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Rocket Pharmaceuticals Announces Publication of Comprehensive Review of Danon Disease Cases in the International Journal of Cardiology


Rocket Pharmaceuticals, Inc. (RCKT) (“Rocket”), a leading U.S.-based multi-platform gene therapy company, today announces the online publication of a comprehensive literature review of published Danon disease cases in the International Journal of Cardiology, co-authored by Eric Adler, M.D., Director of Cardiac Transplant and Mechanical Circulatory Support at UC San Diego Health and Professor of Medicine at University of California San Diego School of Medicine. The manuscript entitled, “Danon Disease: Gender Differences in Presentation and Outcomes” outlines a comprehensive review of 83 published manuscripts describing cases of Danon disease, the target indication for RP-A501. RP-A501 is the Company’s adeno-associated viral vector (AAV)-based gene therapy for the treatment of Danon disease that is in development under a collaboration with the University of California San Diego School of Medicine.

The review consisted of 90 male patients and 56 female patients for a total of 146 patients with positive genetic testing for Danon disease or positive muscle biopsy. The results show that despite being an X-linked disease, Danon is as devastating for females as it is males, with 92.5% of overall patients presenting with cardiac abnormalities.

The primary outcome of the analysis was defined as a composite outcome of death, heart transplantation, or long-term left ventricular assist device (LVAD) implantation. The composite outcome occurred in 34.9% of patients, with similar findings seen in males (36.7%) and females (32.1%). 29 patients received a heart transplantation with complications including allograft rejection leading to re-transplant, muscle weakness and respiratory failure that required mechanical ventilatory support. These findings underscore the limitations of transplantation, which is not considered curative and associated with considerable morbidity and mortality.

“Together with Dr. Eric Adler and the UC San Diego team, Rocket is building a robust natural history database to help elucidate the best markers of disease progression in Danon patients and to serve as a supportive reference as part of the registrational package for RP-A501,” said Gaurav Shah, M.D., Chief Executive Officer and President of Rocket. “We’ve now initiated a retrospective patient chart review as well as a prospective natural history study (further information available here). This extensive disease history will inform our clinical development plans for RP-A501 and ultimately, patient care.”

About Danon Disease

Danon disease is caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. It is estimated to have a prevalence of 15,000 to 30,000 patients in the U.S. and the European Union. The disease is often fatal in patients in the second or third decade of life due to progressive heart failure unless treated with a cardiac transplantation, which is associated with numerous complications and is not considered curative. There are no specific therapies available for the treatment of Danon disease.

About Rocket Pharmaceuticals, Inc.

Rocket Pharmaceuticals, Inc. (RCKT) (“Rocket”) is an emerging, clinical-stage biotechnology company focused on developing first-in-class gene therapy treatment options for rare, devastating diseases. Rocket’s multi-platform development approach applies the well-established lentiviral vector (LVV) and adeno-associated viral vector (AAV) gene therapy platforms. Rocket's lead clinical program is a LVV-based gene therapy for the treatment of Fanconi Anemia (FA), a difficult to treat genetic disease that leads to bone marrow failure and potentially cancer. Rocket’s additional pipeline programs for bone marrow-derived disorders are for Pyruvate Kinase Deficiency (PKD), Leukocyte Adhesion Deficiency-I (LAD-I) and Infantile Malignant Osteopetrosis (IMO). Rocket is also developing an AAV-based gene therapy program for a devastating, pediatric heart failure indication, Danon disease. 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, effectiveness and timing of product candidates that Rocket may develop, including in collaboration with academic partners, to treat Fanconi Anemia (FA), Leukocyte Adhesion Deficiency-I (LAD-I), Pyruvate Kinase Deficiency (PKD) and Infantile Malignant Osteopetrosis (IMO), 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 successfully demonstrate the efficacy and safety of such products and pre-clinical studies and clinical trials, its gene therapy programs, the preclinical and clinical results for its product candidates, which may not support further development and marketing approval, Rocket's ability to commence a registrational study in FA within the projected time periods, the potential advantages of Rocket's product candidates, 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 and its licensors ability to obtain, maintain and protect its and their respective intellectual property, the timing, cost or other aspects of a potential commercial launch of Rocket's product candidates, Rocket's ability to manage operating expenses, Rocket's ability to obtain additional funding to support its business activities and establish and maintain strategic business alliances and new business initiatives, 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, 2017. 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.

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