SANTHERA PHARMACEUTICALS HOLDING AG modifies its downstream activity. This novel mode of action is believed to dissociate efficacy from typical steroid safety concerns [2-5]. In the currently completed studies, a total of 48 patients have received various doses of vamorolone; of which 41 patients have been treated and evaluated for a period of 2.5 years. Aggregate clinical data from these open label studies in DMD published to date showed sustained efficacy and clinical improvement with vamorolone across multiple endpoints coupled with a reduction of certain corticoid-specific side effects [6]. The Company believes vamorolone has the potential to become a foundational therapy in DMD for patients irrespective of the underlying gene mutation and a promising alternative to existing corticosteroids, the current standard of care in children and adolescent patients with DMD. In September 2020, Santhera secured full worldwide rights for vamorolone in all indications through agreements with Idorsia and ReveraGen, and assumed overall responsibility for the development program towards approval and commercialization. Based on the encouraging data available from previous studies, the Company is confident that the ongoing fully recruited pivotal Phase 2b VISION-DMD study [7-9], which enrolled 121 patients and compares vamorolone to a corticosteroid and placebo, will confirm previous efficacy and safety findings. A defined regulatory path for vamorolone has been pre-agreed in discussions with the US Food and Drug Administration (FDA) and on this basis Santhera is hopeful that a positive topline readout expected in Q2-2021 will meet the clinical requirements for a US new drug application (NDA) and approval. Vamorolone has been granted fast track status and rare pediatric disease designation, a requirement for a Priority Review Voucher, with the FDA. The assembly of the technical module of the NDA (Common Technical Document, CTD) will require additional technical work to be performed by Santhera since bioequivalence between the licensors' formulation used in clinical trials and the formulation intended for upscaling and commercialization has not yet been demonstrated. This involves validation work with the existing clinical formulation to enable initial commercialization and is expected to result in a delay of NDA submission by approximately one quarter to Q1-2022 but, at the same time, will also allow for inclusion of 12-month data from the VISION-DMD trial for which a topline readout is expected in Q4-2021. In parallel, Santhera is initiating development of a next-generation follow-on formulation aiming at an improved scalability, cost reduction and potential additional intellectual property. In Europe, discussions with the European Medicines Agency (EMA) indicated that the regulators will accept positive 12-month data from the VISION-DMD trial as a basis for a regulatory filing anticipated by mid-2022. Further pipeline update and business development opportunities Aside from vamorolone, Santhera continues to advance its clinical-stage candidate lonodelestat, a potent and selective peptide inhibitor of human neutrophil elastase (hNE), developed in cystic fibrosis. Data readout of the double-blind, placebo-controlled dose escalation Phase 1b study in patients with cystic fibrosis, to investigate safety, tolerability, pharmacokinetics and pharmacodynamics of orally inhaled multiple doses of lonodelestat for up to four weeks, is expected in the coming weeks (clinicaltrials.gov identifier: NCT03748199). Based on data from this early clinical proof of concept trial, the Company plans to design the further clinical development program. Available data from previous studies already demonstrated that single dose inhalation of lonodelestat can lead to high drug concentrations within the lung, resulting in inhibition of hNE in sputum of patients, and more results from this important biomarker are expected in the Phase 1b study. Neutrophil elastase is an enzyme associated with lung tissue inflammation, leading to degradation of the lung tissue in cystic fibrosis and several other pulmonary diseases [10]. With regard to the Company's proactive portfolio management strategy as an additional source of future non-dilutive income streams, the clarity achieved in the ownership structure of vamorolone lends itself to increased partnering opportunities for further expansion across different geographies (outside USA and the EU) and additional non-DMD indications for the benefit of patients worldwide. Similarly, the Company is currently also seeking opportunities for outlicensing agreements for lonodelestat in pulmonary indications beyond cystic fibrosis. Note The Invitation to the Bondholders' Meeting can be viewed under https://www.globenewswire.com/Tracker?data=GY95UblAPTRBZAkCUHgO-bQ7DYgolSyuaXB1caGnkD3RHG4ueTHAHpZ83u3_mR1wbRSnbD9PlByG2wte6SHglamunfxroz5W1Ii-o2tT2dlxrfhxVkweJNM9UElG2IVvqZN9SL-Y59p0ZTPw04M0q2wheIzBOlAS8dR86bZATVNwH_P3D65HgVK8i8MqSKgAyWduhP_C-1sj8HDF2pVcx_pQh-qrbVVfo9ypiE0lJXHH65Uwc4GmFexlLA2bdHiZ www.santhera.com/investors-and-media/investor-toolbox/share-bondholder-meetings from February 17, 2021, 07:00 hrs. The Financial Status with preliminary unaudited financial figures as of December 31, 2020, has been prepared exclusively in view of the upcoming bondholders' meeting of Santhera Pharmaceuticals Holding AG and can be accessed under https://www.globenewswire.com/Tracker?data=GY95UblAPTRBZAkCUHgO-bQ7DYgolSyuaXB1caGnkD3RHG4ueTHAHpZ83u3_mR1wbRSnbD9PlByG2wte6SHglamunfxroz5W1Ii-o2tT2dmFXJB0KXCitwBOxHv8BWWCS1rdRK4F0uuklxFuhOkBRzYYNL-bvjrYNhEeZH1IQGwFeGspHU5diexCCiV5P6v_-nX0fFO-QOMX-pnYI8-7jVc_T4RdK7Pz-QuR-38lpRZLj95AzTi0BI6QQW_40ReE www.santhera.com/investors-and-media/investor-toolbox/share-bondholder-meetings. The Company expects to publish its 2020 Annual Report, with audited financial statements, on April 27, 2021. Corporate calendar February 17, 2021 Publication of invitation to Bondholder Meeting March 8, 2021 Bondholder Meeting Mid to end March, 2021 Extraordinary General Meeting April 27, 2021 Publication of annual results and Annual Report 2020 June 22, 2021 Annual General Meeting References: [1] Key bond data, https://www.globenewswire.com/Tracker?data=3ytQIthssLXkhq4q9N5jTgmFMUOFU3s_9XRZsuA-tSG0xJxg82s4u8HdD97OnedI2r2aQEfMDfkTXYRyj9jb8B2Vs6H1D3zR5ri98kR4DIoQB-EFM52PAYdDFdWkNpOX Link [2] Heier CR at al. (2013). EMBO Mol Med 5: 1569--1585. [3] Reeves EKM, et al (2013). Bioorg Med Chem 21(8):2241-2249 [4] Heier CR et al. (2019). Life Science Alliance DOI 10.26508/lsa.201800186. [5] Liu X et al. (2020). Proc Natl Acad Sci USA. https://www.globenewswire.com/Tracker?data=3ytQIthssLXkhq4q9N5jTrOrj_dn_Q5raahsuh_crCgaF_NhBsTeRzLJVNbeJR7LDfvgASPKPo27KVCh7JtRA-I3TzZtUMFdqdq9TULc234= Link [6] Smith E, et al. (2020). PLOS Medicine, https://www.globenewswire.com/Tracker?data=3ytQIthssLXkhq4q9N5jTgUiTOo8TijyJRiPJ0vDwY0NQr5aPwbVeKzCMRzdYS7IgC0M9psmtq_TSXffkqm0x6GRg4de3kZGTqCsh-Dxs54= Link [7] Clinicaltrials.gov. https://www.globenewswire.com/Tracker?data=3ytQIthssLXkhq4q9N5jTjpsnL2eBaiEvSc9nRJ5qjQUjFwGmEL5nHrRaIEdhaOredAY8lC2JkCAlZ31_zmynbfj83ReV6se1nPBhKlQ0FM= Link [8] Santhera press release, September 11, 2020, https://www.globenewswire.com/Tracker?data=3ytQIthssLXkhq4q9N5jTvrqwqtwVarSzpQlIVP0b9LGZdwdcsOkGNfqmruCVYBK6kX_oSkpwbpYCt1-j9LPOjqBcXSHp7xKAJFSKLCOR2JapABAe2XBa1hdVbYAr8RkWIa6kxkDZUTwXAnOipnNff1ngwK1qix37RI8TGHxqWFzGdrBD3u5nrjetSCpZ8sc Link [9] VBP15-004, https://www.globenewswire.com/Tracker?data=BAW5k95W23I1vY5Ae8hX1QaEn26Uo5nFOQ35VrIHH_H9FACWhgDaspwt8mhEtjZEMBp07wSNoL_AC8PAvZSt_zWES73kBSnEgA59uCLVv5kYJhxE5gtdR0Es9gJUqaX6Hf_ZpPBX-MKDkp8EsEoXTMXW_mgFoQoHI-_4qcGnr98= https://vision-dmd.info/2b-trial-information [10] Barth P. et al (2019). Journal of cystic fibrosis, https://www.globenewswire.com/Tracker?data=3ytQIthssLXkhq4q9N5jTkOQvN_jsW-Mc7-gg1YomhmTJCbypWXzZtR7-xAr1b2we-YhnYcUvlp5Pt7v0IQOnpuwMTfKdPb3JjE8rie64nA= Link About Santhera Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical need. Santhera has an exclusive license for all indications worldwide to vamorolone, a first-in-class dissociative steroid with novel mode of action, currently investigated in a pivotal study in patients with DMD as an alternative to standard corticosteroids. The clinical stage pipeline also includes lonodelestat (POL6014) to treat cystic fibrosis (CF) and other neutrophilic pulmonary diseases as well as an exploratory gene therapy approach targeting congenital muscular dystrophies. Santhera out-licensed ex-North American rights to its first approved product, Raxone(R) (idebenone), for the treatment of Leber's hereditary optic neuropathy (LHON) to Chiesi Group. For further information, please visit https://www.globenewswire.com/Tracker?data=GY95UblAPTRBZAkCUHgO-eMYFeXE7QfTCQVfg2DZDAvSWUSMJK_dV6o-VUhZwLaTG6KwwyUvXo-wQSLjm35AR3W2nK9heUUJn9abouwRk0c= www.santhera.com. Raxone(R) is a trademark of Santhera Pharmaceuticals. For further information please contact: https://www.globenewswire.com/Tracker?data=k7-iwtOnV_c4KrlpvlxZ9Gx053adGfny3GVUeGkrNrUW_4LqSmgDElxKkNHvtFzKQABqFa3q1zSqegSmebt9ox2a-9NR1xMHy5TgceCP8LGxiHHNYRjIZPVNm-K9gHPN public-relations@santhera.com or Eva Kalias, Head External Communications Phone: +41 79 875 27 80 eva.kalias@santhera.com Disclaimer / Forward-looking statements This publication is not intended to constitute an offer or solicitation to purchase or invest in securities of Santhera Pharmaceuticals Holding AG in any jurisdiction. The securities referred to in this publication, including in connection
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