MT-TL1

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

Khondrion announces sonlicromanol Phase IIb progress supporting Phase III development in MELAS spectrum disorders

Retrieved on: 
Tuesday, November 22, 2022

Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.

Key Points: 
  • Sonlicromanol, Khondrions wholly-owned lead asset, is being investigated in the Phase IIb programme in adult patients with MELAS spectrum disorders as genetically confirmed by the m.3243A>G mutation in the mitochondrial DNA.
  • In line with previous studies, sonlicromanol was found to be safe and well tolerated in the Phase IIb programme, with no serious adverse effects.
  • These longer-term patient data are, therefore, instrumental in providing valuable insights for the design of the upcoming Phase III trial.
  • One of the most advanced disease-modifying drug candidates for mitochondrial disease in development, sonlicromanol has recently completed a Phase IIb study in adult patients with m.3243A>G MELAS spectrum disorders.

Khondrion announces first patients dosed in 6-month paediatric Phase II study of sonlicromanol for mitochondrial diseases

Retrieved on: 
Wednesday, April 21, 2021

First patients are being dosed at the Radboud Centre for Mitochondrial Medicine, Nijmegen, The Netherlands.

Key Points: 
  • First patients are being dosed at the Radboud Centre for Mitochondrial Medicine, Nijmegen, The Netherlands.
  • Despite advances in the understanding of mitochondrial disorders, treatment options are extremely limited and, to date, largely consist of supportive care.
  • A Phase IIb clinical trial in adult patients with MELAS (mitochondrial encephalopathy, lactic acidosis, and stroke-like episodes) spectrum disorders is ongoing, investigating the effect of sonlicromanol on cognitive functioning.
  • It has also been granted a Rare Pediatric Disease (RPD) designation in the US for the treatment of MELAS.\n'

Cyclerion Therapeutics Reports Third Quarter 2020 Financial Results and Recent Corporate Updates

Retrieved on: 
Thursday, November 5, 2020

Cyclerion expects to begin enrolling its Phase 2 clinical trial in patients with Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) this quarter.

Key Points: 
  • Cyclerion expects to begin enrolling its Phase 2 clinical trial in patients with Mitochondrial Encephalomyopathy, Lactic Acidosis, and Stroke-like episodes (MELAS) this quarter.
  • Cyclerion intends to complete its analysis of the study results and present or publish them in a future medical forum.
  • General and Administrative Expenses:General and administrative expenses wereapproximately $8.0 millionfor the third quarter of 2020, as compared toapproximately $7.1 millionfor the third quarter of 2019.
  • Net Loss:Net loss wasapproximately $18.8 millionfor the third quarter of 2020, as compared to$27.3 millionfor the third quarter of 2019.