Santhera Pharmaceuticals : Delivered Solid Performance in 2016 and Advances All Programs

Hammerstrasse 49

4410 Liestal / Switzerland Phone +41 61 906 89 50

Fax +41 61 906 89 51

www.santhera.com

"In the past year Santhera made excellent progress in all programs and 2016 marked our first full year as a commercial company," commented Thomas Meier, PhD, Chief Executive Officer of Santhera. "We are equally excited about Santhera's future. With our expanded geographic reach in Europe and the US, additional clinical trials and regulatory filings, we paved the way for further growth and value generation. Our strong financial position allows us to aggressively pursue our business strategy, to advance our products and establish Santhera as a leader in mitochondrial medicine."

Net sales grew to CHF 19.0 million (2015: CHF 4.3 million) driven by strong sales of the lead prod- uct Raxone® for the treatment of Leber's hereditary optic neuropathy (LHON) in the EU.

Cash-flow from operating activities amounted to CHF -27.1 million (2015: CHF -22.4 million). As of December 31, 2016, Santhera had cash and cash equivalents of CHF 49.8 million (2015: CHF 76.9 million). In February 2017, after the balance sheet date, Santhera successfully placed CHF 60 mil- lion senior unsecured convertible bonds due 2022, resulting in a cash position by end of February 2017 of CHF 100.8 million. These cash reserves provide the financial flexibility to support the Com- pany's, development, regulatory and commercial projects as planned.

2016 was marked by a strong internationalization of Santhera's business activities. In parallel to the commercial roll-out of Raxone, Santhera expanded its operations across Europe and, by the end of 2016, sold Raxone into 15 countries. In February 2017, Santhera established presence in the United States with its US-subsidiary Santhera Pharmaceuticals (USA), Inc. in the Boston metropoli- tan area, one of the main centers for pharmaceutical companies in North America. The US team currently intensifies relations to patient advocacy groups, prepares market access and provides regulatory and medical affairs expertise.

In 2016, Santhera submitted Marketing Authorization Applications (MAA) for Raxone for the treat- ment of Duchenne muscular dystrophy (DMD) in the EU and Switzerland and significantly advanced all product development programs. Santhera also started the randomized, double-blind, placebo- controlled phase III trial (SIDEROS) to assess the efficacy of Raxone in delaying the loss of respira- tory function in DMD patients receiving concomitant glucocorticoid therapy. Details on progress made with Raxone and the development programs in 2016 were previously announced (press release of January 26, 2017) and are described in the Annual Report 2016.

For the Company's Annual Shareholders' Meeting on April 4, 2017, the Board proposes Philipp Gutzwiller, Elmar Schnee, Patrick Vink, MD, and Thomas Meier, PhD, CEO of Santhera, for election as new members of the Board of Directors. Subject to his election, Thomas Meier will also be ap- pointed Delegate of the Board. With these nominees, the Board will gain additional experienced senior executives to strengthen the strategic expertise in global pharmaceutical business growth. Jürg Ambühl, Board Member of Santhera since 2009, has decided not to stand for re-election. The Board and management thank Mr. Ambühl for his many valuable contributions and his strong com- mitment to the Company during these decisive years.

Santhera will continue to grow its international business. The Company expects 2017 net sales of Raxone in the currently approved indication alone to reach CHF 21 to 23 million. Besides focusing on commercialization and reimbursement of Raxone in LHON in Europe, additional priorities in 2017 will entail preparations for market entry and launch for Raxone in the second indication DMD, and advancement of all currently ongoing clinical trials.

Santhera 2016 Annual Report see www.santhera.com/investors-and-media/investor-toolbox/financial-reports.

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The Santhera Annual Report 2016 is available for download on the Company's website at www.santhera.com/investors-and-media/investor-toolbox/financial-reports.

The Annual Shareholders' Meeting of Santhera will be held on April 4, 2017, in Basel, Switzerland (shareholders will receive a separate invitation).

Santhera Pharmaceuticals (SIX: SANN) is a Swiss specialty pharmaceutical company focused on the development and commercialization of innovative pharmaceutical products for the treatment of orphan mitochondrial and neuromuscular diseases. Santhera's lead product Raxone is authorized in the European Union, Norway, Iceland and Liechtenstein for the treatment of Leber's hereditary optic neuropathy (LHON). For Duchenne muscular dystrophy (DMD), the second indication for Raxone, Santhera has filed a Marketing Authorization Application (MAA) in the European Union and Switzer- land. In collaboration with the US National Institute of Neurological Disorders and Stroke (NINDS) Santhera is developing Raxone in a third indication, primary progressive multiple sclerosis (PPMS), and omigapil for congenital muscular dystrophy (CMD), all areas of high unmet medical need. For further information, please visit the Company's website www.santhera.com.

Raxone® is a trademark of Santhera Pharmaceuticals.

Thomas Meier, PhD, Chief Executive Officer Christoph Rentsch, Chief Financial Officer Phone +41 61 906 89 64 Phone +41 61 906 89 65

thomas.meier@santhera.com christoph.rentsch@santhera.com

hans@lifesciadvisors.com jgillespie@rxmedyn.com

This communication does not constitute an offer or invitation to subscribe for or purchase any secu- rities of Santhera Pharmaceuticals Holding AG. This publication may contain certain forward-looking statements concerning the Company and its business. Such statements involve certain risks, uncer- tainties 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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