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Investing.com - Goldman Sachs has reiterated its Sell rating and $2.00 price target on Rocket Pharmaceuticals (NASDAQ:RCKT) following the FDA’s decision to lift a clinical hold on the company’s pivotal Phase 2 study for Danon disease. The stock, currently trading at $3.69, has fallen nearly 85% over the past year, though InvestingPro analysis suggests the company may be undervalued at current levels.
The FDA lifted the clinical hold on RP-A501’s pivotal Phase 2 study on Wednesday, less than three months after issuing it in May. The study will now resume using a lower dose of 3.8E13 GC/kg, compared to the previous 6.7E13 GC/kg dose. According to InvestingPro data, the company maintains a strong liquidity position with a current ratio of 6.39 and holds more cash than debt on its balance sheet, providing financial flexibility for its clinical programs.
The next three patients will be sequentially dosed with a minimum four-week interval between each treatment. Rocket Pharmaceuticals will use its original Phase 2 immunomodulatory protocol without the prophylactic use of a C3 complement inhibitor, while eculizumab will be used in qualifying patients with a lower threshold for intervention.
Six patients have been treated in the Phase 2 study to date, representing half of the planned 12 patients. The FDA has not requested additional patients be added to the study, though the total number will be reassessed after successful dosing of the next three patients.
Rocket Pharmaceuticals plans to provide further updates after treating these patients and is scheduling a comprehensive Danon disease program update in 2026 that will include epidemiology insights. While analysts maintain mixed views with targets ranging from $2 to $16, InvestingPro subscribers can access 12 additional key insights about RCKT’s financial health and market position through the platform’s comprehensive Pro Research Report.
In other recent news, Rocket Pharmaceuticals announced a significant development as the FDA lifted a clinical hold on its pivotal Phase 2 trial of RP-A501 for Danon disease. This decision came after the company addressed the FDA’s concerns, allowing the study to resume with an adjusted dose for three patients. Following this news, BofA Securities upgraded Rocket Pharmaceuticals from Neutral to Buy, raising its price target to $10.00, citing the FDA’s decision as a "positive surprise." Additionally, Jefferies adjusted its price target for the company to $3.00, maintaining a Hold rating, while noting potential stock drivers for the future.
In another strategic move, Rocket Pharmaceuticals revealed a reorganization plan that includes a workforce reduction of approximately 30%. This restructuring aims to concentrate on the company’s cardiovascular gene therapy programs, particularly those targeting Danon disease and other heart-related conditions. The company will also continue regulatory efforts for KRESLADI, its treatment for severe leukocyte adhesion deficiency-I. These recent developments reflect Rocket Pharmaceuticals’ ongoing efforts to advance its gene therapy pipeline and streamline its focus on key therapeutic areas.
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