According to a Town News press release, Sound Pharmaceuticals, a biopharmaceutical company based in Seattle, received FDA approval for their Phase 3 clinical trial on SPI-1005. The trial, STOPMD-3, is the first of its kind to look at an investigational new drug for the treatment of patients with Meniere’s disease. Additionally, if found to be effective, SPI-1005 would become the first FDA-approved treatment for this disorder.

Meniere’s Disease

Doctors aren’t entirely sure what causes Meniere’s disease, a rare disorder that impacts the inner ears. However, either inner ear inflammation or the presence of an abnormal amount of fluid (endolymph) may be the cause. Your inner ear is responsible for hearing and for balance. When fluid builds up in the ear, it causes pain, dizziness, and pressure. Fluid may accumulate because of allergies, an immune reaction, viral infections, migraines, or genetics.

Symptom onset usually occurs between one’s 40s and 50s. However, symptoms often vacillate between “remission” and “attacks.” When experiencing a Meniere’s disease episode or attack, symptoms include:

• Tinnitus (ringing in the ear)
• Vertigo
• Hearing loss
• Ear pressure
• Imbalance
• Involuntary eye movements
• Headaches or migraines
• Nausea and vomiting

There is no cure for Meniere’s disease. Rather, treatments include low-sodium diets, diuretics, steroids, or surgery.

Learn more about Meniere’s disease here.

SPI-1005

SPI-1005 is an investigational drug. According to the FDA, this means that the drug is still being studied to determine its safety, efficacy, and tolerability. However, the FDA also notes that investigational drugs may be good options for patients who have:

experienced side effects that are too severe to continue taking [approved drugs], limited treatment options available, heard about promising early study results for a specific investigational drug, or [have] no approved drugs available to treat [a rare] disease or medical condition.

In this case, SPI-1005 is an orally-administered drug containing ebselen, a selenorganic compound that mimics glutathione peroxidase (GPx). GPx plays a role in inner ear, retina, kidney, brain, lung cell, and tissue health. But cells and tissues can degenerate or become damaged if GPx activity is low. In some cases, low GPx activity has been linked to hearing loss.

As a result, ebselen use could prevent cellular harm and inhibit hearing loss for patients with Meniere’s disease. The National Library of Medicine also notes that ebselen displays:

anti-inflammatory, anti-oxidant and cytoprotective activity.

The FDA considers ebselen to be a new chemical entity (NCE), which you can learn more about in the FDA’s published guide.

Phase 3 Trial: Treating Meniere’s Disease

The Phase 3 clinical trial was approved following positive results from SPI-1005’s Phase 1b and Phase 2b clinical trials. In these randomized and placebo-controlled studies:

• 39 patients participated in the Phase 1b trial.
• 126 patients participated in the Phase 2b trial.
• Patients were either given a placebo or SPI-1005 for 21 or 28 days (approximately 3-4 weeks).
• Researchers based results on pure-tone audiometry, the words-in-noise tests, the Tinnitus Functional Index (TFI), and the Vertigo Symptoms Scale (VSS).
• In the Phase 1b trial, SPI-1005 significantly reduced hearing loss and tinnitus in patients with Meniere’s disease as compared to those receiving a placebo.
• In the Phase 2b trial, non-placebo patients received 400mg SPI-1005 for 8 weeks.
  • Based on pure-tone audiometry results, 47% of patients showed auditory improvements within 1 month. This rose to 61% of patients by 8 weeks.
    • Improvements = at least ≥10 dB gain at one low frequency
  • Based on words-in-noise tests, 57% of patients improved within 1 month, which rose to 68% by 8 weeks.
    • Improvements = at least ≥20% increase in word recognition
• SPI-1005 also reduced tinnitus significantly (30%) compared to the placebo (10%).

In addition to the upcoming Phase 3 trial, SPI-1005 is also being tested in a Phase 2 trial for patients with cystic fibrosis.

What are your thoughts on this trial? Share your stories, thoughts, and hopes with the Patient Worthy community!