Western Sydney University

Data presented at the 2024 FMF (Fetal Medicine Foundation) World Congress in Lisbon, Portugal shows the potential for an automated system to deliver genomic profiles of fetal cells that is highly concordant with the genomic analysis obtained from invasive procedures. Results of a large clinical validation study show that Menarini Silicon Biosystems' fetal cell based noninvasive prenatal screening (NIPT) technology can accurately detect both fetal genome-wide pathogenic copy number variants greater than 600Kb in size and the commonly screened trisomy conditions. BOLOGNA, Italy and HUNTINGDON VALLEY, Pa., July 2, 2024 /PRNewswire/ --The Reproductive Precision Medicine Unit of Menarini Silicon Biosystems (MSB) presented last week, at the 21st World Congress of the Fetal Medicine Foundation meeting in Lisbon, Portugal, results of a large multicenter study describing their next generation non-invasive prenatal testing technology using fetal cells isolated from maternal blood. The genomic analysis of these fetal cells showed a high concordance with the analysis of fetal cells obtained from invasive diagnostic procedures. Moreover, MSB's cell-based test, under development, demonstrated its potential validity for screening genomic conditions not easily found with currently available, state of the art, non-invasive screening technologies based on cell-free DNA (cfDNA) analysis. This large study, which enrolled over 1,000 women, was centered on isolating individual fetal (trophoblast) cells from maternal blood and analyzing them for both common trisomic conditions and genome-wide microdeletions and microduplications, called pathogenic copy number variants (pCNVs), that account for significant perinatal morbidity and mortality. The results presented showed that MSB's fetal cell based NIPT could deliver information beyond core "common" trisomies detected by standard non-invasive cfDNA analysis, as well as detect with a high level of accuracy and granularity genome-wide microdeletions and microduplications down to a size of at least 600Kb. The cell-based test was compared with chromosomal microarray analysis (CMA) and karyotype from chorionic villus sampling (CVS) or amniocentesis, the clinical gold - standard fetal diagnostic methodologies to detect genomic chromosomal abnormalities in the prenatal setting. According to Professor Jon Hyett, Head of Maternal and Fetal Medicine at Liverpool Hospital and Professor of Obstetrics and Gynaecology at Western Sydney University, who looks after pregnancies that have a high risk of complication – either for the mother, or for the fetus: "This data is exciting because it shows the potential to deliver clinically relevant and actionable information about fetal genomic abnormalities at higher resolution and accuracy than existing screening tests and at an early gestational age when almost no pCNVs are currently detected." Menarini's new study thereby opens the door to a whole new paradigm in prenatal screening. For Thomas Musci, MD, Chief Medical Officer, Head of Menarini Silicon Biosystems' Reproductive Precision Medicine Business Unit, who presented the results of the study, "Isolating intact fetal cells from maternal blood for prenatal screening has long been perceived as an extremely challenging goal. Our highly automated system for the isolation and single–cell analysis of circulating extravillous trophoblasts (cEVTs) supports the feasibility of a cell–based NIPT for fetal genomic profiling that can lead to more informed decision-making at all levels." Menarini has been actively investing to advance single cell analysis and sequencing in the field of reproductive care. For Fabio Piazzalunga, President of Menarini Silicon Biosystems, "The results of this study, which confirmed the potential ability of our cell based NIPT to identify fetal abnormalities with high sensitivity, accuracy, and specificity, show the potential of Menarini to significantly impact women's health. Our continuous commitment and efforts to advance our scientific findings in this field aim to provide, in the future, a potentially revolutionary solution that brings more information to women and their doctors". These new activities fully support the company's vision to become a leader in minimally invasive cell-based applications that can allow for easier, faster, and more precise diagnostic and therapeutic approaches in multiple therapeutic areas. About Menarini Silicon Biosystems (MSB) MSB offers unique rare cell technologies and solutions that provide clinical researchers with access to unparalleled resolution in the study of cells and their molecular characterization. Menarini Silicon Biosystems, based in Castel Maggiore (Bologna, Italy), and Huntingdon Valley, PA., U.S., is a wholly owned subsidiary of the Menarini Group, a multinational pharmaceutical, biotechnology and diagnostics company headquartered in Florence, Italy, with more than 17,000 employees in 140 countries. Logo: Contact: PAVY Consulting Linda PAVY [email protected]

CAMBRIDGE, Mass. & SALISBURY, England--( BUSINESS WIRE )--KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors, today announced that it presented real-world data from US patient surveys that assessed the experience of HAE patients using injectable on-demand treatments at the 2024 American Academy of Allergy, Asthma & Immunology (AAAAI) Annual Meeting that took place in Washington, DC. The following presentations occurred at AAAAI 2024: Characteristics of Hereditary Angioedema Attacks Among Long-Term Prophylaxis Users: Bob Geng, Allergy and Immunology, University of California, San Diego, California, United States (Poster Presentation) Among HAE patients who had treated a recent attack, the location and duration of the most recent attacks were similar between long-term prophylaxis (LTP) and on-demand only users Earlier on-demand treatment was associated with a shorter attack duration, regardless of LTP use Delayed On-demand Treatment of Hereditary Angioedema Attacks: Patient Perceptions and Associated Barriers: Sandra Christiansen, University of California San Diego, La Jolla, CA, United States (Poster Presentation) Despite their perception of treating attacks “early”, many patients did not meet guideline recommendations for prompt on-demand treatment after recognition of an HAE attack The most common barriers to earlier treatment were uncertainty if attack was real, thinking the attack would be mild, and wanting to save treatment for a severe attack Anxiety Associated with On-Demand Treatment for Hereditary Angioedema (HAE) Attacks: James Wedner, Washington University School of Medicine, St Louis, MO, United States (Poster Presentation) Both adults and adolescents with HAE reported moderate to extreme anxiety when anticipating use of parenteral on-demand treatment, irrespective of use of on-demand only or on-demand plus LTP The results of this study highlighted the association between delayed treatment and treatment-related anxiety, with a higher proportion of patients who delayed treatment experiencing moderate to extreme anxiety Anxiety Associated with Refilling On-demand Therapy for HAE Attacks Contributes to Treatment Delay and Non-Treatment: Autumn Burnette, Division of Allergy and Immunology, Howard University Hospital, Washington, DC, United States (Poster Presentation) Anxiety associated with not being able to refill on-demand treatment quickly impacted treatment decisions, which contributed to treatment delay or resulted in non-treatment of HAE attacks One-third of patients using icatibant did not treat or delayed treatment due to anxiety about refills Characterizing the Negative Impact of Delayed On-Demand Treatment of HAE Attacks: Princess Ogbogu, Division of Pediatric Allergy, Immunology, and Rheumatology, University Hospitals Rainbow Babies and Children's Hospital, Cleveland, Ohio, United States (Poster Presentation) People living with HAE understood the importance of treating HAE attacks early and recognized that earlier treatment translates to quicker recovery and resolution Delayed on-demand treatment (> 1 hour) lengthened the time to “feeling in control” of an attack and the time to feeling fully recovered The Impact of On-demand Treatment on Quality of Life of People with HAE: Paula Busse, Department of Medicine, Division of Clinical Immunology, Mount Sinai, New York, United States (Poster Presentation) People with HAE needed to make adjustments to their daily lives including avoiding situations where their injectable on-demand treatment may be discovered by others Embarrassment associated with carrying on-demand treatment was among the reasons HAE patients delayed administration of on-demand treatment Characterizing the Perspective of Patients With HAE on Prophylactic Treatment: Stephen Betschel, Division of Allergy and Immunology, Department of Medicine, St. Michael’s Hospital, University of Toronto, Toronto, Ontario, Canada (Poster Presentation) Only 35% of LTP patients always carried on-demand treatment when away from home, while 43% of prophylaxis patients cited avoiding potential triggers as a reason for not carrying on-demand treatment at all times Nearly half of patients on LTP patients experienced moderate to extreme levels of anxiety when anticipating on-demand treatment administration Treatment Patterns of Patients Requiring Redosing of an On-demand Treatment After the Return of an HAE Attack: Constance Katelaris, Department of Medicine, Campbelltown Hospital and Western Sydney University, Sydney, NSW, Australia (Poster Presentation) Almost one third of people with HAE experienced the return of an HAE attack requiring ≥1 additional dose of on-demand treatment HAE attacks treated within 1 hour returned less frequently compared with attacks treated after ≥1 hour “The results of these surveys clearly conveyed the challenges faced by patients trying to manage their HAE attacks with injectable on-demand treatments. The resulting non-compliance with treatment guidelines may lead to poor clinical outcomes, even among patients receiving LTP,” said Andrew Crockett, Chief Executive Officer of KalVista. “We believe the efficacy and safety data from our phase 3 trial for sebetralstat show a potential path forward to address these persisting unmet needs.” Links to all posters and presentations can be found on the KalVista website under “Publications”. About KalVista Pharmaceuticals, Inc. KalVista Pharmaceuticals, Inc. is a pharmaceutical company focused on the discovery, development, and commercialization of oral, small molecule protease inhibitors for diseases with significant unmet need. KalVista disclosed positive phase 3 data for the KONFIDENT trial for its oral, on-demand therapy sebetralstat in February 2024. The Company anticipates submitting a new drug application to the U.S. FDA for sebetralstat in the first half of 2024 and expects to file for approval in Europe and Japan later in 2024. In addition, KalVista’s oral Factor XIIa inhibitor program represents a new generation of therapies that may further improve the treatment for people living with HAE and other diseases. For more information about KalVista, please visit www.kalvista.com . Forward-Looking Statements This press release contains "forward-looking" statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Forward-looking statements can be identified by words such as: "anticipate," "intend," "plan," "goal," "seek," "believe," "project," "estimate," "expect," "strategy," "future," "likely," "may," "should," "will" and similar references to future periods. These statements are subject to numerous risks and uncertainties that could cause actual results to differ materially from what we expect. Examples of forward-looking statements include, among others, timing or outcomes of communications with the FDA, our expectations about safety and efficacy of our product candidates and timing of clinical trials and its results, our ability to commence clinical studies or complete ongoing clinical studies, including the KONFIDENT-S trial, and to obtain regulatory approvals for sebetralstat and other candidates in development, the success of any efforts to commercialize sebetralstat, the ability of sebetralstat and other candidates in development to treat HAE or other diseases, and the future progress and potential success of our oral Factor XIIa program. Further information on potential risk factors that could affect our business and financial results are detailed in our filings with the Securities and Exchange Commission, including in our annual report on Form 10-K for the year ended April 30, 2023, our quarterly reports on Form 10-Q, and our other reports that we may make from time to time with the Securities and Exchange Commission. We undertake no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

TUESDAY, Jan. 23, 2024 -- Soy, enterolactone, and green tea are associated with reductions in adverse breast cancer outcomes, according to a systematic review and meta-analysis published in the February issue of JNCI Cancer Spectrum . M. Diana van Die, Ph.D., from Western Sydney University in Penrith, Australia, and colleagues reviewed prospective and retrospective observational studies examining the impact of soybean, lignans, cruciferous vegetables, green tea, or their phytonutrients on breast cancer survival outcomes. Data were included from 32 studies. The researchers found that the risk for recurrence was reduced in association with soy isoflavones (hazard ratio, 0.74), especially among postmenopausal and estrogen receptor-positive survivors (hazard ratios, 0.72 and 0.82, respectively), with the greatest reduction in risk seen at 60 mg/day. The reduction was mainly at 20 to 40 mg/day for mortality outcomes. An inverse association was seen for soy protein and soy products with cancer-specific mortality for estrogen receptor-positive disease (hazard ratio, 0.75). Serum or plasma enterolactone, measured prediagnosis and early postdiagnosis, was inversely associated with cancer-specific mortality and all-cause mortality (hazard ratios, 0.72 and 0.69, respectively). There were no effects observed for cruciferous vegetables. Prediagnostic green tea intake was associated with a reduced risk for recurrence for stage I and II breast cancer (hazard ratio, 0.56). "To further inform clinical practice, evidence is required on the impact of dietary and supplemental intakes of phytonutrients introduced or substantially increased following diagnosis and treatment," the authors write. Several authors disclosed ties to industry. Abstract/Full Text Disclaimer: Statistical data in medical articles provide general trends and do not pertain to individuals. Individual factors can vary greatly. Always seek personalized medical advice for individual healthcare decisions. Posted January 2024 More news resources FDA Medwatch Drug Alerts Daily MedNews News for Health Professionals New Drug Approvals New Drug Applications Drug Shortages Clinical Trial Results Generic Drug Approvals Subscribe to our newsletter Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.