Ad hoc announcement pursuant to Art. 53 LR

A conference call will be held on April 25, 2024, at 14:30 CEST / 13:30 BST / 08:30 EDT. Details are at the end of this news release.

Santhera Announces Preliminary Unaudited 2023 Annual Results and Provides Corporate Update Ahead of Full Report Publication in May

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-- 2023 financial key figures:        Revenue from contracts with customers 
of CHF 103.4 million (2022: CHF 7.5 million)        Net result of 
CHF 54.8 million (2022: CHF -71.1 million)         Cash flow from 

operating activities of CHF 47.6 million (2022: CHF -29.8 million)

-- Regulatory approvals in key territories -- AGAMREE(R) (vamorolone)
approved for Duchenne muscular dystrophy (DMD) treatment in the U.S., EU
and UK, showcasing safety benefits over traditional corticosteroids

-- Strategic partnership -- licensing agreement signed with Catalyst
Pharmaceuticals Inc. (NASDAQ: CPRX) for North America, focusing on
commercialization of AGAMREE in DMD and exploring joint development of
indications beyond DMD

-- First launches -- first launch in Germany, followed by U.S. launch by
Catalyst, plans for gradual roll-out in larger European countries by
Santhera and partnering for other territories

-- Cash reach into 2025 -- significant financial gains from Catalyst deal,
divestment of RAXONE business, and product sales, overall strengthening
the balance sheet and securing financing of operations (excluding
maturing convertible bonds)

-- First quarter 2024 -- positive uptake with approx. 150 patients treated
with AGAMREE in Germany and Austria; revenue of CHF 4.7 million, cash on
March 31, 2024 of CHF 26.8 million

-- Outlook -- expected peak annual sales over EUR 150 million in Europe for
AGAMREE in DMD alone, with additional revenue from partnerships, aiming
for financial breakeven by mid-2026
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Pratteln, Switzerland, April 25, 2024 -- Santhera Pharmaceuticals (SIX: SANN) announces the Company's preliminary unaudited financial results for the year ended December 31, 2023, reports on business progress achieved in 2023 and extending into 2024, and provides updates on its strategic and financing initiatives. As permitted by SIX Exchange Regulation, the Company will publish the full 2023 Annual Report in May.

The last 15 months were a pivotal phase for Santhera, characterized by remarkable successes and important milestones. We have successfully navigated the regulatory landscapes and secured approvals for AGAMREE in key territories and across continents within just three months. Our collaboration with Catalyst Pharmaceuticals has strategically positioned us to enable product availability to patients in North America as well as to explore strategy of joint development of AGAMREE in indications beyond DMD. In addition, by divesting non-core assets and focusing on AGAMREE, we have streamlined our operations and strengthened our financial base," said Dario Eklund, CEO of Santhera. "Looking ahead, we are poised to continue this momentum, driving forward our mission to improve care for the DMD community and enhance the quality of life for patients. I am incredibly proud of our team's dedication and innovative spirit and am optimistic about the future as we move toward profitability and expand AGAMREE's therapeutic reach."

In 2023 and into 2024, Santhera achieved critical milestones, securing regulatory approvals for AGAMREE in DMD across the U.S., EU and UK, and submitting the product for approval in China. Strategic moves included licensing AGAMREE to Catalyst Pharmaceuticals in North America and focusing operations on Europe, culminating in AGAMREE's first global market launch in Germany in January 2024. Financially, Santhera reported a 2023 revenue of CHF 103.4 million and net income of CHF 54.8 million, driven by the Catalyst licensing deal. With CHF 30.4 million in cash reserves at year-end, and anticipated revenue from product sales, this is expected to support operations into 2025. Additional funding will be necessary to meet debt obligations (maturing convertible bonds) and support further market launches, aiming for cash breakeven by the first half of 2026.

REVIEW OF PIPELINE AND BUSINESS PROGRESS

2023 key events and post-period updates

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-- AGAMREE approved in the U.S., EU and the UK for the treatment of DMD

-- European regulators acknowledged safety benefits of AGAMREE with regards
to preserving bone health and maintaining growth compared to standard of
care corticosteroids

-- China's regulatory authority accepted and granted priority review for
vamorolone NDA in DMD

-- Exclusive North America license for AGAMREE granted to Catalyst in deal
valued at up to USD 231 Million plus royalties

-- AGAMREE launched in Germany as first market for the treatment of DMD,
with encouraging early uptake

-- Santhera's partner Catalyst launched AGAMREE in the U.S. in Q1-2024

-- Divestment of RAXONE/idebenone business to Chiesi Group completed

-- Focusing on AGAMREE and following portfolio review, lonodelestat license
terminated by Santhera and asset to be returned to Spexis
===
AGAMREE (vamorolone) approved across the U.S., EU and UK

AGAMREE was approved by the U.S. FDA (on October 26, 2023), the European Medicines Agency (EMA) in the EU (on December 18, 2023) and the Medicines and Healthcare products regulatory agency (MHRA) in the UK (on January 11, 2024). Thereby, it became the first DMD treatment approved across these three territories. In the EU, AGAMREE is the first and only approved medication for treating all patients from age 4 years with DMD.

The EMA and the MHRA acknowledged clinically important safety benefits of AGAMREE with regards to maintaining normal bone metabolism, density and growth compared to standard of care corticosteroids, while demonstrating similar efficacy. In March 2024, the Center for Drug Evaluation (CDE) of China's National Medical Products Administration (NMPA) accepted the NDA for vamorolone, granting priority review. Subject to a positive outcome, this could lead to approval by Q1-2025.

North America license for AGAMREE granted to Catalyst Pharmaceuticals

In June 2023, Santhera announced the signing of an exclusive license and collaboration agreement for AGAMREE in North America (NA) with Catalyst, a commercial-stage biopharmaceutical company focused on novel medicines for patients living with rare diseases. The agreement covers the development and commercialization of AGAMREE in DMD and rights to all potential future indications in NA. Total consideration to Santhera is up to USD 231 million (including equity investment) plus royalty payments from product sales.

After closing of the transaction in July 2023, Santhera received an upfront payment of USD 90 million (USD 75 million in cash and USD 15 million equity investment). Upon U.S. FDA approval of AGAMREE in DMD on October 26, 2023, Santhera received an additional USD 36 million from Catalyst, of which Santhera paid contractually agreed third-party regulatory milestone obligations (USD 26 million). Furthermore, Catalyst may pay Santhera sales-based milestones of up to USD 105 million as well as up to low-teen percentage royalties and will assume corresponding third-party royalty obligations of Santhera on AGAMREE sales in all indications in NA.

In March 2024, following the U.S. FDA approval on October 26, 2023, Catalyst announced that AGAMREE is now available by prescription and dispensed throughout the United States through a specialty pharmacy network.

Santhera and Catalyst have made considerable progress to define the strategic framework of a joint clinical development program and shared funding of AGAMREE for global indications, in addition to DMD.

First launch in Germany in early 2024--pre-commercialization measures advancing across Europe

On January 15, 2024, Santhera launched AGAMREE as a 40 mg/ml oral suspension for the treatment of DMD in Germany as the first market worldwide. Since February 2024, AGAMREE is also available in Austria. This significant milestone represented Santhera's commitment to fill a high unmet medical need by providing a safe and effective treatment for DMD patients. For Santhera, this launch signified a leap forward as the Company entered the commercial stage in the DMD space.

Santhera plans to make AGAMREE available to patients in additional key geographies in Europe (France, UK, Italy, Spain, Benelux and Switzerland), and is in the late stages of negotiations with distribution partners for commercialization in other European countries.

Activities surrounding market access, stakeholder and key opinion leader engagement in the target countries progressed throughout the period under review. After Germany, the build-up of a core commercial organization is well underway in the UK, France, Italy and the Benelux countries.

Full divestment of RAXONE/idebenone business to Chiesi Group

In a transaction closed on July 28, 2023, Chiesi Group acquired all assets and certain liabilities related to idebenone in all indications worldwide. This included RAXONE in Leber hereditary optic neuropathy (LHON), for which Chiesi already held exclusive license rights globally since 2019, except for North America and France. Under the terms of the agreement, Chiesi Group assumed the responsibility for the settlement agreed between Santhera and the French reimbursement authorities relating to RAXONE in LHON amounting to EUR 25.3 million. The transaction significantly reduced debt and strengthened Santhera's balance sheet. Furthermore, the cessation of RAXONE-related activities allowed Santhera to streamline business processes, reducing operating costs and freeing up resources for AGAMREE and its European launch.

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