On October 14, 2025 AB Science SA (Euronext – FR0010557264 – AB) reported an update on the Phase 1 study of the molecule AB8939 and, in particular, on the initial clinical data for the combination of AB8939 + Venetoclax in the first three patients with acute myeloid leukemia (AML) associated with a very unfavorable genetic profile.

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AB8939 is a drug candidate that jointly targets cancer cells by destabilizing microtubules, which are essential for cell division, and also targeting cancer stem cells by inhibiting enzymes (ALDH1A1 and ALDH2) that are essential for maintaining their physiological state and survival.

AB8939 is currently being evaluated in a Phase 1 clinical trial (study AB18001, NCT05211570) in patients with refractory and relapsed AML.

The Phase 1 clinical trial of AB8939 has completed its first two stages, which consisted of determining the maximum tolerated dose (MTD) after 3 and 14 consecutive days of monotherapy, respectively. In both cases, the MTD was 21.3 mg/m².

The third stage, currently underway, involves evaluating the combination of AB8939 + Venetoclax. Three patients were evaluated at the first dose level (AB8939 14 days at a dose of 16 mg/m² + Venetoclax 14 days).

Nicholas J. Short, MD, Associate Professor and Co-Lead of Section of Developmental Therapeutics, Department of Leukemia, MD Anderson Cancer Center, said, "The results observed in these high-risk AML patients are impressive, particularly in the two patients whose leukemia had progressed on venetoclax. These initial results are very encouraging and justify the continuation of patient treatment with additional cycles, particularly in view of the mechanism of AB8939 on cancerous hematopoietic stem cells."

At the end of this third stage, an AB8939 + venetoclax expansion phase will be initiated in a group of about 15 patients with a more homogeneous profile than in the previous stages of phase 1, namely patients in their second- or third-line of treatment and with a poor prognosis (TP53 mutant, MECOM, NRAS mutant) in order to confirm the initial promising clinical data before initiating a registration clinical trial.

Virtual conference

AB Science will hold a virtual conference on Thursday, October 16, 2025, from 2pm to 3pm CET.

ZOOM link to the conference (audio + presentation): Access to the conference.

The purpose of this virtual conference will be to present in more detail the initial clinical data on the combination of AB8939 + venetoclax in the first three patients with refractory and relapsed AML associated with a very unfavorable genetic profile.

The following individuals will participate in the virtual conference:

Nicholas J. Short, MD, Associate Professor and Co-Lead of Section of Developmental Therapeutics, Department of Leukemia, MD Anderson Cancer Center

Professor Short is a clinical and translational investigator in adult acute leukemias, with a particular emphasis on the development of phase I and II investigator-initiated clinical trials of novel agents and combinations for patients with acute myeloid leukemia (AML) and acute lymphoblastic leukemia (ALL). His major contributions to leukemia research include: developing new immunotherapy-based frontline regimens in Philadelphia chromosome-negative B-cell ALL, developing chemotherapy-free regimens in Philadelphia chromosome-positive ALL, establishing the clinical utility of high-sensitivity next-generation sequencing-based MRD assays in ALL, developing novel MRD-directed therapies in AML and ALL, and developing novel regimens for older adults with FLT3-mutated AML. He serves as principal investigator or co-principal investigator on over a dozen phase I and II clinical trials and has authored over 250 peer-reviewed manuscripts in the field of leukemia. For his clinical and translational accomplishments in the field of leukemia, he has been awarded the ASCO (Free ASCO Whitepaper) Young Investigator Award and the ASH (Free ASH Whitepaper) Junior Faculty Scholar in Clinical Research.

Olivier Hermine, MD, PhD, Head of the Hematology Department at Necker-Enfants Malades Hospital, Paris, France

Olivier Hermine is Professor of Hematology at Paris Descartes University, Head of the Hematology Department at Necker-Enfants Malades Hospital, member of LYSA, and Director of the CALYM team "Cellular and Molecular Mechanisms of Hematological Disorders and Therapeutic Implications" at the IMAGINE Inserm U 116 CNRS ERL 8654 institute. He is also coordinator of the Reference Center for Mastocytosis (CeReMast), co-founder and director of the scientific committee of AB Science.

His research topics include lymphoproliferative disorders linked to the hepatitis C virus, mantle cell lymphomas, and the regulation of erythropoiesis. He is the author or co-author of more than 900 scientific publications.

Christian Auclair, PharmD, PhD, Emeritus Professor

Professor Auclair holds a doctorate in pharmaceutical sciences. He is co-founder and former director of the doctoral school of oncology at the Faculty of Medicine of Paris-Saclay University. He is former director of the biology department at the École Normale Supérieure de Cachan (now ENS Paris-Saclay) and director of UMR 8113 at the CNRS. He was also deputy scientific director of the CNRS’s life sciences department. He is the author of more than 120 publications in the field of antitumor pharmacology and virology. He is co-founder and scientific advisor to AB Science.

Orphan Drug Status

In April 2025, AB Science announced that the molecule AB8939 had been granted orphan drug designation by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA) for the treatment of acute myeloid leukemia (AML).

The molecule AB8939 had already obtained orphan drug designation from the US Food and Drug Administration (FDA) for AML.

About AB8939
AB8939 is a new synthetic molecule which jointly targets cancer cells, by destabilizing the microtubules essential for cell division, and cancer stem cells, by inhibiting enzymes (ALDH1A1 and ALDH2) essential for maintaining their physiological state and survival. The molecule ‘1-{4-[2-(5-ethoxymethyl-2-methylphenylamino)-oxazol-5-yl]phenyl}imidazolidin-2-one’ is the chemical name of AB8939. The intellectual property of AB8939 is 100% owned by AB Science.

(Press release, AB Science, OCT 14, 2025, View Source [SID1234656628])

On October 14, 2025 Privo Technologies, Inc. a leader in localized cancer therapies, reported the completion of enrollment in Arm 1 of the Phase 2 run-in portion of its ongoing Phase 2/3 clinical trial (CLN-004) evaluating PRV111, a nano-engineered chemotherapy patch designed to treat oral cavity cancers across distinct stages of disease (Press release, Privo Technologies, OCT 14, 2025, View Source [SID1234656645]).

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The Phase 2 run-in data are currently being analyzed for submission to the U.S. Food and Drug Administration (FDA) prior to continuing enrollment in the Phase 3 portion of the study.While detailed results remain under review, initial observations from the Phase 2 run-in are highly encouraging with respect to both safety and local tumor response with no evidence of systemic toxicity and among patients who demonstrated a favorable local response. Investigators determined that planned surgical procedures could be cancelled based on the absence of visible or measurable disease following PRV111 treatment. These findings suggest that PRV111 has the potential to offer a localized, tissue-sparing approach for early-stage oral lesions, subject to further clinical evaluation and regulatory review.

These early results support Privo’s mission to advance targeted, localized cancer therapies aimed at reducing the need for invasive procedures and improving patients’ quality of life.

Privo extends its gratitude to the investigators, clinical staff, and patients who made this milestone possible.

About the CLN-004 Phase 2/3 Clinical Trial

CLN-004 is an adaptive, open-label Phase 2/3 clinical study evaluating the safety, tolerability, and preliminary efficacy of PRV111 for the localized treatment of oral cavity lesions.

In the Phase 2 run-in (Arm 1), enrolled patients were treated with PRV111 as a stand-alone, non-surgical topical therapy for oral carcinoma in situ (CIS)/ high-grade oral dysplasia (HGD). The goal of this stage is to determine whether localized delivery of PRV111 can safely and effectively eliminate pre-cancerous and early-stage cancerous lesions while minimizing the need for surgery.

Completion of the Phase 2 enrollment marks an important milestone in Privo’s mission to develop therapies that spare patients from invasive surgery and potentially reduce recurrence risk.

"Completing enrollment in the Phase 2 portion of the CLN-004 study is a pivotal achievement for Privo and our clinical partners," said Dr. Manijeh Goldberg, Chief Executive Officer of Privo Technologies. "The initial observations are encouraging, and we look forward to sharing the full dataset with the FDA as we prepare for the Phase 3 evaluation. PRV111 reflects our vision to transform oral cancer treatment through precise, localized therapies designed to preserve function and quality of life."

Transforming Oral Cancer Treatment Through Localized Delivery

PRV111 is part of Privo’s proprietary PRV platform, a family of nano-engineered drug-delivery systems designed to deliver high concentrations of chemotherapy directly to tumor tissue while minimizing systemic exposure.

PRV111 is a topical, transmucosal patch that adheres directly to oral lesions, enabling the delivery of cisplatin nanoparticles through the mucosa to achieve targeted, localized drug penetration. Each PRV111 application is customized to the size and shape of the patient’s tumor, allowing precise coverage of the affected area and consistent drug delivery across complex anatomical surfaces.

This non-invasive, localized approach is intended to reduce systemic toxicity compared to conventional chemotherapy and to preserve surrounding healthy tissue. By potentially avoiding extensive surgical excision, PRV111 aims to help patients maintain normal speech, swallowing, and appearance—functions often affected by standard treatment options.

The CLN-004 study builds upon Privo’s earlier clinical experience with PRV111 (CLN-001), which showed promising local tumor responses with no systemic toxicity in a first-in-human setting. The results of that earlier trial were published in Nature Communications and highlighted by Forbes for their innovative approach to localized cancer drug delivery.

Looking Ahead

With Phase 2 enrollment complete, Privo Technologies is preparing to submit the CLN-004 dataset to the U.S. FDA. The data from this phase will help inform the design of the pivotal Phase 3 trial, which is planned to further evaluate PRV111 as a localized, non-surgical treatment approach.

"This milestone moves us another step toward providing patients with treatments designed to minimize the life-altering consequences of major surgery," said Dr. Manijeh Goldberg, Chief Executive Officer of Privo Technologies. "Our platform continues to demonstrate the promise of localized, patient-focused cancer care that aims to improve outcomes while preserving quality of life."

On October 14, 2025 Pilatus Biosciences Inc., a biopharmaceutical company developing novel metabolic checkpoint immunotherapies for liver and gastrointestinal cancers, reported the granting of its foundational patent in Europe and Australia, entitled "Methods for Modulating Regulatory T Cells and Inhibiting Tumor Growth."

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Pilatus has an exclusive license on the patent from the Ludwig Institute for Cancer Research Ltd and the University of Lausanne, Switzerland, based on work by Dr. Ping-Chih Ho’s laboratory at the Ludwig Lausanne Branch. The patent covers Pilatus’ first-in-class antibody program targeting CD36, a key metabolic checkpoint expressed on regulatory T cells (Tregs) that play a critical role in suppressing anti-tumor immune responses.

This intellectual property milestone provides broad protection for the company’s pioneering work in Treg modulation through metabolic reprogramming, representing a new therapeutic strategy in immuno-oncology.

Tregs are essential for maintaining immune balance but can also enable tumor immune evasion by suppressing effector T cell activity within the tumor microenvironment. CD36, a fatty acid transporter highly expressed on Tregs acts as a metabolic gatekeeper that supports their survival and suppressive function under hypoxic and nutrient-deprived tumor conditions.

Pilatus’ antibody program is designed to disrupt CD36-mediated lipid uptake and signaling in Tregs, thereby reawakening the immune system’s ability to attack tumors without triggering systemic autoimmunity. This approach builds upon discoveries made in the laboratory of Dr. Ping-Chih Ho, Full Member at LICR Lausanne, and forms the scientific foundation for Pilatus’ proprietary pipeline of metabolic checkpoint inhibitors.

Nobel Prize Underscores the Field’s Importance

This announcement comes at a particularly timely moment: the 2025 Nobel Prize in Physiology or Medicine was awarded for groundbreaking discoveries in Regulatory T cell biology, underscoring the growing recognition of immune regulation as a cornerstone of modern medicine.

In fact, one of this year’s Nobel Laureates, Prof. Shimon Sakaguchi, the discoverer of Tregs, was a recent keynote speaker at the Ho Lab anniversary symposium in Lausanne, where he presented on "Targeting Tregs for Cancer Immunotherapy."

"The recognition of Treg biology by the Nobel Committee highlights the importance of immune regulation in health and disease," said Dr. Ping-Chih Ho, Co-Inventor and Full Professor and member at the Ludwig Institute for Cancer Research at University of Lausanne. "Our CD36 discoveries bridge metabolic control and immune suppression, paving the way for transformative treatments in oncology and beyond."

"This patent issuance solidifies Pilatus’ leadership position in metabolic checkpoint immunotherapy," said Raven Lin, Ph.D., Co-Founder and CEO, Pilatus Biosciences. "By targeting the metabolic dependencies of regulatory T cells, we are developing a new class of therapies designed to unlock potent and durable anti-tumor immunity, while demonstrating synergistic potential with PD-1 blockade to benefit patients with difficult to treat cancers."

(Press release, Pilatus Biosciences, OCT 14, 2025, View Source [SID1234656660])

On October 14, 2025 Aanastra, Inc., a biotechnology company leveraging its proprietary targeted peptide delivery technology (PEP-NP) to advance in vivo targeting and reprogramming of cells with RNA therapeutics, reported multiple presentations showcasing preclinical data in its lead programs for anti-CD19 in vivo CAR-T (AAN-14x) and for in vivo P53 rescue in P53 mutant tumors (AAN-53x), at upcoming conferences.

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"Among the broad applications of the PEP-NP technology, we are particularly excited by the preclinical data in our in vivo CAR-T program as well as our ability to target wide-ranging P53 driver mutations in our P53 program for which no therapy exists today ," said Neil Desai Ph.D., Founder and CEO at Aanastra. "These preclinical data highlight the remarkable versatility of the PEP-NP targeted peptide RNA delivery technology, from in vivo reprogramming of immune cells to cause B cell depletion, to in vivo restoration of mutant tumor suppressor function directly in tumors, all with a mRNA approach. Importantly the PEP-NP system can completely bypass the liver, without immunogenicity or safety issues upon repeat administration, uses no lipid, viral or protein components, and is fully owned by Aanastra."

Featured Presentations and Conferences:

Advancing Cell and Gene Therapies for Cancer Conference, Philadelphia, PA, Oct 15-16, 2025

Poster presentation title: In Vivo CAR-T generation of anti-CD19 CAR-T-cells using CAR mRNA delivered with the PEP-NP peptide-based (non-lipid, non-viral) delivery system targeted to CD3/CD5+ T-cells
Presenter: Gilles Divita, Ph.D
Date and time: Oct 15, 2025, 5:00 p.m. – 6.30 p.m. ET

Poster presentation title: P53 mutant tumor regression across P53 mutations with AAN-53x, a P53 mRNA delivered with the PEP-NP tumor-targeted peptide (non-lipid) delivery system
Presenter: Gilles Divita, Ph.D
Date and time: Oct 15, 2025, 5:00 p.m. – 6.30 p.m. ET

mRNA Vaccines and Therapeutics Summit, Barcelona, Spain, Oct 23-24, 2025

Oral Presentation Title: A novel peptide-based nanoparticle platform for systemic targeted delivery of therapeutic mRNA : invivo CAR-T cell engineering and Tumor selectivity
Presenter: Gilles Divita, Ph.D.
Date and Time: Oct 23, 2025, 4.40 pm – 5.20pm. CET

About P53
Known as the "Guardian of the Genome," P53 is a crucial tumor suppressor that preserves DNA integrity by regulating DNA repair and cell division. Loss of functional p53 causes DNA damage to accumulate, driving uncontrolled cell growth and tumor development. It is mutated or inactivated in over 50% of cancers, affecting 400,000 new U.S. patients annually and over 10 million globally. Mutations span hundreds of variants, making broad therapies difficult. These mutations are markers of poor prognosis and key cancer drivers. No existing treatments restore p53 function across this spectrum, leaving a significant unmet need. Aanastra’s RNA-based approach using its targeted PEP-NP delivery offers a promising strategy to broadly restore p53 function, potentially transforming outcomes for aggressive, treatment-resistant cancers.

About In vivo CAR-T
In vivo CAR-T therapy engineers a patient’s T cells inside the body to target and destroy cancer cells, avoiding the costly, complex ex vivo CAR-T process that today is available to less than 20% of eligible patients in the US. Current lentiviral methods for in vivo CAR-T allow only single treatments due to immune responses against viral components. Lipid nanoparticle (LNP)-based mRNA delivery enables transient CAR expression that could be repeated but is limited by liver toxicity and immune related side effects like inflammation. These safety and dosing challenges can restrict sustained treatment effects. Aanastra’s PEP-NP system efficiently targets CAR RNA to T cells for safe, repeat dosing without these toxicities, potentially overcoming current limitations and expanding access for hematological cancers and autoimmune diseases.

About PEP-NP
Aanastra’s PEP-NP technology is based on short, amphipathic peptides that form stable nanoparticles with RNA for efficient delivery. These peptides are designed with distinct hydrophobic and hydrophilic regions, adopting α-helical conformations facilitating membrane interaction and cellular entry. Importantly, PEP-NP peptides can include specific receptor-targeting domains, enabling precise cell and tissue targeting without using antibodies or other large proteins. This antibody-free targeting allows rapid design flexibility and avoids immunogenicity associated with protein-based ligands. PEP-NP nanoparticles can enter cells though a non-endosomal mechanism, enhancing RNA release into the cytoplasm for effective therapeutic action. Together, these features make PEP-NP a highly versatile, scalable, and safe platform for delivering diverse RNA payloads for treating cancer, autoimmune, and genetic diseases.

(Press release, AANASTRA, OCT 14, 2025, View Source [SID1234662316])

On October 14, 2025 Actithera, a biotechnology company pioneering next-generation radiopharmaceutical therapies, reported an exclusive license agreement with Yeda, the commercial arm of the Weizmann Institute of Science, for two patent families covering breakthrough covalent chemistry technologies (Press release, Actithera, OCT 14, 2025, View Source [SID1234656629]). Actithera will apply these innovations to advance its proprietary platform for radiopharmaceutical drug discovery and development.

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The licensed technologies were developed in the laboratory of Professor Nir London, an internationally recognized leader in covalent drug design. These approaches enable a highly differentiated way to introduce radioactivity selectively and durably onto tumor-specific proteins, offering the potential to redefine how radiopharmaceuticals are conceived, optimized, and deployed in the clinic.

"This agreement represents an important step forward for Actithera," said Andreas Goutopoulos, PhD, Founder and Chief Executive Officer of Actithera. "By adding Professor London’s pioneering chemistry to our toolkit of proprietary covalent and non-covalent approaches, we are uniquely positioned to unlock new opportunities in cancer treatment. This innovation from the Weizmann Institute enables us to introduce radioactivity directly into tumor cells by irreversibly and tracelessly radiolabeling tumor-specific proteins while keeping them in their native state. We believe that this combination of irreversibility and tracelessness can translate into prolonged retention of radiation within tumors and ultimately improve therapeutic outcomes."

Elik Chapnik, PhD, Chief Executive Officer of Yeda, commented: "We are pleased to partner with Actithera in bringing Professor London’s groundbreaking covalent chemistry technologies into the radiopharmaceutical field. Actithera’s vision and expertise make them an ideal partner to translate these scientific advances into impactful cancer therapies that can benefit patients worldwide."

The technologies will be integrated into Actithera’s existing discovery platform as the company advances its lead FAP-targeting radioligand candidate toward clinical development in multiple indications. This strategic expansion is supported by Actithera’s recent oversubscribed $75.5 million Series A financing completed in July 2025, which is enabling the continued development of the Company’s proprietary RLT discovery platform and preclinical pipeline.

Professor Nir London, Associate Professor, Department of Chemical and Structural Biology, Weizmann Institute of Science, added: "My lab has long been dedicated to expanding the possibilities of covalent drug design. Seeing our work translated into the radiopharmaceutical space through Actithera’s innovative platform is particularly exciting given the field’s potential to deliver targeted radiation directly to tumors while sparing healthy tissue. I look forward to supporting their progress as they advance these technologies toward clinical validation."
The covalent chemistry technologies from the Weizmann Institute represent one of several cutting-edge approaches integrated into Actithera’s discovery engine, expanding the Company’s toolkit for building a pipeline of precision therapies addressing areas of high unmet need in oncology.