Medicines discovery for auditory disorders: Challenges for industry.

Area of Science:

• Otolaryngology and Pharmaceutical Sciences
• Drug Discovery and Development

Background:

• No approved pharmaceutical treatments currently exist for preventing or treating hearing loss.
• The pharmaceutical industry exhibits historically low success rates, with a 90% failure rate for drugs entering clinical evaluation.

Purpose of the Study:

• To review current strategies and challenges in developing medicines for hearing loss and tinnitus.
• To highlight initiatives aimed at improving the success rate of drug discovery and clinical translation.

Main Methods:

• Analysis of advancements in target selection, clinical candidate selection, and clinical study design.
• Review of therapeutic strategies for hearing protection, restoration, and tinnitus treatment.
• Discussion of improved diagnostics and methodologies for assessing inner ear drug delivery and pharmacokinetics.

Main Results:

• Initiatives focus on data-informed decisions to translate preclinical findings into effective medicines.
• Emphasis on selecting optimal biological targets, appropriate chemical entities, and correct patient cohorts.
• Investigational drug examples illustrate key discovery challenges and applied learnings.

Conclusions:

• Improved diagnostics and in-ear measurement methodologies are crucial for patient selection and assessing treatment efficacy.
• Strategic advancements in drug discovery aim to increase the probability of successfully launching new medicines for hearing disorders.