Ascentage Pharma to Present Updated Data of Olverembatinib in Patients with SDH-Deficient GIST during a Mini Oral Presentation at the 2024 ESMO Congress

On September 8, 2024 Ascentage Pharma (6855.HK), a global biopharmaceutical company engaged in discovering, developing and commercializing both first-in-class and best-in-class therapies for malignancies, reported that it will present the latest clinical data of olverembatinib (HQP1351), the company’s novel drug candidate, in patients with succinate dehydrogenase- (SDH-) deficient gastrointestinal stromal tumor (GIST), in a Mini Oral at the 2024 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress (Press release, Ascentage Pharma, SEP 8, 2024, View Source;ascentage-pharma-to-present-updated-data-of-olverembatinib-in-patients-with-sdh-deficient-gist-during-a-mini-oral-presentation-at-the-2024-esmo-congress-302241379.html [SID1234646410]).

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As one of the world’s leading and most influential oncology congresses, the ESMO (Free ESMO Whitepaper) Congress showcases the latest results in some of the most cutting-edge cancer research from around the world. This year, the ESMO (Free ESMO Whitepaper) Congress will be held from September 13 to 17 in Barcelona, Spain.

"We are pleased to have this opportunity to showcase olverembatinib’s therapeutic potential for the treatment of SDH-deficient GIST," said Dr. Yifan Zhai, Chief Medical Officer of Ascentage Pharma. "As a third-generation TKI developed in house by Ascentage Pharma, olverembatinib was recently cleared by the China CDE to enter a registrational Phase III study in patients with SDH-deficient GIST. We will expeditiously advance this clinical development program in efforts to bring more treatment options to patients around the world."

Details of the Mini Oral presentation at this year’s ESMO (Free ESMO Whitepaper) Congress are as follows:

Updated efficacy results of olverembatinib (HQP1351) in patients with succinate dehydrogenase (SDH)-deficient gastrointestinal stromal tumors (GIST) and potential mechanisms of action (MOA)

Format: Mini oral
Presentation#: 1722MO
Category: Sarcoma
Date & Time: Friday September 13, 2024, 16:30 – 16:35 CEST
Speaker: Haibo Qiu, MD, PhD, Sun Yat-sen University Cancer Center

Antennova to Present Latest Data of ATN-037 in a Mini Oral Presentation at ESMO Congress 2024

On September 8, 2024 Antennova, a clinical-stage biotech company focused on oncology reported that it will present the latest data of CD73 small molecule inhibitor ATN-037 in a Mini Oral presentation at the 2024 European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress (ESMO Congress 2024), taking place from September 13th to September 17th at the Fira Barcelona Gran Via in Barcelona, Spain (Press release, Antennova, SEP 8, 2024, View Source [SID1234646411]).

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Details of the Presentation:

ATN-037 (also known as ATG-037, CD73 Oral Small Molecule Inhibitor)

Title: A First-In-Human Phase I/Ib study of ATG-037 Monotherapy and Combination Therapy with Pembrolizumab in Patients with Advanced Solid Tumours – STAMINA-01

Abstract: 6067

Presentation Number: 997MO

Date: September 16, 2024

Time:

10:50 AM – 10:55 AM (Central European Summer Time)

4:50 AM – 4:55 AM (US Eastern Time)

ATN-037 is a highly potent oral small molecule inhibitor of CD73. The STAMINA-01 Phase I/II study is investigating the safety, pharmacokinetics and optimal dose of ATN-037 as a monotherapy or in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with refractory/relapsed solid tumors. Antennova has initiated the dose optimization and dose expansion portion of the Phase II STAMINA trial of ATN-037 in Australia and plans to initiate the study in China at the end of October 2024.

As of February 29, 2024, 32 patients have been enrolled, receiving doses ranging from 20mg BID to 600mg BID. 20 of these patients who acquired checkpoint inhibitor (CPI) resistance were treated with combination therapy.
Efficacy data showed that in the 32 patients in the monotherapy group who were all evaluable, 14 achieved stable disease (SD) with a 43.8% disease control rate (DCR). In the 15 evaluable patients among the 20 patients in the combination therapy group, 3 patients (2 with melanoma and 1 with NSCLC) achieved confirmed partial response (PR) and 1 patient with non-small cell lung cancer (NSCLC) achieved unconfirmed PR with an overall response rate (ORR) of 20.0%. Additionally, 9 patients achieved SD, contributing to a 65.0% DCR.

40.6% of patients on monotherapy and 25.0% on combination therapy reported treatment-related adverse events (TRAEs). Only one patient receiving combination therapy experienced a dose-limiting toxicity grade 3 rash, while all other TRAEs were grades 1-2.
The updated results as of July 26, 2024 will be presented in the Mini Oral Session of ESMO (Free ESMO Whitepaper) Congress 2024, scheduled on September 16.
KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merk & Co., Inc., Rahway, NJ, USA.

Senhwa Biosciences Presents Clinical Data Abstract on Pidnarulex at 2024 ESMO Congress

On September 8, 2024 Senhwa Biosciences, Inc. (TPEx: 6492), a drug development company dedicated to developing first-in-class therapeutics for oncology, rare diseases, and infectious diseases, reported the presentation of a poster abstract of its investigational drug, Pidnarulex, at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress (Press release, Senhwa Biosciences, SEP 8, 2024, View Source [SID1234646412]). The abstract highlights Pidnarulex’s demonstrated efficacy in treating various solid tumors harbouring BRCA1/2 or PALB2 gene defects. This significant milestone underscores the promising potential of Pidnarulex in the realm of precision medicine.

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The findings, presented both on-site and online via the ESMO (Free ESMO Whitepaper) platform, involve patients with advanced-stage cancer who have undergone multiple prior treatments. Notably, a portion of the patients have achieved stable disease and are continuing to receive Pidnarulex as part of the ongoing clinical trial. This development marks a remarkable achievement for both Senhwa Biosciences and the collaborating Canadian and the US clinical team, reinforcing the drug’s relevance in the evolving landscape of targeted cancer therapies.

The title of the poster abstract is "Phase 1b expansion study of CX-5461 in patients with solid tumours and BRCA2 and/or PALB2 mutation." The full abstract content has been published on the ESMO (Free ESMO Whitepaper) website at 00:05 CEST on September 9, 2024, at the following link: View Source The trial report indicates that the preliminary results from Senhwa Biosciences’ clinical trials of Pidnarulex, conducted in Canada and the USA, shows that out of the first 28 enrolled patients, 22 completed at least one cycle of treatment and were evaluated for dose-limiting toxicity (DLT). The patients had previously undergone multiple lines of cancer treatment, with a median of 6 lines (ranging from 2 to 10 lines) of prior therapy, including 77% of patients who had received platinum-based chemotherapy, 41% of patients who had been treated with bevacizumab, and 86% of patients who had previously received PARP inhibitors without success. These were end-stage oncology patients with no other suitable treatment options. The median number of Pidnarulex treatments received by the patients was 4 doses (ranging from 2 to 36 doses).

### Trial Results:

Among the 15 patients who were evaluable for drug response, 40% achieved clinical benefit, with stable disease (SD) being the best therapeutic response. Among these stable disease patients, there were 5 ovarian cancer patients, including 3 with BRCA1 somatic mutations, 1 with a BRCA1 germline mutation, and 1 with HRD-related gene mutations. All 5 patients had previously failed platinum chemotherapy and PARP inhibitor treatments, with 2 of them maintaining stable disease for at least 6 months following Pidnarulex treatment, offering renewed hope for advanced-stage ovarian cancer patients.

### Trial Objectives:

This clinical trial is designed as an open-label, multicenter, multinational study, divided into the Main Study Cohort and the Exploratory Cohort. It aimed to recruit patients with BRCA2 and/or PALB2 gene deficiencies from various tumor types (pancreatic cancer, ovarian cancer, prostate cancer, and breast cancer), as well as ovarian cancer patients with BRCA1 deficiencies and/or other HRD-related homologous recombination defects. The primary goal of the trial was to determine the recommended Phase II trial dose for patients with specific genetic deficiencies, while secondary endpoints include evaluating the safety, tolerability, and antitumor activity of Pidnarulex (CX-5461).

### Trial Conclusion:

This Phase Ib study demonstrated that CX-5461 exhibit acceptable clinical tolerability and shows preliminary signs of activity, even in patients who had previously failed treatment with PARP inhibitors. Photosensitivity was found to be manageable through preventive measures.

Currently, several PARP inhibitors have been approved by the FDA for the treatment of pancreatic, breast, ovarian, and prostate cancers with BRCA1/2 gene deficiencies. In addition to BRCA1/2, the PALB2 mutated gene represents a critical factor in in triple-negative breast cancer, ranking the third most significant gene associated with this subtype. Moreover, mutations in the PALB2 gene are also associated with a higher risk of developing ovarian and pancreatic cancers, further underscoring its importance in oncology.

Senhwa’s Pidnarulex is a next-generation novel DDR drug with the potential to be developed as a rescue medication for patients who have developed resistance to PARP inhibitors. In preclinical studies, Pidnarulex has also demonstrated the ability to modulate tumor microenvironment, thereby enhancing sensitivity and efficacy of immunotherapy, including anti-PD-1 and anti-PD-L1. Senhwa looks forward to combining Pidnarulex with immunotherapy drugs such as Keytruda, which has been approved in the United States for the treatment of over 30 types of cancer. In 2023, Keytruda achieved global sales exceeding $25 billion, making it the highest-grossing drug worldwide. However, immunotherapy has its limitations, with only 20% of patients experiencing significant effects. If Pidnarulex can enhance the efficacy of immunotherapeutic agents, it is expected to provide new options for future treatment plans.

Akeso’s Ivonescimab Head-to-Head Phase III Data Against Pembrolizumab Unveiled at WCLC 2024

On September 8, 2024 Akeso (9926. HK) reported that it is thrilled to unveil groundbreaking data on its internally developed ivonescimab (a first-in-class PD-1/VEGF bispecific antibody) at the IASLC 2024 World Conference on Lung Cancer (WCLC24) (Press release, Akeso Biopharma, SEP 8, 2024, View Source [SID1234646413]). The data, presented as a Late-Breaking Abstract (LBA) with an oral presentation, comes from a registration Phase III head-to-head clinical trial comparing ivonescimab monotherapy to pembrolizumab monotherapy as a first-line treatment for PD-L1 positive (PD-L1 TPS ≥1%) locally advanced or metastatic non-small cell lung cancer (NSCLC).

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The trial results were presented by Professor Caicun Zhou, MD, PhD, Chief Physician and Director of the Department of Medical Oncology at Shanghai Pulmonary Hospital, Tongji University School of Medicine, and President-Elect of IASLC. Furthermore, Professor Zhou participated in the official WCLC press conference, discussing the clinical significance of this study.

In the HARMONi-2 study, baseline characteristics were well-balanced between the experimental and control groups. In the intention-to-treat (ITT) population, ivonescimab significantly improved progression-free survival (PFS) compared to pembrolizumab, markedly reducing the risk of disease progression or death. Subgroup analyses revealed that ivonescimab outperformed pembrolizumab across various factors, including age, sex, ECOG performance status, PD-L1 expression, histological type, and the presence of liver or brain metastases. This is the first randomized phase III study to demonstrate a clinically significant improvement in efficacy with a novel drug compared to pembrolizumab in NSCLC.

PFS Stratified HR 0.51, mPFS 11.14 Months

Ivonescimab as first-line treatment for PD-L1 positive NSCLC significantly extends median progression-free survival (mPFS) compared to pembrolizumab, with both statistical and clinical significance.

In the ITT population, ivonescimab demonstrated a median progression-free survival (mPFS) of 11.14 months compared to 5.82 months for pembrolizumab. The PFS hazard ratio (HR) was 0.51 (P<0.0001), indicating a significant 49% reduction in the risk of disease progression or death.
Ivonescimab significantly improved the objective response rate (ORR) and disease control rate (DCR) compared to pembrolizumab in the first-line treatment of PD-L1 positive non-small cell lung cancer (NSCLC) patients. The ORR for ivonescimab was 50.0%, versus 38.5% for pembrolizumab, while the DCR was 89.9% for ivonescimab versus 70.5% for pembrolizumab. These results highlight ivonescimab’s effective anti-tumor effect.
Overall survival data was not yet mature at the time of the data cutoff and will be evaluated in the future.
Significant Progression-Free Survival Benefits of Ivonescimab Across PD-L1 Expression Subgroups

Ivonescimab demonstrated significant progression-free survival benefits in patients with both low and high PD-L1.

The HARMONi-2 study enrolled participants with PD-L1 TPS ≥50%, accounting for 42.2%, aligning with the distribution in real-world populations.
In the PD-L1 TPS ≥50% population, the PFS HR for ivonescimab versus pembrolizumab was 0.46, reducing the risk of disease progression/death by 54%.
In the PD-L1 TPS 1-49% population, the PFS HR for ivonescimab versus pembrolizumab was 0.54, reducing the risk of disease progression/death by 46%.
Ivonescimab showed meaningful progression-free survival benefits in both squamous and non-squamous subgroups

In the HARMONi-2 study, 45.5% of the enrolled patients had sq-NSCLC, and 54.5% had nsq-NSCLC.
In sq-NSCLC subgroup, the PFS HR for ivonescimab and pembrolizumab was 0.48.
In nsq-NSCLC subgroup, the PFS HR for ivonescimab and pembrolizumab was 0.54.
Ivonescimab monotherapy demonstrated significant efficacy regardless of liver metastasis, brain metastasis, or a history of hemoptysis.

In this study, 12.6% of patients treated with ivonescimab had liver metastasis, and 16.7% had brain metastasis.
The PFS HR for ivonescimab and pembrolizumab in patients with brain metastasis was 0.55; in those without brain metastasis, the PFS HR was 0.53.
The PFS HR for ivonescimab and pembrolizumab in patients with liver metastasis was 0.47; in those without liver metastasis, the PFS HR was 0.53.
Ivonescimab Demonstrated Acceptable and Manageable Safety Profile Across Subgroups

In the HARMONi-2 study, 72.2% of patients with squamous cell carcinoma (sq-NSCLC) treated with ivonescimab had centrally located tumors, 10.0% had tumors with cavitation or necrosis, and 6.7% had tumors encasing large blood vessels. Despite these factors, which typically contraindicate traditional anti-VEGF therapies due to bleeding risks, ivonescimab did not significantly increase bleeding compared to the control group. The study confirms that ivonescimab has an acceptable and manageable overall safety profile, consistent with prior research.

The incidence of grade 3 or higher treatment-related adverse events (TRAEs) in sq-NSCLC patients treated with ivonescimab was comparable to that in the pembrolizumab group (22.2% vs. 18.7%).
HRQoL with ivonescimab was comparable to pembrolizumab.
Based on the results of HARMONi-2, Akeso’s partner, Summit, plans to initiate HARMONi-7 in early 2025. HARMONi-7 is currently planned as a multi-regional Phase III clinical trial that will compare ivonescimab monotherapy to pembrolizumab monotherapy in metastatic NSCLC patients whose tumors have high PD-L1 expression (PD-L1 TPS≥50%)

Dr. Michelle Xia, Founder, Chairwoman, President, and CEO of Akeso, said: "The research outcomes of HARMONi-2 are very encouraging. Pembrolizumab has been one of the most commercially successful innovative drugs globally in recent years. The clinically meaningful outcomes of ivonescimab versus pembrolizumab in a randomized, double-blind Phase III study, coupled with its demonstrated superior efficacy and manageable safety profile, affirm the immense potential of ivonescimab as a cornerstone treatment in cancer immunotherapy. This further substantiates the clinical benefit and market potential of ivonescimab on a global scale. The HARMONi-2 results presented today encourages Akeso and our partner SUMMIT to expand the clinical development of ivonescimab globally in more cancer indications for the benefit of patients with unmet medical needs. We are very impressed by Summit’s ability to execute on clinical development since the start of our collaboration, and we are excited to work with them to pave the way for approvals in the US, Europe, and the rest of the license territories in the future."

About ivonescimab
Ivonescimab is a novel global first-in-class PD-1/VEGF bi-specific immunotherapy drug internally developed by Akeso. Ivonescimab has been approved in China for treating EGFR mutated locally advanced or metastatic non-squamous NSCLC patients who have progressed after EGFR TKI treatment. It is the world’s first approved bispecific antibody with a "cancer immunotherapy + anti-angiogenesis" synergistic mechanism.

Akeso out-licensed Summit Therapeutics exclusive rights to ivonescimab for the development and commercialization in certain territories including United States, Canada, Europe, Japan, Latin America, Africa and the Middle East. Ivonescimab is known as AK112 within Akeso and SMT112 in the territories licensed to Summit.

Currently, a Phase III study of ivonescimab monotherapy versus pembrolizumab monotherapy as first-line treatment for PD-L1+ NSCLC has met its primary endpoint of progression-free survival (PFS) in an interim analysis, achieving a decisive positive outcome. Based on this study, a supplemental New Drug Application (sNDA) for ivonescimab monotherapy as first-line treatment for PD-L1+ NSCLC has been submitted and granted priority review. Additionally, a Phase III clinical study of ivonescimab combined with chemotherapy versus tislelizumab combined with chemotherapy as first-line treatment for squamous NSCLC is ongoing. The HARMONi study, an international multicenter Phase III clinical study led by Akeso’s partner Summit, is investigating ivonescimab combined with chemotherapy for EGFR-mutated, locally advanced or metastatic nsq-NSCLC that has progressed after third-generation EGFR-TKI therapy. Another international multicenter Phase III study is comparing ivonescimab combined with chemotherapy to pembrolizumab combined with chemotherapy as first-line treatment for squamous NSCLC.

Furthermore, 3 new Phase III clinical studies are either initiated or about to start, including ivonescimab combined with AK117 (CD47) as first-line treatment for PD-L1 positive squamous cell carcinoma of the head and neck (vs. pembrolizumab), ivonescimab combined regimen as first-line treatment for cholangiocarcinoma (vs. durvalumab combined regimen), and ivonescimab combined regimen as first-line treatment for pancreatic cancer. Overall, ivonescimab is engaged in over 25 clinical trials across 17 indications, including lung cancer, pancreatic cancer, breast cancer, hepatocellular carcinoma, and colorectal cancer, through both monotherapy and combination therapy approaches.

RYBREVANT® (amivantamab-vmjw) plus LAZCLUZE™ (lazertinib) show strong favorable overall survival trend versus osimertinib in EGFR-mutated advanced lung cancer

On September 8, 2024 Johnson & Johnson (NYSE: JNJ) reported longer follow-up data from the landmark Phase 3 MARIPOSA study which showed first-line treatment with RYBREVANT (amivantamab-vmjw) combined with LAZCLUZE (lazertinib) provided consistent benefit across long-term outcomes compared to osimertinib monotherapy in adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or L858R substitution mutations (Press release, Johnson & Johnson, SEP 8, 2024, View Source [SID1234646414]). The data show a strong and improving overall survival (OS) trend favoring RYBREVANT plus LAZCLUZE at approximately three years of follow-up. These results were presented in a late-breaking oral presentation at the International Association for the Study of Lung Cancer (IASLC) 2024 World Conference on Lung Cancer (WCLC) (Abstract #1146).1

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At three years (a median follow-up of 31.1 months), 61 percent of patients receiving RYBREVANT plus LACLUZE were alive compared to 53 percent of those treated with osimertinib based on an analysis performed at the request of a health authority (Median OS not estimable vs 37.3 months; hazard ratio [HR], 0.77; [95 percent confidence interval [CI], 0.61-0.96]; nominal P=0.019). Overall survival will continue to be assessed with longer term follow-up as a key secondary endpoint. The primary efficacy outcome measure was progression-free survival (PFS) as assessed by blinded independent central review (BICR).1

"By combining the multi-targeted mechanism of RYBREVANT with LAZCLUZE, a central nervous system-penetrant third-generation tyrosine kinase inhibitor, we are advancing a chemotherapy-free regimen for the first-line treatment of patients with EGFR-mutant NSCLC. This approach blocks EGFR and MET pathways and leverages the immune system, offering patients an opportunity for prolonged benefits," said Shirish M. Gadgeel, M.D., Chief of Division of Hematology and Oncology, Associate Director at Henry Ford Cancer Institute and presenting author.* "Even more encouraging is the marked improvement in the hazard ratio and the ongoing separation of survival curves, showing an eight percent improvement at three years for RYBREVANT plus LAZCLUZE compared to osimertinib. This supports the long-term benefit of the combination as a first-line treatment option in this setting."

Results further showed RYBREVANT plus LAZCLUZE demonstrated a trend toward improved central nervous system disease control compared to osimertinib at three years (HR, 0.82; [95 percent CI, 0.62-1.09]; nominal P=0.165). At the three-year landmark, intracranial PFS was double for RYBREVANT plus LAZCLUZE versus osimertinib (38 percent vs 18 percent, respectively). More patients remained on treatment at three years with the RYBREVANT combination compared to osimertinib (40 percent vs 29 percent, respectively; HR, 0.80; [95 percent CI, 0.68-0.96]; nominal P=0.014). Additionally, more patients receiving RYBREVANT and LAZCLUZE at the three-year follow-up had not started a subsequent therapy versus osimertinib (45 percent vs 32 percent, respectively; HR, 0.77; [95 percent CI, 0.65-0.93]; nominal P=0.005). Progression-free survival after first subsequent therapy was 57 percent for the RYBREVANT combination compared to 49 percent for osimertinib (HR, 0.73; [95 percent CI, 0.59-0.91]; nominal P=0.004).1

"Promising results like these presented at WCLC reinforce our mission to improve the lives of patients diagnosed with lung cancer," said Joshua Bauml, M.D., Vice President, Lung Cancer Disease Area Stronghold Leader, Johnson & Johnson Innovative Medicine. "We are encouraged by the favorable overall survival trend observed with RYBREVANT plus LAZCLUZE and are eager to see how these data evolve as we continue to follow patients over time."

As previously reported in the MARIPOSA study, the safety profile was consistent with the safety profiles of the individual treatments. The rate of discontinuation of all study treatments due to treatment-related adverse events for RYBREVANT plus LAZCLUZE was 10 percent. The rate of interstitial lung disease (including pneumonitis) was less than three percent in both arms.2

In August 2024, RYBREVANT combined with LAZCLUZE was approved following a Priority Review by the U.S. Food and Drug Administration as a first-line therapy for patients with EGFR-mutated NSCLC based on the favorable efficacy and safety profile demonstrated in this 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 ex19del or L858R substitution mutations. The primary endpoint of the study is PFS (using RECIST v1.1 guidelines) as assessed by BICR. Secondary endpoints include OS, overall response rate (ORR), duration of response (DOR), second progression-free survival (PFS2) and intracranial PFS.3

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 in 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.4

RYBREVANT is approved in the U.S., Europe and in 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. 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 L858R substitution mutations, as detected by an FDA-approved test. A marketing authorization application (MAA) and type II extension of indication application were submitted to the European Medicines Agency (EMA) seeking approval of LAZCLUZE in combination with RYBREVANT based on the MARIPOSA study.

In November 2023, Johnson & Johnson submitted a supplemental Biologics License Application (sBLA) to the U.S. FDA for RYBREVANT in combination with chemotherapy for the treatment of patients with EGFR-mutated NSCLC who progressed on or after osimertinib based on the MARIPOSA-2 study. This indication was approved in Europe in August 2024.

In June 2024, Johnson & Johnson submitted a BLA to the U.S. FDA for the subcutaneous formulation of RYBREVANT in combination with LAZCLUZE for all currently approved or submitted indications of intravenous (IV) RYBREVANT in certain patients with NSCLC. A submission for the extension of the RYBREVANT marketing authorization (line extension) was also submitted to the EMA seeking approval for this indication.

The 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 chemotherapy as a preferred (Category 1 preferred recommendation) subsequent therapy for patients with locally advanced or metastatic NCSLC with EGFR exon 19 deletions or exon 21 L858R mutations who experienced disease progression after treatment with Osimertinib.5 †‡
Amivantamab-vmjw (RYBREVANT) plus carboplatin and pemetrexed as a preferred (Category 1 preferred recommendation) first-line therapy in treatment-naive patients with newly diagnosed advanced or metastatic EGFR exon 20 insertion mutation-positive advanced NSCLC, or as a subsequent therapy option (Category 2A recommendation) for patients that have progressed on or after platinum-based chemotherapy with or without immunotherapy and have EGFR exon 20 insertion mutation-positive advanced NSCLC.5 †‡
Amivantamab-vmjw (RYBREVANT) as a subsequent therapy option (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.5 †‡
In addition to the Phase 3 MARIPOSA study, RYBREVANT is being studied in multiple clinical trials in NSCLC, including:

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 EGFR ex19del or L858R substitution NSCLC after disease progression on or after osimertinib.6
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.7
The Phase 3 PALOMA-3 (NCT05388669) study assessing LAZCLUZE with subcutaneous amivantamab compared to intravenous amivantamab in patients with EGFR-mutated advanced or metastatic NSCLC.8
The Phase 2 PALOMA-2 (NCT05498428) study assessing subcutaneous amivantamab in patients with advanced or metastatic solid tumors including EGFR-mutated NSCLC.9
The Phase 1 PALOMA (NCT04606381) study assessing the feasibility of subcutaneous administration of amivantamab based on safety and pharmacokinetics and to determine a dose, dose regimen and formulation for amivantamab subcutaneous delivery.10
The Phase 1 CHRYSALIS (NCT02609776) study evaluating RYBREVANT in patients with advanced NSCLC.11
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.12
The Phase 1/2 METalmark (NCT05488314) study assessing RYBREVANT and capmatinib combination therapy in locally advanced or metastatic NSCLC.13
The Phase 1/2 PolyDamas (NCT05908734) study assessing RYBREVANT and cetrelimab combination therapy in locally advanced or metastatic NSCLC.14
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.15
The Phase 1/2 swalloWTail (NCT06532032) study assessing RYBREVANT and docetaxel combination therapy in patients with metastatic NSCLC.16
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.17
The Phase 1b/2 OrigAMI-4 (NCT06385080) study assessing RYBREVANT monotherapy and in addition to standard-of-care therapeutic agents in patients with recurrent/metastatic head and neck squamous cell carcinoma.18
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 LACLAZA in 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.

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.19,20 The main subtypes of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma.21 Among the most common driver mutations in NSCLC are alterations in EGFR, which is a receptor tyrosine kinase controlling cell growth and division.22 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.21,22,23,24,25,26 EGFR ex19del or EGFR L858R mutations are the most common EGFR mutations.27 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.28,29 EGFR exon 20 insertion mutations are the third most prevalent activating EGFR mutation.30 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.31

IMPORTANT SAFETY INFORMATION4,32

WARNINGS AND PRECAUTIONS

Infusion-Related Reactions

RYBREVANT can cause infusion-related reactions (IRR); 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

In PAPILLON (n=151), infusion-related reactions occurred in 42% of patients treated with RYBREVANT in combination with carboplatin and pemetrexed, including Grade 3 (1.3%) adverse reactions. The incidence of infusion modifications due to IRR was 40%, and 0.7% of patients permanently discontinued RYBREVANT.

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.

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

In PAPILLON, Grade 3 ILD/pneumonitis occurred in 2.6% of patients treated with RYBREVANT in combination with carboplatin and pemetrexed, all patients required permanent discontinuation.

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%) 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 (VTEs) 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

In PAPILLON, rash occurred in 89% of patients treated with RYBREVANT in combination with carboplatin and pemetrexed, including Grade 3 (19%) adverse reactions. Rash leading to dose reductions occurred in 19% of patients, and 2% permanently discontinued RYBREVANT and 1.3% 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, dose reduce 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

In PAPILLON, ocular toxicity including blepharitis, dry eye, conjunctival redness, blurred vision, and eye pruritus occurred in 9%. All events were Grade 1-2.

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, dose reduce 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 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%).

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.