Latest news with #PNH
Business Wire
16 hours ago
- Health
- Business Wire
Atropos Health Enters into Strategic Collaboration to Reduce Time to Rare Disease Diagnosis with Artificial Intelligence (AI) Algorithm
PALO ALTO, Calif.--(BUSINESS WIRE)--Atropos Health, a leader in translating real-world clinical data into personalized real-world evidence (RWE) and insights, today announced a collaboration with Novartis. Through this strategic collaboration, Atropos Health will build, train and evaluate multiple models to help streamline identification of people with a rare disease who have not yet been diagnosed, ultimately reducing time from symptom reporting to testing, diagnosis and treatment. The collaboration will specifically focus on paroxysmal nocturnal hemoglobinuria (PNH), a rare, serious blood disorder. Adults with PNH often experience lengthy diagnostic delays—many wait over a year, and some more than five—because the disease is rare and presents varied symptoms affecting multiple organs. 1,2 As a part of this collaboration, Atropos Health has developed an initial AI model for finding potential patients with PNH who have not yet been diagnosed, aimed to save critical time for both patients and providers by helping health systems accelerate appropriate diagnosis. The AI model is now available for integration into health systems. 'Building AI models tested and trained on high-quality real-world data is truly the next frontier in precision medicine,' said Dr. Brigham Hyde, CEO and co-founder at Atropos Health. 'The accuracy of the models reduces the guesswork and patients who are able to get testing sooner provides a potentially life-changing experience. For providers and health systems, faster time to diagnosis and treatment equate to higher patient satisfaction.' The goal of the strategic collaboration is to build and publish patient-finding models aimed at reducing time from initial symptoms to testing, diagnosis and treatment. The models created by Atropos Health in connection with this collaboration will be implemented across health system members of the Atropos Evidence Network and be seamlessly integrated at the point of care to improve the provider and patient experience. Through this collaboration, Atropos Health will build models trained on real-world data (RWD) from the Atropos Evidence™ Network, which includes GENEVA OS ®. 'At Novartis, we are committed to delivering meaningful impact for patients. Accelerating diagnosis and treatment through AI and machine learning has the power to significantly improve patient outcomes by enabling faster access to appropriate care,' said Rodney Gillespie, Head of Oncology, Novartis US. 'Our collaboration with Atropos Health to develop an AI model for identifying PNH embodies this commitment as it advances precision health, potentially enabling earlier diagnosis and timely care, reducing delays that can greatly affect patients' lives.' About paroxysmal nocturnal hemoglobinuria (PNH) PNH is a rare, chronic and serious complement-mediated blood disorder 3. People with PNH have an acquired mutation in some of their hematopoietic stem cells (which are located in the bone marrow and can grow and develop into red blood cells [RBCs], white blood cells and platelets) that causes them to produce RBCs that are susceptible to premature destruction by the complement system. 4 It is estimated that approximately 10-20 people per million worldwide live with PNH. Although PNH can develop at any age, it is often diagnosed in people between 30-40 years old. 5,6 Today's announcement follows Atropos Health's AI model training capabilities released earlier this year. Atropos Evidence Network membership benefits include the ability to leverage the network to deliver AI models to clinicians. Atropos Health is collaborating with Arcadia to help leading healthcare organizations deliver the latest care protocols and advanced precision medicine at scale. The partnership enables healthcare providers to accelerate clinical decision-making with actionable, AI-powered insights at the point of care using the combination of real-world evidence (RWE) and longitudinal patient records to improve outcomes and drive high-value, low-cost care. Healthcare organizations are also engaged with Atropos Health on leveraging AI for precision medicine, and building MOTOR, CLMBR and Foundation models on the Atropos Evidence Network. About Atropos Health Atropos Health is the developer of GENEVA OS™, the operating system for rapid healthcare evidence across a robust network of real-world data. Healthcare and life science organizations work with Atropos Health to close evidence gaps from bench to bedside, improving individual patient outcomes with data-driven care, expediting research that advances the field of medicine, and more. We aim to transform healthcare with timely, relevant real-world evidence. To learn more about Atropos Health, visit or connect through LinkedIn or follow on X (Twitter) @AtroposHealth. _________________________ 1 Bektas M, Copley-Merriman C, Khan S, Sarda SP, Shammo JM. Paroxysmal nocturnal hemoglobinuria: patient journey and burden of disease. J Manag Care Spec Pharm. 2020 Dec;26(12-b Suppl):S8-S14. doi: 10.18553/jmcp.2020.26.12-b.s8. PMID: 33356781; PMCID: PMC10408416 2 Mancuso S, Sucato G, Carlisi M, Santoro M, Tarantino G, Iannitto E, Napolitano M, Siragusa S. Paroxysmal nocturnal hemoglobinuria: When delay in diagnosis and long therapy occurs. Hematol Rep. 2018 Mar 29;10(1):7523. doi: 10.4081/hr.2018.7523. PMID: 29721255; PMCID: PMC5907647. 3 Cançado RD, Araújo ADS, Sandes AF, et al. Consensus statement for diagnosis and treatment of paroxysmal nocturnal haemoglobinuria. Hematol Transfus Cell Ther. 2021;43(3):341-348. 4 Dingli D, Matos JE, Lehrhaupt K, et al. The burden of illness in patients with paroxysmal nocturnal hemoglobinuria receiving treatment with the C5-inhibitors eculizumab or ravulizumab: results from a US patient survey. Ann Hematol. 2022;101(2):251-263. 5 Hill A, DeZern AE, Kinoshita T, Brodsky RA. Paroxysmal nocturnal haemoglobinuria. Nat Rev Dis Primers. 2017;3:17028. 6 Röth A, Maciejewski J, Nishimura JI, Jain D, Weitz JI. Screening and diagnostic clinical algorithm for paroxysmal nocturnal hemoglobinuria: Expert consensus. Eur J Haematol. 2018;101(1):3-11. Expand
Herald Malaysia
06-08-2025
- Politics
- Herald Malaysia
Irish missionary among group kidnapped in Haiti
Concern is deepening for the plight of an Irish missionary and eight other people including a child, who have been kidnapped by gunmen in Haiti. Aug 07, 2025 Members of the Haitian National Police (PNH) patrol after an attack by an armed gang in Kenscoff, Haiti (ANSA) By James Blears in Mexico CityGena Heraty, who runs the St Helene Orphanage near Haiti's Capital Port Au Prince, was kidnapped on Sunday along with a three year old child and seven of her staff. The orphanage cares for more than 200 children and is renowned for its kind, loving and dedicated work. Authorities say that the kidnappers broke through an outer wall and then headed straight for the main building in the compound, executing a carefully crafted plan. Gena Heraty has been on mission in Haiti since 1993, helping young and often vulnerable children. Aware for years of the deteriorating situation, the crisis and the risks involved, she has bravely said: ''The children are why I'm still here. I've no intention whatsoever of leaving, because we're all in this together.'' Lawlessness and gang violence plague Haiti, the Western Hemisphere's poorest nation. The United Nations says that more than eighty percent of the Capital Port Au Prince is under the stranglehold control of the street gangs and organized crime. Kidnapping is an everyday, commonplace crime in Haiti, often aimed at gaining large sums of cash for guns and other equipment. Nations, especially those from the Caribbean, have sent police officers to Haiti, to bolster its fragile security forces. But no major nation has yet offered it a troop reinforcement to form a peacekeeping operation. Haiti wants to hold Presidential elections, but this isn't currently possible due to widespread instability. --Vatican News
Yahoo
10-07-2025
- Business
- Yahoo
Apellis Sells Aspaveli Ex-US Royalties to Sobi for Up to $300M
Apellis Pharmaceuticals Inc. (NASDAQ:APLS) is one of the most promising stocks according to Wall Street analysts. On July 1, Apellis Pharmaceuticals announced a capped royalty purchase agreement with Sobi. Under this agreement, Apellis will receive up to $300 million in exchange for 90% of its future ex-US royalties for Aspaveli (systemic pegcetacoplan). This non-dilutive financing includes an upfront payment of $275 million in cash and up to $25 million in milestone payments contingent upon European Medicines Agency/EMA approval of Aspaveli for C3 glomerulopathy (C3G) and primary immune complex membranoproliferative glomerulonephritis (IC-MPGN). Apellis will retain exclusive commercialization rights for systemic pegcetacoplan in the US, where it is marketed as EMPAVELI. A biomedical scientist in a lab coat conducting research on biopharmaceutical compounds. The transaction reinforces the shared confidence between Apellis and Sobi in the growth potential of Aspaveli/EMPAVELI in rare kidney diseases. Aspaveli/EMPAVELI is currently approved in the European Union, other global countries, and the US for the treatment of paroxysmal nocturnal hemoglobinuria/PNH, which is a rare blood disorder. Apellis Pharmaceuticals Inc. (NASDAQ:APLS) is a commercial-stage biopharmaceutical company that discovers, develops, and commercializes novel therapeutic compounds for diseases with unmet needs. While we acknowledge the potential of APLS as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
08-07-2025
- Health
- Yahoo
Market Barriers Continue to Challenge PNH Treatment Optimization Despite Emerging Options, According to Spherix Global Insights
New research highlights significant insurance-driven hurdles, increasing second-line uptake of Fabhalta (Novartis), and strong physician interest in pipeline therapies including zaltenibart (Omeros), pozelimab-cemdisiran (Regeneron/Alnylam), and ruxaprubart (NovelMed). EXTON, PA, July 08, 2025 (GLOBE NEWSWIRE) -- Paroxysmal nocturnal hemoglobinuria (PNH) is a rare, chronic blood disorder that can affect individuals of any age, race, or gender, though it is most commonly diagnosed in adults in their 30s and 40s. In the United States, an estimated 400 to 500 new cases are identified annually. The clinical presentation of PNH varies widely—some patients experience only mild symptoms, while others face life-threatening complications requiring interventions such as immunosuppressive therapy or blood transfusions. ¹ Hematologists are actively adapting their prescribing strategies in paroxysmal nocturnal hemoglobinuria (PNH), with future pipeline entrants poised to further reshape the treatment landscape. According to Spherix Global Insights' Q2 2025 update from RealTime Dynamix™: PNH (US), the availability and adoption of biosimilars are expected to significantly expand the overall treated PNH population—bringing more patients onto complement inhibitor therapy than ever before. The Spherix research projects that biosimilar eculizumab products—Bkemv (Amgen) and Epysqli (Teva/Samsung Bioepis)—will continue to gain traction, capturing share from both branded Soliris and Ultomiris (Alexion/AstraZeneca), as hematologists grow increasingly confident in the clinical utility and cost-effectiveness of biosimilar alternatives. Use of novel entrants in the second line is growing as well, with therapies such as Fabhalta (Novaratis) and Voydeya (AstraZeneca) capturing an increasing share of recent prescribing. These agents are selected for patients who progress or are sub-optimally controlled on C5 inhibitors. Despite these gains, access remains a major commercial hurdle. Many patients with PNH are believed to be suboptimally managed due to persistent insurance-related barriers. Nearly all prescribers report significant challenges navigating the prior authorization process, citing inconsistent payer requirements, delays in drug procurement, and a growing reliance on artificial intelligence by insurers to further restrict coverage. This evolving dynamic has become a major source of frustration for hematologists, often delaying timely access to appropriate therapies and complicating clinical decision-making. The competitive landscape in PNH continues to intensify, with future therapies gaining interest among both academic and community hematologists. The latest Spherix study incorporates target product profiles and prescriber expectations for emerging agents, including zaltenibart (Omeros), a MASP-3 inhibitor that has captured strong physician interest. Most hematologists anticipate zaltenibart will be adopted alongside existing first- and second-line therapies upon approval, driven by high efficacy expectations and its potential to address persistent unmet needs. Other late-stage candidates—such as pozelimab-cemdisiran (Regeneron/Alnylam) and ruxaprubart (NovelMed)—also register meaningful commercial and clinical potential among surveyed specialists. 'These results underscore that physicians aren't just passively watching the market evolve – they're preparing to act,' said Sarah Hendry, Hematology Franchise Head at Spherix. 'With rising expectations for access support and guideline alignment, manufacturers that combine clinical value with commercial enablement will be best positioned for success.' As was seen in Paris at the IPIG international conference when Spherix presented data from their Patient Chart Dynamix™, membership in critical professional societies is growing as hematologists seek the guidance in PNH treatments and care. The most recent release offers a comprehensive look at the U.S. PNH market, including prescribing patterns, brand dynamics, access challenges, and future projections. Delivered quarterly, RealTime Dynamix™ helps commercial teams anticipate shifts, benchmark against competitors, and support strategic planning ahead of key market events. RealTime Dynamix™ is an independent service providing strategic guidance through quarterly or semiannual reports, which include market trending and a fresh infusion of event-driven and variable content with each wave. The reports provide an unbiased view of the competitive landscape within rapidly evolving specialty markets, fueled by robust HCP primary research and our in-house team of experts. Patient Chart Dynamix™ is an independent service that includes robust patient chart audits and integrated specialist surveys fielded biannually. This research provides an in-depth, real-world view of treatment practices by combining verified patient data with attitudinal insights from physicians. The series highlights clinical decision-making, treatment sequencing, and outcomes for targeted patient populations across key therapeutic areas. About Spherix Global Insights Spherix is a leading independent market intelligence and advisory firm that delivers commercial value to the global life sciences industry, across the brand lifecycle. The seasoned team of Spherix experts provides an unbiased and holistic view of the landscape within rapidly evolving specialty markets, including dermatology, gastroenterology, rheumatology, nephrology, neurology, ophthalmology, and hematology. Spherix clients stay ahead of the curve with the perspective of the extensive Spherix Physician Community. As a trusted advisor and industry thought leader, Spherix's unparalleled market insights and advisory services empower clients to make better decisions and unlock opportunities for growth. To learn more about Spherix Global Insights, visit or connect through LinkedIn. For more details on Spherix's primary market research reports and interactive dashboard offerings, visit or register here: NOTICE: All company, brand or product names in this press release are trademarks of their respective holders. The findings and opinions expressed within are based on Spherix Global Insight's analysis and do not imply a relationship with or endorsement of the companies or brands mentioned in this press release. CONTACT: Sarah Hendry, Hematology Franchise Head Spherix Global Insights 4848794284 in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Yahoo
13-06-2025
- Health
- Yahoo
Novartis reports positive results from Phase IIIB study of iptacopan for PNH
Novartis has reported positive outcomes from the multi-centre Phase IIIB APPULSE-PNH study of oral Fabhalta (iptacopan) in adults with paroxysmal nocturnal haemoglobinuria (PNH). These findings are set to be presented at the European Hematology Association (EHA) Congress in 2025. The single-arm, open-label, multinational trial assessed the oral therapy in PNH subjects with haemoglobin (Hb) levels of ≥10g/dL, who transitioned from anti-C5 therapies (eculizumab or ravulizumab). It enrolled 52 subjects and aimed to expand the clinical evidence for the therapy by including patients with higher baseline Hb levels than those in the pivotal Phase III programme. Following 24 weeks of treatment with the therapy, subjects experienced an average increase in Hb levels of 2.01g/dL, with a significant number achieving normal or near-normal levels. During the trial, none of the participants needed blood transfusions, and 92.7% of them reached Hb levels of at least 12g/dL. In addition, subjects reported improvements in fatigue, with Functional Assessment of Chronic Illness Therapy (FACIT)-Fatigue scores aligning with those of the general population by day 168. Novartis noted that Fabhalta-treated subjects also maintained intravascular haemolysis control and resolved extravascular haemolysis control, indicated by lactate dehydrogenase levels remaining below 1.5 times the upper limit of normal and a decrease in absolute reticulocyte count. Along with these findings, the company will also present longer-term data from the APPLY-PNH and APPOINT-PNH Phase III trials at the EHA Congress. Novartis development president and chief medical officer Shreeram Aradhye said: 'New data from APPULSE-PNH, combined with findings from the Phase III roll-over extension of the APPLY-PNH and APPOINT-PNH studies, reinforce the efficacy and safety profile of Fabhalta in delivering real benefits to patients.' Fabhalta was discovered by Novartis and acts as a Factor B inhibitor of the alternative complement pathway. It also offers a new treatment option for the rare and life-threatening complement-mediated blood disorder, PNH. The company recently shared topline outcomes from the Phase III PSMAddition trial's pre-specified interim analysis, in which Pluvicto plus standard of care (SoC) treatment demonstrated a benefit in treating prostate-specific membrane antigen (PSMA)-positive metastatic hormone-sensitive prostate cancer (mHSPC). "Novartis reports positive results from Phase IIIB study of iptacopan for PNH" was originally created and published by Clinical Trials Arena, a GlobalData owned brand. The information on this site has been included in good faith for general informational purposes only. It is not intended to amount to advice on which you should rely, and we give no representation, warranty or guarantee, whether express or implied as to its accuracy or completeness. You must obtain professional or specialist advice before taking, or refraining from, any action on the basis of the content on our site. Sign in to access your portfolio
