FDA Clears Investigational New Drug Application for Actinium Pharmaceuticals’ Iomab-ACT in Patients with Sickle Cell Disease

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The FDA has cleared Actnium Pharmaceuticals’ Investigational New Drug (IND) application to study Iomab-ACT, a targeted radiotherapy conditioning agent, developed in a partnership with Columbia University for the treatment of patients with sickle cell disease (SCD) who are undergoing allogeneic bone marrow transplants (BMT). The study, which will be led by Markus Mapara, MD, PhD, professor of medicine, Colombia University Irving Medical Center, director, Bone Marrow Transplantation and Cell Therapy Program, Vagelos College of Physicians and Surgeons, will evaluate the safety of Iomab-ACT in this patient population.¹

"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,” said Mapara, in a press release. “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."

Iomab-ACT targets CD45, a marker on blood cancer and immune cells, aiming to replace traditional chemotherapy and total body irradiation conditioning methods, which typically result in extreme adverse events (AEs), according to Actinium. The company stated that this trial has the potential to influence broader access to curative therapies for SCD, such as BMT and gene therapy. Actinium further stated that this therapeutic approach may revolutionize treatment by providing a low-toxicity method for engrafting genetically engineered autologous stem cells in SCD patients.

Iomab-ACT’s targeted conditioning approach is also currently being studied for broader applications in cellular therapies, such as chimeric antigen receptor (CAR) T-cell therapies and gene therapies, with Actinium citing the rapidly expanding pipeline of these therapies. The number of patients treated with cellular therapies is estimated to reach 93,000 in the United States by 2030 based on the current pipeline, with an estimated CAGR growth rate of 11% over the next five years, according to Actinium.¹

In the United States, SCD effects an estimated 100,000 people, according to the Centers for Disease Control and Prevention, over 90% of whom are non-Hispanic Black or African American, while 3%–9% are Hispanic or Latino. Currently, life expectancy in the United States for people with SCD is 20 years lower than the global average. SCD is most common in Saudi Arabia, India, Sub-Saharan Africa, Spanish-speaking regions in the Western Hemisphere, and a number of Mediterranean countries.²

"We are honored to collaborate with Dr. Mapara on this important initiative to address the significant number of patients with sickle cell disease who do not pursue or are not able to access transplant or gene therapies due to the required chemotherapy-based conditioning and its severe toxicities. Sickle cell disease represents a large and high unmet need that needs better treatment options and outcomes. This exciting program in sickle cell disease adds to our recently announced commercial CAR T trial addressing the large patient population with blood cancers that can also benefit from broader access to cellular therapy via targeted conditioning. We look forward to further expanding Iomab-ACT's already large addressable patient opportunity in transplant, cell therapy and gene therapy conditioning,” said Sandesh Seth, chairman, CEO, Actinium Pharmaceuticals, in the press release.

References

1. 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. Actinium. July 25, 2024. Accessed July 25, 2024. https://ir.actiniumpharma.com/press-releases/detail/484 

2. Data and Statistics on Sickle Cell Disease. CDC. Accessed July 25, 2024. https://www.cdc.gov/sickle-cell/data/index.html#:~:text=Sickle%20cell%20disease%20(SCD)%20affects,shorter%20than%20the%20average%20expected.