DMD is the most common form of muscular dystrophy and is a genetic disorder characterised by progressive muscle degeneration; symptom onset is in early childhood, usually between the ages of two and three
Microscopic image of cross-sectional calf muscle from a person with Duchenne muscular dystrophy. (Credit: Dr. Edwin P. Ewing, Jr./ Wikipedia)
MetrioPharm, a pharmaceutical company developing drugs for inflammatory and infectious diseases, announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation for MP1032 for the treatment of Duchenne muscular dystrophy (DMD).
DMD is the most common form of muscular dystrophy. It is a genetic disorder characterised by progressive muscle degeneration; symptom onset is in early childhood, usually between the ages of two and three.
The condition predominantly impacts boys, resulting in substantial muscle loss and heart failure, although girls may also exhibit milder symptoms. DMD significantly reduces life expectancy. Currently, the standard treatment approach involves administering high doses of corticosteroids over an extended period, which can lead to severe side effects and merely slow down the progression of the disease.
MetrioPharm CSO and co-founder Dr Wolfgang Brysch said: “Currently, DMD cannot be cured, but it can be treated. These treatments have serious side effects that heavily impact patients’ quality of life.
“With MP1032 we aim to improve the tolerability of treatment while also further slowing down disease progression. Our goal is to improve both safety and efficacy in the long-term treatment of DMD patients.”
MetrioPharm CEO Thomas Christély said: “The orphan drug designation is granted by the FDA for drug candidates that the FDA considers a promising new treatment. In a designated orphan development, timelines are shorter, and costs are lower compared to indications with larger patient populations.
“The Orphan Drug Designation for MP1032 in DMD by the FDA is a very important achievement; it takes us one crucial step closer to obtaining an accelerated market approval of our lead compound for Duchenne patients. MetrioPharm plans to initiate a Phase II clinical trial in DMD in 2024.”
DMD is categorised as an orphan disease with a high level of unmet medical need. The FDA grants orphan drug designation to investigational treatments targeting rare medical conditions that affect fewer than 200,000 individuals in the US. Obtaining orphan drug status offers advantages to drug developers, such as assistance in the development process, exemptions from FDA fees, and a seven-year period of exclusive marketing rights following approval.
Details of MP1032 in DMD:
MetrioPharm has conducted preclinical studies in collaboration with the patient organisation Duchenne UK. These in vivo experiments involved testing MP1032 in an mdx model for DMD and comparing its effects to corticosteroids. MP1032 demonstrated the ability to improve muscle strength, similar to the corticosteroid Prednisolone, but without the serious side effects associated with the latter.
In a separate preclinical study conducted by Eurofins, several biomarkers indicated that combining MP1032 with a 90% reduced dose of Prednisolone resulted in an efficacy increase of more than two and a half times the normal dose of Prednisolone. This synergistic effect, known as supra-additivity, signifies that the combination of MP1032 and Prednisolone is more effective than either substance alone.