X4 Pharmaceuticals, Inc. (XFOR) Q2 2024 Earnings Call

Investor webinar replay

Replay transcript excerpt

meaningful levels. Finally, we'll be also looking for continued tolerability and safety of Mavericks IV as seen in all of our other clinical programs to date. In June, we also announced the initiation of our Global Pivotal Phase III clinical trial, branded the FORWARD Study, evaluating the efficacy, safety, and tolerability of oral once daily Mavericks IV with and without stable doses of GCSF in people with congenital acquired primary autoimmune or idiopathic CN who are experiencing recurrent and or serious infections. The 52-week forward trial is a randomized, double-blinded, placebo-controlled, multicenter study aiming to enroll 150 participants. The clinical operations team is in full swing, opening up sites, and we are on track to fully enroll the trial in mid-2025. We believe that the interim Phase II CN data presented to date strongly support our decision to initiate the Phase III trial as well as its design. Importantly, we believe that the interim results de-risk the Phase III trial, given that the ANC increases seen in CN patients to date mirror those in our successfully completed IV-WIM Phase III trial. where similar levels of ANC increases led to clinical benefit by reducing the frequency, duration, and severity of infections. As we focus our efforts on the forward CN trial, we are also looking to maximize the global opportunity in WHIM syndrome. We expect an MAA submission to the European Medicines Agency for Mavericks IV and WHIM syndrome by early 2025, while we also explore additional potential opportunities and geographies where we may be able to efficiently leverage our U.S. FDA approval. With that, I'll now turn it over to our CFO, Adam Mostafa, to review the second quarter financials. Adam? Thanks, Paula, and thanks to all of you for being on the ca...