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Our pipeline

We are passionate about coupling our deep understanding of the neuroscience of the ear with our drug delivery and development expertise to advance a broad product pipeline to address significant unmet needs in neurotology.

(Target Indication)
Phase 2

OTO-313 is a sustained-exposure formulation of gacycylidine, a potent and selective NMDA receptor antagonist, developed for the treatment of tinnitus. In August 2022, we announced negative top-line results from a Phase 2 clinical trial of OTO-313 in patients with persistent tinnitus of at least moderate severity. Based on these results, we have discontinued the development of OTO-313.

(hearing loss)
Phase 2a

OTO-413 is a sustained-exposure formulation of brain-derived neurotrophic factor (BDNF) in development for the treatment of hearing loss. In April 2022, we announced positive top-line results from a Phase 2a clinical trial of OTO-413, demonstrating that a single intratympanic injection of OTO-413 provided clinically meaningful treatment benefit versus placebo across multiple speech-in-noise hearing tests. We are currently conducting a clinical evaluation of higher dosing of OTO-413 with top-line results expected in the fourth quarter of 2022.

(congenital hearing loss)
GJB2 gene therapy

OTO-825 is a gene therapy targeting mutations in the gap junction beta 2 gene (GJB2), which is the most common cause of congenital hearing loss. Based on preclinical studies including positive results in two proof-of-concept hearing loss models presented at the ASGCT Annual Meeting in May 2021, we have selected OTO-825 as a candidate for clinical development. Together with our corporate collaborator, Applied Genetic Technologies Corporation (AGTC), we have completed a Pre-IND meeting with the FDA, initiated IND-enabling activities, and expect to file an IND for this program in the first half of 2023.


OTO-510 is a product candidate in preclinical development for the prevention of hearing loss due to chemotherapy with cisplatin (CIHL). This novel molecule has demonstrated improved otoprotection in preclinical studies compared to other agents in development, and is being formulated to provide sustained exposure from a single intratympanic injection. The goal of the OTO-510 program is to preserve hearing without protecting the tumor.

(severe hearing loss)

The OTO-6XX program is focused on cochlear hair cell repair for the treatment of severe hearing loss.