On September 8, 2020 The CIBMTR (Center for International Blood and Marrow Transplant Research) reported that it has reached a study enrollment goal 2 years early, and 1,500 patients have now enrolled in the post-marketing safety study for Yescarta (axicabtagene ciloleucel) (Press release, Kite Pharma EU, SEP 8, 2020, View Source [SID1234564773]). The study is part of a collaboration with Kite, a Gilead Company.

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!

This collaboration between the CIBMTR and Kite tracks long-term outcomes of patients treated with Yescarta, a chimeric antigen receptor T-cell (CAR T) therapy. This study will generate real-world evidence to assess long-term safety and effectiveness. Enrollment is the first step in the study; next, researchers will follow the participants for at least 15 years. The U.S. Food and Drug Administration (FDA) has required this long-term study for Yescarta.

Yescarta is the first CAR T therapy approved by the FDA for the treatment of adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma (DLBCL) not otherwise specified, primary mediastinal large B-cell lymphoma, high grade B-cell lymphoma, and DLBCL arising from follicular lymphoma. Yescarta is not indicated for the treatment of patients with primary central nervous system lymphoma.

"This prospective study was created to meet a post-marketing requirement for this therapy, and the accrual completion is an important milestone," said Marcelo Pasquini, MD, MS, co-lead investigator and Senior Scientific Director of the CIBMTR; Cellular Therapy Registry Director; and Professor of Medicine, Division of Hematology / Oncology at the Medical College of Wisconsin. "Together, the CIBMTR and Kite are the first to have reached our target enrollment, and we are pleased to have done so ahead of our initial projections."

"Even though we’ve met our initial goal for this study, we continue to enroll people in the CIBMTR’s Cellular Immunotherapy Data Resource (CIDR). The CIBMTR will continue to reimburse medical centers for reporting cell therapy data," said Dr. Pasquini. The CIDR is part of a federal initiative to accelerate cancer research; medical centers and pharmaceutical companies can participate.

Interim and final study results will be shared with the FDA, and eventually, the public. For more information about the CIBMTR’s Cellular Immunotherapy Data Resource, visit cibmtr.org/About/WhatWeDo/CIDR.

On September 8, 2020 Median Technologies (ALMDT) (Paris:ALMDT), The Imaging Phenomics Company, reported the promising results of a first retrospective study on the implementation of the iBiopsy platform for the analysis of patients with solid tumor cancer who are likely to be responsive to immuno-oncology treatments such as those based on immune checkpoint inhibitors (ICIs) (Press release, MEDIAN Technologies, SEP 8, 2020, View Source [SID1234564772]).

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!

These results relate to one of three clinical development plans for the iBiopsy imaging biomarker discovery platform, which integrates cutting-edge artificial intelligence technologies. The various clinical development plans for iBiopsy were previously communicated on April 20.

The mode of action of immuno-oncology is to stimulate the immune system in cancer patients to elicit an immune response that destroys cancer cells. Immunotherapy, which brings together several categories of molecules already on the market or under development, is a very big trend in the therapeutic arsenal in oncology and today represents real hope for patients. However, only 20 to 40% of patients respond to these therapies1. In these small populations, and despite the side effects that can be extremely penalizing (toxicity), the success rates are then high.

In clinical routine, the implementation of the different types of immuno-oncological treatments are based on the identification of patients who are likely to respond. This is also the case in the context of drug development, where stratification of patients to be included in clinical trials is vital to ultimately increase the chances of successful commercialization of immuno-oncology treatments. The identification of susceptible responder patients is currently based on immunohistopathological tests and specific tumor genetic tests (companion diagnostics of the molecules used) derived from tumor biopsies, an invasive procedure with sampling problems. The non-invasive identification of responder / non-responder patients to immunotherapies is a public health issue for patients, payers and the pharmaceutical industry.

The infiltration of CD8+ cells into tumor tissue plays a key role in building anti-tumor immunity. The retrospective study conducted by Median Technologies used CT images of a cohort of 44 patients with primary liver cancer. The objective of this study was to combine the extraction of visual signatures and the construction of a deep learning model optimizing the prediction of the infiltration of CD8+ cells in the tumor microenvironment. In the study conducted by Median, the expression of the CD8 gene was verified by biopsy and was used to quantify CD8+ cells in the tumor microenvironment and for training the predictive model of iBiopsy. It was thus shown that in this cohort, the CD8+ signature of iBiopsy compared to that obtained by traditional radiomic methods2 was a better predictor of the tumor microenvironment with AUC 0.93 vs AUC 0.67 respectively. Finally, iBiopsy CD8+ signature was also shown to be predictive of patient prognosis.

This non-invasive predictive biomarker shows promise in predicting the immune phenotype of tumors and in evaluating the effectiveness of anti-PD-1 and PD-L1 treatments for cancer patients. These results need to be confirmed in larger independent patient cohorts.

"These first results are extremely promising and demonstrate, once again, the relevance of our iBiopsy approach, which allows for AI technologies to extract the content hidden in standard medical images and to generate non-invasive biomarkers. iBiopsy makes it possible to quantify in real time the tumor microenvironment and in particular the immune signature relevant to immunotherapy approaches," said Fredrik Brag, CEO and co-founder of Median. "The identification of predictive biomarkers of the response to immuno-oncology treatments has the potential to significantly improve the management of a large population of cancer patients, and so far, not one solution has done a good job addressing this need. These predictive biomarkers are the foundation of precision medicine. For pharmaceutical companies, the stakes are also colossal: between 2017 and 2019, the number of molecules under development in immuno-oncology increased by 91% worldwide and more than 5,200 active clinical trials in immuno-oncology are currently listed in the global trials database clinicaltrials.gov.3"

About iBiopsy: Based on the most advanced AI technologies and with expertise in data science, Median’s iBiopsy proprietary imaging platform allows for the extraction of non-invasive imaging biomarkers, which are the disease "signatures". These biomarkers, obtained from standard medical imaging modalities are used both in the field of clinical development and clinical routine in which medical needs regarding disease detection, treatment options and follow-up of patients are still unmet and have yet to foster the promise of predictive and precision medicine. Several indications are already targeted for liver diseases (NASH and HCC) and for the use of immuno-oncology drugs.
Median’s iBiopsy development program is supported by the European Investment Bank (EIB) through a financial loan of €35 million under the Juncker Plan, the European Fund for Strategic Investments, which aims to support research and innovation projects developed by companies with high growth potential.

On September 8, 2020 VECT-HORUS, a biotechnology company that designs and develops vectors that facilitate the targeted delivery of therapeutic molecules and imaging agents, reported that it has raised €6.7 million (Press release, Vect-Horus, SEP 8, 2020, View Source [SID1234564771]). In this new round of financing participated historic shareholders and also new entrants . It will strengthen the company’s equity capital, enabling it to develop its programs and the upcoming clinical trial of its first theragnostic agent (contraction of the terms "therapeutic" and "diagnostic").

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 proceeds of this new round of financing will enable the company to increase its R&D investments in order to optimize its VECTrans technology and, more specifically, to identify new vectors that will expand its portfolio. It will also enable the development of new conjugates associating the company’s vectors with therapeutic biomolecules: antibodies, mRNAs and various antisense oligonucleotides, including siRNAs. Therefore, the vectorization potential of the VECTrans platform will be significantly enriched in order to achieve its targeted therapy objectives in multiple organs and indications.

Moreover, with its co-development partner RadioMedix (a company based in Houston USA and specialized in nuclear medicine), VECT-HORUS is preparing the pre-IND dossier of its theragnostic agent for clinic trials in 2021 for the diagnosis of glioblastoma.

Finally, several collaboration agreements have been signed or are currently under negotiation with various pharmaceutical partners. The company aims to balance its R&D programs with an increasing flow of revenues from these industrial partnerships.

"This new chapter in our growth will enable our company to strengthen its ties with the pharmaceutical industry in the field of nervous system pathologies and also in other areas, with a view to developing targeted therapy to other organs", stated Alexandre Tokay, Chairman of VECT-HORUS.

VECT-HORUS is consolidating its growth via a mixed business model, based on its proprietary VECTrans technology, both to make its technology platform available to pharmaceutical and biotechnology company partners and to develop an internal pipeline of products, which are growth drivers for the company.

On September 8, 2020 Imago BioSciences, Inc. ("Imago"), a private clinical stage biopharmaceutical company developing innovative treatments for myeloid diseases, reported that Hugh Young Rienhoff, Jr. M.D., chief executive officer, will present a corporate overview at two upcoming investor conferences (Press release, Imago BioSciences, SEP 8, 2020, View Source [SID1234564770]).

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!

Citi’s 15th Annual BioPharma Virtual Conference
Date: Tuesday, September 8th 2020
Time: 2:20 p.m. ET
Logistics: If you’d like to register and attend the company presentation, click here to learn more.
H.C. Wainwright 22nd Annual Global Investment Virtual Conference
Date: Monday, September 14th 2020
Time: 10:00 a.m. ET
Logistics: If you’d like to register and attend the company presentation, click here to learn more.

On September 8, 2020 Caribou Biosciences, Inc., a leading CRISPR genome editing company, reported that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for CB-010, an off-the-shelf allogeneic anti-CD19, genome-edited CAR-T cell therapy for patients with relapsed/refractory B cell non-Hodgkin lymphoma (B-NHL) (Press release, Caribou Biosciences, SEP 8, 2020, View Source [SID1234564769]).

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!

Upon initiation of the ANTLER Phase 1 trial, CB-010 will be Caribou’s first clinical-stage product candidate. CB-010 is manufactured from healthy donor T cells using Caribou’s next-generation CRISPR genome editing technology. Through genome editing, PD-1 is deleted from the CAR-T cells, which in preclinical studies promoted an increase in the durability of antitumor activity. Genome editing is used to remove the endogenous T cell receptor in order to prevent graft-versus-host disease and to site-specifically insert the CAR into the CAR-T genome.

"We are excited to receive clearance for our first IND, a pivotal milestone in the continued evolution of Caribou," said Rachel Haurwitz, Ph.D., Caribou’s President and Chief Executive Officer. "Caribou’s genome-edited cell therapies hold tremendous potential for patients and we are eager to begin clinical studies for CB-010."

ANTLER is an open-label study designed to evaluate the safety and efficacy of a single dose of CB-010 in adult patients with different subtypes of relapsed/refractory B-NHL. The study will be conducted at multiple clinical trial sites in the United States, and enrollment is expected to begin later this year.

"B-NHL comprises multiple serious and life-threatening diseases, and CB-010 therapy has the potential to extend lives," said Steve Kanner, Ph.D., Caribou’s Chief Scientific Officer. "CB-010 is distinct among allogeneic CAR-Ts since the critical checkpoint PD-1 is eliminated to reduce CAR-T cell exhaustion, which we believe offers a crucial therapeutic advantage."