On December 8, 2025 Amphista Therapeutics ("the Company" or "Amphista"), a leader in the discovery of next generation targeted protein degradation (TPD) medicines, reported that full details of its new preclinical data with its lead Targeted Glue AMX-883, an orally bioavailable, potent and selective degrader of BRD9 were presented on 6 December during the 67th American Society of Haematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition in Orlando, Florida.

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The compelling findings support the potential of AMX-883 as a first-line treatment option in the earlier disease setting of acute myeloid leukaemia (AML), one of the most aggressive blood cancers where 5-year survival rates remain at just 33% and where resistance to standard of care treatments like venetoclax remains a major clinical challenge. Based on these data, the Company will advance AMX-883 as a karyotype-independent, pro-differentiation agent into the clinic for AML in H2 2026.

The key data presented at ASH (Free ASH Whitepaper) showed that:

AMX-883 demonstrates potent degradation as a monotherapy and synergistic benefit in combination with venetoclax

AMX-883 (30mg/kg twice daily and 100mg/kg once daily) significantly reduced cancer growth by achieving picomolar potency degradation of BRD9 across a panel of AML cell lines and significantly reduced leukemic burden in bone marrow and blood in vivo, compared to venetoclax alone.
Synergistic efficacy was observed when each dose of AMX-883 was combined with venetoclax (75mg/kg once daily), leading to significant blocking of tumour growth at therapeutically relevant concentrations.
AMX-883 prevents resistance to venetoclax when dosed in combination

The combination of AMX-883 with venetoclax in a venetoclax resistant cell line, prevented the emergence of resistance, and cells had comparable sensitivity to venetoclax from their first exposure.
AMX-883 actively degrades BRD9 in venetoclax resistant cells, prevents upregulation of key resistance markers, including MCL-1 and BCL-2, and increases levels of cell death markers.
Martin Pass, Chief Development Officer at Amphista Therapeutics said, "The important preclinical data presented for the first time show that our Targeted Glue AMX-883, a selective degrader of BRD9, extends the durability and efficacy of combination therapy with venetoclax and prevents AML cancer cells from becoming resistant to venetoclax. We are very pleased to receive positive feedback from experts in the field on this compelling profile, which gives us strong reason to believe that AMX-883 could be a viable treatment option for patients with AML. We look forward to commencing the first clinical trial for this devastating blood cancer in H2 2026."

To deliver the first clinical study of AMX-883, Amphista has built its development capabilities with three key strategic leadership appointments, including Dr. Lisa Butler, Senior Vice President of Clinical Operations who was previously Head of Study Leadership, early Oncology Clinical at AstraZeneca.

The abstract presented at ASH (Free ASH Whitepaper) is available on the congress website.

(Press release, Amphista Therapeutics, DEC 8, 2025, View Source [SID1234661249])

On December 8, 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 that Prof. Malard, MD, PhD, hematology professor at Saint-Antoine Hospital and Sorbonne University and ARES Trial lead investigator, presented the results for the pivotal ARES, single arm, open label trial evaluating MaaT013 (Xervyteg) in aGvHD during an oral session at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition currently taking place in Orlando, Florida, USA. In addition, the Company announced new data from the pivotal ARES trial including a 1-year overall survival rate of 54%, confirming the global clinical benefit of MaaT013 (Xervyteg).

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"These results confirm that MaaT013 (Xervyteg) offers a durable clinical benefit for patients with GI-aGvHD who have exhausted all currently approved treatment options. Achieving a 62% gastrointestinal response at Day 28, maintaining responses over time, and reaching a 54% one-year overall survival represent a meaningful step forward in addressing this critical unmet need," said Prof. Florent Malard, MD, PhD, Professor of Hematology at Saint-Antoine Hospital and Sorbonne University, and lead investigator of the ARES trial who presented the findings.

Prof. Malard detailed primary and secondary endpoints, noting that GI-Overall Response Rate (GI-ORR) at Day 28 (62% including 38% of complete response) remains high over time, indicating a durable response with a GI-ORR of 47% and all-organ ORR of 45% at Day 56. At three months, GI-ORR and all-organ ORR were both still high at 44%. These results indicate that responses to MaaT013 (Xervyteg) are durable and result in improved survival outcomes, which translates into a 54% 1-year overall survival rate in the study population.

Final efficacy data of MaaT013 (Xervyteg) in the ARES study are summarized below.
The ARES trial is a single-arm, open label trial evaluating MaaT013 (Xervyteg) as third-line treatment in 66 adult patients with severe GI-aGvHD refractory to corticosteroids and ruxolitinib across 50 sites in six European countries:

Patient profile:

91% (n=60) presented with Grade III–IV aGvHD with GI involvement
86% (n=57) were steroid-resistant and 14% (n=9) steroid-dependent; all were refractory to ruxolitinib
Male: 53%, Female: 47%
Final results:

GI-ORR at Day 28 occurred in 41/66 patients (62%) and prevalently consisted of complete response (CR) (38%, 25/66 patients), and very good partial response (VGPR) (20%, 13/66 patients).
All-organ ORR at Day 28 occurred in 42/66 patients (64%) patients and was similarly driven by high rates of CR (36%, 24/66 patients) and VGPR (18%, 12/66 patients).
GI-ORR at Day 56 was maintained at 47% (31/ 66 patients) and prevalently consisted of CR (35%, 23/66 patients).
All-organ ORR at Day 56 was 45% (30/66 patients) and prevalently consisted of CR (35%, 23/66 patients).
GI-ORR and all-organ ORR at 3 months were both 44% (29/ 66 patients), with a prevalence of CR (36%, 24/66 patients).
Overall survival (OS) at 12 months was 54% (median survival not reached), this confirms the 12-month probability of survival of 54% announced in January 2025 for the topline results.
Median overall survival was not reached, indicating that more than half of the patients were still alive at the end of the study. This suggests a durable survival benefit and reinforces the strong efficacy signal observed in the pivotal ARES study. The median OS of responders was not reached, while it was only 54 days for non-responders.
The OS was significantly higher in patients who had a GI response at Day 28 than those who did not respond: 68% vs 28% respectively (p <0.0001), indicating a strong association between early GI response and improved survival in refractory GI-aGvHD.
Safety data showed that MaaT013 (Xervyteg) was associated with an acceptable tolerability profile in severe aGvHD patient population (as reviewed continuously by a Data and Safety Monitoring Board).
The pivotal ARES trial results will soon be submitted for publication in a leading peer-reviewed medical journal. MaaT013 (Xervyteg) is currently under review by the European Medicines Agency (EMA) following the submission of a marketing authorization application in June 2025, with a decision expected in mid- 2026, as previously announced. If approved, MaaT013 (Xervyteg) would become the first microbiotherapy in oncology in the world and the first 3rd line therapy in aGvHD addressing a critical unmet need.

(Press release, MaaT Pharma, DEC 8, 2025, View Source [SID1234661265])

On December 8, 2025 Sumitomo Pharma America, Inc. (SMPA) reported new clinical data supporting further development of enzomenib, an investigational, oral selective menin inhibitor being researched for the treatment of relapsed or refractory acute leukemia, and nuvisertib, an oral investigational highly selective small molecule PIM1 kinase inhibitor, being researched for the treatment of relapsed or refractory myelofibrosis (MF), at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition.

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Updated Data Shared with the Use of Enzomenib in Acute Myeloid Leukemia
Updated preliminary data were presented from the ongoing Phase 1/2 study of enzomenib monotherapy, which as of October 4, 2025, included 116 total patients with acute leukemia, most of whom (93.1%, 108/116) had acute myeloid leukemia (AML) with a median of two prior regimens. Genomic abnormalities of leukemia subtypes including KMT2A rearrangement (KMT2Ar) were documented in 61 patients (52.6%), NPM1 mutation (NPM1m) in 34 patients (29.3%), and other HOXA9/MEIS1-driven abnormalities in 21 patients (17.7%).

Enzomenib was escalated from 40 mg twice a day (BID) to 400 mg BID with no dose-limiting toxicities (DLTs). Treatment-related adverse events (TRAEs) observed in at least 10% of patients were nausea (16.4%), and vomiting (11.2%). Differentiation syndrome (DS) was reported in 12.9% of patients and did not result in patient deaths, study discontinuations, or dose reductions of enzomenib. No treatment-related deaths were observed in the study. Dose-dependent increases in exposure were observed, particularly at doses greater than 140 mg BID. Little to no drug accumulation was observed, and CYP3A4 inhibitor azoles did not have a significant impact on exposure.

In patients with KMT2Ar, dose optimization of 200, 300, and 400 mg BID is complete, and the RP2D has been determined as 300 mg BID. At RP2D, in patients with KMT2Ar who had not received prior treatment with a menin inhibitor (n = 15), the objective response rate (ORR) was 73.3% and CR+CRh was 40%. Across the optimization dose levels, the duration of CR+CRh (n = 11) was 12.5 months and in all optimization patients (n = 39) median overall survival (mOS) was 11.8 months.

For patients with NPM1m AML, dose optimization is ongoing at 200, 300, and 400 mg BID with a focus on 200 and 300 mg BID in patients who have not received a prior menin inhibitor. In the NPM1m dose optimization population (n=25, pts who received ≥ 200 mg BID), ORR is 52% and CR+CR is 44% with a duration of CR+CRh of 5.7 months. The mOS was 8.5 months.

"Despite improved understanding of the genetic factors of certain high-risk subtypes in acute leukemias, poor prognosis for patients remains a significant unmet need," said Naval G. Daver, M.D., professor and director of the Leukemia Research Alliance Program in the Department of Leukemia at MD Anderson Cancer Center in Houston. "The data from this ongoing Phase 1/2 study continue to show that enzomenib exhibits promising clinical activity, with encouraging overall and complete response rates, duration of response, and no significant drug interactions with azoles in patients with relapsed or refractory KMT2Ar or NPM1m AML. As an intentionally designed oral therapy to inhibit menin and KMT2A protein interaction, these encouraging clinical results combined with a promising safety profile support the potential of enzomenib as a therapeutic option for relapsed or refractory acute leukemia patients with KMT2A-rearranged or NPM1-mutated subtypes of the disease."

Also presented were preliminary results of a Phase 1 study of enzomenib at dose levels of 140 mg, 200 mg, and 300 mg BID in combination with azacitidine and venetoclax (VEN/AZA) in patients with relapsed or refractory AML with KMT2Ar or NPM1m. VEN was administered on days 1-14 of a 28-day study cycle, AZA on days 1-7, and enzomenib was administered on days 1-28 with and without azole antifungal agents.

A total of 40 patients were enrolled, of which 18 had KMT2Ar (45%) and 22 (55%) had NPM1m. The median number of prior regimens was 2, with 15 patients having received prior venetoclax (37.5%) and 11 patients (27.5%) a prior menin inhibitor.

There were no DLTs observed in the 40 patients enrolled. Hematologic TRAEs related to any regimen component observed in at least 15% of patients included thrombocytopenia (45%), leukopenia (35%), neutropenia (30%), anemia (22.2%), and lymphopenia (15%). Non-hematologic TRAEs were nausea (25%), diarrhea (20%), and AST increased and constipation (15% each). Any-cause QT interval prolongation was reported in 4 patients (10%) with no grade 3 or higher events; no cases were considered related to enzomenib. There were also 4 events of non-serious DS with 0 events grade 3 or higher. Pharmacokinetic data indicated that there were no significant drug-drug interactions between enzomenib and VEN.

As of the clinical data cutoff on October 4, 2025, clinical activity data is available for 26 of the 40 total patients as 11 patients were still in Cycle 1 (n = 9) or Cycle 2 (n = 2), and 3 patients had cutaneous leukemia without measurable disease in the bone marrow (bone marrow blasts <5%).

Promising preliminary clinical activity has been observed, particularly in patients without prior VEN or menin inhibitor exposure (N=13). The ORR is 85% (11/13) and the composite complete remission (CRc) rate is 62% (8/13). Local MRD assessments were available in 9 patients and 7/9 (78%) achieved MRD negativity as of the cut-off.

These data support evaluating enzomenib with VEN/AZA in patients with newly diagnosed AML. Study arms are being added to investigate the combination regimen for patients with newly diagnosed disease. Enzomenib will be administered at 200 mg or 300 mg BID using VEN/AZA administered according to the FDA label. Enrollment of newly diagnosed patients with KMT2Ar or NPM1m AML will begin in early 2026.

Nuvisertib in Patients with Relapsed or Refractory (R/R) MF
For the first time, clinical data were presented from the ongoing global Phase 1/2 study evaluating the safety and efficacy of nuvisertib in combination with momelotinib (MMB) in 18 patients with R/R MF with anemia. All enrolled patients in the study had previously been treated with a JAK inhibitor, the current standard of care for patients with MF, and 61% of patients had high molecular risk mutation. Preliminary data showed that the treatment with nuvisertib and MMB combination appeared well tolerated, with early clinical activity observed, including ³50% total symptom score reduction (TSS50 response) in 58% of patients with an absolute reduction in all individual symptoms, a spleen volume reduction ³ 25% (SVR25 response) in 50% of patients, anemia improvement, and cytokine modulation in patients with relapsed or refractory MF with anemia. These preliminary data, collected as of October 15, 2025, support further development of nuvisertib in combination with MMB as a potential treatment option for patients with MF.

Additionally, data presented from the ongoing global Phase 1/2 study of nuvisertib in patients with relapsed or refractory MF (N=77) showed that nuvisertib monotherapy continued to be well tolerated with no DLTs and limited myelosuppression. The results showed that treatment with nuvisertib monotherapy led to SVR25 response in 20% of patients, TSS50 response in 45% of patients with absolute reduction in all individual symptoms. Data also showed that treatment with nuvisertib resulted in significant cytokine modulation over time, correlating with spleen and symptom responses and one-year overall survival rate of 81%.

"Patients living with myelofibrosis with anemia usually have a dismal prognosis, still continue to face limited treatment options," said John Mascarenhas, M.D., Director of the Center of Excellence for Blood Cancers and Myeloid Disorders, Icahn School of Medicine at Mount Sinai, New York. "The promising preliminary results from the combination of nuvisertib and momelotinib underscore the urgent need for new therapeutic approaches that may offer meaningful clinical benefits to a difficult to treat disease."

"We are excited to present the first-ever myelofibrosis data with a momelotinib-based combination, specifically nuvisertib in combination with momelotinib. The development of innovative therapies—both alone and in combination with other treatments—are critical for physicians and patients with blood cancers such as AML or MF who are in urgent need of new effective therapies," said Jatin Shah, M.D., Chief Medical Officer, Oncology, SMPA. "Based on these updated preliminary data presented at ASH (Free ASH Whitepaper), which continue to show promising clinical activity and safety profiles for both enzomenib and nuvisertib, we remain committed to accelerate the clinical development in these programs with the ultimate goal of improving patient outcomes."

About Leukemia
Leukemia is a type of cancer that forms in blood-forming tissue, characterized by the uncontrolled growth of blood cells, usually white blood cells, in the bone marrow. Acute leukemia, a form of leukemia, requires immediate treatment as blood cells multiply rapidly leading to a sudden onset of symptoms.1 Approximately 30% of patients with AML have NPM1 mutations,2 and 5%-10% of patients with AML have KMT2A rearrangements.3

About Myelofibrosis (MF)
MF is a rare type of blood cancer that is characterized by the buildup of fibrous tissue in the bone marrow, which can affect the production of blood cells. This buildup is caused by dysregulation in the JAK signaling pathway. MF is a serious and rare disease with 0.7 new cases per 100,000 people worldwide each year.4

About Enzomenib (DSP-5336)
Enzomenib is an investigational, oral, small molecule inhibitor of the menin and Lysine (K)-specific methyltransferase 2A (KMT2A) protein interaction, a key interaction in acute leukemia and other tumor cell proliferation and growth. Menin is a scaffold nuclear protein, which plays key roles in gene expression and protein interactions involved in many biological pathways, including cell growth, cell cycle, genomic stability, and hematopoiesis.5,6 In preclinical studies, enzomenib has shown selective growth inhibition in human acute leukemia cell lines with KMT2A rearrangements or NPM1 mutations.5,7 Enzomenib reduced the expression of the leukemia-associated genes HOXA9 and MEIS1 and increased the expression of the differentiation gene CD11b in human acute leukemia cell lines with KMT2A rearrangements and NPM1 mutation.8,9 The safety and efficacy of enzomenib is currently being clinically evaluated in a Phase 1/2 dose-escalation/dose-expansion study in patients with relapsed or refractory acute leukemia (NCT04988555). Additionally, the registrational Phase 2 Horizen-1 R/R mono AML/ALL (KMT2Ar + NPM1m) study is now open for enrollment. The FDA granted Orphan Drug Designation for enzomenib for the indication of acute myeloid leukemia in June 2022. The FDA granted Fast Track Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in June 2024. Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) granted Orphan Drug Designation for enzomenib for the indication of relapsed or refractory acute myeloid leukemia with KMT2Ar or NPM1m in September 2024.

About Nuvisertib (TP-3654)
Nuvisertib is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.10,11 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.10 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization and reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.11 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate- and high-risk myelofibrosis (NCT04176198). The FDA granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024. The FDA granted Fast Track Designation to nuvisertib for the indication of myelofibrosis in July 2025.

(Press release, Sumitomo Pharmaceuticals, DEC 8, 2025, View Source [SID1234661282])

On December 8, 2025 Qihan Biotech, a clinical-stage biotechnology company developing off-the-shelf cell therapies for autoimmune diseases and cancer, including allogeneic CAR-T and in vivo CAR-T therapies, reported an oral and several poster presentations at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting. The data included encouraging clinical results with the company’s allogeneic CAR-T program and foundational preclinical work supporting its emerging in vivo CAR-T program.

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Qihan’s oral presentation highlighted clinical data from 20 patients enrolled in three investigator-initiated trials evaluating QT-019B in multiple autoimmune diseases. Treatment was well tolerated, with no events exceeding Grade 1 CRS and no reported ICANS or serious infections.

Across multiple severe autoimmune indications, QT-019B demonstrated clinical benefit in patients dosed at therapeutically relevant levels (n=19), with one subtherapeutic-dose patient excluded from the efficacy summary.

SLE-ITP (n=6): 5 complete responses (CR) with normalized platelet counts and 1 partial response (PR)
APS-associated ITP (n=5): 4 CR with normalized platelet counts
AIHA (n=3): 2 CR with normalized hemoglobin
SLE (n=2): 1 DORIS remission and 1 SRI-4 response at Month 2
Neurological autoimmune diseases (NMOSD and MS; n=3): 2 EDSS stabilization; 1 too soon to assess
Pharmacokinetic and pharmacodynamic readouts reinforce the potential of Qihan’s immune-privileged platform to deliver durable, systemic immune reset.

Robust expansion of allogeneic CAR-T cells observed in 17 of 19 patients treated at therapeutic dose levels
Deep and sustained depletion of pathogenic autoantibodies, consistent with the intended immune-reset mechanism
In MS, early CSF analyses showed rapid and durable clearance of oligoclonal bands (OCB) and kappa free light chains (kFLC)
Qihan’s QT-019B program is supported by IND clearance for rSLE from both the US FDA and the China NMPA. The FDA has also granted Fast Track Designation for the SLE-ITP indication. Phase 1 enrollment is actively underway.

Qihan’s unique "immune-privileged" platform – built on the company’s world-leading capabilities in multiplexable gene editing and deep understanding of transplantation immunology – enables creation of next-generation cells capable of evading immune attack. The platform is uniquely designed to address the biological and translational barriers that have limited progress across the CAR-T field. A primary barrier is immune rejection, where host T and NK cells rapidly eliminate donor cells, limiting expansion and persistence, and preventing cells from surviving long enough to achieve durable clinical benefit. Another major challenge is the need for lymphodepletion, which is associated with organ damage and broad toxicities. Qihan is developing the next generation product QT-019C, which aims to overcome both immune rejection and the dependence on lymphodepletion. Across its oral and poster sessions, the company has demonstrated scientific rigor, clinical relevance, and platform scalability that collectively address these critical obstacles.

"Autologous therapies have shown what cell therapy can achieve, but they are not scalable for autoimmune disease. Efforts to develop off-the-shelf allogeneic approaches have been challenging due to immune rejection, limited expansion and persistence, and the need for lymphodepletion. Our data show a different path," said Luhan Yang, Ph.D., Chief Executive Officer of Qihan Biotech. "Our first-generation program demonstrates strong safety, robust in vivo expansion, meaningful efficacy, and evidence of immune reset. Our next-generation product, which is now in human testing in an IIT, goes further. By driving deeper hypoimmunity and enhanced expansion and persistence, it provides the possibility to eliminate lymphodepletion. We believe we are overcoming the key barriers that have constrained the field and opening the door to broadly accessible, off-the-shelf cell therapies."

Details of the oral and posters being presented are as follows:

Designing immune-evasive UCAR-T cells to overcome allogeneic barriers and advance off-the-shelf immunotherapy
Publication Number: 1045 (Oral Session)
Session Title: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Refining CAR-T Cells and Engineered HSPCs; New Approaches to HSPC mobilization
Session Date & Time: December 8, 2025, at 4:30PM – 4:45 PM EST

Engineering Quiescent Viral Entry Pathways for in Vivo CAR-T Generation via Binder-Fusogen Combinatorics
Publication Number: 2404
Session Title: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Poster I
Session Date & Time: December 6, 2025, at 5:30PM – 7:30 PM EST

Cytokine receptor armored CAR-T cells enable robust T cell expansion and function in the absence of lymphodepletion
Publication Number: 4104
Session Title: 702. CAR-T Cell Therapies: Basic and Translational: Poster II
Session Date & Time: December 7, 2025, at 6:00PM – 8:00 PM EST

Enhanced vsvg variants for binder‑dependent and high‑specificity transduction of primary T cells
Publication Number: 4181
Session Title: 711. Cell Collection and Manufacturing of HSPCs, CAR-T Cells, and Other Cellular Therapy Products: Poster II
Session Date & Time: December 7, 2025, at 6:00PM – 8:00 PM EST

Rapid normalization of platelet counts in patients with refractory thrombocytopenia associated with systemic lupus erythematosus (SLE) following treatment with allogeneic dual-target CD19/BCMA CAR T-cell therapy (QT-019B)
Publication Number: 3031
Session Title: 311. Disorders of Platelet Number or Function: Clinical and Epidemiological: Poster II
Session Date & Time: December 7, 2025, at 6:00PM – 8:00 PM EST

Allogeneic Dual-Target CD19/BCMA CAR T-Cell Therapy (QT-019B) for Refractory Autoimmune Hemolytic Anemia
Publication Number: 5922
Session Title: 704. Cellular Immunotherapies: Early Phase Clinical Trials and Toxicities: Poster III
Session Date & Time: December 8, 2025, at 6:00PM – 8:00 PM EST

About QT-019B
QT-019B is Qihan Biotech’s first-generation allogeneic CAR-T therapy designed to treat severe autoimmune diseases. Engineered using Qihan’s immune-privileged platform, QT-019B incorporates multiplex gene edits to enhance expansion, persistence, and resistance to immune rejection. QT-019B has demonstrated encouraging safety and clinical activity in multiple investigator-initiated trials and has been cleared by US FDA and China NMPA for further clinical development. QT-019B has received FDA Fast Track Designation for SLE-ITP.

QT-019B has been evaluated in the following autoimmune diseases:
SLE: Systemic Lupus Erythematosus
SLE-ITP: Systemic Lupus Erythematosus–associated Immune Thrombocytopenia
APS-ITP: Antiphospholipid Syndrome–associated Immune Thrombocytopenia
AIHA: Autoimmune Hemolytic Anemia
MS: Multiple Sclerosis
NMOSD: Neuromyelitis Optica Spectrum Disorder

(Press release, Qihan Biotech, DEC 8, 2025, View Source [SID1234661298])

On December 8, 2025 Bristol Myers Squibb (NYSE: BMY) reported its hematology leadership through new research across multiple therapies for patients with lymphoma at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

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Highlights include updates from the company’s targeted protein degradation pipeline, including data on first-in-class investigational lymphoma CELMoD agent golcadomide and first-in-class BCL6 ligand-directed degrader BMS-986458; alongside long-term results for Breyanzi (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy, from the Phase 3 TRANSFORM and Phase 2 TRANSCEND FL trials.

"The data presented at ASH (Free ASH Whitepaper) represent a significant step forward in our pursuit of transformative outcomes for patients with lymphoma, who urgently need more effective and durable treatment options," said Anne Kerber, senior vice president, head of development, Hematology, Oncology and Cell Therapy, Bristol Myers Squibb. "From the potential of golcadomide to offer meaningful benefit in aggressive B-cell and follicular lymphomas, to the innovative mechanism of our BCL6 ligand-directed degrader, BMS-986458, and the proven, long-term efficacy of Breyanzi – these collective results underscore our dedication to pioneering therapies that make a real difference for patients."

Targeted Protein Degradation

Two-year follow-up of golcadomide plus R-CHOP in patients with previously untreated aggressive B-cell lymphoma (Abstract #476): At median follow-up of 24 months, golcadomide 0.4 mg plus R-CHOP continued to demonstrate deep, durable responses and promising progression-free survival (PFS).

PFS rate of 79% across overall and high-risk populations.
Complete metabolic response rate (CMR) was 88% and minimal residual disease (MRD) negativity rate was 90%, irrespective of cell of origin.
In high-risk patients, CMR was 89% and MRD negativity was 93%.
Predictable and manageable safety was observed with no new safety signals observed.
Data support the ongoing Phase 3 GOLSEEK-1 study in this high-risk population.
Extended follow-up of golcadomide plus rituximab in patients with relapsed or refractory follicular lymphoma (Abstract #1006) and relapsed or refractory diffuse large B-cell lymphoma (Abstract #479): Golcadomide plus rituximab continued to show promising efficacy and durable responses in heavily pre-treated patients with relapsed or refractory follicular lymphoma (R/R FL) and relapsed or refractory diffuse large B-cell lymphoma (R/R DLBCL), including in those with prior T-cell redirecting therapy.

In FL, golcadomide 0.4 mg plus rituximab showed an overall response rate (ORR) of 97% and a complete response rate (CRR) of 78%.
In DLBCL, golcadomide 0.4 mg plus rituximab showed an ORR of 58% and a CRR of 44%.
No new safety signals in either patient population were observed.
Data support the ongoing Phase 3 GOLSEEK-4 study in FL who have received at least one prior line of systemic therapy.
Updated results from dose-escalation study of BMS-986458 monotherapy in heavily pre-treated relapsed or refractory non-Hodgkin lymphoma (Abstract #480): BMS-986458, an investigational ligand-directed-degrader targeting BCL6, continued to show promising preliminary efficacy and acceptable tolerability in patients with heavily pre-treated R/R DLBCL and FL, with mainly low-grade adverse events.

Strong antitumor activity was confirmed, with an ORR of 65% (54% in DLBCL and 80% in FL) and a CRR of 21% (7% in DLBCL and 40% in FL).
Data support continued development of BMS-986458 as a monotherapy or combination therapy for non-Hodgkin lymphoma (NHL).
"The updated data for our BCL6-targeting ligand-directed degrader reinforce the promise of ligand-directed degradation as a novel approach for patients with relapsed or refractory non-Hodgkin lymphoma," said Michael Pourdehnad, senior vice president, head of early clinical development, Hematology, Oncology and Cell Therapy, Bristol Myers Squibb. "Seeing strong antitumor activity and meaningful responses, even in heavily pre-treated populations, underscores the potential of this mechanism to address critical unmet needs and advance the standard of care."

Cell Therapy

Long-term follow-up results of patients treated with Breyanzi in TRANSFORM study (Abstract #3710): In a four-year follow-up, combining data from TRANSFORM and long-term follow-up (LTFU) studies, Breyanzi continued to demonstrate long-term clinical benefit with high PFS and overall survival (OS) rates in patients with second-line relapsed or refractory large B-cell lymphoma (LBCL). Upon completion of TRANSFORM, patients treated with Breyanzi could choose to enroll in a separate LTFU study.

Median PFS (95% CI: 12.6–NR) and OS (95% CI: NR–NR) were not reached (NR).
The four-year landmark PFS and OS rates were 52.2% (95% CI: 41.5–62.8) and 61.5% (95% CI: 51.2–71.7), respectively.
Breyanzi continued to demonstrate a consistent safety profile with no new safety signals observed compared with previous results from TRANSFORM.
Three-year follow-up with Breyanzi in the TRANSCEND FL study (Abstract #467): Three-year follow-up results from TRANSCEND FL showed that a single infusion of Breyanzi continued to demonstrate high rates of deep and durable responses.

Complete response (CR) rate was 94% with 70% of patients still in response at 36-months (duration of response [DOR]).
36-month OS was 86% and PFS was 68% in patients with third-line or later R/R FL.
Consistently high efficacy was seen across subgroups with ORR of 96%-100% and three-year ongoing response rates of 60%-83%, including in patients with high-risk characteristics (progression of disease within 24 months (POD24), bulky disease, double-refractory disease).
Safety was consistent with the primary and two-year follow-up analyses.
"Results from the TRANSFORM and TRANSCEND FL trials are a remarkable display of the treatment Breyanzi can provide for patients living with certain B-cell lymphomas, offering improved outcomes and consistent safety profile," said Lynelle B. Hoch, president, Cell Therapy Organization, Bristol Myers Squibb. "These trial results are reinforced by what we’ve seen in the real world – cell therapy continues to revolutionize the treatment of certain types of blood cancer with deep and durable responses, further inspiring us at BMS to continue driving this modality forward for patients."

Bristol Myers Squibb thanks the patients and investigators involved in these clinical trials.

About Breyanzi

Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR T cells. Breyanzi is made from a patient’s own T cells, which are collected and genetically reengineered to become CAR T cells that are then delivered via infusion as a one-time treatment. The treatment process includes blood collection, CAR T cell creation, potential bridging therapy, lymphodepletion, administration, and side-effect monitoring.

Breyanzi is approved in the U.S. for the treatment of relapsed or refractory large B-cell lymphoma (LBCL) after at least one prior line of therapy, has received accelerated approval for the treatment of relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) after at least two prior lines of therapy, relapsed or refractory follicular lymphoma (FL) after two or more prior lines of systemic therapy, relapsed or refractory mantle cell lymphoma (MCL) after at least two prior lines of systemic therapy, and relapsed or refractory marginal zone lymphoma (MZL) after at least two prior lines of systemic therapy. Breyanzi is also approved in Japan, the European Union (EU), Switzerland, Israel, the United Kingdom (UK), and Canada for the treatment of relapsed or refractory LBCL after at least one prior line of therapy; in Japan for the treatment of patients with relapsed or refractory high-risk FL after one prior line of systemic therapy, and in patients with relapsed or refractory FL after two or more lines of systemic therapy; in the EU, Switzerland, Israel, and the UK for the treatment of relapsed or refractory FL after two or more lines of systemic therapy; and in the EU and Israel for the treatment of relapsed or refractory MCL after at least two lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor.

Breyanzi U.S. FDA-Approved Indications

BREYANZI is a CD19-directed genetically modified autologous T cell immunotherapy indicated for the treatment of:

adult patients with large B-cell lymphoma (LBCL), including diffuse large B-cell lymphoma (DLBCL) not otherwise specified (including DLBCL arising from indolent lymphoma), high-grade B cell lymphoma, primary mediastinal large B-cell lymphoma, and follicular lymphoma grade 3B, who have:
refractory disease to first-line chemoimmunotherapy or relapse within 12 months of first-line chemoimmunotherapy; or
refractory disease to first-line chemoimmunotherapy or relapse after first-line chemoimmunotherapy and are not eligible for hematopoietic stem cell transplantation (HSCT) due to comorbidities or age; or
relapsed or refractory disease after two or more lines of systemic therapy.
Limitations of Use: BREYANZI is not indicated for the treatment of patients with primary central nervous system lymphoma.

adult patients with relapsed or refractory chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) who have received at least 2 prior lines of therapy, including a Bruton tyrosine kinase (BTK) inhibitor and a B-cell lymphoma 2 (BCL-2) inhibitor. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
adult patients with relapsed or refractory follicular lymphoma (FL) who have received 2 or more prior lines of systemic therapy. This indication is approved under accelerated approval based on response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in confirmatory trial(s).
adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have received at least 2 prior lines of systemic therapy, including a Bruton tyrosine kinase (BTK) inhibitor.
adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least 2 prior lines of systemic therapy.
Breyanzi U.S. Important Safety Information

WARNING: CYTOKINE RELEASE SYNDROME, NEUROLOGIC TOXICITIES, AND SECONDARY HEMATOLOGICAL MALIGNANCIES

Cytokine Release Syndrome (CRS), including fatal or life-threatening reactions, occurred in patients receiving BREYANZI. Do not administer BREYANZI to patients with active infection or inflammatory disorders. Treat severe or life-threatening CRS with tocilizumab with or without corticosteroids.
Neurologic toxicities, including fatal or life-threatening reactions, occurred in patients receiving BREYANZI, including concurrently with CRS, after CRS resolution, or in the absence of CRS. Monitor for neurologic events after treatment with BREYANZI. Provide supportive care and/or corticosteroids as needed.
T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI.
Cytokine Release Syndrome

Cytokine release syndrome (CRS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. In clinical trials of BREYANZI, which enrolled a total of 769 patients with non-Hodgkin lymphoma (NHL), CRS occurred in 56% of patients, including ≥ Grade 3 CRS in 3.4% of patients. The median time to onset was 5 days (range: 1 to 63 days). CRS resolved in 99% of patients with a median duration of 5 days (range: 1 to 37 days). One patient had fatal CRS and 5 patients had ongoing CRS at the time of death. The most common manifestations of CRS (≥10%) were fever, hypotension, chills, tachycardia, hypoxia, and headache.

Serious events that may be associated with CRS include cardiac arrhythmias (including atrial fibrillation and ventricular tachycardia), cardiac arrest, cardiac failure, diffuse alveolar damage, renal insufficiency, capillary leak syndrome, hypotension, hypoxia, and hemophagocytic lymphohistiocytosis/macrophage activation syndrome (HLH/MAS).

Ensure that 2 doses of tocilizumab are available prior to infusion of BREYANZI.

Neurologic Toxicities

Neurologic toxicities that were fatal or life-threatening, including immune effector cell-associated neurotoxicity syndrome (ICANS), occurred following treatment with BREYANZI. Serious events including cerebral edema and seizures occurred with BREYANZI. Fatal and serious cases of leukoencephalopathy, some attributable to fludarabine, also occurred.

In clinical trials of BREYANZI, CAR T cell-associated neurologic toxicities occurred in 32% of patients, including ≥ Grade 3 cases in 10% of patients. The median time to onset of neurotoxicity was 8 days (range: 1 to 63 days). Neurologic toxicities resolved in 88% of patients with a median duration of 7.5 days (range: 1 to 119 days). Of patients developing neurotoxicity, 83% also developed CRS.

The most common neurologic toxicities (≥5%) included encephalopathy, tremor, aphasia, delirium, and headache.

CRS and Neurologic Toxicities Monitoring

Monitor patients daily for at least 7 days following BREYANZI infusion for signs and symptoms of CRS and neurologic toxicities and assess for other causes of neurological symptoms. Continue to monitor patients for signs and symptoms of CRS and neurologic toxicities for at least 2 weeks after infusion and treat promptly. At the first sign of CRS, institute treatment with supportive care, tocilizumab, or tocilizumab and corticosteroids as indicated. Manage neurologic toxicity with supportive care and/or corticosteroid as needed. Advise patients to avoid driving for at least 2 weeks following infusion. Counsel patients to seek immediate medical attention should signs or symptoms of CRS or neurologic toxicity occur at any time.

Hypersensitivity Reactions

Allergic reactions may occur with the infusion of BREYANZI. Serious hypersensitivity reactions, including anaphylaxis, may be due to dimethyl sulfoxide (DMSO).

Serious Infections

Severe infections, including life-threatening or fatal infections, have occurred in patients after BREYANZI infusion. In clinical trials of BREYANZI, infections of any grade occurred in 33% of patients, with Grade 3 or higher infections occurring in 12% of all patients. Grade 3 or higher infections with an unspecified pathogen occurred in 7%, bacterial infections in 3.5%, viral infections in 2%, and fungal infections in 0.7% of patients. One patient who received 4 prior lines of therapy developed a fatal case of John Cunningham (JC) virus progressive multifocal leukoencephalopathy 4 months after treatment with BREYANZI. One patient who received 3 prior lines of therapy developed a fatal case of cryptococcal meningoencephalitis 35 days after treatment with BREYANZI.

Febrile neutropenia developed after BREYANZI infusion in 8% of patients. Febrile neutropenia may be concurrent with CRS. In the event of febrile neutropenia, evaluate for infection and manage with broad-spectrum antibiotics, fluids, and other supportive care as medically indicated.

Monitor patients for signs and symptoms of infection before and after BREYANZI administration and treat appropriately. Administer prophylactic antimicrobials according to standard institutional guidelines. Avoid administration of BREYANZI in patients with clinically significant, active systemic infections.

Viral reactivation: Hepatitis B virus (HBV) reactivation, in some cases resulting in fulminant hepatitis, hepatic failure, and death, can occur in patients treated with drugs directed against B cells. In clinical trials of BREYANZI, 35 of 38 patients with a prior history of HBV were treated with concurrent antiviral suppressive therapy. Perform screening for HBV, HCV, and HIV in accordance with clinical guidelines before collection of cells for manufacturing. In patients with prior history of HBV, consider concurrent antiviral suppressive therapy to prevent HBV reactivation per standard guidelines.

Prolonged Cytopenias

Patients may exhibit cytopenias not resolved for several weeks following lymphodepleting chemotherapy and BREYANZI infusion. In clinical trials of BREYANZI, Grade 3 or higher cytopenias persisted at Day 29 following BREYANZI infusion in 35% of patients, and included thrombocytopenia in 25%, neutropenia in 22%, and anemia in 6% of patients. Monitor complete blood counts prior to and after BREYANZI administration.

Hypogammaglobulinemia

B-cell aplasia and hypogammaglobulinemia can occur in patients receiving BREYANZI. In clinical trials of BREYANZI, hypogammaglobulinemia was reported as an adverse reaction in 9% of patients. Hypogammaglobulinemia, either as an adverse reaction or laboratory IgG level below 500 mg/dL after infusion, was reported in 30% of patients. Monitor immunoglobulin levels after treatment with BREYANZI and manage using infection precautions, antibiotic prophylaxis, and immunoglobulin replacement as clinically indicated.

Live vaccines: The safety of immunization with live viral vaccines during or following BREYANZI treatment has not been studied. Vaccination with live virus vaccines is not recommended for at least 6 weeks prior to the start of lymphodepleting chemotherapy, during BREYANZI treatment, and until immune recovery following treatment with BREYANZI.

Secondary Malignancies

Patients treated with BREYANZI may develop secondary malignancies. T cell malignancies have occurred following treatment of hematologic malignancies with BCMA- and CD19-directed genetically modified autologous T cell immunotherapies, including BREYANZI. Mature T cell malignancies, including CAR-positive tumors, may present as soon as weeks following infusion, and may include fatal outcomes. Monitor lifelong for secondary malignancies. In the event that a secondary malignancy occurs, contact Bristol-Myers Squibb at 1-888-805-4555 for reporting and to obtain instructions on collection of patient samples for testing.

Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS)

Immune Effector Cell-Associated Hemophagocytic Lymphohistiocytosis-Like Syndrome (IEC-HS), including fatal or life-threatening reactions, occurred following treatment with BREYANZI. Seven out of 769 (0.9%) patients with R/R NHL exposed to BREYANZI developed IEC-HS. Time to onset of IEC-HS ranged from 7 to 32 days. Of the 7 patients, 3 patients developed IEC-HS with overlapping occurrence of CRS and neurotoxicity, 2 patients developed IEC-HS with overlapping occurrence of neurotoxicity, and 1 patient developed IEC-HS with overlapping occurrence of CRS. IEC-HS was fatal in 2 of 7 patients. One patient had fatal IEC-HS and one had ongoing IEC-HS at time of death. IEC-HS is a life-threatening condition with a high mortality rate if not recognized and treated early. Treatment of IEC-HS should be administered per current practice guidelines.

Adverse Reactions

The most common adverse reaction(s) (incidence ≥30%) in:

LBCL are fever, CRS, fatigue, musculoskeletal pain, and nausea. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, platelet count decrease, and hemoglobin decrease.
CLL/SLL are CRS, encephalopathy, fatigue, musculoskeletal pain, nausea, edema, and diarrhea. The most common Grade 3-4 laboratory abnormalities include neutrophil count decrease, white blood cell decrease, hemoglobin decrease, platelet count decrease, and lymphocyte count decrease.
FL is CRS. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, and white blood cell decrease.
MCL are CRS, fatigue, musculoskeletal pain, and encephalopathy. The most common Grade 3-4 laboratory abnormalities include neutrophil count decrease, white blood cell decrease, and platelet count decrease.
MZL is CRS. The most common Grade 3-4 laboratory abnormalities include lymphocyte count decrease, neutrophil count decrease, and white blood cell decrease.
Please see full Prescribing Information, including Boxed WARNINGS and Medication Guide.

(Press release, Bristol-Myers Squibb, DEC 8, 2025, View Source [SID1234661250])