Chugai Launches FoundationOne Liquid CDx Cancer Genomic Profile as the First Blood-based Comprehensive Genomic Profiling Test for Solid Tumors in Japan

On August 2, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has launched FoundationOne Liquid CDx Cancer Genomic Profile as a liquid biopsy-based comprehensive genomic profiling (CGP) test for solid tumor, following the product’s listing on the national health insurance (NHI) reimbursement price list on August 1, 2021 (Press release, Chugai, AUG 2, 2021, View Source [SID1234585500]). In addition, SRL Inc., the clinical laboratory testing company has started providing testing services for the product today. FoundationOne Liquid CDx Cancer Genomic Profile was approved by the Ministry of Health, Labour and Welfare (MHLW) on March 22, 2021 for use as a companion diagnostic (CDx) for certain approved targeted therapies in Japan, making it the first MHLW-approved blood-based test with both CDx and solid tumor CGP indications.

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"We are very pleased that we can start providing FoundationOne Liquid CDx Cancer Genomic Profile, a blood-based CGP testing option for patients today. The test provides meaningful information that can help inform treatment for patients with advanced or recurrent cancer, which is especially valuable if they are not eligible for tissue-based CGP testing," said Chugai’s president and CEO Dr. Osamu Okuda. "We are committed to advance personalized healthcare through expanding access to CGP testing."

Developed by Foundation Medicine Inc. based in Cambridge, USA, FoundationOne Liquid CDx Cancer Genomic Profile is a blood-based diagnostic test that uses next-generation sequencing. It identifies genomic alterations in 324 cancer-related genes for cancer patients with solid tumors through detection of blood circulating tumor DNA (ctDNA). FoundationOne Liquid CDx Cancer Genomic Profile provides an integrated test report informing alterations matched to MHLW-approved targeted therapies.

As a leading company in the field of oncology, Chugai is committed to advance personalized healthcare in oncology and contributing to patients and healthcare professionals through improving access to CGP.

Approval information

Brand name FoundationOne Liquid CDx Cancer Genomic Profile
Japanese medical device nomenclature (JMDN)
Software for gene variants analysis (for cancer genome profiling)
Software for analysis of somatic cell gene variants (for eligibility identification of antineoplastic agents)
Intended uses or indications
The Product is used for comprehensive genomic profiling of blood samples in patients with solid tumors.
The Product is used for detecting gene mutations and other alterations to support the assessment of drug indications listed in the table below.
Alterations Cancer type Relevant drugs
Activated EGFR alterations Non-small cell lung cancer (NSCLC) afatinib maleate, erlotinib hydrochloride, gefitinib, osimertinib mesilate
EGFR exon 20 T790M alterations osimertinib mesilate
ALK fusion genes alectinib hydrochloride, crizotinib, ceritinib
ROS1 fusion genes entrectinib
NTRK1/2/3 fusion gene Solid tumors entrectinib
BRCA1/2 alteration Prostate cancer olaparib
Conditions for approval
The necessary measures must be taken to ensure that the product is used by a physician with adequate knowledge and experience of cancer genomic medicine at a medical institution with a cancer genome profiling-based medical system pursuant to the "Guidelines for the Development of Core Hospitals and Other Facilities for Cancer Genomic Medicine," and in compliance with the scope and timing of testing stipulated in the most recent guidelines, etc., of relevant academic societies.
Appropriate procedures and controls to protect personal information and up-to-date security and privacy protection measures to prevent unauthorized access must be implemented for blood samples sent to the laboratory and for information obtained from these specimens.
Quality control of input data must be performed as described in the Remarks column of the attached Application Form. Any changes to the quality control of input data as described in the Remarks column of the Application Form (excluding minor changes specified by Order of the MHLW in Article 23-2-5, paragraph (15) of the Act on Securing Quality, Efficacy and Safety of Products Including Pharmaceuticals and Medical Devices ["the Act"]) must be approved by the MHLW Minister pursuant to Article 23-2-5, paragraph (15) of the Act. Note that this approval applies mutatis mutandis to the provisions of Article 23-2-5 paragraph (17), Article 23-2-6, and Article 23-2-7 of the Act.
Date of NHI reimbursement price listing: August 1, 2021
About FoundationOne Liquid CDx Cancer Genomic Profile
Developed by Foundation Medicine Inc. based in Cambridge, USA, FoundationOne Liquid CDx Cancer Genomic Profile is a next-generation sequencing based in vitro diagnostic device using blood samples for advanced cancer patients with solid tumors. It is intended to identify genomic alterations in 324 cancer-related genes through detection of blood circulating tumor DNA (ctDNA). The test is approved by the MHLW for use in cancer genome profiling to report substitutions, insertion and deletion alterations, and select gene rearrangements for short variants in 324 genes. It is also indicated for use as a companion diagnostic to identify patients who may benefit from treatment with specific targeted therapies (listed in Table above of Intended uses or indications). For the latest information about the product, including companion diagnostic indications, please refer to the prescribing information.

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

Ipsen and Exicure Enter Into Exclusive Collaboration Targeting Rare Neurodegenerative Disorders

On August 2, 2021 Ipsen (Euronext: IPN; ADR: IPSEY) and Exicure Inc. (NASDAQ: XCUR) reported that they have signed an exclusive collaboration agreement to research, develop, and commercialize novel Spherical Nucleic Acids (SNAs) as potential investigational treatments for Huntington’s disease and Angelman syndrome (Press release, Ipsen, AUG 2, 2021, View Source [SID1234585494]).

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Oligonucleotides are synthetic structures of nucleic acids that can be used to modulate gene expression via a range of processes, including gene activation, inhibition, and splice-modulation. These molecules have demonstrated potential in many different therapeutic areas.1 Achieving efficient oligonucleotide delivery to target organs and tissues, including the brain, remains a major limitation to their use.1,2 Exicure’s SNAs provide distinct chemical and biochemical properties to oligonucleotides. In preclinical models, SNAs have been shown to enhance the cell penetration, biodistribution and organ persistence properties of oligonucleotides,3,4 which may potentially enhance drug delivery to previously inaccessible target tissues, including deep brain regions.5,6

Philippe Lopes-Fernandes, Chief Business Officer at Ipsen, said "Neuroscience is deeply rooted within Ipsen as a key strategic driver for our business. We are pleased to partner with Exicure to progress development of investigational treatment options for Huntington’s disease and Angelman syndrome, two areas of significant unmet need. This collaboration marks an important step in maximizing the potential of this novel technology, bringing together the expertise of Exicure and the robust heritage of Ipsen in neuroscience. With this new collaboration we will deepen our commitment to people living with neurological conditions around the world."

"We are thrilled to partner with Ipsen, a leading global company with significant expertise and commitment to developing treatments for patients with rare neurological diseases," said David Giljohann, Ph.D., Chief Executive Officer, Exicure, Inc. "In collaboration with Ipsen, we have the opportunity to apply our technology to Huntington’s disease and Angelman syndrome, both indications requiring deep brain penetration and technological advances to reach previously hard-to-drug targets. We believe our platform technology with its deep penetration and persistence of medicinal effect will allow Exicure and Ipsen to overcome challenges from first-generation oligonucleotides and bring new medicines to patients in need."

Under the agreement, Ipsen will receive exclusive options to license SNA-based therapeutics arising from two collaboration programs for Huntington’s disease and Angelman syndrome. Ipsen will pay Exicure a cash upfront payment of $20m upon closing and Exicure will be responsible for discovery and certain pre-clinical development activities. In the event Ipsen exercises its option, Ipsen will be responsible for further development and commercialization of the licensed products. Exicure will receive a $20m upfront payment and is eligible to receive up to $1B in option exercise fees and milestone payments should Ipsen opt into both programs, as well as tiered royalties.

Huntington’s disease

Huntington’s disease (HD) is a progressive, fatal neurodegenerative disorder and the most common monogenic neurological disorder in the developed world, affecting about 40,000 individuals in the US.7 HD is caused by an expanded CAG trinucleotide repetition in the huntingtin (HTT) gene in chromosome 4. HD is characterized by involuntary movements, psychiatric disorders, cognitive deterioration, and early mortality, with death often occurring within 10 to 20 years after motor symptoms appear. Mean age of onset of motor symptoms is around 40 years of age, with longer CAG repeats causing earlier disease onset. 8 There is currently no approved therapy to address the underlying molecular cause of HD to slow or stop disease progression.9

Angelman syndrome

Angelman syndrome (AS) is a severe neurodevelopmental disorder. The prevalence of Angelman syndrome is estimated to be 1 in 12,000-20,000 people in the general population.10 The disorder is characterized by severe intellectual deficit, speech impairment, epilepsy, ataxic movements and behavioral abnormalities. AS results from loss of function of the maternally inherited copy of the ubiquitin-protein ligase E3A (UBE3A) gene on chromosome 15.11 Disruption of UBE3A function in neurons prevents synapse formation and remodeling, leading to significant neurodevelopmental disability. There is currently no approved disease-modifying therapy for AS and standard-of-care treatment is supportive, such as medications for seizures and behavioral abnormalities.12

Evotec SE Announces Confidential Submission of Draft Registration Statement on Form F-1 with the U.S. Securities and Exchange Commission for a Proposed Offering of American Depositary Shares

On August 1 2021 Evotec SE (Frankfurt Stock Exchange; EVT, MDAX/TecDAX, ISIN: DE0005664809, WKN 566480) reported that it confidentially submitted a Registration Statement on Form F-1 ("Registration Statement") with the U.S. Securities and Exchange Commission ("SEC") for a proposed offering and sale in the United States of shares of Evotec represented by American Depositary Shares ("ADSs") (Press release, Evotec, AUG 1, 2021, View Source;announcements/ad-hoc-releases/p/evotec-se-announces-confidential-submission-of-draft-registration-statement-on-form-f-1-with-the-us-securities-and-exchange-commission-for-a-proposed-offering-of-american-depositary-shares-6080 [SID1234585507]). The Registration Statement has not yet become effective and the final number of ADSs to be offered and their price have not yet been determined.

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Evotec’s shares are listed on the regulated market of the Frankfurt Stock Exchange in Germany with additional admission obligations of the Prime Standard Segment. Evotec will seek a secondary listing of the ADSs on the Nasdaq Global Select Market in the United States under the ticker symbol "EVO". The new shares underlying the ADSs will be issued from Evotec’s authorized capital. The first day of trading of the ADSs is expected to occur during the second half of 2021, however, this timing remains subject to change.

The ADSs referred to in this announcement are to be offered only by means of a registration statement on Form F-1 approved by the SEC. In accordance with applicable capital markets requirements, Evotec will announce when the registration statement has been approved by the SEC, including further details, such as final number and price of ADS.

This announcement does not constitute an offer to sell or the solicitation of an offer to buy any securities. Any offers, solicitations, or offers to buy, or any sales of securities will be made in accordance with the registration requirements of the U.S. Securities Act of 1933, as amended ("Securities Act"), and other applicable securities laws. This announcement is being issued in accordance with Rule 135 under the Securities Act and other applicable securities laws. This public disclosure of inside information does not constitute a prospectus.

Antengene Announces the Approval of First-in-Class Oral XPO1 Inhibitor Selinexor in South Korea for the Treatment of Relapsed or Refractory Multiple Myeloma and Diffuse Large B-Cell Lymphoma

On August 1, 2021 Antengene Corporation Limited ("Antengene", SEHK: 6996.HK), a leading innovative biopharmaceutical company dedicated to discovering, developing and commercializing global first-in-class and/or best-in-class therapeutics in hematology and oncology, reported that through a priority review process, the South Korean Ministry of Food and Drug Safety (MFDS) has approved the company’s New Drug Application (NDA) for the Orphan Drug-designated first-in-class oral inhibitor of XPO1, selinexor (XPOVIO), in combination with dexamethasone for the treatment of adult patients with relapsed or refractory multiple myeloma (rrMM) who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, at least two immunomodulatory agents, and an anti-CD38 monoclonal antibody (penta-refractory); and as a monotherapy for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (rrDLBCL) who have received at least two prior lines of treatment (Press release, Antengene, AUG 1, 2021, View Source [SID1234585496]).

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Introduction of a practice-changing therapy for MM and DLBCL patients

Most patients with MM eventually suffer from relapse or become refractory diseases. For patients who have failed in one therapy, diffuse large B-cell lymphoma (DLBCL) has poor prognosis and the chance of cure or long-term disease-free survival declined every time getting worse after treatment. At present, there is no cure for rrMM and rrDLBCL and it is in dire need for safer and more effective therapies.

In October 2020, selinexor was granted an Orphan Drug Designation (ODD) in South Korea, where the term "Orphan Drug" is defined by the MFDS as therapies used to treat diseases affecting 20,000 or fewer patients and therapies used to treat diseases for which no appropriate therapy has been developed, or therapies that have demonstrated significant improvement in safety and/or efficacy compared to existing treatments.

A new mechanism of action with a broad therapeutic window

Selinexor’s novel mechanism of action (MoA) which is based on the selective inhibition of the nuclear export protein XPO1, is different from that of all currently approved therapies. The drug can be combined with various therapies to deliver improved treatment outcomes in these diseases. To date, five selinexor-based regimens have been added to the National Comprehensive Cancer Network (NCCN) Guidelines.

In addition, a number of international multicenter clinical trials in respect of selinexor are jointly carried out in places such as North America, Europe, Australia, Asia. These include the study of XPORT-DLBCL-030 in international multicenter clinical trials in combination with R-GDP for the treatment of rrDLBCL, and the study of SIENDO in international multicenter clinical trials of monotherapy of endometrial neoplasms.

Minyoung Kim, General Manager of Antengene, commented: "We are very encouraged by the MFDS’ approval of selinexor. I am confident that this oral selective inhibitor of nuclear export protein, with its practice-changing therapeutic utility, will improve the quality of life of patients with rrMM and rrDLBCL in South Korea, and bring renewed hope to this patient population."

About XPOVIO (selinexor)

XPOVIO is a first-in-class, oral Selective Inhibitor of Nuclear Export (SINE) compound. XPOVIO functions by selectively binding to and inhibiting the nuclear export protein exportin 1 (XPO1, also called CRM1). XPOVIO blocks the nuclear export of tumor suppressor, growth regulatory and anti-inflammatory proteins, leading to accumulation of these proteins in the nucleus and enhancing their anti-cancer activity in the cell. The forced nuclear retention of these proteins can counteract a multitude of the oncogenic pathways that, unchecked, allow cancer cells with severe DNA damage to continue to grow and divide in an unrestrained fashion.

LISCure Biosciences Announces Know-How License Agreement and Stock Purchase Agreement with Mayo Clinic

On August 1, 2021 LISCure Biosciences, Inc. ("LISCure") reported that it has entered into a know-how license and development collaboration agreement, and stock purchase agreement with Mayo Clinic to advance LISCure’s microbiome therapeutics in non-alcoholic steatohepatitis (NASH) (Press release, LISCure Biosciences, AUG 1, 2021, View Source [SID1234585495]).

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The two entities previously entered a research collaboration agreement in the first half of 2021 for NASH candidates.

Under the agreement, the company will receive Mayo Clinic’s know-how to establish a new drug development of NASH program. As part of the agreement, Mayo Clinic will become a shareholder in LISCure, the first Korean biotech company receiving equity investment from Mayo Clinic.

The two entities will pursue the common goal of maximizing the value of LISCure’s therapeutics in metabolic diseases by expanding research and development activities such as discovering new indications and additional value-added candidates. LISCure plans to minimize trial and error in preparation for phase 2 clinical trial of its NASH candidate in the US in 2022 by jointly cooperating with Mayo Clinic.

"This collaboration will provide innovative treatment opportunities to patients with high medical needs in various metabolic diseases. We are very excited to establish a collaboration with Mayo Clinic," said a company official. "We are also proud of Mayo Clinic becoming our shareholder and look forward to continuing a long-term collaboration."

LISCure plans to enter the global clinical trial for NASH candidate as well as auto-immune disease candidate in the second half of this year, and to initiate clinical trial in the US for the world’s first novel microbe-mediated cancer therapeutics in the first half of 2022.