Advancing leading pipeline of six
disclosed programs across AAV cardiovascular and LV hematology
portfolios; all milestones remain on track for 2024
Expanding commercial capabilities to support
launch of LV portfolio beginning with KRESLADI™ (marnetegragene
autotemcel) for severe LAD-I; PDUFA date of June 30, 2024
Cash, cash equivalents and investments of
approximately $407.5M; expected operational runway into 2026
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a fully integrated,
late-stage biotechnology company advancing a sustainable pipeline
of genetic therapies for rare disorders with high unmet need, today
reported financial and operational results for the fourth quarter
and year ended December 31, 2023.
“I am pleased with the strong results Rocket delivered in 2023,
as we closed another successful year of progress across all six
disclosed gene therapy programs spanning our AAV cardiovascular and
LV hematology portfolios,” said Gaurav Shah, M.D., Chief Executive
Officer, Rocket Pharma. “We are well poised to make further strides
in 2024, notably expanding our commercial and operational
capabilities to support the anticipated launch of KRESLADI™
(marnetegragene autotemcel) for severe LAD-I, representing Rocket’s
first step in making our gene therapies available to patients who
need them most. At the same time, we are laser focused on
regulatory filings in Fanconi Anemia and continuing to advance our
clinical programs in Danon Disease, PKP2-ACM and PKD in the year
ahead. As we close the quarter, Rocket continues to solidify our
industry leadership in gene therapy across multiple therapeutic
areas and platforms as we seek to meet the needs of patients living
with rare and devastating diseases.”
Recent Pipeline and Operational Updates
- Milestones remain on track for 2024 across pipeline of
adeno-associated virus (AAV) cardiovascular and lentiviral (LV)
vector hematology portfolios. The Marketing Authorisation
Application (MAA) and Biologics License Application (BLA) for
Fanconi Anemia remain on track for filing with the European
Medicines Agency (EMA) and U.S. Food and Drug Administration (FDA),
respectively, in the first half of 2024. Rocket is also progressing
its Phase 2 pivotal study of RP-A501 for Danon Disease, Phase 2
pivotal study of RP-L301 for Pyruvate Kinase Deficiency (PKD),
Phase 1 study of RP-A601 for PKP2-arrhythmogenic cardiomyopathy
(ACM), and IND-enabling studies for BAG3-associated dilated
cardiomyopathy (DCM).
- KRESLADI™ for severe Leukocyte Adhesion
Deficiency-I (LAD-I) on track for PDUFA date of June 30, 2024.
Based on the positive top-line efficacy and safety data from the
global Phase 1/2 study and high unmet need in patients with severe
LAD-I, the FDA accepted the BLA and granted Priority Review for
KRESLADI™. On February 13, 2024, Rocket announced the FDA extended
the Priority Review period by three months, to June 30, 2024, to
allow additional time to review clarifying Chemistry,
Manufacturing, and Controls (CMC) information submitted by Rocket
in response to FDA information requests. The FDA further confirmed
that an advisory committee meeting is not needed. In support of its
first product launch, Rocket continues to expand its commercial and
operational infrastructure, including Qualified Treatment Center
initiation, channel strategy, disease education, and payer
engagement.
- Published data from preclinical study of RP-A601 in
PKP2-ACM. “AAV-Mediated Delivery of Plakophilin-2a Arrests
Progression of Arrhythmogenic Right Ventricular Cardiomyopathy in
Murine Hearts: Preclinical Evidence Supporting Gene Therapy in
Humans” was published in Circulation: Genomic and Precision
Medicine. Results from the study demonstrated the potential of
RP-A601 as a gene therapy for patients with PKP2-ACM.
- Bolstered commercial and operational expertise of Board of
Directors with appointment of R. Keith Woods. Mr. Woods is a
seasoned executive with more than 30 years of experience spanning
commercialization, global operations, business strategy and supply
chain. He recently served as Chief Operating Officer of argenx,
where he led its global commercial organization, including
marketing, market access, medical affairs, program management, and
supply chain, during the company’s successful transition to
commercial stage. Mr. Woods launched Vyvgart, a treatment for a
rare autoimmune condition that causes muscle weakness, generating
more than $1 billion in sales.
Upcoming Investor Conferences
- Cowen’s 44th Annual Health Care Conference: March 5, 2024
- Leerink Partners Global Biopharma Conference: March 12,
2024
- Needham 23rd Annual Virtual Healthcare Conference: April 9,
2024
Fourth Quarter and Full Year 2023 Financial Results
- Cash position. Cash, cash equivalents and investments as
of December 31, 2023, were $407.5 million.
- R&D expenses. Research and development expenses were
$41.7 million and $186.3 million for the three and twelve months
ended December 31, 2023, respectively, compared to $50.0 million
and $165.6 million for the three and twelve months ended December
31, 2022, respectively. The increase in R&D expenses for the
twelve months ended December 31, 2023, was primarily driven by
increased compensation and benefits expense of $16.9 million due to
increased R&D headcount, increased clinical trial costs of
$14.5 million, and increased non-cash stock compensation expense of
$5.0 million, offset by a decrease in manufacturing and development
costs of $17.0 million.
- G&A expenses. General and administrative expenses
were $21.5 million and $73.3 million for the three and twelve
months ended December 31, 2023, respectively, compared to $19.0
million and $58.8 million for the three and twelve months ended
December 31, 2022, respectively. The increase in G&A expenses
for the twelve months ended December 31, 2023, was primarily driven
by increases in commercial preparation expenses which consists of
commercial strategy, medical affairs, market development and
pricing analysis of $8.4 million and increased non-cash stock
compensation expense of $3.4 million.
- Net loss. Net loss was $59.7 million and $245.6 million
or $0.64 and $2.92 per share (basic and diluted) for the three and
twelve months ended December 31, 2023, compared to $66.7 million
and $221.9 million or $0.92 and $3.26 per share (basic and diluted)
for the three and twelve months ended December 31, 2022.
- Shares outstanding. 90,282,267 shares of common stock
were outstanding as of December 31, 2023.
Financial Guidance
- Cash position. As of December 31, 2023, Rocket had cash,
cash equivalents and investments of $407.5 million. Rocket expects
such resources will be sufficient to fund its operations into 2026,
including producing AAV cGMP batches at the Company’s Cranbury,
N.J. R&D and manufacturing facility and continued development
of its six clinical and/or preclinical programs.
About Rocket Pharmaceuticals, Inc.
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT) is a fully
integrated, late-stage biotechnology company advancing a
sustainable pipeline of investigational genetic therapies designed
to correct the root cause of complex and rare disorders. Rocket’s
innovative multi-platform approach allows us to design the optimal
gene therapy for each indication, creating potentially
transformative options that enable people living with devastating
rare diseases to experience long and full lives.
Rocket’s lentiviral (LV) vector-based gene therapies target
hematologic diseases and consist of late-stage programs for Fanconi
Anemia, a difficult to treat genetic disease that leads to bone
marrow failure and potentially cancer, Leukocyte Adhesion
Deficiency-I (LAD-I), a severe pediatric genetic disorder that
causes recurrent and life-threatening infections which are
frequently fatal, and Pyruvate Kinase Deficiency (PKD), a monogenic
red blood cell disorder resulting in increased red cell destruction
and mild to life-threatening anemia.
Our adeno-associated virus (AAV)-based cardiovascular portfolio
includes a late-stage program for Danon Disease, a devastating
heart failure condition resulting in thickening of the heart, an
early-stage program in clinical trials for PKP2-arrhythmogenic
cardiomyopathy (ACM), a life-threatening heart failure disease
causing ventricular arrhythmias and sudden cardiac death, and a
pre-clinical program targeting BAG3-associated dilated
cardiomyopathy (DCM), a heart failure condition that causes
enlarged ventricles.
For more information about Rocket, please visit
www.rocketpharma.com and follow us on LinkedIn, YouTube and X.
Rocket Cautionary Statement Regarding Forward-Looking
Statements
This press release contains forward-looking statements
concerning Rocket’s future expectations, plans and prospects that
involve risks and uncertainties, as well as assumptions that, if
they do not materialize or prove incorrect, could cause our results
to differ materially from those expressed or implied by such
forward-looking statements. We make such forward-looking statements
pursuant to the safe harbor provisions of the Private Securities
Litigation Reform Act of 1995 and other federal securities laws.
All statements other than statements of historical facts contained
in this release are forward-looking statements. You should not
place reliance on these forward-looking statements, which often
include words such as "believe," "expect," "anticipate," "intend,"
"plan," "will give," "estimate," "seek," "will," "may," "suggest"
or similar terms, variations of such terms or the negative of those
terms. These forward-looking statements include, but are not
limited to, statements concerning Rocket’s expectations regarding
the safety and effectiveness of product candidates that Rocket is
developing to treat Fanconi Anemia (FA), Leukocyte Adhesion
Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD), Danon
Disease (DD) and other diseases, the expected timing and data
readouts of Rocket’s ongoing and planned clinical trials, the
expected timing and outcome of Rocket’s regulatory interactions and
planned submissions, Rocket’s plans for the advancement of its DD
program, including its planned pivotal trial, and the safety,
effectiveness and timing of related pre-clinical studies and
clinical trials, Rocket’s ability to establish key collaborations
and vendor relationships for its product candidates, Rocket’s
ability to develop sales and marketing capabilities or enter into
agreements with third parties to sell and market its product
candidates and Rocket’s ability to expand its pipeline to target
additional indications that are compatible with its gene therapy
technologies. Although Rocket believes that the expectations
reflected in the forward-looking statements are reasonable, Rocket
cannot guarantee such outcomes. Actual results may differ
materially from those indicated by these forward-looking statements
as a result of various important factors, including, without
limitation, Rocket’s dependence on third parties for development,
manufacture, marketing, sales and distribution of product
candidates, the outcome of litigation, unexpected expenditures,
Rocket’s competitors’ activities, including decisions as to the
timing of competing product launches, pricing and discounting,
Rocket’s ability to develop, acquire and advance product candidates
into, enroll a sufficient number of patients into, and successfully
complete, clinical studies, Rocket’s ability to acquire additional
businesses, form strategic alliances or create joint ventures and
its ability to realize the benefit of such acquisitions, alliances
or joint ventures, Rocket’s ability to obtain and enforce patents
to protect its product candidates, and its ability to successfully
defend against unforeseen third-party infringement claims, as well
as those risks more fully discussed in the section entitled "Risk
Factors" in Rocket’s Annual Report on Form 10-K for the year ended
December 31, 2022, filed February 28, 2023 with the SEC and
subsequent filings with the SEC including our Quarterly Reports on
Form 10-Q. Accordingly, you should not place undue reliance on
these forward-looking statements. All such statements speak only as
of the date made, and Rocket undertakes no obligation to update or
revise publicly any forward-looking statements, whether as a result
of new information, future events or otherwise.
Three Months Ended December 31, Twelve Months Ended
December 31,
2023
2022
2023
2022
Operating expenses: Research and development
$
41,744
$
50,037
$
186,342
$
165,570
General and administrative
21,535
19,044
73,317
58,773
Total operating expenses
63,279
69,081
259,659
224,343
Loss from operations
(63,279
)
(69,081
)
(259,659
)
(224,343
)
Research and development incentives
-
500
-
500
Interest expense
(470
)
(467
)
(1,875
)
(1,862
)
Interest and other income, net
814
1,245
5,288
3,889
Accretion of discount and amortization of premium on investments,
net
3,275
1,081
10,651
(47
)
Net loss
$
(59,660
)
$
(66,722
)
$
(245,595
)
$
(221,863
)
Net loss per share - basic and diluted
$
(0.64
)
$
(0.92
)
$
(2.92
)
$
(3.26
)
Weighted-average common shares outstanding - basic and diluted
93,336,541
72,889,548
84,009,004
68,148,925
December 31, December 31,
2023
2022
Cash, cash equivalents, and investments
$
407,495
$
399,670
Total assets
566,341
551,807
Total liabilities
73,767
62,121
Total stockholders' equity
492,574
489,686
View source
version on businesswire.com: https://www.businesswire.com/news/home/20240226940459/en/
Media Kevin Giordano media@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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