On January 12, 2026 Eureka Therapeutics, Inc., a clinical-stage biotechnology company advancing next-generation T-cell therapies for cancer, reported that the U.S. Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to ECT204, a GPC3-targeting ARTEMIS CAR T-cell therapy, for the treatment of patients with advanced hepatocellular carcinoma (HCC), the most common form of liver cancer.

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The RMAT designation followed the FDA’s review of data from the completed Phase I cohort, together with additional clinical data, from Eureka’s ongoing Phase I/II ARYA-3 trial evaluating ECT204, an autologous ARTEMIS CAR T-cell therapy based on the AbTCR (Antibody-T Cell Receptor) architecture, in adult patients with GPC3-positive advanced HCC. RMAT designation, granted based on preliminary clinical evidence of potential to address unmet medical needs, provides the benefits of Breakthrough Therapy designation for regenerative medicines, including enhanced FDA interaction, alignment on development and manufacturing strategy, discussion of surrogate or intermediate clinical endpoints that may support accelerated approval, and eligibility for priority review of a future biologics license application (BLA).

"This represents an important inflection point for Eureka and for cell therapy in solid tumors," said Dr. Cheng Liu, Founder and Chief Executive Officer of Eureka Therapeutics. "ARTEMIS was designed to overcome the limitations of conventional CAR T-cell therapies in solid tumors, and early clinical observations with ECT204 in liver cancer reinforce our belief that the ARTEMIS architecture can meaningfully expand the therapeutic reach of cell therapy."

HCC is among the most biologically resistant solid tumors in oncology, characterized by an immunosuppressive tumor microenvironment that has posed significant challenges to the effectiveness of cell-based therapies. Unlike conventional CAR T cells that rely on a synthetically fused CD3-zeta (ζ) signaling domain linked to co-stimulatory molecules, which may contribute to constitutive activation and limited persistence in solid tumors, ARTEMIS CAR T cells were engineered using the AbTCR architecture and incorporate dual receptors that leverage human gamma delta (γ/δ) T cell receptor (TCR) signaling with co-stimulation delivered through separate receptors. This more natural TCR signaling design combines the precision of antibody-redirected tumor targeting with balanced and sustained T-cell activation, which may explain the improved tumor infiltration, persistence, and functional activity of the engineered T cells in hostile solid tumor environments.

The advantages of ARTEMIS CAR T cells over conventional CAR T cells have been validated by independent research conducted in collaboration with the National Cancer Institute, including NCI-led, peer-reviewed studies published in Cell Reports Medicine. In preclinical solid tumor models, ARTEMIS CAR T cells demonstrated significantly greater tumor infiltration and anti-tumor activity than conventional CAR T cells using the same antigen-binding domain, including in low antigen density settings that typically limit conventional CAR T efficacy.

ECT204 previously received FDA Orphan Drug Designation (ODD) in January 2022, reflecting the significant unmet need in advanced HCC. With RMAT designation in place, Eureka will work closely with the FDA to advance ECT204 through the ongoing ARYA-3 trial while continuing to build on the broader applicability of the ARTEMIS platform across different cancer indications.

About ECT204

ECT204 is an autologous T-cell therapy engineered with Eureka’s proprietary ARTEMIS CAR platform. Unlike conventional CAR T cells, ARTEMIS T cells are designed to mimic the natural biological regulation of T-cell activation, potentially improving safety, tumor infiltration, and persistence in solid tumors. ECT204 targets GPC3 (Glypican-3), an oncofetal antigen found on more than 70% of HCC cells but rarely on healthy tissues. The GPC3 protein is also expressed in other solid tumors, including ovarian and lung cancer. ECT204 is currently being investigated in Eureka’s ongoing ARYA-3 study, an open-label, dose escalation, multi-center Phase I/II clinical trial in adult patients with GPC3-positive HCC.

(Press release, Eureka Therapeutics, JAN 12, 2026, View Source [SID1234661999])

On January 12, 2026 Actuate Therapeutics, Inc. (NASDAQ: ACTU) ("Actuate" or the "Company"), a clinical-stage biopharmaceutical company focused on developing therapies for the treatment of high-impact, difficult-to-treat cancers through the inhibition of glycogen synthase kinase-3 beta (GSK-3β), reported that new additional and promising patient survival and biomarker data from treatment with elraglusib for metastatic pancreatic cancer were featured in oral and poster presentations at the 2026 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Gastrointestinal Cancers Symposium (ASCO GI) on January 9, 2026. The presentations highlighted new, expanded data from the Company’s Phase 2 clinical program (Actuate 1801-Part 3B) evaluating elraglusib in combination with gemcitabine/nab-paclitaxel (GnP), a first-line chemotherapy regimen for metastatic pancreatic cancer, providing additional evidence that suggests that elraglusib combined with GnP is associated with improved survival when compared with GnP alone.

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"Pancreatic cancer continues to have a high unmet medical need, particularly at the metastatic stage," said Dr. Devalingam Mahalingam, MD, PhD. "The results from this Phase 2 study suggest that adding elraglusib to standard gemcitabine and nab-paclitaxel chemotherapy may improve survival outcomes while maintaining a manageable safety profile. The scope of this trial, which enrolled 286 patients across 60 sites in six countries, underscores the robustness of the dataset. We are grateful to the patients and their families whose participation made this research possible."

"Metastatic pancreatic cancer remains one of the most difficult cancers to treat, with few effective first-line options," said Dan Schmitt, Chief Executive Officer of Actuate Therapeutics. "In this randomized Phase 2 study, the addition of elraglusib to gemcitabine and nab-paclitaxel resulted in a meaningful improvement in overall survival compared with chemotherapy alone. As of November 2025, seventeen patients are still alive in the elraglusib/GnP arm, and three of them have remarkably passed the 24-month mark on first-line treatment, while no patients remain on GnP treatment alone. Current patient survival for metastatic pancreatic cancer is less than 12 months, so our findings are especially encouraging. We look forward to building from this data and continuing to advance elraglusib for patients with this devastating disease."

Key Findings:

Efficacy: The primary efficacy endpoints were met in this study:

· The median overall survival (OS) showed significant benefit in the elraglusib/GnP vs GnP alone arms (mOS 10.1 months vs. 7.2 months, p=0.02, HR=0.62)
· 12-month survival rate doubled to 44.4% (elraglusib/GnP) vs 22.3% (GnP)
· 24-month survival rate increased fivefold to 12.9% in the elraglusib/GnP arm compared to 2.6% in the GnP arm, emphasizing the potential for long-term clinical benefit.

Safety: The safety and tolerability profile of elraglusib was consistent with previously reported data at the 9.3 mg/kg dose, and no new safety signals were identified:

· Serious TEAE were similar between the elraglusib/GnP (56.1%) and the GnP alone arm (56.4%),
· TEAE resulting in death were similar between treatment arms: elraglusib/GnP (12.3%) and GnP alone (16.7%)
· Grade-3 or higher TEAE leading to stoppage of any study drug: elraglusib/GnP (16.8%) and GnP alone (21.8%)

Genomic biomarkers: Several mutations commonly associated with pancreatic cancer were identified as potential predictive biomarkers of overall survival in the elraglusib/GnP arm, but not in the GnP-alone arm. Actionable mutations like KRAS or ARID1A could suggest novel drug combinations for future clinical studies.

Immunological biomarkers: The percentage of CD8+ and Granzyme B+ cells, as well as NK cells, was increased in tumors obtained from elraglusib/GnP-treated patients but not in GnP-treated patients, suggesting that combination therapy may potentiate improved anti-tumor immune response.

Oral Presentation

Title: Results from the randomized Phase 2 study (1801 Part 3B) of elraglusib plus gemcitabine/nab-paclitaxel (GnP) versus GnP in previously untreated metastatic pancreatic ductal adenocarcinoma (mPDAC).
Abstract: 653
Session: Rapid Oral Abstract Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract
Date/Time: January 9, 2026, 4:15 PM-5:00 PM (PST)
First Author: Devalingam Mahalingam, MD, PhD, Gastrointestinal Oncologist and Professor of Medicine at Northwestern University Feinberg School of Medicine

Poster Presentation

Title: Mutational analysis and identification of potential biomarkers in patients with metastatic pancreatic cancer treated with the combination of the GSK-3 inhibitor elraglusib and gemcitabine/nab-paclitaxel in the 1801 Part 3β Phase 2 study.
Abstract: 761
Poster Bd: L5
Session: Poster Session B: Cancers of the Pancreas, Small Bowel, and Hepatobiliary Tract
Date/Time: January 9, 2026, 11:30 AM-1:00 PM; 5:00 PM-6:00 PM (PST)
First Author: Andrey Ugolkov, MD, PhD, Senior Director, Clinical Science at Actuate Therapeutics

About Actuate-1801 Part 3B Study

The Actuate-1801 Part 3B study (NCT03678883) is a randomized, controlled Phase 2 trial of elraglusib with GnP versus GnP alone in first-line mPDAC. The trial enrolled 286 mPDAC patients with no prior systemic treatment for metastatic disease, who were randomized 2:1 to the elraglusib treatment arm (elraglusib/GnP) or the control arm (GnP alone). Elraglusib is administered at a dose of 9.3 mg/kg by IV infusion on Day 1 of each week of a 28-day cycle. The primary endpoint for this study is median overall survival, with OS summarized throughout the study by estimates of 1-year survival. Secondary endpoints are DCR, ORR, PFS, and AE.

(Press release, Actuate Therapeutics, JAN 12, 2026, View Source [SID1234661934])

On January 12, 2026 Relmada Therapeutics, Inc. (Nasdaq: RLMD, "Relmada" or the "Company"), a clinical-stage biotechnology company advancing innovative therapies for oncology and central nervous system disorders, reported that it has received written feedback from the U.S. Food and Drug Administration (FDA) regarding the registrational development pathway for NDV-01 in 2nd-line refractory, high-grade BCG-unresponsive non-muscle invasive bladder cancer (NMIBC) with carcinoma in situ (CIS) — one of the highest-risk and most treatment-resistant NMIBC populations — and in intermediate risk NMIBC in the adjuvant setting, where there are currently no approved therapies.

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In its written response to Relmada’s Type B pre-IND submission, the FDA indicated that a single-arm, open-label clinical trial in this high-grade, BCG-unresponsive with CIS population is an appropriate registrational approach for NDV-01. This feedback provides a clear and efficient development path toward a potential NDA submission for NDV-01 as a bladder-sparing therapeutic option in a patient population with significant unmet need.

The FDA also provided separate, supportive written feedback on the Company’s planned single registrational study in intermediate-risk NMIBC in the adjuvant setting, which is expected to follow an open-label, randomized-to-observation design.

Relmada continues to anticipate initiating both registrational trials in the first half of 2026.

"We are very pleased with the FDA’s alignment on the registrational design for NDV-01 in high-grade BCG-unresponsive NMIBC," said Raj S. Pruthi, MD, Chief Medical Officer – Oncology at Relmada Therapeutics. "A single-arm pivotal study in this setting represents a meaningful opportunity to advance an in-office, bladder-sparing therapy for patients who have few if any effective alternatives. This study represents the fastest path to approval for NDV-01."

Dr. Pruthi continued, "We are also encouraged by the FDA’s feedback on our intermediate-risk registration plans, where we believe NDV-01 could potentially provide meaningful clinical benefit to patients where no approved treatments currently exist."

About the Planned High-Grade Registrational Study

The planned pivotal Phase 3 study in 2nd-line, refractory, high-grade BCG-unresponsive NMIBC with carcinoma in situ (CIS) will be an open-label, single-arm trial evaluating:

Primary endpoint: Complete response (CR) rate at any time
Key secondary endpoint: Duration of response (DOR)
Assessments: Cystoscopy, cytology, and biopsy per protocol

The design reflects FDA’s written guidance on the study population, endpoint selection, and evaluation methodology and is consistent with prior FDA precedents for single-arm registrational trials in NMIBC.

About the Planned Intermediate-Risk Registrational Study

The planned pivotal Phase 3 study in intermediate-risk NMIBC in the adjuvant setting will be an open label randomized-to-observation study:

Primary endpoint: Disease Free Survival (DFS)
Key secondary endpoint: Duration of response (DOR)
Assessments: Cystoscopy, cytology, and biopsy per protocol

The design reflects FDA’s written guidance on the study population, endpoint selection, and evaluation methodology.

About NDV-01

NDV-01 is a sustained-release, intravesical formulation of gemcitabine and docetaxel (Gem/Doce), in development for the treatment of non-muscle invasive bladder cancer. It is designed to enable Gem/Doce bladder retention and gradual drug release over ten days. The formulation creates a soft matrix that enhances local exposure while minimizing systemic toxicity. The NDV-01 formulation is a ready to use, convenient to administer in-office in less than five minutes, and does not require anesthesia or specialized equipment. It is protected by patents through 2038.

About NMIBC

NMIBC represents 75-80% of all bladder cancer cases and is associated with high recurrence (50 – 80% over 5 years). With over 744,000 prevalent cases in the U.S. and limited treatment options, the market opportunity is significant. High-grade BCG-unresponsive disease represents one of the most difficult-to-treat NMIBC subtypes, with limited bladder-sparing options. Intermediate-risk NMIBC in the adjuvant setting has no currently approved therapies. NDV-01 has the potential to serve as a frontline or salvage therapy and could be applicable across multiple NMIBC subtypes.

On January 12, 2026 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on developing innovative CAR T-cell therapies, and Dispatch Bio, a biotech company engineering a universal treatment across solid tumors leveraging its first-in-class Flare platform, reported a clinical collaboration to conduct a Phase 1 trial in China, planned to begin in 2026.

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The trial will evaluate DISP-11, an investigational therapy leveraging Dispatch’s first-in-class Flare platform – including DV-10, the company’s novel tumor-specific virus – and CARsgen’s zevorcabtagene autoleucel (zevor-cel, R&D code: CT053), an autologous B-cell maturation antigen (BCMA)-targeting CAR T-cell therapy, in patients suffering from solid tumors. Zevor-cel is approved by the National Medical Products Administration (NMPA) in China for the treatment of multiple myeloma.

"CARsgen is working to transform outcomes for patients with solid tumors by advancing the potential of CAR T technologies," said Zonghai Li, M.D., Ph.D., Founder, Chairman of the Board, Chief Executive Officer and Chief Scientific Officer of CARsgen. "Dispatch’s Flare platform offers a differentiated and highly complementary approach to expanding where and how CAR T can be applied, particularly for solid tumors lacking specific targets. We are excited to explore this combination as part of our broader efforts to unlock meaningful benefit for patients."

In the planned study, patients with solid tumors of epithelial origin, which account for 90% of all solid tumors, will first receive DV-10, followed by zevor-cel.

"We are working to expand the impact of the Flare platform, and this collaboration represents a step forward in a region with infrastructure for efficient and impactful oncology development and significant unmet medical need," said Sabah Oney, Ph.D., Chief Executive Officer of Dispatch. "CARsgen’s scientific rigor and track record of rapid clinical advancement make them an ideal partner. As our second collaboration with a company that has successfully commercialized a CAR T therapy, this partnership reinforces the growing confidence in Flare’s potential across a variety of immunotherapies and will help shape our global development strategy."

About Zevor-cel

Zevor-cel is a fully human, autologous BCMA CAR T-cell product for the treatment of multiple myeloma (MM). Zevor-cel was approved by the NMPA on February 23, 2024, for the treatment of adult patients with relapsed or refractory MM who have progressed after at least three prior lines of therapy (including a proteasome inhibitor and an immunomodulatory agent). Zevor-cel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. FDA in 2019.

(Press release, Carsgen Therapeutics, JAN 12, 2026, View Source [SID1234661985])

On January 12, 2026 ConcertAI, a leading oncology real-world evidence data and AI SaaS technology company, and Foundation Medicine, a global, patient-focused precision medicine company, reported a collaboration to combine their data assets for life sciences research. The collaboration brings Foundation Medicine’s expansive de-identified multimodal dataset derived from its genomic testing portfolio together with ConcertAI’s high-quality clinical data, to support more efficient drug development and real-world evidence research. With the addition of Foundation Medicine’s data, ConcertAI now offers the largest and most comprehensive clinically-linked dataset with nearly half a million patients.

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The collaboration comes as oncology drug programs face rising trial complexity and tighter budgets, which puts more weight on high-quality data that can guide early decisions. Pairing Foundation Medicine and ConcertAI’s data captures the full journey of cancer patients from pre-diagnosis through treatment and outcomes, allowing life science researchers and biopharmaceutical developers to reduce guesswork in early research, tighten trial plans, and bring new therapies to patients faster, while ensuring the rigor of their research.

"Drug developers are increasingly pushed to accelerate their research pipelines but require more robust insights into both early genomic signals and patient outcomes to make this a reality," said Eron Kelly, CEO of ConcertAI. "By combining the largest and deepest set of clinical data with genomic insights, including whole slide imaging, we can help teams set a clearer plan and speed up their projects. Our collaboration with Foundation Medicine complements our full suite of partnerships aimed at advancing the development of vital therapies."

Foundation Medicine has U.S Food and Drug Administration-approved companion diagnostic indications for all four major classes of genomic alterations and for multiple genomic signatures. Detection of these major classes of alterations provide partners with a comprehensive view of their cohort’s genomic landscape, including difficult-to-detect alterations, such as MTAP loss, that are not detected well by all next-generation sequencing tests.1

In addition to genomic data, the combined dataset includes gene expression data, immunohistochemistry (IHC) results, and whole-slide images, alongside one of the most diverse clinical datasets from ConcertAI, whether measured by socioeconomic diversity, ethnic diversity, or rural vs. urban communities. By connecting these views, researchers can better study how cancers act in real life and how patients respond to treatments over time.

"By integrating our high-quality genomic data with ConcertAl’s electronic health data, we have created one of the most powerful new real-world data sets that enables insights to be turned into strategic decisions at critical milestones for our partners," said Dan Malarek, CEO of Foundation Medicine. "AI is the future and through our Al-driven analytical capabilities via FoundationInsights, our biopharmaceutical and research partners can access the right data when they need it and uncover answers faster. This accelerates development and helps patients benefit sooner from advances in precision medicine."

(Press release, Foundation Medicine, JAN 12, 2026, View Source [SID1234662000])