Results from LENVIMA® (lenvatinib) plus KEYTRUDA® (pembrolizumab) Trials in Unresectable Hepatocellular Carcinoma and Advanced Renal Cell Carcinoma to be Presented at 2020 ASCO Annual Meeting

On May 29, 2020 Eisai (CEO: Haruo Naito) and Merck & Co., Inc., Kenilworth, N.J., U.S.A. known as MSD outside the United States and Canada, reported that results from two trials evaluating LENVIMA, an orally available multiple receptor tyrosine kinase inhibitor discovered by Eisai, plus KEYTRUDA, the anti-PD-1 therapy from Merck & Co., Inc., Kenilworth, N.J., U.S.A., will be presented at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) (ASCO20 Virtual Scientific Program) from May 29 to 31, 2020 (Press release, Eisai, MAY 29, 2020, View Source [SID1234558745]). The two trials, Study 116/KEYNOTE-524 and Study 111/KEYNOTE-146, examined patients with unresectable hepatocellular carcinoma (HCC) with no prior systemic therapy and patients with metastatic clear cell renal cell carcinoma (ccRCC) who progressed following immune checkpoint inhibitor therapy, respectively.

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"The tumor response rates demonstrated with KEYTRUDA plus LENVIMA in these studies underscore the potential of this combination regimen in certain types of hepatocellular and renal cell carcinoma," said Dr. Jonathan Cheng, Vice President, Oncology Clinical Research, Merck & Co., Inc., Kenilworth, N.J., U.S.A. Research Laboratories. "KEYTRUDA plus LENVIMA is an important pillar of our broad oncology research program, and we continue to advance the study of the combination across multiple types of cancers and stages of disease."

"As data from our combination trials continue to read out, our enthusiasm for and belief in the potential of LENVIMA plus KEYTRUDA are strengthened by the growing body of evidence observed in multiple advanced cancers," said Dr. Takashi Owa, Chief Medicine Creation and Chief Discovery Officer, Oncology Business Group at Eisai. "Our ongoing clinical study efforts on this combination exemplify our commitment to following the science and exploring possible solutions for patients affected by difficult-to-treat cancers."

Results from Study 116/KEYNOTE-524 (Abstract #4519) are being presented in a poster discussion session, and results from Study 111/KEYNOTE-146 (Abstract #5008) are being presented in an oral abstract session of the virtual scientific program of the 2020 ASCO (Free ASCO Whitepaper) Annual Meeting.

Study 116/KEYNOTE-524 Trial Design and Data (Abstract #4519)

Study 116/KEYNOTE-524 (ClinicalTrials.gov, NCT03006926(New Window)) is a Phase 1b, open-label, single-arm trial evaluating the LENVIMA plus KEYTRUDA combination in 100 patients with unresectable HCC with no prior systemic therapy. Patients were treated with LENVIMA 8 or 12 mg (based on baseline body weight ˂60 kilograms or ≥60 kilograms, respectively) orally once daily in combination with KEYTRUDA 200 mg intravenously every three weeks. The primary endpoints are ORR and duration of response (DOR) by modified Response Evaluation Criteria in Solid Tumors (mRECIST) and RECIST v1.1 per independent imaging review (IIR). Secondary endpoints include progression-free survival (PFS), time to progression (TTP) and overall survival (OS). At data cutoff (October 31, 2019) and a median duration of follow-up of 10.6 months (95% CI: 9.2-11.5), 37 patients were still on study treatment (LENVIMA plus KEYTRUDA: n=34; LENVIMA only: n=3), and median duration of treatment exposure to the LENVIMA plus KEYTRUDA combination was 7.9 months (range: 0.2-31.1).

The final analysis of the study’s primary endpoints showed the LENVIMA plus KEYTRUDA combination demonstrated an ORR of 36% (n=36) (95% CI: 26.6-46.2), with a complete response rate of 1% (n=1) and a partial response rate of 35% (n=35), and a median DOR of 12.6 months (95% CI: 6.9-not estimable [NE]), using RECIST v1.1 criteria per IIR. As assessed using mRECIST criteria per IIR, the LENVIMA plus KEYTRUDA combination demonstrated an ORR of 46% (n=46) (95% CI: 36.0-56.3), with a complete response rate of 11% (n=11) and a partial response rate of 35% (n=35), and a median DOR of 8.6 months (95% CI: 6.9-NE).

Treatment-related adverse events (TRAEs) led to discontinuation of LENVIMA and KEYTRUDA in 6% of patients, of LENVIMA in 14% of patients, and of KEYTRUDA in 10% of patients. Grade ≥3 TRAEs occurred in 67% of patients (Grade 3: 63%; Grade 4: 1%; Grade 5: 3%). There was one Grade 4 TRAE (leukopenia/neutropenia), and there were three treatment-related deaths (acute respiratory failure/acute respiratory distress syndrome, intestinal perforation and abnormal hepatic function; n=1 for each). The most common TRAEs of any grade (≥20%) were hypertension (36%), diarrhea (35%), fatigue (30%), decreased appetite (28%), hypothyroidism (25%), palmar-plantar erythrodysesthesia syndrome (23%), decreased weight (22%), dysphonia (21%), increased aspartate aminotransferase (20%) and proteinuria (20%).

Study 111/KEYNOTE-146 Trial Design and Data from the RCC Cohort (Abstract #5008)

KEYNOTE-146/Study 111 (ClinicalTrials.gov, NCT02501096(New Window)) is a Phase 1b/2, open-label, single-arm trial evaluating the LENVIMA plus KEYTRUDA combination in patients with selected solid tumors. Results from the RCC cohort of the Phase 2 part of the study are based on 104 patients with metastatic ccRCC with disease progression following PD-1/PD-L1 immune checkpoint inhibitor therapy using RECIST v1.1 criteria. Patients were treated with LENVIMA 20 mg orally once daily in combination with KEYTRUDA 200 mg intravenously every three weeks until unacceptable toxicity or disease progression. The primary endpoint is ORR at week 24 by immune-related RECIST (irRECIST) per investigator review. The key secondary endpoints include ORR, PFS, OS, safety and tolerability for a maximum of 35 cycles/treatments (approximately two years).

At data cutoff (April 9, 2020), results from the Phase 2 part of the study showed the LENVIMA plus KEYTRUDA combination demonstrated an ORR at week 24 of 51% (95% CI: 41-61) by irRECIST per investigator review. As assessed by irRECIST per investigator review, ORR was 55% (95% CI: 45-65), with a partial response rate of 55%, stable disease rate of 36% and progressive disease rate of 5% (5% were not evaluable). Median DOR was 12 months (95% CI: 9-18). Median PFS was 11.7 months (95% CI: 9.4-17.7), and the 12-month PFS rate was 45% (95% CI: 32-57). Median OS was not reached (95% CI:16.7-NR), and the 12-month OS rate was 77% (95% CI: 67-85).

As assessed by RECIST v1.1 per investigator review, ORR was 52% (95% CI: 42-62), with a partial response rate of 52%, stable disease rate of 38% and progressive disease rate of 6% (5% were not evaluable). Median DOR was 12 months (95% CI: 9-18). Median PFS was 11.3 months (95% CI: 7.6-17.7), and the 12-month PFS rate was 44% (95% CI: 31-55).

Treatment-related adverse events (TRAEs) led to discontinuation of LENVIMA and KEYTRUDA in 15% of patients, discontinuation of LENVIMA in 12% of patients, and discontinuation of KEYTRUDA in 12% of patients (2% due to proteinuria). Grade 4 TRAEs included lipase increased, diverticulitis, large intestine perforation and myocardial infarction, and Grade 5 TRAEs included upper gastrointestinal hemorrhage and sudden death. The most common TRAEs of any grade (≥20%) were fatigue (53%), diarrhea (46%), proteinuria (39%), dysphonia (35%), hypertension (34%), nausea (32%), stomatitis (32%), arthralgia (29%), decreased appetite (28%), palmar-plantar erythrodysesthesia syndrome (25%), hypothyroidism (23%) and headache (22%).

This release discusses investigational uses for FDA-approved products. It is not intended to convey conclusions about efficacy and safety. There is no guarantee that any investigational uses of FDA-approved products will successfully complete clinical development or gain FDA approval.

LENVIMA, discovered and developed by Eisai, is a kinase inhibitor that inhibits the kinase activities of vascular endothelial growth factor (VEGF) receptors VEGFR1 (FLT1), VEGFR2 (KDR), and VEGFR3 (FLT4). LENVIMA inhibits other kinases that have been implicated in pathogenic angiogenesis, tumor growth, and cancer progression in addition to their normal cellular functions, including fibroblast growth factor (FGF) receptors FGFR1-4, the platelet derived growth factor receptor alpha (PDGFRα), KIT, and RET. In syngeneic mouse tumor models, lenvatinib decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity in combination with an anti-PD-1 monoclonal antibody compared to either treatment alone. Currently, LENVIMA has been approved for monotherapy as a treatment for thyroid cancer in over 60 countries including Japan, the United States, in Europe and Asia, and for unresectable hepatocellular carcinoma in over 55 countries including Japan, the United States, in Europe, China and in Asia. Additionally, LENVIMA is also approved in combination with everolimus as a treatment for renal cell carcinoma following prior antiangiogenic therapy in over 55 countries, including the United States and in Europe. In Europe, the agent was launched under the brand name Kisplyx for renal cell carcinoma. In addition, LENVIMA is approved in combination with KEYTRUDA (pembrolizumab) as a treatment for advanced endometrial carcinoma that is not microsatellite instability-high (MSI-H) or mismatch repair deficient (dMMR) who have disease progression following prior systemic therapy and are not candidates for curative surgery or radiation in countries including the United States, Australia, and Canada. This indication is approved under accelerated approval based on tumor response rate and durability of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in the confirmatory trials.

About KEYTRUDA (pembrolizumab) Injection

KEYTRUDA is an anti-PD-1 therapy that works by increasing the ability of the body’s immune system to help detect and fight tumor cells. KEYTRUDA is a humanized monoclonal antibody that blocks the interaction between PD-1 and its ligands, PD-L1 and PD-L2, thereby activating T lymphocytes which may affect both tumor cells and healthy cells.

Merck & Co., Inc., Kenilworth, N.J., U.S.A has the industry’s largest immuno-oncology clinical research program. There are currently more than 1,200 trials studying KEYTRUDA across a wide variety of cancers and treatment settings. The KEYTRUDA clinical program seeks to understand the role of KEYTRUDA across cancers and the factors that may predict a patient’s likelihood of benefitting from treatment with KEYTRUDA, including exploring several different biomarkers.

About the Eisai and Merck & Co., Inc., Kenilworth, N.J., U.S.A. Strategic Collaboration

In March 2018, Eisai and Merck & Co., Inc., Kenilworth, N.J., U.S.A., known as MSD outside the United States and Canada, through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of LENVIMA. Under the agreement, the companies will jointly develop, manufacture and commercialize LENVIMA, both as monotherapy and in combination with Merck & Co., Inc., Kenilworth, N.J., U.S.A.’s anti-PD-1 therapy KEYTRUDA.

In addition to ongoing clinical studies evaluating the LENVIMA plus KEYTRUDA combination across several different tumor types, the companies have jointly initiated new clinical studies through the LEAP (LEnvatinib And Pembrolizumab) clinical program and are evaluating the combination in 13 different tumor types (endometrial carcinoma, hepatocellular carcinoma, melanoma, non-small cell lung cancer, renal cell carcinoma, squamous cell carcinoma of the head and neck, urothelial cancer, biliary tract cancer, colorectal cancer, gastric cancer, glioblastoma, ovarian cancer and triple-negative breast cancer) across 18 clinical trials.

Eisai’s Focus on Cancer

Eisai focuses on the development of anticancer drugs, targeting the tumor microenvironment (with experience and knowledge from Halaven (Eribulin mesilate) and Lenvima) and the driver gene mutation and aberrant splicing (leveraging RNA Splicing Platform) as areas (Ricchi) where real patient needs are still unmet, and where Eisai can become a frontrunner in oncology. Eisai will discover innovative new drugs with new targets and mechanisms of action from these Ricchi, with the aim of contributing to the cure of cancers.

ENHERTU® Demonstrated Meaningful Clinical Activity in Patients with HER2 Mutant Non-Small Cell Lung Cancer in Interim Analysis of Phase 2 DESTINY-Lung01 Trial

On May 29, 2020 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) and AstraZeneca reported that Results from the ongoing phase 2 DESTINY-Lung01 trial showed ENHERTU (fam-trastuzumab deruxtecan-nxki) achieved a clinically meaningful tumor response in patients with HER2 mutant unresectable and/or metastatic non-squamous non-small cell lung cancer (NSCLC) whose disease had progressed following one or more systemic therapies (Press release, Daiichi Sankyo, MAY 29, 2020, View Source [SID1234558744]). These results were presented today at the 2020 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Virtual Scientific Program (#ASCO20).

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Lung cancer is the leading cause of cancer death among both men and women, and accounts for about one-fifth of all cancer deaths, with 80 to 85 percent classified as NSCLC.[1],[2] There are currently no medicines approved specifically for the treatment HER2 mutant NSCLC, which affects approximately two to four percent of patients with NSCLC.[3],[4]

The primary endpoint of confirmed objective response rate (ORR), also called a tumor response rate, which was assessed by independent central review (ICR), was 61.9% for patients treated with ENHERTU monotherapy (6.4 mg/kg). Patients achieved a disease control rate (DCR) of 90.5% with a median progression-free survival (PFS) of 14.0 months. Median duration of response (DoR) and overall survival (OS) had not yet been reached at the time of data cut-off.

The overall safety and tolerability profile of ENHERTU in DESTINY-Lung01 was consistent with that seen in the phase 1 trial and previously reported ENHERTU trials. The most common grade 3 or higher treatment emergent adverse events were decreased neutrophil count (26.2%) and anemia (16.7%). There were five cases (11.9%) of confirmed treatment-related interstitial lung disease (ILD) and pneumonitis as determined by an independent adjudication committee. All ILD and pneumonitis were grade 2. One grade 1 ILD is still undergoing adjudication.

"While there have been important advances in the treatment of lung cancer over the past decade, there are still patients whose tumors continue to progress despite treatment with newer targeted agents or immunotherapies," said Egbert F. Smit, MD, PhD, Professor, Department of Thoracic Oncology at the Netherlands Cancer Institute, Amsterdam, Netherlands and principal investigator of the DESTINY-Lung01 trial. "Understanding additional molecular targets for treatment, such as HER2, is critical to advancing treatment options for these patients and the results seen in the DESTINY-Lung01 trial are very encouraging."

Patients were treated with a median of two prior lines of therapy (range, 1-6) with most receiving platinum-based chemotherapy (90.5%) and anti-PD-1 or PD-L1 treatment (54.8%). Median treatment duration was 7.75 months (0.7-14.3) with a median duration of follow-up of 8.0 months (1.4-14.2). As of data cut-off on November 25, 2019, 45.2% of patients with HER2 mutated metastatic NSCLC remained on treatment with ENHERTU.

"While the role of anti-HER2 treatment is well-established in breast and gastric cancers, there are no HER2 directed therapies specifically approved for lung cancer," said Antoine Yver, MD, MSc, Executive Vice President and Global Head, Oncology Research and Development, Daiichi Sankyo. "These results validate HER2 mutations as actionable targets in lung cancer and offer further evidence that ENHERTU has the potential to transform outcomes for these patients."

"The results seen with ENHERTU in patients with metastatic HER2 mutant non-small cell lung cancer are very exciting and highlight the role ENHERTU may have as a new treatment option for patients facing a devastating prognosis," said José Baselga, MD, PhD, Executive Vice President, Oncology R&D, AstraZeneca. "Further, the results demonstrate the potential of ENHERTU to treat another tumor type among patients with extremely high unmet need."

ENHERTU recently was granted a Breakthrough Therapy Designation in the U.S. for the treatment of patients with metastatic NSCLC whose tumors have a HER2 mutation and with disease progression on or after platinum-based therapy.

About DESTINY-Lung01

DESTINY-Lung01 is a global, phase 2, open-label, multicenter, two-cohort trial evaluating the safety and efficacy of ENHERTU in 170 patients with HER2 mutant or HER2 overexpressing (defined as IHC 3+ or IHC 2+) unresectable and metastatic non-squamous NSCLC. Patients had progressed after one or more systemic therapies including chemotherapy, molecular targeted therapy or immunotherapy. The primary endpoint is ORR by independent central review. ORR, or tumor response rate, represents the percentage of patients whose disease decreased and/or disappears. Key secondary endpoints include DoR, DCR, PFS and OS.

About HER2

HER2 is a tyrosine kinase receptor growth-promoting protein expressed on the surface of many types of tumors including breast, gastric, lung and colorectal cancers. In some tumors, HER2 overexpression is associated with a specific HER2 gene alteration known as amplification and is often associated with aggressive disease and poorer prognosis.[5]

Other HER2 gene alterations (called HER2 mutations) have been identified in NSCLC, specifically adenocarcinomas, as distinct molecular targets and have been reported in approximately two to four percent of patients with NSCLC.3,4,[6],[7] These acquired HER2 gene mutations have been independently associated with cancer cell growth and poor prognosis.3,4

About NSCLC

Lung cancer is the leading cause of cancer death among both men and women and accounts for about one-fifth of all cancer deaths.1 NSCLC accounts for approximately 80 to 85 percent of all lung cancers.2 For patients with metastatic disease, prognosis is particularly poor, as only six to 10 percent will be alive five years after diagnosis.[8] The introduction of targeted therapies and checkpoint inhibitors in recent years has improved the treatment landscape for patients with advanced NSCLC; however, new approaches are needed for those who are not eligible for available treatments, or whose cancer continues to progress.[9] Currently, no medicine is specifically approved to treat patients with HER2 mutant NSCLC.

About ENHERTU

ENHERTU (fam-trastuzumab deruxtecan-nxki in the U.S. only; trastuzumab deruxtecan outside the U.S.) is a HER2 directed antibody drug conjugate (ADC) and is the lead ADC in the oncology portfolio of Daiichi Sankyo and the most advanced program in AstraZeneca’s ADC Scientific platform.

ADCs are targeted cancer medicines that deliver cytotoxic chemotherapy ("payload") to cancer cells via a linker attached to a monoclonal antibody that binds to a specific target expressed on cancer cells. Designed using Daiichi Sankyo’s proprietary DXd ADC technology, ENHERTU is comprised of a HER2 monoclonal antibody attached to a novel topoisomerase I inhibitor payload by a tetrapeptide-based linker.

ENHERTU (5.4 mg/kg) is approved in the U.S. and Japan for the treatment of adult patients with unresectable or metastatic HER2 positive breast cancer who received two or more prior anti-HER2 based regimens based on the DESTINY-Breast01 trial.

ENHERTU has not been approved in the EU, or countries outside of Japan and the U.S., for any indication. It is an investigational agent globally for various indications. Safety and effectiveness have not been established for the subject proposed use.

About the ENHERTU Clinical Development Program

A comprehensive development program for ENHERTU is underway globally with six pivotal trials evaluating the efficacy and safety of ENHERTU monotherapy across multiple HER2 targetable cancers including breast, gastric and lung cancers. Trials in combination with other anticancer treatments, such as immunotherapy, also are underway.

In May 2020, ENHERTU received BTD from the U.S. Food and Drug Administration (FDA) for the treatment of patients with HER2 positive unresectable or metastatic gastric or gastroesophageal junction adenocarcinoma who have received two or more prior regimens including trastuzumab, and Orphan Drug Designation for gastric cancer, including gastroesophageal junction cancer. In March 2018, ENHERTU received a SAKIGAKE designation for potential use in the same HER2 positive patient population and a supplemental New Drug Application (NDA) was submitted to the Japan Ministry of Health, Labour and Welfare (MHLW) for approval in April 2020.

In May 2020, ENHERTU also received a BTD for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have a HER2 mutation and with disease progression on or after platinum-based therapy.

About the Collaboration between Daiichi Sankyo and AstraZeneca

In March 2019, Daiichi Sankyo and AstraZeneca entered into a global collaboration to jointly develop and commercialize ENHERTU worldwide, except in Japan where Daiichi Sankyo maintains exclusive rights. Daiichi Sankyo is solely responsible for the manufacturing and supply.

U.S. FDA-Approved Indication for ENHERTU

ENHERTU is a HER2-directed antibody and topoisomerase inhibitor conjugate indicated for the treatment of adult patients with unresectable or metastatic HER2-positive breast cancer who have received two or more prior anti-HER2-based regimens in the metastatic setting.

This indication is approved under accelerated approval based on tumor response rate and duration of response. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

WARNING: INTERSTITIAL LUNG DISEASE and EMBRYO-FETAL TOXICITY

● Interstitial lung disease (ILD) and pneumonitis, including fatal cases, have been reported with ENHERTU. Monitor for and promptly investigate signs and symptoms including cough, dyspnea, fever, and other new or worsening respiratory symptoms. Permanently discontinue ENHERTU in all patients with Grade 2 or higher ILD/pneumonitis. Advise patients of the risk and to immediately report symptoms.

● Exposure to ENHERTU during pregnancy can cause embryo-fetal harm. Advise patients of these risks and the need for effective contraception.

Interstitial Lung Disease / Pneumonitis
Severe, life-threatening, or fatal interstitial lung disease (ILD), including pneumonitis, can occur in patients treated with ENHERTU. In clinical studies, of the 234 patients with unresectable or metastatic HER2-positive breast cancer treated with ENHERTU, ILD occurred in 9% of patients. Fatal outcomes due to ILD and/or pneumonitis occurred in 2.6% of patients treated with ENHERTU. Median time to first onset was 4.1 months (range: 1.2 to 8.3).

Advise patients to immediately report cough, dyspnea, fever, and/or any new or worsening respiratory symptoms. Monitor patients for signs and symptoms of ILD. Promptly investigate evidence of ILD. Evaluate patients with suspected ILD by radiographic imaging. Consider consultation with a pulmonologist. For asymptomatic ILD/pneumonitis (Grade 1), interrupt ENHERTU until resolved to Grade 0, then if resolved in ≤28 days from date of onset, maintain dose. If resolved in >28 days from date of onset, reduce dose one level. Consider corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥0.5 mg/kg prednisolone or equivalent). For symptomatic ILD/pneumonitis (Grade 2 or greater), permanently discontinue ENHERTU. Promptly initiate corticosteroid treatment as soon as ILD/pneumonitis is suspected (e.g., ≥1 mg/kg prednisolone or equivalent). Upon improvement, follow by gradual taper (e.g., 4 weeks).

Neutropenia

Severe neutropenia, including febrile neutropenia, can occur in patients treated with ENHERTU. Of the 234 patients with unresectable or metastatic HER2-positive breast cancer who received ENHERTU, a decrease in neutrophil count was reported in 30% of patients and 16% had Grade 3 or 4 events. Median time to first onset was 1.4 months (range: 0.3 to 18.2). Febrile neutropenia was reported in 1.7% of patients.

Monitor complete blood counts prior to initiation of ENHERTU and prior to each dose, and as clinically indicated. Based on the severity of neutropenia, ENHERTU may require dose interruption or reduction. For Grade 3 neutropenia (Absolute Neutrophil Count [ANC] <1.0 to 0.5 x 109/L) interrupt ENHERTU until resolved to Grade 2 or less, then maintain dose. For Grade 4 neutropenia (ANC <0.5 x 109/L) interrupt ENHERTU until resolved to Grade 2 or less. Reduce dose by one level. For febrile neutropenia (ANC <1.0 x 109/L and temperature >38.3ºC or a sustained temperature of ≥38ºC for more than 1 hour), interrupt ENHERTU until resolved. Reduce dose by one level.

Left Ventricular Dysfunction

Patients treated with ENHERTU may be at increased risk of developing left ventricular dysfunction. Left ventricular ejection fraction (LVEF) decrease has been observed with anti-HER2 therapies, including ENHERTU. In the 234 patients with unresectable or metastatic HER2-positive breast cancer who received ENHERTU, two cases (0.9%) of asymptomatic LVEF decrease were reported. Treatment with ENHERTU has not been studied in patients with a history of clinically significant cardiac disease or LVEF <50% prior to initiation of treatment.

Assess LVEF prior to initiation of ENHERTU and at regular intervals during treatment as clinically indicated. Manage LVEF decrease through treatment interruption. Permanently discontinue ENHERTU if LVEF of <40% or absolute decrease from baseline of >20% is confirmed. When LVEF is >45% and absolute decrease from baseline is 10-20%, continue treatment with ENHERTU. When LVEF is 40-45% and absolute decrease from baseline is <10%, continue treatment with ENHERTU and repeat LVEF assessment within 3 weeks. When LVEF is 40-45% and absolute decrease from baseline is 10-20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF has not recovered to within 10% from baseline, permanently discontinue ENHERTU. If LVEF recovers to within 10% from baseline, resume treatment with ENHERTU at the same dose. When LVEF is <40% or absolute decrease from baseline is >20%, interrupt ENHERTU and repeat LVEF assessment within 3 weeks. If LVEF of <40% or absolute decrease from baseline of >20% is confirmed, permanently discontinue ENHERTU. Permanently discontinue ENHERTU in patients with symptomatic congestive heart failure.

Embryo-Fetal Toxicity
ENHERTU can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risks to a fetus. Verify the pregnancy status of females of reproductive potential prior to the initiation of ENHERTU. Advise females of reproductive potential to use effective contraception during treatment and for at least 7 months following the last dose of ENHERTU. Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ENHERTU and for at least 4 months after the last dose of ENHERTU.

Adverse Reactions
The safety of ENHERTU was evaluated in a pooled analysis of 234 patients with unresectable or metastatic HER2-positive breast cancer who received at least one dose of ENHERTU 5.4 mg/kg in DESTINY-Breast01 and Study DS8201-A-J101. ENHERTU was administered by intravenous infusion once every three weeks. The median duration of treatment was 7 months (range: 0.7 to 31).

Serious adverse reactions occurred in 20% of patients receiving ENHERTU. Serious adverse reactions in >1% of patients who received ENHERTU were interstitial lung disease, pneumonia, vomiting, nausea, cellulitis, hypokalemia, and intestinal obstruction. Fatalities due to adverse reactions occurred in 4.3% of patients including interstitial lung disease (2.6%), and the following events occurred in one patient each (0.4%): acute hepatic failure/acute kidney injury, general physical health deterioration, pneumonia, and hemorrhagic shock.

ENHERTU was permanently discontinued in 9% of patients, of which ILD accounted for 6%. Dose interruptions due to adverse reactions occurred in 33% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose interruption were neutropenia, anemia, thrombocytopenia, leukopenia, upper respiratory tract infection, fatigue, nausea, and ILD. Dose reductions occurred in 18% of patients treated with ENHERTU. The most frequent adverse reactions (>2%) associated with dose reduction were fatigue, nausea, and neutropenia.

The most common adverse reactions (frequency ≥20%) were nausea (79%), fatigue (59%), vomiting (47%), alopecia (46%), constipation (35%), decreased appetite (32%), anemia (31%), neutropenia (29%), diarrhea (29%), leukopenia (22%), cough (20%), and thrombocytopenia (20%).

Use in Specific Populations

● Pregnancy: ENHERTU can cause fetal harm when administered to a pregnant woman. Advise patients of the potential risks to a fetus. There are clinical considerations if ENHERTU is used in pregnant women, or if a patient becomes pregnant within 7 months following the last dose of ENHERTU.

● Lactation: There are no data regarding the presence of ENHERTU in human milk, the effects on the breastfed child, or the effects on milk production. Because of the potential for serious adverse reactions in a breastfed child, advise women not to breastfeed during treatment with ENHERTU and for 7 months after the last dose.

● Females and Males of Reproductive Potential: Pregnancy testing: Verify pregnancy status of females of reproductive potential prior to initiation of ENHERTU. Contraception: Females: ENHERTU can cause fetal harm when administered to a pregnant woman. Advise females of reproductive potential to use effective contraception during treatment with ENHERTU and for at least 7 months following the last dose. Males: Advise male patients with female partners of reproductive potential to use effective contraception during treatment with ENHERTU and for at least 4 months following the last dose. Infertility: ENHERTU may impair male reproductive function and fertility.

● Pediatric Use: Safety and effectiveness of ENHERTU have not been established in pediatric patients.

● Geriatric Use: Of the 234 patients with HER2-positive breast cancer treated with ENHERTU 5.4 mg/kg, 26% were ≥65 years and 5% were ≥75 years. No overall differences in efficacy were observed between patients ≥65 years of age compared to younger patients. There was a higher incidence of Grade 3-4 adverse reactions observed in patients aged ≥65 years (53%) as compared to younger patients (42%).

● Hepatic Impairment: In patients with moderate hepatic impairment, due to potentially increased exposure, closely monitor for increased toxicities related to the topoisomerase inhibitor.

Forma Therapeutics Eyes IPO to Advance Sickle Cell, Prostate Cancer Drugs

On May 29, 2020 Forma Therapeutics reported that kit has developed a pipeline of small molecules aimed at rare blood disorders and cancers, but it aims to advance two—investigational treatments for sickle cell disease and for advanced prostate cancer—and find partners for the rest of them (Press release, Forma Therapeutics, MAY 29, 2020, View Source [SID1234558743]).

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Now the Watertown, MA-based company is considering tapping the public markets to add enough funds to move one of its pair of core drug candidates further along in the clinic and to start human tests of the other. Forma set a preliminary $150 million goal for its IPO and says it plans to apply for a Nasdaq listing under the stock symbol "FMTX."

The company priorities are its oral, once-daily drug, FT-4202, which it is evaluating in a Phase 1 trial as a treatment for sickle cell disease, and FT-7051, a preclinical-stage treatment for metastatic castration-resistant prostate cancer (mCRPC).

Forma’s sickle cell drug is designed to increase hemoglobin levels and reduce the episodes of acute pain known as vaso-occlusive crises, caused when sickle-shape cells aggregate and block the supply of oxygen to tissues. The drug is intended to activate an enzyme, pyruvate kinase-R, that plays a role in the metabolism, function, and survival of red blood cells.

The biotech is studying the drug in patients age 12 and older who have the blood disorder. About 100,000 people in the US are affected by the inherited condition. A slew of companies are also pursuing new treatments for the disease, including Cambridge, MA-based Imara (NASDAQ: IMRA), which went public in March to raise funds to advance its investigational small molecule; Bluebird Bio (NASDAQ: BLUE), which is developing a gene therapy; and a handful of competitors with experimental gene-editing fixes. Late last year Global Blood Therapeutics (NASDAQ: GBT) won FDA approval for its once-daily sickle cell disease drug.

In its preliminary prospectus, Forma said it also aims to expand clinical development of FT-4202 into beta thalassemia, another rare blood disorder.

Forma’s prostate cancer treatment is designed to inhibit a protein complex, CBP/p300, that plays a role in the activity of the androgen receptor, which drives prostate cancer cell growth. The FDA in April cleared Forma to begin human tests of the drug.

Prostate cancer is the second leading cause of cancer death for men in the US; the type that Forma’s drug is intended to treat is the most advanced form of the disease. The UK’s CellCentric is also aiming to treat mCRPC with a CBP/p300 inhibitor of its own; Cambridge, MA-based Constellation Pharmaceuticals (NASDAQ: CNST) is advancing a clinical-stage drug for the indication, too.

Money raised in an IPO would be deployed to get the sickle cell drug through Phase 1 and, if results are as anticipated, proceed to a pivotal Phase 2/3 clinical trial. The funds would also be used to advance the cancer therapy into a planned Phase I clinical trial.

Some cash would also go toward Forma’s non-core programs—those already partnered and those seeking partners to take them forward. Programs still in search of partners include its most advanced, olutasidenib, which is currently in a pivotal study testing it as a treatment for acute myeloid leukemia. The investigational cancer drug is also being studied in an exploratory Phase I trial for glioma. In addition, Forma is advancing two potential treatments for nonalcoholic steatohepatitis, the liver disease better known as NASH.

Forma previously licensed a molecule it discovered, a potential treatment for non-Hodgkin lymphoma, to Celgene (now a subsidiary Bristol Myers Squibb (NYSE: BMY)) and a KRAS inhibitor, meant to treat solid tumors, to Boehringer Ingelheim.

The company, which was founded in 2007, is headed by president and CEO Frank Lee, who previously ran global development and commercial strategy for Genentech’s immunology, ophthalmology and infectious diseases divisions. Its chief medical officer, Patrick Kelly, who has been with the company since 2016, served previously in the oncology unit at Takeda Pharmaceutical (NYSE: TAK). Forma last month added to its C-suite David Cook, who was most recently at Seres Therapeutics (NASDAQ: MCRB), as its chief scientific officer.

Forma’s founding backers were the investment arms of Eli Lilly (NYSE: LLY) and Novartis (NYSE: NVS. Today, according to the prospectus, RA Capital owns 21.16 percent of the company; Novartis holds 12.63 percent; Baker Bros. Advisors, 10.58 percent; and Lilly, 10.15 percent. Other shareholders include Cormorant Asset Management and Singapore’s Biomedical Sciences Investment Fund.

As of the end of March the company reported about $142 million in cash, cash equivalents, and short-term investments.

Lilly’s CYRAMZA® (ramucirumab) Receives FDA Approval as First-Line Treatment for Metastatic EGFR-Mutated Non-Small Cell Lung Cancer

On May 29, 2020 Eli Lilly and Company (NYSE: LLY) reported that the U.S. Food and Drug Administration (FDA) has approved CYRAMZA (ramucirumab injection, 10 mg/mL solution), in combination with erlotinib, for the first-line treatment of people with metastatic non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) mutations (Press release, Eli Lilly, MAY 29, 2020, View Source [SID1234558742]). With this approval, CYRAMZA has now received six FDA approvals to treat certain types of lung, liver, stomach and colorectal cancers.

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CYRAMZA plus erlotinib is the first and only FDA-approved anti-VEGFR/EGFR TKI combination therapy for metastatic EGFR-mutated NSCLC. This approval is based on the efficacy and safety from the global, randomized, placebo-controlled Phase 3 RELAY trial. In the RELAY study, CYRAMZA, a VEGF receptor 2 antagonist, in combination with erlotinib, a globally approved EGFR-targeting tyrosine kinase inhibitor (TKI), demonstrated a statistically significant and clinically meaningful improvement in progression-free survival (PFS) – the time patients lived without their cancer growing or spreading after starting treatment – compared to placebo in combination with erlotinib [19.4 months in the CYRAMZA-containing arm compared to 12.4 months in the placebo-containing arm (HR=0.59; 95% CI, 0.46, 0.76; p<0.0001)]. The PFS treatment effect was consistent across exon 19 and exon 21 subgroups. The overall safety profile observed in the RELAY study was consistent with that of its individual components. RELAY is the second positive Phase 3 trial of CYRAMZA in metastatic NSCLC. The first was REVEL, which supported the approval of CYRAMZA plus docetaxel as a treatment for people with metastatic NSCLC whose cancer has progressed after prior platinum-based chemotherapy.

"The approval of this new first-line metastatic EGFR-mutated non-small cell lung cancer regimen, which inhibits the VEGFR and EGFR pathways together, is an important milestone in the treatment of this disease. It is wonderful that patients now have multiple options for initial therapy capable of delaying disease progression for considerably longer than erlotinib, which has been our traditional standard approach," said Edward Garon, M.D., David Geffen School of Medicine, University of California, and North America lead investigator of the RELAY trial. "Ramucirumab, in combination with erlotinib, is a welcomed first-line option to offer our patients with metastatic EGFR-mutated non-small cell lung cancer."

"This CYRAMZA combination regimen represents a new and meaningful treatment option for people with metastatic EGFR-mutated non-small cell lung cancer, and we are proud that it has been approved by the FDA for patients with this disease and the doctors who treat them," said Anne White, president of Lilly Oncology. "Today’s approval underscores Lilly’s continued commitment to people living with lung cancer and to delivering meaningful medicines that are tailored for those with advanced or metastatic cancers. It also further reinforces the value that CYRAMZA can provide in treating certain advanced or metastatic cancers."

Fifty percent of people with NSCLC present with advanced or metastatic disease at diagnosis.1 The five-year survival rate for metastatic NSCLC patients is six percent.2 In the U.S., it is estimated that approximately 15 percent of people diagnosed with NSCLC have an EGFR mutation.3

"We’re encouraged by CYRAMZA’s latest approval, which represents one step towards our goal of making EGFR-mutated non-small cell lung cancer into a manageable chronic disease," said Ivy Elkins, cofounder of EGFR Resisters. "Each new treatment option gives hope to those living with this disease and provides oncologists with more options that may help slow the spread of this deadly cancer, which is an important goal for many patients."

The labeling for CYRAMZA contains warnings and precautions for hemorrhage and gastrointestinal (GI) hemorrhage, including severe and sometimes fatal events; GI perforations, a potentially fatal event; impaired wound healing; arterial thromboembolic events (ATEs), including serious and sometimes fatal events; hypertension; infusion-related reactions (IRR) including severe and life-threatening reactions; worsening of pre-existing hepatic impairment; Posterior Reversible Encephalopathy Syndrome (PRES); proteinuria including nephrotic syndrome; thyroid dysfunction; and embryo-fetal toxicity. CYRAMZA should be permanently discontinued in patients who experience severe bleeding, a GI perforation, an ATE, uncontrolled hypertension, Grade 3 or 4 IRR, PRES, or nephrotic syndrome. Withhold CYRAMZA for 28 days prior to elective surgery. Do not administer CYRAMZA for at least two weeks following a major surgical procedure and until adequate wound healing.

The most common adverse reactions (all grades) observed in CYRAMZA with erlotinib-treated patients at a rate of ≥30% of patients and ≥2% higher than placebo with erlotinib-treated patients were infections, hypertension, stomatitis, proteinuria, alopecia, and epistaxis. The most common laboratory abnormalities ≥30% and ≥2% higher than the placebo were increased alanine aminotransferase, increased aspartate aminotransferase, anemia, thrombocytopenia, and neutropenia. Please see Important Safety Information below.

In addition to a recent approval for CYRAMZA in the European Union based on the RELAY results, Lilly has made a submission in Japan with regulatory action expected by the end of 2020.

Click here to view the EGFR-mutated NSCLC fact sheet.
Click here to view the CYRAMZA product photo.
Click here to view the CYRAMZA logo.

About the RELAY Trial
RELAY is a global randomized, double-blind, placebo-controlled Phase 3 study of CYRAMZA in combination with erlotinib, compared to placebo in combination with erlotinib, as a first-line treatment in previously untreated patients with metastatic NSCLC whose tumors have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations. EGFR-targeting TKIs are the current standard treatment options for EGFR-mutated NSCLC. Erlotinib, the TKI included in the RELAY trial regimen, is a globally approved treatment option for this type of lung cancer.

Initiated in 2015, the study randomized 449 patients across North America, Europe and Asia. The primary endpoint of the RELAY trial is PFS; key secondary endpoints include safety, overall response rate (ORR), duration of response (DoR), and overall survival (OS). On the primary endpoint of investigator-assessed PFS, CYRAMZA plus erlotinib (N=224) demonstrated statistically significant and clinically meaningful improvement in median PFS – the time patients lived without their cancer growing or spreading after starting treatment – by seven months compared to placebo plus erlotinib (N=225) [19.4 months in the CYRAMZA-containing arm compared to 12.4 months in the placebo-containing arm (HR=0.59; 95% CI, 0.46, 0.76; p<0.0001)]. The PFS treatment effect was consistent across exon 19 and exon 21 subgroups. At the time of the final analysis of PFS, OS data were not mature as only 26 percent of planned events for the final analysis had occurred (HR=0.83, 95% CI: 0.53, 1.30). A final OS analysis is planned when at least 300 events have occurred.

RELAY Trial Efficacy Results Supporting Approval

Endpoint

CYRAMZA + erlotinib

N=224

Placebo + erlotinib

N=225

Progression-Free Survival (primary outcome measure)

Number of events (%)a

122 (55%)

158 (70%)

Median – months (95% CI)

19.4 (15.4, 21.6)

12.4 (11.0, 13.5)

Hazard Ratio (95% CI)

0.59 (0.46, 0.76)

Stratified Log-rank p-value

<0.0001

Overall Response Rate (Complete Response + Partial Response) (secondary outcome measure)

Rate – percent (95% CI)

76% (71, 82)

75% (69, 80)

Duration of Response (DoR) (secondary outcome measure)

N=171

N=168

Median – months (95% CI)

18.0 (13.9, 19.8)

11.1 (9.7, 12.3)

Abbreviations: CI = confidence interval

a 4 of 122 events in CYRAMZA-treated patients and 1 of 158 events in placebo-treated patients were deaths.

Treatment discontinuation of all study drugs due to adverse reactions occurred in 13 percent of CYRAMZA with erlotinib-treated patients, with increased alanine aminotransferase (1.4%) and paronychia (1.4%) being the most common. The most common adverse reactions leading to treatment discontinuation of CYRAMZA were proteinuria (8.6%) and hyperbilirubinemia (6%).

Detailed RELAY efficacy and safety results were published in The Lancet Oncology.

Notes to Editors

About Lung Cancer and EGFR Mutations
Globally, lung cancer is the leading cause of cancer death, killing nearly 1.8 million people worldwide each year.4 In the U.S., lung cancer is the second most common cancer (not counting skin cancer) and the leading cause of cancer death, responsible for almost 25 percent of all cancer deaths – more than those from colorectal, breast and prostate cancers combined.5 It is estimated that there will be 228,820 new cases of lung cancer and 135,720 deaths from lung cancer in the U.S. in 2020.4 Non-small cell lung cancer (NSCLC) is much more common than other types of lung cancer and accounts for about 85 percent of all lung cancers.6 Stage IV NSCLC is a very difficult-to-treat cancer and the prognosis is poor for metastatic NSCLC.7 Fifty percent of NSCLC patients present with advanced or metastatic disease at diagnosis.1 The five-year survival rate for metastatic NSCLC is six percent.2

EGFR is a protein that helps cells grow and divide. When the EGFR gene is mutated it can cause the protein to be overactive, causing cells to grow and divide more quickly. EGFR mutations may occur in 10 to 35 percent of NSCLC tumors globally.8 In the U.S., it is estimated that approximately 15 percent of people diagnosed with NSCLC have an EGFR mutation.3 Activating EGFR mutations are found in about 10 to 20 percent of Caucasian patients with lung adenocarcinomas and in up to 40 to 60 percent of Asian patients.9,10,11 Regardless of ethnicity, these mutations are commonly found in females, non-smokers and those with adenocarcinoma histology.12,13 The most common activating mutations in EGFR are deletions within exon 19 and a substitution in exon 21 (L858R). These mutations are present in 90 percent of EGFR-mutated NSCLC tumors. The presence of these activating EGFR mutations in advanced NSCLC is associated with sensitivity to small-molecule EGFR TKIs.10,11

About CYRAMZA (ramucirumab)
In the U.S., CYRAMZA (ramucirumab) has six FDA approvals to treat four different types of cancers. CYRAMZA is being investigated in a broad global development program that has enrolled more than 15,000 patients across more than 100 trials worldwide. These include several studies investigating CYRAMZA in combination with other anti-cancer therapies for the treatment of multiple tumor types. To date, more than 150,000 patients have been treated with CYRAMZA.

CYRAMZA is an antiangiogenic therapy. It is a vascular endothelial growth factor (VEGF) Receptor 2 antagonist that binds specifically to VEGFR-2, thereby blocking the binding of the receptor ligands (VEGF-A, VEGF-C, and VEGF-D) – which may slow tumor growth. CYRAMZA inhibited angiogenesis in an in vivo animal model.

About Angiogenesis and VEGF Protein
Angiogenesis is the process of making new blood vessels. In a person with cancer, angiogenesis creates new blood vessels that give a tumor its own blood supply, allowing it to grow and spread.

Some tumors create proteins called VEGF. These proteins attach to the VEGF receptors of blood vessel cells causing new blood vessels to form around the tumors, enabling growth. Blocking the VEGF protein from binding to the receptors located on the surface of blood vessels helps to inhibit tumor growth by slowing angiogenesis and the blood supply that feeds tumors. Of the three known VEGF receptors, VEGF Receptor 2 is linked most closely to VEGF-induced tumor angiogenesis.

INDICATIONS FOR CYRAMZA
Gastric Cancer
CYRAMZA, as a single agent, or in combination with paclitaxel, is indicated for the treatment of patients with advanced or metastatic gastric or gastroesophageal junction (GEJ) adenocarcinoma with disease progression on or after prior fluoropyrimidine- or platinum-containing chemotherapy.

Non-Small Cell Lung Cancer
CYRAMZA, in combination with erlotinib, for first-line treatment of metastatic non-small cell lung cancer with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 (L858R) mutations.

CYRAMZA, in combination with docetaxel, is indicated for the treatment of patients with metastatic non-small cell lung cancer (NSCLC) with disease progression on or after platinum-based chemotherapy. Patients with epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) genomic tumor aberrations should have disease progression on FDA-approved therapy for these aberrations prior to receiving CYRAMZA.

Colorectal Cancer
CYRAMZA, in combination with FOLFIRI (irinotecan, folinic acid, and fluorouracil), is indicated for the treatment of patients with metastatic colorectal cancer (mCRC) with disease progression on or after prior therapy with bevacizumab, oxaliplatin, and a fluoropyrimidine.

Hepatocellular Carcinoma
CYRAMZA, as a single agent, is indicated for the treatment of patients with hepatocellular carcinoma (HCC) who have an alpha-fetoprotein (AFP) of ≥400 ng/mL and have been treated with sorafenib.

IMPORTANT SAFETY INFORMATION FOR CYRAMZA (ramucirumab)
Warnings and Precautions

Hemorrhage

CYRAMZA increased the risk of hemorrhage and gastrointestinal hemorrhage, including Grade ≥3 hemorrhagic events. In 2137 patients with various cancers treated with CYRAMZA, the incidence of all Grade hemorrhage ranged from 13-55%. Grade 3-5 hemorrhage incidence ranged from 2-5%.
Patients with gastric cancer receiving nonsteroidal anti-inflammatory drugs (NSAIDs) were excluded from enrollment in REGARD and RAINBOW; therefore, the risk of gastric hemorrhage in CYRAMZA-treated patients with gastric tumors receiving NSAIDs is unknown.
Patients with NSCLC receiving therapeutic anticoagulation or with evidence of major airway invasion by cancer were excluded from REVEL. In addition, patients with NSCLC with a recent history of gross hemoptysis, those receiving chronic therapy with NSAIDs or other anti-platelet therapy other than once daily aspirin or with radiographic evidence of major blood vessel invasion or intratumor cavitation were excluded from REVEL and RELAY; therefore the risk of pulmonary hemorrhage in these groups of patients is unknown.
Permanently discontinue CYRAMZA in patients who experience severe (Grade 3 or 4) bleeding.
Gastrointestinal Perforations

CYRAMZA can increase the risk of gastrointestinal perforation, a potentially fatal event. In 2137 patients with various cancers treated with CYRAMZA, the incidence of all Grade and Grade 3-5 gastrointestinal perforations ranged from <1-2%.
Permanently discontinue CYRAMZA in patients who experience a gastrointestinal perforation.
Impaired Wound Healing

CYRAMZA has the potential to adversely affect wound healing. CYRAMZA has not been studied in patients with serious or non-healing wounds.
Withhold CYRAMZA for 28 days prior to elective surgery. Do not administer CYRAMZA for at least 2 weeks following a major surgical procedure and until adequate wound healing. The safety of resumption of CYRAMZA after resolution of wound healing complications has not been established.
Arterial Thromboembolic Events (ATEs)

Serious, sometimes fatal, ATEs, including myocardial infarction, cardiac arrest, cerebrovascular accident, and cerebral ischemia, occurred across clinical trials. In 2137 patients with various cancers treated with CYRAMZA, the incidence of all Grade ATE was 1-3%. Grade 3-5 ATE incidence was <1-2%.
Permanently discontinue CYRAMZA in patients who experience an ATE.
Hypertension

An increased incidence of severe hypertension occurred in patients receiving CYRAMZA. Across five clinical studies, excluding RELAY, in 1916 patients with various cancers treated with CYRAMZA, the incidence of all Grade hypertension ranged from 11-26%. Grade 3-5 hypertension incidence ranged from 6-15%. In 221 patients with NSCLC receiving CYRAMZA in combination with erlotinib in the RELAY study, the incidence of new or worsening hypertension was higher (45%), as was the incidence of Grade 3-5 hypertension (24%). Of the patients experiencing new or worsening hypertension in RELAY (N=100 CYRAMZA and erlotinib; N=27 placebo and erlotinib), 13% of those treated with CYRAMZA and erlotinib required initiation of 3 or more antihypertensive medications compared to 4% of patients treated with placebo and erlotinib.
Control hypertension prior to initiating treatment with CYRAMZA. Monitor blood pressure every two weeks or more frequently as indicated during treatment. Withhold CYRAMZA for severe hypertension until medically controlled. Permanently discontinue CYRAMZA for medically significant hypertension that cannot be controlled with antihypertensive therapy or in patients with hypertensive crisis or hypertensive encephalopathy.
Infusion-Related Reactions (IRR)

IRR, including severe and life-threatening IRR, occurred in CYRAMZA clinical trials. Symptoms of IRR included rigors/tremors, back pain/spasms, chest pain and/or tightness, chills, flushing, dyspnea, wheezing, hypoxia, and paresthesia. In severe cases, symptoms included bronchospasm, supraventricular tachycardia, and hypotension. In 2137 patients with various cancers treated with CYRAMZA in which premedication was recommended or required, the incidence of all Grade IRR ranged from <1- 9%. Grade 3-5 IRR incidence was <1%.
Premedicate prior to each CYRAMZA infusion. Monitor patients during the infusion for signs and symptoms of IRR in a setting with available resuscitation equipment. Reduce the infusion rate by 50% for Grade 1-2 IRR. Permanently discontinue CYRAMZA for Grade 3- 4 IRR.
Worsening of Pre-existing Hepatic Impairment

Clinical deterioration, manifested by new onset or worsening encephalopathy, ascites, or hepatorenal syndrome, was reported in patients with Child-Pugh B or C cirrhosis who received single agent CYRAMZA. Use CYRAMZA in patients with Child-Pugh B or C cirrhosis only if the potential benefits of treatment are judged to outweigh the risks of clinical deterioration.
Based on safety data from REACH-2, in patients with Child-Pugh A liver cirrhosis, the pooled incidence of hepatic encephalopathy and hepatorenal syndrome was higher for patients who received CYRAMZA (6%) compared to patients who received placebo (0%).
Posterior Reversible Encephalopathy Syndrome (PRES)

PRES (also known as Reversible Posterior Leukoencephalopathy Syndrome [RPLS]) has been reported in <0.1% of 2137 patients with various cancers treated with CYRAMZA. Symptoms of PRES include seizure, headache, nausea/vomiting, blindness, or altered consciousness, with or without associated hypertension.
Permanently discontinue CYRAMZA in patients who develop PRES. Symptoms may resolve or improve within days, although some patients with PRES can experience ongoing neurologic sequelae or death.
Proteinuria Including Nephrotic Syndrome

In 2137 patients with various cancers treated with CYRAMZA, the incidence of all Grade proteinuria ranged from 3-34%. Grade ≥3 proteinuria (including 4 patients with nephrotic syndrome) incidence ranged from <1-3%.
Monitor for proteinuria. Withhold CYRAMZA for urine protein levels that are 2 or more grams over 24 hours. Reinitiate CYRAMZA at a reduced dose once the urine protein level returns to less than 2 grams over 24 hours. Permanently discontinue CYRAMZA for urine protein levels greater than 3 grams over 24 hours or in the setting of nephrotic syndrome.
Thyroid Dysfunction

In 2137 patients with various cancers treated with CYRAMZA, the incidence of Grade 1-2 hypothyroidism ranged from <1-3%; there were no reports of Grade 3-5 hypothyroidism. Monitor thyroid function during treatment with CYRAMZA.
Embryo-Fetal Toxicity

CYRAMZA can cause fetal harm when administered to pregnant women. Advise pregnant women of the potential risk to a fetus. Advise females of reproductive potential to use effective contraception during treatment with CYRAMZA and for 3 months after the last dose.
Lactation

Because of the potential risk for serious adverse reactions in breastfed children from ramucirumab, advise women not to breastfeed during treatment with CYRAMZA and for 2 months after the last dose.
Adverse Reactions

REGARD:

The most common adverse reactions (all Grades) observed in single agent CYRAMZA-treated gastric cancer patients at a rate of ≥5% and ≥2% higher than placebo were hypertension (16% vs 8%), diarrhea (14% vs 9%), headache (9% vs 3%), and hyponatremia (6% vs 2%).
The most common serious adverse reactions with CYRAMZA were anemia (3.8%) and intestinal obstruction (2.1%). Red blood cell transfusions were given to 11% of CYRAMZA-treated patients vs 8.7% of patients who received placebo.
Clinically relevant adverse reactions reported in ≥1% and <5% of CYRAMZA-treated patients in REGARD were neutropenia (4.7%), epistaxis (4.7%), rash (4.2%), intestinal obstruction (2.1%), and arterial thromboembolic events (1.7%).
Across clinical trials of CYRAMZA administered as a single agent, clinically relevant adverse reactions (including Grade ≥3) reported in CYRAMZA-treated patients included proteinuria, gastrointestinal perforation, and IRR. In REGARD, according to laboratory assessment, 8% of CYRAMZA-treated patients developed proteinuria vs 3% of placebo-treated patients. Two patients discontinued CYRAMZA due to proteinuria. The rate of gastrointestinal perforation in REGARD was 0.8% and the rate of IRR was 0.4%.
RAINBOW:

The most common adverse reactions (all Grades) observed in patients treated with CYRAMZA with paclitaxel at a rate of ≥5% and ≥2% higher than placebo with paclitaxel were fatigue/asthenia (57% vs 44%), neutropenia (54% vs 31%), diarrhea (32% vs 23%), epistaxis (31% vs 7%), hypertension (25% vs 6%), peripheral edema (25% vs 14%), stomatitis (20% vs 7%), proteinuria (17% vs 6%), thrombocytopenia (13% vs 6%), hypoalbuminemia (11% vs 5%), and gastrointestinal hemorrhage events (10% vs 6%).
The most common serious adverse reactions with CYRAMZA with paclitaxel were neutropenia (3.7%) and febrile neutropenia (2.4%); 19% of patients who received CYRAMZA with paclitaxel received granulocyte colony-stimulating factors.
Adverse reactions resulting in discontinuation of any component of the CYRAMZA with paclitaxel combination in ≥2% of patients in RAINBOW were neutropenia (4%) and thrombocytopenia (3%).
Clinically relevant adverse reactions reported in ≥1% and <5% of patients receiving CYRAMZA with paclitaxel were sepsis (3.1%), including 5 fatal events, and gastrointestinal perforations (1.2%), including 1 fatal event.
REVEL:

The most common adverse reactions (all Grades) observed in patients treated with CYRAMZA with docetaxel at a rate of ≥5% and ≥2% higher than placebo with docetaxel were neutropenia (55% vs 46%), fatigue/asthenia (55% vs 50%), stomatitis/mucosal inflammation (37% vs 19%), epistaxis (19% vs 7%), febrile neutropenia (16% vs 10%), peripheral edema (16% vs 9%), thrombocytopenia (13% vs 5%), lacrimation increased (13% vs 5%), and hypertension (11% vs 5%).
The most common serious adverse reactions with CYRAMZA with docetaxel were febrile neutropenia (14%), pneumonia (6%), and neutropenia (5%). The use of granulocyte colony-stimulating factors was 42% in CYRAMZA with docetaxel- treated patients versus 37% in patients who received placebo with docetaxel.
Treatment discontinuation due to adverse reactions occurred more frequently in CYRAMZA with docetaxel-treated patients (9%) than in placebo with docetaxel-treated patients (5%). The most common adverse reactions leading to treatment discontinuation of CYRAMZA were IRR (0.5%) and epistaxis (0.3%).
For patients with non-squamous histology, the overall incidence of pulmonary hemorrhage was 7% and the incidence of Grade ≥3 pulmonary hemorrhage was 1% for CYRAMZA with docetaxel compared to 6% overall incidence and 1% for Grade ≥3 pulmonary hemorrhage for placebo with docetaxel. For patients with squamous histology, the overall incidence of pulmonary hemorrhage was 10% and the incidence of Grade ≥3 pulmonary hemorrhage was 2% for CYRAMZA with docetaxel compared to 12% overall incidence and 2% for Grade ≥3 pulmonary hemorrhage for placebo with docetaxel.
Clinically relevant adverse reactions reported in ≥1% and <5% of CYRAMZA with docetaxel-treated patients in REVEL were hyponatremia (4.8%) and proteinuria (3.3%).
RELAY:

The most common adverse reactions (all Grades) observed in patients treated with CYRAMZA with erlotinib at a rate of ≥ 5% and ≥2% higher than placebo with erlotinib were infections (81% vs 76%), diarrhea (70% vs 71%), hypertension (45% vs 12%), stomatitis (42% vs 36%), alopecia (34% vs 20%), epistaxis (34% vs 12%), proteinuria (34% vs 8%), peripheral edema (23% vs 4%), headache (15% vs 7%), gastrointestinal hemorrhage (10% vs 3%), gingival bleeding (9% vs 1%), and pulmonary hemorrhage (7% vs 2%).
The most common serious adverse reactions with CYRAMZA with erlotinib were pneumonia (3.2%), cellulitis (1.8%), and pneumothorax (1.8%). Red blood cell transfusions were given to 3.2% of CYRAMZA-treated patients versus 0 patients who received placebo.
Treatment discontinuation of all study drugs due to adverse reactions occurred in 13% of CYRAMZA with erlotinib-treated patients, with increased alanine aminotransferase (1.4%) and paronychia (1.4%) being the most common. The most common adverse reactions leading to treatment discontinuation of CYRAMZA were proteinuria (8.6%) and hyperbilirubinemia (6%).
Of the 221 patients who received CYRAMZA with erlotinib, 119 (54%) were 65 and over, while 29 (13%) were 75 and over. Adverse reactions occurring at a 10% or higher incidence in patients receiving CYRAMZA with erlotinib and with a 10% or greater difference between patients aged 65 or older compared to patients aged less than 65 years were: diarrhea (75% versus 65%), hypertension (50% versus 40%), increased ALT (49% versus 35%), increased AST (49% versus 33%), stomatitis (46% versus 36%), decreased appetite (32% versus 19%), dysgeusia (23% versus 12%), and weight loss (19% versus 6%).
RAISE:

The most common adverse reactions (all Grades) observed in patients treated with CYRAMZA with FOLFIRI at a rate of ≥5% and ≥2% higher than placebo with FOLFIRI were diarrhea (60% vs 51%), neutropenia (59% vs 46%), decreased appetite (37% vs 27%), epistaxis (33% vs 15%), stomatitis (31% vs 21%), thrombocytopenia (28% vs 14%), hypertension (26% vs 9%), peripheral edema (20% vs 9%), proteinuria (17% vs 5%), palmar-plantar erythrodysesthesia syndrome (13% vs 5%), gastrointestinal hemorrhage events (12% vs 7%), and hypoalbuminemia (6% vs 2%). Twenty percent of patients treated with CYRAMZA with FOLFIRI received granulocyte colony- stimulating factors.
The most common serious adverse reactions with CYRAMZA with FOLFIRI were diarrhea (3.6%), intestinal obstruction (3.0%), and febrile neutropenia (2.8%).
Treatment discontinuation of any study drug due to adverse reactions occurred more frequently in CYRAMZA with FOLFIRI-treated patients (29%) than in placebo with FOLFIRI-treated patients (13%). The most common adverse reactions leading to discontinuation of any component of CYRAMZA with FOLFIRI as compared to placebo with FOLFIRI were neutropenia (12.5% vs 5.3%) and thrombocytopenia (4.2% vs 0.8%). The most common adverse reactions leading to treatment discontinuation of CYRAMZA were proteinuria (1.5%), and gastrointestinal perforation (1.7%).
Clinically relevant adverse reaction reported in ≥1% and <5% of patients receiving CYRAMZA with FOLFIRI was gastrointestinal perforation (1.7%), including 4 fatal events.
Thyroid-stimulating hormone (TSH) levels were evaluated in 224 patients (115 CYRAMZA with FOLFIRI-treated patients and 109 placebo with FOLFIRI-treated patients) with normal baseline TSH levels. Increased TSH levels were observed in 53 (46%) patients treated with CYRAMZA with FOLFIRI compared with 4 (4%) patients treated with placebo with FOLFIRI.
REACH-2:

The most common adverse reactions (all Grades) observed in single agent CYRAMZA-treated HCC patients at a rate of ≥10% and ≥2% higher than placebo were fatigue (36% vs 20%), peripheral edema (25% vs 14%), hypertension (25% vs 13%), abdominal pain (25% vs 16%), decreased appetite (23% vs 20%), proteinuria (20% vs 4%), nausea (19% vs 12%), ascites (18% vs 7%), headache (14% vs 5%), epistaxis (14% vs 3%), insomnia (11% vs 6%), pyrexia (10% vs 3%), vomiting (10% vs 7%), and back pain (10% vs 7%).
The most common serious adverse reactions with CYRAMZA were ascites (3%) and pneumonia (3%).
Treatment discontinuations due to adverse reactions occurred in 18% of CYRAMZA-treated patients, with proteinuria being the most frequent (2%).
Clinically relevant adverse reactions reported in ≥1% and <10% of CYRAMZA-treated patients in REACH-2 were IRR (9%), hepatic encephalopathy (5%) including 1 fatal event, and hepatorenal syndrome (2%) including 1 fatal event.
RB-P HCP ISI 29MAY2020

Please see full U.S. Prescribing Information for CYRAMZA.

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EORTC 1325-MG/Keynote 054: Pembrolizumab versus placebo after complete resection of high-risk stage III melanoma: New recurrence-free survival results at three-year median follow-up

On May 29, 2020 EORTC reported that Eggermont presented the updated efficacy results of the phase III double-blind EORTC 1325/KEYNOTE-054 trial to evaluate pembrolizumab vs placebo in patients with resected high-risk stage III melanoma (Press release, EORTC, MAY 29, 2020, View Source [SID1234558741]).

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Patients with stage III melanoma usually undergo surgery to remove the primary melanoma and the nearby metastasized lymph nodes. However, successful removal of melanoma through surgery, does not guarantee that patients will be cancer free. Pembrolizumab, an immune checkpoint inhibitor antibody, has been shown to stimulate the body’s immune system to remove remaining melanoma cells. After recovery from the lymph node surgery, the phase III double-blind EORTC 1325/KEYNOTE-054 study compared the administration of pembrolizumab and placebo, for a maximum of year. In 2018, at a 1.25-year median follow-up, this study showed that pembrolizumab treatment yielded a 43% reduction of their risk of melanoma recurrence or death as compared to placebo treatment, i.e. prolonging recurrence-free survival. These findings led to the approval of pembrolizumab for resected high-risk stage III melanoma patients by EMA in 2018 and by FDA in 2019.

In this study, 1019 patients were randomized to pembrolizumab (514 patients) or placebo (505 patients). A dose of 200 mg was given every three weeks for over a year or until there was disease recurrence or unacceptable toxicity. Two primary endpoints were assessed, recurrence free survival from randomization in the overall population and those patients with PD-L1 positive tumours. This abstract presents, a new, up-to-date evaluation of recurrence free survival at a 3-year median follow up.

Professor Eggermont showed that, adjuvant administration of pembrolizumab provided a sustained prolongation of recurrence free survival compared to placebo. In the overall population, the 3-year recurrence-free survival (RFS) rate was 64% (pembrolizumab group) vs 44% (placebo group). The improvement was consistent across all tumour and stage subgroups. Importantly, in BRAFmutant patients the RFS rate difference was 25% (62% vs 37%) and in BRAFwild type patients it was 16% (62%-46%), demonstrating a significant benefit irrespective of mutational status In the overall population, the 3-year cumulative incidence of distant metastasis as the first recurrence was 22% (pembrolizumab group) vs 37% (placebo group). These results confirm the clinical utility of pembrolizumab in the adjuvant setting in resected high-risk stage III melanoma.

"Pembrolizumab adjuvant therapy provides a clear and sustained relapse free survival benefit to stage III melanoma patients irrespective of stage subgroup or mutational status. We expect these benefits to be also consistent across endpoints at long term follow up" said Professor Alexander M.M. Eggermont, study chair of this study, Chief Scientific Officer and Professor of Surgical Oncology at Princess Máxima Center, Utrecht, The Netherlands

Research Funding: Merck
Session Type: Oral Abstract Session
Session Title: Melanoma/Skin Cancers
Track: Melanoma/Skin Cancers
Subtrack: Other
Abstract #: 10000
Clinical Trial Registry Number: NCT02362594
Citation: J Clin Oncol 38: 2020 (suppl; abstr 10000)
DOI: 10.1200/JCO.2020.38.15_suppl.10000