Asia FX dithers as dollar steadies before Powell speech; yen muted after CPI data
Investing.com - Rocket Pharmaceuticals (NASDAQ:RCKT) shares surged 23.8% this week after the company announced the FDA lifted its clinical hold on the RP-A501 program for Danon disease. According to InvestingPro data, the stock remains significantly below its 52-week high of $22.01, suggesting potential recovery room despite recent volatility.
The clinical hold, which had been in place for approximately three months, was removed with conditions that Rocket proceed with a lower dose of 3.8e13 GC/kg, down from the previously used 6.7e13 GC/kg, and adjust the immunomodulatory regimen.
While patients remain enrolled and ready for treatment, the company expects to resume dosing in early 2026 after completing IRB processes, rescreening, and the three-month baseline run-in period through the remainder of 2025.
The company plans to dose three additional patients, with four weeks between each, before providing the FDA with an update to determine next steps for the program.
Canaccord Genuity maintained its Buy rating and $10.00 price target on Rocket Pharmaceuticals, with the firm noting that while the lower dose creates some uncertainty, it believes the current valuation does not reflect the program’s potential value.
In other recent news, Rocket Pharmaceuticals has seen significant developments concerning its pivotal Phase 2 trial for Danon disease. The U.S. Food and Drug Administration (FDA) lifted a clinical hold on Rocket’s RP-A501 therapy, allowing the study to resume with a recalibrated dose. This decision came after Rocket addressed the FDA’s concerns, enabling the treatment of three patients with a minimum four-week interval between treatments. Following this development, BofA Securities upgraded Rocket Pharmaceuticals from Neutral to Buy, raising its price target from $4.00 to $10.00, describing the FDA’s decision as a "positive surprise."
Goldman Sachs maintained a Sell rating for Rocket Pharmaceuticals, setting a price target of $2.00, despite the FDA’s recent action. Meanwhile, Jefferies increased its price target from $2.50 to $3.00, maintaining a Hold rating, and highlighted potential stock drivers for the future. These recent changes reflect the varied perspectives of different financial firms on Rocket Pharmaceuticals’ prospects.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.