PentixaFor by Pentixapharm AG receives Orphan Drug Designation for Marginal Zone Lymphoma from the EMA

Pentixapharm Holding AG, the future holding company of Pentixapharm AG, a clinical-stage biotech company discovering and developing novel targeted radiopharmaceuticals, announced that the European Medicines Agency (EMA) and the European Commission (EC) have granted an Orphan Drug Designation for the diagnostic tracer PentixaFor, also known as Gallium (⁶⁸Ga) boclatixafortide, for the diagnosis and staging of Marginal Zone Lymphoma.

Marginal Zone Lymphoma (MZL) is a type of non-Hodgkin’s Lymphoma (NHL) and has been recognized by the EMA as a chronically debilitating and life-threatening condition. Pentixapharm currently sponsors a European Phase III clinical trial to assess the performance of PentixaFor for the imaging and staging in patients with confirmed MZL.

PentixaFor is a novel tracer for positron emission tomography (PET) imaging targeting the C-X-C receptor 4 (CXCR4), which is overexpressed on MZL cells. Preclinical and clinical studies indicate that PentixaFor has a high sensitivity and specificity for the diagnosis and staging of MZL, with potentially better outcomes than the currently used [¹⁸F]FDG. An improved staging of the malignant disease is key to a more successful treatment.

The European Orphan Drug Program aims to support companies that develop medicinal products for diseases which affect no more than 5 in 10,000 people in the EU. The benefits of an Orphan Drug Designation include the possibility of seeking EMA protocol advice for PentixaFor development in MZL, regulatory fee reductions and waivers, and the access to the European centralized marketing authorization application. Additionally, upon market approval, PentixaFor could benefit from 10 years of market exclusivity within the EU for the diagnosis and staging of MZL.

About Pentixapharm Holding AG

Pentixapharm Holding AG was founded in 2024 to incorporate all shares held by Eckert & Ziegler SE in Pentixapharm AG, based in Würzburg, following the spin-off resolved by the Annual General Meetings of Eckert & Ziegler SE and Pentixapharm Holding AG on June 26, 2024. It is intended to list Pentixapharm Holding AG in the Prime Standard of the Frankfurt Stock Exchange.

Pentixapharm AG is a clinical-stage radiopharmaceutical development company founded in 2019 with its headquarters in Würzburg, Germany. It is committed to developing CXCR4 ligand-based first-in-class radiopharmaceutical approaches for diagnostic and therapeutic programs in a number of hematological and solid cancers, as well as cardiovascular, endocrine and inflammatory diseases.

Pentixapharm’s clinical pipeline encompasses PentixaTher, an Yttrium-90 based therapeutic against Non-Hodgkin Lymphomas (NHL), and PentixaFor, a Gallium-68-based companion diagnostic. Clinical studies for both compounds have already commenced in Europe, including a dose-finding study for PentixaTher and a Phase III registration study for PentixaFor in Marginal Zone Lymphoma. Additionally, PentixaFor is being developed as a diagnostic tool for Primary Aldosteronism (PA), a significant cause of hypertension. Pentixapharm is currently preparing a US-centric Phase III registration study with PentixaFor in PA that will start in 2025.