Orphan drug developer Minoryx raises €51m

The Series C round was co-led by Columbus Venture Partners and Caixa Capital Risc, and supported by CDTI Innvierte, Fund+, Ysios Capital and other current investors.According to Barcelona-headquartred Minoryx Therapeutics, the funds will support the EU and US applications for marketing authorisation of  the company's Phase III candidate leriglitazone for male X-ALD (X-linked adrenoleukodystrophy), an orphan neurodegenerative disease. Proceeds will also cover activities for extension of indication to entire X-ALD population.

The company is currently holding discussions with the FDA to define the next steps for its US 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.

Leriglitazone, a novel brain penetrant PPAR gamma agonist, has shown significant clinical benefit in Minoryx’s ADVANCE Phase II/III 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 II/III 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, Minoryx.

Leriglitazone has been granted orphan drug status from the FDA and the EMA and fast track and rare pediatric disease designation from the FDA for the treatment of X-ALD.