Santhera inks exclusive agreement with USD 20M upfront payment

Santhera Pharmaceuticals is a Swiss pharmaceutical company focused on the development and commercialization of innovative medicines for rare neuromuscular and pulmonary diseases with high unmet medical needs. The company developed vamorolone, a first-in-class dissociative steroid with novel mode of action, which was investigated in a pivotal study in patients with Duchenne muscular dystrophy (DMD) as an alternative to standard corticosteroids. Vamorolone has been granted Orphan Drug status in the US and in Europe for DMD, and has received Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.

DMD is one of the most common and devastating types of muscular degeneration and results in progressive muscle weakness, starting at young age. DMD is also on the Chinese list of rare diseases with a high medical need recognized by the Chinese government. Approximately 70’000 patients in china have been diagnosed with DMD, and there is currently no approved treatment for DMD in the country. The partnership with Santhera allows China-based Sperogenix to proactively engage with the health authorities in China to achieve an accelerated regulatory pathway for vamorolone. Under the terms of the agreement, Sperogenix will receive exclusive rights for the development and commercialization of vamorolone for the treatment of DMD and any other rare disease. Sperogenix plans to initiate a regulatory filing for vamorolone for DMD in China upon US FDA approval which could lead to market entry in China as early as in 2024. 

With a total deal consideration of USD 124 million, Santhera will receive a double-digit upfront cash compensation andDMD-related US-regulatory milestone payments amounting to a combined USD 20 million, as well as further double-digit royalties on net sales.

Continued efforts to push vamorolone in the US and Europe
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. The Company will commence a rolling NDA submission in the US in Q1-2022, paving the way for a first launch as early as beginning of 2023 in the US, followed by a European marketing authorization application in Q2-2022. 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.

(Press release/RAN)