Investigational Oral Treatment Leriglitazone Gains Orphan Drug Status for FA

Leriglitazone (MIN-102), an investigational oral treatment by Minoryx Therapeutics, has been granted orphan drug status by the U.S. Food and Drug Administration (FDA) for treating patients with Friedreich’s ataxia (FA).

Orphan drug status qualifies Minoryx for various incentives meant to expedite clinical testing and possible approval of the treatment. If eventually authorized, it also provides seven years of marketing exclusivity in the U.S.

“Orphan Drug Designation by the FDA for Friedreich’s ataxia is yet another important milestone for the company. It is recognition of the disease-modifying potential of leriglitazone and of our commitment to changing the lives of patients suffering from severe orphan diseases with high unmet medical needs,” Marc Martinell, CEO of Minoryx said in a press release.

Leriglitazone (also known as MIN-102) is a novel PPAR gamma (γ) activator, or agonist, that is able to cross the blood-brain barrier — a barrier that controls the movement of substances and cells between the blood and the brain — and penetrate the brain.

By activating PPARγ, a receptor involved in regulating cells’ metabolism, leriglitazone is believed to counteract specific impairments within cells, thought to be central for neurodegeneration in FA. Specifically, it can help restore the functioning of mitochondria (the so-called “power plants” of cells) and protect against oxidative stress and neuroinflammation.

In animal models of neurological disease, the compound showed proof of such benefits, demonstrating anti-oxidant, anti-inflammatory, and neuroprotective properties.

Leriglitazone is currently in late-stage clinical development for the treatment of people with adrenomyeloneuropathy (AMN) — the most common form of adrenoleukodystrophy (ALD) and for which orphan drug status was granted previously — and Friedreich’s ataxia. ALD and AMN are genetic neurodegenerative disorders thought to share common pathways of nerve cell damage.

Earlier this month, Minoryx completed enrollment of patients in the Phase 2 clinical trial FRAMES (NCT03917225), which will test leriglitazone as a potential oral treatment for FA.

The study enrolled 39 participants, 12–60 years old, recruited in Belgium, France, Germany, and Spain.

Patients will be assigned randomly to either leriglitazone (15 mg/ml) or a placebo, given daily as an oral suspension, for one year.

Minoryx also is testing leriglitazone in patients with AMN in the pivotal Phase 2/3 ADVANCE trial (NCT03231878).

“We recently completed enrollment in the Phase 2 study of leriglitazone in Friedreich’s Ataxia and the pivotal study in patients with adrenomyeloneuropathy is progressing as planned. We are looking forward to reporting the topline data for both studies in late 2020,” Martinell said.