Santhera Pharmaceuticals (SIX: SANN) announces that it has entered into an exclusive license agreement with Sperogenix Therapeutics, a China-based company specializing in orphan diseases. Under this agreement, Sperogenix will in-license vamorolone for rare disease indications for a total consideration of up to USD 124 million, including a double-digit upfront cash compensation and DMD-related US-regulatory milestone payments amounting to a combined USD 20 million, as well as further double-digit royalties on net sales.
Under the terms of the agreement, Santhera will grant Sperogenix Therapeutics exclusive development and commercialization rights to vamorolone in DMD and all other rare disease indications for Greater China (including mainland China, Hong Kong, Macau, and Taiwan). Santhera will remain responsible for manufacturing and supply while Sperogenix, a China-based dedicated rare disease company financed by Lilly Asia Ventures and Morningside Ventures, will focus on regulatory and development work and future commercialization. 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.
There are significant opportunities in the healthcare business in China due to the large patient base and high unmet needs. Regulatory reforms have led to an increasing number of approvals and launches of innovative medicines . DMD is on the Chinese list of rare disease with a high medical need recognized by the Chinese government . The estimated prevalence of DMD could be as high as 70,000 patients with increasing rate of diagnosis giving more patients access to care in expert centers. There is currently no approved treatment for DMD in China. Sperogenix will proactively engage with the health authorities in China in order to achieve an accelerated regulatory pathway for vamorolone.
“We look forward to working with Sperogenix, a strong rare disease partner and well positioned for bringing vamorolone to patients in China,” said Dario Eklund, Chief Executive Officer of Santhera. “This agreement enables us to continue our focus on US and EU regulatory activities on our own while also gaining access to one of the largest pharmaceutical markets globally.”
“We are pleased to partner with Santhera. We believe in the potential of vamorolone as a treatment for Duchenne muscular dystrophy and are excited about its potential in the treatment of other rare diseases. The extensive regulatory, clinical and commercialization experience in rare disease products of our team will help to bring this differentiated product candidate to benefit patients in the Greater China region,” said Alan (Zhiyu) Yan, Co-founder, Chairman and Chief Executive Officer of Sperogenix.
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.