Press Release: Santhera Announces Half-Year 2022 Financial Results and Provides Corporate Update

Ad hoc announcement pursuant to Art. 53 LR

A conference call will be held on November 7, 2022, at 14:30 CET, 13:30 GMT, 08:30 EST. Details are at the end of this news release.

-- Revenue from contracts with customers of CHF 6.3 million (H1-2021:

CHF 4.5 million)

-- Operating result of CHF -25.5 million (H1-2021: CHF -19.5 million) and

net result of CHF -29.7 million (H1-2021: CHF -20.5 million)

-- Cash and cash equivalents of CHF 12.7 million (June 30, 2022), together

with existing facilities enabling cash reach into Q1-2023

-- Key milestones reached with U.S. and EU regulatory submissions (NDA, MAA)

for vamorolone in Duchenne muscular dystrophy (DMD)

-- Financing initiatives ongoing to support vamorolone launch and other

ongoing activities

Pratteln, Switzerland, October 31, 2022 -- Santhera Pharmaceuticals (SIX: SANN) announces the Company's financial results for the six months ended June 30, 2022, reports on the regulatory and clinical progress with its lead drug candidate vamorolone for the treatment of DMD in the U.S. and Europe, and provides updates on its financing initiatives.

"The year 2022 to date was fully geared towards registration and approval of vamorolone in Duchenne muscular dystrophy (DMD) and I am delighted that, over the last month, we submitted and received validation for a marketing authorization application (MAA) in the EU followed by completion of the rolling submission of a new drug application (NDA) in the U.S. This represents a tremendous achievement for Santhera and a major step towards our goal of bringing this investigational therapy to patients living with DMD," said Dario Eklund, CEO of Santhera. "With equally high priority, we are pursuing additional near-term financing, primarily to allow us to fund market entry preparations for vamorolone. We are evaluating various non-dilutive options including but not limited to licensing agreements and monetization of assets in addition to debt and royalty financing and, depending on market conditions, may also consider equity-based funding options."

PIPELINE MILESTONES AND PROGRESS REVIEW

Half-year key events and post-period updates

-- New drug application (NDA) submitted to the U.S. Food and Drug

Administration (FDA) for vamorolone in DMD

-- Marketing authorization application (MAA) submitted to and validated by

the European Medicines Agency (EMA) for vamorolone in DMD

-- Efficacy, safety and bone health data with vamorolone published in JAMA

Neurology and presented at scientific conferences

-- Activities advanced to establish launch readiness for vamorolone in the

U.S.

-- Exclusive license agreement concluded with Sperogenix for vamorolone in

rare diseases in the Greater China Region

-- Phase 2 trial started with vamorolone in boys aged 2 to <4 years and 7 to

<18 years with DMD to assess drug effects in wider age range

-- First participant dosed in FDA-funded study with vamorolone in Becker

muscular dystrophy

-- Lonodelestat development program deprioritized owing to limited resources

The primary operational focus of Santhera in 2022 continues to be the advancement of the regulatory submissions for vamorolone in the U.S. and the EU towards approval. In parallel, the Company plans to advance operational preparations for launches anticipated for 2023 in both regions.

NDA submission to the U.S. FDA for vamorolone in DMD completed

In October, Santhera completed the rolling submission of an NDA to the U.S. FDA, seeking priority review for vamorolone for the treatment of DMD. Typically, within 60 days of the receipt of the dossier, the FDA will inform if a priority review will be granted. A priority review designation indicates FDA's goal to take action on an application within six months (compared to ten months under standard review) which would set an anticipated approval date for as early as mid-2023. Subject to approval, vamorolone is set to become available to patients in the U.S. in H2-2023.

European MAA for vamorolone in DMD submitted -- review by the EMA has started

In September, Santhera submitted an MAA for vamorolone for the treatment of DMD to the EMA. Validation, received in October, confirms that the submission is complete and that the review by the EMA's Committee for Medicinal Products for Human Use (CHMP) has begun. Santhera expects the CHMP to complete the review and issue an opinion regarding approval to EMA's European Commission (EC) in late Q3-2023. Subject to EC approval later in 2023, vamorolone will receive marketing authorization in all member states of the European Union, as well as in Norway, Liechtenstein and Iceland.

Findings on bone health published for vamorolone alongside efficacy and safety data

Vamorolone is under joint development by ReveraGen and Santhera for DMD patients with the objective to provide an anti-inflammatory and muscle preserving treatment with a favorable safety and tolerability profile as an alternative to the current standard of care. In addition to long-term efficacy and safety data with vamorolone, recent publications and presentations further characterized vamorolone's differentiated profile especially with regard to bone health [1-5].

In July 2022, data assessing the impact of long-term treatment with vamorolone on bone health were presented at the https://www.globenewswire.com/Tracker?data=t6e7C6l09ZmwiQauyiJDP-NldtE3fhG2b36SgJJt56vVX2s9tMffJKHa5HfG4oN_j5J71PW5RII_y0ZiRcsP5dg1CZGlCt93u4NCBMN_8FlEZ5FbEqrXojlVRdaRnXkcdyx1-1q6ktc2j5NUxYhyq8vcxSRfBSDpj7N0q4CI4jxaUTspJ0b0p9idgS0pmPLV3l20_L3Ew-IaC0mdQWD1hw== 10th International Conference on Children's Bone Health [1]. After 2.5 years of treatment with vamorolone, bone turnover markers were not suppressed, bone age delay was minimal, and the vertebral fracture burden was lower compared with published data on daily prednisolone. Efficacy data showed that vamorolone can maintain muscle function in boys with DMD, similar to standard of care glucocorticoid treatment.

In August 2022, https://www.globenewswire.com/Tracker?data=H36itLp7nO-6kuBg-3zJKnEnltPj47IJFXtgUA3LDalBoFgEIPA_Wk5bD161UBAioPB-jO76R8zS91j-CuoOzmzEUU5lYFDyEQH5gd13VUI8gVu1J4Z1zVekxJVvSaLiMjEd5_f9_sfHBaDzyNETCIU458FWk7XcpNTCBVG7vrZD2-yrHEErv-4V0K_txZzHiqtv8Vjj3xQJMInp-6CcwGUqxvdHNsG8_wNTDyOfYTUxlkT6KFfkZhECBfwS6b8ECmUGdDk0Az3ipadfJq87-XYnliIDrrH1595v_ZZe8Zs= JAMA Neurology published the positive 24-week results from the pivotal VISION-DMD study evaluating vamorolone in patients with DMD compared to placebo and prednisone [2]. Vamorolone met its primary endpoint by demonstrating statistically significant and clinically relevant improvement in time to stand from floor compared to placebo and showed consistent results across multiple secondary endpoints. The relative efficacy of vamorolone 6 mg/kg/day was comparable to that seen with prednisone 0.75 mg/kg/day across primary and secondary efficacy endpoints. Importantly, no negative impact on biomarkers of bone health and no loss of linear growth were observed with vamorolone. Patients treated with prednisone experienced reductions in serum biomarkers of bone formation, which promptly recovered to baseline values when subjects were switched from prednisone to vamorolone.

In October 2022, key opinion leaders further highlighted the bone-related profile of vamorolone in different presentations at the https://www.globenewswire.com/Tracker?data=S2pPzZ9kmxPlrmeKB0ROZg15ygjuc_gq3mEvNK5ZWyWmHmVGgeCOuY5r0BLrEn3JfJPU13OAp48iGTFHpGeKjtLubaH4re8NuylFjnVH3yquHtPncylpVQTNgRTLifLD0gcJKTfidM3Ysc1xN6LbKO72gJ72YFniQVM18z3ykINEizHmHjU1vXCRQFtyJhvo7gP93BTpiY-CiabLHI5ITTyOYv1YE7ADikokABqOTOk= World Muscular Society Congress 2022 which can be viewed on Santhera's website here https://www.globenewswire.com/Tracker?data=yF8yk-PWXlWEwnSHHTxqwlxXxLuGnwPdUKF7USKxWB4YIkvqpdUU-3CttPXqwalHcqY2-MvdSfgdvbuFwnHAMFYY-YtD1vH4zdQ-zRgsfNXOE5wpgawKWFcf_cXIrqoMtcy8ZPUxlsP4n4leivk0VjsSw0kNfdShH123Ixd_ZFQ=.

Vamorolone, an investigational drug, was generally safe and well tolerated. The most commonly reported adverse events versus placebo were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.

U.S. pre-commercialization measures advanced

The U.S. subsidiary made further progress in establishing launch readiness with the hiring into critical roles and a focus on long lead-time priority projects. These include medical and market access activities, working closely with key clinical opinion leaders to facilitate presentations and papers as well as engaging with patient advocacy groups.

Started Phase 2 study evaluating vamorolone in a wider age range of patients with DMD

Health care professionals routinely prescribe glucocorticoid steroids in DMD to preserve muscle strength and function in ambulant boys, starting at an early stage. In most cases treatment is continued until deleterious side effects prevent further therapy and lead to early discontinuation. The clinical development program for vamorolone included patients 4-7 years old and this new study aims at collecting information on vamorolone outside the original age range. The ongoing Phase 2 VBP-006 study (ClinicalTrials.gov ID: NCT05185622) is an open-label, multiple dose study to evaluate the clinical efficacy, safety and tolerability of vamorolone 2 or 6 mg/kg/day over a treatment period of 12 weeks in 44 steroid-naïve boys ages 2 to <4 years, and glucocorticoid-treated and currently untreated boys ages 7 to <18 years with DMD. The estimated study completion date is end of 2023.

First patient dosed in FDA-funded study with vamorolone in Becker muscular dystrophy (BMD)

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October 31, 2022 02:00 ET (06:00 GMT)