Everyone, this is David Amsalem from the Piper Sandler Biopharma team, and welcome to Day 2 of the Piper Sandler Healthcare Conference. Our next company is Fennec Pharmaceuticals, and we're delighted to have CEO Jeff Hackman and CFO Robert Andrade joining us. And it's been an exciting time at the company. So just starting at a high level here, we're going to talk about all things PEDMARK.
Sure.
But wanted to get your thoughts, for those who are not as familiar with the story, on how the underlying molecule here exerts its efficacy in preventing/mitigating Cisplatin-induced ototoxicity?
Sure. Good question. Well, thank you for having us here, and we really appreciate this, and PEDMARK's always exciting to talk about, and it's because it's what it's doing. And so for those of you who are not familiar, it's the first and only approved product from the FDA to prevent ototoxicity focused around Cisplatin treatment. And Cisplatin is a treatment that's given to many cancer patients, and so this product, this is for localized non-metastatic tumors. It's the only therapeutic agent that has proven efficacy and safety data, and it's been established in a paradigm with two phase III trials, and we just recently or yesterday, we just announced another trial from Japan that had also showed efficacy and safety data, so it's designed to be safe at higher doses. It's a product that's used for otoprotection for these patients during chemotherapy, given with Cisplatin.
It's given six hours after Cisplatin is given, and the product right now is indicated for ages one to 18 years of age.
In terms of the overall incidence, severity of Cisplatin-induced ototoxicity, just help us understand how it impacts pediatric patients relative to adolescents and young adult patients. In other words, is there sort of a differential impact based on the different age groups?
We didn't see that in the studies. We haven't had, w e're now going out and doing some investigator-initiated trials in some of the older AYA or adolescent young adult population. But when you look at the populations that this product's used for, it's used in really two segments, o ne is the pediatric segment. There's about 2,000 patients annually in the U.S. that are treated with Cisplatin in the ages one to 18 years of age. And this product was approved back in September of 2022. About six months after approval, we received guidelines that approved the product in these guidelines. They're called National Comprehensive Cancer Network Guidelines, NCCN 2A Guidelines, to approve the product between ages of 15 and 39 years of age. So while that's not an FDA indication, what it is, is it's a recommendation from NCCN, which gives us the opportunity to be able to use the product there.
That market is about 20,000 patients that get Cisplatin. You can see the two markets and the sizes and the size differences that we have. We market the product in both of those populations.
And just to be clear, I mean, the NCCN guidelines, and again, this is sort of more for the edification of folks who are less familiar with the story, I mean, that essentially is good as an approval, as a label expansion.
Sure.
In the oncology space .
Yeah. Exactly right.
So with that in mind, one of the parts of the AYA market that I think about is the germ cell tumor/testicular cancer population. Obviously, the most common cancer has been under the age of 40. Cisplatin is the standard of care here. So can you talk about that opportunity? I understand you're approved in localized malignancies, but again, Cisplatin is the standard of care here. So help us frame that opportunity in the germ cell setting.
Yeah. When you look at that population, ages 15 to 39, and you look at that 20,000, about 4,000 of those patients are germ cell patients, in particular testicular patients. And so these young men get a significant amount of Cisplatin. Cisplatin works really well in testicular cancer, really well. But it does have the impact, as we all know, to hearing. And it's toxic in that way. So we find that that population is one that we see early use now of PEDMARK. As we start to go into this population, it seems like those 4,000 patients, those testicular patients, are almost kind of that bull's-eye that you see so far that's being utilized for PEDMARK in that population.
And just to clarify, the 4,000, those are the testicular or germ cell patients who are on Cisplatin?
Correct.
Okay.
Yeah.
Got it, so talk more broadly about the commercial infrastructure that you have in place to support PEDMARK in the U.S.
Sure.
And I'll l et Robert talk a little bit about that.
Yeah. Let me give you a little overview of Fennec itself to start because Fennec has one product, PEDMARK. We spent several decades working to develop it. So we're very proud of it. As Jeff mentioned, we got it approved in September of 2022. Today, we stand at just under 40 employees total in Fennec, of which half of those are customer-facing, what we call customer-facing. And break it out into a couple of key components: traditional sales reps, medical science liaisons, or MSLs, as we call them, that help educate and train on PEDMARK. And then what we established recently are called key account directors. And so these key account directors are responsible for creating the awareness for the larger academic centers that really need the red carpet service for the large oncology networks. And so if you step back a year ago, we've doubled our MSL presence.
We didn't even have the key account directors because 2025 has really been that year of growth and awareness in the AYA market. We brought a new commercial leadership team here with Jeff spear heading it just a year ago. Our team has really focused on creating awareness across all age categories because I think something that's very important is that it is agnostic to the age and it is agnostic to the tumor type in terms of the damage that Cisplatin can do to your hearing. Our goal is hopefully to provide the availability and the awareness to patients, any patient out there that takes Cisplatin.
How are you helping practitioners and patients navigate the infusion-related logistics here? There are some aspects of the treatment regimen that are, how should I say, unique for PEDMARK. So just help us understand how PEDMARK is administered and when after the Cisplatin infusion and how you're handling and helping patients and practitioners navigate these unique logistics?
Yeah.
I'll let Robert take it because I know you were involved in designing the trials early on back in the day.
Correct. So Cisplatin itself is done IV. It can be done over several days or one course. Effectively, after two hours, all of the Cisplatin that's attacking the tumor cells has gone intracellular. The only thing that's left is extracellular. And what PEDMARK does is it goes in as a scavenger and it mops up, for lack of a better word, or it's a binding agent to any excess Cisplatin before that Cisplatin gets into your cochlea and causes the stress and eliminates these hair cells. And importantly, once you lose your hearing, it's irreversible. So the only alternatives prior to PEDMARK were hearing aids, cochlear ear implants, or living with it for the rest of your life. PEDMARK itself is given six hours later to your question. And it's a 15-minute IV infusion.
The challenge, what we've especially dealt with in the AYA space, is a large portion of these patients are seen at the community oncology centers. So to the extent that there's any limitations in terms of opening, if a patient, as an example, gets Cisplatin through 12 o'clock to noon, then you have to wait the six hours, that center might not be open. So we've enabled, through some valuable partnerships, to do what's called home health administration and to a certain extent also even work with those centers. So maybe we could start the Cisplatin earlier because of that six-hour time gap. In the pediatric, a little different because most of those patients are kept overnight. So the logistics is something that we're very proud of from the beginning of 2025 until now in terms of the efforts that we've made there.
What portion of your PEDMARK patients now are actually given the infusion at home versus they're given in the cancer centers?
It has certainly grown from the beginning of the year. I would say a 1/3 to 40% of those patients are growing towards the home health pharmacy.
Okay, and what's the mix between pediatric and the older patients at present, and how's that evolving?
Yeah. From a market perspective, to repeat, pediatrics' incidents rate on a yearly basis is about 2,000 patients that receive Cisplatin. From an AYA perspective, it's roughly 20,000 patients a year. And these are both per year that receive cisplatin. We received the NCCN 2A endorsement after approval. So we like to say we are really in the beginning of a launch after we had been in the beginning of another launch, which was the pediatric market. So I think the best way to frame it now, although we don't dissect pediatric versus AYA in our financials, is 2024 Financials, largely all pediatric. The growth that you've seen, and I know Jeff is very humble, but we've had four quarters of straight growth, and we've grown from roughly $7 million quarterly sales, product sales, net product sales, to $12.5 million is all in that AYA market.
That's all in AYA. Okay. And I guess as a follow-up to that, part of a supportive care product in oncology, when you have oncologists, they're thinking about all the different toxicities that they have to manage, is sort of just raising awareness. So when you think about awareness of PEDMARK among oncologists and among nurses, how are you working towards growing that awareness? Obviously, the NCCN guidelines are a big part of that. But can you talk about just promotional efforts that are targeting to practitioners, be that the actual medical oncologists or supportive nurses?
Yeah. Because when we were first licensed, which is a great question, David, when we were first licensed, it was a small group of hospitals really seeing kids, right? These children's hospitals, and there aren't that many. And so when we decided to now go ahead and launch in the AYA space, you now go from 100 or so hospitals now to thousands of locations, upwards of 4,000 locations out in the community setting. And so first off was, okay, so how do we get out there and how do we do that and what's the message and what does this sound like and look like and who should we target? So that was all as we started to develop this a s Robert said, we were kind of relaunching the product in this new space.
The other part of this is really important to create this awareness. There was really very limited awareness because we didn't do any trials with these physicians where we did in the pediatric space. So they somewhat knew us or knew we were coming. But for these, we call them these practitioners that are out in the community, we did not. Awareness levels were low and they still are. And so it's one of the things that we've got to get out and have conversations with these potential users of PEDMARK. They use a lot of Cisplatin. They understand that there's a toxicity there. In a lot of cases, they don't know that PEDMARK exists. And so we call this just a huge awareness phase for the product, and that's going to continue.
The reach has to get out to even more physicians, and our plan in 2026 is to continue to build as much awareness as we can, specifically with these users of Cisplatin in this setting.
So another follow-up regarding practitioner behavior. The incidence of, well, really any toxicity with a platinum-based chemo, I mean, it's not uniform. I mean, some patients are going to experience Cisplatin differently than others. Ototoxicity is no different. So I guess my question here is, as you think about ototoxicity and whether the incidence rate is 40% or 50% and there's varying degrees and severities, you have oncologists who are kind of pushing back and just saying, "I'll look at it as an option if my patient is actually starting to experience ototoxicity as opposed to thinking about it as a prophylaxis for ototoxicity"?
Yeah. The problem is once you start to show signs of hearing loss, there's no turning back and there's no reversing it. But maybe you could follow up a little bit on that question because I know you guys.
Yeah. Certainly, oncologist first, survival, second survival, third survival. But part of why the question early on, our infrastructure, what we've really focused and emphasized is the medical piece because it takes that education. That simple question, "Well, let me see if he has hearing loss." That's too late by that point, and it's cumulative. And so by beefing up our MSLs, our medical education, all that's really important to get over that hurdle and that understanding. The second component is, "Oh, this is a pediatric-only product." Well, your ear itself is the same really from any age. And so it's a lot of that a nd also oncologists themselves, Cisplatinum is so effective that some of them don't even know why it's so effective or how it actually works or how it actually goes systemic and what part goes intracellular versus extracellular.
And so this all circles back around to the medical education in part because our trials were focused on pediatrics only. But you're going to hear some more, and hopefully can ask that question next. Some exciting news of what we have in the pipeline.
Yeah. I did want to get to that. But first, can you talk about payer access and also about pricing on PEDMARK and what net realized price looks like?
Sure. Yeah. From a payer perspective, we are quite pleased. As an example, last quarter, we had 100% reimbursement across the large carriers: Blue Cross Blue Shield, Aetna, Cigna, United. They understand the impact to losing your hearing. They understand the impact of carrying that the rest of your life, especially when you see survival rates with cisplatinum that get close to 90%. So you survived. And I love this part of our marketing team. We put survival with an asterisk. You look at the asterisk, "Whoa, that's not full survival," right? Because I had an impact to my hearing. And so I think that that is critical and crucial to what we're trying to address. But that's on the payer side. Quite pleased with it. And I think that was part of the early phases on the trial adopters even. I understand PEDMARK now.
Let me identify a patient, and now let me see if I can get it reimbursed. You cover all three of those, then you get the repeat customers, which is what we want. On your other question in terms of pricing, so PEDMARK itself, the WAC price is $11,000 per vial. Important to note there's two big components that are going to impact the overall price per patient. One is the amount of Cisplatin that you get, and the other one is the size of the patient because some patients for each round of Cisplatin may get one vial, obviously the younger, smaller patients, or they may get two or three vials. So we see ranges from six vials up to 30 vials on that $11,000 WAC. On a gross to net basis, there's about a 20% step down.
That's largely some discounts and then some rebates on the government side.
Okay. That's helpful. What about nausea and vomiting? I mean, it can be emetogenic. I mean, obviously, Cisplatin is emetogenic. And obviously, anybody on Cisplatin is going to be on antiemetic agents, quite a number of them. But is nausea and vomiting in practice been a problem?
I want to say this. I'll say it for most people who have relatives or people who've been involved in watching treatment. It's very linked to oncology treatments, right? Nausea and vomiting, and it is something that you see with Cisplatin significantly because this product's given six hours afterwards. In a lot of cases, these patients are still nauseous, right? Are still going through nausea. Our product does cause a little bit of nausea as well. We make sure that prior to administration, we have an antiemetic protocol that's put out there that works fine, which really helps the patients, not only their Cisplatin potential nausea that they're dealing with, but also the potential nausea that they'll get from PEDMARK, so we do that proactively. We make sure that the physicians understand the antiemetic protocol that goes along with our product.
We want to make sure that these patients can get all of the doses that they need to get. And if you get really sick, right, you don't want to take any more of the product. It's really important that the antiemetic protocol is administered to the patient.
Yep. Just before I get to the recent data for supportive in Japan, just wanted to touch on the intellectual property on PEDMARK. And it's an old molecule, right? So I think it's a reasonable question. How should we think about exclusivity runway in the U.S.?
We have a very unique formulation. So I appreciate the old molecule, but we have a very unique formulation within our product. So to be specific, we have orphan drug exclusivity that was seven years from approval. So that carries us until September 2029. Then we have six Orange Book patents that carry us through July of 2039. We are currently in ANDA litigation with a generic manufacturer. We've been in it for a couple of years. Ultimately, the question is, is it 2029? Is it 2039? And we know how a lot of these things get settled at the end of the day. And stay tuned.
Okay. I'll leave it at that. All right. The Japan data. Let's talk about it.
That's great news. Yeah. Yesterday, a trial that was started, I think, what, three years ago in Japan. It's an investigator-initiated trial, STSJ01. It evaluated the efficacy as well as safety of PEDMARK, reducing cisplatin-related ototoxicity in Japan. We had 10 sites, 27 available patients in the primary cohort. We had 6 also that were in exploratory cohorts as well. But looked at PEDMARK administered 6 hours after cisplatin as well. The product met its primary endpoint. But one of the things that it also showed that there's no interference of PEDMARK with the cisplatin anti-tumor activity, one of the questions that you had earlier. So by evidence, the 95% clinical response rate. So that's great news. The data is compelling enough to move forward for approval in Japan. We're going to meet with the PMDA and others to start that process forward with our investigators there.
But the data, now this is the third trial that's been conducted, very similar that shows clinical efficacy and safety for PEDMARK. So we're excited. We plan to find a partner in Japan. We're in discussions, obviously, throughout, and we will continue those discussions and explore partnering and licensing opportunities. You'll need somebody on the ground in Japan to file. That's the regulations there, so.
I'm just wondering out loud, I mean, is there potentially a path down the road for further exploration in metastatic disease? I know it's locally advanced and non-metastatic disease. I know the body of data is not lost on me, but how do you think about that?
Yeah. We've had multiple institutions approach us here in the U.S., large institutions to want to conduct investigator-initiated trials. And these trials, so stay tuned on that. It's a good question. We hope to announce some of these as we go forward. But they want to look at metastatic. They want to look at potentially PEDMARK in adults. They want to look at just not non-localized, but metastatic disease. So it's exciting. These trials can come out and they can be fast, right? They don't have to take that long, and we can get results quickly for the physicians, which I think is important. So stay tuned on that.
I'll try to think of one more question. Just ex-U.S. launches, European markets. Just remind us where you launched in Europe and how that's going?
Yeah. Yeah. Robert, you.
Yeah. We have a great partner, and I think it's a good comparable or precedent to Japan, but we went out and had results globally from our two phase three trials. Got approval in Europe. We have a partner in Norgine. If you're not familiar with them, they do about $500 million in sales, backed by Goldman Sachs, so they licensed it from us for Europe, UK, Australia, New Zealand, $43 million upfront, $200 million in milestones, which I believe right now is about our enterprise value coming to us based on certain metrics, and they are just getting going with launch, so really, they just launched in the UK and Germany. We have that first milestone that's going to hit hopefully in the first quarter of 2026 that we look forward to, and we're excited to see what comes. It's a market that needs it.
And for similar reasons in the U.S. to provide that availability for patients to prevent that hearing loss for the rest of their life. And then one last thing on the pricing because we get a lot of questions on this. The pricing in Europe. There's one formal price today in the U.K. I mentioned the $11,000 per vial WAC in the U.S. In the U.K., it's at GBP 10,000, which is versus precedents or comparables. It's a really good small gap between the two. And the next pricing, as I mentioned, will be in Germany.
Okay. Very helpful. Well, thanks, Robert. Thanks, Jeff. Thanks, everyone in the audience.
Yeah. Thank you, everybody.
Cheers.
Appreciate it.