Tagrisso plus chemotherapy demonstrated a median overall survival of nearly four years, the longest benefit ever reported in a global Phase III trial in EGFR-mutated advanced lung cancer

On September 7, 2025 Astrazeneca reported positive results from the final overall survival (OS) analysis of the FLAURA2 Phase III trial showed AstraZeneca’s Tagrisso (osimertinib) with the addition of pemetrexed and platinum-based chemotherapy demonstrated a statistically significant and clinically meaningful improvement in the key secondary endpoint of OS compared to Tagrisso monotherapy in the 1st-line treatment of patients with locally advanced or metastatic epidermal growth factor receptor-mutated (EGFRm) non-small cell lung cancer (NSCLC) (Press release, AstraZeneca, SEP 7, 2025, View Source [SID1234655802]).

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These results will be presented today during the Presidential Symposium at the IASLC 2025 World Conference on Lung Cancer (WCLC) hosted by the International Association for the Study of Lung Cancer in Barcelona, Spain (abstract #PL02.04).

In the final OS analysis, Tagrisso plus chemotherapy demonstrated a median OS of nearly four years (47.5 months) compared to approximately three years (37.6 months) for Tagrisso monotherapy. At 57% data maturity, results showed Tagrisso plus chemotherapy reduced the risk of death by 23% compared to Tagrisso monotherapy (based on a hazard ratio [HR] of 0.77; 95% confidence interval [CI] 0.61-0.96; p=0.0202). An estimated 63.1% of patients treated with the combination were alive at three years and 49.1% of patients were alive at four years compared to 50.9% and 40.8%, respectively, in the monotherapy arm. Importantly, the observed OS benefit for Tagrisso plus chemotherapy versus Tagrisso monotherapy was consistent across all prespecified subgroups. Patients in the control arm received standard of care, including chemotherapy, upon progression, supporting the relevance of the OS results.

David Planchard, MD, PhD, Thoracic Oncologist at Gustave Roussy Institute of Oncology, Villejuif, France, and principal investigator for the trial, said: "The fundamental goals of lung cancer treatment are to extend survival while preserving patients’ quality of life. These compelling results, which demonstrated unprecedented median overall survival, show this combination can achieve both of these goals and support osimertinib, with or without the addition of chemotherapy, as the standard of care for patients with 1st-line advanced EGFR-mutated lung cancer. With two highly effective osimertinib-based options for these patients, physicians can better tailor treatment to individual needs and help ensure the best possible outcome for each patient."

Susan Galbraith, Executive Vice President, Oncology Haematology R&D, AstraZeneca, said: "The latest FLAURA2 trial results set a new survival standard for patients, with Tagrisso plus chemotherapy demonstrating a median overall survival of nearly four years in 1st-line advanced EGFR-mutated lung cancer—surpassing the three-year benchmark established in the FLAURA trial. Over the past decade, Tagrisso has consistently delivered strong survival benefits and tolerable safety across all stages of non-small cell lung cancer, cementing its role as the backbone therapy in EGFR-mutated lung cancer."

Summary of OS results: FLAURA2


Tagrisso plus chemotherapy (n=279)

Tagrisso monotherapy (n=278)

Median OS (in months)i,ii, iii

47.5 (41.0-NCiv)

37.6 (33.2 ,43.2)

Hazard ratio (95% CI)

0.77 (0.61-0.96)

Stratified log-rank p-valuev

0.0202

Number of deaths, n (%)

144 (51.6)

171 (61.5)

Data maturity

57%

OS rate at 24 months (%)

79.7 (74.5-84.0)

71.5 (65.8-76.5)

OS rate at 36 months (%)

63.1 (57.1-68.5)

50.9 (44.8-56.6)

OS rate at 48 months (%)

49.1 (43.0-55.0)

40.8 (34.9-46.6)

i. OS data cut-off date was 12 June 2025
ii. Median follow-up duration for OS in censored patients at data cut-off: 51.2 (0.2-60.4) months for Tagrisso plus chemotherapy and 51.3 (0.1-60.1) months for Tagrisso monotherapy
iii. Calculated by Kaplan–Meier method
iv. Not calculable
v. For statistical significance, a 2-sided p-value of less than 0.04953, as determined by the O’Brien and Fleming spending rule, was required

With longer follow-up, the safety profile of Tagrisso plus chemotherapy continued to be manageable and consistent with the established profiles of the individual medicines. Grade 3 or higher adverse events (AEs) from all causes occurred in 70% of patients in the Tagrisso plus chemotherapy arm, driven by well-characterised chemotherapy-related AEs, versus 34% in the Tagrisso monotherapy arm, similar to the rates reported at the primary analysis presented at the IASLC 2023 WCLC (64% versus 27%, respectively). Discontinuation rates due to AEs and on-target toxicities were low in both trial arms (12% versus 7%).

Notes

NSCLC
Lung cancer is the leading cause of cancer death among men and women, accounting for about one-fifth of all cancer deaths.1 Lung cancer is broadly split into small cell lung cancer or NSCLC, the latter accounting for 80-85% of cases.1-2 Approximately 75% of people are diagnosed with advanced NSCLC.3 Additionally, about 10-15% of NSCLC patients in the US and Europe, and 30-40% of patients in Asia have EGFRm NSCLC.4-6

While EGFR-tyrosine kinase inhibitors (TKI) have significantly improved outcomes in the 1st-line setting, mechanisms of resistance and disease progression are extremely common, and a significant unmet need exists in later-line settings for effective and well-tolerated treatment options.7-10

FLAURA2
FLAURA2 is a randomised, open-label, multi-centre, global Phase III trial in previously untreated patients with locally advanced (Stage IIIB-IIIC) or metastatic (Stage IV) NSCLC whose tumors have EGFR exon 19 deletion or exon 21 L858R mutations. Patients were treated with Tagrisso 80mg QD oral tablets with the addition of chemotherapy (pemetrexed (500mg/m2) plus cisplatin (75mg/m2) or carboplatin (AUC5) every three weeks for four cycles, followed by Tagrisso with pemetrexed maintenance every three weeks.

The trial enrolled 557 patients in more than 150 centres across more than 20 countries, including in the US, Europe, South America and Asia. The primary endpoint is PFS with OS as the key secondary endpoint.

Tagrisso
Tagrisso (osimertinib) is a third-generation, irreversible EGFR-TKI with proven clinical activity in NSCLC, including against central nervous system metastases. Tagrisso (40mg and 80mg QD oral tablets) has been used to treat more than one million patients across its indications worldwide and AstraZeneca continues to explore Tagrisso as a treatment for patients across multiple stages of EGFRm NSCLC.

Tagrisso is approved as monotherapy in more than 120 countries including the US, EU, China and Japan. Approved indications include for 1st-line treatment of patients with locally advanced or metastatic EGFRm NSCLC, locally advanced or metastatic EGFR T790M mutation-positive NSCLC, adjuvant treatment of early-stage EGFRm NSCLC and locally advanced, unresectable NSCLC following platinum-based chemoradiation therapy (CRT). Tagrisso is also approved in combination with chemotherapy in more than 80 countries, including the US, EU, China and Japan, for 1st-line treatment of patients with locally advanced or metastatic EGFRm NSCLC.

There is an extensive body of evidence supporting the use of Tagrisso in EGFRm NSCLC, and it is the only targeted therapy shown to improve patient outcomes across all stages of the disease.

In late-stage disease, Tagrisso demonstrated improved outcomes as monotherapy in the FLAURA Phase III trial and in combination with chemotherapy in the FLAURA2 Phase III trial. Tagrisso is also being investigated in this setting in combination with Orpathys (savolitinib) in the SAFFRON Phase III trial and in combination with Datroway (datopotamab deruxtecan or Dato-DXd) in the TROPION-Lung14 and TROPION-Lung15 Phase III trials.

Tagrisso also showed improved outcomes in early-stage disease in the NeoADAURA and ADAURA Phase III trials and in locally advanced stages in the LAURA Phase III trial. As part of AstraZeneca’s ongoing commitment to treating patients as early as possible in lung cancer, Tagrisso is also being investigated in the early-stage adjuvant resectable setting in the ADAURA2 Phase III trial.

Ifinatamab Deruxtecan Demonstrated Clinically Meaningful Response Rates in Patients with Extensive-Stage Small Cell Lung Cancer in IDeate-Lung01 Phase 2 Trial

On September 7, 2025 Merck & Co. reported results from the IDeate-Lung01 Phase 2 trial showed ifinatamab deruxtecan (I-DXd) demonstrated clinically meaningful response rates in patients with previously treated extensive-stage small cell lung cancer (ES-SCLC) (Press release, Merck & Co, SEP 7, 2025, View Source [SID1234655801]). These data were presented today during a late-breaking presentation (OA06.03) and included as part of the press program at the 2025 World Conference on Lung Cancer hosted by the International Association for the Study of Lung Cancer (#WCLC25).

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Ifinatamab deruxtecan is a specifically engineered, potential first-in-class B7-H3 directed DXd antibody drug conjugate (ADC) discovered by Daiichi Sankyo (TSE: 4568) and being jointly developed by Daiichi Sankyo and Merck (NYSE: MRK), known as MSD outside of the United States and Canada.

SCLC is aggressive and progresses rapidly to the distant metastatic stage, which has a low five-year survival rate. While conventional standard of care treatments for patients with advanced SCLC may help improve outcomes, there is a need for additional subsequent treatment approaches.

In August 2025, ifinatamab deruxtecan was granted Breakthrough Therapy Designation (BTD) by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with ES-SCLC with disease progression on or after platinum-based chemotherapy.

Response was assessed in patients who received ifinatamab deruxtecan (12 mg/kg) in both the dose optimization and single-arm expansion parts of the trial. A confirmed objective response rate (ORR) of 48.2% (95% confidence interval [CI]: 39.6–56.9) was observed with ifinatamab deruxtecan in 137 patients with previously treated ES-SCLC as assessed by blinded independent central review (BICR). Three complete responses (CRs), 63 partial responses (PRs) and 54 cases of stable disease (SD) were seen. A median duration of response (DOR) of 5.3 months (95% CI: 4.0–6.5) and a disease control rate (DCR) of 87.6% (95% CI: 80.9–92.6) were observed. Median progression-free survival (PFS) was 4.9 months (95% CI: 4.2–5.5) and median overall survival (OS) was 10.3 months (95% CI: 9.1–13.3). Disease progression and time-to-event results support further randomized, controlled assessment.

In a subset of patients (n=32) receiving ifinatamab deruxtecan as a second-line treatment, a confirmed ORR of 56.3% (95% CI: 37.7–73.6) was observed as assessed by BICR. Eighteen PRs and 13 cases of SD were seen in this subset of patients. A median DOR of 7.2 months (95% CI: 3.6–NE) and a DCR of 96.9% (95% CI: 83.8–99.9) were observed. Median PFS of 5.6 months (95% CI: 3.9–8.1) and median OS of 12.0 months (95% CI: 7.3–19.1) were seen.

In a subset of patients (n=105) receiving ifinatamab deruxtecan in a third-line and beyond setting, a confirmed ORR of 45.7% (95% CI: 36.0–55.7) with three CRs, 45 PRs and 41 cases of SD were seen. A DCR of 84.8% (95% CI: 76.4–91.0) was observed in these patients.

In an exploratory analysis, an intracranial ORR of 46.2% (95% CI: 33.7–59.0) was observed by CNS RECIST v1.1 in a subset of patients (n=65) with brain metastases at baseline. A full subgroup analysis (2760MO) will be presented at 2025 European Society for Medical Oncology (#ESMO25).

"Patients with extensive-stage small cell lung cancer have an extremely poor prognosis despite current standard of care treatment options," said Myung-Ju Ahn, MD, PhD, Professor, Department of Hematology & Oncology, Samsung Medical Center, Sungkyunkwan University School of Medicine, Seoul, Republic of Korea. "The impressive response rates observed in IDeate-Lung01 provide further evidence of the potential role that ifinatamab deruxtecan could play in treating this aggressive form of lung cancer."

The safety profile observed in IDeate-Lung01 was consistent with that seen for ifinatamab deruxtecan in the Phase 1 trial with no new safety signals identified. Grade 3 or higher treatment-related adverse events (TRAEs) occurred in 36.5% of patients. The most common (>10%) grade 3 or higher TRAEs were neutropenia (13.9%), lymphopenia (12.4%), and anemia (10.2%). Seventeen patients (12.4%) had confirmed treatment-related interstitial lung disease (ILD)/pneumonitis as determined by an independent adjudication committee. The majority of ILD/pneumonitis events were low grade, with 11 grade 1 or 2 (8.0%), four grade 3 (2.9%), and two grade 5 (1.5%) ILD/pneumonitis events observed as of the data cutoff of March 3, 2025.

"In these primary results from IDeate-Lung01, ifinatamab deruxtecan produced clinically meaningful responses in patients with previously treated extensive-stage small cell lung cancer," said Ken Takeshita, MD, Global Head, R&D, Daiichi Sankyo. "These data reinforce the potential benefit of this B7-H3 directed antibody drug conjugate in patients who have received one or more lines of platinum-based chemotherapy and will support our ongoing discussions with global regulatory authorities."

"Small cell lung cancer is the second most common type of lung cancer globally, with 15 percent of patients impacted by this particularly devastating form of the disease," said Eliav Barr, MD, Senior Vice President, Head of Global Clinical Development and Chief Medical Officer, Merck Research Laboratories. "With limited advances over the last 30 years, there is a high unmet need for new medicines and novel mechanisms of action that could provide additional options to patients with extensive-stage small cell lung cancer."

A majority of patients (54.7%) in IDeate-Lung01 received a median of two prior lines of treatment, including immunotherapy (81%), topoisomerase I inhibitor (32.1%), lurbinectedin (21.2%), amrubicin (8.8%) and DLL3-targeting T-cell engager (8.0%). As of the data cutoff, the median treatment duration was 4.8 months (range: 0.7–22.7) and 14 patients remain on treatment.

Efficacy Measure Ifinatamab Deruxtecan 12 mg/kg
Total Population (N=137) Second-Line Subset (n=32) Third-Line Plus and Beyond Subset (n=105)
Confirmed ORR, % (95% CI) 48.2% (39.6–56.9) 56.3% (37.7–73.6) 45.7% (36.0–55.7)
CR, n (%) 3 (2.2%) 0 3 (2.9%)
PR, n (%) 63 (46.0%) 18 (56.3%) 45 (42.9%)
SD, n (%) 54 (39.4%) 13 (40.6%) 41 (39.0%)
DCR (95% CI), % 87.6% (80.9–92.6) 96.9% (83.8–99.9) 84.8% (76.4–91.0)
DOR, median (95% CI), months 5.3 months (4.0–6.5) 7.2 months (3.6–NE) 4.3 months (3.7–5.8)
TTR, median (95% CI), months 1.4 months (1.0–8.1) 1.4 months (1.2–4.0) N/A
PFS, median (95% CI), months 4.9 months (4.2-5.5) 5.6 months (3.9–8.1) N/A
OS, median (95% CI), months 10.3 months (9.1-13.3) 12.0 months (7.3–19.1) N/A
CR, complete response; DCR, disease control rate; DOR, duration of response; N/A, not available; NE, not evaluable; ORR, objective response rate; OS, overall survival; PR, partial response; PFS, progression-free survival; SD, stable disease; TTR, time to response
About IDeate-Lung01

IDeate-Lung01 is a global, multicenter, randomized, open-label, two-part Phase 2 trial evaluating the safety and efficacy of ifinatamab deruxtecan in patients with ES-SCLC previously treated with at least one prior line of platinum-based chemotherapy and a maximum of three prior lines of therapy. Patients with asymptomatic brain metastases (untreated or previously treated) were eligible to participate.

In the first part of the trial (dose optimization), patients were randomized 1:1 to receive ifinatamab deruxtecan (8 or 12 mg/kg) given intravenously once every three weeks. In the second part of the trial (dose expansion), patients received ifinatamab deruxtecan (12 mg/kg) intravenously at the same dosing interval.

The primary endpoint is ORR as assessed by BICR per RECIST v1.1. Secondary endpoints included DOR, PFS, DCR, TTR, OS, pharmacokinetics and safety. Intracranial ORR was assessed by BICR as an exploratory analysis.

IDeate-Lung01 enrolled 187 patients in Asia, Europe and North America. For more information about the trial, visit ClinicalTrials.gov.

About small cell lung cancer

More than 2.48 million lung cancer cases were diagnosed globally in 2022. Small cell lung cancer (SCLC) is the second most common type of lung cancer, accounting for approximately 15% of cases. SCLC is aggressive and progresses rapidly to the distant metastatic stage, which has a low five-year survival rate. While conventional standard of care treatments for patients with advanced SCLC may help improve outcomes, there is a need for additional subsequent treatment approaches.

About B7-H3

B7-H3 is a transmembrane protein that belongs to the B7 family of proteins, which bind to the CD28 family of receptors that includes PD-1. B7-H3 is overexpressed in a wide range of cancer types, including SCLC, and its overexpression has been shown to correlate with poor prognosis, making B7-H3 a promising therapeutic target. There are currently no B7-H3 directed medicines approved for the treatment of any cancer.

About ifinatamab deruxtecan

Ifinatamab deruxtecan is an investigational potential first-in-class B7-H3 directed ADC. Designed using Daiichi Sankyo’s proprietary DXd ADC Technology, ifinatamab deruxtecan is comprised of a humanized anti-B7-H3 IgG1 monoclonal antibody attached to a number of topoisomerase I inhibitor payloads (an exatecan derivative, DXd) via tetrapeptide-based cleavable linkers.

Ifinatamab deruxtecan was granted Breakthrough Therapy Designation by the U.S. Food and Drug Administration (FDA) for the treatment of adult patients with extensive-stage SCLC with disease progression on or after platinum-based chemotherapy.

Ifinatamab deruxtecan has been granted orphan drug designation by the U.S. FDA, European Commission, Japan Ministry of Health, Labour and Welfare and Taiwan Food and Drug Administration for the treatment of SCLC.

About the ifinatamab deruxtecan clinical development program

A comprehensive global clinical development program is underway evaluating the efficacy and safety of ifinatamab deruxtecan monotherapy and in combination with other cancer medicines across multiple cancers.

RYBREVANT® (amivantamab-vmjw) plus LAZCLUZE® (lazertinib) prevents acquired resistance versus osimertinib in first-line EGFR-mutated non-small cell lung cancer

On September 6, 2025 Johnson & Johnson (NYSE:JNJ) reported new analyses from the Phase 3 MARIPOSA study showing that first-line treatment with RYBREVANT (amivantamab-vmjw) plus LAZCLUZE (lazertinib) significantly reduces the development of epidermal growth factor receptor (EGFR)- and MET-driven resistance compared with osimertinib in patients with EGFR-mutated non-small cell lung cancer (NSCLC) with exon 19 deletion (ex19del) or L858R mutations (Poster Abstract PT1.03) (Press release, Johnson & Johnson, SEP 6, 2025, View Source [SID1234655791]). These resistance data build on the combination’s previously reported and unmatched overall survival benefit in a chemotherapy-free regimen, which is projected to exceed four years, one year beyond the median observed with osimertinib, and underscore its potential to change the biology of the disease by preventing acquired resistance.2,3 Late-breaking results are being presented at the International Association for the Study of Lung Cancer (IASLC) 2025 World Congress on Lung Cancer (WCLC).

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Resistance to third-generation EGFR tyrosine kinase inhibitors (TKIs), such as osimertinib given alone or with chemotherapy, remains a common and major barrier to long-term disease control.4 This ongoing challenge underscores the need for next-generation strategies that can more effectively prevent the development of resistance to EGFR and MET and extend survival for patients with EGFR-mutated lung cancer.

"We now have a body of evidence that suggests TKI monotherapy is no longer enough in the first-line treatment of EGFR-mutated lung cancer," said Professor Sanjay Popat*, FRCP, Ph.D., medical oncologist at the Royal Marsden Hospital and the Institute of Cancer Research in the United Kingdom. "The MARIPOSA results show that combining RYBREVANT with LAZCLUZE is an important step forward, reducing EGFR- and MET-driven resistance seen with TKI-based therapy and giving patients a longer, stronger first response."

Consistent with prior data presented at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2024 Congress,5 these updated analyses from the MARIPOSA study confirm that patients treated with RYBREVANT plus LAZCLUZE were less likely to develop the two main types of resistance (MET amplification and EGFR mutations) compared to those treated with osimertinib alone. MET amplifications occurred in three percent of patients on the combination versus 13 percent on osimertinib (P=0.002), and secondary EGFR mutations (such as C797S) were significantly lower for RYBREVANT plus LAZCLUZE (1 percent vs 8 percent; P=0.01). Acquired MET amplification led to early discontinuation in 23 percent of patients on osimertinib within six months, compared with four percent on RYBREVANT plus LAZCLUZE. Among patients who stayed on the combination for at least six months, acquired resistance was rare, with two percent developing MET amplification and no EGFR C797S mutations observed. The analysis also found greater overall genetic diversity of resistance in patients treated with osimertinib, particularly among patients with EGFR- and MET-based alterations.1

"Choosing the first treatment for EGFR-mutated NSCLC is one of the most important decisions we make. It can influence how the disease progresses over time," said Joshua Bauml, M.D., Vice President, Lung Cancer Disease Area Leader, Johnson & Johnson Innovative Medicine. "These data show RYBREVANT plus LAZCLUZE changes the biology of disease by blocking the resistance pathways cancers typically use to overcome treatment. By preventing resistance in the frontline, we can extend survival and keep future treatment options open for patients. These are benefits not seen with prior therapies or emerging combinations."

The safety profile of RYBREVANT plus LAZCLUZE was consistent with the primary analysis and no new safety signals emerged with longer-term follow-up. Most AEs (grade 3 or higher) occurred early in treatment. RYBREVANT studies suggest that using preemptive or prophylactic measures can help lower the overall number and severity of skin reactions, infusion-related reactions and venous thromboembolic events.6,7,8

RYBREVANT plus LAZCLUZE is approved in the United States, Europe and other markets around the world for patients with first-line EGFR-mutated NSCLC based on the Phase 3 MARIPOSA study.

About the MARIPOSA Study
MARIPOSA (NCT04487080), which enrolled 1,074 patients, is a randomized, Phase 3 study evaluating RYBREVANT in combination with LAZCLUZE versus osimertinib and versus LAZCLUZE alone in first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR exon 19 deletion (ex19del) or substitution mutations. The primary endpoint of the study is progression-free survival (PFS) (using RECIST v1.1 guidelines**) as assessed by BICR. Secondary endpoints include overall survival, overall response rate, duration or response, progression-free survival after first subsequent therapy (PFS2) and intracranial PFS.9

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 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.11 †‡
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.11 †‡
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. 11 †‡
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. 11 †‡
RYBREVANT is being studied in multiple clinical trials in NSCLC, 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.12
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.13
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.14
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.15
The Phase 2 PALOMA-2 (NCT05498428) study assessing SC amivantamab in patients with advanced or metastatic solid tumors including EGFR-mutated NSCLC.16
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.17
The Phase 1 CHRYSALIS (NCT02609776) study evaluating RYBREVANT in patients with advanced NSCLC.18
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.19
The Phase 1/2 METalmark (NCT05488314) study assessing RYBREVANT and capmatinib combination therapy in locally advanced or metastatic NSCLC.20
The Phase 1/2 swalloWTail (NCT06532032) study assessing RYBREVANT and docetaxel combination therapy in patients with metastatic NSCLC.21
The Phase 1/2 PolyDamas (NCT05908734) study assessing RYBREVANT and cetrelimab combination therapy in locally advanced or metastatic NSCLC.22
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.23
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.24
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.25
The legal manufacturer for RYBREVANT is Janssen Biotech, Inc.

For more information, visit: View Source

About LAZCLUZE

In 2018, Janssen Biotech, Inc., entered into a license and collaboration agreement with Yuhan Corporation for the development of LAZCLUZE (marketed as LECLAZA in South Korea). LAZCLUZE is an oral, third-generation, brain-penetrant EGFR TKI that targets both the T790M mutation and activating EGFR mutations while sparing wild-type EGFR. An analysis of the efficacy and safety of LAZCLUZE from the Phase 3 LASER301 study was published in The Journal of Clinical Oncology in 2023.26

The legal manufacturer for LAZCLUZE is Janssen Biotech, Inc. and Yuhan Corporation.

About Non-Small Cell Lung Cancer

Worldwide, lung cancer is one of the most common cancers, with NSCLC making up 80 to 85 percent of all lung cancer cases.27,28 The main subtypes of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma.29 Among the most common driver mutations in NSCLC are alterations in EGFR, which is a receptor tyrosine kinase controlling cell growth and division.30 EGFR mutations are present in 10 to 15 percent of Western patients with NSCLC with adenocarcinoma histology and occur in 40 to 50 percent of Asian patients.27,28,31,32,33,34 EGFR ex19del or EGFR L858R mutations are the most common EGFR mutations.35 The five-year survival rate for all people with advanced NSCLC and EGFR mutations treated with EGFR tyrosine kinase inhibitors (TKIs) is less than 20 percent.36,37 EGFR exon 20 insertion mutations are the third most prevalent activating EGFR mutation.38 Patients with EGFR exon 20 insertion mutations have a real-world five-year overall survival (OS) of eight percent in the frontline setting, which is worse than patients with EGFR ex19del or L858R mutations, who have a real-world five-year OS of 19 percent.39

IMPORTANT SAFETY INFORMATION10,40

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

RYBREVANT can cause infusion-related reactions (IRR) 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

RYBREVANT in combination with LAZCLUZE can cause infusion-related reactions. In MARIPOSA (n=421), IRRs occurred in 63% of patients treated with RYBREVANT in combination with LAZCLUZE, including Grade 3 in 5% and Grade 4 in 1% of patients. The incidence of infusion modifications due to IRR was 54% of patients, and IRRs leading to dose reduction of RYBREVANT occurred in 0.7% of patients. Infusion-related reactions leading to permanent discontinuation of RYBREVANT occurred in 4.5% of patients receiving RYBREVANT in combination with LAZCLUZE.

RYBREVANT with Carboplatin and Pemetrexed

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

RYBREVANT as a Single Agent

In CHRYSALIS (n=302), IRR occurred in 66% of patients treated with RYBREVANT. Among patients receiving treatment on Week 1 Day 1, 65% experienced an IRR, while the incidence of IRR was 3.4% with the Day 2 infusion, 0.4% with the Week 2 infusion, and cumulatively 1.1% with subsequent infusions. Of the reported IRRs, 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 due to IRR was 62% and 1.3% of patients permanently discontinued RYBREVANT due to IRR.

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 infusion-related reactions. Monitor patients for signs and symptoms of infusion reactions during RYBREVANT infusion 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 treated with RYBREVANT in combination with LAZCLUZE, 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% treated with RYBREVANT in combination with carboplatin and pemetrexed 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 treated with RYBREVANT, 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 (VTE) 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. The majority of these events occurred during the first four months of therapy.

In MARIPOSA, VTEs occurred in 36% of patients receiving RYBREVANT in combination with LAZCLUZE, 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 treated with RYBREVANT in combination with LAZCLUZE, 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 treated with RYBREVANT in combination with carboplatin and pemetrexed, 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 treated with RYBREVANT as a single agent, including Grade 3 rash 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% of patients, and RYBREVANT was permanently discontinued due to rash in 0.7% of patients.

Toxic epidermal necrolysis occurred in one patient (0.3%) treated with RYBREVANT as a single agent.

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 RYBREVANT treatment with or without LAZCLUZE, administer alcohol-free emollient cream to reduce the risk of dermatologic adverse reactions. Consider prophylactic measures (e.g. use of oral antibiotics) to reduce the risk of dermatologic reactions. 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% of patients treated with RYBREVANT in combination with LAZCLUZE, 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 treated with RYBREVANT in combination with carboplatin and pemetrexed. 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 treated with RYBREVANT. 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 its mechanism of action and findings from 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 female patients of reproductive potential to use effective contraception during treatment and for 3 months after the last dose of RYBREVANT.

Advise females of reproductive potential to use effective contraception during treatment with LAZCLUZE and for 3 weeks after the last dose. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with LAZCLUZE and for 3 weeks after the last dose.

Adverse Reactions

RYBREVANT with LAZCLUZE

For the 421 patients in the MARIPOSA clinical trial who received RYBREVANT in combination with LAZCLUZE, the most common adverse reactions (≥20%) were rash (86%), nail toxicity (71%), infusion-related reactions (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%), nausea (21%), and ocular toxicity (16%). 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 adverse reactions occurred in 49% of patients who received RYBREVANT in combination with LAZCLUZE. Serious adverse reactions occurring in ≥2% of patients included VTE (11%), pneumonia (4%), ILD/pneumonitis and rash (2.9% each), COVID-19 (2.4%), and pleural effusion and infusion-related reaction (RYBREVANT) (2.1% each). Fatal adverse reactions occurred in 7% of patients who received RYBREVANT in combination with LAZCLUZE 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

For the 130 patients in the MARIPOSA-2 clinical trial who received RYBREVANT in combination with carboplatin and pemetrexed, the most common adverse reactions (≥20%) were rash (72%), infusion-related reactions (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 adverse reactions occurred in 32% of patients who received RYBREVANT in combination with carboplatin and pemetrexed. Serious adverse reactions in >2% of patients included dyspnea (3.1%), thrombocytopenia (3.1%), sepsis (2.3%), and pulmonary embolism (2.3%). Fatal adverse reactions occurred in 2.3% of patients who received RYBREVANT in combination with carboplatin and pemetrexed; these included respiratory failure, sepsis, and ventricular fibrillation (0.8% each).

For the 151 patients in the PAPILLON clinical trial who received RYBREVANT in combination with carboplatin and pemetrexed, the most common adverse reactions (≥20%) were rash (90%), nail toxicity (62%), stomatitis (43%), infusion-related reaction (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 adverse reactions occurred in 37% of patients who received RYBREVANT in combination with carboplatin and pemetrexed. Serious adverse reactions in ≥2% of patients included rash, pneumonia, ILD, pulmonary embolism, 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

For the 129 patients in the CHRYSALIS clinical trial who received RYBREVANT as a single agent, the most common adverse reactions (≥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 adverse reactions occurred in 30% of patients who received RYBREVANT. Serious adverse reactions in ≥2% of patients included pulmonary embolism, 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.

Please read full Prescribing Information for RYBREVANT.

Please read full Prescribing Information for LAZCLUZE.

Roche receives CE IVDR approval for HER2 (4B5) companion diagnostic test to identify HER2-ultralow breast cancer and biliary tract cancer patients

On September 5, 2025 Roche (SIX: RO, ROG; OTCQX: RHHBY) reported it has received CE IVDR approval for two label expansions for its VENTANA HER2 (4B5) Rabbit Monoclonal Primary Antibody RxDx* assay (Press release, Hoffmann-La Roche, SEP 5, 2025, View Source [SID1234655795]). HER2 is a receptor protein expressed in a variety of cancers and serves as a predictive biomarker to help determine if a patient will respond to HER2-targeted therapy.

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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!

The VENTANA HER2 (4B5) test is the first and only companion diagnostic approved to identify patients with HR-positive metastatic breast cancer that are HER2-ultralow. These patients may be eligible for treatment with ENHERTU (trastuzumab deruxtecan), a specifically engineered HER2-directed antibody drug conjugate (ADC) discovered by Daiichi Sankyo and being jointly developed and commercialised by Daiichi Sankyo and AstraZeneca. In addition, this test is now the first and only companion diagnostic to aid in the assessment of HER2-positive status to identify biliary tract cancer patients with an immunohistochemistry score of 3+ who are eligible for treatment with Jazz Pharmaceuticals’ ZIIHERA (zanidatamab-hrii).

"This is about creating new options for patients facing some of the toughest cancers," said Jill German, Head of Pathology Lab at Roche Diagnostics. "Our understanding of HER2 is rapidly evolving, and this expanded approval ensures our diagnostics are leading the way. We’re enabling clinicians to unlock personalized, life-altering treatments for patients who urgently need them."

Advancing Science in HER2-ultralow Breast Cancer
In Europe this year, more than 564,000 people will be diagnosed with breast cancer, and more than 145,000 are estimated to die from the disease.1 Metastatic breast cancer incidence is rising in younger populations and is the leading cause of breast cancer related death.2,3

HER2 interpretation in breast cancer continues to evolve beyond the traditional "positive" or "negative" classifications. The test now enables the identification of a new patient population designated as "HER2-ultralow," referring to patients who have very low levels of HER2 expression, even lower than the existing HER2-low category. Approximately 20-25 percent of hormone receptor (HR)-positive, HER2-negative breast cancer patients may be considered HER2-ultralow.4 These patients may now be eligible for ENHERTU.

The VENTANA HER2 (4B5) test was used in the DESTINY-Breast06 trial,5 which demonstrated a significant improvement in progression-free survival with ENHERTU compared to standard of care chemotherapy in patients with HER2-low and HER2-ultralow metastatic breast cancer.6

Addressing Unmet Needs in Biliary Tract Cancer
In Europe, the incidence of biliary tract cancer (BTC) and mortality rates from the disease have been increasing in the past few decades.7 BTC is often diagnosed at an advanced stage, and patients currently have very few treatment options.8 The prognosis for these patients is generally poor.9 The VENTANA HER2 (4B5) test is now approved to identify BTC patients with HER2-positive status who may be eligible for treatment with ZIIHERA.

About VENTANA HER2 (4B5) Rabbit Monoclonal Primary Antibody RxDx
The VENTANA HER2 (4B5) Rabbit Monoclonal Primary Antibody RxDx assay delivers timely, clear and reliable results, driving diagnostic certainty and enabling therapeutic decisions that can lead to better outcomes for patients. Previously indicated as an aid to identify certain breast cancer patients eligible for HER2-targeted treatment with Herceptin, KADCYLA, PERJETA, or ENHERTU,10 and gastric cancer patients eligible for treatment with Herceptin, the test is used in combination with the fully automated VENTANA BenchMark slide staining instrument.

The assay standardizes all immunohistochemistry (IHC) processes from baking through staining, and reduces the possibility of human error.10 It also minimizes inherent variability resulting from individual reagent dilution and other processes found in manual and semi-automated IHC methods. The HER2 (4B5) clone achieves consistently high proficiency assessment scores compared to other clones11 and demonstrates high concordance with HER2 FISH,12,13 empowering laboratories to employ the most widely adopted and reliable HER2 IHC primary antibody.

For more information about the portfolio, please visit the Roche Diagnostics Pathology Lab companion diagnostics page.

Senhwa Biosciences CX-5461 Secures NCI Support to Advance into MYC-Driven Lymphoma, Driving Cross-Cancer and Global Licensing Value

On September 5, 2025 Senhwa Biosciences, Inc. (TPEx: 6492), a clinical-stage biopharmaceutical company reported that the U.S. Food and Drug Administration (FDA) has granted IND clearance for its innovative drug candidate Pidnarulex(CX-5461) ina Phase 1b/2 clinical trial in patients with B-cell lymphoma subtypes that harbors MYC gene aberrations (Press release, Senhwa Biosciences, SEP 5, 2025, View Source [SID1234655794]).

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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

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Given that nearly 30% of all cancers are associated with MYC oncogene overexpression, the clinical development of CX-5461 is generating significant anticipation. If the trial delivers positive results, CX-5461 could become the first pan-cancer therapy specifically targeting MYC-driven tumors, unlocking substantial licensing opportunities and market potentials for Senhwa.

MYC as a Cancer Driver – Genetic Aberrants in Nearly One-Third of Patients

MYC is recognized as a critical oncogene, with gene amplification or related alterations present in approximately 28% of tumors across multiple cancer types, including lung, breast, liver, lymphoma, prostate, and endometrial cancers. MYC functions as a transcription factor, notably enhancing glycolysis and biosynthetic pathways to meet the energy and biomass demands of rapidly proliferating cancer cells, which accelerates malignant growth, increases recurrence rates, and reduces overall survival.

First-in-Class G4 Targeting Therapy Against Hard-to-Treat Lymphomas

CX-5461 is the world’s first and most advanced G-quadruplex (G4) stabilizer in clinical development. Preclinical studies have demonstrated its ability to suppress MYC expression and inhibit tumor progression. Moreover, in a blood cancer trial conducted by Senhwa’s clinical partner Peter MacCallum Cancer Center (PMCC) in Australia, CX-5461 delivered promising results in patients with B-cell lymphoma.

"From preclinical models to haematologic malignancies study, CX-5461 has demonstrated potential efficacy in MYC gene-driven tumors. With FDA’s clearance for this Phase 1b/2 trial, we are excited to broaden its potential application from refractory lymphomas to multiple cancer indications in the future, offering patients a truly groundbreaking treatment option," said Jason Huang, Chief Medical Officer of Senhwa.

B-cell Lymphoma Market Potential Exceeds USD 10 Billion

According to BioSpace, the global B-cell lymphoma therapeutics market reached USD 4.9 billion in 2024, and is projected to grow to USD 8.9 billion by 2035, representing a steady CAGR of 5.79% over the next decade. Demand for innovative targeted therapies is particularly strong among relapsed and refractory lymphoma patients, underscoring an unmet medical need.

With its differentiated mechanism of action and precision oncology potential, CX-5461 could significantly enhance Senhwa’s global licensing value and emerge as a next-generation standard of care in B-cell lymphoma treatment.