On December 8, 2025 Incyte (Nasdaq:INCY) reported updated clinical data from two Phase 1 studies evaluating the safety, tolerability and efficacy of INCA033989, a first-in-class mutant calreticulin (mutCALR)-targeted monoclonal antibody, as a treatment for patients with mutCALR-expressing myeloproliferative neoplasms (MPNs). These data, which are featured in oral presentations at the 2025 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in Orlando (Session 634, Publication #1024; Session 631, Publication #71), focus on the dose escalation portion of the studies in patients with essential thrombocythemia (ET) harboring a CALR mutation who are resistant or intolerant to at least one cytoreductive therapy.

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

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

                  Schedule Your 30 min Free Demo!

"The compelling efficacy and safety data presented today at the 2025 ASH (Free ASH Whitepaper) Annual Meeting provide additional evidence of the potential for INCA033989 to provide disease modification for high-risk ET patients harboring a CALR mutation," said Pablo J. Cagnoni, M.D., President, Head of Research and Development, Incyte. "We are proud to be advancing a portfolio of novel targeted therapies, including INCA033989, that could offer a mutation-specific treatment for patients with mutCALR-expressing MPNs."

Results presented today evaluate the safety and efficacy of INCA033989 in patients with ET, as measured by hematologic response and reduction in mutCALR variant allele frequency (VAF), and build upon previously announced data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress.

The new results (data cut-off September 25, 2025) demonstrate rapid and durable normalization of platelet counts across all dose levels in patients with ET treated with INCA033989 (n=55), with optimal responses at higher doses. Of note, most (90%) ET patients treated with starting doses of 400 to 2,500 mg (n=30) INCA033989 achieved a hematologic response. Of those, 83.3% achieved a complete hematologic response (CHR), defined as platelet count ≤400 × 109/L and leukocytes <10 × 109/L, and nearly half (46.4%) achieved a durable (≥12 weeks) CHR. Among patients treated at lower doses (24 to 250 mg, n=25), 88% achieved a hematologic response, with 68% achieving a CHR and of those 44% achieving a durable CHR.

Additionally, molecular responses were frequent, rapid, durable and correlated with hematologic responses seen with INCA033989 monotherapy. A reduction in mutCALR VAF from baseline occurred in 96.2% of ET patients with ≥1 post-baseline VAF measurement, with approximately half (52%) achieving a ≥25% best reduction in VAF and nearly one third (31%) achieving a ≥50% best reduction in VAF. Reduction in mutCALR VAF was observed within three to six months and has maintained over time in most patients, with deeper and more consistent mutCALR VAF reductions observed at higher doses of INCA033989.

Exploratory analyses in a subset of ET patients showed that INCA033989 reduced circulating mutCALR-positive hematopoietic stem and progenitor cells (HSPC) and mutCALR-positive platelet producing cells called megakaryocytes (MK) in the bone marrow, and improved MK hyperplasia. Together, these findings demonstrate the disease-modifying activity of INCA033989 in patients with mutCALR-expressing ET.

The safety analysis (n=55) showed that INCA033989 was well tolerated across all dose cohorts (24 to 2,500 mg), with no dose-limiting toxicities observed. Only one (1) patient discontinued treatment due to treatment emergent adverse events (TEAEs). One (1) dose reduction and one (1) infusion interruption due to TEAEs were reported, and a maximum tolerated dose was not reached. Fifty-three (53) patients across the dose cohorts reported a TEAE, thirty-six (36) of which were treatment related. The most common TEAEs were fatigue (30.9%), headache (27.3%), upper respiratory tract infection (27.3%) and anemia (20%). Grade >3 anemia and/or neutropenia TEAEs (>1 patient) occurred in five (5) patients (3.6% and 7.3%, respectively). No Grade ≥3 thrombocytopenia TEAEs were observed.

"Approximately 25 to 35 percent of ET patients have mutCALR-expressing ET, yet current treatments are broadly myelosuppressive, not mutant targeted and have limited efficacy in reducing mutCALR allele frequency," said John Mascarenhas, M.D., Professor of Medicine at the Icahn School of Medicine at Mt. Sinai and Director, Center of Excellence for Blood Cancers and Myeloid Disorders, The Tisch Cancer Institute. "These emerging data suggest that INCA033989 could offer a novel treatment approach by selectively targeting mutCALR in a way that enables rapid and durable hematologic responses, while maintaining safety and tolerability for ET patients who are resistant or intolerant to prior cytoreductive therapy. I’m encouraged by these findings and the potential for INCA033989 to redefine treatment paradigms for patients with ET."

INCA033989 was recently granted Breakthrough Therapy designation by the U.S. Food and Drug Administration (FDA) for the treatment of patients with ET harboring a Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy. Incyte plans to develop INCA033989 for patients with Type 1 and non-Type 1 CALR mutations and, following discussions with regulatory agencies, plans to initiate a registrational program evaluating patients with ET with a Type 1 or non-Type 1 CALR mutation who are resistant or intolerant to at least one cytoreductive therapy in the first half of next year.

More information regarding the 2025 ASH (Free ASH Whitepaper) Annual Meeting can be found on the ASH (Free ASH Whitepaper) website: View Source

About Essential Thrombocythemia
Essential thrombocythemia (ET) is a chronic myeloproliferative neoplasm (MPN) characterized by persistently elevated platelet counts due to abnormal blood cell production in the bone marrow. People living with ET are at increased risk for blood clots and bleeding and a proportion of patients may progress over time to myelofibrosis or acute leukemia.

About Mutations in Calreticulin (mutCALR)
Calreticulin (CALR) is a protein involved in the regulation of cellular calcium levels and normal protein folding. Somatic, or non-inherited, DNA mutations in the CALR gene (mutCALR) can result in abnormal protein function and lead to the development of myeloproliferative neoplasms (MPNs),1 a closely related group of clonal blood cancers in which the bone marrow functions abnormally, overproducing blood cells.2,3 Among two types of MPNs, essential thrombocythemia (ET) and myelofibrosis (MF), mutCALR drives 25-35% of all cases.2,3

Incyte is at the forefront of developing novel therapies for patients with mutCALR ET or MF that target only malignant cells, sparing normal cells, including INCA033989, a first-in-class, mutCALR-specific therapy.

About the INCA033989 Trial Program
The clinical trial program for INCA033989 includes two multicenter, open-label Phase 1 studies, INCA33989-101 (NCT05936359) and INCA33989-102 (NCT06034002). The studies are evaluating the safety, tolerability and efficacy of INCA033989 in ~455 adult (≥18 years old) patients with mutCALR-expressing myeloproliferative neoplasms (MPNs), including essential thrombocythemia (ET) and myelofibrosis (MF).

The primary endpoint of the studies is measured by the number of participants with dose limiting toxicities (DLTs), treatment-emergent adverse events (TEAEs) and the number of participants with TEAEs leading to dose modification or discontinuation. Secondary endpoints include response rates, mean change of ET total symptom score, percentage of MF patients achieving spleen volume reduction, MF patient anemia response, mean change in disease-related allele burden and various pharmacokinetics measures.

For more information on the studies, please visit: View Source and View Source

(Press release, Incyte, DEC 8, 2025, View Source [SID1234661289])

On December 8, 2025 Akeso, Inc. (9926.HK) ("Akeso" or the "Company") reported that at the 2025 ESMO (Free ESMO Whitepaper) Asia Congress, updated results from the pivotal Phase III HARMONi-6 study (AK112-306) were shared in an oral presentation by Professor Shun Lu from Shanghai Chest Hospital. The study evaluates ivonescimab (a first-in-class PD-1/VEGF bispecific antibody) combined with chemotherapy versus tislelizumab combined with chemotherapy in first-line treatment for advanced squamous non-small cell lung cancer (sq-NSCLC).

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

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

                  Schedule Your 30 min Free Demo!

Beyond the previously reported efficacy data presented at the ESMO (Free ESMO Whitepaper) 2025 Presidential Symposium and simultaneously published in The Lancet, this presentation further disclosed patient-reported quality of life outcomes based on the EORTC QLQ-C30 questionnaire.

Both prolonging survival and improving quality of life are core indicators for evaluating cancer treatments. The results published at 2025 ESMO (Free ESMO Whitepaper) Asia demonstrate that, compared to the tislelizumab-based regimen, treatment with ivonescimab plus chemotherapy not only significantly prolongs progression-free survival (PFS) but also offers better tolerability, enables higher treatment adherence, and provides patients to maintain better overall health status and quality of life over a longer period. These findings highlight the comprehensive clinical value of the ivonescimab regimen in delivering both survival and quality-of-life benefits for patients.

Quality of life (QoL) assessments from the HARMONi-6 study show that, compared with PD-1 inhibitor plus chemotherapy, ivonescimab plus chemotherapy not only significantly prolongs progression-free survival (PFS) but also helps patients maintain better overall health status. Time to deterioration in "Global Health Status/Quality of Life" was meaningfully delayed in the ivonescimab arm (HR = 0.94), indicating a trend toward reduced risk of QoL worsening versus the control group.
The ivonescimab-based regimen met the primary PFS endpoint versus the tislelizumab-based regimen, delivering a decisive, strongly positive outcome with both statistical significance and clear clinical benefit. PFS was substantially prolonged with ivonescimab plus chemotherapy compared with tislelizumab plus chemotherapy.
The hazard ratio for PFS between the ivonescimab and tislelizumab arms was 0.60 (P < 0.0001), corresponding to an absolute PFS improvement (ΔPFS) of 4.24 months (11.14 months vs. 6.90 months). This benefit was consistent across all PD-L1 expression subgroups.
The HARMONi-6 study enrolled 532 patients with well-balanced baseline characteristics. Among these patients, 92.3% had stage IV disease at enrollment. The squamous histology profile of the patients reflected real-world patterns, with approximately 63% of patients exhibiting the central squamous subtype (66.9% in the ivonescimab arm vs. 59.4% in the control arm). PD-L1 expression levels were also aligned with clinical expectations.

The results from the HARMONi-6 study further validate the breakthrough clinical value of the ivonescimab-plus-chemotherapy regimen compared to PD-1-plus chemotherapy regimen. The ivonescimab-plus chemotherapy regimen addresses a critical clinical gap when anti-angiogenic agents such as bevacizumab demonstrated severe safety considerations in the treatment of sq-NSCLC. Since ivonescimab’s initial approval in 2024, it has been evaluated in multiple clinical studies and used in real-world settings involving over 40,000 patients, where its transformative clinical benefits have been consistently demonstrated.

Across the immuno-oncology landscape, ivonescimab has shown clinical superiority to both PD-1 based treatments, which are currently the optimal standard of care for many cancers, and to also VEGF-targeted therapies in anti-angiogenesis based treatments.

In July 2025, based on the HARMONi-6 study results, the supplemental New Drug Application (sNDA) for ivonescimab in combination with chemotherapy as first-line treatment for sq-NSCLC was accepted for review by the Center for Drug Evaluation (CDE) of China’s National Medical Products Administration (NMPA). Akeso’s partner, Summit Therapeutics, is currently carrying out a global multicenter Phase III HARMONi-3 study, evaluating ivonescimab plus chemotherapy versus pembrolizumab plus chemotherapy as first-line therapy for advanced NSCLC (both squamous and non-squamous subtypes).

(Press release, Akeso Biopharma, DEC 8, 2025, View Source [SID1234661288])

On December 8, 2025 HanchorBio Inc. (TPEx: 7827), a global clinical-stage biotechnology company advancing next-generation immunotherapies for oncology and autoimmune diseases, reported new first-in-human data from its ongoing Phase 1 monotherapy study of HCB101, a 3.5th-generation SIRPα-Fc fusion protein, in relapsed/refractory non-Hodgkin lymphoma (R/R NHL) at the 67th Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper), held December 6-9, 2025, in Orlando, Florida.

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

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

                  Schedule Your 30 min Free Demo!

The ASH (Free ASH Whitepaper) Annual Meeting is the world’s premier platform for clinical and translational advances in hematology. This year, over 8,200 abstracts were accepted globally, reaffirming ASH (Free ASH Whitepaper)’s position as one of the most competitive and influential medical congresses in hematology and oncology, with historical rejection rates of approximately 28%.

The accepted abstract (#3299) features a focused sub-analysis from HanchorBio’s ongoing multinational, open-label Phase 1 study (NCT05892718) evaluating HCB101 monotherapy across solid and hematologic malignancies, highlighting results from the R/R NHL cohort.

Key Results (data cutoff: October 14, 2025):

Thirteen patients with R/R NHL received HCB101 (5.12 – 24.0 mg/kg QW). No dose-limiting toxicities (DLTs) were observed, and the maximum tolerated dose (MTD) was not reached.
All treatment-related adverse events were Grade 1-2, confirming a cytopenia-sparing safety profile.
CD47 receptor occupancy (RO) reached ≥ 75-85% at 1.2 mg/kg and ≥ 90% at 8 mg/kg, demonstrating a broad pharmacologic window.
A confirmed partial response (PR) was observed in a patient with marginal zone B-cell lymphoma at 8.00 mg/kg, with -43.3% tumor reduction at Week 8 deepening to -89.5% by Week 16 at the same dose.
The results were presented by Alvin Luk, PhD, MBA, CCRA, President & Chief Medical Officer and Chief Executive Officer (U.S.) of HanchorBio during the ASH (Free ASH Whitepaper) 2025 poster session. Dr. Luk recently joined HanchorBio to lead the company’s global development of late-stage products and U.S. operations, advancing its next-generation immuno-oncology pipeline.

"Despite ASH (Free ASH Whitepaper)’s highly competitive selection process, the inclusion of HCB101 monotherapy data reflects the strong translational foundation and clinical potential of our SIRPα-CD47 backbone," said Scott Liu, Ph.D., Founder, Chairman, and Chief Executive Officer of HanchorBio. "We’re encouraged by the favorable safety and early signs of clinical activity seen in the heavily pretreated patient population. These findings validate the translational strength of our FBDB platform, and we look forward to engaging with the global hematology community as we expand HCB101 into hematologic malignancies and macrophage/T-cell combination strategies."

"Presenting these data at ASH (Free ASH Whitepaper) marks an important step for HanchorBio and our team," added Dr. Alvin Luk. "Moving from molecular design to early clinical validation highlights the potential of selective SIRPα-CD47 blockade to achieve both safety and effective immune activation in patients with limited treatment options."

About HCB101: A Differentiated CD47-SIRPα Blockade
HCB101 is a 3.5th-generation, affinity-optimized SIRPα-Fc fusion protein with an intact IgG4 Fc backbone, developed using HanchorBio’s proprietary FBDB platform. It is engineered for selective CD47 targeting with low red blood cell (RBC) binding, thereby avoiding the anemia and thrombocytopenia commonly associated with earlier anti-CD47 monoclonal antibodies, while preserving strong antibody-dependent cellular phagocytosis (ADCP) and innate-to-adaptive immune bridging. Key differentiators of HCB101:

Enhanced safety: Cytopenia-sparing profile, with no DLTs observed up to 30 mg/kg and receptor occupancy >90% at ≥1.28 mg/kg, supporting a broad therapeutic window.
Robust immune activation: Engineered to enhance ADCP and bridge innate-to-adaptive immunity, with evidence of durable immune-mediated tumor control in monotherapy.
Broad tumor applicability: Demonstrated activity across >80 PDX and CDX preclinical models, with early clinical signals in gastric cancer, TNBC, HNSCC, non-Hodgkin lymphoma, and ovarian cancer.
Clinical translation: Shows durable disease control as monotherapy and a 100% confirmed partial response rate (6/6) in 2L gastric cancer when combined with ramucirumab and paclitaxel, with additional confirmed responses in 1L TNBC and 2L HNSCC, substantially exceeding historical benchmarks.

(Press release, Hanchor Bio, DEC 8, 2025, View Source [SID1234661287])

On December 8, 2025 TuHURA Biosciences, Inc. (NASDAQ:HURA) ("TuHURA"), a Phase 3 immune-oncology company developing novel technologies to overcome resistance to cancer immunotherapy, reported that its research on the potential role of the Delta Opioid Receptor (DOR) in controlling the immunosuppressive capabilities of MDSCs was presented in an oral presentation at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting and Exposition that took place on Sunday, December 7 in Orlando, FL. The Company presented these updated results, along with a poster presentation highlighting the effects of DOR inhibition on TAMs, another immunosuppressive cellular component critical to the tumorigenic microenvironment.

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

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

                  Schedule Your 30 min Free Demo!

In the oral presentation, entitled: Delta Opioid Receptor (DOR) Expression on Myeloid-Derived Suppressor Cells (MDSCs) Represents a Novel Target to Overcome Resistance to Immune Checkpoint Inhibitors (ICIs), Dr. Michael Turner, Vice President Immunology at TuHURA Biosciences presented updated data validating DOR expression on MDSCs and further showing that the pharmacological antagonism of DOR reduced the suppressive activity of MDSCs. MDSCs are a heterogeneous population of immature myeloid cells that contribute to creating an immunosuppressive tumor microenvironment (TME) by suppressing anti-tumor immune responses. The study showed that antagonism of the DOR with a specific inhibitor modulated a variety of direct and indirect MDSC-mediated immunosuppressive factors and reversed T cell suppression, suggesting the DOR may be a novel target to reprogram MDSC induced immunosuppression in the TME.

In the poster, entitled: Delta Opioid Receptor (DOR): A Novel Target for Reprogramming Tumor-Associated Macrophage (TAM) Immunosuppressive Phenotype to Overcome Acquired Resistance and Enhance the Effectiveness of Cancer Immunotherapies, Dr. Krit Ritthipichai, Director of Immunology at TuHURA Biosciences, presented the results of a study that showed how the DOR is highly expressed in tumor-infiltrating myeloid cells, particularly TAMs, indicating that the TME induces DOR upregulation relative to peripheral macrophages, and that targeting the DOR provides a promising strategy to reprogram suppressive TAMs and MDSCs, alleviate T-cell dysfunction, and potentially overcome resistance to checkpoint blockade and other immunotherapies.

Dr. James Bianco, President and Chief Executive Officer of TuHURA Biosciences, said "Innate immune cells, MDSCs, TAMs, and adaptive regulatory T cells (Tregs) are among the most important cellular components of the body’s immune system that provide the ability to regulate inflammation, autoimmunity and immune tolerance. Our discovery of the expression of the DOR on MDSCs and TAMs, and that its activation is coupled to mechanisms by which these cells contribute to immunosuppression, makes the DOR a compelling target for pharmacologic intervention to overcome acquired resistance to cancer immunotherapy. Data demonstrating that the DOR is also expressed on T regs and controls the expression of FOXP3, a critical immunosuppressive gene, provides a shared mechanism by which endogenous opioids, via the DOR, control the immunosuppressive tone of both innate and adaptive immune responses critical in the pathology associated with cancer and autoimmune disease. TuHURA is the first to demonstrate that this single target shares control of the immune suppressive capabilities of these cells and provides us a unique position to exploit pharmacologic modulation of the DOR to overcome resistance to cancer immunotherapy and the treatment of autoimmune and inflammatory diseases."

"The Company has developed a library of highly selective (>1,200 fold) and potent (<1.0 ng/ml) DOR antagonists and is in position to advance our first-in-class immune modulating bi-functional, bi-specific antibody drug conjugates (ADCs). We anticipate our lead ADC candidate to consist of a DOR inhibitor conjugated to our VISTA inhibiting antibody. TuHURA’s updates at this ASH (Free ASH Whitepaper) meeting demonstrate that elucidating the role of VISTA on macrophages and MDSCs in promoting the progression of myelodysplasia (MDS) to acute leukemia, as well as VISTA’s documented role in being central to how leukemia escapes immune recognition, makes it an ideal candidate to link to a DOR inhibitor. Our ADCs have the potential to not only remove the immunosuppressive tone of the TME but to also checkpoint release resting T cells, allowing them to recognize and kill leukemic cells." Dr. Bianco concluded, "We are excited to be at the forefront of these discoveries and look forward to working on the development of a whole new class of ADCs that could meaningfully change the treatment of cancer."

(Press release, TuHURA Biosciences, DEC 8, 2025, View Source [SID1234661286])

On December 8, 2025 Pierre Fabre Pharmaceuticals Inc., reported updated results presented at the 67th American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting of the pivotal Phase 3 ALLELE study of tabelecleucel in adults and children with R/R EBV+ PTLD following HCT or SOT. The two abstracts included sub-analyses of pediatric patients and by prior therapy.

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

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

                  Schedule Your 30 min Free Demo!

"These two sub-analyses highlight the potential of tabelecleucel to improve outcomes for patients, both pediatric and adult, with R/R EBV+ PTLD who after undergoing a potentially life-saving solid organ or hematopoietic cell transplant suddenly face yet another life-threatening illness," said presenter and clinical investigator, Dr. Sarah Nikiforow, Assistant Professor, Stem Cell Transplantation, Dana-Farber Cancer Institute. "The updated data support the potential of tabelecleucel as an important advancement in addressing the significant unmet need for patients with EBV+ PTLD, who currently have no FDA-approved treatment options and may experience poor overall survival of only weeks to a few months following the failure of standard treatment."

ALLELE is an ongoing multicenter, open-label Phase 3 study. This analysis encompassed a larger cohort of 86 patients (29 HCT, 57 SOT). In the study, patients received a median of two treatment cycles. Each cycle of tabelecleucel consisted of three infusions given on days 1, 8 and 15 with an imaging assessment of efficacy on day 28.

The updated findings showed patients receiving tabelecleucel had an objective response rate (ORR) of 47.7% with the HCT cohort achieving an 48.3% ORR and SOT cohort achieving a 47.4% ORR. The median (95% CI) overall survival (OS) from Kaplan-Meier survival estimates for the HCT cohort was 18.6 months. For the SOT cohort, median OS was not estimable as more than half of the patients remained in follow-up. In a sub-analysis of treatment response by prior therapy in SOT patients, ORR was 52.4% for those receiving rituximab and 44.4% for those receiving rituximab and chemotherapy.

Safety findings presented were consistent with previously published data. Serious adverse events (SAEs) were reported in 58.6% of HCT and 66.7% of SOT patients. These events were related to study treatment in 1 HCT patient and 7 SOT patients. Fatal SAEs were reported in 5 HCT and 9 SOT patients, and none were reported by investigators to be treatment related. Pyrexia was reported in 1 SOT patient and considered as a potential sign of cytokine release syndrome possibly related to treatment. In the updated ALLELE study data, there were no reports of tumor flare or infusion reactions, immune effector cell-associated neurotoxicity syndrome, or transmission of infectious diseases. No events of graft vs host disease or organ rejection were reported as tabelecleucel related.

A new sub-analysis of 12 pediatric patients less than 17 years of age found the ORR was 50.0% with complete response reported in 4 of 12 patients, and partial response observed in 2 of 12 patients. Efficacy and safety observed in the pediatric sub-group was consistent with that reported in the overall population. Six patients developed treatment emergent adverse events (TESAEs); 4 TESAEs in 2 patients were considered treatment related. Fatal SAEs were reported in 2 patients, neither considered related to treatment. There were no reports of tumor flare, infusion reactions, immune effector cell-associated neurotoxicity syndrome, graft vs. host disease, transmission of infection or bone marrow/solid organ transplant rejection.

"The growing body of evidence supporting the efficacy and safety of tabelecleucel in treatment of people living with EBV+PTLD enhances our confidence in the potential of this innovative cell therapy. These patients have undergone a difficult journey to receive a life-saving transplant only to be diagnosed with this ultra rare form of cancer and they deserve an effective treatment option following failure of standard-of-care therapy," said Adriana Herrera, Chief Executive Officer of Pierre Fabre Pharmaceuticals Inc., the Pierre Fabre Laboratories pharmaceutical subsidiary in the United States. "We look forward to our FDA target action date in January 2026 and if approved, the opportunity to address the significant unmet medical need of these patients who urgently need new treatment options."

A BLA for tabelecleucel is currently under FDA review with a target action date of January 10, 2026. The application is granted priority review for tabelecleucel indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with EBV+ PTLD who have received at least one prior therapy including an anti-CD20 containing regimen. The BLA is supported by pivotal study data from the ALLELE study and supportive data covering more than 430 patients treated with tabelecleucel.

About Tabelecleucel
Tabelecleucel is an allogeneic, off-the-shelf, EBV-specific T-cell immunotherapy designed to selectively target and eliminate EBV-infected cells. Unlike autologous CAR-T therapies, allogeneic T-cells are derived from third-party donors and are not genetically modified. T-cells are collected from the blood of healthy donors and activated by exposure to Epstein-Barr virus antigens, enriching for a population of T-cells that recognize EBV. These EBV-specific T-cells are then expanded, characterized, and cryopreserved for future use to treat patients.

About EBV+PTLD
EBV+ PTLD is an ultra-rare, acute, and potentially deadly blood malignancy that occurs after transplantation when patient T-cell immune responses are compromised by immunosuppression. It can impact patients who have undergone allogeneic HCT or SOT. Poor median survival of 3 weeks and 4.1 months for HCT and SOT, respectively, is reported in EBV+ PTLD patients for whom standard of care failed, underscoring the significant need for new therapeutic options.

(Press release, Pierre Fabre, DEC 8, 2025, View Source [SID1234661285])