On April 22, 2025 Onward Therapeutics SA, a biotechnology company dedicated to advancing innovative cancer immunotherapies, reported that its subsidiary, Emercell SAS, has received Investigational Medicinal Product Dossier (IMPD) approval from the European Medicines Agency (EMA) to initiate a Phase 1 clinical trial of OT-C001, an allogeneic natural killer (NK) cell therapy, in combination with an anti-CD20 monoclonal antibody, Rituximab (Press release, Onward Therapeutics, APR 22, 2025, View Source [SID1234652024]).

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The upcoming phase 1 study (C00101 study) will enroll patients with diffuse large B-cell lymphoma (DLBCL), who have relapsed after, or are refractory to CAR-T cell therapy, or ineligible for CAR-T cell infusion. OT-C001 will be evaluated with Rituximab across various dose levels. The trial will also examine cellular kinetics and biomarkers to characterize the specific mechanism associated with therapeutic responses.

"We are delighted to have reached an important milestone of entering clinical development of OT-C001 following our strategic investments in Emercell since 2021," said Dr. C. Grace Yeh, Chairman and CEO of Onward Therapeutics,"This Phase 1 trial may validate Emercell’s patented platform technology for meaningful clinical outcomes. Along with the continued optimization of industrial manufacturing, these efforts will strengthen our ability to form strategic partnerships and advance this promising therapy."

"OT-C001 sets itself apart by utilizing a pool of multiple umbilical cord blood (UCB) samples, offering both diversity in the product and consistency across GMP batches," said Dr. Patrick Henno, Founder and ex-President of Emercell, "unlike the complex manufacturing processes of many engineered cell therapy products, OT-C001 provides a safe and scalable solution to unmet medical needs in targeted indications."

"We are excited to initiate this multicenter phase 1 trial in one of the most common and aggressive forms of non-Hodgkin lymphoma," said Dr. Alain Herrera, President of Emercell and CMO of Onward Therapeutics, "despite challenges in the cell therapy market, this achievement advances our mission to provide novel treatments for DLBCL patients in need of new therapeutic options. OT-C001 has the versatility to act as a universal platform through combinations with various monoclonal antibodies or targeted therapies; it may benefit patients across multiple indications beyond DLBCL."

About OT-C001

OT-C001 is an allogeneic cell therapy of highly activated and expanded NK cells from UCB, manufactured using a patented expansion and activation process. It targets tumor cells through direct and indirect cytotoxicity, when combined with a monoclonal antibody targeting tumor specific antigens. OT-C001 is being evaluated as a potential new treatment for advanced hematologic malignancies with monoclonal antibodies. In non-clinical in vitro, in vivo models, and toxicology studies, it was well tolerated and displayed promising anti-tumor activities.

On April 22, 2025 Tagworks Pharmaceuticals BV ("Tagworks"), a clinical-stage precision oncology company using its proprietary Click-to-Release treatment platform to develop a new standard of care for patients suffering from solid tumors, reported the U.S. Food and Drug Administration (FDA) cleared the investigational new drug (IND) application for a Phase 1 clinical trial evaluating TGW101, a first-in-class antibody drug-conjugate (ADC) targeting tumor associated glycoprotein 72 (TAG-72) with an monomethyl auristatin E (MMAE) payload, in patients with advanced solid tumors (Press release, Tagworks Pharmaceuticals, APR 22, 2025, View Source [SID1234652023]). The Company also announced the appointment of Keith Orford, MD, PhD, as Chief Medical Officer (CMO).

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Tagworks initiated first-in-human Phase 1 clinical development of TGW101 in a multicenter, open-label, dose-escalation trial in patients with advanced solid tumors. The primary objectives of the study are to evaluate the safety and tolerability of TGW101 and identify the maximum tolerated dose, recommended dose, and regiment for cohort expansion. The study is currently recruiting in the U.S. and plans to enroll up to 50 patients.

"TAG-72 is a validated target with overexpression in many high unmet need solid tumors. However, due to its non-internalizing nature, this target has remained unreachable by current ADCs and other therapies which require uptake by tumor cells to activate their anti-tumor activity. TGW101’s highly differentiated profile enables controlled MMAE payload release and activation in the tumor microenvironment without internalization by tumor cells. This controlled drug release is achieved through a click reaction of the ADC linker with a trigger molecule, which is administered in a second step," said Marc Robillard, Chief Scientific Officer and Co-Founder of Tagworks. "TGW101 has demonstrated a favorable safety profile as well as effective and durable responses in preclinical solid tumor models. The IND clearance and initiation of our Phase 1 clinical trial is a significant milestone for our lead program and Tagworks, the pioneer of Click-to-Release chemistry."

Anthony Tolcher, MD, FRCPC, CEO, Founder, and Director of Clinical Research of NEXT Oncology San Antonio and the TGW101 Phase 1 Primary Investigator commented, "TGW101 represents an attractive new potential treatment option in advanced solid tumors with high unmet need, overcoming certain challenges and limitations of other ADC therapies. TGW101 has the potential to offer an improved anti-tumor effect and safety profile which may lead to better clinical outcomes. I am excited to collaborate with Tagworks’ team to evaluate TGW101 in patients with advanced solid tumors in the Phase 1 clinical trial."

In addition, Tagworks further strengthened its management team with the appointment of Keith Orford, MD, PhD, as CMO. Dr. Orford’s proven track record as a physician, scientist, and executive leader at several biotechnology companies will support Tagworks’ position as a leader and innovator in cancer drug development.

"We are pleased to welcome Keith and look forward to his contributions to the clinical development of our pipeline of innovative ADCs and targeted radiopharmaceuticals," said Ken Mills, Chief Executive Officer of Tagworks. "As we enter into clinical development, Keith’s development expertise, strategic insights, and leadership in the oncology community will be critical in positioning Tagworks for long-term success."

"I am thrilled to join the experienced team at Tagworks to develop and expand on the transformative potential of the Click-to-Release technology with the clear goal of providing safe and efficacious new therapies for patients with cancer," said Keith Orford, MD, PhD, CMO of Tagworks. "I look forward to immediately working closely with all of our investigators to evaluate the therapeutic utility of TGW101 in multiple advanced solid tumor indications."

Dr. Orford brings extensive experience in leadership and oncology development, having served as CMO at public and private cancer biotechnology companies. Most recently, he was CMO, Executive Vice President, Clinical and Translational Science at Parabilis Medicines (formerly Fog Pharma), where he oversaw translational medicine and clinical development. Prior to Parabilis, he held a series of clinical development roles of increasing responsibility, including CMO of Calithera Biosciences, Clinical Development Lead and Clinical Head of Immuno-Oncology and Combinations DPU at GlaxoSmithKline, and Associate Director of Clinical Pharmacology and Experimental Medicine and Oncology at Merck. Before joining industry, he held various roles as a Research Fellow and Instructor at Massachusetts General Hospital and Harvard Medical School, where he trained in internal medicine and completed a postdoctoral fellowship. He received his BS, MD, and PhD from Georgetown University.

About TGW101
TGW101 is an antibody-drug conjugate (ADC) targeting TAG-72, a non-internalizing marker found on the surface of many solid tumor cells. TGW101 consists of a TAG-72-binding diabody conjugated with monomethyl auristatin E (MMAE) toxin. TGW101 is administered intravenous (IV) first and allowed to bind to TAG-72 in the tumor. Then a small molecule trigger is administered IV, resulting in selective chemical cleavage of the linker of the tumor-bound TGW101, release of the MMAE in the extracellular tumor microenvironment and diffusion into surrounding tumor cells. TGW101 is being studied in an open-label, multicenter, Phase 1 dose-escalation clinical trial designed to evaluate safety, tolerability, pharmacokinetics, and preliminary efficacy in patients with advanced solid tumors.

On April 22, 2025 GRAIL, Inc. (Nasdaq: GRAL), a healthcare company whose mission is to detect cancer early when it can be cured, reported that it will present new data highlighting the latest real-world evidence with the Galleri multi-cancer early detection (MCED) test and additional data from GRAIL’s circulating tumor DNA (ctDNA)-based targeted methylation platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting in Chicago, April 25-30, 2025 (Press release, Grail, APR 22, 2025, View Source [SID1234652022]).

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"GRAIL has an extensive evidence program that is setting the standard for multi-cancer early detection development that includes a large real-world dataset demonstrating Galleri test performance and implementation," said Josh Ofman, MD, MSHS, President of GRAIL. "The real-world findings being presented at AACR (Free AACR Whitepaper) support those observed in our previous clinical studies, highlighting the test’s ability to screen for deadly cancers that do not have recommended screening tests. Additional data presented will underscore the potential of GRAIL’s ctDNA-based targeted methylation assay for quantifying abnormal promoter methylation, which is a known hallmark of cancer and has shown potential utility as a biomarker in precision oncology."

Data Presentations

Title: Real-world data and clinical experience from over 100,000 multi-cancer early detection tests
Abstract Number: 7202
Session Title: Targeted Therapies and Combinations 4
Date/Time: April 30, 2025, 9 am-12 pm
Location: Poster section 35

The latest data from a real-world study of more than 100,000 participants with the Galleri test, consistent with previously reported large-scale clinical data, affirming the MCED test can reliably detect a cancer signal across a wide range of cancer types, including cancers without recommended screening, with a high Cancer Signal of Origin prediction accuracy to help guide diagnostic evaluations.
Title: Estimated Post-Test Probabilities of Cancers For Individuals Receiving Multi-Cancer Early Detection (MCED) Tests
Abstract Number: 7132
Session Title: Immune Monitoring / Clinical Correlates
Date/Time: April 30, 2025, 9 am-12 pm
Location: Poster section 31

This modeling analysis shows that individuals receiving a no cancer signal detected MCED test result have a reduced risk of cancer diagnosis for one year post-blood draw; this risk increases as screening interval goes beyond one year, highlighting the importance of annual screening with the MCED test.
Title: Promoter Methylation as a Cancer Biomarker: Insights From ctDNA-Based Targeted Methylation Data
Abstract Number: 1943
Session Title: Liquid Biopsy: Circulating Nucleic Acids 5 / Circulating Tumor Cells 2
Date/Time: April 28, 2025, 9 am-12 pm
Location: Poster section 28

Leveraging insights from the Circulating Cell-free Genome Atlas (CCGA) study, this early proof-of-concept study underscores the potential of GRAIL’s ctDNA-based targeted methylation assay for detecting clinically informative promoter methylation signals in a plasma sample.
Title: Assessment of Cancer Subtypes Across Multiple Cancer Types Using a Circulating Tumor DNA (ctDNA)-Based Targeted Methylation Assay
Abstract Number: 1947
Session Title: Liquid Biopsy: Circulating Nucleic Acids 5 / Circulating Tumor Cells 2
Date/Time: April 28, 2025, 9 am-12 pm
Location: Poster section 28

Based on this proof-of-concept study, GRAIL’s ctDNA-based targeted methylation assay enables subtyping across cancers using a single blood draw, without the need for invasive tissue biopsy.

On April 22, 2025 Akeso, Inc. (9926.HK) ("Akeso" or the "Company") reported that its global first-in-class PD-1/VEGF bispecific antibody, ivonescimab, in combination with chemotherapy, has demonstrated strongly positive results in the Phase III clinical trial (AK112-306/HARMONi-6) for first-line treatment of advanced squamous non-small cell lung cancer (sq-NSCLC) (Press release, Akeso Biopharma, APR 22, 2025, View Source [SID1234652021]). The Independent Data Monitoring Committee (IDMC) declared that the study had met its primary endpoint of progression-free survival (PFS) at the first pre-specified interim analysis. The results of HARMONi-6 study are both statistically significant and clinically meaningful.

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Detailed results from the HARMONi-6 study will be presented at an upcoming medical conference later this year.

Data from the HARMONi-6 study show that, in the intention-to-treat (ITT) population, ivonescimab plus chemotherapy decisively beat tislelizumab plus chemotherapy in terms of progression-free survival (PFS)
The ivonescimab combination showed clinically meaningful PFS benefits in both PD-L1-positive and PD-L1-negative populations
The trial enrolled a total of 532 patients, approximately 63% of whom had centrally located squamous cell carcinoma, a distribution consistent with real-world patient populations
Ivonescimab demonstrated a favorable safety profile, with no new safety signals identified. The incidence of treatment related serious adverse events and the incidence of bleeding events of grade 3 or higher were comparable to those of the control group
The HARMONi-6 study is ivonescimab’s third Phase III clinical trial with positive results in lung cancer, highlighting that treatment with ivonescimab can overcome the limitations of bevacizumab in treating squamous-NSCLC.

The HARMONi-6 study is also ivonescimab’s second Phase III clinical trial with positive results in lung cancer in head-to-head comparisons vs. PD-1 inhibitors, further establishing ivonescimab as a comprehensive treatment option for both first-line and later-line NSCLC. This result further positions ivonescimab to improve upon and replace the current standard of care for the treatment of NSCLC. The study was conducted at 66 clinical research centers across China.

Professor Lu Shun, Director of Shanghai lung cancer, Shanghai Chest Hospital, Shanghai Jiao Tong University School of Medicine and principal investigator of the AK112-306 / HARMONi-6 study, commented:

"It is a great honor for us to witness ivonescimab once again successfully challenge the optimal standard of care. This breakthrough not only advances the treatment of non-small cell lung cancer but also marks a significant milestone in global oncology immunotherapy."

"The interim analysis results from the HARMONi-6 study show that ivonescimab in combination with chemotherapy significantly prolonged progression-free survival (PFS) compared to tislelizumab with chemotherapy. In patients with up to 63% central squamous carcinoma, ivonescimab demonstrated a safety profile comparable to the control group. This highlights its potential to overcome the limitations of bevacizumab in treating squamous non-small cell lung cancer, ultimately enhancing the clinical benefits of immunotherapy for NSCLC. With its combined immune and antiangiogenic mechanisms, ivonescimab offers a promising new treatment option for patients with advanced squamous carcinoma."

Professor Lu added,

"Ivonescimab has gained widespread recognition for treating EGFR-TKI-resistant, advanced nsq-NSCLC since its approval in China nearly a year ago. The HARMONi-2 study showed strong positive results for PD-L1-positive squamous and non-squamous NSCLC in first-line treatment. Additionally, the recent HARMONi-6 study demonstrated significant positive outcomes in first-line squamous NSCLC, further confirming ivonescimab’s exceptional efficacy in both squamous and non-squamous cancers. This positions ivonescimab as a new standard of care for the treatment of these types of cancer. I am confident and excited about its potential in global Phase III trials and its ability to positively reshape the global oncology landscape with a Chinese solution."

Dr. Xia Yu, Founder, Chairwoman, President, and CEO of Akeso, said:

"Today, we are incredibly excited to announce the third significant positive result for ivonescimab in a Phase III study. PD-1 combined with chemotherapy remains the global standard of care for first-line treatment of NSCLC. Ivonescimab has once again demonstrated its breakthrough clinical value and market competitiveness as a next-generation cancer therapy through compelling clinical data. We sincerely thank all the investigators, participants, and patients who have contributed to this clinical study."

Dr. Xia continued,

"Beyond its demonstrated superior efficacy and safety in non-small cell lung cancer, ivonescimab is currently being tested in multiple Phase II and III trials across other cancer types, establishing a clear leadership in both improving patients’ lives and addressing critical unmet need across multiple cancer types. The success of the HARMONi-6 study validates our very high confidence in continuing to integrate global resources and advancing ivonescimab’s role as a next-gen immunotherapy. We look forward to working with our partner Summit on expanding global access to ivonescimab. We are impressed by and also appreciative of their progress in developing ivonescimab in the US, Europe, and Japan. We are committed to improving the standard of care, changing treatment approaches, and offering safer, more unwaveringly effective solutions for patients worldwide."

On April 22, 2025 Oncotelic Therapeutics, Inc. (OTCQB:OTLC), a biotechnology company focused on nanomedicine and RNA-based therapeutics, reported that its Chairman and CEO, Dr. Vuong Trieu, will deliver a keynote presentation at the 21st Annual Congress of International Drug Discovery Science & Technology (IDDST-Europe), scheduled for June 18-20, 2025, in Stockholm, Sweden (Press release, Oncotelic, APR 22, 2025, View Source [SID1234652020]).

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Dr. Trieu’s presentation, titled " Deciparticles: Novel Sub-20 nm Nanoparticles for Advanced Drug Delivery" will highlight the groundbreaking Deciparticles platform developed by Oncotelic Therapeutics and Sapu Nano. Deciparticles are a novel class of sub-20 nm nanoparticles specifically engineered to overcome challenges associated with delivering insoluble drugs to solid tumors. Deciparticles platform is able to formulate more than 90% of all water insoluble drugs tested – including the taxanes,

The presentation will outline key findings demonstrating that Deciparticles significantly improve tumor penetration and achieve superior antitumor efficacy compared to conventional formulations. A central feature of this innovation is the integration of biomarker-driven strategies, leveraging DNA methylation signatures and gene-expression profiles predictive of patient survival, potentially enabling personalized therapeutic interventions. Oncotelic’s lead candidate, Sapu-001, has shown promising preclinical results, demonstrating a higher maximum-tolerated dose and reduced toxicity relative to existing therapies.

Dr. Trieu emphasized, "With Deciparticles, we’ve entered a new era of targeted drug delivery-leveraging sub-20 nm nanoparticles combined with epigenetic biomarkers to significantly enhance treatment outcomes for patients. We anticipate Phase 1 clinical trials for Sapu-001 later this year."

Presentation Details:

Event: IDDST-Europe 2025 Conference
Date: June 19, 2025, at 10:30 CET
Speaker: Vuong Trieu, Ph.D., Chairman & CEO, Oncotelic Therapeutics
Title: " Deciparticles: Novel Sub-20 nm Nanoparticles for Advanced Drug Delivery"