Category: 3. Business

Results from the phase 2 PARADIGM trial (NCT04801797) that were shared at the 2025 American Society of Hematology Annual Meeting and Exposition demonstrated that treatment with azacitidine plus venetoclax (Venclexta) improved event-free survival (EFS) compared with intensive induction chemotherapy for patients with acute myeloid leukemia (AML).

In the trial, patients receiving azacitidine/venetoclax had a 39% reduction in risk of progressive disease, persistent disease prompting therapy change, relapse, hospice, or death compared with those receiving conventional intensive induction chemotherapy (HR, 0.61; P =.017). Patients also had improved quality of life (QOL) with azacitidine/venetoclax.

Induction chemotherapy with cytarabine plus anthracycline has been the standard of care for decades, but is not tolerable in older or less fit patients and can still yield suboptimal long-term outcomes in fitter patients. The hypomethylating agent (HMA) azacitidine plus the BCL2 inhibitor venetoclax was established as the regimen of choice for patients who were not eligible for induction chemotherapy based on the phase 3 VIALE-A trial (NCT02993523), but no prospective randomized trials have shown an advantage with direct comparison.

“Intensive induction chemotherapy comes with substantial burden and cost,” said Amir Fathi, MD, program director of the Center for Leukemia at the Massachusetts General Hospital Cancer Center, in a press briefing. “It’s associated with significant morbidity, including deep and prolonged marrow suppression, weeks’ long hospitalizations, frequent infections and bleeding complications, mucositis, malnutrition, deleterious psychosocial effects, and an increased risk of cardiac injury and secondary malignancies.”

PARADIGM Trial Design

The open-label, multicenter, investigator-initiated PARADIGM clinical trial randomly assigned 172 patients with previously untreated AML at 9 centers in the United States to receive induction chemotherapy or azacitidine/venetoclax. The primary end point was EFS with secondary end points including response rates, overall survival, toxicity, measurable residual disease, hospitalization metrics, and QOL. Patients were excluded if they were below 60 years old and had NPM1 mutations, or if they had core binding factor fusions or FLT3 mutations.

The median age was 64 years in the experimental arm and 65 years in the comparator arm, and 55% vs 60% were male in these respective arms. Seventy-two percent had adverse risk with 15% having intermediate and 12% having favorable risk status. The arms were balanced for risk status as well as TP53, NPM1, and IDH1/IDH2 mutations.

The induction chemotherapy used was a 7+3 regimen (cytarabine plus an anthracycline) in 54% with the remaining 46% receiving liposomal daunorubicin plus cytarabine (CPX351).

Efficacy Outcomes in PARADIGM

Results were reported as of July 25, 2025. The median number of treatment cycles was 4 with azacitidine/venetoclax vs 2 with induction chemotherapy.

The overall response rate was 88% with azacitidine/venetoclax vs 62% with the comparator (P <.001). The rate of complete response (CR), CR with partial hematologic recovery, and CR with incomplete hematologic recovery was 81% vs 55%, respectively (P <.001). Although these were statistically significant, the CR rates were not significantly different (59% vs 50%; P =.066). Both arms were allowed to proceed to hematopoietic cell transplant (HCT); 52 patients (61%) received HCT after azacitidine/venetoclax vs 34 patients (40%) after induction chemotherapy. HCT had a significant protective effect for EFS, but after adjustment in univariate and multivariate models, azacitidine/venetoclax still showed protective effect on EFS (HR, 0.67; P =.0302).

The median EFS was 14.6 months for azacitidine/venetoclax vs 6.2 months for induction chemotherapy, and the 1-year rate of EFS was 53% vs 39%, respectively, with the Cox proportional hazard model showing no age effect on EFS with the HR remaining at 0.61 (P =.018).

Fathi stated that overall survival improvement was not statistically significant, but this was a difficult end point to interpret compared with EFS because patients could benefit from receiving the trial regimens as subsequent therapy. “Even if we were to do a larger phase 3 study, overall survival, in my opinion is going to be fraught with challenge because of this extensive crossover in both directions, but mainly from intensive therapy to azacitidine/venetoclax,” he said.

Tolerability, QOL, and Hospitalization

In terms of adverse events (AEs), there were similar rates of grade 3 or 4 treatment-related AEs, which were mainly hematologic. Grade 3 or 4 lung infections were reported in 12% in the experimental arm vs 15% in the comparator arm, and grade 3/4 sepsis occurred in 7% vs 11%, respectively. There was no 30- and 60-day mortality with azacitidine/venetoclax, whereas 30-day mortality was 3.5% and 60-day mortality was 4.7% with induction chemotherapy.

Quality of life was also assessed, with patients in the experimental arm reporting significantly better QOL (P =.001) in the first 2 weeks. Symptom burden and depression symptoms were also significantly improved.

Intensive care unit care during index hospitalization was not needed in any patients receiving azacitidine/venetoclax compared with 9.8% with induction chemotherapy (P =.003). They also had fewer days of inpatient index hospitalization vs those receiving induction chemotherapy (15 vs 36; P <.001) and fewer days hospitalized in the first 6 months (41 vs 58; P <.001).

Fathi noted that those with favorable risk, such as CEBPA-associated AML and those with targetable mutations, were excluded to maintain equipoise and characterize the patient population who would likely receive HCT as consolidation. “This was really a study aimed at patients who were transplant eligible for consolidation, and in that patient population, it met its primary end point,” he said.

“We therefore believe these data support the use of azacitidine and venetoclax in functionally fit patients eligible for transplant with intermediate or adverse risk, FLT3 wild-type AML,” Fathi concluded.

Reference

Fathi A, Perl A, Fell G, et al. Results from paradigm – a phase 2 randomized multi-center study comparing azacitidine and venetoclax to conventional induction chemotherapy for newly diagnosed fit adults with acute myeloid leukemia. Presented at: American Society of Hematology Annual Meeting; December 3-7, 2025; Orlando, Florida. Abstract 6.

Fixed-duration epcoritamab (Epkinly) plus rituximab (Rituxan) and lenalidomide (Revlimid; R²) demonstrated significant improvements in progression-free survival (PFS) and response rates compared with R² alone in patients with relapsed/refractory follicular lymphoma, according to findings from the phase 3 EPCORE FL-1 trial (NCT05409066) presented in a press briefing during the 2025 American Society of Hematology (ASH) Annual Meeting and Exposition.^1,2

At a median follow-up of 14.8 months, results showed that the median PFS per independent review committee (IRC) was not estimable with epcoritamab (95% CI, NE-NE) plus R² compared with 11.7 months (95% CI, 11.1-15.1) with R² alone, leading to a 79% reduction in the risk of disease progression or death (HR, 0.21; 95% CI, 0.14-0.31; P <.0001). The concordance rate was 94% for PFS between IRC and investigator assessment. The estimated 16-month PFS rates were 85.5% (95% CI, 79.7%-89.7%) and 40.2% (95% CI, 31.8%-48.4%), respectively.

The epcoritamab regimen elicited a 95% objective response rate (ORR) compared with 79% with R² alone (P <.0001). In the epcoritamab arm, the complete response (CR) rate was 83% and the partial response (PR) rate was 12%; 1 patient had stable disease (SD), 3% of patients had progressive disease (PD), and 2% were not evaluable. In the R² arm, the CR rate was 50% and the PR rate was 29%; 7% of patients each had SD, PD, and were not evaluable.

“Epcoritamab and R² is a novel chemotherapy-free, fixed-duration therapy that is suitable for outpatient administration, and we believe sets a new benchmark as standard of care for second-line [and beyond] follicular lymphoma,” lead study author Lorenzo Falchi, MD, of the Lymphoma Service at Memorial Sloan Kettering Cancer Center in New York, NY, said in the oral presentation. “We’re happy to see that the FDA granted full approval to epcoritamab and R² on November 18, 2025, for [this patient population].”

R² is the only second-line alternative to chemoimmunotherapy-based treatment for patients with relapsed/refractory follicular lymphoma. The FDA initially approved epcoritamab, a CD3xCD20 bispecific antibody, as a single agent for the treatment of patients with relapsed/refractory follicular lymphoma following at least 2 lines of systemic therapy.³

Early data from the phase 1b/2 EPCORE NHL-2 trial (NCT04663347), which evaluated fixed-duration epcoritamab plus R² for relapsed/refractory follicular lymphoma, demonstrated deep and durable responses and a manageable safety profile.⁴

In November 2025, the FDA also approved epcoritamab in combination with R2 in relapsed/refractory follicular lymphoma, based on the phase 3 EPCORE FL-1 findings, which is what Falchi presented during the press briefing.⁵ In the international, open-label, EPCORE FL-1 trial, patients underwent a 1:1 randomization to receive fixed-duration epcoritamab at 48 mg plus R² (n = 243) or R² alone (n = 245).

Epcoritamab was administered in a 3-step-up, 2-dosing schedule at 0.16 mg on day 1, 0.8 mg on day 8, and 3 mg on day 15, all in cycle 1. Cycles 2 and 3 were administered weekly, and cycles 4 to 12 were administered every 4 weeks. Rituximab was given at 375 mg/m² for 5 cycles; the agent was given weekly in cycle 1 and every 4 weeks in cycles 2 through 5. Lastly, lenalidomide was given at 20 mg for 12 cycles; treatment was given daily in cycles 1 through 12 on days 1 through 21.

The dual primary end points were ORR and PFS, both per IRC; key secondary end points were CR rate per IRC, overall survival (OS), and minimal residual disease. Additional secondary end points were duration of response (DOR), duration of complete response, time to next lymphoma treatment (TTNLT), safety, and patient-reported outcome assessments.

The data cutoff date was May 24, 2025, and patients were enrolled between October 2022 and January 2025.

To be eligible for enrollment, patients had to have histologically confirmed CD20-positive follicular lymphoma that was grade 1 to 3a and stage II to IV. At least 1 prior treatment was required, including an anti-CD20 monoclonal antibody plus an alkylating agent, and at least 1 Groupe d’Etude des Lymphomes Folliculaires criterion needed to be met.

Patients were stratified by disease status in second line (> or ≤ 2 years since last therapy), third line (> or <6 months since last therapy), and region (US/EU vs rest of world).

Baseline characteristics showed that the median age was 61 years (range, 24-89), and 40% of patients were at least 65 years old; 57% of patients were male, and 72% were White. Thirty-one percent of patients had an ECOG performance status of 1 to 2, while 83% had Ann Arbor stage III to IV disease. Regarding Follicular Lymphoma International Prognostic Index, the breakdown of scores was 0 to 1 (24%), 2 (32%), and 3 to 5 (44%). Twenty-two percent of patients had bulky disease, consisting of at least 7 cm.

The median time from initial diagnosis to randomization was 5 years (range, 0.1-43.0), and the median number of prior lines of therapy was 1 (range, 1-7), with most patients receiving 1 (59%), 2 (24%), or at least 3 (17%). All patients received a prior anti-CD20 antibody, and 3% had received prior R². Forty-one percent of patients experienced disease progression within 2 years from starting frontline therapy; 34% of patients were refractory to their first-line therapy, and 37% of patients had double-refractory disease.

Additional efficacy data showed that the median DOR with epcoritamab plus R² was NE (95% CI, NE-NE) compared with 11.5 months (95% CI, 8.5-18.6) with R² alone (HR, 0.19; 95% CI, 0.12-0.30; P <.0001).

The epcoritamab regimen also extended TTNLT; the median was NE (NE-NE) compared with 24.3 months (95% CI, 18.2-NE) with R² alone (HR, 0.15; 95% CI, 0.09-0.27; P <.0001). At 16 months, 92.8% of patients on epcoritamab plus R² remained free from new anti-lymphoma therapy vs 64.9% of those on R² alone.

Falchi noted there is a positive trend for OS with the epcoritamab arm, which is NE (95% CI, NE-NE) in both arms (HR, 0.38; 95% CI, 0.18-0.80; P = .0039), adding that there are 10 and 25 events in the epcoritamab and R²-alone arms, respectively. Sixteen-month estimates for OS were 95.8% for epcoritamab plus R² vs 88.8% with R² alone.

Regarding safety, any-grade adverse events occurred in 100% and 99% of patients on epcoritamab plus R² and R² alone, with grade 3 or higher AEs at 90% and 68%, respectively. Serious AEs occurred in 56% and 29%, respectively, and AEs that led to treatment discontinuation occurred in 19% and 12% of patients.

Any-grade and grade 3 or higher AEs of clinical interest occurring in at least 20% of patients on epcoritamab/R² and R² alone included infections (77% and 33% vs 53% and 16%), neutropenia (74% and 69% vs 52% and 42%), cytokine release syndrome (any-grade, 35% vs <1%) thrombocytopenia (28% and 9% vs 18% and 6%), pyrexia (24% and <1%; 14% and 1%), rash (24% and 8% vs 22% and 4%), and COVID-19 (22% and 3% vs 13% and 2%).

On the epcoritamab arm, 3% of patients discontinued due to neutropenia vs 2% in the R²-alone arm. Febrile neutropenia occurred in 6% and 3% of patients, respectively. Discontinuation rates due to infections occurred in 6% and 1% of patients, respectively.

Two percent and 4% of patients on epcoritamab and R² and R² alone experienced fatal AEs.

“Despite these higher rates of AEs in the epcoritamab and R² arm, the median relative dose intensity was greater than 90%,” Falchi added.

Disclosures: Falchi cited consultancy, including expert testimony, with Johnson & Johnson, Roche, ADC Therapeutics, Evommune, F. Hoffman-La Roche Ltd., AbbVie, Sanofi, Genentech, AstraZeneca, Seagen, Merck, and Genmab; honoraria from Roche, Kite Pharma, F. Hoffman-La Roche Ltd., AbbVie, and Genmab; and research funding from Roche, F. Hoffman-La Roche Ltd., AbbVie, Innate Pharma, Genentech, BeiGene, AstraZeneca, and Genmab.

References

1. Falchi L, Nijland M, Huang H, et al. Primary phase 3 results from the EPCORE- FL-1 trial of epcoritamab with rituximab and lenalidomide (R2) versus R2 for relapsed or refractory follicular lymphoma. Blood. 2025;146(supplement 1):466. doi:10.1182/blood-2025-466
2. Falchi L, Nijiland M, Huang H, et al. Epcoritamab, lenalidomide, and rituximab versus lenalidomide and rituximab for relapsed/refractory follicular lymphoma (EPCORE FL-1): a global, open-label, randomised, phase 3 trial. Lancet. Published online December 7, 2025. doi:10.1016/50140-6736(25)02360-8
3. EPKINLY™ (epcoritamab-bysp) injection prescribing information. FDA. Updated May 2023. Accessed December 7, 2025. https://tinyurl.com/mrvfmaur
4. Falchi L, Sureda A, Leppä S, et al. Fixed-duration epcoritamab plus R² drives favorable outcomes in relapsed or refractory follicular lymphoma. Blood. 2025;146(22):2629-2640. doi:10.1182/blood.2025029909
5. FDA approves epcoritamab-bysp for follicular lymphoma indications. News release. FDA. November 18, 2025. Accessed December 7, 2025. https://tinyurl.com/y8t4hh3a

Early data from the phase 1 trial (NCT05651932) of the MMSET/NSD2 inhibitor gintemetostat (KTX-1001) revealed that the agent was tolerable and produced encouraging antitumor activity in a heavily pretreated population of patients with relapsed or refractory multiple myeloma, including those with triple-class–refractory disease and high-risk features such as t(4;14). These results were presented at the 2025 ASH Annual Meeting, and Exposition.

Among 40 patients treated with gintemetostat, 1 patient had a very good partial response, 1 patient had a partial response, 2 patients had a minimal response, and 12 patients had stable disease.

“Single-agent activity was demonstrated in heavily pretreated R/R multiple myeloma including t(4;14) positive patients across different dose-escalation cohorts,” said lead author Saad Usmani, MD, MBA, chief of Myeloma Service at Memorial Sloan Kettering Cancer Center.

Safety and Tolerability

The safety profile from the phase 1 study showed that 75% of patients experienced treatment-emergent adverse events (TEAEs) potentially related to gintemetostat and 45% experienced grade 3 or higher potentially gintemetostat-related TEAEs. Three patients had TEAEs that required a dose reduction.

Twelve patients remained on treatment at the data cutoff date of June 13, 2025. Of the 28 patients who discontinued treatment, progressive disease was the cause for 82%, physician decision for 7.1%, consent withdrawal for 7.1%, and TEAE for 3.6% (1 patient).

The most common grade 3/4hematologic TEAEs were thrombocytopenia (grade 3, 10%; grade 4, 20%), anemia (25%; 0%), neutropenia (25%; 5%), and febrile neutropenia (5%; 0%). The most common grade 3 nonhematologic TEAEs were infections (12.5%) and fatigue (10%).

There were 2 patient deaths, both of which were not related to gintemetostat treatment. One patient death was due to respiratory failure and the other was due to pleural effusion.

Study Background, Design, and Patient Characteristics

Regarding the rationale for the study, Usmani explained, “Overexpression of MMSET—also known as NSD2—often results from t(4;14) and is associated with poor clinical outcomes in patients with multiple myeloma.”

Accordingly, Usmani et al sought to explore the safety and efficacy of gintemetostat, an oral, first-in-class, potent and selective inhibitor of MMSET4 in patients with R/R multiple myeloma.

The ongoing, open-label, multicenter phase 1 dose escalation/expansion study of gintemetostat has accrued 40 patients with R/R multiple myeloma. The study is enrolling patients aged ≥18 years with R/R multiple myeloma who have received at least 3 prior therapies, including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 monoclonal antibody.

The median age of the 40 patients in the dose-escalation phase was 69 years (range, 50–83) and 52.5% of patients were female. The ECOG performance status was 0 (22.5%) and 1 (77.5%). The median time since initial diagnosis was 8 years (range, 2–20). About one-third (32.5%) of patients had extramedullary disease and 30% had high-risk multiple myeloma per IMWG criteria.

Regarding cytogenetic abnormalities, 47.5% of patients had t(4;14), comprising 20% with t(4;14) with 1q+ or del(1p32), and 27.5% with t(4;14) alone. Further, 1 patient had t(14;20), 5 had 1q21 amplification, and 4 had del(17p).

The median number of prior lines of therapy was 6.5 (range, 3–25), with 77.5% of patients having received at least 5 lines of therapy. Seventy percent of patients had prior stem cell transplant.

Prior drug classes of therapy received included IMiD/PI (100%), anti-CD38 (98%), BCMA CAR-T (42.5%), BCMA-targeted bispecific antibodies (BsAb) and antibody-drug conjugates (57.5%), GPRC5D-targeted BsAb (32.5%), and FcRH5-targeted BsAb (7.5%). All patients except 1 were triple-drug exposed and 80% were penta-drug exposed.

Overall, 9 dose levels have been assessed using a 3+3 dose-escalation design. Gintemetostat is being administered orally in a 28-day cycle. Usmani did not provide the specific details of the what the different dose levels were.

Regarding pharmacokinetics, Usmani said, “Gintemetostat plasma concentrations increased with dose across all 9 dose levels tested, and at steady-state, moderate variability in exposure of gintemetostat was observed.”

Summary and Next Steps

In a news release accompanying the presentation at the ASH meeting, a statement from Usmani summarized the findings and next steps with gintemetostat.

“In the dose-escalation phase, gintemetostat monotherapy showed a favorable safety and tolerability profile and demonstrated disease control and efficacy. Pharmacodynamic data confirm target engagement, and we look forward to advancing into the dose-expansion phase to evaluate combinations with proteasome inhibitors, IMiDs, and next-generation CELMoDs such as mezigdomide,” stated Usmani.²

References

1. Usmani S, Bories P, Gasparetto C, et al. Phase 1 study of ktx-1001, a first-in-class oral MMSET/NSD2 inhibitor, demonstrates clinical activity in relapsed/refractory multiple myeloma. Blood. 2025;146(suppl 1): 250. doi:10.1182/blood-2025-250

2. K36 Therapeutics announces presentation of First-in-Human Clinical Data for Gintemetostat (KTX-1001) Demonstrating Target Engagement and Clinical Activity in Multiple Myeloma at ASH 2025 and the Appointment of Dr. Shinta Cheng, M.D., Ph.D., as Chief Medical Officer. Published online December 5, 2025. Accessed December 7, 2025. https://tinyurl.com/3wzem4nx

Preliminary activity and favorable safety were observed among patients with heavily pretreated, triple-class refractory, relapsed/refractory multiple myeloma who received single-agent gintemetostat (KTX-1001), according to a presentation on findings from a phase 1 study (NCT05651932) at the 2025 American Society of Hematology (ASH) Annual Meeting and Exposition.¹

Among 40 patients treated with gintemetostat, 1 patient had a very good partial response, 1 patient had a partial response, 2 patients had a minimal response, and 12 patients had stable disease.

“Single-agent activity was demonstrated in heavily pretreated R/R multiple myeloma including t(4;14) positive patients across different dose-escalation cohorts,” said lead author Saad Usmani, MD, MBA, chief of Myeloma Service at Memorial Sloan Kettering Cancer Center.

Safety and Tolerability

The safety profile from the phase 1 study showed that 75% of patients experienced treatment-emergent adverse events (TEAEs) potentially related to gintemetostat and 45% experienced grade ≥3 potentially gintemetostat-related TEAEs. Three patients had TEAEs that required a dose reduction.

Twelve patients remained on treatment at the data cutoff date of June 13, 2025. Of the 28 patients who discontinued treatment, progressive disease was the cause for 82%, physician decision for 7.1%, consent withdrawal for 7.1%, and TEAE for 3.6% (1 patient)

The most common grade 3/4hematologic TEAEs were thrombocytopenia (grade 3, 10%; grade 4, 20%), anemia (25%; 0%), neutropenia (25%; 5%), and febrile neutropenia (5%; 0%). The most common grade 3 nonhematologic TEAEs were infections (12.5%) and fatigue (10%).

There were 2 patient deaths, both of which were not related to gintemetostat treatment. One patient death was due to respiratory failure and the other was due to pleural effusion.

Study Background, Design, and Patient Characteristics

Regarding the rationale for the study, Usmani explained, “Overexpression of MMSET—also known as NSD2—often results from t(4;14) and is associated with poor clinical outcomes in patients with multiple myeloma.”

Accordingly, Usmani et al sought to explore the safety and efficacy of gintemetostat, an oral, first-in-class, potent and selective inhibitor of MMSET4 in patients with R/R multiple myeloma.

The ongoing, open-label, multicenter phase 1 dose escalation/expansion study of gintemetostat has accrued 40 patients with R/R multiple myeloma. The study is enrolling patients aged ≥18 years with R/R multiple myeloma who have received at least 3 prior therapies, including a proteasome inhibitor (PI), an immunomodulatory drug (IMiD), and an anti-CD38 monoclonal antibody.

The median age of the 40 patients in the dose-escalation phase was 69 years (range, 50-83) and 52.5% of patients were female. The ECOG performance status was 0 (22.5%) and 1 (77.5%). The median time since initial diagnosis was 8 years (range, 2-20). About one-third (32.5%) of patients had extramedullary disease and 30% had high-risk multiple myeloma per IMWG criteria.

Regarding cytogenetic abnormalities, 47.5% of patients had t(4;14), comprising 20% with t(4;14) with 1q+ or del(1p32), and 27.5% with t(4;14) alone. Further, 1 patient had t(14;20), 5 had 1q21 amplification, and 4 had del(17p).

The median number of prior lines of therapy was 6.5 (range, 3-25), with 77.5% of patients having received at least 5 lines of therapy. Seventy percent of patients had prior stem cell transplant.

Prior drug classes of therapy received included IMiD/PI (100%), anti-CD38 (98%), BCMA CAR-T (42.5%), BCMA-targeted bispecific antibodies (BsAb) and antibody-drug conjugates (57.5%), GPRC5D-targeted BsAb (32.5%), and FcRH5-targeted BsAb (7.5%). All patients except 1 were triple-drug exposed and 80% were penta-drug exposed.

Overall, 9 dose levels have been assessed using a 3+3 dose-escalation design. Gintemetostat is being administered orally in a 28-day cycle. Usmani did not provide the specific details of the what the different dose levels were.

Regarding pharmacokinetics, Usmani said, “Gintemetostat plasma concentrations increased with dose across all 9 dose levels tested, and at steady-state, moderate variability in exposure of gintemetostat was observed.”

Summary and Next Steps

In a news release accompanying the presentation at the ASH meeting, a statement from Usmani summarized the findings and next steps with gintemetostat.

“In the dose-escalation phase, gintemetostat monotherapy showed a favorable safety and tolerability profile and demonstrated disease control and efficacy. Pharmacodynamic data confirm target engagement, and we look forward to advancing into the dose-expansion phase to evaluate combinations with proteasome inhibitors, IMiDs, and next-generation CELMoDs such as mezigdomide,” stated Usmani.²

References

1. Usmani S, Bories P, Gasparetto C, et al. Phase 1 study of ktx-1001, a first-in-class oral MMSET/NSD2 inhibitor, demonstrates clinical activity in relapsed/refractory multiple myeloma. Blood. 2025;146(suppl 1): 250. doi:10.1182/blood-2025-250
2. K36 Therapeutics announces presentation of First-in-Human Clinical Data for Gintemetostat (KTX-1001) Demonstrating Target Engagement and Clinical Activity in Multiple Myeloma at ASH 2025 and the Appointment of Dr. Shinta Cheng, M.D., Ph.D., as Chief Medical Officer. Published online December 5, 2025. Accessed December 7, 2025. https://tinyurl.com/3wzem4nx