Minoryx, a biotech company focused on developing innovative therapies for rare diseases, has received clearance from the US Food and Drug Administration (FDA) to initiate a Phase III clinical trial of its therapy for cerebral adrenoleukodystrophy (cALD). This is a significant milestone for the company and for patients suffering from this devastating disease.
cALD is a rare genetic disorder that affects the nervous system and can lead to severe disability or death. It is caused by a mutation in the ABCD1 gene, which leads to the accumulation of very long-chain fatty acids in the brain and other organs. The disease primarily affects young boys and can progress rapidly, with symptoms including behavioral changes, loss of vision, hearing, and motor function.
Minoryx’s therapy, known as MIN-102, is a small molecule that targets the underlying cause of cALD by reducing the accumulation of very long-chain fatty acids in the brain. The drug has shown promising results in preclinical studies and in a Phase II clinical trial, where it demonstrated a significant reduction in disease progression compared to placebo.
The Phase III trial will enroll approximately 120 patients with early-stage cALD and will evaluate the safety and efficacy of MIN-102 over a period of two years. The primary endpoint of the trial is the proportion of patients who experience disease progression, as measured by changes in neurological function and brain imaging.
“We are thrilled to receive FDA clearance to initiate our Phase III trial of MIN-102 in cALD,” said Marc Martinell, CEO of Minoryx. “This is a major step forward in our mission to develop a much-needed therapy for patients with this devastating disease. We look forward to working with our clinical partners to advance this important program.”
The Phase III trial is expected to begin in the second half of 2021 and will be conducted at multiple sites in the US and Europe. Minoryx has also received orphan drug designation from the FDA and the European Medicines Agency for MIN-102 in cALD, which provides regulatory and financial incentives to support the development of treatments for rare diseases.
In addition to cALD, Minoryx is also developing therapies for other rare diseases, including X-linked adrenoleukodystrophy (X-ALD) and Friedreich’s ataxia. The company’s approach is based on identifying and targeting specific pathways that are disrupted in these diseases, with the goal of developing precision therapies that can address the underlying cause of the disease and improve patient outcomes.
Overall, the clearance of Minoryx’s Phase III trial for cALD therapy represents a significant step forward in the development of treatments for rare diseases. With continued investment and innovation in this field, there is hope that more effective therapies will be developed to improve the lives of patients and their families.
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