On June 12, 2025 GlycoNex, Inc. (4168, hereinafter referred to as GNX), a clinical-stage biotechnology company specializing in glycan-directed cancer immunotherapies, reported that its first-in-class antibody-drug conjugate (ADC), GNX1021, has demonstrated superior efficacy in preclinical studies, including potent tumor growth inhibition in gastric cancer animal models (Press release, GlycoNex, JUN 12, 2025, View Source [SID1234653859]). GNX1021 may offer a novel treatment option for gastric cancer patients who are ineligible for HER2 or CLDN18-targeted therapies, and its broad activity suggests therapeutic potential across multiple solid tumor types.

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In safety assessments, GNX1021 has completed a pilot toxicology study in cynomolgus monkeys, demonstrating a safety profile comparable to that of currently approved ADCs. GlycoNex plans to initiate GLP-compliant toxicology studies by year-end and aims to submit clinical trial applications in Taiwan and Japan in the first quarter of 2026, with first-in-human Phase I trials anticipated to commence in the second quarter.

"With our GNX1021 program advancing rapidly and backed by our deep expertise in antibody-drug development, we are actively pursuing early-stage licensing and strategic partnerships," said Dr. Mei-Chun Yang, CEO of GlycoNex. "These partnerships will provide upfront funding, mitigate development risk, and help accelerate our path to the clinic."

Recent landmark deals in the ADC field highlight the strong licensing potential of early-stage assets, with total deal sizes reaching several billion dollars. As the global ADC market exceeded USD 160 billion in 2023, GlycoNex remains committed to seizing this significant market opportunity, driving innovation in oncology, and addressing unmet medical needs worldwide. GlycoNex will actively engage in strategic collaboration discussions with international pharmaceutical partners at the BIO International Convention in June 2025.

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.

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 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 Autolus Therapeutics plc (Nasdaq: AUTL), an early commercial-stage biopharmaceutical company developing, manufacturing and delivering next-generation programmed T cell therapies, reported updated long term data (up to approximately three years of follow up) from the FELIX study of obecabtagene autoleucel (obe-cel) in adult patients with relapsed/refractory (r/r) B-cell acute lymphoblastic leukemia (B-ALL), to be presented in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress between June 12-15, 2025, in Milan, Italy (Press release, Autolus, JUN 12, 2025, View Source [SID1234653855]). Autolus will also present an additional oral and poster presentation, the details of which are included below.

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"Obe-cel’s durability of response without any subsequent therapy in two out of every five responders is a key factor leading the transformation of therapy for adult r/r B-ALL patients. At a median follow up of 33 months, we are encouraged to see a continuation of the long-term plateau we observed at the last data cut," said Dr. Christian Itin, Chief Executive Officer of Autolus. "A well-tolerated, effective, durable treatment option for ALL patients who often have a poor prognosis and have had multiple prior treatments is of significant clinical benefit."

Oral S113:
Title: Can CAR T-cell therapy be a definitive treatment for adult r/r B-ALL without transplant? Long-term findings and predictors of sustained remission for obecabtagene autoleucel
Session Name: s447 Immunotherapy and CAR-T cells for ALL
Session room: Coral 6
Session Date and Time: Sunday, June 15; 11:00 – 12:15 CEST
Presenting Author: Jae H Park, MD

Summary: At the updated median follow up of 32.8 months, 38.4% of responders were in ongoing remission without consolidative SCT or other therapies (versus the previously reported 40% at a median follow up of 21.5 months). The 24-month probability of Event Free Survival was 43%, and for Overall Survival was 46%, with an emerging long-term plateau observed. A substantial subset of patients benefit from standalone treatment with obe-cel, achieving long-term remission. No new safety signals or Grade ≥3 secondary malignancies were observed at the extended follow-up. These results suggest that obe-cel may be a definitive treatment for some patients with r/r B-ALL – specific analysis will be needed to determine which patients may need additional treatments.

The multivariate analysis demonstrated that Philadelphia chromosome-positive disease, earlier obe-cel use, and relapsed disease correlated with achieving higher remission rates. Lower disease burden at lymphodepletion and ongoing CAR T-cell persistence were independent factors associated with long-term remission and survival.

Oral S114:
Title: Efficacy and Safety Outcomes of Obecabtagene Autoleucel (obe-cel) Stratified by Age in Patients with r/r B-ALL
Session Name: Immunotherapy and CAR-T cells for ALL
Session Room: Coral 6
Session Date and Time: Sunday, June 15; 11:00 – 12:15 CEST
Presenting Author: Bijal D. Shah, MD

Summary: Obe-cel treatment was associated with deep and durable remissions resulting in favorable overall remission rate, event free survival, and overall survival with low incidence of Grade ≥3 CRS and ICANS in both age groups (<55 years and ≥55 years). These findings indicate that obe-cel is effective and has a positive benefit and risk profile regardless of patient age, including in older adults with R/R B-ALL.

Poster PF378:
Title: Predicting Hematotoxicity Risk and Outcomes in Relapsed/Refractory B-Cell Acute Lymphoblastic Leukemia (r/r B-All): Should Hematotox Models be CAR Specific Rather than Disease Specific
Session Title: Poster Session 1
Session date and time: Friday, June 13; 18:30 – 19:30 CEST.
Presenting Author: Claire Roddie, MD

Summary: Although both the CAR-Hematotox (CAR-HT) model, and the ALL-Hematotox (ALL-HT) model show potential, ALL-HT appears to improve risk stratification and may be a better predictor of response, survival and safety outcomes in adult patients with r/r B-ALL treated with obe-cel, than CAR-HT. Taken together with other published reports, our data suggest that the strength of HT-model predictions may be CAR T-cell product specific. Further analyses are needed.