On June 8, 2021 Astellas Pharma Inc. (TSE: 4503, President and CEO: Kenji Yasukawa, Ph.D., "Astellas") reported the presentation of new data in acute myeloid leukemia (AML) at the European Hematology Association (EHA) (Free EHA Whitepaper) virtual congress, taking place June 9-17 (Press release, Astellas, JUN 8, 2021, View Source [SID1234583695]).

Schedule your 30 min Free 1stOncology Demo!
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

                  Schedule Your 30 min Free Demo!

Eight Astellas-sponsored abstracts focused on patients with AML are being presented, comprising two oral presentations, four posters and two online-only abstract publications.

"We’re pleased to present new investigational research at EHA (Free EHA Whitepaper) 2021 that examines how patients and healthcare providers value different treatment approaches in AML," said Erhan Berrak, M.D., Vice President of Medical Affairs, Oncology, Astellas. "For example, one oral presentation sheds light on patients’ preferences for treatment, which may help to inform decisions of healthcare professionals when considering a plan for treatment after hematopoietic stem cell transplantation."

"Clinical trial results to be presented reflect our deep commitment to AML research, where we are investigating gilteritinib as monotherapy or in combination with other treatments, and across the range of patients with FLT3 mutation-positive AML, including patients whose AML is newly diagnosed or relapsed or refractory," said Andrew Krivoshik, M.D., Ph.D., Senior Vice President and Oncology Therapeutic Area Head, Astellas.

Oral Presentations
Oral presentations are available online from Friday, June 11 at 9 a.m. CEST, when all prerecorded presentations will be published on the virtual congress platform.

Title: Patient and Physician Preferences for Post–Hematopoietic Stem Cell Transplantation Maintenance Treatment of Acute Myeloid Leukemia (Abstract S313)

Presenting author: Manasee V. Shah, Astellas Pharma Inc., Northbrook, Ill., USA
Title: Efficacy and Safety of Venetoclax in Combination with Gilteritinib for Relapsed/Refractory FLT3-Mutated Acute Myeloid Leukemia: Updated Analyses of a Phase 1b Study (Supported by AbbVie, Astellas and Genentech) (Abstract S135)

Presenting author: Jessica K. Altman, Robert H. Lurie Comprehensive Cancer Center, Northwestern University, Chicago, Ill., USA
E-Poster Presentations
E-poster presentations are available from Friday, June 11 at 9 a.m. CEST, when the e-posters are published on the virtual congress platform.

Title: Follow-up of Patients with FLT3-Mutated Relapsed or Refractory Acute Myeloid Leukemia in the Phase 3 ADMIRAL Trial (Abstract EP438)

Presenting author: Mark J. Levis, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Md., USA
Title: Clinical Outcomes in Patients with Relapsed/Refractory Acute Myeloid Leukemia Treated with Gilteritinib Who Received Prior Midostaurin or Sorafenib (Abstract EP448)

Presenting author: Alexander E. Perl, Abramson Comprehensive Cancer Center, University of Pennsylvania, Philadelphia, Pa., USA
Title: Outcomes in Gilteritinib-Treated FLT3-Mutated R/R AML Patients Who Underwent Transplantation (Abstract EP441)

Presenting author: Alexander E. Perl, Abramson Comprehensive Cancer Center, University of Pennsylvania, Philadelphia, Pa., USA
Title: A Phase 1 Study of Gilteritinib in Combination with Induction and Consolidation Chemotherapy in Patients with Newly Diagnosed AML: Final Results Update (Abstract EP437)

Presenting author: Keith W. Pratz, Sidney Kimmel Comprehensive Cancer Center, Johns Hopkins University, Baltimore, Md., USA
Online-only abstract publications
Online-only abstracts are available via the virtual congress platform.

Title: Retrospective Assessment of Treatment Patterns and Resource Utilization for Patients Newly Diagnosed with Acute Myeloid Leukemia in Canada, UK, France, Germany, Italy, and Spain (Abstract PB1390)

Title: Frequency of FLT3-ITD and FLT3-TKD Mutations in Patients with Acute Myeloid Leukemia: A Systematic Literature Review and Meta-Analysis (Abstract PB1405)

The EHA (Free EHA Whitepaper) 2021 virtual congress abstracts are available in the EHA (Free EHA Whitepaper) Library.

About Acute Myeloid Leukemia (AML)
Acute myeloid leukemia (AML) is a type of cancer that affects the bone marrow and blood. It is deemed "acute," meaning that this type of leukemia can progress quickly.1 In the European Union, the incidence rate of AML is 3.7 per 100,000 per year, resulting in an estimated 16,800 individuals diagnosed.2

About Gilteritinib
Gilteritinib was discovered through a research collaboration with Kotobuki Pharmaceutical Co., Ltd., and Astellas has exclusive global rights to develop, manufacture and commercialize gilteritinib. Gilteritinib is available as XOSPATA in the U.S., Japan, China and selected European countries, among others, for the treatment of adult patients who have relapsed or refractory FLT3mut+ AML.3,4,5 Gilteritinib is an FMS-like tyrosine kinase 3 (FLT3) inhibitor with demonstrated activity against FLT3-ITD, a common driver mutation that presents with a high burden and poor prognosis, and FLT3-TKD mutations.6

European Union Important Safety Information
For important Safety Information for gilteritinib please see the full Summary of Product Characteristics at: View Source

United States Important Safety Information
For important Safety Information for gilteritinib please see Important Safety Information at: View Source

On June 8, 2021 Chugai Pharmaceutical Co., Ltd. (TOKYO: 4519) reported that it has been selected as one of the "Digital Transformation Stocks (DX Stocks) 2021" by the Ministry of Economy, Trade and Industry (METI) and the Tokyo Stock Exchange (TSE) (Press release, Chugai, JUN 8, 2021, View Source [SID1234583694]). From among TSE-listed companies, "DX Stocks" are designated to those companies, in each industrial category, that have established internal systems to promote digital transformation (DX) leading to increased corporate value, and have outstanding achievements in digital utilization. Chugai is selected for the second consecutive year as DX Stock.

Schedule your 30 min Free 1stOncology Demo!
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

                  Schedule Your 30 min Free Demo!

28 TSE-listed companies were selected in consideration of contribution to corporate value, and capability of DX realization. Under the positive commitment of top management, clear digital vision and investment policy, Chugai’s efforts to reform the drug discovery development process using AI etc., strengthen human resources and culture, and provide new value to patients were highly evaluated.

In order to realize its vision of becoming a top innovator in the healthcare industry, Chugai formulated a new growth strategy "TOP I 2030," which is consisted of two pillars of "global first-class drug discovery" and a "futuristic business model." DX is positioned as one of the key drivers to achieve the vision. In accelerating DX, Chugai is committed to transforming its business and providing healthcare solutions that will change society through three basic strategies of CHUGAI DIGITAL VISION 2030: 1) strengthening digital platforms, 2) optimize all value chains, and 3) digital transformation for drug discovery and development.

On June 7, 2021 C&Cure, a startup that possesses a bacterial platform for cancer treatment and radiopharmaceutical technology for diagnosis, reported the company developed a precision immunotherapy platform (SAM-04) using the characteristics of bacteria that cluster in tumors (Press release, CNCure, JUN 7, 2021, View Source [SID1234649032]). This technology activates surrounding immune cells and removes cancerous tissues when bacteria infiltrate the tumor.

Schedule your 30 min Free 1stOncology Demo!
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

                  Schedule Your 30 min Free Demo!

C&Cure has developed a radiopharmaceutical that can diagnose malignant melanoma and infections by utilizing radiolabeling technology as well as a bacterial platform for cancer treatment. A radiopharmaceutical for malignant melanoma diagnosis ([ 18 F] DMPY2) with a benzamide-based chemical skeleton structure detected malignant melanoma with ultrahigh sensitivity in small animal tumor and metastasis models. When comparing the degree of distribution in vivo with [ 18 F] FDG, which is currently the most widely used in clinical practice , [ 18 F] DMPY2 was found to have superior selectivity for detecting malignant melanoma and a significantly higher signal ratio in normal tissue compared to tumor tissue.

Also, radiopharmaceuticals for diagnosing fungal and gram-negative bacterial infections ( [ 18 F] FDS) are radiopharmaceuticals developed based on the fact that sorbitol is used as a nutrient for microorganisms. Positron emission tomography (PET) images can be used to rapidly and highly visualize fungal and gram-negative bacterial infections. Compared to existing diagnostic methods, it can quickly and non-invasively confirm infection, and it can even diagnose the location of infection with high sensitivity and specificity.

C&Cure has secured technology and resources for each platform, and has global competitiveness, and the total investment attracted to date has reached 9 billion won.

On June 7, 2021 Defence Therapeutics reported, after very encouraging preliminary in vivo results, the establishment of a collaboration with the HUS Comprehensive Cancer Center at Helsinki, Finland for the optimization of Defence’s Accum-T-DM1 ADC Therapeutic (Press release, Defence Therapeutics, JUN 7, 2021, View Source [SID1234626235]). This Cancer Center is Finland’s largest and most versatile cancer treatment center, their expertise is internationally highly valued and their treatment results are world-class.

Schedule your 30 min Free 1stOncology Demo!
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

                  Schedule Your 30 min Free Demo!

The parties entered into a collaboration for the preclinical selection of an optimized Accum-TDM1 conjugate based on in vitro assessments. These studies will not only highlight the additive effect mediated by Defence’s, Accum technology, it will also guide the selection of optimal Accum-T-DM1 for further in vivo testing on breast and gastric cancer animal models.

T-DM1 (Ado-trastuzumab, Kadcyla) approved by the FDA in 2013 to treat women with metastatic HER2-positive breast cancer surpassed $1 billion in annual sales in 2019 to become the first ADC to hit blockbuster status. The Kadcyla ADC antibody portion of this drug serves as a homing device to the chemotherapeutic drug emtansine. Clinical and pre-clinical studies have shown that intracellular drug delivery to breast cancer cells may not be optimal causing treatment resistance and recurrence.

Combining the Accum technology to the Kadcyla ADC is expected to amplify the therapeutic index of the drug while potentially minimizing side effects observed in patients undergoing the therapy. Defence’s Accum platform has been developed and tested in vitro to enhance the intracellular drug delivery on multiple ADCs that are FDA approved or under development.

"We have a long-standing experience in the field of ADC therapeutics, and we believe that the HUS Comprehensive Cancer Center represents an ideal collaborator for the ongoing development of the optimized Accum-T-DM1 conjugate," said Sebastien Plouffe, Chief Executive Officer of Defence Therapeutics. "We look forward to the continued advancement of our Accum-modified pipeline," he added.

Currently, the T-DM1 regiment consists of a 3-week treatment cycle for up to 14 cycles. By linking Defence’s Accum to T-DM1, the regiment will most likely be shorter due to the enhanced therapeutic effect of the ADC.

The Global cancer immunotherapy market is worth over $100 billion annually.

On June 7, 2021 Novartis flexed its clinical muscles over the weekend, reported that results from multiple studies across various indications, including different types of cancer and kidney disease (Press release, Novartis, JUN 7, 2021, View Source [SID1234583935]).

Schedule your 30 min Free 1stOncology Demo!
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

                  Schedule Your 30 min Free Demo!

Some of the oncology data were presented at the virtual 2021 Annual American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) meeting, including data from the ongoing Phase II GEOMETRY mono-1 study assessing Tabrecta in patients with EGFR wild-type, ALK-negative rearrangement, metastatic NSCLC harboring mutations that lead to MET exon-14 skipping.

The ASCO (Free ASCO Whitepaper) presentation includes new data from a treatment-naïve expansion cohort (7) and previously-treated cohort (6) and mature data from previously reported cohorts. Novartis said the latest data demonstrates Tabrecta’s effectiveness as a treatment option for patients with this particular form of lung cancer.

Data showed that Tabrecta, which was approved by the U.S. Food and Drug Administration last year, showed significant improvements in overall survival for this cancer type. The study revealed that Tabrecta provided 20.8 months of OS for treatment-naïve patients and 13.6 months for previously-treated patients.

Jeff Legos, Head of Oncology Drug Development for Novartis Oncology, touted the data as additional proof for the use of Tabrecta in this patient population.

"The introduction of Tabrecta a year ago dramatically changed the treatment landscape for patients with METex14 NSCLC. Now we have further evidence that Tabrecta… has the potential to help people live longer," Legos said in a statement.

In addition to OS, Novartis noted the median duration of response was 12.6 months among treatment-naïve patients in one of the cohorts. In two cohorts of previously treated patients, the DOR was 9.7 months and 8.4 months. The overall response rate was much higher in treatment naïve patients, 67.9% and 65.6% across two cohorts. For previously treated patients, ORR was 40.6% and 51.6% across two cohorts.

Esophageal Cancer

Also presented at ASCO (Free ASCO Whitepaper) was data from the pivotal Phase III RATIONALE 302 study that showed the company’s checkpoint inhibitor tislelizumab improved overall survival in patients with unresectable recurrent locally advanced or metastatic esophageal squamous cell carcinoma (ESCC) who had received prior systemic therapy. In the study, tislelizumab, an investigational anti-PD-1 immune checkpoint inhibitor, was assessed against chemotherapy.

The study showed tislelizumab extended median OS by 2.3 months compared to chemotherapy with a 30% reduction in the risk of death. In PD-L1 positive patients, tislelizumab extended median OS by 3.5 months with a 46% reduction in the risk of death.

Legos expressed his excitement about the Phase III study. He said data shows tislelizumab has the potential to help patients with esophageal squamous cell carcinoma live longer. Legos said data from the study would be shared with regulatory agencies.

Chemotherapy did best tislelizumab when it came to progression-free survival. Treatment with tislelizumab demonstrated median progression-free survival of 1.6 months compared to 2.1 months for chemotherapy. Tislelizumab showed a higher and more durable anti-tumor activity than chemotherapy, 20.3% vs. 9.8%, and a greater median duration of response, 7.1 months compared to 4 months.

Prostate Cancer

Data from the Phase III VISION study showed Lu-PSMA-617, a targeted radioligand therapy, plus best standard of care (SOC), demonstrated significant improvement in overall survival in patients with advanced PSMA-positive metastatic castration-resistant prostate cancer (mCRPC). Lu-PSMA-617 plus SOC was evaluated against SOC alone.

Novartis said the OS difference between the Lu-PSMA-617 combination and SOC was statistically significant, with an estimated 38% reduction in risk of death in the 177Lu-PSMA-617 arm. Data from the Phase III VISION study was presented at ASCO (Free ASCO Whitepaper) over the weekend.

Patients who received Lu-PSMA-617 also demonstrated a statistically significant 60% risk reduction for radiographic progression-free survival or death, compared to the best standard of care only arm.

John Tsai, Head of Global Drug Development and Chief Medical Officer for Novartis, said the VISION study is the first to demonstrate the potential of a radioligand therapy in advanced prostate cancer. Novartis is planning to assess Lu-PSMA-617 in earlier line treatment settings for metastatic prostate cancer. The company will investigate the radioligand therapy in the mCRPC pre-taxane setting (PSMAfore) and the metastatic hormone-sensitive setting (PSMAddition).

Kidney Disease

In kidney disease, Phase II data showed the investigational asset iptacopan reduced protein in the urine (proteinuria), which is a surrogate marker correlating with progression in kidney failure. It also showed promise in stabilizing kidney function in patients with IgA nephropathy (IgAN), Novartis said. Iptacopan is a first-in-class, oral, targeted factor B inhibitor that targets the alternative complement pathway, a key driver of complement-driven renal diseases.

In the Phase II IgAN study presented at the ERA-EDTA Congress, iptacopan met its primary endpoint by demonstrating a statistically significant dose-response effect on reduction in proteinuria compared to placebo after 90 days. A 200mg dose of iptacopan twice per day achieved a 23% reduction in proteinuria.

Iptacopan also demonstrated a trend towards stabilizing kidney function. Additionally, iptacopan showed a favorable safety and tolerability profile, the company said.

"Complement-driven renal diseases, such as IgAN, are devastating and mostly affect young adults, imposing a high disease burden. These new data in IgAN add to the growing body of evidence around the potential of iptacopan to target a key driver in these rare renal diseases," Tsai said in a statement.

Iptacopan is also in development for the blood disorder paroxysmal nocturnal hemoglobinuria.