X4 Pharmaceuticals

During the parallel #ESID2024 and #IPOPI #GPM2024 meetings we engaged with medical, scientific and patient advocacy partners from across the global #immunodeficiency community. At both events we enjoyed fostering new collaborations and learning from each other, all with the shared goal of serving patients.    At ESID, we enjoyed sharing more about our Phase 3 4WARD trial in chronic #neutropenia and hearing updates from multiple IEI (inborn errors of immunity) registries around the world. At IPOPI we had the privilege of sitting down with representatives of the international advocacy community to listen to their needs and hopes, and exchange ideas on how to improve awareness, diagnosis and treatment of #immunodeficiencies. Thank you to everyone who came together with us! European Society for Immunodeficiencies (ESID) IPOPI - International Patient Organisation for Primary Immunodeficiencies

We announced an upcoming conference call and webcast to report Q3 2024 financials, as well as results from our concluded Phase 2 clinical trial of mavorixafor in people with chronic #neutropenia on November 13. Learn more in our latest release: https://bit.ly/40hFHnr

Last week the X4 team heard virtually from Kara who has been living with #neutropenia since childhood. Now in college, Kara shared her experiences of balancing her health needs with student life and the importance of prioritizing your mental health when living with a chronic illness.

This The New York Times piece takes a deep dive into what it means to live #immunocompromised and having to manage a higher risk of serious illness from common infections, and frequent disruptions in daily life. https://nyti.ms/4dMlRUt Our mission is to drive progress for people living with rare #immunodeficiencies and we are passionate about delivering medicines for this often overlooked community. Thank you to Knvul Sheikh for sharing their stories.

Thank you, Eszter and the Shwachman-Diamond Syndrome Alliance for your dedication to people living with this #raredisease. We're honored to support organizations dedicated to amplifying the needs of patient communities we aim to serve. We look forward to continuing to listen and learn from you.

We are joining the Shwachman-Diamond Syndrome Alliance summit this weekend which will feature information about our ongoing Phase 3 clinical trial for chronic #neutropenia.   Unfortunately, most people with Schwachman-Diamond have neutropenia, or lower-than-normal levels of neutrophils, and we’re looking forward to listening and learning from researchers, patients, physicians and other community members at this important #raredisease gathering.   Learn about the event here: https://bit.ly/48qKHbH

Jennifer has lived with WHIM syndrome her entire life, but until just a decade ago didn’t know the name of her condition. Now in her sixties, and after multiple tests that made her feel like a “science experiment,” she finally knows the reason behind her unexplained and chronic infections. Read her story in this Patient Worthy piece: https://bit.ly/4eKMAS9   We are grateful for and inspired by Jennifer’s words and working hard to ensure that her diagnostic journey becomes the exception rather than the rule for those living with rare diseases such as WHIM syndrome.

Coming up this month: We’re focused on #WHIMsyndrome at multiple industry events. At #AMCPNexus, X4 is leading encore presentations of data from our Phase 3 open-label extension (OLE) and sharing neutrophil and lymphocyte data in WHIM syndrome at #ESID2024, as well as connecting with healthcare providers to discuss this rare #immunodeficiency at #IgNS2024.

ICYMI: September kicked off the investor conference season, including Cantor Fitzgerald's Global Healthcare Conference where our CFO Adam Mostafa outlined our plans for the US launch of the first approved product in WHIM syndrome and to advance our newly initiated pivotal Phase 3 clinical trial for a potential second indication in chronic neutropenia. Catch the replay here: https://bit.ly/3XVxXoC #CantorHCC

Last week we attended Life Science Cares Boston’s Mini Golf for Good Tournament in Technology Square! The event supports LSC’s nonprofit partners who are each dedicated to ending poverty across Greater Boston. As a Boston-based company, we appreciate the opportunity to join our fellow members of the #biotech community to support such an important cause.