Krystal Biotech Announces Second Quarter 2023 Financial Results and Operational Highlights

临床1期财报快速通道基因疗法临床申请
Krystal Biotech Announces Second Quarter 2023 Financial Results and Operational Highlights
VYJUVEK™ approved in the U.S. as the first and only topical redosable gene therapy for the treatment of
dystrophic epidermolysis bullosa
• 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 $505.9 million in cash, cash equivalents and investments
PITTSBURGH, Aug. 07, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, today reported financial results and key operational progress updates for the second quarter ended June 30, 2023.
“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 Krish S. Krishnan, Chairman and CEO of Krystal Biotech. “I am particularly thrilled with the enthusiasm that physicians and patients are expressing for VYJUVEK in these early days of launch. With 121 Patient Start Forms in the first six weeks since approval, I am pleased with the initial pace of the launch, and we look forward to promptly initiating treatment for these patients.”
On 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 Agency in Japan accepted the open label extension study of B-VEC, and the Company intends to start an open label extension study of B-VEC in Japan in the second half of 2023 and file for approval in Japan in 2024. The Company will provide details of the open label extension study when it doses the first patient in Japan.
Respiratory
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 Chicago in 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)
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.
Oncology
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.
Dermatology
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
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.
Aesthetics
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 June 30, 2023:
Cash, cash equivalents, and investments totaled $505.9 million on June 30, 2023.
Research and development expenses for the quarter ended June 30, 2023 were $12.1 million, inclusive of $2.9 million of stock-based compensation, compared to $10.9 million, inclusive of stock-based compensation of $2.0 million for the quarter ended June 30, 2022.
General and administrative expenses for the quarter ended June 30, 2023 were $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, 2023 and 2022 were $33.2 million and $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 June 30, 2023:
Research and development expenses for the six months ended June 30, 2023 were $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 million for the six months ended June 30, 2022.
General and administrative expenses for the six months ended June 30, 2023 were $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 million for the six months ended June 30, 2022.
Net losses for the six months ended June 30, 2023 and 2022 were $78.5 million and $78.1 million or $(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.
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 http://www.fda.gov/medwatch.
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.
For more information, see full U.S. Prescribing Information.
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.
Krystal Biotech, Inc. (NASDAQ: KRYS) is a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs. VYJUVEKTM is the Company’s first commercial product, the first-ever redosable gene therapy, and the only medicine approved by the FDA for the treatment of
dystrophic epidermolysis bullosa
. The Company is rapidly advancing a robust preclinical and clinical pipeline of investigational genetic medicines in respiratory,
oncology
, dermatology, ophthalmology, and aesthetics. Krystal Biotech is headquartered in Pittsburgh, Pennsylvania. For more information, please visit http://www.krystalbio.com, and follow @KrystalBiotech on LinkedIn and Twitter.
Jeune Aesthetics, Inc., a wholly-owned subsidiary of Krystal Biotech, Inc., is a biotechnology company leveraging a clinically validated gene-delivery platform to develop products to fundamentally address – and reverse – the biology of aging and/or damaged skin. For more information, please visit http://www.jeuneinc.com.
Forward-Looking Statements
Any statements in this press release about future expectations, plans and prospects for Krystal Biotech, Inc. or Jeune Aesthetics, Inc., including statements about our commercial launch of VYJUVEK; our plans to report on Patient Start Forms and Patients on Therapy; our plans to submit a market authorization application to the EMA in the second half of 2023 and our anticipation of a potential launch of B-VEC in the EU in the second half of 2024; our plans for an open label extension study of B-VEC in Japan in the second half of 2023, filing for approval in Japan in 2024, and the timing of our provision of details of the open label extension study; our expectation that we will announce data from the Phase 1 study of KB407 in 2024; our intention to file an 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; our expectations regarding dosing the first patient with KB707 in the second half of 2023; our plans to file an amendment to the existing KB707 IND in the second half of 2023 to evaluate inhaled KB707 in a clinical trial in the first half 2024; our plans to initiate the Phase 2 cohort of the KB105-02 (JADE-1) trial in 2024; our plans to file an IND application and initiate a clinical trial of KB104 in late 2024; our expectation that we will announce results of the Phase 1, Cohort 3 study of KB301 for the improvement of lateral canthal lines at rest in the second half of 2023, and other statements containing the words “anticipate,” “believe,” “estimate,” “expect,” “intend,” “may,” “plan,” “predict,” “project,” “target,” “potential,” “likely,” “will,” “would,” “could,” “should,” “continue,” and similar expressions, constitute forward-looking statements within the meaning of The Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including: uncertainties associated with regulatory review of clinical trials and applications for marketing approvals, the availability or commercial potential of product candidates including VYJUVEK, the sufficiency of cash resources and need for additional financing and such other important factors as are set forth under the caption “Risk Factors” in the Company’s annual and quarterly reports on file with the U.S. Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company’s views as of the date of this release. The Company anticipates that subsequent events and developments will cause its views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, it specifically disclaims any obligation to do so. These forward-looking statements should not be relied upon as representing the Company’s views as of any date subsequent to the date of this release.
Disclosures
The Company is using the Aerogen Solo® Nebulizer System and Aerogen® Ultra in its Phase 1 CORAL-1/U.S. study evaluating KB407.
CONTACT:
Investors and Media:
Meg Dodge
mdodge@krystalbio.com
Consolidated Balance Sheet Data:
(In thousands)June 30,
2023 December 31,
2022 Balance sheet data: Cash and cash equivalents$275,875 $161,900 Short-term investments 201,642 217,271 Long-term investments 28,410 4,621 Total assets 684,026 558,450 Total liabilities 30,912 36,219 Total stockholders’ equity$653,114 $522,231
Consolidated Statements of Operations:
Three Months Ended
June 30, (In thousands, except shares and per share data) 2023 2022 ChangeExpenses Research and development$12,144 $10,890 $1,254 General and administrative 25,904 17,863 8,041 Total operating expenses 38,048 28,753 9,295 Loss from operations (38,048) (28,753) (9,295)Other Income Interest and other income, net 4,838 645 4,193 Net loss$(33,210) $(28,108) $(5,102) Net loss per common share: Basic and diluted$(1.25) $(1.10) Weighted-average common shares outstanding: Basic and diluted 26,656,883 25,545,167
Six Months Ended June 30, 2023 2022 Change(In thousands)(unaudited) Expenses Research and development$24,432 $20,204 $4,228 General and administrative 49,939 33,771 16,168 Litigation settlement 12,500 25,000 (12,500)Total operating expenses 86,871 78,975 7,896 Loss from operations (86,871) (78,975) (7,896)Other Income Interest and other income, net 8,364 902 7,462 Net loss$(78,507) $(78,073) $(434) Net loss per common share: Basic and diluted$(3.00) $(3.08) Weighted-average common shares outstanding: Basic and diluted 26,187,161 25,331,000


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适应症
营养不良性大疱性表皮松解,囊性纤维化,实体瘤
[+9]
靶点
A1AT,PD-1,TGM1
[+1]
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