On February 19, 2025 Krystal Biotech, Inc. (the "Company") (NASDAQ: KRYS), a commercial-stage biotechnology company, reported financial results for the fourth quarter and full year ending December 31, 2024 and provided a business update (Press release, Krystal Biotech, FEB 19, 2025, View Source [SID1234650387]).
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"Last year, our commercial and financial strength allowed us to deliver significant earnings growth, continue to build out a global footprint, and advance multiple clinical stage programs from our industry-leading HSV-1 based gene delivery platform," said Krish S. Krishnan, Chairman and CEO of Krystal Biotech. "Building on this foundation, our focus in 2025 will be on executing the global launch of VYJUVEK and progressing our rare disease and oncology programs through key milestones to bring our redosable genetic medicines closer to patients."
VYJUVEK (beremagene geperpavec-svdt, or B-VEC)
for the Treatment of Dystrophic Epidermolysis Bullosa (DEB)
The Company recorded $91.1 million and $290.5 million in VYJUVEK net product revenue for the fourth quarter and full year of 2024. Gross margin for the fourth quarter was 95%.
As of February 2025, the Company has secured over 510 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 85% as of the end of 2024.
The Company expects a Committee for Medicinal Products for Human Use opinion on its European Marketing Authorization Application in 1Q 2025.
The Pharmaceuticals and Medical Devices Agency’s review of the Company’s Japan New Drug Application is ongoing and on track for a decision in 2H 2025.
Ophthalmology
KB803 for ocular complications of DEB
The Company has enrolled approximately 50 DEB patients in an 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 effect on corneal abrasions of DEB. The registrational Phase 3 study, IOLITE, is a single arm, open-label study that is expected to commence in 1H 2025.
Pipeline expansion
The Company is actively evaluating multiple, internal preclinical-stage genetic medicine candidates for the treatment of diseases of the front and back of the eye.
Respiratory
KB407 for the treatment of cystic fibrosis (CF)
In January 2025, the Cystic Fibrosis Foundation (CFF) Therapeutic Development Network (TDN) Clinical Research Executive Committee granted full sanctioning of the Company’s KB407 Phase 1 CORAL-1 study protocol.
In December 2024, the Company announced that single and repeat dosing of KB407 was safe and well-tolerated by patients in both Cohort 1 and Cohort 2 of the ongoing KB407 Phase 1 CORAL-1 study. The Company expects to report interim molecular data for Cohort 3 patients in mid-2025. CORAL-1 is a multi-center, dose escalation study evaluating KB407 in patients with CF, regardless of their underlying genotype. 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
In December 2024, the Company announced successful SERPINA1 gene delivery and functional alpha-1 antitrypsin expression reaching therapeutic levels as part of an interim clinical update for Cohorts 1 and 2 of its ongoing KB408 Phase 1 SERPENTINE-1 study. Inhaled KB408 was safe and well-tolerated at both tested dose levels. Based on this promising initial data, the Company has simultaneously expanded Cohort 2 and opened Cohort 3 of SERPENTINE-1 for more comprehensive molecular assessments at both dose levels and expects to report results from both cohorts in 2H 2025. SERPENTINE-1 is an open label, single dose escalation study in adult patients with AATD with a Pi*ZZ or a Pi*ZNull genotype. Details about the study can be found at www.clinicaltrials.gov under NCT identifier: NCT06049082.
Oncology
Inhaled KB707 for the treatment of solid tumors of the lung
In December 2024, the Company announced an initial clinical update for the monotherapy dose escalation and expansion cohorts of KYANITE-1, an ongoing, 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. Early clinical evidence of monotherapy activity was observed in heavily pre-treated patients with advanced non-small cell lung cancer treated with inhaled KB707, achieving an objective response rate of 27% and disease control rate of 73% as of data cut-off. Inhaled KB707 was also reported to be safe and generally well tolerated and amenable to administration in an outpatient setting, with no Grade 4 or 5 adverse events observed. Enrollment in KYANITE-1 is ongoing. 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 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
Building on the previously reported positive interim safety and efficacy results for KB301 in the treatment of dynamic wrinkles of the décolleté, Jeune Aesthetics, Inc. ("Jeune Aesthetics"), a wholly-owned subsidiary of the Company, has initiated development of a décolleté-specific evaluation scale necessary for advanced clinical development. Jeune Aesthetics expects to complete scale development and dose the first subject in a randomized, placebo-controlled Phase 2 study evaluating KB301 for the treatment of dynamic wrinkles of the décolleté in 2H 2025.
KB304 for the treatment of aesthetic indications
In November 2024, Jeune Aesthetics dosed the first subject in an ongoing, randomized and placebo-controlled Phase 1 study PEARL-2 evaluating its second clinical-stage investigational aesthetic product KB304 for the treatment of wrinkles. KB304 was developed using the Company’s novel replication-defective, non-integrating HSV-1-based vector and is designed to deliver COL3A1 and ELN transgenes following intradermal injection to increase both type III collagen and elastin levels in aging skin. 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
The Company is now planning on initiating 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.
Financial Results for the Quarter Ended December 31, 2024:
Cash, cash equivalents, and investments totaled $749.6 million as of December 31, 2024.
Product revenue, net totaled $91.1 million and $42.1 million for the quarters ended December 31, 2024 and December 31, 2023, respectively.
Cost of goods sold totaled $4.9 million and $2.9 million for the quarters ended December 31, 2024 and December 31, 2023, respectively.
Research and development expenses for the quarter ended December 31, 2024 were $13.5 million, inclusive of $2.3 million of stock-based compensation, compared to $11.4 million, inclusive of stock-based compensation of $2.4 million for the quarter ended December 31, 2023.
Selling, general, and administrative expenses for the quarter ended December 31, 2024 were $31.3 million, inclusive of stock-based compensation of $11.0 million, compared to $24.8 million, inclusive of stock-based compensation of $7.5 million, for the quarter ended December 31, 2023.
Net income for the quarter ended December 31, 2024 was $45.5 million, or $1.58 per common share (basic) and $1.52 per common share (diluted). Net income for the quarter ended December 31, 2023 was $8.7 million, or $0.31 per common share (basic) and $0.30 per common share (diluted).
Financial Results for the Twelve Months Ended December 31, 2024:
Product revenue, net totaled $290.5 million and $50.7 million for the twelve months ended December 31, 2024 and December 31, 2023, respectively.
Cost of goods sold totaled $20.1 million and $3.1 million for the twelve months ended December 31, 2024 and December 31, 2023, respectively.
Research and development expenses for the twelve months ended December 31, 2024 were $53.6 million, inclusive of $9.2 million of stock-based compensation, compared to $46.4 million, inclusive of stock-based compensation of $10.1 million for the twelve months ended December 31, 2023.
Selling, general, and administrative expenses for the twelve months ended December 31, 2024 were $113.7 million, inclusive of stock-based compensation of $39.9 million, compared to $98.4 million, inclusive of stock-based compensation of $29.9 million, for the twelve months December 31, 2023.
Net income for the twelve months ended December 31, 2024 was $89.2 million, or $3.12 per common share (basic) and $3.00 per common share (diluted). Net income for the twelve months ended December 31, 2023 was $10.9 million, or $0.40 per common share (basic) and $0.39 per common share (diluted).
For additional information on the Company’s financial results for the twelve months ended December 31, 2024, please refer to the Form 10-K filed with the SEC.
Financial Guidance
($ in millions) FY 2025 Guidance
Non-GAAP Research and Development ("R&D") and Selling, General and Administrative ("SG&A") expense(1) $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 February 19, 2025, at 8:30 am ET.
Investors and the general public can access the live webcast at:
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 gene therapy 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.
Indication
VYJUVEK is a herpes-simplex virus type 1 (HSV-1) vector-based gene therapy indicated for the treatment of wounds in patients six months of age and older 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 must be applied by a healthcare provider.
After treatment, patients and caregivers should be careful not to touch treated wounds and dressings for 24 hours.
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.