Spotlight on hearing therapeutics blog series: commercialising hearing research

As part of this, we will be publishing a series of blogs focussing on hearing therapeutics from different sector perspectives.

In the second blog, Simon Chandler, CEO at Rinri Therapeutics, shares his thoughts on what he perceives to be some of the major opportunities and challenges when it comes to commercialising hearing research. 

Opportunities and challenges in developing novel therapeutics for hearing loss

Hearing loss is a significant global health issue, affecting over half a billion people worldwide—a figure expected to double by 2050 due to ageing global populations. This presents an unparalleled opportunity in drug development to enhance the quality of life for millions globally. Currently, the standards of care for hearing loss—hearing aids and cochlear implants—only palliate symptoms. These treatments fail to restore natural hearing, leaving many patients with unmet medical needs and suboptimal outcomes. To date, no biological or pharmaceutical treatment is available for hearing loss; leaving a vast white space for therapeutics developers.

Rinri Therapeutics, a pioneering UK Biotech, was established to bridge this critical gap in hearing loss treatment. Led by Professor Marcelo Rivolta from the University of Sheffield, our innovative approach is to use advanced regenerative medicine to restore hearing. Our flagship product, Rincell-1, specifically targets auditory neurons, addressing neural hearing loss, a significant contributor to Sensorineural Hearing Loss (SNHL), including age-related hearing loss known as Presbycusis. This unique approach marks a significant advancement in the field, addressing a widespread and pressing health challenge.

Challenges

Therapeutic development, one of the most challenging activities in modern society, is a complex and lengthy process. Regardless of the indication or modality, the development cycles are long, the complexity is high, and the inherent uncertainties and risks are significant. It requires immense effort, commitment, and financial support to bring new therapeutics to patients. This is a reality that Rinri Therapeutics, like all other therapeutic development programs, must navigate.

All therapeutic development programs that start from fundamental academic research are characterised by unique opportunities, advantages, challenges, and idiosyncrasies. Hearing loss therapeutic development is no different.

The cochlea, the functional organ in human hearing, is not easily accessible for therapeutic delivery as is located behind one of the hardest bones in the human body. Unlike our oncology or even ophthalmology colleagues who can rely on soft tissue injections, delivery of therapeutics to the inner ear requires developers to design completely new routes of administration.

Measuring hearing health and efficacy presents challenges for developers, particularly for effective clinical trial design. There are no blood biomarkers for hearing health, and traditional measures of hearing performance, like word recognition scores, are not ideal for clinical research as they have been set up to detect the loss of hearing rather than the regain of function.

Although the patient populations with hearing loss are substantial, this presents significant challenges for developers of advanced therapies, in how to scale up to meet these needs. Historically, the development of Advanced Therapy Medicinal Products (ATMPs) has primarily targeted rare and orphan diseases, which inherently affect fewer patients. Hearing loss not only carries social and ethical responsibilities but also raises concerns about ensuring equality and access to treatment.

At Rinri, we have tackled these challenges head-on to progress our therapy to clinical trials. We have developed new delivery techniques and novel, powerful, objective measures of hearing health, resulting in powerful trial designs. Recognising the importance of accessibility early, we are leading the ATMP community in developing cell therapy manufacturing processes to meet larger patient numbers in more common conditions.

Opportunities

To date, hearing loss treatments have primarily been device-based, with the clinical field yet to fully embrace New Chemical Entities (NCEs) or biologics, much less the advanced therapies that Rinri is pioneering. However, we are now entering what could be described as a golden era for hearing loss therapeutic development. This shift is driven by a rapidly deepening understanding of the biology of hearing loss, alongside advancements in technology, clinical expertise, and regulatory flexibility, creating a prime opportunity for significant breakthroughs.

The clinical community specialising in hearing loss has recognised and seized this opportunity, working closely with developers to overcome hurdles and expedite the development of new treatments. Additionally, communities affected by hearing loss are proving to be extraordinarily engaged and supportive, understanding that developers need patient input to grasp the true impact of hearing loss and to ensure our therapeutic and clinical strategies effectively address their needs.

The regulatory landscape in the UK, overseen by the MHRA, is now particularly supportive of developing novel treatments for conditions like hearing loss. The regulatory environment is now characterised by a willingness to adopt flexible and pragmatic approaches, which are crucial for fostering innovation and facilitating rapid progress in therapeutic development for hearing loss.

Rinri has benefited enormously from robust interdisciplinary partnerships with leading universities, hospitals, and device manufacturers. We have also taken advantage of multiple formal MHRA scientific advice meetings and were awarded a passport to their Innovative Licensing and Access Pathway (ILAP) accelerator for high-impact and high-potential therapeutics. These collaborations have been essential in Rinri’s successful navigation of challenges, mitigation of risks, and acceleration of the development of our therapeutics.

Conclusion

The landscape for developing hearing therapies is brimming with possibilities, driven by deeper insights into hearing biology, collaborative ventures with clinical communities, and a supportive regulatory framework. Nonetheless, the path is marked by substantial challenges to effectively translate scientific discoveries into clinical therapeutics.  Rinri is actively addressing these obstacles by innovating delivery methods, crafting novel clinical trial frameworks, and enhancing manufacturing processes to broaden the impact of our pioneering treatments. We have capitalised on these emerging opportunities and successfully navigated challenges to make swift preclinical progress. We are now on track to initiate clinical trials in the UK within the next 18 months, aiming to be the first to provide a broad therapeutic solution for the hearing loss community.

Now is an unprecedented time to tackle hearing loss. I urge researchers, pharmaceutical firms, and investors in this domain to champion the advancement of innovative therapeutics for hearing loss that promises to revolutionise the lives of millions.

Want to find out more?

If you enjoyed this blog and want to find out more about hearing loss therapeutics, why not join our Hearing Therapeutics Initiative or contact us directly to discuss how the HTI network could support you and your innovation.