Pharming N.V.(PHAR)2024-10-24 17:44Financial 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 1 million in Q3 compared to a net profit of 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 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]