Latest news with #Agios
Business Wire
8 hours ago
- Business
- Business Wire
Avanzanite Announces Pan-European Partnership with Agios to Launch PYRUKYND ® in Rare Blood Disorders
AMSTERDAM--(BUSINESS WIRE)--Avanzanite Bioscience B.V. ('Avanzanite'), a fast-growing commercial-stage specialty pharmaceutical company dedicated to bringing rare disease medicines to patients across Europe, announced today an exclusive agreement with Agios Pharmaceuticals Inc. (Nasdaq: AGIO), a Boston-based biotech company focused on the development and commercialization of rare disease medicines. Under the agreement, Avanzanite will commercialize and distribute PYRUKYND® (mitapivat) across the European Economic Area, the UK and Switzerland. 'This partnership marks a major milestone in our mission to transform how rare disease therapies reach patients in Europe' Share 'This partnership marks a major milestone in our mission to transform how rare disease therapies reach patients in Europe,' said Adam Plich, Founder and CEO of Avanzanite. 'We provide biotech partners with a capital-efficient path to sustainable patient access – without the burden of building a costly local infrastructure. We look forward to working with Agios to help bring PYRUKYND® to patients across Europe, further advancing our efforts to set a new benchmark for biotech commercial and distribution partnerships in the region. This is just the beginning.' PYRUKYND®, a first-in-class, oral, pyruvate kinase (PK) activator, is approved by the European Commission and the UK's Medicines and Healthcare products Regulatory Agency for the treatment of adult patients with PK deficiency. PK deficiency is an ultra-rare, inherited condition that causes premature red blood cell breakdown, leading to chronic anemia, serious complications, and reduced quality of life. Until recently, there were no approved treatments. Agios also has a robust mid- and late-stage pipeline, with clinical programs focused on other rare diseases, including thalassemia and sickle cell disease. The partnership also includes potential future indications. Avanzanite is rapidly scaling its operations, having tripled its revenue in Q1 2025 year-over-year, with two rare disease medicines already on the market. Over the next 12 months, the company will expand into 32 European countries, including new territories of Italy, France, the UK, Romania, and Spain. 'Avanzanite goes truly pan-European,' concluded Plich. 'And as we expand across the continent, our promise stays the same – making sure no patient is left behind.' About Avanzanite Avanzanite is redefining launches of rare disease medicines across Europe. Founded in 2022 and based in Amsterdam, the company partners with biotech innovators to unlock the full commercial value of orphan medicines continent-wide. With our deep expertise in market access, we navigate Europe's complex landscape like master chess players – ensuring no patient is left behind. Learn more at
Yahoo
5 days ago
- Business
- Yahoo
As Interest Grows for New Agents to Treat Thalassemia, Hematologists Begin Early Identification of Ideal Patients for Gene Therapies Vs. New Therapeutic Options
Spherix Global Insights' new patient audit on transfusion-dependent thalassemia reveals strong physician interest in emerging treatments like mitapivat and etavopivat, and provides insight into ideal candidates for curative gene therapies. EXTON, PA, June 04, 2025 (GLOBE NEWSWIRE) -- Despite the promise of new curative strategies for beta thalassemia, such as Casgevy (Vertex) and Zynteglo (bluebird bio), most transfusion-dependent thalassemia patients remain tethered to long-term transfusion support. Transfusions, while providing symptom relief, in turn create logistical barriers and their own clinical challenges. New data from Spherix Global Insights' independent chart audit, Patient Chart Dynamix™: Transfusion Dependent Thalassemia 2025 (US), is based on insight from 81 real-world patient charts submitted by 49 US-based hematologists. These data paint a complex picture of therapeutic inertia, growing anticipation for emerging treatments, and deep-seated frustration with limitations of current treatment options. Most transfusion-dependent beta thalassemia patients follow some regular transfusion schedule. That said, many receive them at relatively extended intervals: often once a month or even less frequently, though a small subset still require more frequent transfusions. This variation in treatment patterns, along with persistent symptoms and unmet needs highlighted in the chart audit, points to a strong opportunity for new therapies to improve care for a broad range of patients. Among surveyed hematologists, mitapivat (Agios) currently holds a slight edge in name recognition over etavopivat (Novo Nordisk). Interestingly, despite this difference in familiarity, physicians identified slightly more patients as potential candidates for etavopivat, hinting at a broader perceived clinical fit once awareness and understanding of the agent catch up. Chart audit data allows users to probe further into specific patient types which physicians perceive as better fits for either agent, as well as those who they do not perceive as candidates. This growing interest in emerging oral therapies reflects a broader desire among hematologists for more accessible, lower-burden treatment options. While most patients are theoretically eligible for curative approaches like gene therapy, the market still favors lower-barrier alternatives. These alternatives include oral therapies that do not require referral to specialized centers or extensive pre-conditioning, and brands with which they are more familiar given other indications such as Reblozyl (Bristol Myers Squibb). Disease-modifying therapies that are easier to prescribe and integrate into routine practice have the promise to significantly improve the lives of patients who would not qualify or want gene therapy. Access barriers compound the issue, with over half of physicians voicing frustration over payer restrictions that make it difficult to initiate optimal care. These challenges highlight a critical need – not just for clinical innovation, but also for strong manufacturer support in navigating reimbursement and expanding access. As the beta thalassemia landscape continues to evolve, hematologists are increasingly looking toward emerging therapies that are easier to prescribe, carry fewer logistical burdens than gene therapy, and offer clear reductions in transfusion dependency. Products like mitapivat and etavopivat are well-positioned to address this gap, assuming familiarity, confidence, and access grow in tandem – but the goal to cure patients of hemoglobinopathies still carries heavy weight, with desire to treat with gene therapy consistently growing. Patient Chart Dynamix™ is an independent, data-driven service unveiling real patient management patterns through rigorous analysis of large-scale patient chart audits. Insights reveal the 'why' behind treatment decisions, include year over year trending to quantify key aspects of market evolution, and integrate specialists' attitudinal & demographic data to highlight differences between stated and actual treatment patterns. 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 to access your portfolio
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
