# Bench to Bedside

All-Access Saturday — May 2 | Colorado Convention Center | Denver

## **Bench to Bedside — Nonclinical development and new approach methodologies (NAMs)**

_(all times are local MT)_

Advancing an ocular therapeutic from laboratory discovery to first-in-human studies requires more than compelling science. It demands translational rigor, strategic sequencing of nonclinical studies, regulatory alignment, and capital discipline. These sessions are designed for eye and vision researchers seeking to move their research concepts toward clinical development.

Through expert-led sessions and interactive discussions, participants will clarify what constitutes regulatory-grade proof-of-concept, understand the role of pharmacokinetics and toxicology in IND-enabling programs, examine safety considerations unique to ocular therapies, and explore the appropriate integration of New Approach Methodologies (NAMs). Regulatory, industry, CRO, and translational funding perspectives will provide practical insight into when programs are ready to advance and when additional de-risking is needed.

Participants will leave with a structured framework for evaluating their program’s translational readiness and a roadmap toward clinical testing.

**Target Audience** — Academic and early-stage industry eye and vision researchers

**Primary Goal** — Enable researchers to assess translational readiness and identify steps required to advance toward an IND.

### **Learning objectives**

At the conclusion of this activity, participants will be able to:

- **Outline** the major nonclinical steps required to advance an ocular therapeutic from discovery to IND submission.
- **Assess** whether their current proof-of-concept data meet translational readiness standards expected by regulators.
- **Design** an early pharmacology strategy that supports dose selection and clinical relevance.
- **Interpret** basic PK data to determine whether a therapeutic approach is viable for further development.
- **Describe** the purpose, timing, and scope of IND-enabling toxicology studies.
- **Identify** at least two common translational pitfalls that delay or derail ocular therapeutic development.
- **Differentiate** between investor ready and foundation ready data and presentation styles.
- **Evaluate** when and how New Approach Methodologies (NAMs) can appropriately supplement or reduce traditional animal studies.

### Schedule

**8 - 10:10am — Session 1: Is my idea ready for translation? Proof of concept & early pharmacology — [BREAK @ 10:10am]**

This session examines the transition from discovery-stage research to translationally relevant proof-of-concept. Speakers will clarify the distinction between publication-quality data and regulatory-grade evidence and discuss expectations for primary and secondary pharmacology studies in ocular therapeutics. Emphasis will be placed on reproducibility, dose-response characterization, and model selection that reflects human disease biology.

Through case examples and interactive discussion, participants will evaluate whether their current data package supports advancement toward IND-enabling studies and identify common gaps that delay translational progress.

Participants will be able to:

- **Outline** the major nonclinical steps required to advance an ocular therapeutic from discovery to IND submission.
- **Assess** whether their current proof-of-concept data meet translational readiness standards expected by regulators.
- **Identify** at least two common translational pitfalls that delay or derail ocular therapeutic development.

**8am — Welcome Opening**  
Cheryl L. Rowe-Rendleman, PhD, FARVO, FAOPT  
Owner and Managing Consultant, Omar Consulting Group, Durham, NC

**8:02am — Introductions**  
Claire M. Gelfman, PhD _(Session moderator)_  
Principal, CMG Consulting, Associate Adjunct Professor, University of California, Irvine, San Diego, Calif.

**8:03am — Publication-quality data and regulatory-grade data**  
Cheryl L. Rowe-Rendleman, PhD, FARVO, FAOPT

**8:15am — Soliciting regulatory feedback: Formal vs Informal meetings: a view from the FDA pharm/tox** _(i.e., INTERACT vs pre-IND vs EOP meeting)_  
Erin Ruhland, PhD  
Pharmacologist/Toxicologist, FDA, Rockville, Md.

**8:27am — Soliciting regulatory feedback: Formal vs Informal meetings: a medical view** _(i.e., INTERACT vs pre-IND vs EOP meeting)_  
Maryam Mokhtarzadeh, MD  
Chief Medical Officer, Persephoni Bio, Irvine, Calif.

**8:39am — Is mechanistic validation sufficient to justify further development? (Part 1)**  
Ashley Winslow, PhD  
CEO & CSO, Odylia Therapeutics, Atlanta, Ga.

**8:51am — Is mechanistic validation sufficient to justify further development? (Part 2)**  
Shigemi Matsuyama, DVM, PhD  
Professor, Case Western Reserve University, Cleveland, Ohio

**9:03am — Decision tree for advancing or pausing a therapeutic concept**  
Brian Levy, OD, MSc  
Chief Executive Officer, InflammX Therapeutics, Tampa, Fla.

**9:15am — Identifying gaps/challenges that delay translational progress, and how to overcome them**  
Daniel C. Chung, DO, MA  
Chief Medical Officer, Beacon Therapeutics, Newtown Square, Pa.

**CASE STUDIES**

**9:27am — Introductions**  
Linda Alunkal, MBA  
Co-Founder & CEO, BioLattice Ophthalmics Inc., Philadelphia, Pa.

**9:30am — The journey of Xiflam: Path to IND**  
Brian Levy, OD, MSc  
Chief Executive Officer, InflammX Therapeutics, Tampa, Fla.

**9:40am — Development of N-acetyl cysteine amide (NACA) for Retinitis Pigmentosa: Path to IND (US and Australia)**  
Jami R. Kern, MBA, PhD  
SVP, Chief Clinical Officer, Nacuity Pharmaceuticals, Inc.

**9:50am — Orally administered deuterium reinforced docosahexaenoic acid for autosomal dominant RP-path to IND**  
Maureen A. McCall, PhD, FARVO  
Professor, University of Louisville, Louisville, Ky.

**10am — Discussion: “Pressure Test Your Idea”**  
All

**BREAK: 10:10 - 10:25am**

**10:25 - 11:45am — Session 2: Understanding PK and Toxicology - So you can talk to investors**

Pharmacokinetics and toxicology studies are often misunderstood by early-stage investigators yet represent major determinants of clinical viability and investment readiness. This session provides a practical overview of absorption, distribution, metabolism, and excretion (ADME) considerations specific to locally administered ocular therapies, along with the purpose and timing of GLP toxicology studies.

Panel discussion will address when to initiate IND-enabling work and how to avoid costly repetition of studies. Participants will gain clarity on sequencing, budget implications, and the data thresholds that support progression to regulatory engagement.

Participants will be able to

- **Design** an early pharmacology strategy that supports dose selection and clinical relevance.
- **Interpret** basic PK data to determine whether a therapeutic approach is viable for further development.
- **Describe** the purpose, timing, and scope of IND-enabling toxicology studies.

**10:25am — Welcome and introduction**  
Stephen H. Poor, MRCOphth _(Moderator)_  
Head of Translational Medicine, Apellis Pharmaceuticals, Cambridge, Mass.

**10:28am — PK matters more than you think**  
John S. Penn, PhD, FARVO  
Founder & Chief Scientific Officer, Vanderbilt Ophthalmic Contract Research Organization (VO-CRO), Nashville, Tenn.

**10:38am — Toxicology 101**  
Simon Kaja, PhD  
Dr. John P. and Therese E. Mulcahy Endowed Professor in Ophthalmology, Loyola University Chicago, Principal and Founder, K&P Scientific LLC, Chicago, Ill.

**10:48am — Toxicology 201**  
Shivakumar Vasanth, PhD  
Director, Ocular Toxicology, Charles River Laboratories, Portage, Mich.

**10:58am — Safety**  
Brian C. Gilger, DV, MS, FARVO  
Professor, North Carolina State University, Co-Founder, Bedrock Therapeutics, Inc., Raleigh, NC.

**11:08am — PK/Tox: choosing the right model (Part 1)**  
James Bojdo, PhD  
Founding Director / CEO, Medinect Ophtho, Belfast, Northern Ireland, UK.

**11:18am — De-risking formulations for ocular delivery/bioanalytical analysis**  
Uday B. Kompella, PhD, FARVO  
Professor, University of Colorado Anschutz Medical Campus, Chief Scientific Officer, EyeTrans Technologies, Inc., Denver, Colo.

**11:28am — PK/Tox: choosing the right model  (Part 2)**  
Andy Whitlock, PhD  
Chief Scientific Officer, Ocular Services On Demand (OSOD), a MERIT Company, Madison, Wisc.

**11:38am — Discussion**  
All

**11:45am - 12:15pm — Session 3: How to speak to investors and funders — [LUNCH @ 12:15pm]**

Securing support for translational research requires tailoring both data and messaging to the priorities of different funding audiences. This session will explore how to prepare and present scientific programs for investors versus research foundations, highlighting differences in expectations for data maturity, risk framing, and value proposition. Speakers from industry and nonprofit funding organizations will share practical guidance on preparing an effective pitch and positioning translational data for funding discussions. Participants will gain insight into how presentation style, evidence thresholds, and strategic framing differ between investor and foundation audiences.

Participants will be able to: **Differentiate** between investor ready and foundation ready data and presentation styles.

**PANEL DISCUSSION**

**11:45am — Welcome**  
Barbara Wirostko, MD, FARVO _(Moderator)_  
Co-Founder and Chief Medical Officer, Qlaris Bio, Inc., Co-Founder and Medical Director, MyEyes, Clinical Adjunct Professor, University of Utah, Moran Eye Center, Salt Lake City, Utah.

**11:47am — How to prepare your pitch and speak to investors**  
_Panelists_
Maryam Mokhtarzadeh, MD  
Chief Medical Officer, Persephoni Bio, Irvine, Calif.

Firas M. Rahhal, MD  
Partner / Owner, Retina-Vitreous Associates Medical Group, Co-Founder / General Partner, ExSight Ventures, Beverly Hills, Calif.

**11:57am — How to prepare your data to speak to foundations**  
_Panelists_
Diane Bovenkamp, PhD  
Vice President, Scientific Affairs, BrightFocus Foundation, Ellicott City, Md.

Amy Palmer Laster, PhD  
Chief Scientific Officer, Foundation Fighting Blindness, Columbia, Md.

**12:07pm — How to pitch foundations to raise money for your new venture**  
_Panelist_
David Chute, MSc, MBA  
President & Founder, i2C Ventures LLC, Founder / Manager, GRF Treatment Accelerator, Atherton, Calif.

**12:12pm — Discussion**  
All panelists

* * *

**LUNCH: 12:15 – 1pm**

**1 - 2:45pm — Session 4: NAMs**

This session will examine how New Approach Methods (NAMs) are being used to support ophthalmic drug development. Speakers will discuss where NAMs can strengthen translational programs, where animal data remain necessary, and how NAM-generated evidence can be positioned in regulatory discussions, including orphan drug and pre-IND interactions. Case examples will highlight hybrid strategies that combine NAMs with targeted animal studies and ex vivo ocular tissue, including lessons learned on cost, timelines, and development strategy. A translational perspective from the National Eye Institute will address expectations for IND-enabling research and funding mechanisms that support the transition from discovery to regulatory-grade evidence.

Participants will be able to: **Evaluate** when and how New Approach Methodologies (NAMs) can appropriately supplement or reduce traditional animal studies.

**1:15pm — Welcome**  
Michael F. Chiang, MD, FARVO _(Moderator)_  
Director, National Eye Institute, National Institutes of Health, Bethesda, Md.

**1:18pm — What Are NAMs and When Do They Help?**  
Daniel Paull, PhD  
Scientific Director and VP, Discovery and Platform Development, The Jackson Laboratory-NYSCF, New York, NY.

**1:33pm — Positioning NAM data in regulatory discussions-orphan drugs**  
Nana K. Mainoo, PharmD, MA  
Chief Executive Officer, Cleracs Consulting, Cambridge, Mass.

**1:48pm — Hybrid NAM: ex vivo ocular tissue**  
Onkar Sawant, PhD  
Vice President, Research & Development, Eversight, Cleveland, Ohio.

**2:03pm — Engineered corneas to treat corneal blindness**  
Linda Alunkal, MBA

**2:18pm — NIH/NEI translational perspective**  
Michael F. Chiang, MD, FARVO

**2:33pm — Discussion**

**2:43pm — Wrap-up**  
Claire Gelfman, PhD

**2:45pm — Closing**  
Cheryl L Rowe-Rendleman, PhD, FARVO, FAOPT

_\* Speakers and topics are subject to change. Inclusion in the program does not imply an ARVO endorsement of the products and services of any company._