Sperogenix Therapeutics, a platform company dedicated to developing and commercializing rare disease therapeutics in China, and Minoryx Therapeutics, a company that specializes in the development of innovative treatments for orphan Central Nervous System (CNS) diseases, have entered into an exclusive license agreement for the development and commercialization of leriglitazone, Minoryx’s brain penetrating disease-modifying PPAR-γ agonist.
 
Under the terms of the agreement, Sperogenix will receive exclusive rights to develop and commercialize leriglitazone in mainland China, Hong Kong special administrative region (SAR) and Macau SAR, for the treatment of X-linked adrenoleukodystrophy (X-ALD), a life-threatening orphan neurological condition. Minoryx will receive an initial upfront payment and pre-defined regulatory and commercial milestone payments of up to $78 million, as well as double-digit royalties on annual net sales.
 
Leriglitazone is a novel bioavailable and selective PPAR-γ agonist, with the potential to become the world’s first treatment for X-ALD. It has received Orphan Drug Designation for X-ALD in both the U.S. and Europe, and Fast Track Designation as well as Rare Pediatric Disease Designation from the U.S. FDA. A registration enabling trial in adult X-ALD patients with adrenomyeloneuropathy (AMN) is currently ongoing in the EU and in the U.S. (ADVANCE trial). It is a global multi-centric, double-blind, placebo-controlled Phase 2/3 study with data expected by the end of 2020. In addition, leriglitazone is being evaluated in a registration enabling open-label Phase 2 study in pediatric patients with cerebral X-ALD (cALD) in Europe, with topline results anticipated by mid-2021.
 
“This exclusive license agreement entered for leriglitazone further strengthens Sperogenix’s pipeline in neurological rare diseases,” said Mr. Alan (Zhiyu) Yan, co-founder, chairman and CEO of Sperogenix. “It demonstrates our strong dedication and long-term commitment to address the huge unmet medical needs in the rare disease field in China. We look forward to collaborating with all stakeholders to bring this therapy to Chinese patients as early as possible.”
 
“We are very pleased to enter into this exclusive license agreement with Sperogenix based on its deep insights in rare diseases in China and its unique capabilities, which make us highly confident in our partnership,” said Dr. Marc Martinell, co-founder and CEO of Minoryx. “This is a major milestone that underscores the potential of leriglitazone to address an important unmet medical need.”