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 Clarity Pharmaceuticals (ASX: CU6) ("Clarity" or "Company"), a clinical-stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for patients with cancer, reported that the Co-PSMA (NCT06907641)1 Investigator-Initiated Trial (IIT), led by Prof Louise Emmett at St Vincent’s Hospital Sydney, has achieved its primary endpoint with a statistically significant higher number of prostate-specific membrane antigen (PSMA)-positive prostate cancer lesions detected using 64Cu-SAR-bisPSMA compared to standard-of-care (SOC) 68Ga-PSMA-11 positron emission tomography/computed tomography (PET/CT) in patients with low prostate-specific antigen (PSA) levels.

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Co-PSMA’s official study title is "Comparative performance of 64Copper [64Cu]-SAR-bisPSMA vs. 68Ga-PSMA-11 PET CT for the detection of prostate cancer recurrence in the setting of biochemical failure following radical prostatectomy". This Phase II IIT is evaluating the performance of Clarity’s diagnostic product, 64Cu-SAR-bisPSMA, in comparison to SOC 68Ga-PSMA-11 in 50 patients with low PSA who are candidates for curative salvage therapy. Eligible patients were required to have had radical prostatectomy with no salvage therapy and a PSA level between 0.2 and 0.75 ng/mL.

The Co-PSMA trial has successfully met its primary endpoint, demonstrating that 64Cu-SAR-bisPSMA PET/CT detects significantly more lesions per patient than the SOC, 68Ga-PSMA-11 PET/CT. This result supports the hypothesis that 64Cu-SAR-bisPSMA can improve early detection of recurrence and staging of prostate cancer in patients with low PSA who are candidates for curative salvage therapy. Full analysis of all data generated in the Co-PSMA trial is underway, and results of this study will be presented at an upcoming international conference.

The Co-PSMA topline results corroborate findings from the COBRA trial2, which investigated the diagnostic performance of 64Cu-SAR-bisPSMA in patients with biochemical recurrence (BCR) of prostate cancer who had a negative or equivocal SOC scan at study entry. In the COBRA study, a subset of participants underwent follow-up SOC PSMA PET imaging. Only 60% of these participants had a positive SOC PSMA PET, while 70% of participants had a positive 64Cu-SAR-bisPSMA PET on same-day imaging and 90% on next-day imaging. The number of lesions across all participants (average sum of lesions across all readers) identified by 64Cu-SAR-bisPSMA was also higher (26.3 lesions on same-day imaging, 52.6 on next-day imaging) than that detected by SOC PET agents (20 lesions). The COBRA trial results showed that 64Cu-SAR-bisPSMA was able to identify lesions more than 6 months earlier than SOC PSMA PET agents, with 6 months being the last follow-up visit for that trial.

The Co-PSMA trial further builds on the growing body of evidence of the enhanced diagnostic performance of 64Cu-SAR-bisPSMA compared to SOC PSMA PET agents, which are known to have low sensitivity, especially in patients with low PSA levels3,4. Improvements in sensitivity, as with all diagnostic agents, play a pivotal role in guiding more informed treatment decisions, enabling earlier and more accurate detection of prostate cancer recurrence and ultimately improving patient outcomes.

Dr Alan Taylor, Executive Chairperson of Clarity Pharmaceuticals, commented: "Achieving the primary endpoint in the Co-PSMA trial, which was a head-to-head trial against a SOC competing product, is yet another important step in the development of 64Cu-SAR-bisPSMA as we look to further validate this agent as best-in-class through two registrational trials with two Fast Track Designations (FTDs) under our belt for diagnostic applications and a strong focus on commercialisation.

"We look forward to Prof Emmett releasing the full Co-PSMA trial data at world-leading congresses as we continue to adhere to the highest standards of clinical research in our aspirations to bring a best-in-class diagnostic option to men with prostate cancer with clear evidence of superiority. We have already seen in the first-in-human PROPELLER trial an improved diagnostic performance of 64Cu-SAR-bisPSMA compared to 68Ga-PSMA-11 on same-day imaging, including a higher number of lesions identified and 2-3 times higher tumour uptake and tumour-to-background ratio5. We then showed in the COBRA trial that approximately twice the amount of lesions was identified on 64Cu-SAR-bisPSMA PET on next-day vs. same-day imaging, and vs. SOC PSMA PET2. 64Cu-SAR-bisPSMA also allowed for the identification of lesions in the 2-mm range, something that SOC PSMA PET agents often fail to achieve2, 6-9. This improvement was driven by a substantially increased tumour-to-background ratio and lesion uptake over time with next-day imaging2.

"The current market for PSMA PET imaging in the US alone is around US$2 billion per year, and this is expected to further grow to over US$3 billion by 2029. However, this entire market is currently served by products that have low sensitivity, while the development pipeline of new products, excluding 64Cu-SAR-bisPSMA, offers no differentiation from the existing agents, with some new entrants commercialising the unpatented 68Ga-PSMA-11 agent, which has been capitalised on by three separate groups already. We believe that with the clinical and logistical benefits offered by 64Cu-SAR-bisPSMA we could not only become the new SOC in PSMA PET but grow the market opportunity further by diagnosing prostate cancer earlier, while lesions are still very small.

"We look forward to continuing to work with Prof Emmett, a world-renowned thought leader in the radiopharmaceutical space, in our home city of Sydney on progressing clinical development of the 64Cu-SAR-bisPSMA product, including our CLARIFY and AMPLIFY Phase III trials. This partnership will ensure we continue to generate the highest quality of data supporting our New Drug Applications (NDAs) to the US Food and Drug Administration (FDA) as the next step towards our mutual goal of improving treatment outcomes for men with prostate cancer."

(Press release, Clarity Pharmaceuticals, OCT 14, 2025, View Source [SID1234656623])

On October 13, 2025 BriaCell Therapeutics Corp. (Nasdaq: BCTX, BCTXW, BCTXZ) (TSX: BCT) ("BriaCell" or the "Company"), a clinical-stage biotechnology company that develops novel immunotherapies to transform cancer care, reported it will be presenting positive clinical biomarker data of its ongoing pivotal Phase 3 study of Bria-IMT plus an immune check point inhibitor (CPI) in metastatic breast cancer at its poster presentation at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025 Annual Meeting taking place October 17 – 21 in Berlin, Germany.

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The details of the poster presentation is listed below.

#3928: Feasibility and Biomarker Validation of an International Randomized Phase 3 Trial of Bria-IMT Cell Therapy in Late Stage MBC (BRIA-ABC)
Presentation Number: 570P
Speaker: Dr. Giuseppe Del Priore
Date and Time: Oct 20, 2025, at 12:00 – 1:00 pm CET
Presentation venue: Messe Berlin, Messedamm 22, 14055 Berlin, Germany, Hall 25

Abstract Summary
In BriaCell’s pivotal Phase 3 study of Bria-IMT plus an immune check point inhibitor (CPI) in metastatic breast cancer, patients are randomized 1:1:1 to Bria-IMT + CPI, Physician’s Choice, or Bria-IMT monotherapy. As of the time of the abstract submission, data in 68 evaluable patients, with a median of 6 prior lines of treatment (2–13), was available. Additional data will be presented at the ESMO (Free ESMO Whitepaper) conference.

Clinical efficacy data: Treatment arm agnostic biomarker positive subgroups showed significant improvement in progression free survival in patients who developed an immune response to Bria-IMT (as measured by delayed type hypersensitivity) (p=0.0002).

Tolerability profile: Bria-IMT was well tolerated with no treatment-related discontinuations due to adverse events (AEs). Most common AEs include fatigue 22.8%, anemia 22.8%, and nausea 21.5%.

About BriaCell’s Pivotal Phase 3 Clinical Study of Bria-IMT Combination Regimen in MBC patients

BriaCell’s pivotal Phase 3 study of Bria-IMT plus an immune check point inhibitor (CPI) in metastatic breast cancer is ongoing.

Interim data will be analyzed once 144 patient events (deaths) occur, comparing the overall survival (OS) in patients treated with the Bria-IMT combination regimen versus those treated with physician’s choice as the primary endpoint. Positive results of the pivotal Phase 3 study could result in full approval and marketing authorization for Bria-IMT in MBC patients. BriaCell reported positive Phase 2 survival data in a similar MBC patient population treated with the same Bria-IMT combination regimen . The Bria-IMT combination regimen has received FDA Fast Track designation.

For additional information on BriaCell’s pivotal Phase 3 study of Bria-IMT and an immune check point inhibitor in metastatic breast cancer, please visit ClinicalTrials.gov NCT06072612 .

Following the presentations, copies of the presentations will be posted on View Source

(Press release, BriaCell Therapeutics, OCT 13, 2025, View Source [SID1234656935])

On October 13, 2025 Repare Therapeutics Inc. ("Repare" or the "Company") (Nasdaq: RPTX), a clinical-stage precision oncology company, reported it will share initial topline safety, tolerability and early efficacy data from the Phase 1 LIONS trial in a poster presentation at the 37th 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), being held October 22-26, 2025 in Boston, MA.

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The LIONS clinical trial (NCT06232408) is a first-in-human, multicenter, open-label Phase 1 study to investigate safety, pharmacokinetics, pharmacodynamics and the preliminary efficacy of RP-1664, a potential first-in-class, highly selective, oral PLK4 inhibitor, for the monotherapy treatment of adult and adolescent patients with TRIM37-high solid tumors.

Poster Presentation Details:

Title: Preliminary safety and antitumor activity of RP-1664, a first-in-class PLK4 inhibitor, as monotherapy in advanced solid tumors with and without TRIM37 amplification
Presenter: Benjamin Herzberg, MD, Columbia University
Abstract Number: LB-C002
Session: Poster Session C
Session Date and Time: Saturday, October 25 | 12:30 p.m. – 4:00 p.m. ET
Session Location: Level 2, Exhibit Hall D

A copy of the poster presentation will be available on the Scientific Resources page of the Repare Therapeutics website at the start of the poster presentation session.

About RP-1664

RP-1664 is a potential first-in-class, highly selective, oral PLK4 inhibitor designed to harness the synthetic lethal relationship with TRIM37 amplification or overexpression in solid tumors. Tumors rely on PLK4 for centriole biogenesis in S-phase of the cell cycle when TRIM37, an E3 ligase that reduces pericentriolar material, is high. Preclinical studies demonstrate that RP-1664 selectively inhibits PLK4 and drives potent synthetic lethality in TRIM37-high tumor models, both in vitro and in vivo. Elevated TRIM37 is a feature found across a range of solid tumors and in approximately 80% of all high-grade neuroblastomas. RP-1664 is the only selective PLK4 inhibitor known to be in the clinic.

(Press release, Repare Therapeutics, OCT 13, 2025, View Source [SID1234656900])

On October 13, 2025 Immutep Limited (ASX: IMM; NASDAQ: IMMP) ("Immutep" or "the Company"), a late-stage immunotherapy company targeting cancer and autoimmune diseases, reported that positive and straightforward feedback has been received from the US Food and Drug Administration ("FDA") regarding the successful completion of Project Optimus requirements and agreement on 30mg as the optimal biological dose for eftilagimod alfa (efti).

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Immutep’s Chief Development Officer, Christian Mueller, said, "We are very thankful for the FDA’s positive feedback and productive discussions over the past few years. The alignment on efti’s optimal biologic dose has strategic relevance to our efti oncology programs and is a major de-risking event and building block towards future BLA filings. We are excited to successfully conclude this chapter of efti’s clinical development and are intently focused on bringing this novel immunotherapy to market to help address the needs of cancer patients worldwide."

The agreement with the FDA on efti’s optimal biological dosing carries strategic importance in the ongoing and future clinical development of this first-in-class immunotherapy, including the global TACTI-004 (KEYNOTE-F91) Phase III trial evaluating efti in combination with MSD’s (Merck & Co., Inc., Rahway, NJ, USA) anti-PD-1 therapy KEYTRUDA (pembrolizumab), and chemotherapy as first-line treatment for advanced or metastatic non-small cell lung cancer (1L NSCLC), regardless of PD-L1 expression. With the conclusion of Project Optimus, this registrational study is now in process of opening sites in the United States.

About Eftilagimod Alfa (Efti)

Efti is a novel immunotherapy that directly activates antigen-presenting cells or APCs (e.g. dendritic cells, monocytes) via the MHC Class II pathway to fight cancer. As an MHC Class II agonist, its activation of APCs engages the adaptive and innate immune system to initiate a broad anti-cancer immune response. This includes priming and activating cytotoxic T cells as well as generating important co-stimulatory signals & cytokines that further boost the immune system’s ability to combat cancer.

Efti is under evaluation for a variety of solid tumours including non-small cell lung cancer (NSCLC) in a pivotal Phase III trial called TACTI-004 (KEYNOTE-F91), as well as head and neck squamous cell carcinoma (HNSCC), soft tissue sarcoma, and breast cancer. Its favourable safety profile enables various combinations like with anti-PD-[L]1 immunotherapy, radiotherapy, and/or chemotherapy. Efti has received Fast Track designation in first line HNSCC and in first line NSCLC from the United States Food and Drug Administration (FDA).

(Press release, Immutep, OCT 13, 2025, View Source [SID1234656640])