On January 29, 2025 Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients with RAS/MAPK pathway-driven cancers, reported that its management team will participate in a fireside chat at the Guggenheim SMID Cap Biotech Conference on Wednesday, Feb. 5, 2025, at 1:00 pm EST (Press release, Verastem, JAN 29, 2025, View Source [SID1234649932]).

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A live webcast of the fireside chat can be accessed under "Events & Presentations" in the Investors & Media section of the company’s website at www.verastem.com. A replay of the webcast will be archived on the website for approximately 90 days following the presentation.

On January 29, 2025 Agenus Inc. (Nasdaq: AGEN), a leader in immuno-oncology, reported publication in the Journal of Clinical Oncology showcasing data from its study of botensilimab (BOT) in combination with balstilimab (BAL) in patients with relapsed/refractory (R/R) metastatic sarcomas (Press release, Agenus, JAN 29, 2025, View Source [SID1234649933]).

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These findings further reinforce the consistency of the BOT/BAL combination, which has already shown activity and a favorable safety profile across both multiple "warm and cold" tumor types, including colon cancer, lung cancer, melanoma and ovarian cancers.

Patients with advanced sarcomas face poor outcomes and have limited treatment options, underscoring the urgent need for innovative therapies. This Phase 1 study evaluated the safety and efficacy of botensilimab (BOT), an Fc-enhanced anti-CTLA-4 antibody, in combination with balstilimab (BAL), an anti-PD-1 antibody, in this challenging patient population.

"The publication in the Journal of Clinical Oncology further underscores the significant potential of botensilimab and balstilimab to address ‘cold’ tumors like certain subtypes of refractory sarcomas," said Dr. Breelyn A. Wilky, University of Colorado Cancer Center. "These findings highlight the ability of this combination to deliver meaningful clinical benefits, including durable responses and extended survival, for patients who previously had very limited treatment options."

Publication Highlights

Study Overview

This open-label multicenter trial (NCT03860272) enrolled patients across multiple sarcoma subtypes, including angiosarcoma and leiomyosarcoma—tumor types historically resistant to traditional checkpoint inhibitors. Patients were heavily pretreated with a median of three prior lines of therapy and 15% received previous PD(L)-1 therapy.
In this expansion cohort, BOT was administered intravenously at 1 mg/kg or 2 mg/kg every 6 weeks in combination with BAL at 3 mg/kg every 2 weeks for up to 2 years.
All patients were evaluable for safety and 52 patients for efficacy.
Efficacy Highlights

Durable responses were observed across immunologically "cold" soft tissue sarcoma types, including visceral angiosarcoma and leiomyosarcoma.
Overall response rate (ORR) was 19.2% for the overall study population (n=52). Among angiosarcoma patients (n=18), ORR was 27.8%, with 33.3% in visceral and 22.2% in cutaneous subtypes.
Disease control rate (DCR) was 65.4%, with a median progression-free survival (PFS) of 4.4 months and a 36% PFS rate at 6-months.
At a median follow-up of 9.1 months, median overall survival (OS) was not reached; the 12-month OS was 69%.
Median Duration of Response (DOR) of 21.7 months, underscoring durable efficacy in heavily pretreated patients.
Safety Highlights

The BOT/BAL combination was well tolerated, with a manageable safety profile consistent with earlier findings across tumor types.
The most common treatment-related adverse event (TRAE) was diarrhea/colitis (grade 3, 6.3%), generally managed successfully with early intervention using steroids and TNF-alpha inhibitors.
No Grade 4 or 5 TRAEs were reported in this cohort.
These results add to a growing body of evidence supporting the potential of botensilimab plus balstilimab to deliver meaningful, durable benefit in multiple tumor types—especially those resistant to existing checkpoint inhibitors. As this data continues to show consistency and tolerability in colon, lung, melanoma, ovarian, and now sarcoma, it strengthens the rationale for broader investigation of this combination.

On January 29, 2025 Amgen (NASDAQ:AMGN) reported that the European Commission (EC) has approved BLINCYTO (blinatumomab) monotherapy as part of consolidation therapy for the treatment of adult patients with newly diagnosed Philadelphia chromosome-negative CD19-positive B-cell precursor acute lymphoblastic leukemia (B-ALL) (Press release, Amgen, JAN 29, 2025, View Source [SID1234649934]).

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"This approval represents a significant advancement, offering patients the opportunity to receive BLINCYTO earlier in their treatment pathway, with the potential to improve outcomes," said Jean-Charles Soria, senior vice president of Global Oncology Development at Amgen. "The E1910 data that served as the basis of this approval adds to the growing body of evidence of the meaningful survival impact of BLINCYTO."

The Phase 3 E1910 clinical trial led by ECOG-ACRIN Cancer Research Group studied patients with newly diagnosed Philadelphia chromosome-negative B-ALL receiving postinduction consolidation treatment, which aims to deepen remission to achieve durable responses. Study results demonstrated that BLINCYTO added to multiphase consolidation chemotherapy showed superior overall survival (OS) versus chemotherapy alone. With a median follow-up of 4.5 years, the 5-year OS was 82.4% in the BLINCYTO plus chemotherapy arm (n=112) and 62.5% in the chemotherapy arm (n=112).

"While there has been some treatment progress, many patients with newly diagnosed Philadelphia chromosome-negative B-ALL remain at high risk of relapse," said Robin Foà, M.D., emeritus professor of hematology, Sapienza University of Rome. "The E1910 study results highlight that BLINCYTO has the potential to advance frontline consolidation treatment, including patients who are minimal residual disease (MRD)-negative, offering a crucial new option to achieve deeper remissions and improve long-term survival."

The E1910 study was designed and conducted independently from industry. ECOG-ACRIN led the trial with public funding and sponsorship provided by the National Cancer Institute (NCI), part of the National Institutes of Health (NIH). Other NCI-funded network groups took part in the study. In addition, Amgen provided BLINCYTO and support through an NCI Cooperative Research and Development Agreement.

About Acute Lymphoblastic Leukemia (ALL)
ALL, also known as acute lymphoblastic leukemia, is a fast-growing type of blood cancer that develops in the bone marrow and can sometimes spread to other parts of the body, including the lymph nodes, liver, spleen and central nervous system. In Europe, ALL has an estimated prevalence of 1.28 persons per 100,000 people.1 Among both children and adults, the most common subtype of ALL is B-ALL.1 B-ALL begins in immature cells that would normally develop into B-cell lymphocytes, which are white blood cells that grow in bone marrow.2,3 B-ALL is the most common type of ALL, constituting approximately 75% of cases in adults.4

About BLINCYTO (blinatumomab)
BLINCYTO is the first globally approved Bispecific T-cell Engager (BiTE) immuno-oncology therapy that targets CD19 surface antigens on B cells. BiTE molecules fight cancer by helping the body’s immune system detect and target malignant cells by engaging T cells (a type of white blood cell capable of killing other cells perceived as threats) to cancer cells. By bringing T cells near cancer cells, the T cells can inject toxins and trigger cancer cell death (apoptosis). BiTE immuno-oncology therapies are currently being investigated for their potential to treat a wide variety of cancers.

In the European Union (EU), BLINCYTO is indicated as monotherapy for the treatment of:

Adults with Philadelphia chromosome-negative CD19-positive relapsed or refractory B-ALL. Patients with Philadelphia chromosome-positive B-ALL should have failed treatment with at least two tyrosine kinase inhibitors (TKIs) and have no alternative treatment options.
Adults with Philadelphia chromosome-negative CD19-positive B-ALL in first or second complete remission with MRD greater than or equal to 0.1%.
Pediatric patients aged 1 month or older with Philadelphia chromosome-negative CD19-positive B-ALL which is refractory or in relapse after receiving at least two prior therapies or in relapse after receiving prior allogeneic hematopoietic stem cell transplantation.
Pediatric patients aged 1 month or older with high-risk first relapsed Philadelphia chromosome-negative CD19-positive B-ALL as part of the consolidation therapy.
Adult patients with newly diagnosed Philadelphia chromosome-negative CD19-positive B-ALL as part of consolidation therapy.
BLINCYTO was granted breakthrough therapy and Priority Review designations by the U.S. FDA and is approved in the U.S. for the treatment of:

Adult and pediatric patients one month or older with CD19-positive Philadelphia chromosome-negative B-ALL during the consolidation phase of multiphase therapy.
CD19-positive B-ALL in first or second complete remission with MRD greater than or equal to 0.1% in adults and pediatric patients one month or older.
Relapsed or refractory CD19-positive B-ALL in adults and pediatric patients one month or older.
EU SAFETY INFORMATION FOR BLINCYTO
The safety profile of BLINCYTO in the Phase 3 E1910 study was consistent with the known safety profile for BLINCYTO.

See BLINCYTO full Summary of Product Characteristics (SmPC) at www.ema.europa.eu

About BiTE Technology
Bispecific T-cell Engager (BiTE) technology is a targeted immuno-oncology platform that is designed to engage patient’s own T cells to any tumor-specific antigen, activating the cytotoxic potential of T cells to eliminate detectable cancer. The BiTE immuno-oncology platform has the potential to treat different tumor types through tumor-specific antigens. The BiTE platform has a goal of leading to off-the-shelf solutions, which have the potential to make innovative T cell treatment available to all providers when their patients need it. Amgen is advancing multiple BiTE molecules across a broad range of hematologic malignancies and solid tumors, further investigating BiTE technology with the goal of enhancing patient experience and therapeutic potential. To learn more about BiTE technology, visit View Source

On January 29, 2025 Alligator Bioscience (Nasdaq Stockholm: ATORX) reported that clinical data for its 4-1BB agonist, ATOR-1017, has been published in the Journal for ImmunoTherapy of Cancer (JITC) (Press release, Alligator Bioscience, JAN 29, 2025, View Source [SID1234649919]). This publication validates ATOR-1017’s safety and tolerability in patients with advanced cancers and highlights early signs of clinical efficacy. Additionally, pharmacokinetic and pharmacodynamic analyses demonstrate proof of mechanism, further supporting the therapeutic potential of ATOR-1017.

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This publication adds to previously published preclinical data and underscores the scientific foundation of ATOR-1017 as an immunotherapy designed to activate T cells and natural killer (NK) cells in a tumor-specific manner. These findings strengthen the clinical package for ATOR-1017 and support its potential for future development, particularly in combination with other anticancer agents.

Dr. Sumeet Ambarkhane, Chief Medical Officer of Alligator Bioscience, commented:
"These first-in-human clinical data of ATOR-1017 in the Journal for ImmunoTherapy of Cancer demonstrate excellent safety profile and biological activity ATOR-1017. This peer-reviewed publication also reaffirms the scientific value of targeting 4-1BB pathway as a cancer immunotherapy, and further enhances its potential for future development."
Alligator remains focused on advancing its lead candidate, mitazalimab, while exploring strategic opportunities to realize the potential of ATOR-1017.

On January 29, 2025 IASO Biotherapeutics ("IASO Bio"), a biopharmaceutical company focused on the discovery, development, manufacturing, and commercialization of innovative cell therapies and biologics, reported that the Singapore Health Sciences Authority (HSA) has officially accepted the New Drug Application (NDA) for Equecabtagene Autoleucel (Press release, IASO Biotherapeutics, JAN 29, 2025, View Source [SID1234649935]). This treatment is indicated for patients with relapsed and/or refractory multiple myeloma (R/R MM) who have received three or more prior lines of therapies.

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Equecabtagene Autoleucel (trade name: FUCASO) was approved by China’s National Medical Products Administration (NMPA) on June 30, 2023, for the treatment of adult patients with relapsed or refractory multiple myeloma (R/RMM) who have received three or more lines of prior therapies, including at least one proteasome inhibitor and an immunomodulatory agent. As the world’s first commercialized fully human CAR-T product, FUCASO has gained recognition from healthcare professionals and patients in China or its remarkable efficacy and safety since its launch. Furthermore, it has also attracted patients from over ten countries to China to receive this innovative therapy.

Ms. Jinhua Zhang, Founder, Chairwoman, and CEO of IASO Bio, stated, "Expanding our global footprint is a core strategy for IASO Bio. Singapore is the first country where we have submitted an overseas NDA. The official acceptance of the NDA for Equecabtagene Autoleucel by the HSA marks a significant milestone in our journey to ‘go global.’ Equecabtagene Autoleucel has demonstrated outstanding efficacy and safety in both clinical trials and real-world settings. We will actively cooperate with the HSA throughout the regulatory process and strive to expedite the launch of this CAR-T therapy in Singapore. Upon NDA approval, we plan to implement an innovative model of ‘Manufactured in China, supplied overseas’, enabling the export of domestically produced autologous CAR-T therapies to other countries.

There is significant unmet medical need for CAR-T therapy in emerging markets. We have an experienced regulatory team for international registration and possess the capability to efficiently advance our overseas commercialization efforts. As the next step, we plan to initiate registration in multiple countries simultaneously to accelerate our global expansion.. We hope that this innovative therapy will benefit patients in more countries worldwide."

About Multiple Myeloma（MM)

Multiple myeloma (MM) is the second most common hematological malignancy in Singapore as well as globally. According to Globocan data, the global incidence of multiple myeloma in 2022 was 1.8 per 100,000 people, with a 5-year prevalence of 6.8 per 100,000. In Singapore, the incidence of MM in 2022 was 2.2 per 100,000 people, with a 5-year prevalence of 15.0 per 100,000. Despite progress in current anti-myeloma treatments, MM remains largely incurable with multiple relapses and tendency to develop refractoriness to several drug classes, presenting a major therapeutic challenge. Thus, there is an unmet need for new treatment options beyond these current anti-myeloma therapies for the treatment of relapsed or refractory MM, capable of achieving deep and durable responses.

About FUCASO(Equecabtagene Autoleucel)

FUCASO(Equecabtagene Autoleucel) is an innovative fully-human anti- BCMA CAR-T cell therapy which uses lentivirus as a gene vector to transfect autologous T cells. The CAR contains a fully human scFv, CD8a hinge and transmembrane, and 4-1BB co-stimulatory and CD3ζ activation domains. Based on rigorous molecular structure screening and comprehensive in vitro and in vivo functional evaluations, FUCASO demonstrates rapid and potent efficacy, accompanied by exceptional long-term persistence in vivo, enabling patients to achieve higher and deeper responses, providing continuous protection and care for patients with multiple myeloma.