Press Release: Santhera Implements Reorganization -2-

glucocorticoids is limited due to detrimental side-effects.

Vamorolone has been granted Orphan Drug status in the US and in Europe,

has received Fast Track and Rare Pediatric Disease designations by the

US FDA and obtained Promising Innovative Medicine (PIM) status from the

UK MHRA.

References:

[1] Heier CR at al. (2013). VBP15, a novel anti--inflammatory and

membrane--stabilizer, improves muscular dystrophy without side effects.

EMBO Mol Med 5: 1569--1585.

[2] Reeves EKM, et al (2013) VBP15: preclinical characterization of a

novel anti-inflammatory delta 9,11 steroid. Bioorg Med Chem

21(8):2241-2249

[3] Heier CR et al. (2019). Vamorolone targets dual nuclear receptors

to treat inflammation and dystrophic cardiomyopathy. Life Science

Alliance DOI 10.26508/lsa.201800186.

[4] Liu X et al. (2020). Disruption of a key ligand-H-bond network

drives dissociative properties in vamorolone for Duchenne muscular

dystrophy treatment. Proc Natl Acad Sci USA. Link

https://www.globenewswire.com/Tracker?data=c0anYhdf5ZgBXRzmDmb3ywh3nIrKIOe98u3z7SGTiNWp7XqQKiDg_Eeqs6WtX36BPBJCODeirxxEU1S70Gl98LqDoPDP3fRAEyTWJDk1k2o=

[5] ClinicalTrials.gov Identifier: NCT02760277, Link

https://www.globenewswire.com/Tracker?data=c0anYhdf5ZgBXRzmDmb3y-jVGTr4svWTgvktxliJohQ9tQ243BXtJPqsjaNK-TZmT_w2U6GoA9QhCnb3tCoBqraF3dg3-GRTx4Nca3RN4sk=

[6] Hoffman EP et al. (2019). Vamorolone trial in Duchenne muscular

dystrophy shows dose-related improvement of muscle function. Neurology

93: e1312-e1323.

[7] ClinicalTrials.gov Identifier: NCT03038399, Link

https://www.globenewswire.com/Tracker?data=c0anYhdf5ZgBXRzmDmb3y2LASoRTXixf60CbQjhQWEsBaDEsY1x1zNGtneI32l_A8W9vquXJsLIpGM6_GFBb7-FdTzgRJJ-MVaQjglxUgvw=

[8] Smith E, et al. (2020). Efficacy and safety of vamorolone in

Duchenne muscular dystrophy: an 18-month interim analysis of a

non-randomized open-label extension study. PLOS Medicine, Link

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[9] ClinicalTrials.gov Identifier: NCT03439670, Link

https://www.globenewswire.com/Tracker?data=c0anYhdf5ZgBXRzmDmb3yw9zGIP7CkzjLSoXCr9Sq7QYjVg_hpggFgQdi0BTDgh1OKHGCoj0ZKLlrCn4UwazuPQxwN-bTna66IhwPrFg_hZXGDO3ZnkPXPBRisVeffPh0BKoPsq_8wzLPKhrzQWP8g==

[10] ReveraGen website, Link

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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=rLFY4D0Ozdr6Axs0F421GbuuO1ugQrjAJiKfcN70045Aov-S_EbfcdWdkWMPslDuvnjiwGWoT98-6t2iCLFupQ==

www.santhera.com.

Raxone(R) is a trademark of Santhera Pharmaceuticals.

For further information please contact:

https://www.globenewswire.com/Tracker?data=MYO8-KAksbHH4jzusHcMkqoCNa1uuiF-3TttxcTt_EV37DOaWi86EkO9ptXU7DgRLRjpI8vp-Ed6EcqjHGsde-sB87eC5VUxAGENAVE2aLWvQcAZEtDosR9O16sLUtmR

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 communication does not constitute an offer or invitation to

subscribe for or purchase any securities of Santhera Pharmaceuticals

Holding AG. This publication may contain certain forward-looking

statements concerning the Company and its business. Such statements

involve certain risks, uncertainties and other factors which could cause

the actual results, financial condition, performance or achievements of

the Company to be materially different from those expressed or implied

by such statements. Readers should therefore not place undue reliance on

these statements, particularly not in connection with any contract or

investment decision. The Company disclaims any obligation to update

these forward-looking statements.

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(END) Dow Jones Newswires

November 02, 2020 01:00 ET (06:00 GMT)