1stOncology™: Cancer Intelligence Service – Page 485 – 1stOncology is recognized as a Top 10 Global Pharma Analytic Provider

Orca Bio Announces FDA Acceptance and Priority Review of the Biologics License Application (BLA) for Orca-T® to Treat Hematological Malignancies

On October 6, 2025 Orca Bio, a late-stage biotechnology company committed to transforming the lives of patients through high-precision cell therapy, reported the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the Biologics License Application (BLA) seeking approval for Orca-T, its lead investigational allogeneic T-cell immunotherapy, for the treatment of hematological malignancies including acute myeloid leukemia (AML), acute lymphoblastic leukemia (ALL) and myelodysplastic syndromes (MDS) (Press release, Orca Bio, OCT 6, 2025, View Source;utm_medium=rss&utm_campaign=orca-bio-announces-fda-acceptance-and-priority-review-of-the-biologics-license-application-bla-for-orca-t-to-treat-hematological-malignancies [SID1234656463]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

Schedule Your 30 min Free Demo!

The FDA granted the BLA Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of April 6, 2026.

"A stem cell transplant has been the only potentially curative option for many people with AML, ALL or MDS, however treatment-related toxicities too often hinder patient recovery. Acceptance of the Orca-T BLA marks a pivotal moment in our ability to deliver a first-in-class therapy designed to improve survival free from complications like graft versus host disease," said Nate Fernhoff, Ph.D., co-founder and chief executive officer at Orca Bio. "Supported by positive Phase 3 clinical data, today’s regulatory milestone reflects important recognition of the transformative potential of Orca-T. We look forward to working collaboratively with the FDA on the review of our application with the goal of advancing Orca-T and making it available to patients in need."

The BLA submission for Orca-T is supported by positive results from the pivotal Phase 3 study, Precision-T (NCT04013685), a randomized, open-label multi-center study that evaluated the safety, efficacy and tolerability of Orca-T compared to conventional allogeneic hematopoietic stem cell transplant (alloHSCT) in patients with AML, ALL and MDS. The study met its primary endpoint of a statistically significant improvement in survival free of moderate-to-severe chronic graft versus host disease (cGvHD) with Orca-T versus alloHSCT.

About Orca-T
Orca-T is an investigational allogeneic T-cell immunotherapy being evaluated for the treatment of multiple hematological malignancies including acute leukemias and myelodysplastic syndromes. Orca-T is composed of highly purified regulatory T-cells, hematopoietic stem cells and conventional T-cells derived from peripheral blood from either related or unrelated matched donors. Orca-T has received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug Designation for the prevention of graft versus host disease or death in patients eligible for hematopoietic stem cell transplant from the U.S. Food and Drug Administration.

Nanoligent closes €12 million Series A financing to advance its lead oncology nanodrug

On October 6, 2025 Nanoligent reported the completion of a €12 million Series A financing round led by Inveready (Inveready Biotech IV), with participation from CDTI (through the Innvierte program), Clave Capital (Clave Innohealth), and existing investors i&i Biotech Fund I, Italian Angels for Growth / Nanolinvest, and AVANTECA Partners (Press release, Nanoligent, OCT 6, 2025, https://www.nanoligent.com/nanoligent-closes-12-million-series-a-financing-to-advance-its-lead-oncology-nanodrug/ [SID1234656462]).

The proceeds will support completion of the regulatory preclinical package for the company’s lead candidate, NNL1524, enable manufacturing scale-up, and fund the initiation of a Phase Ia clinical study in patients with solid tumors.

NNL1524 is a multivalent cytotoxic nanoparticle designed to selectively target the chemokine receptor CXCR4, a protein involved in tumor growth, invasion, and metastasis, and frequently overexpressed in several hematologic and solid malignancies including colorectal, lung, and breast cancers. In preclinical studies, NNL1524 demonstrated significant antitumor activity as a single agent and a favorable safety profile, supporting its advancement into clinical development.

Dr. Montserrat Cano, Chief Executive Officer of Nanoligent, commented:
"This financing represents an important milestone for Nanoligent. The support of such a strong syndicate of investors will be instrumental as the company progresses toward clinical validation of its technology and first-in-human studies."

Antonio Herce, Director of Investments at Inveready, stated:
"Nanoligent’s nanodrug platform offers a distinctive approach to targeted cytotoxic therapy, combining high selectivity, internalization efficiency, and stability. We are pleased to support the company’s efforts to bring this promising technology closer to patients."

The transaction was advised by Hoffmann Eitle and VentureTech Audit.

Marker Therapeutics Announces First Patient Treated in Off-the-Shelf Program

On October 6, 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 tumor indications, reported that the first patient has been treated in the Company’s OTS program, with encouraging preliminary safety data (Press release, Marker Therapeutics, OCT 6, 2025, View Source [SID1234656461]).

Marker is evaluating the safety and efficacy of escalating doses of MT-401, a multi-antigen recognizing (MAR) T cell product targeting four antigens, as an OTS product in the Phase 1 RAPID study (clinicaltrials.gov Identifier: NCT06552416). The first study participant received the OTS product at the initial dose level (100×106 cells) and was monitored for 28 days. The therapy was well tolerated with no treatment-related adverse events. This observation is consistent with the favorable safety profile and tolerability previously reported for MAR-T cells. The OTS product will be initially tested in patients with acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS), with the potential to be expanded to other indications.

"The initiation of our OTS program represents a major achievement," said Juan Vera, M.D., President and CEO of Marker Therapeutics. "One of the biggest limitations to cell therapy is the time-consuming manufacturing of individualized products. With our OTS product, we are aiming to remove this bottleneck and provide a fast treatment option for patients with aggressive and rapidly progressing diseases. We believe that using commercially available leukapheresis material from healthy donors can facilitate large-scale manufacturing and expedite treatment as fast as 72 hours, while also enabling broader scalability and accessibility of cell therapies at a lower per-dose cost."

"Having a rapid available alternative to individualized T cell production allows us to broaden our clinical investigation of MAR-T cells and to extend the OTS program to other indications," commented Dr. Vera. "Looking ahead, we will remain focused on advancing our clinical investigation of MAR-T cells in lymphoma. We recently reported promising clinical efficacy and durability data from our APOLLO study where we have observed an objective response rate of 66%, with durable complete responses in patients with non-Hodgkin lymphoma, and we believe our lymphoma program has the potential to qualify for expedited approval."

Marker has secured non-dilutive funding from the Food and Drug Administration (FDA), the National Institutes of Health (NIH) Small Business Innovation Research (SBIR) program and the Cancer Prevention and Research Institute of Texas (CPRIT) to support the clinical investigation of the OTS product. Using these allocated non-dilutive funds will allow the Company to proceed with the OTS program without affecting the Company’s runway and its efforts to advance its lead asset, MT-601, in the ongoing Phase 1 APOLLO study in patients with lymphoma. Marker recently reported an update from the APOLLO study (Press Release, August 26, 2025) highlighting a favorable safety profile and durable clinical responses.

To facilitate the OTS program, the Company has established a cellular inventory from commercially available leukapheresis material that was carefully selected to cover a large patient population with partially human leukocyte antigen (HLA) matched material. This approach has been validated and extensively tested in the clinic (Leen et al., Blood, 2013; Tzannou et al, Blood Adv, 2019; Tzannou et al, J Clin Oncol, 2017). Data from the ongoing RAPID trial will help inform future clinical developments of MAR-T cell products and guide their potential use as an OTS product in other indications.

"Behind this milestone is a set of scientific data and a significant body of research and development. This strategy has been tested extensively in the clinic at Baylor College of Medicine in the context of virus-specific T cells (VST). As we enroll additional patients in the Phase 1 RAPID study, we will continue to closely monitor the safety and long-term treatment effects of our OTS product. The collected data from this trial will serve as a foundation for refining and understanding the performance of MAR-T cells as an OTS product to potentially expand the OTS approach to other product candidates in our pipeline with the goal to accelerate time to treatment in other indications," concluded Dr. Vera.

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.

About the Off-the-Shelf Program

MT-401-OTS is an Off-the-Shelf (OTS) multi-antigen recognizing (MAR) T cell product that targets four different tumor antigens upregulated in cancer cells (Survivin, PRAME, NY-ESO-1, WT-1). MT-401-OTS is currently investigated in the Company-sponsored Phase 1 multicenter, open-label RAPID trial (clinicaltrials.gov identifier: NCT06552416) for the treatment of patients with relapsed acute myeloid leukemia (AML) or myelodysplastic syndromes (MDS). The Company’s OTS program is supported by the National Cancer Institute of the National Institutes of Health (Award Number 1R44CA285177), the Food and Drug Administration Department of Health and Human Services (R01FD007272), and the Cancer Prevention and Research Institute of Texas (CPRIT, Award Number DP210042).

Immix Biopharma to Present Abstract at the upcoming ASH 2025 Annual Meeting

On October 6, 2025 Immix Biopharma, Inc. ("ImmixBio", "Company", "We" or "Us" or "IMMX"), the global leader in relapsed/refractory AL Amyloidosis, reported that it will present a NXC-201 abstract at the upcoming American Society of Hematology (ASH) (Free ASH Whitepaper) 67th Annual Meeting and Exposition to be held December 6-9, 2025, in Orlando, Florida (Press release, Immix Biopharma, OCT 6, 2025, View Source [SID1234656460]).

About AL Amyloidosis
AL amyloidosis is caused by abnormal plasma cells in the bone marrow, which produce misfolded amyloid proteins that build-up in the heart, kidney, and liver, causing heart and renal failure, leading to mortality.

The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is estimated to be growing at 12% per year according to Staron, et al Blood Cancer Journal, to approximately 37,270 patients in 2025.

The Amyloidosis market was $3.6 billion in 2017, and is expected to reach $6 billion in 2025, according to Grand View Research.

Defence Therapeutics To Attend Cphi Worldwide In Frankfurt, October 28-30, 2025

On October 6, 2025 Defence Therapeutics Inc. ("Defence" or the "Company"), a leading biotechnology company specialized in drug delivery technologies, reported its participation at CPHI Worldwide 2025, taking place in Frankfurt, Germany from October 28–30, 2025 (Press release, Defence Therapeutics, OCT 6, 2025, View Source;utm_medium=rss&utm_campaign=defence-therapeutics-to-attend-cphi-worldwide-in-frankfurt-october-28-30-2025 [SID1234656458]).

At this premier gathering and global trade fair of pharmaceutical leaders, Defence looks forward to engaging with potential partners across the biopharma value chain and with the global pharma community, learning about the latest trends in pharmaceutical manufacturing, and connecting with its international collaborators and shareholders.

In addition to its conference participation, Defence warmly invites attendees, partners, and shareholders in the region to join its leadership team for an informal Meet & Greet on Wednesday, October 29 from 4:30–6:30 PM at the American May lobby bar of the Kimpton Hotel in Frankfurt. This networking event will provide an opportunity to meet the team, exchange insights, and explore partnership opportunities in a relaxed setting.

Interested in meeting our team? Reach out through the CPHI conference partnering platform or connect with our leaders who will be at the meeting via LinkedIn: Amie Phinney, Strategy and Business Advisory, Mark Lambermon, Head of Quality and Operations (our in-house German speaker!) or Sebastien Plouffe, CEO. And of course, don’t hesitate to just drop-by the Meet & Greet event.

About CPHI:

CPHI (www.cphi.com) is a leading global pharmaceutical trade show and networking platform, bringing together tens of thousands of industry professionals from more than 160 countries. In the 2025 edition in Frankfurt, over 62,000 visitors and around 2,400 exhibiting companies are expected, with more than 180 international speakers featured.