Sound Pharmaceuticals Receives Breakthrough Therapy Designation for SPI-1005 for Meniere’s Disease
Tuesday, December 02, 2025
Sound Pharmaceuticals (SPI) has announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to its investigational drug SPI-1005 for the treatment of hearing loss associated with Meniere’s disease (MD).
Meniere’s disease is a complex inner ear disorder characterised by low-to-mid frequency hearing loss, tinnitus, vertigo and dizziness. There are currently no FDA-approved drug treatments for MD, making this the first therapy to receive BTD for the condition. SPI-1005 is also the first drug to gain BTD for sensorineural hearing loss, which is the most common age-related hearing disorder and is assessed through pure-tone audiometry (PTA) and speech discrimination tests such as words-in-noise (WIN).
SPI-1005 is an investigational therapy containing ebselen, a novel compound that mimics and enhances the activity of glutathione peroxidase (GPx). GPx1 plays a key role in repairing damaged or ageing cells in the inner ear, retina, brain, lungs and kidneys, and its activity is known to decline with exposure to noise, ototoxic medicines or ageing. Reduced GPx function can lead to neuroinflammation, neurodegeneration or maladaptive changes in the nervous system.
SPI-1005 is being developed for multiple neurotologic disorders, including Meniere’s disease, noise-induced hearing loss and various forms of ototoxicity caused by aminoglycoside antibiotics or platinum-based chemotherapy. Across 13 completed clinical studies, 790 patients have been treated with SPI-1005, with over 400 additional participants expected to be enrolled in ongoing trials over the next year. No significant drug-drug interactions have been reported to date across different study populations, including patients with cystic fibrosis, bipolar mania and treatment-resistant depression.
SPI-1005 met its co-primary endpoints in a pivotal Phase 3 randomised, double-blind, placebo-controlled study (STOPMD-3), demonstrating clinically meaningful improvements in both PTA and WIN results. A second open-label Phase 3 study is currently enrolling to gather additional long-term safety data for use in patients with probable and definite MD. The drug has previously shown favourable safety and efficacy outcomes across five separate randomised controlled trials, including Phase 1b and 2b studies in MD, a Phase 2 trial in acute noise-induced hearing loss, and another Phase 2 study in aminoglycoside-induced ototoxicity.
The FDA has also granted Fast Track Designation to SPI-1005 following completion of a Phase 2b trial in MD. While both designations apply to serious or life-threatening conditions, Breakthrough Therapy status is reserved for drugs that demonstrate substantial improvement over existing treatments, whereas Fast Track status focuses on therapies with the potential to address unmet medical needs.
Source: soundpharma.com
