On September 30, 2025 AbbVie (NYSE: ABBV) reported submission of a new Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of investigational Pivekimab sunirine (PVEK) for treatment of Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN) (Press release, AbbVie, SEP 30, 2025, View Source;an-investigational-antibody-drug-conjugate-adc-to-treat-rare-cancer-with-limited-treatment-options-302570103.html [SID1234656356]). The submission is based on data from the Phase 1/2 CADENZA trial, a global study evaluating the safety and efficacy of PVEK in BPDCN. BPDCN is a rare and aggressive blood cancer that has features of both leukemia and lymphoma. Patients typically present with skin lesions and the disease often spreads to the bone marrow, central nervous system and the lymph nodes. First-line treatments are typically intensive chemotherapy and often followed by stem cell transplant. The need for additional and innovative treatment is high for both newly diagnosed patients and for those whose prior treatments have resulted in relapsed or refractory disease.

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PVEK is a CD123-targeting Antibody-Drug Conjugate (ADC) in clinical development for hematological malignancies (blood cancers), including BPDCN and acute myeloid leukemia (AML). ADCs are designed to deliver potent cancer cell death-inducing agents called ‘payloads’ directly to the cancer cells expressing a specific protein. CD123 (IL-3Rα) is a protein overexpressed in BPDCN, making it an ideal target for therapy.

"Meaningful innovations in cancer research and treatment are happening every day. It is important that these innovations reach patients who desperately need them, including those with rare cancers who have limited options," said Roopal Thakkar, executive vice president, research and development and chief scientific officer, AbbVie. "We look forward to next steps in the regulatory process for our latest Antibody-Drug Conjugate (ADC), our first ADC in blood cancer, and how it may advance treatment for those living with Blastic Plasmacytoid Dendritic Cell Neoplasm (BPDCN)."

About the CADENZA Trial
CADENZA is a Phase 1/2 multicenter, open-label study designed to determine the maximum tolerated dose (MTD), recommended Phase 2 dose (RP2D), and recommended dosing schedule for PVEK monotherapy and to assess the safety, tolerability, PK, immunogenicity, and antileukemia activity of PVEK when administered to subjects with CD123+ hematologic malignancies (including subjects with BPDCN, AML, and others).

About Pivekimab Sunirine (PVEK)
PVEK is a CD123-targeting ADC in clinical development for hematological malignancies, including blastic plasmacytoid dendritic cell neoplasm (BPDCN) and acute myeloid leukemia (AML). PVEK is currently being evaluated as monotherapy for patients with BPDCN and in combination with Vidaza (azacitidine) and Venclexta (venetoclax) for patients with untreated and relapsed/refractory AML. In October 2020, the FDA granted PVEK Breakthrough Therapy designation in relapsed/refractory BPDCN.

On September 30, 2025 Astellas Pharma Inc. (TSE: 4503, President and CEO: Naoki Okamura, "Astellas") reported it will present ten abstracts from our portfolio and advancing pipeline at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) congress taking place from 17-21 October 2025, including new data for PADCEV (enfortumab vedotin) plus pembrolizumab in muscle-invasive bladder cancer (MIBC) which will be presented in an ESMO (Free ESMO Whitepaper) Presidential Symposium on 18 October (Press release, Astellas, SEP 30, 2025, View Source [SID1234656355]). In prostate cancer, we are sharing final best-in-class overall survival data for XTANDI (enzalutamide) in high-risk, biochemically recurrent non-metastatic hormone sensitive disease, plus encouraging data for our next-generation bispecific T cell engager ASP2138 in solid tumors, demonstrating continued leadership in CLDN18.2-targeted precision medicine.

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Moitreyee Chatterjee-Kishore, Ph.D., M.B.A., Head of Oncology Development, Astellas
"Astellas focuses on some of the most complex and devastating cancers. These ESMO (Free ESMO Whitepaper) data demonstrate our ‘bench to bedside’ approach in action – from deep disease biology to measurable improvements in patient outcomes. We’re proud to share breakthrough new survival data in muscle-invasive bladder cancer and overall survival data in hormone sensitive prostate cancer, which reflect potentially practice-changing advances that could transform outcomes for patients who need them most. We also continue to advance innovation in gastric and gastroesophageal junction cancer with new clinical data for our next-generation investigational bispecific T cell engager."

Highlights from Astellas at ESMO (Free ESMO Whitepaper) 2025 will include:

Data from the Phase 3 EV-303 (also known as KEYNOTE-905) clinical trial which will be featured in an ESMO (Free ESMO Whitepaper) Presidential Symposium, evaluating enfortumab vedotin in combination with pembrolizumab as neoadjuvant and adjuvant treatment (before and after surgery) versus surgery alone, the current standard of care, in patients with MIBC who are not eligible for or declined cisplatin-based chemotherapy.
Long-term follow-up data from the EV-302 clinical trial exploring the utility of enfortumab vedotin in combination with pembrolizumab for patients with challenging baseline characteristics, including older patients with locally advanced or metastatic urothelial cancer and those with comorbidities such as diabetes and chronic kidney disease.
Final data from the Phase 3 EMBARK trial assessing overall survival with enzalutamide in combination with leuprolide and as monotherapy in patients with non-metastatic hormone-sensitive prostate cancer (nmHSPC; also known as non-metastatic castration-sensitive prostate cancer or nmCSPC) with high-risk biochemical recurrence.
First clinical data from Astellas’ investigational CLDN18.2-targeted, next-generation bispecific CD3 T cell engager ASP2138, both as a monotherapy and in combination with standard of care therapy.
Astellas Presentations at ESMO (Free ESMO Whitepaper) Congress 2025

Enfortumab vedotin

Presentation Title

Presenter

Presentation Details

Perioperative enfortumab vedotin plus pembrolizumab in participants with muscle-invasive bladder cancer who are cisplatin-ineligible: The phase 3 KEYNOTE-905 study

C. Vulsteke

Type: Presidential Symposium 1

Abstract Number: LBA2

Date: October 18,
16:52 – 17:04 CEST

Enfortumab vedotin and pembrolizumab in previously untreated locally advanced or metastatic urothelial cancer: An exploratory analysis in older patients and those with comorbidities from EV-302

N. Mar

Type: Poster

Abstract Number: 3073P

Date: October 18,
12:00-12:45 CEST

Enfortumab vedotin plus pembrolizumab as first-line treatment in recurrent or metastatic head and neck squamous cell carcinoma: Results from a cohort of the EV-202 trial

P.L. Swiecicki

Type: Mini oral

Abstract Number: 1329 MO

Date: October 19,
17:38-17:43 CEST

EV-103 Cohort K: Efficacy and safety of enfortumab vedotin with or without pembrolizumab in cisplatin-ineligible pts with previously untreated locally advanced or metastatic urothelial cancer with a median follow-up of ≈3.5 y

T.W. Friedlander

Type: Poster

Abstract Number: 3074P

Date: October 18,
12:00-12:45 CEST

Real-world use of enfortumab vedotin in patients with locally advanced or metastatic urothelial cancer previously treated with chemotherapy and immunotherapy in France

A. Fléchon

Type: ePoster

Abstract Number: 3111eP

Date: October 18, 12:00-12:45 CEST

Enzalutamide

Presentation Title

Presenter

Presentation Details

Overall survival in EMBARK, a phase 3 randomised trial of enzalutamide or placebo plus leuprolide and enzalutamide monotherapy in patients with nonmetastatic hormone-sensitive prostate cancer with biochemical recurrence at high risk for metastasis

S.J. Freedland

Type: Proffered Paper

Abstract Number: LBA87

Date: October 19,
10:55-11:05 CEST

Baseline features and metastasis-free survival by prior definitive treatment in patients with high-risk biochemically recurrent prostate cancer: EMBARK post hoc analysis

N.D. Shore

Type: Poster

Abstract Number: 2461P

Date: October 18,
12:00-12:45 CEST

Pipeline

Presentation Title

Presenter

Presentation Details

ASP2138 monotherapy in patients with (CLDN18.2)+, advanced solid tumors: Phase 1/1b trial

K. Shitara

Type: Poster

Abstract Number: 2137P

Date: October 19,
12:00-12:45 CEST

ASP2138 monotherapy or in combination with pembrolizumab and mFOLFOX6 or with ramucirumab and paclitaxel in (CLDN18.2)+ locally advanced unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma: Phase 1/1b trial

F. Dayyani

Type: Poster

Abstract Number: 2136P

Date: October 19,
12:00-12:45 CEST

Phase 1 trial of ASP5541 (PRL-02), a long-acting intramuscular depot injection of abiraterone decanoate, in patients with advanced prostate cancer

J. Avitia

Type: Poster

Abstract Number: 2443P

Date: October 18,
12:00-12:45 CEST

On September 30, 2025 Oryzon Genomics, S.A. (ISIN Code: ES0167733015, ORY), a clinical-stage biopharmaceutical company and European leader in epigenetics, reported that the European Patent Office (EPO) has issued a Decision to Grant for its patent application EP20712594.9, entitled "Combinations of iadademstat for cancer therapy" (Press release, Oryzon, SEP 30, 2025, View Source;utm_medium=email&utm_campaign=NdP.30+2025-10-01+EPO+allowance+combos+ICI+ENG784 [SID1234656354]).

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The allowed claims protect the use of iadademstat in combination with PD1 or PD-L1 inhibitors for the treatment of cancer, including small cell lung cancer (SCLC). Once formally granted, the patent will remain in force until at least 2040, excluding potential patent term extensions. Corresponding patents have already been allowed or granted in Australia and Russia, with additional applications pending in the United States, Japan, China, and other territories.

"We are delighted to have secured this grant, which strengthens protection for iadademstat in combination with immune checkpoint inhibitors such as atezolizumab and durvalumab, a therapeutic approach we are actively pursuing in clinical trials", said Neus Virgili, Oryzon’s Chief IP Officer.

Iadademstat is currently being investigated in combination with PD-L1 inhibitors (atezolizumab or durvalumab) in first line, extensive-disease SCLC patients in a Phase I/II trial conducted and sponsored by the U.S. National Cancer Institute (NCI) under a Cooperative Research and Development Agreement (CRADA) with Oryzon. More than 30 sites accross the U.S. participate in the trial, including leading institutions such as Memorial Sloan Kettering Cancer Center, Johns Hopkins, City of Hope, Yale University and the University of Chicago, among others.

On September 30, 2025 SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) ("SELLAS" or the "Company"), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, reported that it will host a virtual R&D Day on Wednesday, October 29, 2025 at 10:00 AM ET featuring key opinion leaders (KOLs), alongside company management, to discuss the unmet medical need and evolving treatment landscape for acute myeloid leukemia (AML) (Press release, Sellas Life Sciences, SEP 30, 2025, View Source [SID1234656352]).

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The event will feature an overview of the ongoing Phase 3 REGAL trial of GPS (results expected by year-end) and a discussion of the unmet needs of AML patients in complete second remission (CR2). SELLAS will also present an update of SLS009, its highly selective cyclin-dependent kinase 9 (CDK9) inhibitor, highlighting recently reported Phase 2 data and plans for a newly diagnosed and frontline AML study anticipated to begin in the first quarter of 2026.

A live Q&A session will follow the formal presentations.

About Omer Jamy, MD
Omer Jamy, MD is Assistant Professor of Medicine at the University of Alabama (UAB) in the Division of Hematology and Oncology and Associate Scientist, Experimental Therapeutics at the O’Neal Comprehensive Cancer Center. He serves as principal investigator of the Phase 3 REGAL study at UAB, one of the trial’s highest enrolling sites, and leads several clinical trials in addition to REGAL, focusing on AML, chronic myelogenous leukemia, and allogeneic stem cell transplantation. Dr. Jamy completed his internal medicine residency at the University of Tennessee in Memphis followed by fellowship training at UAB in hematology/oncology, bone marrow transplantation and cellular therapy.

About Panagiotis Tsirigotis, MD, PhD
Panagiotis Tsirigotis, MD, PhD is Professor of Hematology at the National and Kapodistrian University of Athens, School of Medicine in Athens, Greece, and Scientific Director of the Transplantation Program of the Hematology Unit since 2010. He is an investigator in the Phase 3 REGAL trial and has enrolled the highest number of patients in the study. His clinical work focuses on AML with particular emphasis on cellular therapies and hematopoietic cell transplantation. Dr. Tsirigotis’ research focuses on the application of immunotherapy methods to prevent leukemia relapse after allogeneic transplantation, such as the administration of donor lymphocytes, as well as on mechanisms of immune escape in hematologic malignancies. He is the president of Acute Leukemia Working Party of the Hellenic Society of Hematology, and Vice President of the Hellenic Transplant Organization.

About Philip Amrein, MD
Philip Amrein, MD is Assistant Professor of Medicine at Harvard Medical School and a physician at the Massachusetts General Hospital (MGH), where he is part of the Cancer Center, Leukemia, Cellular Immunotherapy, and Hematology/Oncology departments. Dr. Amrein specializes in treating adults with acute and chronic leukemias, myelodysplasia, and myeloproliferative neoplasms, and leads numerous clinical trials exploring novel treatment approaches. Dr. Amrein has conducted research with SLS009 and is an expert in cyclin-dependent kinases (CDK).

About Sharif Khan, MD
Sharif Khan, MD is a hematologist at Bon Secours Health System in Greenville, SC. He is a highly rated specialist in indications such as AML, myeloproliferative neoplasms, multiple myeloma, non-Hodgkin lymphoma, and bone marrow transplantation. In addition to clinical care, Dr. Khan is a researcher for cutting-edge, breakthrough therapies and started a CAR T-Cell program at Bon Secours Health System. Dr. Khan serves as an investigator in both the REGAL trial of GPS and the SLS009 clinical program.

On September 30, 2025 Star Therapeutics, a clinical stage biotechnology company discovering and developing best-in-class antibodies for bleeding disorders and other diseases, reported an oversubscribed $125 million Series D financing (Press release, Star Therapeutics, SEP 30, 2025, View Source [SID1234656350]). The financing round was co-led by Sanofi Ventures and Viking Global Investors with participation from both existing and other new investors, including Janus Henderson Investors, Frazier Life Sciences and GordonMD Global Investments. Existing investors include Agent Capital, Blue Owl Capital, Catalio Capital Management, Cormorant Asset Management, New Leaf Venture Partners, NYBC Ventures, OrbiMed, Qatar Investment Authority (QIA), RA Capital Management, Redmile Group, Sofinnova Investments, Soleus Capital and Westlake BioPartners.

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Proceeds from the Series D financing will support the continued advancement of Star’s pipeline including its lead program, VGA039, a first-in-class monoclonal antibody that targets Protein S to restore balance in blood clotting. VGA039 is being developed as a universal hemostatic therapy for multiple bleeding disorders, starting with von Willebrand disease (VWD). Star has initiated a pivotal Phase 3 clinical trial of VGA039 in patients with all types of VWD, designed to evaluate VGA039 administered once monthly via subcutaneous injection.

"We’re making significant progress across our pipeline, highlighted by the recent initiation of our Phase 3 trial of VGA039 to prevent bleeding in people with all types of VWD, the most common inherited bleeding disorder, with more than 50,000 patients diagnosed and treated in the U.S.," said Adam Rosenthal, Ph.D., CEO and Founder of Star Therapeutics. "VGA039 has the potential to be transformative for VWD patients and could meaningfully reduce the treatment burden, given its once monthly subcutaneous dosing regimen, in comparison to factor replacement prophylaxis which requires two to three IV infusions per week. This new funding from leading life sciences investors reinforces the urgency and impact of our mission: to bring life-changing therapies to people living with serious bleeding disorders and other diseases in hematology and immunology."

In conjunction with the financing, Jason Hafler, Ph.D., from Sanofi Ventures will join Star’s board of directors, and Maneka Mirchandaney of Viking Global Investors will join as a board observer.

"At Sanofi Ventures, we’re proud to bring meaningful experience in supporting innovative therapies for bleeding disorders and remain deeply committed to exploring best-in-class therapies to better meet the needs of patients. VWD represents a compelling opportunity, with a sizable patient population and significant unmet need," said Jason Hafler, Ph.D., Managing Director at Sanofi Ventures. "With VGA039 advancing into Phase 3 for VWD, Star is well-positioned to deliver both substantial patient impact and long-term value. We’re proud to support a team with a strong track record and a bold vision for transforming care in VWD and beyond."

About VGA039
VGA039 is a monoclonal antibody therapy with a novel mechanism of action that targets Protein S, thereby restoring balance to the blood clotting process. VGA039 has potential to be a universal hemostatic therapy that can treat numerous bleeding disorders, starting with VWD. As a subcutaneously self-administered antibody therapy with a convenient once monthly dosing regimen, VGA039 has the potential to dramatically reduce treatment burden for patients. VGA039 has received Fast Track and orphan drug designations from the United States Food and Drug Administration (FDA).

Interim positive data from VIVID-2, a Phase 1 single ascending dose study of VGA039 in patients with VWD, were previously reported at the Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper) in December 2024. A Phase 1/2 multidose study, VIVID-3, is ongoing (NCT05776069). VGA039 has advanced into a Phase 3 study, VIVID-6 (NCT07115004), a global single arm cross-over study designed to investigate the safety and efficacy of subcutaneous administration of VGA039 as prophylaxis for bleeding in patients with every type of VWD. For additional information on VIVID-6, including how to enroll, please visit the website here.

About von Willebrand disease
Von Willebrand disease (VWD) is the most common inherited bleeding disorder in which the blood does not clot properly, caused by absent or defective von Willebrand factor (VWF). VWD patients may experience excessive bleeding with variability in severity and frequency, negatively impacting their daily lives. Current therapies for VWD prophylaxis include factor replacement therapies requiring multiple intravenous (IV) infusions every week. More than 50,000 people in the United States are estimated to be receiving treatment for VWD.