Dawn Pharma has reached an agreement with Minoryx to innovate the rare disease drug Leriglitazone.

Under the terms of the agreement, Shufang Pharmaceuticals will receive exclusive development and commercialization interests in Chinese mainland, Hong Kong and Macao Special Administrative Regions for Leriglitazone's treatment of X-series adrenal white blood malnutrition (X-ALD), a rare and life-threatening neurological disorder, and Minoryx will receive a down payment of up to $78 million and a two-figure annual net sales share.
Leriglitazone, a new oral selective PPAR gamma agonist, is expected to be the world's first treatment to fill the gap in the field of X-ALD disease, having previously been identified as an orphan drug for X-ALD in the United States and Europe, and as a fast-track and pediatric rare disease identified by the FDA in the United States.
a critical registered clinical trial (ADVANCED) for adult X-ALD patients assessing Leriglitazone's treatment of epinephrine neuropathy (AMN) is being conducted simultaneously in the United States and Europe, and the results of this randomized, double-blind, placebo-controlled, international multi-center Phase 2/3 clinical trial are expected to be published in late 2020.
another open-label Phase 2 key registered clinical trial to assess the efficacy of Leriglitazone in the treatment of brain type X-ALD in children is under way in Europe, with preliminary results expected to be published in mid-2021.
.