SEATTLE, WASHINGTON — Sound Pharmaceuticals (SPI) announced that the U.S. Food and Drug Administration (FDA) has granted its investigational drug, SPI-1005, Breakthrough Therapy Designation (BTD) for the treatment of hearing loss associated with Meniere’s disease (MD). MD is a complex inner ear disorder involving low-to-mid frequency hearing loss, tinnitus, vertigo, and dizziness. This marks the first BTD granted for a treatment targeting MD, which currently has no FDA-approved drug therapies.
SPI-1005, an oral anti-inflammatory, is also the first drug to receive BTD for the treatment of sensorineural hearing loss, the most common form of hearing impairment and a significant contributor to age-related and neurologic decline. Sensorineural hearing loss is evaluated using pure-tone audiometry (PTA) and speech discrimination measures such as the words-in-noise (WIN) test, which are considered diagnostic standards.
“We look forward to working closely with the FDA to get SPI-1005 ready for a future NDA filing”
–Dr. Jonathan Kil, Co-Founder and CEO of SPI
SPI-1005 is the first drug to meet its co-primary endpoints—showing clinically meaningful improvements in PTA and WIN—within a pivotal Phase 3 randomized, double-blind, placebo-controlled trial (STOPMD-3), which included an open-label extension. A second open-label Phase 3 study is enrolling to gather additional safety data supporting chronic dosing for patients with probable and definite MD.
The drug has also shown favorable safety and efficacy results in multiple randomized controlled trials, including Phase 1b and 2b studies in MD, a Phase 2 trial in acute noise-induced hearing loss, and a Phase 2 trial in aminoglycoside-induced ototoxicity.
SPI-1005 previously received Fast Track Designation for the treatment of MD following completion of a Phase 2b trial. The FDA describes Breakthrough Therapy Designation as applying to drugs that treat serious or life-threatening conditions where preliminary clinical evidence indicates substantial improvement over available therapies. Fast Track Designation applies to drugs with the potential to address unmet medical needs in similarly serious conditions.
About SPI-1005
SPI-1005 is an investigational drug containing ebselen, a new chemical entity that mimics and enhances glutathione peroxidase (GPx) activity. GPx1 plays a key role in repairing cellular injury in organs including the inner ear, retina, prefrontal cortex, lung, and kidney. Reduced GPx1 activity following noise exposure, drug toxicity, or aging may contribute to neuroinflammation and neurodegenerative processes.
SPI-1005 is being developed for several neurotologic conditions beyond MD, including noise-induced hearing loss (with and without tinnitus) and forms of ototoxicity caused by aminoglycoside antibiotics (e.g., tobramycin/amikacin) and platinum-based chemotherapies (e.g., cisplatin/carboplatin). No significant drug-drug interactions have been observed across studies involving MD, cystic fibrosis, bipolar mania, and treatment-resistant depression. To date, 13 completed trials have enrolled more than 790 participants, with more than 400 additional participants expected over the next year.
About Sound Pharmaceuticals
Sound Pharmaceuticals is a private biotechnology company developing SPI-1005 under five active Investigational New Drug (IND) applications across multiple neurotologic indications. Information about SPI-1005 clinical trials is available at www.clinicaltrials.gov and www.soundpharma.com.
Source: Sound Pharmaceuticals
