The ultimate goal of the proposed project is to evaluate and commercialize a combination of proprietary magnet assemblies and magnetic nanoparticles (initially developed by our University STTR partner), that has been shown in preliminary animal studies to improve delivery of medications to the inner ear. Initial animal studies using the system (which uses magnetic forces to inject steroid-eluting magnetic nano-particles into the inner ear) have successfully reduced the degree of hearing loss and tinnitus in rats due to acoustic trauma. These initial studies suggest that the amount of steroids reaching the inner ear is increased ten-fold (compared to the current standard of trans-tympanic injection into the middle ear), without side effects. Our strategy is to demonstrate the utility of the magnetic injection system as a platform technology addressing the $4 billion hearing market, starting with a compelling clinical problem that currently is without a cure. Sudden Sensorineural Hearing Loss (SSHL) is an orphan disease without either a clear cause or a satisfactory therapy, and is considered to be an otologic emergency. Current therapies, which include oral and trans- tympanic steroid injection into the middle ear, are only partially effective. There is n widely-recognized animal model for SSHL, and as a result, we have been advised by regulatory experts that we would need to proceed directly to an investigational new drug (IND) application for human testing after showing improved drug delivery and safety in animals. We plan to advance in step-wise fashion by performing proof-of-principle and toxicity studies in the Phase I portion of the STTR project, and then conducting comprehensive pharmaco- kinetic studies in the STTR Phase II portion in preparation for the IND. The current standard of care in SSHL is to inject steroids into the middle ear. We expect that with preclinical demonstrations of low toxicity and high delivered more-uniform inner-ear concentrations using the magnetic injection system, the FDA would approve an IND augmenting trans-tympanic needle injections with magnetic injection of drug-eluting particles. Eventually we anticipate that the magnetic injection platform technology will be approved for other ear, nose and throat and CNS conditions. In Phase I, we will demonstrate the superiority of magnetic injection in attaining and maintaining physiologically-effective concentration of active ingredient in the inner ear (with low serum concentration). We will collect toxicity data for expected levels of steroid-loaded nanoparticles, quantifying levels of apoptotic and inflammatory responses in-vitro. With animal studies we will show significantly improved drug delivery and cochlear drug uniformity, and will demonstrate non-significant incidences of morbidity, mortality, and ototoxicity in-vivo. In Phase II, we will attempt to conduct rigorous pharmacokinetic studies and work with a strategic partner in the (fairly profitable) Orphan drug market, in preparation for IND submission.
Our overall goal is to improve treatment outcomes for patients with sudden hearing loss using a proprietary drug delivery system. Magnetic injection will be used to minimally- invasively deliver drugs to the inner ear, to improve drug concentration and uniformity.