Initiating enrollment of two-patient pediatric
safety run-in for pivotal single-arm Phase 2 study of RP-A501
(Danon Disease); finalizing primary composite endpoint with FDA to
support accelerated approval
RP-L201 (LAD-I) BLA submitted to FDA;
represents Rocket’s first product filing
RP-A601 (PKP2-ACM) advancing towards first
patient treatment in Phase 1 study following FDA clearance of IND;
received FDA Fast Track and Orphan Drug designations
RP-L102 (Fanconi Anemia) U.S. pivotal Phase 2
study completed; product filing on track
RP-L301 (PKD) granted EMA PRIME and FDA RMAT
designations
Cash, cash equivalents and investments of
approximately $307M; expected operational runway into the first
half of 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 June 30, 2023, and updates from the Company’s key pipeline
developments, business operations and upcoming milestones.
“The second quarter of 2023 marked a period of strong forward
momentum across our cardiovascular AAV and hematology LV portfolios
highlighted by the BLA submission for our LAD-I program, Rocket’s
first product filing. In addition, we are in advanced discussions
with the FDA to finalize the components of the primary composite
endpoint for the single-arm, pivotal Phase 2 Danon Disease study.
In parallel, we are initiating enrollment in the trial following
recent alignment with the FDA on our proposed two-patient pediatric
safety run-in,” said Gaurav Shah, M.D., Chief Executive Officer,
Rocket Pharma. “We appreciate the strong and positive collaboration
with the FDA afforded by our RMAT designation, which we believe
will lead to the most optimal global development path forward for
the first pivotal gene therapy trial for a cardiac condition.
Simultaneously, in our second cardiac program, RP-A601 for the
treatment of PKP2-ACM, we are rapidly moving towards first patient
treatment following IND clearance and have also received FDA Fast
Track and Orphan Drug designations.”
Dr. Shah continued, “Building on the BLA submission for LAD-I,
we remain on track to submit the BLA for our LV-based Fanconi
Anemia program in the fourth quarter. Taken together, I am very
pleased with our progress across our entire pipeline of AAV and LV
assets and look forward to continuing our momentum through the
remainder of the year and beyond.”
Key Pipeline and Operational Updates
AAV Cardiovascular Portfolio
Danon Disease
- Received European designations including Priority Medicines
(PRIME) and Advanced Therapy Medicinal Products (ATMP) for
RP-A501. PRIME designation offers the benefits of early and
enhanced support from the European Medicines Agency (EMA) and the
opportunity for accelerated marketing application review. It is
granted to medicines that target an unmet need for which no
treatment option exists and with data showing a meaningful
improvement of clinical outcomes. ATMP designation is intended for
medicines that offer groundbreaking new opportunities and allows
for a single evaluation and authorization procedure.
- Positive clinical data from the Phase 1 trial of RP-A501
presented at the ASGCT Annual Meeting. As of May 2023, all six
optimally enrolled patients in the Danon Disease Phase 1 trial
continued to demonstrate improvement or stabilization in ongoing
follow-up of six to 36 months. Patients would have typically
experienced progressive disease or death without treatment.
- Initiating two-patient pediatric safety run-in for RP-A501
pivotal study; approaching final alignment with FDA on primary
composite endpoint to support accelerated approval. Rocket has
obtained IRB approvals and begun screening activities. Initial
clinical sites are the University of California, San Diego (UCSD)
and Children’s Hospital of Philadelphia.
PKP2 Arrhythmogenic Cardiomyopathy
(PKP2-ACM)
- Received FDA Fast Track and Orphan Drug designations for
RP-A601. Fast Track designation facilitates the development and
expedited review of medicines that treat serious conditions and
fill an unmet medical need. It also enables increased communication
with the FDA, offering the potential for accelerated approval and
priority review if criteria are met, and permits a rolling
Biologics License Application (BLA) review. Orphan Drug designation
is granted to support the development of medicines for rare
disorders and provides certain benefits, including market
exclusivity upon regulatory approval, exemption of FDA application
fees, and tax credits for qualified clinical trials.
- Positive proof of concept from preclinical studies of
RP-A601 presented at the ASGCT Annual Meeting. Data
demonstrated decreased arrhythmias and increased survival in the
PKP2 knockout mouse model. Based on these data and the completion
of Investigational New Drug (IND)-enabling toxicology studies,
Rocket received IND clearance from the FDA for a Phase 1 study of
RP-A601 that 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.
- Phase 1 start-up activities for RP-A601 are well progressed
following IND clearance to allow for first patient treatment.
Rocket has gained IRB approval at UCSD, initiated Phase 1 study
start up activities and is rapidly advancing toward first patient
treatment.
LV Hematology Portfolio
Leukocyte Adhesion Deficiency-I
(LAD-I)
- Positive clinical data from pivotal Phase 2 trial of RP-L201
presented at the ASGCT Annual Meeting. Positive, updated
top-line data demonstrated 100% overall survival at 12 months
post-infusion (and for entire duration of follow-up) via Kaplan
Meier estimate for all nine LAD-I patients with 12 to 24 months of
available follow-up. Data also showed evidence of resolution of
LAD-I-related skin rash and restoration of wound repair
capabilities. The safety profile was highly favorable in all
patients with no RP-L201-related serious adverse events.
- BLA submitted to FDA for RP-L201. Based on the positive
efficacy and safety data from the pivotal study of RP-L201, Rocket
submitted the BLA to the FDA, as its first product filing.
Fanconi Anemia (FA)
- Positive clinical data from pivotal Phase 2 trial of RP-L102
presented at the ASGCT Annual Meeting. Positive, updated
top-line data showed sustained genetic correction in eight of 12
evaluable patients and comprehensive phenotypic correction in seven
of 12 evaluable patients with ≥12 months of follow up as
demonstrated by increased resistance to mitomycin-C (MMC) in bone
marrow (BM)-derived colony forming cells and hematologic
stabilization. The safety profile was highly favorable with no
significant safety signals, and the treatment, administered without
any cytotoxic conditioning, was well tolerated.
- BLA submission for RP-L102 anticipated in Q4 2023. Based
on the positive efficacy and safety data from the pivotal study of
RP-L102, Rocket is on track to submit the BLA during the fourth
quarter of 2023 and anticipates providing an update following FDA
acceptance.
Pyruvate Kinase Deficiency
(PKD)
- Presented positive clinical data from Phase 1 trial of
RP-L301 at the ASGCT Annual Meeting. Data showed robust and
sustained efficacy in both adult patients for up to 30 months.
Results from the first pediatric patient indicate preliminary
efficacy and favorable safety.
- Received EMA Priority Medicines (PRIME) designation for
RP-L301 in July. PRIME designation offers the benefits of early
and enhanced support from the European Medicines Agency (EMA) and
the opportunity for accelerated marketing application review. It is
granted to medicines that target an unmet need for which no
treatment option exists and with data showing a meaningful
improvement of clinical outcomes.
- Received FDA Regenerative Medicine Advanced Therapy (RMAT)
designation for RP-L301. RMAT designation was granted based on
robust safety and efficacy data from the ongoing Phase 1 study and
its potential to cure a life-threatening disease for which no
curative therapies currently exist. The designation will provide
the benefits of added FDA guidance and expedited review through the
program’s development. All four Rocket-sponsored programs with
clinical data have received RMAT designation.
- Adult and pediatric enrollment are completed in the Phase 1
study. Phase 2 pivotal trial initiation activities are
anticipated in the fourth quarter of 2023.
Operational Update
- Entered into data licensing agreement with Invitae to
support timely access to genetic testing for patients who may have
genetic forms of cardiomyopathy. The Invitae Unlock™
Cardiomyopathy and Arrhythmia genetic testing program evaluates
causal mutations in approximately 170 genes, including LAMP2, PKP2
and BAG3, that result in Danon Disease, PKP2-ACM and BAG3-DCM,
respectively. As part of the agreement, Rocket is raising awareness
of this testing program and educating healthcare professionals
across the U.S. on the benefits of screening for genetic forms of
cardiomyopathy.
Second Quarter Financial Results
- Cash position. Cash, cash equivalents and investments as
of June 30, 2023, were $307.0 million.
- R&D expenses. Research and development expenses were
$51.4 million for the three months ended June 30, 2023, compared to
$41.4 million for the three months ended June 30, 2022. The
increase in R&D expenses was primarily driven by increases in
compensation and benefits expense of $5.7 million due to increased
R&D headcount, clinical trial expenses of $2.7 million, and
non-cash stock-based compensation of $1.7 million.
- G&A expenses. General and administrative expenses
were $17.4 million for the three months ended June 30, 2023,
compared to $12.9 million for the three months ended June 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 $1.3 million, compensation and benefits of $0.8 million due to
increased G&A headcount and non-cash stock compensation expense
of $1.1 million.
- Net loss. Net loss was $65.7 million or $0.82 per share
(basic and diluted) for the three months ended June 30, 2023,
compared to $54.4 million or $0.83 (basic and diluted) for the
three months ended June 30, 2022.
- Shares outstanding. 80,521,415 shares of common stock
were outstanding as of June 30, 2023.
Financial Guidance
- Cash position. As of June 30, 2023, Rocket had cash,
cash equivalents and investments of $307.0 million. Rocket expects
such resources will be sufficient to fund its operations into the
first half of 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
childhood 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
vector (LV)-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 has received IND clearance for the AAV-based
gene therapy program for PKP2-arrhythmogenic cardiomyopathy (ACM),
a heart failure condition associated with life-threatening
arrhythmias, cardiac structural abnormalities, and sudden cardiac
death, and is advancing a preclinical program for 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), PKP2-ACM 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 “aim,” “anticipate,” "believe," “can,”
“continue,” “design,” “estimate,” "expect," "intend," “may,”
"plan," “potential,” "will give," "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, 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, our ability to submit regulatory filings
with the U.S. Food and Drug Administration (FDA) and to obtain and
maintain FDA or other regulatory authority approval of our product
candidates, Rocket’s dependence on third parties for development,
manufacture, marketing, sales and distribution of product
candidates, the outcome of litigation, our competitors’ activities,
including decisions as to the timing of competing product launches,
pricing and discounting, our integration of an acquired business,
which involves a number of risks, including the possibility that
the integration process could result in the loss of key employees,
the disruption of our ongoing business, or inconsistencies in
standards, controls, procedures, or policies, our ability to
successfully develop and commercialize any technology that we may
in-license or products we may acquire and any 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 June 30, Six Months Ended June 30,
2023
2022
2023
2022
Operating expenses: Research and development
$
51,383
$
41,356
$
97,754
$
72,150
General and administrative
17,374
12,854
33,197
24,624
Total operating expenses
68,757
54,210
130,951
96,774
Loss from operations
(68,757
)
(54,210
)
(130,951
)
(96,774
)
Interest expense
(468
)
(465
)
(936
)
(928
)
Interest and other income, net
846
669
2,754
1,291
Accretion of discount and amortization of premium on investments,
net
2,678
(396
)
5,097
(973
)
Net loss
$
(65,701
)
$
(54,402
)
$
(124,036
)
$
(97,384
)
Net loss per share - basic and diluted
$
(0.82
)
$
(0.83
)
$
(1.55
)
$
(1.50
)
Weighted-average common shares outstanding - basic and diluted
80,472,362
65,476,531
79,965,755
64,995,797
June 30, December 31,
2023
2022
Cash, cash equivalents, and investments
$
307,040
$
399,670
Total assets
461,781
551,807
Total liabilities
58,811
62,121
Total stockholders' equity
402,970
489,686
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230809684696/en/
Media Kevin Giordano media@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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