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 - 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 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]