Cilcare, a French biotech specializing in auditory sciences, has entered into a significant option agreement with Japanese pharmaceutical company Shionogi & Co., Ltd. for the development of novel treatments for hearing disorders. The deal, announced on June 6, 2024, grants Shionogi exclusive rights to license two promising drug candidates targeting the growing global challenge of hearing loss.
Under the terms of the agreement, Shionogi will make an upfront payment of €15 million (approximately $16 million USD) to Cilcare. If Shionogi exercises its options for both compounds and successfully develops and commercializes them, the total value of the deal could reach approximately €400 million (about $429 million USD), plus royalties on net sales.
Focus on Innovative Hearing Loss Treatments
The agreement centers on two key drug candidates: CIL001 and CIL003. CIL001, originally licensed from Sanofi to Cilcare in 2020, is a disease-modifying small molecule designed to treat cochlear synaptopathy and associated disorders through a single transtympanic administration.
Preclinical studies have demonstrated CIL001's ability to repair inner ear cochlear synapses and improve the first wave of the auditory brainstem response (ABR), which reflects cochlear synapse function. The compound has already generated robust preclinical and clinical safety data, positioning it to enter Phase 2a trials in 2025 in both Europe and the United States.
Meanwhile, CIL003 remains in preclinical development, with studies currently underway to evaluate its potential in addressing hearing disorders.
Shionogi will decide whether to exercise its option rights based on the results of the Phase 2a studies for CIL001 and preclinical data for CIL003, both to be conducted by Cilcare.
Addressing a Critical Global Health Challenge
Hearing loss represents a major global health issue affecting approximately 1.5 billion people worldwide, with projections suggesting that by 2050, a quarter of the global population will experience some degree of hearing deficiency.
Cochlear synaptopathy, also known as "hidden hearing loss," affects approximately 15% of the population. This condition damages the connections between sensory cells and nerve cells in the inner ear, leading to difficulties in speech comprehension despite normal results on standard hearing tests.
The condition is particularly concerning as it has been linked to increased risk of dementia in the elderly and is a significant complication of diabetes. Currently, there are no effective treatments available, highlighting the substantial unmet medical need that this partnership aims to address.
Economic Impact and Strategic Importance
The economic burden of unaddressed hearing loss is estimated at around $1 trillion annually, underscoring the urgent need for innovative therapeutic approaches. By focusing on cochlear synaptopathy, Cilcare and Shionogi are targeting a condition that not only affects quality of life but also has broader implications for cognitive health and healthcare costs.
Célia Belline, Chief Executive Officer of Cilcare, emphasized the company's commitment to advancing therapies for auditory-related diseases through its AI-driven auditory markers known as digital auditory signatures. The partnership with Shionogi represents a strategic alignment with Cilcare's focus on external innovation and collaboration.
For Shionogi, the agreement aligns with its material focus on "Contributing to a Healthy and Prosperous Life" and its commitment to delivering innovative treatments for diseases with high unmet medical needs. The company continues to pursue its vision of creating a society where everyone can lead a longer, more vibrant life.
Development Timeline and Next Steps
Preparatory activities are currently underway to collect auditory data in patients with type 2 diabetes or mild cognitive impairment. From fiscal year 2025, Phase 2a studies are planned to evaluate the safety and efficacy of CIL001 in patients with type 2 diabetes who have cochlear synaptopathy.
The collaboration between Cilcare and Shionogi represents a pivotal step in addressing the urgent global health crisis posed by hearing disorders, potentially offering new hope for millions of individuals worldwide who currently lack effective treatment options.
