GT Biopharma Advances GTB-3650 Phase 1 Trial to Second Cohort Following Promising Immune Activation Signals
• GT Biopharma has successfully completed dosing in the first cohort of its Phase 1 trial for GTB-3650, a second-generation TriKE immunotherapy, with no safety or tolerability issues observed.
• Both patients in Cohort 1 demonstrated early biological signs of immune activation, including increased natural killer cell activity and expansion, supporting the therapy's mechanism of action.
• The trial, which will evaluate approximately 14 patients across seven dose cohorts, aims to treat relapsed or refractory CD33-expressing hematologic malignancies through enhanced NK cell-mediated cancer destruction.
CAR-T Cell Therapies Drive Growth in Adult T-Cell Leukemia-Lymphoma Market Despite Access Challenges
• The global Adult T-cell Leukemia-Lymphoma (ATLL) market is projected to experience significant growth through 2035, driven by increasing HTLV-1 infections and advancements in immunotherapy technologies.
• CAR-T cell therapies from companies like Gilead Sciences, Bristol Myers Squibb, and Novartis are revolutionizing ATLL treatment, though high costs—approximately $525,000 per infusion—create substantial access barriers.
• Pharmaceutical companies are working to reduce CAR-T manufacturing turnaround time by up to 50%, potentially improving outcomes for patients with aggressive forms of ATLL where timely intervention is critical.
Fate Therapeutics to Showcase Innovative Off-the-Shelf CAR T-Cell Platform at ASGCT Annual Meeting
• Fate Therapeutics will present five studies on its iPSC-derived CAR T-cell therapy platform at the upcoming ASGCT Annual Meeting, highlighting potential applications across autoimmune diseases and cancer.
• The company's lead candidate FT522, featuring novel Alloimmune Defense Receptor technology, will be featured in an oral presentation showing how it may eliminate the need for conditioning chemotherapy in patients.
• Preclinical data will demonstrate Fate's progress in developing off-the-shelf cell therapies targeting solid tumors, including FT836, a novel MICA/B-targeting CAR T-cell therapy with broad activity across multiple cancer types.
Design Therapeutics Appoints Dr. Chris Storgard as Chief Medical Officer to Advance GeneTAC® Pipeline
• Design Therapeutics has appointed Chris Storgard, M.D., as Chief Medical Officer, bringing over two decades of leadership in drug development with experience advancing multiple assets through global regulatory approvals.
• Dr. Storgard previously served as CMO at ADARx Pharmaceuticals and Heron Therapeutics, where he secured U.S. and European approvals for several products in oncology and acute care.
• The appointment comes at a strategic time as Design Therapeutics advances its portfolio of GeneTAC® small molecules targeting serious degenerative genetic diseases, including its lead program for Friedreich ataxia.
New Report Reveals Critical Insights into Clinical-Stage Pharma Partnerships from 2020-2025
• A comprehensive new industry report analyzes over 1,670 clinical-stage partnering agreements in pharma and biotech from 2020-2025, providing unprecedented access to deal structures and financial terms.
• The report reveals detailed intelligence on how licensing agreements typically grant exclusive rights across Phase I-III trials, with multi-component structures involving collaborative R&D and commercialization strategies.
• Business development professionals can now access actual contract documents and payment triggers often missing from press releases, enabling more effective negotiation strategies and competitive deal structuring.
NK Cell Therapy Market Poised for Significant Growth with Innovative Pipeline Developments
• The NK cell therapy market is experiencing robust growth driven by increasing interest in immuno-oncology and advancements in cell engineering technologies, with significant expansion expected through 2034.
• Leading companies including Artiva Biotherapeutics, Glycostem, Fate Therapeutics, and Senti Biosciences are developing promising therapies such as AlloNK, oNKord, and FT522, with several receiving FDA designations for expedited development.
• Off-the-shelf NK cell therapies are emerging as a key trend, offering advantages in manufacturing scalability, immediate availability, and potential applications beyond oncology into autoimmune diseases.
Off-the-Shelf CAR-NK Cell Therapy Shows Promise in B-Cell Lymphomas
• A Phase 1 clinical trial of FT596, an off-the-shelf CAR-NK cell therapy, demonstrates its safety in patients with various types of B-cell lymphoma.
• The study indicates that FT596 could be a less toxic alternative to CAR-T cell therapies, with potential for broader accessibility and reduced manufacturing costs.
• Patients with follicular lymphoma showed an 85% complete response rate, with responses lasting nearly 17 months, suggesting significant efficacy.
• FT596 combined with rituximab showed promising results in patients who relapsed after CAR-T therapy, offering a potential treatment option for this group.
CAR T-Cell Therapy Shows Promise in Treating Autoimmune Diseases: Clinical Trials and Future Directions
• UChicago Medicine launched a Phase 2 clinical trial to explore CAR T-cell therapy for systemic lupus erythematosus, inflammatory myositis, and systemic sclerosis.
• CAR T-cell therapy, initially for blood cancers, is being adapted to autoimmune diseases, potentially offering a new solution for resistant cases.
• BMS reported promising Phase 1 trial results using CD19 CAR T-cells for severe autoimmune diseases, showing significant disease activity reduction.
• Researchers are optimistic about CAR T-cell therapy's potential to 'reset' the immune system, but emphasize the need for long-term data and safety monitoring.
Fate Therapeutics Shifts Focus to Autoimmune Diseases with Promising Cell Therapies
• Fate Therapeutics is expanding its focus to autoimmune diseases, leveraging its cell therapy platform with programs like FT819 and FT522.
• Clinical progress includes dosing the first SLE patient in a Phase I study with FT819, with preliminary data expected later this year.
• The company's strong financial position, with a cash runway extended to the end of 2026, supports ongoing research and development activities.
• Upcoming data readouts from FT819 in SLE and FT522 in B-cell malignancies are expected to be critical catalysts for the company.
Novocure's Device Shows Promise in Pancreatic Cancer, FDA Reviews Cytokinetics' Heart Drug
• Novocure's electrical field-emitting device, combined with chemotherapy, significantly extended survival in locally advanced pancreatic cancer patients by two months in a Phase 3 trial.
• The FDA is set to decide on Cytokinetics' aficamten for obstructive hypertrophic cardiomyopathy by September 26, 2025, following positive Phase 3 trial results.
• Merus has granted Partner Therapeutics U.S. rights to zenocutuzumab, an experimental cancer drug currently under FDA review for NRG1 fusion-positive tumors, with a decision expected by February 4, 2025.
• The FDA is investigating blood cancer cases linked to Bluebird bio's gene therapy Skysona, advising caution and consideration of alternative treatments like stem cell transplants.
FDA Accepts Abeona Therapeutics' BLA Resubmission for Prademagene Zamikeracel in RDEB Treatment
• The FDA has accepted Abeona Therapeutics' resubmitted Biologics License Application (BLA) for prademagene zamikeracel (pz-cel) for recessive dystrophic epidermolysis bullosa (RDEB).
• Pz-cel, an autologous cell-based gene therapy, aims to address the unmet needs of RDEB patients by providing collagen VII expression at wound sites.
• The BLA is supported by data from the Phase 3 VIITAL study and a Phase 1/2a study with up to 8 years of follow-up, showcasing clinical efficacy and safety.
• The FDA has set a PDUFA target action date of April 29, 2025, with potential for Abeona to receive a Priority Review Voucher upon approval.
NK Cell Therapy Pipeline Shows Promise with Over 160 Therapies in Development
• The NK cell therapy field is experiencing significant growth, driven by its potential to address unmet needs in cancer treatment with safer, more targeted options.
• Over 140 companies are actively involved in developing more than 160 NK cell therapies, indicating robust R&D investment and confidence in this therapeutic area.
• Clinical trials are progressing for various NK cell therapies, including CAR-NK cell therapies and those targeting autoimmune disorders, showcasing versatility.
• Regulatory support, such as FDA orphan drug designation and fast track designation, is facilitating the development and potential approval of NK cell therapies.
Fate Therapeutics' FT825 Shows Promise in HER2-Targeting CAR T-Cell Therapy for Solid Tumors
• Fate Therapeutics presented initial Phase 1 data for FT825 / ONO-8250, a HER2-targeting CAR T-cell therapy, showing a favorable safety profile in advanced solid tumors.
• Preclinical data highlights FT825's cancer-selective HER2 recognition, potentially reducing off-target toxicities compared to existing HER2-directed therapies.
• The Phase 1 study observed CAR T-cell expansion and activation in patients' peripheral blood, indicating potential for effective tumor targeting.
• FT825 incorporates novel synthetic controls designed to enhance safety and efficacy in treating solid tumors, addressing limitations of current CAR T-cell therapies.
Fate Therapeutics Presents Promising Early Data for FT825/ONO-8250 CAR-T Therapy in Solid Tumors
• Fate Therapeutics' FT825/ONO-8250, a HER2-targeting CAR-T cell therapy, shows a favorable safety profile in an early-stage trial for advanced solid tumors.
• Preclinical data highlights the therapy's cancer-selective HER2 targeting, reducing off-tumor toxicities compared to traditional HER2-directed treatments.
• Initial results from the Phase 1 study indicate CAR T-cell expansion and activation in patients, suggesting potential for clinical efficacy.
• The FT825 / ONO-8250 incorporates seven novel synthetic controls of CAR T-cell function designed to overcome multiple mechanisms.
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