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]

Core Insights - Santhera Pharmaceuticals reported a significant increase in total revenue by 70% to CHF 24 million for the first half of 2025, driven by strong sales of AGAMREE and growing royalty revenues [5][23][24] - The company is advancing the global rollout of AGAMREE, particularly in the US, Germany, Austria, and the UK, with expectations for continued growth in sales and market share [3][6][14] - Santhera has secured additional financing of approximately CHF 20 million to support increased product demand and global launches, maintaining a cash-flow break-even guidance for mid-2026 [21][31] Financial Performance - Total revenue increased to CHF 24 million from CHF 14.1 million in H1 2024, with product sales rising 76% to CHF 11.6 million [5][23][24] - Royalties from licensing partners surged to CHF 5.4 million, a 500% increase compared to CHF 0.9 million in H1 2024, indicating strong growth in the US and China [5][24] - Operating loss widened to CHF 35.4 million from CHF 17.7 million in H1 2024, primarily due to a one-time milestone payment of USD 25 million [31][28] Market Expansion - AGAMREE has achieved over 40% market penetration among steroid-using DMD patients in Germany, with Austria exceeding 50% market share [6][7] - The UK market has positively contributed following the nationwide launch in April 2025, supported by favorable NICE guidance [8] - Santhera is actively pursuing pricing and reimbursement discussions in Spain, Italy, and the Nordic regions, with launches expected from Q4 2025 to Q1 2026 [10][11][12] Strategic Initiatives - The company is focusing on expanding its distribution agreements across multiple regions, including five Gulf Cooperation Council countries, India, and Türkiye [15][17] - Santhera plans to present long-term clinical outcomes from the GUARDIAN study in early Q4 2025, which may further support AGAMREE's market position [18] - The company is not planning near-term investments in additional indication expansions for AGAMREE but is looking to leverage partnerships for future studies [19] Leadership and Governance - Santhera appointed Catherine Isted as CFO in February 2025 and Dr. Melanie Rolli to the Board in May 2025, enhancing its leadership team with experienced professionals [20]

Core Viewpoint - Santhera Pharmaceuticals has secured approximately CHF 20 million in additional funding to support the global rollout of AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD), driven by increased demand and inventory needs [2][3][8] Financing Agreements - Santhera has entered into financing agreements with R-Bridge and Highbridge Capital Management, securing USD 13 million through royalty monetization and CHF 10 million via an upsized convertible bond [4][5][7] - The royalty monetization with R-Bridge allows them to receive 25% of net royalties from AGAMREE sales in North America and China, with a payment of USD 13 million to Santhera upon closing [5][6] - Highbridge's additional CHF 10 million will be provided through a new convertible note, extending the maturity of an existing bond and offering increased flexibility [7] Market Demand and Performance - AGAMREE has treated over 1000 patients globally, with strong sales performance reported in the U.S. at USD 49.4 million for the first half of 2025, driven by physician adoption [2][5] - Demand for AGAMREE is expected to increase in China following its launch into the private-pay market, with expectations for growth in 2025 and 2026 [2][5] Strategic Goals - The additional funding will enable Santhera to accelerate product launches, build inventory, and aim for cash flow break-even by 2026 [5][8] - The company continues to maintain partnerships with high-quality investors to support its growth trajectory [5]

Core Viewpoint - Santhera Pharmaceuticals has signed an exclusive agreement with Ikris Pharma Network to distribute AGAMREE® (vamorolone) in India for treating Duchenne muscular dystrophy (DMD) in patients aged four and older, with sales expected to start in Q4 2025 [1][2][12] Company Overview - Santhera Pharmaceuticals is a Swiss specialty pharmaceutical company focused on developing innovative medicines for rare neuromuscular diseases, particularly DMD [8] - AGAMREE® (vamorolone) is a novel drug that acts similarly to glucocorticoids but aims to reduce associated safety concerns, positioning it as an alternative to existing corticosteroids [4][8] Partnership Details - The agreement with Ikris has a five-year term, and Santhera will receive a percentage of net sales, consistent with previous distribution agreements [1][2] - Ikris Pharma Network is India's largest named patient supply company, specializing in rare disease medications and has experience in DMD [11] Product Information - AGAMREE has shown potential to dissociate efficacy from steroid safety concerns, making it a promising treatment option for DMD [4][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 [5][6] Market Expansion - The agreement with Ikris follows recent distribution deals in Turkey and Gulf Cooperation Council countries, indicating Santhera's commitment to expanding AGAMREE's global access [2][3] - The collaboration aims to provide a regulatory-compliant pathway for access to AGAMREE and enhance patient support and disease awareness in India [12][13]

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]