First Patient Treated in Phase I/II Trial for Leukemia with Lu177-PentixaTher, Expanding Targeted Radiotherapy Evidence

BERLIN and WURZBURG, Germany, Nov. 21, 2024 – Pentixapharm Holding AG, a biopharmaceutical firm focusing on advanced radiopharmaceuticals, has initiated treatment for the first patient in a Phase I/II clinical trial. This trial aims to assess the safety and efficacy of Lu177-PentixaTher, a novel radiopharmaceutical therapy, in adults with relapsed or refractory CXCR4 positive acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). Funded by the French Ministry of Health and led by an expert team from the University Hospital in Nantes, this trial marks a pivotal step in the development of targeted radiotherapy for blood cancers.

The clinical trial, named PENTILULA (ClinicalTrials.gov ID: NCT06356922), is an open-label, multicenter study that plans to enroll up to 21 patients with relapsed or refractory AML or ALL over 24 months, with the study extending to 36 months in total. The primary goal is to establish the maximum tolerated dose (MTD) of Lu177-PentixaTher and to evaluate primary efficacy outcomes such as the overall response rate (ORR) and complete remission (CR) following infusion of this innovative radiopharmaceutical treatment.

In January 2023, a peer-reviewed article in the Journal of Nuclear Medicine reported encouraging clinical outcomes for T-cell lymphoma patients treated with PentixaTher. This has bolstered further investigation into Pentixapharm's leading candidate for treating hematologic cancers. The current study is particularly significant given the higher incidence and medical necessity of treating ALL and AML compared to T-cell lymphoma.

The PENTILULA trial uniquely combines PentixaTher with Lutetium-177, a radioisotope already used successfully in treating castration-resistant prostate cancer and neuroendocrine tumors. Acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL) are aggressive blood cancers affecting many individuals globally each year. AML, the most common acute leukemia in adults, has roughly 20,000 new cases annually in the United States and a low five-year survival rate of 31.9%. ALL is the most prevalent cancer in children but also has a significant incidence in adults. Current therapeutic options typically involve intensive chemotherapy and stem cell transplantation, which are associated with severe side effects.

Effective radiotherapies for other conditions are currently reimbursed at more than $100,000 per patient. Despite advances in treatments for acute leukemia, many patients still face limited options and poor outcomes. Pentixapharm believes that PentixaTher offers substantial potential to address these unmet needs, providing a valuable theranostic approach tailored to individual patients with hematologic cancers.

Dr. Dirk Pleimes, Group CEO and CMO at Pentixapharm, stated, "We are excited about the potential of PentixaTher in addressing the significant needs of leukemia patients. The initiation of this study by leading experts such as Professor Françoise Kraeber-Bodéré and Professor Patrice Chevallier in Nantes underscores the importance of evaluating this novel treatment option. We are fully committed to supporting this research and facilitating access to PentixaTher to advance this important work."

Professor Françoise Kraeber-Bodéré, heading the Nuclear Medicine department at Nantes University Hospital, is a prominent expert in nuclear medicine and oncology, contributing to various prestigious medical committees and research groups. Professor Patrice Chevallier, a specialist in hematology, is actively involved in hemato-oncological research through his roles in HéMA's executive committee and scientific council.

Pentixapharm is a clinical-stage biotech company based in Berlin and Würzburg, Germany, dedicated to developing CXCR4 ligand-based first-in-class radiopharmaceuticals. Their pipeline includes PentixaTher, targeting non-Hodgkin lymphomas with Yttrium-90 or Lutetium-177, and PentixaFor, a Gallium-68 based diagnostic tool for blood cancers and cardiovascular diseases. PentixaTher focuses on the chemokine-4 receptor (CXCR4), showing potential for treating hematologic cancers.

Nantes University Hospital (CHU de Nantes) is one of France's foremost healthcare institutions, excelling in numerous medical specialties and contributing significantly to medical research and clinical trials, driving substantial advancements in healthcare.