Press Release: Santhera Pharmaceuticals Full Year -2-

Introduction

Santhera delivered strong commercial progress in 2025, driven by the continued global rollout of AGAMREE(R) (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD). Total revenue increased by 97% to CHF 77 million, significantly exceeding prior guidance, reflecting strong growth in product sales in Europe and increasing royalties, milestones and product supply revenues from partners as sales outside Europe accelerated.

During 2025 and into 2026, several key foundations were established to support future growth. These included multiple new distribution partnerships, a major licensing agreement covering Japan and key Asia-Pacific markets and agreement on reimbursement in Spain. The Company also presented long-term clinical data on AGAMREE demonstrating improved safety while maintaining efficacy comparable to traditional corticosteroids, and established a second European-based manufacturing site to support future demand.

These developments, together with continued progress on pricing and reimbursement discussions in additional countries expected to launch during 2026, support a strong outlook for Santhera and expand access for patients with DMD globally.

Continued strong uptake in core European markets

Following the initial launches in Germany and Austria in 2024, AGAMREE continued to demonstrate strong adoption in 2025.

In Germany, over 40% of corticosteroid-treated DMD patients have now been treated with AGAMREE, with increasing use across both newly diagnosed patients and in restarts and switches including among older patient populations. Austria continues to lead in terms of penetration, with more than 50% of steroid-treated patients receiving AGAMREE.

Across Germany and Austria, sales continued to grow strongly into Q1 2026, with orders in Q1 2026 over 50% higher than those for the same period in the prior year. While the price per bottle in Germany decreased to EUR 3,001 in January 2026 from EUR 3,612, reflecting a one-time, pre-agreed price adjustment at the time of initial pricing and reimbursement in early 2025, strong volume growth has more than offset this impact.

In the United Kingdom, AGAMREE became available nationwide from April 2025 following positive NICE guidance. Uptake has been supported by updated treatment guidelines and improved access initiatives, including home delivery programs. The UK launch is tracking the early trajectory seen in Germany and has also delivered a strong performance in Q1 2026.

Advancing rollout across Europe

Santhera continued to advance pricing and reimbursement discussions across key European markets throughout 2025.

In Spain, AGAMREE has been available through a named-patient program since mid-2024, with reimbursement discussions progressing during 2025. Post period-end, in February 2026, AGAMREE was proposed for inclusion in Spain's National Health System, representing a key milestone towards full reimbursement and broad patient access, with launch planned imminently. As part of the agreement with the Spanish National Health System and to accelerate patient access to therapy, Santhera will provide product free of charge during Q2 2026, with commercial sales commencing from July 2026.

In Italy, the Company continues to engage with the Italian Medicines Agency. Following submission of the long-term data, pricing and reimbursement discussions have continued positively. In a further positive step in April 2026, the Agency's Board of Directors approved the reimbursement of AGAMREE by the National Health Service. Santhera is now awaiting publication in the official Gazette, which will occur during Q2, after which commercialization will begin.

In other European markets, including the Nordics, Benelux, and Switzerland, pricing and reimbursement processes are progressing, with launches expected during the course of 2026.

Catalyst Pharmaceuticals -- Continuing to beat expectations

Santhera's U.S. partner, Catalyst Pharmaceuticals, continued to deliver strong commercial performance in 2025, reinforcing AGAMREE's positioning as a new standard of care in DMD.

Catalyst reported full-year 2025 AGAMREE sales of USD 117 million, exceeding its initial full-year guidance of USD 100--110 million. As sales exceeded USD 100 million, this triggered a USD 12.5 million milestone payment to Santhera, recognized in 2025 revenue.

Sustained demand and broad penetration continue to support growth in the U.S., with Catalyst noting that approximately 90% of DMD Centers of Excellence have started using AGAMREE for their patients. Catalyst has guided that it expects to achieve USD 140--150 million of AGAMREE sales in 2026, reflecting continued adoption and momentum.

Since mid-2025, Catalyst has also held the license to manufacture AGAMREE for use in North America and has established its own drug product manufacturing capabilities to serve its territory. As a result, from Q1 2026 onwards, Santhera does not expect to generate further product sales to Catalyst.

Sperogenix -- Strong growth in private payor market

In China, Santhera's partner Sperogenix initiated a non-reimbursed commercial rollout of AGAMREE in September 2025, following prior early-access programs. Uptake has been strong, with more than 800 patients treated to date, supporting increasing demand and positioning the product for future reimbursement discussions.

China represents a significant long-term growth opportunity, with ongoing efforts focused on expanding access, preparing for potential inclusion in national reimbursement frameworks from early 2027, and discussing technology transfer to potentially enable local manufacturing from 2028.

Nxera Pharma -- New licensing agreement

Post period-end, in January 2026, Santhera entered into an exclusive licensing agreement with Nxera Pharma covering Japan, South Korea, Australia and New Zealand. The agreement, valued at up to USD 215 million plus royalties, includes a USD 40 million upfront payment (comprising USD 30 million in cash and a USD 10 million equity investment), up to USD 175 million in potential milestone payments, and double-digit tiered royalties on net sales.

Under the terms of the agreement, Nxera will be responsible for regulatory approval, including, if required, conducting a registrational bridging study, as well as manufacturing and commercialization in the licensed territories. This licensing partnership significantly expands Santhera's geographic reach into key Asia-Pacific markets while maintaining a capital-efficient operating model.

Distribution agreements and geographic expansion

During 2024, Santhera signed distribution agreements with GENESIS Pharma SA covering 20 Central and Eastern European markets, as well as regional agreements for Israel and Qatar. These partnerships began contributing revenue during 2025 and are expected to accelerate as rollout progresses across additional countries.

Santhera continued to expand its global footprint through further distribution partnerships during 2025, signing new agreements across regions including the Gulf Cooperation Council countries, Türkiye, Russia and India.

These partnerships support rapid expansion into additional territories while leveraging local expertise in market access and commercialization.

Continued generation of long-term clinical data

During the year, Santhera continued to generate long-term clinical evidence supporting AGAMREE's differentiated profile in DMD. In November 2025, the Company reported positive topline results from the ongoing GUARDIAN study, demonstrating sustained efficacy over extended treatment durations, alongside a markedly improved safety profile.

Post period-end, in March 2026, full results were presented at the Muscular Dystrophy Association (MDA) Conference, including data from patients treated for up to eight years.

Importantly, the data demonstrated clinically meaningful safety advantages versus traditional corticosteroids. Patients treated with AGAMREE showed an approximately 80% reduction in vertebral fracture risk (8.1% vs 41.9% with deflazacort), as well as maintenance of normal growth, with a mean height advantage of over 12 cm compared to corticosteroid-treated patients.

Additional findings included a substantially lower incidence of cataracts, no observed cases of glaucoma, and a generally favorable metabolic profile, with no new safety signals identified. Alongside a markedly improved safety profile, the analyses confirmed comparable long-term effectiveness with no statistically significant difference in time to loss of ambulation compared to prednisone and deflazacort.

These results further reinforce AGAMREE's differentiated benefit-risk profile and support its positioning as a foundational therapy for long-term disease management in DMD.

Expanded Manufacturing footprint

In 2025, Santhera, together with its partners, further expanded its manufacturing network to strengthen supply chain resilience, enhance scalability, and materially improve the cost base.

The approval of a new drug product manufacturer for AGAMREE in the EU enabled a fivefold increase in production scale, driving significant cost efficiencies. In parallel, licensing partners have established, or are in the process of establishing, local manufacturing capabilities in their respective territories. This decentralized approach supports capacity expansion, reduces supply risk, and underpins a robust, globally scalable supply infrastructure through close coordination across territories.

Executive and Board changes

Santhera continued to evolve its leadership team and governance structure during the year.

In February 2025, Catherine Isted was appointed Chief Financial Officer, and following the AGM in May 2025, Dr. Melanie Rolli was elected to the Board of Directors.

Post year-end, Marc Clausse was appointed Chief Commercial Officer, effective 1 June 2026, bringing over 25 years of international life sciences experience across specialty, oncology, and rare diseases to lead the next phase of commercial growth.

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April 28, 2026 01:00 ET (05:00 GMT)