Press Release: Santhera Announces Half Year 2025 Financial Results and Provides Corporate Update

Ad hoc announcement pursuant to Art. 53 LR

A conference call will be held on September 23, 2025, at 14:00 CEST / 13:00 BST / 08:00 BST.

Details are at the end of this news release

Pratteln, Switzerland, September 23, 2025 -- Santhera Pharmaceuticals (SIX: SANN) announces the Company's financial results for the six months ended June 30, 2025, reports on progress with AGAMREE(R) (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) and provides updates on its corporate and financing initiatives.

Highlights

-- Total revenue increased 70% to CHF 24 million (H1 2024: CHF 14.1 million)

driven by strong sales growth in launched markets in addition to growing

royalty and product supply revenues

-- Product sales increased 76% to CHF 11.6 million (H1 2024: CHF 6.6

million), led by Germany and Austria with first contributions from the UK

following the Q2 launch

-- Royalties from licensing partners in the U.S. and China of CHF 5.4

million (H1 2024: CHF 0.9 million), already >25% above full year 2024;

further acceleration expected in H2 2025

-- Global sales (including partners) of AGAMREE exceed USD 100 million on a

four consecutive quarter basis, ahead of expectations, triggering a USD

25 million sales milestone payment due, reflected in cost of sales (COS)

-- Operating expenses (excluding non-cash compensation) of CHF 25 million

(H1 2024: CHF 24.1 million), in line with full year CHF 50-55 million

guidance

-- Operating loss of CHF 35.4 million (H1 2024: loss of CHF 17.7 million)

-- Global rollout advancing with new distribution agreements (post

period-end) across five Gulf Cooperation Council (GCC) countries, India,

and Türkiye

-- Cash and cash equivalents of CHF 18.4 million at June 30, 2025 (Dec 31,

2024: CHF 40.9 million); strengthened by approximately CHF 20 million of

growth capital in September 2025

-- Cash-flow break-even guidance maintained for mid-2026

-- Full year 2025 revenue is expected to exceed the previous guidance range

of CHF 65-70 million

Dario Eklund, CEO of Santhera said: "It has been an exciting period for the business, as the global rollout of AGAMREE has continued at pace. Our partner Catalyst is seeing particularly strong demand in the US, with increasing expectations in China through Sperogenix. Sales in Germany and Austria continue to exceed expectations, and the UK is contributing positively following the Q2 launch. We look forward to the second half of the year, with the publication of our GUARDIAN study on the horizon and the rollout of AGAMREE continuing through direct launches in Europe and via distribution and licensing partners globally. All these efforts are aimed at enabling more patients access to this important treatment around the world."

BUSINESS AND CORPORATE UPDATE

(Including post period events)

-- Direct market sales growing strongly with approximately 40% of steroid

using DMD patients in Germany now treated with AGAMREE. Austria becomes

the first country to have in excess of 50% market share for DMD patients

taking corticosteroids

-- Pricing and reimbursement discussions advancing in Spain, Italy and the

Nordic regions, with launches expected from Q4 2025 into Q1 2026

-- US partner Catalyst Pharmaceuticals, Inc. ("Catalyst") posted strong

AGAMREE H1 sales of USD 49.4 million, on track to reach its guidance for

FY 2025 sales of USD 100-110 million, which would trigger an additional

milestone to Santhera of USD 12.5 million

-- Chinese partner Sperogenix Therapeutics Ltd. ("Sperogenix") commences

commercial rollout on a non-reimbursed basis. Forecasted demand in China

for 2025 and 2026 has increased

-- Continued rollout of AGAMREE worldwide with distribution agreements

signed for five Gulf Corporation Council (GCC) countries, India and

Türkiye post period end

-- Catherine Isted joined Santhera as CFO in February and Dr. Melanie Rolli

joined the Santhera Board in May

Successful launches drive own market sales in Europe

Following successful 2024 launches in Germany and Austria, patient numbers continue to grow, with approximately 450-500 patients having initiated AGAMREE and many of these patients having been on the therapy for well over a year. In Germany, use is broadening beyond the initial pediatric cohort as neurologists increasingly (re)start or switch older DMD patients to AGAMREE. Combination therapy with givinostat is also rising in the country. Roughly 40% of corticosteroid-using DMD patients in Germany have been treated to date with AGAMREE. In Austria, strong uptake has continued into 2025, making it the first country where more than half of steroid-using Duchenne patients are being treated with AGAMREE.

In the United Kingdom, after positive final NICE guidance in January 2025, AGAMREE became available nationwide from April. Updated UK guidelines on "Corticosteroids in Duchenne muscular dystrophy" (April 2025) favor daily regimens and recognize AGAMREE as equally effective with fewer side effects than traditional corticosteroids. In mid-Q3, Santhera introduced a home-delivery program to streamline access and reduce administrative burden on centers. Early feedback has been positive and is expected to support ordering momentum into Q4.

Advancing the rollout of AGAMREE across Europe

A key focus of the business is the continued rollout across Europe, and the Company is advancing multiple national reimbursement submissions and pricing negotiations across the continent.

AGAMREE has been available in Spain through a paid, named-patient program since mid-2024, and the Company has been working to secure full approval and reimbursement so all eligible patients can access the medicine. Santhera's proposal is scheduled for discussion at the upcoming October CIMP meeting. Subject to a positive outcome, the launch into regional and hospital formularies is expected to begin in Q4 2025 and to continue into 2026 as regional rollout progresses. In Portugal, an early access plan is about to start, with pricing and reimbursement discussions running in parallel.

Across the Nordics -- Sweden, Denmark, Finland, and Norway -- pricing and reimbursement filings are progressing alongside pre-marketing activities conducted by local teams. First sales are anticipated between Q4 2025 and Q1 2026 as country launches commence.

In Italy, following discussions with the Italian Medicines Agency (AIFA), the Company has decided to update the reimbursement dossier with long-term GUARDIAN study data once available. Approval is anticipated in late Q1 2026, assuming good progress with regulators over the coming quarter. In the meantime, an early access program is being rolled out with the aim of covering the majority of Italian expert centers, supported by strong engagement with key opinion leaders and patient advocacy groups.

Reimbursement discussions continue in the Benelux region, with launches expected during 2026. In France, pricing negotiations with CEPS are ongoing. As previously disclosed, Santhera expects to include additional GUARDIAN data and potentially other supportive evidence in the dossier to achieve the appropriate ASMR rating and broader positioning. The Company will update the market on expected launch timing as discussions progress. In Switzerland, pre-marketing activities have begun, with marketing authorization expected in H1 2026, with fully reimbursed launch targeted for the second half of 2026.

Strong US partner sales with Catalyst continues

Santhera's U.S. licensing partner, Catalyst, continued to report strong growth, delivering AGAMREE sales of USD 49.4 million in the first half of 2025, driven by accelerated physician adoption. This strong and sustained uptake underscores AGAMREE's positioning as a new standard of care for DMD. Based on current projections, Catalyst has guided USD 100-110 million in 2025 sales. Upon achieving at least USD 100 million, Santhera would receive a USD 12.5 million milestone. The robust U.S. performance is also increasing Santhera's inventory requirements and contributing to higher supply sales and royalties.

Sperogenix non-reimbursed commercial rollout commences

In China, partner Sperogenix commenced a non-reimbursed commercial rollout of AGAMREE in September 2025, in addition to the earlier started Early Access Program. Uptake has been encouraging, with more than 250 patients treated to date. Priorities for the second half of 2025 are to expand sales in the non-reimbursed market. In anticipation of increased demand in 2025-2026, Santhera is bringing forward inventory plans to provide product to this market.

Geographical expansion continues successfully

Across additional territories, Santhera is broadening access through distribution partnerships. In 2024, the Company signed an agreement with GENESIS Pharma SA covering 20 Central and Eastern European markets and established regional distribution agreements for Israel and Qatar. During 2025, Santhera signed further agreements in five Gulf Cooperation Council countries, Türkiye, and India, and continues to engage in various discussions to expand its global footprint and broaden patient access to AGAMREE.

New AGAMREE data to be presented in early Q4 2025

Santhera continues to generate additional long-term clinical outcomes in DMD patients with up to seven years on AGAMREE and looks forward to reporting long-term effectiveness and safety results from the GUARDIAN study in early Q4 2025. In parallel, real-world evidence abstracts from both younger and older patient cohorts in Germany -- where AGAMREE has been available the longest -- have been submitted for presentation at the World Muscle Society Congress in Vienna, October 7--11, 2025.

Pipeline development and business development initiatives

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