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

Rhea-AI Summary

Actinium Pharmaceuticals (NYSE AMERICAN: ATNM) announced FDA clearance for an IND application to study Iomab-ACT for targeted conditioning prior to bone marrow transplant (BMT) in sickle cell disease patients. The study, in collaboration with Columbia University, aims to evaluate Iomab-ACT's safety and potentially inform future gene therapy conditioning trials. Iomab-ACT, an Antibody Radiation Conjugate targeting CD45, could replace current non-targeted chemotherapy and total body irradiation conditioning methods, potentially reducing severe side effects and broadening access to curative cellular therapies. This initiative addresses the high unmet need among approximately 100,000 U.S. sickle cell patients annually. The study aligns with Actinium's strategy to expand Iomab-ACT's applications in transplant, cell therapy, and gene therapy conditioning, tapping into a growing market for CAR-T and gene therapies expected to reach 93,000 U.S. patients by 2030.

Positive

• FDA clearance of IND application for Iomab-ACT in sickle cell disease
• Collaboration with Columbia University for clinical trial
• Potential to broaden access to curative cellular therapies for sickle cell patients
• Iomab-ACT could replace current high-toxicity conditioning methods
• Expanding addressable market in transplant, cell therapy, and gene therapy conditioning
• Aligns with growing CAR-T and gene therapy market, expected to reach 93,000 U.S. patients by 2030

Negative

• Early-stage development, success not guaranteed
• Competitive landscape in cellular and gene therapies

Medical Research Analyst

The FDA clearance of Actinium Pharmaceuticals' IND application for Iomab-ACT in sickle cell disease (SCD) patients is a significant development in the field of targeted conditioning for bone marrow transplants (BMTs). This news has several important implications:

• Iomab-ACT, an Antibody Radiation Conjugate targeting CD45, could potentially revolutionize the conditioning process for SCD patients undergoing BMT or gene therapy.
• The current chemotherapy-based conditioning methods often result in severe side effects, limiting patient access to potentially curative treatments. Iomab-ACT aims to minimize these toxicities while ensuring effective engraftment.
• If successful, this approach could dramatically increase the number of SCD patients eligible for BMT or gene therapies, addressing a significant unmet medical need.

The collaboration with Columbia University adds credibility to this research. However, it's important to note that this is still an early-stage clinical trial and the efficacy and safety of Iomab-ACT in SCD patients remain to be proven. The potential impact on Actinium's market position and financials will depend on the trial outcomes and subsequent developments in gene therapy conditioning.

Financial Analyst

Actinium's advancement of Iomab-ACT into clinical trials for sickle cell disease (SCD) conditioning represents a strategic move to tap into a substantial market opportunity. Key financial implications include:

• The SCD market in the U.S. alone affects approximately 100,000 patients annually, presenting a significant addressable market.
• The broader cellular therapy conditioning market is projected to nearly double, reaching 93,000 patients in the U.S. by 2030.
• The CAR-T market, a related sector, is estimated to grow at a CAGR of 11% over the next 5+ years.
• In 2023, FDA-approved CAR T-cell therapies generated over $3.5 billion in total sales.

If Iomab-ACT proves successful, it could potentially capture a significant portion of this expanding market. However, investors should be cautious as the product is still in early clinical stages and success is not guaranteed. The company's ability to execute on this opportunity and navigate regulatory hurdles will be important for realizing the potential 'blockbuster revenue opportunity' mentioned.

Oncology Doctor

The development of Iomab-ACT for sickle cell disease (SCD) patients represents a potentially transformative approach in hematology-oncology. As an oncologist, I see several critical aspects:

• Current conditioning regimens using chemotherapy or total body irradiation often result in severe toxicities, including organ damage, infections and secondary malignancies. Iomab-ACT's targeted approach could significantly reduce these risks.
• The CD45-targeting mechanism of Iomab-ACT is particularly interesting, as it's the only such agent in clinical development for conditioning.
• If successful, this approach could extend beyond SCD to other hematological conditions requiring BMT or gene therapy.
• The potential to reduce cytokine release syndrome (CRS) and immune effector cell-associated neurotoxicity syndrome (ICANS) in CAR-T therapy is especially noteworthy, as these are significant barriers to broader adoption of cellular therapies.

While promising, it's important to await clinical trial results to assess Iomab-ACT's efficacy and safety profile in SCD patients. The success of this approach could indeed revolutionize treatment paradigms in hematology-oncology, but cautious optimism is warranted at this stage.

- 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.

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."

Sandesh Seth, Actinium's Chairman and CEO, added, "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."

Targeted Radiotherapy Conditioning Opportunity

The opportunity exists for better conditioning in other areas of cellular therapy, such as CAR-T as well as gene therapies. The pipeline of CAR-T and gene therapies has rapidly expanded, with the addressable patient population expected to nearly double and reach approximately 93,000 patients in the U.S. by 2030 based on the current pipeline of cellular therapies. The CAR-T market size in terms of revenue is estimated to grow at a CAGR of approximately 11% over the next 5 plus years. Currently, there are six CAR T-cell therapies approved by the FDA that are used to treat patients with lymphomas, leukemia, and multiple myeloma, which collectively had total sales of over $3.5 billion in 2023 and two gene therapies approved by the FDA for patients with sickle cell disease. The addressable market for Iomab-ACT is in line with the patient population for cell and gene therapies as all patients must receive some type of conditioning prior to these treatments. We will continue to develop Iomab-ACT, our next-generation conditioning program for rapidly growing cell and gene therapies based on early promising results, ultimately with the value proposition of improving overall access and outcomes for patients who need cellular or gene therapies. A potential blockbuster revenue opportunity exists for Iomab-ACT assuming it can provide one or more clinical benefits related to lower cytokine release syndrome (CRS), less immune effector cell-associated neurotoxicity syndrome (ICANS), longer duration of response with selective lymphodepletion or a higher overall success rate of cell and gene therapies due to benefits of targeted conditioning.

About Actinium Pharmaceuticals, Inc.

Actinium develops targeted radiotherapies to meaningfully improve survival for people who have failed existing oncology therapies. Advanced pipeline candidates Iomab-B (pre-BLA & MAA (EU)), an induction and conditioning agent prior to bone marrow transplant, and Actimab-A (National Cancer Institute CRADA), a therapeutic agent, have demonstrated potential to extend survival outcomes for people with relapsed and refractory acute myeloid leukemia. Actinium plans to advance Iomab-B for other blood cancers and next generation conditioning candidate Iomab-ACT to improve cell and gene therapy outcomes. Actinium holds more than 230 patents and patent applications including several patents related to the manufacture of the isotope Ac-225 in a cyclotron.

For more information, please visit: https://www.actiniumpharma.com/

Forward-Looking Statements

This press release may contain projections or other "forward-looking statements" within the meaning of the "safe-harbor" provisions of the private securities litigation reform act of 1995 regarding future events or the future financial performance of the Company which the Company undertakes no obligation to update. These statements are based on management's current expectations and are subject to risks and uncertainties that may cause actual results to differ materially from the anticipated or estimated future results, including the risks and uncertainties associated with preliminary study results varying from final results, estimates of potential markets for drugs under development, clinical trials, actions by the FDA and other governmental agencies, regulatory clearances, responses to regulatory matters, the market demand for and acceptance of Actinium's products and services, performance of clinical research organizations and other risks detailed from time to time in Actinium's filings with the Securities and Exchange Commission (the "SEC"), including without limitation its most recent annual report on form 10-K, subsequent quarterly reports on Forms 10-Q and Forms 8-K, each as amended and supplemented from time to time.

Investors:
investorrelations@actiniumpharma.com 

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SOURCE Actinium Pharmaceuticals, Inc.

FAQ

What is the purpose of Actinium's Iomab-ACT study for sickle cell disease?

The study aims to evaluate the safety of Iomab-ACT as a targeted conditioning agent prior to bone marrow transplant in sickle cell disease patients, potentially informing future gene therapy conditioning trials.

How many sickle cell disease patients could potentially benefit from Actinium's (ATNM) Iomab-ACT therapy?

Approximately 100,000 patients in the U.S. annually are affected by sickle cell disease and could potentially benefit from Iomab-ACT therapy if it proves successful.

What advantage does Iomab-ACT offer over current conditioning methods for sickle cell disease patients?

Iomab-ACT, as a targeted radiotherapy, aims to minimize toxicity compared to current non-targeted chemotherapy and total body irradiation methods, potentially reducing severe side effects and improving access to curative cellular therapies.

How does Actinium's (ATNM) Iomab-ACT fit into the broader cellular therapy market?

Iomab-ACT aligns with the growing CAR-T and gene therapy market, which is expected to reach approximately 93,000 U.S. patients by 2030, offering potential applications in transplant, cell therapy, and gene therapy conditioning.