Krystal Biotech : KB801 First Patient Dosed Update

Published: 2025-07-09 09:57:06 ET KRYS

Published on 07/09/2025 at 09:57

KB801 for Neurotrophic Keratitis First Patient Dosed Update

July 2025

About Neurotrophic Keratitis

Krish Krishnan; Chairman and CEO

Krystal's Platform for Front of Eye

Suma Krishnan, MS, MBA; President, Research & Development

Krystal's KB801 Program

Preclinical Overview

Phase 1/2 Clinical Trial Design

Regulatory and Pipeline Outlook

Trevor Parry, PhD; VP, Product Development

David Sweet, MD, PhD; Director, Clinical Development

Suma Krishnan, MS, MBA; President, Research & Development

Closing

Krish Krishnan, Chairman and CEO

Q&A

All Speakers

NK is a degenerative disease of the cornea that occurs when corneal nerves are damaged and their roles in maintaining the corneal epithelium are compromised

Corneal epithelial impact can range from punctate lesions to recurrent or persistent epithelial defects and ulcers, leading to stromal melting and corneal perforation

All NK associated with some degree of vision impairment, severe cases lead to blindness

Although rare, diagnosis rates are climbing rapidly as awareness grows

There were an estimated 68K patients in U.S. with at least one NK claim

Estimated Patients with

68K

31K

NK Claim in the U.S.

in 2024, more than double the number in 2020 2020 2024

Komodo Healthcare Map, Estimated Patients

with at Least One H1623* Code, 2020-2024

Sacchetti M, et al. Clin Ophthalmol. 2014. 8: 571-579; Bian Y, et al. Ophthalmology. 2022. 129: 1255-1262; Rama P, et al. Orphanet. Neurotrophic Keratopathy. 2017 [accessed Sept 28 2023]; Gablson EE et al. Invest Ophthalmol Vis Sci. 2018. 59: 1800; Komodo Health H16.23* Claims Analysis [June 2025]; Dana R, et al. BMC Ophthalmol. 2021. 21: 327

The only specific FDA approved therapy for NK is Oxervate®

First approved in 2018, Oxervate is an ophthalmic formulation of recombinant human nerve growth factor (cenegermin-bkbj) for topical application as an eye drop

Oxervate® targets underlying nerve defect and has been shown to improve healing

4 week healing rates in the range of 50-60%

8 week healing rates (primary endpoint) in the range of 65%-75%

However, Oxervate must be dosed 6x daily for 8 weeks which is both highly burdensome and may lead to suboptimal outcomes

Eye pain is the most common adverse event, compounding the problem of 6x daily dosing

Over $540M

2023 U.S. Medicaid and Medicare Spend on Oxervate

CMS - Medicare and Medicaid Spending Per

Drug, query 'Oxervate', accessed June 2025

Over 410K

Estimated Days of Reimbursed Oxervate Therapy in US in 2024

Komodo Drug Projections Assuming 14 Day

Supply per Projected Rx

CADTH Review - Cenegermin (Oxervate). 2022. Vol 2, Issue 9; Pflugfelder SC, et al. Ophthalmology. 2020. 127: 14-26; Bonini S, et al. Ophthalmology. 2018. 125: 1332-1343; Oxervate® 2019 FDA Label; Komodo Health Drug Projections [June 2025]

Topical

Eye Drop

Rapid protein clearance and frequent cell turnover have to date limited potential of gene therapy and biologics in the front of the eye

Redosable HSV-1 based vector can overcome those challenges

HSV-1 exhibits natural tropism for epithelial cells of the eye

Vector is amenable to eye drop formulation and administration

Safety and efficacy of repeat dosing with B-VEC eye drops already demonstrated under compassionate use in DEB patient

Cargo capacity allows for delivery of wide variety and combinations of biologic

payloads

Multiple development opportunities targeting genetic diseases as well as those where biologics would be beneficial but not feasible given rapid protein clearance rates

Sabater A et al., Poster # 787 - C0388. at the 2023 Association for Research in Vision and Ophthalmology Annual Meeting; Vetencourt AT, et al. N Engl J Med. 2024;390:530-535

Disclaimer

Krystal Biotech Inc. published this content on July 09, 2025, and is solely responsible for the information contained herein. Distributed via Public Technologies (PUBT), unedited and unaltered, on July 09, 2025 at 13:56 UTC.