Rocket Pharma Stock Slips Despite New Long-Term Data On Phase-I Study; Retail Sentiment Slides

Danon disease is a rare genetic disorder whose complications can ultimately cause heart failure, and for male patients, frequent death.

Shares of biotech company Rocket Pharmaceuticals ($RCKT) were down more than 1% on Monday afternoon after the life sciences company published long-term safety data in the New England Journal of Medicine. Retail sentiment, however, slipped despite the progress.

The Cranbury, N.J.-based late-stage biotechnology company, which focuses on developing gene therapies to treat rare diseases, said it published long-term safety and efficacy results from the Phase 1 of its RP-A501 study that treats Danon disease, showing the drug was generally well tolerated in patients. In a statement, it noted that all six evaluable Phase 1 patients with Danon disease were alive and transplant-free up to age 25 years, and one showed “preliminary evidence” of robust protein expression in heart on five-year biopsy.

Danon disease is a rare genetic disorder and its complications can ultimately cause 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 U.S. and Europe, according to Rocket Pharma.

“As is true for many other recent internal and peer company programs, when gene therapy works, it is life changing. RP-A501 is being developed as a potential one-time gene therapy and the results of the long-term Phase 1 study show the promise of gene therapy across cardiac diseases, including PKP2-ACM, BAG3-DCM and others,” Gaurav Shah, CEO of Rocket Pharma, said in a statement.

Retail Sentiment on the stock inched down to ‘neutral’ (47/100) from ‘extremely bullish’ (84/100) from a day ago, with many users expressing surprise at the little action the stock was seeing. Message volumes, meanwhile, climbed to ‘extremely high’ levels from ‘high.’

In September, Rocket announced the completion of enrollment in the Phase 2 study of RP-A501 to treat Danon Disease.

In its last quarter, the company also said it made progress on the Phase 1 clinical study of RP-A601 for PKP2 arrhythmogenic cardiomyopathy (ACM), and completed patient enrollment in a low dose cohort.

Rocket Pharma shares are down 52% year-to-date.