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What is NV354?

NV354 is a drug that is currently being investigated as a potential treatment for mitochondrial disease. It has been developed by a Swedish pharmaceutical company called NeuroVive Pharmaceuticals as part of their NVP015 programme that aims to create a medicine for patients with mitochondrial disease.

What is it supposed to do?

NV354 is thought to improve energy production within the mitochondria. This could increase the amount of energy produced by the mitochondria, which could reduce some of the symptoms associated with mitochondrial disease.

How does it work?

NV354 works by allowing the energy chain to produce energy when the first part of the chain (known as complex I) is faulty. It does this by providing an important molecule needed by the second part of the energy chain (complex II), meaning that the faulty part of the chain can be bypassed and energy production restored.

Is it a cure?

Unfortunately not. NV354 cannot correct the underlying genetic defect that causes mitochondrial disease.

What types of mitochondrial disease could it treat?

NV354 could be used to treat any mitochondrial disease that is caused by a problem in the first part of the energy chain, often known as complex I deficiency.

Complex I is the largest of the five complexes that make up the energy chain and defects in this complex are common in childhood mitochondrial diseases, including Leigh syndrome.

Have there been any trials of NV354?

There have been no clinical trials of NV354 to date. However, preclinical research has shown that NV354 is well tolerated in an animal model of mitochondrial disease, with other positive findings confirming the potential of NV354 as a therapeutic candidate.

NV354 has also been tested on skin cells taken from patients with Leigh syndrome, with the results of the study demonstrating that NV354 can bypass complex I and support energy production. To read more:

What is the latest?

In August 2018, the Children’s Hospital of Philadelphia was awarded a $4 million grant from the U.S Department of Defense to advance the development of NV354 with NeuroVive Pharmaceuticals. This extensive research programme will allow the clinical efficiency of the drug to be evaluated further and will hopefully lead to ‘first in human’ trials. To read more:

Any other news?

In June 2018, BridgeBio Pharma made an exclusive agreement with NeuroVive Pharmaceuticals to develop candidate drugs based on NV354 for the treatment of Leber’s Hereditary Optic Neuropathy (LHON). BridgeBio launched Fortify Therapeutics to work on the LHON programme and committed $20 million to the study.

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