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Pathfinders in Biopharma

Ocular Therapeutix’ bold vision to redefine retinal disease treatment

24 min episode · 2 min read
·
Praveen Duggal

Episode

24 min

Read time

2 min

Topics

Investing, Leadership, Design & UX

AI-Generated Summary

Key Takeaways

  • Patient dropout economics: 40% of wet AMD patients in the U.S. abandon monthly or bimonthly injection regimens within the first year, resulting in blindness. Reducing dropout by even 10% would prevent approximately 250,000 additional cases of blindness annually in the U.S. alone, generating substantial cost savings for payers who bear escalating care costs for blind patients.
  • Fibrosis as the overlooked failure mode: Even patients who remain compliant with anti-VEGF injections lose vision after two to five years due to fibrosis and atrophy, not rebleeding. The cyclical swelling from monthly injections damages retinal nerve cells similarly to repeated concussions. Continuous VEGF suppression via sustained-release formats reduces this scarring and improves long-term visual outcomes.
  • ELutix platform tunability: The ELutix hydrogel platform releases drug over a programmable duration — one month for DEXTENZA, approximately ten months for EXPaxley — by adjusting PEG bond composition around the implant. The API itself is not pegylated. The hydrogel fully dissolves post-release, leaving no residual implant material, which eliminates a key safety concern for intraocular sustained-delivery devices.
  • Non-proliferative diabetic retinopathy opportunity: In the Healios study, a single EXPaxley injection reduced vision-threatening complication rates from 37.5% in controls to zero at 48 weeks in non-proliferative diabetic retinopathy patients. This population — largely asymptomatic working-age adults — has under 1% treatment penetration today, representing a commercially untapped market that any successful VEGF-targeting drug historically expands into.
  • Regulatory risk mitigation via SPA: Ocular followed FDA's February 2023 draft guidelines for retinal disease trials precisely, earning a Special Protocol Assessment agreement for SOL1. Investors evaluating clinical-stage biopharma should treat SPA agreements as a concrete de-risking signal, particularly in volatile macro environments where regulatory unpredictability elevates the value of pre-agreed trial design alignment with the agency.

What It Covers

Ocular Therapeutix CEO Praveen Duggal outlines how the company's lead drug candidate EXPaxley, a tunable dissolvable hydrogel delivering a tyrosine kinase inhibitor, targets two core failures in wet AMD treatment: 40% patient dropout rates and long-term vision loss from fibrosis, with SOL1 trial readout expected Q1 2026.

Key Questions Answered

  • Patient dropout economics: 40% of wet AMD patients in the U.S. abandon monthly or bimonthly injection regimens within the first year, resulting in blindness. Reducing dropout by even 10% would prevent approximately 250,000 additional cases of blindness annually in the U.S. alone, generating substantial cost savings for payers who bear escalating care costs for blind patients.
  • Fibrosis as the overlooked failure mode: Even patients who remain compliant with anti-VEGF injections lose vision after two to five years due to fibrosis and atrophy, not rebleeding. The cyclical swelling from monthly injections damages retinal nerve cells similarly to repeated concussions. Continuous VEGF suppression via sustained-release formats reduces this scarring and improves long-term visual outcomes.
  • ELutix platform tunability: The ELutix hydrogel platform releases drug over a programmable duration — one month for DEXTENZA, approximately ten months for EXPaxley — by adjusting PEG bond composition around the implant. The API itself is not pegylated. The hydrogel fully dissolves post-release, leaving no residual implant material, which eliminates a key safety concern for intraocular sustained-delivery devices.
  • Non-proliferative diabetic retinopathy opportunity: In the Healios study, a single EXPaxley injection reduced vision-threatening complication rates from 37.5% in controls to zero at 48 weeks in non-proliferative diabetic retinopathy patients. This population — largely asymptomatic working-age adults — has under 1% treatment penetration today, representing a commercially untapped market that any successful VEGF-targeting drug historically expands into.
  • Regulatory risk mitigation via SPA: Ocular followed FDA's February 2023 draft guidelines for retinal disease trials precisely, earning a Special Protocol Assessment agreement for SOL1. Investors evaluating clinical-stage biopharma should treat SPA agreements as a concrete de-risking signal, particularly in volatile macro environments where regulatory unpredictability elevates the value of pre-agreed trial design alignment with the agency.

Notable Moment

Duggal describes a prior standard of care where physicians deliberately lasered the center of a patient's vision — reducing sight to legally blind levels — because the resulting blind spot was smaller than what the disease would eventually cause untreated. This practice was considered medically justified at the time.

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Episode Transcript

Hello, and welcome to Pathfinders, a podcast series from RBC Capital Markets where we uncover the key trends and catalysts shaping the fast moving world of biopharma and health care. I'm your host, Joe Coletti. Today, we're at RBC's global health care conference here in New York. And today, we're gonna be exploring innovative therapeutics intended to improve the lives of patients with serious retinal disease. Joining us is Praveen Duggal, MD, executive chairman, president, and CEO of Ocular Therapeutics, which is seeking to redefine the treatment of retinal diseases. The company has two ongoing registrational trials for its lead program, x Paxley, in wet AMD, with the first of those trials, Sol one, slated to read out in q one of next year. Praveen has over thirty years experience as a retina specialist. He is an internationally recognized clinical researcher with a lifetime achievement award from the American Academy of Ophthalmology and has extensive leadership experience with the Keck School of Medicine and is a founding member of the Spectra Eye Institute. So today, we'll explore how Ocular's innovative therapeutics pipeline is helping drive better long term outcomes in treatment of retinal disease, including wet AMD, which is the leading cause of blindness in The United States. We'll also explore the trend shaping investor strategies in this exciting space. Praveen, it's great to have you here. Joe, thank you. It's an honor, and thank you for having me here today. So Ocular is aiming to transform the experience of retinal disease treatment. Can you talk about what drives that mission and what your latest clinical program's helping to reduce the burden and how it's helping to reduce the burden on patients and providers alike? Yeah. Great question. So I frankly have a long family history of macular degeneration, so this affects me personally. And also having practiced for thirty years, I see what's happening. I'm old enough to remember when we had absolutely no treatment for wet macular degeneration, and all we would do is to watch patients go blind. And our job was to sort of, like, time when they would go blind and say, miss Jones, you ought to get your affairs in order because it may be two months, it may be six months, or whatever it is. There was even a point, Joe, believe it or not, when the standard of care was to actually laser the center of vision. So if somebody would come in with vision of, I don't know, twenty eighty, say, the standard of care was to go ahead and laser the center of vision and make them 2,200, which is legally blind. And we would say, miss Jones, you're gonna thank me two years from now because the blind spot that I just created with the laser is actually smaller than it would have been had I not done anything and left it alone. It was really that barbaric. And then came along this medicine called Lucentis and then Eylea …

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