Cellectar Biosciences Announces Enrollment of the First Patient in Pediatric High-Grade Gliomas Phase 1b Clinical Study

Rhea-AI Summary

Cellectar Biosciences, Inc. (NASDAQ: CLRB) has announced the enrollment of the first patient in its Phase 1b clinical trial for iopofosine I 131, a novel phospholipid radiotherapeutic conjugate, in pediatric patients with relapsed or refractory malignant high-grade gliomas (pHGG). The study is supported by a $2 million Fast Track SBIR grant from the National Institute of Health’s National Cancer Institute (NCI). The company aims to identify the optimal iopofosine I 131 Phase 2 recommended dose and schedule in pHGG patients and evaluate safety and tolerability. The treatment has shown promising results in a relapsed, refractory Bing-Neel Syndrome patient and demonstrated clinical activity across the blood-brain barrier in other hematologic malignancies. Cellectar is hopeful that its novel mechanism of action will provide a meaningful treatment option for pediatric patients with high-grade gliomas.

Oncology Doctor

High-grade gliomas in pediatric patients represent a particularly devastating diagnosis due to the aggressive nature of these tumors and the limited efficacy of current treatment options. The entry of iopofosine I 131 into Phase 1b trials signifies a potentially significant advancement in the field of pediatric oncology. This compound, as a phospholipid radiotherapeutic conjugate, offers a targeted approach that could minimize damage to surrounding healthy brain tissue, a critical consideration in treating children. The ability of iopofosine to cross the blood-brain barrier and its reported efficacy in hematologic malignancies suggest a promising therapeutic avenue for these young patients. The safety and tolerability profile will be crucial, as the pediatric population is particularly sensitive to the adverse effects of cancer treatments. If iopofosine I 131 can demonstrate a favorable balance between efficacy and safety, it could become a vital component of treatment protocols for pediatric high-grade gliomas.

Medical Research Analyst

The $2 million Fast Track Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI) underscores the potential impact of iopofosine I 131. Grants of this nature are competitive and are indicative of a high level of confidence in the therapeutic's potential by the granting body. The fact that this grant was awarded based on promising Phase 1a data is noteworthy, as it suggests that iopofosine has already shown a level of clinical activity that warrants further investigation. From a research perspective, the progression to a Phase 1b trial is an essential step in determining the optimal dosing regimen for efficacy and safety. The trial's outcome will not only affect the future of iopofosine I 131 but could also influence the direction of research and development in targeted radiotherapeutics for pediatric cancers.

Market Research Analyst

Cellectar Biosciences' advancement into Phase 1b clinical trials for iopofosine I 131 could have significant implications for the company's valuation and market position. Investors typically monitor clinical trial progress closely, as positive outcomes can lead to increased stock valuations due to the potential for new and effective treatments in high-need areas. Pediatric high-grade gliomas represent an area of high unmet medical need and the development of effective treatments could open a substantial market opportunity for Cellectar. The company's focus on a novel mechanism of action (MOA) and its ability to secure substantial funding from the NCI are positive indicators for investors. However, it's important to consider that clinical trial success rates are generally low and the path from Phase 1b to final approval is long and uncertain. Stakeholders should be cautious and consider the inherent risks of biopharmaceutical development when evaluating the potential long-term benefits of their investment.

Study of Iopofosine I 131 Supported by $2 Million Grant from the National Institute of Health’s National Cancer Institute (NCI)

FLORHAM PARK, N.J., Jan. 30, 2024 (GLOBE NEWSWIRE) -- Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery, development and commercialization of drugs for the treatment of cancer, today announced the first patient has been enrolled in its Phase 1b clinical trial evaluating iopofosine I 131, a novel phospholipid radiotherapeutic conjugate, in pediatric patients with relapsed or refractory malignant high-grade gliomas (pHGG).

“Treatment of pediatric patients with relapsed and refractory high-grade glioma and ependymoma is extremely challenging. There are limited treatment options and high unmet clinical need for patients suffering from this aggressive cancer,” said Dr. Sameer Farouk Sait, pediatric hematologist-oncologist and assistant attending at Memorial Sloan Kettering Cancer Center. “Iopofosine I-131’s recently reported complete central nervous system clearance in a relapsed, refractory Bing-Neel Syndrome patient and demonstrated clinical activity across the blood-brain barrier in other hematologic malignancies, which included a complete response in a relapsed/refractory primary CNS lymphoma patient, is highly encouraging. The novel MOA is exciting, and we look forward to evaluating iopofosine’s efficacy and safety with the goal of providing a meaningful treatment to pediatric patients and hope to their families.”

The open-label study (NCT05610891) will assess two dosing regimens to identify the optimal iopofosine I 131 Phase 2 recommended dose and schedule in pHGG patients and evaluate safety and tolerability. The study is supported by a $2 million Fast Track SBIR grant from the National Institute of Health’s National Cancer Institute (NCI), which was awarded based in part on the promising Phase 1a trial data.

“We understand the profound impact pediatric high-grade gliomas have on the lives of young patients and their families,” commented James Caruso, president and CEO of Cellectar. “There exists an urgent need for more effective, targeted therapies for this vulnerable population and we are rapidly advancing further evaluation of our lead phospholipid radioconjugate therapy in this important indication. Iopofosine I 131 showed great promise in our Phase 1a study, demonstrating the ability to cross the blood brain barrier and provide sustained progression free survival that compares favorably to historical data. We are hopeful its novel MOA will provide a meaningful treatment option for these young patients.”

About Pediatric High-Grade Gliomas (pHGG)
Pediatric high-grade gliomas are aggressive malignant tumors that form in the brain or spinal cord of children, adolescents, and young adults. Comprising a small but devastating portion of childhood cancers with an approximate incidence rate of 1,300 patients in the US (1.1-1.178 per 100,000 children), these tumors originate in the glial cells of the brain and spinal cord and are known for their rapid growth and resistance to conventional treatments. They are also responsible for over 40% of childhood brain tumor death and are the more common cause of tumor related death for children. The current standard of care for pediatric high-grade gliomas typically involves a combination of extensive brain surgery, radiation therapy, and chemotherapy at the time of initial diagnosis. However, the outcomes remain unsatisfactory for newly diagnosed patients and are dismal for relapsed or progressive disease. The five-year overall survival rate for high-grade gliomas in children is less than 20% and limited improvements were seen over the years. The disease's aggressive clinical nature and inherent genomic resistance often leads to significant challenges in achieving long-term remission and therapies with new mechanisms of action are urgently needed.

About Cellectar Biosciences, Inc.
Cellectar Biosciences is a late-stage clinical biopharmaceutical company focused on the discovery and development of proprietary drugs for the treatment of cancer, independently and through research and development collaborations. The company’s core objective is to leverage its proprietary Phospholipid Drug Conjugate™ (PDC) delivery platform to develop the next-generation of cancer cell-targeting treatments, delivering improved efficacy and better safety as a result of fewer off-target effects.

The company’s product pipeline includes lead asset iopofosine I 131, a small-molecule PDC designed to provide targeted delivery of iodine-131 (radioisotope), proprietary preclinical PDC chemotherapeutic programs and multiple partnered PDC assets.

For more information, please visit www.cellectar.com and www.wmclinicaltrial.com or join the conversation by liking and following us on the company’s social media channels: Twitter, LinkedIn, and Facebook.

Forward-Looking Statement Disclaimer

This news release contains forward-looking statements. You can identify these statements by our use of words such as "may," "expect," "believe," "anticipate," "intend," "could," "estimate," "continue," "plans," or their negatives or cognates. These statements are only estimates and predictions and are subject to known and unknown risks and uncertainties that may cause actual future experience and results to differ materially from the statements made. These statements are based on our current beliefs and expectations as to such future outcomes including our expectations regarding the WM CLOVER-WaM pivotal trial. Drug discovery and development involve a high degree of risk. Factors that might cause such a material difference include, among others, uncertainties related to the ability to raise additional capital, uncertainties related to the disruptions at our sole source supplier of iopofosine, the ability to attract and retain partners for our technologies, the identification of lead compounds, the successful preclinical development thereof, patient enrollment and the completion of clinical studies, the FDA review process and other government regulation, our ability to maintain orphan drug designation in the United States for iopofosine, the volatile market for priority review vouchers, our pharmaceutical collaborators' ability to successfully develop and commercialize drug candidates, competition from other pharmaceutical companies, product pricing and third-party reimbursement. A complete description of risks and uncertainties related to our business is contained in our periodic reports filed with the Securities and Exchange Commission including our Form 10-K for the year ended December 31, 2022, and our Form 10-Q for the quarter ended September 30, 2023. These forward-looking statements are made only as of the date hereof, and we disclaim any obligation to update any such forward-looking statements.

Contacts

MEDIA:
Claire LaCagnina
Bliss Bio Health
315-765-1462
clacagnina@blissbiohealth.com

INVESTORS:
Chad Kolean
Chief Financial Officer
investors@cellectar.com

FAQ

What is the purpose of the Phase 1b clinical trial for iopofosine I 131?

The trial aims to evaluate the optimal recommended dose and schedule of iopofosine I 131 in pediatric patients with relapsed or refractory malignant high-grade gliomas (pHGG) and assess its safety and tolerability.

What is the source of funding for the study?

The study is supported by a $2 million Fast Track SBIR grant from the National Institute of Health’s National Cancer Institute (NCI).

What are the potential benefits of iopofosine I 131 in pediatric patients?

Iopofosine I 131 has shown promising results in a relapsed, refractory Bing-Neel Syndrome patient and demonstrated clinical activity across the blood-brain barrier in other hematologic malignancies, offering hope for a meaningful treatment option for pediatric patients with high-grade gliomas.