On November 11, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported that completed a Type B Pre-Biologics License Application (BLA) meeting with the U.S. Food and Drug Administration (FDA) for omidubicel, a potentially life-saving treatment for patients with blood cancers in need of stem cell transplant (Press release, Gamida Cell, NOV 11, 2021, View Source [SID1234595304]). The FDA requested that Gamida Cell provide revised analysis of the manufacturing data generated at Gamida Cell’s wholly-owned commercial manufacturing facility to demonstrate the comparability to the omidubicel that was produced at the clinical manufacturing sites for the Phase 3 study. The FDA did not request additional clinical data to initiate the BLA submission once analytical comparability is demonstrated. The company will continue to work collaboratively with the FDA and anticipates submitting the BLA in the first half of 2022 in lieu of the company’s previous plan to submit the BLA by the end of 2021.

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!

"Despite the delay in timing to bring omidubicel to patients after a potential FDA approval, we are encouraged by the FDA’s reaction to our Phase 3 data as the pivotal trial of omidubicel. We have gained further clarity with the FDA on the requirements for demonstrating comparability for our commercial manufacturing facility," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "With the FDA’s feedback in hand, we believe that we are one step closer for omidubicel to be made available to patients in need."

Omidubicel, an investigational advanced cell therapy for allogeneic bone marrow transplant

Omidubicel is the foundational product based on Gamida Cell’s proprietary NAM-enabled cell expansion technology. It is the first cell therapy for bone marrow transplant to receive Breakthrough Therapy Designation from the FDA. The BLA submission will be based on the results of an international, randomized Phase 3 study of omidubicel that was designed to evaluate the safety and efficacy of omidubicel in patients with hematologic malignancies undergoing a bone marrow transplant compared to patients who received a standard umbilical cord blood transplant. The study achieved its primary endpoint, a statistically significant reduction in time to neutrophil engraftment, as well as all key secondary endpoints. A key milestone in a patient’s recovery, neutrophil engraftment is a measure of how quickly the stem cells a patient receives in a bone marrow transplant are established and begin to make healthy new cells. In the Phase 3 study, the median time to neutrophil engraftment was 12 days for patients randomized to omidubicel compared to 22 days for the comparator group (p < 0.001). Additionally, the study met key secondary endpoints related to the speed of platelet engraftment, decrease in infections and reduction in hospitalizations, all significant clinical measures in bone marrow transplant.

On November 11, 2021 4D pharma plc (AIM: DDDD, NASDAQ: LBPS), a pharmaceutical company leading the development of Live Biotherapeutic products (LBPs), a novel class of drug derived from the microbiome, reported that 4D pharma management will participate in the Jefferies London Virtual Healthcare Conference, being held November 18-19, 2021 (Press release, 4d Pharma, NOV 11, 2021, View Source [SID1234595303]).

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!

On November 11, 2021 Shasqi, a clinical-stage biotechnology company developing precision activated oncology therapeutics with its proprietary Click Activated Protodrugs Against Cancer (CAPAC) Platform, reported a Series B funding round of $50 million driven by a syndicate of private investors, including Juan Jaen, Ph.D., President of Arcus Biosciences and Bill Rieflin, Executive Chairman of the Board at NGM Biopharmaceuticals (Press release, Shasqi, NOV 11, 2021, View Source [SID1234595302]). The funds will be used to advance the Phase 1/2 clinical study of SQ3370 for the treatment of injectable, advanced solid tumors, and will enable Shasqi to expand its click chemistry platform to create therapeutics that do not require intratumoral injections.

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!

"Data from our lead program shows us that click chemistry works in humans and the early validation gives us even greater confidence in our approach. This round of financing will enable us to advance SQ3370 through clinical development, while simultaneously advancing multiple next-generation click chemistry programs, beginning with an antibody-directed program that will unlock the potential of click chemistry to target tumors that cannot be reached with injections, significantly expanding our platform," said José M. Mejía Oneto, M.D., Ph.D., founder and CEO of Shasqi. "We expect this expansion to open up the path for the platform to antibody-based approaches, immune cell engagers, radiopharmaceuticals and more. We thank all of our investors for their continued support and enthusiasm."

"Shasqi’s approach enables us to look across the spectrum of the industry’s most powerful tumor killing therapies with a new lens," said Dr. Jaen. "The next iterations of the technology will enable Shasqi to use even more powerful payloads and localize drugs at tumors that cannot be injected. There is an enormous opportunity to dramatically improve the efficacy of powerful therapies by localizing them, which could help increase the number of patients who benefit. I look forward to supporting the company’s progress, given the platform’s potential to transform the treatment landscape and patients’ lives."

Mr. Rieflin commented, "The Shasqi team has parlayed $18 million into an ongoing Phase 1/2 clinical study of SQ3370 and a click chemistry platform that is poised for expansion into antibody-based and other targeted modalities. I’ve been impressed with the dedication and capital efficiency of the team and can’t wait to see what’s possible with the proceeds of this Series B financing."

Shasqi plans to initiate two development programs over the next two years and explore a variety of other approaches in partnership with industry leading oncology companies.

With SQ3370, Shasqi advanced the first click chemistry-based therapy into human clinical studies. Interim clinical data from the Phase 1 clinical study of SQ3370, Shasqi’s lead program, in advanced sarcomas and other solid tumors, was presented earlier this year at the European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) Congress and will be presented at the Society of Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)s (SITC) (Free SITC Whitepaper) 36th Annual meeting. The data provides initial validation for Shasqi’s platform, demonstrating an encouraging safety profile and early signs of clinical activity. Based on this data, Shasqi plans to begin a Phase 1 expansion cohort of its clinical study of SQ3370 for the treatment of soft tissue sarcoma in patients who are unable to tolerate chemotherapy in Q1 2022 and expects to share pharmacokinetic and biomarker data by Q2 2022. In 2022, Shasqi is planning to initiate a Phase 2 registration study of SQ3370.

About CAPAC and SQ3370:
SQ3370 is the first click chemistry-based treatment to be tested in humans, and utilizes Shasqi’s proprietary CAPAC platform, an approach that activates cancer drugs at a tumor with decreased systemic toxicity. Shasqi is validating its platform with SQ3370, which is designed to activate a powerful chemotherapeutic, doxorubicin, at the site of the tumor. The investigational product is based on the chemical reaction between a drug protected through a trans-cyclooctene modification (a protodrug) and a tetrazine-modified biopolymer. The biopolymer is injected into the target tumor lesion, where it precisely activates an intravenously infused protodrug. Shasqi believes its click-chemistry approach can improve the efficacy and safety of many existing drugs and various modalities that have a limited therapeutic window.

On November 11, 2021 NanoString Technologies, Inc. (NASDAQ: NSTG), a leading provider of life science tools for discovery and translational research, reported the launch of the nCounter TCR Diversity Panel and companion cloud-based TCR analysis report created for use with the ROSALIND platform (Press release, NanoString Technologies, NOV 11, 2021, View Source [SID1234595300]). This new gene expression panel allows researchers to measure the usage of T cell receptor (TCR) variable regions and shifts in TCR diversity with a simple assay that can deliver results in less than 24 hours.

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!

A diverse population of TCRs is a hallmark of a healthy immune system. The mix and abundance of TCR variable regions changes upon challenge with a pathogen, cancer, or any disease that triggers an adaptive immune response. TCRs are studied to understand disease onset, progression, response or non-response to treatment, and disease recovery. By directly quantifying the expression of each TCR variable region, the nCounter TCR Diversity Panel can rapidly screen samples without the time and cost of laborious sequencing methods.

"The nCounter TCR Diversity Panel provides an intuitive, powerful new tool for use with clinical samples," said Dr. Léon van Kempen, an early access user and scientific lead at the Laboratory for Molecular Pathology at University Medical Center Groningen, Netherlands. "The panel allows for a TCR diversity score to be easily calculated based on expression and objectively quantifies a T cell response allowing for broad utility of the panel throughout all types of research."

The new panel utilizes the unique oligonucleotide barcoding chemistry of the nCounter Analysis System to detect mRNA transcripts directly. It contains probes for 129 human genes covering all TCR alpha, beta, gamma, delta variable regions as well as constant regions. The panel also allows for customization with the ability for researchers to add in and measure unique TCRs of interest. This novel solution is coupled with an automated data analysis report purchased separately and accessible within the ROSALIND platform, allowing researchers to analyze results and generate TCR diversity scores within minutes.

"We remain committed to developing innovative tools to advance our understanding of the human immune response. Leveraging the simplicity of the nCounter workflow makes it possible to gain broader insights with higher throughput," said Joseph Beechem, Ph.D., chief scientific officer, NanoString. "In the future, we expect to enable our GeoMx Digital Spatial Profiler to study TCRs spatially within tissues, providing an entirely new dimension of knowledge."

Learn more about the nCounter TCR Diversity Panel and the latest scientific advances in the field of immuno-oncology using the nCounter and GeoMx Digital Spatial Profiler platforms at the virtual symposium and booth #8 at the 36th annual Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Conference.

Virtual Symposium: "Quantifying T-cell receptor diversity via expression analysis" is on Friday, Nov. 12, from 1:00 – 1:30 pm EST. The symposium is an opportunity to talk with Dr. Léon van Kempen, a clinical scientist in molecular pathology, about getting started with the spatial biology revolution and the GeoMx Whole Transcriptome Atlas. In addition, this workshop will focus on the nCounter TCR Diversity Gene Expression Panel and a case study on the use of the panel for clinical research.

On November 11, 2021 Biodesix, Inc. (Nasdaq: BDSX), a leading data-driven diagnostic solutions company with a focus in lung disease, reported Scott Hutton, Chief Executive Officer of Biodesix, will present in a fireside chat at the Canaccord Genuity Virtual MedTech, Diagnostics and Digital Health & Services Forum being held virtually November 18, 2021 (Press release, Biodesix, NOV 11, 2021, View Source [SID1234595299]).

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!

Canaccord Genuity Virtual MedTech, Diagnostics and Digital Health & Services Forum
Date: Thursday, November 18, 2021
Time: 4:30 PM ET

The fireside chat will be webcast live and available for replay under "News & Events" in the Investors section of the Company’s website at www.biodesix.com.