On March 31, 2025 Marker Therapeutics, Inc. (Nasdaq: MRKR), a clinical-stage immuno-oncology company focusing on developing next-generation T cell-based immunotherapies for the treatment of hematological malignancies and solid tumors, reported corporate updates and financial results for the year ended December 31, 2024 (Press release, Marker Therapeutics, MAR 31, 2025, View Source [SID1234651680]).

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"In 2024, we made substantial progress advancing MT-601, our lead multi antigen recognizing (MAR)-T cell therapy, and laid the groundwork for continued momentum in 2025," said Juan Vera, M.D., President and Chief Executive Officer of Marker Therapeutics. "Preliminary data from our Phase 1 APOLLO study showed encouraging safety and efficacy results in lymphoma patients who relapsed after anti-CD19 CAR-T cell therapy. With a 78% objective response rate and favorable safety profile, we believe MT-601 has the potential to provide a transformative treatment option for this patient population. We look forward to sharing additional insights during a webinar in the second quarter of 2025."

"We also strengthened our financial position through a strategic private placement and additional non-dilutive funding from the NIH and CPRIT. As we move further into 2025, our focus remains on cash preservation and disciplined execution to maximize the impact of our clinical programs," concluded Dr. Vera.

2024 PROGRAM UPDATES & OPERATIONAL HIGHLIGHTS

MT-601 (Lymphoma)

- MT-601, Marker’s lead MAR-T cell therapy, is being evaluated in the nationwide multicenter Phase 1 APOLLO study (clinicaltrials.gov identifier: NCT05798897) in patients with anti-CD19 CAR-T relapsed lymphoma or where CAR-T cells are not an option.

- The Company provided an update on the APOLLO study (Press Release, December 19, 2024). Key findings from the study include:

o Safety: MT-601 was well tolerated across all study participants. No immune-effector cell associated neurotoxicity syndrome (ICANS) and one case of Grade 1 cytokine release syndrome (CRS) were observed. No dose limiting toxicities (DLTs) have been reported to date.

o Efficacy: In the first dose cohort, 7 out of 9 patients achieved objective responses (78%) at first response assessment, with 4 patients demonstrating complete response (CR; 44.4%).

o Time in Follow-Up: Three patients have been followed for 6 to 12 months, with ongoing follow-up underway. All study participants are monitored closely to ensure comprehensive data collection and patient safety.

- The Company is enrolling additional study participants in the Phase 1 APOLLO trial and expects to report further data in the second half of 2025.

MT-601 (Pancreatic)

- Marker received $2 million from NIH SBIR and $9.5 million from CPRIT to support the development of MT-601 in metastatic pancreatic cancer.

- Clinical program launch is anticipated in the second half of 2025.

MT-401-OTS (Acute Myeloid Leukemia or Myelodysplastic Syndrome)

- The Company previously secured non-dilutive funding to support the clinical investigation of MT-401 as an "Off-the-Shelf" (MT-401-OTS) product in patients with Acute Myeloid Leukemia (AML) or Myelodysplastic Syndrome (MDS). MT-401-OTS is manufactured from healthy donors and a cellular inventory has been established with ongoing efforts to expand.

- The Company anticipates clinical program initiation during the second half of 2025.

2024 CORPORATE HIGHLIGHTS

- Announced clinical pipeline prioritization in January 2024 to strategically focus on MT-601 in patients with lymphoma. This announcement also included program updates that highlighted the potential of the Company’s MT-401-OTS program for patients with AML (Press Release, January 8, 2024).

- The United States Adopted Names (USAN) and International Nonproprietary Names (INN) committees approved "neldaleucel" as the nonproprietary (generic) name for MT-601.

- On December 23, 2024, the Company announced a $16.1 million private placement to support the clinical advancements of the Phase 1 APOLLO study. The financing involved participation from new and existing investors, including esteemed firms such as Blue Owl, New Enterprise Associates (NEA) and Aisling Capital.

FISCAL YEAR 2024 FINANCIAL HIGHLIGHTS

Cash Position and Guidance: At December 31, 2024, Marker had cash and cash equivalents of $19.2 million. The Company believes that its existing cash and cash equivalents will fund its operating expenses into the first quarter of 2026, assuming no additional grant funds are received. We anticipate receiving additional grant funding, which we expect could extend our runway beyond Q1 2026.

R&D Expenses: Research and development expenses were $13.5 million for the year ended December 31, 2024, compared to $10.4 million for the year ended December 31, 2023.

G&A Expenses: General and administrative expenses were $4.2 million for the year ended December 31, 2024, compared to $7.5 million for the year ended December 31, 2023.

Net Loss: Marker reported a net loss of $10.7 million for the year ended December 31, 2024, compared to a net loss of $8.2 million for the year ended December 31, 2023.

About MAR-T cells

The multi-antigen recognizing (MAR) T cell platform (formerly known as multiTAA-specific T cells) is a novel, non-genetically modified cell therapy approach that selectively expands tumor-specific T cells from a patient’s/donor’s blood capable of recognizing a broad range of tumor antigens. Unlike other T cell therapies, MAR-T cells allow the recognition of hundreds of different epitopes within up to six tumor-specific antigens, thereby reducing the possibility of tumor escape. Since MAR-T cells are not genetically engineered, Marker believes that its product candidates will be easier and less expensive to manufacture, with an improved safety profile compared to current engineered T cell approaches, and may provide patients with meaningful clinical benefits.

On March 31, 2025 MaaT Pharma (EURONEXT: MAAT – the "Company"), a clinical-stage biotechnology company and a leader in the development of Microbiome Ecosystem TherapiesTM (MET) dedicated to enhancing survival for patients with cancer through immune modulation, reported it will present new non clinical data at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, taking place on April 25-30, 2025 in Chicago, Illinois (Press release, MaaT Pharma, MAR 31, 2025, View Source [SID1234651679]).
MaaT034 is the Company’s next-generation full ecosystem synthetic microbiome therapy and the first candidate from its proprietary MET-C platform. Developed using a co-culturing technology optimized for large-scale production, MaaT034 is designed to improve patient responses to immunotherapy in combination with Immune Checkpoint Inhibitors.

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The key findings, obtained from studies conducted in germ-free mice, demonstrate that MaaT034:

Effectively engrafts, thus ensuring an enduring presence of beneficial bacteria in the gut environment.
Potentiates anti-tumor effects mediated by anti-PD-1 checkpoint blockade.
Leads to the production of key microbial-derived metabolites in germ-free mice.
Improves gastrointestinal physiological functions.
In parallel, the Company is developing donor-derived microbiome therapies that are being explored in multiple clinical trials. Among these, MaaT013 has been tested in patients with acute Graft-versus-Host Disease (aGvHD) and advanced melanoma receiving immune checkpoint inhibitors (PICASSO trial).

"While the PICASSO trial builds on MaaT013’s established safety and efficacy to potentially unlock significant new market opportunities in oncology, our next-generation candidate, MaaT034 — to be featured at AACR (Free AACR Whitepaper) — is designed with an optimized microbiome composition that eliminates the need for donors and incorporates defined and reproducible immunomodulatory properties, offering enhanced therapeutic potential across a broader range of oncology indications," stated Hervé Affagard, CEO and co-founder of MaaT Pharma.

AACR Poster Presentation details:

Title: MaaT034, a new co-cultured microbiome ecosystem therapy candidate, is capable to safely colonize the gastro-intestinal tract of germ-free mice to restore a healthy gut physiology and to stimulate immunity
Abstract Number: 2209
Session Category: Immunology
Session Title: Microbiome, Inflammation, and Cancer
Session Date/ Time: Monday April 28th, 2025 – 9:00:00 AM U.S Central Time
About Picasso

PICASSO is a Phase 2a clinical trial sponsored by AP-HP and in collaboration with INRAE and Institut Gustave Roussy, evaluating MaaT013 in combination with immune checkpoint inhibitors, ipilimumab (Yervoy) and nivolumab (Opdivo). The trial is fully recruited and have enrolled 70 patients. Key study endpoints include MaaT013 safety profile and best-overall response rate vs placebo as add-on treatment to Ipilimumab + Nivolumab. Clinical Trials.gov: NCT04988841

About MaaT034

MaaT034, currently in preclinical development, is a next-generation donor-independent full ecosystem synthetic microbiome therapy, dedicated to improving patient responses to immunotherapy in combination with Immune Checkpoint Inhibitors. Developed using the Company’s co-culturing proprietary MET-C platform, MaaT034 is optimized for large-scale production in oncology. Previous presented preclinical data showed that MaaT034 produced key metabolites, recognized as promoting gut barrier restoration and modulating immune responses, such as Short-Chain Fatty Acids (SCFA), secondary bile acids, and tryptophan derivatives. These data support the role of MaaT034 in gut barrier repair and in T cell reactivation either in combination with anti-PD1 or with anti-PD-L1. By enhancing gut barrier repair and modulating immune responses, MaaT034 is expected to complement the action of these immunotherapeutic agents, potentially improving their efficacy in treating solid tumors cancer.

On March 31, 2025 LIXTE Biotechnology Holdings, Inc. ("LIXTE" or the "Company") (Nasdaq: LIXT and LIXTW), a clinical stage pharmaceutical company, reported it will conduct a new pre-clinical study in collaboration with Netherlands Cancer Institute (NKI) to test whether "initiated" cells that carry mutations found in cancer cells can be eliminated by treatment with LIXTE’s proprietary compound LB-100 (Press release, Lixte Biotechnology, MAR 31, 2025, View Source [SID1234651678]).

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"In addition to our ongoing clinical trials in ovarian and colorectal cancer, this study represents a new opportunity in cancer prevention," said Bas van der Baan, LIXTE’s Chief Executive Officer. "Thus far in our Phase 1 clinical trials, LB-100 has shown patient tolerance, with little toxicity, making it a promising candidate as a broad and effective cancer prevention modality."

Increasing evidence indicates that as individuals age, certain mutations accumulate that are found in cancer cells. While these "initiated" cells behave essentially normally, they can propagate to form reservoirs of pre-malignant cells from which malignant cells may eventually emerge. Recent data from LIXTE’s ongoing clinical collaboration with NKI shows that LB-100 activates oncogenic signaling and that this is detrimental to cancer cells.

The new study in animal models will investigate whether "initiated" cells, harboring a mutant RAS oncogene, can be eliminated with LB-100. If successful, LB-100 could have a significant role in the elimination of initiated cells in aged individuals and could reduce the risk of developing a wide range of cancers as a person ages.

The study will be led by René Bernards, Ph.D., a global leader in the field of molecular carcinogenesis and Senior Staff Scientist at NKI, one of the world’s leading comprehensive cancer centers. Dr. Bernards also is a member of LIXTE’s Board of Directors.

On March 31, 2025 Kazia Therapeutics Limited (NASDAQ: KZIA) ("Kazia" or "the Company"), an oncology-focused drug development company, reported the sale of all intellectual property and trademarks rights to Cantrixil for USD $1 million (Press release, Kazia Therapeutics, MAR 31, 2025, View Source [SID1234651677]).

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In March 2021, Vivesto licensed the exclusive global development and commercialization rights for Cantrixil from Kazia Therapeutics. Having decided not to pursue development of Cantrixil in ovarian cancer, as originally anticipated under the license, Vivesto is currently exploring Cantrixil preclinically for the treatment of hematological cancers. Cantrixil, a legacy molecule in the Kazia pipeline, is a product candidate consisting of the active molecule, a potent and selective third generation benzopyran SMETI inhibitor named TRXE-002-01, encapsulated in α-cyclodextrin.

"We are pleased to enter into this agreement with Vivesto, which provides a source of non-dilutive funding that will help advance our proprietary, clinical-stage pipeline," said John Friend, M.D., Chief Executive Officer of Kazia Therapeutics.

On March 31, 2025 IN8bio, Inc. (Nasdaq: INAB), a clinical-stage biopharmaceutical company developing innovative gamma-delta T cell therapies for cancer and autoimmune diseases, reported a poster presentation on its potentially breakthrough next generation γδ T cell-based T cell engager (TCE) platform at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting 2025, taking place April 25-30, 2025 in Chicago, IL (Press release, In8bio, MAR 31, 2025, View Source [SID1234651676]).

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"T cell engagers are an exciting area of immunotherapy that remains in the early innings of development. We believe gamma-delta T cells offer unique properties, including tissue residence, phagocytosis and low IL-6 secretion, representing a powerful modality with the potential to overcome the limitations of current CD-3 based engager therapies," said William Ho, CEO, and co-founder of IN8bio. Our novel gamma-delta T cell engager platform, presented for the first time at AACR (Free AACR Whitepaper) 2025, demonstrates how we can combine the innate tumor-recognition capabilities of gamma-delta T cells with the capacity for significant cell expansion and the specificity of bispecific engagers to drive a potent, targeted immune response against multiple target antigens. These early findings in AML and B-ALL support our broader strategy to harness the unique biology of these cells across a range of cancers."

AACR Poster Presentation Details

Poster Title: A novel gamma-delta T cell engager platform for cancer immunotherapy

Abstract Presentation Number: 7321 (Poster Board 7)

Session Title: Immunology/T Cell Engagers and Novel Antibody-Based Therapies

Session Date and Time: Wednesday, April 30, 2025, 9:00 AM – 12:00 PM CT

For more details visit: www.aacr.org/meeting/aacr-annual-meeting-2025/abstracts.