Latest news with #Mitapivat
Web Release
04-07-2025
- Health
- Web Release
A Landmark Global Trial Led from Lebanon: Mitapivat in NTDT Published in The Lancet
In a major advancement for global thalassemia care, the results of a landmark global phase 3 clinical trial of mitapivat in non-transfusion-dependent thalassemia (NTDT) led by the American University of Beirut (AUB) and the Chronic Care Center, the thalassemia center in Lebanon, have been published in The Lancet, one of the world's leading medical journals. The study, titled 'Phase 3 trial of mitapivat in non-transfusion-dependent alpha- or beta-thalassemia,' led by Dr. Ali Taher, professor of medicine at the Division of Hematology and Oncology, director of the Naef K. Basile Cancer Institute, and consultant at the Chronic Care Center served as both global principal investigator and first and corresponding author. It is the first to demonstrate the efficacy of an oral, disease-modifying therapy for both beta and alpha forms of NTDT, positioning mitapivat as a potential game changer in the treatment of this lifelong blood disorder. 'This publication is more than a scientific milestone,' said Dr. Taher. 'It's proof that Lebanon, even in its hardest times, can lead groundbreaking medical advances that change global standards of care. I'm proud this trial was conceived, led, and delivered from AUB and the Chronic Care Center.' NTDT is a lifelong inherited blood disorder that results in chronic anemia. Patients with NTDT do not require blood transfusions, but still suffer from chronic anemia and associated complications, including iron overload and reduced quality of life. Until now, therapeutic options for these patients have been limited. Mitapivat is currently under review by the United States Food and Drug Administration (FDA) for potential marketing authorization. If approved, it would become the first FDA-authorized oral disease-modifying treatment for NTDT, offering a transformative option for thousands of patients worldwide. The phase 3 trial met all of its primary and secondary endpoints, demonstrating statistically and clinically significant improvements in hemoglobin levels, alongside favorable safety and tolerability profiles. This work represents the culmination of over a decade of research, beginning with redefining the disease and understanding the morbidity and mortality risks of untreated anemia and iron overload. It builds on prior global efforts led from AUB that resulted in the approval of key therapies, including iron chelation through the THALASSA trial and luspatercept through the BEYOND trial. Both studies were led by Dr. Ali Taher, whose vision and leadership have been instrumental in transforming the global approach to thalassemia care. Widely recognized as one of the foremost experts in the field, Dr. Taher has played a central role in advancing treatment standards and improving outcomes for patients worldwide. His long-standing commitment to thalassemia research has not only shaped the current therapeutic landscape, but has also positioned Lebanon and AUB at the forefront of international medical innovation. This achievement serves as a powerful affirmation of AUB's role as a beacon of academic and clinical excellence, firmly committed to advancing health through research, education, and patient-centered innovation. It also reflects the resilience and determination of Lebanon's medical and scientific communities to contribute meaningfully to global health, despite the formidable challenges faced at home.
Yahoo
14-02-2025
- Business
- Yahoo
Agios Pharmaceuticals Inc (AGIO) Q4 2024 Earnings Call Highlights: Robust Revenue Growth and ...
Net Pyrukynd Revenue (Q4 2024): $10.7 million, a 51% increase from $7.1 million in Q4 2023. Cost of Sales (Q4 2024): $1.3 million. R&D Expenses (Q4 2024): $82.8 million, an increase of $5.3 million from Q4 2023. SG&A Expenses (Q4 2024): $51.7 million, an increase of $16.4 million from Q4 2023. Cash Equivalents and Marketable Securities (End of Q4 2024): Approximately $1.5 billion. Milestone Payments (2024): Total of $1.1 billion, including $905 million from Royalty Pharma and $200 million from Servier. Warning! GuruFocus has detected 4 Warning Signs with AGIO. Release Date: February 13, 2025 For the complete transcript of the earnings call, please refer to the full earnings call transcript. Agios Pharmaceuticals Inc (NASDAQ:AGIO) reported a 51% increase in net Pyrukynd revenue for Q4 2024 compared to the same quarter in 2023. The company is preparing for two additional commercial launches, with potential approval and launch of Pyrukynd in thalassemia expected in September 2025 and in sickle cell disease in 2026. Agios has a robust early and mid-stage pipeline poised for clinical advancement, including the completion of enrollment in the phase 3 rise up study for sickle cell disease. The company has a strong balance sheet with approximately $1.5 billion in cash, equivalents, and marketable securities, providing financial independence for future growth. Agios announced positive top-line results from the Activate Kids Phase 3 trial of Mitapivat in pediatric patients with PK deficiency, marking its first pediatric clinical program for a rare hemolytic anemia. Agios Pharmaceuticals Inc (NASDAQ:AGIO) expects flat revenues for PK deficiency in 2025 compared to 2024, indicating limited growth in this area. The company noted that Q4 2024 revenues were higher due to year-end stocking and adjustments, which are not expected to repeat in Q1 2025. There are concerns about liver toxicity in the sickle cell trial, leading to changes in the monitoring protocol. The launch in the Gulf region, particularly Saudi Arabia, is expected to take time due to the need for formulary access and healthcare system navigation. Agios faces challenges in pediatric development, which is complex and requires significant logistical considerations. Q: What is Agios Pharmaceuticals' strategy for updating the investment community on the safety profile of Mitapivat, especially concerning hepatocellular injury? A: Sarah Gheuens, Chief Medical Officer, stated that if there are any changes to the safety profile of Mitapivat, the company will update the investment community as they have done in the past when new safety information became available. Q: How does Agios Pharmaceuticals view the peak sales potential for Mitapivat in thalassemia and sickle cell disease? A: Brian Goff, CEO, expressed confidence in the multi-billion-dollar potential of Pyrukynd, citing the significant unmet need in thalassemia, where two-thirds of the U.S. patient population has no approved therapy, and the high unmet need in sickle cell disease, which has increased due to limitations in available therapeutic options. Q: What changes were made to the sickle cell trial protocol following the liver toxicity disclosure? A: Sarah Gheuens explained that monitoring for liver enzymes was already part of the core period of trials. The open-label extension (OLE) portion was adjusted to align with the core period's monthly monitoring for the first six months of exposure. Q: How does Agios Pharmaceuticals plan to approach the development of Tepapivat in sickle cell disease? A: Brian Goff and Tsveta Milanova, Chief Commercial Officer, indicated that they are building a sickle cell disease franchise with both Pyrukynd and Tepapivat. The development path will be guided by data from ongoing studies, and they aim to provide multiple treatment options for this complex disease. Q: How should investors model the launch trajectory for Mitapivat in thalassemia, considering potential pent-up demand? A: Tsveta Milanova noted that while there is a focus on educating and increasing urgency among physicians, they do not expect an initial bolus of patients. The launch will target about 65% of the U.S. thalassemia patient population, capturing patients as they visit their doctors. For the complete transcript of the earnings call, please refer to the full earnings call transcript. This article first appeared on GuruFocus. Sign in to access your portfolio
