Conference Transcript - XFOR 2026-06-04

Tim Odetola, Analyst — Jefferies Healthcare Team

Welcome to the Jefferies Healthcare Global Conference. My name is Tim Odetola, part of the Jefferies Healthcare team, and it's my pleasure today to introduce Adam Craig, Chairman of X4 Pharmaceuticals.

Adam R. Craig, Chairman

Thank you, Tim, and thank you to the Jefferies team for the invitation to join you today. I will be making some forward-looking statements during the presentation today, and I refer you to the text in this slide. So, X4 Pharmaceuticals is primarily a Boston-based company that specializes in rare haematology conditions. Our lead asset is a drug called maverixifor, which is already approved in the ultra rare indication of Wim syndrome, both in the US and in the European Union. But we're currently focusing on a second and broader indication in chronic neutropenia. We have a phase three trial called FORD underway that we expect to complete enrollment at the end of the third quarter of this year and top line data in 2027 with a hopefully an approval for second indication in 2028 x4 over the summer of last year august of last year underwent considerable corporate restructuring there was a introduction of a new management team myself my colleague david kursky and so could i have the slide back to where it was there please because that's the only screen i can see thank you much appreciated so a new restructuring of a team from cti biopharma some of you may know we led the team with a successful acquisition of cti biopharma sobe in 2023 when we came in we had to reduce the head count to to reduce costs and we reduced it by 50 percent we changed the c-suite and we now having raised 240 million in two raises last year we now have enough cash to take us through commercialization to the end of 2028. Maverixifor is an oral agent that's designed to alleviate neutropenia. Neutrophils are moved around the body through a target called CXCR4 and Maverixifor is an antagonist of that target and through the target when it antagonizes the target neutrophils are released from the bone marrow into the bloodstream. So in patients who have low neutrophils and are susceptible to infection this can be quite significant change. It can reduce their risk of infection as I'll talk about later in the presentation. So Maverixer 4 has already been reviewed by the FDA and has been shown to increase neutrophils and also circulating lymphocytes. Client neutropenia is a difficult situation to live with, a difficult condition to live with. Hopefully most people in this room will have neutrophil counts above 1500 which is the normal level but if you have neutrophil counts lower than that if you have severe neutropenia where your counts are 500 microliters per liter or moderate where they're 500 to 1000 you're at risk of increased infections and these infections really can be quite debilitating often requiring hospitalization they can be potentially be fatal but they're very limiting to people's lives because just going out of your home going into society as a whole you're at risk of infections so it's quite a difficult condition to to live with and our aim is to make the lives of these patients a lot better so we've done a fair bit of work on the population we estimate through aviva claims analysis and some work physician survey work that we did with clearview partners that there are 57 000 patients in the u.s who have primary chronic neutropenia i should be clear that's not including patients who get neutropenia from things like chemo that's a secondary cause and that's a much more a much bigger population of that 57 000 there's 22 000 patients who have recurring infection if you take our population that we're studying which is the moderate and severe disease patients with an ANC less than a thousand that's 15 000 patients and that's our target population a significant number of patients and certainly given our specialty in dealing with unmet medical needs a very important population to deal with. So what are the unmet medical needs in chronic neutropenia? It's really very much determined by how patients and whether they're symptomatic and it's also determined by how patients whether they're on GCSF and how they respond to GCSF. We think there's an opportunity for Maverixifor to be used as a single agent in patients who haven't used GCSF or were intolerant of it, weren't able to take it, they felt unwell, they had bone pain. Or for those who are on it, hopefully to reduce the bone pain by reducing the GCSF dose and reducing the long-term risk of malignancy. One of the things we have surveyed in our physician survey we've asked what are the most important things for a new therapy in chronic neutropenia it probably comes as no surprise that an oral therapy is the number one uh desire of new physicians to for a new therapy is an oral therapy and that's because gcsf is an injection and it can't just be given uh as easily as a tablet whereas maverixifor is an oral agent can be given once a day. So we have some proof of concept data, 23 patients, that phase two study that was already conducted when David and I joined the company. And what they looked at was really this data set, although small, really answers two questions. Can Maverixifor be given in chronic neutropenia as a single agent and increased neutrophil counts? And then this second is, can it be given safely with GCSF and can GCSF doses be reduced while still maintaining neutrophil counts? So 23 patients, 15 of them were idiopathic. 10 of the patients were given Mavericks 4 alone and 13 were given Mavericks 4 in combination with GCSF. so if you look at the monotherapy patient you can see here at baseline the mean ANC was less than 1500 on the left hand side and when the drug was given over one to six month period the ANC was at 1500 or above so this demonstrates that mavericks of four alone can increase the ANC if we look at the gcs gcsf question remember the question was can you change the dose of gcsf safely and can you give the drug safely in combination the answer is yes the mean gcsf reduction in the gcsf patients was 70 percent and in fact three patients managed to come off gcsf and still maintain their counts so if we look at the data here now this is the green box the lower part of the green box the green box is showing a normal range for ANC so the objective here is to maintain the ANC whilst GCSF doses go down as you can see over a six month period from left to right despite significant reductions in GCSF dose and the elimination of the dose in some patients the ANC was still maintained within normal range a very powerful very important finding so obviously every drug has to be viewed on the efficacy side and also the safety side we have found to date that there are the drug is pretty well tolerated there is some gi toxicity nausea and diarrhea in the first few weeks of therapy most patients it resolves with a with over-the-counter medication and despite you know as we enroll more patients on the phase three and we see more patients. We haven't seen any new signals from the early trials. We haven't seen any new indications of toxicity, which is a good position to be in. So this is the trial that 99% of the time and resources of the company are spent on, which is the forward, the phase three trial of chronic neutropenia. What we're aiming here is to get a second indication for the drug in the US, which is much larger and potentially more valuable to the company than the WIM indication. So FORD is a double-blinded placebo-controlled study. Patients are split between two arms. Mavericks are four, plus or minus GCSF, and placebo, plus or minus GCSF. We're including congenital autoimmune idiopathic patients, and we have co-primary endpoints. The first is a reduction in the annual infection rate as independently assessed by a blinded committee. And then the other one is an increase in ANC of less than 500 cells per microlitre. Both endpoints are very well powered, greater than 96% for the ITT population. We do have some other interesting endpoints. I'll point one out to you, which is fatigue. We have quite a few anecdotal reports of improvement in fatigue and patients feeling better on Maverixor 4 in the early phase studies. And this is something we're looking at in the latter phase, in the phase three trial. So when we joined in August of last year, enrollment was an issue for the company. Since that time, we have expanded our clinical trial to 110 active sites. We've opened sites in the US. We have over 20 sites in the US. We moved our medical science liaisons from commercialization of WIM to the recruitment activities for the trial. We've worked on getting dedicated patient referral mechanisms from hospitals to our treatment sites. We've consolidated our CROs to improve efficiency. and we continue to use tools including some AI driven tools to identify new patients because this is obviously rare disease 15,000 patients is our population in the US so we have to work hard to find the patients. On the financial side before I summarize a cash position is strong thanks to the prudent cost management by David Kursky our CFO currently we last reported 233 million in cash and that's based on 144.8 million fully diluted stock outstanding so just to finish this is a short presentation we are on track to deliver data in the second half of 2027 this will be done by a new c-suite team who specialize in hematology drug approval and drug launch. We are now supported by blue chip investors and we have cash run way through 2028. We already have clear proof of principle of the role of Mavericks affording the increased individual counts from our phase two study and from the WIMS studies but the forward study is the registration study to get the new indication approved with the FDA and we're on track to have a FDA submission and probably approval in 2028. This is a pretty large opportunity. It is of around 15,000 patients. There's certainly a clear unmet medical need. And I do believe very strongly a well-tolerated oral agent would be very useful in this setting, either as monotherapy or as combination. And that's it. Thank you for your listening to the presentation. Tim.

Tim Odetola, Analyst — Jefferies Healthcare Team

Thank you, Adam. We can now open up for questions. We can pass the mic around. Please just raise your hand and Jeffries will bring a mic over to you shortly.

Speaker 3

So the presentation was, are there a possibility for indications beyond the one you're going for now?

Adam R. Craig, Chairman

Yes. So the question was, are there possibilities for indications? Absolutely. I have a quite long list in my head. The priority at the moment is to get the enrollment completed in the trial. What we hope to do in 2028, maybe through some ISTs and other programs, is increase the use of drug and generate data in other areas before we launch the second indication in 2028. But absolutely, there are a number of other neutropenias that we think we could get some very interesting data in. But as of today, it's not a priority. We're focusing the company on the execution of the forward trial.

Speaker 3

Thank you.

Tim Odetola, Analyst — Jefferies Healthcare Team

Do we have any other questions? If not, we can wrap up here. I think we're all set.