On March 23, 2021 AC Immune SA (NASDAQ: ACIU), a clinical-stage biopharmaceutical company pioneering precision medicine for neurodegenerative diseases, reported its financial results for the year ended December 31, 2020 (Press release, AC Immune, MAR 23, 2021, View Source [SID1234577010]). The Company also provided an overview of its execution strategy and anticipated clinical and preclinical milestones for 2021, as well as the strong progress being made across its broad portfolio of therapeutic and diagnostic product candidates.

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Prof. Andrea Pfeifer, CEO of AC Immune SA, commented: "We began 2021 with strong momentum based on the effective execution of our multi-pronged clinical development growth strategy. This is exemplified by our anti-pTau vaccine, which recently demonstrated highly potent immune responses against pathological Tau and remarkable safety in patients with early Alzheimer’s disease (AD). We are also creating future value by accelerating development of our proprietary, first-in-class candidates addressing novel targets in neurodegeneration, such as our promising alpha-synuclein PET tracer, which will generate initial clinical results this year, and our highly valued programs targeting the NLRP3 inflammasome. Our strong track record shows that expanding our efforts to advance these key early-stage programs may lead to multiple future opportunities for strategic partnership as well as in-house clinical development for select indications. In parallel, we continue to collaborate with our global partners to advance our later-stage clinical programs toward key inflection points. Looking forward in 2021, we expect to build upon our successes and continue innovating as a leader in precision medicine for neurodegenerative disease."

2020 and Q1 2021 Research & Development Highlights

Clinical Pipeline advancement

Reported promising interim Phase 1b/2a results for ACI-35.030, a novel anti-phospho-Tau (pTau) vaccine candidate showing strong safety and high titers of antigen-specific antibodies in 100% of older patients with early AD. The study is currently enrolling patients into the highest dose group, with further clinical readouts expected this year.
Advanced next-generation alpha-synuclein positron emission tomography (PET) tracer candidate, ACI-12589, into a first-in-human clinical study, with an expected data readout in Q3 2021
Reported topline Phase 2 results for semorinemab, the Company’s investigational monoclonal anti-Tau antibody candidate for the treatment of prodromal to mild AD, partnered with Genentech, a member of the Roche Group. These represent the first-ever Phase 2 results for an anti-Tau antibody therapeutic in AD. Primary completion of the second Phase 2 study in moderate AD patients is expected in Q2 2021.
Completed a Phase 1 clinical study in healthy volunteers for ACI-3024, an oral small molecule Morphomer Tau aggregation inhibitor, which achieved target brain exposure. The Companies have decided to pursue other promising Tau Morphomer candidates from AC Immune’s research platform for potential clinical development in AD. ACI-3024 will be further evaluated for efficacy in models of rare Tauopathies.
Partnership milestone payments and grants

Received a CHF 10 million milestone payment from Eli Lilly and Company related to ACI-3024
Amended the collaboration agreement with Lilly for Tau Morphomers to include a new CHF 60 million Phase 2 milestone payment, which increased the total potential deal value by CHF 40 million to CHF 1.86 billion
Received multiple prestigious and highly competitive grants in 2020 focused on acceleration of the Company’s proprietary and potentially game-changing diagnostic programs
Won Ken Griffin Alpha-synuclein Imaging Competition from The Michael J. Fox Foundation for Parkinson’s Research (MJFF) and is able to receive together with its clinical partner USD 3.2 million (CHF 3.1 million) to support AC Immune’s alpha-synuclein-PET tracers
Awarded a EUR 1.45 million grant to support the partnership between AC Immune and the EU Joint Programme – Neurodegenerative Disease Research (JPND) ImageTDP-43 Consortium to advance its first-in-class TAR DNA-binding protein 43 (TDP-43) PET tracers
Awarded a USD 600,000 grant from Target ALS to develop novel immuno-assays to detect pathological TDP-43 in cerebrospinal fluid (CSF) and blood based on AC Immune’s SupraAntigen-derived anti-TDP-43 antibodies
Strengthening of Management and Board

Appointed Prof. Johannes R. Streffer, former UCB Biopharma Head of Translational Neuroscience, to the new role of Chief Medical Officer
Welcomed Prof. Carl H. June, world authority on immune tolerance and adoptive immunotherapy, to the Company’s Board of Directors
Appointed renowned Neurologist with a specific focus in the emerging field of Down syndrome (DS)-related Alzheimer’s disease, Dr. Juan Fortea to AC Immune’s Clinical Advisory Board
Future Value Creation

Reported key advancements for several therapeutic programs targeting the NLRP3 inflammasome, including small molecule inhibitors, which showed the first evidence of in vivo activity in a model of peripheral inflammation, as well as high-affinity monoclonal antibodies that bind extracellular components of the (NOD)-like receptor protein 3 (NLRP3) pathway and inhibit inflammasome-mediated immune response in vitro
Identified and characterized the first biologically active small molecule Morphomer alpha-synuclein aggregation inhibitors, which significantly decreased alpha-synuclein aggregate formation in cellular assays by interfering with the fibrillation process
Strengthened strategic partnership with WuXi Biologics to accelerate advancement of TDP-43 antibody into clinical development for NeuroOrphan indications
2021 execution strategy to maximize value creation

AC Immune’s execution strategy is focused on three key initiatives, which support the Company’s overarching goal of enabling precision medicine for neurodegenerative diseases:

The Company plans to accelerate the development of its late-stage therapies in AD in collaboration with its strategic partners, including its novel pTau vaccine with Janssen Pharmaceuticals Inc., which continues to show great promise.
AC Immune is sharpening its strategic focus on non-AD indications with high unmet need. Currently this includes its anti-Abeta vaccine in people with DS, as well as its therapeutic and diagnostic candidates targeting TDP-43 and alpha-synuclein, where the Company may focus in-house efforts on select NeuroOrphan indications while seeking potential partnerships for larger indications like LATE (limbic-predominant age-related TDP-43 encephalopathy) and Parkinson’s disease (PD). Furthermore, AC Immune’s NLRP3 inflammasome-targeted programs have broad applicability both within central nervous system (CNS) and non-CNS indications.
The Company plans to accelerate advancement of its diagnostic candidates to late-stage development, as continued leadership in precision medicine is a key differentiator for AC Immune. These candidates include its Tau, alpha-synuclein, and TDP-43 PET tracers, which potentially enable earlier disease diagnosis, improved clinical trial outcomes and additional revenue generation for the Company.
Anticipated 2021 milestones
Clinical Milestones

ACI-35.030 anti-pTau vaccine: reported Phase 1b/2a in AD interim results in Q1 (second highest dose); further Phase 1b/2a interim analysis in Q4 (highest dose)
JACI-35.054 alternative anti-pTau vaccine: Phase 1b/2a in AD interim analysis in Q2 (low dose)
Alpha-synuclein imaging agent: advanced third-generation candidate to first-in-human clinical study in Q1; readout expected in Q3
ACI-24 anti-Abeta vaccine in DS: reported Phase 1b top line results in Q1; to present further study results at the Alzheimer’s Association International Conference 2021 in Q2
ACI-24 in AD: reported Phase 2, 12-month interim analysis in Q1; 18-month interim analysis in Q2
Semorinemab anti-Tau antibody: Phase 2 trial primary completion (estimated last patient, last visit) in moderate AD in Q2
ACI-3024 small molecule Morphomer Tau aggregation inhibitor: select NeuroOrphan indication for further development in Q2
ACI-24 in DS: submit investigational new drug (IND) application for optimized vaccine formulation in Q4
Preclinical Milestones

Alpha-synuclein small molecule inhibitor: identified first biologically active small molecule in Q1; start in vivo proof-of-concept studies in Q3
TDP-43 imaging agent: initiate investigational new drug (IND)-enabling studies in Q3
Morphomer NLRP3-ASC: report in vivo proof-of-concept results in a non-CNS disease model and begin in vivo proof-of-concept studies with validated candidate in CNS in Q4
Anti NLRP3-ASC antibody: begin in vivo proof-of-concept studies in Q4
Anti-TDP-43 antibody: initiate IND-enabling toxicology studies in Q4
TDP-43 biofluid diagnostic: establish validation-ready assay in Q4
Therapeutic and Diagnostic Pipeline Overview

AC Immune also provided a comprehensive overview highlighting strong progress across its clinical and preclinical development pipeline. This supplemental material can be viewed and downloaded in the investor section of the Company’s website.

Analysis of Financial Statements for the year ended December 31, 2020

Cash Position: The Company had a total cash balance of CHF 225.9 million, comprised of CHF 160.9 million in cash and cash equivalents and CHF 65 million in short-term financial assets. This compares to a total cash balance of CHF 288.6 million as of December 31, 2019. The decrease of CHF 62.7 million is principally due to continued investments in our R&D pipeline. The total shareholders’ equity position decreased to CHF 215.5 million from CHF 272.4 million as of the prior year. The Company’s cash balance provides enough capital resources to progress through at least Q1 2024 without potential incoming milestone payments.
Contract Revenues: Contract revenues for the year ended December 31, 2020 totaled CHF 15.4 million compared to CHF 110.5 million in 2019, representing a CHF 95 million decrease. The Company recognized a CHF 10 million milestone and CHF 4.3 million for research and development activities in 2020 from its Lilly agreement compared to CHF 103.1 million for an upfront payment and milestone and CHF 2.6 million for research and development activities in 2019.
R&D Expenditures: R&D expenses increased by CHF 9.1 million for the year ended December 31, 2020.
Discovery and preclinical expenses: The Company increased expenditures across a variety of its discovery and preclinical programs. These include investments to advance the second generation of our ACI-24 for Down Syndrome project, the initiation of IND-enabling studies of our anti-TDP-43 antibody project and various other investments across our alpha-synuclein and neuroinflammation programs.
Clinical expenses: The Company also increased expenditures across multiple Clinical programs. These include investments to prepare a follow-on trial for our Abeta vaccine for Down Syndrome project, additional enrollment costs for the Phase 1b/2a study for ACI-35.030 and a full year of clinical activities to complete the Phase 1 of our Morphomer Tau asset in partnership with Lilly.
Salary- and benefit-related costs: The Company’s salary- and benefit-related costs increased by CHF 2.7 million, primarily due to the addition of 13 FTEs, annualization of 2019 hires and increases in share-based compensation.
G&A Expenditures: For the year ended December 31, 2020, G&A increased by CHF 2.5 million to 18.6 million. Of this increase, CHF 1.7 million is due to salary- and benefit-related costs, primarily due to the addition of 3 FTEs, annualization of 2019 hires and increases in share-based compensation. Additionally, the Company incurred a CHF 0.8 million increase in other G&A expenses, predominantly associated with depreciation expense, insurance and professional fees
IFRS Income/(Loss) for the Period: The Company reported a net loss after taxes of CHF 61.9 million for the year ended December 31, 2020, compared with net income of CHF 45.4 million for 2019
2021 Financial Guidance

For the full year 2021, the Company expects its total cash burn to range between CHF 65 million ‒75 million.

On March 23, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it obtained approval from the Ministry of Health, Labour and Welfare (MHLW) for the anticancer agent/antimicrotubule binding anti-CD79b monoclonal antibody Polivy intravenous infusion 30mg and 140mg [generic name: polatuzumab vedotin (genetical recombination)] in combination with bendamustine (freeze-dried formulation) and rituximab (BR therapy) for the treatment of relapsed or refractory (R/R) diffuse large B-cell lymphoma (DLBCL) (Press release, Chugai, MAR 23, 2021, View Source [SID1234577005]).

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"I am very pleased that Polivy in combination with BR therapy now can be offered to patients as a new treatment option for R/R DLBCL, a disease with high unmet medical needs, in the hematologic cancer field following Rituxan and Gazyva," said Chugai’s President and CEO Dr. Osamu Okuda. "We are preparing to bring this first-in-class anti-CD79b antibody-drug conjugate (ADC) to patients so that we may contribute to realize a better treatment."

The approval is based on data including the results from a multicenter overseas phase Ib/II clinical study (GO29365) that evaluated the efficacy and safety of Polivy in combination with BR therapy compared to BR therapy alone, and a multicenter, single-arm Japanese phase II study (JO40762/P-DRIVE study) that evaluated the efficacy and safety of the combination therapy in R/R DLBCL.

The efficacy and safety of Polivy and BR therapy (40 patients) compared with BR therapy alone (40 patients) was studied in the randomized phase II part of the GO29365 study in 80 patients with R/R DLBCL not eligible for autologous stem cell transplantation (ASCT). The primary endpoint of the complete response rate (CRR) at the time point of primary response assessment (PRA; 6 to 8 weeks after last dose of Polivy) as evaluated by an independent assessment committee using positron emission tomography-computed tomography (PET-CT) was 40% (16/40 patients; 95% CI: 24.9-56.7%) in the Polivy + BR therapy group, and 17.5% (7/40 patients; 95% CI: 7.3-32.8%) in the BR therapy group (data cut-off: April 30, 2018). Adverse reactions occurred in 36 (92.3%) patients out of 39 patients who received Polivy. The most common adverse reactions were neutropenia 53.8% (21/39 patients), thrombocytopenia 41.0% (16/39 patients), diarrhea and anemia 33.3% (13/39 patients) each, fatigue and nausea 23.1% (9/39 patients) each, and pyrexia and peripheral neuropathy 20.5% (8/39 patients) each.

In the P-DRIVE study, the efficacy and safety of Polivy + BR therapy were studied in 35 patients with R/R DLBCL not eligible for ASCT. The primary endpoint of the CRR at the PRA as assessed by the principal investigator using PET-CT was 34.3% (12/35 patients), (95% CI: 19.1-52.2%) (data cut-off: December 24, 2019). Adverse reactions occurred in 33 (94.3%) patients out of 35 patients who received Polivy. The most common adverse reactions were anemia 37.1% (13/35 patients), nausea 31.4% (11/35 patients), thrombocytopenia and neutropenia 25.7% (9/35 patients) each, constipation, decreased platelet count and decreased neutrophil count 22.9% (8/35 patients) each, and malaise and decreased appetite 20.0% (7/35 patients) each.

A double-blind, placebo-controlled global phase III study (GO39942/POLARIX study) is ongoing for untreated DLBCL to compare the efficacy and safety of Polivy in combination with rituximab plus cyclophosphamide, doxorubicin, prednisolone (R-CHP) to rituximab plus cyclophosphamide, doxorubicin, vincristine, prednisolone (R-CHOP).

[Reference information]
Polatuzumab Vedotin Achieved Primary Endpoint in the Japanese Phase II study for Relapsed or Refractory Diffuse Large B-cell Lymphoma (Press release issued by Chugai on February 13, 2020)
View Source

European Commission approves Roche’s Polivy for people with previously treated aggressive lymphoma (Press release issued by Roche on January 21, 2020)
View Source

Approval information

Product name: Polivy Intravenous Infusion 30mg, Polivy Intravenous Infusion 140mg

Generic name: polatuzumab vedotin (genetical recombination)

Intended uses or indications: Relapsed or refractory diffuse large B-cell lymphoma

Dosage and administration:
The usual adult dosage is 1.8mg/kg (body weight) polatuzumab vedotin (genetical recombination) administered by intravenous infusion every 3 weeks for 6 doses, in combination with bendamustine hydrochloride and rituximab (genetical recombination). If the first infusion is well tolerated after 90 minutes, subsequent infusions may be administered over a shorter time of at least 30 minutes. Reduce the dose as necessary in accordance with the patient’s condition.

About GO29365 study1)
GO29365 is a global, phase Ib/II study evaluating the safety and tolerability of Polivy in combination with bendamustine and rituximab (BR therapy) or obinutuzumab (BG therapy) in R/R follicular lymphoma or DLBCL. In the phase II randomized part of the study with 80 DLBCL patients, the efficacy and safety of Polivy in combination with BR therapy were studied compared to BR therapy alone. The primary endpoint was complete response at the point of primary response assessment as evaluated by an independent assessment committee using PET-CT. Patients received six cycles of treatment, spaced three weeks apart.

About JO40762 (P-DRIVE) study
JO40762 (P-DRIVE) is an open label, single-arm study investigating Polivy in combination with BR therapy in 35 patients with R/R DLBCL. Primary endpoint is investigator’s assessment of CRR by PET-CT at the timing of primary response assessment. Patients received six cycles of treatment, spaced three weeks apart.

About polatuzumab vedotin
Polatuzumab vedotin was developed by Roche using Seattle Genetics’ ADC technology. It is a first-in-class anti-CD79b antibody-drug conjugate (ADC), comprising the anti-CD79b humanized monoclonal antibody and a tubulin polymerization inhibitor attached together using a linker. The CD79b protein is expressed specifically in the majority of B-cells, making it a promising target for the development of new therapies2,3). Polatuzumab vedotin binds to CD79b and destroys these B-cells through the delivery of an anti-cancer agent, which is thought to suppress the effects on normal cells4,5). Polatuzumab vedotin was granted accelerated approval in the US in June 2019 and conditional marketing authorization in the EU in January 2020, respectively.

About diffuse large B-cell lymphoma (DLBCL)
DLBCL is one of the histologic subtypes of non-Hodgkin’s lymphoma (NHL), which is categorized as an aggressive disease that progresses on a monthly basis. DLBCL is the most common form of NHL, accounting for 30-40 percent of NHL6-8). DLBCL frequently occurs in middle-aged and older people, mainly in their 60’s9). The median age at diagnosis has been reported to be 6410).

The combination of rituximab and chemotherapy is the standard therapy for untreated DLBCL; however, recurrence has been observed in about 40% of patients due to insufficient therapeutic effect11). In addition, although autologous stem cell transplantation (ASCT) is recommended for eligible patients with recurrent or refractory DLBCL, ASCT cannot be performed in about half of these patients due to failure of salvage chemotherapy prior to ASCT12). Furthermore, no standard therapy has been established for patients ineligible for ASCT due to reasons including age or complications13). Therefore, more useful new treatment options for relapsed or refractory DLBCL are in great need.

Salvage chemotherapy: Salvage chemotherapy or salvage therapy is used to treat patients with hematologic malignancy who experienced no therapeutic effects (refractory), or recurrence/relapse of the disease. Applicable treatment may vary depending on the type of cancer. Combination therapies of multiple drugs including anticancer agents14) are generally used.

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

On March 23, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has obtained approval from the Ministry of Health, Labour and Welfare (MHLW) for FoundationOne Liquid CDx Cancer Genomic Profile as the liquid biopsy (LB) test that provides comprehensive genomic profiling (CGP) for solid tumors on March 22, 2021 (Press release, Chugai, MAR 23, 2021, View Source [SID1234577004]). The test was also approved 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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"The approval of FoundationOne Liquid CDx Cancer Genomic Profile, a blood-based CGP option, has a significant meaning for Chugai that aims to advance personalized healthcare based on the genomic profile of a patient’s tumor," said Chugai’s President and CEO, Dr. Osamu Okuda. "In cancer treatment that continues to evolve day-to-day, it enables us to support a wider range of patients to help inform treatment decisions aligning the patient’s status and stage of treatment by utilizing both tissue-based and blood-based tests. We are committed to preparing for the launch of the test as soon as possible and also will continue to expand companion diagnostic functions with the aim of maximizing the value provided to patients."

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 from advanced cancer patients with solid tumors. It identifies genomic alterations in 324 cancer-related genes through detection of blood circulating tumor DNA (ctDNA) in blood. 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 realize advanced 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 dimaleate, 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
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.
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 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.

On March 23, 2021 EORTC reported that GLIOBLASTOMAS represent almost 50% of malignant primary brain tumors in adults, and range among the most lethal cancer types (Press release, EORTC, MAR 23, 2021, https://www.eortc.org/blog/2021/03/23/surviving-glioblastoma-eortc-brain-tumour-group-explores-the-factors-that-contribute-to-long-term-survival/ [SID1234577003]). Approximately three in 100,000 people a year are diagnosed with a glioblastoma. These tumors mainly affect patients in later life, but can occur in younger adults and even in children. Due to the aggressive nature of this tumor, which invades the brain by infiltration and destroys healthy brain tissue, glioblastomas lead to a significantly reduced life span with sometimes considerable loss of quality of life. Almost half of the patients die in the first year of diagnosis, despite the use of a broad therapeutical approach including brain surgery, radiotherapy and sometimes several courses of chemotherapy. Yet, a small percentage of up to 5% of all patients suffering from glioblastoma may survive for more than five years. These patients are referred to as long-term survivors.

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The reasons leading to this survival benefit in this heterogeneous patient group have not been fully identified so far. These patients are now the focus of EORTC 1419, also known as ETERNITY, a large international comprehensive study, assessing potential clinical and biological factors of this long-term survival that may contribute to improved survival in glioblastoma patients in general. Considering the rareness of glioblastoma long-term survival, a multicenter approach, involving a large number of clinical centers collecting patient data, is required. This EORTC study was made possible by a generous grant of US $2,000,000 by the Brain Tumor Funders’ Collaborative (BTFC). The BTFC is a strategic partnership of five private philanthropic and advocacy organisations in the US and Canada: the American Brain Tumor Association, Brain Tumour Foundation of Canada, Children’s Brain Tumor Foundation, James S. McDonnell Foundation, and the Sontag Foundation. EORTC 1419 comprises 33 leading centers for neuro-oncology worldwide, including centers in Europe, Australia and the US.

The participating medical researchers collect extensive clinical data from over 400 patients who survived their disease for more than five years. They record additional information including patient histories and health-related data, and perform extensive neurocognitive assessments to allow for a better understanding of the implications of the disease as well as the therapies in affected patients. Tumor tissue and blood samples from these patients will be collected to study the genetic and immunologic features of glioblastomas. Moreover, an analysis of all neuroimaging studies available from the selected patient group will be performed, assessing tumor growth patterns and development by different imaging tools. All the acquired information will later be compared to the data set of a reference cohort of glioblastoma patients who have not become long-term survivors. The collected clinical data will be inserted in a large database for further comparative analysis. In parallel, all available tissue samples will be catalogued in a central biobank in Germany, and sorted for the planned molecular and genetic investigations. The knowledge gained from this study will allow for a better understanding of the disease, and should help develop better treatment strategies for all glioblastoma patients in the future (contact: [email protected], [email protected]).

On March 23, 2021 Taiho Pharmaceutical Co., Ltd. (hereinafter "Taiho") reported that it has submitted to the Japanese Ministry of Health, Labour and Welfare a new drug application for an NK1 receptor antagonist antiemetic drug (development code: Pro-NETU; generic name: fosnetupitant chloride hydrochloride; hereinafter "fosnetupitant") for gastrointestinal symptoms (nausea and vomiting) associated with cancer chemotherapy (Press release, Taiho, MAR 23, 2021, View Source [SID1234577002]).

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Fosnetupitant is an NK1 receptor antagonist antiemetic drug developed for the prevention of chemotherapy-induced nausea and vomiting. It is a phosphorylated pro-drug preparation (injection) of netupitant, the active component. The prevention of chemotherapy-induced nausea and vomiting is considered important in clinical practice. Taiho acquired exclusive development and marketing rights for fosnetupitant in Japan under a license agreement signed in April 2011 with Helsinn Healthcare SA and has conducted clinical trials in Japan since then.

The new drug application is based on the results of a Phase III clinical trial comparing the efficacy and safety of fosnetupitant and fosaprepitant (NK1 receptor antagonist) in patients receiving highly emetogenic chemotherapy. In the trial, either fosnetupitant (235 mg) or fosaprepitant (150 mg) was administered in a single I.V. dose prior to administration of the chemotherapeutic agent, in combination with palonosetron (5-HT3 receptor antagonist) and dexamethasone. Details of the trial results will be presented at a future medical conference.

Within Taiho’s mainstay field of oncology, the company is also focusing on cancer supportive care. Under a former distribution and license agreement effective as of January 2004 with Helsinn Healthcare SA, Taiho has been marketing the 5-HT3 receptor antagonist Aloxi (generic name: palonosetron hydrochloride) in Japan since April 2010.

Taiho will work to secure approval of fosnetupitant, aiming to deliver the agent as soon as possible as a new treatment which can contribute to improving quality of life for patients, thereby contributing to comprehensive care in cancer.