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.”
VYJUVEKTM
- 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)
- 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 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.
About Krystal Biotech, Inc.
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.
About Jeune Aesthetics,
Inc.
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 DodgeKrystal Biotechmdodge@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 EndedJune 30, |
|
|
(In thousands, except
shares and per share data) |
|
2023 |
|
|
|
2022 |
|
|
Change |
Expenses |
|
|
|
|
|
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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