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

Rhea-AI Summary

Rocket Pharmaceuticals is set to present updated data from its Phase 1 Danon Disease trial on September 30, 2022, at the Heart Failure Society of America Annual Scientific Meeting. The presentation will reveal efficacy and safety updates from both pediatric and adult cohorts. An investor event discussing this data will stream at 8:00 a.m. ET. The trial aims to address the serious pediatric heart condition, Danon Disease, utilizing gene therapy.

Positive

• Upcoming presentation on Phase 1 Danon Disease trial showcasing updated efficacy and safety data.
• Positive initial safety data reported for two pediatric patients in the trial.

Negative

• None.

— 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 —

CRANBURY, N.J.--(BUSINESS WIRE)-- 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

Source: Rocket Pharmaceuticals, Inc.

FAQ

What are the expected updates from Rocket Pharmaceuticals' Phase 1 Danon Disease trial?

Rocket Pharmaceuticals will present updated efficacy and safety data from pediatric and adult cohorts during the Heart Failure Society of America Annual Scientific Meeting.

When will Rocket Pharmaceuticals host its investor webcast?

The investor event will be webcast on September 30, 2022, at 8:00 a.m. ET.

What is the significance of the Danon Disease trial for Rocket Pharmaceuticals?

The trial aims to develop a gene therapy for Danon Disease, a serious condition causing pediatric heart failure.

Where will the presentation of Danon Disease trial data take place?

The presentation will occur at the Heart Failure Society of America Annual Scientific Meeting at Gaylord National Harbor Convention Center, Washington, D.C.

What details are provided about the trial's safety data?

Initial safety data from two pediatric patients in the Phase 1 trial has shown positive results, indicating potential for further development.