Core Viewpoint - Santhera Pharmaceuticals has appointed Marc Clausse as Chief Commercial Officer (CCO), effective June 1, 2026, succeeding Geert Jan van Daal, who will retire after 11 years with the company [1][5]. Group 1: Leadership Transition - Marc Clausse brings over 25 years of international experience in the life sciences industry, with expertise in specialty, oncology, and rare diseases [2]. - Geert Jan van Daal joined Santhera in 2015 and played a significant role in the European launch of AGAMREE® (vamorolone), enhancing patient access [5][6]. Group 2: Marc Clausse's Background - Clausse previously served as VP of International Strategy & Operations at Mirum Pharmaceuticals, where he successfully executed the company's international strategy and built regional commercial infrastructure [3]. - At GSK, he led the ovarian cancer portfolio to a market-leading position and integrated Tesaro UK following its acquisition [4]. Group 3: Company Overview - Santhera Pharmaceuticals focuses on developing and commercializing innovative medicines for rare neuromuscular diseases with high unmet medical needs [7]. - The company has an exclusive license for AGAMREE® (vamorolone), which is approved in multiple regions including the U.S., EU, UK, and Canada for the treatment of Duchenne muscular dystrophy (DMD) [7][8].

Core Insights - Santhera Pharmaceuticals presented long-term real-world data on AGAMREE® (vamorolone) at the MDA Clinical & Scientific Conference 2026, demonstrating its effectiveness and improved safety profile compared to traditional corticosteroids for treating Duchenne muscular dystrophy (DMD) [1][3][12]. Long-Term Effectiveness - Vamorolone shows comparable long-term effectiveness to classic corticosteroids, with no statistically significant difference in time to loss of ambulation between vamorolone and traditional treatments [5][9]. - The mean real-world dose of vamorolone was 4.5 ± 1.8 mg/kg/day, with a median exposure of approximately five years, indicating that patients may tolerate higher doses over the long term [5]. Growth and Development - Patients treated with vamorolone maintained height trajectories, achieving a mean height advantage of +12.17 cm compared to those on classic corticosteroids, where growth stunting was observed [6][9]. - Vamorolone is the first dissociative corticosteroid to demonstrate that patients with DMD can achieve normal growth without compromising efficacy [6]. Safety Profile - There was an 80% reduction in the proportion of patients with vertebral fractures in the vamorolone group (8.1%) compared to those treated with deflazacort (41.9%) [7][9]. - Non-vertebral fractures occurred in 27.5% of patients, indicating a favorable profile for chronic use compared to traditional corticosteroids [8]. - Cataracts were observed in significantly fewer patients treated with vamorolone (5.3%) compared to those on deflazacort (37.8%) [10][9]. Ongoing Research - The GUARDIAN study will continue to assess long-term outcomes, including muscle function, bone health, and additional safety endpoints, contributing to the understanding of vamorolone's long-term benefit-risk profile [11][12]. Company Overview - Santhera Pharmaceuticals focuses on developing innovative medicines for rare neuromuscular diseases, with AGAMREE® positioned as a dissociative anti-inflammatory drug and an alternative to classic corticosteroids for DMD [17].

Group 1: 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, which is being investigated as an alternative to standard corticosteroids for treating Duchenne muscular dystrophy (DMD) [2] - AGAMREE has received regulatory approvals in multiple regions, including the U.S., EU, UK, Switzerland, China, Hong Kong, and Canada [2] Group 2: Recent Developments - Santhera Pharmaceuticals has appointed Stifel Nicolaus Europe Limited as a corporate finance advisor, focusing on capital markets and international institutional investors [1] - Octavian AG will continue to act as a corporate finance advisor alongside Stifel to broaden investor reach [1] Group 3: Licensing Agreements - Santhera has out-licensed the rights to AGAMREE to various companies: Catalyst Pharmaceuticals for North America, Sperogenix Therapeutics for China and certain Southeast Asian countries, and Nxera Pharma for Japan, South Korea, Australia, and New Zealand [2]

Core Viewpoint - Santhera Pharmaceuticals has received approval from Swissmedic for AGAMREE® (vamorolone) to treat Duchenne muscular dystrophy (DMD) in patients aged four and older, with a commercial launch anticipated in the second half of 2026 [2][3]. Company Overview - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing innovative medicines for rare neuromuscular diseases with high unmet medical needs [8]. - The company has an exclusive license for AGAMREE® (vamorolone) from ReveraGen for all indications worldwide [8]. Product Details - AGAMREE is a novel drug that binds to the same receptor as glucocorticoids but modifies its downstream activity, potentially dissociating efficacy from steroid safety concerns [4]. - The drug has shown no growth restriction and no negative effects on bone metabolism, unlike traditional corticosteroids [6]. Clinical Study Results - In the pivotal Phase 2b VISION-DMD study, AGAMREE met the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo (p=0.002) at 24 weeks and demonstrated a good safety and tolerability profile [5]. - Common side effects included cushingoid features, vomiting, weight increase, and irritability, generally of mild to moderate severity [5]. Market Context - There are over 200 individuals affected by DMD in Switzerland, with limited treatment options available [3]. - Following the Swissmedic approval, Santhera has been granted a 15-year exclusivity period under Swiss orphan drug status, retaining exclusive distribution rights for AGAMREE in Switzerland [3].

Core Viewpoint - Santhera Pharmaceuticals has entered into an exclusive licensing agreement with Nxera Pharma UK Ltd. for the development and commercialization of AGAMREE® (vamorolone) for Duchenne muscular dystrophy (DMD) in Japan, South Korea, Australia, and New Zealand, which includes a total upfront payment of USD 40 million [1][2][4]. Financial Terms - The agreement includes an upfront payment of USD 40 million, consisting of USD 30 million in cash and USD 10 million as an equity investment at CHF 14.91 per share, representing a 20% premium to the 30-day VWAP [1][2]. - Santhera is also eligible for up to USD 165 million in sales and regulatory milestone payments, along with double-digit tiered royalties on net sales of AGAMREE in the licensed territories [2]. Responsibilities and Strategic Partnership - Nxera will handle regulatory approval for AGAMREE in the licensed territories and will conduct a registrational bridging clinical study, as well as lead commercial and manufacturing activities [3]. - This partnership leverages Nxera's expertise in the APAC region, particularly in Japan, where there is a significant unmet medical need for DMD patients [3]. Product Overview - AGAMREE is a novel drug that modifies the activity of glucocorticoid receptors, aiming to dissociate efficacy from steroid safety concerns, making it a potential alternative to existing corticosteroids for DMD treatment [5]. - 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 [6]. Market Potential - AGAMREE has shown no growth restriction or negative effects on bone metabolism, positioning it favorably compared to traditional corticosteroids [7]. - The partnership aims to expand global access to AGAMREE, addressing critical limitations of existing steroid therapies and potentially changing the standard of care for DMD patients in the targeted regions [4].

Core Viewpoint - Santhera Pharmaceuticals has successfully closed a USD 13 million royalty monetization agreement to support the global launch of AGAMREE® (vamorolone) [1][4]. Group 1: Agreement Details - The royalty monetization agreement was initially secured in September 2025 with R-Bridge, covering 25% of net royalties on AGAMREE from Catalyst Pharmaceuticals in North America and Sperogenix Therapeutics in China [2]. - Partners Group, a major player in the global private markets, has joined the financing, contributing a majority of the USD 13 million raised [2]. - Payments to Partners Group and R-Bridge are capped, and once the ceiling or duration is reached, royalties revert to Santhera [3]. Group 2: Company Background - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing innovative medicines for rare neuromuscular diseases with high unmet medical needs [4]. - The company holds an exclusive license for AGAMREE® (vamorolone), which is approved for treating Duchenne muscular dystrophy (DMD) in multiple regions including the U.S., EU, UK, China, Hong Kong, and Canada [4].

Core Insights - Santhera Pharmaceuticals reported positive topline results from the long-term analysis of the GUARDIAN study evaluating AGAMREE® (vamorolone) for Duchenne muscular dystrophy (DMD) patients [1][5] Long-term Analysis - The analysis included corticosteroid-naïve patients aged four to seven who received AGAMREE for up to eight years, with a median follow-up of about five years [2] - Up to 110 patients were analyzed, with most remaining on higher doses (4–6 mg/kg/day) during the observation period [2] Efficacy - Patients treated with AGAMREE maintained motor function over extended follow-up, showing durable efficacy comparable to standard corticosteroids in terms of time to loss of ambulation (p=0.91) [3] - No significant differences were observed in pre-specified subgroup analyses compared to daily deflazacort or prednisone [3] Safety and Tolerability - AGAMREE demonstrated a differentiated safety profile, with a significantly lower rate of vertebral fractures (p=0.0061) and normal growth without stunting, unlike standard corticosteroids (p<0.0001) [4] - Fewer cases of cataracts were reported compared to glucocorticoids, including a notably lower incidence versus deflazacort (p<0.015) [4] - No cases of glaucoma were observed, and changes in BMI or weight did not differ when matched for height [4] Future Developments - Detailed results will be presented at a major international scientific conference in Q1 2026, with complete data to be shared publicly afterward [5] - Further readouts from the GUARDIAN study are planned over the next three years, focusing on a broader set of efficacy and safety outcomes [5] Expert Commentary - Experts in the field expressed encouragement regarding the long-term efficacy and safety profile of AGAMREE, highlighting its potential benefits over conventional corticosteroids [7][9]

Core Insights - Santhera Pharmaceuticals has signed an exclusive distribution agreement with Biomedica for AGAMREE® (vamorolone) in Russia, aimed at treating Duchenne muscular dystrophy (DMD) in patients aged four and older [1][2][3] Agreement Details - Under the agreement, Santhera will receive a percentage of net sales, with sales expected to commence in Q1 2026 [2] - The collaboration will facilitate access to AGAMREE for approximately 1,400 DMD patients in Russia through managed access programs [2] Company Statements - Dario Eklund, CEO of Santhera, emphasized the importance of this agreement in increasing access to DMD treatment and noted it as part of a series of global agreements made in 2025 [3] - Oleg Parosin, CEO of Biomedica, highlighted their expertise in neurology and DMD therapies, positioning them as a strong partner for Santhera in Russia [3] Product Information - AGAMREE is a novel drug that modifies the action of glucocorticoids, aiming to dissociate efficacy from safety concerns associated with traditional corticosteroids [4] - In the pivotal VISION-DMD study, AGAMREE demonstrated significant efficacy and a favorable safety profile, with a primary endpoint met at 24 weeks [5] Safety and Efficacy - AGAMREE has shown no growth restrictions or negative effects on bone metabolism, distinguishing it from traditional corticosteroids [6]

Core Viewpoint - Health Canada has approved AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older, marking it as the first approved treatment for DMD in Canada [1][9]. Company Overview - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing innovative medicines for rare neuromuscular diseases with high unmet medical needs [11]. - Kye Pharmaceuticals holds exclusive Canadian commercial rights to AGAMREE for DMD following a sub-license agreement with Santhera's commercialization partner, Catalyst Pharmaceuticals [2]. Product Information - AGAMREE is a novel drug that binds to the same receptor as glucocorticoids but modifies its downstream activity, potentially dissociating efficacy from steroid safety concerns [5]. - In the pivotal VISION-DMD study, AGAMREE met the primary endpoint of Time to Stand (TTSTAND) velocity versus placebo at 24 weeks of treatment, with a p-value of 0.002, and demonstrated a good safety and tolerability profile [6]. Market Context - The Canadian Neuromuscular Disease registry estimates over 800 boys and young men living with DMD in Canada [1]. - AGAMREE is now the sixth independent approval by local health authorities, following approvals in the U.S., Europe, the UK, China, and Hong Kong [3].

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 has an exclusive license for AGAMREE® (vamorolone), a dissociative steroid, which is approved for treating Duchenne muscular dystrophy (DMD) in multiple regions including the U.S., EU, UK, China, and Hong Kong [2] Convertible Bond Update - The conversion price for the newly announced convertible bond is set at CHF 13.5446, which represents a 10% premium to the volume-weighted average price (VWAP) of Santhera's shares on 23 September 2025 [6] - The coupon rate for the bond is 7%, indicating a reduction compared to the previous bond [6] - The total principal amount of the convertible bond, including the exchange at parity of the prior bond, is CHF 20.132 million [6]