinical trial to answer an important question about labeling and one that we know parents will be asking. That data set turned out to be pretty much the breakthrough in the discussion with the agency that it's not about adhesion, it's about wear time. Wear time is bimodal. For most patients, it's not an issue at all. For those who have a bit more struggle, data can elucidate what should be expected for best clinical response.
We got the 4 - 7 year olds, data coming for Q, BLA submission first half of 2026. Where are we on 1 - 3 year olds? You got the COMFORT safety study kicking off soon.
Yeah.
When could that data come, BLA submission follow?
We're aiming to file that BLA late in 2026 on 1 - 3. It's a lot of work, but we think we can get it done. Faris and his team are very good at running trials in this space. We expect to recruit those patients in a bit less than a year, six months of assessment. Everything else about the BLA will have been pretty much put in place at that point in time. We're going to learn a lot from our discussions with the agency about 1 - 3. That's why about 4 - 7 has to make the BLA sort of assembly process to be a bit more efficient the second time around. We're aiming for late 2027, late 2026, sorry, for 1 - 3.
What's the prevalence in these two age groups in the U.S.?
Per year of birth cohorts, about 100,000 kids per year are diagnosed with a peanut allergy. The exact numbers or the best estimation we have is 280,000 kids age 1 to 3 in the US have a peanut allergy, 390,000 kids age 4 - 7, making that seven-year population to be 670,000. It is a very large population. Again, to repeat what I said earlier on, 1 - 3 and 4 - 7 is the age that the patient needs to be to initiate treatment. There is no need to switch over on your fourth birthday from one patch to the other or drop off therapy at the age of 7. To be clear, a lot of the OLE data would be bringing the agency to support the BLA would be in kids who have aged out of those groups, obviously, through our open label extension studies.
I wanted to dig in a little bit more, but we're bumping up on time on 4 March.
We are okay.
I think a good one to end on is your recent financing and what does that give the company? Can you talk a little bit about that transaction? Because it was fairly complex, but you got something done that most companies aren't able to do in this environment. Talk about kind of cash runway and where it gets you.
Thank you. We raised $125 million in the first tranche of financing. That money was raised in early April. With that comes warrant coverage for $181 million. Those warrants are triggered if VITESSE, the efficacy trial in 4-7 year olds, reads out to be positive in the fourth quarter. Those warrants are two-year warrants, get to be accelerated. Investors will then have 30 days to exercise the warrants. It's at the same price as the first tranche was. It's a good deal. Again, we had to get the deal done here. With that second tranche of $181 million, if it comes in in its entirety, we will have enough money to get comfortably to our first approval and be a commercial company. At that point in time, our cost of capital drops and our capital needs would change.
The first and second tranche add up to a financial situation that is quite comfortable for us. Right now, the $125 million we have, we've got it, is enough to take us to June of 2026, comfortably on the other side of the VITESSE readout and obviously the exercise of the warrants.
We need success in 4Q to trigger that, which gets us to the first launch potentially early 2027.
That's it.
Perfect.
Yeah.
We covered a lot. It's been a long road coming, but we're getting close to the finish line. And now.