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
Pharming Group N.V. (NASDAQ:PHAR) Q4 2024 Earnings Conference Call March 13, 2025 8:30 AM ET Company Participants Fabrice Chouraqui - CEO Stephen Toor - CCO Anurag Relan - Chief Medical Officer Jeroen Wakkerman - CFO Conference Call Participants Jeff Jones ??? Oppenheimer Ben Jackson ??? Jefferies Alistair Campbell - RBC Capital Markets Joe Pantginis - H.C. Wainwright Simon Scholes - First Berlin Operator Good day and thank you for standing by. Welcome to the Pharming Group Fourth Quarter and Full-year 2024 ...
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 to participate in March investor conference
Newsfilter· 2025-02-28 07:00
Leiden, the Netherlands, February 28, 2025: Pharming Group N.V. ("Pharming") (NASDAQ:PHAR) announces that Pharming's management will participate in the following investor conference in the month of March: 37th Annual Roth Conference, Dana Point, CA, USA, March 16-18, 2025Anurag Relan, Chief Medical Officer, and Michael Levitan, VP Investor Relations and Corporate Communications, will participate in a fireside chat question and answer session on Monday, March 17 at 01:30pm PT/21:30 CET. For more information ...
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 N.V.(PHAR) - 2024 Q3 - Earnings Call Transcript
2024-10-24 17:44
Financial Data and Key Metrics Changes - Revenues in Q3 increased by 12%, with RUCONEST growing by 6% and Joenja by 73%, primarily driven by volume growth [26][27] - Gross profit increased by $9.7 million, aligning with revenue growth, while gross margin saw a slight increase [26] - The company reported a net loss of $1 million in Q3 compared to a net profit of $3.4 million in the previous year, mainly due to higher finance expenses [27][29] - Total revenues for the first nine months of 2024 increased by 25% to $204.5 million, with RUCONEST up 12% and Joenja up 210% compared to the same period last year [27][28] Business Line Data and Key Metrics Changes - RUCONEST generated approximately $64 million in Q3 and nearly $173 million for the first nine months of 2023, indicating strong cash flow generation [4][5] - Joenja achieved $32 million in sales year-to-date, reflecting significant growth since its launch [15][28] - The company continues to expand its prescriber base, adding 57 new prescribers this year, totaling around 800 [14] Market Data and Key Metrics Changes - The company is actively identifying and diagnosing new patients for Joenja, with approximately 50% of known patients identified in the US [15][19] - The market for hereditary angioedema (HAE) treatments remains competitive, with RUCONEST being the second most prescribed acute product in the US [13] Company Strategy and Development Direction - The company aims to expand its portfolio through in-licensing or acquiring clinical-stage opportunities in rare diseases [6][34] - There is a focus on launching Joenja in additional markets, including the UK and pediatric populations, with significant growth expected in 2026 [15][33] - The company is also initiating a Phase 2 trial for leniolisib in primary immune deficiencies, indicating a strategic move to broaden its therapeutic offerings [24][34] Management's Comments on Operating Environment and Future Outlook - Management expressed confidence in the continued growth of RUCONEST, particularly in a market where many patients still require acute therapies [13][38] - The company anticipates a steady increase in revenues, particularly from Joenja, with expectations to reach the midpoint of the revenue guidance range for the year [31][32] - Management highlighted the importance of ongoing patient education and engagement to ensure therapy adherence and successful market penetration [43] Other Important Information - The company is undergoing a leadership transition, with the CEO announcing plans to step down after 16 years, emphasizing the company's strong position for a successor [3] - The company has made adjustments to its operating expenses in response to market conditions and growth expectations [30][57] Q&A Session Summary Question: What is the outlook for RUCONEST and the on-demand market? - Management noted that refills for RUCONEST are occurring, reflecting patient needs, and the on-demand market remains stable despite new prophylactic therapies [36][38] Question: How are discussions with NICE progressing for Joenja in the UK? - Discussions with NICE are ongoing and productive, but specific details remain confidential [39][40] Question: What are the potential headwinds for Joenja's growth? - Short-term headwinds include a slowing growth rate as the company has identified a significant portion of the patient population, but upcoming launches in the UK and pediatric indications are expected to drive future growth [42][43] Question: What percentage of RUCONEST is used for rescue therapy versus prophylactic therapy? - It is estimated that almost all patients using RUCONEST are utilizing it for rescue therapy, as effective prophylactic options are available [46][48] Question: What is the average time for patients to be enrolled in therapy? - The average time for patients to be on paid therapy is between four to six weeks, depending on insurance processes [63]
Pharming N.V.(PHAR) - 2024 Q3 - Earnings Call Presentation
2024-10-24 15:13
Pharming Group N.V. Third quarter 2024 financial results and business update October 24, 2024 NASDAQ: PHAR | EURONEXT Amsterdam: PHARM � Pharming® SPEAKERS Sijmen de Vries, MD Stephen Toor Chief Executive Officer Chief Commercial Officer Anurag Relan, MD Jeroen Wakkerman Chief Medical Officer Chief Financial Officer Forward-looking statements This presentation may contain forward-looking statements. Forward-looking statements are statements of future expectations that are based on management's current expec ...