On June 19, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported a business update and highlights its key milestones expected for the second half of 2025 (Press release, MaaT Pharma, JUN 19, 2025, View Source [SID1234654006]).

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!

"Following the submission of the Marketing Authorization Application to the EMA for our lead asset, Xervyteg, earlier this month, we are excited to advance Xervyteg toward commercialization — a potential world first for a microbiota therapeutic in oncology — and are now fully focused on progressing registration activities across Europe ", said Hervé Affagard, CEO and co-founder of MaaT Pharma. " This marks a major step in confirming our commitment to address high unmet medical needs and, importantly, it serves as a stepping stone toward international expansion, as we aim to bring our therapies to patients worldwide ".

Pipeline highlights

In Hemato-Oncology

Acute Graft-versus-Host Disease (aGvHD) – Xervyteg (MaaT013)

In January 2025, the Company announced positive topline results from the pivotal Phase 3 ARES Study evaluating Xervyteg (MaaT013) in aGvHD. The study met its primary endpoint with a significant gastrointestinal overall response rate at Day 28 of 62% and demonstrates the unprecedented efficacy of Xervyteg as third-line treatment of aGvHD with gastrointestinal involvement (GI-aGvHD) consistent with communicated EAP results.
On June 02, 2025, the Company announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency (EMA) for its lead drug candidate MaaT013, under the registered brand name of Xervyteg. If approved, the Marketing Authorization would establish Xervyteg as the first microbiota therapeutic approved by the EMA, the first one in hemato-oncology worldwide and the first approved therapy in third-line GI-aGvHD.
On June 13, 2025, the Company presented positive updated data in Early Access Program for 173 patients at the 2025 annual EHA (Free EHA Whitepaper) Congress supporting the high efficacy and good safety profile of Xervyteg. This dataset confirms the breakthrough potential of Xervyteg for aGvHD patients with limited treatment options.
Final results from the pivotal ARES study, including 12-month overall survival data, are expected before the end of 2025 and will be incorporated into the filing dossier.
The potential marketing authorization could be delivered around mid-2026, enabling the start of the commercialization of Xervyteg in Europe.
MaaT Pharma is advancing discussions with potential partners to accelerate the commercialization plan across Europe.
MaaT Pharma primarily focuses on the commercialization of its most advanced asset with the completion of regulatory steps in Europe, and dedicated preparation activities for the European launch of Xervyteg.
In parallel, the Company continues discussions with the FDA to optimize a dedicated pivotal study in the U.S., with the objective of enabling the earliest possible access to Xervyteg for U.S. patients. Such a study could be initiated in 2026 (instead of Q4 2025), subject to regulatory confirmation as MaaT Pharma continues watching the evolving regulatory policies and process in the United States.
The Company continues the ongoing Early Access Program in the United States, initiated in December 2024.
Allogenic Hematopoietic Stem Cell Transplant (allo-HSCT) – MaaT033

Over the past 12 months, three DSMB safety assessments were conducted for MaaT033 in the Phase 2b PHOEBUS randomized trial designed to be pivotal: two routine evaluations and one interim analysis focused on excess mortality. All confirmed a favorable safety profile and recommended continuation of the trial without modifications.
The last patient enrollment in the trial is anticipated for mid-2026 while the 1-year OS results are expected in H2 2027.
In Immuno-Oncology

Xervyteg and MaaT033 – Proof-of-Concept trials using the MET-N platform (donor derived conducted as Investigator-Sponsored Trials (ISTs).

In March 2024, the Company completed patient recruitment for the Phase 2a randomized clinical trial (NCT04988841) (PICASSO) sponsored by AP-HP in Paris and in collaboration with INRAE and Institut Gustave Roussy, evaluating Xervyteg in combination with immune checkpoint inhibitors (ICI), ipilimumab (Yervoy) and nivolumab (Opdivo), in metastatic melanoma patients. The Company provided its Xervyteg drug candidate and placebo and contributes to the microbiome profiling of patients using its proprietary gutPrint AI research engine, while the trial investigator-sponsor handled recruitment, treatment and is overseeing data collection and analysis. Data readout is expected in H2 2025 as previously announced.
In May 2024, the Company announced its participation in the IMMUNOLIFE ‘RHU’ (university hospital trial) program, a consortium including academic partners, such as Institut Gustave Roussy (IGR), a world-renowned center in the field of cancer treatment, and biotech companies. MaaT033 will be tested as a concomitant treatment to cemiplimab (Regeneron), an anti-PD1 therapy, to assess the potential increase in response rate in patients having received antibiotics. This investigator-sponsored, randomized, multicenter Phase 2 trial will evaluate MaaT033 in patients with advanced non-small cell lung cancer (NSCLC), with MaaT Pharma supplying the investigational product. The trial is expected by the sponsor to start mid-2025.
MaaT034 – Next-generation drug candidates with co-cultured technology (MET-C platform)

In April 2025, the Company presented new preclinical data for MaaT034, its next generation product, showing compelling anti-tumor efficacy results in germ-free mice at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025. Key results included:
Metagenomic analysis shows that MaaT034 reproduces the microbial functions of Xervyteg
MaaT034 improves DC-mediated T cell activation and potentiates anti-tumor effects mediated by anti-PD-1 checkpoint blockade in vitro.
70% of MaaT034 microbial species engraft in mice, ensuring an enduring presence of beneficial bacteria in the gut environment.
MaaT034 increases the production of key microbial-derived metabolites such as short-chain fatty acids in germ-free mice. This translates into an improved gastrointestinal physiology as evidenced by gut mucosal restoration.
MaaT034 optimizes anti-PD1 mediated activity in tumor-bearing, germ-free mice. While anti-PD1 alone reduced tumor growth by 10%, the combination of anti-PD1 and MaaT034 resulted in a 83.7% tumor growth reduction (compared to a 24.2% reduction when using a single strain of Akkermansia muciniphilabacteria).
In Neurodegenerative Diseases

In May 2025, MaaT Pharma announced positive final Phase 1b results for MaaT033 in Amyotrophic Lateral Sclerosis (ALS), showing a favorable safety and tolerability profile supported by biomarker and microbiome analyses. Moving forward, the Company is seeking a partner to further advance clinical evaluation in ALS.
As a reminder, the Company’s Annual General Meeting will take place on Friday, June 20, 2025, at 9:30am CET at the Company’s headquarter in France located at 70 avenue Tony Garnier, 69007 Lyon and will also be broadcasted live. A presentation by the management team on recent developments and perspectives will take place from 9:00 to 9:30 a.m. CET, prior to the General Meeting. For more information, please visit the investors section on the Company’s website.

On June 19, 2025 Amplia Therapeutics Limited (ASX: ATX), ("Amplia" or the "Company"), reported further data from the ongoing ACCENT trial investigating the Company’s best-in-class FAK inhibitor narmafotinib in advanced pancreatic cancer (Press release, Amplia Therapeutics, JUN 19, 2025, View Source [SID1234653983]). A second patient of the 55 patients enrolled in the trial has recorded a confirmed complete response (CR). This finding follows the announcement earlier this week1 of a separate patient who achieved a pathological CR in the trial.

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!

A confirmed complete response is a formal designation of response where there is a complete disappearance of all tumour lesions that is maintained for >2 months. This is a rare outcome in advanced pancreatic cancer where the disease has spread to other parts of the body. For example, the seminal study demonstrating efficacy of the chemotherapies gemcitabine and Abraxane in advanced pancreatic cancer reported only one (1) CR out of 431 patients.

Amplia CEO and MD Dr Chris Burns commented: "To see a second complete response in the ACCENT trial is really wonderful news, particularly given how rare these are observed in advanced pancreatic cancer. Along with the pathological CR announced earlier in the week, this outcome further demonstrates the promising activity narmafotinib, on top of standard-of-care, is showing in pancreatic cancer."

This ASX announcement was approved and authorised for release by the Board of Amplia Therapeutics.

About Narmafotinib

Narmafotinib (AMP945) is the company’s best-in-class inhibitor of the protein FAK, a protein over-expressed in pancreatic cancer and a drug target gaining increasing attention for its role in solid tumours. The drug, which is a highly potent and selective inhibitor of FAK, has shown promising data in a range of preclinical cancer studies.

About the ACCENT Trial

The ACCENT trial is entitled ‘A Phase 1b/2a, Multicentre, Open Label Study of the Pharmacokinetics, Safety and Efficacy of AMP945 in Combination with Nab-paclitaxel and Gemcitabine in Pancreatic Cancer Patients’.

The trial is a single-arm open label study conducted in two stages. The first stage (Phase 1b), completed in November 2023, determined an optimal dose of narmafotinib (AMP945) by assessing the safety, tolerability, pharmacokinetics and preliminary efficacy when dosed in combination with gemcitabine and Abraxane in first-line patients with advanced pancreatic cancer.

The second stage (Phase 2a) of the trial is designed to assess efficacy in combination with gemcitabine and Abraxane. The primary endpoints are Objective Response Rate (ORR) and Duration on Trial (DOT) with secondary endpoints being Progression Free Survival (PFS) and Overall Survival (OS). Safety and tolerability will continue to be assessed.

The trial is being conducted at seven sites in Australia and five sites in South Korea.

More information about the ACCENT trial can be found via the ACCENT trial site, the Amplia Therapeutics website and at ClinicalTrials.gov under the identifier NCT05355298.

On June 18, 2025 Akari Therapeutics, Plc (Nasdaq: AKTX), an oncology biotechnology company developing novel immuno-oncology payload antibody drug conjugates (ADCs) for the treatment of cancer, reported the Intellectual Property India (IPI) has issued Patent No. 562,919 titled,"Thailanstatin Analogs" (Press release, Akari Therapeutics, JUN 19, 2025, View Source [SID1234654005]).

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 issued patent covers claims for the Company’s potent immuno-oncology PH1 payload, proprietary non-cleavable and cleavable linkers and ADC technology which has applications across various cancer targets.

"We believe our innovative ADC payload PH1 has the potential to provide therapies that disrupt cellular function, trigger cancer cell death and most importantly, enhance the immune system to fight cancer beyond the cells targeted by the ADC molecule. The issuance of this patent further strengthens our global intellectual property estate and importantly, provides additional patent protection around our CMC manufacturing activities for this PH1payload and related analogs. Importantly, as cancer rates and diagnosis continue to rise significantly in India, we believe the need for innovative cancer therapies continue to increase. We are pleased to bolster our intellectual property portfolio to include this key market for future opportunities to potentially help cancer patients with our immuno-oncology ADCs," commented Abizer Gaslightwala, President and CEO of Akari Therapeutics.

The issued India patent is a divisional patent of the Company’s R&D toxin portfolio on PH1 and builds on the portfolio of previously issued patents (Patent No. US 10,815,246 B2, Patent No. US 10,301,319 B2 and Patent No. US 11,691,982 B2). Grant Patent right for PCT/US2018/051721 family was issued by China National Intellectual Property Administration in August 2023, and in Israel in September 2023. Corresponding foreign patent applications are pending and currently undergoing examination in Patent Cooperation Treaty ("PCT")countries – Brazil, Canada, member states of the European Patent Organisation ("EPO"), Hong Kong, Japan, New Zealand, Singapore, & South Africa.

Leveraging its innovative payload platform, the Company is advancing a pipeline of potentially first-in-class, best-in-class ADC candidates across a wide range of cancer tumor targets. These initial candidates have shown significant tumor-killing activity in preclinical models with the ability to robustly activate the immune system to drive durable, and sustained outcomes.

On June 18, 2025 FUJIFILM Healthcare Americas Corporation, a leading provider of diagnostic and enterprise imaging solutions, reported that use of its AFP-L3 and DCP in vitro diagnostic tests is being evaluated in the National Liver Cancer Screening Trial (known as TRACER), a study funded by the National Cancer Institute, part of the National Institute of Health (Press release, Fujifilm, JUN 18, 2025, View Source [SID1234653998]). The multi-center study compares liver cancer screening strategies in patients with cirrhosis or chronic hepatitis B.

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!

Hepatocellular cancer (HCC) is the third leading cause of cancer-related mortality worldwide and the most rapidly growing cause of cancer deaths in the U.S.1 HCC mortality continues to escalate due to the increased prevalence of chronic liver diseases, inadequate diagnostic precision in the early stages of disease, and historical lack of effective systemic therapies for late-stage HCC. Surveillance of high-risk patients is typically conducted via liver ultrasound in combination with serum alpha-fetoprotein (AFP) serum testing. However, ultrasound sensitivity can be compromised by the severity and underlying cause of liver disease, and serum AFP levels can be affected by hepatic inflammation, patient characteristics, and hereditary and other non-hepatic disorders,2 among other factors, compromising early-stage HCC diagnostic accuracy.

The use of serum biomarkers such as AFP-L3 and des-gamma-carboxyprothrombin (DCP) in surveillance can significantly improve early diagnosis capabilities, and the GALAD score3 – derived from Gender, Age, AFP-L3, AFP and DCP – has shown promise as a more accurate model for screening and early detection of HCC in at-risk patients in recent studies, including the HCC Early Detection Strategy (HEDS) Study conducted through the EDRN (View Source). FUJIFILM Healthcare Americas Corporation In Vitro Diagnostics division offers FDA-cleared AFP-L3 and DCP biomarker tests that are utilized as an aid in HCC risk assessment in conjunction with other laboratory findings, imaging studies, and clinical assessment.

The GALAD model continues to be studied in the TRACER study. The Phase IV prospective validation study, which began enrollment in January 2024 and is being conducted in the U.S., will enroll 5,500 at-risk patients across 15 sites that will be randomly assigned semi-annual screening with ultrasound ± AFP arm (the standard of care) or semi-annual screening via the GALAD model. Patients will be actively recruited over a 3-year period, with assessment of the data at year 5.5; the primary aim is a reduction in the proportion of late-stage HCC diagnosis. The TRACER study will measure secondary outcomes of interest including psychological and financial harms utilizing validated patient surveys.

"We are pleased that the TRACER study is evaluating the use of Fujifilm’s biomarker tests as a potential way to improve effectiveness in HCC screening in at-risk patients," said Henry (Hidetoshi) Izawa, president and chief executive officer, FUJIFILM Healthcare Americas Corporation. "HCC is often diagnosed at an advanced stage, with limited treatment options. Our hope is that this study will further underscore the benefits of HCC-specific blood tests in the detection of early-stage HCC, and patients will benefit from earlier diagnosis and treatment options."

The use of the Fujifilm’s biomarkers in the GALAD model is a novel concept and for research use only.
TRACER is supported by a grant within the Early Detection Research Network, a program of the National Cancer Institute, National Institutes of Health.

On June 18, 2025 CEL-SCI Corporation (NYSE American: CVM) reported the U.S. Food and Drug Administration’s (FDA) approval of Merck’s KEYTRUDA (pembrolizumab), an anti-PD-1 therapy, for the treatment of adult patients with resectable locally advanced head and neck squamous cell carcinoma (HNSCC) whose tumors express PD-L1 (Combined Positive Score [CPS] ≥1) as determined by an FDA-approved test (Press release, Cel-Sci, JUN 18, 2025, View Source [SID1234653997]). Merck’s application was granted the FDA’s priority review on February 25, 2025, and regulatory approval was granted on June 13, 2025, based on interim results from Keytruda’s Phase 3 KEYNOTE-689 trial.

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!

Last week the FDA approved Keytruda as a perioperative (before and after surgery) treatment for resectable locally advanced head and neck cancer patients whose tumors express PD-L1 at a positive level. In Merck’s Phase 3 KEYNOTE-689 trial, Keytruda reduced the risk of recurrence and progression by 30%, compared with standard of care, in patients whose tumors expressed PD-L1 (CPS ≥1). The study did not show an improvement in overall survival. Patients with low to zero levels of PD-L1 did not benefit from Keytruda.

In contrast to the results of the KEYNOTE-689, CEL-SCI’s Phase 3 study showed that Multikine* treated patients whose tumors expressed low (Tumor Proportion Score [TPS <10]) to zero PD-L1, had their risk of death reduced by 66% (hazard ratio 0.34, 95% CI [0.18, 0.65], p=0.0012) and extended the 5-year overall survival to 73% compared to 45% in the standard of care, log rank p=0.0015. About 70% of the patients in CEL-SCI’s Phase 3 study had low to zero levels of PD-L1.

"It is encouraging for CEL-SCI that Merck’s Keytruda application received the FDA’s priority review and that marketing approval was given based on a Phase 3 study’s first pre-specified interim analysis. CEL-SCI has received the FDA’s go-ahead for a confirmatory Registration Study with 212 patients based on results from the completed Multikine Phase 3 study in head and neck cancer patients. The patients in the Phase 3 study that benefited from Multikine pre-surgery treatment showed an almost 4-year median overall survival advantage over control, and pre-surgery tumor responses to Multikine predicted survival benefit. The Keytruda approval based on pre-specified interim results strongly implies that Multikine has the potential for accelerated regulatory approval based on favorable post-surgical tumor responses," stated CEL-SCI’s CEO, Geert Kersten.