Natera to Present New Signatera™ Colorectal Cancer Data at ESMO Showing 10X Advantage in Overall Survival

On September 8, 2024 Natera, Inc. (NASDAQ: NTRA), a global leader in cell-free DNA and genetic testing, reported it will present new data on its personalized and tumor-informed molecular residual disease (MRD) test, Signatera, at the 2024 European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress taking place Sept. 13-16 in Barcelona, Spain (Press release, Natera, SEP 8, 2024, View Source [SID1234646417]).

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Natera and its collaborators will present a total of nine abstracts, including five poster presentations from the GALAXY observational arm of the CIRCULATE-Japan trial, one of the largest and most comprehensive prospective studies of MRD testing in resectable colorectal cancer (CRC).

The GALAXY data includes an updated analysis of more than 2,100 patients with stage I-IV CRC, reporting 36-month disease-free survival (DFS), and, for the first time, overall survival (OS). Key highlights include:

Signatera-positive patients had significantly shorter OS compared to those who were Signatera-negative (hazard ratio of ~10), suggesting that Signatera-negative patients had an almost 10-fold advantage in OS. This compares favorably to all known guideline recommended biomarkers that typically have hazard ratios for overall survival in the 1-4 range.
Overall survival stratified by adjuvant chemotherapy (ACT) will also be presented. This will build on prior GALAXY DFS data which has shown significant benefit of ACT in Signatera-positive patients, but lack of benefit in Signatera-negative patients. The current standard of care is based on studies showing that most CRC patients derive a small ~0-5% absolute and ~10-20% relative OS benefit from ACT. Identifying subgroups where this benefit is concentrated is critical for advancing clinical management of early-stage CRC.
"ESMO 2024 will showcase some of our most impactful data in colorectal cancer to-date," said Minetta Liu, MD, chief medical officer of oncology at Natera. "Notably, we will present an updated analysis from the GALAXY arm of CIRCULATE-Japan with the first prospective read-out of overall survival based on MRD. These findings underscore the potential for Signatera to predict long-term outcomes."

Other presentations at ESMO (Free ESMO Whitepaper) will highlight new Signatera data in breast cancer and squamous cell carcinoma of the head and neck. Below is the full list of presentations:

Presentation Highlights

Poster Presentation #553P | CRC | Sept. 16 | Presenter: Jun Nagata, MD
Association of ctDNA-based MRD detection and MRD clearance with short-term overall survival in patients with resectable colorectal cancer: Updated analysis of CIRCULATE-Japan GALAXY

Poster Presentation #558P | CRC | Sept. 16 | Presenter: Kozo Kataoka, MD, PhD
Survival benefit of adjuvant chemotherapy based on molecular residual disease detection in resected colorectal liver metastases; subgroup analysis from CIRCULATE-Japan GALAXY

Oral Presentation and Additional Presentations

Oral Presentation | CRC | Presenter: Myriam Chalabi, MD
Sept. 15, 09:10 – 09:20
Neoadjuvant immunotherapy in locally advanced MMR-deficient (dMMR) colon cancer (CC): 3-year disease-free survival (DFS) from NICHE-2

Poster Presentation #923P | SCCHN | Sept. 14 | Presenter: Natasha Honoré, MD, PhD
Tumor-informed ctDNA assay to predict recurrence in locally advanced SCCHN

Poster Presentation #555P | CRC | Sept. 16 | Presenter: Tomoya Harima, MD
Association Between Copy Number Aberration and ctDNA MRD in Colorectal Cancer: CIRCULATE-Japan GALAXY

Poster Presentation #554P | CRC | Sept. 16 | Presenter: Yoshiaki Nakamura, MD, PhD
Novel Clinical Decision Support (CDS) System Optimizing Adjuvant Chemotherapy (ACT) for Colorectal Cancer (CRC) by Integrating Deep Learning and circulating tumor DNA (ctDNA) molecular residual disease (MRD): GALAXY Histotyping

Poster Presentation #545P | CRC | Sept. 16 | Presenter: Chiara M. Loeffler, MD
HIBRID: Histology and ct-DNA based Risk-stratification with Deep Learning

Poster Presentation #338TiP | Breast Cancer | Sept. 16 | Presenter: Michail Ignatiadis, MD, PhD
EORTC-2129-BCG: Elacestrant for treating ER+/HER2- breast cancer patients with ctDNA relapse (TREAT ctDNA)

Poster Presentation #314P | Breast Cancer | Sept. 16 | Presenter: Mark Magbanua, PhD
The impact of changes in tumor mutational landscape during neoadjuvant therapy on tumor-informed ctDNA testing in breast cancer patients

About Signatera

Signatera is a personalized, tumor-informed, molecular residual disease test for patients previously diagnosed with cancer. Custom-built for each individual, Signatera uses circulating tumor DNA to detect and quantify cancer left in the body, identify recurrence earlier than standard of care tools, and help optimize treatment decisions. The test is available for clinical and research use and is covered by Medicare for patients with colorectal cancer, breast cancer, ovarian cancer and muscle invasive bladder cancer, as well as for immunotherapy monitoring of any solid tumor. Signatera has been clinically validated across multiple cancer types and indications, with published evidence in more than 70 peer-reviewed papers.

Ivonescimab Monotherapy Reduced the Risk of Disease Progression or Death by 49% Compared to Pembrolizumab Monotherapy in First-Line Treatment of Patients with PD-L1 Positive Advanced NSCLC in China

On September 8, 2024 Summit Therapeutics Inc. (NASDAQ: SMMT) ("Summit," "we," or the "Company") reported data from the primary analysis of the Phase III HARMONi-2 trial featuring the novel, potential first-in-class investigational bispecific antibody, ivonescimab (Press release, Summit Therapeutics, SEP 8, 2024, View Source [SID1234646416]). The data was presented this morning as part of the Presidential Symposium at the International Association for the Study of Lung Cancer’s (IASLC) 2024 World Conference on Lung Cancer (WCLC 2024) in San Diego, California.

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The HARMONi-2 presentation, Phase 3 Study of Ivonescimab (AK112) vs. Pembrolizumab as First-line Treatment for PD-L1-positive Advanced NSCLC: Primary Analysis of HARMONi-2, evaluated monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose tumors have positive PD-L1 expression (PD-L1 TPS >1%). HARMONi-2 is a single region, multi-center, double-blinded Phase III study conducted in China sponsored by our collaboration partner, Akeso, Inc. (Akeso, HKEX Code: 9926.HK), with data generated and analyzed by Akeso.

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.

Clinically Meaningful Efficacy

In the HARMONi-2 primary analysis, ivonescimab monotherapy demonstrated a statistically significant improvement in the trial’s primary endpoint, progression-free survival (PFS) by Independent Radiologic Review Committee (IRRC), when compared to monotherapy pembrolizumab, achieving a hazard ratio (HR) of 0.51 (95% CI: 0.38, 0.69; p<0.0001). A clinically meaningful benefit was demonstrated across clinical subgroups, including those with PD-L1 low expression (PD-L1 TPS 1-49%), PD-L1 high expression (PD-L1 TPS ≥ 50%), squamous and non-squamous histologies, as well as other high-risk patients. Both the overall response rate (ORR) measured according to RECIST v1.1 criteria as well as the disease control rate (DCR) were higher in patients treated with ivonescimab compared to those treated with pembrolizumab.

HARMONi-2 ITT (n=398);

Median Follow-up: 8.67 mos

Ivonescimab

(n=198)

Pembrolizumab

(n=200)

Median PFS

11.14 mos

(95% CI: 7.33, NE)

5.82 mos

(95% CI: 5.03, 8.21)

PFS Stratified HR

0.51

(95% CI: 0.38, 0.69; p<0.0001)

ORR

50.0%

(95% CI: 42.8%, 57.2%)

38.5%

(95% CI: 31.7%, 45.6%)

DCR

89.9%

(95% CI: 84.8%, 93.7%)

70.5%

(95% CI: 63.7%, 76.7%)

HARMONi-2 Subgroup Analyses

Ivonescimab vs. Pembrolizumab

PD-L1 High (PD-L1 TPS ≥50%) PFS stratified HR

Ivonescimab n=83; Pembrolizumab n=85

0.46

(95% CI: 0.28, 0.75)

PD-L1 Low (PD-L1 TPS 1-49%) PFS stratified HR

Ivonescimab n=115; Pembrolizumab n=115

0.54

(95% CI: 0.37, 0.79)

Squamous histology PFS stratified HR

Ivonescimab n=90; Pembrolizumab n=91

0.48

(95% CI: 0.31, 0.74)

Non-Squamous histology PFS stratified HR

Ivonescimab n=108; Pembrolizumab n=109

0.54

(95% CI: 0.36, 0.82)

Overall survival data was not yet mature at the time of the data cutoff and will be evaluated in the future.

Manageable Safety Profile

Ivonescimab demonstrated an acceptable and manageable safety profile, which was consistent with previous studies. There were three patients (1.5%) who discontinued ivonescimab due to TRAEs compared to six patients (3.0%) who discontinued pembrolizumab due to TRAEs. There was one patient in the ivonescimab arm and two patients in the pembrolizumab arm who died as a result of TRAEs in this Phase III study. The most frequent treatment-related adverse events (TRAEs) for ivonescimab treatment were proteinuria (Grade 3+: ivonescimab, 3.0%; pembrolizumab 0.0%), hypertension (Grade 3+: ivonescimab, 5.1%; pembrolizumab 0.5%), and various laboratory abnormalities, including AST increases, hypercholesterolemia, anemia, and bilirubin increases. Grade 3 or higher immune-related adverse events occurred in 7.1% of patients receiving ivonescimab and 8.0% of patients receiving pembrolizumab. Grade 3 or higher adverse events that were possibly VEGF-related in the ivonescimab monotherapy arm were 10.2% vs. 1.0% for pembrolizumab, all of which were classified as Grade 3. Of note, Grade 3 hemorrhage events were observed in two patients in the ivonescimab arm (both were of non-squamous histology) compared to one patient in the pembrolizumab arm in this study.

HARMONi-2

(n=396)

Ivonescimab

(n=197)

Pembrolizumab

(n=199)

TRAEs Grade 3+

29.4%

15.6%

Serious TRAEs (TRSAEs)

20.8%

16.1%

TRAEs Leading to Drug Discontinuation

1.5%

3.0%

TRAEs Leading to Death

0.5%

1.0%

Grade 3+ Immune-related

7.1%

8.0%

Grade 3+ Possibly VEGF-related*

10.2%

1.0%

*All Grade 3+ adverse events that were possibly VEGF-related were classified as Grade 3 events in both arms; there were no Grade 4 or 5 adverse events that were possibly VEGF-related observed in either arm.

"This is a historic moment for ivonescimab, Team Summit, our partners at Akeso, and most importantly, we believe this is the beginning of a landscape shift for treatment options for patients living with cancer," stated Robert W. Duggan, Chairman and Chief Executive Officer of Summit. "We are incredibly proud of our partnership with Akeso and their accomplishment with HARMONi-2."

Based on the results of HARMONi-2, Summit announced its intention 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 patients with metastatic NSCLC whose tumors have high PD-L1 expression (PD-L1 TPS > 50%).

"HARMONi-2 clearly demonstrates that ivonescimab has the potential to be the next generation in PD-1 directed immunotherapy, and potentially make a significant difference in the lives of patients with lung cancer and prospectively other tumors," added Dr. Maky Zanganeh, Chief Executive Officer and President of Summit. "We want to again congratulate Akeso for this incredible result and their work to advance the patient-friendly standards of care today and well into the future. We look forward to initiating HARMONi-7 and sharing additional details about our expanded clinical development plan in early 2025."

Phase II Perioperative NSCLC

In addition to the HARMONi-2 data presentation, a second oral presentation featuring ivonescimab was presented by Xiaoliang Zhao, MD, Deputy Chief Physician, Department of Lung Cancer at Tianjin Medical University Cancer Institute & Hospital, and Visiting Scholar at the Lombardi Comprehensive Cancer Center at Georgetown University in Washington, D.C. The presentation was entitled, A Phase II Study of Perioperative Ivonescimab Alone or Combined with Chemotherapy in Resectable Non-Small Cell Lung Cancer, presenting the results from AK112-205, a single-region (China), multi-center, open-label Phase II study of patients with Stage II or III resectable NSCLC.

The study is designed to assess patients receiving either ivonescimab monotherapy or ivonescimab plus chemotherapy prior to surgical resection and then ivonescimab monotherapy after surgery. Due to the maturity of the data and the timing of the data cutoff, the results were limited to the neo-adjuvant, or pre-surgery, portion of the clinical trial. At the time of data cutoff, 49 patients had been enrolled into the ivonescimab plus chemotherapy arm in the neo-adjuvant setting; of these 49 patients, 39 went on to complete surgery.

Of the 39 patients who received ivonescimab plus chemotherapy in the neo-adjuvant stage and completed surgery, 71.8% of patients experienced a major pathological response (MPR) and 43.6% of patients experienced a pathological complete response (pCR). In the 49 patients enrolled in this cohort, median event-free survival (EFS) was not yet reached after 8.9 months of median follow-up time; the 12-month EFS rate was 80.3% (95% CI: 59.6, 91.1). These results are encouraging compared to the historical data that has been observed in global pivotal studies in a similar setting. The safety profile was manageable: of the 49 patients who received ivonescimab plus chemotherapy in the neo-adjuvant setting, Grade 3 or higher adverse events were observed in 32.7% of patients; there was one patient who experienced a treatment-related serious adverse event. There were no TRAEs leading to delayed or cancelled surgery or that led to the death of a patient.

Additional Phase II Data to be Presented at ESMO (Free ESMO Whitepaper) 2024

Beyond the data recently featured at WCLC 2024, Phase II data featuring ivonescimab will be presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2024. ESMO (Free ESMO Whitepaper) 2024 will take place in Barcelona, Spain, from September 13 – 17, 2024.

These presentations, which will feature data from studies sponsored by Akeso including first-line treatment for triple-negative advanced breast cancer (TNBC), first-line treatment for advanced head and neck squamous cell carcinoma (HNSCC), and first-line treatment of advanced colorectal cancer (CRC).

About the ESMO (Free ESMO Whitepaper) 2024 Presentations

Presentation Title: The efficacy and safety of ivonescimab with or without ligufalimab in combination with FOLFOXIRI (chemotherapy) as first-line treatment for metastatic colorectal cancer (mCRC)

ESMO Presentation No.: 514MO

Session Date & Time: Saturday, September 14, 3:50 to 3:55pm CET

Presentation Title: The safety and efficacy of ivonescimab in combination with chemotherapy as first-line treatment for triple-negative breast cancer (TNBC)

ESMO Presentation No.: 347MO

Session Date & Time: Monday, September 16, 8:30 to 8:35pm CET

Poster Title: Evaluation of the safety and efficacy of ivonescimab in combination with ligufalimab as first-line treatment for PD-L1 positive recurrent/metastasis head and neck squamous cell carcinoma (R/M HNSCC)

ESMO Poster No.: 876P

Poster Display Date & Time: Saturday, September 14, 12:00 to 1:00pm CET

Conference Call

Summit Therapeutics Inc. will host a conference call to discuss recent updates related to ivonescimab, including data released at WCLC, on Monday September 9, 2024, before the market opens.

Summit will host a live webcast of the conference call at 8:30am ET, which will be accessible through our website www.smmttx.com, and can also be accessed via the following link: View Source

The dial-in information for US attendees is toll-free at (800) 715-9871. Additionally, all attendees may access through the toll number, (646) 307-1963. The Conference ID is 9820029.

An archived edition of the webcast will be available on our website later in the day on Monday.

About Ivonescimab

Ivonescimab, known as SMT112 in Summit’s license territories, the United States, Canada, Europe, Japan, Latin America, including Mexico and all countries in Central America, South America, and the Caribbean, the Middle East, and Africa, and as AK112 in China and Australia, is a novel, potential first-in-class investigational bispecific antibody combining the effects of immunotherapy via a blockade of PD-1 with the anti-angiogenesis effects associated with blocking VEGF into a single molecule. Ivonescimab displays unique cooperative binding to each of its intended targets with multifold higher affinity when in the presence of both PD-1 and VEGF.

This could differentiate ivonescimab as there is potentially higher expression (presence) of both PD-1 and VEGF in tumor tissue and the tumor microenvironment (TME) as compared to normal tissue in the body. Ivonescimab’s tetravalent structure (four binding sites) enables higher avidity (accumulated strength of multiple binding interactions) in the TME with over 18-fold increased binding affinity to PD-1 in the presence of VEGF in vitro, and over 4-times increased binding affinity to VEGF in the presence of PD-1 in vitro (Zhong, et al, SITC (Free SITC Whitepaper), 2023). This tetravalent structure, the intentional novel design of the molecule, and bringing these two targets into a single bispecific antibody with cooperative binding qualities have the potential to direct ivonescimab to the tumor tissue versus healthy tissue. The intent of this design, together with a half-life of 6 to 7 days (Zhong, et al, SITC (Free SITC Whitepaper), 2023), is to improve upon previously established efficacy thresholds, in addition to side effects and safety profiles associated with these targets.

Ivonescimab was engineered by Akeso Inc. (HKEX Code: 9926.HK) and is currently engaged in multiple Phase III clinical trials. Over 1,800 patients have been treated with ivonescimab in clinical studies globally.

Summit has begun its clinical development of ivonescimab in non-small cell lung cancer (NSCLC), commencing enrollment in 2023 in two multi-regional Phase III clinical trials, HARMONi and HARMONi-3, with a plan to initiate HARMONi-7 in early 2025.

HARMONi is a Phase III clinical trial which intends to evaluate ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with a 3rd generation EGFR TKI (e.g., osimertinib).

HARMONi-3 is a Phase III clinical trial which is designed to evaluate ivonescimab combined with chemotherapy compared to pembrolizumab combined with chemotherapy in patients with first-line metastatic squamous NSCLC.

HARMONi-7 is a planned Phase III clinical trial which is intended to evaluate ivonescimab monotherapy compared to pembrolizumab monotherapy in patients with first-line metastatic NSCLC whose tumors have high PD-L1 expression (PD-L1 TPS > 50%).

In addition, Akeso has recently had positive read-outs in two single-region (China), randomized Phase III clinical trials for ivonescimab in NSCLC, HARMONi-A and HARMONi-2.

HARMONi-A was a Phase III clinical trial which evaluated ivonescimab combined with chemotherapy compared to placebo plus chemotherapy in patients with EGFR-mutated, locally advanced or metastatic non-squamous NSCLC who have progressed after treatment with an EGFR TKI.

HARMONi-2 is a Phase III clinical trial evaluating monotherapy ivonescimab against monotherapy pembrolizumab in patients with locally advanced or metastatic NSCLC whose tumors have positive PD-L1 expression (PD-L1 TPS >1%).

Ivonescimab is an investigational therapy that is not approved by any regulatory authority in Summit’s license territories, including the United States and Europe. Ivonescimab was approved for marketing authorization in China in May 2024.

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.

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.

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.