Pharming N.V.(PHAR)

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Pharming N.V.(PHAR) - 2024 Q4 - Annual Report
2025-04-03 10:04
UNITED STATES SECURITIES AND EXCHANGE COMMISSION Washington, D.C. 20549 FORM 20-F (Mark One) ☐ REGISTRATION STATEMENT PURSUANT TO SECTION 12(b) OR (g) OF THE SECURITIES EXCHANGE ACT OF 1934 OR ☒ ANNUAL REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the fiscal year ended December 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to OR ☐ SHELL COMPANY REPORT PURSUANT TO SECTION 13 OR 15(d) OF ...
Pharming Group announces first patient dosed in Phase II clinical trial of leniolisib for common variable immunodeficiency (CVID) with immune dysregulation
Newsfilter· 2025-03-20 06:00
Core Insights - Pharming Group N.V. has initiated a Phase II clinical trial for leniolisib targeting common variable immunodeficiency (CVID) patients with immune dysregulation, marking a significant step in addressing unmet medical needs in this patient population [1][4]. Group 1: Clinical Trial Details - The Phase II trial is a single-arm, open-label study involving approximately 20 patients aged 12 and older, focusing on safety, tolerability, pharmacokinetics, pharmacodynamics, and clinical efficacy of leniolisib in CVID patients [2]. - The trial specifically targets CVID patients exhibiting lymphoproliferation and at least one additional clinical manifestation of immune dysregulation, such as interstitial lung disease or autoimmune cytopenias [2][3]. - The lead investigator is Dr. Jocelyn Farmer, with clinical sites located in the US, UK, and EU [2]. Group 2: Disease Context and Unmet Needs - CVID is the largest group of symptomatic primary immunodeficiency patients, with about 50% experiencing autoimmune and lymphoproliferative complications due to immune dysregulation [3]. - Patients with CVID and immune dysregulation have an 11-fold increased mortality rate compared to those with infectious manifestations alone, highlighting the critical need for effective therapies [3]. - The global prevalence of CVID with immune dysregulation is estimated at approximately 39 patients per million [3]. Group 3: Leniolisib Overview - Leniolisib is an oral small molecule phosphoinositide 3-kinase delta (PI3Kδ) inhibitor, currently approved in the U.S. for treating activated phosphoinositide 3-kinase delta syndrome (APDS) [5][6]. - The drug has shown statistically significant improvement in clinical trials for APDS, and its safety and tolerability for long-term use have been supported by interim data [6]. - Leniolisib is under regulatory review in multiple regions, including the European Economic Area, Canada, and Australia, with plans for further approvals in Japan and South Korea [6]. Group 4: Company Background - Pharming Group N.V. is a global biopharmaceutical company focused on developing therapies for rare and life-threatening diseases, with a portfolio that includes protein replacement therapies and precision medicines [7]. - The company is headquartered in Leiden, the Netherlands, and operates in over 30 markets worldwide [7].
Pharming N.V.(PHAR) - 2024 Q4 - Earnings Call Transcript
2025-03-13 23:44
Financial Data and Key Metrics Changes - Full-year 2024 revenues increased by 21% to $297 million, exceeding guidance, with strong performance in Q4 [7][42] - Operating profit increased by $5.6 million, marking the second consecutive quarter of positive operating profit [43] - Net results shifted from a loss in Q4 2023 to a net profit in Q4 2024, with positive operating cash flow for the second quarter in a row [44] Business Line Data and Key Metrics Changes - RUCONEST revenue grew 11% to $252 million for the year, with a 9% increase in Q4, driven by new patient enrollments and prescriber base expansion [7][11] - Joenja revenue surged by 147% to $45 million in 2024, with a 65% increase in Q4 [9][12] Market Data and Key Metrics Changes - The prescriber base for RUCONEST increased by 11% and new patient enrollments rose by 24% [11] - Joenja's patient pipeline is expanding, with over 240 identified patients in the US, 40% of whom are on paid therapy [20] Company Strategy and Development Direction - The company aims to develop a leading global rare disease portfolio, leveraging a proven clinical development and commercial infrastructure [7] - The acquisition of Abliva is seen as a strategic move to enhance the company's pipeline and revenue potential [6][46] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in RUCONEST's continued growth despite increasing competition, citing its unique profile and strong patient experience [50][79] - The company anticipates total revenues for 2025 to be between $315 million and $335 million, reflecting continued growth in RUCONEST and Joenja [47] Other Important Information - The company is actively monitoring potential US tariffs on drugs and is taking steps to mitigate any impact on its supply chain [70] - The anticipated operating expenses related to the Abliva acquisition are estimated at $30 million for 2025, including $17 million for R&D [49][69] Q&A Session Summary Question: Insights on KL1333 and patient population - The addressable population for KL1333 is estimated at 30,000 patients in the US and Europe, focusing on those with specific mitochondrial DNA mutations [59] Question: Breakdown of patients in the expanded access program - Patients in the expanded access program are in multiple countries, with some on paid therapy through name patient programs, but specific numbers are not disclosed [63] Question: Recurring costs from Abliva acquisition - Of the $30 million anticipated OpEx from the Abliva acquisition, approximately $17 million will be for R&D, with the remainder being non-recurring costs [69] Question: Impact of potential US tariffs - The company is monitoring the situation regarding US tariffs and is exploring ways to minimize potential impacts on its supply chain [70] Question: RUCONEST's growth despite competition - Management believes RUCONEST will remain a treatment of choice due to its unique profile and strong patient experience, supported by market research [79][81] Question: Timeline for CVID trials - A Phase 2 study is anticipated for CVID, with a Phase 3 study likely required for registration [102] Question: Future expectations for CVID and fast-track designation - The company plans to explore fast-track designation options for CVID, given the severity of the disease [105]
Pharming Group reports fourth quarter and full year 2024 financial results and provides business update
Newsfilter· 2025-03-13 06:00
Financial Performance - Total revenues for 2024 increased by 21% to US$297.2 million, exceeding guidance of US$280-$295 million, driven by record RUCONEST® revenue and strong Joenja® growth [3][8][52] - RUCONEST® revenue for the full year 2024 was US$252.2 million, an 11% increase compared to 2023, while Joenja® revenue reached US$45.0 million, a 147% increase in its first full year of sales [8][52] - Fourth quarter 2024 total revenues increased by 14% to US$92.7 million compared to the same quarter in 2023, with RUCONEST® revenue at US$79.6 million (9% increase) and Joenja® revenue at US$13.1 million (66% increase) [8][44][49] Clinical Development and Pipeline - The company is advancing regulatory reviews for leniolisib in key markets and for pediatric use, with two ongoing Phase II trials for additional primary immunodeficiencies (PIDs) [4][27] - The acquisition of Abliva has been completed, adding KL1333 for mitochondrial diseases to the clinical pipeline, which has blockbuster potential [5][34] - Positive top-line results were announced for a Phase III trial of leniolisib in children aged 4 to 11, with global regulatory filings planned for the second half of 2025 [21][19] Market and Product Insights - The U.S. market contributed 98% of 2024 revenues, with the company focusing on expanding its portfolio in the U.S. and Joenja® in key countries [10][8] - The company identified over 880 diagnosed APDS patients globally, with ongoing initiatives to diagnose additional patients through genetic testing programs [14][15] - The strong growth in RUCONEST® revenue is attributed to new physician prescriptions and patient enrollments, with a 24% increase in total enrollments in 2024 [11][9] Financial Outlook - For 2025, the company anticipates total revenues between US$315 million and US$335 million, representing a growth of 6% to 13% [78] - The company plans to continue investing in its product portfolio and R&D pipeline, particularly for PIDs and primary mitochondrial diseases [7][78] - Operating expenses are expected to remain below the prior year's levels, with an estimated US$30 million in Abliva-related expenses [78]
Pharming Group reports on results of the 2025 Extraordinary General Meeting of Shareholders
Globenewswire· 2025-03-04 14:15
Core Points - Pharming Group N.V. announced the adoption of all proposals at the Extraordinary General Meeting of Shareholders, including the appointment of Mr. Fabrice Chouraqui as Executive Director and CEO for a four-year term [1][2] - Mr. Chouraqui succeeds Mr. Sijmen de Vries, who will remain as a strategic advisor until December 31, 2025, to ensure a smooth transition [2] - The remuneration package for Mr. Chouraqui was also approved by shareholders [2] Company Overview - Pharming Group N.V. is a global biopharmaceutical company focused on transforming the lives of patients with rare and life-threatening diseases [5] - The company is engaged in the commercialization and development of protein replacement therapies and precision medicines, including small molecules and biologics [5] - Pharming is headquartered in Leiden, the Netherlands, and operates in over 30 markets across North America, Europe, the Middle East, Africa, and Asia-Pacific [5] Leadership Commentary - Dr. Richard Peters, Chairman of the Board, expressed a warm welcome to Mr. Chouraqui and acknowledged the significant contributions of the outgoing CEO, Mr. de Vries, over the past 16 years [4]
Pharming Group to participate in March investor conference
Newsfilter· 2025-02-28 07:00
Core Insights - Pharming Group N.V. will participate in the 37th Annual Roth Conference in Dana Point, California, from March 16-18, 2025, with a fireside chat scheduled for March 17 at 01:30 PM PT [1] - The company is focused on developing and commercializing innovative therapies for patients with rare and life-threatening diseases [3] Company Overview - Pharming Group N.V. is a global biopharmaceutical company headquartered in Leiden, the Netherlands, with a presence in over 30 markets across North America, Europe, the Middle East, Africa, and Asia-Pacific [3] - The company specializes in protein replacement therapies and precision medicines, including small molecules and biologics [3]
Pharming Group to report fourth quarter and full year 2024 financial results on March 13
Newsfilter· 2025-02-27 07:00
Company Overview - Pharming Group N.V. is a global biopharmaceutical company focused on transforming the lives of patients with rare, debilitating, and life-threatening diseases [5] - The company is engaged in the commercialization and development of an innovative portfolio of protein replacement therapies and precision medicines, including small molecules and biologics [5] - Pharming is headquartered in Leiden, the Netherlands, and operates in over 30 markets across North America, Europe, the Middle East, Africa, and Asia-Pacific [5] Financial Reporting - Pharming will report its preliminary (unaudited) fourth quarter and full year 2024 financial results for the period ended December 31 on March 13, 2025 [1] - A presentation for analysts and investors will be hosted on the same day at 13:30 CET/08:30 am EDT [1] Conference Call Participation - Participants interested in the conference call must register in advance to receive dial-in information and a unique PIN [2] - The company will only take questions from dial-in attendees during the conference call [3]
Pharming to nominate biopharmaceutical leader Fabrice Chouraqui as new Executive Director and Chief Executive Officer
Newsfilter· 2025-01-21 06:00
Core Viewpoint - Pharming Group N.V. has nominated Fabrice Chouraqui as the new Executive Director and CEO, succeeding Sijmen de Vries, with the appointment to be confirmed at an upcoming Extraordinary General Meeting of Shareholders [1][2][3] Company Leadership Transition - Fabrice Chouraqui will be appointed for a term of four years, and Sijmen de Vries will resign from the Board but remain as a strategic advisor until December 31, 2025, to ensure a smooth transition [2][3] - Dr. Richard Peters, Chairman of the Board, expressed confidence in Chouraqui's ability to lead Pharming and implement its growth strategy in the rare disease sector [4] Fabrice Chouraqui's Background - Chouraqui is a seasoned global pharmaceutical executive with experience at Flagship Pioneering, Novartis, and Bristol-Myers Squibb, known for his leadership in launching treatments across various therapeutic areas [6][8][9] - He has a strong track record in developing commercial strategies and improving profitability, particularly during his tenure at Novartis [9][10] Strategic Vision - Chouraqui aims to build on Pharming's existing foundation and enhance its portfolio of products and commercial opportunities in the rare disease market [6][8] - His leadership is expected to drive Pharming's growth and position it as a leading company in the rare disease space [6][4]
European Biotech Pharming Group Touts Positive Data From Pivotal Pediatric Study Of FDA-Approved Immune System Disorder Drug
Benzinga· 2024-12-11 14:34
Core Insights - Pharming Group N.V. announced topline results from a Phase 3 trial of leniolisib for children aged 4 to 11 with activated phosphoinositide 3-kinase delta syndrome (APDS), a genetic disorder affecting the immune system [1][2] - Leniolisib, marketed as Joenja in the U.S., received FDA approval for APDS in patients aged 12 and older in March 2023, with plans to seek approval for younger patients starting in 2025 [2] Study Details - The Phase 3 study enrolled 21 children aged 4 to 11 years, focusing on primary efficacy endpoints such as reduction in lymph node size and increased proportion of naïve B cells after 12 weeks [3][5] - Secondary endpoints included evaluating the impact of leniolisib on health-related quality of life [4] Results - All 21 patients completed the 12-week treatment, showing improvement in lymphoproliferation and immunophenotype correction, consistent with previous results in older patients [5][6] - Treatment-emergent adverse events were mild to moderate, with no serious drug-related adverse events reported [6] Market Reaction - Following the announcement, PHAR stock increased by 11.6%, reaching $9.75 [7]
Pharming Group reports second quarter and first half 2024 financial results and provides business update
Newsfilter· 2024-08-01 05:00
Second quarter 2024 total revenues increased by 35% to US$74.1 million, compared to the second quarter 2023, driven by strong RUCONEST® and Joenja® revenue growth RUCONEST® second quarter revenue increased by 23% to US$63.0 million, compared to the second quarter 2023 Joenja® (leniolisib) second quarter revenue increased by 16% to US$11.1 million, compared to the first quarter 2024 First half total revenues increased by 33% to US$129.7 million, compared to the first half 2023 On track for 2024 total revenue ...