On November 3, 2025 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS) reported financial results for the third quarter ending September 30, 2025 and provided a business update.
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"It is immensely gratifying to see a growing number of DEB patients worldwide benefit from access to VYJUVEK, and we look forward to rapidly and sustainably expanding that number in the months ahead," said Krish S. Krishnan, Chairman and CEO of Krystal Biotech. "With multiple near-term readouts, starting with cystic fibrosis in Q4, and a strong balance sheet, Krystal is well positioned to advance our pipeline and deliver transformative therapies to patients with serious and rare diseases."
VYJUVEK (beremagene geperpavec-svdt, or B-VEC)
for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
The Company recorded $97.8 million in VYJUVEK net product revenue for the third quarter of 2025. Gross margin for the quarter was 96%.
The Company has secured over 615 reimbursement approvals for VYJUVEK in the United States and continues to maintain strong access nationwide.
In September, the United States Food and Drug Administration (FDA) approved a label update which expanded the VYJUVEK eligible patient population to include DEB patients from birth and provided patients greater flexibility with respect to VYJUVEK application, including the option for patients or their caregivers to apply VYJUVEK at home on their own.
In late August, the Company launched VYJUVEK in Germany, its first commercial launch of VYJUVEK outside of the United States. The Company estimates that approximately 20 patients have been prescribed VYJUVEK therapy across Germany, with over 10 German centers prescribing VYJUVEK to date. The Company has started discussions with payers in Germany and expects pricing negotiations to continue until at least 2H 2026.
In September, the Haute Autorité de Santé (HAS) in France approved early reimbursed access to VYJUVEK under the post-marketing authorization Accès Précoce AP2 program, including the option to dispense VYJUVEK outside of the hospital setting, and, in October, the Company launched VYJUVEK in France. Also in October, the HAS appraised VYJUVEK as Amélioration du Service Médical Rendu (ASMR) III, a designation which recognizes the added clinical benefit of VYJUVEK and is an important milestone as the Company advances access discussions in France. According to the Transparency Committee of the HAS, only 11% of new medicines reviewed in 2024 were appraised as ASMR I-III. The Company continues to engage with payers in France and expects negotiations to continue for at least the next 15 months.
In October, the Company launched VYJUVEK in Japan following successful completion of pricing negotiations with Japan’s Ministry of Health, Labour and Welfare.
Also in October, VYJUVEK was awarded the Prix Galien Italia in the Advanced Therapy Medicinal Products category in Italy. The Prix Galien is an international awards program recognizing excellence in scientific innovation that improves the state of human health.
The Company is also preparing regulatory filings for the United Kingdom and Switzerland, as well as initiating pricing discussions with relevant authorities in other key Western European markets. The timing of European launches outside of France and Germany will depend on the cadence and outcomes of pricing negotiations.
In addition to the Company’s direct VYJUVEK launches in the United States, major European markets, and Japan, the Company is also building a specialty distributor network to support commercialization of VYJUVEK in the rest of world and has executed agreements with leading regional specialty distributors covering key markets in the Middle East, Turkey, and Central and Eastern Europe, with additional network expansion expected in 2026.
Respiratory
KB407 for the treatment of cystic fibrosis (CF)
The Company continues to enroll in Cohort 3 of CORAL-1, the Company’s multi-center, dose escalation study evaluating KB407 in patients with CF, regardless of their underlying genotype, and expects to provide an interim data readout for Cohort 3 patients before year end. 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 repeat dose Cohort 2B of SERPENTINE-1, the Company’s open label dose escalation study evaluating KB408 in adult patients with AATD with a Pi*ZZ or a Pi*ZNull genotype, and expects to report interim data for this cohort in 1H 2026. Cohort 2B is designed to evaluate the safety and tolerability of repeat KB408 dosing at the same dose level that was previously shown to safely deliver SERPINA1 to the lungs of AATD patients after a single dose. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06049082.
Ophthalmology
KB803 for the treatment and prevention of corneal abrasions in DEB patients
The Company expects to complete enrollment in IOLITE, the Company’s intra-patient, double-blind, multicenter, placebo-controlled Phase 3 study with crossover design evaluating KB803 for the treatment and prevention of corneal abrasions in DEB patients, before year end. The primary study endpoint will be the change in the average number of days per month with corneal abrasion symptoms while receiving KB803 versus placebo. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT07016750.
KB801 for the treatment of neurotrophic keratitis (NK)
The Company continues to enroll in EMERALD-1, the Company’s 2:1 randomized, double-masked, multicenter, placebo-controlled study evaluating KB801 for the treatment of NK. The primary objective of EMERALD-1 is to evaluate the safety and tolerability of topical ocular administration of KB801 in patients with NK. The secondary objective is evaluation of efficacy based on the proportion of patients with complete durable healing of corneal epithelium at eight weeks. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT06999733.
In October, the FDA granted platform technology designation to the genetically modified, non-replicating herpes simplex virus type 1 viral vector used in KB801. The FDA’s platform technology designation program is intended to provide efficiencies in drug development, manufacturing, and review processes for drug product applications that incorporate designated platform technologies, with potential benefits including more frequent engagement with the FDA during clinical development, as well as opportunities to leverage manufacturing and nonclinical safety data from FDA-approved products that incorporate designated platform technologies, such as VYJUVEK, in submissions to the FDA.
Oncology
Inhaled KB707 for the treatment of solid tumors of the lung
In August, the Company announced that the FDA had granted the Company an End of Phase 2 meeting to discuss the inhaled KB707 program and early evidence of efficacy for the treatment of non-small cell lung cancer (NSCLC) from KYANITE-1, the Company’s ongoing open label, multi-center, dose escalation and expansion Phase 1/2 study. Based on the FDA’s feedback, the Company now expects that a single Phase 3 registrational study, evaluating inhaled KB707 in combination with chemotherapy against chemotherapy alone in patients with advanced NSCLC, would be sufficient to support potential registration of inhaled KB707 in combination with chemotherapy as a second-line treatment for NSCLC.
In support of this potential registrational pathway, the Company has opened a new cohort in KYANITE-1 to evaluate a fixed inhaled dose of KB707 in combination with chemotherapy in patients with advanced NSCLC. Enrollment in KYANITE-1 is ongoing. The Company expects to report interim efficacy data and potential registrational study plans in 2H 2026. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT06228326.
Intratumoral KB707 for the treatment of injectable solid tumors
The Company has paused enrollment in OPAL-1, the Company’s Phase 1/2 open label, multi-center, dose escalation and expansion study evaluating intratumoral KB707 in patients with locally advanced or metastatic solid tumor malignancies. Patients enrolled in OPAL-1 continue to be followed and based on safety and efficacy results from the study, the Company may adjust development plans for intratumoral KB707. Details of the study can be found at www.clinicaltrials.gov under NCT identifier NCT05970497.
Aesthetics
KB304 for the treatment of wrinkles of the décolleté
Jeune Aesthetics, Inc. (Jeune), a wholly-owned subsidiary of the Company, remains on track to initiate a Phase 2 study of its lead program KB304 in 1H 2026 following feedback from the FDA on Jeune’s validated décolleté-specific photonumeric scale and Phase 2 study design in 2H 2025.
Dermatology
KB111 for the treatment of Hailey-Hailey disease (HHD)
In October, the FDA cleared the Company’s investigational new drug application to evaluate KB111 for the treatment of HHD, a rare genetic disease of the skin linked to mutations in the ATP2C1 gene and low expression levels of ATP2C1-encoded calcium-transporting ATPase type 2C member 1 (ATP2C1) in keratinocytes. HHD is characterized by painful rash and blistering in skin folds, with a relapsing-remitting clinical course aggravated by heat and sweating. Patients with HHD report debilitating symptoms of pain, itch, burning, infections, and body odor, as well severe, negative impacts on quality of life and psychological distress. The prevalence of HHD is not well characterized and is most commonly estimated at roughly 1 per 50,000, although underreporting is possible. Current disease management is supportive in nature and no specific therapy for HHD has been approved by the FDA or the European Medicines Agency. KB111 was developed using the Company’s novel replication-defective, non-integrating HSV-1-based vector and is designed to deliver two copies of the full-length, wild-type ATP2C1 gene following topical application with the goal of increasing functional ATP2C1 levels in the skin to accelerate lesion healing and meaningfully reduce disease burden for HHD patients. The Company presented preclinical data on the KB111 program at the Society for Investigative Dermatology 2025 Annual Meeting earlier this year. Data presented at the meeting demonstrated that KB111 could efficiently deliver ATP2C1 to keratinocytes in vitro and in vivo resulting in increased expression of functional ATP2C1.
The Company expects to dose HHD patients in an intra-patient randomized, double-blind, placebo-controlled, multi-center study evaluating KB111 in 1H 2026.
Financial Results for the Quarter Ended September 30, 2025:
Cash, cash equivalents, and investments totaled $864.2 million as of September 30, 2025.
Product revenue, net totaled $97.8 million and $83.8 million for the quarters ended September 30, 2025 and September 30, 2024, respectively.
Cost of goods sold totaled $4.3 million and $6.7 million for the quarters ended September 30, 2025 and September 30, 2024, respectively.
Research and development expenses for the quarter ended September 30, 2025 were $14.6 million, inclusive of $2.6 million of stock-based compensation, compared to $13.5 million, inclusive of stock-based compensation of $2.3 million for the quarter ended September 30, 2024.
Selling, general, and administrative expenses for the quarter ended September 30, 2025 were $37.6 million, inclusive of stock-based compensation of $10.6 million, compared to $28.7 million, inclusive of stock-based compensation of $11.0 million, for the quarter ended September 30, 2024.
Net income for the quarter ended September 30, 2025 was $79.4 million, or $2.74 per common share (basic) and $2.66 per common share (diluted). Net income for the quarter ended September 30, 2024 was $27.2 million, or $0.95 per common share (basic) and $0.91 per common share (diluted).
For additional information on the Company’s financial results for the three months ended September 30, 2025, please refer to the Form 10-Q filed with the SEC.
Financial Results for the Nine Months Ended September 30, 2025:
Product revenue, net totaled $282.0 million and $199.4 million for the nine months ended September 30, 2025 and September 30, 2024, respectively.
Cost of goods sold totaled $16.5 million and $15.1 million for the nine months ended September 30, 2025 and September 30, 2024, respectively.
Research and development expenses for the nine months ended September 30, 2025 were $43.3 million, inclusive of $7.7 million of stock-based compensation, compared to $40.1 million, inclusive of stock-based compensation of $6.9 million for the nine months ended September 30, 2024.
Selling, general, and administrative expenses for the nine months ended September 30, 2025 were $105.3 million, inclusive of stock-based compensation of $33.1 million, compared to $82.3 million, inclusive of stock-based compensation of $28.9 million, for the nine months ended September 30, 2024.
Net income for the nine months ended September 30, 2025 was $153.4 million, or $5.31 per common share (basic) and $5.14 per common share (diluted). Net income for the nine months ended September 30, 2024 was $43.7 million, or $1.53 per common share (basic) and $1.47 per common share (diluted).
For additional information on the Company’s financial results for the nine months ended September 30, 2025, please refer to the Form 10-Q filed with the SEC.
Financial Guidance
($ in millions) FY 2025 Guidance
Non-GAAP Research and Development ("R&D") and Selling, General and Administrative ("SG&A") expense(1) $145.0 – $155.0
(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 November 3, 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.
About VYJUVEK
VYJUVEK is a non-invasive, topical, redosable genetic medicine designed to deliver two copies of the COL7A1 gene when applied directly to DEB wounds. VYJUVEK was designed to treat DEB at the molecular level by providing the patient’s skin cells the template to make normal COL7 protein, thereby addressing the fundamental disease-causing mechanism. VYJUVEK is approved in the United States, Europe, and Japan.
U.S. INDICATION
VYJUVEK is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in adult and pediatric patients with dystrophic epidermolysis bullosa with mutation(s) in the collagen type VII alpha 1 chain (COL7A1) gene.
IMPORTANT SAFETY INFORMATION
Adverse Reactions
The most common adverse drug reactions (incidence >5%) were itching, chills, redness, rash, cough, and runny nose. These are not all the possible side effects with VYJUVEK. Call your healthcare provider for medical advice about side effects.
To report SUSPECTED ADVERSE REACTIONS, contact Krystal Biotech, Inc. at 1-844-557-9782 or FDA at 1-800-FDA-1088 or View Source
Contraindications
None.
Warnings and Precautions
VYJUVEK gel may be applied by a healthcare provider, a caregiver, or the patient.
After treatment, patients and caregivers should be careful not to touch treated wounds and dressings until the next dressing change.
Wash hands and wear protective gloves when changing wound dressings. Disinfect bandages from the first dressing change with a virucidal agent, and dispose of the disinfected bandages in a separate sealed plastic bag in household waste. Dispose of the subsequent used dressings in a sealed plastic bag in household waste.
Patients should avoid touching or scratching wound sites or wound dressings.
In the event of an accidental exposure flush with clean water for at least 15 minutes.
For more information, see full U.S. Prescribing Information.
(Press release, Krystal Biotech, NOV 3, 2025, View Source [SID1234659289])