• Vamorolone is approved and marketed as AGAMREE® for the treatment of Duchenne Muscular Dystrophy (DMD) in the US, European Union, UK and China
• New five-year data on vamorolone highlighted substantially improved safety profile with reduced vertebral fractures, lower incidence of cataracts and that normal growth rate was maintained.   With adverse effects being a key limitation of conventional corticosteroid therapy, vamorolone’s safety profile combined its comparable effectiveness, has the potential to displace existing steroid treatments
• Transaction advances Nxera’s mission to bring innovative medicines to patients in Japan/APAC in line with 2030 vision to build high-growth, highly profitable Japanese biopharma company
  
  

Nxera Pharma Co. Ltd announces that it has entered an exclusive licensing agreement for the development, manufacturing and commercialization of vamorolone for the treatment of Duchenne Muscular Dystrophy (DMD) in Japan, South Korea, Australia and New Zealand with Santhera Pharmaceuticals Holding (“Santhera”) (SIX: SANN). Vamorolone is approved and marketed as AGAMREE® for the treatment of DMD, a rare inherited neurodegenerative disease, in the US, European Union, UK and China.

The addition of vamorolone brings into Nxera’s portfolio of innovative medicines for rare and specialty diseases, a late-stage development candidate with the potential to address significant unmet needs of patients in Japan and the Asia-Pacific (“APAC”) region living with DMD. This portfolio includes PIVLAZ™ (clazosentan) and QUVIVIQ™ (daridorexant), both of which are approved and marketed in Japan and under commercial development in other APAC countries.

Under the terms of the agreement, Nxera will be responsible for obtaining regulatory approval of vamorolone in the licensed territories, including undertaking regulatory activities such as clinical trials where required, and will lead commercialization and manufacturing activities for those territories. This strategic partnership leverages Nxera's proven commercial capabilities and development and regulatory expertise across the APAC region, particularly in Japan. In addition, Nxera’s team brings significant prior development and manufacturing experience with vamorolone through its 2023 acquisition of Idorsia’s Japan and APAC business.

Christopher Cargill, President and Chief Executive Officer of Nxera, commented: “We are excited to partner with Santhera to bring vamorolone to DMD patients in Japan, South Korea, Australia and New Zealand. Vamorolone’s differentiated safety and efficacy profile has the potential to fundamentally change the standard of care by enabling early use, full dosing, and long-term treatment, addressing critical limitations of existing steroid therapies currently used in the region.

“Furthermore, the transaction expands our portfolio of late- and commercial-stage products for Japan/APAC and advances our mission to bring innovative medicines to patients in these important regions in line with our 2030 vision to build high-growth, highly profitable Japanese biopharma company.”

Dario Eklund, Chief Executive Officer of Santhera, added: “This strategic partnership represents a significant milestone in our mission to expand global access to AGAMREE® (vamorolone) and bring meaningful new treatment options to patients with DMD worldwide. Nxera's deep expertise and established infrastructure in Japan and across the wider APAC region, as well as prior experience of vamorolone, make them an ideal partner to unlock the full commercial and clinical potential of AGAMREE® (vamorolone) in these markets and accelerate access for patients with DMD.”

Additional transaction terms
In return for development, manufacturing and commercialization rights for vamorolone in the licensed territories, Nxera will make an upfront payment of USD 40 million to Santhera, consisting of USD 30 million in cash and USD 10 million as a strategic equity investment. Santhera is also eligible to receive sales and regulatory milestone payments of up to USD 165 million from Nxera as well as tiered royalties starting in the low teens on net sales of vamorolone in the licensed territories.