Chugai Files for Additional Indication of ALK Inhibitor ALECENSA for Recurrent or Refractory ALK Fusion Gene-Positive Anaplastic Large Cell Lymphoma (ALCL)

On June 3, 2019 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it filed an application for ALK Inhibitor, Alecensa capsule 150 mg (generic name: alectinib) to MHLW for an additional indication of recurrent or refractory ALK fusion gene-positive anaplastic large cell lymphoma (ALK-positive ALCL) (Press release, Chugai, JUN 3, 2019, View Source [SID1234536788]). Alecensa obtained an orphan drug designation from the MHLW on May 30.

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"While chemotherapy is known to be effective for ALCL, there is a strong need for development of new therapeutic options since recurrent ALCL often carries a poor prognosis," said Dr. Yasushi Ito, Chugai’s Executive Vice President, Co-Head of Project & Lifecycle Management Unit. "We hope that we can contribute to improving prognosis of these patients with the use of Alecensa that has the possibility of becoming a promising molecular target drug for ALK-positive ALCL."

This filing is based on the results from the investigator initiated study (the ALC-ALCL study) started in May 2015. The study has been conducted as a part of "Research Project on Practical Application of Innovative Cancer Therapy" by Japan Agency for Medical Research and Development. The ALC-ALCL study was a phase II multicenter clinical study in which the response rate (primary endpoint) was assessed by the central review committee and safety was investigated in 10 patients with recurrent or refractory ALK-positive ALCL. It was conducted in three sites, led by Nagoya Medical Center, National Hospital Organization.

As a leading pharmaceutical company in the oncology field in Japan, Chugai will work to provide Alecensa as a new treatment option for ALK-positive ALCL as soon as possible.

About ALK-positive ALCL
ALCL is one of four subtypes of peripheral T-cell lymphoma classified as malignant lymphoma, which is non-Hodgkin’s lymphoma originated in T-cells in lymphocytes. The malignancy is categorized as "intermediate-grade," in which disease progression is observed on a monthly basis. In Japan, the percentage of ALCL in malignant lymphomas is from 1.5 to 2.0%1, 2), about half of which have been reported as ALK-positive.3,4) Considering that the 5-year survival rate with successful treatment has been reported to be 60% in patients with ALK-positive ALCL who received chemotherapy in another global study, the percentage of recurrent and refractory cases is estimated to be 40%.3)

Trademarks used or mentioned in this release are protected by law.

Lymphoma Study Group of Japanese Pathologists. The World Health Organization classification of malignant lymphomas in Japan: Incidence of recently recognized entities. Pathol Int. 2000 Sep; 50(9): 696-702
Aoki R, Karube K, Sugita Y, Nomura Y, Shimizu K, Kimura Y, et al. Distribution of malignant lymphoma in Japan: Analysis of 2260 cases. 2001-2006. Pathol Int. 2008 Mar; 58(3): 174-82
Savage KJ, Harris NL, Vose JM, Ullrich F, Jaffe ES, Connors JM, et al. ALK- anaplastic large-cell lymphoma is clinically and immunophenotypically different from both ALK+ ALCL and peripheral Tcell lymphoma, not otherwise specified: report from the International Peripheral T-Cell Lymphoma Project. Blood. 2008 Jun 5; 111(12): 5496-504
Sibon D, Fournier M, Brière J, Lamant L, Haioun C, Coiffier B, et al. Long-Term Outcome of Adults With Systemic Anaplastic Large-Cell Lymphoma Treated Within the Groupe d’Étude des Lymphomes de l’Adulte Trials. J Clin Oncol. 2012 Nov 10; 30(32): 3939-46

Final Progression-Free Survival Data and the Second Interim Analysis of Overall Survival from the J-ALEX Study for Alecensa Presented at the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting

On June 3, 2019 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) announced that the final progression-free survival (PFS) data and the second interim analysis of overall survival (OS) from the Japanese phase III J-ALEX study for Alecensa were presented on June 2 (local time) at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place from May 31 to June 4 in Chicago, IL, United States (Press release, Chugai, JUN 3, 2019, View Source [SID1234536782]). J-ALEX study compared Alecensa and crizotinib as the first-line treatment for patients with ALK fusion gene positive non-small cell lung cancer (NSCLC).

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Abstract #9092;
Final PFS analysis and safety data from the phase III J-ALEX study of Alectinib (ALC) vs. Crizotinib (CRZ) in ALK-inhibitor naïve ALK-positive Non-Small Cell Lung Cancer (ALK+NSCLC)

"The long-term data from the J-ALEX study confirms the benefits of Alecensa as the first-line treatment for patients with ALK-positive NSCLC," said Dr. Yasushi Ito, Chugai’s Executive Vice President, Co-Head of Project & Lifecycle Management Unit.

The J-ALEX study is an open-label, randomized phase III study that compares the efficacy and safety between Alecensa and crizotinib. The J-ALEX study enrolled 207 ALK-inhibitor naïve patients with ALK fusion gene positive advanced or recurrent NSCLC, who either had not undergone chemotherapy or had undergone one chemotherapy regimen. The primary endpoint of the J-ALEX study was PFS as assessed by an independent review facility (IRF). The secondary endpoints included OS, time to disease progression of brain metastases in patients with brain metastases at baseline, and safety.

In February, 2016, Chugai carried out a prospectively defined interim analysis, and had an independent data monitoring committee examine the results. Since the results showed that Alecensa significantly prolonged PFS to a higher extent than anticipated, the committee decided to recommend an early discontinuation of the study [Alecensa arm: not estimable (95% CI: 20.3-not estimable), crizotinib arm: 10.2 months (95% CI: 8.2-12.0), HR=0.34 (99.7% CI: 0.17-0.70), stratified log-rank test, p<0.0001)].

Latest data from the J-ALEX study shows:

Risk of progression or death was reduced by 63% (HR=0.37, 95% CI: 0.26-0.52) in the Alecensa arm compared to the crizotinib arm. Median PFS (primary endpoint) was 34.1 months in the Alecensa arm (95% CI: 22.1-not estimable) versus 10.2 months (95% CI: 8.3-12.0) in the crizotinib arm.
In the second interim analysis, the superiority of OS in the Alecensa arm over the crizotinib arm was not conclusive (stratified HR=0.80, 95% CI: 0.35-1.82). The investigation on OS will be continued.
The safety profile of Alecensa was consistent with previous reports.
[Reference information]
Media release issued by Chugai on May 11, 2017:
Results of the J-ALEX Study for Chugai’s Alecensa are Published in "The Lancet" Online;
View Source

New Data from GEOMETRY mono-1 Study Show Clinically Meaningful Results in Patients with Non-Small Cell Lung Cancer with MET exon-14 Skipping Mutation Treated with Capmatinib

On June 3, 2019 Incyte (Nasdaq:INCY) reported primary efficacy results from the Novartis-sponsored GEOMETRY mono-1 Phase 2 clinical trial of capmatinib, an investigational, selective MET inhibitor (Press release, Incyte, JUN 3, 2019, View Source [SID1234536781]). The results demonstrate that capmatinib shows promise as a potential treatment option for patients with locally advanced or metastatic non-small cell lung cancer (NSCLC) that harbor a MET exon-14 skipping mutation. There are currently no approved targeted therapies to treat this particularly aggressive form of NSCLC.

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Results of the Phase 2 study will be presented at an oral session today, June 3, 2019, at the American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) 2019 Annual Meeting at 8:00 a.m. CDT (Abstract #9004)1.

GEOMETRY mono-1 is an international, prospective, multi-cohort, non-randomized, open-label study evaluating 97 adult patients with locally advanced or metastatic NSCLC harboring a MET exon-14 skipping mutation who received capmatinib tablets 400 mg orally twice daily. Primary efficacy results among treatment-naïve patients (Cohort 5b: 28 patients) included a 68 percent overall response rate (ORR) based on the Blinded Independent Review Committee (BIRC) assessment per RECIST v1.1 (95% [CI: [47.6-84.1]) and 41 percent of previously-treated NSCLC patients (Cohort 4: 69 patients) also responded (95% CI: [28.9 – 53.1]). Data on median duration of response (DOR), a key secondary endpoint, was 11.14 months (95% CI: [5.55-NE]) and 9.72 months (95% CI: [5.55-12.98]), in the treatment-naïve and previously-treated groups, respectively. Intracranial activity in 54 percent (n=7/13) of patients, including some cases of complete resolution of brain lesions, was also observed by ad hoc neuro-radiologist review in patients with brain lesions. All results were based on independent assessment by the BIRC, and all tumor CT scans were evaluated in parallel by two radiologists to confirm the response.

The most common treatment-related adverse events (AE) (≥10% all grades) across all cohorts (n=334), were peripheral edema (42%), nausea (33%), creatinine increase (20%), vomiting (19%), fatigue (14%), decreased appetite (13%) and diarrhea (11%); the majority of the AEs were grades 1/2.

"In the absence of approved targeted therapies, patients with advanced or metastatic NSCLC harboring a MET exon-14 skipping mutation must rely on existing treatment approaches and, as a result, face a particularly poor prognosis," said Steven Stein, M.D., Chief Medical Officer, Incyte. "The results of the GEOMETRY mono-1 study to be presented at ASCO (Free ASCO Whitepaper) underscore the potential of capmatinib to meaningfully improve outcomes for this underserved subset of NSCLC patients."

The U.S. Food and Drug Administration (FDA) recently granted capmatinib Breakthrough Therapy designation for patients with metastatic NSCLC harboring a MET exon-14 skipping mutation with disease progression on or after platinum-based chemotherapy. Previously, both the U.S. FDA and Japan’s Pharmaceuticals and Medical Devices Agency recognized capmatinib with Orphan Drug status. It is estimated that 3 to 4 percent of all patients with NSCLC have an identified MET mutation2.

Novartis expects to submit a new drug application to the FDA for capmatinib as a treatment for patients with advanced NSCLC harboring a MET mutation in 2019.

About GEOMETRY mono-1

The Novartis-sponsored GEOMETRY mono-1 trial is an international, prospective, multi-cohort, non-randomized, open-label Phase 2 study to evaluate the efficacy and safety of single-agent capmatinib in adult patients with EGFR wildtype, ALK-negative rearrangement, advanced NSCLC harboring a MET amplification and/or mutation. Patients with locally advanced or metastatic NSCLC harboring a MET exon-14 skipping mutation (centrally confirmed) were assigned to Cohorts 4 (previously treated patients) or 5B (treatment-naïve), regardless of MET amplification/gene copy number and received 400 mg capmatinib tablets orally twice daily.

The primary endpoint was ORR based on BIRC assessment per RECIST v1.1. The key secondary endpoint was DOR by BIRC. The GEOMETRY mono-1 study found an ORR in the treatment-naïve patients (n=28) of 67.9 percent (95% CI: [47.6 – 84.1]) and an ORR of 40.6 % (95% CI: [28.9 – 53.1]) in the previously treated patients (n=69). Median DOR was 11.14 months (95% CI: [5.55-NE]) in treatment-naïve patients and 9.72 months (95% CI: [5.55-12.98]) in previously treated patients1.

The most common treatment-related AEs included peripheral edema, nausea, creatinine increase and vomiting. Of patients treated with capmatinib, 84 percent experienced an AE, with 36 percent having grade 3/4 AEs (only 4.5% were Grade 4)1.

About Capmatinib

Capmatinib is an investigational, oral and selective MET inhibitor discovered by Incyte that was licensed to Novartis in 2009. Under the terms of the Agreement, Incyte granted Novartis exclusive worldwide development and commercialization rights to capmatinib and certain back-up compounds in all indications. If capmatinib is successfully developed by Novartis, Incyte may become eligible for over $500 million in future milestones as well as royalties of between 12 and 14 percent on global sales by Novartis.

corporate presentation

On June 2, 2019, Corvus Pharmaceuticals presented the corporate presentation (Presentation, Corvus Pharmaceuticals, JUN 2, 2019, View Source [SID1234536804]).

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Daiichi Sankyo Presents Preliminary Phase 1 Data for TROP2 Targeting ADC DS-1062 in Patients with Non-Small Cell Lung Cancer at 2019 ASCO Annual Meeting

On June 2, 2019 Daiichi Sankyo Company, Limited (hereafter, Daiichi Sankyo) reported that preliminary results from the dose escalation part of a phase 1 study with DS-1062, an investigational TROP2 targeting antibody drug conjugate (ADC), in 39 patients with advanced non-small cell lung cancer (NSCLC) were presented today during a Poster Session at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL (Abstract #9051) (Press release, Daiichi Sankyo (Brazil), JUN 2, 2019, https://www.prnewswire.com/news-releases/daiichi-sankyo-presents-preliminary-phase-1-data-for-trop2-targeting-adc-ds-1062-in-patients-with-non-small-cell-lung-cancer-at-2019-asco-annual-meeting-300860250.html [SID1234536785]).

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Preliminary efficacy data for 35 evaluable patients who received DS-1062 at the dose levels between 0.27 mg/kg and 8.0 mg/kg every 21 days showed three confirmed and four not yet confirmed partial responses at the time of data cut-off on April 12, 2019.

An additional three partial responses have been reported with the 8.0 mg/kg dose of DS-1062 since data cut-off, bringing the total partial responses to 10. The median number of prior therapies for the partial responders is 3.5 and includes patients with prior EGFR or ALK inhibitors and checkpoint inhibitors. A maximum tolerated dose of DS-1062 has not yet been reached and the dose escalation portion of the study is ongoing. Sixteen patients remain on-treatment at the time of data cut-off.

"Despite recent advances in the treatment of NSCLC, we need new precision treatment options for patients with advanced disease who currently have no available standard treatment options," said Jacob Sands, MD, Physician, Dana-Farber Cancer Institute, Instructor, Harvard Medical School, and lead investigator for the study. "These early results are encouraging as we have seen increased activity with higher doses of DS-1062 including several partial responses. Additional study is warranted to further determine the potential for targeting TROP2 with DS-1062 in these patients with advanced NSCLC."

Preliminary data for 39 patients evaluable for safety as of April 12, 2019 showed that DS-1062 was well-tolerated at a median treatment exposure time of 8.86 weeks (range 3.0-31.1). Common treatment emergent adverse events (occurring in ≥ 30 percent of patients) included fatigue (33.3 percent) and nausea (30.8 percent). Sixteen patients (41.0 percent) experienced at least one treatment emergent adverse event ≥ grade 3. One dose-limiting toxicity of maculopapular rash, grade 3 was observed in a patient in the 6.0 mg/kg cohort. Ten patients (25.6 percent) experienced serious adverse events not related to study drug, and one patient (2.5 percent) in the 4.0 mg/kg cohort experienced a treatment-related serious adverse event of pyrexia.

"We are encouraged by these preliminary results with DS-1062, which was designed using our proprietary DXd ADC technology to target and deliver treatment directly to tumors that express TROP2, a promising therapeutic target for many types of cancer, including NSCLC," said Eric Slosberg, PhD, Head, Global Translational Development, Oncology Research and Development, Daiichi Sankyo. "Currently, there are no TROP2 targeting therapies approved for any cancer, and we will continue to study DS-1062 as part of our commitment to developing new targeted therapy options for patients with non-small cell lung and other cancers."

DS-1062 is designed using Daiichi Sankyo’s proprietary DXd ADC technology, which consists of a humanized monoclonal antibody attached to a novel topoisomerase I inhibitor payload by a tetrapeptide-based linker. DS-1062 was designed to target and deliver chemotherapy inside cancer cells that express TROP2 as a cell surface antigen. The DXd ADC technology provides flexibility to adapt the drug-to-antibody ratio (DAR) or the number of DXd molecules conjugated per antibody. DS-1062 has a DAR of four, which is based on initial research into the construct necessary for intended efficacy and safety in TROP2 expressing tumors.

About the Phase 1 Study
The phase 1, first-in-human open-label study is investigating the safety and tolerability of DS-1062 in patients with unresectable advanced NSCLC who are refractory to or have relapsed following standard treatment or for whom no standard treatment is available. The first part of the study (dose escalation) is assessing the safety and tolerability of increasing doses of DS-1062 to determine the maximum tolerated dose and recommended dose for expansion. The second part of the study (dose expansion) will evaluate the safety and tolerability of DS-1062 at the recommended dose for expansion. Study endpoints include safety, pharmacokinetics, objective response rate, duration of response, disease control rate, time to response, progression-free survival, overall survival, biomarker analysis and immunogenicity. The study is currently enrolling patients with unresectable advanced NSCLC in the United States and Japan.

Based on the results of the study, additional cohorts may be initiated for other solid tumors where high expression of TROP2 is frequently observed. For more information about the study, visit ClinicalTrials.gov.

Unmet Need in Non-Small Cell Lung Cancer (NSCLC)
Lung cancer is the most common cancer and the leading cause of cancer mortality worldwide; there were an estimated 2.1 million new cases of lung cancer in 2018 and 1.8 million deaths.1 Most lung cancers are diagnosed at an advanced or metastatic stage.2 Non-small cell lung cancer (NSCLC) accounts for 80 to 85 percent of all lung cancers.3 The introduction of targeted therapies and checkpoint inhibitors in the past decade has improved the treatment landscape for patients with advanced or metastatic NSCLC; however, for those who are not eligible for current treatments, or whose cancer continues to progress, new therapeutic approaches are needed.4

TROP2 (trophoblast cell-surface antigen 2) is a transmembrane glycoprotein that is highly expressed on several types of solid tumors, including NSCLC.5,6 Researchers have recognized TROP2 as a promising molecular target for therapeutic development in various types of malignancies, including NSCLC.6,7 Overexpression of TROP2 has been associated with increased tumor aggressiveness and decreased survival in several cancers.8 High TROP2 expression was identified in 64 percent of non-small cell adenocarcinomas and 75 percent of non-small cell squamous cell carcinomas in one study.5 Currently, no TROP2 targeting therapy is approved for NSCLC or any cancer.

About DS-1062
Part of the investigational ADC Franchise of the Daiichi Sankyo Cancer Enterprise, DS-1062 is an investigational TROP2 targeting ADC. 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, DS-1062 is comprised of a humanized anti-TROP2 monoclonal antibody attached to a novel topoisomerase I inhibitor payload by a tetrapeptide-based linker. It is designed to target and deliver chemotherapy inside cancer cells and reduce systemic exposure to the cytotoxic payload (or chemotherapy) compared to the way chemotherapy is commonly delivered.

DS-1062 is an investigational agent that has not been approved for any indication in any country. Safety and efficacy have not been established.

About Daiichi Sankyo Cancer Enterprise
The mission of Daiichi Sankyo Cancer Enterprise is to leverage our world-class, innovative science and push beyond traditional thinking to create meaningful treatments for patients with cancer. We are dedicated to transforming science into value for patients, and this sense of obligation informs everything we do. Anchored by three pillars including our investigational Antibody Drug Conjugate Franchise, Acute Myeloid Leukemia Franchise and Breakthrough Science, we aim to deliver seven distinct new molecular entities over eight years during 2018 to 2025. Our powerful research engines include two laboratories for biologic/immuno-oncology and small molecules in Japan, and Plexxikon Inc., our small molecule structure-guided R&D center in Berkeley, CA. Compounds in pivotal stage development include: [fam-] trastuzumab deruxtecan, an antibody drug conjugate (ADC) for HER2 expressing breast, gastric and other cancers; quizartinib, an oral selective FLT3 inhibitor, for newly-diagnosed and relapsed/refractory FLT3-ITD acute myeloid leukemia (AML); and pexidartinib, an oral CSF1R inhibitor, for tenosynovial giant cell tumor (TGCT). For more information, please visit: www.DSCancerEnterprise.com.