Mitochondrial Pharmacology

Question 1

What type of drug is Idebehone (Raxone)?

Answer 1

• OXPHOS modulator
• Facilitates the transfer of electrons from complex 2 to complex 3

Question 2

What disease has Idebehone received fast track and orphan drug designation for?

Answer 2

Duchenne Muscular Dystrophy (DMD)
- received fast track and orphan drug designation by FDA for treatment of DMD in patients NOT using glucocorticois

Question 3

What is Imeglimin?

Answer 3

OXPHOS modulator

Question 4

For was disease state has Imeglimin shown efficacy in clinical trials?

Answer 4

Type 2 Diabetes
- targeting and correcting mitochondrial dysfunction in pancreas, liver and skeletal muscle in T2D

Question 5

What is MOA of Imeglimin?

Answer 5

Precise mechanism not fully understood.
- complex inhibitor of Complex 1 and restores hyperglycaemia-induced reduction in complex 3 content and activity

Question 6

What do antioxidants do?

Answer 6

Decrease the amount of reactive oxygen species being produced

Question 7

What is Nicotinimide’s MOA

Answer 7

Replenish NAD+ stocks

Question 8

What is Leriglitazone?

Answer 8

Pioglitazone metabobolite

Question 9

What does Pioglitazone do?

Answer 9

Increases PGCIalpha expression leading to an up-regulation of mitochondrial biogenesis

Question 10

What disease is Ventoclax used for?

Answer 10

Chronic Lymphocytic Leukemia

Question 11

What is Ventoclax MOA

Answer 11

Induces apoptosis

Question 12

What is Sonlicromanol’s MOA

Answer 12

Alters mitochondrial cell signalling - targets the Trx system
- orphan drug designation
- used to treat maternally inherited diabetes and deafness