On September 25, 2025 JW Therapeutics (HKEx: 2126), an independent and innovative biotechnology company focusing on developing, manufacturing and commercializing cell immunotherapy products, reported that its enhanced Fast CAR-T product JWCAR239, targeting CD19/CD20, has completed the first patient infusion in its clinical study for relapsed or refractory B-cell non-Hodgkin lymphoma (r/r B-NHL).

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Targeted CD19 CAR-T cell therapy represents a breakthrough treatment for patients with refractory/relapsed (r/r) B-cell non-Hodgkin lymphoma (B-NHL), demonstrating remarkable short-term efficacy. However, many patients remain unresponsive to initial treatment or experience relapse, with long-term progression-free survival (PFS) rates approximately 40%¹. Common mechanisms of refractory/relapse include antigen escape (e.g., loss of CD19 antigen expression), immunosuppressive microenvironments (e.g., inhibitory TGF-β signaling), and dysregulation of CAR-T cell-mediated cytotoxic pathways (e.g., apoptosis)².

Simultaneously targeting CD19/CD20 and enhancing CAR-T cells’ resistance to immunosuppressive microenvironments and apoptotic signals represent key strategies for overcoming CAR-T resistance. In recent years, dual-targeted CAR-T cells targeting CD19/CD20 and fourth-generation CAR-T cells incorporating various enhancement elements have yielded preliminary safety and efficacy data in lymphoma patients.

Additionally, Fast CAR-T possesses unique characteristics compared to traditional CAR-T in terms of production processes and cellular properties, offering multiple advantages. It is gradually demonstrating promising prospects in the treatment of cancer and autoimmune diseases3.

JWCAR239 is a fast CAR-T cell product derived from patient-specific T cells genetically engineered to target CD19 and CD20. It prevents antigen escape while incorporating two exogenously transduced enhancement elements—dnFas-CD2 and dnTGFβRII—that enhance CAR-T cells’ resistance to apoptosis (via CD58 downregulation) and their ability to counteract immunosuppressive signals in the tumor microenvironment. Its rapid manufacturing process not only significantly shortens production cycles but also yields CAR-T cells with a younger phenotype, preserving a high proportion of youthful Tscm cells. This results in enhanced in vivo expansion and tumor cell killing capacity. Preclinical studies have preliminarily validated its efficacy and safety. This study aims to conduct a preliminary assessment of JWCAR239’s safety and/or efficacy in patients with r/r B-NHL.

(Press release, JW Therapeutics, SEP 25, 2025, View Source [SID1234671258])

On September 25, 2025 Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) reported that the U.S. Food and Drug Administration (FDA) approved PALSONIFY (paltusotine) for the first-line treatment of adults with acromegaly who had an inadequate response to surgery and/or for whom surgery is not an option. PALSONIFY, a selectively-targeted somatostatin receptor type 2 nonpeptide (SST2) agonist, is now the first once-daily, oral treatment approved for adults with acromegaly.

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"With the FDA approval of our lead therapy Palsonify, today marks a new era for those living with acromegaly and also for Crinetics as a company," said Scott Struthers, Ph.D., Founder and Chief Executive Officer of Crinetics. "We are very pleased to be fulfilling our commitment to transforming patient lives. This approval is the first to come from our deep pipeline of first-in-class, small molecule drugs. This would not be possible without the help and partnership of people living with acromegaly, their caretakers, our employees, and the clinical researchers and health care professionals who contributed to Palsonify’s successful development program. Thank you to all involved."

The approval is based on data from the PATHFNDR-1 and PATHFNDR-2 Phase 3 pivotal trials, which evaluated PALSONIFY’s safety and efficacy in previously treated and medically untreated adults with acromegaly. Across both trials, PALSONIFY consistently demonstrated rapid onset, reliable biochemical control, and sustained efficacy.

Participants also reported significant reductions in signs and symptoms associated with acromegaly as measured by the Acromegaly Symptom Diary (ASD) — an FDA-aligned patient-reported outcome tool developed to capture the symptoms that matter to people living with acromegaly. Symptoms include headaches, joint pain, sweating, fatigue, weakness, swelling, and/or numbness/tingling. PALSONIFY was generally well-tolerated, with no serious adverse events reported in the randomized controlled portion of the trial.

Long-term results from the open-label extension (OLE) phases of both trials were presented at this year’s Endocrine Society’s annual meeting, ENDO 2025, providing further evidence of PALSONIFY’s ability to deliver durable IGF-1 control, sustained improvements in patient symptom burden, and a consistent safety profile. Ninety-one percent of patients from PATHFNDR-1 and 97 percent of completers from PATHFNDR-2 enrolled in the OLE.

"The PATHFNDR clinical development program set a new standard for acromegaly treatment by demonstrating the ability of Palsonify to drive both biochemical and symptom control, regardless of the degree of underlying disease severity," said Dr. Shlomo Melmed, Executive Vice President of Medicine and Health Sciences and Dean of the Medical Faculty at Cedars-Sinai, "The approval of Palsonify is a significant advancement for our patients, as there is an unmet need for an easy-to-administer and safe therapeutic option with a rapid action and durable response that can consistently manage acromegaly."

"For people living with acromegaly, treatment once meant burdensome injections, breakthrough symptoms, and lifestyle sacrifices just to stay on track," said Jill Sisco, President of Acromegaly Community. "What matters most to our community – maintaining consistent control so the disease doesn’t control us – led us to partner with the FDA on Externally Led Patient-Focused Drug Development meetings. This new treatment reflects that our voices have been heard in shaping the next generation of acromegaly care."

PALSONIFY is expected to be available in the U.S. in early October. Crinetics is ensuring broad access to PALSONIFY by working closely with payers, healthcare providers, and patient advocacy organizations to support those who may benefit from this treatment.

As part of this commitment, Crinetics has launched CrinetiCARE, a comprehensive support program designed to assist people living with acromegaly throughout their treatment journey. CrinetiCARE provides disease and product education, benefit verification, financial assistance resources, and access to dedicated nurse educators who can offer support with treatment onboarding and ongoing adherence. For more information, visit www.CrinetiCARE.com or the PALSONIFY product website Palsonify.com.

A Marketing Authorization Application (MAA) for paltusotine in acromegaly is currently under review for use in the European Union, and the current timeline for the Committee for Medicinal Products and Human Use (CHMP) opinion is the first half of 2026. Crinetics is in partnership with Sanwa Kagaku Kenkyuso (SKK) to develop and commercialize paltusotine for acromegaly in Japan.

Paltusotine is also being evaluated for the treatment of carcinoid syndrome in the pivotal Phase 3 CAREFNDR trial. Global enrollment for CAREFNDR is expected throughout 2025.

(Press release, Crinetics Pharmaceuticals, SEP 25, 2025, View Source [SID1234669419])

On September 25, 2025 Kura Oncology, Inc. (Nasdaq: KURA) and Kyowa Kirin Co., Ltd. (TSE: 4151, "Kyowa Kirin") reported the Journal of Clinical Oncology published the full results from the pivotal KOMET-001 clinical trial (NCT04067336) evaluating ziftomenib, an investigational, once-daily, oral menin inhibitor, in adult patients with relapsed/refractory (R/R) NPM1-mutated (NPM1-m) acute myeloid leukemia (AML) (Press release, Kyowa Hakko Kirin, SEP 25, 2025, View Source [SID1234656275]). Although newly diagnosed patients with NPM1-m AML have high response rates to approved standard of care, relapses are common and survival outcomes are poor. There is currently no approved therapy to specifically target NPM1-m AML. Ziftomenib is currently under priority review by the Food and Drug Administration (FDA) for treatment of R/R NPM1-m AML.

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"Relapsed or refractory NPM1-mutated AML remains very challenging to treat, particularly after venetoclax-based therapy or transplant," said Eunice Wang, M.D., Chief of Leukemia Service, Professor of Oncology, Roswell Park Comprehensive Cancer Center, Buffalo, NY. "The manuscript describes deep responses, signals of clinical activity across relevant subgroups and a generally manageable tolerability profile, which is important in treatment of late line AML patients where accumulated toxicity can limit treatment options. The benefit-risk profile of ziftomenib is highly encouraging and, if replicated in additional treatment settings, has potential to be transformative for a large population of patients with menin pathway-driven AML."

"Publication in ASCO (Free ASCO Whitepaper)’s Journal of Clinical Oncology is an important advancement for adult patients with NPM1-m AML," said Mollie Leoni, M.D., Chief Medical Officer of Kura Oncology. "In addition to evidence of monotherapy activity, the safety and tolerability profile of ziftomenib from this trial is encouraging, marked by the absence of clinically meaningful QTc prolongation as well as low rates of both myelosuppression and treatment discontinuation. No clinically meaningful drug-drug interactions were observed, including with commonly used supportive-care medications, which may simplify co-administration in a polypharmacy setting. We continue to conduct studies in earlier line settings and in combination with multiple therapeutic agents in close collaboration with investigators, study teams and our partner Kyowa Kirin."

Summary of the published data
The publication, entitled "Ziftomenib in Relapsed or Refractory NPM1-Mutated AML", includes positive data from 92 adult patients with R/R NPM1-m AML in the phase 2 portion of the clinical trial as of the primary data cutoff date of October 28, 2024.

The KOMET-001 phase 2 trial met its primary endpoint with a complete remission with full or partial hematologic recovery (CR/CRh) rate of 22% (95% CI, 14 to 32; P=0.0058), which was significantly higher than the 12% historical standard-of-care response rate for patients with R/R NPM1-m AML. One additional response of CRh occurred after the primary analysis data cutoff resulting in a cumulative CR/CRh rate of 23% (95% CI, 15 to 33). 61% of evaluable CR/CRh responders were negative for measurable residual disease (MRD). Overall response rate (ORR) was 33% (95% CI, 23 to 43), with a median duration of overall response of 4.6 months (95% CI, 2.8 to 7.4).

Median overall survival (OS) was 6.6 months (95% CI, 3.6 to 8.6). Among ORR responders, median OS was 18.4 months (95% CI, 8.6 to not estimable) vs. 3.5 months (95% CI, 2.7 to 4.2) among non-responders. Two responders received subsequent allogeneic stem cell transplant and both resumed ziftomenib maintenance after transplant. At the time of data cutoff, nine patients (two after transplantation) remained on ziftomenib treatment. Prespecified subgroup analyses showed comparable CR/CRh rates regardless of lines of therapy, prior venetoclax exposure, or presence of co-mutations, including FLT3m or IDH1/2m.

Ziftomenib was well tolerated with a safety profile consistent with previously disclosed data. The most common grade ≥3 treatment-emergent adverse events were febrile neutropenia (26%), anemia (20%), and thrombocytopenia (20%). Differentiation syndrome occurred in 25% of patients (15% grade 3; no grade 4-5) and was manageable with protocol-defined mitigation. Three patients (3%) discontinued treatment because of ziftomenib-related adverse events.

These findings formed part of the data set used for the New Drug Application for ziftomenib as a potential treatment for patients with R/R NPM1-m AML. The FDA target action date is November 30, 2025. There is currently no FDA-approved treatment for patients with R/R NPM1-m AML.

"The publication of the investigational ziftomenib data adds important scientific context for clinicians and patients," said Takeyoshi Yamashita, Ph. D., Executive Vice President and Chief Medical Officer, Kyowa Kirin. "Together with Kura Oncology, we are committed to rigorous, globally coordinated evidence generation to support the benefit-risk profile of menin inhibition across the treatment landscape. Our shared goal is to advance development rapidly and generate the evidence needed to deliver ziftomenib to appropriate patients in need."

The publication is now available on the Journal of Clinical Oncology website and in the Scientific Manuscripts section on Kura’s website.

Ziftomenib is currently under clinical development, and its safety and efficacy have not been evaluated by any regulatory authority.

On September 25, 2025 Pfizer Inc. (NYSE: PFE) reported it will highlight data across its extensive Oncology portfolio at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2025, being held October 17-21 in Berlin, Germany (Press release, Pfizer, SEP 25, 2025, View Source [SID1234656250]). Data from more than 45 company-sponsored, investigator-sponsored, and collaborative research abstracts, including 11 oral/mini oral presentations and five late-breaking sessions, will be presented across Pfizer’s core scientific modalities and key tumor areas.

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"At ESMO (Free ESMO Whitepaper), Pfizer is demonstrating how earlier interventions with our innovative medicines have the potential to deliver greater impact to even more patients," said Jeff Legos, Chief Oncology Officer, Pfizer. "The survival benefits we’re seeing across certain cancer types reinforce our commitment to accelerating innovative medicines that bring new hope to patients everywhere, while pipeline data highlight the next wave of potential breakthroughs that could transform care for even more people living with cancer."

Pfizer will share highlights from its leading Oncology portfolio at ESMO (Free ESMO Whitepaper), including:

In a Presidential Symposium, unprecedented survival results from the Phase 3 EV-303 trial (KEYNOTE-905) evaluating PADCEV (enfortumab vedotin-ejfv), a Nectin-4 directed antibody-drug conjugate (ADC), plus KEYTRUDA (pembrolizumab)* in patients with muscle-invasive bladder cancer who are ineligible for or declined cisplatin-based chemotherapy, showing potential to redefine standard of care in these patients (Presentation #LBA2)
Final overall survival results from the Phase 3 EMBARK trial evaluating XTANDI (enzalutamide)** in combination with leuprolide and as monotherapy in non-metastatic hormone-sensitive prostate cancer with high-risk biochemical recurrence, highlighting the benefit of XTANDI in this earlier line of treatment (Presentation #LBA87)
Updated overall survival data from the Phase 2 PHAROS study of BRAFTOVI (encorafenib) plus MEKTOVI (binimetinib)*** in patients with BRAF V600E-mutant metastatic non-small cell lung cancer (NSCLC), reinforcing this combination as a potential key treatment option for these patients (Presentation #1849MO)
Information on significant Pfizer and partner-sponsored abstracts, including date and time of presentation, follows in the chart below. A complete list of Pfizer and partner-sponsored abstracts and presentations is available here.

Presentation Title

Details

Genitourinary Cancer

Perioperative (periop) enfortumab vedotin (EV) plus pembrolizumab (pembro) in participants (pts) with muscle-invasive bladder cancer (MIBC) who are cisplatin-ineligible: The phase 3 KEYNOTE-905 study [Merck/MSD-led]

Vulsteke et. al

Presidential Symposium (Presentation #LBA2)

Saturday, October 18, 2025, 4:30 PM CEST

Disitamab vedotin (DV) plus toripalimab (T) versus chemotherapy (C) in first-line (1L) locally advanced or metastatic urothelial carcinoma (la/mUC) with HER2-expression [Remegen-led]

Sheng et. al

Presidential Symposium (Presentation #LBA7)

Sunday, October 19, 2025, 4:30 PM CEST

Overall survival with enzalutamide in biochemically recurrent prostate cancer

Freedland et. al

Oral Presentation (Presentation #LBA87)

Sunday, October 19, 2025, 10:55 AM CEST

Randomised phase 3 trial of androgen deprivation therapy (ADT) with radiation therapy with or without enzalutamide for high risk, clinically localised prostate cancer: ENZARAD (ANZUP 1303)****

Nguyen et. al

Oral Presentation (Presentation #LBA86)

Sunday, October 19, 2025, 10:15 AM CEST

Thoracic Cancer

Updated overall survival analysis from the phase 2 PHAROS study of encorafenib plus binimetinib in patients with BRAF V600E-mutant metastatic NSCLC (mNSCLC)

Johnson et. al

Mini Oral Presentation (Presentation #1849MO)

Sunday, October 19, 2025, 8:30 AM CEST

Enfortumab vedotin plus pembrolizumab (EV + P) as first-line (1L) treatment in recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC): results from a cohort of the EV-202 trial [Astellas led]

Swiecicki et. al

Mini Oral Presentation (Presentation #1329MO)

Sunday, October 19, 2025, 4:30 PM CEST

Breast Cancer

Health-Related Quality of Life (HRQoL) from the PATINA Trial (AFT-38): Impact of Adding Palbociclib to HER2 and Endocrine Therapy (ET) after Induction in HR+/HER2+ Metastatic Breast Cancer (MBC)

Ines Vaz-Luis et. al

Mini Oral Presentation (Presentation #485MO)

Monday, October 20, 2025, 10:15 AM CEST

Patient-reported outcomes (PROs) with vepdegestrant (VEP) vs fulvestrant (FUL) in patients (pts) with estrogen receptor (ER) 1 gene mutated (ESR1m) ER+/human epidermal growth factor receptor 2 (HER2)−advanced breast cancer (aBC) in the phase 3 VERITAC-2 trial*****

Campone et. al

Mini Oral Presentation (Presentation #489MO)

Monday, October 20, 2025, 10:15 AM CEST

Gastrointestinal Cancer

Circulating tumor (ct) DNA analysis of BRAF V600E dynamics and changes in genomic landscape in patients (pts) with first-line (1L) BRAF V600E-mutant metastatic colorectal cancer (mCRC) treated in BREAKWATER******

Kopetz et. al

Mini Oral Presentation (Presentation #729O)

Monday, October 20, 2025, 08:30 AM CEST

Cancer-Related Conditions

Efficacy and safety of ponsegromab in patients with cancer-associated cachexia: Results from the open-label extension of a randomized, placebo-controlled, Phase 2 study

Oral Presentation (Presentation #LBA102)

Friday, October 17, 2025, 4:00 PM CEST

On September 25, 2025 TAE Life Sciences (TLS), a global leader in advancing next-generation boron neutron capture therapy (BNCT), and the University of Wisconsin–Madison (UW) reported to have signed a memorandum of understanding (MOU) announcing the intention to launch the first accelerator-based BNCT center in the United States (Press release, TAE Life Sciences, SEP 25, 2025, View Source [SID1234656249]). As part of this collaboration, UW would install the Alphabeam compact accelerator-based BNCT system developed by TAE Life Sciences. This significant milestone has the potential to pave the way for the adoption of BNCT in the United States.

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As part of this proposal, UW and TAE Life Sciences will collaborate on research and development of Alphabeam and novel boron-10 drugs through pre-clinical and clinical studies on several cancer indications with high unmet need, including brain and head and neck cancers. Available in Japan, and currently being evaluated in clinical trials in several other countries, BNCT is a cutting-edge cancer treatment that selectively targets cancer cells while minimizing harm to healthy tissue. TAE Life Sciences, with its compact accelerator-based neutron source and novel targeted boron-10 drug development, is the only company to have a comprehensive BNCT solution that replaces earlier challenges with nuclear reactor-based BNCT and has the potential to establish accelerator-based BNCT as a mainstay cancer treatment in the United States. This announcement further builds upon the success TAE Life Sciences has had deploying its technology globally. To date, the company has installed its neutron beam system in China’s Xiamen Humanity Hospital, which is currently running human clinical trials, and its Alphabeam will soon be installed at Italy’s National Center of Oncological Hadrontherapy (CNAO).

"Our collaboration with the esteemed University of Wisconsin–Madison underscores the growing momentum and recognition of BNCT as a transformative cancer therapy," stated Robert Hill, Chief Executive Officer of TAE Life Sciences. "By working closely with UW and the University of Wisconsin Carbone Cancer Center, a leading cancer center renowned for its commitment to innovation, we aim to revolutionize the landscape for cancer treatment, and make even the most challenging cancers treatable with minimal side effects."

Alphabeam is a patented neutron source developed for clinical BNCT that offers a breakthrough approach to cancer treatment. This combinational therapy utilizes targeted boron-10 drugs and low-energy neutrons to destroy tumors with no damage to healthy tissues. With this system, patients can undergo a short, minimally invasive treatment in just one or two sessions, providing a streamlined alternative to traditional cancer therapies.

"Today marks a significant milestone in the field of oncology in the United States," said Dr. Zachary Morris, Chair and Paul Harari Professor of Human Oncology at the University of Wisconsin School of Medicine and Public Health. "Our collaboration with TAE Life Sciences brings together our expertise in clinical radiation medicine, translational research, and theranostics with the accelerator-based BNCT system, enabling us to harness the full potential of this advanced cancer treatment. Together, we aim to accelerate the development and clinical implementation of this therapy, ultimately providing patients with what we hope will be a markedly improved cancer treatment option that is currently not available anywhere else in North America."

The Alphabeam BNCT system and novel boron-10 drugs are for research purposes and not available for sale.

About BNCT

BNCT is a combination treatment based on the reaction that occurs when a non-toxic compound containing boron-10 is irradiated with a low-energy neutron beam. BNCT differs radically from other radiation therapy and shows promise in becoming the next-generation cancer treatment. Research has shown BNCT has the capability of killing cancer cells that are resistant to traditional radiation therapy with limited harm to healthy tissue. Current advances in both neutron radiation technology and medicinal boron drug targeting are enabling BNCT’s potential to improve patient care while also improving treatment economics. To date, approximately 2,000 patients have been treated with BNCT at research sites worldwide.