Sound Pharmaceuticals Receives FDA Breakthrough Therapy Designation for SPI-1005 to Treat Meniere’s Disease

SEATTLE--(BUSINESS WIRE)--Sound Pharmaceuticals (SPI) is pleased to announce that the FDA has granted its investigational new drug, SPI-1005, Breakthrough Therapy Designation (BTD) for the treatment of hearing loss in patients with Meniere’s disease (MD). MD is a complex inner ear disease that involves low-to-mid frequency hearing loss, tinnitus, vertigo, and dizziness. This is the first BTD granted for the treatment of MD, for which there are no FDA approved drug treatments. Definite MD requires audiometric documentation of low-to-mid frequency hearing loss while probable MD does not. In addition, SPI-1005, an oral anti-inflammatory, is the first drug to receive BTD involving the treatment of sensorineural hearing loss, the most common disease of aging and of the peripheral and central nervous system. Sensorineural hearing loss is documented by pure-tone audiometry (PTA) and speech discrimination testing including the words-in-noise (WIN) test which together serve as the gold standards for determining hearing impairment and other auditory dysfunction. “We look forward to working closely with the FDA to get SPI-1005 ready for a future NDA filing,” said Dr. Jonathan Kil, Co-Founder and CEO of SPI.

SPI-1005 is the first drug to achieve its co-primary endpoints, involving clinically relevant improvements in PTA and WIN, in a pivotal Phase 3 randomized, double blinded, placebo-controlled trial (RCT with Open Label Extension) to treat Meniere’s Disease (STOPMD-3). A second open-label Phase 3 is currently enrolling to accrue additional safety data to allow for the chronic dosing of patients with probable and definite MD. SPI-1005 has also achieved favorable safety and efficacy results in five different RCTs involving Phase 1b & 2b RCTs in MD, a Phase 2 RCT in acute noise-induced hearing loss, and a Phase 2 RCT in aminoglycoside-induced ototoxicity.

SPI-1005 also received Fast Track Designation for the Treatment of MD after completing a Phase 2b in MD. According to the FDA, “A breakthrough therapy designation is for a drug that treats a serious or life-threatening condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement on a clinically significant endpoint(s) over available therapies. In contrast, a fast track designation is for a drug that treats a serious or life-threatening condition, and nonclinical or clinical data demonstrate the potential to address unmet medical needs for the serious condition.”

About SPI-1005

SPI-1005 is an investigational new drug that contains ebselen, a new chemical entity that mimics and induces glutathione peroxidase (GPx) activity. GPx1 is a critical enzyme that repairs injured and aging cells in the inner ear, retina, prefrontal cortex, lung, and kidney, and is reduced during and after exposure to environmental insults such as noise, drugs, or aging. Consequently, neuroinflammation can progress through the peripheral and central nervous system leading to neurodegeneration and/or maladaptive plasticity in the brain. SPI-1005 is being developed for several neurotologic indications beyond MD including noise-induced hearing loss (including tinnitus) and two types of ototoxicity (hearing loss, tinnitus, dizziness, or vertigo) caused by aminoglycoside antibiotics (including tobramycin/amikacin) and platinum-based chemotherapy (including cisplatin/carboplatin). To date, no significant drug-drug interactions have been observed across multiple study populations including MD, cystic fibrosis, bipolar mania, and treatment-resistant depression. Thirteen completed SPI-1005 trials have enrolled more than 790 patients with 400+ more enrolled patients anticipated over the next year.

About Sound Pharmaceuticals

Sound Pharma is a private biotechnology company studying SPI-1005 under five active INDs involving several neurotologic indications. Details of the SPI-1005 clinical trials can be viewed online at www.clinicaltrials.gov or www.soundpharma.com. Please contact [e-mail] for further information.