Agios Pharmaceuticals (NasdaqGS:AGIO) Q4 2025 Earnings call February 12, 2026 08:00 AM ET Company ParticipantsBrian Goff - CEOCecilia Jones - CFOMark Breidenbach - Managing DirectorMorgan Sanford - Head of Investor RelationsSarah Gheuens - Chief Medical Officer and Head of Research and DevelopmentTsveta Milanova - Chief Commercial OfficerConference Call ParticipantsAlec Stranahan - Biotechnolology Equity Research AnalystEmily Bodnar - Biotechnolology Equity Research AnalystEric Schmidt - Biotechnology Analy ...

Core Viewpoint - The Committee for Medicinal Products for Human Use (CHMP) has issued a positive opinion for the 2mg/kg every-4-weeks dosing regimen of pegunigalsidase alfa for adult patients with Fabry disease, pending review by the European Commission, with a decision expected by March 2026 [1] Group 1: Company Updates - Chiesi Global Rare Diseases and Protalix BioTherapeutics announced the positive CHMP opinion, which aims to reduce the treatment burden for patients and their families by extending the time between infusions [2] - Protalix is eligible for a $25 million regulatory milestone payment from Chiesi if the new dosing regimen is approved by the European Commission [2] Group 2: Product Information - Pegunigalsidase alfa is intended for adult patients with Fabry disease who are stable on enzyme replacement therapy, and the positive opinion follows a re-examination of the dosing regimen application [1][2] - The CHMP's opinion is based on results from the BRIGHT study, which assessed the safety, efficacy, and pharmacokinetics of the alternative dosing regimen over 52 weeks [2] Group 3: Industry Context - Fabry disease is a rare inherited lysosomal storage disorder caused by mutations in the GLA gene, leading to serious health complications, including kidney failure and heart issues [3] - Early detection and access to appropriate treatments are critical for managing symptoms and slowing disease progression in Fabry disease [3]

Company Overview - Zevra Therapeutics is a commercial-stage company focused on impacting individuals with rare diseases [2] - The company is currently in a growth phase, with the operational capacity to pursue its commercial and development goals [2] Product and Revenue - Zevra is commercializing its lead product, MIPLYFFA, in the U.S. for Niemann-Pick disease type C and has an MAA under review by the EMA [3] - The company is expanding access through geographic opportunities and has a pipeline of rare disease programs [3] - In the first nine months of the previous year, Zevra generated net revenues of $72.3 million and ended Q3 with a strong cash position of $230.4 million [3]

Company Overview - Catalyst Pharmaceuticals is a commercial stage biopharmaceutical company focused on rare diseases, aiming to in-license, purchase, and develop products for these conditions [4]. Product Portfolio - The company has two key molecules: FIRDAPSE, which is the only evidence-based product approved for Lambert-Eaton myasthenic syndrome (LEMS), and AGAMREE [5]. Industry Context - The presentation took place at the 44th Annual JPMorgan Healthcare Conference, highlighting the company's engagement with investors and the healthcare community interested in rare diseases [2][3].

Core Viewpoint - Protalix BioTherapeutics is focused on advancing its innovative pipeline for rare diseases, particularly targeting uncontrolled gout and rare kidney diseases, while maintaining a profitable commercial business through strategic partnerships [2][14]. Pipeline and Product Development - The company is advancing PRX-115 for uncontrolled gout, which has shown a rapid and durable urate-lowering effect in Phase 1 trials, indicating its potential as a differentiated treatment option [3][15]. - PRX-119 is being developed as a long-acting DNase I for treating inflammation and fibrosis in rare kidney diseases, reflecting the company's strategic focus on this area [4][13]. - The company has submitted an Investigational New Drug (IND) application for PRX-115, which has been approved by the FDA, and the first clinical sites are now activated [15]. Commercial Execution and Market Positioning - Protalix has achieved solid performance with its partner Chiesi Global Rare Diseases, particularly with the launch of Elfabrio for Fabry disease, tracking well against core indicators such as treated patient counts and market share [6][7]. - The global Fabry market is projected to reach approximately $3.4 billion by 2030, with Protalix expecting Elfabrio to capture 15% to 20% of this market [7]. - An appeal regarding a negative opinion from the European Medicines Agency on Elfabrio's dosing regimen is underway, with an outcome expected in Q1 2026 [8]. Strategic Partnerships and Collaborations - The company continues to benefit from established revenue streams, including Elelyso, through collaborations with Pfizer and Fundação Oswaldo Cruz, which support its operational resilience [9]. - A recent collaboration with Secarna Pharmaceuticals aims to develop novel therapeutic candidates for rare renal indications using AI technology [15]. Long-Term Growth and Value Creation - Protalix enters 2026 with a profitable commercial foundation and a focused pipeline addressing high unmet medical needs, aiming to create long-term value for patients and shareholders [14]. - The company emphasizes its commitment to delivering meaningful therapies while pursuing strategic partnerships to enhance its impact and scale [5][14].

Core Insights - Pharming Group N.V. reported a strong financial performance for Q3 2025, with total revenues increasing by 30% to US$97.3 million compared to Q3 2024, driven by growth in both RUCONEST® and Joenja® [9][30][31] Financial Performance - Total revenues for Q3 2025 reached US$97.3 million, a 30% increase from US$74.8 million in Q3 2024 [30] - RUCONEST® revenue increased by 29% to US$82.2 million, reflecting sustained growth in patients and prescribers [9][10] - Joenja® revenue grew by 35% to US$15.1 million, driven by a significant increase in patients on paid therapy [13][31] - Operating profit surged by 285% to US$15.8 million compared to US$4.1 million in Q3 2024 [32] - The company generated US$32 million in cash flow from operations during the quarter [34] Strategic Developments - Pharming announced a significant reduction in general and administrative headcount to optimize capital deployment towards high-growth initiatives [5][23] - The company is focusing on expanding its pipeline, particularly for leniolisib in broader primary immunodeficiency populations and addressing unmet needs in primary mitochondrial disease with KL1333 [4][5] Market Position and Outlook - The company raised its full-year revenue guidance to between US$365 million and US$375 million, up from the previous range of US$335 million to US$350 million [6][44] - The U.S. market contributed 89% of Q3 revenue, while the EU and Rest of World contributed 11% [14] - Pharming plans to launch a pediatric formulation of leniolisib for children aged 4 to 11 years, with FDA priority review expected by January 2026 [20][44] Organizational Changes - Leverne Marsh has been appointed as Chief Commercial Officer, effective January 1, 2026, succeeding Stephen Toor [7][24] - Kenneth Lynard was appointed as Chief Financial Officer, effective October 1, 2025, enhancing the company's financial leadership [22] Corporate Highlights - Pharming was promoted from the Euronext AScX® to the AMX® index, reflecting its growing market capitalization and trading activity [25] - The company is working on options to mitigate the impact of recently announced U.S. tariffs, although it does not expect a material impact on its business [26]

Drug Development - Avidity is developing experimental drugs [1] - The drugs target rare diseases, including myotonic dystrophy type 1 [1]

Core Insights - Novo Nordisk has entered into a definitive asset purchase and license agreement with Omeros Corporation for the drug zaltenibart, which is in clinical development for rare blood and kidney disorders [1][2] Financial Terms - Under the agreement, Omeros is eligible to receive $340 million in upfront and near-term milestone payments, with a total potential of $2.1 billion including development and commercial milestones, plus tiered royalties on net sales [2][9] Drug Mechanism and Development - Zaltenibart is an antibody targeting MASP-3, a key activator of the complement system's alternative pathway, which is implicated in various rare diseases [3][10] - Positive phase 2 data has been reported for zaltenibart in paroxysmal nocturnal hemoglobinuria (PNH), showing multiple advantages over other alternative pathway inhibitors [4][11] - Novo Nordisk plans to initiate a global phase 3 program for zaltenibart in PNH and explore its development in other rare blood and kidney disorders [6][7] Strategic Positioning - The acquisition of zaltenibart enhances Novo Nordisk's Rare Disease portfolio and supports its ambition to lead in this therapeutic area [7][9] - Omeros retains rights to its preclinical MASP-3 programs unrelated to zaltenibart, allowing for continued development of small-molecule MASP-3 inhibitors [5]

Core Insights - Novo Nordisk has entered into a definitive asset purchase and license agreement with Omeros Corporation for the drug zaltenibart, which is in clinical development for rare blood and kidney disorders [1][2] Financial Terms - Under the agreement, Omeros is eligible to receive $340 million in upfront and near-term milestone payments, with a total potential of $2.1 billion including development and commercial milestones, plus tiered royalties on net sales [2][9] Drug Mechanism and Development - Zaltenibart is an antibody targeting MASP-3, a key activator of the complement system's alternative pathway, which is implicated in various rare diseases [3][10] - Positive Phase 2 data has been reported for zaltenibart in paroxysmal nocturnal hemoglobinuria (PNH), showing multiple advantages over other alternative pathway inhibitors [4][11] Strategic Positioning - Novo Nordisk aims to leverage its expertise to maximize the value of zaltenibart and develop it into a best-in-class treatment for rare blood and kidney disorders [4][7] - The transaction is expected to close in the fourth quarter of 2025, subject to customary closing conditions and regulatory approvals [7] Broader Implications - Zaltenibart has potential applications across various rare blood and kidney disorders, enhancing Novo Nordisk's Rare Disease portfolio and supporting its growth ambitions in this area [11][12]

Company Overview - Quince Therapeutics focuses on developing treatments for rare diseases [2] Leadership - The event features key executives from Quince Therapeutics, including CEO Dirk Thye and COO Brendan Hannah [1]