Continued to advance leading pipeline of AAV
cardiology and LV hematology assets; all 2023 milestones remain on
track
Updates on all four clinical programs and
PKP2-ACM preclinical program to be presented at the 26th Annual
Meeting of the American Society of Gene and Cell Therapy
(ASGCT)
Mark White, MB.ChB, named Chief Medical Officer
following more than 25 years at AstraZeneca; Jonathan Schwartz,
M.D., appointed Chief Gene Therapy Officer
Became founding consortium member of Rady
Children’s Institute for Genetic Medicine’s BeginNGSTM newborn
screening program to increase speed of diagnosis for genetic
diseases
Cash, cash equivalents and investments of
approximately $360M; 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 March 31, 2023, and updates from the Company’s key pipeline
developments, business operations and upcoming milestones.
“I am very pleased with our first quarter progress, as we
continued to advance our industry leading pipeline of now six
programs across both AAV and LV platforms addressing life
threatening cardiac and hematologic rare diseases, including two LV
programs nearing fillings and four programs either demonstrating
robust clinical results or strong preclinical proof of concept,
“said Gaurav Shah, M.D., Chief Executive Officer, Rocket Pharma.
“Corporate and program milestones remain on track, including the
planned initiation of the Phase 2 pivotal trial in Danon Disease
this second quarter and regulatory filings for LAD-I and Fanconi
Anemia this second quarter and fourth quarter, respectively.”
Dr. Shah continued, “In addition, we look forward to presenting
data across all four of our clinical programs at the upcoming ASGCT
meeting this month, as well as our recently announced preclinical
program for PKP2-ACM, one of the most prevalent and devastating
forms of heart disease impacting nearly 50,000 adults and children
in the U.S. and EU. Further, I am proud to announce that we became
a Founding Member of BeginNGSTM, a national program that advocates
for and facilitates newborn screening for approximately 400 genetic
diseases. As we diligently work towards cures for rare diseases,
encouraging early genetic screening is essential in providing rare
disease patients and their families with an accurate and early
diagnosis so they can identify and activate a disease management
plan with their healthcare providers as soon as possible.”
“Lastly, following a recent ATM sale of $17.2M in net proceeds
and in-house manufacturing efficiencies, I am pleased to announce
that we have extended our cash runway into the first half of 2025.
We look forward to leveraging this strong cash position, which will
take us through planned launches in 2024," said Dr. Shah. “Taken
together, I am incredibly proud of the tremendous progress we have
made in expeditiously developing one of the broadest and deepest
pipeline of assets in gene therapy and building integrated
capabilities spanning discovery through manufacturing and
commercial to help address the unmet needs of patients facing these
rare and devastating diseases. We look forward to continuing this
progress into the next quarter and year.”
Key Pipeline and Operational Updates
- Danon, FA, LAD-I, PKD trials and plans for PKP2-ACM and
BAG3-DCM preclinical programs remain on track. All 2023
milestones remain on track including anticipated initiation of the
Phase 2 pivotal trial for Danon Disease during this second quarter
and Biologics License Application (BLA) filing for LAD-I in Q2 2023
and BLA filing for FA in Q4 2023.
- Updated data across all clinical trials and PKP2-ACM
preclinical program to be presented at ASGCT. Updates
anticipated at the 26th Annual Meeting of the American Society of
Gene and Cell Therapy (ASGCT) at the Los Angeles Convention Center,
May 16-20.
Details for oral presentations are as
follows:
Title: Danon Disease Phase 1
RP-A501 Results: The First Single-Dose Intravenous (IV) Gene
Therapy with Recombinant Adeno-Associated Virus (AAV9:LAMP2B) for a
Monogenic Cardiomyopathy Session: Clinical Trials Spotlight
Symposium Presenter: Joseph Rossano, M.D., M.S., FAAP, FACC,
Co-Director of the Cardiac Center and Chief of the Division of
Cardiology at Children's Hospital of Philadelphia Session date
and time: Thursday, May 18, 2023, 8:00 a.m. – 9:45 a.m. PT
Location: Concourse Hall 152 & 153 Presentation
number: 9
Title: Lentiviral-Mediated Gene
Therapy for Fanconi Anemia [Group A]: Results From Global RP-L102
Clinical Trials Session: Hematologic and Immunologic
Diseases Presenter: Agnieszka Czechowicz, M.D., Ph.D.,
Department of Pediatrics, Division of Hematology/ Oncology, Stem
Cell Transplantation and Regenerative Medicine, Stanford University
School of Medicine Session date and time: Thursday, May 18,
2023, 3:45 p.m. – 5:30 p.m. PT Location: Room 501 ABC
Presentation number: 217
Title: Global Phase 1 Study Results of
Lentiviral Mediated Gene Therapy for Severe Pyruvate Kinase
Deficiency (PKD) Session: Hematologic and Immunologic
Diseases Presenter: Ami J. Shah, M.D., Clinical Professor of
Pediatrics, Division of Hematology/ Oncology, Stem Cell
Transplantation and Regenerative Medicine, Stanford University
School of Medicine Session date and time: Thursday, May 18,
2023, 3:45 p.m. – 5:30 p.m. PT Location: Room 501 ABC
Presentation number: 218
Title: Preclinical Efficacy of
AAVrh.74-PKP2a (RP-A601): Gene Therapy for PKP2-associated
Arrhythmogenic Cardiomyopathy Session: Late-Breaking
Abstracts 1 Presenter: Christopher Herzog, Ph.D., Associate
Vice President, AAV R&D, Rocket Pharma Session date and
time: Friday, May 19, 2023, 8:00 a.m. – 9:45 a.m. PT
Location: Room 515AB Presentation number: 2
Details for the poster presentation are as
follows:
Title: Autologous Ex-Vivo
Lentiviral Gene Therapy for Pediatric Patients with Severe
Leukocyte Adhesion Deficiency-I (LAD-I): Interim Results from an
Ongoing Phase 1/2 Study Session: Friday Poster Session
Presenter: Donald B. Kohn, M.D., Distinguished Professor of
Microbiology, Immunology & Molecular Genetics (MIMG),
Pediatrics and Molecular & Medical Pharmacology; Director of
the UCLA Human Gene and Cell Therapy Program, University of
California, Los Angeles Session date and time: Friday, May
19, 2023, 12:00 p.m. PT Location: Exhibit Hall/West Hall A
Presentation number: 1547
- Expanded leadership team to support evolution towards
Commercial stage. Jonathan Schwartz, M.D., Rocket’s founding
Chief Medical Officer, was appointed Chief Gene Therapy Officer to
enhance the Company’s focus on the strategic application of gene
therapy technologies to current and future therapeutic areas. Dr.
Schwartz oversees research, deepens relationships with external
collaborators, and offers a pointed focus on clinical strategy and
pipeline expansion. In addition, Mark White, MB.ChB, was named
Chief Medical Officer following more than 25 years at AstraZeneca
and brings expertise in clinical development, global regulatory
submissions, and commercial and business strategy. At AstraZeneca,
Dr. White most recently served as Global Franchise Head,
Respiratory and Inflammation and has been the program lead for
multiple innovative medicines guiding them through late-stage
development, approvals and launches around the globe.
- Became founding consortium member of a diagnostic and
precision medicine guidance tool to accelerate early diagnosis of
rare genetic diseases. Rocket entered into a collaboration with
Rady Children’s Institute for Genetic Medicine to advance
BeginNGSTM, a national program that advocates for and facilitates
newborn screening for approximately 400 genetic diseases using
rapid Whole Genome Sequencing (rWGS®). As part of the
collaboration, Rocket joins the BeginNGSTM consortium, which serves
as a think tank across public and private institutions and
participates in the Frontiers in Pediatric Genomic Medicine
Conference. As a founding consortium member, Rocket will play a
leading role advocating for early diagnosis of patients with
genetic rare diseases.
- Celebrated annual Rare Disease Day with multi-faceted
awareness campaign. On February 28, 2023, Rocket hosted its
annual Rare Disease Day recognition program highlighting the theme,
“Stories That Need To Be Shared: The Human Side of Rare Disease.”
More than 300 members of the global rare disease community gathered
in person and virtually at the Make-A-Wish New Jersey Samuel &
Josephine Plumeri Wishing Place to hear inspirational stories from
patients living with rare diseases, caregivers and patient
advocates. The Company also continued to build upon its Light Up
for Rare initiative in collaboration with global partners to light
up buildings and landmarks in more than 100 countries across the
globe in the Rare Disease Day colors, including the Empire State
Building and Niagara Falls. Rocket remains committed to supporting
the rare disease community through patient-focused events,
education and advancing science to bring potential treatments to
patients with unmet needs.
First Quarter Financial Results
- Cash position. Cash, cash equivalents and investments as
of March 31, 2023, were $360.0 million.
- R&D expenses. Research and development expenses were
$46.4 million for the three months ended March 31, 2023, compared
to $30.8 million for the three months ended March 31, 2022. The
increase in R&D expenses was primarily driven by increases in
compensation and benefits expense of $6.6 million due to increased
R&D headcount, manufacturing and development costs of $2.8
million, direct materials of $0.9 million, and laboratory supplies
of $0.9 million.
- G&A expenses. General and administrative expenses
were $15.8 million for the three months ended March 31, 2023,
compared to $11.8 million for the three months ended March 31,
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 $1.1 million, compensation and benefits of $0.7
million due to increased G&A headcount and non-cash stock
compensation expense of $1.1 million.
- Net loss. Net loss was $58.3 million or $0.73 per share
(basic and diluted) for the three months ended March 31, 2023,
compared to $43.0 million or $0.67 (basic and diluted) for the
three months ended March 31, 2022.
- Shares outstanding. 80,412,194 shares of common stock
were outstanding as of March 31, 2023.
Financial Guidance
- Cash position. As of March 31, 2023, Rocket had cash,
cash equivalents and investments of $360.0 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 our 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 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 preclinical 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 “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 March 31,
2023
2022
Operating expenses: Research and development
$
46,371
$
30,794
General and administrative
15,823
11,770
Total operating expenses
62,194
42,564
Loss from operations
(62,194
)
(42,564
)
Interest expense
(468
)
(464
)
Interest and other income, net
1,908
623
Accretion of discount and amortization of premium on investments,
net
2,419
(577
)
Net loss
$
(58,335
)
$
(42,982
)
Net loss per share - basic and diluted
$
(0.73
)
$
(0.67
)
Weighted-average common shares outstanding - basic and diluted
79,453,519
64,509,721
March 31, December 31,
2023
2022
Cash, cash equivalents, and investments
$
360,041
$
399,670
Total assets
514,757
551,807
Total liabilities
55,881
62,121
Total stockholders' equity
458,876
489,686
View source
version on businesswire.com: https://www.businesswire.com/news/home/20230504005960/en/
Media Kevin Giordano kgiordano@rocketpharma.com
Investors Brooks Rahmer investors@rocketpharma.com
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