On April 30, 2025 Genocury Biotech reported groundbreaking clinical data from its noval in vivo CD19 CAR-T therapy in relapsed/refractory diffuse large B-cell lymphoma (R/R DLBCL), a notoriously aggressive blood cancer (Press release, Shenzhen Genocury Biotech, APR 30, 2025, View Source [SID1234652399]). In an investigator-initiated trial (IIT) led by the Hematology Department at Tongji Hospital, a heavily pretreated relapsed/refractory (R/R) patient with advanced DLBCL achieved complete remission (CR) after 1 month of the in vivo CAR-T treatment, with durable response sustained over three months. Notably, the treatment eliminated the need for lymphodepletion, a standard yet toxic preconditioning step in traditional CAR-T protocols.

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Clinical Breakthrough: Redefining Safety, Efficacy, and Speed

Key trial findings:

1. Rapid and Deep Response:

A relapsed DLBCL male patient achieved complete hematological remission (CR) within 28 days following a single dose of Genocury’s CD19 in vivo CAR-T without lymphodepletion. Flow cytometric analysis revealed significant CAR-T cell expansion kinetics, with sustained therapeutic efficacy confirmed through 90-day follow-up monitoring.

2. Unprecedented Safety Profile:

Unlike conventional CAR-T, which carries a ~50% risk of cytokine release syndrome (CRS) and neurotoxicity, this novel therapeutic approach demonstrated complete absence of:

Cytokine release syndrome (CRS)
Immune effector cell-associated neurotoxicity syndrome (ICANS)
Lymphodepletion-related complications – typically associated with increased infection risk- further underscores the therapy’s safety profile.
Reimagining CAR-T: In Vivo Engineering Breakthroughs

Genocury’s in vivo CAR-T platform overcomes two critical roadblocks in current CAR-T therapy:

Eliminates Ex Vivo Manipulation: Traditional CAR-T requires 3-4 week vein-to-vein timeline by harvesting, modifying, and expanding a patient’s T cells outside the body, and $400K price tag stems from this time-consuming process. Genocury’s proprietary in vivo CAR-T vector delivers CAR payload directly into circulating T cells in vivo, enabling functional CAR-T generation.
Lymphodepletion-Free Protocol: Current protocols require the harsh preconditioning chemotherapy for immune reset – a leading cause of hospitalization. Genocury’s lymphodepletion-free approach reduces treatment-related complications and logistical burdens, as well as enabling significant CAR-T cell expansion.
Statement from Prof. Jia Wei , M.D., Ph.D.

PI (principal investigator), Tongji Hospital Hematology Department (Wuhan)
"In this groundbreaking case, we observed the patient treated with Genocury’s CD19 in vivo CAR-T achieved complete remission through 90-day follow-up – achieved without any lymphodepletion, which fundamentally challenges current cellular therapy dogma" said Dr. Jia Wei, "This therapy combines the benefits of autologous CAR-T with the accessibility of universal therapies, potentially ending the era of unaffordable cancer treatments. This could democratize access to CAR-T globally, we are very excited to advance this paradigm-shifting approach."

On April 30, 2025 Endevica Bio, a privately held company developing first-in-class peptide drug candidates, reported the dose administration for the first patient in a Phase 2 trial for its experimental drug TCMCB07 (B07) to prevent weight loss in cancer patients undergoing chemotherapy (Press release, Endevica Bio, APR 30, 2025, View Source [SID1234652383]).

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The trial, being conducted in partnership with WuXi Clinical, will include 20 sites and 100 patients who are diagnosed with stage 4 metastatic colorectal cancer.

In the trial, patients are dosed with B07 as they begin chemotherapy and during the first several rounds of chemotherapy. The primary endpoint is preventing weight loss, which can lead to a debilitating condition called cachexia, a life-threatening wasting syndrome associated with chronic diseases, including cancer.

"This marks an important milestone in our commitment in developing a potentially life changing treatment for cachexia," said Russell Potterfield, Chief Executive Officer and Executive Chair of Endevica. "Each trial brings us closer to offering a viable solution for this debilitating disease, and we remain dedicated to making a lasting impact on the lives of those affected."

"We are incredibly excited to have our first patient dosed with B07 in individuals diagnosed with metastatic colorectal cancer who are undergoing chemotherapy," said Dr. Daniel Marks, Chief Medical and Scientific Officer of Endevica Bio. "Since there is no FDA approved therapeutic for cancer cachexia, this trial is a crucial step to provide a therapy for an area of huge unmet clinical need, and we look forward to the results."

In 2024, Endevica Bio completed its Phase 1 clinical trial with preliminary findings supporting its strong safety and efficacy. Last November, the Journal of Clinical Investigation, showed that B07 improved the appetite and preserved lean mass and fat mass in rodent models of cancer and its associated combination chemotherapy. This same study showed the strong potential of B07 to alleviate chemotherapy-induced anorexia and weight loss for millions of patients worldwide.

About TCMCB07
TCMCB07 is a melanocortin‐3/4 antagonist peptide candidate in clinical development for the treatment of cachexia. It is designed to be a first-in-class peptide drug with the ability to cross the blood-brain barrier and act on previously inaccessible target receptors to modulate the body’s behavioral and metabolic response to chronic illness. Pre-clinical animal trial results show significant lean muscle mass retention (e.g., a reversal of the cachectic condition) during administration of the drug. The results are consistent in cachexia arising from many different types of chronic disease.

On April 30, 2025 J INTS BIO, a company specializing in anticancer and orphan drugs, reported its research findings for the next-generation EGFR-TKI therapeutic ‘JIN-A02’ (clinical Phase 1/2 results) and the ovarian cancer treatment candidate ‘JIN-001’ (preclinical results) at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) 2025, held in Chicago from April 25 to 30 (Press release, J INTS BIO, APR 30, 2025, View Source [SID1234652400]). In this presentation, J INTS BIO demonstrated its innovative drug development capabilities on a global stage, attracting significant attention from both academia and the industry.

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‘JIN-A02’: New Hope for Overcoming Resistance – 4th Generation EGFR-TKI Clinical Results

Non-small cell lung cancer (NSCLC) accounts for approximately 85% of all lung cancer cases worldwide, and among these, EGFR mutations are considered a major therapeutic target. JIN-A02, introduced at AACR (Free AACR Whitepaper) 2025, showed outstanding therapeutic efficacy in patients who developed resistance to existing third-generation EGFR-TKIs. In particular, JIN-A02 demonstrated remarkable antitumor activity in patients with EGFR C797S mutation—currently a mutation for which no approved targeted therapies exist—thus opening a new avenue for treatment options.

In the clinical trial, a patient in the 300 mg cohort showed complete clearance of circulating tumor DNA (ctDNA) harboring either the C797S or Ex19del mutation, and ctDNA carrying the T790M mutation was reduced by over 90%. Clinically, a partial response (PR) was observed, with tumor size reductions of up to 39.7%, and notable shrinkage of intracranial metastatic lesions. These findings are particularly important for NSCLC patients, in whom brain metastases are common. Furthermore, no dose-limiting toxicities (DLTs) or serious adverse events were observed up to the 300 mg dose, highlighting the excellent safety profile of JIN-A02. This safety profile is expected to be particularly beneficial for patients requiring long-term treatment and combination therapies.

Currently, JIN-A02 is undergoing multinational clinical trials in Korea, the United States, Thailand, and other countries, and it is being closely watched as a potential groundbreaking treatment option for EGFR-mutant NSCLC patients.

‘JIN-001’: An Innovative New Drug Targeting Refractory Ovarian Cancer

Also presented was ‘JIN-001,’ J INTS BIO’s second-generation synthetic HSP90 inhibitor, developed as a new strategy to overcome resistance to existing ovarian cancer treatments. Ovarian cancer remains one of the deadliest gynecological malignancies, as it is often diagnosed at an advanced stage when effective treatment options are extremely limited.

The preclinical study demonstrated that JIN-001, when combined with the chemotherapeutic agent cisplatin, significantly enhanced tumor suppression compared to cisplatin alone. Notably, even at low concentrations (≤0.1 μM), JIN-001 enhanced the antitumor activity of cisplatin against both cisplatin-sensitive and cisplatin-resistant ovarian cancer cells, compared to cisplatin alone. Moreover, the combination therapy significantly reduced the expression of key signaling proteins related to cisplatin resistance, contributing to a sustained antitumor effect.

Based on these promising results, J INTS BIO is planning additional preclinical studies and aims to rapidly advance JIN-001 into clinical trials, with the goal of establishing a new standard of care for the treatment of refractory and multidrug-resistant ovarian cancer.

Led by the presentation at AACR (Free AACR Whitepaper) 2025, J INTS BIO aims to further strengthen its competitiveness in the global oncology market and solidify its position as a frontrunner in next-generation anticancer drug development. The company also outlined its vision to continue delivering innovative therapies through ongoing research and development efforts, offering new hope to cancer patients worldwide.

On April 30, 2025 FORE Biotherapeutics, a registration stage biotherapeutics company dedicated to developing targeted therapies to treat patients with cancer, reported that a plixorafenib abstract has been selected for poster presentation at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (ASCO) (Free ASCO Whitepaper), taking place May 30-June 3, 2025 in Chicago (Press release, Fore Biotherapeutics, APR 30, 2025, View Source [SID1234652384]).

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At ASCO (Free ASCO Whitepaper) 2025, Karisa Schreck, M.D., Ph.D., from the Sidney Kimmel Comprehensive Cancer Center at Johns Hopkins University, will present a trials-in-progress poster highlighting the study design of the global registration-intended FORTE Master Protocol, which includes four sub-protocol baskets evaluating plixorafenib in distinct patient populations. The three monotherapy indications currently under evaluation are BRAF V600 Recurrent Primary Central Nervous System Tumors, Rare BRAF V600 Mutated Solid Tumors and Solid Tumors with BRAF Fusions.

Poster Presentation Details:

Title: FORTE: A phase 2 master protocol assessing plixorafenib for BRAF-altered cancers
Poster Session: Central Nervous System Tumors
Date and Time: Saturday, May 31, 2025, 9:00 a.m. – 12:00 p.m. CT
Abstract Number: TPS2091
Presenter: Karisa Schreck, M.D., Ph.D., Johns Hopkins University

On April 30, 2025 Ingenium Therapeutics, a clinical-stage biotechnology company advancing allogeneic natural killer (NK) cell therapies, reported that it has received positive regulatory feedback from the U.S. Food and Drug Administration (FDA) following a Pre-Investigational New Drug (Pre-IND) meeting (Press release, Ingenium Therapeutics, APR 30, 2025, View Source [SID1234652401]).

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Ingenium has conducted multiple investigator-initiated trials in South Korea, enrolling over 140 patients with acute myeloid leukemia (AML), including a Phase 2 randomized clinical trial with long-term overall survival follow-up. These studies have generated robust non-clinical and clinical data supporting the safety and efficacy profile of Gengleucel.

Based on its review of these data, the FDA has provided positive feedback on Ingenium’s proposal to initiate a Phase 2 trial of Gengleucel in the United States without a preceding Phase 1 study. This regulatory milestone may streamline Gengleucel’s clinical development pathway and may accelerate access for patients with measurable residual disease-positive (MRD+) AML.

The planned multicenter study will be conducted at leading cancer centers across the United States, including a world-renowned institution in Texas. Gengleucel is positioned to become the first NK cell therapy in AML to use MRD negativity as a primary endpoint, underscoring its potential to improve outcomes by eradicating minimal residual disease and reducing relapse risk.

"We are thrilled with the FDA’s guidance, which reflects the rigor of our clinical research and the significant therapeutic potential of Gengleucel, especially in targeting minimal residual disease," said Kevin Koh, CEO of Ingenium Therapeutics. "Launching our U.S. Phase 2 trial at premier cancer centers marks a major milestone in our mission to deliver novel immunotherapies to AML patients with MRD."

The trial is expected to begin in early 2026 and will evaluate Gengleucel’s ability to achieve MRD negativity and reduce relapse and mortality. To ensure reliable clinical operations and product supply, Ingenium is transferring its manufacturing technology to a Texas-based manufacturing facility.

About Gengleucel

Gengleucel is a non-engineered, allogeneic NK cell therapy composed of memory NK cells with enhanced activating receptor expression and increased secretion of IFN-gamma, granzyme, and perforin. These features drive potent anti-cancer activity and sustained in vivo persistence. Clinical trials have demonstrated Gengleucel’s favorable safety, tolerability, and efficacy in high-risk AML and MDS populations. Gengleucel has been granted Orphan Drug Designation by the U.S. FDA for the treatment of AML.