Santhera Full Year Results for the Year Ended 31 December 2024

Core Insights - The year 2024 was a transformational period for Santhera Pharmaceuticals, marked by the successful launch of AGAMREE® for Duchenne muscular dystrophy (DMD) in Germany, Austria, and the U.S. [1][4][12] - The company secured financing of up to CHF 69 million, which is expected to support growth initiatives and provide liquidity until cash flow break-even in 2026 [1][10][67] - Santhera aims to expand its product portfolio and establish itself as a leading specialty pharmaceutical company in rare diseases [1][4] Operational Highlights - AGAMREE was launched in Germany and Austria, with over 300 patients on treatment by year-end, representing nearly 30% of those on steroid treatment [4][16] - Catalyst Pharmaceuticals launched AGAMREE in the U.S. in March 2024, achieving USD 46 million in revenue for the year, exceeding guidance [4][19] - The partnership with Sperogenix in China initiated an early access program, with commercial rollout expected in mid-2025 [4][20] Financial Highlights - Total revenue for 2024 was CHF 39.1 million, a decrease from CHF 103.4 million in 2023, primarily due to significant licensing milestones recognized in the previous year [4][49] - Product sales amounted to CHF 14.8 million, driven by the successful launch of AGAMREE in Germany and Austria [4][49] - Operating loss for 2024 was CHF 33.2 million, compared to a profit of CHF 68.8 million in 2023, influenced by the absence of prior out-licensing milestones [10][56] Market Access and Reimbursement - AGAMREE received full reimbursement in Germany and Austria, facilitating patient access [22][23][24] - In the UK, AGAMREE was recommended as a cost-effective treatment by NICE, with availability for prescription starting in April 2025 [25][18] - Ongoing negotiations for pricing and reimbursement in other EU countries are expected to progress through 2025 and 2026 [17][26] Partnerships and Licensing - Santhera's partnership with Catalyst Pharmaceuticals in the U.S. includes tiered royalties and potential milestone payments, enhancing market access [34][66] - The collaboration with Sperogenix in China is set to provide tiered royalties and milestone payments, supporting commercialization efforts [35][66] - Santhera is pursuing a hybrid model of direct commercialization in key markets while establishing distribution agreements in secondary territories [36][38] Manufacturing and Supply Chain - Santhera is validating additional manufacturing sites to meet global demand and improve supply chain efficiencies [39][40] - A secondary manufacturing site in the U.S. is being qualified to support growing demand and mitigate supply risks [41] - The company is also exploring local production options in China to enhance pricing and reimbursement discussions [42] R&D Strategy and Pipeline Development - Santhera will focus on maximizing AGAMREE's potential in DMD and generating long-term safety data, with results from the GUARDIAN study expected in Q4 2025 [46] - The company is actively seeking to expand its late-stage pipeline through licensing and distribution agreements, particularly in the rare disease field [47] Financial Guidance and Outlook - For 2025, Santhera expects total revenues in the range of CHF 65-70 million, driven by continued global rollout of AGAMREE [71] - The company anticipates operating expenses to be between CHF 50-55 million, excluding non-cash share-based compensation [71] - Long-term revenue guidance includes expectations of EUR 150 million by 2028, excluding milestones [72]