Pharming Group (NasdaqGM:PHAR) FY Conference Transcript

Summary of Pharming Group FY Conference Call Company Overview - Company: Pharming Group (NasdaqGM:PHAR) - CEO: Fabrice Chouraqui - Date of Conference: September 08, 2025 Key Points Company Transformation - Pharming has evolved from a one-asset company with RUCONEST to a company with two commercial assets: RUCONEST and Joenja, both experiencing double-digit growth [2][3] - The company has a high-value pipeline with two programs projected to have sales potential exceeding $1 billion [2] Financial Performance - Sales grew by 26% in the last quarter, driven by RUCONEST and Joenja [4] - Revenue guidance for the year has been raised to between $335 million and $350 million [4] RUCONEST - RUCONEST is a cornerstone treatment for hereditary angioedema, with 97% of attacks treated with one dose and 93% of attacks stopped for at least three days [5][6] - The drug has been on the market for 10 years and is growing at a rate of 28% year-on-year [6] - RUCONEST's unique manufacturing process from transgenic rabbits makes it difficult to replicate [7] Joenja - Joenja is indicated for activated PI3K delta syndrome (APDS) and has shown significant patient uptake, with more new patients in the first half of the year than in the entire previous year [10][11] - Three growth catalysts for Joenja include: 1. Reclassification of VUS patients, with an estimated 20% of VUS patients potentially reclassified as APDS [12][13] 2. Expansion of the label to pediatric patients, with an NDA submitted to the FDA [14] 3. Geographic expansion, including recent launches in the UK and an NDA filed in Japan [15] Pipeline and Future Prospects - Pharming is studying Joenja's efficacy in adjacent indications, which could significantly broaden its market potential [16][17] - KL1333, acquired from Ableva, is being developed for primary mitochondrial disease, with a Phase 2 registrational trial ongoing [18][19] - The company aims to self-fund the development of its pipeline and launch of new drugs, leveraging cash flows from RUCONEST [20] Growth Outlook - The company has raised its revenue guidance and expects continued strong growth, with significant short-term catalysts for both Joenja and KL1333 [21][22] Additional Insights - The company is focused on creating a world leader in rare diseases, leveraging its capabilities in clinical development, supply chain, and commercial operations [3] - The strong performance of RUCONEST is critical for funding the expansion of the portfolio and pipeline [20]