On January 10, 2022 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company mainly focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that the United States Food and Drug Administration (FDA) granted Regenerative Medicine Advanced Therapy (RMAT) designation to CT041 for the treatment of patients who have advanced gastric or gastroesophageal junction adenocarcinoma (GC/GEJ) with Claudin18.2-positive tumor (Press release, Carsgen Therapeutics, JAN 10, 2022, View Source [SID1234598548]). CT041 is the first CAR T-cell product candidate against solid tumor with RMAT designation*, according to public resources.

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!

With the RMAT designation, CT041 enjoys all the benefits of Fast Track and Breakthrough Therapy designations including early and intensive FDA guidance on efficient product candidate development and eligibility for rolling review and priority review. Products granted RMAT designation may also be eligible for accelerated approval.

CT041 is an autologous CAR T-cell product candidate developed by CARsgen, which has the potential to be the first-in-class globally. As of the date of this announcement, CT041 is the only CLDN18.2-targeted CAR T-cell product candidate globally that is being studied in clinical trials with investigational new drug (IND) or clinical trial application approvals from the FDA, the NMPA, and Health Canada.

In October 2019, CARsgen’s CT053 CAR T-cell product candidate was first granted RMAT designation by the FDA for the treatment of relapsed and/or refractory multiple myeloma. CT041 is CARsgen’s second product candidate with RMAT designation.

CARsgen currently has 11 in-house developed product candidates and obtained 8 IND approvals for CAR T-cell therapies. In addition to the existing clinical pipeline product candidates, CARsgen continues to develop innovative technologies, such as CycloCAR technology to enhance efficacy of CAR T cells against solid tumors, and allogeneic THANK-uCAR technology to reduce costs and increase affordability. As of December 31, 2021, CARsgen owns over 300 patent applications and 65 issued patents in more than 19 countries and regions, including the United States, Europe, China, and Japan.

Dr. Zonghai Li, Founder, Chairman of the Board, Chief Executive Officer, and Chief Scientific Officer of CARsgen Therapeutics Holdings Limited, stated that, "The RMAT designation indicates that CT041 has the potential to solve the unmet medical needs of patients with GC/GEJ. RMAT is not only important for accelerating product development and the review of the biologics license application, but also helps patients obtain this advanced therapy as soon as possible. We hope that the RMAT and PRIME designations obtained by CT041 will enable us to work closely with the FDA and the EMA, to obtain approvals from regulatory agencies globally, and thus benefit patients worldwide as soon as possible."

About CT041

CT041 is an autologous CAR T-cell product candidate developed by CARsgen. CT041 targets the treatment of CLDN18.2-positive solid tumors with a primary focus on GC/GEJ and pancreatic cancer (PC). CT041 has demonstrated promising therapeutic efficacy and favorable safety in ongoing clinical trials. CT041 has the potential to become a backbone therapy for GC/GEJ and PC and benefit a large population of patients worldwide.

According to the results of the investigator-initiated trial of CT041 that was orally presented at the European Society of Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress 2021 (ESMO 2021), 18 patients with GC/GEJ who failed at least two prior lines of therapy (including 8 [44%] patients exposed to an anti-PD-1 or PD-L1 antibody) at the dose of 2.5×108 CAR T cells achieved an objective response rate (ORR) of 61.1%. Historical data shows that for patients with GC/GEJ who fail at least two prior lines of therapy, the efficacy rate of chemotherapy is about 4% to 8%, and the efficacy rate of anti-PD-1 antibody is about 11%. Therefore, compared with other treatments for patients with GC/GEJ who fail at least two prior lines of therapy, CT041 shows a significant improvement in ORR.

In addition to the investigator-initiated trials in China, CARsgen has initiated a Phase Ib/II clinical trial for advanced GC/GEJ and PC in China and a Phase 1b clinical trial for advanced gastric or pancreatic adenocarcinoma in North America. CARsgen also intends to conduct a pivotal Phase 2 clinical trial in North America in 2022.

In 2020 and 2021, CT041 received Orphan Drug designation from the FDA for the treatment of GC/GEJ and Orphan Medicinal Product designation from the European Medicines Agency (EMA) for the treatment of advanced gastric cancer. In November 2021, CT041 was granted Priority Medicines (PRIME) eligibility by the EMA for the treatment of advanced gastric cancer.

About Regenerative Medicine Advanced Therapy Designation

The RMAT designation program is intended to help the FDA facilitate an efficient development program for any drug that (1) qualifies as a RMAT, which is defined as a cell therapy, therapeutic tissue engineering product, human cell and tissue product, or any combination product using such therapies or products; (2) is intended to treat, modify, reverse, or cure a serious or life-threatening disease or condition; and (3) has preliminary clinical evidence to indicate the drug has the potential to address unmet medical needs for such a disease or condition.

On January 10, 2022 BioAtla, Inc. (Nasdaq: BCAB), a global clinical-stage biotechnology company focused on the development of Conditionally Active Biologic (CAB) antibody therapeutics, reported that it has entered into a clinical collaboration with Bristol Myers Squibb (NYSE: BMY) to investigate BioAtla’s two lead CAB-ADC candidates, BA3011 and BA3021, in combination with Bristol Myers Squibb’s anti-PD-1 therapy Opdivo (nivolumab) (Press release, BioAtla, JAN 10, 2022, View Source [SID1234598547]).

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!

Under the terms of the agreement, BioAtla and Bristol Myers Squibb will collaborate on clinical trials of separate combination therapies using two of BioAtla’s Conditionally Active Biologic Antibody Drug Conjugates, BA3011 and BA3021, each in combination with Opdvio. BioAtla will serve as the study sponsor and will be responsible for costs associated with the trial execution. Bristol Myers Squibb will provide Opdivo clinical drug supply for the study.

BA3011 (CAB-AXL-ADC) and BA3021 (CAB-ROR2-ADC) are CAB antibody drug conjugates that target receptor tyrosine kinase AXL and ROR2, respectively. AXL and ROR2 are important targets as they are expressed in many solid tumors with higher frequency of expression observed in tumors previously treated with anti-PD-1 therapy. This, coupled with the therapeutic index advantages provided by BioAtla’s proprietary CAB technology, lends strong rationale for investigating BA3011 and BA3021 in combination with Opdivo.

"BioAtla is very pleased to enter into this collaboration with Bristol Myers Squibb. Identification of optimal combination regimens holds significant promise for cancer patients, and we look forward to expanding investigation of our CAB-ADCs in combination with Opdivo," stated Jay M. Short, Ph.D., Chairman, Chief Executive Officer and co-founder of BioAtla, Inc.

"This collaboration supports our strategy to aggressively drive development of therapeutics in areas of high unmet medical need and underscores the potential broad applicability of our CABADCs to provide benefit to many patients across a wide range of tumor types," added Scott Smith, President of BioAtla.

Opdivo is a trademark of Bristol-Myers Squibb Company.

About Mecbotamab Vedotin (BA3011)
BA3011, CAB-AXL-ADC, is a CAB antibody drug conjugate targeting the receptor tyrosine kinase AXL that is overexpressed across multiple different solid tumors. We are developing BA3011 as a potential therapeutic for multiple solid tumor types, including soft tissue and bone sarcoma, nonsmall cell lung cancer (NSCLC) and ovarian cancer, with other potential indications in the future. We are enrolling a potentially registration-enabling Phase 2 clinical trial (NCT03425279) of BA3011 given as monotherapy or in combination with a PD-1 inhibitor in soft tissue and primary bone sarcoma patients 12 years and older that are high AXL tumor membrane expressors (AXL high). We also are conducting a Phase 2 study (NCT04681131) in AXL high NSCLC patients who have previously progressed on PD-1/L1, EGFR, or ALK inhibitor therapy. In addition, a multicenter investigator-initiated Phase 2 clinical trial of BA3011 in combination with a PD-1 inhibitor in patients with platinum-resistant ovarian cancer has been initiated in Canada and in the US (NCT04918186).

About Ozuriftamab Vedotin (BA3021)
BA3021, CAB-ROR2-ADC, is a conditionally and reversibly active antibody drug conjugate directed against ROR2, a receptor tyrosine kinase that is overexpressed across many different solid tumors including lung, head and neck, melanoma, and breast. We are developing BA3021 as a potential therapeutic for multiple solid tumor types, including NSCLC, melanoma, squamous cell cancer of the head and neck (SCCHN) and ovarian cancer. We are enrolling a Phase 2 trial (NCT03504488) of BA3021 monotherapy or in combination with a PD-1 inhibitor in patients with ROR2 high melanoma who have previously progressed on PD-1/L1 inhibitor and patients with ROR2 high NSCLC who have previously progressed on PD-1/L1, EGFR or ALK inhibitor therapy. A Phase 2 study in patients with ROR2 high SCCHN will be initiated in early 2022. In addition, a multi-center investigator-initiated Phase 2 clinical trial of BA3021 in combination with a PD-1 inhibitor in patients with platinum-resistant ovarian cancer has been initiated in Canada and in the US (NCT04918186).

On January 10, 2022 Illumina Inc. (NASDAQ: ILMN) reported a partnership with Boehringer Ingelheim to develop companion diagnostics (CDx) for several programs in Boehringer Ingelheim’s oncology pipeline (Press release, Illumina, JAN 10, 2022, View Source [SID1234598546]). The partnership aims to accelerate the development of therapy selection and precision medicines for patients with advanced cancer.

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!

We are delighted that Boehringer Ingelheim has chosen Illumina to be its long-term partner in the development of companion diagnostics for the treatment of cancers
We are delighted that Boehringer Ingelheim has chosen Illumina to be its long-term partner in the development of companion diagnostics for the treatment of cancers
The partnership spans current and future CDx programs, with plans to add CDx claims to an in vitro diagnostic test Illumina is developing that is based on the content of TruSight Oncology 500 (TSO 500). The first program will co-develop a CDx for a Boehringer Ingelheim investigational medicine.

"We are delighted that Boehringer Ingelheim has chosen Illumina to be its long-term partner in the development of companion diagnostics for the treatment of cancers," said Joydeep Goswami, Chief Strategy and Corporate Development Officer of Illumina. "Illumina continues to provide the leading distributable assay enabling comprehensive genomic profiling for both tissue and liquid biopsy and together we will unlock the potential of new biomarkers to identify patients most likely to benefit from new precision medicines."

About TruSight Oncology 500
TSO 500 is a Research Use Only comprehensive pan-cancer assay designed to identify 523 known and emerging tumor biomarkers. TSO 500 utilizes both DNA and RNA from tumor samples to identify key variants critical for cancer development and progression, such as small DNA variants, fusions, and splice variants. Based on the content of TSO 500, Illumina will be adding an in vitro diagnostic test to the TruSight Oncology product family. This comprehensive tumor profiling assay will have similar chemistry and analytics to TSO 500. To learn more about TSO 500, click here.

On January 10, 2022 Agendia, Inc., a commercial stage company focused on precision oncology for breast cancer, reported a multi-year partnership with Illumina, Inc. (NASDAQ: ILMN), to co-develop in vitro diagnostic (IVD) tests for oncology testing (Press release, Agendia, JAN 10, 2022, View Source [SID1234598545]). The partnership is intended to advance the use of next-generation sequencing (NGS) for decentralized oncology testing and aligns with Illumina’s approach to IVD partnerships in oncology. Agendia joins Illumina’s growing portfolio of more than 30 IVD partners developing over 40 sequencing-based solutions for cancer prognosis, therapy selection, and other applications.

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!

Illumina and Agendia plan to develop new tests to enhance the care and management of breast cancer patients, utilizing the Illumina MiSeqDx sequencing platform to expand the range of gene panels available for solid tumor analysis. The company expects its flagship test, MammaPrint, which is FDA-cleared and currently offered via Agendia’s central laboratory, will be the first decentralized NGS-based breast cancer recurrence risk test cleared by the FDA.

"This partnership represents a major step in Agendia’s integration of NGS technologies and brings us even closer to our promise of providing crucial genomic information to physicians and their patients to guide care at every single step in a patient’s treatment journey," said Mark Straley, Chief Executive Officer of Agendia. "We believe our current FDA-cleared, broadly reimbursed MammaPrint test provides the perfect foundation for incorporation with Illumina’s MiSeqDx platform. Together, we plan to expand what is possible in breast cancer care and ensure that the essential insights that come from genomic testing are delivered to patients around the world."

Illumina’s robust diagnostic NGS product portfolio, including the MiSeqDx platform, covers a broad range of clinical applications designed for a variety of clinical lab settings. As NGS testing becomes more integrated in routine patient care, Illumina is committed to providing clinical solutions, through both products and partnerships, that enable the diagnosis and management of human diseases.

Agendia’s proprietary testing solutions empower physicians to more accurately diagnose the disease drivers and predict the progression of a woman’s breast cancer. The company’s scientific approach provides a holistic view of the biology underlying an individual patient’s breast cancer, enabling physicians to personalize treatment. The company believes the decentralized MammaPrint NGS test, developed on the Illumina MiSeqDx platform, will enable major clinical hospitals worldwide to offer the clinical utility and benefits of MammaPrint to their physicians and patients in-house. In addition, the MammaPrint NGS test will leverage nearly two decades of clinical evidence drawn from 30 clinical studies featuring over 30,000 patients with breast cancer, as well as a 15-year history of FDA clearance, top tier medical society guideline inclusion, and well-established and unique CPT codes for broad reimbursement.

"We are pleased to partner with Agendia, with its deep expertise in genomic testing in breast cancer, to expand the reach of NGS-based genomic testing in cancer care," said Joydeep Goswami, Chief Strategy and Corporate Development Officer of Illumina. "We believe enabling in-house clinical testing will strengthen the knowledge of the healthcare network, reduce turnaround times, and ultimately better support more patients with improved guidance on care pathways."

On January 10, 2022 Nashville Biosciences, a wholly-owned subsidiary of Vanderbilt University Medical Center (VUMC), reported an agreement with Illumina, a leading genomic technology company, to realize the full potential of VUMC’s DNA databank, BioVU (Press release, Nashville Biosciences, JAN 10, 2022, View Source [SID1234598544]).

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 agreement is a continuation of Vanderbilt’s long-term effort around BioVU and will accelerate genomic sequencing, resulting in new discoveries to advance new methods for diagnosing, preventing and treating an array of human diseases.

Under the terms of the agreement, Nashville Biosciences and Illumina will seek to establish an alliance with multiple pharmaceutical industry partners to perform whole genome sequencing on approximately 250,000 de-identified DNA samples collected under an Institutional Review Board (IRB)-approved process over the past 15 years.

The resulting library of de-identified health and genomic data, collected from patients diverse in gender, race, age and clinical conditions will be available to industry partners and researchers at Vanderbilt to discover and advance new methods for diagnosing and treating an array of human diseases.

"Through BioVU and other initiatives by talented individuals from across our campus, Vanderbilt holds a special place at the forefront of personalized medicine. This agreement with Illumina is a next step, allowing us to utilize their global presence and technology to join with industry partners, realizing our vision to achieve the full promise of clinical and genomic data we have developed," said Jeff Balser, MD, PhD, President and Chief Executive Officer for Vanderbilt University Medical Center and Dean of Vanderbilt University School of Medicine.

"Nashville Biosciences’ enriched biobank data, coupled with Illumina’s extensive genomics and bioinformatics expertise will enable scientists and researchers to generate new insights into disease and develop new medicines," said Joydeep Goswami, Chief Strategy and Corporate Development Officer of Illumina. "Together, we will be able to identify potential targets that could lower costs, improve development timelines, and make therapies more effective for patients."

BioVU is the most comprehensive collection of human DNA and other biological materials stored at a single site. DNA samples are extracted from discarded blood collected at VUMC during routine clinical testing and linked to a "synthetic derivative" – a copy of VUMC’s electronic medical records from which patients’ personal identifying information has been removed while still preserving the clinical information generated in the ongoing delivery of healthcare.

The linking of specific DNA sequences to clinical information has enabled researchers to understand a wide range of health conditions, from cancer to heart disease, as well as genetic factors that increase the risk for severe illness following infections, including COVID-19.

Nashville Biosciences is a clinical and genomic analytics company providing access to VUMC’s research resources, notably BioVU, and is helping biopharmaceutical, diagnostics, and other life sciences partners accelerate their R&D activities. Since its founding in 2018, Nashville Biosciences has partnered with more than 60 clients to utilize BioVU to identify new disease biomarkers and potential drug targets, optimize clinical trials and conduct other studies using real-world data.

In 2016, for example, VUMC researchers and Illumina collaborated to analyze approximately 100,000 BioVU DNA samples using Illumina’s Multi-Ethnic Genotyping Array (MEGA). At the time, it was the largest single use of the MEGA array and has provided BioVU researchers a rich platform for genetic discovery since its completion.