On September 21, 2022 Propath UK, Europe’s leading CRO for spatial biology, and Nucleai, a leader in AI-powered spatial biology transforming precision medicine by unlocking spatial biology insights from pathology data, reported their collaboration to develop and validate a 30-plex immunofluorescence (IF) panel focused on protein targets relevant to immuno-oncology (Press release, Nucleai, SEP 21, 2022, View Source [SID1234621317]). For our pharmaceutical and biotechnology partners, this groundbreaking panel, in conjunction with Nucleai’s validated AI assay, can be used to unlock insights from immunotherapy trials and inform the development of novel biomarkers and companion diagnostics (CDx).

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"Our strategic partnership with Propath has far-reaching implications in biomarker discovery," said Avi Viedman, CEO of Nucleai. "The collaboration allows us to develop and deploy AI-based multi-modal models to identify unique sets of previously unavailable biomarkers and drug targets. Together, we are pushing the boundaries of pathology tissue analytics."

Applying its expertise in multiomic spatial biology and high-plex immunofluorescence, Propath will develop a novel protocol using the innovative Lunaphore COMET platform for analysis of 30 high-value protein targets from a single tissue section. The COMET enables rapid development of highly multiplexed panels, with robust and reproducible staining. Propath UK will use experience gained across a range of spatial proteomic and transcriptomic projects completed on its in-house multiomic platforms.

"Spatial biology is transforming biopharma research by enabling deeper analysis of cell phenotypes with full spatial context in the tissue microenvironment," commented Dr Krish Soni, Chief Executive of Propath UK. "We are delighted to collaborate with Nucleai on this ambitious project which will empower researchers to unlock the potential of spatial biology, with relevance to programs from discovery through to translational and clinical research."

Nucleai’s collaboration with Propath comes on the heels of the recent news announcement that it made with Lunaphore, announcing their partnership to provide AI-powered spatial biology analysis to accelerate drug discovery. This three-way synergy between Propath, Lunaphore and Nucleai brings new capabilities to pharma companies. The partnership utilizes Lunaphore’s flagship COMET platform for hyperplex staining and imaging with Nucleai’s cutting-edge AI spatial models to derive new insights from tissue biopsies, including novel drug targets, mechanisms of action, and biomarkers to advance the field of precision medicine. Demonstrating the utility of the technology, Propath provides high-plex tissue analytics using the Lunaphore COMET as a fully managed service.

Nucleai’s proprietary AI-powered, pathology-based biomarker discovery platform combined with Propath’s multiomic spatial biology and high-plex immunofluorescence will accelerate the pharmaceutical industry’s drug development and research programs. A fundamental aspect of precision oncology is predicting drug response for a patient cohort. The combined expertise will allow computational analysis of the tumor microenvironment, feature selections to predict disease prognosis and drug response including cancer sub-type determination, to stratify patients and support early detection and prognosis.

On September 21, 2022 CARsgen Therapeutics Holdings Limited (Stock Code: 2171.HK), a company focused on innovative CAR T-cell therapies for the treatment of hematologic malignancies and solid tumors, reported that at the 7th Annual CAR-TCR Summit, in Boston, Massachusetts, the Company gave an oral presentation on the North America Phase 2 clinical trial of zevorcabtagene autoleucel ("zevor-cel", R&D code: CT053), an autologous CAR T-cell product candidate against BCMA (Press release, Carsgen Therapeutics, SEP 21, 2022, View Source [SID1234621316]). This multi-center, open-label, Phase 1b/2 study (NCT03915184) is being conducted to evaluate the safety and efficacy in North American patients with relapsed and/or refractory multiple myeloma (R/R MM).

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As of August 31, 2022, 17 patients with R/R MM received zevor-cel infusion in the Phase 2 portion of the clinical trial and have been followed for a median of 113 days (range: 9 to 373). Of these 17 patients, five patients (29.4%) had extramedullary disease (EMD; ≥1 plasmacytoma) and nine patients (52.9%) had high-risk cytogenetic features. Patients were heavily pretreated with a median of six prior lines of therapy (range: 4 to 17). All patients were refractory to their last line of therapy. Prior to zevor-cel infusion, patients received lymphodepletion regimen of fludarabine (30mg/m2 for three consecutive days) and cyclophosphamide (500mg/m2 for two consecutive days).

Efficacy

In the 11 evaluable patients who had at least eight weeks follow-up, including four patients with EMD, the objective response rate was 100% (very good partial response, complete response, or stringent complete response) and responses deepened for patients with longer follow-up. Since all responses are ongoing, the median progression-free survival, median overall survival and median duration of response have not been reached, and the complete response/stringent complete response rate is not mature. All patients with available MRD results at week 4 were MRD negative by next-generation of sequencing.

Safety

No death occurred and no patient experienced Grade 3 or higher cytokine release syndrome. Cytokine release syndrome was observed in 10 of 17 patients (59%), all reported as Grade 1 or 2. One transient Grade 3 immune effector cell-associated neurotoxicity syndrome event was reported and the patient fully recovered; no neurological toxicity with parkinsonian features was observed. For the management of post-infusion symptoms, five of 17 patients (29%) received tocilizumab and one patient (5.9%) received corticosteroids. Notably, three patients have received outpatient zevor-cel treatment in this study, and the two patients were admitted into the hospital for symptom management for 1 or 2 days.

Conclusion

Zevor-cel at a dose of 1.8×108 CAR T cells was well tolerated in the initial 17 Phase 2 patients with R/R MM, with promising efficacy and MRD negativity. Outpatient treatment is currently undergoing further exploration.

Dr. Raffaele Baffa, Chief Medical Officer of CARsgen Therapeutics, said: "Multiple myeloma is the second most common hematologic malignancy. People in the United States (US) living with or in remission from multiple myeloma is expected to increase to about 160 thousand in 2024. There remains a significant unmet medical need of multiple myeloma patients that call for alternative options. The results of the initial 17 patients treated with zevor-cel in the LUMMICAR-2 Phase 2 clinical trial, reported at the CAR-TCR Summit, indicates a consistent trend of clinical benefit with the data of earlier trials conducted on zevor-cel, such as the investigator-initiated trials and LUMMICAR-1 phase 1 trials in China as reported previously in ASH (Free ASH Whitepaper). We are very encouraged by the competitive efficacy and favorable safety profiles and will continue to dedicate our efforts in successfully developing and launching zevor-cel for multiple myeloma patients worldwide."

About Zevor-cel

Zevor-cel (CT053) is a fully human, autologous BCMA CAR T-cell product candidate for the treatment of R/R MM. CARsgen is conducting a Phase 1b/2 clinical trial (LUMMICAR STUDY 2) in North America to evaluate the safety and efficacy of zevor-cel for R/R MM. The Company also plans to conduct additional clinical trials to develop zevor-cel as an earlier line of treatment for multiple myeloma.

Zevor-cel received Regenerative Medicine Advanced Therapy (RMAT) and Orphan Drug designations from the U.S. Food and Drug Administration (FDA) in 2019, as well as the PRIority MEdicines (PRIME) and Orphan Medicinal Product designations from the European Medicines Agency (EMA) in 2019 and 2020, respectively. Zevor-cel also received Breakthrough Therapy designation from the NMPA in 2020.

The Company believes that zevor-cel is well positioned to potentially reshape the treatment paradigm for multiple myeloma and become a foundational treatment for multiple myeloma patients.

On September 21, 2022 Abpro, a biotech company with the mission of improving the lives of mankind facing severe and life-threatening diseases with next-generation antibody therapies, reported a strategic partnership with Celltrion, a biopharmaceutical company headquartered in Incheon, South Korea, today, for its cancer molecule ABP 102, an antibody therapy for patients suffering from HER2+ cancer, including breast, gastric, and pancreatic cancer (Press release, Abpro Therapeutics, SEP 21, 2022, View Source [SID1234621315]). Through this global partnership, Abpro will receive payments from Celltrion of up to $1.75 Billion, including an equity investment, development and commercial milestone payments and worldwide profit sharing. Celltrion will be in charge of the development of ABP 102 following the completion of in vitro studies by Abpro and will have world-wide commercialization rights. HER2+ type cancer is implicated in up to 30% of all cases in breast, gastric, pancreatic, and other forms of cancer.

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"We are thrilled to be able to enter this collaboration with Celltrion and are excited to work with such an excellent partner on bringing this treatment to market. Through our commitment to patients, we strive to enable them to live life to the fullest, and this collaboration with Celltrion, a world class pharmaceutical company, will help us meet that goal," said Ian Chan, co-founder and CEO of Abpro. "This is a significant validation of our technology platform and also our pipeline of t-cell engagers. We aim to deliver therapies to help patients facing severe disease that leads to a better quality of life."

"Abpro’s ABP 102 molecule has shown preliminary data of better efficacy and less toxicity compared to other therapies treating the same indication," said Dr. Sooyoung Lee, VP and head of the new drug research division of Celltrion. "We are confident this partnership will allow us to deliver the best possible care to patients suffering from HER2+ cancer. Abpro’s platform looks highly promising and potentially best-in-class. Together, we will go great lengths to ensure people live longer, healthier lives."

On September 21, 2022 BostonGene reported that it will participate in the 81st Annual Meeting of the Japanese Cancer Association (Press release, BostonGene, SEP 21, 2022, View Source [SID1234621314]). The event, to be held at Pacific Convention Plaza Yokohama from September 29 – October 1, will bring together leading oncologists and basic and clinical researchers in academia or industry to discuss cutting-edge findings in cancer research and recent progress in cancer, such as genomic medicine and immune therapy. BostonGene will also exhibit in Hall B, booth 87.

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BostonGene will showcase how its innovative computational platform performs AI-based molecular and immune profiling to discover correlations between tumor genomics, a patient’s immune system and the effectiveness of all available approved and investigational treatments can advance how physicians treat their patients and dramatically improve clinical outcomes.

"I look forward to participating at the Annual Meeting of the Japanese Cancer Association and to presenting the clinical utility of the BostonGene Tumor PortraitTM Tests," said Nathan Fowler, MD, Chief Medical Officer at BostonGene. "We firmly believe BostonGene’s deep molecular and immune profiling, computational power, and analytics will positively impact the standard of care for cancer patients worldwide."

Details about the abstracts selected for presentation can be found below:

Abstract Number: E-1044
Title: Analytical Validation of the BostonGene Tumor Portrait Assay
Date and Time: Thursday, September 29 at 3:15 PM JST
Location: Room 7
English Oral Session Title: E7-2 Elucidating the significance of mutations through informatics and novel technologies

With the increasing use of molecular profiling to guide personalized decision-making, the analytical capabilities of next-generation sequencing (NGS) assays are critical. This presentation describes the analytical performance of the BostonGene Tumor PortraitTM Test, which integrates whole-exome sequencing (WES) and RNA sequencing (RNA-seq).

Abstract Number: E-2073
Title: Combinatory Technology for Single Sample Gene Expression Projection onto a Cohort Sequenced with a Different Technology
Date and Time: Friday, September 30 at 1:00 PM JST
Location: Room 13
English Oral Session Title: E14-7 Gastric cancer (1)

This presentation highlights the importance of BostonGene Batch Correction technique that enables the analysis of single tumor gene expressions by comparison with datasets collected from cohorts of patients with a similar diagnosis to support personalized treatment decisions.

Abstract Number: E-3075
Title: Urothelial carcinoma (UC) subtypes defined by genomic and tumor microenvironment (TME) features predict therapy response properties for precision oncology
Date and Time: Saturday, October 1 at 1:30 PM JST
Location: Room 10
English Oral Session Title: E14-10 Basic and clinical research for urologic cancer (1)

This presentation demonstrates the ability of BostonGene’s novel transcriptome-based classification platform to identify tumor microenvironment (TME) subtypes for a specific cancer type, urothelial carcinoma, that are predictive of response to immunotherapy.

Abstract Number: P-3081
Title: Astraea: A biomarker database integrating genomic, transcriptomic, and TME properties for precision oncology
Date and Time: Saturday, October 1 at 5:15 PM
Location: Room P
Poster Session Title: P25-1 Towards Understandings of Cancer Biology from Viewpoints of Mathematical Modelings and Computational Biology

This presentation underscores the application of BostonGene’s cutting-edge analytical tools to improve treatment for cancer patients by identifying biomarkers for diverse therapies and ultimately helping guide decision-making for personalized medicine.

On September 21, 2022 Foundation Medicine, Inc., a pioneer in molecular profiling for cancer, and Day One Biopharmaceuticals (Nasdaq: DAWN), a clinical-stage biopharmaceutical company dedicated to developing and commercializing targeted therapies for people of all ages with life-threatening diseases, reported a collaboration to develop FoundationOneCDx as a companion diagnostic for Day One’s lead investigational therapy, tovorafenib (DAY101) (Press release, Foundation Medicine, SEP 21, 2022, View Source [SID1234621313]).

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Tovorafenib is an investigational, oral, brain-penetrant, highly selective type II pan-RAF kinase inhibitor, which is currently being evaluated in an ongoing pivotal Phase 2 clinical trial (FIREFLY-1) for the treatment of pediatric, adolescent and young adult patients with relapsed pediatric low-grade glioma (pLGG). In June 2022, Day One reported positive initial data from the FIREFLY-1 study and topline results for the full pivotal study population are expected in the first quarter of 2023. Day One has also initiated a pivotal Phase 3 study (FIREFLY-2/LOGGIC) with tovorafenib in newly-diagnosed patients with pLGG and tovorafenib is additionally being evaluated alone and as a combination therapy for other recurrent or progressive solid tumors with MAPK pathway aberrations.

"New advancements in pediatric cancer research, including novel targeted therapies and companion diagnostics, have the ability to provide a more precise perspective into a patient’s disease helping to inform a tailored therapeutic strategy that may increase the odds of long-term success for children with these diseases," said Samuel Blackman, M.D., Ph.D., co-founder and chief medical officer of Day One. "Combining our purposeful approach to drug development with Foundation Medicine’s genomic profiling expertise and global reach, will enable greater patient access to tovorafenib once approved, and ultimately help more children living with this debilitating form of cancer."

Foundation Medicine’s portfolio of FDA-approved comprehensive genomic profiling tests offer physicians both blood- and tissue-based testing options for detecting genomic alterations that help guide personalized treatment decisions. Currently, Foundation Medicine’s tissue and blood-based tests have more than 60 companion diagnostic indications collectively in the United States and Japan and are available in more than 100 countries globally.

"Foundation Medicine and Day One Biopharmaceuticals have a shared commitment to deepening our understanding of cancer biology so we can deliver more treatment options to patients of all ages, faster," said Sanket Agrawal, Chief Biopharma Business Officer at Foundation Medicine. "We’re proud to partner with Day One to provide access to tovorafenib through companion diagnostic development once approved and look forward to our ongoing collaboration to address significant unmet needs in pediatric oncology."

If tovorafenib, and this companion diagnostic indication are approved, this would be the first companion diagnostic indication for FoundationOne CDx in pediatric oncology.