The first US patient has been dosed with the drug MIN-102 as part of a clinical trial into its effects in patients with adrenomyeloneuropathy. For more information about this, you can view the original press release here, at Minoryx Therapeutics’ website.
About Adrenomyeloneuropathy (AMN)
AMN is a condition caused by mutations in the ABCD1 gene. This gene is also linked to adrenoleukodystrophy (ALD). AMN can cause damage to nerves, which may cause difficulties with movement, including stiffness, weakness, and pain. Other areas that may be affected include the bladder, bowel, and sexual organs. AMN is thought to be most common in men, although women may also be affected by the condition. This information is sourced from ALD Life.
About the Drug Being Studied
MIN-102 is an investigational drug that is being researched as a potential treatment for AMN. It is a selective PPAR gamma agonist that has previously been studied in animal models. According to Minoryx, researchers believe it may have the potential to treat AMN and cerebral ALD. A phase 1 study that was carried out indicated that MIN-102 is well tolerated, and has the ability to cross the blood-brain barrier. Both the EU and US have granted MIN-102 Orphan Drug status for the treatment of X-ALD.
The ADVANCE Trial
The ADVANCE trial is an ongoing Phase 2/3 clinical study that is investigating MIN-102 as a potential treatment for patients with AMN. Recently, the researchers dosed a patient taking part in the US arm of the study, making him the first US patient to ever receive MIN-102.
The clinical trial is a randomised and double-blind study that will compare the effects of MIN-102 to those of a placebo drug. The study also has an open-label extension phase. The trial is designed to investigate how safe and effective MIN-102 is as a treatment for AMN; the main outcome researchers will track is the progression of AMN in male patients, as measured by a motor function test. In total, researchers hope to enrol over one hundred patients to take part in the study. The trial is taking place over sites in Europe and the US, and results are hoped to become available by the end of 2020.
For more information, you can view the original press release here.