News

KH176 by Khondrion had a favorable safety profile and improved depression and migraine in some forms of mitochondrial disease, according to preliminary results from an ongoing Phase 2 trial. The study included carriers of a gene defect called m.3243A>G who have MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) and…

Defective cellular energy production by mitochondria can result from abnormal expression of enzymes involved in the synthesis of the essential co-factor coenzyme Q, a study with mice revealed. This finding resulted from the collaborative work between researchers at the Max Planck Institute for Biology of Ageing, in Germany, and at…

The National Organization for Rare Disorders (NORD) says it’s “disappointed and dismayed” after the House of Representatives voted 227-205 last week to repeal the Orphan Drug Tax Credit as part of a U.S. tax reform package. A similar package before the Senate Finance Committee does not repeal the credit…

The U.S. Patent and Trade Mark Office (USPTO) sent a notice of allowance — meaning it intends to issue a patent — to Mitochon Pharmaceuticals regarding the clinical development of its investigative drug MP101 for the treatment of several mitochondrial-related diseases, including Huntington’s disease and Duchenne muscular dystrophy, among others.

Researchers found that patients with different types of mitochondrial disease have higher rates of headaches compared to the general population. Tension-type headaches (TTH) were more frequent than migraines among this patient population, the study found. The study, “Prevalence of Headache in Patients With Mitochondrial Disease: A Cross-Sectional Study,” was…

NeuroVive Pharmaceutical has received a signal from European regulators that its mitochondrial disease therapy KL1333 is likely to receive Orphan Drug Designation, which could accelerate its development and approval. The company is seeking Orphan Drug Designation for KL1333 as treatment for a condition known as mitochondrial myopathy, encephalopathy, lactic acidosis and stroke-like…

Khondrion has secured three new patents that protect several products, including the Dutch pharmaceutical company’s lead candidate, KH176, to treat mitochondrial disease and related conditions. KH176 is a small molecule that targets reactive oxygen species, or byproducts of mitochondria. The compound belongs to a new class of drugs that…

Mitobridge’s investigative PPARδ modulator MA-0217 has been shown to improve mitochondria activity and kidney function in animal models of acute kidney injury. The findings were the subject of two poster presentations titled, “Modulation of PPARδ with MTB-2 Post-Reperfusion Attenuates IR-Induced AKI Injury Biomarkers and Histopathology in…

Presenilin-2 (PS2) plays a crucial role in ensuring mitochondria work as intended in a cell, and may contribute to mitochondrial dysfunction in patients with Alzheimer’s disease (AD), new research reports. The study “Presenilin 2-Dependent Maintenance of Mitochondrial Oxidative Capacity and Morphology” was published in the journal Frontiers in…

Researchers have discovered that mitophagy – the selective degradation of damaged mitochondria – allows liver cancer stem cells to survive and contribute to liver tumor growth. Their findings suggest that halting mitophagy temporarily is a potential new therapeutic target for liver cancer. The study “Mitophagy Controls the…