Rocket Pharmaceuticals to Present Updated Data from Phase 1 Danon Disease Trial for RP-A501 at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2022
September 16 2022 - 8:00AM
Business Wire
— Updated Data from Phase 1 Danon Disease trial
expected to include efficacy and safety data from pediatric cohort
and longer-term data from adult cohort —
— Company to webcast investor event on Sept.
30, at 8:00 a.m. ET —
Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading
late-stage, clinical biotechnology company advancing an integrated
and sustainable pipeline of genetic therapies for rare childhood
disorders with high unmet need, today announces that the Company
will present updated clinical data on its Phase 1 Danon Disease
Trial for RP-A501 during an oral poster session at the Heart
Failure Society of America (HFSA) Annual Scientific Meeting 2022 to
be held Sept. 30 – Oct. 3, 2022. On the first day of the meeting at
6:00 a.m., the oral poster presentation will be made available on
the congress online portal and the Company will issue a press
release. An investor event will subsequently be webcast at 8:00
a.m. Rocket recently announced positive initial safety data from
two pediatric patients in its Phase 1 Danon Disease trial at the
25th Annual Meeting of the American Society of Gene and Cell
Therapy (ASGCT).
HFSA Presentation Details
Title: Safety Profile of the First Pediatric
Cardiomyopathy Gene Therapy Trial: RP-A501 (AAV9:LAMP2B) for Danon
Disease Session: Inherited Cardiomyopathies, Amyloidosis,
Myocarditis, CardioOncology 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: Saturday, Oct. 1, 12:00 – 1:00 p.m.
ET Location: Gaylord National Harbor Convention Center,
Washington, D.C. Poster Number: 375
Investor Webcast Information
Company management will discuss the Danon Disease data via
webcast on Sept. 30, 2022, at 8:00 a.m. ET. To access the webcast,
please register online at:
https://ir.rocketpharma.com/events-presentations. Participants are
requested to register a minimum of 15 minutes before the start of
the call.
A simultaneous webcast of the presentation will be available
under “Events” in the Investors section of the Company’s website
at: https://ir.rocketpharma.com/. The webcast replay will be
available on the Rocket website upon completion of the event.
About Rocket Pharmaceuticals, Inc. Rocket
Pharmaceuticals, Inc. (NASDAQ: RCKT) is advancing an integrated and
sustainable pipeline of genetic therapies that 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 (LVV)-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. 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 its guidance
for 2022 in light of COVID-19, 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), and Danon Disease, 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 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, 2021, filed
February 28, 2022 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220916005090/en/
Media Kevin Giordano Director, Corporate Communications
kgiordano@rocketpharma.com
Investors Jessie Yeung, M.B.A. Vice President, Investor
Relations and Corporate Finance investors@rocketpharma.com
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