KRYS Begins Dosing With Gene Therapy in Rare Eye Disease, Stock Up

Shares of Krystal Biotech KRYS gained 3.9% yesterday after the company announced that it had dosed the first patient in a phase I/II study evaluating its eye drop KB801 for the treatment of neurotrophic keratitis (NK), a rare, progressive corneal disease.

KB801 is an investigational, redosable eye drop gene therapy developed to offer a novel treatment approach for NK. The therapy is designed to promote the sustained, localized production and secretion of nerve growth factor (NGF) by epithelial cells in the anterior segment of the eye. By enhancing NGF expression, KB801 aims to support corneal healing and prevent complications such as epithelial defects, ulcers and corneal perforation associated with NK.

Although a rare disease, if not treated in time, NK can lead to severe vision loss. The disease affects an estimated 10 to 50 people out of every 100,000.

However, recent claims data suggest that awareness and diagnosis of NK are steadily increasing in the United States. In 2024, around 68,000 people had a recorded NK claim in the United States, more than double the 31,000 reported cases in 2020.

KRYS' Price Performance

Shares of Krystal Biotech have lost 5% year to date compared with the industry’s 2% decline.

More on KRYS' Phase I/II Study for KB801 Eyedrop

The double-masked, placebo-controlled phase I/II EMERALD-1 study is evaluating KB801, given as an eye drop in up to 27 adults for the treatment of NK. The main goal of the EMERALD-1 study is to investigate the safety and tolerability of topical ocular administration of KB801 in people with NK.

This early-stage study marks an important step toward developing a simple eye drop option for patients affected by this rare eye disease.

In April, the FDA cleared the company’s investigational new drug application to evaluate KB801 for the treatment of NK.

Notably, KB801 is Krystal Biotech’s second clinical-stage ophthalmology program for the treatment of NK.

Last month, the company dosed the first patient in the phase III IOLITE study evaluating its investigational candidate, KB803, administered as an eye drop, for the treatment and prevention of corneal abrasions in dystrophic epidermolysis bullosa (DEB).

DEB is a rare and severe monogenic disease that affects the skin and mucosal tissues.

Krystal Biotech’s only marketed product, Vyjuvek, is currently FDA-approved for treating patients aged six months or older with DEB. The drug is referred to as B-VEC outside the United States.

KRYS' Zacks Rank

Krystal Biotech currently carries a Zacks Rank #5 (Strong Sell).

Stocks to Consider

Some better-ranked stocks from the biotech sector are Amarin Corporation AMRN, Alkermes ALKS and Keros Therapeutics KROS, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, Amarin's 2025 loss per share estimates have improved from $3.85 to $2.30. Loss per share estimates for 2026 have narrowed from $3.59 to $1.50 during the same period. AMRN stock has surged 78.7% year to date.

AMRN’s earnings beat estimates in two of the trailing four quarters, met the mark once and missed on the other occasion, delivering an average surprise of 29.11%.

In the past 60 days, estimates for Alkermes’ earnings per share have increased from $1.74 to $1.79 for 2025. During the same time, earnings per share estimates for 2026 have risen from $1.79 to $1.91. Year to date, shares of ALKS have gained 4.2%.

ALKS earnings beat estimates in one of the trailing four quarters while missing the same on the remaining three occasions, the negative average surprise being 8.24%.

In the past 60 days, Keros Therapeutics’ 2025 loss per share estimates have narrowed from 79 cents to 29 cents. Loss per share estimates for 2026 have narrowed from $4.52 to $4.29 during the same period. KROS stock has lost 9.6% year to date.

KROS’ earnings beat estimates in one of the trailing four quarters, while missing the same on the remaining three occasions, the average surprise being 9,065.97%.

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This article originally published on Zacks Investment Research (zacks.com).

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