Rocket Pharmaceuticals stock rating reiterated at Overweight by Cantor Fitzgerald

Investing.com - Cantor Fitzgerald has reiterated an Overweight rating on Rocket Pharmaceuticals (NASDAQ:RCKT) with a price target of $8.00. The stock, currently trading at $3.07, has seen significant pressure, falling over 75% year-to-date. According to InvestingPro data, seven analysts have recently revised their earnings estimates upward, with price targets ranging from $2 to $16.

The firm maintained its positive outlook despite recent complications in the company’s Danon disease clinical trial, where two patients experienced capillary leak syndrome after receiving an immunosuppressive regimen that included Empaveli, a complement C3 inhibitor.

One of these patients died from infection complications, which led to a clinical hold on the trial for less than three months. The hold was lifted on August 20, 2025, with updated dosing parameters of 3.8E13 GC/kg and a requirement of 4 weeks between treated patients.

The trial modifications also included discontinuation of the C3 inhibitor use that had been part of the previous treatment protocol. According to Cantor Fitzgerald, the complications appeared to result from a combination of underlying disease, AAV capsid, and C3 inhibition.

The clinical hold being lifted allows Rocket Pharmaceuticals to continue its development program for Danon disease with the modified protocol designed to address the safety concerns identified during the trial.

In other recent news, Rocket Pharmaceuticals announced that the FDA has lifted the clinical hold on its RP-A501 program for Danon disease. This decision allows the company to resume its pivotal Phase 2 study, although with a lower dose and adjusted immunomodulatory regimen. The clinical hold had been in place for approximately three months. Following this development, BofA Securities upgraded Rocket Pharmaceuticals from Neutral to Buy and increased its price target to $10.00 from $4.00. Conversely, Goldman Sachs maintained its Sell rating with a $2.00 price target, even after the FDA’s decision. In another update, Rocket Pharmaceuticals entered into separation and consulting agreements with Dr. Kinnari Patel, who is departing to join RTW Investments LP. Dr. Patel will receive her current salary and benefits through the end of 2025, along with a lump sum payment and healthcare allowance. These recent developments have captured significant attention in the investment community.

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