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.

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 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 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 Vincerx Pharma, Inc. (Nasdaq: VINC), a biopharmaceutical company aspiring to address the unmet medical needs of patients with cancer through paradigm-shifting therapeutics, and Oqory, Inc., a private biopharmaceutical company dedicated to developing next-generation antibody drug conjugates (ADCs) for the treatment of cancer, reported Phase 1a/1b data for Oqory’s anti-TROP2 ADC, OQY-3258, for patients with solid tumors (Press release, Vincerx Pharma, JAN 29, 2025, View Source [SID1234649931]). The companies also provided insights into their proposed strategic merger, which aims to advance OQY-3258 into global Phase 3 trials and build a differentiated ADC pipeline by leveraging their combined expertise.

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OQY-3258, also known as ESG401, is an anti-TROP2 ADC currently under evaluation in three clinical trials:

A Phase 1a/1b clinical trial for patients with solid tumors (NCT04892342).
A Phase 3 study in patients with locally advanced or metastatic HR+/​HER2- breast cancer (NCT06383767).
A Phase 3 study as a first-line treatment in patients with unresectable recurrent or metastatic triple-negative breast cancer (NCT06732323), which garnered Breakthrough Designation from China’s National Medical Products Administration (NMPA) on November 6, 2024.

As of August 2024, the Phase 1a/1b study had enrolled approximately 150 patients, with promising preliminary results in multiple breast cancer subtypes. Recent data highlights, including those presented at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), include:

Evaluable patients with previously untreated TNBC (n=25)
As presented at ESMO (Free ESMO Whitepaper), the confirmed overall response rate (ORR) was 76% and disease control rate (DCR) was 100%. Median duration of response (DOR) and progression-free survival (PFS) had not yet been reached.
A recent data cut from January 2025, with ten additional patients (n=35) evaluable for efficacy, showed an improved confirmed ORR of 80%, with median DOR and PFS still not reached.
Evaluable patients with late-stage TNBC (n=37)
As presented at ESMO (Free ESMO Whitepaper), the confirmed ORR was 27% and DCR was 62%, with 6-month DOR and PFS rates of 39% and 25%, respectively.
Evaluable patients with HR+/HER2- breast cancer (n=58)
As presented at ESMO (Free ESMO Whitepaper), the confirmed ORR was 39%, DCR was 78%, and 6-month DOR and PFS rates of 70% and 55%, respectively, with a median (range) PFS of 7.4 (3.7-9.2) months.
Also presented at ESMO (Free ESMO Whitepaper), OQY-3258 demonstrated meaningful activity in patients with brain metastases, reporting an intracranial ORR of 41% (n=17), which included three complete and four partial transcranial responses. Additionally, in patients with brain metastases, PFS (95% confidence interval) was 4.6 (2.0-9.8) months with OQY-3258 compared with 2.8 (1.5-3.9) months for historical data with Trodelvy. OQY-3258 has demonstrated a favorable safety profile, with the most common Grade ≥3 adverse events being manageable hematologic toxicities, such as neutropenia and leukopenia, that did not lead to treatment discontinuation. Notably, no Grade ≥3 rash or interstitial lung disease/pneumonitis was observed, and there was only one case each of Grade 3 diarrhea and stomatitis. This differentiated safety profile sets OQY-3258 apart from other TROP2 ADCs.

"The compelling clinical activity demonstrated in our Phase 1a/1b trial highlights the potential of OQY-3258 to address significant unmet needs in TROP2-expressing tumors," said Michael King, CEO of Oqory, Inc. "With its optimized serum-stable linker design, OQY-3258 has shown a markedly lower incidence of severe off-target toxicity compared with other marketed TROP2 therapies, positioning it as a differentiated late-stage ADC for metastatic breast cancer and other TROP2-expressing cancers. Our proposed merger with Vincerx represents a pivotal step toward advancing this asset into global Phase 3 trials and driving innovation in next-generation ADCs."

Raquel Izumi, Ph.D., Acting CEO of Vincerx, added, "The proposed merger with Oqory reflects our commitment to develop transformative therapies for patients with cancer. The promising efficacy and favorable safety profile demonstrated by OQY-3258 highlights its potential as a best-in-class anti-TROP2 ADC. By bringing together Vincerx’s development expertise and Oqory’s ADC technologies, we aim to accelerate the development of OQY-3258, while building a pipeline of next-generation ADCs that address significant unmet patient needs."

About Proposed Merger
Vincerx and Oqory are parties to a binding term sheet, as amended, pursuant to which Oqory would merge into Vincerx, with Oqory equity holders expected to own approximately 95% of the combined entity and Vincerx equity holders expected to hold approximately 5%. The proposed merger provides for a minimum fully diluted equity value of $13.66 million for existing Vincerx stockholders at closing and, as a condition to the closing of the merger, completion of a concurrent private offering of Vincerx equity securities of at least $20 million. Additionally, Oqory-designated investors will provide interim financing to Vincerx of $1.5 million in two tranches, approximately $1,000,000 of which was provided on December 27, 2024, and approximately $500,000 of which is to be provided on or prior to January 31, 2025. The entry into a definitive merger agreement is contingent on a number of conditions, including satisfactory completion of due diligence by Vincerx and Oqory, interim financing for Vincerx in the amount of at least $500,000 on or prior to January 31, 2025, commitments by investors for the concurrent financing, and negotiation of the terms of a definitive merger agreement. Once a definitive merger agreement is executed, the closing of any merger will be subject to customary closing conditions, including regulatory approvals, stockholder approval from both parties, completion of the minimum $20 million financing, and the continued listing of Vincerx’s common stock on Nasdaq.

The description of the binding term sheet and proposed merger contained herein is only a summary and is qualified in its entirety by reference to the binding term sheet, as amended, a copy of which has been filed by Vincerx with the Securities and Exchange Commission.

About OQY-3258 (also known as ESG401)
OQY-3258 is Oqory’s anti-TROP2 ADC with an optimized enzyme-dependent linker technology and an SN-38 payload with established efficacy and manageable side effect profile. As of August 2024, OQY-3258 has completed Phase 1a/1b development in about 150 patients with solid tumors, including metastatic HR+/HER2- and triple-negative breast cancer. OQY-3258 has shown efficacy in these patients, including reduction of brain metastasis and responses in heavily pretreated patients. To date, OQY-3258 has exhibited a differentiated safety profile versus other marketed TROP2 ADCs. Notably, no Grade ≥3 interstitial lung disease/pneumonitis or rash have been observed. Gastrointestinal effects have been mild and mainly Grade 1/2. Neutropenia and leukopenia have been the major adverse events, which were manageable and did not result in discontinuation of study drug. OQY-3258 is being evaluated in a Phase 3 study as first-line treatment in patients with unresectable recurrent or metastatic triple-negative breast cancer (NCT06732323) and in a Phase 3 study in patients with unresectable locally advanced or metastatic HR+/HER2- breast cancer (NCT06383767); both currently conducted by Shanghai Escugen Biotechnology Co., Ltd., Oqory’s development partner.