CRANBURY, N.J.– Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a clinical-stage company advancing an integrated and sustainable pipeline of genetic therapies for rare childhood disorders, today announces an oral presentation of previously disclosed data from the ongoing Phase 1 trial of RP-A501 for the treatment of Danon Disease was given at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2021. The presentation overviewed data from the low-dose (6.7e13 vg/kg) cohort which demonstrated the investigational gene therapy RP-A501 was well tolerated and showed progressive and durable clinical benefit.
“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 Jonathan Schwartz, M.D., chief medical officer of Rocket Pharma. “We continue to be encouraged by the safety and durable clinical benefit RP-A501 has demonstrated in the low dose, with one patient now followed to 24 months. Additionally, we are working expeditiously to commence treatment in the low-dose (6.7e13 vg/kg) pediatric patient cohort in the third quarter and expect to provide a comprehensive clinical update from the Phase 1 trial in the fourth quarter.”
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.
