Press Release: Santhera Full Year Results for the -2-
Financially, the Company secured additional funding to support ongoing operations and market launches and has guided to cash break-even by mid-2026. Total revenues for the year were CHF 39.1 million and included CHF 15.0 million from own market product sales driven by the successful launches in Germany and Austria.
The year also marked important advances in manufacturing scale-up and supply chain management as well as long-term data generation. This will be important for the continued successful global rollout of AGAMREE over the coming years, as the Company seeks to maximize the potential of the drug in helping DMD patients globally.
AGAMREE(R) commercial rollout
The commercial rollout of AGAMREE in 2024 represented a landmark in Santhera's evolution. The Company's ability to translate clinical success into real-world patient access across multiple regions demonstrated both the clinical value of AGAMREE and Santhera's strong regulatory and commercial capabilities.
In Europe, the product was first launched in Germany in January 2024. Germany, as the largest pharmaceutical market in Europe, served as a critical bellwether for AGAMREE's clinical adoption. By year-end, over 300 patients in Germany had transitioned to AGAMREE, representing approximately 30% of those currently treated with corticosteroids. This rapid uptake was supported by broad reimbursement agreements and favorable feedback from prescribers, highlighting AGAMREE's benefits in terms of bone health, growth, and behavior compared to traditional steroids.
Austria followed closely behind, with national reimbursement secured and uptake gradually increasing as pre-scriber familiarity and patient demand grew. Parallel efforts were initiated in additional EU markets, with pricing and reimbursement negotiations advancing in Italy, Spain, Benelux, the Nordics and other EU countries. These launches are expected to progress through 2025 and 2026, in line with local market access and reimbursement negotiation timelines.
In the United Kingdom, AGAMREE received Medicines and Healthcare products Regulatory Agency MHRA marketing authorization in January 2024. In December 2024 AGAMREE went on to receive a draft positive recommendation from the National Institute for Health and Care Excellence (NICE). Following positive final guidance from NICE in January 2025 AGAMREE became available for prescription across the UK from April 2025.
In North America, Santhera's U.S. licensee, Catalyst Pharmaceuticals (Catalyst), launched AGAMREE in March 2024 following FDA approval in October 2023. Catalyst reported strong early demand from the Duchenne community and healthcare providers, achieving USD 46 million in net sales by year-end. These results exceeded initial forecasts and reflect AGAMREE's strong positioning as a new standard of care for DMD patients. Based on current projections, Catalyst has provided guidance for 2025 sales of USD 100-110 million, which would trigger additional milestones for Santhera.
In China, Santhera's partner Sperogenix Therapeutics initiated early access programs for AGAMREE in mid-2024, while awaiting full regulatory approval. In December, China's National Medical Products Administration (NMPA) granted marketing authorization for AGAMREE, positioning Sperogenix to launch commercially on a non-reimbursed basis during the latter half of 2025, with plans to secure reimbursement in early 2026. These early efforts will establish a critical foothold in one of the world's largest rare disease markets.
The commercial launch of AGAMREE in 2024 underscored the global medical community's recognition of its differentiated clinical profile. Santhera continues to engage with clinicians, patient advocacy groups, and regulators to ensure rapid and equitable access to this important treatment globally, either directly or through its strategic partnerships.
Market access and reimbursement
Achieving broad and sustainable access for AGAMREE across international markets was a core priority for Santhera in 2024. Building on foundational work undertaken in the previous year, the Company advanced multiple national reimbursement submissions and pricing negotiations, positioning AGAMREE for long-term availability through public healthcare systems.
In Germany, AGAMREE was granted full reimbursement under the AMNOG process following rapid consensus pricing negotiations with the GKV-SV. The product was listed with an agreed price supporting access for all eligible DMD patients. Santhera's early engagement with key opinion leaders and health authorities helped expedite access pathways and ensured clinical and economic evidence was well aligned with payer expectations.
Austria followed a similar path, with positive decisions from the Hauptverband der österreichischen Sozialversi-cherungsträger (HVSV), enabling inclusion in the national reimbursement system. Santhera also initiated submissions in Belgium, the Netherlands, and Luxembourg, where national health authorities indicated a willingness to consider AGAMREE under existing frameworks for rare disease treatments.
In the United Kingdom, NICE published its final guidance recommending AGAMREE as a cost-effective option for treating DMD in steroid-naive and previously treated patients. The appraisal committee recognized AGAMREE's differentiated benefit-risk profile, particularly around growth preservation, bone health, and behavior. In parallel, NHS England confirmed product inclusion in the national formulary, supporting broad patient access.
In Spain and Italy, Santhera progressed dossier submissions and pricing negotiations with the Spanish Agency of Medicines and Medical Devices (AEMPS) and the Italian Medicines Agency (AIFA), respectively. Engagements with the Spanish regional authorities and Italian Technical-Scientific Committee (CTS) are ongoing and expected to lead to reimbursed access in 2025 or early 2026.
In the Nordics, Santhera pursued market access pathways in Sweden, Denmark, Norway, and Finland through the Nordisk Läkemedelsstatistik collaboration. While timelines vary by country, all four health systems indicated interest in the treatment, particularly for pediatric patients transitioning from standard steroids.
Outside of Europe, Santhera worked closely with its commercial partners to align reimbursement and pricing strategies. Catalyst in the U.S. undertook formulary negotiations with public and private payers, achieving broad coverage across Medicaid, Medicare Part D, and key commercial plans. The Centers for Medicare & Medicaid Services (CMS) classified AGAMREE as a preferred treatment for eligible pediatric DMD patients, supporting strong early adoption.
In China, Sperogenix received marketing authorization approval from the NMPA in December 2024. The Company began preparation for provincial listing submissions, with initial pricing discussions proceeding during 2025. Full reimbursement is targeted for 2026 through China's National Reimbursement Drug List.
Market access workstreams are ongoing across the Middle East, Central and Eastern Europe, and Latin America to establish new distribution partners or additional regional licensees. These efforts are backed by a central medical affairs and health economics team based in Switzerland, who continue to develop real-world evidence packages and pharmacoeconomic dossiers to support local submissions.
Santhera's commitment to access goes beyond pricing. The Company has established an early access framework in select countries and provided product through compassionate use programs where regulatory conditions allow. Additionally, educational outreach to clinicians, patient groups, and payer stakeholders has reinforced understanding of AGAMREE's clinical differentiation.
Partnerships and licensing
Santhera's commercial success in 2024 was underpinned by a robust network of strategic partnerships that continued to deliver value across core regions. These collaborations provided capital-efficient routes to market, accelerated geographic expansion, and strengthened global brand presence for AGAMREE.
In the U.S., Catalyst led the commercial launch of AGAMREE following FDA approval. Under the licensing agreement, Santhera is eligible to receive low to high teens tiered royalties on U.S. net sales and up to USD 105 million in further commercial milestone payments. Catalyst's experienced rare disease commercial infrastructure proved instrumental in achieving USD 46 million in net sales in its first year, exceeding its market guidance. The partnership also supported post-marketing commitments and real-world data generation in the U.S. market.
In China, Santhera's exclusive regional partner, Sperogenix Therapeutics, advanced AGAMREE towards commercialization following regulatory approval in late 2024. Sperogenix initiated early access programs and began preparing for commercial launch in the second half of 2025, with reimbursement planned for 2026. As with the Catalyst agreement, Santhera receives tiered royalties on sales as well as milestone payments relating to commercial success.
In Europe, Santhera pursued a hybrid model of direct commercialization in key markets (e.g. Germany, UK, France, Spain, Italy, Switzerland) and out-licensing or distribution agreements in secondary territories.
Santhera entered into a distribution agreement with GENESIS Pharma SA in July 2024, for 20 markets in Central and Eastern Europe. Genesis has since successfully introduced AGAMREE in several of their markets, either via Named Patient Supply or via full reimbursement, with Slovenia being the first country in the territory to achieve the latter in April 2025. The two companies continue their close collaboration to ensure the availability of AGAMREE across all countries in the region.
In 2024 the Company also signed regional distribution agreements covering the Israel and Qatar. These partnerships aim to extend AGAMREE's reach while ensuring efficient local market access and regulatory navigation. Santhera remains active in expanding territories through additional partnerships.
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April 29, 2025 01:00 ET (05:00 GMT)