On June 12, 2025 Specialised Therapeutics (ST) reported the expansion of its existing supply and distribution agreement with Incyte Biosciences International Sàrl, the Swiss-based affiliate of Incyte (NASDAQ:INCY), to launch and distribute two additional medicines from its oncology portfolio in Australia, New Zealand and Singapore, with an option to add further countries in the Asia-Pacific region (Press release, Specialised Therapeutics Australia, JUN 12, 2025, https://www.prnewswire.com/news-releases/specialised-therapeutics-expands-partnership-with-incyte-to-include-two-additional-therapies-for-hard-to-treat-conditions-302479723.html [SID1234653856]). The expanded agreement will see new therapies axatilimab (registered as Niktimvo in the United States) and retifanlimab (registered as Zynyz in the U.S. and European Union) added to the current partnered portfolio of Minjuvi (tafasitamab) and Pemazyre (pemigatinib).

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Under the terms of the expanded agreement, Incyte will be responsible for the development, manufacture and supply of both axatilimab and retifanlimab to the region, while ST will have responsibility for regulatory, distribution and local marketing and medical affairs related activities.

ST Chief Executive Officer, Mr Carlo Montagner, welcomed the expansion of the partnership agreement with Incyte, a leading global biopharmaceutical company, and the opportunity to bring these important medicines to eligible patients in the local region.

"Specialised Therapeutics has partnered with Incyte since 2021 and we are delighted to be extending this successful partnership," he said. "As part of our initial agreement, our teams have worked collaboratively to commercialise Minjuvi, which has been approved for use in Australia, and Pemazyre, which has been approved in Australia and Singapore, and we look forward to expanding the portfolio to help more patients with rare and hard-to-treat conditions."

"The addition of axatilimab and retifanlimab to the partnership agreement with Incyte recognises our strong track record of working with local stakeholders to bring innovative medicines to patients where unmet medical needs persist, despite the availability of existing treatments," said Mr Montagner. "Our focus will be on seeking regulatory and reimbursement approval for all four medicines in the region, to ensure equitable access to these treatments are available for patients as soon as practicable."

ST anticipates submitting axatilimab and retifanlimab for local regulatory and reimbursement approval in 2025.

Incyte CEO, Mr Hervé Hoppenot said the expanded partnership agreement reflected the synergies between the two companies, with a shared goal of improving outcomes for patients with unmet medical needs.

"We are pleased to extend our partnership with ST to include axatilimab and retifanlimab," he said. "ST has already demonstrated its ability to navigate complex regional regulatory pathways for Minjuvi and Pemazyre, and we look forward to continuing our work together to make these new therapies available for the oncology community in the Asia-Pacific region."

Axatilimab is a first-in-class colony stimulating factor-1 receptor (CSF-1R)-blocking antibody. It was approved by the US Food and Drug Administration (FDA) in August 2024 as a treatment for adults and children with chronic graft-versus-host disease (GVHD) who have received at least two prior treatments (systemic therapy) and require additional treatment.1 Chronic GVHD usually occurs 3 months after a transplant – typically haematopoietic stem cell or bone marrow transplantation, but occasionally also solid organ transplants – where the donor cells ("graft") attack the graft recipient’s cells ("host").2 Chronic GVHD can affect all organs, but commonly impacts the skin, mouth, eyes, lungs, stomach, bowel, and liver.2,3 The Phase II clinical trial for axatilimab involved 79 patients from 13 countries, including Australia and Singapore.4

Retifanlimab is an intravenous immune checkpoint (PD-1) inhibitor that has been approved in the US in combination with carboplatin and paclitaxel (platinum-based chemotherapy) for the first-line treatment of adult patients with inoperable locally recurrent or metastatic squamous cell carcinoma of the anal canal (SCAC) and as a single agent for the treatment of adult patients with locally recurrent or metastatic SCAC with disease progression or intolerance to platinum-based chemotherapy.5 Retifanlimab is also approved in the US6 and Europe7 for the treatment of adults with metastatic or recurrent locally advanced Merkel cell carcinoma (MCC). MCC is a rare and aggressive type of skin cancer, that is often difficult to diagnose due to the lack of specific features to distinguish it from other common skin cancers.8 Australia has the highest incidence of MCC in the world.9 The pivotal Phase II clinical trial of retifanlimab in MCC enrolled 101 chemotherapy-naïve patients from 12 countries, including Australia.10 In addition to MCC, retifanlimab, in combination with chemotherapy, is currently being investigated as a potential therapeutic option in other hard-to-treat cancers, including metastatic non-small cell lung cancer (NSCLC).

On June 12, 2025 Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, reported 5 posters presented at the EHA (Free EHA Whitepaper) 2025 annual meeting in Milan, Italy (Press release, Disc Medicine, JUN 12, 2025, View Sourcenews-releases/news-release-details/disc-medicine-presents-positive-clinical-data-updates-across" target="_blank" title="View Sourcenews-releases/news-release-details/disc-medicine-presents-positive-clinical-data-updates-across" rel="nofollow">View Source [SID1234653841]). This year’s presentations included data from HELIOS, an ongoing open-label extension study of bitopertin in EPP, which showed favorable long-term efficacy and safety with sustained protoporphyrin IX (PPIX) reductions, improvement in quality of life, and improved liver biomarkers. Disc is advancing development and registrational activities for bitopertin in EPP, with plans to submit an NDA in H2 2025. The company has initiated APOLLO, a confirmatory clinical trial of bitopertin in adults and adolescents with EPP. Disc recently launched a campaign to raise awareness of EPP among physicians, patients, and caregivers with emphasis on the causative role of PPIX accumulation in the disease. Learn more at PPIXisWhy.com.

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Disc also presented longer term data from the continuation phase of its Phase 1b trial in MF anemia, showing sustained activity on key biomarkers and durable anemia response among major responders. Enrollment for the Phase 2 RALLY-MF trial of DISC-0974 is ongoing with the exploratory cohort for patients on concomitant momelotinib or pacritinib fully enrolled. The company expects to present initial RALLY-MF data in H2 2025. Additionally, Disc shared an update of the Phase 1 healthy volunteer trial of DISC-3405, initially presented at ASH (Free ASH Whitepaper) 2024, which demonstrated deep, sustained reductions in serum iron and meaningful changes in hematologic parameters, establishing proof of mechanism. Based on these data, the company has initiated a Phase 2 trial of DISC-3405 in polycythemia vera (PV). Disc also presented an iron pulse study of DISC-3405 in healthy volunteers which showed inhibition of dietary iron uptake, further confirming the mechanism of action and supporting DISC-3405’s potential in diseases of iron overload.

The collection of data presented at EHA (Free EHA Whitepaper) adds to the evidence base supporting Disc’s continued advancement of all three clinical candidates and provides support for expansion opportunities in new indications. Management will host a call to review these updates on June 16 at 8:00 am ET. Please register for the event on the Events and Presentations page of Disc’s website (View Source)

Bitopertin, DISC-0974, and DISC-3405 are investigational agents and are not approved for use as therapies in any jurisdiction worldwide.

Summary of Poster Presentations

Bitopertin:

HELIOS:

HELIOS is an ongoing Phase 2, open-label, long-term extension trial that enrolled 86 adult and adolescent patients with EPP from the BEACON and AURORA trials. Patients were randomized to receive 20 mg or 60 mg bitopertin in BEACON and 20 mg or 60 mg bitopertin or placebo in AURORA, with all patients transitioning to a 60 mg daily dose of bitopertin in HELIOS.

Longer term treatment with bitopertin was associated with sustained reductions in the disease-causing toxin PPIX, with additional benefit for patients receiving the 60 mg dose continuously
Continuous treatment with 60 mg of bitopertin reduced ALT and other exploratory hepatobiliary biomarkers
Nearly all participants reported substantial improvements in quality of life measures
Bitopertin exhibited a favorable longer-term safety profile with up to 2+ years of exposure and similar safety across adults and adolescents with EPP and XLP
DISC-0974:

DISC-0974 Phase 1b in MF anemia:

Data from the continuation phase of the Phase 1b trial of DISC-0974 in MF anemia as of October 2024 were presented. This multi-center, open-label trial included patients who were: non-transfusion dependent receiving no transfusions (nTD, n=23), transfusion dependent with low transfusion burden (TD Low, n=5) and transfusion dependent with high transfusion burden (TD High, n=7). The trial was comprised of both patients receiving concomitant JAK inhibitor therapy (n=13) and not receiving JAK inhibitor therapy (n=22). DISC-0974 was administered subcutaneously at 14 mg (n=1), 28 mg (n=7), 50 mg (n=12), 75 mg (n=9), or 100 mg (n=6) every 4 weeks for up to 6 treatments. Long-term results demonstrated:

Sustained hepcidin suppression, iron mobilization, and reduction in Zinc PPIX, a measure of iron restricted hemoglobin production
nTD patients:
50% of evaluable patients achieved major response of sustained mean hemoglobin increase of ≥1.5 g/dL
Hematologic improvement was durable through the continuation phase
TD Low patients:
80% of evaluable patients achieved major response of transfusion independence over 16 weeks
Hematologic improvement was durable, and all major responders remained transfusion independent during the continuation phase
TD High patients:
40% of evaluable patients achieved major response of transfusion independence over 12 weeks
1 of 2 patients with a major response entered the continuation phase and remained transfusion independent at Day 225, with follow-up ongoing
DISC-0974 was safe and well-tolerated at all evaluated dose levels
DISC-0974 in combination with luspatercept and ESA (mouse model):

The effect of DISC-0974, luspatercept, and ESA as single agents and DISC-0974 combined with luspatercept or ESA on hematological parameters in wild type mice was evaluated. Results showed:

Treatment with DBIO-100 (a mouse analog of DISC-0974) suppressed hepcidin, enhanced iron availability, and boosted erythropoiesis in wild-type mice
Combining with DPO (ESA) or RAP-536 (mouse analog of luspatercept) led to additional hematological benefits beyond those agents alone
These results demonstrate that DISC-0974 has a distinct mechanism of action among anemia-targeted agents and highlight the potential for synergistic anemia benefits when combining DISC-0974 with other anemia-targeted agents.

DISC-3405:

Phase 1 Healthy Volunteer Study:

Updated SAD/MAD data from the Phase 1 trial of DISC-3405 in healthy volunteers were presented. In the SAD portion of this trial, healthy males and females ages 18 to 65 were given a single dose of placebo (n=10) or DISC-3405 at 75 mg intravenously (IV) (n=6), 37.5 mg subcutaneously (SC) (n=6), 75 mg SC (n=6), 150 mg SC (n=6), or 300 mg SC (n=6). The MAD portion included placebo (n=4), 75 mg SC (n=6), and 150 mg SC (n=6) cohorts dosed every 4 weeks for a total of 2 doses. Consistent with data presented at ASH (Free ASH Whitepaper) 2024, results showed:

DISC-3405 produced dose-related increases in serum hepcidin with corresponding reductions in serum iron across all dose levels
DISC-3405 resulted in deep reductions in serum iron (ranging from 50-80% from baseline) that were sustained and support a once-monthly SC dosing regimen
Single and repeat dosing of DISC-3405 demonstrated meaningful reductions in hematologic parameters, including reticulocyte hemoglobin, hemoglobin, and hematocrit
DISC-3405 was generally well-tolerated at all evaluated dose levels, with no serious adverse events (AEs), greater than Grade 2 AEs, or AEs leading to study withdrawal
These results provide proof of mechanism and support the advancement DISC-3405 into proof of concept studies. Disc has now initiated a Phase 2 trial of DISC-3405 in PV with initial results expected in 2026.

Iron Pulse Study:

Disc conducted an iron pulse study in healthy volunteers to evaluate the effectiveness of DISC-3405 in inhibiting dietary iron uptake. N=8 healthy volunteers received placebo treatment followed by oral ferrous sulfate tablets on two sequential occasions to establish baseline iron absorption profiles. Participants were then given a single 150mg IV dose of DISC-3405, followed by oral ferrous sulfate "iron pulses" on the 2nd and 15th days following treatment. DISC-3405 resulted in an average 94% reduction in iron absorption at Day 2 and 68% at Day 15.

These results confirm the mechanism of DISC-3405 and demonstrate its ability to block dietary iron absorption, supporting the potential for treating diseases associated with iron overload.

On June 12, 2025 Sumitomo Pharma America, Inc. (SMPA) reported that the U.S. Food and Drug Administration (FDA) granted Fast Track Designation to nuvisertib (TP-3654) for the treatment of patients with intermediate or high-risk myelofibrosis (MF) (Press release, Sumitomo Pharmaceuticals, JUN 12, 2025, View Source [SID1234653857]). The FDA Fast Track Designation is granted to investigational therapies being developed to treat serious or life-threatening conditions that demonstrate the potential to address unmet medical needs. Nuvisertib is an oral, investigational, highly selective inhibitor of PIM1 kinase, which demonstrated clinical activity including symptom and spleen responses correlating with cytokine modulation in the updated preliminary Phase 1/2 data presented at the European Hematology Association (EHA) (Free EHA Whitepaper) 2025 Congress in Milan, Italy.

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MF, a serious and rare type of blood cancer, is characterized by the buildup of fibrous tissues in the bone marrow which is caused by dysregulation in the Janus-associated kinase (JAK) signaling pathway. The clinical manifestations of MF include an enlarged spleen, debilitating symptoms and reduction in hemoglobin and/or platelets. MF affects 1 in 500,000 people worldwide.1

"This positive momentum for nuvisertib signals strong promise in our pipeline and reflects our dedication to addressing unmet medical needs on behalf of patients with myelofibrosis and their families," said Tsutomu Nakagawa, Ph.D, President and Chief Executive Officer of SMPA. "Receiving FDA Fast Track Designation for nuvisertib in the treatment of myelofibrosis reinforces our confidence in its potential as a treatment option for patients facing a poor prognosis with limited treatment options. We are committed to working closely with the FDA to progress the clinical development of nuvisertib and bring an alternative treatment option to patients with myelofibrosis."

Updated data from the ongoing Phase 1/2 study of nuvisertib in patients with relapsed/refractory MF were presented at the EHA (Free EHA Whitepaper) Congress on June 12, 2025. Preliminary data showed that nuvisertib monotherapy appears to be well tolerated with no dose-limiting toxicities (DLTs). Evaluable patients showed clinical activity including a ≥25% spleen volume reduction (SVR25) in 22.2% of patients and a ≥50% reduction in total symptom score (TSS50) of 44.4% of patients, as well as improvement of bone marrow fibrosis (42.9% patients), hemoglobin (24% patients) and platelet count (26.7% patients). Data also showed that nuvisertib treatment led to significant cytokine modulation [reduction of pro-inflammatory cytokines (e.g. EN-RAGE, MIP-1β) and increase of anti-inflammatory cytokines (e.g. adiponectin)], which demonstrated significant (p<0.001) correlation with symptom and spleen responses. Preclinical2 and emerging clinical data support the development of nuvisertib in combination with JAK inhibitors for the treatment of patients with MF.

"The data observed to date demonstrate promising clinical activity for nuvisertib and the strong potential for selective PIM1 inhibition to slow the progression of myelofibrosis," said Jatin Shah, MD, Chief Medical Officer, Oncology. "Patients with myelofibrosis are in need of new therapeutic approaches, including combination treatment options, that can provide increased and durable response rates with limited hematologic adverse events. The FDA Fast Track Designation reinforces the potential of nuvisertib to provide clinical benefits for patients with myelofibrosis, an unmet medical need."

About Nuvisertib (TP-3654)
Nuvisertib (TP-3654) is an oral investigational selective inhibitor of PIM1 kinase, which has shown potential antitumor and antifibrotic activity through multiple pathways, including induction of apoptosis in preclinical models.2,3 Nuvisertib was observed to inhibit proliferation and increase apoptosis in murine and human hematopoietic cells expressing the clinically relevant JAK2 V617F mutation.3 Nuvisertib alone and in combination with ruxolitinib showed white blood cell and neutrophil count normalization, and also reduced spleen size and bone marrow fibrosis in JAK2 V617F and MPLW515L murine models of myelofibrosis.2 The safety and efficacy of nuvisertib is currently being clinically evaluated in a Phase 1/2 study in patients with intermediate and high-risk myelofibrosis (NCT04176198). The U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to nuvisertib for the indication of myelofibrosis in May 2022. The Japan Ministry of Health, Labour and Welfare (MHLW) granted Orphan Drug Designation to nuvisertib for the treatment of myelofibrosis in November 2024.

On June 12, 2025 Enterome SA, a clinical-stage company developing first-in-class OncoMimics immunotherapies to treat cancer, reported it has raised $19 million in a new private financing to advance its lead clinical program EO2463 OncoMimics immunotherapy to treat indolent non-Hodgkin lymphoma (iNHL) (Press release, Enterome, JUN 12, 2025, View Source [SID1234653842]). The new funds will be used to expand and finalize the ongoing Phase 1/2 SIDNEY clinical trial of EO2463 and prepare the candidate for a registrational trial.

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New U.S. investor The Institute for Follicular Lymphoma Innovation (IFLI), a global non-profit foundation dedicated to advancing research and treatment for follicular lymphoma, invested $9 million in the round, of which $5 million will be allocated to Enterome upon closing and an additional $4 million in conditional tranched funding.

Existing shareholders invested an additional $10 million including: SymBiosis, a U.S. venture capital firm; Seventure Partners, based in France; Lundbeckfonden BioCapital from Denmark; Primo Capital, an Italian venture capital and private equity firm; and The U.S. Leukemia & Lymphoma Society Therapy Acceleration Program (LLS TAP).

"Attracting highly specialized blood cancer investor IFLI to this financing demonstrates the conviction of our new and existing investors in the potential of OncoMimics for blood and solid tumor cancers," said Pierre Bélichard, Enterome’s Chief Executive Officer. "We currently are generating exciting clinical proof of concept data for EO2463 monotherapy in several iNHL patient populations included in the Phase 1/2 SIDNEY clinical trial. Most importantly, EO2463 has shown robust clinical efficacy and exceptional safety and tolerability – which is especially impressive for such a potent immunotherapy. This offers a new hope for these patients and a rare opportunity to create an entirely new market segment for an impactful therapeutic. This financing will enable us to continue the SIDNEY trial of EO2463 and prepare to launch a first pivotal Phase 3 trial of this candidate for the ‘watch-and-wait’ iNHL population."

The company presented interim SIDNEY dataat the American Society of Hematology (ASH) (Free ASH Whitepaper) conference in December 2024, showing highly encouraging responses in the Cohort 2 of "watch and wait" iNHL patients in the ongoing SIDNEY study. This population, as the name suggests, is generally not eligible to receive other treatments due to the unacceptable risk-benefit ratio (in this iNHL sub-population) of the most commonly used blood cancer therapies.

The company also recently disclosed having held positive meetings with both FDA (Type C meeting) and EMA (Scientific Advice), outlining a clear regulatory path to registration for marketing authorizations in "watch-and-wait" iNHL.

"This investment aligns with IFLI’s mission to accelerate the development of innovative therapies and precision biomarkers for follicular lymphoma," said Michel Azoulay, MD, Chief Medical Officer at IFLI. "EO2463 represents a novel class of synthetic, off-the-shelf Immunotherapeutics with a unique mechanism of action that selectively targets malignant B cells. We are particularly interested in supporting Enterome’s efforts to demonstrate EO2463’s clinical efficacy across multiple lines of therapy, including in relapsed and refractory settings."

Enterome recently announced that it will present new data showing EO2463 also has a meaningful impact when tested in combination with standard of care in relapsed and refractory iNHL patients at the International Conference on Malignant Lymphoma (ICML) in Lugano on June 21. Previous findings presented at ASCO (Free ASCO Whitepaper) in 2024 in the relapsed and refractory patient population further suggested the potential to identify individuals most likely to benefit from EO2463 treatment, supported by biomarker analyses.

Lore Gruenbaum, Chief Scientific Officer at LLS, said, "It is important for us to continue to support Enterome, a company working to develop novel therapeutics based on our shared commitment to create better therapies for blood cancers. LLS has invested over $1.8 billion in groundbreaking research since our inception in 1949. Our active partnership with Enterome, through our Therapy Acceleration Program, will continue to advance the clinical development of the OncoMimics family of novel immunotherapeutics for the benefit of blood cancer patients. We are particularly excited to help advance EO2463 which has shown promising signs of efficacy as monotherapy with excellent safety and tolerability in ‘watch-and-wait’ iNHL patients, who currently have no approved treatment options."

EO2463 is an innovative, off-the-shelf immunotherapy candidate that combines four synthetic OncoMimics peptides. These non-self, microbial-derived peptides correspond to CD8 HLA-A2 epitopes that mimic the B lymphocyte-specific lineage markers CD20, CD22, CD37, and CD268 (BAFF receptor). EO2463 also includes the helper peptide (CD4+ epitope) universal cancer peptide 2 (UCP2). The unique ability of EO2463 immunotherapy to selectively target multiple B cell markers enables the destruction of malignant B lymphocytes. By ensuring broad target coverage across malignant B cells, this novel approach aims to simultaneously improve safety and maximize efficacy, reducing the tumor cells’ capacity to develop immune-resistance mechanisms such as antigen escape.

SIDNEY is an ongoing 12-month open label Phase 1/2 study that aims to assess safety, tolerability, immunogenicity, and preliminary efficacy of EO2463 monotherapy and combination therapy in up to about 55 patients with follicular lymphoma and marginal zone lymphoma including divided into three cohorts: newly diagnosed patients eligible to watch-and-wait (monotherapy); newly diagnosed patients in need of therapy / first line (combo with rituximab); patients with relapsed/refractory disease (combo with R2). In addition to safety, survival, response rates and other measures of efficacy are being collected.

OncoMimics were inspired by the microbial origin of certain autoimmune diseases. The Company uses AI and machine learning to identify microbial proteins that closely mimic the structure, effect or actions of specific cancer antigens (as well as hormones or cytokines). Memory T cells against microbial antigen are created during early development, sometimes leading to autoimmune disorders. In the case of OncoMimics, however, this means that the immune system can mount a rapid, robust and durable immune response that is highly targeted and specific for the OncoMimics and the cancer antigens they closely resemble.

This is possible because, unlike cancer antigens, OncoMimics bypass the biological process, known as thymic deletion, that prevents the immune system from mounting an attack against the "self" proteins (e.g. antigen) on tumor and blood cancer cells. Once activated, the immune system attacks with high specificity and potency the cancer antigens targeted by the OncoMimics, killing the cancer cells that carry them. OncoMimics are synthetic peptides that are easy to manufacture, store, distribute and administer as an off-the-shelf subcutaneous injection. In clinical testing to date they have been shown to be extremely well tolerated, especially compared to other potent immunotherapies.

On June 12, 2025 Johnson & Johnson (NYSE: JNJ) reported new Phase 1b data showing encouraging antileukemic activity and a promising safety profile for bleximenib (JNJ-75276617) in combination with venetoclax and azacitidine (VEN + AZA) for the treatment of acute myeloid leukemia (AML) harboring KMT2A gene rearrangements (KMT2Ar) or NPM1 gene mutations (NPM1m) (Press release, Johnson & Johnson, JUN 12, 2025, View Source;johnsons-bleximenib-demonstrate-promising-antileukemic-activity-in-combination-with-venetoclax-and-azacitidine-for-acute-myeloid-leukemia-302480190.html [SID1234653858]). The study evaluated patients with newly diagnosed, intensive chemo-ineligible AML and relapsed or refractory AML.1 The results were featured in an oral presentation at the 2025 European Hematology Association (EHA) (Free EHA Whitepaper) Congress (S137).

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Even though AML is the most common type of acute leukemia in adults, it has the lowest survival rate and is associated with poor patient outcomes, despite treatment advances to date – especially for patients with KMT2Ar and NPM1m.

"AML encompasses a spectrum of genetically diverse cancers affecting the bone marrow and blood, which progress rapidly, making it an extremely challenging cancer to treat," said Andrew M. Wei*, MBBS, PhD, Peter MacCallum Cancer Centre, Royal Melbourne Hospital, Walter and Eliza Hall Institute of Medical Research and University of Melbourne, Australia. "These data highlight the potential of this targeted therapy in combination with VEN + AZA for patients with newly diagnosed AML who are ineligible for intensive chemotherapy or with disease that has relapsed after prior therapy."

The Phase 1b dose-finding study (NCT05453903) evaluated 125 patients with relapsed or refractory AML and newly diagnosed, intensive chemo-ineligible AML who harbored KMT2Ar (n=52) or NPM1m (n=73). Bleximenib in combination with VEN + AZA was evaluated across multiple dose levels without step-up dosing. Of the 85 relapsed or refractory patients, 36 percent received one, 42 percent received two and 12 percent received three lines of prior treatment; 47 percent had previously been treated with venetoclax.1

The bleximenib data at 100 mg twice a day in combination with VEN + AZA showed higher efficacy and a similar safety profile in comparison to other dose levels. At the recommended Phase 2 dose (RP2D), patients with relapsed or refractory AML achieved an overall response rate (ORR) of 82 percent and a composite complete response (cCR) rate of 59 percent.1 The newly diagnosed, intensive chemo-ineligible patient population showed an ORR of 90 percent and a cCR rate of 75 percent.1

Safety analysis of the study population showed a profile comparable among dose groups, genetic subtypes and disease settings. At the RP2D in combination with VEN+AZA, differentiation syndrome events were reported in two of 49 patients (4 percent). Bleximenib safety data continued to support a lack of QTc prolongation signal, with no events of Grade 3 or higher and only three Grade 1 events (6 percent) at the RP2D.1 The most common all-grade treatment-emergent adverse events (TEAEs) were nausea (65 percent), thrombocytopenia (61 percent), neutropenia (59 percent) and anemia (49 percent).1 The most common Grade 3 or higher TEAEs were thrombocytopenia (59 percent), neutropenia (59 percent), and anemia (49 percent).1

"Building on our heritage of leadership and innovation in hematologic malignancies, we are committed to delivering transformative treatment options that address the significant unmet needs of patients with acute myeloid leukemia," said Jeffrey Infante, M.D., Vice President of Early Clinical Development and Translational Research at Johnson & Johnson Innovative Medicine. "We continue to explore the potential of this compound as a monotherapy and in combination with standard of care regimens in additional Phase 2 and 3 studies, which are currently enrolling patients."

About Phase 1b Bleximenib Combination Dosing Study

This bleximenib combination trial (NCT05453903) is an ongoing Phase 1b open-label, non-randomized sequential assignment multicenter study to determine the recommended Phase 2 dose (RP2D) and further evaluate the safety and tolerability of bleximenib in combination with VEN + AZA in approximately 200 patients with either newly diagnosed or relapsed/refractory acute myeloid leukemia harboring KMT2A or NPM1 alterations.

Patients received VEN + AZA in combination with oral bleximenib twice daily at 15–150 mg (relapsed/refractory) or 30–100 mg (newly diagnosed) over a 28-day cycle and during count recovery. Bleximenib was started on day 4 without the need for step-up dosing. Primary outcome measures included adverse events and dose-limiting toxicity. Secondary efficacy measures included depletion of leukemic blasts, percentage of patients achieving complete response (CR), and percentage of patients who achieve overall response.

About Bleximenib (JNJ-75276617)

Bleximenib is an investigational oral menin inhibitor being evaluated for the treatment of patients with newly diagnosed and relapsed or refractory AML. It targets a key oncogenic interaction between menin and KMT2A fusion proteins, disrupting a pathway that drives leukemic cell growth in patients with KMT2Ar or NPM1m mutations.

It is currently being investigated in Phase 1, 2, and 3 trials, both as a monotherapy and in combination with AML-directed therapies to further explore its potential in both relapsed or refractory and newly diagnosed AML populations.

About Acute Myeloid Leukemia (AML)

Acute myeloid leukemia is an aggressive, fast-growing blood cancer that originates in the bone marrow and is marked by the uncontrolled proliferation of immature white blood cells known as myeloblasts.2, 5 These malignant cells crowd out healthy blood-forming cells, leading to complications such as anemia, infections and bleeding.6 Acute myeloid leukemia progresses rapidly, often requiring immediate treatment after diagnosis.5 It is the most common type of acute leukemia in adults, with a median age of diagnosis around 70 years.2

Despite treatment advances, acute myeloid leukemia remains associated with poor patient outcomes, particularly in older adults or those with high-risk genetic profiles.7 The five-year survival rate remains the lowest among leukemias, with outcomes especially poor in patients with KMT2Ar or NPM1m where relapse/refractory disease survival can be as short as 2 to 3 months after a second relapse – highlighting a significant unmet medical need.