On October 26, 2021 CANbridge Pharmaceuticals, Inc., a leading China-based global rare disease-focused biopharmaceutical company committed to the research, development, and commercialization of transformative therapies, reported that it has dosed the first patient in the CAN008 Phase 2 clinical trial to treat glioblastoma multiforme (GBM) in Mainland China (Press release, CANbridge Life Sciences, OCT 26, 2021, View Source [SID1234591981]).

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The multi-center, randomized, double-blind, placebo-controlled Phase 2 trial will compare standard-of-care (tumor removal, followed by radiation therapy plus temozolomide (TMZ)) with placebo, to standard-of-care with CAN008. The trial will investigate efficacy, as well as explore how select biomarkers correlate to outcome, to provide potential benefits to GBM patients.

The compound has been recognized by regulatory bodies in key international markets as potentially promising in the treatment of glioblastoma. It was granted orphan drug designation by the United States Food and Drug Administration (FDA) and received an orphan medicinal product designation by the European Medicines Agency (EMA) for the treatment of glioblastoma multiforme. In addition, it also was accepted into the EMA’s PRIME (Priority Medicines) program, which provides early and enhanced support to medicines that have the potential to address patients’ unmet needs. CANbridge completed the Phase 1 clinical trial of CAN008 plus temozolomide (TMZ), during and after radiation therapy, in patients with newly diagnosed glioblastoma multiforme (GBM). The results demonstrate that CAN008 has excellent safety and tolerability in patients with GBM and could potentially improve the quality life and the survival time of the patients

"Glioblastoma multiforme is the most common primary intracranial malignant tumor," said Professional Wenbin Li, Director of Comprehensive Treatment Ward of Neuro-Oncology, Beijing Tiantan Hospital, Capital Medical University. "Under the current standard treatment, the media survival time of patients is only 14.6 months, and the two-year survival rate is only 27%. The incidence in China has been increasing in recent years. The preliminary clinical trial results of CAN008 show that it has a good treatment effect trend, as well as very good safety and tolerability. We are pleased to participate in the domestic clinical study of CAN008 to provide a potential new treatment option for patients with glioblastoma."

"CAN008 is our first clinical product approved to commence clinical trials that has received a Class 1 new drug designation by China Food’s National Medical Products Administration," said James Xue, Ph.D., Founder, Chairman and CEO of CANbridge Pharmaceuticals, Inc. "The dosing of the first patient in CAN008 Phase 2 trial for the treatment of GBM in China represents a major milestone for CANbridge. We look forward to advancing this truly novel, fusion protein treatment for glioblastoma along the clinical pathway in China, where it might provide new options for patients."

About Glioblastoma (GBM)

Glioblastoma (GBM) is the most common malignant tumor of the central nervous system, accounting for 35.26% – 60.9% of intracranial tumors. It is classified by the World Health Organization (WHO) as a stage-IV cancer, according to the latest classification standard of intracranial tumors. GBM is highly infiltrative and aggressive and is not surgically curable, which leads to a high recurrence rate. Although the treatment of GBM, through resection radiotherapy and chemotherapy, has continuously improved, prognosis has not been substantially improved. The median survival time is still only 14.6 months.

According to the American Association of Neurological Surgeons AANS Statistics, the number of patients with GBM in western countries is 2-3/100,000 every year, accounting for 52% of intracranial tumors. The two-year and five-year survival rates are about 30% and 4-5%, respectively.

According to Frost & Sullivan, GBM patients in China accounted for 46.6 % of intracranial tumors, totaling 54,700 patients in 2020. With the aging population and aggravated levels of air pollution and ionizing radiation exposure, the number of patients with newly diagnosed glioblastoma in China is expected to reach 59,800 in 2025, and 64,400 in 2030.

About CAN008

CAN008 is a CD95 Fc fusion protein that blocks the interaction between CD95 receptor, and its cognate ligand CD95L, through binding to CD95L. CAN008 has a unique dual mechanism of action, which can not only inhibit the invasive growth and migration of tumor cells, but could also reduce the apoptosis of T cells induced by Caspase, and enhance immune recognition. Previous clinical trial data show that CAN008 has good safety, can effectively improve the quality-of-life of GBM patients, and can greatly prolong the survival time of patients. Based on this, European Medicines Agency (EMA) included CAN008 in the Priority Medicines (PRIME) program.

Note: The European Medicines Agency (EMA) officially launched the "PRIority MEdicines (PRIME)" plan on March 7, 2016, to accelerate the review process of drugs that could address medically underserved conditions.

On October 26, 2021 SimBioSys reported it raised $15 million in Series A funding to accelerate the development and commercialization of its TumorScope software platform (Press release, Northpond Ventures, OCT 26, 2021, View Source [SID1234591980]). The company’s novel, simulation-based, precision medicine platform enables individualized treatment planning for cancer patients. This Series A was co-led by Genoa Ventures and Northpond Ventures, with participation from AV8 Ventures, Heritage Medical Group, and Mayo Clinic. Existing investors and founders also participated in this financing round, bringing the company’s total capital raised to $21 million.

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In the first half of 2021, SimBioSys tripled its headcount, bringing in expert clinicians, scientists, and executives from the life sciences industry. SimBioSys is also working in collaboration with 20 leading cancer institutions across the country to run clinical validation studies and recently published results from independent validation performed by prestigious cancer centers, demonstrating over 90 percent accuracy in predicting response to therapy in its first indication of early breast cancer.

"We are honored to have the support of such prestigious and thoughtful investors, validating the novelty and promise of our science and its potential to improve outcomes for millions of patients in the future," said Tushar Pandey, CEO of SimBioSys.

Despite the crowded landscape of precision medicine, treatment decisions continue to be made based on trial and error, and the resulting uncertainty among clinicians leads to sub-optimal outcomes for patients. SimBioSys aims to individualize care and eliminate uncertainty by assessing response to therapy at the time of treatment planning.

"The rate of innovation in oncology is truly inspiring, but it doesn’t always translate to benefit for most patients," said Tushar. "SimBioSys firmly believes we can do more with what is currently available while accounting for the rapidly evolving standard of care to ensure all patients have access to precision medicine."

SimBioSys’ early validation data and approach with standard-of-care data alone provides a glimpse into the future of oncology and drug development. With this new funding, the company now has the resources to move one step closer to delivering on its mission.

"Since meeting Tushar and the SimBioSys team, Genoa Ventures has been excited about the enormous potential for the TumorScope platform to democratize insights for precision medicine in cancer care," said Vikram Chaudhery, Principal at Genoa Ventures. "For the first time, any hospital, clinic or cancer center can make truly informed decisions in choosing the best treatment protocols for patients, based on the standard pre-existing patient data available, eliminating the need for additional, expensive wet-lab testing."

Unlike current approaches, SimBioSys’ first-of-its-kind application combines artificial intelligence with biophysical simulations to model the impact of phenomena such as drug delivery, metabolism, and spatial heterogeneity in a comprehensive model using standard-of-care data alone. The results are generated within minutes, enabling physicians to make a well-informed decision while improving patient experience and shared decision-making. In addition, the technology can support the drug development process across pre-clinical and clinical trial settings.

"Robust clinical and patient-reported data is critical to assess and prescribe the best options of cancer care for patients," said Andrea Jackson, Director at Northpond Ventures. "The SimBioSys TumorScope virtualizes cancer to simulate – in minutes – a patient’s tumor response to therapies. Simulating response before prescribing treatment is a significant stride in personalized treatment planning. Northpond is grateful to partner with Tushar and the SimBioSys team on this important work."

The company’s name, SimBioSys, and logo capture its core scientific approach – Simulating Biological Systems. SimBioSys’ TumorScope virtualizes cancer in 3D and can accurately simulate how a patient’s tumor will respond to a variety of therapies following diagnosis. The new funding will allow SimBioSys to expand its state-of-the-art technology into other solid tumors beyond its current focus on breast cancer. Additionally, the new capital will drive commercialization efforts to bring the technology to patients and the biopharma industry. Andrea Jackson at Northpond Ventures and Vikram Chaudhery at Genoa Ventures will join the SimBioSys Board of Directors.

On October 26, 2021 Indivumed, the developer of the world’s leading discovery platform for precision oncology, and Biognosys, a leader in next-generation proteomics solutions for discovery and drug development, reported the extension of their strategic partnership to provide biopharmaceutical customers with immunopeptidomic perspectives in drug discovery in oncology (Press release, Indivumed, OCT 26, 2021, View Source [SID1234591979]).

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The partnership builds on each company’s unique capabilities in omics research, with Biognosys providing proteomic technology and solutions based on mass spectrometry, and Indivumed providing high-quality samples and data, as well as its multi-discovery AI system. -omics, nRavel .

The initial partnership created in December 2019 aimed to enrich IndivuType, Indivumed’s multi-omic cancer database, with proteomic data from thousands of samples analyzed by Biognosys. Since then, Indivumed and Biognosys have successfully collaborated on various clinical research projects and published the results of a large-scale lung cancer study at AACR (Free AACR Whitepaper) 2020 , unveiling new biomarkers for tumor biology. Building on this success, the companies are extending their partnership with immunopeptidomics research.

Immunopeptides play an essential role in the immune system, and can be analyzed to support the development of personalized treatments, in particular for cancers, vaccines, and infectious and autoimmune diseases. Mass spectrometry is currently the only technology to reliably measure and identify immunopeptide profiles from large-scale biological samples.

Biognosys Immunopeptide Stream deploys its patented Hyper Reaction Monitoring (HRM ) mass spectrometry technology for proteome quantification, in conjunction with its proprietary data analysis software Spectronaut and SpectroMine to identify over 10,000 unique immunopeptides from small quantity samples. Highly efficient and scalable, the flux can be used in large-scale clinical studies.

Indivumed can then provide biopharmaceutical customers with further validation and insight on the immunopeptide signatures identified through the Biognosys feed, leveraging IndivuType, the multi-omics cancer database , and the nRavel analytical platform. in order to optimize R&D activities and new discovery programs for individualized therapy.

Biognosys present validate the sensitivity and reproducibility of the workflow during the annual conference of the American Society of Mass Spectrometry (ASMS) on 1 st November Philadelphia. Indivumed will present a clinically relevant multi-omic analysis at the BioData World Congress on November 2 in Basel.

The new immunopeptidomics offer is already being applied successfully to third-party biopharmaceutical companies in order to optimize their drug discovery efforts.

Lukas Reiter, PhD, Chief Technology Officer, Biognosys: " Biognosys HRM technology uniquely addresses key challenges in immunopeptidomics research. Using only small amounts of tissue, our optimized flow provides a comprehensive biological perspective on the immune system. We are delighted to apply this solution to Indivumed’s valuable tissue samples and to support drug discovery in areas of significant unmet need. "

Roald Forsberg, Sales Manager and Head of IndivuType Business Unit at Indivumed: "With our IndivuType database and our nRavel AI system , we have the perfect opportunity to make new discoveries about the role of the immune response in the tumor microenvironment, and to develop novel immunotherapies against cancer.This extended partnership with Biognosys gives us the opportunity to validate and deepen our findings using mass spectrometry technology that will accelerate the transition between these discoveries and the practice of personalized oncology. "

On October 26, 2021 Castle Biosciences, Inc. (Nasdaq: CSTL), a company applying innovative diagnostics to inform disease management decisions and improve patient outcomes, reported new data further validating the performance of the Company’s DecisionDx DiffDx-Melanoma test as a highly accurate, ancillary tool to aid in the diagnosis of melanocytic lesions with ambiguous histopathology (Press release, Castle Biosciences, OCT 26, 2021, View Source [SID1234591978]). The data was shared in a poster presentation at The American Society of Dermatopathology (ASDP) 58th Annual Meeting, held virtually Oct. 20-24.

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DiffDx-Melanoma is part of Castle’s Comprehensive Diagnostic Offering (CDO), leveraging the strengths of both myPath Melanoma and DiffDx-Melanoma. These two gene expression profile (GEP) tests were designed to provide a highly accurate, objective result to aid dermatopathologists and dermatologists in characterizing difficult-to-diagnose melanocytic lesions.

The poster, titled "Performance of the 35 Gene Expression Profile (GEP) Test for Use as an Adjunctive Melanoma Diagnostic Tool in a New Independent Validation Cohort," describes the performance of DiffDx-Melanoma, independent of myPath Melanoma and also as part of the clinical CDO workflow. The poster can be viewed here.

"In clinical practice, difficult-to-diagnose melanocytic lesions often present a challenge for dermatopathologists, particularly when there is hesitation in the diagnosis of melanoma," said Gregory A. Hosler, M.D., Ph.D., dermatopathologist at ProPath in Dallas. "In this study, DiffDx-Melanoma demonstrated its ability to provide dermatopathologists additional information to help guide and potentially increase confidence in a diagnosis, if any uncertainty exists, to help clinicians provide their patients with the most appropriate care."

Study background and findings:

The Institutional Review Board (IRB)-approved study reviewed 644 melanocytic lesions and associated de-identified clinical data from patients ≥ 18 years of age.
Lesions were independently reviewed by at least two dermatopathologists for diagnostic adjudication and included in the study if they received at least two out of three reviews that were diagnostically concordant.
Accuracy metrics demonstrated highly accurate performance of the DiffDx-Melanoma test (96% sensitivity and 92.2% specificity; intermediate result 2.3%) across lesion subtypes reported (excluding spitzoid lesions), including those with subtype discordance.
Separately, the clinical Comprehensive Diagnostic Workflow leverages myPath Melanoma with DiffDx-Melanoma. Clinical order data from June 3-Aug. 31, 2021, showed that this workflow was able to increase the reporting of actionable test results of either benign or malignant from 78% for myPath Melanoma alone to 99% when used in conjunction with DiffDx-Melanoma.
Overall, the study data demonstrated that DiffDx-Melanoma was an accurate, objective ancillary tool that provided information to aid in the diagnosis of melanocytic lesions of uncertain malignant potential, thus enhancing diagnostic confidence and the delivery of clinically, actionable results to healthcare providers.
About Castle Biosciences’ Comprehensive Diagnostic Offering for Difficult-to-Diagnose Melanocytic Lesions

Castle Biosciences’ comprehensive diagnostic offering leverages the strengths of myPath Melanoma and DecisionDx DiffDx-Melanoma. These gene expression profile tests are designed to provide a highly accurate, objective result to aid dermatopathologists and dermatologists in characterizing difficult-to-diagnose melanocytic lesions. Of the approximately 2 million suspicious pigmented lesions biopsied annually in the U.S., Castle estimates that approximately 300,000 of those cannot be confidently classified as either benign or malignant through traditional histopathology methods. For these cases, the treatment plan can also be uncertain. Obtaining highly accurate, objective ancillary testing can mean the difference between a path of overtreatment or the risk of undertreatment. Interpreted in the context of other clinical, laboratory and histopathologic information, myPath Melanoma and DecisionDx DiffDx-Melanoma are designed to reduce uncertainty and provide confidence for dermatopathologists and help dermatologists deliver more informed patient management plans.

More information about the test and disease can be found at www.CastleTestInfo.com.

On October 26, 2021 Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to cures for cancer and other serious diseases, reported that it will be hosting a virtual R&D Day event detailing the company’s proprietary NAM-enabled natural killer (NK) cell therapy pipeline today, Tuesday, October 26, at 8:00 a.m. ET (Press release, Gamida Cell, OCT 26, 2021, View Source [SID1234591977]). During the event, the company will highlight Gamida Cell’s new programs leveraging next-generation, NAM-enabled, genetically modified NK cells in development for solid tumors and hematological cancers, as well as provide an update on the clinical development of GDA-201, its lead cryopreserved, off-the-shelf cell therapy candidate for the treatment of patients with follicular and diffuse large B cell lymphomas, including an update on the status of its Phase 1/2 Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA).

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Update Regarding IND Application for GDA-201:

The company recently submitted an IND application to FDA for a Phase 1/2 trial with a cryopreserved formulation of GDA-201 in patients with diffuse large B cell lymphoma and follicular lymphoma and was notified that the IND application has been placed on Clinical Hold prior to the initiation of patient dosing. The FDA has requested modifications in donor eligibility procedures and sterility assay qualification. Gamida Cell is in active communication with the FDA with the objective to promptly address these requests to potentially enable the requirements for IND acceptance and study initiation. The initiation of the planned Phase 1/2 trial of GDA-201 may be delayed beyond the end of 2021, pending the outcome of FDA interactions.

"While we work to resolve outstanding issues with our IND, we are pleased to be able to share updates regarding our NAM-enabled NK cell programs," said Julian Adams, Ph.D., Chief Executive Officer of Gamida Cell. "We believe that the issues raised by FDA are addressable and can hopefully be resolved in an expeditious manner. In the meantime, we are pleased to elaborate on the power of NAM combined with the genomic tools that we have harnessed to enable us to create potentially transformative immuno-oncological therapies that may move beyond what is currently possible with existing approaches. These advances in our NK cell pipeline will help to further our mission to bring cancer patients potentially curative cell therapies."

Update Regarding NK Cell Therapy Programs:

During today’s virtual event, Gamida Cell management and partners will provide an overview of the company’s NK cell programs, including:

Objective to improve treatment of both hematological cancers and solid tumors in which genetic modifications to allogeneic NK cells may overcome immunosuppressive microenvironments.
Review of Gamida Cell’s proprietary NAM expansion process, which enhances the potency, function and persistence of NK cells while improving homing to and retention in lymphoid tissues.
Descriptions of Gamida Cell’s genetically modified NK cell immunotherapy programs (GDA-301, GDA-401, GDA-501 and GDA-601), which utilize CAR- and CRISPR-mediated strategies to increase targeting, potency and persistence against hematologic malignancies and solid-tumors.
Discuss a research collaboration with the Dana-Farber Cancer Institute studying the in vitro natural killer (NK) cell killing activity of GDA-601, Gamida Cell’s nicotinamide (NAM)-enabled genetically modified NK cell therapy in multiple myeloma. GDA-601 is a CD38 CRISPR knockout combined with a CD38 CAR NK cell construct that has demonstrated promising preclinical results, including reduced fratricide and increased cytotoxicity against a multiple myeloma cell line.
Phase 1 data on the safety and efficacy of GDA-201, a NAM-enabled, unmodified allogeneic NK cell therapy that has produced positive clinical results in the treatment of diffuse large B cell lymphoma and follicular lymphoma, both of which have significant unmet need.
"This is an exciting time for Gamida Cell as we have expanded R&D activities to augment our NK cell pipeline," said Ronit Simantov, M.D., Chief Medical Officer of Gamida Cell. "During today’s event, we will share our plans to advance the clinical development of GDA-201 based on highly encouraging clinical data in patients with lymphoma that have arisen from a physician sponsored study. We also plan to illustrate how our proprietary NAM expansion process, combined with our advanced genetic modifications, differentiate our NK cell programs as may meaningfully help patients with solid tumors and hematologic malignancies."

A replay of the webcast will be available on the "Investors & Media" section of Gamida Cell’s website at www.gamida-cell.com, and will be available for at least 14 days following the event.

About GDA-201

Gamida Cell applied the capabilities of its nicotinamide (NAM)-enabled cell expansion technology to develop GDA-201, an innate NK cell immunotherapy for the treatment of hematologic and solid tumors in combination with standard of care antibody therapies. GDA-201, the lead candidate in the NAM-enabled NK cell pipeline, has demonstrated promising initial clinical trial results, as reported at the 2020 American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting & Exposition1. GDA-201 addresses key limitations of NK cells by increasing the cytotoxicity and in vivo retention and proliferation in the bone marrow and lymphoid organs. Furthermore, GDA-201 improves antibody-dependent cellular cytotoxicity (ADCC) and tumor targeting of NK cells. For more information about GDA-201, please visit View Source

GDA-201 is an investigational therapy, and its safety and efficacy have not been established by the FDA or any other health authority.