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New CHORUS data published in Blood show HHT is a serious multisystem bleeding disorder requiring earlier diagnosis, expert care, and research. This publication gives the HHT community stronger national evidence for what patients and families have experienced all along.” — Marianne Clancy, MPA MONKTON, MD, UNITED STATES, March 25, 2026 / EINPresswire.com / -- Cure HHT proudly announces the publication of “ Clinical Spectrum of Hereditary Hemorrhagic Telangiectasia : Data from the Comprehensive HHT Outcomes Registry of the US (CHORUS)” in Blood, the flagship journal of the American Society of Hematology (ASH). The study reports early findings from CHORUS, the first national U.S. registry designed to capture the real-world clinical impact of hereditary hemorrhagic telangiectasia (HHT). HHT is a genetic blood vessel disorder that causes abnormal connections between arteries and veins, known as arteriovenous malformations (AVMs), in organs including the lungs, brain, liver, and gastrointestinal tract. It is the second most common inherited bleeding disorder in the U.S. and worldwide, resulting in frequent and severe nosebleeds, chronic gastrointestinal bleeding, iron deficiency anemia, and other serious complications. Because HHT often goes unrecognized for years, delayed diagnosis can lead to preventable, life-threatening outcomes, including brain and lung hemorrhage. This publication marks a major milestone for the HHT community. By documenting the clinical spectrum of HHT using data from patients receiving care across U.S. HHT Centers of Excellence, the CHORUS study helps strengthen medical understanding of the disease, elevates awareness among clinicians, and reinforces the urgent need for earlier diagnosis, expert care, and continued research investment. “This publication gives the HHT community something incredibly important: stronger national evidence for what patients and families have been experiencing all along,” said Marianne Clancy, MPA, Chief Executive Officer of Cure HHT. “It shows that HHT is a serious, multisystem inherited bleeding disorder that demands earlier recognition, expert care, and continued investment in research. We hope this publication helps drive greater awareness, better care, and better outcomes for every family affected by HHT.” Key Takeaways from the Publication • Patients with HHT experience a substantial burden from chronic bleeding, iron deficiency, anemia, and organ involvement. • Delayed or missed diagnosis can leave serious complications undetected and untreated. • Researchers found that intracranial hemorrhage occurred in 3% of those studied, with most first brain bleeds happening by age 25. This rate is very high among bleeding disorders. • Genetic testing was performed in only 60% of patients in the study’s cohort, highlighting ongoing gaps in diagnosis and management. • 76% of patients with HHT develop moderate-to-severe nose bleeding and/or gastrointestinal bleeding requiring treatment. • One-sixth of patients develop serious abnormal blood clots like strokes or pulmonary emboli. • The incidence of potentially life-threatening brain bleeding is similar to that of hemophilia in the 1990s, before those patients received long-term treatment to prevent bleeding. “CHORUS is helping us better understand the true burden of HHT,” said Hanny Al-Samkari, MD, the study’s first author who is a classical hematologist, The Peggy S. Blitz Endowed Chair of Hematology/Oncology, and Co-Director of the HHT Center of Excellence at Massachusetts General Hospital and an Associate Professor of Medicine at Harvard Medical School. “These findings underscore in dramatic fashion the serious unmet need in HHT, and therefore the urgency of developing new medications to treat this disease that currently has no FDA-approved therapies.” CHORUS was launched through federal funding secured after years of advocacy by Cure HHT and the broader HHT community. The registry was created to generate a stronger evidence base for HHT, improve care delivery, and accelerate progress toward better treatments. This publication represents an important step forward in that work and a powerful example of what is possible when patient advocacy, expert clinical care, and research infrastructure come together. Valaree Machen Cure HHT +1 410-357-9932 ext. 116 valaree.machen@curehht.org Visit us on social media: LinkedIn Instagram Facebook YouTube Other Legal Disclaimer: EIN Presswire provides this news content "as is" without warranty of any kind. We do not accept any responsibility or liability for the accuracy, content, images, videos, licenses, completeness, legality, or reliability of the information contained in this article. If you have any complaints or copyright issues related to this article, kindly contact the author above.

Atavistik Bio, a Cambridge, Massachusetts-based biotechnology company focused on developing selective allosteric therapeutics, has closed a USD 40 million extension to its Series B financing, bringing total proceeds from the round to USD 160 million. The company is advancing a pipeline of mutant-selective small molecule inhibitors targeting hereditary hemorrhagic telangiectasia and myeloproliferative neoplasms, the company said. The extension was funded by new investor RA Capital Management, which joins existing Series B participants Nextech Invest, The Column Group, Lux Capital, and Regeneron Ventures. The initial Series B close was announced in December 2025. Atavistik Bio said it plans to use the combined proceeds to fund clinical development of its lead candidate, ATV-1601, an oral allosteric AKT1-selective inhibitor for hereditary hemorrhagic telangiectasia, and to advance its JAK2 V617F mutant-selective inhibitor program for myeloproliferative neoplasms through clinical proof of concept. ATV-1601 has completed a Phase I study in oncology, where the company said it demonstrated a safety profile consistent with its AKT1-selective mechanism, avoiding the hyperglycemia associated with pan-AKT inhibitors driven by AKT2 inhibition. The company is now redirecting development toward HHT, a severe inherited bleeding disorder affecting more than 1.6 million people globally with no currently approved therapies. AKT1 hyperactivation has been identified as a driver of the vascular pathology underlying HHT. The company’s preclinical-stage second program targets the JAK2 V617F mutation, the most common driver mutation in myeloproliferative neoplasms, found in approximately 95% of polycythemia vera patients, 60% of essential thrombocythemia patients, and 55% of myelofibrosis patients. Approved pan-JAK inhibitors such as ruxolitinib provide symptom relief but inhibit both mutant and wild-type JAK2, limiting disease modification and contributing to adverse events. Atavistik Bio’s platform centers on designing small molecules that exploit structural differences between mutant and wild-type proteins, targeting allosteric sites rather than conserved active sites to achieve selectivity. This approach aims to widen the therapeutic window by enabling higher effective doses against disease-driving targets without the toxicities associated with wild-type inhibition. The company was co-founded by Philip Cole, a professor at Harvard Medical School whose academic work on allosteric kinase regulation and isoform-selective inhibition informs the platform’s scientific foundation. Specific intellectual property licensing arrangements between the company and academic institutions have not been publicly disclosed. Your email address will not be published. Required fields are marked * Comment * Name * Email * Website