Conference Day Two

Overcoming Hurdles in Drug Delivery & Manufacturing

8:00 am Chair’s Opening Address

8:15 am Panel Discussion: Overcoming Drug Delivery & Administration Challenges in Otology


This panel will be a structured conversation designed to answer key questions such as:
• A key challenge in hearing loss trials is choice of delivery method. The decision will depend on the pharmacokinetic profile of the individual agent, and the balance of risks associated with delivery against the potential benefit of the treatment
• The current administration routes of inner ear disorders consist of systemic drug delivery, intratympanic injection, nanoparticle approaches, oral administration, and others. Despite the assortment of options, current approaches have been associated with inconsistent efficacy and safety
• This panel will explore the differing routes of delivery and administration methods, current challenges and how to select the best method based on your candidate

9:00 am Optimizing Clinical Outcomes through an Effective Drug Delivery Approach


• Successful technologies for otic 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 payors?
• What lessons cam be learned from drug delivery for other conditions including ocular disorders?

9:30 am Fireside Chat: Managing Scale-Up & CMC Challenges for Novel Therapeutics in Otology


• As the industry moves towards late-stage clinical trials, it is important to iron out complex manufacturing challenges associated with developing inner ear therapeutics
• This discussion will share insights about how to overcome scale-up and manufacturing challenges

10:00 am Structured Networking & Morning Coffee Break


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.

Leveraging Genetic & Cellular Approaches to Combat Inner Ear Disorders

11:15 am Leveraging Single Cell Genomics in Silico to Identify & Screen Drug Targets for Protection & Regeneration of Hearing

  • Jian Zuo Professor, Creighton University & Co-founder, Ting Therapeutics


• Highlighting in silico drug screening methods
• Characterization in preclinical animal models
• Drug Pk/Pd in the inner ear

11:45 am Precision Gene Therapy for Hair Cell Regeneration

  • Joseph Burns Vice President, Discovery, Decibel Therapeutics


  • We are engineering gene therapies to convert supporting cells, the cells adjacent to hair cells, into both cochlear and vestibular hair cells in order to restore hearing and balance function through selective expression of reprogramming factors that control cell fate
  • We have observed that selective expression of reprogramming factors via proprietary support cell selective promoters resulted in regeneration of vestibular hair cells in vivo
  • We are evaluating whether a single AAV vector expressing a combination of reprogramming factors may further enhance maturation of hair cell types

12:15 pm Cell Therapy 2.0: Delivering a Revolution in the Regenerative Treatment of Hearing Loss


• After years of false starts, cell therapies are no longer distant scientific explorations – they are becoming a reality for patients and healthcare providers. Expectations are growing worldwide for discoveries that will harness the tremendous potential of cell therapy, which has already brought breakthroughs in therapeutic areas where there is high unmet need, from oncology, to ophthalmology and rare disease
• Cell therapies have the potential to treat diseases that cannot be adequately addressed by traditional medicines
• Using stem cells to recover the damaged sensory circuitry of the cochlea of patients with sensorineural hearing loss is a potential therapeutic strategy for treating deafness
• Rinri is the leading company exploring the potential of regenerative cell therapy for hearing loss

12:45 pm Lunch Break

Emerging Biologics & Small Molecule Therapies in Otology to Overcome Hearing Loss

1:45 pm Audible-S, A Study to Evaluate the Use of SENS-401 in SSNHL Disease: Results & Lessons Learned


• SENS-401 is well tolerated and safe
• Control for spontaneous hearing recovery is essential for SSNHL studies
• Future clinical studies should focus on the severe-to-profound SSNHL population

2:15 pm Sustained Drug Delivery to the Inner Ear Using Novel Bone-Binding Small Molecules with Neurotrophic Activity

  • Judith Kempfle Research Associate, Department of Otolaryngology, Massachusetts Eye and Ear


• Bisphosphonates enable sustained drug delivery to the inner ear
• Bone binding, neurotrophic small molecules improve neurite outgrowth and synapse regeneration in vitro
• Novel small molecule neurotrophic agonists retain activity in the inner ear in vivo

2:45 pm Novel Treatment to Targeting Acute Sensorineural Hearing Loss


• Small lipophilic pyridoindole activates neurotrophins and performance of respiratory chain in mitochondria
• This improves cell maintenance, energy supply and restores cell damage in noise-induced hearing loss

3:15 pm Results of Four Single-Dose Trials of FX-322 Show Improved Speech Perception

  • Carl LeBel Chief Development Officer, Frequency Therapeutics


• This presentation showcases clinical case studies of FX-322, a small-molecule combination designed to regenerate cochlear hair cells
• This summary combines the results of four single-dose trials of FX-322, three of which showed speech perception improvements in some patients
• These positive results support the continued evaluation of FX-322 in multiple populations to determine the full range of benefit to SNHL patients as a therapeutic for hearing restoration

3:45 pm Break

4:15 pm Small Molecule, KCNQ4 Agonist ACOU085 for the Treatment & Prevention of Hearing Loss


• The KCNQ4 ion channel, a biologically validated drug target, plays a key role in regulating excitability and homeostasis of cochlear sensorineural cells
• This presentation highlights the otoprotective potential and development of the small molecule, clinical stage drug candidate ACOU085 for the treatment and prevention of hearing loss

Assessing Therapeutics Targeting Tinnitus

4:45 pm Novel Therapeutic Treatments to Targeting Tinnitus


• Background on tinnitus population, disease burden and pathophysiology
• Potential treatment using sustained-exposure formulation of gacyclidine (OTO-313)
• Review of OTO-313 Phase 1/2 clinical results

5:15 pm Chair’s Closing Remarks