Jazz Pharmaceuticals Presents Overall Survival and Longer Follow-Up Data from HERIZON-BTC-01 Trial Evaluating Zanidatamab in Previously Treated HER2-Positive Biliary Tract Cancer at ASCO 2024

On June 1, 2024 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported long-term follow-up results, including the first-ever overall survival (OS) findings, from the Phase 2b HERIZON-BTC-01 clinical trial of zanidatamab in previously treated, unresectable, locally advanced, or metastatic HER2-positive biliary tract cancer (BTC) (Press release, Jazz Pharmaceuticals, JUN 1, 2024, View Source [SID1234643956]). These data will be featured at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in a poster presentation during the Gastrointestinal Cancer – Gastroesophageal, Pancreatic, and Hepatobiliary session on June 1, 2024, at 1:30-4:30 p.m. CDT. Zanidatamab is a human epidermal growth factor receptor 2 (HER2)-targeted bispecific antibody being studied in multiple solid tumors.

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For the trial’s primary endpoint, results demonstrated that a confirmed objective response rate (cORR) by independent central review (ICR) was maintained at 41.3% (95% confidence interval (CI): 30.4, 52.8) and one additional patient achieved a complete response (n=2; 2.5%) since initial findings were presented at the ASCO (Free ASCO Whitepaper) Annual Meeting in 2023. The median duration of response (DoR), one of the trial’s key secondary endpoints, increased by approximately 2 months to 14.9 months (95% CI: 7.4, not reached), compared to the previously reported findings. In this data cut, zanidatamab demonstrated a median estimated OS, another secondary endpoint, of 15.5 months (95% CI: 10.4, 18.5) in all patients with HER2+ BTC, 18.1 months (95% CI:12.2, 23.2) in patients with IHC 3+ tumors, and 5.2 months (95% CI: 3.1, 10.2) in patients with IHC 2+ tumors. Results highlight the clinically meaningful benefits of sustained and durable responses with continued treatment with zanidatamab.

"Patients with BTC are typically diagnosed when their disease is at an advanced stage, which is associated with a poor prognosis," said Shubham Pant, M.D., M.B.B.S. professor in the Department of Gastrointestinal Medical Oncology and Investigational Cancer Therapeutics at The University of Texas MD Anderson Cancer Center. "Historically, overall survival with standard-of-care chemotherapy in second-line patients with advanced BTC is reported to be between 6-9 months1 so there is a significant unmet need for targeted therapies that can potentially improve survival. The deep and durable responses seen in this study signal the potential to fill a critical unmet patient need with zanidatamab, a chemotherapy-free option, among patients with HER2-expressing cancers."

"We are encouraged by the updated results from the pivotal HERIZON-BTC-01 trial demonstrating sustained clinical activity in previously treated patients with advanced HER2-positive BTC. Results from HERIZON-BTC-01 were included in the Biologics License Application (BLA) for zanidatamab, which was granted Priority Review by the FDA earlier this week, as well as in the Marketing Authorization Application for zanidatamab which was recently submitted to the European Medicines Agency," said Rob Iannone, M.D., M.S.C.E., executive vice president, global head of research and development of Jazz Pharmaceuticals. "Clinical development of zanidatamab for the treatment of advanced HER2-positive BTC continues with HERIZON-BTC-302 (NCT06282575), the ongoing, global, randomized Phase 3 trial of zanidatamab in combination with standard-of-care therapy in the first-line setting for patients with HER2-positive BTC. We also look forward to investigating zanidatamab’s potential in other HER2-expressing solid tumors, including in cases resistant to prior HER2-targeted therapies."

Trial Results

Results from this long-term analysis of the Phase 2b HERIZON-BTC-01 trial (NCT04466891) indicate that zanidatamab monotherapy demonstrated sustained and durable antitumor responses in previously treated patients with HER2-positive unresectable, locally advanced, or metastatic BTC and support the clinically meaningful benefit of continued treatment with zanidatamab. The safety profile in all enrolled patients remained manageable with favorable tolerability compared with the initial analysis. Two (2.3%) patients discontinued treatment due to treatment-related adverse events (TRAEs).

The trial evaluated zanidatamab (20 mg/kg IV once every 2 weeks) in patients with HER2-positive, locally advanced unresectable, or metastatic BTC who had received prior gemcitabine-containing therapy. Patients with prior HER2-targeted therapy use were excluded from the trial. All patients were required to have centrally confirmed HER2-amplified tumors (assessed by in situ hybridization). Patients (n=87) were assigned into two cohorts based on tumor IHC status: Cohort 1 (n=80) included patients who were IHC 2+/3+ (HER2-positive) and Cohort 2 (n=7) included patients who were IHC 0/1+. The median duration of follow-up was 21.9 (16-34) months. Tumors were assessed every 8 weeks per RECIST v1.1. Updated efficacy analyses include only Cohort 1, while safety analyses include Cohorts 1 and 2.

As of July 28, 2023, data from HER2-positive BTC patients enrolled in Cohort 1 (n=80) demonstrated:

With longer follow-up, the cORR was maintained from the initial analysis (n=33; 41.3%) (95% CI: 30.4, 52.8), with one additional complete response (n=2; 2.5%).
Although the trial was not designed to detect treatment effects by HER2 status, as previously reported, in a pre-planned subgroup analysis of cORR by HER2 expression, responses were observed in patients with IHC 3+ tumors (cORR: 51.6% [95% CI: 38.6%-64.5%]) and IHC 2+ tumors (cORR: 5.6% [95% CI: 0.1-27.3%])2.
A two-month increase in the median DoR to 14.9 months (95% CI: 7.4, not reached).
In patients with IHC3+ tumors, the median DoR was 14.9 months (95% CI: 7.4, not reached).
The DOR in the 1 responder with IHC 2+ tumors was 7.5 months.
A median OS (95% CI) of 15.5 months (95% CI: 10.4, 18.5).
The median OS in patients with IHC 3+ was 18.1 months (95% CI: 12.2, 23.2).
The median OS in patients with IHC 2+ was 5.2 months (95% CI: 3.1, 10.2).
Median progression-free survival (PFS) was maintained (5.5 months [95% CI: 3.6, 7.3]) compared with the initial analysis, which had a data cutoff of October 10, 2022.
In patients with IHC 3+ tumors, the median PFS was 7.2 months (95% CI: 5.4, 9.4) months.
In patients with IHC 2+ tumors, the median PFS was 1.7 months (95% CI: 1.0, 3.3) months.
As previously reported for Cohorts 1 and 2, zanidatamab demonstrated a manageable and tolerable safety profile, with no new safety signals identified and no deaths that were treatment related. TRAEs leading to dose reductions remained infrequent. Serious TRAEs occurred in eight (9.2%) patients. One patient experienced serious TRAEs since the initial analysis (alanine aminotransferase increased and aspartate aminotransferase increased).Treatment discontinuation rate was 2.3% and no additional patients discontinued treatment due to TRAEs since the initial analysis.

About BTC
BTC, including gallbladder cancer and intrahepatic and extrahepatic cholangiocarcinoma, account for <1% of all adult cancers globally and are often associated with a poor prognosis.3,4 The human epidermal growth factor receptor 2 (HER2) is a well-validated target for antitumor therapy in other cancers. Across the U.S., Europe, and Japan, approximately 12,000 people are diagnosed with HER2+ BTC annually.5,6,7,8

About Zanidatamab
Zanidatamab is an investigational HER2-targeted bispecific antibody that can simultaneously bind two non-overlapping epitopes of the HER2 receptor, known as biparatopic binding. This unique design and increased binding results in multiple mechanisms of action, including dual HER2 signal blockade, removal of HER2 protein from the cell surface, and immune-mediated cytotoxicity leading to encouraging antitumor activity in patients. Zanidatamab is being developed in multiple clinical trials as a targeted treatment option for patients with solid tumors that express HER2. Zanidatamab is being developed by Jazz and BeiGene, Ltd. (BeiGene) under license agreements from Zymeworks, which first developed the molecule.

The U.S. Food and Drug Administration (FDA) has accepted and granted Priority Review of the Biologics License Application (BLA) for zanidatamab with a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2024. Zanidatamab was also granted Breakthrough Therapy designation in patients with previously treated HER2 gene-amplified biliary tract cancers (BTC) and given two Fast Track designations: one as a single agent for refractory BTC and one in combination with standard of care chemotherapy for 1L gastroesophageal adenocarcinoma (GEA). Additionally, zanidatamab has received Orphan Drug designations from FDA for the treatment of BTC and GEA, as well as Orphan Drug designation from the European Medicines Agency for the treatment of BTC and gastric cancer. Zanidatamab was also granted Breakthrough Therapy designation from the Center for Drug Evaluation (CDE) in China.

Updated Data from the Phase 1/2 Study of Olomorasib in KRAS G12C-Mutant Advanced Solid Tumors Presented at the 2024 ASCO® Annual Meeting

On June 1, 2024 Eli Lilly and Company (NYSE: LLY) reported updated data from the Phase 1/2 clinical trial evaluating olomorasib as a monotherapy in patients with KRAS G12C-mutant advanced solid tumors and in combination with Merck’s anti-PD-1 therapy KEYTRUDA (pembrolizumab) in patients with KRAS G12C-mutant advanced non-small cell lung cancer (NSCLC) (Press release, Eli Lilly, JUN 1, 2024, View Source [SID1234643924]). Olomorasib is an investigational, oral, potent, and highly selective second-generation inhibitor of the KRAS G12C protein. These data will be shared in oral presentations at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting.

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"Despite recent advances, there remains a significant unmet need for patients with KRAS G12C-mutant cancers," said Timothy Burns, M.D., Ph.D., Associate Professor of Medicine, University of Pittsburgh Medical Center Hillman Cancer Center. "These data show efficacy with olomorasib across tumor types and, importantly, tolerability that suggests it can be combined with immunotherapy, the backbone of first-line treatment for KRAS-mutant NSCLC. In NSCLC, it is also exciting to see promising activity in patients previously treated with a KRAS G12C inhibitor as well as central nervous system (CNS) activity, consistent with the improved potency of this second generation KRAS G12C inhibitor. Collectively, these data point to a promising emerging profile for olomorasib, particularly in NSCLC where new options are needed to improve outcomes for patients."

"As a second generation KRAS G12C inhibitor, olomorasib was specifically designed to offer a differentiated profile that could potentially overcome limitations of currently available treatment options," said David Hyman, M.D., chief medical officer, Lilly. "With these updated data, we are pleased to see our thesis for olomorasib continuing to translate clinically. Through our SUNRAY-01 study, we look forward to further investigating the potential of olomorasib in combination with pembrolizumab-containing regimens in first-line NSCLC, where there remains great need to further impact the disease trajectory for patients with KRAS G12C-mutant lung cancers."

Data from the LOXO-RAS-20001 Phase 1/2 Study
Results presented at ASCO (Free ASCO Whitepaper) utilized a cutoff date of March 18, 2024. Efficacy results are based on investigator response assessments, and objective response rates (ORR) include responses that are confirmed, as well as those pending confirmation and ongoing.

Olomorasib as Monotherapy in KRAS G12C-Mutant Advanced Solid Tumors

An oral presentation (abstract #3007) detailed updated data for olomorasib monotherapy in patients with KRAS G12C-mutant advanced solid tumors. This dataset consisted of 184 patients, including 42 with NSCLC naïve to a KRAS G12C inhibitor (six with active brain metastases), 41 with NSCLC who had received a prior KRAS G12C inhibitor, 32 with colorectal cancer (CRC), 24 with pancreatic cancer, and 45 with other solid tumors. Patients had received a median of three prior lines of therapy (range 0-11).

Efficacy for olomorasib monotherapy was consistent across a range of solid tumors with an ORR of 35% (37/105) in patients with non-CRC solid tumors. Median progression free survival (PFS) in all patients with KRAS G12C inhibitor-naive non-CRC solid tumors was 7.1 months (95% CI: 5.5-8.9) and 7.9 months (95% CI: 4.1-NE) in patients with KRAS G12C inhibitor-naive NSCLC. In patients with NSCLC previously treated with a KRAS G12C inhibitor, the ORR was 41% (16/39), with 63% having received a KRAS G12C inhibitor as their immediate prior therapy, and median PFS was 8.1 months (95% CI: 5.6-15.6). Preliminary CNS activity was seen, with CNS responses observed in patients with NSCLC and measurable brain metastases.

Patients were on treatment for a median of 4.7 months and treatment-related adverse events (TRAEs) were predominantly grade 1 with only diarrhea seen in greater than 15% of patients. The most common TRAEs of any grade were diarrhea (23%), nausea (11%), and fatigue (10%). The safety profile for patients who discontinued a prior KRAS G12C inhibitor due to toxicity was similar to all patients treated with olomorasib monotherapy including patients who discontinued their previous first KRAS G12C inhibitor due to toxicity. TRAEs led to discontinuation of olomorasib in 1% of patients.

Olomorasib in Combination with Pembrolizumab in KRAS G12C-Mutant Advanced NSCLC

An oral presentation (abstract #8510) detailed updated data for olomorasib in combination with pembrolizumab in patients with KRAS G12C-mutant advanced NSCLC, studying the two doses (50mg and 100mg BID) under ongoing investigation in first-line NSCLC. This dataset consisted of 64 patients, including patients with first-line metastatic disease and those previously treated (prior KRAS G12C inhibitor, chemotherapy, and/or immunotherapy). Patients received a median of two prior lines of therapy (range: 0-8).

In patients with first-line metastatic NSCLC, across a range of PD-L1 levels, the ORR for olomorasib in combination with pembrolizumab was 77% (13/17) and median PFS was not reached with follow-up ongoing. The most common TRAEs of any grade were diarrhea (23%), increased ALT (20%), pruritus (19%), increased AST (16%), and fatigue (16%). TRAEs led to discontinuation of olomorasib only in 3% of patients (2/64), pembrolizumab only in 11% of patients (7/64) and both drugs in 5% of patients (3/64).

The SUNRAY-01 trial (NCT06119581), a global, registrational study investigating olomorasib in combination with pembrolizumab or pembrolizumab plus chemotherapy in first-line NSCLC, is currently enrolling.

KEYTRUDA is a registered trademark of Merck Sharp & Dohme LLC, a subsidiary of Merck & Co., Inc., Rahway, NJ, USA.

About the LOXO-RAS-20001 Study
LOXO-RAS-20001 is an open-label, multicenter, Phase 1/2 study evaluating the safety, tolerability and preliminary efficacy of olomorasib in patients with KRAS G12C-mutant advanced solid tumors (NCT04956640). The study includes a Phase 1a dose escalation phase of olomorasib monotherapy in KRAS G12C-mutant solid tumors and a Phase 1b dose expansion and optimization phase which are evaluating olomorasib as a monotherapy and in combination with other treatments.

About Olomorasib
Olomorasib (LY3537982) is an investigational, oral, potent, and highly selective second-generation inhibitor of the KRAS G12C protein. KRAS is the most common oncogene across all tumor types, and KRAS G12C mutations occur in 13% of patients with non-small cell lung cancer (NSCLC) and 1-3% of patients with other solid tumors.1,2 Olomorasib was specifically designed to target KRAS G12C mutations and has pharmacokinetic properties which allow for high predicted target occupancy and high potency when used as monotherapy or in combination.3

Olomorasib is currently being studied in the LOXO-RAS-20001 Phase 1/2 trial (NCT04956640) in patients with KRAS G12C-mutant NSCLC and other advanced solid tumors and in the pivotal, registrational SUNRAY-01 global study (NCT06119581) investigating olomorasib in combination with pembrolizumab with or without chemotherapy for first-line treatment of KRAS G12C-mutant advanced NSCLC. For additional information about olomorasib clinical trials, please refer to clinicaltrials.gov.

First results from late-breaking Phase 3 PALOMA-3 study show five-fold reduction in infusion-related reactions with five-minute subcutaneous amivantamab administration

On May 31, 2024 Johnson & Johnson reported first data from the Phase 3 PALOMA-3 study evaluating subcutaneous (SC) amivantamab combined with lazertinib in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletion (ex19del) or L858R mutations (Press release, Johnson & Johnson, MAY 31, 2024, View Source [SID1234643910]). Study results showed non-inferior efficacy and pharmacokinetics for SC amivantamab combined with lazertinib compared to intravenous (IV) administration, the currently approved formulation of RYBREVANT (amivantamab-vmjw). Administration time for SC amivantamab was reduced to approximately five minutes from five hours (across two days) and showed a five-fold reduction in infusion-related reactions (IRRs). These late-breaking results, which are the Company’s fourth positive Phase 3 readout for the RYBREVANT clinical program, were featured as an oral presentation at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Abstract #LBA8505).1 Data were also selected for the Best of ASCO (Free ASCO Whitepaper) 2024 Meetings, highlighting the cutting-edge science and leading research in oncology from Johnson & Johnson.

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"The PALOMA-3 data show that subcutaneous amivantamab offers shorter infusion times and lower rates of infusion-related reactions and venous thromboembolism with pharmacokinetics and efficacy comparable to the current IV administration," said Dr. Natasha B. Leighl*, medical oncologist at the Princess Margaret Cancer Centre in Toronto, Canada, and the presenting author. "I look forward to seeing how these findings can make a meaningful difference in clinical practice by potentially improving the treatment experience for patients with EGFR-mutated non-small cell lung cancer."

Results showed SC amivantamab was non-inferior to IV amivantamab, meeting both co-primary pharmacokinetic (PK) efficacy endpoints as measured by amivantamab levels in the blood (Ctrough and area under the serum concentration time curve from day 1 to 15).1

At a median follow-up of seven months, the overall response rate was 30 percent (95 percent confidence interval [CI], 24–37) in the subcutaneous arm and 33 percent (95 percent CI, 26–39) for IV (relative risk, 0.92; 95 percent CI, 0.70-1.23; P=0.001), meeting the noninferiority criteria. SC amivantamab also demonstrated longer duration of response (DoR), progression-free survival (PFS) and significant improvement in overall survival (OS) compared to IV administration during this time. Specifically, median DoR was numerically longer for SC amivantamab combined with lazertinib compared to IV (median, 11.2 vs 8.3 months among confirmed responders) as was PFS (median, 6.1 vs 4.3 months; hazard ratio [HR], 0.84; 95 percent CI, 0.64–1.10; P=0.20). A pre-specified exploratory endpoint showed patients treated with SC amivantamab had significantly longer OS compared with IV (HR, 0.62; 95 percent CI, 0.42–0.92; nominal P=0.02). At 12 months, 65 percent of patients who received SC amivantamab combined with lazertinib were alive compared with 51 percent of those treated with the IV regimen. It is theorized that the efficacy seen with SC amivantamab may be linked to SC absorption via the lymphatic system, potentially enhancing immune-mediated activity.1

Of particular note, administration time was substantially shorter for SC amivantamab (median less than approximately five minutes) compared to IV administration (up to five hours), with significantly more patients reporting convenience with the SC administration (85 percent with SC amivantamab vs 35 percent with IV administration at end of treatment; P<0.001).1

The overall safety profile of SC amivantamab is consistent with the known profile of IV administration. The most common all-grade adverse events (≥ 20 percent) for SC amivantamab compared to IV were paronychia (54 percent vs 51 percent), hypoalbuminemia (47 percent vs 37 percent) and rash (46 percent vs 43 percent), respectively. No Grade 4 or 5 IRRs were reported. The rate of IRRs for patients treated with SC amivantamab combined with lazertinib was shown to be approximately five-fold lower than that observed with the IV formulation (13 percent vs 66 percent, respectively). Prophylactic anticoagulation was used in most patients in the study and was found to be safe and effective in reducing the rate of venous thromboembolic events (VTE). Patients receiving prophylactic anticoagulation had lower rates of VTE (10 percent) than those without prophylaxis (21 percent). Furthermore, VTE incidence was lower in the SC arm compared to the IV arm (9 percent vs 14 percent, respectively) regardless of anticoagulation use. Severe bleeding risk was low and similar among patients receiving anticoagulants in the SC (2 percent) and IV (1 percent) arms.1

"We are always exploring innovative approaches to meet the urgent needs of patients living with EGFR-mutated non-small cell lung cancer and these compelling findings reinforce the potential for a new route of administration for amivantamab," said Yusri Elsayed, M.D., M.H.Sc., Ph.D., Global Therapeutic Area Head, Oncology, Johnson & Johnson Innovative Medicine. "We look forward to pursuing regulatory submissions for this formulation, as we advance our ambition to transform the first-line treatment of EGFR-mutated NSCLC."

Today, Janssen-Cilag International NV, a Johnson & Johnson company, announced the submission of an application for the extension of the RYBREVANT marketing authorization (line extension) to the European Medicines Agency (EMA) seeking approval of SC amivantamab in combination with lazertinib for the first-line treatment of adult patients with locally advanced or metastatic NSCLC with EGFR ex19del or L858R mutations and as monotherapy in adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after failure of platinum-based therapy based on the PALOMA-3 data. Johnson & Johnson will submit regulatory applications seeking the approval of SC amivantamab in other markets, including the United States.

About PALOMA-3

PALOMA-3 (NCT05388669), which enrolled 418 patients, is a randomized, open-label Phase 3 study evaluating the pharmacokinetics (PK), efficacy and safety of subcutaneous amivantamab (administered via manual injection) combined with lazertinib compared to IV amivantamab and lazertinib in patients with EGFR-mutated advanced or metastatic NSCLC after progression on osimertinib and chemotherapy. The co-primary PK endpoints of the study were trough concentration (Ctrough on Cycle [C] 2 Day [D] 1 or C4D1) and C2 area under the curve (AUCD1-D15). Key secondary endpoints were objective response rate and progression-free survival. Overall survival was a predefined exploratory endpoint. Prophylactic anticoagulation was recommended for the first four months of treatment.2

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.3 In the subcutaneous formulation, amivantamab is co-formulated with recombinant human hyaluronidase PH20 (rHuPH20), Halozyme’s ENHANZE drug delivery technology.

RYBREVANT is also approved in the U.S. 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. In October 2023, a type II extension of indication application was submitted to the European Medicines Agency (EMA) seeking approval of RYBREVANT for this indication.

In December 2023, Johnson & Johnson submitted a supplemental Biologics License Application (sBLA) together with a New Drug Application (NDA) to the U.S. FDA for RYBREVANT in combination with 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. This submission is based on the Phase 3 MARIPOSA study and was granted Priority Review in February 2024. A marketing authorization application (MAA) and type II extension of indication application were also submitted to the EMA seeking approval of lazertinib in combination with RYBREVANT based on the MARIPOSA study.

In November 2023, Johnson & Johnson submitted an 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. A type II extension of indication application was also submitted to the EMA seeking approval of RYBREVANT 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 carboplatin and pemetrexed as a preferred (Category 1 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.4 †‡
Amivantamab-vmjw (RYBREVANT) plus chemotherapy as a preferred (Category 1 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.4 †‡
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.4 †‡
RYBREVANT is being studied in multiple clinical trials in NSCLC, including:

The Phase 3 PALOMA-3 (NCT05388669) study assessing lazertinib with subcutaneous amivantamab compared to intravenous amivantamab in patients with EGFR-mutated advanced or metastatic NSCLC.2
The Phase 2 PALOMA-2 (NCT05498428) study assessing subcutaneous amivantamab in patients with advanced or metastatic solid tumors including EGFR-mutated NSCLC.5
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.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 MARIPOSA-2 (NCT04988295) study assessing the efficacy of RYBREVANT (with or without lazertinib) 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.8
The Phase 3 MARIPOSA (NCT04487080) study assessing RYBREVANT in combination with lazertinib versus osimertinib and versus lazertinib alone in the first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR ex19del or L858R substitution mutations.9
The Phase 1 CHRYSALIS (NCT02609776) study evaluating RYBREVANT in patients with advanced NSCLC.10
The Phase 1/1b CHRYSALIS-2 (NCT04077463) study evaluating RYBREVANT in combination with lazertinib and lazertinib as a monotherapy in patients with advanced NSCLC with EGFR mutations.11
The Phase 1/2 METalmark (NCT05488314) study assessing RYBREVANT and capmatinib combination therapy in locally advanced or metastatic NSCLC.12
The Phase 1/2 PolyDamas (NCT05908734) study assessing RYBREVANT and cetrelimab combination therapy in locally advanced or metastatic NSCLC.13
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 lazertinib in relapsed or refractory EGFR-mutated advanced or metastatic NSCLC.14
For more information, visit: View Source

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.15, 16 The main subtypes of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma.17 Among the most common driver mutations in NSCLC are alterations in EGFR, which is a receptor tyrosine kinase controlling cell growth and division.18 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.17,18,19,20,21,22 EGFR ex19del or EGFR L858R mutations are the most common EGFR mutations.23 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.24,25 EGFR exon 20 insertion mutations are the third most prevalent activating EGFR mutation.26 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.27

RYBREVANT IMPORTANT SAFETY INFORMATION3

WARNINGS AND PRECAUTIONS

The safety population of RYBREVANT with carboplatin and pemetrexed described in Warnings and Precautions was based on 151 patients in the PAPILLON study.

The safety population of RYBREVANT as a single agent described in Warnings and Precautions was based on 129 patients in the CHRYSALIS study.

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.

RYBREVANT with Carboplatin and Pemetrexed

RYBREVANT in combination with carboplatin and pemetrexed can cause infusion-related reactions. Based on the safety population, 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

Based on the safety population, 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. Monitor patients for any 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 interstitial lung disease (ILD)/pneumonitis.

RYBREVANT with Carboplatin and Pemetrexed

Based on the safety population, 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

Based on the safety population, 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). Immediately withhold RYBREVANT in patients with suspected ILD/pneumonitis and permanently discontinue if ILD/pneumonitis is confirmed.

Dermatologic Adverse Reactions

RYBREVANT can cause rash (including dermatitis acneiform), pruritus, and dry skin.

RYBREVANT with Carboplatin and Pemetrexed

RYBREVANT in combination with carboplatin and pemetrexed can cause dermatologic adverse reactions. Based on the safety population, 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; 2% permanently discontinued RYBREVANT, and 1.3% discontinued pemetrexed.

RYBREVANT as a Single Agent

Based on the safety population, rash occurred in 74% of patients treated with RYBREVANT, 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. Advise patients to wear protective clothing and use broad-spectrum UVA/UVB sunscreen. Alcohol-free emollient cream is recommended for dry skin.

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. Withhold, dose reduce, or permanently discontinue RYBREVANT based on severity.

Ocular Toxicity

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

RYBREVANT with Carboplatin and Pemetrexed

Based on the safety population, RYBREVANT in combination with carboplatin and pemetrexed can cause ocular toxicity including blepharitis, dry eye, conjunctival redness, blurred vision, and eye pruritus. All events were Grade 1-2.

RYBREVANT as a Single Agent

Based on the safety population, 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 presenting with 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 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.

Adverse Reactions

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.

Please read the full Prescribing Information for RYBREVANT.

Rakovina Therapeutics Announces 2024 Q1 Financial Results and Provides Corporate Update

On May 30, 2024 Rakovina Therapeutics Inc. (TSX.V: RKV), (the "Company") a biopharmaceutical company committed to advancing new cancer therapies based on novel DNA-damage response technologies reported the financial results for the first quarter of 2024 ending March 31, and provides an update to corporate activities (Press release, Rakovina Therapeutics, MAY 31, 2024, View Source;utm_medium=rss&utm_campaign=rakovina-therapeutics-announces-2024-q1-financial-results-and-provides-corporate-update [SID1234643876]).

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"This year has started with significant changes for Rakovina Therapeutics in the most positive sense," said Executive Chairman Jeffrey Bacha. "Our strategic shift to include AI in our drug developments, additions to the scientific team and expansion of our research collaborations all position Rakovina Therapeutics to successfully achieve our goal of improving the lives of patients with cancer."

Q1 2024 Financial Highlights

For the three months ended March 31, 2024 the Company reported a net loss of $752,383, inline with Company estimates.
Research and development operating expenses were $438,983 and general and administrative expenses were $251,090.
Total cash operating expenses related to research and development and general and administrative expenses for the three months ended March 31, 2024 were $522,705.
Recent Corporate Highlights

On March 27, 2024, we announced a collaboration agreement with Dr. Artem Cherkasov granting Rakovina Therapeutics exclusive access to the proprietary Deep Docking (trademarked) artificial intelligence ("AI") Platform for DNA-damage response targets. Using the Deep Docking platform Rakovina Therapeutics is analyzing billions of molecular structures to evaluate their potential as targeted cancer drugs. The Company plans to validate the activity of the most promising candidates using its established research and development infrastructure at the University of British Columbia. Rakovina Therapeutics intends to advance validated drug candidates to human clinical trials in collaboration with pharmaceutical partners.
On May 8, 2024, we announced the expansion of contracts with the University of British Columbia ("UBC") and Pharma Inventor Inc. to support the Company’s Deep Docking AI Drug Discovery Platform. The wet lab infrastructure at UBC combined with the medicinal chemistry expertise will allow the Company to quickly synthesize and evaluate lead drug candidates identified by the AI platform.
On May 23, 2024, the Company announced the undertaking of a non-brokered Private Placement (the "Offering") for gross proceeds of up to $1.5 million at a purchase price of $0.10 per Unit which includes lead orders totalling approximately $1.25 million from two new investors. Each Unit will be comprised of one (1) common share of Rakovina Therapeutics common share stock and one (1) share purchase warrant at a purchase price of $0.20 for a period of three years (36 months) after the closing date of the Offering. If the closing price for the Common Shares on the TSX Venture Exchange (the "TSXV") is $0.25 or greater for five consecutive trading days, the expiry date of the Warrant shall be accelerated to the date that is 30 days following the last day of the five-trading day period.
The Company plans to use the proceeds primarily to support research and development activities related to its recently announced Artificial Intelligence (AI) collaboration that provides exclusive access to the proprietary Deep Docking platform for DNA-damage response targets.

Summary Financial Results for the quarter ended March 31, 2024

Selected Financial Information As at
March 31, 2024
$
Cash & cash equivalents 177,188
Working capital (87,183)
Intangible assets 4,381,391
Total Assets 4,608,212
Total liabilities 1,667,051
Deficit (11,667,694)
Total equity 2,941,161
Statements of net loss and
comprehensive loss data: For the three
months ended
March 31, 2024
$
Research & Development 438,983 433,344
General and Administrative 251,090 212,043
Net loss and comprehensive loss (752,383) (641,790)
Basic and diluted income (loss) per share (0.01) (0.01)
Operating cash burn 522,705 472,628
Weighted average shares outstanding 70,084,925 69,829,500

Further to the Company’s press release on April 1, 2024, the Company wishes to clarify that the capital market advisory services agreement entered into with Insight Capital Partners Inc. ("ICP") also includes a separate market making services agreement with ICP Securities Inc. ("ICP Securities"). ICP Securities provides market making services to the Company, and ICP Securities receives a fee of $7,500 plus applicable taxes per month. The market making services agreement between the company and ICP Securities is for a term of three months commencing on March 25, 2024, and may continue on a month-to-month basis. ICP is acting as a collection agent on behalf of ICP Securities.

Rakovina Therapeutics’ financial statements as filed with SEDAR can be accessed from the Company’s website at: View Source

RYBREVANT® (amivantamab-vmjw) plus lazertinib is the only chemotherapy-free regimen showing longer progression-free survival versus osimertinib in first-line treatment of patients with high-risk EGFR-mutated non-small cell lung cancer

On May 31, 2024 Johnson & Johnson reported new data from the Phase 3 MARIPOSA study demonstrating the benefit of first-line treatment with RYBREVANT (amivantamab-vmjw) in combination with lazertinib in patients with high-risk disease or clinical features, which occur in nearly 85 percent of patients with non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) mutations (Press release, Johnson & Johnson, MAY 31, 2024, View Source [SID1234643911]). Results from the new analysis show the RYBREVANT combination consistently and significantly improved progression-free survival (PFS) compared to osimertinib in patients with NSCLC with EGFR exon 19 deletion (ex19del) or L858R mutations. These data were presented at the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (Abstract #8504).1

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"These new data demonstrate the efficacy of RYBREVANT plus lazertinib, showing a significant reduction in the risk of disease progression or death compared to osimertinib in several high-risk subgroups of patients with EGFR-mutated NSCLC," said Byoung Chul Cho, M.D., Ph.D., medical oncologist and professor in the Division of Medical Oncology at Yonsei Cancer Center, Yonsei University College of Medicine in Seoul, Republic of Korea, and study author.* "These findings support the potential of this combination as an important first-line option for these patients who face significant unmet needs."

The MARIPOSA study enrolled treatment-naïve patients with EGFR-mutant (ex19del or L858R) advanced NSCLC. Overall, results showed RYBREVANT plus lazertinib resulted in a significant reduction in the risk of disease progression or death compared to osimertinib as previously reported. High-risk features, such as liver or brain metastases, baseline TP53 co-mutations, and circulating tumor DNA (ctDNA) shedding are common in patients with EGFR-mutated advanced NSCLC and associated with poor prognoses. In the study, 89 percent of enrolled patients had one or more of these high-risk disease or clinical features at baseline. Specifically, 41 percent had brain metastases, 16 percent had liver metastases, 54 percent had TP53 co-mutations, 70 percent had ctDNA present at baseline and 15 percent continued to shed ctDNA after two cycles of treatment.1

Results from the analysis showed treatment with RYBREVANT plus lazertinib significantly reduced the risk of disease progression or death consistently across all high-risk subgroups1:

31 percent compared to osimertinib in patients with a history of brain metastases (18.3 vs 13.0 months; hazard ratio [HR], 0.69; [95 percent confidence interval [CI], 0.53-0.92]; P=0.010)
42 percent compared to osimertinib in patients with liver metastases at baseline (18.2 vs 11.0 months; HR, 0.58 [95 percent CI, 0.37-0.91]; P=0.017)
35 percent compared to osimertinib among patients with TP53 co-mutations (18.2 vs 12.9 months; HR, 0.65 [95 percent CI, 0.48-0.87]; P=0.003)
32 percent compared to osimertinib in patients with detectable ctDNA at baseline (20.3 vs 14.8 months; HR, 0.68 [95 percent CI, 0.53-0.86]; P=0.002)
51 percent compared to osimertinib in patients without cleared ctDNA at C3D1 (16.5 vs 9.1 months; HR, 0.49 [95 percent CI, 0.27-0.87]; P=0.015)
As reported at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) 2023 Congress, the safety profile of the combination of RYBREVANT and lazertinib was consistent with the safety profiles of the individual treatments, with mostly Grade 1 or 2 adverse events (AEs). Toxicity was largely manageable with dose interruptions and reductions, along with supportive care measures commonly used in the treatment of patients with NSCLC. The most common Grade 3 or higher treatment-related AEs were rash and paronychia. RYBREVANT plus lazertinib had higher rates of EGFR- and MET-related AEs (hypoalbuminemia and peripheral edema) and venous thromboembolism compared to osimertinib, with higher rates of diarrhea being observed with osimertinib. The rate of discontinuation of all study treatments due to treatment-related AEs for the RYBREVANT combination was 10 percent. The rate of interstitial lung disease (including pneumonitis) was less than three percent in both arms.2

"With the majority of patients with EGFR-mutated lung cancer having high-risk disease and clinical features, ensuring that all patients receive the most appropriate treatment in the first-line setting is critical. The results presented at ASCO (Free ASCO Whitepaper) suggest RYBREVANT plus lazertinib offer a new standard of care in this patient population," said Craig Tendler, M.D., Vice President, Late Development and Global Medical Affairs, Johnson & Johnson Innovative Medicine. "These new findings continue to demonstrate how RYBREVANT-based regimens are transforming treatment for patients with EGFR-mutated non-small cell lung cancer and add to the growing body of evidence that supports the promise of this chemotherapy-free approach."

About the MARIPOSA Study

MARIPOSA (NCT04487080), which enrolled 1,074 patients, is a randomized, Phase 3 study evaluating RYBREVANT in combination with lazertinib versus osimertinib and versus lazertinib alone in first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR ex19del or substitution mutations. The primary endpoint of the study is PFS (using RECIST v1.1 guidelines) as assessed by BICR. Secondary endpoints include overall survival (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 also approved in the U.S. 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. In October 2023, a type II extension of indication application was submitted to the European Medicines Agency (EMA) seeking approval of RYBREVANT for this indication.

In December 2023, Johnson & Johnson submitted a supplemental Biologics Lincense Application (sBLA) together with a New Drug Application (NDA) to the U.S. FDA for RYBREVANT in combination with 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. This submission is based on the Phase 3 MARIPOSA study and was granted Priority Review in February 2024. A marketing authorization application (MAA) and type II extension of indication application were also submitted to the EMA seeking approval of lazertinib in combination with RYBREVANT based on the MARIPOSA study.

In November 2023, Johnson & Johnson submitted an 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. A type II extension of indication application was also submitted to the EMA seeking approval of RYBREVANT 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 carboplatin and pemetrexed as a preferred (Category 1 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) plus chemotherapy as a preferred (Category 1 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) 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 †‡
RYBREVANT is being studied in multiple clinical trials in NSCLC, including:

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.6
The Phase 3 MARIPOSA-2 (NCT04988295) study assessing the efficacy of RYBREVANT (with or without lazertinib) 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.7
The Phase 3 MARIPOSA (NCT04487080) study assessing RYBREVANT in combination with lazertinib versus osimertinib and versus lazertinib alone in the first-line treatment of patients with locally advanced or metastatic NSCLC with EGFR ex19del or L858R substitution mutations.3
The Phase 1 CHRYSALIS (NCT02609776) study evaluating RYBREVANT in patients with advanced NSCLC.8
The Phase 1/1b CHRYSALIS-2 (NCT04077463) study evaluating RYBREVANT in combination with lazertinib and lazertinib as a monotherapy in patients with advanced NSCLC with EGFR mutations.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 2 PALOMA-2 (NCT05498428) study assessing subcutaneous amivantamab in patients with advanced or metastatic solid tumors including EGFR-mutated NSCLC.11
The Phase 3 PALOMA-3 (NCT05388669) study assessing lazertinib with subcutaneous amivantamab compared to intravenous amivantamab in patients with EGFR-mutated advanced or metastatic NSCLC.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 lazertinib in relapsed or refractory EGFR-mutated advanced or metastatic NSCLC.15
For more information, visit: View Source

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.16,17 The main subtypes of NSCLC are adenocarcinoma, squamous cell carcinoma, and large cell carcinoma.18 Among the most common driver mutations in NSCLC are alterations in EGFR, which is a receptor tyrosine kinase controlling cell growth and division.19 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.18,19,20,21,22,23 EGFR ex19del or EGFR L858R mutations are the most common EGFR mutations.24 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.25,26 EGFR exon 20 insertion mutations are the third most prevalent activating EGFR mutation.27 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.28

RYBREVANT IMPORTANT SAFETY INFORMATION4

WARNINGS AND PRECAUTIONS

The safety population of RYBREVANT with carboplatin and pemetrexed described in Warnings and Precautions was based on 151 patients in the PAPILLON study.

The safety population of RYBREVANT as a single agent described in Warnings and Precautions was based on 129 patients in the CHRYSALIS study.

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.

RYBREVANT with Carboplatin and Pemetrexed

RYBREVANT in combination with carboplatin and pemetrexed can cause infusion-related reactions. Based on the safety population, 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

Based on the safety population, 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. Monitor patients for any 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 interstitial lung disease (ILD)/pneumonitis.

RYBREVANT with Carboplatin and Pemetrexed

Based on the safety population, 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

Based on the safety population, 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). Immediately withhold RYBREVANT in patients with suspected ILD/pneumonitis and permanently discontinue if ILD/pneumonitis is confirmed.

Dermatologic Adverse Reactions

RYBREVANT can cause rash (including dermatitis acneiform), pruritus, and dry skin.

RYBREVANT with Carboplatin and Pemetrexed

RYBREVANT in combination with carboplatin and pemetrexed can cause dermatologic adverse reactions. Based on the safety population, 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; 2% permanently discontinued RYBREVANT, and 1.3% discontinued pemetrexed.

RYBREVANT as a Single Agent

Based on the safety population, rash occurred in 74% of patients treated with RYBREVANT, 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. Advise patients to wear protective clothing and use broad-spectrum UVA/UVB sunscreen. Alcohol-free emollient cream is recommended for dry skin.

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. Withhold, dose reduce, or permanently discontinue RYBREVANT based on severity.

Ocular Toxicity

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

RYBREVANT with Carboplatin and Pemetrexed

Based on the safety population, RYBREVANT in combination with carboplatin and pemetrexed can cause ocular toxicity including blepharitis, dry eye, conjunctival redness, blurred vision, and eye pruritus. All events were Grade 1-2.

RYBREVANT as a Single Agent

Based on the safety population, 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 presenting with 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 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.

Adverse Reactions

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.

Please read the full Prescribing Information for RYBREVANT.