AZ confirms cardiovascular safety with roxadustat results

pharmatimes | May 16, 2018

AstraZeneca has announced top-line results from the pooled cardiovascular (CV) safety analyses of its global Phase III roxadustat programme. One of the key CV safety endpoints is major adverse CV events (defined as MACE), and another key CV safety endpoint evaluated MACE plus heart failure requiring hospitalisation and unstable angina requiring hospitalisation (defined as MACE+). The first-in-class hypoxia-inducible-factor prolyl hydroxylase inhibitor (HIF-PHI) was evaluated in three separate tests: In the pooled analysis of over 4,300 patients of MACE/MACE+ in non-dialysis-dependent patients, based on the totality of the adjudicated evidence, the MACE/MACE+ analyses between roxadustat and placebo showed no clinically-meaningful difference. In the pool of 1,500 incident (newly-initiated) dialysis patients, MACE/MACE+ results indicate that incident dialysis patients on roxadustat do better than those who are on epoetin alfa. In the pooled analysis of around 4,000 dialysis-dependent patients, the MACE/MACE+ analyses between roxadustat and epoetin alfa showed no clinically-meaningful difference.

Spotlight

What happens when a patient receives blood transfusions for sickle cell disease? Watch this video to learn more about blood transfusions for people with sickle cell disease and test your knowledge with a quiz at the end.

Spotlight

What happens when a patient receives blood transfusions for sickle cell disease? Watch this video to learn more about blood transfusions for people with sickle cell disease and test your knowledge with a quiz at the end.

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PHARMA TECH

Salarius Pharmaceuticals and VolitionRx Enter into R&D Collaboration Agreement

Salarius Pharmaceuticals, Inc. | August 03, 2022

Salarius Pharmaceuticals, Inc. a clinical-stage biopharmaceutical company developing therapies for patients with cancer in need of new treatment options, and VolitionRx Limited a multinational epigenetics company, today announced the signing of a research and development collaboration to advance rapid epigenetic profiling using Volition’s Nu.Q® technology to support further development of Salarius’ clinical stage drug, seclidemstat. Seclidemstat, a novel oral, reversible, targeted LSD1 inhibitor that affects gene expression, is currently in Phase 1/2 clinical studies for solid and hematologic cancers where LSD1 is implicated in disease progression. Nu.Q® has been developed as simple, easy-to-use, cost-effective blood tests to diagnose and monitor a range of life-altering diseases including cancer in humans and animals. “We are delighted to collaborate with Volition and believe its Nu.Q® technology may provide valuable biomarker information as we seek to advance the development of seclidemstat in multiple clinical indications. Biomarkers allow for a noninvasive method for determining target engagement and potential drug activity in patients. So, this exciting research collaboration with Volition Rx Limited provides another tool to aid in the development of seclidemstat in clinic.” David Arthur, Chief Executive Officer of Salarius Gael Forterre, Chief Commercial Officer of Volition, added, “We are excited to be collaborating with Salarius as part of our Nu.Q® Discover program which offers biopharma companies and academia access to our state-of-the-art assays for rapid epigenetic profiling. We are looking forward to supporting Salarius in the development and release of their groundbreaking seclidemstat therapy, to directly benefit patients with cancer.” About VolitionRx VolitionRx Limited is a multinational epigenetics company that applies its Nucleosomics™ platform through its subsidiaries to develop simple, easy-to-use, cost-effective blood tests to help diagnose and monitor a range of life-altering diseases including some cancers and diseases associated with NETosis such as sepsis and COVID-19. Early diagnosis and monitoring have the potential not only to prolong the life of patients but also to improve their quality of life. The tests are based on the science of Nucleosomics, which is the practice of identifying and measuring nucleosomes in the bloodstream or other bodily fluid to indicate that disease is present. Volition is primarily focused on human diagnostics and monitoring but also has a subsidiary focused on animal diagnostics and monitoring. Volition's research and development activities are centered in Belgium, with an innovation laboratory and office in the U.S. and additional offices in London and Singapore. For more information, please visit the Volition Rx Limited website. About Salarius Pharmaceuticals Salarius Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company developing therapies for patients with cancer in need of new treatment options. Salarius’ product portfolio includes seclidemstat, Salarius’ lead candidate, which is being studied as a potential treatment for pediatric cancers, sarcomas and other cancers with limited treatment options, and SP-3164, an oral small molecule protein degrader. Seclidemstat is currently in a Phase 1/2 clinical trial for relapsed/refractory Ewing’s sarcoma and certain additional sarcomas that share a similar biology, also referred to as Ewing’s-related or FET-rearranged sarcomas. Seclidemstat has received fast track, orphan drug and rare pediatric disease designations for Ewing’s sarcoma from the U.S. Food and Drug Administration. Salarius is also exploring seclidemstat’s potential in several cancers with high unmet medical need, with an investigator-initiated Phase 1/2 clinical study in hematologic cancers underway at MD Anderson Cancer Center. Salarius has received financial support from the National Pediatric Cancer Foundation to advance the Ewing’s sarcoma clinical program and was a recipient of a Product Development Award from the Cancer Prevention and Research Institute of Texas.

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BUSINESS INSIGHTS

Cosán and Genomind Partner to Help Prevent Adverse Prescription Drug Events for Older Adults

Cosán and Genomind | July 20, 2022

Cosán, a provider of technology-driven preventative care services for older adults and the physicians that support them, and Genomind, a leading precision healthcare company, have partnered to fill an existing gap in aging adult care management by providing Cosán’s network of physicians with Genomind’s integrated precision medication management platform. The platform will provide insights that will help Cosán to achieve the best outcomes for their patient population, prevent adverse drug events and reduce avoidable costs of medication mismanagement. With over 40% of U.S. adults 65 years of age and older taking five or more prescription medicines, aging populations are at high-risk for adverse drug events. Through this partnership, Genomind’s analytic capabilities will provide Cosán with risk stratification insights, enabling Cosán clinicians to target individuals that are most likely to benefit from medication reviews and pharmacogenetictesting, also known as drug-gene testing. Genomind’s PGx test adds a layer of precision medicine that leverages genetics to help providers select appropriate medications and dosing. Equipped with advanced pharmacogenetic screening, clinical concierge services and access to expert pharmacists, Cosán’s care teams can then incorporate individualized insights into each patient’s chronic care management plan so medications are administered safely. “This partnership brings a new level of pharmacy and genetic expertise to the thousands of providers and the patients they support, marking yet another stride in the delivery of effective preventative care management services. These unique drug analytic and genetic testing capabilities provide Cosán’s network of physicians and care coordinators with another tool designed to help eliminate avoidable drug risks and improve overall care quality for vulnerable patients receiving care from their homes.” David Hunt, Founder & President at Cosán According to the CDC, adverse drug events cause approximately 1.3 million emergency department visits each year, and older adults are nearly seven times more likely to end up hospitalized after such events than younger persons. Many of these adverse events are caused by just a few drugs. This new platform will help physicians develop personalized therapeutic approaches for patients based on their individual genetic makeup and are designed to improve patient and physician satisfaction and reduce medical expenses. With the recent expansions by Medicare to cover PGx testing in certain circumstances, this population is particularly suited to dramatically benefit from this platform. “The prevalence and cost of medication mismanagement is unacceptable, costing thousands of lives and billions of dollars annually. We’re thrilled to join Cosán in helping to improve care management for older adults, who are at high risk for these adverse drug events,” said Cynthia Kilroy, Co-CEO, Genomind. About Cosán Group Cosán Group, established in 2015, is an industry-leading healthcare organization creating new pathways to modern aging with technology-driven preventative care services, offering concierge home care for older adults. Early market exposure in the delivery of technology and services to support the Chronic Care Management (CCM) program, and Behavioral Health Integration (BHI) programs with Remote Patient Monitoring (RPM) has propelled Cosán to deliver a practice and patient-centric approach to remote care coordination. About Genomind Genomind is a leading precision health company focused on optimizing the treatment experience for individuals and healthcare providers through actionable genetic insights and innovative health technology. Genomind’s platform integrates precision health technology and pharmacogenetic testing on an individual and population level beyond mental health. Built on the foundation of compassionate care and clinical expertise, Genomind is empowering a more targeted and personalized approach to medication management and supporting providers in a new standard of care.

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BUSINESS INSIGHTS

Tris Pharma Announces Development of a Very Low Sodium and Once-Nightly Oxybate Formulation

Tris Pharma, Inc. | July 28, 2022

Tris Pharma, Inc. a fully integrated pharmaceutical company with a robust portfolio of CNS products announced that it has successfully developed an oxybate formulation for the treatment of cataplexy or excessive daytime sleepiness n patients 7 years of age and older with narcolepsy that has the potential to be significantly advantageous to existing approved therapies, such as Jazz Pharmaceuticals' blockbuster XYWAV® and XYREM® franchise. The Tris oxybate formulation is believed to provide an advantage over approved therapies by utilizing Tris's proprietary LiquiXR technology platform and our newly developed RaftWorks design to provide a unique oxybate formulation offering both once-nightly dosing, and very low sodium content. The Tris oxybate formulation is investigational and not yet approved by any regulatory agency. Both XYWAV® and XYREM® require twice a night dosing – once at bedtime and again 2.5 to 4 hours later for the second dose. Further, XYREM contains an express warning based on its high sodium content and associated risk to patients with heart failure, hypertension or impaired renal function. The Tris oxybate formulation seeks to improve on existing therapies by providing convenient once per night dosing, and significantly reduced sodium content such that it will contain less than 100 mg of sodium per 9.0 gm of maximum sodium oxybate equivalent dose. Based on the significant potential advantage the Tris oxybate formulation could provide over existing available therapies, Tris is pursuing an Orphan Drug Designation for its unique oxybate formulation. In a human clinical study, Tris established about 100% bioavailability for one dose of its formulation against two doses of XYREM at 4.5 gm each, equivalent to the 9.0 gm maximum dose of sodium oxybate. If approved, the Tris product will provide significant advantages in that patients will not have to wake up in the middle of the night to take a second dose and will have all the benefits of a very low sodium product. The Tris oxybate product achieved its clinical objectives by combining two distinct proprietary Tris technologies: the LiquiXR platform, and the newly developed RaftWorks design. Working together, these unique formulation platforms provide an interpenetrating network raft in-situ, in which sodium-free drug particles are entrapped in a raft that allows for extended release of the active ingredient. Many drugs, such as oxybate, are poorly absorbed because they have a narrow window of absorption within the human gastro-intestinal tract. The RaftWorks approach permits a slow and extended release of oxybate from a floating raft in-situ, thereby facilitating absorption of the active ingredient within the targeted area in the gastro-intestinal tract. Further, the raft disintegrates at a predetermined time due to the trigger mechanism that is built into the formulation. "This unique formulation design by Tris scientists potentially achieves drug delivery capabilities that have never previously been achieved within our industry. We are looking to build on this approach and develop extended-release formulations for other drugs that are limited by their narrow window of absorption." Ketan Mehta, Founder and Chief Executive Officer of Tris About Narcolepsy Narcolepsy, a chronic and disabling neurological disorder that affects the stability of sleep and wakefulness, is characterized primarily by excessive daytime sleepiness, with or without episodic loss of muscle tone usually triggered by strong emotions during wakefulness (cataplexy) 1-4. Additional symptoms can vary by person and may include disrupted nighttime sleep, and REM-sleep phenomena (sleep paralysis, hallucinations). The exact number of individuals with narcolepsy in the United States is unknown. Several studies have estimated the prevalence of narcolepsy in the United States to be approximately 1 (range: 0.6 to 1.6) in 2,000 people 2,4-6. There is currently no cure for narcolepsy; treatment of symptoms includes medications and lifestyle modifications 1. About Tris Pharma Tris is a fully integrated, innovation-driven CNS company that provides a differentiated approach to target unmet medical needs, including the application of novel technologies designed to enhance patient benefits across therapeutic categories. Tris's CNS portfolio includes treatments for pain; addiction; spasticity in multiple sclerosis, cerebral palsy; narcolepsy; and ADHD.

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