Date: 2017-03-21
Type of
information: Completion of the trial
phase: 1
Announcement: completion of the trial
Company: Minoryx Therapeutics (Spain)
Product: MIN-102 (5-[4-[2-(5-(1-hydroxyethyl)-2-pyridinyl)ethoxy]benzyl]-2,4-thiazolidinedione hydrochloride)
Action
mechanism:
- PPAR agonist. MIN-102 is a novel, orally bioavailable and selective PPAR gamma agonist. This metabolite from pioglitazone showed a superior brain penetration and safety profile, allowing PPAR gamma engagement above the level that can be safely achieved with pioglitazone and other glitazones. It showed robust preclinical proof of concept in several animal models.
- There are two main clinical phenotypes of X-ALD: adrenomyeloneuropathy (AMN), characterized by progressive motor dysfunction, and inflammatory cerebral ALD, characterized by severe neuroinflammation leading to early death. There are currently no pharmacological treatments for X-ALD. MIN-102 is the only product in development for potential use across all the main phenotypes.
- In X-ALD, mutations on ABCD1 trigger a cascade of events leading to mitochondrial dysfunction, oxidative stress, neuroinflammation, demyelination and axonal degeneration. MIN-102, through its PPAR gamma activity, prevents such dysfunctions, thus it has the potential to treat both adrenomyeloneuropathy (AMN) and cerebral ALD (cALD).
Disease: X-linked adrenoleukodystrophy
Therapeutic
area: Rare diseases - Genetic diseases
Country:
Trial
details:
- The phase 1 trial was a combined single- and multiple-ascending dose study with the aim of assessing pharmacokinetics, safety and tolerability of MIN-102 in healthy male volunteers. Additionally, the trial included assessment of food effect, evaluation of brain penetration and biomarkers for PPAR Gamma engagement.
Latest
news:
- • On March 21, 2017, Minoryx Therapeutics announced that it has successfully completed its phase 1 trial with MIN-102. MIN-102 targets X-linked adrenoleukodystrophy (X-ALD), a rare and chronically debilitating life threatening neurodegenerative disease. The results show that MIN-102 was generally safe and well tolerated at exposures exceeding the levels required for efficacy. No serious adverse events were observed, any adverse events were mild and similar to those observed with the placebo. Results from brain penetration and biomarker assessment confirmed earlier results, showing that MIN-102 reaches its target in the central nervous system and exerts a broad range of effects to treat various aspects of X-ALD aligned with those observed in preclinical studies. No relevant food effects were observed and pharmacokinetics showed good linearity with dose providing a simple and convenient dosing regimen for patients.
- Based on the successful completion of the phase 1 trial, a phase 2/3 trial in adult AMN patients will be launched in the coming months.
- • On July 6, 2016, Minoryx Therapeutics announced the initiation of its first-in-man Phase 1 clinical trial for MIN-102. The aim of the Phase 1 study is to assess pharmacokinetics, safety, tolerability and brain penetration of MIN-102 after administration of single ascending doses and multiple ascending doses in healthy volunteers. Results are expected by the end of the year
Is
general: Yes
