On December 8, 2025 Geron Corporation (Nasdaq: GERN), a commercial-stage biopharmaceutical company aiming to change lives by changing the course of blood cancer, reported new data presented at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) 2025 Annual Meeting being held in Orlando, FL. Collectively, the oral and poster presentations reinforce the growing body of evidence supporting the potential of RYTELO (imetelstat), Geron’s first-in-class telomerase inhibitor, across lower-risk myelodysplastic syndromes/neoplasms (LR-MDS) and myelofibrosis (MF).

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"These new data confirm our understanding of how imetelstat works and highlight the potential long-term benefit in appropriate patients," said Joseph E. Eid, M.D., Executive Vice President, Research and Development and Chief Medical Officer of Geron. "The data suggesting the association between early treatment-emergent cytopenias and subsequent hemoglobin and transfusion independence response with imetelstat, together with the long-term outcomes from IMerge presented at ASH (Free ASH Whitepaper), continue to reinforce imetelstat as a differentiated treatment option for eligible patients with lower-risk MDS."

The oral presentation by Amer Zeidan, MBBS, MHS, Yale School of Medicine, and a principal investigator of the IMerge trial, featured pooled analyses of the IMerge population suggesting that early treatment-emergent neutropenia and thrombocytopenia may be associated with greater hemoglobin increases, which emerged as a main driver for achieving red blood cell transfusion independence (RBC-TI).

"What stands out in these analyses is the consistency between the clinical response we see in patients and the early treatment patterns observed in their blood counts when we use imetelstat in non-del5q lower-risk MDS patients with transfusion dependent anemia. This is reminiscent of similar trends we see with lenalidomide, which is a disease-modifying agent in del5q lower-risk MDS patients," said Amer Zeidan, Chief of the Division of Hematologic Malignancies and Professor of Medicine at Yale Cancer Center. "For patients with lower-risk MDS, transfusion dependence places a considerable physical and logistical burden on daily life, and treatment options beyond ESAs remain limited. The association we observed between early blood count changes and later improvements in hemoglobin provides valuable insight for clinicians and underscores the meaningful benefit imetelstat may offer to this population."

Additional poster presentations provided long-term outcomes data, including survival data in LR-MDS, exploratory biomarker analyses in MF, and ongoing combination and investigator-sponsored studies across the myeloid malignancy spectrum.

Highlights from the ASH (Free ASH Whitepaper) 2025 Presentations

Correlation between Treatment-Emergent Cytopenias and Clinical Response with Imetelstat in Patients with Lower-Risk Myelodysplastic Syndromes: Analysis from the IMerge Trial (Zeidan et al., Oral #490)

This oral presentation reported findings from post-hoc analyses examining the potential relationship between treatment-emergent cytopenias within the first two cycles of imetelstat treatment and key clinical outcomes in the pooled IMerge patient population. These analyses evaluated reductions in platelets and neutrophils alongside hemoglobin (Hb) rise, achievement of ≥8- or ≥24-week RBC-TI, and achievement of Hb rise ≥1.5 g/dL lasting ≥8 weeks.

Across analyses looking at single clinical factors, patients who experienced ≥75% reductions in neutrophils or ≥50% reductions in platelets during the first two cycles of imetelstat had greater maximum Hb increases compared with those who did not experience such reductions. Numerically higher rates of ≥8- or ≥24-week RBC-TI were also observed in these groups. When the analyses accounted for multiple clinical factors at the same time, the associations between early cytopenias and Hb improvements remained consistent: ≥75% neutrophil reduction was associated with greater Hb increase, whereas maximum ≥50% platelet reduction was associated with achieving Hb rise ≥1.5 g/dL lasting ≥8 weeks. Further, Hb rise emerged as a main driver of achieving transfusion independence. A strong association between platelet and neutrophil reductions was also observed.

These findings offer deeper context for interpreting patient responses and provide insight into how imetelstat’s biological effects may translate into meaningful clinical outcomes.

Long-Term Outcomes and Overall Survival from the Randomized, Double-Blind, Placebo-Controlled, Phase 3 IMerge Trial of Imetelstat for Lower-Risk Myelodysplastic Syndromes (Santini et al., Poster #2074)

This poster reported updated long-term outcomes data from the double-blind, placebo-controlled IMerge Phase 3 trial assessing overall survival (OS), progression-free survival (PFS), and time to progression to acute myeloid leukemia (AML) in patients with transfusion-dependent LR-MDS treated with imetelstat.

At a median follow-up of 45 months, the analysis showed a favorable trend toward improved secondary endpoints of OS, PFS, and time to AML progression for imetelstat-treated patients compared with placebo recipients in the overall population. OS favored imetelstat versus placebo in most predefined subgroups, regardless of transfusion burden, serum EPO (erythropoietin) level, and ring sideroblast positive or negative status. Additionally, OS outcomes were numerically improved in patients who achieved RBC-TI or hemoglobin improvement.

These results suggest that imetelstat may provide meaningful long-term benefit for patients with lower-risk MDS who are transfusion independent, although the trial was not powered to detect statistical significance for OS. The data support continued clinical exploration of telomerase inhibition as a potential disease-modifying approach for LR-MDS.

Correlation between IL-8 and TNF-Alpha Levels and Overall Survival in Patients with Myelofibrosis Relapsed or Refractory to a JAKi Treated with Imetelstat in the IMbark Trial​ (Mascarenhas et al., Poster #5585)

This poster reported results from a post hoc analysis of inflammatory cytokines in the Phase 2 IMbark trial of patients with JAK inhibitor relapsed/refractory myelofibrosis treated with imetelstat. The analysis evaluated how cytokine changes corresponded with clinical endpoints, including symptom improvement, spleen volume reduction, and survival trends.

The findings showed that patients treated with imetelstat experienced dose-dependent reductions in IL-8 and TNF-a (proinflammatory cytokines), with the 8.9 mg/kg dose showing more pronounced effects compared to the 4.4 mg/kg dose. Reductions in these cytokines corresponded with reductions in total symptom score or spleen volume, consistent with previously reported clinical activity. A longer survival trend was observed in patients receiving the higher dose, particularly among patients with elevated baseline IL-8 and TNF-a levels, although subgroup sizes were small.

These results suggest that cytokine modulation may contribute to the clinical activity of imetelstat and provide further support for its potential disease-modifying properties in myelofibrosis. These findings build on prior biomarker work demonstrating imetelstat’s impact on malignant progenitor cells in myelofibrosis.

IMproveMF: Phase 1b Trial of Imetelstat Plus Ruxolitinib in Patients with Intermediate-1/2 or High-Risk Myelofibrosis (Mascarenhas, et al., Poster #2052)

This poster reported enrollment progress and emerging observations from the Phase 1b IMproveMF trial evaluating imetelstat in combination with ruxolitinib for patients with intermediate-2 or high-risk myelofibrosis. The study is designed to determine safety, tolerability, and recommended dosing for future development.

As of the July 2025 cutoff, the trial had enrolled three patients in part 2 of the study, with the first patient treated in January 2025. The part 1 dose escalation phase of the study is complete, with 8.9 mg/kg imetelstat IV every 4 weeks identified as the recommended dose to be combined with ruxolitinib.

Advanced Myelodysplastic Neoplasms or AML Failing HMA-based Therapy – Preliminary Results of the IMpress Study​ (Ades, et al, Poster #5115)

This poster reported interim results from an investigator-sponsored Phase 2 study evaluating imetelstat in patients with advanced myelodysplastic neoplasms or acute myeloid leukemia who were refractory to, relapsed after, or intolerant to hypomethylating agent, or HMA, therapy.

The results showed that imetelstat administered at 7.1 mg/kg IV every 2 weeks in this patient population did not appear to be associated with additional toxicity, and that treatment-emergent adverse events were manageable in this heavily pre-treated population. While limited single-agent clinical activity was observed – consistent with the aggressive disease biology and prior treatment history – several patients were able to complete protocol-specified visits, and one remained on treatment at the time of the analysis.

These findings suggest that single agent imetelstat has a predictable and manageable safety profile at this exposure in these advanced diseases and provides important insights for potential combination strategies in high-risk populations, where unmet needs remain substantial.

The ASH (Free ASH Whitepaper) presentations are available on Geron’s website in the Investor section under publications.

Dr. Zeidan has served as a consultant for Geron and has received honoraria. The views expressed in this press release and in the presentation are his own and do not necessarily reflect those of his employer.

About RYTELO (imetelstat)
RYTELO is an oligonucleotide telomerase inhibitor approved in the U.S. for the treatment of adult patients with LR-MDS with transfusion-dependent anemia requiring four or more red blood cell units over eight weeks who have not responded to or have lost response to or are ineligible for erythropoiesis-stimulating agents (ESAs). It is indicated to be administered as an intravenous infusion over two hours every four weeks.
In addition, RYTELO is approved in the European Union as a monotherapy for the treatment of adult patients with transfusion-dependent anemia due to very low, low or intermediate risk myelodysplastic syndromes without an isolated deletion 5q cytogenetic (non-del 5q) abnormality and who had an unsatisfactory response to or are ineligible for erythropoietin-based therapy.

RYTELO is a first-in-class treatment that works by inhibiting telomerase enzymatic activity. Telomeres are protective caps at the end of chromosomes that naturally shorten each time a cell divides. In LR-MDS, abnormal bone marrow cells often express the enzyme telomerase, which rebuilds those telomeres, allowing for uncontrolled cell division. Developed and exclusively owned by Geron, RYTELO is the first and only telomerase inhibitor approved by the U.S. Food and Drug Administration and the European Commission.

US IMPORTANT SAFETY INFORMATION ABOUT RYTELO

WARNINGS AND PRECAUTIONS

Thrombocytopenia

RYTELO can cause thrombocytopenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased platelets occurred in 65% of patients with MDS treated with RYTELO.
Monitor patients with thrombocytopenia for bleeding. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer platelet transfusions as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.

Neutropenia

RYTELO can cause neutropenia based on laboratory values. In the clinical trial, new or worsening Grade 3 or 4 decreased neutrophils occurred in 72% of patients with MDS treated with RYTELO.
Monitor patients with Grade 3 or 4 neutropenia for infections, including sepsis. Monitor complete blood cell counts prior to initiation of RYTELO, weekly for the first two cycles, prior to each cycle thereafter, and as clinically indicated. Administer growth factors and anti-infective therapies for treatment or prophylaxis as appropriate. Delay the next cycle and resume at the same or reduced dose, or discontinue as recommended.

Infusion-Related Reactions

RYTELO can cause infusion-related reactions. In the clinical trial, infusion-related reactions occurred in 8% of patients with MDS treated with RYTELO; Grade 3 or 4 infusion-related reactions occurred in 1.7%, including hypertensive crisis (0.8%). The most common infusion-related reaction was headache (4.2%). Infusion-related reactions usually occur during or shortly after the end of the infusion.
Premedicate patients at least 30 minutes prior to infusion with diphenhydramine and hydrocortisone as recommended and monitor patients for at least one hour following the infusion as recommended. Manage symptoms of infusion-related reactions with supportive care and infusion interruptions, decrease infusion rate, or permanently discontinue as recommended.

Embryo-Fetal Toxicity

Based on animal findings, RYTELO can cause embryo-fetal harm when administered to a pregnant woman. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with RYTELO and for 1 week after the last dose.

ADVERSE REACTIONS

Serious adverse reactions occurred in 32% of patients who received RYTELO. Serious adverse reactions in >2% of patients included sepsis (4.2%) and fracture (3.4%), cardiac failure (2.5%), and hemorrhage (2.5%). Fatal adverse reactions occurred in 0.8% of patients who received RYTELO, including sepsis (0.8%).
Most common adverse reactions (≥10% with a difference between arms of >5% compared to placebo), including laboratory abnormalities, were decreased platelets, decreased white blood cells, decreased neutrophils, increased AST, increased alkaline phosphatase, increased ALT, fatigue, prolonged partial thromboplastin time, arthralgia/myalgia, COVID-19 infections, and headache.

(Press release, Geron, DEC 8, 2025, View Source [SID1234661260])

On December 8, 2025 Genmab A/S (Nasdaq: GMAB) reported new and updated data from three arms of the ongoing Phase 1b/2 EPCORE CLL-1 trial (NCT04623541) evaluating the efficacy and safety of epcoritamab-bysp, a T-cell engaging bispecific antibody administered subcutaneously, as a monotherapy and in combination for the treatment of patients with Richter transformation (RT), a rare complication in which chronic lymphocytic leukemia (CLL) evolves into an aggressive lymphoma, most often diffuse large B-cell lymphoma (DLBCL). The results were presented today in two oral presentations (abstracts 1015 and 1017) at the 67th Annual Meeting and Exposition of the American Society of Hematology (ASH) (Free ASH Whitepaper), in Orlando, Florida.

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EPCORE CLL-1, Arm 2A (Epcoritamab Monotherapy)
In Arm 2A of the trial, patients with RT (n=42) received epcoritamab monotherapy in the first-line setting or in second- or later-line settings, with a median follow-up of 22.9 months. In the first-line setting (n=21), patients achieved an overall response rate (ORR) of 57%, with 52% experiencing a complete response (CR). The median overall survival (OS) was 27.5 months, progression-free survival (PFS) was 8.5 months, and the median duration of response (DOR) and duration of complete response (DOCR) were not reached. Among RT patients who received epcoritamab monotherapy in second- or third-line settings (n=21), ORR was 38% and the CR rate was 29%. The median DOR was 6.6 months, median PFS was 2.9 months, and median OS was 9.8 months. The results from Arm 2A have been simultaneously published in The Lancet Haematology.

"Patients with Richter transformation, an aggressive form of lymphoma, have limited treatment options and face a poor prognosis," said Arnon Kater, M.D., Ph.D., Department of Hematology, Amsterdam UMC. "The response and survival rates observed in this trial evaluating epcoritamab as a monotherapy treatment are encouraging, especially as a potential option for patients with Richter transformation."

In this arm, cytokine release syndrome (CRS) occurred in 86% of patients (79% with Grade 1/2), immune effector cell-associated neurotoxicity syndrome (ICANS) in 12% of patients (all Grade 1/2), and clinical tumor lysis syndrome (CTLS) in 5%. Most CRS events occurred after the first full dose and resolved within a median of three days in 97% of patients.

EPCORE CLL-1, Arm 2B (Epcoritamab Lenalidomide Combination)
In Arm 2B, previously-treated patients with RT (n=11) ineligible to receive chemoimmunotherapy who had two or less prior lines of therapy received epcoritamab in combination with lenalidomide. With a median follow-up of 16.7 months, the ORR was 82% and the CR rate was 73%. The median OS at nine months was not reached, and the median PFS was 5.7 months. The estimated median DOR and DOCR were not reached.

In this arm of the trial, CRS events were primarily low grade and resolved in 10 patients, with a median time to resolution of four days. One patient discontinued due to CRS. ICANS occurred in two patients (Grade 1/2) and resolved in a median of 2.5 days. There was one treatment-related Grade 5 event.

"With no standard of care for patients with Richter transformation, clinicians are in need of new, therapeutic options with the potential for patients to achieve and maintain remissions," said Philip A. Thompson, MB, MS, Peter MacCallum Cancer Center Melbourne, Australia. "These first results from the combination arms of the EPCORE CLL-1 study demonstrate the potential of epcoritamab combination regimens as potential therapeutic options for those living with Richter transformation."

EPCORE CLL-1, Arm 2C (Epcoritamab R-CHOP Combination)
In Arm 2C, previously untreated patients with RT (n=30) received epcoritamab in combination with rituximab, cyclophosphamide, doxorubicin, vincristine, and prednisone (R-CHOP). With a median follow-up of 13.6 months, the ORR was 77% and the CR rate was 63%. The median OS was 16.4 months and the median PFS was 16.0 months. The estimated median DOR and median DOCR were not reached.

In this arm, CRS events were primarily low Grade (Grade 1, 7; Grade 2, 8; Grade 3, 2) and median time to resolution was 2.0 days. No patients discontinued due to CRS. ICANS occurred in four patients (Grade 1, 3; Grade 3, 1); three cases resolved in a median of one day, and one was ongoing at time of death. There were three treatment-related Grade 5 events.

"The results from these trials demonstrate the potential of epcoritamab as a monotherapy, and in combination, in patients with Richter transformation, a rare, often fatal, transformation of chronic lymphocytic leukemia into an aggressive lymphoma, mostly diffuse large B-cell lymphoma," said Dr. Judith Klimovsky, Executive Vice President and Chief Development Officer of Genmab. "We are deeply committed to exploring epcoritamab as a potential core therapy across a range of B-cell malignancies, both as an initial treatment and as a later line of therapy."

The safety and efficacy of epcoritamab have not been established for these investigational uses.

In all three study arms, safety was consistent with the known profiles of each agent. In Arm 2A, the most common treatment-emergent adverse events (TEAEs) were infection (74%), anemia (50%), thrombocytopenia (48%), neutropenia (45%), diarrhea (36%), and fatigue (31%). Four patients (10%) discontinued treatment due to a TEAE, and three (7%) experienced fatal events, none considered related to study treatment. In Arm 2B, common TEAEs were CRS (100%), neutropenia (82%), thrombocytopenia (73%), anemia and hypokalemia (45% each). Grade ≥3 TEAEs occurred in all patients, serious TEAEs in 10/11, and epcoritamab-related discontinuations and fatal TEAEs in one patient each. In Arm 2C, common TEAEs were CRS (56%), anemia (60%), neutropenia (73%), thrombocytopenia (46%), diarrhea (33%), and febrile neutropenia (30%). Grade ≥3 TEAEs occurred in 27 (90%) patients and serious TEAEs in 25 (83%). TEAEs led to epcoritamab discontinuations in six (20%) patients and there were three fatal TEAEs (one epcoritamab related). CTLS was not reported in Arms 2B or 2C.

About Richter Transformation (RT)
Richter transformation (RT) is a rare but aggressive evolution of chronic lymphocytic leukemia (CLL), most often into CD20+ diffuse large B-cell lymphoma (DLBCL).i Prognosis of RT is poor, with complete remission rates of approximately 20% and median survival often less than one year following chemoimmunotherapy.ii,iii

About the EPCORE CLL-1 Trial
EPCORE CLL-1 is a global, Phase 1b/2, open-label, multi-center trial to evaluate the safety and preliminary efficacy of epcoritamab as a monotherapy and in combination with standard of care agents in patients with difficult-to-treat relapsed/refractory (R/R) chronic lymphocytic leukemia (CLL), R/R small lymphocytic lymphoma (SLL) and Richter transformation (RT). The trial consists of two parts: a dose-escalation phase (Phase 1b) and an expansion phase (Phase 2). Patients with RT are only included in the expansion phase. In patients with RT, epcoritamab monotherapy (Arm 2A) and combination therapy with lenalidomide (Arm 2B) or R-CHOP (Arm 2C) will be evaluated to assess their efficacy, safety and tolerability profiles.

More information on this trial can be found at www.clinicaltrials.gov (NCT: 04623541).

About Epcoritamab
Epcoritamab is an IgG1-bispecific antibody created using Genmab’s proprietary DuoBody technology and administered subcutaneously. Genmab’s DuoBody-CD3 technology is designed to direct cytotoxic T cells selectively to elicit an immune response toward target cell types. Epcoritamab is designed to simultaneously bind to CD3 on T cells and CD20 on B cells and induces T-cell-mediated killing of CD20+ cells.iv

Epcoritamab (approved under the brand name EPKINLY in the U.S. and Japan, and TEPKINLY in the EU) has received regulatory approval in certain lymphoma indications in several territories. Where approved, epcoritamab is a readily accessible therapy. Epcoritamab is being co-developed by Genmab and AbbVie as part of the companies’ oncology collaboration. The companies will share commercial responsibilities in the U.S. and Japan, with AbbVie responsible for further global commercialization. Both companies will pursue additional international regulatory approvals for the investigational R/R FL indication and additional approvals for the R/R DLBCL indication.

Genmab and AbbVie continue to evaluate the use of epcoritamab as a monotherapy, and in combination, across lines of therapy in a range of hematologic malignancies. This includes four ongoing Phase 3, open-label, randomized trials, among them a trial evaluating epcoritamab as a monotherapy in patients with R/R DLBCL compared to investigators choice chemotherapy (NCT04628494), a trial evaluating epcoritamab in combination with R-CHOP in adult patients with newly diagnosed DLBCL (NCT05578976), a trial evaluating epcoritamab in combination with R2 compared to chemoimmunotherapy in patients with previously untreated FL (NCT06191744), and a trial evaluating epcoritamab in combination with lenalidomide compared to chemotherapy infusion in patients with R/R DLBCL (NCT06508658). The safety and efficacy of epcoritamab has not been established for these investigational uses. Please visit www.clinicaltrials.gov for more information.

(Press release, Genmab, DEC 8, 2025, View Source [SID1234661259])

On December 8, 2025 Galmed Pharmaceuticals Ltd. (NASDAQ: GLMD) ("Galmed" or the "Company"), a clinical-stage biopharmaceutical company for liver, cardiometabolic diseases and GI oncological therapeutics, reported the presentation of a late breaking abstract for its lead drug candidate, Aramchol at HEP-DART 2025 Meeting.

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Previously, Galmed announced that Aramchol significantly enhances Bayer’s regorafenib effect in GI cancer models to kill GI tumor cells. SCD1 inhibition augments regorafenib (Stivarga) activity through ATM-AMPK-autophagy signaling. These findings provide the scientific and translational rationale for the initiation of a Phase 1/2 clinical trial of the combination of standard of care regorafenib with the SCD1 inhibitor Aramchol in HCC and other GI cancers. Once a recommended Phase 2 dose is found, Galmed plans to add a dose expansion cohort that will include Metformin and will evaluate the 3-drugs’ combination efficacy.

"The acceptance of our late-breaking abstract to the HEP-DART prestigious scientific meeting underscores the significance of our data. The research work presented has directly informed VCU Massey Comprehensive Cancer’s decision to initiate an investigator-initiated Phase 1/2 clinical trial of Aramchol and regorafenib in advanced GI cancers, including HCC, with planned enrollment starting in 2026. Positive findings would not only lay the groundwork for subsequent accelerated clinical development of Aramchol in key three GI cancers, but could potentially expand Galmed’s oncology pipeline and create value for investors and stakeholders" said Allen Baharaff, CEO of Galmed Pharmaceuticals.

About HEP-DART

HEP-DART started in 1995 as the "FIRST International Conference on Therapies for Viral Hepatitis." Since its inception in 1995, HEP-DART has provided a cutting-edge platform for tackling challenges in drug development for viral hepatitis and chronic liver disease. The aim of HEP-DART 2025 is to assemble clinicians, researchers, and physician together to advance our knowledge of the ongoing drug development processes in the treatment of viral hepatitis, fibrosis, Metabolic Dysfunction-Associated Steatohepatitis MASH), and hepatocellular carcinoma (HCC) and to provide the scientific community with an increased understanding of the current and future challenges in therapeutics for liver infection, disease and cancer.

(Press release, Galmed Pharmaceuticals, DEC 8, 2025, View Source [SID1234661258])

On December 8, 2025 Galapagos NV (Euronext & NASDAQ: GLPG) reported new and updated Phase 2 data from the ongoing ATALANTA-1 study with its CD19 CAR T-cell therapy candidate, GLPG5101, during an oral presentation (#662) at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting.

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"The new and updated results from the Phase 2 ATALANTA-1 study demonstrate that GLPG5101 offers timely treatment with low rates of high-grade toxicities and durable responses for patients with relapsed or refractory MCL," said Marie José Kersten, MD, ATALANTA-1 Principal Investigator and Professor of Hematology at Amsterdam University Medical Center. "The short 7-day vein-to-vein time enabled a low dropout rate and eliminated the need for bridging therapy, allowing more patients to receive treatment who otherwise might not have been able to access CAR T-cell therapy."

Summary of ATALANTA-1 data from the MCL cohort (pooled data across two dose levels):
As of September 2, 2025 (data cut-off date), 26 heavily pretreated MCL patients had undergone leukapheresis and 25 had received an infusion of GLPG5101 (4% dropout rate). Of these, 24 patients received a fresh product, with 23 infused within seven days after apheresis.

Among infused patients (N=24), the objective response rate (ORR) was 100%, with a complete response rate (CRR) of 96%. Duration of response (DOR) and progression-free survival (PFS) rates were both 83% at a median follow-up of 9 months.
9 of 10 (90%) of minimal residual disease (MRD)-evaluable patients were MRD-negative at CR and 7 of 9 MRD-negative patients remained in CR at the time of the data cut-off.
GLPG5101 showed an encouraging safety profile (N=24). The most common Grade ≥ 3 treatment-emergent adverse events were hematologic. No Grade ≥ 3 CRS was observed, and only one case of Grade ≥ 3 ICANS occurred.
GLPG5101 demonstrated robust in vivo CAR T-cell expansion and long-term persistence with an enrichment of early memory phenotypes.
Intention to wind down Galapagos’ cell therapy activities
As announced on October 21, 2025, and following a comprehensive strategic and evaluation and sales process, Galapagos remains focused on the intention to wind down the cell therapy activities. This intention is subject to the conclusion of consultations with works councils in Belgium and the Netherlands, during which Galapagos will continue to operate the business and conduct ongoing clinical studies. Galapagos would still consider any viable proposal to acquire all, or part of the cell therapy business, should such a proposal emerge during the wind down process.

About GLPG5101 and ATALANTA-1 (EudraCT 2021-003272-13; NCT 06561425)

GLPG5101 is a second generation anti-CD19/4-1BB CAR-T product candidate, administered as a single fixed intravenous dose. The safety, efficacy and feasibility of decentralized manufactured GLPG5101 are currently being evaluated in the ATALANTA-1 Phase 1/2 study in eight hematological malignancies with high unmet need. The primary objective of the Phase 1 part of the study is to evaluate safety and to determine the recommended dose for the Phase 2 part of the study. Secondary objectives include assessment of efficacy and feasibility of decentralized manufacturing of GLPG5101. The dose levels that were evaluated in Phase 1 are 50×106 (DL1), 110×106 (DL2) and 250×106 (DL3) CAR+ viable T-cells. The primary objective of the Phase 2 part of the study is to evaluate the Objective Response Rate (ORR) while the secondary objectives include Complete Response Rate (CRR), duration of response, progression free survival, overall survival, safety, pharmacokinetic profile, and the feasibility of decentralized manufacturing. Each enrolled patient will be followed for 24 months. The ATALANTA-1 study is currently enrolling patients in the U.S. and Europe.

(Press release, Galapagos, DEC 8, 2025, View Source [SID1234661257])

On December 8, 2025 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived off-the-shelf cellular immunotherapies to patients for broad accessibility, reported updated clinical data from its ongoing Phase 1 trial evaluating its FT819 off-the-shelf iPSC-derived CAR T-cell program in systemic lupus erythematosus (SLE) and unveiled new preclinical data from next-generation off-the-shelf iPSC-derived CAR T-cell programs for hematologic malignancies and autoimmune diseases at the 2025 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition in Orlando, Florida.

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"We are very pleased with the accelerating patient enrollment, the expansion of U.S. clinical sites, and the addition of international clinical sites, which together are enabling broader access to FT819 for patients suffering with lupus," said Bob Valamehr, Ph.D., M.B.A., President and Chief Executive Officer of Fate Therapeutics. "The updated FT819 clinical data continue to demonstrate meaningful and durable responses with the use of less-intensive conditioning chemotherapy and a differentiated safety profile, reinforcing our goal to commence a registrational study for FT819 in 2026 and highlighting the potential of FT819 as an ideal CAR T-cell therapy for various autoimmune diseases. At the same time, our next-generation programs, FT836 and FT839, are showing substantial progress, with enhanced potency, functional persistence, and multifunctional engineering that are designed to extend the benefits of our platform across hematologic malignancies and solid tumors. These advances highlight the continued momentum of our iPSC-derived off-the-shelf CAR T-cell pipeline and our commitment to delivering scalable, on-demand and broadly accessible CAR T-cell therapies worldwide."

The clinical update includes data from the Company’s ongoing Phase 1 basket trial of FT819, its lead product candidate, across 13 enrolled patients; 12 with SLE (10 of whom have at least one month of post-treatment follow-up) and one with systemic sclerosis. The updated results demonstrate sustained clinical responses, durable B-cell depletion in a potential dose-response manner, and a differentiated safety profile without the need for intensive-conditioning chemotherapy that typically consists of multiple days of combined doses of cyclophosphamide and fludarabine. With the strength of this clinical data, the Company continues to advance preparations for a pivotal study and is engaged in discussions with the United States Food and Drug Administration (FDA) under its Regenerative Medicine Advanced Therapy (RMAT) designation regarding plans to initiate registrational trial of FT819 in 2026.

At ASH (Free ASH Whitepaper), the Company also presented new preclinical data for two next-generation iPSC-derived CAR T-cell programs designed for use in both oncology and autoimmunity. These programs demonstrate substantial improvements in functional activity and persistence, drug product consistency and uniformity, and breadth of antigen-targeting mechanisms compared with existing autologous and in vivo CAR T-cell platforms.

FT819-102 Clinical Trial Update

FT819 is an off-the-shelf CD19-targeting chimeric antigen receptor (CAR) T-cell product engineered to improve safety and efficacy. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, a precisely engineered clonal master iPSC line serves as the starting cell source to manufacture FT819, overcoming numerous limitations associated with patient- and donor-sourced CAR T-cell therapies. FT819 is well-defined and uniform in composition, produced at a low cost of goods, and can be stored in inventory for off-the-shelf, on-demand availability to potentially reach a broad patient population.

As of a November 25, 2025 data cut off date, 12 SLE patients were treated by 5 enrolling clinical sites, with 14 clinical sites in total (11 in United States and 3 in United Kingdom) now activated. Baseline characteristics were consistent with a high disease burden patient population:

median SLE duration was 8.7 years, median 7 prior therapies;
median 14 Systemic Lupus Erythematosus Disease Activity Index 2000 (SLEDAI-2K) (baseline range 8-20);
mean 2.3±0.4 Physician Global Assessment (PGA); and
mean 23±13 Functional Assessment of Chronic Illness Therapy Fatigue (FACIT-Fatigue) score.
As of an October 22, 2025 data cut off date, 10 SLE patients had ≥ 1 month follow up, with 6 of the 10 patients having active lupus nephritis. Preliminary data in Regimen A (with patients receiving a single FT819 dose after pretreatment with either a single dose of cyclophosphamide or two doses of bendamustine) show mean SLEDAI-2K score across both dose levels (DL1, 360 million cells; DL2, 900 million cells) decreased progressively from baseline:

DL1 SLEDAI-2K score decreased from a mean of 15.2 (n=5) at baseline to a mean of 10 at month 3 (n=2), and to a mean of 6 at month 6 (n=2), representing mean percent drops of 50% and 70%, respectively; and
DL2 SLEDAI-2K score decreased from a mean of 14.3 (n=3) at baseline to a mean of 6 at month 3 (n=2) and to 4 at month 6 (n=1), representing mean percent drops of 65% and 78%, respectively.
Clinical SLEDAI-2K (excluding anti-dsDNA and complement) of 0 was achieved in 5 out of 10 patients, two of whom had resumed an immunosuppressive agent that had previously failed to achieve a clinical SLEDAI-2K of 0 prior to FT819. Two lupus nephritis patients had greater than 3-month follow up, with both achieving complete renal response (CRR) at 2 months and 6 months, respectively. FACIT-fatigue scores improved meaningfully for all patients who had more than one assessment. B-cell depletion was observed, with reconstitution towards predominately naïve cells within the first 3 months. There were no observed dose limiting toxicities; no Grade >2 CRS, ICANS, or GVHD were reported. All patients were treated with FT819 that was available on-demand.

Below are links to the Company Presentations at the 2025 ASH (Free ASH Whitepaper) Annual Meeting & Exposition:

Saturday December 6, 2025

Targeting of tumor antigen CD38 and stress antigens MICA/B by CAR T cells provides a unique approach for the comprehensive treatment of multiple myeloma
Poster Presentation Number: 2350

Session Title: CAR-T Cell Therapies: Basic and Translational: Poster I

Session Time: 5:30 PM – 7:30 PM ET

Sunday December 7, 2025

Development of next generation multi-antigen targeting off-the-shelf CAR T cells for conditioning-free treatment of B-cell lymphoma
Poster Presentation Number: 4121

Session Title: CAR-T Cell Therapies: Basic and Translational: Poster II

Session Time: 6:00 PM – 8:00 PM ET

Monday December 8, 2025

The development of an off-the-shelf CAR T-cell therapy targeting CD19 and CD38 for broad application in autoimmune disease
Poster Presentation Number: 5895

Session Title: CAR-T Cell Therapies: Basic and Translational: Poster III

Session Time: 6:00 PM – 8:00 PM ET

(Press release, Fate Therapeutics, DEC 8, 2025, View Source [SID1234661256])