RARE Daily

Minoryx and Sperogenix Enter Agreement On X-ALD Drug for China, Hong Kong, and Macau for up to $78 Million

September 24, 2020

Rare Daily Staff

Sperogenix Therapeutics will pay Minoryx Therapeutics up to $78 million in upfront and milestone payments for an exclusive license agreement for the development and commercialization in China, Hong Kong, and Macau for the experimental therapy leriglitazone, which could become the first treatment for the rare neurodegenerative condition X-linked adrenoleukodystrophy.

X-linked adrenoleukodystrophy (X-ALD) affects the nervous system and adrenal glands.  AMN (45 percent) and cALD (35 percent) are the two most common phenotypes of X-ALD. The age of onset of cALD patients is typically 4 to 8 years old. Untreated patients progress quickly, as severe neurological function impairment appears 6 to 24 months after disease onset, leading to early death in 2 to 4 years.

AMN is characterized by progressive spastic paraparesis, sensory dysfunction, and incontinence. This form progresses chronically with onset of symptoms typically in adulthood and poor prognosis.

There is currently no therapeutic treatment available for X-ALD. Several observational studies have demonstrated that hematopoietic stem cell transplantation (HSCT) may improve the five-year overall survival for cALD patients. However, there is no evidence that HSCT improves clinical outcomes of patients with AMN.

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 United States and Europe, and Fast Track Designation as well as Rare Pediatric Disease Designation from the U.S. Food and Drug Administration. A registration enabling trial in adult X-ALD patients with adrenomyeloneuropathy (AMN) is currently ongoing in the European Union and the United States.

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.

In addition to the upfront and milestone payments, the agreement provides for Minoryx to receive double-digit royalties on annual net sales if the product is approved.

“This is a major milestone that underscores the potential of leriglitazone to address an important unmet medical need,” Marc Martinell, co-founder and CEO of Minoryx.

Photo: Marc Martinell, co-founder and CEO of Minoryx

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