Rocket Pharmaceuticals Maintains Hold Rating Amid Clinical and Financial Uncertainties

Rocket Pharmaceuticals has a Hold rating from Leerink Partners due to clinical and financial uncertainties. The company faces a clinical hold on its pivotal trial for RP-A501 and challenges in the lentiviral gene therapy partnering environment. However, Rocket has a substantial cash reserve and strategic reorganization efforts, extending its cash runway. Analyst Mani Foroohar maintains the Hold rating with a $9.00 price target.

Rocket Pharmaceuticals, Inc. (RCKT), a biotechnology company developing gene therapies for rare diseases, has recently been subject to significant setbacks. On May 27, 2025, the U.S. Food and Drug Administration (FDA) placed a clinical hold on the company's Phase 2 pivotal trial for RP-A501, an experimental therapy intended to treat patients with Danon disease, a multi-organ lysosomal-associated disorder leading to early death due to heart disorder [1].

The clinical hold was imposed following a Serious Adverse Event (SAE) involving a patient's death. This event, along with the revelation of significant protocol changes in the trial, led to a substantial drop in the company's stock price. The stock plummeted 62% in a single day and down 75% from its February 27, 2025 closing price [1].

In response to these developments, a securities class action lawsuit styled Ho v. Rocket Pharmaceuticals, Inc., et al., No. 3:25-cv-10049 (D. N.J.) has been filed. The lawsuit seeks to represent investors who purchased or otherwise acquired Rocket Pharmaceuticals securities between February 27, 2025 and May 26, 2025. The litigation focuses on the propriety of Rocket Pharmaceuticals' disclosures about the trial protocol being implemented in the RP-A501 Phase 2 trial [1].

The class action complaint alleges that while Rocket publicly highlighted its advances and expressed confidence in meeting trial endpoints, it failed to alert investors to significant protocol changes and known risks of SAEs, including patient deaths. The company's shares closed at $9.40 on February 27, 2025, buoyed by these statements, but the stock price later plummeted due to the disclosure of the clinical hold and protocol changes [1].

Rocket Pharmaceuticals faces additional challenges in the lentiviral gene therapy partnering environment. Analyst Mani Foroohar maintains a Hold rating for the company, citing clinical and financial uncertainties. Despite these challenges, Rocket Pharmaceuticals has a substantial cash reserve and strategic reorganization efforts, extending its cash runway [2].

The deadline to seek lead plaintiff status in the securities class action lawsuit is August 11, 2025. Investors who suffered substantial losses or have knowledge that may assist the firm's investigation are urged to submit their losses now. For more information, contact Hagens Berman at RCKT@hbsslaw.com or 844-916-0895 [1].

References:
[1] https://www.globenewswire.com/news-release/2025/08/08/3130175/32716/en/Rocket-Pharmaceuticals-Inc-RCKT-Faces-Securities-Class-Action-Amid-Protocol-Change-and-FDA-s-Clinical-Hold-on-Gene-Therapy-Trial-Hagens-Berman.html
[2] Leerink Partners, "Rocket Pharmaceuticals, Inc. (RCKT) - Hold Rating, July 2, 2025"