Potential DMD therapy MP1032 granted orphan drug status by FDA

Phase 2 trial of MetrioPharm's oral treatment expected to start in 2024

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by Patricia Inácio, PhD |

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MP1032, MetrioPharm’s oral therapy candidate for Duchenne muscular dystrophy (DMD), has been designated an orphan drug by the U.S. Food and Drug Administration (FDA).

A Phase 2 trial testing MP1032 in DMD patients is expected to start next year.

“Currently, DMD cannot be cured, but it can be treated,” Wolfgang Brysch, MD, co-founder, chief scientific officer, and chief medical officer of MetrioPharm, said in a press release. “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.”

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Long-term use of standard corticosteroids can lead to serious side effects

Corticosteroids, such as prednisolone, are widely used to slow the decline in muscle strength in DMD patients. They work by reducing inflammation and suppressing the immune system. However, long-term use of these therapies may lead to serious side effects, such as weight gain, weakening of the bones, and fluid retention.

Metabolic dysfunction affecting immune cells has been identified as a factor that may fuel the development of several diseases, including DMD.

MP1032 is a small molecule that acts as a self-regulating modulator of macrophages — a key cell in immune response — and inflamed tissues to lower oxidative stress and inflammation. Oxidative stress occurs when the production of damaging toxic molecules, called reactive oxygen species, outweighs the body’s antioxidant defenses. Due to its self-regulated way of activation, the effect of MP1032 is limited to the site of inflammation, according to MetrioPharm.

In preclinical studies conducted with Duchenne UK, MP1032 enhanced muscle strength in mdx mice, a model of DMD, with similar efficacy as prednisolone, but without its serious side effects.

In another study, conducted by Eurofins, adding MP1032 to a 90% reduced dose of prednisolone boosted the corticosteroid’s efficacy by more than 2.5 times relative to the normal dose. This indicates that MP1032 and prednisolone are more effective when combined than when used alone, according to MetrioPharm.

With MP1032 we aim to improve the tolerability of treatment while also further slowing down disease progression.

Orphan drug status provides incentives to develop rare disease treatments

Orphan drug status encourages the development of therapies for rare diseases — those affecting fewer than 200,000 people in the U.S. — by providing certain benefits, including support throughout development, exemptions from FDA fees, and seven years of market exclusivity if the therapy is approved.

“In a designated orphan development, timelines are shorter, and costs are lower compared to indications with larger patient populations,” said Thomas Christély, MetrioPharm’s CEO. “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.”

MP1032 is also being developed to treat juvenile idiopathic arthritis, COVID-19, and multiple sclerosis, among other indications.