Conference Day One

Seeking Improved Outcomes for Patients with Inner Ear Diseases

8:00 am Chair’s Opening Address

8:10 am The War on Hearing Loss

  • Steven Rauch Professor & Vice Chair for Clinical Research, Department of Otolaryngology, Harvard Medical School

Synopsis

• The dominant experience of patients with hearing loss is the sheer effort and frustration of trying to understand verbal communication
• Hearing loss arises from myriad abnormalities throughout the entirety of the auditory system. It is, therefore, unrealistic to expect remedy of any one abnormality to have broad impact across a majority of hearing loss patients
• New treatment will never reach patients unless physicians are willing to administer it and healthcare systems are willing to pay for it

8:40 am Living with Hearing Loss: Challenges & Hopes

  • Brenda Battat Ex Executive Director, Hearing Loss Association of America

Synopsis

• Sharing a personal story of living with profound hearing loss caused by rare genetic mutation – DFNA5.
• An evaluation of the current status of attitudes, tech and delivery systems that impact those suffering with hearing loss
• Speaking from experience of what patients want and don’t want from future therapeutics combating hearing loss

9:00 am Panel Discussion: Looking Beyond 2022 – Future Directions for Inner Ear Therapeutics

  • David Lucchino Co-Founder, President & Chief Executive Officer, Frequency Therapeutics
  • Steven Rauch Professor & Vice Chair for Clinical Research, Department of Otolaryngology, Harvard Medical School
  • Laurence Reid Chief Executive Officer , Decibel Therapeutics
  • Alan Foster Vice President of Research , Otonomy

Synopsis

• Showcasing the future directions in inner ear therapeutics from industry, what are the latest innovations happening in the space in the next 5 years?
• What are the key milestones companies are trying to achieve in 2022/2023 and beyond?
• What are the challenges to come post-regulatory?

9:40 am The Hearing Therapeutics Initiative – Sparking Cross-Sector Collaborations to Bring New Treatments to Patients, Faster

Synopsis

• Building on 2 years of Hearing Medicines Discovery Syndicate: our impact to date
• Our current and future areas of focus: identifying and addressing key sectoral challenges
• Working with the Hearing Therapeutics Initiative: how to engage with us and the support we can offer you

10:00 am Structured Networking & Morning Coffee Break

Synopsis

This session is the perfect opportunity to get face-to-face time with the key opinion leaders in the inner ear disorders space.
Establish meaningful business relationships to build upon for the rest of the conference and gain individual insight into the pioneering work ongoing.

11:10 am Sensorineural Hearing Loss as a Comorbidity of Chronic Inflammatory Diseases

  • Mathieu Schue Project Director, Drug Development & Translational Research, CILcare

Synopsis

• Raise awareness on the increased risk for sensorineural hearing loss as a complication of chronic inflammatory diseases
• Recent advances on identifying the underlying mechanisms
• Clinical perspectives with most promising pharmacological drug classes

11:40 am Panel Discussion: Lessons Learned from Clinical Trials to Enlighten Drug Development for Inner Ear Therapeutics

Synopsis

This panel will be a structured conversation designed to answer key questions such as:
• In 2021 we saw a number of clinical trials advance through all phases of clinical development. This panel will look at what the industry learned from both successes and setbacks to inform how can we use this to improve drug development moving forward
• What is the role of novel mechanisms and combination approaches targeting these diseases and how can we gather clinical data to demonstrate a favorable benefit/risk ratio of novel therapeutics to regulatory bodies?

Overcoming Preclinical & Translational Challenges to Develop Clinically Successful Therapeutics For Inner Ear Diseases

12:10 pm Case Study: Translating Preclinical Research into Successful Clinical Trials: The Story of SPI-1005

  • Jonathan Kil Chief Executive Officer, Sound Pharmaceuticals

Synopsis

• Optimizing drug target studies
• Showcasing phase 2 study populations
• Pivotal Phase 3 Protocol design and considerations

12:40 pm Panel Discussion: De-risking the Early Development Process a Robust & Comprehensive Early Development Process is Essential to Minimize Setbacks Faced in the Clinic.

Synopsis

This panel will be a structured conversation designed to answer key questions such as:
• What metrics should we be measuring in the lab to predict good outcomes in the clinic?
• Which animal models are the best predictors of success in the clinic?
• How do we pick the right approaches, the right drugs, the right doses before attempting the clinical development?
• What factors do we need to consider choosing the right approach, candidate, and dosing regimen preclinically before moving into clinical development?

1:10 am Lunch Break

2:40 pm Inner Ear Therapeutics By Design

Synopsis

• Pay attention to physicochemical properties by design for successful drug discovery
• Understand drug distribution and target engagement
• Design appropriate pharmacokinetic-pharmacodynamic relationship

3:10 pm Enhancing the Predictability of Animal Models for Inner Ear Therapeutics

  • Fabrice Piu Senior Vice President - Research & Pre-clinical Development, Otonomy

Synopsis

• The use of ex vivo cochlear tissue-based assays to improve drug selection for inner ear therapeutics
• Optimizing the pharmacokinetic and pharmacodynamic product profile for clinical evaluations using animal models
• Review of case studies for hearing loss and tinnitus therapeutics demonstrating translation of preclinical findings into clinical proof-of-concept

3:40 pm Primate models and Remote Clinical Trials: Strategy and Tactics for Minding Species Gaps in between Rodents and Human

  • Masato Fujioka Professor & Chair, Department of Molecular Genetics & Department of Otorhinolaryngology- HNS, Kitasato University School of Medicine

Synopsis

  • Pharma history reminds us that the deepest valley of death lies in around Phase I/II trial.
  • Primate models may avoid from “unexpected” failure at the stage due to the species differences.
  • Human iPSC-based in vitro model can be utilized at any stage of the drug development; target ID, choice in the synthetic development and estimate of minimal effective concentration.
  • In vivo non-human primate models offer a comprehensive and easy-handling reliable platform.
  • Daily monitoring of hearing and balance in the early phase of clinical trial by remote data collection may reveal us “real-world” needs of patients, which should be reflected to the pipeline selection, choice of target molecules and clinical endpoints of the trials.

4:10 pm Harnessing the Power of Artificial Intelligence to Transform Hearing Research & Healthcare

  • Nishchay Mehta Associate Professor - UCL, Scientific consultant - ENT Clinical

Synopsis

• What role can big data, machine learning and optimized algorithms play in advancing preclinical and clinical research?
• Where to find and access hearing data?
• How can we understand big data sets and what role can small datasets play

4:40 pm Refreshment Break

Ensuring Success in Clinical Development

5:00 pm Clinical Trial Design Considerations for Inner Ear Therapeutics

  • Carl LeBel Chief Development Officer, Frequency Therapeutics

Synopsis

• Protocol design considerations for hearing loss trials
• Choice of outcome measures and establishing a clinically meaningful improvement level
• Patient selection strategies to optimize target population and patient homogeneity

5:30 pm Addressing the Lack of Clinically Validated Endpoints

  • Colleen Le Prell Professor of Hearing Science, Chair, Department of Speech, Language, & Hearing, University of Texas at Dallas

Synopsis

• Qualitative and quantitative endpoint options
• Matching endpoints to indications
• Standardization of functional measures

6:00 pm Panel Discussion: Optimizing Functional Testing & Outcomes Measures in Clinical Trials

  • Carl LeBel Chief Development Officer, Frequency Therapeutics
  • Douglas Hartley Professor of Otology & Consultant ENT surgeon, School of Medicine, University of Nottingham
  • Chieri Hayashi Medical Director, Astellas

Synopsis

This panel will be a structured conversation designed to answer key questions such as:
• What is the correct choice of outcome measures for novel hearing therapeutics?
• What are the early signals of efficacy and how are functional changes in hearing best measured?
• Evaluating the rationale behind why companies have chosen to opt for certain outcome measures over others

6:30 pm Chair’s Closing Address

6:35 pm Scientific Poster Session

Synopsis

As the landscape of innovative discovery and development efforts to target inner ear disorders for therapeutic application expands, it is more important than ever to collaborate and learn with your peers as we continue to advance therapies through preclinical and clinical development. Join our dedicated session to share your latest data, continue networking and have a first look about what your peers
are working on!