Minoryx Raises $55 Million to Support MAA and Launch for X-ALD Therapy

Spanish rare CNS disease focused biotech Minoryx Therapeutics raised $55 million (€51 million) to finance the marketing authorization application and launch preparations of its drug candidate leriglitazone for adult male X-ALD patients with adrenomyeloneuropathy (AMN) in the European Union.

Photo: Marc Martinell, CEO of Minoryx

Minoryx will also use the funds towards the approval of leriglitazone in the United States for the same indication. The company is currently holding discussions with the U.S. Food and Drug Administration to define the next steps for its U.S. approval path. Finally, the proceeds will support the continuation of leriglitazone’s development in pediatric patients with cerebral ALD (cALD) as well as label expansion into women affected by X-ALD.

X-linked adrenoleukodystrophy (X-ALD) is an orphan, inherited neurodegenerative disease. The most common form is adrenomyeloneuropathy (AMN), which is a highly debilitating chronic disease affecting male and female X-ALD patients reaching adulthood. AMN affects all patients reaching adulthood and is characterized by progressive spastic paraparesis, sensory dysfunction and incontinence. This form progresses chronically with onset of symptoms typically in adulthood, affecting both men and women and has poor prognosis. There is currently no approved treatment for AMN patients. In male patients, both pediatric and adult, X-ALD can also manifest in its acute cerebral form, cALD, resulting in aggressive brain inflammation, leading to permanent disability and death within 2-4 years. The global incidence of X-ALD is approximately 6.2 per 100,000 live births. AMN and cALD are the two most common phenotypes.

Leriglitazone, a novel brain penetrant PPAR gamma agonist, has shown significant clinical benefit in Minoryx’s ADVANCE phase 2/3 clinical trial in adult male patients with AMN. In this study, leriglitazone reduced the progression of cerebral lesions and myelopathy symptoms. These data also support Minoryx’s ongoing NEXUS study, an open-label phase 2/3 trial assessing leriglitazone in male pediatric patients with early stage cALD.

“Minoryx’s series C investment round will enable us to move forward at full speed towards the approval and commercialization of leriglitazone in X-ALD, a devastating orphan disease with a major unmet medical need,” said Marc Martinell, CEO of Minoryx. “These funds will also enable Minoryx to proceed towards US approval based on FDA guidance and investigate the benefits of leriglitazone in further X-ALD patient populations.”

The financing, which includes series C equity financing and complementary bank debt, was co-led by Columbus Venture Partners and Caixa Capital Risc. Damià Tormo, representing Columbus Venture Partners, has joined Minoryx’s Board of Directors. CDTI, through its Innvierte program, also joined the series C round, which included existing series B investors led by Fund+ and its Belgian Public co-investors, and series A investors, led by Ysios Capital.

Leriglitazone has been granted Orphan Drug status from the FDA and the EMA and Fast Track and Rare Pediatric Disease designations from the FDA for the treatment of X-ALD.

Author: Rare Daily Staff