Ad hoc announcement pursuant to Art. 53 LR 


   -- Cash and cash equivalents of CHF 8.0 million (as of June 30, 2021) 
 
   -- Net revenue CHF 4.5 million (H1-2020: CHF 7.8 million) 
 
   -- Net result for period of CHF -20.5 million (H1-2020: CHF -31.8 million)

Pratteln, Switzerland, October 15, 2021 -- Santhera Pharmaceuticals (SIX: SANN) announces the Company's financial results for the first half-year ended June 30, 2021, and provides an update on corporate progress.

"Our achievements in the first half of 2021 have moved us significantly closer to accomplishing key goals. The highlights in the period under review were undoubtedly the positive results of the Phase 2b VISION-DMD study at 24 weeks with vamorolone compared to placebo in Duchenne muscular dystrophy, paving the way for an NDA submission in the U.S. We are equally excited about the favorable tolerability profile of vamorolone versus prednisone, potentially addressing the high medical need for steroid efficacy with a safety profile differentiated from steroids on some key clinically meaningful parameters. On the financing side, we successfully restructured the Company's balance sheet and secured adequate funding to take us to mid-2022 or beyond key upcoming milestones," said Dario Eklund, Chief Executive Officer of Santhera. "These successes are crucial for both patients who are in great need of a better tolerated steroid therapy suitable for chronic treatment and for Santhera as they lay the foundation for renewed future growth. We are grateful for the continued support of patients and their caregivers, health care staff and investors, which will allow us to move forward with our strategy and execute our plans and ambitions."

OPERATIONAL HIGHLIGHTS

Recent developments 


   -- Statistically highly significant 24-week results across multiple 
      endpoints with vamorolone in pivotal Phase 2b VISION-DMD study 
 
   -- Long-term treatment data with vamorolone demonstrating maintenance of 
      effect over 2.5 years 
 
   -- Positive results with lonodelestat in Phase 1b cystic fibrosis (CF) trial 
 
   -- Primary endpoint met in Phase 4 LEROS trial with Raxone(R) in Leber's 
      hereditary optic neuropathy (LHON) 
 
   -- Restructuring activities (announced in October 2020) completed 
 
   -- Completion of 2017/22 convertible bond exchange offer and issuance of a 
      new 2021/24 convertible bond 
 
   -- Share capital increases implemented as a basis to enable additional 
      financing to secure operations, fund prelaunch activities for vamorolone 
      and support advancement of pipeline 
 
   -- Completion of financing to provide up to CHF 42 million net of fees and 
      expenses to fund operations and current debt obligations

Upcoming milestones 


   -- Q4-2021: Completion of vamorolone 48-week VISION-DMD study providing 
      additional safety data 
 
   -- Q4-2021: Conclusion of Raxone PAMs (post-authorization measures) 
 
   -- Q1-2022: NDA (new drug application) filing in the US for vamorolone in 
      DMD 
 
   -- Q2-2022: Regulatory submission in Europe for vamorolone in DMD

Vamorolone--nearing regulatory submission to the US FDA

In June 2021, Santhera and ReveraGen BioPharma announced positive 24-week results from the VISION-DMD study, a pivotal Phase 2b study comparing vamorolone (2 or 6 mg/kg/day) to placebo and prednisone (0.75 mg/kg/day) in the treatment of Duchenne muscular dystrophy (DMD). The results demonstrated robust efficacy as the study met its primary endpoint of superiority in change of time from supine positioning to standing (TTSTAND) velocity with vamorolone 6 mg/kg/day versus placebo (p=0.002) with a treatment difference of 0.06 rises/second [95% CI: 0.02--0.10] from baseline. Likewise, the study also demonstrated superiority of vamorolone versus placebo across multiple secondary endpoints and established the efficacy of vamorolone at 2 and 6 mg/kg/day. Vamorolone is the only steroid to have shown efficacy for two doses across a three-fold dose range, allowing physicians for the first time to tailor treatment to the individual.

Already in April 2021, Santhera and ReveraGen announced new clinical data of 2.5-year treatment outcome with vamorolone in patients with Duchenne muscular dystrophy (DMD). These Phase 2a long-term treatment data demonstrated a maintenance of treatment effect, equivalent to a delay of about two years in decline for time to stand (TTSTAND) velocity, and confirmed safety and tolerability benefits of vamorolone over the 2.5-year follow up period. In comparison to reports from clinical trials with other corticosteroids, long-term treatment with vamorolone resulted in fewer of the side effects that are typically observed with those drugs.

Based on clinical trial results, including long-term safety data up to 30 months, vamorolone at doses up to 6 mg/kg/day was generally well-tolerated. Vamorolone treatment has been shown to preserve height trajectory and had a significantly lower adverse impact on measures of bone health and behavior changes compared to prednisone.

On the basis of the positive 24-week efficacy results from the pivotal VISION-DMD study and the demonstration of long-term benefits of vamorolone, Santhera is preparing for submission of a New Drug Application (NDA) in the US in Q1-2022, for which fast track designation was granted by the FDA.

In Q4-2021, the 48-week data readout delivering supplementary safety and tolerability data for vamorolone is expected upon completion of the VISION-DMD study. During the second period of the study, where all participants receive vamorolone treatment on either of the two dose levels, additional longer-term tolerability data is captured. The 48-week data will support the submission of a marketing authorization application in Europe in Q2-2022.

In September 2021, the FDA awarded a USD 1.2 million grant to ReveraGen under their "Clinical Studies of Orphan Products Addressing Unmet Needs of Rare Diseases (R01)" grants program to initiate a clinical trial of vamorolone in adults and children with Becker muscular dystrophy (BMD), a progressive muscle wasting disease similar to DMD, but usually milder. The mechanisms of actions, which provide the basis of vamorolone's efficacy as demonstrated in the pivotal VISION-DMD study in the more severe DMD, are hypothesized to be relevant to BMD too.

Santhera intends to commercialize vamorolone for the treatment of DMD through its own organization in the United States and main markets in Europe, and is seeking collaborations outside those regions for DMD and for additional indications worldwide. Santhera estimates the peak product sales potential for vamorolone in the indication DMD alone to be in excess of USD 500 million in the US and the largest five European countries combined.

Lonodelestat--positive results in early phase cystic fibrosis trial

In March 2021, Santhera announced positive results of a Phase 1b study with lonodelestat, a potent and selective peptide inhibitor of human neutrophil elastase (hNE) in development to treat cystic fibrosis (CF). Neutrophil elastase is an enzyme associated with tissue inflammation, leading to degradation of the lung tissue in cystic fibrosis and several other acute and chronic inflammatory conditions of the lung where neutrophils play a prominent role in the disease process. The double-blind, placebo-controlled multiple ascending dose Phase 1b study in patients with CF established a safe dose regimen and provided promising data on the safety of lonodelestat. Furthermore, the study demonstrated that lonodelestat is well tolerated at 40 mg and 80 mg daily doses and achieves the desired effect of near complete inhibition of elastase without any drug/metabolite accumulation.

The results from the safety analyses and the confirmed effect on the hNE biomarker by lonodelestat are very encouraging for further development in CF and other inflammatory lung diseases. On this basis, Santhera will now be refining the further clinical development program to advance lonodelestat for the treatment of CF and potentially for other inflammatory pulmonary conditions, whether acute or chronic.

Post-authorization measures (PAMs) with Raxone successful and nearing completion

In June 2021, Santhera announced positive topline results from its long-term Phase 4 LEROS study with Raxone (idebenone) in the treatment of LHON. The primary endpoint, the proportion of eyes with clinically relevant benefit after 12 months treatment with Raxone versus untreated patients from an external natural history control group, was met with high statistical significance (p=0.002). The efficacy data confirm and extend previous findings which demonstrated that Raxone can prevent further vision loss and promote recovery of vision in LHON patients.

The study, which was designed with guidance and approval from the European Medicines Agency (EMA), was part of a post-authorization commitment. The strong evidence of efficacy is expected to support market access in countries where this is not yet the case, allowing patients who have no therapeutic alternative to benefit from treatment with Raxone.

Santhera holds the EU marketing authorization for Raxone (idebenone) and out-licensed rights to the product outside North America and France for the treatment of LHON to Chiesi Group. Santhera is still commercializing Raxone for LHON in France in a transitional phase and, as previously communicated, is supplying the product free of charge since August 2021 following its removal from the list of reimbursed products and while reimbursement discussions are ongoing. The Company is entitled to contingent variable near- to mid-term milestone payments from Chiesi Group of up to EUR 49 million subject to the achievement of certain commercial milestones for Raxone.

Corporate restructuring completed and organization realigned to future priorities

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October 15, 2021 14:00 ET (18:00 GMT)