Eisai Demonstrates Commitment to Oncology Innovation at ASCO 2025

On May 20, 2025 Eisai reported the presentation of clinical research across its oncology portfolio and pipeline during the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting (#ASCO25), which is taking place in Chicago, Illinois from May 30 to June 3 (Press release, Eisai, MAY 20, 2025, View Source [SID1234653245]).

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Notable data include findings from the Phase 3 LEAP-002 study, which evaluated lenvatinib (LENVIMA), the orally available multiple receptor tyrosine kinase inhibitor (TKI) discovered by Eisai, plus pembrolizumab (KEYTRUDA), Merck’s anti-PD-1 therapy, versus lenvatinib monotherapy for the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC). The poster presentation will feature long-term follow-up data providing further insights into lenvatinib’s role in the treatment landscape for patients affected by HCC (NCT03713593; Abstract #4095).

Additional research from Eisai’s pipeline will focus on E7386, a CBP/β-catenin interaction inhibitor, in combination with lenvatinib. This includes a dose optimization trial-in-progress presentation (Abstract #TPS5632) and dose expansion findings (Abstract #5599) in patients with advanced or recurrent endometrial carcinoma (NCT04008797).

"At Eisai, our pursuit of scientific advancement is fueled by a deep commitment to our human health care philosophy. We believe patients deserve our best efforts, and we endeavor to deliver that by pushing boundaries in oncology research, particularly in challenging areas," said Dr. Corina Dutcus, Senior Vice President, Oncology Global Clinical Development Lead at Eisai Inc. "Our data at ASCO (Free ASCO Whitepaper) 2025 showcase this principle in action. The long-term follow-up data from LEAP-002 contribute to our ongoing body of research and further reinforce our understanding of LENVIMA’s established role in unresectable hepatocellular carcinoma, while our pipeline work in advanced endometrial carcinoma represents our continued dedication to addressing areas with unmet medical needs through innovative therapeutic approaches."

An oral presentation will feature data from the final analysis of the Phase 3 LEAP-015 study evaluating lenvatinib plus pembrolizumab and chemotherapy versus chemotherapy in patients with advanced, metastatic gastroesophageal adenocarcinoma (NCT04662710; Abstract #4001).

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

The full list of Eisai presentations is included below. These abstracts will be made available via the ASCO (Free ASCO Whitepaper) website on Thursday, May 22, 2025, at 5:00 PM EDT.

Cancer Type

Study/Compound

Abstract Title

Abstract Type & Details

Lenvatinib Plus Pembrolizumab

Gastrointestinal Cancer

LEAP-002

LEAP-002 long-term follow-up: Lenvatinib
plus pembrolizumab versus lenvatinib plus placebo for advanced hepatocellular carcinoma

Poster Session

Abstract #4095

May 31, 2025

10:00 AM EDT /

9:00 AM CDT

LEAP-015

Lenvatinib plus pembrolizumab and chemotherapy versus chemotherapy in advanced, metastatic gastroesophageal adenocarcinoma: The Phase 3, randomized LEAP-015 study

Oral Abstract Session

Abstract #4001

May 31, 2025

4:12 PM EDT /

3:12 PM CDT

Melanoma

LEAP-003

First-line lenvatinib plus pembrolizumab
versus placebo plus pembrolizumab in Chinese patients with unresectable or metastatic melanoma: results from LEAP-
003

Poster Session

Abstract #9553

June 1, 2025

10:00 AM EDT /

9:00 AM CDT

Pipeline

Gynecologic Cancer

E7386

Randomized study evaluating optimal dose,
efficacy and safety of E7386 + lenvatinib
versus treatment of physicians’ choice in
advanced/recurrent endometrial carcinoma
previously treated with anti-PD-(L)1 immunotherapy

Poster Session

Abstract #TPS5632

June 1, 2025

10:00 AM EDT /

9:00 AM CDT

E7386 Study 102: Global dose-expansion
cohort of E7386 + lenvatinib (LEN) in
patients (pts) with advanced endometrial
cancer (aEC) that progressed on platinum-
based chemotherapy (chemo) and an anti-
PD-(L)1 immunotherapy (IO)

Poster Session

Abstract #5599

June 1, 2025

10:00 AM EDT /

9:00 AM CDT

Pan-Tumor

Systematic Review

The PRO-CTCAE in oncology clinical trials:
Insights from a targeted literature review

For Online Publication

In March 2018, Eisai and Merck (known as MSD outside of the United States and Canada), through an affiliate, entered into a strategic collaboration for the worldwide co-development and co-commercialization of lenvatinib, both as monotherapy and in combination with Merck’s anti-PD-1 therapy, pembrolizumab. Eisai and Merck are studying the lenvatinib plus pembrolizumab combination through the LEAP (LEnvatinib And Pembrolizumab) clinical program in HCC and esophageal cancer across multiple clinical trials.

About E7386
E7386 is thought to block the protein-protein interaction between a transcriptional co-activator, cAMP response element-binding protein (CREB) binding protein (CBP) and β-catenin, resulting in the inhibition of Wnt / β-catenin pathway-dependent gene expression. Since E7386 is thought to act on the CBP / β-catenin transcription complex located at the most downstream of the Wnt signaling, it is expected to inhibit not only ligand-dependent activation but also activation caused by gene mutations in Wnt signaling factors such as adenomatous polyposis coli (APC) and β-catenin. E7386 is created through collaboration research between Eisai and PRISM BioLab Co., Ltd. (Headquarters: Kanagawa)

About LENVIMA (lenvatinib) Capsules

LENVIMA is indicated:

For the treatment of adult patients with locally recurrent or metastatic, progressive, radioactive iodine-refractory differentiated thyroid cancer (DTC)
In combination with pembrolizumab, for the first line treatment of adult patients with advanced renal cell carcinoma (RCC)
In combination with everolimus for the treatment of adult patients with advanced renal cell carcinoma (RCC) following one prior anti-angiogenic therapy
For the first-line treatment of patients with unresectable hepatocellular carcinoma (HCC)
In combination with pembrolizumab, for the treatment of patients with advanced endometrial carcinoma (EC) that is mismatch repair proficient (pMMR) or not microsatellite instability-high (MSI-H), as determined by an FDA-approved test, who have disease progression following prior systemic therapy in any setting and are not candidates for curative surgery or radiation.
LENVIMA, discovered and developed by Eisai, is a multiple receptor tyrosine 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 (PDGFRA), KIT, and RET. Lenvatinib also exhibited antiproliferative activity in hepatocellular carcinoma cell lines dependent on activated FGFR signaling with a concurrent inhibition of FGF-receptor substrate 2 alpha (FRS2) phosphorylation. In syngeneic mouse tumor models, the combination of lenvatinib with an anti-PD-1 monoclonal antibody decreased tumor-associated macrophages, increased activated cytotoxic T cells, and demonstrated greater antitumor activity compared to either treatment alone. The combination of LENVIMA and everolimus showed increased antiangiogenic and antitumor activity as demonstrated by decreased human endothelial cell proliferation, tube formation, and VEGF signaling in vitro and tumor volume in mouse xenograft models of human renal cell cancer greater than each drug alone.

Important Safety Information for LENVIMA

Warnings and Precautions

Hypertension. In DTC (differentiated thyroid cancer), hypertension occurred in 73% of patients on LENVIMA (44% grade 3-4). In RCC (renal cell carcinoma), hypertension occurred in 42% of patients on LENVIMA + everolimus (13% grade 3). Systolic blood pressure ≥160 mmHg occurred in 29% of patients, and 21% had diastolic blood pressure ≥100 mmHg. In HCC (hepatocellular carcinoma), hypertension occurred in 45% of LENVIMA-treated patients (24% grade 3). Grade 4 hypertension was not reported in HCC.

Serious complications of poorly controlled hypertension have been reported. Control blood pressure prior to initiation. Monitor blood pressure after 1 week, then every 2 weeks for the first 2 months, and then at least monthly thereafter during treatment. Withhold and resume at reduced dose when hypertension is controlled or permanently discontinue based on severity.

Cardiac Dysfunction. Serious and fatal cardiac dysfunction can occur with LENVIMA. Across clinical trials in 799 patients with DTC, RCC, and HCC, grade 3 or higher cardiac dysfunction occurred in 3% of LENVIMA-treated patients. Monitor for clinical symptoms or signs of cardiac dysfunction. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Arterial Thromboembolic Events. Among patients receiving LENVIMA or LENVIMA + everolimus, arterial thromboembolic events of any severity occurred in 2% of patients in RCC and HCC and 5% in DTC. Grade 3-5 arterial thromboembolic events ranged from 2% to 3% across all clinical trials.

Among patients receiving LENVIMA with pembrolizumab, arterial thrombotic events of any severity occurred in 5% of patients in CLEAR, including myocardial infarction (3.4%) and cerebrovascular accident (2.3%).

Permanently discontinue following an arterial thrombotic event. The safety of resuming after an arterial thromboembolic event has not been established, and LENVIMA has not been studied in patients who have had an arterial thromboembolic event within the previous 6 months.

Hepatotoxicity. Across clinical studies enrolling 1327 LENVIMA-treated patients with malignancies other than HCC, serious hepatic adverse reactions occurred in 1.4% of patients. Fatal events, including hepatic failure, acute hepatitis, and hepatorenal syndrome, occurred in 0.5% of patients. In HCC, hepatic encephalopathy occurred in 8% of LENVIMA-treated patients (5% grade 3-5). Grade 3-5 hepatic failure occurred in 3% of LENVIMA-treated patients; 2% of patients discontinued LENVIMA due to hepatic encephalopathy, and 1% discontinued due to hepatic failure.

Monitor liver function prior to initiation, then every 2 weeks for the first 2 months, and at least monthly thereafter during treatment. Monitor patients with HCC closely for signs of hepatic failure, including hepatic encephalopathy. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Renal Failure or Impairment. Serious including fatal renal failure or impairment can occur with LENVIMA. Renal impairment was reported in 14% and 7% of LENVIMA-treated patients in DTC and HCC, respectively. Grade 3-5 renal failure or impairment occurred in 3% of patients with DTC and 2% of patients with HCC, including 1 fatal event in each study. In RCC, renal impairment or renal failure was reported in 18% of LENVIMA + everolimus–treated patients (10% grade 3).

Initiate prompt management of diarrhea or dehydration/hypovolemia. Withhold and resume at reduced dose upon recovery or permanently discontinue for renal failure or impairment based on severity.

Proteinuria. In DTC and HCC, proteinuria was reported in 34% and 26% of LENVIMA-treated patients, respectively. Grade 3 proteinuria occurred in 11% and 6% in DTC and HCC, respectively. In RCC, proteinuria occurred in 31% of patients receiving LENVIMA + everolimus (8% grade 3). Monitor for proteinuria prior to initiation and periodically during treatment. If urine dipstick proteinuria ≥2+ is detected, obtain a 24-hour urine protein. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Diarrhea. Of the 737 LENVIMA-treated patients in DTC and HCC, diarrhea occurred in 49% (6% grade 3). In RCC, diarrhea occurred in 81% of LENVIMA + everolimus–treated patients (19% grade 3). Diarrhea was the most frequent cause of dose interruption/reduction, and diarrhea recurred despite dose reduction. Promptly initiate management of diarrhea. Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Fistula Formation and Gastrointestinal Perforation. Of the 799 patients treated with LENVIMA or LENVIMA + everolimus in DTC, RCC, and HCC, fistula or gastrointestinal perforation occurred in 2%. Permanently discontinue in patients who develop gastrointestinal perforation of any severity or grade 3-4 fistula.

QT Interval Prolongation. In DTC, QT/QTc interval prolongation occurred in 9% of LENVIMA-treated patients and QT interval prolongation of >500 ms occurred in 2%. In RCC, QTc interval increases of >60 ms occurred in 11% of patients receiving LENVIMA + everolimus and QTc interval >500 ms occurred in 6%. In HCC, QTc interval increases of >60 ms occurred in 8% of LENVIMA-treated patients and QTc interval >500 ms occurred in 2%.

Monitor and correct electrolyte abnormalities at baseline and periodically during treatment. Monitor electrocardiograms in patients with congenital long QT syndrome, congestive heart failure, bradyarrhythmias, or those who are taking drugs known to prolong the QT interval, including Class Ia and III antiarrhythmics. Withhold and resume at reduced dose upon recovery based on severity.

Hypocalcemia. In DTC, grade 3-4 hypocalcemia occurred in 9% of LENVIMA-treated patients. In 65% of cases, hypocalcemia improved or resolved following calcium supplementation with or without dose interruption or dose reduction. In RCC, grade 3-4 hypocalcemia occurred in 6% of LENVIMA + everolimus–treated patients. In HCC, grade 3 hypocalcemia occurred in 0.8% of LENVIMA-treated patients. Monitor blood calcium levels at least monthly and replace calcium as necessary during treatment. Withhold and resume at reduced dose upon recovery or permanently discontinue depending on severity.

Reversible Posterior Leukoencephalopathy Syndrome (RPLS). Across clinical studies of 1823 patients who received LENVIMA as a single agent, RPLS occurred in 0.3%. Confirm diagnosis of RPLS with MRI. Withhold and resume at reduced dose upon recovery or permanently discontinue depending on severity and persistence of neurologic symptoms.

Hemorrhagic Events. Serious including fatal hemorrhagic events can occur with LENVIMA. In DTC, RCC, and HCC clinical trials, hemorrhagic events, of any grade, occurred in 29% of the 799 patients treated with LENVIMA as a single agent or in combination with everolimus. The most frequently reported hemorrhagic events (all grades and occurring in at least 5% of patients) were epistaxis and hematuria. In DTC, grade 3-5 hemorrhage occurred in 2% of LENVIMA-treated patients, including 1 fatal intracranial hemorrhage among 16 patients who received LENVIMA and had CNS metastases at baseline. In RCC, grade 3-5 hemorrhage occurred in 8% of LENVIMA + everolimus–treated patients, including 1 fatal cerebral hemorrhage. In HCC, grade 3-5 hemorrhage occurred in 5% of LENVIMA-treated patients, including 7 fatal hemorrhagic events. Serious tumor-related bleeds, including fatal hemorrhagic events, occurred in LENVIMA-treated patients in clinical trials and in the postmarketing setting. In postmarketing surveillance, serious and fatal carotid artery hemorrhages were seen more frequently in patients with anaplastic thyroid carcinoma (ATC) than other tumors. Safety and effectiveness of LENVIMA in patients with ATC have not been demonstrated in clinical trials.

Consider the risk of severe or fatal hemorrhage associated with tumor invasion or infiltration of major blood vessels (eg, carotid artery). Withhold and resume at reduced dose upon recovery or permanently discontinue based on severity.

Impairment of Thyroid Stimulating Hormone Suppression/Thyroid Dysfunction. LENVIMA impairs exogenous thyroid suppression. In DTC, 88% of patients had baseline thyroid stimulating hormone (TSH) level ≤0.5 mU/L. In patients with normal TSH at baseline, elevation of TSH level >0.5 mU/L was observed post baseline in 57% of LENVIMA-treated patients. In RCC and HCC, grade 1 or 2 hypothyroidism occurred in 24% of LENVIMA + everolimus–treated patients and 21% of LENVIMA-treated patients, respectively. In patients with normal or low TSH at baseline, elevation of TSH was observed post baseline in 70% of LENVIMA-treated patients in HCC and 60% of LENVIMA + everolimus–treated patients in RCC.

Monitor thyroid function prior to initiation and at least monthly during treatment. Treat hypothyroidism according to standard medical practice.

Impaired Wound Healing. Impaired wound healing has been reported in patients who received LENVIMA. Withhold LENVIMA for at least 1 week prior to elective surgery. Do not administer for at least 2 weeks following major surgery and until adequate wound healing. The safety of resumption of LENVIMA after resolution of wound healing complications has not been established.

Osteonecrosis of the Jaw (ONJ). ONJ has been reported in patients receiving LENVIMA. Concomitant exposure to other risk factors, such as bisphosphonates, denosumab, dental disease, or invasive dental procedures, may increase the risk of ONJ.

Perform an oral examination prior to treatment with LENVIMA and periodically during LENVIMA treatment. Advise patients regarding good oral hygiene practices and to consider having preventive dentistry performed prior to treatment with LENVIMA and throughout treatment with LENVIMA.

Avoid invasive dental procedures, if possible, while on LENVIMA treatment, particularly in patients at higher risk. Withhold LENVIMA for at least 1 week prior to scheduled dental surgery or invasive dental procedures, if possible. For patients requiring invasive dental procedures, discontinuation of bisphosphonate treatment may reduce the risk of ONJ.

Withhold LENVIMA if ONJ develops and restart based on clinical judgement of adequate resolution.

Embryo-Fetal Toxicity. Based on its mechanism of action and data from animal reproduction studies, LENVIMA can cause fetal harm when administered to pregnant women. In animal reproduction studies, oral administration of lenvatinib during organogenesis at doses below the recommended clinical doses resulted in embryotoxicity, fetotoxicity, and teratogenicity in rats and rabbits. Advise pregnant women of the potential risk to a fetus and advise females of reproductive potential to use effective contraception during treatment with LENVIMA and for 30 days after the last dose.

Adverse Reactions
In DTC, the most common adverse reactions (≥30%) observed in LENVIMA-treated patients were hypertension (73%), fatigue (67%), diarrhea (67%), arthralgia/myalgia (62%), decreased appetite (54%), decreased weight (51%), nausea (47%), stomatitis (41%), headache (38%), vomiting (36%), proteinuria (34%), palmar-plantar erythrodysesthesia syndrome (32%), abdominal pain (31%), and dysphonia (31%). The most common serious adverse reactions (≥2%) were pneumonia (4%), hypertension (3%), and dehydration (3%). Adverse reactions led to dose reductions in 68% of LENVIMA-treated patients; 18% discontinued LENVIMA. The most common adverse reactions (≥10%) resulting in dose reductions were hypertension (13%), proteinuria (11%), decreased appetite (10%), and diarrhea (10%); the most common adverse reactions (≥1%) resulting in discontinuation of LENVIMA were hypertension (1%) and asthenia (1%).

In RCC, the most common adverse reactions (≥20%) observed in LENVIMA + pembrolizumab-treated patients were fatigue (63%), diarrhea (62%), musculoskeletal pain (58%), hypothyroidism (57%), hypertension (56%), stomatitis (43%), decreased appetite (41%), rash (37%), nausea (36%), decreased weight (30%), dysphonia (30%), proteinuria (30%), palmar-plantar erythrodysesthesia syndrome (29%), abdominal pain (27%), hemorrhagic events (27%), vomiting (26%), constipation (25%), hepatotoxicity (25%), headache (23%), and acute kidney injury (21%). The most common serious adverse reactions (≥2%) were hemorrhagic events (5%), diarrhea (4%), hypertension (3%), myocardial infarction (3%), pneumonitis (3%), vomiting (3%), acute kidney injury (2%), adrenal insufficiency (2%), dyspnea (2%), and pneumonia (2%). Fatal adverse reactions occurred in 4.3% of patients receiving LENVIMA in combination with pembrolizumab, including cardio-respiratory arrest (0.9%), sepsis (0.9%), and one case (0.3%) each of arrhythmia, autoimmune hepatitis, dyspnea, hypertensive crisis, increased blood creatinine, multiple organ dysfunction syndrome, myasthenic syndrome, myocarditis, nephritis, pneumonitis, ruptured aneurysm and subarachnoid hemorrhage. Serious adverse reactions occurred in 51% of patients receiving LENVIMA and pembrolizumab. Serious adverse reactions in ≥2% of patients were hemorrhagic events (5%), diarrhea (4%), hypertension (3%), myocardial infarction (3%), pneumonitis (3%), vomiting (3%), acute kidney injury (2%), adrenal insufficiency (2%), dyspnea (2%), and pneumonia (2%). Permanent discontinuation of LENVIMA, pembrolizumab, or both due to an adverse reaction occurred in 37% of patients; 26% LENVIMA only, 29% pembrolizumab only, and 13% both drugs. The most common adverse reactions (≥2%) leading to permanent discontinuation of LENVIMA, pembrolizumab, or both were pneumonitis (3%), myocardial infarction (3%), hepatotoxicity (3%), acute kidney injury (3%), rash (3%), and diarrhea (2%). Dose interruptions of LENVIMA, pembrolizumab, or both due to an adverse reaction occurred in 78% of patients receiving LENVIMA in combination with pembrolizumab. LENVIMA was interrupted in 73% of patients and both drugs were interrupted in 39% of patients. LENVIMA was dose reduced in 69% of patients. The most common adverse reactions (≥5%) resulting in dose reduction or interruption of LENVIMA were diarrhea (26%), fatigue (18%), hypertension (17%), proteinuria (13%), decreased appetite (12%), palmar-plantar erythrodysesthesia (11%), nausea (9%), stomatitis (9%), musculoskeletal pain (8%), rash (8%), increased lipase (7%), abdominal pain (6%), vomiting (6%), increased ALT (5%), and increased amylase (5%).

In RCC, the most common adverse reactions (≥30%) observed in LENVIMA + everolimus–treated patients were diarrhea (81%), fatigue (73%), arthralgia/myalgia (55%), decreased appetite (53%), vomiting (48%), nausea (45%), stomatitis (44%), hypertension (42%), peripheral edema (42%), cough (37%), abdominal pain (37%), dyspnea (35%), rash (35%), decreased weight (34%), hemorrhagic events (32%), and proteinuria (31%). The most common serious adverse reactions (≥5%) were renal failure (11%), dehydration (10%), anemia (6%), thrombocytopenia (5%), diarrhea (5%), vomiting (5%), and dyspnea (5%). Adverse reactions led to dose reductions or interruption in 89% of patients. The most common adverse reactions (≥5%) resulting in dose reductions were diarrhea (21%), fatigue (8%), thrombocytopenia (6%), vomiting (6%), nausea (5%), and proteinuria (5%). Treatment discontinuation due to an adverse reaction occurred in 29% of patients.

In HCC, the most common adverse reactions (≥20%) observed in LENVIMA-treated patients were hypertension (45%), fatigue (44%), diarrhea (39%), decreased appetite (34%), arthralgia/myalgia (31%), decreased weight (31%), abdominal pain (30%), palmar-plantar erythrodysesthesia syndrome (27%), proteinuria (26%), dysphonia (24%), hemorrhagic events (23%), hypothyroidism (21%), and nausea (20%). The most common serious adverse reactions (≥2%) were hepatic encephalopathy (5%), hepatic failure (3%), ascites (3%), and decreased appetite (2%). Adverse reactions led to dose reductions or interruption in 62% of patients. The most common adverse reactions (≥5%) resulting in dose reductions were fatigue (9%), decreased appetite (8%), diarrhea (8%), proteinuria (7%), hypertension (6%), and palmar-plantar erythrodysesthesia syndrome (5%). Treatment discontinuation due to an adverse reaction occurred in 20% of patients. The most common adverse reactions (≥1%) resulting in discontinuation of LENVIMA were fatigue (1%), hepatic encephalopathy (2%), hyperbilirubinemia (1%), and hepatic failure (1%).

In EC, the most common adverse reactions (≥20%) observed in LENVIMA and pembrolizumab–treated patients were hypothyroidism (67%), hypertension (67%), fatigue (58%), diarrhea (55%), musculoskeletal disorders (53%), nausea (49%), decreased appetite (44%), vomiting (37%), stomatitis (35%), decreased weight (34%), abdominal pain (34%), urinary tract infection (31%), proteinuria (29%), constipation (27%), headache (26%), hemorrhagic events (25%), palmar‐plantar erythrodysesthesia (23%), dysphonia (22%), and rash (20%). Fatal adverse reactions occurred in 4.7% of those treated with LENVIMA and pembrolizumab, including 2 cases of pneumonia, and 1 case of the following: acute kidney injury, acute myocardial infarction, colitis, decreased appetite, intestinal perforation, lower gastrointestinal hemorrhage, malignant gastrointestinal obstruction, multiple organ dysfunction syndrome, myelodysplastic syndrome, pulmonary embolism, and right ventricular dysfunction. Serious adverse reactions occurred in 50% of patients receiving LENVIMA and pembrolizumab. Serious adverse reactions with frequency ≥3% were hypertension (4.4%), and urinary tract infection (3.2%). Discontinuation of LENVIMA due to an adverse reaction occurred in 26% of patients. The most common (≥1%) adverse reactions leading to discontinuation of LENVIMA were hypertension (2%), asthenia (1.8%), diarrhea (1.2%), decreased appetite (1.2%), proteinuria (1.2%), and vomiting (1.2%). Dose reductions of LENVIMA due to adverse reactions occurred in 67% of patients. The most common (≥5%) adverse reactions resulting in dose reduction of LENVIMA were hypertension (18%), diarrhea (11%), palmar-plantar erythrodysesthesia syndrome (9%), proteinuria (7%), fatigue (7%), decreased appetite (6%), asthenia (5%), and weight decreased (5%). Dose interruptions of LENVIMA due to an adverse reaction occurred in 58% of these patients. The most common (≥2%) adverse reactions leading to interruption of LENVIMA were hypertension (11%), diarrhea (11%), proteinuria (6%), decreased appetite (5%), vomiting (5%), increased alanine aminotransferase (3.5%), fatigue (3.5%), nausea (3.5%), abdominal pain (2.9%), weight decreased (2.6%), urinary tract infection (2.6%), increased aspartate aminotransferase (2.3%), asthenia (2.3%), and palmar-plantar erythrodysesthesia (2%).

Use in Specific Populations
Because of the potential for serious adverse reactions in breastfed children, advise women to discontinue breastfeeding during treatment and for 1 week after the last dose. LENVIMA may impair fertility in males and females of reproductive potential.

No dose adjustment is recommended for patients with mild (CLcr 60-89 mL/min) or moderate (CLcr 30-59 mL/min) renal impairment. LENVIMA concentrations may increase in patients with DTC, RCC, or EC and severe (CLcr 15-29 mL/min) renal impairment. Reduce the dose for patients with DTC, RCC, or EC and severe renal impairment. There is no recommended dose for patients with HCC and severe renal impairment. LENVIMA has not been studied in patients with end-stage renal disease.

No dose adjustment is recommended for patients with HCC and mild hepatic impairment (Child-Pugh A). There is no recommended dose for patients with HCC with moderate (Child-Pugh B) or severe (Child-Pugh C) hepatic impairment. No dose adjustment is recommended for patients with DTC, RCC, or EC and mild or moderate hepatic impairment. LENVIMA concentrations may increase in patients with DTC, RCC, or EC and severe hepatic impairment. Reduce the dose for patients with DTC, RCC, or EC and severe hepatic impairment.

LENVIMA (lenvatinib) is available as 10 mg and 4 mg capsules.

Aptose Announces Dosing of First Patient with 120 mg of Tuspetinib in Phase 1/2 Tuscany Trial of Frontline Triple Drug Therapy after Dose Escalation Decision by Safety Review Committee

On May 20, 2025 Aptose Biosciences Inc. ("Aptose" or the "Company") (OTC: APTOF, TSX: APS), a clinical-stage precision oncology company developing the tuspetinib (TUS)-based triple drug frontline therapy to treat patients with newly diagnosed AML, reported that the Cohort Safety Review Committee (CSRC) monitoring Aptose’s Phase 1/2 TUSCANY trial of tuspetinib in combination with standard of care dosing of venetoclax and azacitidine (TUS+VEN+AZA triplet) has approved escalating from 80 mg dose TUS to 120 mg dose TUS based on its favorable review of safety and efficacy data from patients in the first two cohorts of the trial (Press release, Aptose Biosciences, MAY 20, 2025, View Source [SID1234653244]). Dosing of the first subject at the 120 mg TUS dose level has commenced. The TUS+VEN+AZA triplet is being developed as a one-of-a-kind, safe and mutation agnostic frontline therapy to treat large, mutationally diverse populations of newly diagnosed AML patients who are ineligible to receive induction chemotherapy.

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No significant safety concerns or dose limiting toxicities (DLTs) have been reported in the TUSCANY trial, including no prolonged myelosuppression of subjects in remission. Patients treated in the 40 mg and 80 mg dose cohorts remain on study while enrollment is open for the 120 mg dose cohort. Aptose has reported that the first two dose cohorts have demonstrated safety, CRs, and minimal residual disease (MRD) negativity across patients with diverse mutations (press release here). The Company will be presenting updated data in an oral presentation at the European Hematology Association (EHA) (Free EHA Whitepaper) Congress (EHA 2025), being held June 12-15, 2025, in Milan, Italy, which will include updated safety, CRs, MRD, and pharmacokinetic (PK) clinical findings and longer duration of follow up.

"Data from the first two cohorts, with a 40 mg or 80 mg dose of TUS in the TUS+VEN+AZA triplet, reveal promising clinical safety and antileukemic activity even in some of the most difficult-to-treat AML populations," said Rafael Bejar, M.D., Ph.D., Chief Medical Officer of Aptose. "With these significant findings, our CSRC – comprised of key leaders in the development of therapeutic agents for AML – recommended we dose escalate further, and we have now opened the 120 mg dose cohort of TUS in the triplet therapy."

TUSCANY: TUS+VEN+AZA Triplet Phase 1/2 Study

The tuspetinib-based TUS+VEN+AZA triplet therapy is being advanced in the TUSCANY Phase 1/2 trial with the goal of creating an improved frontline therapy for newly diagnosed AML patients that is active across diverse AML populations, durable, and well tolerated. Earlier APTIVATE trials of TUS as a single agent and in combination as TUS+VEN demonstrated favorable safety and broad activity in diverse relapsed or refractory (R/R) AML populations that went beyond the more prognostically favorable NPM1 and IDH mutant subgroups. Indeed, responses were also in R/R AML patients with highly adverse TP53 and RAS mutations, and those with mutated or unmutated (wildtype) FLT3 genes.

The TUSCANY triplet Phase 1/2 study, being conducted at 10 leading U.S. clinical sites by elite clinical investigators, is designed to test various doses and schedules of TUS in combination with standard dosing of AZA and VEN for patients with AML who are ineligible to receive induction chemotherapy. A convenient, once daily oral agent, TUS is being administered in 28-day cycles. Multiple U.S. sites are enrolling in the TUSCANY trial with anticipated enrollment of 18-24 patients by mid-late 2025. Data will be released as it becomes available.

More information on the TUSCANY Phase 1/2 study can be found on www.clinicaltrials.gov (here).

ALX Oncology Highlights Differentiated Design, Preclinical Data and Development Plans for EGFR-Targeted ADC, ALX2004, in R&D Webcast Event

On May 20, 2025 ALX Oncology Holdings Inc., ("ALX Oncology" or the "Company") (Nasdaq: ALXO), a clinical-stage biotechnology company advancing a pipeline of novel therapies designed to treat cancer and extend patients’ lives, reported an overview of the clinical development program for its highly-differentiated antibody-drug conjugate (ADC), ALX2004, at the Company’s R&D webcast event today (Press release, ALX Oncology, MAY 20, 2025, View Source [SID1234653243]). ALX Oncology leadership will discuss the unique design profile, preclinical data and clinical development plans for ALX2004, a potential best- and first-in-class ADC for the treatment of epidermal growth factor receptor (EGFR)-expressing solid tumors, that is planned to enter Phase 1 studies in mid-year 2025.

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"ALX Oncology is driving a new, highly differentiated approach to the treatment of EGFR-expressing tumors, harnessing our proprietary linker-payload platform to bring forward an ADC candidate that we believe may be able to overcome challenges seen with EGFR-ADCs in the past and drive a best-in-class molecule forward," said Jason Lettmann, Chief Executive Officer at ALX Oncology. "As we advance ALX2004 into the clinic, we are excited to share the development program and highlight how we rigorously designed all components of this ADC with an optimized, validated payload and antibody to maximize its potential for success across a variety of tumor types."

The Company’s ALX2004 R&D webcast event will include presentations on:

ALX2004 Unique Design: Optimized for Anti-Tumor Activity and Improved Outcomes

Developed by ALX Oncology’s protein engineers utilizing the Company’s proprietary topoisomerase I inhibitor payload and linker-payload platform, ALX2004 has been rigorously designed to maximize the therapeutic window and overcome toxicity challenges observed in this class.

This includes an affinity-tuned EGFR antibody with a binding epitope distinct from approved EGFR antibodies. ALX2004 also has a proprietary linker-Top1i payload engineered to offer enhanced bystander effect with improved linker stability for on-target delivery of payload.

Robust Preclinical Data: Supporting Differentiation in EGFR-ADC Class

Preclinical data supports ALX2004’s differentiated linker-payload construct, which has demonstrated superior stability versus other ADCs in its class, with dose-dependent activity and a favorable safety profile. In addition, ALX2004 has shown dose-dependent activity across a range of tumors, EGFR expression levels and mutations. Potent activity in tumor models supports its potential for treating patients with EGFR-expressing tumors. Preclinical model findings did not demonstrate EGFR-related skin toxicity at clinically relevant doses or payload-related interstitial lung disease, supportive of a potentially differentiated safety profile.

Initiation of Phase 1 Clinical Development Program

The U.S. Food and Drug Administration (FDA) cleared ALX Oncology’s Investigational New Drug (IND) application for ALX2004 in April 2025. ALX Oncology will provide an overview of its Phase 1 clinical trial design for ALX2004 planned to initiate mid-2025. The Company anticipates initiating a Phase 1 dose escalation trial in EGFR-expressing solid tumors, including non-small cell lung cancer, colorectal cancer, head and neck squamous cell carcinoma and esophageal squamous cell carcinoma, targeting patients with relapsed/refractory cancers. The Company anticipates initial safety data in the first half of 2026.

ALX2004 Webcast Information
The virtual event will be webcast live and a replay will be available after the event by visiting the "Investors" section of ALX Oncology’s website and selecting "Events and Presentations."

Date & Time: Tuesday, May 20, 2025, 8:00 a.m. PT/11:00 a.m. ET
Webcast Access: View Source

Registrational Phase III AMPLIFY trial in biochemical recurrence of prostate cancer commences

On May 20, 2025 Clarity Pharmaceuticals (ASX: CU6) ("Clarity" or "Company"), a clinical-stage radiopharmaceutical company with a mission to develop next-generation products that improve treatment outcomes for patients with cancer, reported that it has commenced its second registrational Phase III 64Cu-SAR-bisPSMA diagnostic trial in prostate cancer, AMPLIFY (NCT06970847)1, with the initiation of the first clinical site at XCancer in Omaha, NE (Press release, Clarity Pharmaceuticals, MAY 20, 2025, View Source [SID1234653232]).

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AMPLIFY is a study of 64Cu-SAR-bisPSMA Positron Emission Tomography: A Phase 3 Study of Participants with Biochemical Recurrence of Prostate Cancer. It is a non-randomised, single-arm, open-label, multi-centre, diagnostic clinical trial of 64Cu-SAR-bisPSMA positron emission tomography (PET) in participants with rising or detectable prostate-specific antigen (PSA) after initial definitive treatment.

The aim of this Phase III trial is to investigate the ability of 64Cu-SAR-bisPSMA PET/computed tomography (CT) to detect recurrence of prostate cancer. Evaluation will be across 2 imaging timepoints, Day 1 (day of administration, same-day imaging) and Day 2 (approximately 24 hours post administration, next-day imaging).

The study will enroll approximately 220 participants at multiple clinical sites across the United States (US) and Australia, and the first participant is expected to be imaged in the coming weeks. As a pivotal trial, the final study results are intended to provide sufficient evidence to support an application to the US Food and Drug Administration (FDA) for approval of 64Cu-SAR-bisPSMA as a new diagnostic imaging agent in biochemical recurrence (BCR) of prostate cancer.

Clarity’s Executive Chairperson, Dr Alan Taylor, commented, "We are very excited to commence our second registrational Phase III trial with the optimised SAR-bisPSMA agent in patients that are experiencing the return of their disease following treatment of the primary cancer.

"The recent devastating news that former US President, Joe Biden, has been diagnosed with aggressive prostate cancer that has metastasised to the bone is yet another reminder that no man is safe from this insidious disease, and he joins over 3.3 million men in the US who live with prostate cancer today2. Australia is also not immune. A recent public announcement revealed that MP Barnaby Joyce has recently been diagnosed with prostate cancer, and he joins over 250,000 Australian men who are living with prostate cancer today3. These large numbers highlight the need for more timely and accurate diagnosis to safely and effectively treat the cancer with suitable therapies."

The AMPLIFY trial is based on favourable preclinical and clinical data generated to date, including the Phase I/II COBRA trial in patients with BCR of prostate cancer and the Phase I PROPELLER trial in patients with confirmed prostate cancer pre-prostatectomy4,5. These earlier studies demonstrated an excellent safety profile and exciting efficacy results, especially in comparison to current standard of care imaging. PROPELLER showed improved diagnostic performance of 64Cu-SAR-bisPSMA compared to 68Ga-PSMA-11 on same-day imaging, including higher number of lesions identified and 2-3 times higher uptake and tumour-to-background ratio, favouring 64Cu-SAR-bisPSMA. The COBRA trial showed that more lesions and more patients with a positive scan were identified on 64Cu-SAR-bisPSMA PET compared to conventional scans and on next-day vs. same-day imaging. 64Cu-SAR-bisPSMA also allowed for the identification of lesions in the 2-mm range. The most recent findings from the COBRA trial demonstrated that 64Cu-SAR-bisPSMA was able to detect lesions from 29 days to more than 6 months earlier than standard-of-care (SOC) prostate-specific membrane antigen (PSMA) PET agents.

"These compelling findings, along with preliminary theranostic SECuRE trial data6, laid the foundation for the receipt of three Fast Track Designations (FTD) for SAR-bisPSMA from the US FDA within six months7-9, highlighting how impressive our science and clinical development are, the significance of the data so far and the high unmet need for better therapies and diagnostics in prostate cancer. We believe 64Cu-SAR-bisPSMA could become a best-in-class product and look forward to progressing its clinical development through several trials in prostate cancer: AMPLIFY in BCR, our first Phase III CLARIFY study in pre-prostatectomy patients, the investigator-initiated trial led by Prof Louise Emmett, Co-PSMA (head-to-head study comparing the diagnostic performance of 64Cu-SAR-bisPSMA vs. SOC 68Ga-PSMA-11 in BCR), and the theranostic SECuRE study in metastatic castration-resistant prostate cancer (mCRPC). The FTDs will enable us to accelerate the development of this comprehensive program to be used in patients with prostate cancer throughout the management of their cancer, from initial to late-stage disease, with an opportunity to completely change the entire treatment landscape for the large prostate cancer market.

"We have received a lot of interest in the AMPLIFY trial and look forward to seeing this study progress in such an important patient population. Knowing whether or not their prostate cancer has returned following initial treatment and where the cancer lesions are located are essential for the appropriate treatment of BCR patients. Identifying lesions as early as possible can arm clinicians with important information to help determine the right treatment regimen and ensure positive long-term treatment outcomes. We would like to thank all clinicians and patients who participate in our clinical trials and who trust us in delivering on our promise of developing best-in-class products. Our team and collaborators look forward to bringing this next-generation PSMA diagnostic to prostate cancer patients around the world," said Dr Taylor.

About SAR-bisPSMA
SAR-bisPSMA derives its name from the word "bis", which reflects a novel approach of connecting two PSMA-targeting agents to Clarity’s proprietary sarcophagine (SAR) technology that securely holds copper isotopes inside a cage-like structure, called a chelator. Unlike other commercially available chelators, the SAR technology prevents copper leakage into the body. SAR-bisPSMA is a Targeted Copper Theranostic (TCT) that can be used with isotopes of copper-64 (Cu-64 or 64Cu) for imaging and copper-67 (Cu-67 or 67Cu) for therapy.

Q1 2025 Earnings

On May 19, 2025 Oscotec reported first quarter 2025 results (Presentation, Oscotec, MAY 19, 2025, View Source [SID1234654102]).

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Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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