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Agios’ PYRUKYND® (mitapivat) Approved for Adults with Thalassemia in Saudi Arabia
GlobeNewswire· 2025-08-04 20:01
Core Viewpoint - Agios Pharmaceuticals has received approval from the Saudi Food and Drug Authority (SFDA) for PYRUKYND (mitapivat) to treat adult patients with both non-transfusion-dependent and transfusion-dependent alpha- or beta-thalassemia, marking a significant milestone in providing new treatment options for thalassemia patients in Saudi Arabia [1][2]. Company Overview - Agios Pharmaceuticals is a commercial-stage biopharmaceutical company focused on innovative medicines for rare diseases, headquartered in Cambridge, Massachusetts [13]. - The company has partnered with NewBridge Pharmaceuticals to facilitate the distribution and commercialization of PYRUKYND in the Gulf Cooperation Council (GCC) region, which includes Saudi Arabia, UAE, Kuwait, Qatar, Oman, and Bahrain [3][5]. Product Information - PYRUKYND is a pyruvate kinase activator indicated for the treatment of hemolytic anemia in adults with pyruvate kinase deficiency and is now approved for thalassemia in Saudi Arabia [5][6]. - The approval is based on results from the global, randomized, double-blind, placebo-controlled ENERGIZE and ENERGIZE-T Phase 3 trials [2][4]. Market Potential - The SFDA's approval is significant as it is the first of its kind for thalassemia treatment in Saudi Arabia, addressing a community with high prevalence and limited treatment options [2][4]. - Regulatory applications for PYRUKYND are currently under review in the U.S., UAE, and European Union, with a PDUFA goal date of September 7, 2025, for the U.S. [4].
Agios Reports Second Quarter 2025 Financial Results and Provides Business Update
Globenewswire· 2025-07-31 10:30
Core Viewpoint - Agios Pharmaceuticals reported its financial results for Q2 2025, highlighting progress in its drug pipeline, particularly with PYRUKYND for thalassemia and ongoing trials for sickle cell disease [1][2]. Financial Performance - The company generated $12.5 million in net revenue for Q2 2025, an increase from $8.6 million in Q2 2024 [6][11]. - The net loss for Q2 2025 was $112.0 million, compared to a net loss of $96.1 million in Q2 2024 [7][17]. - As of June 30, 2025, Agios had $1.3 billion in cash, cash equivalents, and marketable securities, down from $1.5 billion at the end of 2024 [11][14]. Product Development and Pipeline - PYRUKYND's supplemental New Drug Application (sNDA) for thalassemia is under active review by the FDA, with a PDUFA goal date of September 7, 2025 [5][6]. - The topline results from the RISE UP Phase 3 trial for PYRUKYND in sickle cell disease are expected by year-end 2025, with a potential U.S. commercial launch in 2026 [5][6]. - The first patient has been dosed in the Phase 2 trial for tebapivat in sickle cell disease, which is currently enrolling patients [11]. Operational Highlights - There was a 6% increase in unique patients completing prescription enrollment forms compared to Q1 2025, totaling 248 patients [6]. - The number of patients on PYRUKYND therapy increased by 4% over Q1 2025, reaching 142 patients [6]. - Agios entered into a distribution agreement with Avanzanite Bioscience B.V. to commercialize PYRUKYND across the European Economic Area, the UK, and Switzerland [6]. Research and Development Expenses - R&D expenses for Q2 2025 were $91.9 million, up from $77.4 million in Q2 2024, primarily due to a $10 million regulatory milestone payment [11][17]. - Selling, General and Administrative (SG&A) expenses increased to $45.9 million in Q2 2025 from $35.5 million in Q2 2024, attributed to increased commercial activities [11][17]. Strategic Vision - Agios aims to redefine the future of rare disease treatment by developing innovative medicines that can transform patients' lives [9].
Agios Appoints Dr. Jay Backstrom to Board of Directors
GlobeNewswire News Room· 2025-07-08 11:00
Core Insights - Agios Pharmaceuticals has appointed Dr. Jay Backstrom to its Board of Directors, which is expected to enhance the company's strategic direction and clinical program advancements [1][2][3] Company Overview - Agios Pharmaceuticals is a leader in cellular metabolism and pyruvate kinase (PK) activation, focusing on therapies for rare diseases [1][4] - The company markets a first-in-class PK activator for adults with PK deficiency, representing the first disease-modifying therapy for this condition [4] Leadership and Expertise - Dr. Backstrom brings extensive experience from his previous roles, including President and CEO of Scholar Rock and Executive Vice President of Research and Development at Acceleron Pharma [2][3] - His leadership has been instrumental in advancing clinical programs to regulatory approval, particularly in rare diseases [2][3] Strategic Focus - Agios is focused on maximizing the potential of its PK activator, PYRUKYND (mitapivat), and advancing its pipeline of rare disease medicines [2][3] - The company is developing investigational medicines for conditions such as alpha- and beta-thalassemia, sickle cell disease, and myelodysplastic syndromes [4]