Core Viewpoint - Pharming Group N.V. is under investigation for potential securities fraud following the FDA's Complete Response Letter regarding its drug Joenja® [1][2] Group 1: Company Overview - Pharming Group N.V. is a biopharmaceutical company focused on developing innovative therapies for rare diseases [2] - The company’s stock, represented by American Depositary Receipts (ADRs), experienced a significant decline of 17.07% following negative FDA news [2] Group 2: FDA Response - On February 1, 2026, the FDA issued a Complete Response Letter for Pharming's supplemental New Drug Application for Joenja® [2] - The FDA raised concerns about potential underexposure in lower weight pediatric patients and requested additional pharmacokinetic data [2] - An issue was also identified with one of the analytical methods used for production batch testing, necessitating further data and clarification [2] Group 3: Legal Investigation - Pomerantz LLP is investigating claims on behalf of Pharming investors regarding possible securities fraud or unlawful business practices by the company and its officers [1] - The firm is recognized for its expertise in corporate, securities, and antitrust class litigation, having a long history of fighting for victims of securities fraud [3]

2026 guidance above Street: Pharming forecasted revenue of $405–$425 million for 2026 (8–13% growth vs. 2025), beating an analyst consensus of about $398 million, with an implied ~90% gross margin and operating expenses of $300–$335 million.Commercial momentum but regulatory caution: RUCONEST remains a durable U.S.-driven franchise ($318M in 2025, +26%), while Joenja is accelerating ($58M in 2025, +29%) but faces FDA questions on pediatric APDS dosing — Pharming will request a Type A meeting in March and h ...

I have a strong inclination towards high-growth companies, often treading in sectors poised for exponential expansion. My expertise lies in understanding and investing in disruptive technologies and forward-thinking enterprises. My approach is a mix of fundamental analysis and future trend prediction. I believe in the power of innovation to yield substantial returns and aim to provide insightful analysis on such companies here on SeekingAlpha.Analyst’s Disclosure: I/we have no stock, option or similar deriv ...

Core Viewpoint - The focus is on high-growth companies in sectors expected to experience exponential expansion, emphasizing the importance of innovation and disruptive technologies for substantial returns [1]. Group 1: Investment Strategy - The investment approach combines fundamental analysis with predictions of future trends, aiming to identify and invest in forward-thinking enterprises [1]. Group 2: Market Perspective - There is a strong belief in the potential of innovation to drive significant financial returns, highlighting the importance of staying ahead in rapidly evolving industries [1].

A top executive at German utility Uniper has played down European concerns over increasing dependence on liquefied natural gas (LNG) from the United States, saying companies sign contracts with firms ... ...

Core Insights - Pharming is presenting its financial guidance for the year and highlighting its clinical stage pipeline programs during the 2026 Investor Day [1][2] - The company has evolved from a single asset company to a fast-growing biotech with two commercial assets experiencing double-digit growth and a late-stage pipeline with two programs each having over $1 billion sales potential [3] Financial Update - A short business and financial update will be provided, focusing on the financial guidance for the year [2] - The company emphasizes its growth trajectory and the potential of its pipeline programs [3] Pipeline Overview - The presentation will include two longer sessions dedicated to the pipeline, specifically on leniolisib for high prevalence primary immunodeficiencies (PIDs) and KL1333, napazimone [2]

Pharming Group (NasdaqGM:PHAR) 2026 Investor Day February 03, 2026 10:00 AM ET Company ParticipantsAmel Karaa - Director of Mitochondrial Disease ProgramAngela Qian - VPAnurag Relan - Chief Medical OfficerFabrice Chouraqui - CEO and Executive DirectorJocelyn Farmer - Director of Clinical Immunodeficiency ProgramJoe Pantginis - Managing Director of Equity ResearchKenneth Lynard - CFOMagnus Hansson - Executive Medical Director and Global Program LeadMichael Levitan - VP Investor Relations and Corporate Commun ...

Core Insights - Pharming Group N.V. has announced its financial guidance for 2026, projecting total revenues between US$405 million and US$425 million, representing an 8% to 13% growth [3][7][8] - The company will highlight its advancing clinical-stage pipeline, focusing on two major programs targeting primary immunodeficiencies and mitochondrial diseases during its Investor Day [2][4][6] Financial Guidance - Total revenue guidance for 2026 is set between US$405 million and US$425 million, driven by growth in its commercial products [3][8] - Total operating expenses are expected to be between US$330 million and US$335 million, primarily due to increased R&D expenses related to ongoing clinical trials [7][8] Pipeline Overview - Leniolisib is being developed for primary immunodeficiencies with immune dysregulation, with ongoing Phase II trials expected to yield top-line data in the second half of 2026 [4][5] - Napazimone (KL1333) is being developed for mtDNA-driven mitochondrial disease, with a pivotal clinical trial ongoing and results anticipated in 2027 [6][9] Clinical Programs - Leniolisib is currently approved as Joenja in the U.S. and is the first targeted treatment for activated PI3Kδ syndrome, with potential applicability across broader patient populations [5][6] - Napazimone (KL1333) aims to become the first standard of care for mtDNA-driven mitochondrial disease, addressing significant unmet medical needs [6][9] Investor Day Details - The Investor Day will feature presentations from clinical experts discussing disease biology and the potential impact of Pharming's programs [10][16] - The event is scheduled for February 3, 2026, from 10:00 a.m. to 12:00 p.m. EST, and will be available via webcast [11]

Core Viewpoint - Pharming Group announced that the FDA issued a Complete Response Letter (CRL) for its supplemental New Drug Application (sNDA) for Joenja® (leniolisib) as a treatment for children aged 4 to 11 years with activated phosphoinositide 3-kinase delta syndrome (APDS) [1][5] Regulatory Update - The FDA raised concerns about potential underexposure in lower weight pediatric patients and requested additional pharmacokinetic data to reassess proposed doses [2] - An issue was identified with one of the analytical methods used for production batch testing, prompting the FDA to request further data and clarification [2] - Pharming plans to address the issues outlined in the CRL and intends to request a Type A meeting with the FDA to discuss next steps for resubmission [3] Clinical Data - The sNDA submission was based on positive results from a Phase III study showing improvements in lymphadenopathy and naïve B cells over 12 weeks, indicating a correction of the immune defect in children [4] - Safety data from 8 months of treatment indicated that all treatment-emergent adverse events were mild to moderate, with no serious drug-related adverse events reported [4] Market Context - Currently, there are no approved treatments for children with APDS under the age of 12 globally, highlighting the significance of Joenja's potential impact [5] - Joenja received FDA approval for treating APDS in patients aged 12 years and older in March 2023, which remains unaffected by the recent regulatory action [3][5] Disease Overview - APDS is a rare primary immunodeficiency characterized by severe recurrent infections and immune dysregulation, affecting approximately 1 to 2 people per million worldwide [6] - The condition is caused by variants in the PIK3CD or PIK3R1 genes, leading to hyperactivity of the PI3Kδ pathway, which impairs immune cell function [6] Product Information - Leniolisib is an oral PI3Kδ inhibitor approved in several countries as the first targeted treatment for APDS in patients aged 12 years and older [7] - The drug is currently under regulatory review in multiple regions and is being evaluated in clinical trials for other primary immunodeficiencies [8]

Core Viewpoint - Pharming Group N.V. reported preliminary, unaudited revenues for 2025, estimating approximately US$376 million, which exceeds previous guidance and indicates a 27% growth compared to 2024 [2][5] Financial Performance - Total revenues for 2025 are estimated at US$376 million, surpassing the revised guidance of US$365 to US$375 million [2] - This revenue growth is attributed to the continued success of RUCONEST® and increased demand for Joenja®, particularly in the U.S. market [2] - Full-year operating expenses are expected to be between US$304 million and US$308 million, reflecting the company's commitment to cost discipline [2] Future Outlook - The company anticipates sustained revenue growth and further advancements in its clinical pipeline for 2026 [2] - A complete financial report for Q4 and full year 2025 is scheduled for release on March 12, 2026 [2] Investor Day - Pharming will host a virtual Investor Day on February 3, 2026, to provide updates on its clinical pipeline and financial guidance for 2026 [1][3] - The agenda will include discussions on leniolisib and KL1333, focusing on their clinical trials and the unmet needs in related diseases [3][4] - Presentations will feature leading clinical experts discussing immune dysregulation and mitochondrial diseases, providing context for Pharming's development programs [4]