On September 12, 2022 Bluestar Genomics, Inc., an early cancer detection company leading the development and commercialization of next-generation liquid biopsy tests initially focused on non-invasive detection of high-mortality cancers in high-risk patients, reported the results from multiple studies highlighting the expansion of the company’s technology platform into ovarian cancer detection and tumor tissue analysis (Press release, Bluestar Genomics, SEP 12, 2022, View Source [SID1234619456]).

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These new data highlight promising research on the 5hmC biomarker-based approach to noninvasive detection of a broad range of cancers including those with poor survival like pancreatic and ovarian. They show the utility of epigenomics in cancer detection beyond the company’s growing body of evidence associated with its lead pancreatic cancer detection program. The latest scientific presentations underscore the clinical promise of the 5hmC biomarker in cancer detection and monitoring – including high-mortality and common cancers, such as breast, colon, lung, ovary, and pancreas.

"Pancreatic cancer is only one of many cancer types that can be detected using epigenomics and the 5hmC biomarker using our core platform technology," said Samuel Levy, PhD, chief scientific officer of Bluestar Genomics. "Our current results demonstrate the accuracy of our blood-based cancer detection technology and the feasibility of widespread deployment of epigenomic technology, not only for early cancer detection, but also for understanding other biological changes, such as developing resistance to cancer treatments."

The Bluestar Genomics approach to detecting cancer early is anchored in the process of tracking changes in cells in the body through unprecedented measurement of levels of the biomarker 5-hydroxymethylcytosine (5hmC) in a person’s blood. The 5hmC biomarker is stable, sensitive, and precise enough to detect not just the presence of cancer, but to also specify the location.

Advances in Genome Biology and Technology (AGBT) Precision Health, September 8-10, San Diego, Calif., U.S: 5hmC Technology Platform Shows Promise as Ovarian Cancer Detection Tool

At the AGBT Precision Health Conference, Bluestar Genomics presented a poster titled: Detection of Early-Stage Ovarian Cancer Using 5-Hydroxymethylation Profiles in Plasma-Derived Cell." The results from a research study looking at isolating cell-free DNA and 5hmC from blood samples of ovarian cancer patients and non-cancer controls and leveraging whole genome sequencing in conjunction with proprietary bioinformatics and machine learning algorithms to identify samples from patients with ovarian cancer.

Ovarian cancer is the fifth-leading cause of cancer-related deaths among women and is often diagnosed in the advanced stage due to the lack of early detection tools.

The early development study results showed strong performance (83% sensitivity at 98% specificity) demonstrating the potential of the Bluestar Genomics 5hmC technology platform as an effective tool for ovarian cancer detection.

European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper), September 9-13, Paris, France: 5hmC-based Epigenomic Analysis Reveals Strong Biological Association Between cell free DNA and Tumor Tissue

For the first time at this conference, Bluestar Genomics’ technology was highlighted in an oral presentation discussing research looking at tissue samples to understand how genes possess distinct 5hmC features that are different for cancer tissues and normal tissues. The large study included 1,009 cancer subjects and 1,678 non-cancer controls. The results showed that the differences in tissue-specific genes and 5hmC persisted across several cancer types studied (breast, colon, lung, ovary, and pancreas) and enabled highly accurate classification of cancer and normal tissues when compared with a machine learning model. Collectively, these features enable the identification of cancer from cell-free DNA across these cancer types. "5-hydroxymethycytosine Analysis Reveals Stable Epigenetic Changes in Tumor Tissue that Enable cfDNA Cancer Predictions" is available here.

American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Pancreatic Cancer, September 13-16, Boston, MA, U.S.: Large Study Validates 5hmC-based Algorithm for Early Detection

At the American Association for Cancer Research (AACR) (Free AACR Whitepaper): Special Conference on Pancreatic Cancer, a poster on the clinical validation efforts for the Bluestar Genomics pancreatic cancer classification model. The study validated an early-stage pancreatic cancer classification model using 5hmC profiles in plasma-derived cell-free DNA.

The researchers isolated cell-free DNA and analyzed 5hmC profiles from whole blood samples from 4,408 patients inclusive of both patients with pancreatic cancer and non-cancer controls. The validation of the pancreatic cancer detection algorithm on 2,150 patients showed a performance of 68 % sensitivity in stage I-II pancreatic cancer patients. All-stage performance results showed sensitivity of 67% and a specificity of 97%.

Pancreatic cancer outcomes are poor mostly due to the detection of cancer at late stages, underscoring the need for early detection.

The poster "Validation of an early-stage pancreatic cancer classification model using 5 Hydroxy-methylation profiles in plasma-derived cell-free DNA" can be found here.

On September 12, 2022 Scorpion Therapeutics, Inc. ("Scorpion Therapeutics"), a pioneering oncology company redefining the frontier of precision medicine, reported the appointment of Michael Streit, M.D., M.B.A as the Company’s first Chief Medical Officer ("CMO") (Press release, Scorpion Therapeutics, SEP 12, 2022, View Source [SID1234619455]).

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"We are pleased to welcome Michael to Scorpion at this exciting time in the Company’s evolution, as we shape our first clinical development plans and prepare to submit investigational new drug ("IND") applications for both of our development candidates, STX-478 and STX-721, in 2023," said Axel Hoos, M.D., Ph.D., Chief Executive Officer of Scorpion Therapeutics. "Michael is an industry veteran with extensive experience in ‘bench to bedside’ oncology drug development. He has also helped advance the field of personalized medicine by developing novel targeted therapies paired with companion diagnostic assays. Over the course of his career, Michael has led clinical studies for innovative medicines including both best-in-class and first-in-class molecules, and he will play a critical role in advancing our broad discovery pipeline into efficient and well-designed clinical trials."

Dr. Streit brings over 20 years of oncology and clinical development expertise to Scorpion Therapeutics. Prior to joining the Company, he served as Vice President, Senior Global Project Head at Sanofi, where he was responsible for leading the development of SAR444245 (THOR-707), Sanofi’s precisely PEGylated, not-alpha interleukin-2 candidate. Before that, Dr. Streit served in roles of increasing responsibility at GlaxoSmithKline, including Vice President of Development, where he led the development of multiple oncology medicines. Previously, he was a Senior Director at Janssen, where he worked as the early development lead for the company’s prostate cancer and hematology portfolios and supported the Phase 1 development of multiple early development assets. Dr. Streit received his M.D. from the Free University of Berlin and his M.B.A. in International Business from Golden Gate University, San Francisco. He completed a post-doctoral fellowship in at the Cutaneous Biology Research Center at Massachusetts General Hospital.

"I’m thrilled to join the talented team at Scorpion Therapeutics and look forward to advancing the Company’s portfolio of next-generation cancer medicines into the clinic, beginning with its PI3Kα and EGFR Exon 20 programs," said Dr. Streit. "In preclinical studies, these compounds have demonstrated superior selectivity against well-validated oncogenic drivers, which may enable them to overcome the toxicity issues that limit currently available therapies and deliver superior safety, tolerability and efficacy. I look forward to advancing STX-478 and STX-721, as well as the Company’s more than 15 additional programs, through discovery and clinical development and fulfilling Scorpion’s mission to deliver optimized and transformational therapies to patients living with cancer."

On September 12, 2022 Strand Therapeutics reported that Fierce Biotech has named it as one of 2022’s Fierce 15 biotechnology companies, designating it as one of the most promising early-stage biotechnology companies in the industry (Press release, Strand Therapeutics, SEP 12, 2022, View Source [SID1234619454]).

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Founded by leaders in mRNA-based synthetic biology at MIT, Strand is creating the first platform for programmable, long-acting mRNA therapeutics. The company’s mRNA therapies combine genes for self-replication with genetically programmed logic circuits. By sensing and classifying unique expression signatures of cell types, the breakthrough technology enables precise and controlled delivery of multiple disease treatments in a single mRNA drug.

"It is an absolute honor to be recognized by Fierce Biotech as one of the most exciting and innovative companies in the biotech industry this year," said Jake Becraft, CEO & Co-Founder, Strand Therapeutics. "We’re unlocking the potential of mRNA technology, by developing next-generation programmable mRNA therapies for use beyond vaccines, but as potential treatments in oncology and broader indications with significant unmet need for patients."

The Fierce 15 celebrates the spirit of being "fierce" – championing innovation and creativity, even in the face of intense competition. This is Fierce Biotech’s 20th annual Fierce 15 selection.

An internationally recognized daily report reaching a network of over 450,000 biotech and pharma industry professionals, Fierce Biotech provides subscribers with an authoritative analysis of the day’s top stories. Every year Fierce Biotech evaluates hundreds of early-stage companies from around the world for its annual Fierce 15 list, which is based on a variety of factors such as the strength of its technology, partnerships, venture backers and a competitive market position.

On September 12, 2022 Universal Diagnostics (Universal DX), a bioinformatics and multi-omics company on a mission to transform cancer into a curable disease, reported the results of a proof of principle study demonstrating that the analysis of microbiome signatures in plasma can assist in early detection of colorectal cancer (CRC) (Press release, Universal Diagnostics, SEP 12, 2022, View Source [SID1234619453]).

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This advancement follows the company’s promising early findings that revealed early-stage colorectal cancer detection through analysis of cell-free circulating tumor DNA (ctDNA) methylation, mutation and fragmentation patterns by using targeted sequencing analysis, advanced computational biology and machine learning algorithms to detect colorectal cancer and advanced adenomas.

"While methylation, mutation and fragmentation are still the core of early CRC detection, we think microbiome is an interesting addition to our proprietary technological platform Signal-X as we build out the platform," said Christian Hense, COO at Universal DX. "As we learn more about microorganisms and how they interact in communities within our bodies to change the way we feel and function, we hope this type of data analysis will help patients get access to earlier and more sensitive screening, individualized guidance for treatment, and advanced monitoring techniques."

Over the last two decades, microbiome has become a key focus in the nutrition field, giving clinicians and patients a window into how diet and gut bacteria plays into overall health. The data presented by Universal DX shows that microbiome research can have a profound impact on other areas of healthcare, such as oncology. This body of research from Universal DX is being used to build "Signal-X", a platform to detect multiple types of cancer. Its first product, "Signal-C", detects early-stage colorectal cancer and adenomatous polyps.

"The data are clear: by detecting cancer earlier, we can save more lives," said Dr. James Kinross, Consultant Surgeon at Imperial College London and co-author of the study. "While we have a way to go until we can catch all cancers in their earliest stages, we continue to make great and promising progress. The work Universal DX is doing on Signal-X is one example. Much less invasive than a colonoscopy, accurate liquid biopsies will likely increase screening rates, improve outcomes and save lives."

Study Conclusions:

Changes in gut microbiota have been shown to have a link into colorectal cancer development and progression.
Measuring cancer-related microbiome alterations in plasma cell-free DNA (cfDNA) could offer an accurate, non-invasive approach for early cancer detection, leading to decreased cancer mortality.
cfDNA analysis coupled with model building achieved high sensitivity, including at stage I/II and stage III/IV, at equally accurate specificity.
Universal DX leverages proprietary, state-of-the-art computational biology tools combined with targeted next generation sequencing (NGS) assay platform that allows for simultaneous detection of methylation and microbiome signals for highly-sensitive cancer signal scoring of cell-free DNA regions linked to cancer of interest.

Hense continued: "Colorectal cancer is the third deadliest cancer in the United States. When it is detected early, the 5-year survival rate increases from 60% to 90%. This staggering difference is something we can’t ignore. At Universal DX, we are committed to finding better detection methods that catch dangerous cancers as early as possible, so that we can save lives. Microbiome signatures are a promising avenue, but this is just the beginning."

The company presented it findings live at ESMO (Free ESMO Whitepaper) in Paris on September 11th; abstract 358P – Analysis of microbiome signatures in plasma for early CRC detection.

On September 12, 2022 Gamida Cell Ltd. (Nasdaq: GMDA), the leader in the development of NAM-enabled cell therapies for patients with hematologic and solid cancers and other serious diseases, reported the presentation of data supporting the potential of omidubicel for the treatment of patients with blood cancers in need of an allogeneic hematopoietic stem cell transplant at the 2022 Cord Blood Connect Meeting, being held in South Beach, Florida (Press release, Gamida Cell, SEP 12, 2022, View Source [SID1234619452]). "We continue to be encouraged by the growing body of evidence supporting the improved outcomes and lower infection rates seen in patients treated with omidubicel as well as the superior health-related quality of life scores compared to transplantation with UCB. We also demonstrated the potential role for omidubicel to address the unmet need for patients who are currently eligible for transplant, but cannot find a match," said Julian Adams, chief executive officer of Gamida Cell. "Our omidubicel BLA was accepted by the FDA and granted priority review with a PDUFA date of January 30, 2023, which we believe further underscores the unmet need for patients with blood cancers in need of a stem cell transplant."

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Gamida Cell presented a poster titled "Health-Related Quality of Life (HRQL) Following Transplantation with Omidubicel Versus Umbilical Cord Blood (UCB) in Patients with Hematological Malignancies: Results from a Phase III Randomized, Multicenter Study," which included an analysis of 75 patients to evaluate changes in HRQL measures between the two study arms. Outcomes evaluated included Functional Assessment of Cancer Therapy General (FACT-G) domain scores for physical, social/family, functional and emotional well-being, and EQ-5D-3L index scores at days 42, 100, 180 and 365 post-transplant. During the first-year post-transplant, patients receiving omidubicel had numerically superior average FACT-G domain and EQ-5D-3L index scores compared to UCB, with mean differences across time points ranging from 1.4-3.1 for physical well-being, 0-1.3 for social/family well-being, 0.5-1.4 for emotional well-being, 1.6-3.2 for functional well-being, and 0.03-0.09 for the EQ-5D-3L index score. The data suggest meaningfully greater preservation or improvement of important HRQL domains in patients treated with omidubicel compared to UCB. Learn more

Gamida Cell also presented a poster titled "Projected Impact of Omidubicel on Racial and Ethnic Disparities in Allogeneic Hematopoietic Cell Transplant Access and Outcomes for Patients with Hematologic Malignancies in the US," which featured an analysis of projected impact of allogeneic hematopoietic cell transplant (allo-HCT) access and clinical outcomes in a hypothetical population of 10,000 allo-HCT-eligible patients with hematologic malignancies lacking an HLA-matched related donor. Assuming 20% omidubicel use, the proportion of patients receiving allo-HCT increased by 71% in Black, 43% in Asian, 30% in Hispanic, and 5% in white patients. The model suggests that access to omidubicel, upon approval, is projected to decrease time to allo-HCT and improve patient outcomes, with the greatest improvements among the racial and ethnic groups underserved by the current standard of care. Learn more

In a poster titled "Hematopoietic Stem Cell Transplantation (HSCT) with Omidubicel Leads to Robust Recovery and Diversity of T cells" patients treated with omidubicel were found to have robust and diverse T cell constitution. In an analysis of the T-cell development of 37 patients, patients transplanted with omidubicel demonstrated higher numbers of Recent Thymic Emigrants (RTEs) in peripheral blood at one year post transplant compared to transplantation with UCB, which suggest faster thymopoiesis and provide mechanistic rational for the lower infection rates and improved outcomes in these patients. Learn more

All three posters were made available beginning Saturday, September 10, 2022, 6:15-7:45 p.m. ET, during the 2022 Cord Blood Connect Meeting.

About Omidubicel

Omidubicel is an advanced cell therapy candidate developed as a potential life-saving allogeneic hematopoietic stem cell (bone marrow) transplant for patients with blood cancers. Omidubicel demonstrated a statistically significant reduction in time to neutrophil engraftment in comparison to standard umbilical cord blood in an international, multi-center, randomized Phase 3 study (NCT0273029) in patients with hematologic malignancies undergoing allogeneic bone marrow transplant. The Phase 3 study also showed reduced time to platelet engraftment, reduced infections and fewer days of hospitalization. One-year post-transplant data showed sustained clinical benefits with omidubicel as demonstrated by significant reduction in infectious complications as well as reduced non-relapse mortality and no significant increase in relapse rates nor increases in graft-versus-host-disease (GvHD) rates. Omidubicel is the first stem cell transplant donor source to receive Breakthrough Therapy Designation from the FDA and has also received Orphan Drug Designation in the US and EU.

Omidubicel is an investigational stem cell therapy candidate, and its safety and efficacy have not been established by the FDA or any other health authority. For more information about omidubicel, please visit View Source

About NAM Technology

Our NAM-enabling technology is designed to enhance the number and functionality of targeted cells, enabling us to pursue a curative approach that moves beyond what is possible with existing therapies. Leveraging the unique properties of NAM (nicotinamide), we can expand and metabolically modulate multiple cell types — including stem cells and natural killer cells — with appropriate growth factors to maintain the cells’ active phenotype and enhance potency. Additionally, our NAM technology improves the metabolic fitness of cells, allowing for continued activity throughout the expansion process.