On June 19, 2025 ExCellThera Inc. (ExCellThera), a world leader in blood stem cell expansion and metabolic fitness, reported the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion, recommending granting conditional marketing authorization for Zemcelpro for the treatment of adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available (Press release, ExCellThera, JUN 19, 2025, View Source [SID1234654009]). The European Commission (EC) is expected to make a final decision within approximately two months following CHMP recommendation, and the decision will apply to all 27 European Union (EU) Member States, Iceland, Norway and Liechtenstein.

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Zemcelpro, also known as UM171 Cell Therapy, is a novel cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit.

If approved, Zemcelpro is expected to be the first and only therapy in the EU with marketing authorization for adults with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation following myeloablative conditioning for whom no other type of suitable donor cells is available. Every year in Europe there are over 10,000 new cases of patients with haematological malignancies, including leukemias and myelodysplastic syndromes, requiring bone marrow transplant, and a number of these patients do not have access to suitable donor cells for different reasons, including the absence or unavailability of suitably matched donors.

The positive CHMP opinion was based on the conditional Marketing Authorization Application (MAA) for Zemcelpro. Additional filings are planned for Zemcelpro with other health authorities, including in the US, Canada, the UK, and Switzerland.

"Each year, thousands of people in Europe are diagnosed with haematological malignancies requiring an allogeneic haematopoietic stem cell transplantation, and it’s an upsetting reality that a number of them don’t have access to suitable donor-derived stem cells," said Dr. Guy Sauvageau, CSO and Founder of ExCellThera.

"With today’s positive opinion, we are closer to bringing the life-changing potential of Zemcelpro to patients with at-risk haematological malignancies in the EU," said David Millette, CEO of ExCellThera. "We are proud to bring our transformative cell therapy innovation to patients who continue to have unmet medical needs."

The safety of Zemcelpro is consistent with the well-characterized safety profile of conventional allogeneic blood stem cell transplantation for haematological malignancies following myeloablative conditioning.

ExCellThera extends its sincere gratitude to the patients and investigators who have contributed to the clinical development of Zemcelpro.

About Zemcelpro

Zemcelpro, also known as UM171 Cell Therapy, is a novel cryopreserved haematopoietic stem cell transplantation product containing two components, namely UM171-expanded CD34+ cells (dorocubicel) and unexpanded CD34- cells, each derived from the same cord blood unit.

Zemcelpro, developed by Cordex Biologics, a wholly owned subsidiary of ExCellThera, has been evaluated in 120 patients with haematologic malignancies in clinical trials in the United States, Europe and Canada. Zemcelpro has received orphan drug designation and regenerative medicine advanced therapy (RMAT) designations from the FDA as well as orphan medicinal product designation, advanced therapy medicinal product (ATMP) classification and priority medicines (PRIME) designation from the EMA.

Zemcelpro has been tested in Phase 2 trials in patients with high and very high-risk acute leukemias and myelodysplasias who have limited treatment options with low survival outcomes and high incidence of relapse under the current standard of care, including patients with patients with TP53 mutations or other genetic abnormalities, patients requiring a second transplant, and patients with refractory or active disease. A pivotal Phase 3 trial in this patient population will be initiated as soon as possible.

The use of Zemcelpro in other patient populations, including pediatric patients and patients with non-malignant haematological diseases, is also being explored.

The product is not yet approved for marketing by the EMA and remains subject to European Commission decision. Its safety and efficacy have not been established by other regulatory agencies, such as the FDA and Health Canada.

On June 19, 2025 Whitehawk Therapeutics, Inc. (Nasdaq: WHWK), an oncology therapeutics company applying advanced technologies to established tumor biology to efficiently deliver improved ADC cancer treatments, reported that Dave Lennon, PhD, President and CEO, and David Dornan, PhD, Chief Scientific Officer, will participate in the virtual H.C. Wainwright "HCW@Home" series on Thursday, June 26, 2025, at 10 AM ET (Press release, Whitehawk Therapeutics, JUN 19, 2025, View Source [SID1234654008]).

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To register in advance, please use this link. A live webcast of the event can be accessed by visiting the "Investors & News" page on the Whitehawk Therapeutics website and will be available for replay for approximately 30 days following the event.

On June 19, 2025 Dizal reported the completion of patient enrollment for its WU-KONG28 clinical trial: a multinational, randomized phase III study evaluating the efficacy and safety of sunvozertinib versus platinum-based doublet chemotherapies as a first-line treatment for non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 20 insertion mutations (exon20ins) (Press release, Dizal Pharma, JUN 19, 2025, View Source [SID1234654007]).

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EGFR exon20ins have been difficult to treat due to their unique spatial conformation, diverse mutation subtypes, and high heterogeneity. Patients with EGFR exon20ins NSCLC face a poor prognosis and limited treatment options due to the limited efficacy of first- to third-generation EGFR tyrosine kinase inhibitors (TKIs).

Sunvozertinib, the only oral treatment for NSCLC patients with EGFR exon20ins approved, has demonstrated significant efficacy and a favorable safety profile in this patient population. Both the U.S. Food and Drug Administration (FDA) and the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA) have granted Breakthrough Therapy designation to sunvozertinib for the treatment of EGFR exon20ins NSCLC.

WU-KONG28 is a phase III, multinational, randomized clinical trial investigating sunvozertinib compared to platinum-based doublet chemotherapies in treatment-naïve NSCLC patients with EGFR exon20ins. The study is being conducted across 16 countries and regions in Asia, Europe, North America, and South America.

At the 2023 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Meeting, Dizal reported that sunvozertinib, as a single oral agent, achieved 100% reduction of target lesions, a confirmed objective response rate (ORR) of 78.6%, and a median progression-free survival (mPFS) of 12.4 months, in treatment-naïve patients with advanced or metastatic NSCLC with EGFR exon20ins. In addition, sunvozertinib also demonstrated a well-tolerated safety profile compaired to other EGFR TKIs.

Sunvozertinib is approved in China for the treatment of relapsed and refractory NSCLC with EGFR exon20ins. U.S. FDA granted priority review for its NDA for the same indication with a PDUFA date of July 7, 2025.

About sunvozertinib (DZD9008)

Sunvozertinib is an irreversible EGFR inhibitor discovered by Dizal scientists targeting a wide spectrum of EGFR mutations with wild-type EGFR selectivity. In August 2023, sunvozertinib received approval from NMPA to treat advanced NSCLC with EGFR exon20ins after platinum-based chemotherapies. The approval is based on the results of WU-KONG6 study, the pivotal study of sunvozertinib in platinum-based chemotherapy pretreated NSCLC with EGFR exon20ins. In addition, sunvozertinib also demonstrated encouraging anti-tumor activity in NSCLC patients with EGFR sensitizing, T790M, and uncommon mutations (such as G719X, L861Q, etc.), as well as HER2 exon20ins.

Sunvozertinib showed a well-tolerated and manageable safety profile in the clinic. The most common drug-related TEAEs (treatment-emergent adverse event) were Grade 1/2 in nature and clinically manageable.

Two global pivotal studies are ongoing in ≥ 2nd line (WU-KONG1 Part B) and 1st line setting (WU-KONG28), respectively, in NSCLC patients with EGFR exon20ins.

Pre-clinical and clinical results of sunvozertinib were published in peer-reviewed journals Cancer Discovery and The Lancet Respiratory Medicine.

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]).

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"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.

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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.