Rocket Pharmaceuticals Presents Clinical Data from RP-A501 Trial in Danon Disease at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021
— Previously disclosed positive data from the low-dose (6.7e13 vg/kg) cohort of the ongoing Phase 1 trial presented at HFSA’s Annual Scientific Meeting 2021 —
— Data demonstrated RP-A501 was well tolerated and showed increasing and durable benefit —
— Low-dose pediatric patient cohort expected to commence in Q3; full update anticipated Q4 —
“Patients with Danon Disease desperately need a treatment option that provides meaningful therapeutic benefit with a manageable safety profile, and we are excited to be a part of the HFSA Annual Meeting and to share this important work,” said
Data from the Low-Dose Adult Cohort (n=3) of the Phase 1 Trial of RP-A501 in Danon Disease
- RP-A501 was generally well tolerated with a manageable safety profile. All observed adverse effects were transient and reversible with no lasting clinical sequelae. Steroid-induced myopathy was observed in two of the three patients >2 weeks after dosing, which also resolved.
- All three low-dose patients demonstrated evidence of sustained cardiac LAMP2B expression by Western blot and/or immunohistochemistry with decreased vacuoles and improved tissue architecture on electron microscopy.
- All three patients demonstrated improvements in the 6 minute walk test (6MWT). One patient improved from a pretreatment baseline of 443 meters (m) to 467 m at 24 months. The second patient improved from a pretreatment baseline of 405 m to 410 m at 18 months. The third patient improved from a pretreatment baseline of 427 m to 435 m at 15 months.
- An improvement in NYHA class in two of the three low-dose patients was observed. In these two patients, a substantial improvement of a key marker of heart failure, B-type natriuretic peptide (BNP), was also observed. BNP decreased from a pretreatment baseline by 75 percent in one patient and 79 percent in the other. The third patient has demonstrated stabilization of NYHA class and BNP.
RP-A501 is an investigational gene therapy product being developed for Danon Disease and the first potential gene therapy for monogenic heart failure. It consists of a recombinant adeno-associated serotype 9 (AAV9) capsid containing a functional version of the human LAMP2B transgene (AAV9.LAMP2B). RP-A501 is currently being evaluated in an ongoing Phase 1 clinical trial, from which preliminary data of the low-dose cohort showed it was generally well tolerated and provided evidence of improved cardiac function in patients.
About Danon Disease
Danon Disease is a rare X-linked inherited disorder caused by mutations in the gene encoding lysosome-associated membrane protein 2 (LAMP-2), an important mediator of autophagy. This results in accumulation of autophagosomes and glycogen, particularly in cardiac muscle and other tissues, which ultimately leads to heart failure, and for male patients, frequent death during adolescence or early adulthood. It is estimated to have a prevalence of 15,000 to 30,000 patients in the
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
Director, Corporate Communications