On March 27, 2025 REGiMMUNE Limited, a biotech company focused on the regulatory T cell targeting drugs for immunotherapy, reported the termination of its potential merger with Kiji Therapeutics (Press release, REGimmune, MAR 27, 2025, View Source [SID1234651537]). This decision was made after thorough discussions and mutual agreement, considering strategic adjustments and business objectives.

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Kenzo Kosuda, CEO of REGiMMUNE stated: "We appreciated Kiji’s cooperation and shared achievements throughout this potential merger. While this chapter closes, we remain open to other potential collaborations in the future."

On March 27, 2025 Rakovina Therapeutics Inc. (TSX-V: RKV) (FSE: 7JO), a biopharmaceutical company advancing innovative cancer therapies through artificial intelligence (AI)-powered drug discovery, reported that two of its abstracts have been accepted for presentation at the upcoming 2025 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place April 25-30 in Chicago, Illinois (Press release, Rakovina Therapeutics, MAR 27, 2025, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-unveils-preclinical-data-at-the-aacr-worlds-premier-cancer-research-forum [SID1234651536]).

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The AACR (Free AACR Whitepaper) Annual Meeting is widely considered the most prestigious global forum for cancer research. It is where the world’s leading scientists, clinicians, and biotech innovators gather to unveil next-generation oncology breakthroughs. Rakovina’s acceptance to present, highlights growing recognition for its pioneering work at the intersection of AI technology and precision cancer therapy.

AI-Designed Therapies Poised to Transform Cancer Treatment

Rakovina will present two abstracts at the meeting, the first: Discovery of novel PARP1-selective inhibitors for treatment of brain tumors using artificial intelligence, explores the use of artificial intelligence (AI) to develop a novel, brain-penetrant, PARP1-selective inhibitor for treating brain tumors. Current PARP inhibitors face challenges such as poor blood-brain barrier (BBB) permeability and off-target effects due to PARP2 inhibition. By leveraging Deep Docking, a deep learning-based virtual screening method, alongside generative AI algorithms, researchers rapidly identified potential compounds with strong PARP1 selectivity and BBB penetration. The study presents findings from in silico screening of billions of compounds, followed by in vitro and in vivo validation of their efficacy, selectivity, and pharmacokinetic properties. This AI-driven approach enhances the efficiency of drug discovery, offering a promising new treatment option for brain tumors.

The second abstract: Utilizing artificial intelligence for the discovery of a novel CNS-penetrating ATR inhibitor, highlights the use of artificial intelligence (AI) to develop a novel CNS-penetrating ATR inhibitor for treating brain tumors. ATR plays a key role in DNA damage repair, and while ATR inhibitors show therapeutic potential, current options have poor blood-brain barrier (BBB) permeability, limiting their effectiveness against brain tumors and metastases. The Enki platform, an AI-driven approach utilizing generative models and deep learning, was employed to design de novo molecules with optimized potency, selectivity, and BBB penetration. Preliminary results include AI-generated ATR inhibitors validated for target specificity, metabolic stability, and permeability.

"This is an exceptional accomplishment for Rakovina," said Jeffrey Bacha, Executive Chairman of Rakovina Therapeutics. "Being selected by AACR (Free AACR Whitepaper) for two projects highlights the significance of our innovations in drug discovery and their potential to impact patients battling aggressive, treatment-resistant cancers."

The company’s proprietary integration of Deep Docking and Enki AI platforms allows its scientists to evaluate billions of potential compounds at 100x the speed of traditional methods, with 6,000x greater enrichment of viable candidates. These innovations are further supported by Rakovina’s access to the University of British Columbia’s state-of-the-art wet lab infrastructure, enabling rapid in-house testing and optimization.

"What we’re seeing with Rakovina’s AI-enabled pipeline is the future of oncology—faster, smarter, and more precise," said Dr. Mads Daugaard, President and CSO of Rakovina "This integration of generative AI and biological insight is transforming how—and how quickly—we can identify and advance new therapies."

With a world-class team, including the creator of Deep Docking and a former AstraZeneca DDR program director, Rakovina is driving innovation in a space projected to reach $18 billion annually by 2030. The company’s preclinical pipeline is focused on therapies that target DNA-repair vulnerabilities present in up to 75% of solid tumors, with an emphasis on hard-to-treat cancers such as breast, ovarian, prostate, and brain.

On March 27, 2025 Portage Biotech Inc. (NASDAQ: PRTG), a clinical-stage immuno-oncology company with a portfolio of innovative therapeutics, reported new preclinical data for PORT-7 (TT-4), a selective Adenosine A2B receptor inhibitor, generated by Dr. Luciano Mutti, Gruppo Italiano Mesotelioma e Oncologia Ambientale, at the 2025 European Lung Cancer Congress (ELCC), held in Paris, France March 26-29 (Press release, Portage Biotech, MAR 27, 2025, View Source [SID1234651535]). The new data demonstrate both single agent activity for PORT-7, and a dramatic >90% inhibition of tumor growth when PORT-7 was combined with an anti-PD1 antibody in a murine model of mesothelioma. Immunohistochemistry of the tumors revealed a significant infiltration of both CD3 and CD45 positive immune effector cells. Mesothelioma is an aggressive cancer with limited treatment options in need of novel approaches to overcome immune resistance. To our knowledge, this is the first report of antitumor activity against mesothelioma using a selective A2B receptor inhibitor. Portage is making preparations to commence a first-in-human clinical trial with PORT-7.

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Combining PORT-6 and PORT-7 for a More Comprehensive Immunotherapy Approach

In parallel, Portage is advancing the dose escalation of PORT-6, a potent and selective inhibitor of the A2A adenosine receptor. Portage’s plan is to ultimately co-administer PORT-6 with PORT-7 in the ongoing ADPORT-601 trial. This will mark the first time two highly selective A2A and A2B antagonists are combined in patients, with the aim of achieving a complete blockade of adenosine-induced immunosuppression in the tumor microenvironment. This innovative approach is designed to fully neutralize adenosine-mediated immune suppression, enhance anti-tumor responses, and broaden the impact of immunotherapy in solid tumors.

On March 27, 2025 PDS Biotechnology Corporation (Nasdaq: PDSB) ("PDS Biotech" or the "Company"), a late-stage immunotherapy company focused on transforming how the immune system targets and kills cancers, reported a clinical programs update and announced financial results for the full year ended December 31, 2024 (Press release, PDS Biotechnology, MAR 27, 2025, View Source [SID1234651534]).

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"We are pleased with the progress made over the past year and in recent weeks, culminating with the initiation of our VERSATILE-003 Phase 3 clinical trial evaluating Versamune HPV in recurrent/metastatic ("R/M") HPV16-positive head and neck squamous carcinoma ("HNSCC")," said Frank Bedu-Addo, PhD, President and Chief Executive Officer of PDS Biotech. "Patients with HPV16-positive HNSCC represent a large and rapidly growing subgroup in need of targeted therapies to treat the underlying cause of the cancer. Considering the strength and durability of the clinical responses observed in our VERSATILE-002 study, we are excited that this registrational trial is underway and are confident in the potential of our innovative combination therapy to improve patient outcomes and enhance the standard of care. In the coming weeks, we expect to continue activating additional clinical sites, and look forward to the progression of this registrational trial."

Clinical Strategy Summary

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Phase 3 VERSATILE-003 Trial in HPV16-positive first-line recurrent and/or metastatic HNSCC

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Announced activation of the first site in March 2025

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Trial designed to include approximately 350 patients

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PDS Biotech is aligned with the U.S. Food and Drug Administration ("FDA") on the design of the registrational trial and clinical endpoints.

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Two-arm controlled trial with 2:1 randomization

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Median overall survival is primary endpoint

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Design based on strong and durable responses seen in the VERSATILE-002 trial

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Interim readouts included in study design

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The Company received Fast Track designation from the FDA for the combination of Versamune HPV and pembrolizumab in R/M HNSCC.

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For more information on VERSATILE-003, visit ClinicalTrials.gov (Identifier: NCT06790966)

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In March 2025, announced FDA Clearance of Investigational New Drug ("IND") application for the combination of Versamune MUC1 and PDS01ADC to treat MUC1-positive unresectable, metastatic colorectal carcinoma.

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Phase 1/2 clinical trial evaluating the proprietary combination to be run under PDS Biotech’s Cooperative Research and Development Agreement with the National Cancer Institute

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Second Versamune platform candidate targets MUC1-positive solid tumors

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In October 2024, updated results from the IMMUNOCERV Phase 2 clinical trial in locally advanced cervical cancer patients treated with Versamune HPV and chemoradiotherapy were presented at the 66th American Society for Radiation Oncology (ASTRO) Annual Meeting.

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In October 2024, the rationale and design of a recurrent prostate cancer trial combining Xtandi + PDS01ADC vs. Xtandi were presented at the 12th Annual Meeting of the International Cytokine and Interferon Society (Cytokines 2024).

Full Year 2024 Financial Results

Net loss for the year ended December 31, 2024, was approximately $37.6 million, or $1.03 per basic and diluted share, compared to a net loss of $42.9 million, or $1.39 per basic and diluted share, for the year ended December 31, 2023. The reduced net loss was primarily the result of decreased operating expenses, partially offset by increased net interest expense.

Research and development expenses for the year ended December 31, 2024, were $22.6 million, compared to $27.8 million for the year ended December 31, 2023. The decrease of $5.2 million was primarily attributable to decreases in clinical costs of $4.1 million, personnel costs of $1.0 million and professional fees of $0.1 million.

General and administrative expenses for the year ended December 31, 2024, were $13.8 million, compared to $15.3 million for the year ended December 31, 2023. The $1.5 million decrease was primarily attributable to decreases in professional fees of $1.3 million and facilities costs of $0.2 million.

Total operating expenses for the year ended December 31, 2024, were $36.3 million, compared to $43.0 million for the year ended December 31, 2023.

Net interest expense was $2.2 million for the year ended December 31, 2024, compared to $1.3 million for the year ended December 31, 2023. The change was due to increased debt interest and lower interest income on the Company’s cash balances.

On February 27, 2025, the Company announced an up to $22 million registered direct offering. The securities purchase agreements with new and existing healthcare focused institutional investors included $11 million of upfront gross proceeds, with up to an additional $11 million of aggregate gross proceeds upon cash exercise in full of warrants issued to the investors.

The Company’s cash balance as of December 31, 2024, was $41.7 million.

Conference Details

Date: March 27, 2025
Time: 8:00 a.m. Eastern Time
Dial-in: 1-877-704-4453 (Domestic) or 1-201-389-0920 (International)
Webcast Registration: Click Here
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On March 27, 2025 NANOBIOTIX (Euronext: NANO –– NASDAQ: NBTX – the ‘‘Company’’), a late-stage clinical biotechnology company pioneering disruptive, nanotherapeutic approaches to revolutionize treatment outcomes for millions of patients, reported the first data from the completed dose escalation part of a Phase 1 study sponsored by The University of Texas MD Anderson Cancer Center ("MD Anderson") evaluating radiotherapy-activated NBTXR3 (JNJ-1900)1 as a second or later line (2L+) therapy for patients with locally advanced non-small cell lung cancer ("NSCLC") amenable to re-irradiation (Press release, Nanobiotix, MAR 27, 2025, View Source [SID1234651532]). These data will be presented at the 2025 European Lung Cancer Conference by study principal investigator Dr. Saumil Gandhi.

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ABSTRACT #207P: Phase 1 Study of Reirradiation ("ReRT") with NBTXR3 (JNJ-1900) for Inoperable Locoregional Recurrent Non-Small Cell Lung Cancer ("NSCLC")
Saumil J. Gandhi, MD, PhD, Enoch Chang, MD, Aileen Chen, MD, Stephen G. Chun, MD, Steven H. Lin, MD, PhD, Rachel C. Maguire, BS, Matthew S. Ning, MD, MPH, Julianna K. Bronk, MD, PhD, David Qian, MD, Joe Y. Chang, MD, PhD, James W. Welsh, MD, Zhongxing Liao, MD, Rahul A. Sheth, MD, Roberto F. Casal, MD

Locoregional recurrence occurs in 30-40% of patients with locally advanced NSCLC after treatment with definitive chemoradiation. Historically, patients who are amenable to re-irradiation are often limited to palliative doses due to associated toxicities. As such, strategies to enhance the therapeutic ratio of radiotherapy are needed to improve treatment outcomes.

"Patients with recurrence after prior radiation therapy for locally advanced lung cancer face limited therapeutic options and significant challenges in achieving durable disease control," said Saumil Gandhi, MD, PhD, Department of Thoracic Radiation Oncology, Division of Radiation Oncology at MD Anderson. "These data underscore the need for continued therapeutic innovation for these patients and highlight the potential of NBTXR3 (JNJ-1900) as a novel approach to improving patient outcomes."

Results from the completed dose escalation part of the study demonstrated a favorable safety profile with no dose-limiting toxicities (DLTs), and no Grade 3 or higher SAEs related to NBTXR3. Injection feasibility was confirmed, and the recommended phase 2 dose (RP2D) was established at 33% of gross tumor volume.

Promising early efficacy signals were observed. Preliminary review of survival data from 12 patients showed 12-month LPFS of 64% (median 18.6 months) and 12-month OS of 83% (median 30.2 months), further supporting the potential clinical benefit of NBTXR3 (JNJ-1900) in this patient population.

"The Nanobiotix team is encouraged by these early findings, which suggest NBTXR3 (JNJ-1900) could offer a new therapeutic option for patients with no alternatives after prior treatments have failed," said Louis Kayitalire, MD, Nanobiotix Chief Medical Officer. "Notably, these results were observed in patients who resisted prior curative radiation doses and that were treated with JNJ-1900 (NBTXR3) activated by a lower radiation dose. As we advance the study’s expansion phase, we look forward to further evaluating NBTXR3 (JNJ-1900)’s potential to improve patient outcomes."

The expansion phase of the study is ongoing, with 5/12 patients injected to date.

About JNJ-1900 (NBTXR3)

NBTXR3 is a novel, potentially first-in-class oncology product composed of functionalized hafnium oxide nanoparticles that is administered via one-time intratumoral injection and activated by radiotherapy. Its proof-of-concept was achieved in soft tissue sarcomas for which the product received a European CE mark in 2019. The product candidate’s physical mechanism of action (MoA) is designed to induce significant tumor cell death in the injected tumor when activated by radiotherapy, subsequently triggering adaptive immune response and long-term anti-cancer memory. Given the physical MoA, Nanobiotix believes that NBTXR3 could be scalable across any solid tumor that can be treated with radiotherapy and across any therapeutic combination, particularly immune checkpoint inhibitors.

Radiotherapy-activated NBTXR3 is being evaluated across multiple solid tumor indications as a single agent or in combination with anti-PD-1 immune checkpoint inhibitors, including in NANORAY-312—a global, randomized Phase 3 study in locally advanced head and neck squamous cell cancers. In February 2020, the United States Food and Drug Administration granted regulatory Fast Track designation for the investigation of NBTXR3 activated by radiation therapy, with or without cetuximab, for the treatment of patients with locally advanced HNSCC who are not eligible for platinum-based chemotherapy—the same population being evaluated in the Phase 3 study.

Given the Company’s focus areas, and balanced against the scalable potential of NBTXR3, Nanobiotix has engaged in a collaboration strategy to expand development of the product candidate in parallel with its priority development pathways. Pursuant to this strategy, in 2019 Nanobiotix entered into a broad, comprehensive clinical research collaboration with The University of Texas MD Anderson Cancer Center to sponsor several Phase 1 and Phase 2 studies evaluating NBTXR3 across tumor types and therapeutic combinations. In 2023, Nanobiotix announced a license agreement for the global co-development and commercialization of NBTXR3 with Janssen Pharmaceutica NV, a Johnson & Johnson company.