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IRVINE, Calif., Oct. 14, 2024 (GLOBE NEWSWIRE) -- Helio Genomics (“Helio” or “the Company”), an AI-driven healthcare company specializing in diagnostics technology and test development for cancer detection, showcased the results from their latest collaboration with Ochsner Health, demonstrating the utility of cell-free DNA (cfDNA) methylation-based biomarkers for assessing biological aggressiveness and prognosis in patients with hepatocellular carcinoma (HCC) on Friday, October 11, 2024 at the 2024 San Antonio Liver Cancer Symposium in San Antonio, Texas. Dr. Shivani Mahajan, Head of Computational Science at Helio Genomics, presented a poster from the longitudinal study, entitled “Cell-free DNA methylation biomarkers for evaluating prognosis and treatment response in patients with hepatocellular carcinoma”. Additionally, Helio’s abstract was awarded as one of the top three abstracts by the Symposium Committee. “We are thrilled to be one of three top abstracts selected at the San Antonio Liver Cancer Symposium,” said Dr. Shivani Mahajan, Helio’s Head of Computational Science. “This award underscores the importance of having reliable biomarkers that can evaluate the biological aggressiveness in HCC patients to help physicians make better treatment decisions for improved patient outcomes. In this study, Helio’s methylation score outperformed other established metrics in predicting patients at high risk of progression and in identifying treatment responders, demonstrating that cfDNA methylation-based biomarkers have both diagnostic and prognostic utility.” “We were thrilled to collaborate with Helio Genomics, a company that is at the forefront of diagnostic technologies for early cancer detection,” said Paul Thevenot, PhD, Associate Professor of Research at Ochsner Health. “Reliable prognostic biomarkers for HCC are lacking and this study demonstrates that cfDNA methylation-based biomarkers have tremendous potential for addressing this unmet need in the field,” said Ari J. Cohen, MD, FACS, FAASLD, Director of the Multi-Organ Transplant Institute and Director of Transplant Research at Ochsner Health. Data Highlights: In this longitudinal study cohort (n=108), all enrolled patients were previously diagnosed with liver cirrhosis with varying underlying etiologies.All patients received liver-directed therapies.Blood specimens were drawn from patients at multiple time points starting at diagnosis (pre-treatment).Helio’s automated wet lab platform, ECLIPSE™, was used to generate targeted cfDNA methylation sequencing data.A model utilizing cell-free DNA (cfDNA) methylation-based features was trained (using a separate cohort) to distinguish between HCC and cirrhotic patients and applied to an independent longitudinal cohort of HCC patients to calculate a methylation score for each patient at multiple time points.Helio’s methylation model score from pre-treatment blood specimens outperformed other metrics such as the ALBI score, GALAD score, MELD score, Child-Pugh score, cumulative lesion size and biomarkers like AFP, AFP-L3 and DCP in predicting patient outcomes and can accurately classify patients with low or high risk of progression.Patients who responded to liver-directed therapy showed a significantly larger decline in their methylation score compared to those who did not respond and progressed to advance-stage disease.With biopsy contraindicated in early-to-intermediate-stage disease (BCLC A-B), circulating mediators (biomarkers / ctDNA) provide the best potential targets for assessing prognosis, identifying treatment responders, and ultimately developing personalized approaches to treatment. Helio was founded in 2016 and is headquartered in Irvine, CA. For more information, please visit our website at www.heliogenomics.com or www.linkedin.com/company/heliogenomics; information that may be important to investors will be routinely posted in both locations. About ECLIPSE™The ECLIPSE platform is Helio’s proprietary laboratory process designed to improve the accuracy and efficiency of cfDNA methylation pattern detection and quantification by optimizing each step of the cfDNA methylation capture process, which includes automated cfDNA extraction, library generation and enzymatic conversion, targeted capture and NGS. In particular, our ECLIPSE platform uses an enzymatic conversion approach as opposed to a traditional bisulfite conversion, which yields a higher quantity and quality of converted DNA. About Helio Genomics Helio Genomics is a commercial stage, AI-driven healthcare company specializing in diagnostics technology and test development for cancer detection. HelioLiver is a multi-analyte blood test that evaluates cell free DNA (cfDNA) methylation patterns, serum protein markers, and demographic information for the detection of hepatocellular carcinoma (HCC). For more information, please visit us at www.heliogenomics.com. Investor Contact: Jeremy Feffer Managing Director LifeSci Advisors jfeffer@lifesciadvisors.com

October 1, 2024 – Santhera Pharmaceuticals (SIX: SANN) announces the positive outcome of the LIONHEART study, confirming vamorolone’s distinctive action also as a mineralocorticoid receptor antagonist, setting it apart from other corticosteroids. The LIONHEART study, an open-label randomized, placebo- and eplerenone-controlled study involving 30 healthy adult male subjects, met its primary endpoint. It demonstrated a statistically significant increase in the urinary sodium/potassium ratio in the vamorolone arm compared to placebo (p<0.0001) following a fludrocortisone challenge. This increased ratio in urine provides clinical evidence for vamorolone’s unique mode of action also as a mineralocorticoid receptor antagonist (MRA) in humans. Combined with its known properties as a dissociative glucocorticoid receptor agonist, these findings further differentiate vamorolone’s pharmacological profile, distinguishing it from other corticosteroids. Cardiac complications such as cardiomyopathy are a leading cause of morbidity and mortality in boys with Duchenne muscular dystrophy (DMD). While treatment with corticosteroids (and ACE inhibitors) has demonstrated a delay in the onset of cardiomyopathy, the addition of MRAs including eplerenone to standard of care has also shown an improvement in left ventricular systolic dysfunction, the benefit of which increases with earlier initiation [1-3]. “Mineralocorticoid receptor antagonists are strongly recommended but late when cardiac function is already reduced and tend to be used in the presence of myocardial fibrosis as detected in magnetic resonance imaging,” explained Prof Karim Wahbi, PhD, MD, Cardiologist at the APHP Hospital Cochin, Paris, France. “What is intriguing about this mechanistic study is whether there is a synergistic benefit of the anti-inflammatory and MRA effects of vamorolone on the evolution of cardiac disease in children who started treatment early or if vamorolone could be of benefit to those who are already experiencing cardiac symptoms and wish to remain on a corticosteroid” “The LIONHEART study is an important milestone to establish the proof of concept for a cardioprotective potential of vamorolone,” stated Shabir Hasham, MD, Chief Medical Officer of Santhera. “We continue to collect data from patients who have been on vamorolone for up to seven years allowing us to better characterize long-term outcomes including any beneficial impact on cardiac complications in DMD.” AGAMREE is a novel drug with a mode of action based on binding to the same receptor as glucocorticoids but modifying its downstream activity. Moreover, it is not a substrate for the 11-β-hydroxysteroid dehydrogenase (11β-HSD) enzymes that may be responsible for local drug amplification and corticosteroid-associated toxicity in local tissues [4-7]. This mechanism has shown the potential to ‘dissociate’ efficacy from steroid safety concerns and therefore AGAMREE is positioned as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD [4-7]. In the pivotal VISION-DMD study, AGAMREE met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks of treatment and showed a good safety and tolerability profile [4]. The most commonly reported side effects were cushingoid features, vomiting, weight increase and irritability. Side effects were generally of mild to moderate severity. Currently available data show that AGAMREE, unlike corticosteroids, has been shown to be a dual mineralocorticoid antagonist and a dissociative glucocorticoid agonist based on animal experiments [7]. It has no restriction of growth [8] and no negative effects on bone metabolism as demonstrated by normal bone formation and bone resorption serum markers [9]. AGAMREE (vamorolone), an orphan medicinal product, is approved for use in the United States (Prescribing Information), the European Union (Summary of Product Characteristics) and the United Kingdom for the treatment of DMD. The LIONHEART study (SNT-I-VAM-026) is an open label randomized, 3-arm, parallel-group, placebo and eplerenone controlled study to evaluate the mineralocorticoid receptor antagonism (MRA) effect of vamorolone in 30 healthy adult male subjects after challenge with fludrocortisone, a known mineralocorticoid receptor agonist. The primary endpoint was defined as the ratio of sodium to potassium (Na/K) and the corresponding logarithm of the ratio (log10(10*Na/K) in urine at different time-points. Further analysis of the data is ongoing and will be presented at medical conferences. References: [1] Birnkrant et al. (2018) Lancet Neurol. 2018 Apr;17(4):347-361. doi: 10.1016/S1474-4422(18)30025-5. [2] Raman et a. (2015) Lancet Neurol 14: 153–161 doi:10.1016/s1474-4422(14)70318-7 [3] Raman et al. (2017) Orphanet J Rare Dis 12: 39. doi:10.1186/s13023-017-0590-8 [4] Dang UJ et al. (2024) Neurology 2024;102:e208112. doi.org/10.1212/WNL.0000000000208112. Link. [5] Guglieri M et al (2022). JAMA Neurol. 2022;79(10):1005-1014. doi:10.1001/jamaneurol.2022.2480. Link. [6] Liu X et al (2020). Proc Natl Acad Sci USA 117:24285-24293 [7] Heier CR et al (2019). Life Science Alliance DOI: 10.26508 [8] Ward et al., WMS 2022, FP.27 - Poster 71. Link. [9] Hasham et al., MDA 2022 Poster presentation. Link. Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure. Corticosteroids are the current standard of care for the treatment of DMD. Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular diseases with high unmet medical need. The Company has an exclusive license from ReveraGen for all indications worldwide to AGAMREE® (vamorolone), a dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. AGAMREE for the treatment of DMD is approved in the U.S. by the Food and Drug Administration (FDA), in the EU by the European Medicines Agency (EMA), and in the UK by the Medicines and Healthcare products Regulatory Agency (MHRA). Santhera has out-licensed rights to AGAMREE for North America to Catalyst Pharmaceuticals and for China to Sperogenix Therapeutics. For further information, please visit www.santhera.com. The content above comes from the network. if any infringement, please contact us to modify.

WEDNESDAY, Sept. 11, 2024 -- Conditions offering alternative explanations for lung cancer symptoms are associated with increased diagnostic intervals, according to a study published online Aug. 23 in the British Journal of Cancer . Imogen Rogers, from Brighton and Sussex Medical School in the United Kingdom, and colleagues examined the diagnostic interval from time of first presentation in primary care with a relevant sign/symptom to diagnosis date for 11,870 people with lung cancer diagnoses and associations between the diagnostic interval and 10 “competing demand” comorbidities and four “alternative explanation” conditions. The "competing demand" comorbidities included coronary heart disease, depression/anxiety, heart failure, hypertension, chronic kidney disease, osteoporosis, dementia, serious mental illness, epilepsy, and diabetes. The "alternative explanation" conditions were chronic obstructive pulmonary disease, asthma, chronic fatigue syndrome, and receiving a prescription for an angiotensin-converting enzyme inhibitor. The researchers found that the diagnostic interval was longer for patients with “alternative explanation” conditions, by 31 and 74 days in patients with one and two or more conditions, respectively, compared with those with none. In an adjusted analysis, the number of “competing demand” conditions did not remain significantly associated with the diagnostic interval. "This research underscores the need for heightened vigilance in patients with chronic respiratory conditions," Rogers said in a statement. "Recognizing that these conditions can mask the symptoms of lung cancer is crucial in ensuring timely diagnosis and treatment." Abstract/Full Text Whatever your topic of interest, subscribe to our newsletters to get the best of Drugs.com in your inbox.