- 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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