On December 10, 2025 NEC Bio Therapeutics reported results from the Phase I basket clinical trial of an orally administered cancer vaccine, NECVAX-NEO1, used in combination with checkpoint inhibitors (CPIs) for treating patients with solid tumors. The findings are being presented in a poster at the ESMO (Free ESMO Whitepaper) Immuno-Oncology Congress in London, United Kingdom, from December 10 to 12, 2025.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

NECVAX-NEO1 is a personalized bacteria-based oral DNA therapeutic vaccine, developed using AI prediction of the most immunogenic patient-specific neoepitopes. This vaccine is designed to activate a patient’s immune system, prompting a T-cell response that can precisely target and eliminate tumor cells based on an individual’s unique neoantigens.

In the phase I study, 6 patients with melanoma, renal cell cancer, or head and neck cancer, who have been on CPI treatment for at least three months, were treated with NECVAX-NEO1. The safety run-in phase showed no treatment-related toxicities, allowing a dose increase. Clinically, 83% of the patients achieved stable disease at the end of 24 weeks of treatment, which was followed by a 12 week follow-up period. In all patients immunogenic neoepitopes used in the vaccine, were detected, as demonstrated by ELISPOT analysis.

Regarding the results of the clinical study, Dr. Heinz Lubenau, CEO of NEC Bio Therapeutics, commented, "Our clinical Phase I data are demonstrating promising immune responses in treated cancer patients. The translational biomarker data are in line with the clinical data so that we are presenting a consistent data set. The progress is encouraging as we advance two additional clinical studies in 3 European countries in both early- and later-stage cancer settings. We look forward to further evaluating NECVAX-NEO1 as a potential treatment option for cancer patients with hard-to-treat tumors."

Motoo Nishihara, Corporate Executive Vice President and CTO of NEC Corporation, further commented, "We are proud to present the progress of the NECVAX-NEO1 trial, which demonstrates safety as well as signs of immunogenicity and early efficacy. NECVAX-NEO1 is the first oral cancer vaccine asset to be clinically developed by NEC. The results of this trial are a testament to our proprietary AI predictive software that supports immunological and clinical readouts. This development aligns closely with NEC’s broader mission to deliver global healthcare solutions using state-of-the-art technologies developed in-house."

Details of the poster are below:

Poster title: NECVAX-NEO1, a bacteria-based personalized neoepitope vaccine combined with PD-1/PD-L1 checkpoint inhibition in a phase I, open-label, multicenter study: safety, immunogenicity and early efficacy signals. NCT05354323

Authors: D. Vaitiekus, E. Juozaityte, L. Puzauskienė, S. Tulyte-Kirzova, L. Gatijatullin, M. Platten, I. Poschke, I.Hülsmeyer, A. Kuhn, A. Aranguren, H. Lubenau, R. Stratford, T. Clancy, H. Fontenelle, B. Simovski, Y. Yamashita, C. Chaput, A. Meiser, V. Urbonas

Poster Number: 258P

(Press release, NEC, DEC 10, 2025, View Source [SID1234661347])

On December 10, 2025 Moleculin Biotech, Inc., (Nasdaq: MBRX) ("Moleculin" or the "Company"), a late-stage pharmaceutical company with a broad portfolio of drug candidates targeting hard-to-treat tumors and viruses, reported it has entered into agreements with certain holders of its existing warrants for the immediate exercise of certain outstanding warrants to purchase up to an aggregate of 727,969 shares of common stock of the Company originally issued in February 2025 at an exercise price of $6.63 per share and 316,360 shares of common stock of the Company originally issued in August 2025 at an exercise price of $6.3219 per share. The shares of common stock issuable upon exercise of the outstanding warrants are registered pursuant to effective registration statements on Form S-1 (File No. 333-286276) and Form S-3 (File No. 333-290418). The aggregate gross proceeds from the exercise of the existing warrants is expected to total approximately $6.5 million, before deducting financial advisory fees.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Roth Capital Partners and Maxim Group LLC are acting as the Company’s financial advisors for this transaction.

In consideration for the immediate exercise of the warrants for cash, the Company will issue new unregistered warrants to purchase shares of common stock. The new warrants will be exercisable for an aggregate of up to 2,610,823 shares of common stock, at an exercise price of $6.63 per share and will be exercisable upon shareholder approval and for a term of five years from the date of shareholder approval.

The transaction is expected to close on or about December 11, 2025, subject to satisfaction of customary closing conditions. The Company intends to use the net proceeds from the offering for working capital and general corporate purposes.

The new warrants described above were offered in a private placement pursuant to an applicable exemption from the registration requirements of the Securities Act of 1933, as amended (the "1933 Act") and, along with the shares of common stock issuable upon their exercise, have not been registered under the 1933 Act, and may not be offered or sold in the United States absent registration with the Securities and Exchange Commission ("SEC") or an applicable exemption from such registration requirements. The Company has agreed to file a registration statement with the SEC covering the resale of the shares of common stock issuable upon exercise of the new warrants.

This press release shall not constitute an offer to sell or a solicitation of an offer to buy nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to the registration or qualification under the securities laws of any such state or jurisdiction.

(Press release, Moleculin, DEC 10, 2025, View Source [SID1234661346])

On December 10, 2025 Kazia Therapeutics Limited ("Kazia" or the "Company") reported new data from two presentations at the 2025 San Antonio Breast Cancer Symposium (SABCS) providing compelling mechanistic and early clinical evidence supporting the activity of paxalisib, the Company’s brain-penetrant dual PI3K/mTOR inhibitor, across both HER2-positive metastatic breast cancer and triple-negative breast cancer (TNBC).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

The results, originating from advanced liquid biopsy profiling, immune phenotyping, and early clinical readouts, highlight paxalisib’s potential to disrupt highly aggressive circulating tumor cell (CTC) clusters, reverse epigenetically-driven resistance pathways, and reinvigorate exhausted T- and B-cell populations, thereby enhancing responsiveness to immunotherapy.

Paxalisib Disrupted Drivers of Metastasis: Vim+/Snail+/NRF2+ CTC Clusters in HER2+ Disease Ex Vivo

In HER2-positive metastatic breast cancer, a population in which nearly all patients eventually relapse despite HER2-directed therapies, investigators observed that even patients who were radiographically responding continued to harbor substantial burdens of therapy-resistant CTC clusters—a key driver of metastatic spread.

Poster Presentation: PS2-10-02 : Liquid Biopsy Tracking of PI3K–mTOR Residual Disease Signatures in Metastatic HER2+ Breast Cancer

Key findings:

•
Paxalisib reduced single CTCs by 42% and CTC clusters by 78% ex vivo, including large clusters (≥5 cells), which are strongly associated with metastatic progression.

•
CTC clusters expressed a highly aggressive mesenchymal phenotype marked by Vimentin⁺/Snail⁺/NRF2⁺, which paxalisib significantly disrupted.

•
Patients with poor clinical response demonstrated impaired cytotoxic function (reduced Granzyme B and Perforin) and expanded exhausted T-cell populations, while paxalisib treatment activated cytotoxic, interferon, chemokine, and inflammatory pathways in samples from these patients, supporting a more immunologically "hot" tumor environment.

"These findings reveal an important biological gap left by existing HER2+ directed therapies," said Prof. Sudha Rao, QIMR Berghofer. "CTC clusters persist even in responding patients, and paxalisib is the first agent we have observed that can directly dismantle this highly aggressive and clinically relevant compartment."

TNBC Phase 1b Trial: Early Clinical Data from First Patient Show Robust Suppression of CTC Clusters and Reversal of T-Cell Exhaustion

Early longitudinal biomarker data from the first patient treated in the PaxPlus-ABC Phase 1b study (paxalisib + pembrolizumab + chemotherapy) indicate that paxalisib has had measurable biological activity after only a single cycle.

Poster Presentation: PS5-08-04: A phase 1b, multi-centre, open-label, randomized study to evaluate the safety, tolerability, and clinical activity of combining paxalisib with olaparib or pembrolizumab/chemotherapy in patients with advanced breast cancer

Highlights from first patient include:

•
Marked reduction in CTC clusters following the first cycle of paxalisib.

•
Epigenetic reprogramming of CTCs toward less aggressive phenotypes, confirmed through digital pathology and Nanostring profiling.

•
Significant reduction of exhausted CD8 T cells, with revitalization of cytotoxic and antigen-presentation pathways.

•
CT imaging has demonstrated overall primary tumor volume reduction from baseline 14mm x 11mm (154mm2) to 12mm x 3 mm (36mm2)

•
Notably, a temporary interruption of paxalisib (necessitated by a chemotherapy-related adverse event) resulted in a rapid resurgence of CTC clusters. Resumption of paxalisib after a short 3-week pause restored suppression of CTC clusters, indicating that pembrolizumab alone could not control these metastatic drivers and highlighting paxalisib’s unique mechanistic role.

Pembrolizumab Alone May Not Control CTC Burden; A Mechanistic Opportunity for Paxalisib

Across HER2+ and TNBC ex-vivo datasets, a consistent theme emerges:

•
Pembrolizumab monotherapy does not meaningfully reduce CTC burden, and in TNBC, CTC clusters increased when paxalisib was withheld.

•
Paxalisib directly targets mesenchymal, metastatic, and epigenetically resistant CTC clusters.

•
It also reinvigorates immune effector cells, potentially overcoming the cytotoxic dysfunction and exhaustion that limit checkpoint inhibitor efficacy.

This mechanistic direct suppression of metastasis-initiating cells plus restoration of immune function positions paxalisib as a potentially transformative immunotherapy-enhancing agent.

Expanding Opportunity Across Breast Cancer: HER2+, TNBC, BRCA-Mutated, and Beyond

Because mesenchymal CTC clusters and T-cell exhaustion are shared resistance mechanisms across multiple breast cancer subtypes, paxalisib’s effects are highly relevant beyond TNBC.

Emerging data suggest:

•
In HER2+ patients, despite targeted therapy, residual disease persists in the form of aggressive CTC clusters—a new therapeutic window for paxalisib.

•
In TNBC, paxalisib’s epigenetic and immunologic effects provide a strong rationale for combination with pembrolizumab, PARP inhibitors, and chemotherapy.

•
In BRCA-mutated and homologous recombination–deficient tumors, PI3K/mTOR inhibition may synergize with synthetic lethal strategies such as olaparib.

"Kazia’s recent clinical and translational findings point to a unifying biology across breast cancer subtypes," said Dr. John Friend, CEO of Kazia Therapeutics. "Paxalisib appears capable of disrupting metastatic machinery that is not adequately addressed by current HER2-targeted therapies, checkpoint inhibitors, or chemotherapies. We believe these discoveries meaningfully expand the potential utility of paxalisib beyond our current development programs."

For investor and media, please contact Alex Star, Managing Director LifeSci Advisors LLC, [email protected], +1-201-786-8795.

(Press release, Kazia Therapeutics, DEC 10, 2025, View Source [SID1234661345])

On December 10, 2025 GSK plc (LSE/NYSE: GSK) reported that its B7-H3-targeted antibody-drug conjugate GSK’227, now referred to by its International Nonproprietary Name, risvutatug rezetecan, has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of small-cell lung cancer (SCLC). The ODD was supported by preliminary clinical data showing durable responses in patients with extensive stage SCLC (ES-SCLC) who were treated with risvutatug rezetecan in the phase I ARTEMIS-001 clinical trial.1

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In the US, SCLC constitutes about 13% of all lung cancers. In 2025, an estimated 29,500 people in the US will be diagnosed with SCLC.2 Of patients with SCLC, 70% have extensive-stage disease, meaning the cancer has spread throughout one or both lungs and/or to other parts of the body.3 ES-SCLC is an aggressive and difficult-to-treat cancer with limited treatment options. The 5-year survival rate is approximately 3%.3 Most patients with ES-SCLC relapse after initial treatment and the median overall survival with standard-of-care treatments for relapsed ES-SCLC is approximately 8 months.4

This designation follows the recent announcement that risvutatug rezetecan was granted ODD from the European Medicines Agency (EMA) for the treatment of pulmonary neuroendocrine carcinoma, a category of cancer that includes SCLC. It is the fifth regulatory designation for risvutatug rezetecan, exemplifying the potential of this B7-H3-targeted ADC, which is being developed in a range of solid tumours, including lung, prostate and colorectal cancers. Previously, risvutatug rezetecan was granted Priority Medicines (PRIME) Designation by the EMA for relapsed or refractory ES-SCLC and Breakthrough Therapy Designations for relapsed or refractory ES-SCLC and relapsed or refractory osteosarcoma granted by the US FDA.5,6,7

About risvutatug rezetecan
Risvutatug rezetecan (GSK5764227) is a novel investigational B7-H3-targeted antibody-drug conjugate composed of a fully human anti-B7-H3 monoclonal antibody covalently linked to a topoisomerase inhibitor payload. GSK acquired exclusive worldwide rights (excluding China’s mainland, Hong Kong, Macau, and Taiwan) from Hansoh Pharma to progress clinical development and commercialisation of risvutatug rezetecan. GSK’s global phase III trial (NCT07099898) for risvutatug rezetecan in relapsed ES-SCLC began in August 2025.

(Press release, GlaxoSmithKline, DEC 10, 2025, View Source [SID1234661344])

On December 10, 2025 GENFIT (Euronext: GNFT), a biopharmaceutical company dedicated to improving the lives of patients with rare and life-threatening liver diseases, reported encouraging preliminary Phase 1b data from its CCA clinical trial evaluating GNS561 in combination.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Clinical trial context and objective
CCA is a rare and aggressive cancer of the bile ducts, often diagnosed at an advanced stage. The unmet medical need is characterized by strong limitations in current treatments and poor prognosis. GNS561 is an investigational small molecule that targets PPT1, leading to autophagy inhibition and lysosomal dysfunction, which disrupt cancer cell survival mechanisms. By blocking autophagy, GNS561 aims to promote cancer cell death and may enhance sensitivity to other treatments. Combining GNS561 with a MEKi aims to unlock synergistic potential by simultaneously targeting autophagy and MAPK signaling pathways. In the on-going Phase 1b study, patients with advanced KRAS mutated CCA who have previously failed one or two lines of prior standard of care therapies are enrolled to evaluate the safety and tolerability of GNS561 when given in combination with trametinib, a MEKi, and to identify the recommended doses of the combination to be administered in Phase 2.

Preliminary results
The analysis evaluated 9 patients with measurable disease at baseline, 4 of them reaching tumor assessment at week 6. At this point, the combination therapy demonstrated:

Disease stabilization observed in all 4 evaluated patients, who had all shown disease progression during previous treatment;
Tumor shrinkage in a subgroup of patients with the best response showing a 20% reduction approaching the partial response (PR) threshold.
Achieving disease control and tumor reduction in such heavily pretreated patient population with advanced CCA is a significant signal of antitumor activity.

Clinical Impact

The results to date show a potential to address a critical unmet medical need in oncology. Patients with advanced solid tumors who have progressed on multiple prior therapies have limited treatment options and poor prognoses. The ability of the investigational drug GNS561 associated with a MEKi to achieve disease control in this challenging patient population would represent a significant advance. The consistent pattern of disease stabilization observed across all evaluated patients, combined with objective tumor shrinkage in a subgroup of heavily pretreated patients, suggests the combination has the potential to provide meaningful clinical benefit. Optimization of dosing and patient selection could lead to further improvement in response rates.

Dr. Mark Yarchoan, Associate Professor of Oncology at John Hopkins Medicine (Baltimore, MD, USA), principal investigator of the program, commented: "Advanced KRAS-mutated cholangiocarcinoma remains a formidable clinical challenge, and the emerging activity seen in this initial study is encouraging. Because MEK inhibition alone has historically shown limited efficacy in this setting, the early signs of benefit with dual targeting of autophagy and MAPK signaling provide meaningful rationale for continued evaluation of this combination strategy."

Pascal Prigent, Chief Executive Officer of GENFIT, added: "These early results suggest a potential breakthrough for patients with limited options, and we are committed to advancing this program rapidly to individuals impacted by cholangiocarcinoma. We will also explore GNS561 potential in combination with other agents and in other tumors where autophagy inhibition plays a central role."

Next development steps

Phase 1b dose escalation will continue as planned to confirm the activity signal, with new data for the next patient cohorts expected in 1Q26. These results will be used to establish the recommended Phase 2 combination doses, with completion expected in 1H26. Phase 2 initiation is targeted for 2H26.

(Press release, Genfit, DEC 10, 2025, https://ir.genfit.com/news-releases/news-release-details/genfit-gns561-shows-promising-antitumor-activity-combination [SID1234661343])