Morgan Stanley (NYSE:MS) has reaffirmed its confidence in Rocket Pharmaceuticals (NASDAQ: NASDAQ:RCKT), maintaining an Overweight rating and a $45.00 price target on the stock.
The firm's stance comes after the biotechnology company completed Phase 2 enrollment for its leading drug candidate, RP-A501, which is being developed to treat Danon disease.
The completion of the enrollment marks a significant milestone for Rocket Pharmaceuticals, setting a clear timeline for the study's outcome.
Morgan Stanley anticipates the pivotal study results by the end of 2025, based on the 12-month endpoint of the trial. The final 10 patients were enrolled in a span of three months following a pediatric safety run-in. This rapid enrollment, despite strict criteria, underscores the high demand and unmet need for treatment in Danon disease.
Rocket Pharmaceuticals' Phase 2 pivotal study is a global, single-arm trial that involves administering RP-A501 at a dose of 6.7x10^13GC/kg to 12 patients with Danon disease. A concurrent global natural history study serves as a comparator.
For the possibility of accelerated approval, the trial has co-primary endpoints: an improvement in LAMP2 protein expression (at least Grade 1 IHC (LON:IHC)) and a reduction in left ventricular mass (at least a 10% decrease).
Morgan Stanley's report indicates optimism regarding the drug's development progress. The firm's analyst highlighted the potential of RP-A501 and the importance of addressing the high unmet need in Danon disease.
In other recent news, Rocket Pharmaceuticals has made significant strides in its operations. The company has completed enrollment for its Phase 2 clinical trial for RP-A501, a gene therapy product aimed at treating males with Danon disease.
In addition, Rocket Pharmaceuticals has appointed Dr. Mikael Dolsten to its Board of Directors. According to recent analyst notes, Canaccord Genuity has adjusted its price target for Rocket Pharmaceuticals due to the prospects of the company's Phase 3 clinical trial for its DESCARTES-08 drug candidate.
Rocket Pharmaceuticals has also received a Complete Response Letter from the FDA regarding its Biologics License Application for KRESLADI™, a gene therapy designed to treat severe leukocyte adhesion deficiency-I. In response to this, Canaccord Genuity expressed optimism that the issues would be addressed satisfactorily.
InvestingPro Insights
As Rocket Pharmaceuticals (NASDAQ:RCKT) navigates through its Phase 2 clinical trial for RP-A501, financial metrics and expert analysis provide additional context for investors monitoring the company's progress. According to InvestingPro data, Rocket Pharmaceuticals holds a market capitalization of approximately $1.75 billion. Despite challenges in profitability with an operating income of around -$269.8 million for the last twelve months as of Q2 2024, the company maintains a solid liquidity position. This is evident as it holds more cash than debt on its balance sheet and its liquid assets exceed short-term obligations.
InvestingPro Tips highlight that while Rocket Pharmaceuticals does not currently pay a dividend and is not profitable over the last twelve months, analysts have noted the company's weak gross profit margins and do not anticipate profitability this year. However, it's worth mentioning that Rocket Pharmaceuticals' fair value is estimated at $18.81 by InvestingPro, with analysts targeting a significantly higher fair value of $48. For investors looking for further insights, there are additional InvestingPro Tips available that could shed more light on the company's financial health and future outlook.
As Rocket Pharmaceuticals continues its journey towards potential accelerated approval of RP-A501, these financial insights could be instrumental for investors to align their expectations with the company's performance and market valuation.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.