On October 27, 2025 Cellectar Biosciences, Inc. (NASDAQ: CLRB), a late-stage clinical biopharmaceutical company focused on the discovery and development of drugs for the treatment of cancer, reported the U.S. Food and Drug Administration (FDA) has granted rare pediatric drug designation (RPDD) for iopofosine I 131 in inoperable relapsed or refractory pediatric high-grade glioma (r/r pHGG).

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Iopofosine I 131 is a potential first-in-class, novel cancer targeting agent utilizing a phospholipid ether as a radioconjugate monotherapy. The FDA previously granted Orphan Drug Designation for iopofosine I 131 for the treatment of pHGG.

"Receiving Rare Pediatric Disease Designation for iopofosine I 131 underscores its potential to address one of the most devastating cancers affecting children and young adults. Combined with the encouraging interim results from our CLOVER-2 pHGG study, which showed meaningful improvements in progression-free and overall survival, this designation further validates the promise of our targeted radiotherapeutic approach," stated James Caruso, president and CEO of Cellectar. "We believe iopofosine I 131 represents a compelling opportunity for strategic collaboration to accelerate development and bring a potentially first-in-class therapy to patients who urgently need new options."

The FDA’s Rare Pediatric Disease Designation program is intended to encourage the development of new therapies for serious and life-threatening diseases that primarily affect individuals under 18 years of age. If a New Drug Application (NDA) for iopofosine I 131 is approved, upon reauthorization of the program Cellectar may be eligible to receive a Priority Review Voucher (PRV), which can significantly expedite the review process for future New Drug Applications or Biologic License Applications, may be redeemed to receive priority review for another marketing application or may be sold or transferred.

Pediatric high-grade gliomas are a collection of aggressive tumors affecting the brain and central nervous system. As reported in the literature, median progression free survival (PFS) and overall survival (OS) for patients with relapsed pHGG is poor; approximately 2.25 months and 5.6 months, respectively.

Interim data from CLOVER-2, the company’s ongoing Phase 1b trial of iopofosine I 131 in children, adolescents and young adults with r/r pHGG at multiple sites in the United States and Canada, were highlighted in an oral presentation at the recent American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Pediatric Cancer that took place in late September 2025.

The company’s chief operating officer, Jarrod Longcor, delivered the update, "Precision Radiotherapy for Incurable Brain Tumors: Phase 1b Dose & Regimen Optimization Study of Iopofosine I 131 in Inoperable Relapsed or Refractory Pediatric High-Grade Glioma, Interim Data Assessment," which showed that all patients receiving a minimum of 55 mCi total administered dose (n=6) experienced an average of 5.4 months of PFS and 8.6 months of OS, ongoing. All patients experienced disease control, which according to the committee for the Response Assessment in Pediatric Neuro-Oncology (RAPNO) does correlate with survival benefit. Three patients who received additional dosing cycles (a minimum of four total infusions) had an average PFS of 8.1 months and an OS of 11.5 months (ranging from 4.9 to 14.9 months), ongoing, with two achieving an objective response (ORR).

Two case studies were highlighted in the oral presentation. Case Study 1 showed a 25-year-old male with diffuse hemispheric glioma with the H3 G34R/V mutation who had three prior therapies and who received a total administered dose of 126.6mCi of iopofosine I 131 over four doses (40mCi/m2/dose) had his target lesion reduced by more than 50% approximately eight months post screening. This patient had PFS of 10.9 months and survival is ongoing at greater than 18 months as of July 25, 2025.

Case Study 2 showed a 15-year-old female with ependymoma who had eight prior therapies and who received a total administered dose of 58.9mCi of iopofosine I 131 over four doses (20mCi/m2/dose) had her target lesion reduced from 252mm2 to approximately 141mm2. This patient had PFS of 11.2 months and her ongoing survival was greater than 17 months as of July 22, 2025.

Iopofosine I 131 was well tolerated and its toxicity profile was consistent with the company’s previously reported safety data. Importantly, patients on iopofosine I 131 did not experience any cardiovascular, renal, or liver toxicities, and no peripheral neuropathy or significant bleeding. The safety profile was consistent with selective targeting of tumor sites with clinically negligible off-target effect outside the hematologic system. The most frequently reported treatment emergent adverse events were hematologic in nature (thrombocytopenia, neutropenia and anemia) and were predictable and manageable. No treatment-related deaths were reported.

The complete presentation can be accessed on the company’s website here.

About Pediatric High-Grade Gliomas
Pediatric high-grade gliomas are a collection of aggressive tumors affecting the brain and central nervous system. The patients enrolled in CLOVER-2 with pHGG (n=14) were diagnosed with diffuse midline gliomas (DMG), ependymomas, diffuse intrinsic pontine gliomas (DIPG), diffuse hemispheric gliomas (DHG) and anaplastic ependymomas. As reported in the literature, median progression free survival (PFS) and overall survival (OS) for patients with relapsed pHGG is poor; approximately 2.25 months and 5.6 months, respectively. While MRI measures of tumor volume change can be helpful and are used as a surrogate in clinical trials, they often fail to predict survival.

About the CLOVER-2 Trial
The Phase 1b trial of iopofosine I 131 consists of children, adolescents and young adults with r/r pHGG at multiple sites in the United States and Canada. The study is designed to evaluate the safety and tolerability of iopofosine I 131 in two dosing cohorts, one cohort receiving two doses at 20mCi/m2 each separated by 14 days for two cycles with a third optional cycle. Patients in the second cohort will receive 10 mCi/m2 each, separated by 14 days for three cycles with a fourth optional cycle. The study will also determine therapeutic activity defined as progression free survival (PFS) and overall survival, antitumor activity defined as the reduction in tumor volume and identify the recommended Phase 2/3 dose of iopofosine I 131 in children, adolescents and young adults with r/r pHGG.

(Press release, Cellectar Biosciences, OCT 27, 2025, View Source [SID1234661196])

On October 27, 2025 Adaptam Therapeutics (‘Adaptam’), a biotech company pioneering cancer immunotherapies specifically targeting immunosuppressive myeloid cells, reported the successful completion of a €3 million (~$3.5 million) pre-seed financing round led by Criteria Bio Ventures.

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This financing round will enable Adaptam to further develop its programs and enter the preclinical phase in multiple oncology indications. As part of the round, Salvatore Cappadona, PhD, and Pablo Cironi, PhD, both from Criteria Bio Ventures, joined the board of directors.

Adaptam is developing first-in-class antibody-based therapeutic programs, including ADCs and bispecific antibodies, that target novel glyco-immune checkpoints, selectively expressed in immunosuppressive myeloid cells.

"Immunosuppressive myeloid cells within tumors present one of the toughest challenges in immunotherapy today. Our goal is to neutralize this obstacle by developing therapies that specifically target these cells, offering patients new hope where traditional approaches have failed," said Asis Palazon, PhD, founder, CEO and CSO of Adaptam. "The support from Criteria Bio Ventures enables us to take a step forward in achieving our mission. We are now actively planning our next funding round to support IND-enabling studies."

Immunotherapy has transformed cancer treatment, offering new solutions to patients worldwide. However, a significant number still fail to respond to these therapies or develop resistance over time. This lack of efficacy is primarily due to the immunosuppressive nature of the tumor microenvironment (TME), which hampers the immune system’s ability to effectively target and destroy cancer cells. Among the main contributors to this immunosuppression are myeloid cells, particularly TAMs, which play a crucial role in inhibiting T-cell responses. This challenge is particularly pronounced in patients with solid tumors, where these myeloid cells create an environment that prevents immune cells from mounting an effective anti-tumoral response.

"We are thrilled to support Adaptam in its mission to transform cancer treatment by targeting the immunosuppressive cells within the TME. The scientific breakthroughs achieved by Prof. Asis Palazon and his team have laid the groundwork for potentially life-changing therapies," said Pablo Cironi, PhD, chairman of the Adaptam board. "With this strong foundation, we believe Adaptam is well-positioned to deliver new, effective treatments for patients with solid tumors, addressing critical gaps in current immunotherapy options."

"Adaptam’s differentiated approach at the intersection of myeloid-mediated immunosuppression and glycobiology represents a one-of-a-kind vision uniquely positioned to unlock the next frontier in cancer immunotherapy. We are proud to have partnered with Asis Palazon to build this exciting new venture and support its next phase of development. We are confident that Adaptam’s emerging pipeline offers a game-changing opportunity to transform cancer treatment for many patients," said Salvatore Cappadona, PhD, board member of Adaptam.

Adaptam originated from research conducted by Prof. Asis Palazon and his team at CIC bioGUNE in Bilbao, Spain, with expert insights from Criteria Bio Ventures on the ideation, corporate strategy and operational setup of the company. Asis Palazon also received a Caixa Research Health grant from "la Caixa" Foundation in 2021. Some of the core structural and mechanistic discoveries of immune-modulating glycan-binding proteins and their interactions with immune cells were previously published in Nature Communications.

"CIC bioGUNE’s long-standing leadership in glycobiology provided the scientific foundation for Adaptam and we have supported the company from its earliest steps through to today’s preclinical maturation," said Prof. Jesús Jiménez-Barbero, scientific director of CIC bioGUNE.

(Press release, Adaptam Therapeutics, OCT 27, 2025, View Source [SID1234657099])

On October 27, 2025 Adaptam Therapeutics (‘Adaptam’), a biotech company pioneering cancer immunotherapies specifically targeting immunosuppressive myeloid cells, reported the successful completion of a €3 million (~$3.5 million) pre-seed financing round led by Criteria Bio Ventures (Press release, Adaptam Therapeutics, OCT 27, 2025, View Source [SID1234657091]).

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This financing round will enable Adaptam to further develop its programs and enter the preclinical phase in multiple oncology indications. As part of the round, Salvatore Cappadona, PhD, and Pablo Cironi, PhD, both from Criteria Bio Ventures, joined the board of directors.

Adaptam is developing first-in-class antibody-based therapeutic programs, including ADCs and bispecific antibodies, that target novel glyco-immune checkpoints, selectively expressed in immunosuppressive myeloid cells.

"Immunosuppressive myeloid cells within tumors present one of the toughest challenges in immunotherapy today. Our goal is to neutralize this obstacle by developing therapies that specifically target these cells, offering patients new hope where traditional approaches have failed," said Asis Palazon, PhD, founder, CEO and CSO of Adaptam. "The support from Criteria Bio Ventures enables us to take a step forward in achieving our mission. We are now actively planning our next funding round to support IND-enabling studies."

Immunotherapy has transformed cancer treatment, offering new solutions to patients worldwide. However, a significant number still fail to respond to these therapies or develop resistance over time. This lack of efficacy is primarily due to the immunosuppressive nature of the tumor microenvironment (TME), which hampers the immune system’s ability to effectively target and destroy cancer cells. Among the main contributors to this immunosuppression are myeloid cells, particularly TAMs, which play a crucial role in inhibiting T-cell responses. This challenge is particularly pronounced in patients with solid tumors, where these myeloid cells create an environment that prevents immune cells from mounting an effective anti-tumoral response.

"We are thrilled to support Adaptam in its mission to transform cancer treatment by targeting the immunosuppressive cells within the TME. The scientific breakthroughs achieved by Prof. Asis Palazon and his team have laid the groundwork for potentially life-changing therapies," said Pablo Cironi, PhD, chairman of the Adaptam board. "With this strong foundation, we believe Adaptam is well-positioned to deliver new, effective treatments for patients with solid tumors, addressing critical gaps in current immunotherapy options."

"Adaptam’s differentiated approach at the intersection of myeloid-mediated immunosuppression and glycobiology represents a one-of-a-kind vision uniquely positioned to unlock the next frontier in cancer immunotherapy. We are proud to have partnered with Asis Palazon to build this exciting new venture and support its next phase of development. We are confident that Adaptam’s emerging pipeline offers a game-changing opportunity to transform cancer treatment for many patients," said Salvatore Cappadona, PhD, board member of Adaptam.

Adaptam originated from research conducted by Prof. Asis Palazon and his team at CIC bioGUNE in Bilbao, Spain, with expert insights from Criteria Bio Ventures on the ideation, corporate strategy and operational setup of the company. Asis Palazon also received a Caixa Research Health grant from "la Caixa" Foundation in 2021. Some of the core structural and mechanistic discoveries of immune-modulating glycan-binding proteins and their interactions with immune cells were previously published in Nature Communications.

"CIC bioGUNE’s long-standing leadership in glycobiology provided the scientific foundation for Adaptam and we have supported the company from its earliest steps through to today’s preclinical maturation," said Prof. Jesús Jiménez-Barbero, scientific director of CIC bioGUNE.

On October 27, 2025 Guardant Health (Nasdaq: GH), a leading precision oncology company, and Zephyr AI, a leader in precision medicine harnessing artificial intelligence to accelerate drug development, reported a strategic partnership to deliver novel, scalable, and actionable insights that support biopharmaceutical innovation. This collaboration will combine unique multimodal molecular data and AI/ML technology from both companies driving advancement of novel cancer biomarkers for drug development, targeted therapies selection, and response monitoring.

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The partnership will be integrated with Guardant’s broader suite of Infinity AI capabilities and exemplifies both companies’ broader commitment to harnessing AI and multimodal data to empower oncologists, researchers, and biopharma developers with solutions to transform cancer patient care.

"At Guardant Health, we’ve always believed that data is key to conquering cancer," said Helmy Eltoukhy, chairman and co-CEO of Guardant Health. "By combining our industry-leading molecular data with Zephyr AI’s advanced analytics platform, we’re taking another major step toward realizing the full potential of precision oncology—helping our biopharma partners accelerate drug development and ultimately deliver better outcomes for patients worldwide."

Through the combination of Guardant Health’s multimodal real-world data and precision oncology insights and Zephyr’s proprietary AI technologies, the partnership will accelerate research and development for biopharma partners. This model is unique in its capability to generate predictions of targeted cancer therapy response validated by real-world data, integrating biologic interpretability features that result in actionable scientific intelligence.

"This collaboration represents the convergence of unmatched real-world data, leading-edge diagnostics and cutting-edge machine learning to enable more precise, scalable, and impactful oncology solutions," said Allen Chao, CEO of Zephyr AI. "By working together, Guardant Health and Zephyr AI can supercharge discovery and development needed to transform cancer treatment and deliver on the promise of personalized medicines for cancer patients."

(Press release, Guardant Health, OCT 27, 2025, View Source [SID1234657049])

On October 27, 2025 OncoNano Medicine, Inc. ("OncoNano") reported encouraging first-in-human results from Part 1 of its Phase 1 ON-5001 trial evaluating ONM-501, a dual-acting STING agonist, as monotherapy and in combination with Libtayo (cemiplimab), Regeneron’s PD-1 inhibitor, in patients with advanced solid tumors and lymphomas. The findings were shared during a poster presentation at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston, Massachusetts.

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ONM-501 is designed to activate the STING pathway and stimulate both innate and adaptive immune responses within the tumor microenvironment. By linking a cyclic dinucleotide (cGAMP) to a proprietary polymer, ONM-501 prolongs STING activation and overcomes key limitations of earlier STING agonists. In preclinical models, this approach produced durable anti-tumor responses without triggering systemic inflammation, an effect now supported by emerging clinical results.

In the dose-escalation and dose-finding study, ONM-501 was well tolerated and demonstrated early signs of clinical benefit. Among 39 patients treated (15 with ONM-501 monotherapy and 24 in combination with cemiplimab), no dose-limiting toxicities were observed, and the most common adverse events were mild fatigue and injection-site reactions. Systemic interferon-γ levels remained within normal limits across all cohorts.

In the monotherapy arm, one patient achieved an objective response and three patients had prolonged stable disease, including a case of recurrent, immunotherapy-refractory uveal melanoma with disease control lasting over one year (390 days). In the combination arm, five patients experienced objective responses, including two complete in patients with cutaneous squamous cell carcinoma (cSCC).

"These data provide the first clinical evidence that sustained STING activation through our proprietary polymer conjugate can drive meaningful immune responses in solid tumors," said Kartik Krishnan, MD, PhD, Chief Executive Officer of OncoNano Medicine. "The safety profile and emerging efficacy signals, particularly in patients with advanced cutaneous malignancies, highlight the potential of ONM-501 to expand the reach of STING-based immunotherapy."

Part 2 of the ON-5001 study (NCT06022029) is now open and enrolling patients with advanced basal cell carcinoma, cutaneous squamous cell carcinoma and melanoma.

About ONM-501
ONM-501 is a dual-activating agonist of the stimulator of interferon genes ("STING") pathway composed of cGAMP (the endogenous activator of STING) linked to a proprietary pH-activated polymer (the OMNI polymer). ONM-501 is presently being studied in a Phase 1 clinical trial (ON-5001). In preclinical models, the dual activation of STING by ONM-501 has been shown to lead to a direct anti-tumor effect, as well as leading to an anti-tumor immune response over an extended period of time. Development of ONM-501 was funded in part by the Cancer Prevention and Research Institute of Texas.

(Press release, OncoNano Medicine, OCT 27, 2025, View Source [SID1234657048])