RARE Daily

Santhera Enters into Exclusive License Agreement with Sperogenix for DMD Therapy in the Greater China Region

January 4, 2022

Santhera Pharmaceuticals said that it has entered into an exclusive license agreement with Sperogenix Therapeutics, a China-based company specializing in rare diseases for its therapy vamorolone for Duchenne muscular dystrophy (DMD) and other rare diseases.

Under the agreement, Sperogenix will in-license vamorolone for rare disease indications for a total consideration of up to $124 million, including a double-digit upfront cash compensation and DMD-related U.S.-regulatory milestone payments amounting to a combined $20 million, as well as further double-digit royalties on net sales.

Santhera will grant Sperogenix 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 be responsible for manufacturing and supply while Sperogenix will focus on regulatory and development work and future commercialization. Sperogenix plans to initiate a regulatory filing for vamorolone for DMD in China upon U.S. Food and Drug Administration approval, which could lead to market entry in China as early as in 2024.

“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,” said Dario Eklund, CEO of Santhera.

DMD is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. The condition leads to the loss of the ability to walk, the loss of the ability for self-feeding, the need for assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the person reaches the forties due to respiratory or cardiac failure.

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.

Vamorolone is a first-in-class drug candidate that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative agonist. This mechanism has the potential to dissociate efficacy from typical steroid safety concerns and therefore vamorolone could emerge as a promising alternative to existing corticosteroids, the current standard of care in children and adolescents with DMD, the company said.

In the pivotal VISION-DMD study, vamorolone met the primary endpoint Time to Stand (TTSTAND) velocity versus placebo at 24 weeks of treatment and showed a good safety and tolerability profile. Compared to prednisone, vamorolone showed comparable efficacy, improvements on multiple safety parameters (including a reversal of growth impairment seen during prednisone treatment and a reduction of behavioral changes) and was associated with fewer adverse events. In clinical studies, vamorolone was generally well tolerated. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.

Vamorolone has been granted Orphan Drug status in the U.S. and in Europe for DMD and has received Fast Track and Rare Pediatric Disease designations by the U.S. FDA and Promising Innovative Medicine status from the United Kingdom’s Medicines and Healthcare products Regulatory Agency for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.

“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 CEO 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 United States in the first quarter of 2022. That could lead to a first launch as early as the beginning of 2023 in the United States, the company said.

Photo: Dario Eklund, CEO of Santhera

Author: Rare Daily Staff

Stay Connected

Sign up for updates straight to your inbox.