Neurovive’s Investigative Compound NV556 Shows Promise to Treat Mitochondrial Myopathy in Preclinical Study

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by Alice Melão |

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NeuroVive Pharmaceutical AB’s investigative compound NV556 holds therapeutic potential for the treatment of mitochondrial myopathy, according to recent data of a preclinical study.

Conducted in collaboration with researchers at Karolinska Instutet in Stockholm, Sweden, the study showed that treatment with NV556 could significantly improve the survival rate of animals with mitochondrial myopathy compared with placebo: 94 percent overall survival compared to 50 percent, respectively. In addition, animals treated with NV556 showed enhanced muscle function compared with the control group.

NV556 is an investigative compound that specifically targets cyclophilin D (CypD), which is involved in mitochondria metabolism regulation and myocardial workload. Previous studies have shown that patients with mitochondrial myopathy have elevated levels of CypD in the muscles.

The Swedish team had already shown that inhibition of this protein with cyclosporin A counteracted disease symptoms by improving muscle strength in experimental models. In addition, cyclophilin inhibitors have also showed beneficial effects for the treatment of other muscle disorders, such as Duchenne muscular dystrophy (DMD).

“The effects of NeuroVive’s cyclophilin inhibitor are very exciting and are in line with results from previous studies, where cyclophilin inhibitors have been shown to counter disruption of mitochondrial function and muscle weakness in different models of muscular disorders,” Håkan Westerblad, professor at Karolinska Institutet and principal investigator of the study, said in a press release. “We are looking forward to continuing the positive collaboration with NeuroVive and taking the project additional steps forward in development.”

The more recent results further demonstrate the therapeutic potential of CypD inhibition to prevent mitochondrial myopathy progression.

Magnus Hansson, MD, PhD, chief medical officer and head of preclinical and clinical development at NeuroVive, called the study results “an important milestone in NeuroVive’s NVP025 project,” which evaluates NV556 compound potential. “The study shows that our cyclophilin inhibitors can be further developed towards the goal of offering mitochondrial myopathy patients a novel treatment option,” Hansson said.

Supported by the positive results, the company will continue developing its CypD inhibitors.

“We are now taking the project into its next phase, where we will optimize a candidate drug suitable for further development for patients with different types of muscle disorders,” Hansson said.