On March 4, 2026 Keros Therapeutics presented its corporate presentation.

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(Presentation, Keros Therapeutics, MAR 4, 2026, View Source [SID1234663248])

On March 4, 2026 INOVIO (NASDAQ: INO), and Akeso, Inc. (9926.HK) ("Akeso") reported that they have entered into a clinical trial collaboration and supply agreement to evaluate INO-5412, INOVIO’s DNA immunotherapy candidate, in combination with cadonilimab, Akeso’s first-in-class PD-1/CTLA-4 bispecific antibody, for the potential treatment of GBM. The combination therapy will be studied as a part of INSIGhT, the innovative Phase 2 adaptive platform trial sponsored by the Dana-Farber Cancer Institute and conducted with Mass General Brigham Cancer Care, Inc., which is designed to quickly and efficiently find new treatments for GBM. Dosing in the combination therapy trial is expected to begin in the second half of 2026.

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INO-5412 is composed of INO-5401 and T cell immune activator INO-9012. When combined with a checkpoint blockade, targeted DNA immunotherapy has the potential to overcome the challenges of immune checkpoint therapy alone by stimulating an immune response against tumor antigens and driving T cell infiltration into the glioblastoma tumor microenvironment. In an ongoing Phase 2 trial in newly diagnosed GBM patients, INO-5401 plus INO-9012 in combination with a PD-1 checkpoint inhibitor elicited robust immune responses that potentially correlate with enhanced survival. The novel combination of INO-5412 with cadonilimab to treat GBM builds on this promising data and could potentially benefit patients by providing additional checkpoint inhibition through CTLA-4 binding.

Cadonilimab has received marketing approval in China for several indications, including first-line gastric cancer, first-line cervical cancer, and second/third-line cervical cancer, demonstrating effectiveness irrespective of PD-L1 expression status. As the world’s first approved bispecific antibody for cancer immunotherapy developed in China, its clinical value is well-established through real-world application and validation across multiple Phase 3 trials. The drug is currently involved in over 11 Phase 3/registration clinical studies. Among these are two international multicenter trials: a Phase 3 registrational trial for the first-line treatment of gastric cancer, and a Phase 2 registrational trial for the second-line treatment of liver cancer in patients who exhibit resistance to immune checkpoint inhibitors (IO).

Dr. David Reardon, Director of the Center for Neuro-Oncology at the Dana-Farber Cancer Institute and a professor at Harvard Medical School said, "The INSIGhT trial was designed to help quickly advance cutting-edge treatments for GBM, the most common and aggressive form of brain cancer for which there are few effective treatments currently available or in development. We are excited to include INOVIO and Akeso’s novel combination immunotherapy in the trial and welcome their efforts to help improve potential outcomes for patients."

Dr. Michael Sumner, INOVIO’s Chief Medical Officer, said, "This collaboration is an important step forward for our cancer immunotherapy research and we are delighted to partner with two trailblazing organizations to advance this promising candidate in our late-stage clinical pipeline. Combining INO-5412 with Akeso’s novel checkpoint modality represents an important evolution of our research in GBM, builds on our previous data showing the potential to improve patient outcomes and highlights our ongoing commitment to advancing innovative treatments for diseases with significant unmet need."

Yu (Michelle) Xia, PhD, founder, chairwoman, president, and Chief Executive Officer of Akeso, said, "We are truly excited to collaborate with INOVIO for the treatment of GBM. We are advancing cadonilimab worldwide through Akeso’s ‘in-house innovation + global partnership’ strategy to realize its breakthrough clinical benefits for patients all around the world across multiple cancer types. By collaborating with INOVIO, we aim to harness the benefit of combining INOVIO’s DNA medicine with cadonilimab’s dual checkpoint inhibition for the treatment of GBM, a particularly challenging central nervous system malignancy. We also look forward to working with one of the world’s leading cancer centers in the clinical development of the new cadonilimab and INO-5412 combination treatment for GBM."

Under the terms of the agreement, INOVIO and Akeso will provide support for the INSIGhT study, including supplying their respective therapeutic products, while the investigative sponsors will oversee the day-to-day clinical operations.

About INSIGhT
The INdividualized Screening trial of Innovative Glioblastoma Therapy (INSIGhT) is investigating novel treatments in patients with newly diagnosed GBM. INSIGhT uses a shared control arm to test multiple investigational therapies at one time, with each arm independently compared only to the control. The arm of the trial that involves the collaboration between INOVIO and Akeso will investigate the safety and efficacy of INO-5412 in combination with cadonilimab in patients with histologically confirmed intracranial glioblastoma or gliosarcoma. Dosing is expected to begin in the second half of 2026. For more information about the clinical study, see www.clinicaltrials.gov, identifier NCT02977780.

About GBM
Glioblastoma (GBM) is the most common and aggressive type of brain cancer and remains a devastating disease for both patients and caregivers. Its prognosis is extremely poor, with few therapies approved over the last 10 years. The median overall survival for patients receiving standard of care therapy is approximately 15 months in patients with an unmethylated MGMT (O-6-methylguanine-DNA methyltransferase) promoter and the median progression-free survival is approximately 4 to 6 months. The estimated annual incidence of GBM in the U.S. is 11,362 cases or 3.21 cases per 100,000 persons and the median age at diagnosis is 65 years.

About INO-5412
INO-5412 is an investigational DNA medicine that combines INO-5401 and INO-9012 into a single vial as a potentially powerful cancer immunotherapy particularly when given in combination with checkpoint inhibitors. INO-5401 plus INO-9012 has been previously investigated as a potential therapeutic treatment targeting a number of cancers, including GBM and cancers exhibiting BRCA1 and BRCA2 mutations. Data from an ongoing Phase 2 trial in newly diagnosed GBM patients, highlighted in a 2022 oral presentation at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper), demonstrated that INO-5401 plus INO-9012 in combination with a PD-1 checkpoint inhibitor elicited robust immune responses that potentially correlated with enhanced survival. (Reardon D. et al, ASCO (Free ASCO Whitepaper) 2022).

INO-5401 encodes for INOVIO’s SynCon antigens for hTERT, WT1, and PSMA, which are antigens The National Cancer Institute has highlighted as important targets and designated as high priorities for cancer immunotherapy development. These three antigens have been reported to be over-expressed and often mutated in a variety of human cancers, including GBM. INO-9012 encodes for IL-12, which is a T cell immune activator.

About Cadonilimab
Cadonilimab, independently developed by Akeso, is the world’s first PD-1/CTLA-4 bispecific immuno-oncology therapy. It has been approved and reimbursed in China for three indications: recurrent/metastatic cervical cancer after platinum-based chemotherapy failure, first-line gastric cancer, and first-line cervical cancer. The drug has also been investigated in over 30 clinical trials spanning more than 20 tumor types. Currently, 11 Phase III or registrational trials are ongoing, with three already having met primary endpoints. With a novel dual-targeting mechanism against PD-1 and CTLA-4, cadonilimab demonstrates superior efficacy and a favorable safety profile compared to combination therapies. It has shown strong anti-tumor activity regardless of PD-L1 status and holds breakthrough potential in IO-resistant and immunologically "cold" tumors.

(Press release, Inovio, MAR 4, 2026, View Source [SID1234663247])

On March 4, 2026 HUTCHMED (China) Limited ("HUTCHMED") (Nasdaq/AIM:HCM; HKEX:13) reported that it has initiated a Phase I/IIa clinical trial of HMPL-A580, HUTCHMED’s second novel Antibody-Targeted Therapy Conjugate ("ATTC"), in patients with unresectable, advanced or metastatic solid tumors in China and the US. The first patient received the first dose on March 4, 2026.

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HMPL-A580 is a first-in-class ATTC comprising a highly selective and potent PI3K/PIKK small-molecule inhibitor payload linked to an anti-EGFR antibody via a cleavable linker, HUTCHMED’s second ATTC based on this highly novel PI3K/PIKK inhibitor payload. EGFR is highly expressed in multiple types of solid tumors and is well recognized as a driving force in tumorigenesis and disease progression. Preclinical data have shown that PAM pathway inhibition synergizes with anti-EGFR therapy to enhance anti-tumor activity, and will be presented at an upcoming scientific conference.

This first-in-human Phase I/IIa, multicenter, open-label study evaluates the safety, tolerability, pharmacokinetics, immunogenicity and preliminary efficacy of HMPL-A580. The study consists of two parts. In the Phase I dose escalation part, patients will receive HMPL-A580 intravenously at predefined dose levels to determine the maximum tolerated dose and recommended dose for expansion. The subsequent Phase IIa dose expansion/​optimization part is to further characterize the safety, tolerability and preliminary anti-tumor activity of HMPL-A580 in selected solid tumors, and to determine the recommended dose for the next phase. Additional details may be found at clinicaltrials.gov, using identifier NCT07396584.

About the ATTC Platform

HUTCHMED’s ATTC platform represents a next-generation approach to precision oncology, combining monoclonal antibodies with proprietary small-molecule inhibitor payloads to deliver dual mechanisms of action. Unlike traditional cytotoxin-based Antibody Drug Conjugates, ATTCs combine targeted therapies to achieve synergistic anti-tumor activity and durable responses in preclinical models, outperforming standalone antibody or small-molecule inhibitor components in efficacy and safety.

Built on over 20 years of targeted therapy expertise, the platform enables development of drug candidates for diverse cancer types. By leveraging antibody-guided delivery and tumor-specific payload release, ATTCs improve the accessibility to tumors and reduce off-tumor toxicity. This overcomes challenges of traditional small-molecule inhibitors, ensures safer long-term use, and supports combinations with chemotherapy and immunotherapy, unlocking potential for early-line treatments.

HUTCHMED has demonstrated how its partnerships leverage the expertise of multinational pharmaceutical companies to accelerate bringing novel medicines to address large unmet needs around the world, and plans to apply this strategy to its ATTC technology this year.

About the PAM Pathway and HMPL-A580

The PI3K/AKT/mTOR ("PAM") pathway is a critical intracellular network involved in cell growth, survival, and division. Alterations in the PAM pathway are frequently associated with poor prognosis and resistance to treatment across various cancers. However, existing PAM-targeted drugs face significant challenges, including on-target toxicities that restrict dosing, feedback loops that enable pathway reactivation, and insufficient tumor-specific delivery.

By conjugating this highly novel payload to an anti-EGFR antibody, HMPL-A580 is designed to deliver targeted pathway inhibition directly into EGFR-expressing tumor cells, thereby potentially overcoming the systemic toxicity and narrow therapeutic index historically associated with PI3K/PIKK inhibitors. This approach aims to achieve deeper and more durable target inhibition while improving the overall tolerability profile.

(Press release, Hutchison China MediTech, MAR 4, 2026, View Source [SID1234663246])

On March 4, 2026 Fennec Pharmaceuticals Inc. (NASDAQ:FENC; TSX: FRX), a specialty pharmaceutical company, reported that the Tampa General Hospital (TGH) Cancer Institute is initiating a study evaluating the real-world clinical utility of sodium thiosulfate injection (PEDMARK) in reducing the risk of ototoxicity in Adolescent and Young Adult (AYA) and adult cancer patients receiving cisplatin-based treatment.

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"The TGH Cancer Institute, in collaboration with the USF Health Morsani College of Medicine, is committed to better understanding and advancing otoprotective strategies aimed at preserving auditory function in patients receiving cisplatin chemotherapy," said Gene A. Wetzstein, PharmD, BCOP, Director of Supportive Care Research & Scientific Affairs at the TGH Cancer Institute and principal investigator of the initiative. "This evaluation will examine real-world clinical data and audiology monitoring that will help inform future clinical research and quality efforts in assessing, preventing and managing ototoxicity."

PEDMARK is currently approved for pediatric patients one month of age and older with localized non-metastatic solid tumors and is also recognized by the National Comprehensive Cancer Network with a 2A recommendation for use in AYA patients.

"Real-world evidence plays a critical role in demonstrating the clinical utility of PEDMARK across diverse patient populations and tumor types," said Pierre S. Sayad, PhD, M.S., chief medical officer of Fennec Pharmaceuticals. "As more institutions generate data on its use outside of controlled trials, clinicians will gain a clearer picture of the important role of PEDMARK in providing hearing loss protection for patients receiving cisplatin."

The following sections provide general background information and labeling approved by the U.S. Food and Drug Administration for PEDMARK and are not intended to solicit participation in any research activity.

About Cisplatin-Induced Ototoxicity

Cisplatin and other platinum-based chemotherapies are widely used to treat solid tumors and have been vital in improving survival rates. Unfortunately, these life-saving treatments often result in permanent, irreversible hearing loss, also known as ototoxicity.i

Hearing loss from cisplatin treatment is not rare. Studies show that between 60-90% of patients treated with cisplatin may develop hearing loss, depending upon the dose and duration of chemotherapyii. Many of those treated with cisplatin will require lifelong hearing aids or cochlear implants, which can be helpful for some, but do not reverse the hearing loss and can be costly over time.iii Treatment-induced hearing loss can reduce quality of survivorship as it impacts many aspects of life, such as speech and language skills, academic performance, social-emotional development, career potential and the ability to live independently.iv,v While audiologic monitoring is recommended to help manage ototoxicity, it is currently underutilized in certain cancer patient populations.

PEDMARK (sodium thiosulfate injection)

PEDMARK is the first and only U.S. Food and Drug Administration (FDA) approved therapy indicated to reduce the risk of ototoxicity associated with cisplatin treatment in pediatric patients 1 month of age and older with localized, non-metastatic, solid tumors. It is a unique formulation of sodium thiosulfate in single-dose, ready-to-use vials for intravenous use in pediatric patients. PEDMARK is also the first and only therapeutic agent with proven efficacy and safety data with an established dosing regimen, across two open-label, randomized Phase 3 clinical studies, the Children’s Oncology Group (COG) Protocol ACCL0431 and SIOPEL 6.

Additionally, PEDMARK is recommended for the adolescent and young adult (AYA) population by the National Comprehensive Cancer Network, or NCCN, with a 2A endorsement.

Approximately 500,000 patients in the U.S. are diagnosed annually with cancers that could be treated with a platinum-based chemotherapy.vi,vii The incidence of ototoxicity depends upon the dose and duration of chemotherapy, and many of those treated will require lifelong hearing aids. Until the FDA approval of PEDMARK, there were no preventative agents for this hearing loss. Patients with hearing loss resulting from cancer treatment have a statistically significant worse quality of life compared with peers who have no hearing loss.viii,ix

PEDMARK has been studied by co-operative groups in two Phase 3 clinical studies of survival and reduction of ototoxicity, COG ACCL0431 and SIOPEL 6. Both studies have been completed. The COG ACCL0431 protocol enrolled childhood cancers typically treated with intensive cisplatin therapy for localized and disseminated disease, including newly diagnosed hepatoblastoma, germ cell tumor, osteosarcoma, neuroblastoma, medulloblastoma, and other solid tumors. SIOPEL 6 enrolled only hepatoblastoma patients with localized tumors.

Indications and Usage

PEDMARK (sodium thiosulfate injection) is indicated to reduce the risk of ototoxicity associated with cisplatin in pediatric patients 1 month of age and older with localized, non-metastatic solid tumors.

Limitations of Use

The safety and efficacy of PEDMARK have not been established when administered following cisplatin infusions longer than 6 hours. PEDMARK may not reduce the risk of ototoxicity when administered following longer cisplatin infusions, because irreversible ototoxicity may have already occurred.

Important Safety Information

PEDMARK is contraindicated in patients with history of a severe hypersensitivity to sodium thiosulfate or any of its components.

Hypersensitivity reactions occurred in 8% to 13% of patients in clinical trials. Monitor patients for hypersensitivity reactions. Immediately discontinue PEDMARK and institute appropriate care if a hypersensitivity reaction occurs. Administer antihistamines or glucocorticoids (if appropriate) before each subsequent administration of PEDMARK. PEDMARK may contain sodium sulfite; patients with sulfite sensitivity may have hypersensitivity reactions, including anaphylactic symptoms and life-threatening or severe asthma episodes. Sulfite sensitivity is seen more frequently in people with asthma.

PEDMARK is not indicated for use in pediatric patients less than 1 month of age due to the increased risk of hypernatremia or in pediatric patients with metastatic cancers.

Hypernatremia occurred in 12% to 26% of patients in clinical trials, including a single Grade 3 case. Hypokalemia occurred in 15% to 27% of patients in clinical trials, with Grade 3 or 4 occurring in 9% to 27% of patients. Monitor serum sodium and potassium levels at baseline and as clinically indicated. Withhold PEDMARK in patients with baseline serum sodium greater than 145 mmol/L.

Monitor for signs and symptoms of hypernatremia and hypokalemia more closely if the glomerular filtration rate (GFR) falls below 60 mL/min/1.73m2.

Administer antiemetics prior to each PEDMARK administration. Provide additional antiemetics and supportive care as appropriate.

The most common adverse reactions (≥25% with difference between arms of >5% compared to cisplatin alone) in SIOPEL 6 were vomiting, nausea, decreased hemoglobin, and hypernatremia. The most common adverse reaction (≥25% with difference between arms of >5% compared to cisplatin alone) in COG ACCL0431 was hypokalemia.

Please see full Prescribing Information for PEDMARK at: www.PEDMARK.com.

(Press release, Fennec Pharmaceuticals, MAR 4, 2026, View Source [SID1234663245])

On March 4, 2026 Citius Oncology, Inc. ("Citius Oncology") (Nasdaq: CTOR), an oncology-focused biopharmaceutical company and majority-owned subsidiary of Citius Pharmaceuticals, Inc. ("Citius Pharma") (Nasdaq: CTXR), reported positive topline safety and efficacy results from an investigator-initiated Phase 1 trial evaluating LYMPHIR (E7777, denileukin diftitox-cxdl) administered prior to commercial CD19-directed CAR-T therapy in patients with high-risk relapsed or refractory diffuse large B-cell lymphoma (DLBCL). The trial was conducted by lead investigator, Dr. Veronika Bachanova, at the University of Minnesota and City of Hope. Full results were presented at the 2026 ASTCT & CIBMTR Tandem Meetings1.

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The Phase 1 trial was designed to augment the lymphodepletion regimen prior to CAR-T infusion through the administration of LYMPHIR to potentially improve the anti-tumor activity of CAR-T therapies. LYMPHIR, an engineered fusion toxin that preferentially binds to the IL-2 receptor expressed on regulatory T-cells (Tregs), is currently FDA-approved and commercially available for the treatment of relapsed or refractory cutaneous T-cell lymphoma (CTCL) after one prior systemic therapy.

"Enhancing Treg depletion prior to CAR-T infusion with LYMPHIR represents a promising immunomodulatory strategy in patients with high-risk DLBCL, and these Phase 1 data provide an encouraging signal of the potential to enhance current CAR-T regimens," said Dr. Myron Czuczman, Executive Vice President and Chief Medical Officer of Citius Oncology and Citius Pharma. "These positive data support our broader strategy of exploring LYMPHIR’s modulatory effect on Tregs in combination with other approved therapies to potentially enhance the body’s own immune system to fight cancerous tumors," he added.

Topline Results & Study Design

● All patients (n=14) completed treatment and proceeded to CAR-T infusion;

● LYMPHIR was well tolerated, with no dose-limiting toxicities observed;

● No Grade ≥3 LYMPHIR-related immune adverse events or infusion reactions were reported; and,

● Data demonstrated effective Treg depletion, and promising efficacy signals of enhanced standard lymphodepletion with the use of Treg-targeting LYMPHIR.

The Phase 1, open-label, dose-escalation study (NCT04855253), enrolled 14 patients with relapsed or refractory DLBCL exhibiting poor prognostic features, including double/triple hit genetics, primary refractory disease, and extranodal involvement. Participants received one dose of LYMPHIR (E7777) at 5, 7, or 9 µg/kg followed by low dose chemotherapy prior to standard commercial CD19-directed CAR-T cell therapy. All patients received an infusion of one of the following FDA-approved, commercially manufactured CAR-T products: axicabtagene ciloleucel (Yescarta; Kite Pharma/Gilead Sciences), lisocabtagene maraleucel (Breyanzi; Bristol Myers Squibb), or tisagenlecleucel (Kymriah; Novartis).

The use of LYMPHIR in this study was investigational and outside of its FDA-approved indication. The Phase 1 study was not designed or powered to evaluate clinical efficacy, and no conclusions can be drawn regarding comparative effectiveness or long-term outcomes.

Key Findings from the Phase 1 Trial

● Overall response rate (ORR) was 86% at one month, including 57% complete responses (CR) and 29% partial responses (PR);

● One-year progression-free survival (PFS) was 77% (95% CI: 43–92%);

● One-year overall survival (OS) was 84% (95% CI: 49–96%);

● A single LYMPHIR dose resulted in depletion of circulating Tregs in all but one patient;

○ Median reduction of 24 Tregs/µL (range 8–65);

○ Treg nadir was observed 24 hours post-LYMPHIR;

● LYMPHIR was well tolerated with no dose-limiting toxicities (DLTs) observed up to 9 µg/kg; and,

● Reported adverse events included manageable Grade 1–2 capillary leak syndrome, fever, and transient liver enzyme elevations; Grade 3 cytopenias were consistent with expected lymphodepletion. CAR-T related cytokine release syndrome (CRS) occurred in 43% of patients (all Grade 1/2), and immune effector cell-associated neurotoxicity syndrome (ICANS) occurred in 21% (primarily low grade).

"In this high-risk population, LYMPHIR showed a favorable safety profile and promising pharmacodynamic effects when administered prior to CAR-T therapies. This data sets the stage for a larger study to assess its potential to enhance CAR-T efficacy through longer duration of LYMPHIR use," said Dr. Veronika Bachanova, Principal Investigator and Professor of Medicine at the University of Minnesota.

Dr. Bachanova presented the topline data at the 2026 Tandem Meetings | ASTCT CIBMTR.

Title: E7777 to Enhance Regulatory T-Cell Depletion Prior to CAR-T for High-Risk LBCL

Presentation ID: 677608

Abstract ID: 296369

About Diffuse Large B-Cell Lymphoma (DLBCL)

Diffuse large B-cell lymphoma (DLBCL) is the most common subtype of non-Hodgkin lymphoma (NHL), accounting for approximately 30%–40% of newly diagnosed cases in the United States. DLBCL is an aggressive and rapidly growing cancer of B lymphocytes, a type of white blood cell responsible for producing antibodies. While frontline chemoimmunotherapy regimens such as R-CHOP can be curative for many patients, up to 40% experience relapse or refractory disease. High-risk features are associated with poor outcomes and limited responses to standard therapies, including CAR-T cell therapy. Novel strategies that modulate the tumor microenvironment, such as transient regulatory T-cell depletion, are under investigation to improve treatment efficacy and long-term remission rates in this difficult-to-treat population.

About LYMPHIR (denileukin diftitox-cxdl)

LYMPHIR is a targeted immune therapy for relapsed or refractory cutaneous T-cell lymphoma (CTCL) indicated for use in Stage I-III disease after at least one prior systemic therapy. It is a recombinant fusion protein that combines the IL-2 receptor binding domain with diphtheria toxin (DT) fragments. The agent specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein synthesis. After uptake into the cell, the DT fragment is cleaved and the free DT fragments inhibit protein synthesis, resulting in cell death. Denileukin diftitox-cxdl demonstrated the ability to deplete immunosuppressive regulatory T lymphocytes (Tregs) and antitumor activity through a direct cytocidal action on IL-2R-expressing tumors.

In 2021, denileukin diftitox received regulatory approval in Japan for the treatment of relapsed or refractory CTCL and peripheral T-cell lymphoma (PTCL). Subsequently, in 2021, Citius acquired an exclusive license with rights to develop and commercialize denileukin diftitox in all markets except for India, Japan and certain parts of Asia. LYMPHIR (denileukin diftitox-cxdl) was approved by the FDA and subsequently launched in the U.S. in December 2025.

(Press release, Citius Pharmaceuticals, MAR 4, 2026, View Source [SID1234663244])