News briefing: Frequency posts extended positive results for hearing loss program; Kiniksa earns ODD for GCA candidate
Frequency Therapeutics presented results from a longer-term durability study of its acquired sensorineural hearing loss candidate, saying FX-322 showed significant improvement for some patients in key measures of hearing that were sustained for up to 21 months.
The data build upon Phase I/II results from 2019 where the program demonstrated statistically significant improvements in word recognition scores in patients with chronic SNHL, Frequency said. That study lasted for only 90 days, whereas Tuesday’s new data were part of subsequent testing of a subset of these patients between 13 and 21 months after initial dosing.
Acquired SNHL typically results from damage to the hair cells of the inner ear. These auditory sensory hair cells may be lost due to chronic noise exposure, aging, certain viral infections or exposure to drugs that are toxic to the ear. FX-322 is still being studied in a Phase IIa trial to determine appropriate dosing levels and confirm the earlier results.
Kiniksa nets ODD for giant cell arteritis program
The FDA has granted a new orphan drug designation to a program from Kiniksa Pharmaceuticals.
Tuesday’s winner is mavrilimumab, a monoclonal antibody inhibitor being studied to treat giant cell arteritis, an inflammatory disease of medium-to-large arteries. The compound targets granulocyte macrophage colony stimulating factor receptor alpha, or GM-CSFRα.
Chronic GCA in the head area can typically result in recurrent headaches and jaw cramping, as well as constitutional symptoms of fever and fatigue. The condition can also result in permanent vision loss from diminished blood flow to the eye. The large vessel form of GCA affects the branches of the aorta supplying blood to the rest of the body.
Mavrilimumab is currently in Phase II worldwide studies in GCA and pneumonia related to Covid-19.