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]

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].