Bouncing back from late-phase fail, Minoryx pockets €51M to guide rare disease drug to market

Minoryx has persuaded investors to overlook the failure of its phase 2/3 clinical trial, enabling it to reel in 51 million euros ($55 million) to support attempts to bring the rare disease drug to market in Europe and the U.S. 

Roche helped put Minoryx on the map back in 2015, when its venture fund contributed to a 19.4 million euro round that set the biotech up to take PPAR gamma agonist MIN-102 into the clinic in patients with the genetic nervous system disorder X‐linked adrenoleukodystrophy (X-ALD). Seven years later, the drug candidate, now called leriglitazone, is approaching the final steps on its path to market.

The latest financing is intended to support Minoryx through those steps. Columbus Venture Partners and Caixa Capital Risc co-led the round. The mix of series C equity financing and complementary bank debt has given Minoryx 51 million euros to play with.

The company will use the money to fund a filing for approval and launch preparations in Europe as well as to support its efforts to bring the drug candidate to market in the U.S. While Minoryx shared top-line data from a phase 2/3 clinical trial of the candidate in January 2021, it is still talking to the FDA about the next steps for U.S. approval. 

The phase 2/3 clinical trial in adrenomyeloneuropathy (AMN), which, along with cerebral ALD (cALD), is one of the two most common phenotypes of X-ALD, missed its primary six-minute walk test endpoint. Minoryx looked past the primary endpoint miss, pointing to the effects in early symptomatic patients and on secondary measures to make the case that the drug has a future. Some VCs buy the argument. 

“We are impressed by the clinical data showing the potential of leriglitazone to treat both AMN and cALD,” Damià Tormo, managing director and co-founder of Columbus Venture Partners, said in a statement. “We are eager to support the development of what could be the first approved treatment for the most prevalent form of X-ALD.”

Minoryx plans to advance leriglitazone toward approval on both sides of the Atlantic in AMN while taking steps to generate data in cALD. An open-label phase 2/3 clinical trial in male pediatric patients with early stage cALD is already underway, and a label expansion into women is planned.