Rocket Pharmaceuticals, Inc. RCKT announced that it has completed patient enrollment in a pivotal phase II study evaluating its investigational gene therapy candidate, RP-A501, for treating male patients with Danon disease.
Danon disease is a rare X-linked inherited disorder caused by mutations in the LAMP2 gene.
Per the company, an estimated 15,000 to 30,000 patients suffer from Danon disease in the United States and Europe. Currently, the only available treatment for the disease is cardiac transplantation, which is not considered curative and may lead to complications.
Shares of the company were up 13.5% on Sept. 17 following the announcement of the news.
Year to date, shares of Rocket have plunged 27.2% against the industry’s increase of 1%.
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More on RCKT’s RP-A501 Danon Disease Study
The global multi-center phase II study is evaluating the safety and efficacy of RP-A501 in male patients with Danon disease.
The co-primary endpoint of the study is to check the improvements in LAMP2 protein expression and reductions in left ventricular mass as measured by the biomarker while assessing the efficacy of RP-A501. This will be used to support the accelerated approval for RP-A501.
The key secondary endpoint of the study is to observe the change in troponin. Other secondary endpoints include natriuretic peptides event-free survival to 24 months and treatment-emergent safety events.
Per management, RP-A501 is the first gene therapy product to demonstrate safety and efficacy in clinical studies for a cardiovascular condition.
The FDA and European Medical Agency previously granted regenerative medicine advanced therapy designation and PRIority MEdicines (PRIME) scheme designation to RP-A501, respectively.
RCKT’s Other Pipeline Development Activities
The company is developing its gene therapy candidate, Kresladi (marnetegragene autotemcel), to treat patients with severe leukocyte adhesion deficiency-I (LAD-I), a rare genetic disorder.
In June 2024, the FDA issued a complete response letter (CRL) to Rocket’s biologics license application (BLA) seeking approval for Kresladi to treat LAD-I.
Per the CRL, the FDA sought "limited" additional information on the Chemistry Manufacturing and Controls (CMC), which is part of the BLA submission. The goal of CMC is to ensure that every batch of the final product is identical in terms of its safety, purity, potency and performance.
Rocket is also evaluating two other gene therapies, namely RP-L102 and RP-L301, for the treatment of Fanconi anemia and pyruvate kinase deficiency, respectively. Both candidates are in mid-stage development.
Zacks Rank & Stocks to Consider
Rocket currently carries a Zacks Rank #3 (Hold).
Some top-ranked stocks in the biotech sector are Illumina, Inc. ILMN, Krystal Biotech, Inc. KRYS and Fulcrum Therapeutics, Inc. FULC, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for Illumina’s 2024 earnings per share have moved up from $1.84 to $3.62. Earnings per share estimates for 2025 have improved from $3.22 to $4.43. Year to date, shares of ILMN have lost 6.5%.
ILMN’s earnings beat estimates in each of the trailing four quarters, the average surprise being 463.46%.
In the past 60 days, estimates for Krystal Biotech’s 2024 earnings per share have increased from $2.09 to $2.38. Earnings per share estimates for 2025 have improved from $4.33 to $7.31. Year to date, shares of KRYS have risen 52.6%.
KRYS’ earnings beat estimates in three of the trailing four quarters while missing on the remaining occasion, the average surprise being 45.95%.
In the past 60 days, estimates for Fulcrum Therapeutics’ 2024 loss per share have narrowed from $1.24 to 33 cents. Loss per share estimates for 2025 have narrowed from $1.71 to $1.14. Year to date, shares of FULC have plunged 48.3%.
FULC’s earnings beat estimates in each of the trailing four quarters, the average surprise being 393.18%.
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