On May 28, 2025 The Experimental Drug Development Centre (EDDC), Singapore’s national platform for drug discovery and development, reported that the U.S. Food and Drug Administration (FDA) has granted Fast Track Designation for EBC-129 for the treatment of pancreatic ductal adenocarcinoma (PDAC) (Press release, Experimental Drug Development Centre, MAY 28, 2025, View Source [SID1234654012]). EBC-129 is a first-in-class antibody drug conjugate (ADC) targeting a novel, tumour-specific N256-glycosylated epitope on CEACAM5 and CEACAM6. It is currently undergoing Phase 1 clinical trials for the treatment of patients with solid tumours with high unmet medical need.

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The Fast Track Designation facilitates the expedited development of EBC-129, enabling more frequent engagement with the FDA to discuss the clinical development plan. It also provides potential eligibility for Priority Review and Accelerated Approval, as well as rolling review of any future Biologic License Application (BLA).

"The FDA’s Fast Track Designation for EBC-129 underscores the promise of this novel ADC in addressing the critical need for expanded treatment options for PDAC patients and represents an important step in our efforts to accelerate its development. We view this as both a validation of our efforts and a responsibility to move decisively to advance EBC-129 as a new option to patients in need," said Professor Damian O’Connell, Chief Executive Officer of EDDC.

Updated clinical data from the ongoing Phase 1 study of EBC-129 will be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place in Chicago from 30 May to 3 June 2025.

Presentation Details at ASCO (Free ASCO Whitepaper) 2025:

Title: Clinical activity of EBC-129, a first-in class, anti-N256-glycosylated CEACAM5 and CEACAM6 antibody-drug conjugate (ADC), in patients with pancreatic ductal adenocarcinoma (PDAC) in a Phase 1 study

Rapid Oral Session Title: Gastrointestinal Cancer—Gastroesophageal, Pancreatic, and Hepatobiliary

Date and Time: Monday, June 2, 2025, 11:30 AM – 1:00 PM GMT-5

Abstract Number: 4018

Presenter: Assistant Professor Robert W. Lentz, MD, Division of Medical Oncology, Department of Medicine, University of Colorado Anschutz School of Medicine

About EBC-129

EBC-129 is an ADC that targets a tumour-specific N256-glycosylation site conserved on CEACAM5 and CEACAM6. CEACAM5 and CEACAM6 are known to have functional importance in tumour formation, migration and metastasis. In the ongoing trial, the tumour-specific marker is found to be widely expressed in multiple solid tumour types, including gastric, oesophageal, pancreatic, lung, colorectal, and appendiceal cancers, based on an analytically validated immunohistochemistry (IHC) assay. The payload used in EBC-129 is monomethyl auristatin E (MMAE) which has been extensively tested and approved for clinical use in other marketed ADCs and has demonstrated synergy with PD-1 inhibitors. The ongoing Phase 1 trial of EBC-129 is assessing the safety and tolerability of EBC-129 as a single agent and in combination with pembrolizumab in patients with advanced solid tumours. Enrolment for the PDAC cohort in the Phase 1 dose expansion study is now complete, while recruitment continues for the gastroesophageal adenocarcinoma (GEA) and IHC-positive cohorts.

On May 28, 2025 Exact Sciences Corp. (NASDAQ: EXAS), a leading provider of cancer screening and diagnostic tests, reported new data to be presented from the Beta-CORRECT clinical validation study at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Press release, Exact Sciences, MAY 28, 2025, View Source [SID1234653438]). Results from Beta-CORRECT, a subset of the GALAXY cohort, validate the performance of its tumor-informed molecular residual disease (MRD) test, Oncodetect, in predicting recurrence in stage II–IV colorectal cancer. These data confirm the test’s role in supporting treatment and surveillance decisions.

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Building on this momentum and its commitment to innovation, Exact Sciences will introduce a next-generation version of the test leveraging the Broad Institute’s MAESTRO technology. Early data show the test will track up 5,0002 patient specific variants and detect ctDNA* levels below 1 part per million.1 The test will be available to both new and existing customers in 2026.

"We launched the Oncodetect test to give clinicians and patients a powerful tool for detecting cancer recurrence earlier and with greater precision—progress that’s already being realized," said Brian Baranick, Executive Vice President and General Manager, Precision Oncology at Exact Sciences. "We continue to innovate and look forward to introducing the next iteration of the Oncodetect test, which leverages whole-genome sequencing and proprietary technology developed in collaboration with the Broad Institute to enhance sensitivity and expand clinical utility."

The Beta-CORRECT study demonstrates that the Oncodetect test significantly improves prognosis prediction compared to traditional standard of care methods †,3,4,5

Data presented at ASCO (Free ASCO Whitepaper) from the Beta-CORRECT clinical validation study confirm that the Oncodetect test accurately predicts recurrence in stage III colorectal cancer3—consistent with findings from the Alpha-CORRECT study—and extends this association to stages II and IV.4

Exact Sciences’ largest MRD clinical study to date, with more than 400 patients, demonstrates those with ctDNA-positive results after therapy and during surveillance showed a 24- and 37-fold increased risk of recurrence, respectively.4 By quantifying ctDNA levels across multiple timepoints, the Oncodetect test enables physicians to more effectively guide treatment decisions and surveillance strategies in clinical practice.3,4

Advancing the Oncodetect test with next generation innovation

The next-generation MRD test, currently in validation across multiple solid tumor types, will track up to 5,000 patient-specific variants2 with a limit of detection below 1 part per million,1 enabling scalable monitoring and broad clinical utility. Exact Sciences holds exclusive rights to the Broad Institute’s MAESTRO technology, a whole-genome sequencing method able to detect low-frequency ctDNA mutations with high accuracy. This technology advances the ability to look broadly across thousands of mutations while reducing the sequencing depth required to achieve an ultra-low limit of detection at a highly attractive cost point. Through continued innovation in MRD, Exact Sciences is advancing solutions with the potential to change clinical practice.

"The precision and sensitivity seen in the next generation test reflect deep scientific collaboration and a shared commitment to advancing MRD technology," said Viktor Adalsteinsson, Ph.D., Director, Gerstner Center for Cancer Diagnostics at the Broad Institute. "This approach to innovation will continue to raise the bar for recurrence monitoring, treatment response assessment, and, ultimately, patient outcomes."

On May 28, 2025 Halozyme Therapeutics, Inc. (NASDAQ: HALO) (Halozyme) reported that Bristol Myers Squibb received European Commission (EC) approval of a new Opdivo (nivolumab) subcutaneous formulation developed with ENHANZE, Halozyme’s proprietary recombinant human hyaluronidase enzyme (rHuPH20), for use across multiple adult solid tumors as monotherapy, monotherapy maintenance following completion of intravenous nivolumab plus Yervoy (ipilimumab) combination therapy, or in combination with chemotherapy or cabozantinib (Press release, Halozyme, MAY 28, 2025, View Source [SID1234653455]).

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"The European approval of Opdivo SC represents an advancement for certain cancer patients, who now have the option to receive their Opdivo as a 3-to-5-minute SC injection," said Dr. Helen Torley, president and chief executive officer of Halozyme. "This approval is just one of the 11 growth catalysts for our commercialized SC products expected this year."

The positive EC decision is supported by positive results from the Phase 3 CheckMate -67T trial. For more information on the study and its findings, please view Bristol Myers Squibb’s press release issued on May 28, 2025.

The approval by the EC is valid in all 27 member states of the European Union (EU), as well as Iceland, Liechtenstein and Norway.

On December 27, 2024, subcutaneous nivolumab and hyaluronidase-nvhy, marketed under the brand name Opdivo Qvantig, was approved by the U.S. Food and Drug Administration.

On May 28, 2025 InnoCare Pharma (HKEX: 09969; SSE: 688428), a leading biopharmaceutical company focusing on the treatment of cancer and autoimmune diseases, reported the approval of the Investigational New Drug (IND) by the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) to conduct the clinical trial of B-cell lymphoma-2 (BCL2) inhibitor mesutoclax (ICP-248) in combination with azacitidine for the treatment of myeloid malignancies, including but not limited to myelodysplastic syndromes (MDS) (Press release, InnoCare Pharma, MAY 28, 2025, View Source [SID1234653471]).

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Mesutoclax is a novel, orally bioavailable BCL2 selective inhibitor. BCL2 is an important regulatory protein in the apoptosis pathway, and its abnormal expression is associated with the development of various hematologic malignancies. Mesutoclax exerts anti-tumor activity by selectively inhibiting BCL2 and restoring the normal apoptosis process in cancer cells.

Myelodysplastic syndrome (MDS) is a group of heterogeneous clonal myeloid diseases characterized by the abnormal proliferation of hematopoietic stem cells, recurrent genetic abnormalities, myelodysplasia, ineffective hematopoiesis, peripheral-blood cytopenia, and a high risk of progression to acute myeloid leukemia (AML). The annual incidence of myelodysplastic syndromes is about 4 cases/100,000 people/year (reaching 40–50/100,000 in patients aged ≥ 70 years).

Mesutoclax has been granted Breakthrough Therapy Designation (BTD) by the CDE for the treatment of BTKi-treated relapsed or refractory mantle cell lymphoma (R/R MCL). This marks the first BCL2 inhibitor to receive BTD recognition in China. The Company is accelerating patient enrollment of a Phase III registrational trial of mesutoclax in combination with orelabrutinib as a first line therapy for the treatment of chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL), as well as a clinical trial of mesutoclax for the treatment of AML.

Dr. Jasmine Cui, the Co-founder, Chairwoman, and CEO of InnoCare, said, "Mesutoclax is an important global asset of our company in the field of hematology. We are delighted to receive approval to initiate the clinical trial for the fourth indication of our BCL2 inhibitor. We will accelerate the clinical development of mesutoclax across multiple indications in China and globally to bring benefits to patients as early as possible."

On May 28, 2025 Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, reported that its research collaborators presented positive preclinical data and research from studies of GPX-002, the Company’s diabetes gene therapy drug candidate, in an oral presentation at the American Society of Gene and Cell Therapy’s (ASGCT) (Free ASGCT Whitepaper) 28th Annual Meeting which took place May 13-17, 2025 in New Orleans, Louisiana (Press release, Genprex, MAY 28, 2025, View Source [SID1234653439]).

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"We are pleased with the preclinical studies evaluating our novel gene therapy in diabetes, and we are proud to have been selected to present this data before an audience of innovators in cell and gene therapy," said Ryan Confer, President and Chief Executive Officer at Genprex. "We believe GPX-002 offers a promising opportunity for curative therapy in diabetes, which could impact the millions of patients suffering from this debilitating disease."

The oral presentation details for the Genprex-supported abstract:

Abstract Title: Immune Modulation Sustains Alpha Cell Reprogramming and Mitigates Immune Responses to AAV in a Diabetic Non-Human Primate Model

Session Title: Challenges in Immunological Responses to Therapeutic Interventions

Presenter: Hannah Rinehardt, MD, University of Pittsburgh Medical Center

Presentation Date: May 16, 2025

Presentation Time: 4:15 – 4:30 p.m. CT

Location: Room 278-282

In the oral presentation, Dr. Rinehardt discussed GPX-002, the diabetes gene therapy, which uses intraductal infusion of recombinant adeno-associated virus (rAAV) to deliver the Pdx1 and MafA genes. The gene therapy converts alpha cells into beta-like cells that secrete insulin physiologically, reversing diabetes in mouse models, where immunosuppression was not necessary. Researchers evaluated the immune response to direct infusion of rAAV into the pancreatic duct of Non-Human Primates (NHPs) with streptozotocin-induced diabetes and evaluated how to best manage immune responses.

Diabetes was induced with streptozotocin (STZ) in cynomolgus macaques, a type of NHP. NHPs received retrograde intraductal infusion of rAAV via laparotomy for precise delivery to the pancreas. rAAV capsids were chosen based on tropism for endocrine cells, and pre-existing neutralizing antibody titers (NAbs) were negative. Blood work, including serum C peptide and IV glucose tolerance tests, were serially obtained to monitor therapeutic efficacy. Immune response monitoring was performed for up to 4 months post-infusion and included serial NAbs, ELISpot assays, and immunophenotyping. Pancreatic tissues were analyzed using IHC and RNA-scope for beta cell markers, as well as single-cell RNA transcriptomics.

Expression of viral proteins occurs for a limited period of time after rAAV infection, since the infection doesn’t produce new AAV virus. One-month post-infusion, NHPs showed improved glucose tolerance and reduced insulin requirements. In the following months, using steroid-sparing regimens, increases in pancreatic B and T lymphocyte populations were noted on scRNA sequencing. Temporary immunosuppression (IS), using a combination of rituximab, rapamycin, and steroids for a 3-month course is largely effective at preventing anti-viral immunity. However, discontinuation of IS at 3 months post-infusion led to an immune response afterwards, indicating that IS in NHPs may need to be continued longer.

In conclusion, the novel rAAV gene therapy demonstrated that infusion of rAAV directly into the pancreatic duct of NHPs improves glucose tolerance but induces an anti-viral immune response which can degrade the improvement in glucose tolerance. The anti-viral immune response in NHPs can be largely prevented by administration of a multi-agent IS that leads to sustained therapeutic effects.

Researchers are continuing preclinical studies of GPX-002 therapy in NHP models of both Type 1 and Type 2 diabetes to generate additional data, and present studies are evaluating viral efficacy after six months of immunosuppression.

For more in-depth preclinical data supporting GPX-002, please review Genprex’s Corporate Presentation.

About GPX-002

GPX-002, which has been exclusively licensed from the University of Pittsburgh, is currently being developed using the same construct for the treatment of both Type 1 diabetes (T1D) and Type 2 diabetes (T2D). The same general novel approach is used in each of T1D and T2D whereby an adeno-associated virus (AAV) vector containing the Pdx1 and MafA genes is administered directly into the pancreatic duct. In humans, this can be done with a routine endoscopy procedure. In T1D, GPX-002 is designed to work by transforming alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body’s immune system. In vivo, preclinical studies show that GPX-002 restored normal blood glucose levels for an extended period of time in T1D mouse models. In T2D, where autoimmunity is not at play, GPX-002 is believed to rejuvenate and replenish exhausted beta cells.