Krystal Biotech Announces Second Quarter 2023 Financial Results and Operational Highlights
• VYJUVEK™ approved in the
• 121 Patient Start Forms in the first six weeks of launch
• First cystic fibrosis patient dosed in Phase 1 Clinical Trial of KB407
• IND for KB707, Krystal’s first oncology candidate for solid tumors, accepted by FDA and granted Fast Track Designation
• Strong balance sheet, closing the quarter with
“2023 is off to a very strong start and we expect the momentum to continue for the rest of the year. In the first half of the year, we received FDA approval for VYJUVEK, dosed our first patient in the cystic fibrosis trial and obtained IND clearance for our first oncology candidate,” said
May 19, the Company announced that VYJUVEK (beremagene geperpavec-svdt, or B-VEC) was approved by the U.S. Food & Drug Administration(FDA) for the treatment of patients six months of age or older with either recessive or dominant dystrophic epidermolysis bullosa (DEB) to be administered by a healthcare professional either in a healthcare professional setting (e.g., clinic) or a home setting.
- As of
June 30, the Company received 121 Patient Start Forms of which 30 start forms were generated for patients with dominant dystrophic epidermolysis bullosa. The Company will continue to report on the number of Patient Start Forms submitted to the Company for the first three quarters following VYJUVEK approval and will transition to reporting on the number of Patients on Therapy beginning in the first quarter of 2024.
- The Company also received positive coverage determinations from several of the national health plans, including UnitedHealthcare, as well as regional plans such as BlueCross BlueShield, state Medicaid plans and other smaller regional health plans. The Company expects that additional payer policies will continue to publish that cover VYJUVEK for both recessive and dominant DEB patients.
- In July, the Company received a positive opinion from the
European Medical Agency(EMA) on the Pediatric Investigation Plan for B-VEC for the treatment of DEB with no additional studies required. The Company plans to submit a market authorization application to the EMA in the second half of 2023 and anticipates a potential launch in the EU in the second half of 2024.
- In July, the Pharmaceuticals and
Medical Agencyin Japanaccepted the open label extension study of B-VEC, and the Company intends to start an open label extension study of B-VEC in Japanin the second half of 2023 and file for approval in Japanin 2024. The Company will provide details of the open label extension study when it doses the first patient in Japan.
KB407 for the treatment of Cystic Fibrosis (CF)
- In July, the Company announced that the first patient had been dosed at the
Cystic Fibrosis Institute of Chicagoin the Company’s Phase 1 CORAL-1/ U.S.study evaluating KB407, a mutation agnostic genetic medicine, delivered via a nebulizer, for the treatment of patients with CF. The Phase 1/CORAL-1 study is a multi-center, dose-escalation trial of KB407 in patients with CF, regardless of their underlying genotype. The Company anticipates announcing data from the Phase 1 study in 2024. Details of the Phase 1 study can be found at www.clinicaltrials.gov under NCT identifier NCT05504837.
KB408 for the treatment of Alpha-1 Antitrypsin Deficiency (AATD)
- The Company intends to file an Investigational New Drug (IND) application with the FDA and initiate a Phase 1 clinical trial of KB408 for the treatment of AATD in the second half of 2023.
KB707 for the treatment of solid tumors
- In July, the Company announced that the FDA had accepted its IND application to evaluate intratumoral KB707 in a clinical trial to treat patients with locally advanced or metastatic solid tumor malignancies. KB707 targets solid tumors that are accessible via intratumoral injection or inhalation, and we intend to advance both routes of administration into clinical studies. Details of the Phase 1 (OPAL-1) study can be found www.clinicaltrials.gov under NCT identifier NCT05970497. The Company expects to dose the first patient in the second half of 2023.
- The FDA also granted KB707 fast track designation to delay disease progression in the treatment of patients with anti-PD-1 relapsed/refractory locally advanced or metastatic melanoma.
- The Company plans to file an amendment to the existing KB707 IND in the second half of 2023 to allow the Company to evaluate inhaled (nebulized) KB707 in a clinical trial to treat tumors in a patient’s lungs. The Company expects to dose the first patient with inhaled KB707 in the first half of 2024.
KB105 for the treatment of TGM1 deficient autosomal recessive congenital ichthyosis
- The Company plans to initiate the Phase 2 cohort of its KB105-02 (JADE-1) trial in 2024 and is currently working with the FDA to ensure alignment on the clinical endpoints in the pivotal trial prior to initiating a Phase 2 study in pediatric patients. The Phase 2 cohort will enroll both pediatric and adult patients with TGM1 deficient autosomal recessive congenital ichthyosis for assessment of KB105 safety and efficacy. Details of the Phase 1/2 study can be found at www.clinicaltrials.gov under NCT identifier NCT04047732.
KB104 for the treatment of Netherton Syndrome
- With an expanding pipeline portfolio, the Company now anticipates filing an IND application with the FDA and initiating a clinical trial of KB104 to treat patients with Netherton Syndrome in late 2024.
KB301 for the treatment of aesthetic indications
- In April,
Jeune Aesthetics, Inc., a wholly-owned subsidiary of the Company, announced the dosing of the first subject in the Phase 1, Cohort 3 study of KB301 for the improvement of lateral canthal lines at rest. The study is on-going, and the Company plans to announce results from this study in the second half of 2023. Details of the Phase 1 study can be found at www.clinicaltrials.gov under NCT identifier NCT04540900.
Financial results for the quarter ended
- Cash, cash equivalents, and investments totaled
$505.9 millionon June 30, 2023.
- Research and development expenses for the quarter ended
June 30, 2023were $12.1 million, inclusive of $2.9 millionof stock-based compensation, compared to $10.9 million, inclusive of stock-based compensation of $2.0 millionfor the quarter ended June 30, 2022.
- General and administrative expenses for the quarter ended
June 30, 2023were $25.9 million, inclusive of stock-based compensation of $8.5 million, compared to $17.9 million, inclusive of stock-based compensation of $6.2 million, for the quarter ended June 30, 2022.
- Net losses for the quarters ended
June 30, 2023and 2022 were $33.2 millionand $28.1 million, or $(1.25)and $(1.10), respectively, per common share (basic and diluted).
- For additional information on the Company’s financial results for the quarter ended
June 30, 2023, please refer to the Form 10-Q filed with the SEC.
Financial results for the six months ended
- Research and development expenses for the six months ended
June 30, 2023were $24.4 million, inclusive of stock-based compensation of $5.4 million, compared to $20.2 million, inclusive of stock-based compensation of $3.4 millionfor the six months ended June 30, 2022.
- General and administrative expenses for the six months ended
June 30, 2023were $49.9 million, inclusive of stock-based compensation of $16.4 million, compared to $33.8 million, inclusive of stock-based compensation of $11.3 millionfor the six months ended June 30, 2022.
- Net losses for the six months ended
June 30, 2023and 2022 were $78.5 millionand $78.1 millionor $(3.00)and $(3.08), respectively, per common share (basic and diluted).
- For additional information on the Company’s financial results for the six months ended
June 30, 2023, please refer to the Form 10-Q filed with the SEC.
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.
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
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
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.
For more information, see full
About Fast Track Designation
Fast Track Designation is designed to facilitate the development and expedite the review of drugs to treat serious conditions and treat a serious or unmet medical need, enabling drugs to reach patients sooner. Clinical programs with Fast Track designation may benefit from early and frequent communication with the FDA throughout the regulatory review process, and such clinical programs may be eligible to apply for Accelerated Approval and Priority Review if relevant criteria are met.
Any statements in this press release about future expectations, plans and prospects for
The Company is using the Aerogen Solo® Nebulizer System and Aerogen® Ultra in its Phase 1 CORAL-1/
Investors and Media:
Consolidated Balance Sheet Data:
|Balance sheet data:|
|Cash and cash equivalents||$||275,875||$||161,900|
|Total stockholders’ equity||$||653,114||$||522,231|
Consolidated Statements of Operations:
|Three Months Ended
|(In thousands, except shares and per share data)||2023||2022||Change|
|Research and development||$||12,144||$||10,890||$||1,254|
|General and administrative||25,904||17,863||8,041|
|Total operating expenses||38,048||28,753||9,295|
|Loss from operations||(38,048||)||(28,753||)||(9,295||)|
|Interest and other income, net||4,838||645||4,193|
|Net loss per common share: Basic and diluted||$||(1.25||)||$||(1.10||)|
|Weighted-average common shares outstanding: Basic and diluted||26,656,883||25,545,167|
|Six Months Ended
|Research and development||$||24,432||$||20,204||$||4,228|
|General and administrative||49,939||33,771||16,168|
|Total operating expenses||86,871||78,975||7,896|
|Loss from operations||(86,871||)||(78,975||)||(7,896||)|
|Interest and other income, net||8,364||902||7,462|
|Net loss per common share: Basic and diluted||$||(3.00||)||$||(3.08||)|
|Weighted-average common shares outstanding: Basic and diluted||26,187,161||25,331,000|
Source: Krystal Biotech, Inc.