Jefferies cuts Rocket Pharmaceuticals stock rating, slashes target

Published 28/05/2025, 08:56
Jefferies cuts Rocket Pharmaceuticals stock rating, slashes target

On Wednesday, Jefferies analyst Andrew Tsai adjusted the firm’s stance on Rocket Pharmaceuticals (NASDAQ:RCKT), downgrading the stock from Buy to Hold. This decision was influenced by a recent FDA clinical hold on a pivotal Phase II program for RP-A501, a gene therapy treatment for Danon heart disease. Alongside the downgrade, Jefferies significantly reduced the price target for Rocket Pharmaceuticals to $2.50, a steep decline from the previous $29.00. The stock, currently trading at $2.33, has fallen nearly 65% in the past week alone, according to InvestingPro data, with the RSI indicating oversold conditions.

The FDA’s clinical hold on RP-A501 has introduced uncertainties regarding the drug’s development timeline and its benefit/risk assessment. Tsai noted that while the drug could eventually receive approval, Jefferies requires greater clarity on the steps necessary to resolve the FDA’s concerns and the time it will take to produce pivotal Phase II data. Despite these challenges, InvestingPro data shows the company maintains a strong liquidity position with a current ratio of 9.19, and holds more cash than debt on its balance sheet. For deeper insights into RCKT’s financial health and 14+ additional ProTips, consider exploring InvestingPro’s comprehensive analysis.

The clinical hold is a critical regulatory action that can impact a pharmaceutical company’s research and development progress. It is issued when the FDA identifies concerns that must be addressed before a trial can continue. In this case, the hold on Rocket Pharmaceuticals’ RP-A501 program has significant implications for the company’s future operations and its stock valuation.

Tsai’s comments provide insight into Jefferies’ decision-making process: "An FDA clinical hold on a pivotal Phase II program of RP-A501 (AAV9) gene therapy for Danon heart disease pushes back timelines and raises some questions around the drug’s overall benefit/risk profile, prompting us to downgrade to HOLD (from BUY). To be clear, we do think the drug could be eventually approved, but we’d like more clarity on the path forward, such as the time required to lift the FDA clinical hold and then generate pivotal Phase II data."

Rocket Pharmaceuticals’ stock performance and investor sentiment may be influenced by this news as market participants digest the implications of the FDA’s decision and Jefferies’ subsequent downgrade. The company’s path forward is now contingent on addressing the FDA’s concerns to resume the RP-A501 program’s development. With a market capitalization of $251 million and trading significantly below its 52-week high of $26.98, InvestingPro’s Fair Value analysis suggests the stock may be undervalued at current levels, though investors should note the company’s rapid cash burn rate and uncertain path to profitability.

In other recent news, Rocket Pharmaceuticals has faced significant developments affecting its clinical trials and stock ratings. The company reported a serious adverse event resulting in a patient’s death during a trial for its RP-A501 treatment for Danon disease, leading to a clinical hold by the FDA. This incident has prompted several analysts to adjust their outlooks on the company. Leerink Partners downgraded Rocket Pharmaceuticals from Outperform to Market Perform and slashed the price target from $37 to $8, citing safety concerns and the need to resolve the clinical hold. Similarly, TD Cowen downgraded the stock from Buy to Hold, emphasizing the unresolved safety risks associated with thrombotic microangiopathies observed in the trial.

Despite these challenges, some firms maintain a positive outlook. William Blair continues to rate Rocket Pharmaceuticals as Outperform, expressing optimism that the adverse event was linked to a C3 inhibitor and not the therapy itself. BofA Securities also remains supportive, reducing its price target slightly to $32 while maintaining a Buy rating, highlighting the company’s strong financial position and promising developments in other programs. BMO Capital Markets reiterated its Outperform rating with a $30 price target, underscoring the potential therapeutic benefits of RP-A501 despite the recent setback. These developments have placed Rocket Pharmaceuticals under close scrutiny as it navigates the regulatory and safety challenges ahead.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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