Welcome to the DBV conference call. All participants will be in a listen-only mode. Should you need assistance, please signal a conference specialist by pressing the star key followed by zero. After today's presentation, there will be an opportunity to ask questions. To ask a question, you may press Star, then one on your telephone keypad. To withdraw your question, please press Pound, then one. Please note, this event is being recorded. I would now like to turn the conference over to Katie Matthews, Investor Relations. Please go ahead.
Thank you. This afternoon, DBV Technologies issued a press release on recent FDA feedback on its VIASKIN Peanut program for toddlers and the regulatory path forward. This press release is available in the Press Releases section of the DBV Technologies website. Before we begin, please note that today's call may include a number of forward-looking statements, including, but not limited to, comments regarding our clinical and regulatory development plans, the design of our anticipated clinical trials, the timing and results of interactions with regulatory agencies, our forecast of our cash runway, and the ability of any of our product candidates, if approved, to improve the lives of patients with food allergies. These forward-looking statements are based on assumptions that are subject to risks and uncertainties that could cause the company's actual results to differ significantly from those suggested by these statements.
Given these risks and uncertainties, you should not place undue reliance on these forward-looking statements. Please refer to the company's filings with the SEC and the French AMF for information concerning risk factors that could cause the company's actual results to differ materially from expectations, including any forward-looking statements made on this call. Except as required by law, the company disclaims any obligation to publicly update or revise any forward-looking statements to account for or reflect events or circumstances that occur after this call. Joining me on the call today are Daniel Tassé, Chief Executive Officer of DBV, and Dr. Pharis Mohideen, DBV's Chief Medical Officer. I will now pass the call over to Daniel. Daniel?
Thank you, Katie, and thank you everyone for joining our call this evening as we discuss some very exciting news pertaining to the development of our VIASKIN Peanut patch. After several months of highly productive dialogue with the FDA, I am delighted to announce that FDA has provided us guidance to pursue an accelerated approval pathway for VIASKIN Peanut in toddlers, which we intend to pursue. In addition, our phase three study in four to seven-year-olds, VITESSE, is fully enrolled, and as we have said in the past, we would not want to begin the supplemental safety study, COMFORT Children, until VITESSE was closed to fully enroll. We can now focus on getting COMFORT Children started. Furthermore, we have also received favorable guidance from the European Medicines Agency on a path forward for a single indication in one to seven-year-old, utilizing the circular or the modified VIASKIN patch.
One patch, the modified one, in children one to seven. Pharis will shed more light on this exciting development later, as well as provide more details on VIASKIN Peanut programs in both toddlers and children. Next slide. Before diving into the history of our dialogue with the FDA regarding our VIASKIN Peanut program in toddlers, let's quickly recap the clinical development pathway for VIASKIN Peanut in toddlers and in children. Recall that we have two VIASKIN Peanut patches, the original square patch for toddlers, ages one to three years old, and the modified circular patch for children, ages four to seven. Each patch is regarded as a separate product by FDA and hence follows its own regulatory pathway, meaning that two BLAs will be submitted, one for each age group.
There's a symmetry to the clinical programs, where each BLA will be supported by 12-month phase 3 efficacy and safety study, EPITOPE in toddlers and VITESSE in children, and each with a 36-month open label extension, coupled with a supplemental safety study, namely COMFORT Toddlers and COMFORT Children. Turning now to a bit of history regarding our toddlers program and what has transpired since April of 2023 . At that time, the FDA informed us through written feedback that the positive results from our phase 3 EPITOPE study were sufficient for the efficacy portion of the BLA and that no additional efficacy data were required.
The agency did, however, request that we conduct a supplemental safety study in one to three-year-olds, not because of any safety signal, but simply to bring the total number of participants on active treatment close to six hundred, as per ICH guidelines. This study we called COMFORT Toddlers. At that time, we requested a Type C meeting with the FDA to review relevant details of that safety study. Historically, the agency focused on adhesion and wear time, and so we want to gain alignment on the collection methodology and on clinical assessment. We propose a methodology that we were already using in our ongoing VITESSE phase three study in four to seven-year-olds. A methodology that the FDA had already approved when the agency signed off on that study protocol.
At that time, the agency agreed with the approach of using VITESSE methodology to assess adhesion and wear time in COMFORT, and with adhesion being an exploratory endpoint in that study. With that as the basis, we expeditiously and formally submitted the COMFORT Toddlers safety study protocol to the FDA in November of last year. After several months, we received written feedback indicating that the agency was no longer in agreement with the proposed VITESSE methodology, which thus necessitated more dialogue between DBV and the FDA. FDA also requested that adhesion be elevated from an exploratory assessment to a study objective. We were concerned that the requested FDA changes would result in a study that would be very difficult to recruit and would lead to significant missing data. All these factors led us to conclude that the conduct of this study was just not feasible.
Since then, much of the dialogue between DBV and FDA regarding the COMFORT Toddlers study has focused on adhesion and patch wear-time experience, including how prescribers would advise parents and caregivers to manage potential day-to-day variability in patch wear time. Specifically, the hierarchy of an adhesion assessment within the COMFORT Toddlers study and the agency's request adhesion be a study objective versus exploratory assessment. Second, the amount and details surrounding wear time data points and the methods and the time windows used in the protocol to collect them, and importantly, the clinical relevance and regulatory use of adhesion data collected in the study that does not include an efficacy assessment.
After sharing these concerns with FDA, we wish to acknowledge that they understood our concerns about study feasibility, and with the involvement of senior clinicians and regulators at FDA, we then entered into a problem-solving dialogue in exchange, which resulted a few months later to the FDA guidance I just shared on accelerated approval pathway. With that as background, the regulatory issue we were facing and the dialogue that followed, let me ask Pharis, our Chief Medical Officer, whose team has done such a nice job of engaging that dialogue, to provide more granularity.
Thanks, Daniel. On June 28th of this year, DBV proposed to the FDA an approach crafted on a post-hoc analysis of the existing EPITOPE efficacy and wear-time data. This labeling proposal identifies patients who are most likely to benefit from VIASKIN Peanut, based on their wear-time experience over the first ninety days on treatment. This brings a more holistic approach that includes efficacy, safety, and wear-time experience, and what we believe is an overall broader view of the VIASKIN Peanut patch. As we have always said, efficacy is an essential element in understanding adhesion and the wear-time experience with the VIASKIN Peanut patch. We believe this data played a significant role in progressing the dialogue with the FDA and importantly, provided a concrete example of how a future label might read if the product is approved.
On September 9th, in a written advice letter, FDA proposed the accelerated approval pathway to DBV, stating that VIASKIN Peanut already met two of the three criteria for accelerated approval. One, the product candidate treats a serious condition, and two, the product candidate generally provides a meaningful advantage over available therapies. Defining the 3rd criteria, which is the product candidate, demonstrates an effect on a surrogate endpoint or an intermediate clinical endpoint that is reasonably likely to predict clinical benefit, has been the subject of our most recent interactions with the FDA, and last week, during a teleconference, the agency offered guidance on the 3rd criteria.
The FDA offered that the efficacy data from the phase 3 EPITOPE study, which you recall was published in the New England Journal of Medicine, can serve as an intermediate clinical endpoint, which is deemed to be highly predictive of the clinical benefit that will be confirmed in a future post-marketing confirmatory study. On the call, DBV told the agency that we agreed with this guidance to accelerated approval. At the agency's request, DBV will submit a meeting request to FDA based on the accelerated approval guidance shortly. Thus, the path forward for a VIASKIN Peanut patch in one to three-year-old with an accelerated approval pathway to licensure is anticipated to be supported by the following: Successful completion of a six-month safety study, COMFORT Toddlers. But importantly, patch adhesion will not be a study objective.
Successful completion of a post-marketing confirmatory study to confirm the clinical benefit observed in the EPITOPE study. At this point, it may be helpful to review the FDA's accelerated approval pathway for those who may be less familiar with it. The FDA instituted its accelerated approval pathway to allow for faster approval of treatments that treat serious conditions and fill an unmet medical need based on a surrogate endpoint or an intermediate clinical endpoint. This approach can considerably shorten the time required prior to receiving FDA approval. We are delighted to have the agency propose the accelerated approval pathway. We believe this provides a clear and straightforward pathway moving forward. Let me say again, the 2nd criteria for accelerated approval is that the product candidate generally provides a meaningful advantage over available therapies.
We sincerely thank the agency for its very high level of engagement and responsiveness during these past months as we worked through the specifics. Although these discussions took longer than anticipated, we believe both sides have built stronger relationships and a much greater mutual understanding of each other's positions, which should serve both us and the FDA very well, especially since VIASKIN is a platform, and we have other indications in the pipeline. Let me continue with a few more details on the one to three-year-old toddler indication. The COMFORT Toddlers safety study has already begun study startup activities within DBV, and we anticipate initiation in the second quarter of 2025. This study intends to generate six months of placebo-controlled safety data to support a BLA submission per the accelerated approval pathway.
300 to 350 subjects on active treatment will be recruited to increase the total toddler safety database up to approximately 600 subjects with at least six months of treatment. The FDA has been very consistent with this number per the ICH guidance. We will incorporate the patch wear time collection methodology developed jointly with the FDA over the past several months and formalized in the September written advice letter. We believe this methodology provides a practical approach for families and subjects, is intended to generate sufficient data to support a BLA submission, and places wear time into an acceptable clinical hierarchy relative to other study endpoints. We expect to submit a final study protocol to the FDA very soon. As Daniel discussed, the accelerated approval pathway guidance from FDA includes a confirmatory study.
DBV and FDA are in general agreement that the confirmatory study will need to demonstrate effectiveness of VIASKIN Peanut. DBV told FDA we agree with this approach, and we will formalize a meeting request to FDA based on the accelerated approval guidance shortly. In the short term, we will prioritize starting the COMFORT Toddlers safety study since it is required for a BLA submission in one to three-year-olds, and the confirmatory study will not start until the BLA is submitted. We will finalize the confirmatory study design elements with the FDA soon after. Let me turn now to our four to seven-year-old indication. This is a separate development program, which will have its own BLA submission relative to the one to three-year-olds. This program uses the modified circular patch. We announced last month the completion of screening for the phase 3 VITESSE study in four to seven-year-olds with peanut allergies.
We observed a tremendous increase in subjects wanting to participate in this study, and we closed recruitment, having enrolled 654 subjects, a bit more than the anticipated 600. We thank our study centers for their incredible hard work and, of course, our caregivers, parents, and subjects for their willingness to participate. I'd also like to recognize and thank the patient advocacy groups and academic societies for their ongoing support. VITESSE will be the largest registration trial in this age range ever conducted in peanut allergy. We expect top-line results for VITESSE by the fourth quarter of 2025. As we have said in the past, recruitment for the COMFORT Children safety study would not begin until we have completed enrollment in VITESSE.
Given that the test is fully recruited, along with the alignment with the FDA on the COMFORT Toddlers safety study, we will now prioritize starting the COMFORT Children Safety Study. We anticipate study initiation in the second quarter of 2025. So both COMFORT safety studies will start and run in parallel. The COMFORT Children Study is planned as a six-month placebo-controlled study to enroll approximately 250 subjects in total. This will increase our four to seven-year-old safety database with the modified patch to approximately 600 subjects, which is aligned with the FDA request to follow the ICH guidelines. The test in COMFORT Children will then constitute the two core studies for a future BLA submission for the modified patch in four to seven-year-olds with peanut allergy. Okay, let's move now to the update for Europe.
We sought scientific advice from the European Medicines Agency, known as EMA, regarding the components of a Marketing Authorization Application, or MAA, for VIASKIN Peanut patch. The EMA confirmed that the successfully completed EPITOPE phase 3 study in one to three-year-olds and a positive test study in four to seven-year-olds could constitute an MAA for a one to seven-year-old indication, in addition to a new safety study in one to three-year-olds using the modified circular patch. Thus, we believe there is a clear regulatory path for VIASKIN in Europe.
In addition, I should mention the most recent European Academy of Allergy and Clinical Immunology, known as EAACI, a draft guidelines on the management of IgE-mediated food allergy, has the following recommendations, and I quote, "In children and adolescents with IgE-mediated peanut allergy, peanut epicutaneous immunotherapy is suggested to achieve desensitization if available." This recommendation was stated as being supported with the same level of evidence that exists for oral immunotherapy, OIT. We were thrilled to see this acknowledgment from EAACI, which we think further emphasizes the unmet medical need in food allergies and the potential for the VIASKIN Peanut patch, if approved, to help fill the need for new therapies. The unmet need for peanut allergy in Europe is significant.
It is estimated that 615,000 children, ages one to seven in Europe, have peanut allergies, with an incidence of new diagnosis of approximately 81,000 per year. We are currently discussing the timing for this new safety study in one to three-year-olds with the modified patch, taking into consideration the start-up activities needed for the COMFORT Toddlers and COMFORT Children studies. At this point, I'll turn the call back to Daniel for closing remarks. Daniel?
Thank you, Pharis. Before doing the call for your questions, I would like to close by reiterating how pleased we are to have arrived at this point in our agreement with FDA's proposed guidance with respect to accelerated approval pathway for our toddlers program in one to three-year-olds, while continuing to make significant progress with our second program in four to seven-year-olds. Did this take longer than expected? I'm sure many of you are asking. Yes, it did. But this was a choice we made, and it was a necessary choice. We strongly believe the regulatory process, and thus our ability to help patients, require an investment of time to work through the critically important aspects of the regulatory program between DBV and FDA, and I wish to again acknowledge the engagement from division leadership in this dialogue.
We believe that with this clarity on the path forward for our toddlers program, we can now focus on completing the remaining regulatory steps, which are relatively straightforward, towards two separate and distinct BLAs. We believe VIASKIN Peanut, if approved, has the potential to be a game changer, not just for children with, that are peanut allergic, but for the food allergy community more broadly. Many of us were just back from the biggest patient advocacy group meeting in the U.S., called FARE, Food Allergy Research and Education, and if anything, attending meetings like this really reminds us of what matters here, which is the patients and their families who live with the daily burden, and it's a burden of peanut allergy.
While the landscape has shifted recently with the availability of approved treatments, providing families with much needed options and more of them, and it's clear that more options such as VIASKIN Peanut are needed. We at DBV are 100% committed to doing the studies needed to get to that approval. But they're not just about satisfying the FDA, they're also about helping our patients. I want to thank everyone on my team, everyone on the phone and webcast for joining us today. I will now ask Pharis to join me for question and answers.
At this time, we will conduct a question and answer session. If you would like to ask a question, please press star one on your telephone keypad now. You'll be placed into the queue in the order received. Once again, if you have a question, press star one at this time.... Our first question will come from Sushila Hernandez with Berenberg.
Yes, thank you for taking my questions. I have a few. So when do you expect to meet with the FDA?
Hi, Sushila.
Hello. When do you expect to meet with the FDA to confirm the safety study and confirmatory effectiveness study, and are there any other formal steps needed before you can start the safety studies?
Yes, Sushila, we plan to meet with them, going through the safety study very shortly. We have all the information we need in writing already. The FDA asked us to formalize the AA pathway through meeting requests, which we will do here. And that's going to be done, again, very shortly. First , finalizing the COMFORT Toddlers protocol, because that's job one, and then soon after that, the confirmatory study. Does that answer your question?
Yes, thank you. And are there then any other formal steps needed before you can start the safety study in toddlers?
No, when it comes to the agency, just for Pharis and his team to initiate the trial, but there's no other regulatory steps that are required.
Okay, that's clear, and could you elaborate on the patch wear method, methodology and, adhesion not being a subject anymore in the safety study, or how this has changed?
Yes, an important question. You want me to take it or we want to take it first to sort of describe what VITESSE has as highlights, what the FDA asked us to do and where we landed? I think that would be helpful color.
Yeah, okay. Sure. So basically, Sushila, what we had proposed originally was to do the same methodology as we're doing in VITESSE. So there's a two twenty-eight day period and some intensive monitoring that required more monitoring of different specific time points during those intensive periods. And the FDA then, as Daniel mentioned, sort of had a change in whether they wanted to accept that. And then they had asked for more than that and they sort of asked for shorter windows of time, as well as more data to be collected.
They also asked that some of that data be reported by the investigator data also. So the combination of all of those factors, we felt would put a lot of burden on our patients. It would put added resources at the site level, and it would also result in a lot of missing data points, and it would be hard for us to understand how that then would be analyzed. So all of that made it very difficult to move forward. And as Daniel said, we have had really good dialogue with the agency. They understood where we were coming from, and we have come to a really nice compromise position that we think is very feasible and practicable, and that we can execute in the COMFORT Toddlers study. Does that help to put some flavor around it?
Yes, thank you.
May I add some... Let me add some more color, if I may, Sushila. To give an example, the assessments we do in VITESSE are within a two-hour window, so four-hour window. So the 20 hour -- let's check if the patch is there at 20 hours, is between 18 and 22. The FDA want it to be at 20 hours, between 19 hours and 45 minutes and 20 hours and 15 minutes. That would be, you know, at 12, 16, 18, 20, 24, for days on end. So obviously those windows are just too tight. Moreover, the agency wanted this data to be brought from the patient to the investigator. The investigator then would upload it to the study site. Again, very cumbersome.
The kids could not take a bath, at least with no exposure to water on those days, which obviously would make the data collection or just the life of these families to be difficult, and as Pharis said, by making this study to have adhesion as an objective, missing data becomes a really significant problem, because the missing data obviously complicates the interpretation of the study, so again, that's what they asked. After dialogue with the agency, and I want to make sure this is so clear to everybody, the FDA understood that what was being asked of us was just not realistic. Adhesion cannot be a study objective. It should be an element that we measure as part of an overall safety assessment, and what we have landed on now, on September 9th advice, is much lighter than even what we do in VITESSE.
It's not a compromise, actually. It's lighter than what we do in VITESSE, and I think absolutely appropriate for a safety study in this population. Is that a fair way to put it, Pharis?
Yes, that is, Daniel. Thank you for the added comments.
Thank you. That's clear. Just to clarify, for the confirmatory effectiveness study, the recruitment for the study does not interfere with the safety study. Am I correct?
They will be done sequentially, correct. Yeah. The confirmatory study has to be started by the time the BLA is approved or plan at least to very much do that. But the safety study needs to be completed by the time we file the BLA, so there's no competition for patients here. It's an important question you're asking.
Yeah. And then just on your capital needs, as you mentioned in your PR, you have a runway into Q1 next year. So how much capital would you need to execute all your plans set forward in the PR?
Yeah, we'll be communicating that with more precision when we announce a fundraising, which is not what we're doing today here. So obviously the quantum is a function of how far we want to go in running these programs and then data rollout. We have a clear sense of that, Sushila, but that will be shared with the market, if and when we choose to finance.
Okay, that's clear. Thank you. Those were all my questions.
Thank you.
As a reminder, if you would like to ask a question, please signal by pressing star one at this time. It appears there are no further questions at this time. Mr. Tassé, I'll turn the conference back to you for any additional remarks.
My additional remarks will be, again, thank you to everyone for joining us. We're very much always excited by the outcome we have from the agency and from EMA in Europe. We conclude the call. We thank you again, and we wish everybody a great evening.
This does conclude today's conference. Thank you for attending.