Cecile Geuijen
Chief Scientific Officer, Merus N.V.
Merus N.V. Geuijen
Chief Scientific Officer, Merus N.V.
Product: BIZENGRI®
Company: Merus N.V.
About the Product:
Merus has been an innovator in the field of multispecific antibodies for over a decade. Our proprietary Biclonics® platform generates diverse antibody binding domains against almost any target, which can be combined to create novel bispecific antibodies. In 2024, the U.S. FDA approved BIZENGRI® (zenocutuzumab), the first and only treatment for pancreatic cancer or non–small cell lung cancer (NSCLC) harboring a neuregulin 1 (NRG1) gene fusion. Before approval, BIZENGRI® received Breakthrough Therapy Designations for NRG1+ pancreatic cancer and NSCLC. Advancing a discovery in the lab to approval validates our Biclonics® platform and addresses a critical unmet need for patients.
Islam Hassan
Global Head of Development in Neuro-Oncology, Servier Pharmaceuticals
Islam Hassan
Global Head of Development in Neuro-Oncology, Servier Pharmaceuticals
Product: VORANIGO®
Company: Servier Pharmaceuticals
About the Product:
VORANIGO (vorasidenib)® is the only targeted therapy for Grade 2 glioma harboring isocitrate dehydrogenase 1 or 2 (IDH1/2) mutations and represents a monumental treatment breakthrough. VORANIGO was designed to target mutant IDH enzymes and is taken as a once-daily oral pill. After more than two decades without any new treatment advancements, VORANIGO signifies an undeniable shift in glioma care and addresses a significant unmet need by offering patients a treatment option that addresses the biology of their tumor, delays disease progression, and potentially delays the need for radiation and chemotherapy. Please refer to Full Prescribing Information at voranigo.com.
Peter Ordentlich
Founder and Chief Scientific Officer, Syndax
Peter Ordentlich
Founder and Chief Scientific Officer, Syndax
Product: REVUFORJ® (revumenib)
Company: Syndax
About the Product:
REVUFORJ® (revumenib) is a first-in-class, oral, potent, and selective menin inhibitor that is FDA approved for the treatment of relapsed or refractory acute leukemia with a lysine methyltransferase 2A gene (KMT2A) translocation in adult and pediatric patients 1 year and older. REVUFORJ® inhibits menin-KMT2A interactions by binding to menin and blocking interactions with KMT2A fusion proteins resulting in inactivation of a leukemogenic pathway. With a targeted mechanism of action, oral formulation, established safety profile, and a robust development plan investigating other uses, REVUFORJ® represents a groundbreaking advancement in the treatment of acute leukemia.
Christophe Arbet-Engels
Chief Medical Officer, X4 Pharmaceuticals Inc.
Christophe Arbet-Engels
Chief Medical Officer, X4 Pharmaceuticals Inc.
Product: XOLREMDI® (mavorixafor)
Company: X4 Pharmaceuticals, Inc.
About the Product:
XOLREMDI® (mavorixafor) is the first treatment approved in the U.S. for patients with WHIM syndrome (warts, hypogammaglobulinemia, infections and myelokathexis), a rare and serious primary immunodeficiency. In the Phase 3 4WHIM trial, mavorixafor increased circulating neutrophils and lymphocytes, reducing the frequency of bacterial and viral infections and reducing the use of antibiotics and antivirals in trial participants. Beyond WHIM, by enhancing both innate (neutrophil-mediated) and adaptive (lymphocyte-mediated) immune responses, mavorixafor holds promise to treat a broad spectrum of immunodeficiencies and immune-related conditions and is currently being studied in the Phase 3 4WARD trial for certain chronic neutropenic conditions.
Neil F. McFarlane
President & Chief Executive Officer, Zevra Therapeutics
Neil F. McFarlane
President & Chief Executive Officer, Zevra Therapeutics
Product: MIPLYFFA®
Company: Zevra Therapeutics
About the Product:
MIPLYFFA®, developed by Zevra Therapeutics, is a first-in-class oral therapy approved for use in combination with miglustat for the treatment of neurological manifestations of Niemann-Pick disease type C, an ultra-rare neurodegenerative disorder affecting about 900 individuals in the U.S. Addressing a critical unmet need, MIPLYFFA halts the progression of disease by penetrating the CNS and targeting disease pathophysiology through a novel mechanism that enhances autophagy and cellular cholesterol clearance. By activating transcription factors TFEB and TFE3, it upregulates the Coordinated Lysosomal Expression and Regulation (CLEAR) gene network, offering a disease-modifying approach that advances the treatment landscape for lysosomal storage disorders.