The study (2018-004405-64), named FRAMES, was approved by the Spanish Agency of Medicines and Medical Devices (AEMPS) and is expected to initiate patient recruitment in a few weeks at the Hospital La Paz, in Madrid.
Additional study sites are expected to open soon at clinical institutions across Belgium, Germany, and France.
The trial is going to assess the safety and efficacy of MIN-102 in approximately 36 patients, ages 12 to 60. Participants will be randomized to take either MIN-102 or placebo orally for about 48 weeks.
During this period, researchers will assess changes in patients’ cervical spinal cord area, as determined by magnetic resonance imaging (MRI) measurements, comparing treatment with MIN-102 to placebo.
They will also evaluate the impact of the treatment on the levels of several biomarkers related with Friedreich’s ataxia, as well as the treatment’s effect on fatigue, ataxia severity, and patients’ quality of life.
More information about the study can be found here.
“The FRAMES trial design uses meaningful endpoints that were developed using state of the art knowledge of the disease and its natural progression,” Alexandra Durr, MD, PhD, principal investigator at the Brain and Spine Institute of La Pitié-Salpêtrière University Hospital (ICM) in France and a coordinator of the Phase 2 trial, said in a press release.
“I welcome this approach to provide an awaited treatment option for our patients with the potential to influence disease progression,” she said.
MIN-102 is a selective PPAR gamma activator that has shown an ability to effectively pass the blood brain barrier and reach the central nervous system.
In preclinical models of several diseases, it was seen to modulate cellular signals involved in mitochondrial dysfunction, oxidative stress, neuroinflammation, nerve cell degeneration and demyelination (destruction of the nerve cells’ protective myelin layer).
Results from a Phase 1 study demonstrated that MIN-102 is well-tolerated. It also confirmed that MIN-102 can cross the blood brain barrier and activate PPAR gamma signals within the central nervous system, suggesting that it may hold therapeutic activity for several neurological disorders.
“We are pleased to have received approval for the launch of our Phase 2 trial for Friedreich’s Ataxia with our lead candidate, MIN-102,” Uwe Meya, MD, PhD, chief medical officer at Minoryx, said. “We believe our drug candidate may address the high unmet medical need in multiple rare CNS diseases.”
Minoryx is currently testing MIN-102 in a Phase 2/3 trial (ADVANCE, NCT03231878) in adult patients with adrenomyeloneuropathy, the most common form of X-linked adrenoleukodystrophy (X-ALD). Results of the study are expected to be announced by the end of 2020.
The U.S. Food and Drug Administration and the European Medicines Agency have granted orphan drug status to MIN-102 for the treatment of X-ALD.