On May 6, 2025 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) reported financial results for the first quarter ending March 31, 2025 and provided a business update (Press release, Krystal Biotech, MAY 6, 2025, View Source [SID1234652602]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

                  Schedule Your 30 min Free Demo!

"We were thrilled to receive VYJUVEK approval in Europe, and with the potential expansion to Japan later in the year, we continue to make tremendous progress on our goal of delivering profound long-term benefit to DEB patients around the world," said Krish S. Krishnan, Chairman and CEO of Krystal Biotech. "With today’s announcement of our second clinical-stage ophthalmology program, the near-term initiation of our registrational study in DEB patients with eye lesions, and upcoming molecular readouts for our rare respiratory disease product candidates, KB407 and KB408, we are pushing forward a broad and expanded pipeline which we expect will ultimately demonstrate the power of HSV-1 based gene delivery in the lung, eye, and skin, and – most importantly – deliver meaningful benefit to patients."

VYJUVEK (beremagene geperpavec-svdt, or B-VEC)
for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)

In April, the European Commission approved VYJUVEK for the treatment of wounds in patients with DEB who have mutations in the collagen type VII alpha 1 chain (COL7A1) gene, starting from birth. The Company is on track for its first European launch in Germany in mid-2025.
The Company recorded $88.2 million in VYJUVEK net product revenue for the first quarter of 2025. Gross margin for the quarter was 94%.
As of April, the Company has secured over 540 reimbursement approvals for VYJUVEK in the U.S. and continues to maintain strong access nationwide including positive access determinations for 97% of lives covered under commercial and Medicaid plans.
High patient compliance with weekly treatment while on drug continued at 83% as of the end of the quarter.
The Company continues to expect a decision by Japan’s Pharmaceuticals and Medical Devices Agency (PMDA) on its Japan New Drug Application (JNDA) in 2H 2025.
In April, the British Journal of Dermatology published a case highlighting the success of VYJUVEK in promoting durable wound healing following surgical excision of a large squamous cell carcinoma (SCC) in a recessive DEB patient. The treated patient reported complete healing of their over 100 cm2 post-surgical wound after seven weeks of VYJUVEK therapy. Wound healing benefits were durable, with complete wound closure also observed upon clinical examination at four and ten month post-operative visits.
In April, the results of the Company’s open label extension (OLE) study of VYJUVEK in DEB patients were published in the American Journal of Clinical Dermatology.
Respiratory

KB407 for the treatment of cystic fibrosis (CF)

The Company continues to enroll Cohort 3 of CORAL-1, the Company’s multi-center, dose escalation study evaluating KB407 in patients with CF, regardless of their underlying genotype. The Company received full sanctioning of the study protocol by the Cystic Fibrosis Foundation Therapeutic Development Network in January and remains on track for an interim molecular data readout for Cohort 3 patients in mid-2025. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT05504837.
KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD) lung disease

The Company continues to enroll in Cohort 2 and is working to enroll in Cohort 3 of SERPENTINE-1, the Company’s open label, single dose escalation study in adult patients with AATD with a Pi*ZZ or a Pi*ZNull genotype. Late last year, the Company announced successful SERPINA1 gene delivery and functional alpha-1 antitrypsin (AAT) expression reaching therapeutic levels as part of an interim clinical update for Cohorts 1 and 2 of SERPENTINE-1. Inhaled KB408 was safe and well-tolerated at both tested dose levels. The Company expects to report molecular results for the additional Cohort 2 and 3 patients later this year. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT06049082.
Ophthalmology

KB803 for the treatment of ocular complications of DEB

The Company continues to enroll in its ongoing natural history study to prospectively collect data on the frequency of corneal abrasions in patients with DEB and serve as a run-in period for patients who may be eligible to participate in the Company’s registrational Phase 3 study evaluating KB803’s effect on ocular complications of DEB. The Company expects to dose the first patient in the registrational KB803 Phase 3 IOLITE study later this month.
KB801 for the treatment of neurotrophic keratitis (NK)

In April, the U.S. Food and Drug Administration (FDA) cleared the Company’s investigational new drug (IND) application to evaluate KB801, the Company’s second clinical-stage ophthalmology program, for the treatment of NK. NK is a rare, degenerative corneal disease caused by nerve damage in the eye leading to corneal epithelial defects, ulcers, and perforation, with an estimated prevalence in the range of 10 to 50 cases per 100,000. KB801 was developed using the Company’s novel replication-defective, non-integrating HSV-1-based vector and is designed to deliver two transgene copies to the corneal epithelium as an eye drop to enable local nerve growth factor (NGF) production and corneal healing. Recombinant NGF eye drops have been shown to significantly improve corneal healing and are approved for the treatment of NK in multiple jurisdictions worldwide including the United States, but the short half-life of recombinant protein results in rapid clearance from the eye, thereby necessitating burdensome administration six times a day, and may lead to suboptimal treatment outcomes. Eye pain during treatment is also frequently reported.
In preclinical development, KB801 was shown to efficiently transduce corneal epithelial cells in vitro and in vivo leading to sustained NGF production in the front of the eye. By transducing the cells of the corneal epithelium to produce and secrete NGF, KB801 has the potential to significantly reduce the treatment burden for patients while also maintaining more consistent NGF levels in the front of the eye. Yesterday, the Company presented preclinical safety and efficacy data supporting the clinical development of KB801 at the Association for Research in Vision and Ophthalmology (ARVO) 2025 Annual Meeting.
The Company expects to dose the first patient in EMERALD-1, the Company’s randomized, double-blind, placebo-controlled, multi-center Phase 1/2 study evaluating KB801 in moderate-to-severe NK patients, later this month.
Oncology

Inhaled KB707 for the treatment of solid tumors of the lung

Enrollment is ongoing in the Company’s KYANITE-1 study, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating inhaled KB707, as monotherapy or in combination, in patients with locally advanced or metastatic solid tumors of the lung. Near the end of last year, the Company announced early clinical evidence of monotherapy activity in heavily pre-treated patients with advanced non-small cell lung cancer (NSCLC) treated with inhaled KB707, achieving an objective response rate of 27% and disease control rate of 73% as of data cut-off. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06228326.
A clinical update on the monotherapy cohort from KYANITE-1 is expected to be presented at the 2025 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting next month.
Intratumoral KB707 for the treatment of injectable solid tumors

The Company continues to enroll in OPAL-1, a Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating intratumoral KB707, either as monotherapy or in combination, in patients with locally advanced or metastatic solid tumor malignancies. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT05970497.
Aesthetics

KB301 for the treatment of dynamic wrinkles of the décolleté

Jeune Aesthetics is currently developing a décolleté-specific photo numeric scale for advanced clinical development of KB301. Jeune Aesthetics expects to align with the FDA on the scale and enroll the first subject in a multi-center, randomized, placebo-controlled Phase 2 study evaluating KB301 for the treatment of dynamic wrinkles of the décolleté in Q4 2025.
KB304 for the treatment of wrinkles

In February, Jeune Aesthetics completed enrollment in PEARL-2, an ongoing, randomized and placebo-controlled Phase 1 study evaluating KB304 for the treatment of wrinkles. Jeune Aesthetics expects to report top-line results from the study in 2H 2025. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06724900.
Dermatology

KB105 for the treatment of lamellar ichthyosis

The Company expects to initiate the Phase 2 portion of its KB105 Phase 1/2 JADE-1 trial evaluating KB105 for the treatment of TGM1-deficient lamellar ichthyosis in pediatric patients in 2026.
Pipeline expansion

The Company will be presenting preclinical data at the Society for Investigative Dermatology (SID) 2025 Annual Meeting later this week on early-stage dermatology genetic medicine candidates for the treatment of Hailey-Hailey and Darier diseases.
Financial Results for the Quarter Ended March 31, 2025:

Cash, cash equivalents, and investments totaled $765.3 million as of March 31, 2025.
Product revenue, net totaled $88.2 million and $45.3 million for the quarters ended March 31, 2025 and March 31, 2024, respectively.
Cost of goods sold totaled $5.0 million and $2.4 million for the quarters ended March 31, 2025 and March 31, 2024, respectively.
Research and development expenses for the quarter ended March 31, 2025 were $14.3 million, inclusive of $2.5 million of stock-based compensation, compared to $11.0 million, inclusive of stock-based compensation of $1.9 million for the quarter ended March 31, 2024.
Selling, general, and administrative expenses for the quarter ended March 31, 2025 were $32.7 million, inclusive of stock-based compensation of $11.0 million, compared to $26.1 million, inclusive of stock-based compensation of $7.4 million, for the quarter ended March 31, 2024.
Net income for the quarter ended March 31, 2025 was $35.7 million, or $1.24 per common share (basic) and $1.20 per common share (diluted). Net income for the quarter ended March 31, 2024 was $0.9 million, or $0.03 per common share (basic) and $0.03 per common share (diluted).
Financial Guidance

($ in millions) FY 2025 Guidance
Non-GAAP Research and Development ("R&D") and Selling, General and Administrative ("SG&A") expense(1) $150.0 – $175.0

(1) Refer to Non-GAAP Financial Measures section below for additional information. Non-GAAP combined R&D and SG&A expense guidance does not include stock-based compensation as we are currently unable to confidently estimate Full Year 2025 stock-based compensation expense. As such, we have not provided a reconciliation from forecasted non-GAAP to forecasted GAAP combined R&D and SG&A Expense in the above. This could materially affect the calculation of forward-looking GAAP combined R&D and SG&A Expense as it is inherently uncertain.

Conference Call

The Company will host an investor webcast on May 6, 2025, at 8:30 am ET.

Investors and the general public can access the live webcast at:
View Source

For those unable to listen to the live conference call, a replay will be available for 30 days on the Investors section of the Company’s website at www.krystalbio.com.