Agreement reached with FDA on Phase 2 pivotal
study design of RP-L301 for PKD to support accelerated approval;
Initiating single-arm, 10-patient pivotal study with primary
endpoint of ≥1.5 point Hgb improvement at 12 months
Initiated Phase 2 pivotal trial of RP-A501 for
Danon Disease following FDA alignment
Initiated Phase 1 study of RP-A601 for
PKP2-ACM
FDA accepted BLA with Priority Review for
KRESLADITM (marnetegragene autotemcel) for severe LAD-I; PDUFA
target action date is March 31, 2024
Product filings for RP-L102 for Fanconi Anemia
anticipated in the first half of 2024 in the U.S. and Europe
Cash, cash equivalents and investments of
approximately $437.2M; expected operational runway through 2025
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading
late-stage biotechnology company advancing an integrated and
sustainable pipeline of genetic therapies for rare disorders with
high unmet need, today reported financial results for the quarter
ending September 30, 2023, and updates from the Company’s key
pipeline developments, business operations and upcoming
milestones.
“I’m very pleased with Rocket’s results this quarter, as we
realized significant regulatory milestones across our pipeline,
including reaching FDA alignment on the study design for the
10-patient Phase 2 pivotal trial of RP-L301 for the treatment of
severe PKD and securing the FDA’s acceptance of Rocket’s BLA for
RP-L201, now named KRESLADITM, for the treatment of severe LAD-I,”
said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma. “At
the same time, we initiated the Phase 2 pivotal trial of RP-A501
for Danon Disease following FDA alignment and the Phase 1 study of
RP-A601 for PKP2-ACM, which move us substantially closer to
delivering potential one-time, curative therapies to patients
facing these two devastating cardiac diseases.”
Dr. Shah continued, “As we bring 2023 to a close, we have made
remarkable progress across the breadth of our AAV cardiovascular
and LV hematology portfolios. Four programs have now reached
pivotal studies, one of which is under review for approval, thanks
to strong collaboration with health authorities and scientific
partners, as we collectively seek to fulfill the high unmet needs
of patients with rare and life-threatening diseases.”
Key Pipeline and Operational Updates
AAV Cardiovascular Portfolio
Danon Disease
- Initiated Phase 2 pivotal trial of RP-A501 for Danon Disease
following FDA alignment. The global Phase 2 multi-center trial
is a single arm study with an external comparator arm and includes
12 male patients with Danon Disease. The trial is evaluating the
safety and efficacy of RP-A501 at a dose level of 6.7 x 1013 GC/kg
and includes a pediatric safety run-in (n=2). The co-primary
endpoint is composed of LAMP2 protein expression and left
ventricular (LV) mass index and will be assessed at 12 months for
accelerated approval.
- Supported peer-reviewed expert consensus paper to increase
awareness of diagnosis and clinical management of patients with
Danon Disease. “International Consensus on Differential
Diagnosis and Management of Patients with Danon Disease: JACC
State-of-the-Art Review” was published in the Journal of the
American College of Cardiology. Highlights of the publication
include a review of diagnosing Danon Disease emphasizing the
importance of genetic testing upon clinical suspicion, natural
history, management recommendations and recent advances in
potential gene therapy treatment.
- Filed Clinical Trial Application (CTA)/Investigational
Medicinal Product Dossier (IMPD) for RP-A501 to EMA and MHRA.
Rocket is working towards initiation of Phase 2 pivotal trial
activities in Europe and the UK.
PKP2 Arrhythmogenic Cardiomyopathy
(PKP2-ACM)
- Initiated Phase 1 trial of RP-A601. The multi-center
Phase 1, dose escalation trial will evaluate the safety and
preliminary efficacy of RP-A601 in at least six adult PKP2-ACM
patients with ICDs and overall high risk for arrhythmias. The study
will assess the impact of RP-A601 on PKP2 myocardial protein
expression, cardiac biomarkers, and clinical predictors of
life-threatening ventricular arrhythmias and sudden cardiac death.
Patients in the dose-escalation trial will receive a single dose of
RP-A601. The starting dose will be 8 x 1013 GC/kg.
LV Hematology Portfolio
Leukocyte Adhesion Deficiency-I
(LAD-I)
- BLA accepted by FDA with Priority Review for KRESLADITM
(marnetegragene autotemcel). Based on the positive top-line
efficacy and safety data from the global Phase 1/2 study, the FDA
accepted the BLA and also granted Priority Review for KRESLADITM,
also known as RP-L201. This represents Rocket’s first product
filing and is a significant milestone for both the Company and
patients whose only current available treatment option is bone
marrow transplant, which has substantial morbidity and mortality,
and may not be available in time. The PDUFA date set by the FDA is
March 31, 2024.
Fanconi Anemia (FA)
- Product filings for RP-L102 anticipated in the first half of
2024 in the U.S. and Europe. Rocket is finalizing the CMC
package with the FDA.
Pyruvate Kinase Deficiency
(PKD)
- Reached agreement with FDA on study design of Phase 2
pivotal trial of RP-L301. Based on positive safety and efficacy
data from the Phase 1 study, Rocket has aligned with the FDA on the
pivotal study design to support accelerated approval and is
initiating a 10-patient, single-arm Phase 2 pivotal trial with a
primary endpoint of ≥1.5 point Hgb improvement at 12 months.
- Presented positive updated clinical data from Phase 1
trial of RP-L301 at the ESGCT Annual Meeting. Updated data
demonstrated sustained efficacy in both adult patients with up to
36 months follow-up. Hemoglobin improvement relative to
pre-treatment baseline was observed in both patients in the
pediatric cohort with up to six months of available follow-up.
Results also indicated favorable safety profile for RP-L301 in all
four patients treated in the Phase 1 study.
Operational Updates
- Published peer-reviewed risk assessment demonstrating low
incidence of busulfan-related secondary malignancies in pediatric
transplant recipients. “Busulfan and Subsequent Malignancy: An
Evidence-based Risk Assessment” was published in Pediatric Blood
& Cancer. Busulfan is utilized as conditioning prior to gene
therapy in nonmalignant hematologic and related disorders.
Researchers conducted a literature-based assessment of busulfan and
subsequent late effects, concluding the incidence of
busulfan-related secondary malignancies has been less than 1% in
pediatric transplant recipients, and that the incidence of
secondary cancers in pediatric patients receiving single-agent
busulfan in non-malignant settings is likely to be substantially
lower than 1%.
- Raised net proceeds of $188.9M through public offering.
On September 15, 2023, the Company completed a public offering of
approximately 9.5 million shares of our common stock at a public
offering price of $16.00 per share and pre-funded warrants to
purchase 3.1 million shares of common stock at a price of $15.99
per warrant. The gross proceeds to Rocket from the public offering
were approximately $201.3 million, net of $12.4 million of offering
costs, commissions, legal and other expenses for net proceeds from
the offering of $188.9 million.
Upcoming Investor Conferences
- Stifel Healthcare Conference 2023, November 15
- 6th Annual Evercore ISI HealthCONx Conference 2023, November
30
Third Quarter Financial Results
- Cash position. Cash, cash equivalents and investments as
of September 30, 2023, were $437.2 million.
- R&D expenses. Research and development expenses were
$46.8 million for the three months ended September 30, 2023,
compared to $43.4 million for the three months ended September 30,
2022. The increase in R&D expenses was primarily driven by
increases in compensation and benefits expenses of $4.5 million due
to increased R&D headcount, clinical trial expenses of $7.4
million, license expenses of $2.2 million, and non-cash stock-based
compensation of $1.6 million. Increases noted were offset by
decreases in manufacturing and development costs of $9.0 million
and cost of direct materials of $3.3 million.
- G&A expenses. General and administrative expenses
were $18.6 million for the three months ended September 30, 2023,
compared to $15.1 million for the three months ended September 30,
2022. The increase in G&A expenses was primarily driven by
increases in commercial preparation expenses which consists of
commercial strategy, medical affairs, market development and
pricing analysis of $2.5 million and non-cash stock compensation
expense of $1.0 million, partially offset by reduction in
acquisition related expenses of $1.3 million.
- Net loss. Net loss was $61.9 million or $0.75 per share
(basic and diluted) for the three months ended September 30, 2023,
compared to $57.8 million or $0.87 per share (basic and diluted)
for the three months ended September 30, 2022.
- Shares outstanding. 90,146,602 shares of common stock
were outstanding as of September 30, 2023.
Financial Guidance
- Cash position. As of September 30, 2023, Rocket had
cash, cash equivalents and investments of $437.2 million. Rocket
expects such resources will be sufficient to fund its operations
through 2025, 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 advancing an
integrated and sustainable pipeline of investigational genetic
therapies designed to correct the root cause of complex and rare
disorders. The Company’s platform-agnostic approach enables it to
design the best therapy for each indication, creating potentially
transformative options for patients afflicted with rare genetic
diseases. Rocket's clinical programs using lentiviral (LV)
vector-based gene therapy are for the treatment of Fanconi Anemia
(FA), 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 rare,
monogenic red blood cell disorder resulting in increased red cell
destruction and mild to life-threatening anemia. Rocket’s first
clinical program using adeno-associated virus (AAV)-based gene
therapy is for Danon Disease, a devastating, pediatric heart
failure condition. Rocket also is developing AAV-based gene therapy
programs in PKP2-arrhythmogenic cardiomyopathy (ACM) and
BAG3-associated dilated cardiomyopathy (DCM). For more information
about Rocket, please visit www.rocketpharma.com.
Rocket Cautionary Statement Regarding Forward-Looking
Statements
Various statements in this release concerning Rocket’s future
expectations, plans and prospects, including without limitation,
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 Danon Disease program, including its planned
pivotal trial, and the safety, effectiveness and timing of related
pre-clinical studies and clinical trials, may constitute
forward-looking statements for the purposes of the safe harbor
provisions under the Private Securities Litigation Reform Act of
1995 and other federal securities laws and are subject to
substantial risks, uncertainties and assumptions. 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. 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
ability to monitor the impact of COVID-19 on its business
operations and take steps to ensure the safety of patients,
families and employees, the interest from patients and families for
participation in each of Rocket’s ongoing trials, our expectations
regarding the delays and impact of COVID-19 on clinical sites,
patient enrollment, trial timelines and data readouts, our
expectations regarding our drug supply for our ongoing and
anticipated trials, actions of regulatory agencies, which may
affect the initiation, timing and progress of pre-clinical studies
and clinical trials of its product candidates, Rocket’s dependence
on third parties for development, manufacture, marketing, sales and
distribution of product candidates, the outcome of litigation, and
unexpected expenditures, 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 September 30, Nine Months Ended
September 30,
2023
2022
2023
2022
Operating expenses: Research and development
$
46,844
$
43,383
$
144,598
$
115,533
General and administrative
18,585
15,105
51,782
39,728
Total operating expenses
65,429
58,488
196,380
155,261
Loss from operations
(65,429
)
(58,488
)
(196,380
)
(155,261
)
Interest expense
(469
)
(465
)
(1,405
)
(1,395
)
Interest and other income, net
1,720
1,353
4,474
2,644
Accretion of discount and amortization of premium on investments,
net
2,279
(156
)
7,376
(1,128
)
Net loss
$
(61,899
)
$
(57,756
)
$
(185,935
)
$
(155,140
)
Net loss per share - basic and diluted
$
(0.75
)
$
(0.87
)
$
(2.30
)
$
(2.37
)
Weighted-average common shares outstanding - basic and diluted
82,636,120
66,215,535
80,865,658
65,406,844
September 30, 2023 December 31, 2022
Cash, cash equivalents, and investments
$
437,171
$
399,670
Total assets
598,762
551,807
Total liabilities
57,676
62,121
Total stockholders' equity
541,086
489,686
View source
version on businesswire.com: https://www.businesswire.com/news/home/20231106135977/en/
Media Kevin Giordano media@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
Rocket Pharmaceuticals (NASDAQ:RCKT)
過去 株価チャート
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Rocket Pharmaceuticals (NASDAQ:RCKT)
過去 株価チャート
から 5 2023 まで 5 2024