Nuvectis Pharma Q3 2026 Earnings Call Transcript

Nuvectis Pharma (NASDAQ:NVCT) reported third-quarter financial results on Wednesday. The transcript from the company’s third-quarter earnings call has been provided below.

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Summary

Nuvectis Pharma reported a transformational third quarter with the addition of two late-stage drug candidates, NXP100 and NXP200, through a licensing agreement.

NXP100, an oral complement factor B inhibitor, received its first marketing approval in China for PNH and is under FDA review for U.S. development.

NXP200, a brain-penetrant BRAF inhibitor, received breakthrough designation in China for high-grade glioma and plans are underway for a U.S. IND filing.

The company raised $187 million through a follow-on offering and ATM facility, ending the quarter with $140 million in cash, expected to fund operations into 2029.

Management highlighted robust clinical data from China and ongoing discussions with the FDA regarding U.S. regulatory paths for NXP100, with potential for significant market presence in PNH and IgA nephropathy.

NXP900’s development focus is on combination studies, with clinical updates expected in the first half of 2027.

The overall financial performance showed a net loss of $30.1 million for the quarter, with a strong cash position to support future strategic initiatives.

Full Transcript

OPERATOR

Good morning and welcome to Nuvectis Pharma third quarter 2026 financial results and business update conference call. At this time, all participants are in a listen-only mode. Following the prepared remarks, we will conduct a question-and-answer session. As a reminder, this call is being recorded. I would like to now turn the call over to Ron Bentsur, Chairman and CEO of Nuvectis Pharma. Please go ahead.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Thank you and good morning everyone. Before we begin, I would like to remind everyone that statements made during this call that are not historical facts are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. These statements are based on management’s current expectations and on our interpretation of information available to date, and they are subject to substantial risks and uncertainties that could cause actual results to differ materially.

For a discussion of these risks, please refer to the section titled Risk Factors in our Quarterly Report on Form 10-Q for the quarter ended September 30, 2026, and our other filings with the SEC. These risks are not exhaustive and new risks emerge from time to time. Any forward-looking statements made on this call speak only as of today, Wednesday, November 4, 2026, and Nuvectis Pharma undertakes no obligation to update or revise them except as required by law.

Okay, now let’s get to the interesting part of this call. Today’s the first time we’re hosting a call alongside the release of our quarterly results and the reason is simple. The third quarter was transformational for Nuvectis Pharma and we have a great deal to share with you. We have transformed Nuvectis Pharma into a late-stage development company with the addition of two exciting drug candidates with best-in-class potential through our licensing agreement for ex-China rights with our new partner jisco.

These additions have fundamentally changed the opportunity ahead of us. Our lead drug candidate, Ciprocapan, which we also interchangeably refer to as NXP100, is an oral complement factor B inhibitor for once-daily oral administration that is approved in China for the treatment of newly diagnosed paroxysmal nocturnal hemoglobinuria, or PNH, in short, and in development for the treatment of other complement-mediated diseases including an ongoing Phase 3 study in IgA nephropathy, a Phase 2 study in lupus nephritis, both being conducted in China by our partner Hyesco.

The second drug, NXP200, is an oral, brain-penetrant paradox breaker BRAF inhibitor for the treatment of BRAF-mutated solid tumors, and I’ll expand on these two products and on our legacy compound NXP900 during the call. We closed the licensing deal with AESCO in late June, after which we completed a follow-on offering of $150 million in gross proceeds that was anchored by leading healthcare-dedicated institutional investors. Also in the third quarter, we placed approximately $37 million with a single healthcare-dedicated fund through our ATM facility. We ended the quarter with approximately $140 million in cash, which we expect will fund our operations into the second half of 2029.

And importantly, beyond multiple value inflection points. Let’s start with the key pipeline development highlights before taking a slightly deeper dive into our plans going forward. In July, Soprocopan, or NXP100, received its first marketing approval in China for the treatment of patients with PNH who were not previously treated with complement inhibitors. The approval was based on a Phase 3 study in which NXP100 demonstrated clear superiority versus Soliris on the primary and all key secondary endpoints.

A second marketing application for ciprocapan in China is currently under review. This application covers patients with PNH who were previously treated with C5 inhibitors and is supported by another very successful Phase 3 study in this patient population. We expect AISCO to announce this approval in the first quarter of 2027. We are in active dialogue with the FDA about our development plan for PNH in the U.S. These discussions are going well and we feel that the FDA is very engaged.

We expect to provide an update on the FDA interaction by year end. Several additional Phase 2 and Phase 3 studies with NXD100 are ongoing. We’re also carefully watching the Phase 2 and Phase 3 studies being conducted with Fabalka, or Tacopan, Novartis’ twice-a-day oral complement factor B inhibitor. All these study results could provide an excellent roadmap for the additional indications that we may want to pursue with NXP100. We expect the coming months to be packed with important relevant news on the NXP100 front.

Turning to NXP200, in September, the drug candidate was granted breakthrough designation in China for the treatment of BRAF V600 mutant radiation-recurrent or progressive high-grade glioma. In October, NXP200 was the subject of an oral presentation at the ESMO conference. Updated Phase 1b data from the ASCO study in China demonstrated deep and durable single-agent activity with NXP200, including complete responses in patients with recurrent or progressive gliomas.

Importantly, responses were also observed in patients previously treated with a BRAF inhibitor. Both of these aspects—complete responses and responses in BRAF-relapse patients—could be meaningful points of differentiation for NXP200 compared to the other second-generation BRAF inhibitors in development. The emerging clinical profile for NXP200 demonstrates best-in-class potential. NXP200 is currently being investigated in a broad clinical program in China to unlock its full therapeutic potential.

For our legacy NXP900 oncology candidate, the Phase 1b program continues. This includes ongoing osimertinib and lorlatinib combination studies and the planned investigator-initiated study of NXP900 in combination with the newly approved KRAS inhibitor diroxanracid in pancreatic cancer. This investigator-initiated study will be led by two top medical centers in the country. So as you can see, we’re off to a very good start on all fronts since completing the licensing deal in late June.

Now let’s take a closer look at our plans for each program. Importantly, we’re doing so from a very strong financial position with estimated cash runway into the second half of 2029. Let’s start with Cipropican, or NXP100. NXP100 inhibits factor B of the complement system. Factor B is a key component of the alternative pathway, one of three pathways of the complement system. By inhibiting factor B, NXP100 blocks the amplification of the alternative pathway while preserving the other two. PNH and other complement-mediated diseases result from uncontrolled complement activation. Blocking different targets within the complement system has proven to be a very successful strategy.

Treatments like Soliris, Ultomiris and Phybalta are approved in several different indications and generate significant revenue. As I mentioned, the first approval of NXP100 in China was based on a randomized head-to-head Phase 3 study versus Soliris. In treatment-naive PNH patients, NXP100 was superior to Soliris on the primary endpoint and all key secondary endpoints. The second marketing application in China that is currently under review is based on a Phase 3 study conducted in patients with PNH who failed treatment with a C5 inhibitor.

This study also met the primary endpoint and key secondary endpoints with plenty of room to spare. It’s important to mention that the overall hemoglobin increase demonstrated by NXP100 in the Phase 3 studies conducted were rather unprecedented, with average hemoglobin increases of 5 and 4.4 grams per deciliter in the treatment-naive and previously treated studies, respectively. Importantly, the safety profile of NXP100 appears consistent with that of other complement inhibitors.

Okay, so what does the opportunity in PNH look like for NXP100? The PNH market is estimated to be around $5 billion today and is expected to double within the next 8 to 10 years. Historically, this market has been dominated by the injectable C5 inhibitors, which were the centerpiece of AstraZeneca’s $39 billion acquisition of Alexion in 2021. Factor B inhibitors offer the potential for better efficacy compared to the C5 inhibitors, along with the convenience of oral administration.

So with much better efficacy and this convenience advantage, we expect the factor B inhibitors to become the leading class in PNH over time. As of today, the only FDA-approved factor B inhibitor is Phibalta from Novartis. Phibalta is approved for PNH, IgA nephropathy and C3G, a smaller nephrology indication, and is off to a fast start. It is on an annualized run rate of approximately a billion dollars already, and this is about two years out of the gate.

We see a potentially substantial advantage for NXP100 in its once-daily dosing compared to the twice per day for Phibalta. This convenience advantage could be very meaningful and should not be underestimated in chronic diseases like PNH that require continuous lifelong treatment. I think most of you would agree that there’s nothing easier for a patient suffering from a condition requiring lifelong therapy than a once-a-day oral treatment. And as you have likely gathered by now, PNH is only the beginning. In China, NXP100 is in an ongoing Phase 3 study in IgA nephropathy following a successful 24-week Phase 2 study. In the study, NXP100 demonstrated a statistically significant, clinically meaningful reduction in proteinuria and, importantly, the study also showed an almost 3-point increase in eGFR in the NXP100 arm compared to a 1.5-point decline in the placebo arm over this 24-week time horizon.

We know that there’s a lot of excitement around the April BAF class of drugs in the IgA nephropathy space and for good reason. In IgA nephropathy, the new standard established by the best April BAF drugs is the ability to stabilize eGFR long term, and that is considered disease-modifying. It appears that Phibalta comes up just shy of being able to do that, whereas NXP100, based on the Phase 2 data generated, definitely has the potential to deliver that.

This is where the favorable pharmacokinetic and pharmacodynamic profile of NXP100 versus Phibalta may be coming into play. Hence, NXP100 has the potential to deliver this competitive efficacy which can compare to the best April bafs but with the clear convenience advantage of once-daily oral dosing versus the April BAFs which are all injectables. Together, the PNH and IgA nephropathy markets alone are estimated to exceed $20 billion within the next 10 years.

Our priority is to advance NXP100 towards a marketing application in the United States for PNH. We also see significant potential in additional indications. Our U.S. regulatory efforts in PNH are well underway. With two positive Phase 3 studies from China, we believe we have a strong clinical data package. This puts us in a good position as we work with the FDA to define the U.S. regulatory path. We continue to have constructive dialogue with the FDA which, as I mentioned before, we feel is very engaged.

We expect to provide an update on the U.S. regulatory path before year end. In addition, we’re planning to submit an IND for another indication outside of PNH later this year. We also plan to initiate clinical trials in additional indications in the first half of 2027. Novartis is currently running five Phase 2 and Phase 3 clinical trials for Phibalta in additional indications beyond the three approvals that they already have. These studies include areas such as dry AMD and myasthenia gravis.

We eagerly await the results from the Phybalta- and JISCO-run clinical trials. These results will help inform additional indications for us to pursue and where to focus our resources. As you can appreciate, there’s no shortage of opportunities where we believe Cepropican, or NXP100, can work and be very impactful. Our job is to prioritize these opportunities carefully, taking into account the clinical and regulatory path, market potential and our resources.

Stay tuned for further updates on PNH and additional opportunities later in the year and into early 2027. To support our development efforts in complement, we have made several key hires towards this end. These include Dr. Dustin Little, who joined us in September. A Senior Vice President, Head of the complement program, Dr. Little is a physician, scientist and board-certified nephrologist with a decade of industry experience at AstraZeneca, having most recently served as the Global Clinical Head, Late Cardiovascular, Renal and Metabolism, providing clinical leadership for renal disease area strategy at AstraZeneca, as well as multiple first-in-class or potential first-in-class treatments for people living with kidney, cardiovascular or hematologic diseases.

Dr. Little brings a wealth of complement knowledge and experience in the nephrology/hematology settings and he has really hit the ground running for us and is fully immersed in all facets of the NXP100 program. Now let’s switch gears and talk about NXP200. BRAF is a commercially validated target in oncology, with first-generation BRAF inhibitors generating well over $4 billion in combined annual revenue. While initially effective, first-generation BRAF inhibitors often cause paradoxical activation of the MAP kinase pathway. This results in treatment resistance and that is why the BRAFs are then combined with a MEK inhibitor to try to prolong the treatment effect.

First-generation inhibitors also do not address Class 2 and Class 3 BRAF mutations. There’s a clear need to try to overcome the paradoxical activation of the existing BRAF inhibitors, and the paradox breakers such as NXP200 represent the next-generation approach to targeting BRAF. Last week we reported updated data from the Phase 1b study in China with BRAF-mutated solid tumors conducted in China by jisco. The study also included patients previously treated with BRAF and MEK inhibitors, and NXP200 generated responses in two of the three patients with this prior BRAF exposure, and this represents an important potential differentiator for NXP200. We also believe that NXP200 has the potential to expand beyond CNS tumors.

Durable responses in non-small cell lung cancer, colorectal and papillary thyroid cancers support the opportunity in a broad range of solid tumors and further data from the ongoing Phase 1b study in China could substantiate this further. We should be receiving an update from this program in the next couple of months. In comparison, looking at data from the competitor paradox breakers being developed, it appears that these drugs are struggling to demonstrate meaningful activity outside of CNS tumors.

Turning to the NXP200 data reported last week at the ESMO Conference: in this 27-patient Phase 1b study, NXP200 demonstrated an objective response rate of 40% in the high-grade glioma cohort, which included 20 patients, and this included three complete responses, two confirmed and one pending. The objective response rate was 42.9% in the low-grade glioma cohort, including one confirmed complete response. The responses were assessed based on RANO 2.0 criteria, the FDA regulatory standard for brain tumors.

I want to spend a moment on this assessment criteria because we’ve been asked how our results compare with data recently reported for another second-generation BRAF inhibitor. That company reported six responses out of nine patients, or a 66.7% response rate, in their glioma study. There’s an important distinction in how the responses were assessed. The other company’s reported results included radiographic responses rather than relying entirely on RANO criteria.

Based on their disclosure, only three of nine patients, or 33%, appear to have achieved a RANO response, with no complete responses. In the high-grade glioma group, only one out of five patients appear to have achieved a RANO response. This comparison reflects our interpretation only of that company’s most recent disclosure. We remain on track to submit a U.S. IND for NXP200 by the end of the year, with the goal of starting a U.S. Phase 1b in early 2027. For some perspective on the opportunity, in April of this year Servier acquired Day One Biopharmaceuticals for $2.5 billion. Day One’s only approved drug is a first-generation BRAF inhibitor approved in pediatric low-grade glioma. At the time of the acquisition, Ojemda was projected to generate $225 million to $250 million in sales this year, representing only approximately 7% of the current BRAF market, providing, we believe, an interesting benchmark for potential deals in this setting.

Given the emerging profile in CNS and the potential in other solid tumors, we believe NXP200 could emerge as a best-in-class next-generation BRAF inhibitor in CNS and also across a much broader range of tumors. Now turning to NXP900: with a shift in our corporate focus towards NXP100, we’re concentrating NXP900’s development on the most promising combination opportunities. Two studies are ongoing with NXP900 with Tagrisso (osimertinib) and Lorbrena (lorlatinib), and we also have an investigator-initiated study planned for NXP900 in combination with a newly approved KRAS inhibitor—RAS-onQ or Derek, I’ll skip it. We expect to provide further NXP900 clinical updates in the first half of 2027.

Now to some financial results and cash runway information. I’d like to briefly review our third quarter financial results. Our net loss for the quarter was 30.1 million. Excluding the upfront license payment and the one-time milestone associated with NXP900 and the non-cash stock-based compensation, our adjusted net loss was 5.8 million. We ended the quarter with approximately $140 million in cash. Based on our current plans we expect that cash to fund—we have cash to fund—operations into the second half of 2029.

With that runway, we have the financial strength to reach multiple value inflection points across our pipeline. So looking ahead, we have a number of important catalysts over the next several months. For NXP100, we expect to provide an update on the U.S. regulatory path for PNH as well as an IND filing by year end. We also anticipate regulatory approval in China for previously treated PNH patients in the first quarter of 2027 and near-term clinical updates from the Phibalta- and the Hiesco-run clinical trials which all could provide important data read-throughs for NXP100.

For NXP200, we’re targeting a U.S. IND filing by year end. We also expect additional data from the Phase 1b study in China across solid tumors including colorectal, melanoma, non-small cell lung cancer and papillary thyroid cancers in early 2027. And for NXP900, we expect clinical updates from the Phase 1b program in the first half of 2027. We have several opportunities to deliver meaningful news in the near term with the potential to create significant value across our pipeline.

As I look at Nuvectis Pharma today, I see a very different company. NXP100 is a once-daily oral factor B inhibitor already approved in China for PNH with a potential to become a best-in-class treatment. NXP200 is a next-generation BRAF inhibitor with breakthrough therapy designation in China for high-grade glioma already achieved. We believe it has the potential for significant differentiation and best-in-class attributes across multiple tumor types.

NXP900 adds further opportunities through our combination studies. At this time I would like to thank our incredible partner Jesco for all their hard work and cooperation. We’re privileged to be the shepherds for the NXP100 and NXP200 programs ex-China. I’m also deeply grateful to our team for an extraordinary last few months, and of course thank you all, our shareholders, for your continued trust and support. With that, operator, let’s open it up for Q&A. Thank you.

OPERATOR

Thank you. We will now begin the question and answer session. We ask that you please limit yourself to one question and one follow up. If you would like to ask a question, please press star one on your telephone keypad. A confirmation tone will indicate your line is in the question queue. You may press star two if you would like to remove your question from the queue. For participants using speaker equipment, it may be necessary to pick up your handset before pressing the star keys.

One moment, please, while we pull for questions. Our first question is from Peter Stavropoulos with Cantor Fitzgerald. Please proceed with your question.

Samantha (Analyst)

Hi. Congrats on the progress, team. This is Samantha on the line for Pete. Thanks so much for taking the question. Good morning. Could you tell us more about interactions with the FDA regarding NXP100 you’ve had so far? You’ve guided to a pre-IND meeting in 2H26 with the FDA and an IND submission in 4Q26. What are the possible outcomes for the path forward in PNH? Any color here would be helpful. Thank you.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Good morning, Samantha. Sure. Thank you for the question. So basically, the first formal interaction that we had with the FDA regarding the PNH program was in late September, and it’s been an open dialogue since then. If you think about the Phase 3 studies that were conducted in China, the results were very robust. There’s no question about that. And then when you look at the Fabhalta studies that were conducted—the two Phase 3 studies that Novartis conducted—it’s important to mention that about 40% of the patient population in those studies combined were Asian.

And one study was entirely ex-U.S. To overlay another aspect on top of that, our drug NXP100 is essentially an analogue of Fabhalta. So AISCO, in a very clever fashion, was able to optimize Fabhalta to come up with a much better half-life and, we think, much better pharmacodynamic properties, etc. Of course, this constitutes a new chemical entity and there’s a composition-of-matter patent issued and so on and so forth. That adds another very strong reason to what we laid out in front of the FDA.

As you can imagine, there are bookends to the possibilities here. One possibility is they’ll ask for two clinical trials, let’s say one in naïve and one in pretreated. If we have to do that, we’re more than capable. We’ll start those studies at the same time, and whatever study reads out first will be the basis for the NDA approval at that time, and then there’ll be an NDA supplement for the study that will be completed afterwards. That’s if they require two studies.

The other end of the spectrum is that they’re going to require no studies based on all this evidence that I just laid out. Now, we believe that from a purely regulatory, statutory perspective, we have a very strong leg to stand on. But at the same time, we also realize that there are other reasons for FDA decision-making—there could be policy issues and not wanting to open up floodgates and things like that. So realistically, we believe that we’re going to have to run a study.

We’re fully prepared to do that. We’re gearing up internally for that, and we’re just fine-tuning and waiting for final marching orders from the FDA. But it could be any one of these three. It still hasn’t been finalized. What I will say is that in the prior interaction, the FDA asked specific questions about the studies that were conducted in China to see if you can level the playing field versus the U.S. guidelines and the studies. For example, one of their questions, just to give you a sense of the types of questions they asked, was regarding the transfusion protocol. They wanted to make sure that the transfusion protocol criteria in the Phase 3 studies in China was basically the same or substantially the same as the U.S. guidelines and ideally also substantially the same as what was done in the Fabhalta studies.

And in fact it was. The criteria for the transfusion was pretty much identical to the Fabhalta protocol and to the U.S. guidelines. They asked a couple questions along those lines, and I think we were able to put all those questions or concerns to bed. So that’s where we are. The FDA, we feel, is very engaged. I think the dialogue is very positive. But again, it hasn’t been finalized yet. It could be any one of these three different alternatives that I described. Hopefully that answers your question.

Samantha (Analyst)

Really helpful. Thanks so much.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Thank you.

OPERATOR

Our next question is from Joe Panganise with H.C. Wainwright. Please proceed with your question.

Joe Panganise (Analyst)

Hey, Ron, good morning.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Hey, Joe. Good morning.

Joe Panganise (Analyst)

Good morning. So two questions, please. I wanted to follow up regarding the FDA. Obviously, these are ongoing discussions. You just gave some important nuance to the prior question. So I guess I’ll ask it this way: What would you consider to be the outstanding questions, and are there any that are still up for debate or you’re finalizing that need to be able to get to IND? And fast forward then, with regard to potential study designs—I emphasize potential because it could change tomorrow—you said maybe randomized.

Could this be a placebo-controlled study with Fabhalta out there? And how would you look to identify sites, and how do you think about potential competition for sites or drug moving forward?

Ron Bentsur (Chairman, Chief Executive Officer and President)

Yeah. So in terms of the key questions or concerns that the FDA has, they asked very specific questions and we were able to answer all those. They were mostly related to trying to even the playing field vis-à-vis the Chinese studies and what’s going on in the U.S., and to some extent the Fabhalta studies. I think we were able to answer those questions fully. That does not mean that they’re not going to require a study because there could be some other concerns or other reasons as to why they would want that.

As we know, there are many drugs that have come in from China recently, and there’s an issue of maybe not wanting to open up the floodgates and setting a precedent and things of that nature. I think our situation is very unique in that sense, but without getting into detail, our expectation is that they will require a study. Now, when you think about the different studies that they could ask for—a randomized study versus placebo versus an active control, in a patient-naïve population versus a C5, something like that—very similar to the study that AISCO ran in China, which is the basis of the first approval that was achieved in July for the patient-naïve population. Another one could be pretreated patients. A third one could be a switch study where you take patients who are on a C5, you wash them out for a week or two, and you put them on treatment.

There’s a pretty wide variety. What I will say is that—and this ties into your second question—when you look at the patient numbers in these studies, typically it’s about 35, maybe 40 patients per arm. That’s what we saw in the AISCO studies; that’s what we saw in the Fabhalta studies. I think that’s the guideline we’re going to follow, more or less. So if you’re thinking about a single-arm study, it’ll be about 35 patients. Obviously a controlled study will be about 70 patients, something like that.

In terms of reaching out to sites, there’s a lot that’s happening behind the scenes. This is where the work of Dr. Little and some of the other folks that have come in is really starting to pay off. They bring their Rolodex, their connections, and their know-how and understanding into the mix. We’ve already established very good contact with a lot of the KOLs, a lot of the top recruiting sites from the prior studies in the U.S., ex-U.S. So there’s a lot going on on that front.

I think we’ll be ready to hit the ground running once we have the final marching orders, and we tie it up in a bow and start whatever program we need to start. I think we’re going to be ready.

Joe Panganise (Analyst)

That’s fantastic detail. I really appreciate it. And my quick follow up is, for 200, since China has breakthrough designation, is it safe to assume you’d be looking to achieve the same in the United States with the FDA? And what sort of data do you think you’ll need to get there?

Ron Bentsur (Chairman, Chief Executive Officer and President)

Regarding NXP200, I think that’s a complete sleeper. I don’t think anyone’s paying any attention to it, and I think that this drug can emerge very quickly—literally within the next few months. Once we get an update from China from the Phase 1B study that’s ongoing in China with respect to the other tumor types, this could very quickly emerge with a best-in-class profile. On top of that, I would add that if we see, for example, data in colorectal or melanoma or what have you in BRAF-relapsed patients, that is not just best-in-class type of potential; that is one of the new hot oncology compounds, period. It’s that kind of a profile. So stay tuned for that, and hopefully the data will support what I just said.

With respect to breakthrough designation in the U.S., we’re chomping at the bit to get started with the Phase 1B. The IND is going to go in this year with the goal of starting the Phase 1B as quickly as possible in early 2027. CNS is a given—we’re going to do that. At the same time, I think it would behoove us to try to file for breakthrough designation. Keep in mind that AISCO already has 27 patients in gliomas—20 of which are high grade, seven of which are low grade.

The data, I’m not going to repeat it; you saw it. I think if we can add another 25, 30, 35-patient core to that, that could be very substantial in terms of driving the case vis-à-vis the FDA, not just for breakthrough designation, but beyond that. So we’re definitely gearing up for that as well. As you can imagine, trying to file two or three INDs for two different drugs for a small company is no small feat, but we’re going to do it and we’re going to achieve it. The goal is to get these things going and moving. We want to start clinical trials. We want to put the wheels in motion.

Joe Panganise (Analyst)

Thank you, Ron.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Thank you.

OPERATOR

Our next question is from Naz Rahman with Maxim Group. Please proceed with your question.

Naz Rahman (Analyst)

Good morning, everyone. Congrats on all the progress. Thanks for taking my question. Pretty straightforward on NXP200. Obviously the data in gliomas is highly positive, and you’re about to see this solid tumor data come out soon. Could you walk us through what data we will see? And, more specifically, what would you consider meaningful or important enough to incorporate that tumor type in your next study in the United States? Thanks.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Thanks, Naz. So the data that we’re going to see is an update from the Phase 1b that started in the early part of this year in additional tumor types. So just to give you a little bit of history here, and I’ll try to do it literally in 30 seconds. So back a year and a half or two years ago, JISCO used an older version of the formulation, what they call the freebase formulation. In this freebase formulation, they saw very good data in CNS, and they saw inklings of activity in other solid tumors, colorectal, melanoma, etc. But it wasn’t good enough. So they knew that they would need to improve the formulation, namely to improve the PK parameters of the drug, to basically improve the activity of the drug in the other solid tumors.

So what they did was they reformulated the drug into what’s called the salt form, and then they conducted a small pilot study, just as a litmus test, to see whether they were basically marching down the right path or barking up the right tree — basically whether they were seeing kind of an uptick in activity to justify starting the Phase 1b in these other solid tumors. And in fact, they did. It was a small pilot, so no one’s saying these were large patient numbers or anything, but they had three out of seven responders, deep responses, excellent durability.

So that gave them the confidence to start the Phase 1b in these other solid tumors. What we’re going to get is an update from that Phase 1b, including a specific arm looking at BRAF-relapsed patients. We expect that there’ll be maybe as many as 15 to 17 patients’ worth of update in that subset. And obviously there are going to be other kind of subgroups that we’re going to receive updates on. And as you can imagine, to be able to rechallenge a patient who was on a BRAF, responded for as long as they did, and then relapsed or failed — that is the holy grail, and that is quite substantial if you’re able to do that.

And in terms of what constitutes success in that setting, I would argue that even a 20% or 25% response rate would definitely constitute success. Anything above that, obviously you start getting into kind of the triple home run, grand slam type of territory. Because it’s never been done before, we saw an inkling of that in CNS at the ESMO presentation where they had three patients that were BRAF-relapsed and two of them responded. So that’s hopefully a good bellwether. We are going to wait patiently for that data and we expect to have it within the next two or three months or so.

And if in fact we do see visible activity in that setting, just as an example, that could really set the tone and really put this drug on, I think, much, much greater visibility, and I truly believe that this could transform the drug’s profile into not just a better paradox breaker, but in fact one of these very new, attractive oncology compounds that are out there in the industry. And there are not many of them at any given point in time, but I think it could in fact join that very prestigious club of drugs that fall into that category.

Naz Rahman (Analyst)

Thank you. That was helpful. Thank you.

OPERATOR

Our next question is from Yale Jen with Laidlaw & Company. Please proceed with your question.

Yale Jen (Analyst)

Good morning, and thanks for taking the questions, and congrats on all the progress. You mentioned IgAN, and the recent ASN meetings had several highlights, including what Vertex did and trying to file for approval later this year for the PDUFA date. How do you see NXP100 in that overall context, as well as in the context of the approved drug? And you mentioned earlier that you may file another IND for NXP100 besides PNH. So would that also be IgAN?

And then I have a follow-up question.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Yeah, thank you. So yeah, we’re fully aware of all the data that’s been generated in IgA nephropathy and the APRIL/BAFFs. And the APRIL/BAFFs — obviously some of them, not all of them — but some of them look absolutely terrific with the ability to control eGFR, that being, as we all know, a very important parameter to be able to control because that obviously can curtail the disease progression. So that’s very good news for patients. But we believe that NXP100 can fall into that category of activity.

So when you look at the Phase 2 data — albeit it was a small study, it was a Phase 2, 25 patients per arm and so on and so forth, and the 24-week endpoint — and we need to see it in the Phase 3, which by the way is a fully powered Phase 3: 370 patients, one-year treatment horizon, including a one-year follow-up. So they will have all the information that’s necessary to be able to make that comparison. But when you look at the Phase 2 data, especially at the key components, which are the proteinuria reduction of 57%, which is pretty unprecedented, and also the eGFR increase of almost three points, that definitely gives you a reason to be encouraged about what you could possibly see come April or May when the data reads out in China from the Phase 3 study.

So we believe that NXP100 could definitely have a seat at the table as it relates to frontline therapy, and being a once-a-day oral treatment — I don’t care if you’re a Democrat, Republican, or an Independent — everyone knows that that’s so much easier than having to deal with injectables. And all the APRIL/BAFFs are injectables, right? So we feel that that would put us in a very good position. And just going back to what I said on the call, when you look at the Fabhalta data in IgAN, they come up just a little bit short.

So they see a drop of about two or three points annually in eGFR. So that puts them kind of below that threshold. It’s almost like having a p-value of 0.049 versus a 0.051, right? Something like that. But there is a distinction. There is a distinction. You want to be eGFR-stabilizing. One thing I will add to that whole equation is that when we talk to KOLs, they believe that patients need to be controlled to a level of less than a one-point drop per year. Why one point? Because not all patients are going to be stabilized or go higher. Some patients are going to drop. But if a patient drops only at an average of one point per year, realistically, they will never get to dialysis. They will die of natural causes and dialysis will not be an issue.

So that’s an acceptable number. So when you look at that APRIL/BAFF data — put our data aside for a second — when you look at the APRIL/BAFF data, not all patients achieve that. Obviously when you have an average of eGFR stabilization, some patients are above that, some patients are below that, some patients are below the one-point drop. And those patients are going to require combination therapy based on what — not quite the formal guidelines yet — but the emerging guidelines that we believe are going to be established based on the data that we’ve seen with the APRIL/BAFF.

So at a minimum, I think that the oral complement factor B’s are going to be the mainstay for those combination therapies because no one’s going to start to combine two injectables. So when you think about the messenger RNAs, factor B’s and all that, it’s great science, don’t get me wrong. I mean, it’s very exciting science. But again, there’s nothing easier on the planet than a once-a-day oral. And I’m not even going to talk about the potential safety issues, but no one’s going to combine those with another injectable, with different timing of the injectables and things like that.

So I think everybody assumes that, at a minimum, the oral factor B’s are going to be the mainstay for the combination therapies. And when you look at the APRIL/BAFF data, roughly half the patients are going to fall into that category. But we have higher aspirations than that. As I just mentioned, we believe that NXP100 could be a frontline therapy, could be a frontline targeted therapy based on the data that we’ve seen. Does that answer your question, Yale?

Yale Jen (Analyst)

Sure, I appreciate that. It’s very comprehensive. I just want maybe just two quick ones. First is when you anticipate potential to start that trial. And the other question is that in terms of manufacturing, both of the 100 and 200, where you guys are in the U.S.? And thanks for taking the questions.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Yeah. So in terms of INDs, we want to file INDs this year. NXP200 is pretty straightforward. NXP100, we want to file at least one IND, maybe even two this year. Obviously nephrology is important — IgAN, C3G, which we believe is kind of low-hanging fruit because of the success that Fabhalta has had in C3G. And there’s also a pediatric component there. So that’s nephrology. With respect to hematology, PNH obviously is front and center. There are also some additional indications that we’re thinking about potentially.

And I think there’s going to be no shortage of activity. But the goal for NXP100 is to file at least one IND by the end of the year and be in a position to start clinical trials as soon as possible in 2027. In terms of manufacturing, over time we want to migrate the manufacturing to our CMO, again just to try to seclude ourselves and insulate ourselves from any political issues or anything like that that may arise a year, two years, five years from now — who knows? So we want to make sure we’re able to secure our own manufacturer. So all that kind of tech transfer is happening as we speak. But that’s not going to stop or slow down the start of clinical trials.

So all that’s going to happen in parallel, and hopefully that answers your question.

Yale Jen (Analyst)

Sure, and appreciate that. Again, congrats on all the progress and the very fast speed.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Thank you. Thank you.

OPERATOR

There are no further questions at this time. I would like to turn the call back over to Ron Bentsur for closing remarks.

Ron Bentsur (Chairman, Chief Executive Officer and President)

Thank you very much, and thank you all for joining us this morning. We look forward to updating you on our progress. Have a good day. And again, we sincerely thank you for your time. Be well. Thanks.

OPERATOR

This concludes today’s conference call. Thank you for your participation. You may now disconnect.

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