Conference Day Two

8:15 am Morning Networking

8:50 am Chair’s Opening Remarks

Uncovering the Challenges & Opportunities in Otology to Develop Therapies for Hearing Loss

9:00 am Targeting the α9α10 Nicotinic Acetylcholine Receptor for Hearing Loss

Synopsis

• Medial olivocochlear efferents provide novel approach to hearing disorders
• Selective expression of α9α10 at terminal MOC synapse provides a compelling target
• The recent functional reconstitution α9α10 in recombinant cells enables drug discovery

9:25 am Sharing Astellas’ Approach to Otology: Challenges & Opportunities

Synopsis

• Session details to be confirmed

9:50 am ORC-13661 is Novel, First in Class Drug to Prevent Aminoglycoside-Induced Hearing Loss

  • Vince Groppi Chief Executive Officer , Oricula Therapeutics

Synopsis

• An innovative phenotypic screening strategy was used to identify a lead chemical series and carry out directed SAR to identify ORC-13661 as a clinical candidate
• Parallel in vivo efficacy and mechanism of action studies established that ORC-13661 confers robust protection from aminoglycoside induced hearing loss by blocking drug entry into hair cells without affecting normal hearing
• IND enabling and Phase 1 clinical studies established that ORC-13661 is safe at exposures needed for efficacy. Proof-of-concept clinical trials in Cystic Fibrosis and Nontuberculous Mycobacterial patients are planned to establish efficacy

10:15 am Expanding Drug Portfolio in Hearing, with Fast-Track Development Strategies

Synopsis

  • Leveraging pharma discovery engine to accelerate innovation in hearing health
  • Repurposing & co-developing strategies to rapidly advance promising assets to market

10:30 am Discussion/Q&A

11:00 am Virtual Speed Networking

11:05 am Morning Break

Optimizing Clinical Outcomes to Gain Regulatory Approval

11:35 am Bringing a New Small Molecule to Patients for Sudden Sensorineural Hearing Loss (SSNHL) Disease

Synopsis

• Preclinical development results
• Clinical trial design: selection of endpoints and population
• Clinical trial execution: challenges

12:00 pm Early Clinical Trial Design Considerations in Hearing Restoration

  • Stephen Huhn Chief Medical Officer & Senior Vice President of Clinical Development, Pipeline Therapeutics

Synopsis

• Review of preclinical data describing effect of PIPE-505 on outer hair cells and the cochlear synapse
• Eligibility criteria and patient population in first-in-human studies
• Measures of auditory function and clinical endpoints to demonstrate preliminary efficacy

12:25 pm Optimizing Clinical Outcomes Through an Effective Drug Delivery Approach

Synopsis

• What technologies are being utilized for local drug delivery?
• How to successfully translate PK studies in animals to an optimal dosing paradigm in human clinical trials
• What drug delivery factors are important to patients, physicians, and payers?
• What lessons can be learned from drug delivery for other conditions including ocular disorders?

12:50 pm Discussion/Q&A

1:10 pm Scientific Poster Session

Understanding the Investment & Budgeting Challenges to Develop Successful Hearing Loss Companies

2:00 pm Developing a Gene Therapy from Bench to Clinic – On a Budget

  • Jim Ayala Founder & Chief Executive Officer , Rescue Hearing

Synopsis

• Building the team
• Generating the data
• Securing investment

2:15 pm Challenges of Early-Stage Investment

  • Elaine Hamm Chief Executive Officer , Ascend BioVentures & Otologic Pharmaceutics

Synopsis

• All start-ups have challenges raising money
• Hearing loss companies are no different
• This presentation will give the investment and entrepreneurial perspective on commercialization of therapeutics focused on hearing loss

2:40 pm Discussion/Q&A

  • Jim Ayala Founder & Chief Executive Officer , Rescue Hearing
  • Elaine Hamm Chief Executive Officer , Ascend BioVentures & Otologic Pharmaceutics
  • Jodi A. Cook Board of Directors, Fennec

3:00 pm Chair’s Closing Remarks & End of Inner Disorders Therapeutics Summit