X4 Pharmaceuticals, Inc.

Greetings and welcome to the X4 Pharmaceuticals fourth quarter and full year 2024 financial and operating results conference call. At this time, all participants are in a listen-only mode. A question-and-answer session will follow the formal presentation. As a reminder, this conference call is being recorded, and it is now my pleasure to turn to your host, Dan Ferry from LifeSite Advisors. Please begin.

Thank you, Operator, and good morning, everyone. Presenting on today's call will be X4's Chief Executive Officer, Dr. Paula Regan, and Chief Financial Officer, Adam Mostafa. Following prepared remarks by each, we will open the call to your questions, and we'll be joined by Chief Commercial Officer, Mark Baldry, Chief Medical Officer, Dr. Christoph Arvid Engels, and Chief Operating Officer, Mary DiBiase. As a reminder on today's call, the company will be making forward-looking statements regarding regulatory and product development plans. These statements are subject to risks and uncertainties that may cause actual results to differ from those forecasted. A description of these risks can be found in X4's most recent filings with the SEC, including this year's Form 10-K, which is expected to be filed after market close today. I'd now like to turn the call over to X4's President and CEO, Dr. Paula Regan. Paula?

Thank you, Dan, and thanks to all of you for joining us this morning. As expected, 2024 was a transformative year for the company and for those with WHIM syndrome, and we are very pleased to continue to deliver as we progress into 2025. As you know, we became a fully integrated company in 2024 with the U.S. approval and launch of our first product, Maverick Zephyr, which we branded as the remedy in the US for the treatment of WHIM syndrome, an ultra-rare primary immunodeficiency. Our commercialization strategy remains focused on a multi-pronged approach, as is typical in the rare disease space. Over the past year, we significantly advanced disease awareness with both treating physicians and WHIM patients through a combination of in-person and digital education campaigns and through strengthened relationships with our rare disease patient advocacy group partners. A peer-to-peer speaker program was also launched in the second half, enabling physicians to share their experiences in managing WHIM syndrome and treating with Zolremdi. In addition, our suite of patient services, including our X4Connect and Nurse Educator programs, continue to provide access and support for patients prescribed Zolremdi. and journal publications and presentations of clinical data at top medical meetings, including those of the American Society of Hematology, or ASH, the American Academy of Allergy, Asthma, and Immunology, or Quad AI, and the Clinical Immunology Society, have provided further visibility. And we've been very encouraged by some recently received testimonials from our patients on Zolremdi. We heard from one patient who described his, quote, normally functioning immune system, and his significant improvement in skin infections, warts, and periodontal issues. He said he now knows what it's like to lead a, quote, normal, unquote, life with lower anxiety about getting sick, ability to travel, and that he, quote, didn't know how sick he was until he finally felt better, unquote. Another patient has told us about his near normal ANC levels, his increased energy, increased productivity, and engagement at work, and the importance of him not having to miss any family or social events anymore, it's really been quite inspiring. Our 2024 Zol Remedy sales topped $2.5 million, which covered the seven and a half months since our mid-May launch last year. Throughout 2024, we successfully engaged with all of our target top-tier immunologists and hematologists, We expect that these efforts will continue to deliver through both increased patient finding and shortening the time to pull through to a Zol Remedy prescription. In addition to our progress across the U.S., we've also made significant progress in our efforts to expand the potential global reach of Mavericks for and WHIM. In January, we announced that our submitted MAA was accepted by the EMA for review. With a typical 12 to 15 month review process, we would expect potential approval for the EMA as early as the first quarter of 2026. We've also entered into two international partnerships. The first with Noragene, a leading European specialty pharmaceutical company. Under this license and supply agreement, Noragene will commercialize Mavericks 4 for WHIM and chronic neutropenia in Europe, Australia, and New Zealand, following regulatory approvals in those areas. We received 28.5 million euros up front and are eligible to receive up to 226 million euros in potential regulatory and commercial milestone payments, in addition to tiered double-digit royalties up to the mid-20% range. We continue to believe that the NeuroGene deal is a great fit for us, given both their focus on rare disease and specialty markets and our shared vision of putting patients first. The second deal we announced was with a company called Teba Rare, another specialty pharmaceutical company. The agreement enables Teba to use its expertise towards orphan drug marketing, sales, and distribution in the Middle East and North Africa, or MENA region. Teba will be marketing Zol Remedy for WHIM syndrome across the region. We expect that the formal approvals will be sought in Saudi Arabia and Kuwait, but in the meantime, pending any regulatory approvals, TABA expects to be able to provide Zol Remedy to WHIM patients through a named patient or compassionate use program that allows physicians to prescribe medicines approved in other countries to local patients with no other treatment options. So, a lot to look forward to in WHIM, but now let's turn our continued advancement of Mavericks for it to treat the larger potential indication of chronic neutropenia. During 2024, we successfully completed a Phase II clinical trial of Mavericks IV across several CN disorders with and without concurrent injectable GCSF, which is the current standard of care and the only drug approved for severe chronic neutropenia. Throughout the year, we announced both interim and then full data from this trial that we believe significantly de-risks our ongoing Phase III CN trial. open-label clinical phase 2 trial ended up enrolling 23 participants and demonstrated, one, that once daily, oral Mavericks 4 was generally well-tolerated with and without GCSF with no drug-related serious adverse events reported and consistent with our previous clinical studies. Two, that Mavericks 4 treatment durably and meaningfully increased participants' mean absolute neutrophil counts, or ANC, across all study populations, and three, that physicians were willing and able to reduce the use of GCSF in participants also treated with Mavericks 4 and were able to maintain mean ANC levels within the normal range. Given these positive data, we initiated a global pivotal phase 3 CN trial called the FORWARD trial last year. The FORWARD trial is a 12-month, placebo-controlled trial aiming to enroll 150 participants with congenital acquired primary autoimmune or idiopathic CN who are experiencing recurrent and or serious infections. More recently, based on FDA and EMA guidance, we have refined and simplified the forward protocol. We are now enrolling only moderate to severe neutropenic participants or those with AMCs below 1,000 cells per microliter. Given the trial's infection inclusion criteria, this ANC level was already consistent with our targeted patient population for Mavericks IV. If approved, focuses on those with the highest unmet need and consistent with almost all of the subjects already enrolled in the trial. In addition, the ANC component of the co-primary endpoint, which comprises both ANC increase and infection benefit, will now be uniform across all participants. This primary endpoint seeks to demonstrate that infection benefit from Maverick SWORD treatment results from a durable increase in ANC of at least 500 cells per microliter over the 12-month duration of the study. As you may recall, participants in our Phase III womb trial were severely neutropenic at baseline, with an average ANC well below 500 cells per microliter. Importantly, Mavericks 4 was able to raise ANC by 6 to 800 cells per microliter on average in the Mavericks 4 treatment population, which resulted in clinical benefit in the rate, duration, and severity of infections in that study. For these reasons, we believe that these trial protocol refinements in CN increase our chances of success in this trial on top of what we believe to be an already highly powered and robustly designed study. We also announced this morning that the forward trial has now been activated at about 90% of our targeted sites worldwide. Given this and the average screening success rates currently being observed, we expect that the trial will be fully enrolled in the third or fourth quarter of this year, which would enable us to disclose top line data in the second half of 2026. Before I turn over the call to Adam to discuss our financials, I do want to briefly touch on our recent strategic restructuring, where we announced that our revised business scope is expected to decrease our spending by about $30 to $35 million annually. We implemented these changes in early February to sharpen our focus on maximizing the opportunity for Mavericks IV in chronic neutropenia. We believe this is a much larger potential market opportunity for us, and given the challenging macro environment we all find ourselves in currently, We believe the best path to maximizing shareholder value and to giving us the greatest chance of success. With that, I'll turn it over to Adam.