$RCKT

4 stories mentioning RCKTUpdated 33d ago

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Rocket Pharmaceuticals' Danon, PKP2-ACM Updates Seen as Key H2 Catalysts, Wedbush Says

Rocket Pharmaceuticals' (RCKT) Q2 update confirmed its programs are on track, with management expecting regulatory updates on RP-A501 for Danon disease and RP-A601 for PKP2-ACM in H2, Wedbush Securities said Tuesday in a report.Danon disease is a rare inherited disorder that causes heart and skeletal muscle weakness, and PKP2-ACM is a genetic form of arrhythmogenic cardiomyopathy that can lead to dangerous heart rhythm disturbances.Rocket plans to dose nine additional Danon patients, bringing the total number of patients on the recalibrated dose in the pivotal Phase 2 study to 12, Wedbush said.For RP-A601, engagement with the FDA remains ongoing to finalize the design of a potential Phase 2 study, while Phase 1 remains open and actively enrolling, the report said. Rocket also expects to dose the first patient in the Phase 1 study of RP-A701 for BAG3-DCM in H2, Wedbush said.BAG3-DCM is a genetic form of dilated cardiomyopathy that causes progressive weakening of the heart muscle.Wedbush lowered its price target on Rocket stock to $15 from $16 and maintained its outperform rating.Price: $3.49, Change: $-0.15, Percent Change: -4.12%

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Sectors93d ago

Sector Update: Healthcare Stocks Advance Pre-Bell Friday

Healthcare stocks were advancing pre-bell Friday, with the State Street Health Care Select Sector SPDR ETF (XLV) 0.5% higher and the iShares Biotechnology ETF (IBB) up 0.3%.BeOne Medicines (ONC) stock was up more than 1% after the company said new data for tacabrutideg, a Bruton's tyrosine kinase degrader, showed "durable responses" in pretreated relapsed or refractory chronic lymphocytic leukemia or small lymphocytic lymphoma patients and promising activity in BTK inhibitor-naive patients.BioCryst Pharmaceuticals' (BCRX) Orladeyo was associated with "early and sustained reductions" in hereditary angioedema or HAE attacks, demonstrated by new clinical data from the ongoing APeX-P study, the company said. BioCryst Pharmaceuticals shares were up 0.1% premarket.Rocket Pharmaceuticals (RCKT) stock was up nearly 2% after the company closed the $180 million sale of its rare pediatric disease priority review voucher.

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Wire93d ago

Rocket Pharmaceuticals Closes $180 Million Sale of Pediatric Priority Review Voucher

Rocket Pharmaceuticals (RCKT) has closed the $180 million sale of its rare pediatric disease priority review voucher, the company said Friday.The voucher was granted by the US Food and Drug Administration in March 2026 in connection with the approval of Kresladi, Rocket's gene therapy for severe leukocyte adhesion deficiency-I, a rare and life-threatening primary immunodeficiency disease, the company said.The sale increases Rocket's cash, cash equivalents and investments to around $322.6 million, which is expected to fund operations into Q2 2028, the company said.Shares of the company were up 5.3% in Friday premarket trading.

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Wire128d ago

Rocket Pharmaceuticals' Cardiac Programs Updates to Offer 'Strong' Share Price Catalysts, Wedbush Says

Rocket Pharmaceuticals' (RCKT) clinical and regulatory updates on its three cardiac gene therapy programs in H2 are expected to serve as "strong" share price catalysts, Wedbush Securities said in a note Friday.All three programs are advancing as planned, with management expected to provide an update on RP-A501 for Danon disease once alignment is reached with the US Food and Drug Administration, which should bring the program back on track, the brokerage said. A discussion of disease prevalence is critical amid questions on the size of the market opportunity, Wedbush added.For RP-A601 targeting PKP2-related arrhythmogenic cardiomyopathy, Rocket is in talks with the FDA for the final design of a potential pivotal phase 2 study, while the phase 1 study remains open due to patients' demand to undergo treatment without waiting for the next study, the brokerage said.Rocket's RP-A701 program for BAG3-associated dilated cardiomyopathy is also on track to begin a phase 1 study with a fast track designation and first patient dosing expected mid-year, according to the note.Wedbush reiterated its outperform rating on the stock, with a price target of $16.Shares of Rocket Pharmaceuticals were down about 2.9% in Friday trading.Price: $3.57, Change: $-0.11, Percent Change: -2.86%

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