Taiho Pharmaceutical Exercises Option for an Exclusive License to Casdatifan in Japan and Certain Territories in Asia

On October 19, 2025 Taiho Pharmaceutical Co., Ltd. ("Taiho") and Arcus Biosciences, Inc. (NYSE:RCUS, "Arcus") reported that Taiho exercised its option to develop and, if approved, commercialize casdatifan (International Nonproprietary Name; development code: AB521), an investigational small molecule HIF (Hypoxia Inducible Factor)-2α inhibitor, in Japan and certain other territories in Asia (excluding mainland China). This option exercise is based on a 2017 option and license agreement between Taiho and Arcus. This is the fifth option exercise by Taiho to an Arcus program.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

In exchange for the exclusive license to casdatifan, Taiho will make an option exercise payment, as well as additional payments upon achievement of clinical, regulatory and commercialization milestones, and, if any products from the program are approved, will pay royalties on net sales of such products.

Casdatifan is an investigational small molecule compound developed by Arcus. Arcus is currently conducting an ongoing global, registrational Phase 3 study, PEAK-1,* comparing the combination therapy of casdatifan and a VEGFR-targeted tyrosine kinase inhibitor (TKI) to monotherapy using a VEGFR-targeted TKI alone in patients with clear cell renal cell carcinoma (ccRCC). Japan is also expected to participate in the study starting in the first half of 2026.

*PEAK-1: A Randomized, Placebo-Controlled, Double-Blind, Multicenter Phase 3 Trial of Casdatifan and Cabozantinib Versus Placebo and Cabozantinib in Patients with Advanced Clear Cell Renal Cell Carcinoma

Through this collaboration, Taiho and Arcus are committed to delivering casdatifan as a potentially innovative new medicine to patients and healthcare professionals as swiftly as possible.

About Casdatifan (AB521)

Casdatifan is a small-molecule inhibitor of HIF-2α, a master switch that turns on hundreds of genes in response to low oxygen levels; when oxygen levels return to normal, HIF-2α is turned off. In a majority of people with the most common form of kidney cancer (clear cell renal cell carcinoma), this shut-off mechanism is deficient and HIF-2α remains activated even in the presence of oxygen, causing normal kidney cells to become cancerous. Casdatifan is designed to provide deeper and more durable inhibition of the HIF-2α pathway.

About clear cell renal cell carcinoma (ccRCC)

Kidney cancer is a disease with a rising worldwide incidence estimated at 400,000 new cases annually, and a worldwide mortality rate approaching 175,000 deaths per year. Current projections suggest incidence continuing to increase over the next decade, emphasizing the urgency of addressing this significant global health trend. ccRCC is the most common type of kidney cancer in adults, making up 75-80% of all cases.

(Press release, Taiho, OCT 19, 2025, View Source [SID1234656800])

Preliminary Clinical Data of CARsgen’s Allogeneic BCMA CAR-T Product CT0596 for the Treatment of Primary Plasma Cell Leukemia

On October 19, 2025 Carsgen therapeutcis reported primary plasma cell leukemia (pPCL) is a rare and highly aggressive plasma cell malignancy, often associated with complex genetic abnormalities. There is currently no standard treatment regimens, and conventional therapies for multiple myeloma are typically used. While targeted agents and autologous hematopoietic stem cell transplantation can slightly extend the overall survival of pPCL patients to 1.5–3 years, relapsed/refractory pPCL after multiple lines of therapy presents a significant clinical challenge due to the extremely limited treatment options and rapid disease progression, making it difficult to re-induce remission with existing therapies.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

CT0596 is an allogeneic BCMA-targeted CAR-T therapy developed using CARsgen’s proprietary THANK-u Plus platform. It is currently being evaluated in investigator-initiated trials for plasma cell malignancy. As of the data cutoff date (Oct 17, 2025), two patients with relapsed/refractory pPCL had been enrolled. Details are as follows:

pPCL-01:

A 62-year-old male with IgG-λ type experienced poor disease control and rapid progression despite previous autologous hematopoietic stem cell transplantation and treatment with triple classes of drugs (proteasome inhibitor, immunomodulatory agent, and CD38 monoclonal antibody). By the screening period, his serum free light chain level had reached 10,374.86 mg/L. The patient underwent two CAR-T cell infusions approximately two months apart: he first received low-dose lymphodepletion and a relatively lower dose of CAR-T cells per protocol, then followed one cycle of bridge therapy. About one month after the failure of bridge therapy, he received a second CAR-T cell infusion. Following this infusion, he developed Grade 2 CRS, Grade 4 cytopenia, and a lung infection, but recovered after supportive treatment including tocilizumab, corticosteroids, autologous stem cell infusion, and anti-infective therapy. CAR-T cells expanded robustly, with a peak copy number (Cmax) of 161,971 copies/μg gDNA, and copy numbers remained at 10³ by Week 8. Efficacy assessments at both Week 4 and Week 8 post-infusion showed stringent complete response (sCR), and bone marrow minimal residual disease (MRD) was negative (< 10⁻⁶) at Week 4.

pPCL-02:

A 70-year-old male with κ light chain had previously been treated with triple classes of drugs (proteasome inhibitor, immunomodulatory agent, and CD38 monoclonal antibody). The patient developed Grade 1 CRS, Grade 4 neutropenia, and thrombocytopenia following lymphodepletion and CAR-T cell infusion. The CRS and cytopenias were resolved after treatment with tocilizumab and other supportive measures. CAR-T cells expanded robustly, with a Cmax of 151,654 copies/μg gDNA. The patient achieved sCR at the Week 4, Week 8, and Week 12 assessments post-infusion, with bone marrow MRD negative (< 10⁻⁶) at both Week 4 and Week 12.

Based on the current findings, CT0596 has exhibited robust and rapid efficacy in heavily pretreated patients with rapidly progressive relapsed/refractory pPCL. Both pPCL patients achieved sCR following CT0596 infusion, with peak CAR-T cell copy numbers exceeding 10⁵ copies/μg gDNA. Aside from expected CAR-T-associated toxicities such as CRS and hematologic adverse events, no significant organ toxicities were observed, indicating a manageable safety profile. These results lend strong support to the continued investigation of CT0596 across a broader spectrum of plasma cell neoplasms.

About CT0596

CT0596 is an allogeneic BCMA-targeted CAR-T therapy developed using CARsgen’s proprietary THANK-u Plus platform. It is currently being evaluated in investigator-initiated trials for relapsed/refractory multiple myeloma (R/R MM) or plasma cell leukemia (PCL). CT0596 demonstrated preliminary favorable tolerability and encouraging efficacy signals. Further investigation is planned in additional plasma cell malignancies and autoimmune diseases mediated by autoreactive plasma cells. The company anticipates submitting an Investigational New Drug (IND) application in the second half of 2025.

(Press release, Carsgen Therapeutics, OCT 19, 2025, View Source;preliminary-clinical-data-of-carsgens-allogeneic-bcma-car-t-product-ct0596-for-the-treatment-of-primary-plasma-cell-leukemia-302588331.html [SID1234656799])

Multitude Therapeutics Announces Encouraging Interim Phase I/II Results from Ongoing First-in-Human Study Evaluating its MUC18-directed Antibody-Drug-Conjugate, AMT-253, in Melanoma and Other Advanced Solid Tumors at the 2025 ESMO Annual Meeting

On October 19, 2025 Multitude Therapeutics, Inc., a clinical stage biopharmaceutical company focused on the development of ADC drugs, reported interim results from its ongoing Phase I/II open-label, multicenter dose escalation and expansion study evaluating AMT-253, a MUC18-directed antibody-drug-conjugate (ADC), in patients with melanoma and other advanced solid tumors. The data are being presented on October 19th at the 2025 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Annual Meeting being held in Berlin, Germany.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Phase I/II clinical trials are ongoing in both Australia and China. This first-in-human study will evaluate the Maximum Tolerated Dose (MTD) / the Recommended Phase II Dose (RP2D), safety, tolerability, anti-tumor activity, pharmacokinetics, pharmacodynamics and immunogenicity of AMT-253, in patients with advanced solid tumors. The Phase Ia portion will determine the recommended doses for expansion, and the Phase Ib/II portion will focus on further characterizing safety and efficacy in select tumor types.

As of September 8th, 2025, safety data are available for 170 patients who have received AMT-253 once every three weeks (Q3W) at doses ranging from 1.6 to 5.6 mg/kg. Primary tumor types are melanoma, endometrial cancer, cervical squamous cancer and uterine leiomyosarcoma. In heavily pretreated melanoma patients, with prior lines of therapy ranging from 1-7, promising efficacy was observed without MUC18 preselection. The overall response rate (ORR) was 28.6% in all melanomas (16/56, 15 confirmed, 1 unconfirmed still on-treatment) across all dose levels. In melanoma patients without prior chemotherapy, ORR was 35% (7/20, all confirmed), 50% (5/10; 4 confirmed, 1 unconfirmed still on-treatment), and 33.3% (1/3, confirmed) for the cutaneous, acral, and mucosal subtypes, respectively; preliminary mPFS was 8.6, 8.3, and 11.0 months. Among 18 cutaneous melanoma patients at the potential expansion doses, ORR was 38.9% (7/18, all confirmed), and preliminary mPFS was 8.6 months. Preliminary antitumor activity was also observed in endometrial cancer, cervical squamous carcinoma and uterine leiomyosarcoma patients. Responses were observed across MUC18 expression levels in all tumor patients evaluated. AMT-253 showed a safety profile consistent with other topoisomerase-1 (TOP1) inhibitor–based ADCs, with manageable hematologic toxicities as the most common treatment-related adverse events. No treatment-related cases of neuropathy or pneumonitis were observed as of the data cutoff.

"We are excited by the durable antitumor activity observed in the early clinical results of AMT-253, particularly in unselected, late-line metastatic melanoma patients, where the unmet medical need remains high," said Dr. Shu-Hui Liu, Co-founder and CSO of Multitude Therapeutics. "These initial data, together with our previously published preclinical findings*, highlight the potential of AMT-253 to offer meaningful outcome improvement to patients who have exhausted standard-of-care options. We look forward to further clinical confirmation of these results and to fully exploring the potential of this novel ADC for greater patient benefit."

*Shi et al., (2023) Cancer Research 83:3783-95

Poster Presentation Details:

Title: Ongoing Phase I trial results of AMT-253, a first-in-class MUC18-targeting antibody-drug conjugate in patients with melanoma and other advanced solid tumors
Session Title: Developmental therapeutics
Date and Time: October 19th – 09:00-17:00
Poster Number: 997P
Location: Hall 25, Level 2

About AMT-253

AMT-253, a MUC18 ADC, is composed of a proprietary antibody with high MUC18 binding affinity, a protease-cleavable linker, and an exatecan payload (a potent and clinically validated topoisomerase-1 inhibitor). The linker is designed to complement the exatecan payload, enabling a stable and homogenous ADC. The payload is a weak substrate for BCRP/P-gp, which are drug efflux pumps that drive chemoresistance to many therapies. In preclinical data, this linker-payload has been shown to have an increased "bystander effect" compared to the equivalent of competitor ADCs**. AMT-253 has a drug-to-antibody ratio of eight. AMT-253 is being evaluated in a Phase I/II study in patients with melanoma and other advanced solid tumors. Additional information on the AUS PhI(NCT05906862) and China PhI/II (NCT06209580) trials can be found at clinicaltrials.gov.

(Press release, Multitude Therapeutics, OCT 19, 2025, View Source [SID1234656798])

SystImmune Announces iza-bren Meets One of the Dual Primary Endpoints in the BL-B01D1-303 Trial in Recurrent or Metastatic NPC Patients with Results Presented as a Late-Breaking Oral Presentation at ESMO

On October 19, 2025 SystImmune Inc. (SystImmune), a clinical-stage biotechnology company, reported positive topline results from the BL-B01D1-301 trial. The trial has met one of the dual primary endpoints (BICR-assessed ORR) as iza-bren has demonstrated a statistically significant and clinically meaningful improvement in BICR-assessed ORR in recurrent or metastatic NPC patients who had progressed after at least two prior lines of chemotherapy, including platinum-based chemotherapy and a PD-1/PD-L1 inhibitor. These results were presented today in a late-breaking oral presentation at the 2025 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Iza-bren is a potentially first-in-class topoisomerase 1 inhibitor-based bispecific antibody-drug conjugate (ADC) which targets both epidermal growth factor receptor and human epidermal growth factor receptor 3 (EGFRxHER3). It is being developed by Biokin in China and jointly developed by SystImmune and Bristol Myers Squibb under a collaboration and exclusive license agreement in territories outside of China.

Iza-bren has shown a BICR-assessed ORR of 54.6% vs. 27.0% (Odds Ratio 3.3; 95% confidence interval 1.9-5.8; p<0.0001). Median duration of response (DoR) was 8.5 months for iza-bren versus 4.8 months for physician’s choice of chemotherapy (Hazard ratio 0.43; 95% CI 0.22 to 0.83). Furthermore, median progression-free survival (PFS) was 8.38 months with iza-bren compared to 4.34 months for chemotherapy (hazard ratio of 0.44; 95% confidence interval 0.32-0.62). The ORR and PFS benefits were consistent across all subgroup analysis. At the time of this analysis, the overall survival (OS) data were immature.

Iza-bren was well-tolerated with a manageable safety profile. The most common adverse events were hematological toxicities, which were effectively managed with standard supportive care. Two patients in the iza-bren arm experienced Grade 2 interstitial lung disease (ILD), whereas two patients in the chemotherapy arm experienced Grade 3 ILD. The safety profile in the BL-B01D1-303 study was consistent with the known profiles of the therapy with no new safety signals identified.

"The positive topline results from the first registrational trial of iza-bren presented at ESMO (Free ESMO Whitepaper) demonstrated clear clinical benefits of iza-bren compared to traditional chemotherapy. These data add to the growing body of clinical evidence that continues to reinforce our confidence in iza-bren’s mechanism of action and therapeutic potential. We believe iza-bren can deliver clinically meaningful benefit by targeting key cancer biological pathways to improve outcomes for patients across a broad range of advanced malignancies," added Dr. Jonathan Cheng, Chief Medical Officer of SystImmune.

The New Drug Application (NDA) for iza-bren for the treatment of recurrent or metastatic nasopharyngeal carcinoma (NPC) has been submitted by Biokin to the Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) in China.

About BL-B01D1-303

BL-B01D1-303 is a phase III, randomized, open-label, multicenter study in China to evaluate the efficacy and safety of BL-B01D1 in patients with recurrent or metastatic nasopharyngeal carcinoma who have failed PD-1/PD-L1 monoclonal antibody and at least two lines chemotherapy (one line must contain platinum-based chemotherapy). For more detailed information, please visit clinical.trials.gov (NCT06118333).

About Nasopharyngeal Carcinoma (NPC)

Nasopharyngeal Carcinoma (NPC) is a type of head and neck cancer that originates in the nose. NPC is uncommon globally, but is endemic in southern China, southeast Asia and parts of Africa, and is rising among the immigrant population in US and Europe. NPC is strongly associated with EBV infection. There is a significant unmet need as 5-year overall survival rate is generally less than 10% in the later line metastatic setting.

About iza-bren

SystImmune, in collaboration with BMS outside of China, is developing iza-bren (BL-B01D1), a bispecific antibody-drug conjugate (ADC) that targets both EGFR and HER3, which are highly expressed in various epithelial cancers and are known to be associated with cancer cell proliferation and survival. Iza-bren’s dual mechanism of action blocks EGFR and HER3 signals to cancer cells, reducing proliferation and survival signals. In addition, upon antibody mediated internalization, iza-bren’s therapeutic novel Topo1i payload is released causing cytotoxic stress that leads to cancer cell death.

(Press release, SystImmune, OCT 19, 2025, View Source [SID1234656797])

Subcutaneous amivantamab delivers promising 45 percent overall response rate with median duration of 7.2 months in recurrent or metastatic head and neck cancer

On October 19, 2025 Johnson & Johnson (NYSE:JNJ) reported promising new results from the Phase 1b/2 OrigAMI-4 study evaluating the efficacy and safety of subcutaneous (SC) amivantamab monotherapy in patients with human papillomavirus (HPV)-unrelated, recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC) after disease progression on a checkpoint inhibitor and platinum-based chemotherapy. The study used the new subcutaneous formulation of amivantamab, which can be delivered in a five-minute manual injection, offering increased patient convenience compared to intravenous RYBREVANT (amivantamab-vmjw). Data were presented during a mini-oral session at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2025 Congress (Oral Abstract #1327MO).1

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

Patients with R/M HNSCC have limited options and poor outcomes after disease progression on a PD-1 or PD-L1 inhibitor and platinum-based chemotherapy.2 Many are unable to receive further treatment, and for those who do, response rates with available therapies are typically only 10 to 24 percent.2,3,4 Survival remains short, with a median of 6 to 9 months, and may be even shorter among patients with HPV-unrelated disease.2,4,5,6 Patients also face a significant burden of symptoms such as difficulty swallowing, impaired speech, pain and fatigue.2 Epidermal growth factor receptor (EGFR) and MET, two key tumor drivers, are overexpressed in 80 to 90 percent of HNSCC tumors, highlighting their role as potential treatment targets.7,8,9 RYBREVANT, the world’s first bispecific antibody approved for lung cancer, inhibits EGFR and MET, in addition to activating the immune system to attack cancer cells.10

"Patients with recurrent or metastatic head and neck cancer face an aggressive disease that significantly impacts their quality of life," said Professor Kevin Harrington*, MBBS, Ph.D., Professor in Biological Cancer Therapies at The Institute of Cancer Research, Royal Marsden Hospital, London, UK, and primary study investigator. "These results represent one of the most encouraging response rates we’ve seen in this difficult-to-treat setting, with durability that could meaningfully extend the time patients live without their disease progressing."

In Cohort 1 of the OrigAMI-4 study, treatment with SC amivantamab resulted in an overall response rate (the primary endpoint) of 45 percent in 38 efficacy-evaluable patients with R/M HNSCC unrelated to HPV with disease progression on or after a PD-1 or PD-L1 checkpoint inhibitor and platinum-based chemotherapy (95 percent confidence interval [CI], 29-62).1 Responses occurred quickly, with a median time to first response of 6.4 weeks (range, 5.7-18.3), and were durable, with a median duration of response of 7.2 months (95 percent CI, 5.3-not estimable [NE]).1,11 Tumor shrinkage of target lesions was observed in 82 percent of patients after 8.3 months follow-up.1 Median progression-free survival was 6.8 months (95 percent CI, 4.2-9.0), while median overall survival had not yet been reached (95 percent CI, 7.7-NE).1

"These data highlight the broader potential of RYBREVANT-based therapies across solid tumors where the EGFR and/or MET pathways are activated," said Kiran Patel, Vice President, Global Head, Solid Tumor Clinical Development and Diagnostics, Johnson & Johnson Innovative Medicine. "RYBREVANT combinations have already demonstrated significant results for certain patients with non-small cell lung cancer, leading to extended survival and delayed treatment resistance. Now we’re building on that progress in other hard-to-treat diseases like head and neck cancer. By targeting EGFR and MET while also engaging the immune system, subcutaneous amivantamab could provide new options for more patients who have few effective treatments."

The safety-evaluable population included 86 patients who received at least one dose of SC amivantamab monotherapy. The safety profile was consistent with prior SC amivantamab monotherapy studies, with no new safety signals observed. The most common treatment-emergent adverse events were fatigue (31 percent), hypoalbuminemia (31 percent) and stomatitis (23 percent). Administration-related reactions occurred in seven percent of patients, all mild to moderate (grade 1-2), with no severe events. Treatment discontinuation due to treatment-related adverse events occurred in two percent of patients.1

Based on these results, Johnson & Johnson is initiating further study of SC amivantamab in head and neck cancer with the Phase 3 OrigAMI-5 study, which is evaluating first-line SC amivantamab with pembrolizumab and carboplatin versus 5-fluorouracil (5FU) plus pembrolizumab and platinum-based chemotherapy (cisplatin or carboplatin) in patients with HPV-unrelated R/M HNSCC. In addition to supporting further evaluation in first-line R/M HNSCC, these findings add to the evidence supporting the role of RYBREVANT-based treatments across multiple solid tumors, including non-small cell lung cancer, colorectal cancer and HNSCC.

About the OrigAMI-4 Study

OrigAMI-4 (NCT06385080) is an open-label Phase 1b/2 study evaluating SC amivantamab in recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). The study includes five cohorts, including Cohort 1, which studied SC amivantamab as monotherapy in patients with human papillomavirus (HPV)-unrelated R/M HNSCC who had received prior platinum-based chemotherapy and PD-1/PD-L1 immunotherapy. Patients with prior anti-EGFR therapy were excluded. Subcutaneous amivantamab was administered every three weeks (Q3W) at 2400 mg, or 3360 mg for patients weighing 80 kg or more. The primary endpoint is overall response rate (ORR), assessed by blinded independent central review (BICR) using RECIST v1.1**.12

About Head and Neck Squamous Cell Carcinoma

Head and neck squamous cell carcinoma (HNSCC) is the most common type of head and neck cancer, accounting for more than 90 percent of cases and approximately 4.5 percent of all cancers worldwide.13 It develops in the mucosal linings of the oral cavity, oropharynx, hypopharynx, and larynx.13 Major risk factors include tobacco and alcohol use, as well as infection with high-risk human papillomavirus (HPV).13 Around 75 percent of cases are HPV-negative, which is typically associated with a poorer prognosis and reduced response to treatment.13,14 Despite advances in surgery, radiation, chemotherapy, and immunotherapy, many patients are diagnosed at an advanced stage, and recurrent or metastatic HNSCC continues to carry a poor prognosis.2,7

About Subcutaneous Amivantamab

Subcutaneous amivantamab is an investigational fixed-dose combination of the bispecific antibody amivantamab and recombinant human hyaluronidase PH20 (rHuPH20), which is part of Halozyme’s ENHANZE drug delivery technology. It targets EGFR and MET with immune cell-directing activity and is being developed to address tumors driven by activating and resistance EGFR mutations as well as MET alterations.

Subcutaneous amivantamab is being studied across multiple tumor types, including head and neck squamous cell carcinoma (HNSCC) and non-small cell lung cancer (NSCLC).

About RYBREVANT

RYBREVANT (amivantamab-vmjw), a fully-human bispecific antibody targeting EGFR and MET with immune cell-directing activity, is approved in the U.S., Europe and other markets around the world as monotherapy for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test, whose disease has progressed on or after platinum-based chemotherapy.10

RYBREVANT is approved in the U.S., Europe and other markets around the world in combination with chemotherapy (carboplatin and pemetrexed) for the first-line treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 20 insertion mutations, as detected by an FDA-approved test.

RYBREVANT is approved in the U.S., Europe and other markets around the world in combination with LAZCLUZE (lazertinib) for the first-line treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations, as detected by an FDA-approved test.

RYBREVANT is approved in the U.S., Europe and other markets around the world in combination with chemotherapy (carboplatin-pemetrexed) for the treatment of adult patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or L858R substitution mutations, whose disease has progressed on or after treatment with an EGFR TKI.

Subcutaneous amivantamab is approved in Europe in combination with LAZCLUZE for the first-line treatment of adult patients with advanced NSCLC with EGFR exon 19 deletions or exon 21 L858R substitution mutations, and as a monotherapy for the treatment of adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after failure of platinum-based therapy. A Biologics License Application (BLA) was submitted to the U.S. FDA for this indication.

The National Comprehensive Cancer Network (NCCN) Clinical Practice Guidelines in Oncology (NCCN Guidelines) for NSCLC§ prefer next-generation sequencing–based strategies over polymerase chain reaction–based approaches for the detection of EGFR exon 20 insertion, exon 19 deletion and L858R variants. The NCCN Guidelines include:

Amivantamab-vmjw (RYBREVANT) plus lazertinib (LAZCLUZE) as a Category 1 recommendation for first-line therapy in patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R mutations.15†‡
Amivantamab-vmjw (RYBREVANT) plus chemotherapy as a Category 1 recommendation for patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or exon 21 L858R mutations who experienced disease progression after treatment with osimertinib.15 †‡
Amivantamab-vmjw (RYBREVANT) plus chemotherapy as a Category 1 recommendation for first-line therapy in treatment-naive patients with newly diagnosed advanced or metastatic EGFR exon 20 insertion mutation-positive advanced NSCLC. 15 †‡
Amivantamab-vmjw (RYBREVANT) as a Category 2A recommendation for patients that have progressed on or after platinum-based chemotherapy with or without an immunotherapy and have EGFR exon 20 insertion mutation-positive NSCLC. 15 †‡
In addition to the Phase 1b/2 OrigAMI-4 study, RYBREVANT is being studied in multiple clinical trials, including:

The Phase 3 MARIPOSA (NCT04487080) study assessing RYBREVANT in combination with LAZCLUZE versus osimertinib and versus LAZCLUZE alone in the first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR ex19del or substitution mutations.16
The Phase 3 MARIPOSA-2 (NCT04988295) study assessing the efficacy of RYBREVANT (with or without LAZCLUZE) and carboplatin-pemetrexed versus carboplatin-pemetrexed alone in patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletions or L858R substitution mutations after disease progression on or after osimertinib.17
The Phase 3 PAPILLON (NCT04538664) study assessing RYBREVANT in combination with carboplatin-pemetrexed versus chemotherapy alone in the first-line treatment of patients with advanced or metastatic NSCLC with EGFR exon 20 insertion mutations.18
The Phase 3 PALOMA-3 (NCT05388669) study assessing LAZCLUZE with subcutaneous (SC) amivantamab compared to RYBREVANT in patients with EGFR-mutated advanced or metastatic NSCLC.19
The Phase 2 PALOMA-2 (NCT05498428) study assessing SC amivantamab in patients with advanced or metastatic solid tumors including EGFR-mutated NSCLC.20
The Phase 1 PALOMA (NCT04606381) study assessing the feasibility of SC amivantamab based on safety and pharmacokinetics and to determine a dose, dose regimen and formulation for SC amivantamab delivery.21
The Phase 1 CHRYSALIS (NCT02609776) study evaluating RYBREVANT in patients with advanced NSCLC.22
The Phase 1/1b CHRYSALIS-2 (NCT04077463) study evaluating RYBREVANT in combination with LAZCLUZE and LAZCLUZE as a monotherapy in patients with advanced NSCLC with EGFR mutations.23
The Phase 1/2 METalmark (NCT05488314) study assessing RYBREVANT and capmatinib combination therapy in locally advanced or metastatic NSCLC.24
The Phase 1/2 swalloWTail (NCT06532032) study assessing RYBREVANT and docetaxel combination therapy in patients with metastatic NSCLC.25
The Phase 1/2 PolyDamas (NCT05908734) study assessing RYBREVANT and cetrelimab combination therapy in locally advanced or metastatic NSCLC.26
The Phase 2 SKIPPirr study (NCT05663866) exploring how to decrease the incidence and/or severity of first-dose infusion-related reactions with RYBREVANT in combination with LAZCLUZE in relapsed or refractory EGFR-mutated advanced or metastatic NSCLC.27
The Phase 2 COPERNICUS (NCT06667076) study combining developments in treatment administration and prophylactic supportive care in representative US patients with common EGFR-mutated NSCLC treated with SC amivantamab in combination with LAZCLUZE or chemotherapy.28
The Phase 2 COCOON (NCT06120140) study assessing the effectiveness of a proactive dermatologic management regimen given with first-line RYBREVANT and LAZCLUZE in patients with EGFR-mutated advanced NSCLC.29
The Phase 1b/2 OrigAMI-1 (NCT05379595) study assessing RYBREVANT monotherapy and in addition to standard-of-care chemotherapy in patients with advanced or metastatic colorectal cancer.30
The legal manufacturer for RYBREVANT is Janssen Biotech, Inc.

For more information, visit: View Source

RYBREVANT IMPORTANT SAFETY INFORMATION 10

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

RYBREVANT can cause infusion-related reactions (IRRs) including anaphylaxis; signs and symptoms of IRR include dyspnea, flushing, fever, chills, nausea, chest discomfort, hypotension, and vomiting. The median time to IRR onset is approximately 1 hour.

RYBREVANT with LAZCLUZE

In MARIPOSA (n=421), IRRs occurred in 63% of patients, including Grade 3 in 5% and Grade 4 in 1% of patients. IRR-related infusion modifications occurred in 54%, dose reduction in 0.7%, and permanent discontinuation of RYBREVANT in 4.5% of patients.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population (n=281), IRRs occurred in 50% of patients including Grade 3 (3.2%) adverse reactions. The incidence of infusion modifications was 46%, and 2.8% of patients permanently discontinued RYBREVANT due to IRR.

RYBREVANT as a Single Agent

In CHRYSALIS (n=302), IRRs occurred in 66% of patients. IRRs occurred in 65% of patients on Week 1 Day 1, 3.4% on Day 2 infusion, 0.4% with Week 2 infusion, and were cumulatively 1.1% with subsequent infusions. 97% were Grade 1-2, 2.2% were Grade 3, and 0.4% were Grade 4. The median time to onset was 1 hour (range 0.1 to 18 hours) after start of infusion. The incidence of infusion modifications was 62% and 1.3% of patients permanently discontinued RYBREVANT due to IRRs.

Premedicate with antihistamines, antipyretics, and glucocorticoids and infuse RYBREVANT as recommended. Administer RYBREVANT via a peripheral line on Week 1 and Week 2 to reduce the risk of IRRs. Monitor patients for signs and symptoms of IRRs in a setting where cardiopulmonary resuscitation medication and equipment are available. Interrupt infusion if IRR is suspected. Reduce the infusion rate or permanently discontinue RYBREVANT based on severity. If an anaphylactic reaction occurs, permanently discontinue RYBREVANT.

Interstitial Lung Disease/Pneumonitis

RYBREVANT can cause severe and fatal interstitial lung disease (ILD)/pneumonitis.

RYBREVANT with LAZCLUZE

In MARIPOSA, ILD/pneumonitis occurred in 3.1% of patients, including Grade 3 in 1.0% and Grade 4 in 0.2% of patients. There was one fatal case (0.2%) of ILD/pneumonitis and 2.9% of patients permanently discontinued RYBREVANT and LAZCLUZE due to ILD/pneumonitis.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population, ILD/pneumonitis occurred in 2.1% of patients with 1.8% of patients experiencing Grade 3 ILD/pneumonitis. 2.1% discontinued RYBREVANT due to ILD/pneumonitis.

RYBREVANT as a Single Agent

In CHRYSALIS, ILD/pneumonitis occurred in 3.3% of patients, with 0.7% of patients experiencing Grade 3 ILD/pneumonitis. Three patients (1%) permanently discontinued RYBREVANT due to ILD/pneumonitis.

Monitor patients for new or worsening symptoms indicative of ILD/pneumonitis (e.g., dyspnea, cough, fever). For patients receiving RYBREVANT in combination with LAZCLUZE, immediately withhold both drugs in patients with suspected ILD/pneumonitis and permanently discontinue if ILD/pneumonitis is confirmed. For patients receiving RYBREVANT as a single agent or in combination with carboplatin and pemetrexed, immediately withhold RYBREVANT in patients with suspected ILD/pneumonitis and permanently discontinue if ILD/pneumonitis is confirmed.

Venous Thromboembolic Events with Concomitant Use of RYBREVANT and LAZCLUZE

RYBREVANT in combination with LAZCLUZE can cause serious and fatal venous thromboembolic (VTE) events, including deep vein thrombosis and pulmonary embolism (PE). Most events occurred during the first four months of therapy.

In MARIPOSA, VTEs occurred in 36% of patients including Grade 3 in 10% and Grade 4 in 0.5% of patients. On-study VTEs occurred in 1.2% of patients (n=5) while receiving anticoagulation therapy. There were two fatal cases of VTE (0.5%), 9% of patients had VTE leading to dose interruptions of RYBREVANT, and 7% of patients had VTE leading to dose interruptions of LAZCLUZE; 1% of patients had VTE leading to dose reductions of RYBREVANT, and 0.5% of patients had VTE leading to dose reductions of LAZCLUZE; 3.1% of patients had VTE leading to permanent discontinuation of RYBREVANT, and 1.9% of patients had VTE leading to permanent discontinuation of LAZCLUZE. The median time to onset of VTEs was 84 days (range: 6 to 777).

Administer prophylactic anticoagulation for the first four months of treatment. The use of Vitamin K antagonists is not recommended. Monitor for signs and symptoms of VTE events and treat as medically appropriate.

Withhold RYBREVANT and LAZCLUZE based on severity. Once anticoagulant treatment has been initiated, resume RYBREVANT and LAZCLUZE at the same dose level at the discretion of the healthcare provider. In the event of VTE recurrence despite therapeutic anticoagulation, permanently discontinue RYBREVANT and continue treatment with LAZCLUZE at the same dose level at the discretion of the healthcare provider.

Dermatologic Adverse Reactions

RYBREVANT can cause severe rash including toxic epidermal necrolysis (TEN), dermatitis acneiform, pruritus, and dry skin.

RYBREVANT with LAZCLUZE

In MARIPOSA, rash occurred in 86% of patients, including Grade 3 in 26% of patients. The median time to onset of rash was 14 days (range: 1 to 556 days). Rash leading to dose interruptions occurred in 37% of patients for RYBREVANT and 30% for LAZCLUZE, rash leading to dose reductions occurred in 23% of patients for RYBREVANT and 19% for LAZCLUZE, and rash leading to permanent discontinuation occurred in 5% of patients for RYBREVANT and 1.7% for LAZCLUZE.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population, rash occurred in 82% of patients, including Grade 3 (15%) adverse reactions. Rash leading to dose reductions occurred in 14% of patients, and 2.5% permanently discontinued RYBREVANT and 3.1% discontinued pemetrexed.

RYBREVANT as a Single Agent

In CHRYSALIS, rash occurred in 74% of patients, including Grade 3 in 3.3% of patients. The median time to onset of rash was 14 days (range: 1 to 276 days). Rash leading to dose reduction occurred in 5% and permanent discontinuation due to rash occurred in 0.7% of patients.

Toxic epidermal necrolysis occurred in one patient (0.3%).

Instruct patients to limit sun exposure during and for 2 months after treatment with RYBREVANT or LAZCLUZE in combination with RYBREVANT. Advise patients to wear protective clothing and use broad-spectrum UVA/UVB sunscreen. Alcohol-free (e.g., isopropanol-free, ethanol-free) emollient cream is recommended for dry skin.

When initiating treatment, administer alcohol-free emollient cream to reduce the risk of dermatologic adverse reactions. Consider prophylactic measures (eg, use of oral antibiotics). If skin reactions develop, start topical corticosteroids and topical and/or oral antibiotics. For Grade 3 reactions, add oral steroids and consider dermatologic consultation. Promptly refer patients presenting with severe rash, atypical appearance or distribution, or lack of improvement within 2 weeks to a dermatologist. For patients receiving RYBREVANT in combination with LAZCLUZE, withhold, reduce the dose, or permanently discontinue both drugs based on severity. For patients receiving RYBREVANT as a single agent or in combination with carboplatin and pemetrexed, withhold, dose reduce or permanently discontinue RYBREVANT based on severity.

Ocular Toxicity

RYBREVANT can cause ocular toxicity including keratitis, blepharitis, dry eye symptoms, conjunctival redness, blurred vision, visual impairment, ocular itching, eye pruritus, and uveitis.

RYBREVANT with LAZCLUZE

In MARIPOSA, ocular toxicity occurred in 16%, including Grade 3 or 4 ocular toxicity in 0.7% of patients. Withhold, reduce the dose, or permanently discontinue RYBREVANT and continue LAZCLUZE based on severity.

RYBREVANT with Carboplatin and Pemetrexed

Based on the pooled safety population, ocular toxicity occurred in 16% of patients. All events were Grade 1 or 2.

RYBREVANT as a Single Agent

In CHRYSALIS, keratitis occurred in 0.7% and uveitis occurred in 0.3% of patients. All events were Grade 1-2.

Promptly refer patients with new or worsening eye symptoms to an ophthalmologist. Withhold, reduce the dose, or permanently discontinue RYBREVANT based on severity.

Embryo-Fetal Toxicity

Based on animal models, RYBREVANT and LAZCLUZE can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential of the potential risk to the fetus.

Advise patients of reproductive potential to use effective contraception during treatment and for 3 months after the last dose of RYBREVANT, and for 3 weeks after the last dose of LAZCLUZE.

ADVERSE REACTIONS

RYBREVANT with LAZCLUZE

In MARIPOSA (n=421) , the most common adverse reactions (ARs) (≥20%) were rash (86%), nail toxicity (71%), infusion-related reactions (IRRs) (RYBREVANT) (63%), musculoskeletal pain (47%), stomatitis (43%), edema (43%), VTE (36%), paresthesia (35%), fatigue (32%), diarrhea (31%), constipation (29%), COVID-19 (26%), hemorrhage (25%), dry skin (25%), decreased appetite (24%), pruritus (24%), and nausea (21%). The most common Grade 3 or 4 laboratory abnormalities (≥2%) were decreased albumin (8%), decreased sodium (7%), increased ALT (7%), decreased potassium (5%), decreased hemoglobin (3.8%), increased AST (3.8%), increased GGT (2.6%), and increased magnesium (2.6%).

Serious ARs occurred in 49% of patients, with those occurring in ≥2% of patients including VTE (11%), pneumonia (4%), ILD/pneumonitis and rash (2.9% each), COVID-19 (2.4%), and pleural effusion and IRRs (RYBREVANT) (2.1% each). Fatal ARs occurred in 7% of patients due to death not otherwise specified (1.2%); sepsis and respiratory failure (1% each); pneumonia, myocardial infarction, and sudden death (0.7% each); cerebral infarction, pulmonary embolism (PE), and COVID-19 infection (0.5% each); and ILD/pneumonitis, acute respiratory distress syndrome (ARDS), and cardiopulmonary arrest (0.2% each).

RYBREVANT with Carboplatin and Pemetrexed

In MARIPOSA-2 (n=130), the most common ARs (≥20%) were rash (72%), IRRs (59%), fatigue (51%), nail toxicity (45%), nausea (45%), constipation (39%), edema (36%), stomatitis (35%), decreased appetite (31%), musculoskeletal pain (30%), vomiting (25%), and COVID-19 (21%). The most common Grade 3 to 4 laboratory abnormalities (≥2%) were decreased neutrophils (49%), decreased white blood cells (42%), decreased lymphocytes (28%), decreased platelets (17%), decreased hemoglobin (12%), decreased potassium (11%), decreased sodium (11%), increased alanine aminotransferase (3.9%), decreased albumin (3.8%), and increased gamma-glutamyl transferase (3.1%).

In MARIPOSA-2, serious ARs occurred in 32% of patients, with those occurring in >2% of patients including dyspnea (3.1%), thrombocytopenia (3.1%), sepsis (2.3%), and PE (2.3%). Fatal ARs occurred in 2.3% of patients; these included respiratory failure, sepsis, and ventricular fibrillation (0.8% each).

In PAPILLON (n=151), the most common ARs (≥20%) were rash (90%), nail toxicity (62%), stomatitis (43%), IRRs (42%), fatigue (42%), edema (40%), constipation (40%), decreased appetite (36%), nausea (36%), COVID-19 (24%), diarrhea (21%), and vomiting (21%). The most common Grade 3 to 4 laboratory abnormalities (≥2%) were decreased albumin (7%), increased alanine aminotransferase (4%), increased gamma-glutamyl transferase (4%), decreased sodium (7%), decreased potassium (11%), decreased magnesium (2%), and decreases in white blood cells (17%), hemoglobin (11%), neutrophils (36%), platelets (10%), and lymphocytes (11%).

In PAPILLON, serious ARs occurred in 37% of patients, with those occurring in ≥2% of patients including rash, pneumonia, ILD, PE, vomiting, and COVID-19. Fatal adverse reactions occurred in 7 patients (4.6%) due to pneumonia, cerebrovascular accident, cardio-respiratory arrest, COVID-19, sepsis, and death not otherwise specified.

RYBREVANT as a Single Agent

In CHRYSALIS (n=129), the most common ARs (≥20%) were rash (84%), IRR (64%), paronychia (50%), musculoskeletal pain (47%), dyspnea (37%), nausea (36%), fatigue (33%), edema (27%), stomatitis (26%), cough (25%), constipation (23%), and vomiting (22%). The most common Grade 3 to 4 laboratory abnormalities (≥2%) were decreased lymphocytes (8%), decreased albumin (8%), decreased phosphate (8%), decreased potassium (6%), increased alkaline phosphatase (4.8%), increased glucose (4%), increased gamma-glutamyl transferase (4%), and decreased sodium (4%).

Serious ARs occurred in 30% of patients, with those occurring in ≥2% of patients including PE, pneumonitis/ILD, dyspnea, musculoskeletal pain, pneumonia, and muscular weakness. Fatal adverse reactions occurred in 2 patients (1.5%) due to pneumonia and 1 patient (0.8%) due to sudden death.

LAZCLUZE Drug Interactions

Avoid concomitant use of LAZCLUZE with strong and moderate CYP3A4 inducers. Consider an alternate concomitant medication with no potential to induce CYP3A4.

Monitor for adverse reactions associated with a CYP3A4 or BCRP substrate where minimal concentration changes may lead to serious adverse reactions, as recommended in the approved product labeling for the CYP3A4 or BCRP substrate.

(Press release, Johnson & Johnson, OCT 19, 2025, View Source [SID1234656796])