Actinium Announces FDA Clearance of Iomab-ACT Targeted Conditioning IND Application for Sickle Cell Disease Patients Undergoing Bone Marrow Transplant in Collaboration with Columbia University

In This Article:

- Sickle cell disease affects approximately 100,000 patients in the U.S. annually and is a debilitating and life-threatening condition with high unmet need

- Current conditioning with non-targeted chemotherapies provides limited access to potentially curative bone marrow transplant and recently approved gene therapies for sickle cell disease patients

- Initial trial focused on conditioning for bone marrow transplant intended to inform subsequent gene therapy conditioning study and provide broader access to cellular therapy for sickle cell patients

NEW YORK, July 25, 2024 /PRNewswire/ -- Actinium Pharmaceuticals, Inc. (NYSE AMERICAN: ATNM) (Actinium or the Company), a leader in the development of Antibody Radiation Conjugates (ARCs) and other targeted radiotherapies, today announced the FDA clearance of an Investigational New Drug (IND) application to study Iomab-ACT for targeted conditioning prior to a bone marrow transplant (BMT), in patients with sickle cell disease. The study will be conducted in collaboration with the Columbia University and led by Markus Mapara, M.D., Ph.D., Professor of Medicine, Columbia University Irving Medical Center, Director, Bone Marrow Transplantation and Cell Therapy Program, Vagelos College of Physicians and Surgeons. This trial will evaluate the safety of Iomab-ACT in patients with sickle cell disease who are to receive an allogeneic BMT. If successful, the trial is expected to inform a clinical trial to evaluate Iomab-ACT as a targeted conditioning agent prior to gene therapy for which there are two approved agents for patients with sickle cell disease, Casgevy (Vertex Pharmaceuticals, Inc.) and Lyfgenia (Bluebird Bio, Inc.). This collaboration with Dr. Mapara is intended to enable patients with sickle cell disease broader access to allogeneic BMT and gene therapy, potentially curable cellular therapies, via Iomab-ACT. Iomab-ACT is an ARC that targets CD45, a marker expressed on blood cancer cells and immune cells that is intended to enable conditioning prior to cell and gene therapies replacing the non-targeted chemotherapy and total body irradiation that is currently used for conditioning. It is the only CD45 targeting conditioning agent in clinical development.

(PRNewsfoto/Actinium Pharmaceuticals, Inc.)
(PRNewsfoto/Actinium Pharmaceuticals, Inc.)

Dr. Mapara, stated, "Undergoing chemotherapy- or total body irradiation-based conditioning for curative allogeneic bone marrow transplant or gene therapy often brings severe side effects for patients with sickle cell disease. These toxicities include organ damage, infections, infertility, and the potential for secondary malignancies. Leveraging extensive data from CD45 ARC conditioning in allogeneic bone marrow transplants, I am thrilled to lead this pioneering study using Iomab-ACT, a non-chemotherapeutic targeted radiotherapy conditioning, for patients with sickle cell disease. This innovative approach aims to minimize toxicity while ensuring complete donor hematopoiesis engraftment. Success in this trial could revolutionize treatment, enabling the use of a low-toxicity method for the engraftment of genetically engineered autologous stem cells in SCD patients."