Core Viewpoint - Santhera Pharmaceuticals has signed an exclusive agreement with Uniphar for the distribution of AGAMREE® (vamorolone) in several Gulf Cooperation Council (GCC) countries, aiming to provide treatment for Duchenne muscular dystrophy (DMD) patients aged four and older, with sales expected to start in Q1 2026 [1][2]. Group 1: Agreement Details - The agreement allows Uniphar to manage the distribution of AGAMREE in the UAE, Saudi Arabia, Kuwait, Oman, and Bahrain, with sales beginning on a named patient basis in early 2026 and broader commercial sales anticipated later that year [1]. - Santhera will receive a percentage of net sales as payment, consistent with previous distribution agreements [1]. Group 2: Company Commitment and Strategy - Santhera's CEO expressed excitement about the partnership, highlighting Uniphar's regional presence and expertise in addressing the unmet needs of DMD patients in the GCC [2]. - The agreement is part of Santhera's global expansion strategy, which includes multiple regions across North America, Europe, and Asia [2]. Group 3: Product Information - AGAMREE is a novel drug that acts on the same receptor as glucocorticoids but modifies its downstream activity, potentially dissociating efficacy from steroid safety concerns [3]. - In the pivotal VISION-DMD study, AGAMREE met its primary endpoint and demonstrated a good safety profile, with common side effects being mild to moderate [4]. Group 4: Market Position and Regulatory Status - AGAMREE is approved in various regions, including the U.S., EU, UK, China, and Hong Kong, and is positioned as an alternative to existing corticosteroids for DMD treatment [8]. - Santhera has out-licensed rights to AGAMREE for North America and parts of Asia, indicating a strategic approach to market penetration [8].

Company Overview - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing and commercializing innovative medicines for rare neuromuscular diseases with high unmet medical needs [2] - The company holds an exclusive license for AGAMREE® (vamorolone), a dissociative steroid, for all indications worldwide from ReveraGen [2] - AGAMREE is approved for the treatment of Duchenne muscular dystrophy (DMD) in multiple regions, including the U.S., EU, UK, China, and Hong Kong [2] Recent Developments - Santhera Pharmaceuticals and Highbridge Capital Management have mutually agreed to extend the maturity date of an existing CHF 7 million private convertible bond to 30 September 2025 [1]

Core Insights - Santhera Pharmaceuticals has signed an exclusive agreement with Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. for the distribution and promotion of AGAMREE® (vamorolone) in Turkey for treating Duchenne muscular dystrophy (DMD) in patients aged four and older [1][2] - The supply and sales by GEN are expected to commence in the first half of 2026, starting with named patient sales followed by commercial sales [1] - Santhera will receive a small upfront payment and an ongoing percentage of net sales as per previous agreements [1] Company Overview - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing innovative medicines for rare neuromuscular diseases with high unmet medical needs [7] - AGAMREE® (vamorolone) is a dissociative steroid with a novel mode of action, approved in multiple regions including the U.S., EU, UK, China, and Hong Kong for the treatment of DMD [7][9] - The company has out-licensed rights to AGAMREE for North America and certain Southeast Asian countries [9] Product Information - AGAMREE is designed to bind to the same receptor as glucocorticoids but modifies its downstream activity, potentially dissociating efficacy from steroid safety concerns [3] - In the pivotal VISION-DMD study, AGAMREE met the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo with a p-value of 0.002 at 24 weeks, demonstrating a good safety and tolerability profile [4] - Unlike corticosteroids, AGAMREE shows no restriction of growth and has no negative effects on bone metabolism [5] Partner Company Overview - Gen İlaç ve Sağlık Ürünleri San. ve Tic. A.Ş. is Turkey's leading specialty pharmaceutical company, focusing on innovative therapies for neurological and neuromuscular disorders since 1998 [10] - GEN has a GMP-certified facility and invests in original drug development through dedicated R&D centers [10]

Core Points - Santhera Pharmaceuticals' shareholders approved all motions by the Board of Directors at the Annual General Meeting with a participation representing 5,073,050 shares or 37.5% of the Company's share capital [1] Group 1: Financial Approvals - Shareholders approved the 2024 Annual Report, annual financial statements, and consolidated financial statements, along with the appropriation of the annual result against free reserves [2] - Conditional capital for employee participation was increased by CHF 63,000 to CHF 111,280.20 [3] Group 2: Board Elections - Thomas Meier, Philipp Gutzwiller, and Bradley C. Meyer were re-elected to the Board of Directors for another one-year term, with Thomas Meier also re-elected as Chairman [4] - Melanie Rolli was elected as a new member of the Board, and the Nomination & Compensation Committee members were confirmed [4] Group 3: Compensation Approvals - The Compensation Report for 2024 was endorsed in a consultative vote, and shareholders agreed to the fixed compensation for the Board of Directors and Executive Management for the upcoming periods [5]

Company Overview - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing and commercializing innovative medicines for rare neuromuscular diseases with high unmet medical needs [3] - The company holds an exclusive license for AGAMREE® (vamorolone), a dissociative steroid, for all indications worldwide, which is being investigated as an alternative treatment for Duchenne muscular dystrophy (DMD) [3] - AGAMREE has received regulatory approvals in multiple regions, including the U.S., EU, UK, China, and Hong Kong [3] Annual General Meeting (AGM) Details - The AGM is scheduled for May 20, 2025, at 10:00 CEST in Pratteln, Switzerland [1] - Registered shareholders will receive the invitation by mail, and the agenda will be available on the company's website [1] AGM Agenda Items - Approval of the Annual Report, Annual Financial Statements, and Consolidated Financial Statements for 2024 [5] - Appropriation of the Annual Result and Offset of Deficit [5] - Consultative Vote on the Compensation Report for 2024 [5] - Discharge of the Board of Directors and Executive Management from Liability for the Financial Year 2024 [5] - Conditional Capital for Employee Participations and Amendment to the Articles of Incorporation [5] - Election of a new Member and Re-election of two Members of the Board of Directors, including the Chairman [5] - Re-election of the Members of the Nomination & Compensation Committee [5] - Approval of the Compensation for the Members of the Board of Directors and Executive Management [5] - Re-election of the Statutory Auditors and Independent Proxy [5]

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]

Core Insights - Santhera Pharmaceuticals has made significant progress in the commercial rollout of AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD), with funding from NHS England now available for prescribing in England [1][2] - AGAMREE has received positive final guidance from NICE, and initial sales have already been achieved in Scotland and Wales [1][2] - Kye Pharmaceuticals, a sub-licensee of Catalyst Pharmaceuticals, has received acceptance from Health Canada for AGAMREE's New Drug Submission with Priority Review, potentially leading to its approval as the first treatment for DMD in Canada by the end of 2025 [2] Product Overview - AGAMREE (vamorolone) is a novel drug that binds to the same receptor as glucocorticoids but modifies its downstream activity, positioning it as a dissociative anti-inflammatory drug and an alternative to existing corticosteroids [3][7] - The pivotal VISION-DMD study demonstrated that AGAMREE met the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks, with a good safety and tolerability profile [4][5] - Unlike corticosteroids, AGAMREE shows no restriction of growth and has no negative effects on bone metabolism, as indicated by normal serum markers for bone formation and resorption [5] Company Background - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing and commercializing innovative medicines for rare neuromuscular diseases with high unmet medical needs [7] - The company holds an exclusive license for AGAMREE for all indications worldwide and has out-licensed rights for North America to Catalyst Pharmaceuticals and for China and certain Southeast Asian countries to Sperogenix Therapeutics [7][9]