Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a
commercial-stage biotechnology company, today reported financial
results and key business updates for the third quarter ending
September 30, 2024.
"I am pleased to report that our U.S. launch continues to track
towards our ambitious pre-launch target of achieving 720
reimbursement approvals in the first two years of launch, thanks to
our dedicated team and the unambiguous clinical benefit provided by
VYJUVEK” said Krish S. Krishnan, Chairman and CEO of Krystal
Biotech. “With key milestones recently achieved in Europe and Japan
and the anticipated initiation of the registrational trial to treat
corneal abrasions in DEB patients, we are on the verge of unlocking
expansion opportunities for the franchise that will enable us to
treat DEB patients globally and comprehensively. More importantly,
we continue to progress our deep clinical pipeline. We believe that
our successful clinical readout last quarter for Jeune Aesthetics
and the upcoming two clinical readouts later this year will
showcase the breadth and potential of our redosable platform.”
VYJUVEK® for the
treatment of Dystrophic Epidermolysis Bullosa (DEB)
- The Company recorded $83.8 million in
VYJUVEK net product revenue for the third quarter of 2024. Gross
margin for the quarter was 92%.
- As of October, the Company has secured
over 460 reimbursement approvals for VYJUVEK in the U.S. and
positive access determinations have been achieved for 97% of lives
covered under commercial and Medicaid plans.
- High patient compliance with weekly
treatment while on drug continued at 87% as of the end of the
quarter.
- In September, the Haute Autorité de
Santé in France approved pre-marketing early reimbursed access to
B-VEC under the Accès Précoce program (AP1). DEB patient access to
B-VEC under AP1 is expected to start in 4Q 2024. AP1 allows for
early access to innovative therapies in France prior to European
regulatory approval when a positive benefit/risk ratio is
recognized and when no other therapeutic alternatives are
available.
- The European Medicines Agency (EMA)’s
review of the Company’s Marketing Authorization Application (MAA)
for B-VEC for the treatment of DEB is ongoing. Based on recent
interactions with the EMA, the Company expects a Committee for
Medicinal Products for Human Use (CHMP) opinion on the MAA before
year end and anticipates its first European launch in Germany in 1H
2025.
- In October, the Company filed a Japan
New Drug Application (JNDA) with Japan’s Pharmaceuticals and
Medical Devices Agency (PMDA). A decision on the JNDA by the PMDA
is expected in 2H 2025. The JNDA is expected to receive priority
review given the Orphan Drug Designation status granted to B-VEC in
December 2023.
Respiratory
KB408 for the treatment of alpha-1 antitrypsin deficiency (AATD)
lung disease
- In September, after initiating dosing
in Cohort 2, the Company amended the protocol for its KB408 Phase 1
SERPENTINE-1 study to include mandatory bronchoscopies in this
cohort in an effort to measure alpha-1 antitrypsin expression.
SERPENTINE-1 is an open label, single dose escalation study in
adult patients with AATD with a Pi*ZZ or a Pi*ZNull genotype. The
Company is on track to report interim molecular data from the study
before year end. Details about the study can be found at
www.clinicaltrials.gov under NCT identifier: NCT06049082.
KB407 for the treatment of cystic fibrosis (CF)
- The Company recently activated two
additional clinical sites for its KB407 Phase 1 CORAL-1 study and
expects to report interim molecular data from the study in 1H 2025.
CORAL-1 is a multi-center, dose escalation study evaluating KB407
in patients with CF, regardless of their underlying genotype.
Dosing of the first patient in the third and final cohort in
CORAL-1 is expected before the end of the year. Details of the
study can be found at www.clinicaltrials.gov under NCT identifier
NCT05504837.
Ophthalmology
KB803 (B-VEC formulated as an eyedrop) for ocular complications
of DEB
- The Company continues to enroll
patients in a 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 a registrational study evaluating KB803 effect on
corneal abrasions of DEB. The KB803 registrational IOLITE study is
a single arm, open-label study that is expected to commence in 1H
2025.
Pipeline expansion
- The Company is actively evaluating
multiple preclinical-stage genetic medicine candidates for the
treatment of diseases of the front and back of the eye.
Oncology
Inhaled KB707 for the treatment of solid tumors of the lung
- In August, the Company progressed to
dose expansion in KYANITE-1, the Phase 1 open label, multi-center,
monotherapy, dose escalation and expansion study evaluating inhaled
KB707 in patients with locally advanced or metastatic solid tumors
of the lung. Details of the study can be found at
www.clinicaltrials.gov under NCT identifier NCT06228326.
- In May, the FDA granted Rare Pediatric
Disease Designation (RPDD) for inhaled KB707 for the treatment of
osteosarcoma.
Intratumoral KB707 for the treatment of injectable solid
tumors
- The Company continues to enroll
patients in the dose expansion cohort of OPAL-1, the Phase 1 open
label, multi-center, monotherapy, dose escalation and expansion
study evaluating intratumoral KB707 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.
- In August, the FDA granted RPDD for
intratumoral KB707 for the treatment of rhabdoymyosarcoma.
The Company expects to report initial interim data for KB707
before the end of the year. In addition to being granted RPDDs,
both inhaled and intratumoral KB707 have also been granted Fast
Track Designations by the FDA, regulatory designations which confer
multiple benefits, including more frequent FDA interactions, which
may accelerate KB707 clinical development.
Aesthetics
KB301 for the treatment of aesthetic indications
- In August, Jeune Aesthetics, Inc.
(“Jeune Aesthetics”), a wholly-owned subsidiary of the Company,
announced positive interim safety and efficacy results for KB301 in
the treatment of lateral canthal lines at rest and dynamic wrinkles
of the décolleté, evaluated in Cohorts 3 and 4 of the KB301 Phase 1
study PEARL-1, respectively. Meaningful and sustained improvements
in multiple skin aesthetic attributes, including wrinkles,
crepiness, hydration, and radiance, were reported by the study
investigators and subjects alike in both the décolleté and lateral
canthal regions. Increased subject satisfaction with wrinkle
appearance was also reported. Details of the study can be found at
www.clinicaltrials.gov under NCT identifier NCT04540900.
- Based on the results from Cohort 3 and
4 of PEARL-1, the Company has selected treatment of the dynamic
wrinkles of the décolleté for advanced clinical development and
expects to initiate a Phase 2 study evaluating KB301 in this
indication in 2025. The Company expects to report detailed results
of PEARL-1 Cohorts 3 and 4, including redosing, at future
scientific conference(s).
Dermatology
The Company is continuing preparations to enable initiation of
the Phase 2 portion of its KB105 Phase 1/2 JADE-1 trial evaluating
KB105 for the treatment of lamellar ichthyosis in pediatric
patients in 1H 2025.
Financial Results for the Quarter Ended
September 30, 2024:
- Cash, cash equivalents, and investments
totaled $694.2 million as of September 30, 2024.
- Product revenue, net totaled $83.8
million and $8.6 million for the quarters ended September 30,
2024 and September 30, 2023, respectively.
- Cost of goods sold totaled $6.7 million
and $223 thousand for the quarters ended September 30, 2024
and September 30, 2023, respectively.
- Research and development expenses for
the quarter ended September 30, 2024 were $13.5 million,
inclusive of $2.3 million of stock-based compensation, compared to
$10.6 million, inclusive of stock-based compensation of $2.3
million for the quarter ended September 30, 2023.
- Selling, general, and administrative
expenses for the quarter ended September 30, 2024 were $28.7
million, inclusive of stock-based compensation of $11.0 million,
compared to $23.7 million, inclusive of stock-based compensation of
$6.0 million, for the quarter ended September 30, 2023.
- 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). Net income for
the quarter ended September 30, 2023 was $80.7 million, or
$2.88 per common share (basic) and $2.79 per common share
(diluted).
- For additional information on the
Company’s financial results for the quarter ended
September 30, 2024, please refer to the Form 10-Q filed with
the SEC.
Financial Results for the Nine Months Ended
September 30, 2024:
- Product revenue, net totaled $199.4
million and $8.6 million for the nine months ended
September 30, 2024 and September 30, 2023,
respectively.
- Cost of goods sold totaled $15.1
million and $223 thousand for the nine months ended
September 30, 2024 and September 30, 2023,
respectively.
- Research and development expenses for
the nine months ended September 30, 2024 were $40.0 million,
inclusive of $6.9 million of stock-based compensation, compared to
$35.1 million, inclusive of stock-based compensation of $7.7
million for the nine months ended September 30, 2023.
- Selling, general, and administrative
expenses for the nine months ended September 30, 2024 were
$82.4 million, inclusive of stock-based compensation of $28.9
million, compared to $73.6 million, inclusive of stock-based
compensation of $22.4 million, for the nine months
September 30, 2023.
- 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). Net income for
the nine months ended September 30, 2023 was $2.2 million, or
$0.08 per common share (basic and diluted).
- For additional information on the
Company’s financial results for the nine months ended
September 30, 2024, please refer to the Form 10-Q filed with
the SEC.
Financial Guidance
Our non-GAAP R&D and SG&A expense is now expected to be
between $115 million and $125 million for the full year ending
December 31, 2024. 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 2024 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. This could materially affect the
calculation of forward-looking GAAP combined R&D and SG&A
Expense as it is inherently uncertain. Refer to Non-GAAP Financial
Measures section below for additional information.
Conference Call
The Company will host an investor webcast on November 4,
2024, at 8:30 am ET.
Investors and the general public can access the live webcast
at:
https://www.webcaster4.com/Webcast/Page/3018/51458
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
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 Rare Pediatric Disease Designation
The FDA grants Rare Pediatric Disease Designations for serious
or life-threatening diseases with manifestations in individuals
aged from birth to 18 years, and that affect fewer than 200,000
people in the U.S. Under the FDA's Rare Pediatric Disease Priority
Review Voucher program, a sponsor who receives an approval of a new
drug application or biologics license application for a product for
the prevention or treatment of a rare pediatric disease may be
eligible for a voucher, which can be redeemed to obtain priority
review for any subsequent marketing application, and may be sold or
transferred.
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. VYJUVEK® is the Company’s first commercial
product, the first-ever redosable gene therapy, and the first
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 X (formerly 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 expansion opportunities for the B-VEC franchise
and treating DEB patients globally and comprehensively; the
commercial launch of VYJUVEK in the United States; potential
marketing authorizations in Europe and Japan, including timing of
filings and approvals and related commercial launches; showcasing
the breadth and potential of the Company’s redosable platform;
timing of patient access to B-VEC in France under the AP1 program;
the Company’s expectations regarding reporting interim data from
the Company’s KB408 and KB707 studies, as well as dosing the first
patient in the final cohort of its KB407 study before year end; the
timing of the expected commencement of the KB803 registrational
study, reporting detailed results of Cohorts 3 and 4 of the Phase 1
KB301 study, the commencement of the Phase 2 KB301 study, and the
initiation of the Phase 2 portion of the KB105 Phase 1/2 study; 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 VYJUVEK or product candidates; 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 press 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 press release.
Non-GAAP Financial Measures
This press release includes forward-looking combined R&D and
SG&A expense guidance that is not required by, or presented in
accordance with, U.S. GAAP and should not be considered as an
alternative to R&D and SG&A expense or any other
performance measure derived in accordance with GAAP. The Company
defines non-GAAP combined R&D and SG&A expense as GAAP
combined R&D and SG&A expense excluding stock-based
compensation. The Company cautions investors that amounts presented
in accordance with its definition of non-GAAP combined R&D and
SG&A expense may not be comparable to similar measures
disclosed by competitors because not all companies calculate this
non-GAAP financial measure in the same manner. The Company presents
this non-GAAP financial measure because it considers this measure
to be an important supplemental measure and believes it is
frequently used by securities analysts, investors, and other
interested parties in the evaluation of companies in the Company’s
industry. Management believes that investors’ understanding of the
Company’s performance is enhanced by including this forward-looking
non-GAAP financial measure as a reasonable basis for comparing the
Company’s ongoing results of operations. Management uses this
non-GAAP financial measure for planning purposes, including the
preparation of the Company’s internal annual operating budget and
financial projections; to evaluate the performance and
effectiveness of the Company’s operational strategies; and to
evaluate the Company’s capacity to expand its business. This
non-GAAP financial measure has limitations as an analytical tool,
and should not be considered in isolation, or as an alternative to,
or a substitute for R&D and SG&A expense or other financial
statement data presented in accordance with GAAP in the Company’s
consolidated financial statements. The Company has not provided a
quantitative reconciliation of forecasted non-GAAP combined R&D
and SG&A expense to forecasted GAAP combined R&D and
SG&A expense because the Company is unable, without making
unreasonable efforts, to calculate the reconciling item,
stock-based compensation expenses, with confidence. This item,
which could materially affect the computation of forward-looking
GAAP combined R&D and SG&A expense, is inherently uncertain
and depends on various factors, some of which are outside of the
Company’s control.
CONTACTInvestors and
Media:Stéphane Paquette, PhDKrystal
Biotechspaquette@krystalbio.com
Condensed Consolidated Balance Sheet Data:
|
September 30,2024 |
|
December 31,2023 |
(in thousands) |
(unaudited) |
|
|
Balance sheet data: |
|
|
|
Cash and cash equivalents |
$ |
373,966 |
|
|
$ |
358,328 |
|
Short-term investments |
|
214,358 |
|
|
|
173,850 |
|
Long-term investments |
|
105,888 |
|
|
|
61,954 |
|
Total assets |
|
982,318 |
|
|
|
818,355 |
|
Total liabilities |
|
96,472 |
|
|
|
39,714 |
|
Total stockholders’ equity |
$ |
885,846 |
|
|
$ |
778,641 |
|
Condensed Consolidated Statements of
Operations:
|
Three Months Ended September 30, |
|
|
|
|
2024 |
|
|
|
2023 |
|
|
Change |
(in thousands, except per
share data) |
(unaudited) |
|
|
Revenue |
|
|
|
|
|
Product revenue, net |
$ |
83,841 |
|
|
$ |
8,556 |
|
|
$ |
75,285 |
|
Expenses |
|
|
|
|
|
Cost of goods sold |
|
6,684 |
|
|
|
223 |
|
|
|
6,461 |
|
Research and development |
|
13,511 |
|
|
|
10,629 |
|
|
|
2,882 |
|
Selling, general, and administrative |
|
28,713 |
|
|
|
23,697 |
|
|
|
5,016 |
|
Litigation settlement |
|
12,500 |
|
|
|
— |
|
|
|
12,500 |
|
Total operating expenses |
|
61,408 |
|
|
|
34,549 |
|
|
|
26,859 |
|
Income (loss) from operations |
|
22,433 |
|
|
|
(25,993 |
) |
|
|
48,426 |
|
Other
income |
|
|
|
|
|
Gain from Sale of Priority Review Voucher |
|
— |
|
|
|
100,000 |
|
|
|
(100,000 |
) |
Interest and other income, net |
|
7,336 |
|
|
|
6,740 |
|
|
|
596 |
|
Income before income taxes |
|
29,769 |
|
|
|
80,747 |
|
|
|
(50,978 |
) |
Income tax expense |
|
(2,589 |
) |
|
|
— |
|
|
|
(2,589 |
) |
Net income |
$ |
27,180 |
|
|
$ |
80,747 |
|
|
$ |
(53,567 |
) |
|
|
|
|
|
|
Net income per common
share: |
|
|
|
|
|
Basic |
$ |
0.95 |
|
|
$ |
2.88 |
|
|
|
Diluted |
$ |
0.91 |
|
|
$ |
2.79 |
|
|
|
|
|
|
|
|
|
Weighted-average common shares
outstanding: |
|
|
|
|
|
Basic |
|
28,716 |
|
|
|
28,042 |
|
|
|
Diluted |
|
29,902 |
|
|
|
28,892 |
|
|
|
|
Nine Months Ended September 30, |
|
|
|
|
2024 |
|
|
|
2023 |
|
|
Change |
(in thousands, except per
share data) |
(unaudited) |
|
|
Revenue |
|
|
|
|
|
Product revenue, net |
$ |
199,376 |
|
|
$ |
8,556 |
|
|
$ |
190,820 |
|
Expenses |
|
|
|
|
|
Cost of goods sold |
|
15,112 |
|
|
|
223 |
|
|
|
14,889 |
|
Research and development |
|
40,050 |
|
|
|
35,061 |
|
|
|
4,989 |
|
Selling, general, and administrative |
|
82,398 |
|
|
|
73,637 |
|
|
|
8,761 |
|
Litigation settlement |
|
37,500 |
|
|
|
12,500 |
|
|
|
25,000 |
|
Total operating expenses |
|
175,060 |
|
|
|
121,421 |
|
|
|
53,639 |
|
Income (loss) from operations |
|
24,316 |
|
|
|
(112,865 |
) |
|
|
137,181 |
|
Other
income |
|
|
|
|
|
Gain from Sale of Priority Review Voucher |
|
— |
|
|
|
100,000 |
|
|
|
(100,000 |
) |
Interest and other income, net |
|
22,430 |
|
|
|
15,105 |
|
|
|
7,325 |
|
Income before income taxes |
|
46,746 |
|
|
|
2,240 |
|
|
|
44,506 |
|
Income tax expense |
|
(3,066 |
) |
|
|
— |
|
|
|
(3,066 |
) |
Net income |
$ |
43,680 |
|
|
$ |
2,240 |
|
|
$ |
41,440 |
|
|
|
|
|
|
|
Net income per common
share: |
|
|
|
|
|
Basic |
$ |
1.53 |
|
|
$ |
0.08 |
|
|
|
Diluted |
$ |
1.47 |
|
|
$ |
0.08 |
|
|
|
|
|
|
|
|
|
Weighted-average common shares
outstanding: |
|
|
|
|
|
Basic |
|
28,537 |
|
|
|
26,812 |
|
|
|
Diluted |
|
29,669 |
|
|
|
27,385 |
|
|
|
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