On December 6, 2025 Vivacta Bio, a spin-off company of Grit Biotherapeutics advancing in vivo CAR-T therapies for oncology and autoimmune diseases, reported encouraging initial data from an investigator-initiated, first-in-human study of GT801, a T-LNP/mRNA–based in vivo CAR-T candidate targeting CD19. The results were presented in an oral session by Dr. Pin Wang, Chief Scientific Officer of Grit Biotherapeutics, at the 2025 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando, Florida. The presentation, titled "Precision In Vivo CAR-T Generation via CLAMP-Enabled mRNA Delivery: Toward Scalable and Translatable Cell Therapy," highlighted early clinical activity of GT801 and its potential to reshape CAR-T treatment delivery.

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The Phase 1 investigator-initiated study is evaluating the safety, tolerability, and preliminary efficacy of repeat GT801 dosing in patients with relapsed or refractory CD19-positive B-cell malignancies. As of the November 30, 2025 data cutoff, two patients with non-Hodgkin’s lymphoma had completed multiple GT801 administrations without lymphodepleting chemotherapy, demonstrating the feasibility of generating functional CAR-T cells in vivo in the absence of cytotoxic conditioning.

Patient 1 received three doses of 0.5 mg, and Patient 2 received four doses of 1.5 mg. GT801 was generally well tolerated. Both patients showed high CAR expression within circulating T cells. Durable and repeatable CAR-T expansion was observed following each GT801 administration. No CAR expression was detected in peripheral blood monocytes, suggesting negligible off‑target delivery of GT801 and supporting high selectivity of the T‑LNP platform.

GT801 induced rapid and profound pharmacodynamic activity, resulting in deep B-cell depletion in peripheral blood and across multiple tissues, with B cells effectively cleared in bone marrow and lymph node biopsies. By Week 4 following the final GT801 infusion, both patients achieved a partial response based on investigator assessment.

"We are encouraged by the promising safety profile and compelling efficacy signals observed with GT801 in heavily pretreated NHL patients," said Dr. Yarong Liu, Founder and CEO of Vivacta Bio. "These initial findings demonstrate GT801’s ability to generate potent, repeatable CAR-T responses without lymphodepleting chemotherapy – a major step forward for scalability, patient accessibility, and commercial viability."

About GT801

GT801 is an investigational, systemically administered in vivo CAR-T product candidate that uses T-cell–targeted lipid nanoparticles (T-LNPs) to deliver optimized mRNA encoding an anti-CD19 CAR. The platform integrates Grit Bio’s proprietary CLAMP (Controllable Ligand Attachment Modification and Purification) technology, enabling site-specific antibody conjugation, precise ligand density control, and selective T-cell targeting with minimal off-target uptake. GT801 is designed to generate potent and persistent CAR-T cells directly inside the patient, supporting repeat dosing and eliminating the need for conventional ex vivo cell manufacturing.

(Press release, Vivacta, DEC 6, 2025, View Source [SID1234661220])

On December 6, 2025 Kite, a Gilead Company (Nasdaq: GILD), and its partner Arcellx, reported new positive data from its pivotal iMMagine-1 Phase 2 study of anitocabtagene autoleucel (anito-cel), an investigational agent, which continues to show clinically meaningful deep and durable efficacy with predictable and manageable safety observed to date in relapsed or refractory multiple myeloma (RRMM) patients who had received at least three prior lines of therapy. These new findings from the ongoing study will be shared in an oral presentation (Abstract #256) today at 2:45 PM ET during the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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"These data are compelling and are an important advancement for patients living with multiple myeloma," said Dr. Krina Patel, lead investigator, Associate Professor, Department of Lymphoma/Myeloma, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center. "I am encouraged by the depth of responses in the iMMagine-1 study. For clinicians, we rely on therapies that deliver continued meaningful efficacy, a predictable safety profile, and reliable manufacturing. Anito-cel demonstrates that it could become a significant new treatment option in our efforts to improve outcomes for patients with multiple myeloma."

Data from an October 7, 2025 cutoff date, including 117 patients treated with anito-cel, who were followed for a median of 15.9 months, showed an independent review committee (IRC)-assessed overall response rate (ORR) of 96%, with 74% achieving a stringent complete response or complete response (sCR or CR) per International Myeloma Working Group (IMWG) criteria. 102 of 117 patients (87%) were triple refractory, 48 of 117 patients (41%) were penta refractory, 21 of 117 patients (18%) had extramedullary disease, and 47 of 117 patients (40%) had high risk cytogenetics. For many in this heavily pre-treated population, responses began quickly, often within one month. Median time to best response was 4.8 months and median time to sCR or CR was 3.2 months. Of the 96 patients evaluable for minimal residual disease (MRD) testing, 91 (95%) achieved MRD negativity at a median time of 1 month, meaning no cancer cells could be detected even with highly sensitive tests (≤10-5 sensitivity).

The progression-free survival (PFS) rates were 82.1% at 12 months, 67.4% at 18 months and 61.7% at 24 months, meaning many patients were still alive and free from cancer progression at those timepoints. The overall survival (OS) rates showed that a significant majority of patients remained alive, with 94% at 12 months, 88% at 18 months and 83% at 24 months. The median PFS and OS have not yet been reached, suggesting sustained and ongoing benefit for a majority of patients.

Importantly, no delayed (non-ICANS) neurotoxicities, including no Parkinsonism, no cranial nerve palsies, no Guillain-Barré syndrome, and no immune effector cell-associated enterocolitis, have been observed to date, with all patients dosed at least 12 months prior to the cutoff. In an exploratory study sponsored by Kite (Abstract #503), characterization of CD4+ CAR T cell subtypes provide further mechanistic hypotheses supporting the neurologic tolerability profile of anito-cel.

"For multiple myeloma patients in advanced treatment stages, effective options are critical as resistance to treatment grows," said Cindy Perettie, Executive Vice President, Kite. "The deep, durable responses seen with iMMagine-1, combined with a predictable and manageable safety profile and rapid and reliable manufacturing, highlight anito-cel’s potential to redefine care. Together with Arcellx, our goal is to deliver a differentiated, one-time treatment option in 2026 that may reduce patient burden and improve access, including in outpatient and community oncology settings."

Observed side effects were generally consistent with past readouts. Cytokine release syndrome (CRS) was observed in 86% of patients but was generally mild and manageable. In fact, 83% of patients in the study experienced no CRS or Grade 1 CRS (fever only). Immune effector cell-associated neurotoxicity syndrome (ICANS) occurred in 8% of patients, with only one Grade 3 case and all other cases Grade 2 or lower. The most common hematologic adverse events noted during treatment were low white blood cell counts (neutropenia) in 71% of patients, low red blood cells (anemia) in 28%, and low platelets (thrombocytopenia) in 26%. Grade 3 or higher infections occurred in 9% of patients.

Additional research presented at ASH (Free ASH Whitepaper) provided further insights into CAR T-cell therapies, detailing anito-cel’s mechanism and factors influencing treatment outcomes.

Preclinical research (Abstract #7644) shows that anito-cel’s D-Domain binder interacts with BCMA by binding and releasing quickly. Relative to a comparator CAR T-cell therapy in preclinical models, this transient interaction with cancer cells may be associated with decreased inflammation while maintaining the ability to effectively kill cancer cells. Additionally, the abstract shows anito-cel retains its ability to target cancer cells with altered BCMA expression after previous treatments, demonstrating the potential for anito-cel to maintain efficacy in patients previously exposed to BCMA-targeting therapies. Further research, including crystallography and epitope mapping, is ongoing to provide more detail on this mechanism.

About anitocabtagene autoleucel (anito-cel)

Anitocabtagene autoleucel (anito-cel, previously ddBCMA) is the first BCMA-directed CAR T-cell therapy to be investigated in multiple myeloma that utilizes Arcellx’s novel and compact binder known as the D-Domain. The small, stable D-Domain binder enables high CAR expression without tonic signaling and is designed to quickly release from the BCMA target. This combination may allow for the effective elimination of multiple myeloma cells without severe immunotoxicity. Anito-cel has been granted Fast Track, Orphan Drug, and Regenerative Medicine Advanced Therapy Designations by the U.S. Food and Drug Administration.

About Multiple Myeloma

Multiple myeloma (MM) is a type of hematological cancer in which diseased plasma cells proliferate and accumulate in the bone marrow, crowding out healthy blood cells and causing bone lesions, loss of bone density, and bone fractures. These abnormal plasma cells also produce excessive quantities of an abnormal immunoglobulin fragment, called a myeloma protein (M protein), causing kidney damage and impairing the patient’s immune function. MM is the third most common hematological malignancy in the United States and Europe, representing approximately 10% of all hematological cancer cases and 20% of deaths due to hematological malignancies. The median age of patients at diagnosis is 69 years with one-third of patients diagnosed at an age of at least 75 years. Because MM tends to afflict patients at an advanced stage of life, patients often have multiple comorbidities and toxicities that can quickly escalate and become life-endangering.

About iMMagine-1

iMMagine-1 is a Phase 2 registrational, pivotal open-label study of anito-cel in patients with relapsed or refractory multiple myeloma (RRMM) who have received at least three prior regimens of systemic therapy including proteasome inhibitor, immunomodulatory drugs (IMiD) and anti-CD38 antibody and are refractory to the last line of therapy.

The trial assessed both safety and efficacy in 117 patients receiving a single infusion of anito-cel (target dose of 115×106 CAR+ viable T cells). Efficacy was assessed monthly for the first 6 months, then quarterly up to 2 years, or upon patient relapse. Long-term safety data will be collected under a separate long-term follow-up study for up to 15 years.

The primary endpoint is overall response rate (ORR) per International Myeloma Working Group (IMWG) criteria, as assessed by an independent review committee. Secondary endpoints include complete response rate (CR/sCR), progression-free survival, overall survival, duration of response, minimal residual disease negativity and safety.

(Press release, Gilead Sciences, DEC 6, 2025, View Source [SID1234661212])

On December 6, 2025 Kite, a Gilead Company (Nasdaq: GILD), reported Phase 1 data with encouraging efficacy and safety results for its two investigational bicistronic CAR T-cell therapies, KITE-753 and KITE-363, respectively, in patients with relapsed/refractory large B-cell lymphoma (R/R LBCL). The results of the analysis were shared in an oral presentation (Abstract #265) at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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Both KITE-753 and KITE-363 are bicistronic autologous CAR T-cell therapies. They are designed to target two antigens (CD19 and CD20) found on cancer cells and use two co-stimulatory domains (CD28 and 4-1BB) to help the immune system fight cancer more effectively. The KITE DuoCore construct – with two independent CARs working synergistically – may prevent relapse by reducing cancer cells escaping treatment and may help improve safety, making it possible to treat more patients outside of a hospital setting. Additionally, KITE-753 leverages a novel manufacturing process that preserves T-cell fitness. Combined, these attributes may contribute to better efficacy, lower toxicity, durable function, and faster delivery to patients.

"While CAR T-cell therapy has revolutionized treatment for many people with blood cancers, we urgently need options that not only improve upon the curative potential of existing cell therapies for evasive cancers but are also safer and available to a broader patient population," said Dr. Saurabh Dahiya, MD, FACP, the Stanford School of Medicine. "The encouraging response rates, durability and safety profile of KITE-753 and KITE-363 offer strong clinical evidence to support further development."

The open-label, multicenter, umbrella Phase 1 study enrolled 67 patients with R/R BCL. Thirty patients received KITE-753 and 37 received KITE-363.

Results for KITE-753 showed that at a median follow-up of 4.0 months overall and 2.9 months at dose level three (DL3; 0.2×106 CAR T cells/kg), 11 of 14 CAR-naïve patients (79%) receiving DL3 had a complete response, where the cancer completely disappeared. Bridging options in this Phase 1 study were limited to corticosteroid +/- radiation therapy, and all patients had measurable, active disease at the time of their infusion with KITE-753. No patients responded to these bridging therapy measures at DL3. Notably, KITE-753 demonstrated robust expansion despite a tenfold lower dose than DL3 of KITE-363 (2×106 CAR T cells/kg), which highlights the proliferative capacity of KITE-753. Across all dose levels, 14 of 20 CAR-naïve patients achieved a complete response.

Overall, KITE-753 showed an encouraging safety profile with no dose‑limiting toxicities. At DL3, no Grade ≥3 cytokine release syndrome (CRS) or immune effector cell‑associated neurotoxicity syndrome (ICANS) were observed; Grade ≥3 adverse events occurred in 95% of patients (primarily cytopenias), and serious Grade ≥3 adverse events occurred in 26% of patients. Across all dose levels, one Grade 3 CRS event (none Grade ≥4) and no Grade ≥3 ICANS events occurred.

"Kite is dedicated to pushing the boundaries of CAR T-cell therapy to deliver even greater impact and curative potential," said Dr. Gallia Levy, Senior Vice President, Global Head of Development at Kite. "By combining CD19/CD20 targeting with dual co‑stimulation, along with a manufacturing process resulting in a fit product, we aim to improve outcomes. Our goal is to bring CAR T to more patients, such as those with advanced disease who have exhausted other options, and to those who prefer to be treated in outpatient and in community oncology practice settings."

Meanwhile, results for KITE-363 revealed that at a median follow-up of 17.5 months, there was a durable benefit at the highest dose level (2×106 CAR T cells/kg) among CAR-naïve patients, with more than 70% of complete responders evaluable at 12 months remaining in remission. KITE-363 was generally well tolerated with no dose-limiting toxicities or severe side effects that required stopping the study. Grade ≥3 CRS occurred in one patient; Grade 3 ICANS occurred in two patients; no Grade ≥4 CRS/ICANS occurred.

About LBCL

Globally, large B-cell lymphoma (LBCL) is the most common type of non-Hodgkin lymphoma. In the United States, more than 18,000 people are diagnosed with LBCL each year. About 30–40% of patients with LBCL will need second-line treatment, as their cancer will either relapse (return) or become refractory (not respond) to initial treatment.

About the Study

Thirty patients received KITE-753 and 37 received KITE-363. The open-label, multicenter umbrella Phase 1 study enrolled eligible adults with R/R LBCL after ≥2 lines of therapy (patients with LBCL could have second-line primary refractory disease) in dose escalation (1A) and expansion (1B; LBCL only) cohorts. Following leukapheresis and lymphodepletion, patients received dose level (DL) 1, 2, or 3 of KITE-753 (3.0×104, 1.0×105, or 2.0×105 CAR T cells/kg, respectively) or KITE-363 (0.5×106, 1×106, or 2×106 CAR T cells/kg, respectively). Primary endpoints were incidence of dose-limiting toxicities (DLTs; Phase 1A) and investigator-assessed objective response rate (ORR; per Lugano; Phase 1B).

About KITE-753 and KITE-363

KITE-753 and KITE-363 are investigational, bicistronic autologous CAR T-cell therapies engineered to overcome tumor antigen heterogeneity and prevent relapse. The KITE DuoCore construct uniquely combines anti-CD19 and anti-CD20 targeting with dual co-stimulation (CD28 and 4-1BB). KITE-753 is an enhanced KITE DuoCore CAR T utilizing a novel manufacturing process, aiming to preserve T-cell fitness. Additionally, KITE-363 is being investigated for refractory autoimmune conditions.

(Press release, Gilead Sciences, DEC 6, 2025, View Source [SID1234661211])

On December 6, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (NASDAQ: APTO, TSX: APS), a clinical-stage precision oncology company developing highly differentiated targeted agents to treat hematologic malignancies, reported clinical data for its lead compound tuspetinib (TUS) combined with standard dosing of venetoclax (VEN) and azacitidine (AZA) in a poster presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando, FL. Updated data from patients in the TUSCANY trial across all three cohorts, 40 mg, 80 mg or 120 mg TUS dose in TUS+VEN+AZA, reveal promising clinical safety and antileukemic activity and support the use of TUS in combination with standard of care treatment across a broad range of AML populations, including those with adverse mutations regardless of FLT3 mutation status.

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Poster title: "TUSCANY study demonstrates safety and efficacy of tuspetinib plus standard of care venetoclax and azacitidine in patients with newly diagnosed AML ineligible for induction chemotherapy"

Key Findings and Messages:

In newly diagnosed AML patients, TUS+VEN+AZA shows promising safety, tolerability and resilient efficacy, including MRD-negative remissions across a broad mutational spectrum
High-quality clinical responses (CR/CRh):
90% across 40, 80 and 120 mg dose levels
100% at the higher 80 mg and 120 mg dose levels
Observed in FLT3-WT, FLT3-ITD, and NPM1c genetic subgroups
Observed in biallelic TP53/complex karyotype and RAS adverse genetic subgroups
Observed in AML with MDS-related mutations
MRD negativity: 78% by central flow cytometry in responding subjects
TUS targets VEN resistance mechanisms; inhibits kinase-driven abnormal signaling
Two subjects transitioned to stem cell transplantation and both returned for TUS maintenance
TUS+VEN+AZA triplet therapy was well tolerated with no dose-limiting toxicities (DLTs) across all evaluable TUS dose levels
No DLTs including no prolonged myelosuppression for subjects in remission in Cycle 1
No drug-related deaths, differentiation syndrome, QTc prolongation, or CPK elevation reported
8/10 evaluable subjects experienced red cell and platelet transfusion independence for > 8 weeks after their best response
Febrile neutropenia was reported in 2 subjects (16.7%), with 1 subject related to TUS
At the recently enrolled 160 mg dose level, preliminary findings show patients achieving early blast clearance with MRD-negativity and formal responses in the first few weeks of treatment (not included in poster data cut).

"Tuspetinib, as part of a triple drug therapy, continues to perform well, achieving 100% clinical response in the two higher doses we have evaluated to date," said Rafael Bejar, MD, PhD, Chief Medical Officer at Aptose. "We recently commenced treating patients at the highest dose level of 160 mg TUS and have already achieved early responses. With no dose-limiting toxicities and activity across diverse mutations, TUS+VEN+AZA targets AML’s greatest unmet needs and largest populations."

The ASH (Free ASH Whitepaper) poster presentation is available here.

About Tuspetinib

Aptose’s lead compound tuspetinib is a convenient once daily oral agent that potently targets SYK, mutated and wild type forms of FLT3, mutated KIT, JAK1/2, and RSK2 kinases, while avoiding many typical toxicity concerns observed with other agents. The ongoing TUSCANY triplet Phase 1/2 study is designed to test various doses and schedules of TUS in combination with standard dosing of azacitidine and venetoclax in newly diagnosed patients with AML who are ineligible to receive induction chemotherapy. Data from the first three dose cohorts demonstrate safety, CRs and minimal residual disease (MRD) negativity across patients with diverse mutations. The early data showed that 9 out of 10 patients responded to the TUS triplet therapy, with 100% complete remission (CR/CRh) achieved in the 80mg and 120mg cohorts. Notably, patients with difficult-to-treat mutations in TP53, RAS and FLT3 genes also achieved a 100% CR/CRh rate.

(Press release, Aptose Biosciences, DEC 6, 2025, View Source [SID1234661210])

On December 6, 2025 Star Therapeutics, a late clinical-stage biotechnology company discovering and developing best-in-class antibodies for bleeding disorders and other diseases, reported interim data from its ongoing Phase 1/2 multidose study of VGA039 in von Willebrand disease (VWD). The data are being presented in an oral session at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida. VGA039 is a first-in-class monoclonal antibody therapy that targets Protein S, with dual actions promoting platelet attachment and enhancing fibrin deposition to restore hemostasis.

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In the Phase 1/2 multidose study, treatment with VGA039 administered subcutaneously once monthly resulted in a substantial reduction in annual bleeding rate (ABR) across all types of VWD and all types of bleeds, including meaningful improvement in bleed control for patients switching from prior von Willebrand factor (VWF)-containing intravenous (IV) prophylaxis. The study population includes patients with Types 1, 2, and 3 VWD who have high disease burden, including patients with serious gastrointestinal (GI) and hemophilia-like joint and muscle bleeds.

"The data presented at ASH (Free ASH Whitepaper) indicate that VGA039 could be transformative for people living with VWD, a condition with more than 130,000 diagnosed patients in the U.S. alone," said Allison Wheeler, M.D., MSCI, Associate Professor of Pediatrics at the University of Washington. "VWD can have a significant impact on quality of life, with patients experiencing frequent and severe bleeds that can require hospitalization. Current treatment options remain limited, with VWF-containing prophylaxis requiring multiple IV infusions per week. Data from the multidose trial support a once monthly subcutaneous dosing regimen for VGA039, which could meaningfully alleviate the current treatment burden with the potential for even better bleed control over the standard of care."

Key highlights from the presentation include:

As of November 14, 2025, interim data from all 16 patients enrolled in the Phase 1/2 multidose study were available, including safety data on all 16 patients and efficacy data on all 8 patients who had completed treatment with VGA039.
VGA039 once monthly subcutaneous prophylaxis was safe and well tolerated.
VGA039 demonstrated substantial reductions in ABR across all patients, with all types of VWD and all types of bleeds (including serious GI and hemophilia-like joint and muscle bleeds).
Bleed reductions were 73%-87% for all the participants enrolled who have the same ABR as the population being recruited into the Phase 3 trial (ABR ≥ 12, no prior IV prophylaxis).
In patients switching from prior VWF-containing prophylaxis (IV infusions multiple times per week), bleed reductions were 75%-100%, indicating potential efficacy and dosing improvement over standard-of-care.
All participants to date who have completed the multidose study have transitioned to the open-label extension (OLE) study.
"This dataset is compelling, encompassing a diverse patient population across multiple VWD types, a full spectrum of bleed profiles, and individuals transitioning from prior prophylaxis regimens. VGA039’s ability to consistently reduce bleeding across these groups along with its favorable safety and tolerability profile indicate that VGA039 has the potential to improve outcomes for all patients with VWD," said Steven Pipe, M.D., Professor of Pediatrics and Pathology at the University of Michigan. "Importantly, patients who transitioned from IV prophylaxis multiple times per week to once monthly subcutaneous VGA039 experienced marked improvements in bleed control, highlighting the potential to establish a new standard of care for people living with VWD."

"These interim data provide further validation of VGA039 as a potential once monthly subcutaneous treatment for multiple bleeding disorders, starting with VWD. All patients to date have opted to continue treatment as part of our open-label extension study after finishing the multidose trial," said Gary Patou, M.D., Chief Medical Officer of Star Therapeutics. "These data, along with the recent initiation of our pivotal Phase 3 study, VIVID-6, continue to add to the momentum surrounding this program, bringing us another step closer to achieving our mission of creating life-changing therapies for patients with bleeding disorders."
About VGA039
VGA039 is a monoclonal antibody therapy with a novel mechanism of action that targets Protein S, with dual actions promoting platelet attachment and enhancing fibrin deposition to restore hemostasis. VGA039 has potential to be a universal hemostatic therapy that can treat numerous bleeding disorders, starting with von Willebrand disease (VWD). As a subcutaneously self-administered antibody therapy with a convenient once monthly dosing regimen, VGA039 has the potential to dramatically reduce treatment burden for patients. VGA039 has received Fast Track and orphan drug designations from the U.S. Food and Drug Administration (FDA). VGA039 has advanced into a Phase 3 study (NCT07115004), VIVID-6, a global single arm cross-over study designed to investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD. For additional information on our VIVID trials of VGA039, including how to enroll, please visit the website here.

About von Willebrand disease
Von Willebrand disease (VWD) is the most common inherited bleeding disorder in which the blood does not clot properly, caused by absent or defective von Willebrand factor (VWF). VWD patients may experience excessive bleeding with varying severity and frequency, negatively impacting their daily lives. Current therapies for VWD prophylaxis include factor replacement therapies requiring multiple intravenous (IV) infusions every week. More than 130,000 people in the U.S. are diagnosed with VWD.

(Press release, Star Therapeutics, DEC 6, 2025, View Source [SID1234661209])