Minoryx Therapeutics has finished randomizing patients in its ongoing Phase 2/3 clinical trial testing MIN-102, an investigational oral therapy for adrenomyeloneuropathy (AMN) – the most common type of X-linked adrenoleukodystrophy (X-ALD).
A total of 116 men, ages 18 to 65, with confirmed AMN were randomized to receive either MIN-102 or a placebo. Ninety patients are being treated in Europe, at sites across Spain, France, Hungary, Germany, Italy, the United Kingdom, and the Netherlands. Others are being treated in the U.S., at clinical centers in California, Maryland, and Massachusetts.
Minoryx expects to have top-line data from the ADVANCE Phase 2/3 trial (NCT03231878) by the end of 2020.
“The strong interest in this AMN trial in the E.U. and U.S. has resulted in exceeding the target enrolment ahead of schedule,” Uwe Meya, MD, PhD, chief medical officer of Minoryx, said in a press release. “We believe this highlights the very high unmet medical need in this area.”
MIN-102 is an oral, selective PPAR gamma agonist that has enhanced capacity to cross the protective blood-brain barrier, and penetrate the central nervous system. This investigational therapy has the potential to prevent mitochondrial dysfunction, oxidative stress, neuroinflammation, myelin loss, and nerve cell degeneration, all features known to contribute for AMN progression.
MIN-102 was given orphan drug status for the treatment of X-ALD in both the E.U. and the U.S. to speed its development. The company believes it has the potential to treat both AMN and cerebral ALD (cALD).
Results from a Phase 1 clinical study testing single and multiple ascending doses in healthy male volunteers found that MIN-102 to be well-tolerated and safe. No serious treatment-related adverse events were reported, and side effects experienced by the participants were mild in severity and similar to those reported in the placebo-treated group.
Evaluation of brain penetration and cellular biomarkers demonstrated that MIN-102 can reach the cells in the central nervous system. The treatment was also seen to have a broad activity, targeting several mechanisms of X-ALD, in accordance with the data collected in preclinical studies.
The main goal of this Phase 2/3 trial is to evaluate the ability of MIN-102 to prevent the progression of AMN in patients during a follow-up period of 96 weeks. Treatment efficacy will be determined with a motor function test, as well as by patients’ self-reported outcomes and improvements in life quality.
“On behalf of the company, I would like to take this opportunity to thank the patients and their families, the advocacy groups, and the caregivers and their staff for achieving this fast enrollment,” Marc Martinell, CEO of Minoryx, said.
For additional information about the trial, please visit this link.