Rocket Pharmaceuticals to Host Webcast on Nov. 15 at 8:30 a.m. ET to Present Updated Danon Disease Clinical Data

Rhea-AI Summary

Rocket Pharmaceuticals (NASDAQ: RCKT) will present updated data from its ongoing Phase 1 clinical trial of RP-A501 for Danon Disease during the American Heart Association Scientific Sessions 2021, held virtually from November 13-15. A conference call will occur on November 15 at 8:30 a.m. ET to discuss the findings. Rocket's gene therapies target rare genetic disorders, including Fanconi Anemia and Leukocyte Adhesion Deficiency-I. The company emphasizes its integrated approach to developing innovative treatments for pediatric conditions.

Positive

• Presentation of updated data from the Phase 1 clinical trial of RP-A501 highlights progress in treating Danon Disease.
• The ongoing commitment to developing therapies for rare childhood genetic disorders supports long-term growth.

Negative

• The possibility of delays in clinical trial timelines and data readouts due to COVID-19 uncertainties.

—Company to provide comprehensive update on the ongoing Phase 1 clinical trial of RP-A501, including data to be presented at the American Heart Association (AHA) Scientific Sessions 2021—

CRANBURY, N.J.--(BUSINESS WIRE)-- Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, will host a conference call and live webcast on Monday, Nov. 15, 2021, at 8:30 a.m. ET. Rocket will present updated data from the ongoing Phase 1 clinical trial of RP-A501 in Danon Disease, which will include data being presented in an ePoster at the American Heart Association (AHA) Scientific Sessions 2021 taking place virtually Nov. 13-15, 2021.

Investors may access the conference call by dialing (866) 939-3921 from locations in the United States or +1 (678) 302-3550 from outside the United States. Please refer to conference ID number 50255994. A live webcast of the call can be accessed 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 following the completion of the call.

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, Pyruvate Kinase Deficiency (PKD), a rare, monogenic red blood cell disorder resulting in increased red cell destruction and mild to life-threatening anemia, and Infantile Malignant Osteopetrosis (IMO), a bone marrow-derived disorder. 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 2021 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), Infantile Malignant Osteopetrosis (IMO) and Danon Disease, the expected timing and data readouts of Rocket’s ongoing and planned clinical trials, Rocket’s plans for the advancement of its Danon Disease program following the lifting of the FDA’s clinical hold 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, 2020, filed March 1, 2021 with the SEC. 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/20211111005457/en/

Media

Kevin Giordano

Director, Corporate Communications

kgiordano@rocketpharma.com

Investors

Mayur Kasetty, M.D., M.B.A.

Director, Business Development & Operations

investors@rocketpharma.com

Source: Rocket Pharmaceuticals, Inc.

FAQ

What is Rocket Pharmaceuticals presenting at the AHA Scientific Sessions 2021?

Rocket Pharmaceuticals will present updated data from its Phase 1 clinical trial of RP-A501 for Danon Disease.

When is the conference call for Rocket Pharmaceuticals about the RP-A501 trial?

The conference call is scheduled for November 15, 2021, at 8:30 a.m. ET.

What is the focus of the RP-A501 clinical trial?

The RP-A501 clinical trial focuses on treating Danon Disease, a severe pediatric heart failure condition.

What challenges might Rocket Pharmaceuticals face in its clinical trials?

Rocket Pharmaceuticals may face delays in trial timelines and data readouts due to COVID-19 uncertainties and regulatory hurdles.